We are a clinical-stage biotechnology company comprised of experienced biopharma industry leaders with extensive research, development, and rare disease expertise with a mission to develop and commercialize life-transforming therapies for patients with severe and rare diseases.
−Removed: Since our launch in January 2018, we have built a broad pipeline of promising product candidates aimed at addressing diseases with unmet medical need in the areas of maternal fetal health, complement dysregulation, hematology, and metabolic disorders.
−Removed: Our two most advanced programs are in clinical development:
−Removed: RLYB212, an anti-HPA-1a antibody for the prevention of fetal and neonatal alloimmune thrombocytopenia (“FNAIT”) and RLYB116, an inhibitor of complement component 5 (“C5”), with the potential to treat several diseases of complement dysregulation.
−Removed: RLYB212 is currently in a Phase 2 clinical trial in pregnant women, and we plan to initiate a confirmatory pharmacokinetics (“PK”) and pharmacodynamics ("PD") study of RLYB116 in the second quarter of 2025.
−Removed: At Rallybio Corporation (“Rallybio” or the "Company"), we do not accept that millions of patients suffering from devastating rare diseases should have to live without transformative treatments.
−Removed: There are an estimated 25 to 30 million people affected by as many as 7,000 rare diseases in the United States alone, with a significantly greater number affected globally.
−Removed: Our goal is to transform the lives of these individuals through the development of therapeutics that provide meaningful clinical benefits.
−Removed: We believe the long-term success of the Company is built on our key strengths:
−Removed: • Our team’s proven execution capability to drive product candidates through clinical development to marketing approvals.
−Removed: We have assembled a team with a proven history of successfully advancing product candidates from discovery to clinical development and through marketing approval.
−Removed: Members of our team have played critical roles in the approval of more than 30 drugs, including seven approvals for rare disease therapeutics since 2013, and secured approvals from regulatory authorities in the Americas, Europe, Australia, and Asia.
−Removed: In doing so, our employees previously developed and implemented novel clinical trial designs and successfully conducted clinical trials in previously underserved patient populations, including those impacted by diseases lacking approved therapies.
−Removed: We believe this collective prior experience positions us to efficiently and expertly execute at each step in the research and development process and enhances the value we can bring to our product candidates and patients.
−Removed: • Our extensive knowledge of rare diseases and our scientific expertise positions us to identify therapies with the potential for transformative impact.
−Removed: Although we intend to focus our near-term efforts on maximizing the value of our current portfolio, we may in the future opportunistically seek to acquire and develop product candidates that possess a clear mechanism of action and that aim to address diseases with a well-understood pathophysiology for which there is a significant unmet medical need.
−Removed: We believe that a product candidate’s mechanism of action should target the causal biology of the disease to provide the highest probability of dramatically improving the lives of patients.
−Removed: We also believe that our team’s extensive experience in rare diseases and overall deep scientific expertise position us to identify opportunities where these links can be made.
−Removed: In addition, our ability to source these product candidates is facilitated by our extensive network of relationships with leaders in industry and in academic centers worldwide.
−Removed: Our Chief Executive Officer, Stephen Uden, M.D., and Chairman, Martin W.
−Removed: Mackay, Ph.D., previously worked together in different organizations to successfully build, develop, and launch transformative therapies for patients with rare diseases.
−Removed: In addition, several members of our team were integral in the successful development and/or approval of therapies such as Strensiq (asfotase alfa) for patients with perinatal-, infantile-, and juvenile-onset hypophosphatasia ("HPP"), Kanuma (sebelipase alfa) for patients with lysosomal acid lipase deficiency, Nulibry (fosdenopterin) for patients with molybdenum cofactor deficiency, Soliris (eculizumab) for patients with refractory generalized myasthenia gravis ("gMG"), Soliris for patients with relapsing neuromyelitis optica spectrum disorder ("NMOSD"), Ultomiris (ravulizumab-cwvz) for patients with paroxysmal nocturnal hemoglobinuria ("PNH") and Ultomiris for patients with atypical hemolytic uremic syndrome ("aHUS").
−Removed: Our pipeline is illustrated in the chart below:
−Removed: Fetal and neonatal alloimmune thrombocytopenia;
−Removed: Human platelet antigen-1a;
−Removed: Complement component 5;
−Removed: Albumin-binding domain;
−Removed: Ectonucleotide pyrophosphatase/phosphodiesterase 1
−Removed: Prevention of FNAIT
−Removed: We are developing RLYB212 for the prevention of FNAIT.
−Removed: FNAIT is a potentially life-threatening rare disease that can cause uncontrolled bleeding in fetuses and newborns that can arise during pregnancy due to an immune incompatibility between an expectant mother and her fetus in a specific platelet antigen called human platelet antigen 1 ("HPA-1").
−Removed: This incompatibility can cause an expectant mother to develop antibodies that attack the platelets of her fetus.
−Removed: The destruction of platelets in the fetus can result in severely low platelet counts, or thrombocytopenia, potentially leading to devastating consequences including miscarriage, stillbirth, death of the newborn, or severe lifelong neurological disability in those babies who survive.
−Removed: There is currently no approved therapy for the prevention or treatment of FNAIT.
−Removed: We estimate that there are over 30,000 pregnancies at high risk of developing FNAIT each year in the United States, Canada, the United Kingdom (the "UK"), other major European countries and Australia.
−Removed: Since there are no approved therapies to prevent FNAIT, expectant mothers are not currently screened for the risk of FNAIT.
−Removed: As a result, the vast majority of pregnancies at risk for FNAIT go unidentified and untreated.
−Removed: In those pregnancies that are identified as at-risk, typically due to the delivery of a prior FNAIT affected child, expectant mothers may be treated with weekly intravenously-administered high doses of immunoglobulin G ("IVIG"), along with the oral steroid immunosuppressant prednisone.
−Removed: However, IVIG administration does not prevent the immune response, called alloimmunization, and is costly, time-intensive, difficult to tolerate and associated with significant treatment-related complications.
−Removed: Based on the data generated to date and the clinical precedent for effective prevention of maternal alloimmunization by anti-RhD prophylaxis, which has resulted in the virtual elimination of Rhesus disease, we believe that targeting HPA-1a with an anti-HPA-1a antibody has the potential to drive rapid elimination of HPA-1a positive platelets from the circulation of expectant mothers, prevent alloimmunization and thereby prevent the occurrence of FNAIT.
−Removed: We are currently conducting a Phase 2 clinical trial of RLYB212 in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT at sites across Europe.
−Removed: The primary objective of this single-arm Phase 2 trial is to assess the PK and safety of RLYB212 with secondary objectives that include assessments of pregnancy and neonatal/infant outcomes, and the occurrence of emergent HPA-1a alloimmunization.
−Removed: Subcutaneous ("SC") administration of RLYB212 will be initiated by Gestational Week 16 and will continue every four weeks through parturition.
−Removed: The Phase 2 trial is designed to enroll participants in three stages:
−Removed: first with a sentinel pregnant woman, an initial cohort ("Cohort 1") that will include three pregnant women, and a second cohort ("Cohort 2") that will include four pregnant women, for a total target enrollment of eight participants.
−Removed: A data review for participants and infants is planned prior to the initiation of each cohort.
−Removed: The Phase 2 trial follows completion of two RLYB212 clinical trials:
−Removed: a Phase 1 first-in-human clinical trial and a Phase 1b proof-of-concept clinical trial.
−Removed: The Phase 1 first-in-human clinical trial was a single-blind, placebo-controlled study that investigated the safety and PK of SC administration of RLYB212 in HPA-1a negative healthy participants.
−Removed: The clinical trial included a single dose cohort and a multiple dose cohort.
−Removed: In the multiple dose cohort, subjects received SC RLYB212 or placebo every two weeks for 12 weeks.
−Removed: We reported preliminary results from the multi-dose cohort in the fourth quarter of 2023.
−Removed: The data and our clinical pharmacology modeling predictions support a once monthly dosing regimen for the Phase 2 clinical trial.
−Removed: RLYB212 was observed to be generally well-tolerated with no reports of injection site reactions or serious adverse events.
−Removed: The Phase 1b single-blind, placebo-controlled proof-of-concept clinical trial was designed to establish the ability of SC RLYB212 to rapidly eliminate HPA-1a positive platelets transfused to HPA-1a negative healthy subjects.
−Removed: The study included 11 males aged 18 to 65 years, randomized to RLYB212 0.09mg (n=4), RLYB212 0.29mg (n=5), or placebo (n=2).
−Removed: We reported top-line results from this trial at the 31st Congress of the International Society on Thrombosis and Haemostasis ("ISTH") in the second quarter of 2023 and announced publication of the complete dataset in Thrombosis and Haemostasis in the third quarter of 2024.
−Removed: The results showed that SC RLYB212 administration produced a dose-dependent, rapid, and complete elimination of transfused HPA-1a positive platelets in HPA-1a negative subjects, with both doses meeting the prespecified proof-of-concept criteria of ≥90% reduction in mean platelet elimination half-life.
−Removed: Mean platelet elimination half-life was 5.8 hours (0.09mg dose) and 1.5 hours (0.29mg dose) for RLYB212 compared to 71.7 hours for placebo.
−Removed: Consistent with the Phase 1 first-in-human trial, RLYB212 was observed to be well-tolerated with no reports of serious or severe adverse events.
−Removed: Additionally, we are conducting a FNAIT Natural History Alloimmunization Study ("FNAIT natural history study").
−Removed: This prospective, non-interventional, multinational natural history study is designed to screen expectant mothers presenting at gestational week 10 to 14 prenatal visit to determine the frequency of women at higher FNAIT risk among expectant mothers of different racial and ethnic characteristics, as well as the frequency of HPA-1a alloimmunization and pregnancy outcomes among these women.
−Removed: Subject to future discussions with regulatory authorities, we expect that data from this study will contribute to a control dataset for a future single-arm Phase 3 registrational clinical trial for RLYB212.
−Removed: An additional objective of the FNAIT natural history study is to operationalize de novo the laboratory screening test paradigm for FNAIT risk and generate FNAIT laboratory test performance data that we plan to use for future regulatory discussions.
−Removed: As of January 31, 2025, more than 14,300 women have been screened as part of the study.
+Added: Our lead program, RLYB116, is a differentiated complement component 5 (“C5”) inhibitor with the potential to treat diseases of complement dysregulation.
+Added: In addition, RLYB332, a long-acting matriptase-2 ("MTP-2") antibody for the treatment of diseases of iron overload, is currently in preclinical development.
+Added: Recent Developments
+Added: On March 1, 2026, we entered into an Agreement and Plan of Merger and Reorganization (the “Merger Agreement”) with Candid, a clinical-stage biotechnology company advancing a leading portfolio of T-cell engager ("TCE") therapeutics for autoimmune diseases, and Farmington Merger Sub, Inc., a Delaware corporation and wholly-owned subsidiary of Rallybio (“Merger Sub”).
+Added: Upon the terms and subject to the satisfaction of the conditions described in the Merger Agreement, Merger Sub will be merged with and into Candid, with Candid surviving as a wholly owned subsidiary of Rallybio (the “Merger”).
+Added: The Merger is intended to qualify as a tax-free reorganization for U.S.
+Added: federal income tax purposes.
+Added: Concurrently with the execution and delivery of the Merger Agreement, certain investors entered into subscription agreements with Candid, pursuant to which such investors have agreed to purchase, immediately prior to the Merger, shares of Candid common stock representing an aggregate commitment of approximately $505.5 million in the concurrent financing (the “Concurrent Financing”).
+Added: The shares of Candid common stock that are issued in the Concurrent Financing will be or will have the right to be, respectively, converted into shares of Rallybio Common Stock, par value $0.0001 per share ("Rallybio Common Stock), in the Merger.
+Added: Subject to the terms and conditions of the Merger Agreement, at the effective time of the Merger (the “Effective Time”), (a) each then-outstanding share of common stock or preferred stock of Candid (each such share, a “Candid Share”) (excluding any share described in clauses (b) or (c) below and Candid Shares held by stockholders who have exercised and perfected appraisal rights for such shares) will be converted into the right to receive a number of shares of Rallybio Common Stock equal to the Exchange Ratio as set forth in the Merger Agreement (the “Exchange Ratio”), (b) each Candid Share issued in the Concurrent Financing will be converted into the right to receive a number of shares of Rallybio Common Stock equal to the Concurrent Financing Exchange Ratio as set forth in the Merger Agreement (the “Concurrent Financing Exchange Ratio”), (c) any Candid Shares held as treasury shares or held or owned by Rallybio, Merger Sub or any subsidiary of Rallybio or Candid immediately prior to the Effective Time will be canceled and shall cease to exist, and no consideration shall be delivered in exchange therefor.
+Added: Each then-outstanding option to purchase Candid Shares will be converted into an option to purchase Rallybio Common Stock, based on the Exchange Ratio and subject to adjustment as set forth in the Merger Agreement.
+Added: Under the Exchange Ratio and Concurrent Financing Exchange Ratio formulas in the Merger Agreement, immediately after the Closing, on a pro forma basis and based upon the number of shares of Rallybio Common Stock expected to be issued in connection with the Merger, pre-Merger equityholders of Candid (other than investors in the Concurrent Financing) are expected to own approximately 57.55% of the combined company, pre-Merger equityholders of Rallybio are expected to own approximately 3.65% of the combined company and the Investors in the Concurrent Financing are expected to own approximately 38.80% of the combined company (assuming proceeds from the Concurrent Financing of $505.5 million), in each case, calculated on a fully diluted basis, using the treasury stock method, and subject to certain assumptions, including (i) a valuation for Rallybio of $47.5 million (assuming Rallybio has net cash (“Rallybio Net Cash”) of $37.5 million as of the closing of the Merger (the “Closing” and such date, the “Closing Date”)), (ii) a fixed valuation for Candid of $750.0 million, and (iii) the relative capitalization of Rallybio and Candid.
+Added: The percentage of the combined company that each party’s equity holders will own following the Closing is subject to certain adjustments as described in the Merger Agreement, including the amount of the final Rallybio Net Cash at Closing.
+Added: Immediately prior to the Effective Time, Rallybio and a rights agent are expected to enter into a Contingent Value Rights Agreement (the “CVR Agreement”), pursuant to which holders of record of certain Rallybio securities as of the close of business on the last business day prior to the day on which the Effective Time occurs will receive one contingent value right (each, a “CVR”) for each outstanding share of Rallybio Common Stock, prefunded warrant, Rallybio restricted stock unit or In the Money Parent Option (as defined in the CVR Agreement) held as of such date.
+Added: Pursuant to the CVR Agreement, each CVR holder will be entitled to receive
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+Added: their pro rata share of (i) all of the net proceeds (including cash the value of stock to the extent listed on a national exchange, at the time of disposition), if any, received by Rallybio as a result of payments made to Rallybio of any upfront, milestone, royalty and other payments received under any disposition agreement related to Rallybio’s pre-Merger assets (the “Legacy Assets”), and (ii) all of the cash proceeds, if any, received from Recursion Pharmaceuticals, Inc.
+Added: ("Recursion") under the Membership Interest Purchase Agreement, dated July 8, 2025, by and among Recursion, Exscientia Ventures I, Inc., Rallybio Corporation and Rallybio IPB, LLC.
+Added: For a period of one year after the Closing Date, Rallybio will use commercially reasonable efforts to effect the disposition of the Legacy Assets.
+Added: Such net proceeds will be subject to certain permitted deductions, including for applicable tax payments, certain expenses incurred or other liabilities borne by Rallybio or its affiliates in respect of the Legacy Assets, and losses incurred by Rallybio or its affiliates due to a third-party proceeding in connection with such disposition.
+Added: As part of the proposed Merger with Candid the programs in Rallybio's pipeline that are illustrated in the chart below will be subject to the CVR described above.
Treatment of Disorders Due to Complement Dysregulation
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Dysregulation of the complement pathway has been implicated in the pathogenesis of a growing number of diseases, making it an attractive target for therapeutic intervention.
−Removed: Antibody inhibitors of C5 have been successfully developed to treat diseases caused by complement pathway dysregulation, including PNH, aHUS, refractory gMG and relapsing NMOSD.
+Added: Antibody inhibitors of C5 have been successfully developed to treat diseases caused by complement pathway dysregulation, including paroxysmal nocturnal hemoglobinuria ("PNH"), atypical hemolytic uremic syndrome ("aHUS"), refractory generalized myasthenia gravis ("gMG") and relapsing neuromyelitis optica spectrum disorder ("NMOSD").
Despite the approval of antibody-based C5 inhibitors for patients with these diseases, we believe there remains a significant need in the market for safe, effective, patient-friendly, and accessible therapies.
Our team has a track record of success in designing, developing, and securing marketing approval for C5 complement inhibitors, including Soliris and Ultomiris, for patients around the world with severe and rare complement-mediated diseases.
−Removed: Our most advanced product candidate in this therapeutic area is RLYB116, an inhibitor of C5, which is a central component of the terminal complement pathway.
−Removed: RLYB116 is an Affibody molecule attached to an albumin binding domain ("ABD") that has the potential to drive the rapid, complete, and sustained inhibition of C5 with a SC injection.
−Removed: We have completed a Phase 1 clinical trial in healthy participants that included the study of RLYB116 as both a single ascending dose (“SAD”) and a multiple ascending dose (“MAD”).
−Removed: The SAD portion of the RLYB116 Phase 1 trial included five cohorts with doses ranging from 2mg up to 300mg.
−Removed: Data from the SAD portion of the RLYB116 trial showed that all study participants that were administered a single 1 mL SC injection of 100 mg of RLYB116 (n=6) demonstrated a reduction in free C5 greater than 99% within 24 hours of dosing.
−Removed: Subcutaneously administered RLYB116 in the SAD portion of the trial was observed to be generally well-tolerated at the 100 mg dose, with mild adverse events and no drug-related serious adverse events reported.
−Removed: The terminal elimination half-life of RLYB116 was greater than 300 hours.
−Removed: The MAD portion of the RLYB116 Phase 1 trial included an adaptive single-blind design with a four-week treatment duration to evaluate the safety, tolerability, PK, and PD of RLYB116 with multiple dose SC administration.
−Removed: The MAD portion of the trial included four cohorts:
−Removed: Cohort 1 (weekly dosing of 100 mg), Cohort 2 (3 doses of 100 mg the first week followed by weekly dosing), Cohort 3 (150 mg weekly dosing reduced to 125 mg weekly dosing) and
−Removed: Cohort 4 (75 mg twice the first week followed by 100 mg twice per week) with post-treatment for 10 weeks.
−Removed: In December 2023, we reported data from the MAD portion of the trial that demonstrated a 100 mg low volume (1 mL) once-weekly dose of subcutaneously administered RLYB116 achieved sustained mean reductions in free C5 of greater than 93%, including at Day 29 with measurement prior to the last dose.
−Removed: The reduction from pre-treatment free C5 at 24 hours after the first dose of 100 mg was greater than 99%.
−Removed: In the MAD portion of the Phase 1 trial, RLYB116 demonstrated low inter-subject variability and consistent increases in exposure relative to dose with a mean estimated elimination half-life for RLYB116 of >300 hours.
−Removed: RLYB116 administered in the MAD trial as a 100 mg once-weekly dose was also observed to be generally well tolerated.
−Removed: Based on the results of the RLYB116 Phase 1 trial, we conducted a series of biomarker characterization analyses.
−Removed: These analyses indicate the RLYB116 assay used to measure free C5 in the Phase 1 trial overestimated the levels of free C5 by approximately ten-fold, indicating that RLYB116 produced greater complement inhibition than initially reported.
−Removed: We now believe that RLYB116 has the potential to be an effective treatment for patients with a variety of complement-mediated diseases, including PNH, gMG and antiphospholipid syndrome ("APS").
−Removed: We also completed manufacturing process enhancements with a goal of further improving the tolerability of RLYB116.
−Removed: Based on the results of enhanced analytical techniques, including mass spectrometry, these process enhancements have successfully further purified the RLYB116 drug substance.
−Removed: As a result, we believe that RLYB116 will have a favorable tolerability profile at doses at and above those evaluated in the Phase 1 MAD trial.
−Removed: We plan to initiate a RLYB116 confirmatory clinical PK/PD trial in the second quarter of 2025 to demonstrate improved tolerability as well as complete and sustained complement inhibition.
−Removed: This single-blind MAD trial will evaluate a 4-week treatment duration that will include two cohorts of eight participants each.
−Removed: Our current plan is that Cohort 1 will evaluate weekly dosing of 150 mg and Cohort 2 will evaluate weekly dosing of 225 mg with 10 weeks of follow-up after the conclusion of treatment.
+Added: Our most advanced product candidate in this therapeutic area is RLYB116, an innovative, once-weekly, small volume, subcutaneously injected inhibitor of C5, which is a central component of the terminal complement pathway.
+Added: RLYB116 is an Affibody molecule attached to an albumin binding domain ("ABD") that has the potential to drive the rapid, complete, and sustained inhibition of C5 with a subcutaneous ("SC") injection.
+Added: We have completed two Phase 1 clinical trials in healthy participants that included the study of RLYB116 as both a single ascending dose (“SAD”) and a multiple ascending dose (“MAD”).
+Added: After the first Phase 1 clinical trial, we completed manufacturing process enhancements that were designed to improve the tolerability of RLYB116.
+Added: In 2025, we completed the confirmatory Phase 1 clinical trial evaluating the pharmacokinetic ("PK") / pharmacodynamic ("PD") properties of RLYB116.
+Added: The confirmatory trial achieved its two key objectives including:
+Added: a significant improvement in the tolerability of RLYB116 and demonstration of complete and sustained inhibition of terminal complement.
+Added: These results support the study of RLYB116 as a potential best-in-class therapeutic for multiple complement mediated diseases.
Hematological Disorders
−Removed: In May 2022, we obtained worldwide exclusive rights to RLYB331, a preclinical, monoclonal antibody that is designed to inhibit Matriptase-2 ("MTP-2").
+Added: In May 2022, we obtained worldwide exclusive rights to RLYB331, a preclinical, monoclonal antibody that is designed to inhibit MTP-2.
The inhibition of MTP-2 significantly increases levels of hepcidin, decreases iron load and treats ineffective erythropoiesis.
In 2024, we re-engineered RLYB331 to extend its half-life.
−Removed: In the fourth quarter of 2024 we presented preclinical data at the annual meeting of the American Society of Hematology ("ASH") for RLYB332, the long-acting version of RLYB331, including favorable PD data, that support RLYB332 as a long-acting, potentially best-in-class therapy for the treatment of diseases of iron overload.
+Added: In the fourth quarter of 2024 we presented preclinical data at the annual meeting of the American Society of
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+Added: Hematology ("ASH") for RLYB332, the long-acting version of RLYB331, including favorable PD data, that support RLYB332 as a long-acting, potentially best-in-class therapy for the treatment of diseases of iron overload.
We believe RLYB332 has the potential to address a significant unmet need for patients with severe anemia with ineffective red blood cell production or erythropoiesis and iron overload, such as polycythemia vera, beta thalassemia and a subset of myelodysplastic syndromes ("MDS"), amongst others.
2 unchanged sentences
In July 2019, we formed a joint venture with Exscientia Limited ("Exscientia"), an Oxford, UK-based artificial intelligence ("AI") and machine learning drug discovery company with a proprietary chemical design platform to discover novel small molecule drug candidates.
−Removed: Exscientia was acquired by Recursion Pharmaceuticals, Inc.
−Removed: ("Recursion") in 2024 and we have continued work with Recursion on the discovery of a small molecule targeting an Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 ("ENPP1") inhibitor for the treatment of HPP.
−Removed: HPP is a rare, genetic disease characterized by mutations in the ALPL gene.
−Removed: The ALPL gene provides instructions for making an enzyme called tissue-nonspecific alkaline phosphatase, which plays an important role in the growth and development of bones and teeth.
−Removed: The incidence of HPP has been reported to be 1 in 100,000 to 1 in 300,000 (United States and Canada) for severe disease and 1 in 6,370 (European Union) for less severe forms.
−Removed: These mutations lead to diminished activity of the alkaline phosphatase enzyme and the accumulation of inorganic pyrophosphate (“PPi”), which inhibits bone mineralization causing multiple skeletal pathologies.
−Removed: We believe that a small molecule inhibitor of ENPP1 has the potential to bring meaningful benefit to HPP patients by reducing excess levels of pyrophosphate, thereby removing an inhibitor of calcium mineralization and bone formation.
−Removed: In 2024 we presented data at the American Society for Bone and Mineral Research ("ASBMR") from an early lead ENPP1 inhibitor, REV101, in a mouse model of later-onset HPP demonstrating a 30% reduction in PPi, a key biomarker that is elevated in HPP and contributes to poor bone mineralization.
−Removed: Together with Recursion, we also advanced REV102, an ENPP1 inhibitor for the treatment of patients with HPP to position the molecule for additional preclinical development activities in 2025.
+Added: Exscientia was acquired by Recursion in 2024.
+Added: In July 2025, we entered into a Membership Interest Purchase Agreement (the “ENPP1 Purchase Agreement”) with Recursion Exscientia Ventures I, Inc., an indirect wholly-owned subsidiary of Recursion (“Buyer”) and Rallybio IPB, LLC, a wholly-owned subsidiary of Rallybio Corporation to sell our interest in REV102, an Ectonucleotide Pyrophosphatase/ Phosphodiesterase 1 ("ENPP1") inhibitor in preclinical development for the treatment of patients with hypophosphatasia ("HPP"), to Buyer (a subsidiary of our joint venture partner Recursion) (the "JV Sale").
+Added: In connection with the JV Sale, we received a total of $20.0 million in the third quarter of 2025 including $7.5 million from an upfront payment and $12.5 million from a milestone payment related to the initiation of additional preclinical studies.
+Added: We are eligible to receive a $5.0 million milestone payment in connection with the initiation of dosing in a Phase 1 clinical study, as defined in the ENPP1 Purchase Agreement and low single-digit royalties on all future net sales by Recursion of products comprising or incorporating certain compounds developed by RE Ventures I, LLC ("REV-I").
+Added: We may also be eligible to receive certain payments in the event of Recursion’s sale of the REV102 program.
AbCellera Collaboration
In December 2022, we entered into a strategic alliance to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases.
−Removed: This multi-year, multi-target collaboration will combine AbCellera Biologic's
−Removed: ("AbCellera") antibody discovery engine with Rallybio’s clinical and commercial expertise in rare diseases to identify optimal clinical candidates with a goal of delivering therapies to patients.
+Added: This multi-year, multi-target collaboration will combine AbCellera Biologic's ("AbCellera") antibody discovery engine with Rallybio’s clinical and commercial expertise in rare diseases to identify optimal clinical candidates with a goal of delivering therapies to patients.
We and AbCellera intended that the collaboration would co-develop up to five rare disease therapeutic targets, which will be chosen together by both companies with the first program focused on addressing the significant unmet therapeutic needs of patients with rare metabolic diseases.
−Removed: Our mission is aligned with our expertise:
−Removed: to identify and accelerate the development of life-transforming therapies for patients with severe and rare disorders.
−Removed: To achieve this mission, our strategy includes the following key components:
−Removed: • Establish a leading rare disease company with a team that delivers transformative medicines to patients.
−Removed: We believe our team’s expertise and knowledge are fundamental to our long-term success.
−Removed: Our research and development team is led by experienced drug development executives who were integral in the approvals of more than 30 drugs from leading companies, including Alexion Pharmaceuticals, Inc, Astellas Pharma Inc., Pfizer Inc.
−Removed: and Wyeth, LLC.
−Removed: We plan to create value by continuing to leverage our team’s expertise and focus on further development of our current product candidates either alone or through partnerships and collaborations to significantly improve the lives of patients.
−Removed: • Advance RLYB212 for the prevention of FNAIT.
−Removed: There is currently no approved therapy for the prevention of FNAIT.
−Removed: Women at higher risk of FNAIT may experience potentially devastating outcomes and we believe there is a significant unmet need for these patients.
−Removed: We believe the clinical and non-clinical data generated to date indicate that RLYB212 may prevent FNAIT in pregnant women at higher risk for HPA-1a alloimmunization and were supportive of initiating the ongoing Phase 2 clinical trial in pregnant women.
−Removed: We enrolled the sentinel woman in February 2025 and expect to report PK and safety data from the second trimester in the second quarter of 2025 and PK and safety data at the time of delivery in the third quarter of 2025 at which time we will determine the next steps for the program including seeking a partner to continue development.
−Removed: We estimate that the market opportunity for RLYB212 exceeds $1.6 billion.
−Removed: • Advance RLYB116 for the treatment of diseases of complement dysregulation through partnering or other forms of non-dilutive financing.
−Removed: Based on the biomarker characterization analyses and manufacturing process enhancements we completed in 2024, we intend to initiate a confirmatory clinical PK/PD trial in the second quarter of 2025.
−Removed: We will evaluate future development plans for RLYB116 based on the results of this trial but based on the data generated to date, we believe that RLYB116 has the potential to be an effective treatment for patients with a variety of complement-mediated diseases, including PNH, gMG and APS.
−Removed: • Leverage the progress made to date and maximize the value from our preclinical candidates.
−Removed: We believe that our preclinical pipeline programs have significant potential to generate value by meeting the unmet needs of patients and we intend to continue to move these forward in a capital-efficient manner.
−Removed: As we are able to generate additional data, we will make decisions on the most appropriate next steps in development for each of our preclinical programs.
−Removed: • Create value from our pipeline through targeted business development activities.
−Removed: We believe we have assembled a valuable portfolio of product candidates targeting several rare diseases and will seek to unlock that value by leveraging our current and potential future partnerships and collaborations for one or more of these programs.
−Removed: We may also periodically evaluate the potential in-license or acquisition of new programs subject to market conditions and our cash runway.
+Added: In 2025, the Company made a decision to evaluate strategic alternatives, with a goal of enhancing long term shareholder value that culminated with the signing of the Merger Agreement with Candid, a clinical-stage biotechnology company advancing a leading portfolio of TCE therapeutics for autoimmune diseases on March 1, 2026.
+Added: In connection with the planned Merger, we have started to take steps to wind down our general and administrative operations that will not be needed after the close of the Merger.
+Added: We are continuing to move forward with limited research and development operations with a goal of advancing our programs in the near term to maximize the potential value of the CVRs.
Our Product Candidates
−Removed: RLYB212 for the prevention of FNAIT
−Removed: RLYB212 is a subcutaneously administered monoclonal anti-HPA-1a antibody for the prevention of FNAIT, a maternal fetal blood disorder that can cause potentially devastating outcomes in the fetus or neonate including miscarriage, neonatal death, and severe life-long neurological disability in the babies that survive.
−Removed: We have completed two Phase 1 clinical trials for RLYB212 and are currently running a Phase 2 clinical trial in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT.
−Removed: Based on the preclinical and clinical data to date and our understanding of FNAIT, we believe RLYB212 has the potential to prevent maternal alloimmunization and thereby the occurrence of FNAIT.
−Removed: We are also conducting an FNAIT natural history study.
−Removed: This prospective, non-interventional, multinational study is designed to determine the frequency of women at higher FNAIT risk among expectant mothers of different racial and ethnic characteristics, as well as the frequency of HPA-1a alloimmunization and pregnancy outcomes among these women.
−Removed: An additional objective of the FNAIT natural history study is to operationalize de novo the laboratory screening test paradigm for FNAIT risk and generate FNAIT laboratory test performance data for future regulatory discussions.
−Removed: We recently transitioned screening activities from the natural history study to the Phase 2 clinical trial where sites will continue to collect natural history data in women who do not receive RLYB212.
−Removed: We expect that natural history data from both the natural history study and the Phase 2 clinical trial will contribute historical control data to support a planned single-arm Phase 3 registrational clinical trial of RLYB212.
−Removed: Maternal fetal blood disorders
−Removed: FNAIT is one of several devastating disorders that is caused by an immune incompatibility of a mother and fetus during pregnancy.
−Removed: One of the best-characterized prenatal immune incompatibility disorders is Rh disease.
−Removed: This condition arises when the mother is RhD negative and her fetus is RhD positive.
−Removed: RhD incompatibility may lead to destruction of red blood cells in the fetus and can result in severe outcomes including miscarriage or loss of a newborn.
−Removed: Rh disease is treated by giving at-risk expectant mothers low doses of antibodies to RhD.
−Removed: These antibodies remove fetal red blood cells that have crossed into the mother’s circulation, thereby preventing her from developing an immune response that could destroy the red blood cells in the fetus.
−Removed: Since the approval of the first Rho (D) Immune Globulin in 1968, known as RhoGAM, expectant mothers in many countries, including in North America and Europe, are routinely screened for their RhD status, and Rh disease is largely prevented in at-risk expectant mothers.
−Removed: We are pursuing a similar approach to prevent FNAIT.
−Removed: FNAIT disease background
−Removed: Like Rh disease, FNAIT is a disorder that occurs during pregnancy when an expectant mother’s immune system attacks a specific antigen on the blood cells of her fetus, leading to their destruction.
−Removed: This results in an increased risk of bleeding in the fetus and newborn.
−Removed: In the majority of cases, the effects of FNAIT are mild;
−Removed: however, up to 20% of FNAIT cases experience intracranial hemorrhage ("ICH"), which can lead to devastating outcomes such as miscarriage, stillbirth, loss of the newborn and severe lifelong neurological disabilities in those babies that survive.
−Removed: FNAIT is caused by a mismatch in the type of HPA-1 that is expressed by the expectant mother and the fetus.
−Removed: There are two predominant forms of HPA-1, known as HPA-1a and HPA-1b, which are expressed on the surface of platelets.
−Removed: These two alleles differ by a single amino acid.
−Removed: Individuals who are homozygous for HPA-1b, meaning that they have two copies of the HPA-1b allele and no copies of the HPA-1a allele, are also known as HPA-1a negative.
−Removed: Upon exposure to HPA-1a, these individuals can develop antibodies to that antigen in a process known as alloimmunization.
−Removed: In expectant mothers, alloimmunization can occur upon mixing of fetal blood with maternal blood.
−Removed: When alloimmunization occurs in an expectant mother, the anti-HPA-1a antibodies that develop in the mother can cross the placenta and destroy platelets in the fetus.
−Removed: Pathophysiology of FNAIT
−Removed: There are no approved therapies to treat or prevent FNAIT and expectant mothers are not currently screened for FNAIT risk.
−Removed: Today, expectant mothers at risk of FNAIT are typically only identified following the delivery of an FNAIT affected child.
−Removed: These mothers may be treated during subsequent pregnancies with weekly administration of IVIG, along with the oral steroid immunosuppressant prednisone.
−Removed: While IVIG administration can potentially mitigate the detrimental effects of anti-HPA-1a antibodies, it does not prevent alloimmunization, is costly, time-intensive, difficult to tolerate and associated with significant treatment-related complications.
−Removed: Babies with FNAIT are typically diagnosed at the time of delivery by the presence of low platelet counts identified during routine analysis, the presence of petechiae on the skin or due to the manifestations of severe complications such as ICH or gastrointestinal bleeding.
−Removed: Upon diagnosis, babies with FNAIT may receive platelet transfusions and may be admitted to the neonatal intensive care unit.
−Removed: In severe cases, babies may suffer life-long neurological disability or may not survive.
−Removed: Based on previously published data and a 2024 epidemiological analysis that we sponsored, we believe that more than 30,000 pregnancies each year in the United States, Canada, the UK, other major European countries, and Australia are at higher risk for FNAIT and could potentially benefit from administration of a monoclonal anti-HPA-1a antibody such as RLYB212.
−Removed: These pregnancies represent expectant mothers who are HPA-1a negative, who are carrying an HPA-1a positive fetus and who are at high risk of alloimmunization due to the presence of a specific HLA allele, known as DRB3*01:01.
−Removed: Genetic studies have found that expectant mothers who have this specific HLA allele are approximately 25 times more likely to develop antibodies to HPA-1a than those without this allele.
−Removed: We believe that screening for FNAIT risk can be performed routinely and cost effectively as part of standard prenatal testing provided to expectant mothers during pregnancy.
−Removed: Testing for maternal HPA-1 type and presence of the HLA-DRB3*01:01 allele could occur during the first trimester at the same time as other routine blood work and risk screening.
−Removed: We don’t expect that an additional blood draw would be required.
−Removed: Importantly, U.S.
−Removed: and EU physicians have advised that our approach and timing for FNAIT screening would fit well within the established first trimester prenatal testing paradigm and could be performed at the same time as routine blood typing and Rh testing.
−Removed: Based on our global market research with maternal-fetal medicine specialists, obstetricians-gynecologists and payers, we believe there is high awareness of the catastrophic impact of FNAIT, and a strong desire to both screen and provide preventive therapy to at-risk expectant mothers if there were an approved product to prevent FNAIT and affordable screening tests.
−Removed: We believe screening and preventive treatment can have a significant impact on this potentially devastating disease.
−Removed: For example, screening and treatment in Rh disease have been highly effective in reducing the number of affected births.
−Removed: In developed countries with access to prenatal testing and treatment, the prevalence
−Removed: of Rh disease is 2.5 per 100,000 compared to 276 per 100,000 worldwide.
−Removed: We believe that applying a similar approach to the prevention of FNAIT could lead to a significant reduction in the number of babies at risk for FNAIT.
−Removed: In both the FNAIT natural history study and RLYB212 Phase 2 clinical trial, we are using screening tests for maternal HPA-1 type, maternal HLA-DRB3*01:01 status, maternal HPA-1a antibodies and fetal HPA-1 genotype.
−Removed: Our solution:
−Removed: RLYB212 preclinical data
−Removed: A mouse model of FNAIT has been created in which the amino acids comprising the HPA-1a antigen are reconstituted in the mouse gene.
−Removed: These transgenic mice (referred to as APLDQ mice based on the amino acid changes) recapitulate multiple aspects of FNAIT.
−Removed: Administration of anti-HPA-1a antibodies to APLDQ mice leads to destruction of APLDQ platelets and severe thrombocytopenia.
−Removed: Injection of platelets from APLDQ mice into wild-type mice can induce an HPA-1a specific immune response.
−Removed: Finally, wild-type female mice pre-immunized with APLDQ platelets, when bred with APLDQ male mice, give birth to severely thrombocytopenic pups, many of which exhibit an accompanying bleeding phenotype.
−Removed: Treatment of these pregnant female mice with IVIG resulted in lowering the level of anti-APLDQ antibodies in the fetus and a reduction in thrombocytopenia.
−Removed: In a prophylactic treatment model, a single large bolus intravenous injection of 1 x 108 APLDQ platelets (equivalent to about one-sixth of the total blood volume in the host) was administered to wild-type mice.
−Removed: At a dose of 0.4 µg (yielding a peak concentration of approximately 0.2 µg/ml), RLYB212 was able to drive rapid and complete elimination of APLDQ platelets, as shown in the first graph below, and prevent a host antibody response, as shown in the second graph below.
−Removed: Also shown in the graphs below, this dose correlates to a concentration of RLYB212 projected to bind approximately 10% of the HPA-1a antigen present on the transfused APLDQ platelets.
−Removed: Thus, the approximately 10% receptor binding is sufficient to clear platelets and prevent alloimmunization in the mouse model.
−Removed: RLYB212 Induced Rapid Elimination of APLDQ Platelets
−Removed: RLYB212 Prevented the Development of Antibodies to APLDQ Platelets
−Removed: RLYB212 Clinical Development
−Removed: We have completed two RLYB212 clinical trials:
−Removed: a Phase 1 first-in-human clinical trial and a Phase 1b proof-of-concept clinical trial.
−Removed: The Phase 1 first-in-human clinical trial was a single-blind, placebo-controlled study that investigated the safety and PK of SC administration of RLYB212 in HPA-1a negative healthy participants.
−Removed: The clinical trial included a single dose cohort and a multiple dose cohort.
−Removed: In the multiple dose cohort, subjects received SC RLYB212 or placebo every two weeks for 12 weeks.
−Removed: We reported results from the multi-dose cohort in the fourth quarter of 2023.
−Removed: The data demonstrated that multiple dose PK were consistent both within and between subjects.
−Removed: The data and our clinical pharmacology modeling predictions support a once monthly dosing regimen for the ongoing Phase 2 trial.
−Removed: RLYB212 was observed to be generally well-tolerated with no reports of injection site reactions or serious adverse events.
−Removed: The Phase 1b single-blind, placebo-controlled proof-of-concept trial was designed to establish the ability of SC RLYB212 to rapidly eliminate HPA-1a positive platelets transfused to HPA-1a negative healthy subjects.
−Removed: The study included 11 males aged 18 to 65 years, randomized to RLYB212 0.09 mg (n=4), RLYB212 0.29 mg (n=5), or placebo (n=2).
−Removed: The study design and results are summarized below.
−Removed: RLYB212 Phase 1b Trial — Design and Results
−Removed: In the fourth quarter of 2024 we initiated a Phase 2 clinical trial investigating RLYB212 in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT.
−Removed: This is a single-arm trial that is designed to assess the PK and safety of RLYB212 in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT.
−Removed: Secondary objectives include assessments of pregnancy and neonatal/infant outcomes, and the occurrence of emergent HPA-1a alloimmunization.
−Removed: Subcutaneous administration of RLYB212 will be initiated by Gestational Week 16 and will continue every four weeks through parturition.
−Removed: The trial is designed to enroll participants in three stages:
−Removed: first with a sentinel pregnant woman, an initial cohort (Cohort 1) that will include three pregnant women, and a second cohort (Cohort 2) that will include four pregnant women, for a total target enrollment of eight participants.
−Removed: We enrolled the sentinel woman in February 2025 and expect to report PK and safety data from the second trimester in the second quarter of 2025 and PK and safety data at the time of delivery in the third quarter of 2025.
−Removed: A data review for participants and infants is planned prior to the initiation of each cohort and continuation of the trial will be subject to evaluation of the results from each stage of the trial.
−Removed: The trial will seek to enroll participants at sites across Europe.
−Removed: An overview of the Phase 2 clinical trial design is illustrated below.
−Removed: Phase 2 Dose Confirmation Trial of RLYB212 in Pregnant Women
−Removed: Both the U.S.
−Removed: FDA and the EMA have designated RLYB212 as an orphan drug.
−Removed: Orphan drug designations offer certain incentives including tax credits, marketing exclusivity upon any approval, fee waivers, and the ability to interact with both agencies to receive specialized regulatory advice and assistance.
−Removed: Prospective FNAIT Natural History Alloimmunization Study
−Removed: We have an ongoing prospective, non-interventional, multinational natural history study.
−Removed: This study is designed to screen expectant mothers presenting at gestational week 10 to 14 prenatal visit to determine the frequency of women at higher FNAIT risk among expectant mothers of different racial and ethnic characteristics, as well as the frequency of HPA-1a alloimmunization and pregnancy outcomes among these women.
−Removed: An additional objective of the FNAIT natural history study is to operationalize de novo the laboratory screening test paradigm for FNAIT risk and generate FNAIT laboratory test performance data for future regulatory discussions.
−Removed: We recently transitioned screening activities from the natural history study to the Phase 2 clinical trial where sites will continue to collect natural history data in women who do not receive RLYB212.
−Removed: We expect that natural history data from both the natural history study and the Phase 2 clinical trial will contribute historical control data to support a planned single-arm Phase 3 registrational clinical trial of RLYB212.
RLYB116 for the treatment of disorders due to complement dysregulation
2 unchanged sentences
Antibody inhibitors of C5 have been successfully developed to treat diseases caused by immune dysfunction, including PNH, aHUS, refractory gMG and relapsing NMOSD.
−Removed: RLYB116 includes an Affibody molecule, which is an antibody mimetic protein that has a much smaller molecular weight than a traditional antibody and may also be easier and less costly to produce.
−Removed: In contrast to most C5-targeted antibody therapeutics that are administered intravenously or via daily injection, RLYB116 has the potential to be administered as a small volume, once-weekly SC injection.
−Removed: RLYB116 also includes an ABD, which may extend the half-life of the Affibody domain.
−Removed: In addition, amino acid substitutions that are part of RLYB116 are intended to enhance its stability.
−Removed: We view RLYB116 as a potential pipeline-in-a-product with disease areas under consideration including PNH, gMG and APS.
+Added: RLYB116 includes an Affibody molecule, which is an antibody mimetic protein that has a much smaller molecular weight than a traditional antibody and may be easier and less costly to produce.
+Added: In contrast to most C5-targeted antibody therapeutics that are administered intravenously or via daily injection, RLYB116 has the potential to be administered via an autoinjector as a small volume, once-weekly SC injection.
+Added: We view RLYB116 as a potential pipeline-in-a-product for many disease including platelet transfusion refractoriness ("PTR") and antiphospholipid syndrome ("APS").
We believe RLYB116 can address significant unmet needs for patients with these diseases by providing a potential treatment that is more accessible and patient-friendly than existing marketed products, including by reducing the frequency and improving the route of administration.
+Added: T a b le of Contents
Based on our team’s experience studying and developing therapies targeting the complement system, we believe there are five important attributes that could support clinical and commercial success in the treatment of a broad range of patients suffering from complement-mediated diseases.
1 unchanged sentence
once per week) in an easy to use autoinjector, and pricing flexibility to treat a broad range of complement-mediated diseases.
−Removed: Based on the data generated to date and the manufacturing process enhancements completed in 2024, we believe RLYB116 has the potential to demonstrate these attributes, and if so, could have a life-transforming impact on patients.
+Added: Based on the data generated to date and the manufacturing process enhancements completed in 2024, we believe RLYB116 has the potential to demonstrate these attributes as a best-in-class therapeutic, and if so, could have a life-transforming impact on patients.
The complement system
6 unchanged sentences
The binding of C5b to host cells is normally prevented by the presence of specific glycoproteins on the cell surface.
−Removed: PNH disease background
−Removed: PNH is a rare, potentially life-threatening hematologic disease characterized by complement-mediated destruction of red blood cells, or hemolysis.
−Removed: Early signs of PNH include hemoglobinuria, or dark colored urine, resulting from excretion of hemoglobin from lysed red blood cells, which is more prominent in the morning and decreases during the day.
−Removed: More serious symptoms of PNH include anemia, excessive weakness, fatigue, severe abdominal pain, severe headaches and recurrent infections.
−Removed: PNH leads to over a 60-fold increase in the risk of venous thromboembolism compared to the general population and these thrombotic events lead to between 40% and 67% of deaths in PNH patients.
−Removed: Approximately two thirds of patients with PNH develop chronic kidney disease ("CKD"), and kidney failure is the cause of death in 8% to 18% of patients with PNH.
−Removed: In the absence of disease-modifying treatment, PNH results in the death of approximately 35% of affected individuals within five years of diagnosis.
−Removed: The prevalence of PNH has been estimated to be approximately 12-13 people per million.
−Removed: Current treatments for PNH and their limitations
−Removed: The only curative treatment currently available for PNH is a stem cell transplant from a related donor.
−Removed: However, this procedure is associated with significant risk and is typically used only in those patients with severe disease, such as life-threatening thrombosis or dangerously low blood counts.
−Removed: Various supportive therapies include anticoagulants, red blood cell transfusions and supplements of iron and folate.
−Removed: These therapies provide some relief from symptoms but do not address the underlying cause of the disease.
−Removed: There are currently four approved disease-modifying drugs for PNH.
−Removed: Although these drugs have meaningfully improved the lives of patients with PNH, they have limitations including sub-optimal delivery (three of the four
−Removed: are delivered intravenously or via SC infusion) and high cost that result in limitations on access to therapy.
−Removed: Despite these limitations, worldwide sales of these drugs exceeded $5 billion in 2022.
−Removed: We believe that a product that works through a similar mechanism but with weekly dosing and a low-volume, convenient route of administration and improved patient access has the opportunity to further transform PNH therapy for patients.
−Removed: Potential benefits of our approach
−Removed: We believe PNH represents an attractive development opportunity for RLYB116 for several reasons.
−Removed: First, PNH has a well-understood disease pathophysiology driven by complement, providing a sound biological rationale for a C5-targeted intervention.
−Removed: Second, PNH offers the opportunity for early clinical validation using objective endpoints, including impact on lactate dehydrogenase, a component of red blood cells that is increased in circulation as a result of hemolysis.
−Removed: And third – and most importantly – we believe that with a patient-friendly (small volume, once-per-week delivery using an autoinjector) and accessible therapy, RLYB116 could potentially provide transformative therapeutic impact for unserved and underserved patients with PNH globally.
−Removed: gMG disease background
−Removed: gMG is a potentially life-threatening, rare autoimmune neuromuscular disorder.
−Removed: Patients with gMG develop antibodies that attack critical signaling proteins at the junction between nerve and muscle cells, thereby inhibiting the ability of nerves to communicate properly with muscles.
−Removed: This inhibition leads to muscle weakness, which can occur in ocular muscles leading to droopy eyelids as well as blurred or double vision, and can occur in the muscles in the face, neck, throat and jaw, causing difficulty chewing and swallowing as well as respiratory problems, speech difficulties and weakness in skeletal muscles leading to problems in limb function.
−Removed: While symptoms can be transient and remit spontaneously in the early stages of the disease, as the disease progresses, symptom-free periods become less frequent and disease exacerbations can last for months.
−Removed: Up to 20% of gMG patients experience respiratory crisis at least once in their lives.
−Removed: During such a crisis, a decline in respiratory function can become life-threatening and require intubation and mechanical ventilation.
−Removed: According to a comprehensive epidemiological study of gMG in western Denmark from 1975-89, from the time of diagnosis, the overall survival rates at 3, 5, 10, and 20 years are estimated to be 85%, 81%, 69% and 63%, respectively.
−Removed: Patients with gMG suffer from poor quality of life due to the impact of their disease on physical function and the burden of treatment-related adverse events.
−Removed: The prevalence of gMG has been estimated to be at least 100 people per million.
−Removed: Current treatments for gMG and their limitations
−Removed: In the first-line setting, patients presenting with symptomatic gMG are commonly treated with acetylcholinesterase inhibitors such as pyridostigmine, to improve neuromuscular transmission.
−Removed: As the disease progresses, patients may receive immunosuppressive therapies off-label;
−Removed: unfortunately, these therapies are associated with a substantial treatment burden and can even lead to a worsening of the disease.
−Removed: Targeted, therapies approved for treatment of gMG include complement inhibitors (e.g., Soliris, Ultomiris, Zilbrysq) and FcRn inhibitors (e.g., Vyvgart, Rystiggo).
−Removed: These treatments have yielded substantial symptom improvements, though unmet need remains for treatments that are more patient-friendly.
−Removed: Rituximab is another treatment used off-label for gMG, though it is believed to have more benefit in patients with anti-muscle-specific tyrosine kinase ("MuSK") antibodies than those with anti-AChR antibodies.
−Removed: For patients with severe gMG or recurrent exacerbation and crisis, there are a number of methods utilized to reduce circulating IgG antibodies.
−Removed: These procedures include plasma exchange and administration of IVIG.
−Removed: Both procedures are burdensome for patients and repeat administration is usually required to obtain significant reduction in symptoms.
−Removed: In addition, the large volumes of intravenous fluid associated with the administration of IVIG can lead to pulmonary edema and kidney complications.
+Added: Immune platelet transfusion refractoriness (PTR) disease background
+Added: PTR is a condition in which a patient shows poor platelet counts after a platelet transfusion due to an immune-mediated process.
+Added: It can result from antibodies directed against donor platelet antigens—especially human leukocyte antigen ("HLA") class I antigens, or human platelet antigens ("HPAs").
+Added: These antibodies rapidly clear transfused platelets from the circulation, leading to transfusion failure.
+Added: Immune PTR typically develops after prior antigen exposure, such as through previous transfusions.
+Added: It is diagnosed by consistently low post-transfusion platelet count in the absence of non-immune causes like sepsis or splenomegaly.
+Added: We estimate that there are more than 25,000 cases annually in the United States and other major markets.
+Added: Current treatments for PTR and their limitations
+Added: Management of PTR includes using HLA-matched, crossmatched, or HPA-matched platelets, or attempting to address the underlying immune sensitization.
+Added: Platelet matching, the current standard of care is expensive, challenging and is often not an option in local settings not directly connected to a major blood center.
+Added: Controlling the immune response is often expensive and is accompanied by a variety of undesirable side effects.
Potential benefits of our approach
−Removed: We are pursuing gMG as part of our initial development strategy for two reasons.
−Removed: First, complement overactivity is known to contribute to the disease pathophysiology of gMG, providing a sound biological rationale for a C5-targeted intervention.
−Removed: Second – and most importantly – our proprietary market research suggests that significant unmet need exists for more patient-friendly treatment options, which we believe could be addressed by RLYB116 as a once-weekly, small volume, self-administered SC therapeutic.
+Added: We believe PTR represents an attractive development opportunity for RLYB116 for several reasons.
+Added: First, there are data from at least one investigator initiated trial showing C5 blockade has a positive impact on post-transfusion platelet counts, providing a sound biological rationale for a C5-targeted intervention.
+Added: Second, PTR offers the opportunity for early clinical validation using objective endpoints, including impact on post-transfusion platelet counts.
+Added: And third – and most importantly – we believe that RLYB116 could potentially provide transformative therapeutic impact for unserved and underserved patients with PTR globally.
APS disease background
11 unchanged sentences
However, direct oral anticoagulants are generally less effective in preventing recurrent thromboembolic events, most notably strokes.
+Added: T a b le of Contents
Potential benefits of our approach
−Removed: We are pursuing APS as part of our initial development strategy for two reasons.
−Removed: First, there is significant unmet need in patients with primary and secondary APS who continue to suffer from thrombotic events despite standard anticoagulation therapy.
−Removed: Second, we believe that RLYB116 has the potential to be an effective treatment for this patient population.
−Removed: Complement overactivity likely contributes to the disease pathophysiology, providing a sound biological rationale for a C5-targeted intervention, and case reports evaluating the use of C5 inhibitors in patients experiencing recurrent thrombotic events despite standard anticoagulation therapy have demonstrated clinical benefit.
−Removed: RLYB116 as a once-weekly, small volume, self-administered subcutaneous therapeutics could represent a safe, effective, and patient-friendly option for APS patients with an inadequate response to standard of care.
−Removed: Our solution:
−Removed: RLYB116 is an engineered protein that includes an Affibody molecule and an ABD.
+Added: We believe APS is an attractive indication to pursue as part of a comprehensive development strategy for two reasons.
+Added: First, there is significant unmet need in patients with APS given the lack of currently available treatment alternatives and the rapid time to generate initial proof-of-concept data.
+Added: Second, we believe that RLYB116 has the potential to be an effective treatment for this patient population given published data from prior investigational studies using commercially available complement inhibitors not approved in this therapeutic indication.
+Added: The RLYB116 solution:
+Added: RLYB116 is an engineered protein that includes an Affibody molecule and an antibody binding domain ("ABD").
We acquired rights to RLYB116 from Swedish Orphan Biovitrum AB (Publ) ("Sobi").
3 unchanged sentences
The low molecular weight allows for a higher concentration of active molecules than antibodies in an equivalent volume.
−Removed: This increases the probability of being able to deliver RLYB116 in a volume suitable for SC administration in a patient friendly format such as an autoinjector.
+Added: This likely enables the delivery of RLYB116 in a patient friendly format such as an autoinjector.
• Efficiency of manufacturing.
−Removed: RLYB116 is expressed in E coli, providing for a more streamlined and potentially lower cost manufacturing process compared to antibodies or other biologics expressed in mammalian cell culture, which typically require larger scale and longer manufacturing times.
+Added: RLYB116 is expressed in E.
+Added: coli, providing for a more streamlined and potentially lower cost manufacturing process compared to antibodies or other biologics expressed in mammalian cell culture, which typically require larger scale and longer manufacturing times.
• Less frequent dosing .
Linkage of the Affibody domain to an ABD may lengthen the dosing interval of RLYB116 by extending the biological half-life and offer potential dosing benefits to patients.
+Added: Based on the data generated to date, we believe that RLYB116 will be administered once-weekly.
• Potentially lower risk of treatment conversion .
4 unchanged sentences
Clinical development of RLYB116
−Removed: We have completed a Phase 1 clinical trial in healthy participants that included the study of RLYB116 as a SAD and a MAD.
−Removed: The SAD design was a single-blind, placebo-controlled, dose escalation trial investigating the safety, PK and PD of single dose RLYB116 in healthy participants.
−Removed: The SAD portion of the Phase 1 trial included five sequential ascending dose cohorts with doses ranging from 2mg up to 300mg, each enrolling eight subjects (six treated with RLYB116 and two with placebo) with a 10 week post-treatment / safety follow-up period.
−Removed: An overview of the clinical trial’s design is illustrated below.
−Removed: Completed Phase 1 Clinical Trial of RLYB116 in Healthy Participants (Single Ascending Dose)
−Removed: Data from the SAD portion of the trial showed consistent increases in exposure with increasing dose levels, low inter-subject variability, and a mean elimination half-life greater than 300 hours after a single SC, low volume injection.
−Removed: In the 100 mg dose group study participants that received RLYB116 (n=6) had a reduction in free C5 greater than 99% within 24 hours of dosing and RLYB116 was observed to be generally well-tolerated at this dose, with mild adverse events and no drug-related serious adverse events reported.
−Removed: Based on the data from the 100 mg dose and the volume of administration of the 300 mg dose, we elected to proceed with the 100 mg dose to initially evaluate multiple dose administration of RLYB116.
−Removed: Across the five single dose cohorts, adverse events were generally mild to moderate in severity with a dose-related increase in the frequency of gastrointestinal adverse events.
−Removed: There were no drug-related serious adverse events.
−Removed: The MAD portion of the Phase 1 trial included an adaptive single-blind design with a 4-week treatment duration to evaluate the safety, tolerability, PK, and PD of RLYB116 with multiple dose SC administration.
−Removed: The MAD portion of the trial included four cohorts:
−Removed: Cohort 1 (weekly dosing of 100 mg), Cohort 2 (three doses of 100 mg the first week followed by weekly dosing), Cohort 3 (150 mg weekly dosing reduced to 125 mg weekly dosing) and Cohort 4 (75 mg twice the first week followed by 100 mg twice per week) with post-treatment / study follow-up for 10 weeks.
−Removed: Completed Phase 1 Clinical Trial of RLYB116 in Healthy Participants (Multiple Ascending Dose)
−Removed: In December 2023, we reported data from the MAD portion of the Phase 1 trial that demonstrated a 100 mg low volume (1 mL) once-weekly dose of subcutaneously administered RLYB116 achieved sustained mean reductions in free C5 of greater than 93%, including at day 29 with measurement prior to the last dose.
−Removed: The reduction in free C5 at 24 hours after the first dose of 100 mg was greater than 99%.
−Removed: In the MAD portion of the Phase 1 trial, RLYB116 also demonstrated low inter-subject variability and consistent increases in exposure relative to dose with a mean estimated elimination half-life for RLYB116 of >300 hours.
−Removed: RLYB116 administered as a 100 mg once-weekly dose was also observed to be generally well tolerated.
−Removed: Across the MAD portion of the trial, injection site reaction (ISR) was the most common adverse event (all mild in severity) and gastrointestinal
−Removed: adverse events increased with increasing dose.
−Removed: There was one case of severe liver function test ("LFT") elevation in Cohort 3 in a participant with a history of hepatitis that resulted in discontinuation of treatment.
−Removed: Finally, the measurement of anti-drug antibody ("ADA") formation in the study did not demonstrate an effect on PK or PD parameters and did not appear to be associated with an effect on the severity or incidence of adverse events.
−Removed: Based on the results of the RLYB116 Phase 1 trial, we conducted a series of biomarker characterization analyses.
−Removed: These analyses indicate the RLYB116 assay used to measure free C5 in the Phase 1 trial overestimated the levels of free C5 by approximately ten-fold, indicating that RLYB116 produced greater complement inhibition than initially reported.
−Removed: We now believe that RLYB116 has the potential to be an effective treatment for patients with a variety of complement-mediated diseases, including PNH, gMG and APS.
−Removed: We also completed manufacturing process enhancements with a goal of further improving the tolerability of RLYB116.
−Removed: Based on the results of enhanced analytical techniques, including mass spectrometry, these process enhancements have successfully further purified the RLYB116 drug substance.
−Removed: As a result, we believe that RLYB116 will have a favorable tolerability profile at doses at and above those evaluated in the Phase 1 MAD trial.
−Removed: We plan to initiate a RLYB116 confirmatory clinical PK/PD trial in the second quarter of 2025 to demonstrate improved tolerability as well as complete and sustained complement inhibition.
−Removed: This single-blind MAD trial will evaluate a 4-week treatment duration that will include two cohorts of eight participants each.
−Removed: Our current plan is that Cohort 1 will evaluate weekly dosing of 150 mg and Cohort 2 will evaluate weekly dosing of 225 mg with 10 weeks of follow-up after the conclusion of treatment.
−Removed: The planned confirmatory trial is summarized below:
−Removed: Planned Confirmatory Multiple Ascending Dose Phase 1 Clinical Trial of RLYB116 in Healthy Participants
−Removed: Based on market research conducted to date, we believe that an effective, once-weekly, well-tolerated therapy that can be rapidly self-administered with an autoinjector would be an attractive alternative for patients suffering from gMG and other complement mediated diseases.
−Removed: RLYB114 for the treatment of ophthalmic disorders
−Removed: RLYB114 is a C5-targeted Affibody molecule conjugated to polyethylene glycol ("PEG").
−Removed: The addition of PEG to protein therapeutics is a well-established method of extending the half-life and reducing the immunogenicity of molecules in the body.
−Removed: Given the role of the complement system in retinal and ocular pathology, we believe that RLYB114 could be used to treat ophthalmic diseases, including inflammatory and degenerative disorders.
−Removed: Potential role of complement in ocular diseases
−Removed: Dysregulation of the complement system may drive ocular inflammation and contribute to vision loss in multiple diseases such as age-related macular degeneration ("AMD").
−Removed: A number of genetic studies have shown links between alterations in genes encoding various complement factors and the risk of development of AMD.
−Removed: Several clinical trials of inhibitors of the complement pathway including C5 inhibitors have been conducted, with reports of modest efficacy.
−Removed: Reasons for this limited efficacy are unknown but could include the disease stage, level of intervention in the complement pathway, drug delivery mechanism and the ability of the therapeutic to cross Bruch’s membrane and the retinal pigment epithelium.
−Removed: Our research collaboration with EyePoint Pharmaceuticals to evaluate delivery of RLYB114 using EyePoint’s technology for sustained intraocular drug delivery ended in January 2025.
+Added: We have completed two Phase 1 clinical trials in healthy participants that included the study of RLYB116 as a SAD and a MAD.
+Added: After the first Phase 1 clinical trial, we completed manufacturing process enhancements that were designed to improve the tolerability of RLYB116 and enable us to increase the dose.
+Added: In 2025, we completed the confirmatory (second) Phase 1 clinical trial evaluating the PK /PD properties of RLYB116 in healthy volunteers.
+Added: The trial was a single-blind MAD study evaluating a 4-week treatment duration and a 10-week follow-up period.
+Added: There were eight participants in each of 2 cohorts, six of whom received RLYB116 and two of whom received placebo.
+Added: The trial is summarized below.
+Added: T a b le of Contents
+Added: The trial achieved its two key objectives including:
+Added: a significant improvement in the tolerability of RLYB116 and demonstration of complete and sustained inhibition of terminal complement.
+Added: We believe the results, which are summarized in the three graphs below, support the study of RLYB116 as a potential best-in-class therapeutic for multiple complement mediated diseases.
+Added: Serum RLYB116 Concentration
+Added: The PK profile of RLYB116 is highly predictable with low intersubject variability, rapid absorption and long terminal elimination rates.
+Added: RLYB116 to C5 Ratio
+Added: RLYB116 levels significantly exceed C5 within 12 hours of the first dose and are maintained at ratios of greater than 2:1 with a once-weekly, subcutaneously administered dose of 300mg.
+Added: T a b le of Contents
+Added: Hemolytic Activity
+Added: Most importantly, we observed complete and sustained inhibition of hemolysis in subjects who received the 300mg dose.
+Added: Based on market research conducted to date, we believe that an effective, once-weekly, well-tolerated therapy that can be rapidly self-administered with an autoinjector would be an attractive alternative for patients suffering from a wide array of complement mediated diseases.
RLYB332 for the treatment of severe anemia with ineffective erythropoiesis and iron overload
1 unchanged sentence
The inhibition of MTP-2 significantly increases levels of hepcidin, decreases iron load and treats ineffective erythropoiesis.
−Removed: We believe this molecule has the potential to address a significant unmet need for
−Removed: patients with severe anemias with ineffective erythropoiesis and iron overload, including beta thalassemia and a subset of lower risk MDS.
+Added: We believe this molecule has the potential to address a significant unmet need for patients with severe anemias with ineffective erythropoiesis and iron overload, including beta thalassemia and a subset of lower risk MDS.
Currently these patients are underserved by the existing standard of care.
1 unchanged sentence
These findings, which were presented in a poster at the 66th annual meeting of ASH, support the continued development of RLYB332 as a potentially best-in-class therapeutic for treating diseases of iron overload.
−Removed: Given the potential value of this program, we are currently assessing the most effective way to move this program through preclinical development towards a potential investigational new drug ("IND") application filing.
−Removed: AI drug discovery collaboration with Recursion
−Removed: We established a partnership with Exscientia (acquired by Recursion), an AI and machine learning drug discovery company.
−Removed: Our partnership currently consists of a joint venture that is focused on the discovery and development of small molecules for the treatment of patients with rare metabolic diseases.
−Removed: We are targeting ENPP1, an enzyme involved in regulating extracellular levels of pyrophosphate, a natural inhibitor of calcium mineralization in bone formation, for the treatment of patients with HPP.
−Removed: RE Ventures I, LLC :
−Removed: ENPP1 inhibitor program for the treatment of patients with HPP
−Removed: We are developing an ENPP1 inhibitor for the treatment of patients with HPP, a rare, potentially life-threatening genetic disease characterized by mutations in the ALPL gene.
−Removed: These mutations lead to diminished activity of the tissue non-specific alkaline phosphatase ("TNSALP") enzyme and the accumulation of PPi, which inhibits bone mineralization causing multiple skeletal pathologies.
−Removed: ENPP1 is a Type II transmembrane glycoprotein that cleaves ATP, producing PPi, and is a major source of PPi production in cells.
−Removed: We believe that controlling inhibition of ENPP1 may reduce PPi levels and restore balance within the bone mineralization process.
−Removed: In 2024, we identified and advanced REV102, an ENPP1 inhibitor for the treatment of patients with HPP.
−Removed: We also presented data at the ASBMR from an early lead ENPP1 inhibitor, REV101, in a mouse model of later-onset HPP demonstrating a 30% reduction in inorganic PPi, a key biomarker that is elevated in HPP and contributes to poor bone mineralization.
−Removed: HPP disease overview
−Removed: HPP is an inherited disorder that affects the development of bones and teeth.
−Removed: The most severe forms of the disorder tend to occur before birth and in early infancy.
−Removed: Infants afflicted with the disorder have short limbs, an abnormally shaped chest, soft skull bones, poor feeding, failure to gain weight, respiratory complications and high levels of calcium in the blood, or hypercalcemia, which can lead to life-threatening complications.
−Removed: In other cases, the disease is not recognized until later in childhood where it manifests as rickets, pain, decreased mobility, deficits of growth and fractures.
−Removed: Children with less severe HPP can experience early loss of primary teeth and may have short stature with bowed legs or knock knees, enlarged wrist and ankle joints and an abnormal skull shape.
−Removed: Findings in adults include a softening of the bones, known as osteomalacia, and recurrent fractures in the foot and thigh bones that can lead to chronic pain.
−Removed: The incidence of HPP has been reported to be 1 in 100,000 (United States and Canada) to 1 in 300,000 (EU) for severe disease and 1 in 6,370 (EU) for less severe forms.
−Removed: The various manifestations of HPP are caused by the combination of a lack of phosphate and an excess of PPi due to a deficiency of TNSALP, the enzyme that converts PPi to phosphate.
−Removed: This deficiency negatively impacts bone formation by reducing the hydrolysis of PPi to phosphate required for normal bone formation, resulting in a build-up of PPi, a potent inhibitor of mineralization.
−Removed: Strensiq, an enzyme replacement therapy marketed by AstraZeneca plc ("AstraZeneca"), is the only approved therapy to treat patients with perinatal-, infantile-and juvenile-onset HPP.
−Removed: The therapy has been shown to lead to significant improvements in morbidity and mortality in patients with perinatal- and infantile-onset HPP, and improvements in morbidity for patients with juvenile-onset HPP.
−Removed: However, Strensiq has limitations, including its dosing regimen and patient access.
−Removed: Strensiq is administered by SC injection either three or six times per week using a weight-based dosing scale, which can be both onerous and painful for patients.
−Removed: Our solution:
−Removed: REV102, an ENPP1 small molecule inhibitor
−Removed: REV102 is an orally available small molecule inhibitor of ENPP1 for the treatment of patients with HPP.
−Removed: We are developing REV102 to be a safe, tolerable, and accessible option for juvenile- and adult-onset HPP patients as either monotherapy or add-on therapy to ERT.
−Removed: We believe that REV102 has the potential to address a
−Removed: significant unmet need for patients and plan to conduct IND-enabling studies in 2025 to support the initiation of a Phase 1 trial in 2026.
−Removed: Scientific Rational:
−Removed: ENPP1 Inhibition for the Treatment of Hypophosphatasia
−Removed: Under normal conditions, the level of PPi and Pi is kept in balance by activity of TNSALP, ENPP1 and ANKH.
−Removed: In patients with HPP, the reduction of PPi hydrolysis by TNSALP results in a relative increase in PPi, leading to an inhibition of mineralization, and inhibited hydroxyapatite formation.
−Removed: Through controlled inhibition of ENPP1, we aim to reduce PPi and improve mineralization, restoring hydroxyapatite formation.
AbCellera Collaboration
1 unchanged sentence
This multi-year, multi-target collaboration combines AbCellera’s antibody discovery engine with Rallybio’s clinical and commercial expertise in rare diseases to identify optimal clinical candidates with a goal of delivering therapies to patients.
−Removed: AbCellera and Rallybio intend to co-develop up to five rare disease therapeutic targets, which will be chosen together by both companies.
The biotechnology and pharmaceutical industries are highly competitive and subject to significant and rapid technological change.
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The success of our product candidates, if approved, is likely to be a result of their efficacy, safety, convenience, price, the level of biosimilar or generic competition and/or the availability of reimbursement from government and other third-party payors.
−Removed: There are currently no approved therapies for the prevention or treatment of FNAIT.
−Removed: In one frequently used approach to manage pregnancies where the mother is known to have a history of FNAIT, physicians administer high levels of IVIG.
−Removed: Companies that currently market IVIG include ADMA Biologics, Bio Products Laboratory, CSL Behring, Grifols, Kedrion Biopharma, Leadiant Biosciences, Octapharma and Takeda Pharmaceutical Company Limited.
−Removed: In another approach, to treat FNAIT (which is different than Rallybio's approach to prevent FNAIT), Johnson & Johnson is currently evaluating the effectiveness of nipocalimab (compared with placebo) to reduce the risk of FNAIT in women that have a history of at least one prior FNAIT-affected pregnancy.
−Removed: The only curative treatment currently available for PNH is a stem cell transplant from a related donor.
−Removed: However, this procedure is associated with significant risk and is used only in those patients with severe disease, such as life-threatening thrombosis or dangerously low blood counts.
−Removed: Various supportive therapies include anticoagulants, red blood cell transfusions and iron and folate supplements.
−Removed: These therapies provide some relief from symptoms but do not address the underlying cause of the disease.
−Removed: There are four approved drugs for PNH:
−Removed: eculizumab, marketed by AstraZeneca as Soliris;
−Removed: ravulizumab, marketed by AstraZeneca as Ultomiris;
−Removed: pegcetacoplan, marketed by Apellis Pharmaceuticals as Empaveli, and iptacopan, marketed by Novartis AG as Fabhalta.
−Removed: Eculizumab and ravulizumab are antibodies that bind complement C5;
−Removed: pegcetacoplan is a pegylated pentadecapeptide that targets complement C3;
−Removed: and iptacopan is a small molecule that inhibits complement factor B.
−Removed: There are several companies in mid- to late-stage clinical trials developing treatments for PNH.
−Removed: These include Roche, AstraZeneca, and Alnylam Pharmaceuticals, Inc.
−Removed: among others.
−Removed: Very early-stage gMG is symptomatically treated by the use of acetylcholinesterase inhibitors such as pyridostigmine bromide, marketed as Mestinon by Bausch Health.
−Removed: Eculizumab and ravulizumab are also approved for the treatment of gMG in patients who are positive for anti-AChR antibodies.
−Removed: Efgartigimod, marketed as Vyvgart by Argenx SE, is a neonatal Fc receptor blocker also approved for the treatment of patients with generalized MG who are positive for anti-AChR antibodies and Zilbrysq, a macrocyclic peptide marketed by UCB Pharma was approved for the treatment of AChR+ adult patients with gMG.
−Removed: Rozanolixizumab, marketed as Rystiggo by UCB, is a neonatal Fc receptor blocker approved for the treatment of gMG in adult patients who are AChR+ or anti-MuSK antibody positive.
−Removed: There are several other companies developing assets in mid- to late-stage clinical development for the treatment of gMG using a variety of approaches and modalities.
−Removed: These companies include AstraZeneca, Horizon Therapeutics (acquired by Amgen Inc.) and Immunovant, Inc.
−Removed: There is one approved treatment for HPP, asfotase alfa, marketed by AstraZeneca as Strensiq, which is an alkaline phosphotase enzyme replacement therapy, and the only approved therapy for the treatment of perinatal-, infantile- and juvenile-onset HPP.
−Removed: AstraZeneca is developing a second generation enzyme replacement therapy, ALXN-1850 which is currently in clinical development, and Aruvant is developing ARU-2801, an AAV gene therapy currently in preclinical development.
−Removed: There are several companies pursuing ENPP1 small molecule inhibitors for the treatment of cancer, including Angarus Therapeutics, Avammune Therapeutics, Tcino Bioscience, Nanjing Zenshine Pharmaceuticals Co., Ltd.
−Removed: and Stingray Therapeutics, Inc.
−Removed: with all these companies in discovery or preclinical development.
−Removed: Alesta Therapeutics B.V.
−Removed: recently announced work on an ENPP1 small molecule inhibitor for the treatment of patients with HPP.
Many of our competitors may have significantly greater name recognition and financial, manufacturing, marketing, product development, technical, commercial infrastructure, and human resources than we do.
−Removed: Mergers and acquisitions in the pharmaceutical, biotechnology and diagnostic industries may result in even more resources being concentrated among a smaller number of our competitors.
+Added: Mergers and acquisitions in the pharmaceutical, biotechnology and diagnostic industries may result in even
+Added: T a b le of Contents
+Added: more resources being concentrated among a smaller number of our competitors.
These competitors also compete with us in establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies complementary to, or necessary for, our programs.
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Marketing exclusivity may also be granted for new clinical studies (3 years) and pediatric studies (6 months) on approved drugs.
−Removed: Depending on the length of the regulatory approval process and the ability to make use of the procedures for obtaining PTE, any FDA exclusivity period may in part or in whole overlap with any patent exclusivity to which we are entitled.
+Added: Depending on the length of the regulatory approval process and the
+Added: T a b le of Contents
+Added: ability to make use of the procedures for obtaining PTE, any FDA exclusivity period may in part or in whole overlap with any patent exclusivity to which we are entitled.
We intend to pursue relevant marketing exclusivities in the US and in foreign countries in which any candidate product is approved.
2 unchanged sentences
We also cannot be certain that any of our activities will not be subject to the intellectual property rights of others.
−Removed: As of March 1, 2025, we owned two patent families that were acquired from Prophylix AS ("Prophylix") and relate to product candidates in our FNAIT prevention program, RLYB211 and RLYB212.
−Removed: The acquired patent family covering RLYB212 and its use in treating and preventing FNAIT includes patents issued or accepted in Australia, Canada, Europe, Israel, Mexico, New Zealand, Russia, and the United States.
−Removed: A patent application in this family is pending in Brazil.
−Removed: The granted patents in this family will expire in 2035, excluding any PTA or PTE that may be awarded.
−Removed: The acquired patent family covering administration of RLYB211 for the prophylactic treatment of FNAIT includes patents issued in the United States, Europe and Canada.
−Removed: The foreign patents and one of the U.S.
−Removed: patents will expire at the end of 2026, while the other U.S.
−Removed: patent expires in November 2030 due to a PTA granted by the USPTO.
−Removed: We own three patent families directed to dosing and administration of RLYB212.
−Removed: Two of these families each include patent applications pending in Australia, Canada, Europe, Israel, Mexico, New Zealand, and the United States.
−Removed: Patents issuing in these two families will have an expiration date of June 2042, excluding any PTA or PTE that may be awarded.
−Removed: The third family is a pending International (PCT) patent application.
−Removed: In addition, we filed and own a patent family directed to assays for quantifying anti-HPA1a
−Removed: antibodies, with patent applications pending in Australia, Brazil, Canada, Europe, Israel, Mexico, New Zealand, South Africa and the United States.
−Removed: We also filed and own a pending International (PCT) application directed to the formulation of RLYB212.
−Removed: In addition, we exclusively in-license certain rights to technology from Versiti Blood Research Institute Foundation, Inc.
−Removed: pertaining to a mouse model of FNAIT.
As of March 1, 2026, we owned two patent families relating to the current product candidates in our complement program, RLYB114 and RLYB116, and certain aspects of their use that were acquired from Sobi.
5 unchanged sentences
Patents issuing in this patent family will have an expiration date in November 2042, excluding any PTA or PTE that may be awarded.
−Removed: We also filed and own eight pending provisional patent applications directed to methods of treatment of various C5-related conditions by administration of RLYB116.
+Added: We also filed and own nine pending provisional patent applications directed to methods of treatment of various C5-related conditions by administration of RLYB116.
We have also non-exclusively in-licensed certain patent rights relating to our current product candidates from Affibody, including patent rights relating to the Affibody molecule technology and Albumod albumin binding molecule technology.
2 unchanged sentences
The patent family currently includes pending patent applications in the United States and in more than 20 other countries/regions, including Australia, Brazil, Canada, China, Eurasia, Europe, India, Japan, Mexico, and Saudi Arabia.
+Added: Patents have been granted in Eurasia, Japan, and Malaysia, and are scheduled to expire in November 2040.
In addition, we filed and own a pending International (PCT) patent application family directed to use of RLYB332 for treating pathologies of beta-thalassemia.
10 unchanged sentences
Under the PLA, Affibody is the exclusive owner of, and controls prosecution, maintenance, and defense of intellectual property covering, platform technology.
−Removed: We are the exclusive owner of, and control prosecution, maintenance, and defense of intellectual property covering, product technology.
+Added: We are the exclusive owner of, and control prosecution,
+Added: T a b le of Contents
+Added: maintenance, and defense of intellectual property covering, product technology.
Affibody agrees to disclose to us any improvement to the Affibody technology that it deems commercially reasonable for us to practice and grants us an option to license any such improvement.
23 unchanged sentences
If the Sanofi License Agreement is terminated in its entirety, among other things (a) all rights and licenses granted by Sanofi under the License Agreement (including any sublicenses) will terminate and (b) if Sanofi has an interest in developing, manufacturing and commercializing the licensed compounds or products, the parties to the Sanofi License Agreement shall negotiate an arrangement to provide Sanofi rights to the patents, know-how, materials and other properties controlled by Rallybio applicable to any of the licensed product.
+Added: T a b le of Contents
Asset Purchase Agreements
8 unchanged sentences
This right of negotiation will not apply to a transaction to sell all or substantially all of the assets of IPC Research or an affiliate of IPC Research, a pledge of the assets as collateral or a sale or transfer of the assets to an affiliate of IPC Research that agrees to be bound by the right of negotiation.
−Removed: Asset Purchase Agreement with Prophylix
−Removed: In June 2019, though our subsidiary Rallybio IPA, LLC ("Rallybio IPA"), we entered into an agreement with Prophylix to acquire all of Prophylix’s rights, title and interest in, to and under all assets, properties and rights related to Prophylix’s plasma-derived anti-HPA-1a immunoglobulin, and Prophylix’s monoclonal antibody, which we are developing as RLYB212.
−Removed: We paid Prophylix an upfront purchase price of approximately $1.2 million and reimbursed Prophylix approximately $1.8 million for certain manufacturing costs incurred by Prophylix.
−Removed: We are obligated to pay Prophylix an aggregate of up to $ 19.0 million upon achievement of certain development milestones and an aggregate of up to $20.0 million upon achievement of certain sales milestones.
−Removed: We also will pay Prophylix tiered, mid-single-digit royalties on annual net sales of products containing the monoclonal antibody and tiered mid-to-high-single and low-double-digit royalties on annual net sales of products containing plasma-derived anti-HPA-1a immunoglobulin, subject to certain offsets for royalties payable under certain third-party licenses.
−Removed: Furthermore, the then-applicable royalty rate will be reduced by a mid-double digit percentage for the remaining royalty term on a country-by-country basis if it becomes reasonably likely that the Prophylix patents may no longer be enforceable in such country due to a challenge of the enforceability of the patents or the enforceability of the royalty payments following the expiration of all valid claims of the patents in such country.
−Removed: Our obligation to pay royalties terminates on a country-by-country and product-by-product basis on the later of (a) the expiration of the last-to-expire valid claim of a Prophylix patent covering a product, (b) expiration of regulatory exclusivity for the product in such country or (c) the 10 th year anniversary following first commercial sale of such product in such country.
−Removed: In the event the FDA grants a priority review voucher for one of our product candidates developed using the technology acquired from Prophylix, we will pay Prophylix either:
−Removed: (a) if we sell such priority review voucher to a third-party within 12 months of its receipt, a mid-double digit percentage of the proceeds we receive from the sale, net of taxes, or (b) if we do not sell the priority review voucher to a third-party within 12 months of receipt, a mid-double digit percentage of the fair market value of the priority review voucher as determined in accordance with the agreement.
−Removed: We are obligated to use commercially reasonable efforts to develop and commercialize products containing plasma-derived anti-HPA-1a immunoglobulin in the United States and in at least one major European market.
−Removed: The agreement provides that if we provide notice to Prophylix that we determined that commercialization of products containing plasma-derived anti-HPA-1a immunoglobulin is not feasible due to an insufficient plasma supply following our continued and diligent efforts to obtain a sufficient plasma supply, then our obligation to develop products containing plasma-derived anti-HPA-1a immunoglobulin will cease, and we will be obligated to use commercially reasonable efforts to develop and commercialize products containing the monoclonal antibody in the United States and in at least one major European market.
−Removed: If, after using commercially reasonable efforts to develop and commercialize products containing plasma-derived anti-HPA-1a immunoglobulin and the monoclonal antibody in the United States and in at least one major European market, we decide not to pursue any further development or commercialization activities for such products, then Prophylix will have the right to repurchase the remaining assets acquired under the agreement for approximately $1.2 million.
−Removed: Prophylix also will have the right to repurchase the remaining assets acquired under the agreement for approximately $1.2 million if we elect to transfer all or substantially all of the assets acquired under the agreement to a third-party who does not agree to assume our obligations to develop and commercialize the products.
Joint Venture Agreement
−Removed: In July 2019, we entered into a partnership with Recursion (as successor in interest to Exscientia) and created RE Ventures I, LLC ("REV-I"), which is jointly owned by Recursion and one of our wholly-owned subsidiaries, each a Member and collectively the Members.
+Added: In July 2019, we entered into a partnership with Recursion (as successor in interest to Exscientia) and created REV-I, which was jointly owned by Recursion and one of our wholly-owned subsidiaries, each a Member and collectively the Members.
The joint venture was formed to initiate early-stage drug discovery of orally available small molecules targeting ENPP1 for the treatment of HPP, and thereafter for the future research, development, manufacture, sale and exploitation of any company-owned technology and compounds, including any resulting compound identified by the steering committee of the joint venture.
−Removed: Under the REV-I operating agreement, we received a 50% interest in the joint venture in exchange for an initial contribution of £0.5 million ($0.6 million, based on the exchange rate at the time).
−Removed: REV-I used this initial capital to fund stage 1 of the ENPP1 program, and we committed to fund additional amounts if costs of stage 1 exceeded the initial funding.
−Removed: In June 2020, REV-I determined that the stage 1 objective of discovering compounds for ENPP1 with a certain potency had been achieved.
−Removed: In 2020, we contributed £ 1.1 million ($1.3 million, based on the exchange rate at the time) and in 2021, we contributed approximately £1.4 million ($2.0 million, based on the exchange rate at the time) to REV-I in support of ongoing Stage 2 development of the ENPP1 program.
−Removed: In 2022, we contributed £0.2 million ($0.3 million, based on the exchange rate at the time), in 2023, we contributed £1.8 million ($2.3 million, based on the exchange rate at the time) and in 2024 we contributed £1.6 million ($2.0 million, based on the exchange rate at the time) to REV-I in support of ongoing development of the ENPP1 program.
−Removed: The board of managers of REV-I may determine from time to time that additional capital is necessary or appropriate to enable REV-I to conduct its activities, and may seek (but not require) additional capital contributions from the Members.
−Removed: In the event that either Member does not fund a portion of committed additional amounts, the other Member may contribute the unfunded amount and the respective membership interests in REV-I will be adjusted accordingly.
−Removed: A steering committee is responsible for oversight of REV-I’s research and deployment plans as well as intellectual property and regulatory matters.
−Removed: A two-person board of managers manages the business and affairs of REV-I and is responsible for all management and other responsibilities not specifically reserved to the steering committee or to the Members.
−Removed: Each Member designates one member to the board.
−Removed: Each Member is subject to customary restrictions on its transfer of interests in REV-I, including a right of first refusal, co-sale right and drag-along provision.
+Added: In July 2025, we entered into a Membership Interest Purchase Agreement (the “ENPP1 Purchase Agreement”) with Recursion Exscientia Ventures I, Inc., an indirect wholly-owned subsidiary of Recursion (“Buyer”) and Rallybio IPB, LLC, a wholly-owned subsidiary of Rallybio Corporation to sell our interest in REV102, ENPP1 inhibitor in preclinical development for the treatment of patients with HPP, to Buyer (a subsidiary of our joint venture partner Recursion) (the "JV Sale").
+Added: In connection with the JV Sale, we received a total of $20.0 million in the third quarter of 2025 including $7.5 million from an upfront payment and $12.5 million from a milestone payment related to the initiation of additional preclinical studies.
+Added: We are eligible to receive a $5.0 million milestone payment in connection with the initiation of dosing in a Phase 1 clinical study, as defined in the ENPP1 Purchase Agreement and low single-digit royalties on all future net sales by Recursion of products comprising or incorporating certain compounds developed by REV-I.
+Added: We may also be eligible to receive certain payments in the event of Recursion’s sale of the REV102 program.
AbCellera Collaboration Agreement
In December 2022, the Company entered into a strategic alliance to discover, develop, and commercialize novel antibody-based therapeutics for rare diseases.
−Removed: This multi-year, multi-target collaboration will combine AbCellera’s antibody discovery engine with Rallybio’s clinical and commercial expertise in rare diseases to identify optimal clinical candidates and ultimately deliver therapies to patients.
−Removed: Under the terms of the agreement, AbCellera and Rallybio will co-develop up to five rare disease therapeutic targets, which will be chosen together by both companies.
−Removed: The collaboration will allow Rallybio to add product candidates to its existing pipeline and also provides the option for AbCellera to conduct process development and clinical manufacturing activities.
+Added: This multi-year, multi-target collaboration combined AbCellera’s antibody discovery engine with Rallybio’s clinical and commercial expertise in rare diseases to identify optimal clinical candidates and ultimately deliver therapies to patients.
+Added: Under the terms of the agreement, AbCellera and Rallybio were planning to co-develop up to five rare disease therapeutic targets, which will be chosen together by both companies.
+Added: The collaboration was designed to allow Rallybio to add product candidates to its existing pipeline and provide the option for AbCellera to conduct process development and clinical manufacturing activities.
+Added: T a b le of Contents
Collaboration Agreement with Johnson & Johnson
In April 2024, through Rallybio IPA, we entered into a collaboration agreement (the “J&J Collaboration Agreement”) with Johnson & Johnson, through its wholly-owned subsidiary, Momenta Pharmaceuticals, Inc.
−Removed: Under the J&J Collaboration Agreement, the Company and J&J will advance therapeutic solutions for pregnant individuals at risk of FNAIT.
−Removed: The Company will share certain aggregated, anonymized data with J&J, collected from the Company’s FNAIT natural history study and the Company’s Phase 2 FNAIT clinical trial, where the Phase 2 data will be restricted to certain natural history data in support of the FNAIT natural history study.
+Added: Under the J&J Collaboration Agreement, the Company and J&J planned to advance therapeutic solutions for pregnant individuals at risk of fetal and neonatal alloimmune thrombocytopenia ("FNAIT").
+Added: The Company shared certain aggregated, anonymized data with J&J, collected from the Company’s FNAIT natural history study and the Company’s Phase 2 FNAIT clinical trial.
The Company also agreed to disseminate information to its FNAIT study sites related to J&J’s and its affiliates’ research and development of complementary therapeutic approaches aimed at reducing the risk of FNAIT.
Under the terms of the J&J Collaboration Agreement, J&J made an upfront payment of $0.5 million.
−Removed: The Company is also eligible to receive additional payments of up to an aggregate of $3.7 million, based upon certain triggers relating to the FNAIT studies and the Company’s activities under the agreement.
+Added: We were also eligible to receive additional payments upon certain triggers related to both companies' FNAIT studies, however, in connection with our decision in April 2025 to discontinue development of RLYB212, we do not expect any payments regarding the achievement of certain enrollment-related events.
The J&J Collaboration Agreement expires in April 2026.
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We currently employ internal resources to manage our CMOs.
−Removed: We believe that RLYB212, RLYB116, REV102 and RLYB332 can be manufactured through reliable and reproducible biologic and chemical processes from readily available starting materials.
+Added: We believe that RLYB116 and RLYB332 can be manufactured through reliable and reproducible biologic and chemical processes from readily available starting materials.
We believe that our manufacturing processes are amenable to scale-up and will not require unusual or expensive equipment.
We expect to continue to develop, on our own or with our collaborators, product candidates that can be produced cost-effectively at contract manufacturing facilities.
−Removed: We expect to rely on third parties for the manufacture of any in vitro diagnostic device, companion diagnostics or companion drug delivery systems we develop.
−Removed: For example, we have engaged a third-party to assist in developing laboratory screening tests and in our evaluation of potential companion diagnostics in conjunction with our development of RLYB212.
−Removed: Depending on the regulatory pathway and technology solutions we choose, we may engage third parties to continue the development and manufacturing of any device developed to support our therapeutic products.
+Added: T a b le of Contents
Government Regulation
28 unchanged sentences
In such a case, the IND sponsor and the
+Added: T a b le of Contents
FDA must resolve any outstanding concerns before the clinical trial can begin.
23 unchanged sentences
Similarly, an IRB can suspend or terminate approval of a clinical trial at its institution if the clinical trial is not being conducted in accordance with the clinical protocol, GCP or other IRB requirements or if the drug has been associated with unexpected serious harm to patients.
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During the development of a new drug or biological product, sponsors have the opportunity to meet with the FDA at certain points, including prior to submission of an IND, at the end of phase 2 and before submission of an NDA or BLA.
25 unchanged sentences
If the FDA concludes a REMS is needed, the sponsor of the NDA or BLA must submit a proposed REMS and the FDA will not approve the NDA or BLA without a REMS.
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Under the Pediatric Research Equity Act of 2003 ("PREA"), an NDA or BLA or certain supplements thereto must contain data that are adequate to assess the safety and effectiveness of the product for the claimed indications in all relevant pediatric subpopulations, and to support dosing and administration for each pediatric subpopulation for which the product is safe and effective, unless this requirement is waived, deferred or inapplicable.
27 unchanged sentences
Approval may require the
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performance of certain clinical studies, such as clinical usability or human factors studies to demonstrate the safety and/or effectiveness of the device component of the combination product.
21 unchanged sentences
Following approval of a new product, the manufacturer and the approved product are subject to pervasive and continuing regulation by the FDA, governing, among other things, monitoring and recordkeeping activities,
+Added: T a b le of Contents
reporting of adverse experiences with the product and product problems to the FDA, product sampling and distribution, manufacturing and promotion and advertising.
1 unchanged sentence
The FDA and other agencies actively enforce the laws and regulations prohibiting the promotion of off-label uses, and a company that is found to have improperly promoted off-label uses may be subject to significant liability.
+Added: The current presidential administration announced in September 2025 that it intends to prioritize enforcement of pharmaceutical advertising requirements.
If there are any modifications to the product, including changes in indications, labeling or manufacturing processes or facilities, the applicant may be required to submit and obtain FDA approval of a new NDA/BLA or an NDA/BLA supplement, which may require the applicant to develop additional data or conduct additional preclinical studies and clinical trials.
19 unchanged sentences
• fines, warning letters, untitled letters or other enforcement-related letters or clinical holds on post-approval clinical trials;
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• refusal of the FDA to approve pending NDAs/BLAs or supplements to approved NDAs/BLAs, or suspension or revocation of product approvals;
7 unchanged sentences
Depending upon the timing, duration and specifics of FDA approval for our product candidates, some of our U.S.
−Removed: patents may be eligible for limited patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984, commonly referred to as the Hatch-Waxman Amendments.
+Added: patents may be eligible for limited PTE under the Drug Price Competition and Patent Term Restoration Act of 1984, commonly referred to as the Hatch-Waxman Amendments.
The Hatch Waxman Amendments permit restoration of the patent term up to five years as compensation for patent term lost during the FDA regulatory review process.
2 unchanged sentences
Only one patent applicable to an approved drug is eligible for the extension, and the application for the extension must be submitted prior to the expiration of the patent.
−Removed: The USPTO, in consultation with the FDA, reviews and approves the application for any patent term extension or restoration.
+Added: The USPTO, in consultation with the FDA, reviews and approves the application for any PTE or restoration.
Regulatory exclusivity provisions under the FDCA also can delay the submission or the approval of certain applications.
10 unchanged sentences
Biosimilars and Reference Product Exclusivity for Biological Products
−Removed: In March 2010, the Patient Protection and Affordable Care Act was enacted in the United States and included the Biologics Price Competition and Innovation Act of 2009 (the "BPCIA").
+Added: In March 2010, the Patient Protection and Affordable Care Act ("ACA") was enacted in the United States and included the Biologics Price Competition and Innovation Act of 2009 (the "BPCIA").
The BPCIA amended the PHSA to create an abbreviated approval pathway for biological products that are biosimilar to or interchangeable with an FDA-licensed reference biological product.
2 unchanged sentences
The FDA has also issued several guidance documents outlining its approach to reviewing and approving biosimilars and interchangeable biosimilars.
−Removed: Under the BPCIA, a manufacturer may submit an application that is “biosimilar to” or “interchangeable with” a previously approved biological product or “reference product.” In order for the FDA to approve a biosimilar product, it must find that there are no clinically meaningful differences between the reference product and proposed biosimilar product in terms of safety, purity and potency.
−Removed: For the FDA to approve a biosimilar product
−Removed: as interchangeable with a reference product, the agency must find that the biosimilar product can be expected to produce the same clinical results as the reference product and (for products administered multiple times) that the biologic and the reference biologic may be switched after one has been previously administered without increasing safety risks or risks of diminished efficacy relative to exclusive use of the reference biologic.
+Added: Under the BPCIA, a manufacturer may submit an application that is “biosimilar to” or “interchangeable with” a previously approved biological product or “reference product.” In order for the FDA to approve a biosimilar
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+Added: product, it must find that there are no clinically meaningful differences between the reference product and proposed biosimilar product in terms of safety, purity and potency.
+Added: For the FDA to approve a biosimilar product as interchangeable with a reference product, the agency must find that the biosimilar product can be expected to produce the same clinical results as the reference product and (for products administered multiple times) that the biologic and the reference biologic may be switched after one has been previously administered without increasing safety risks or risks of diminished efficacy relative to exclusive use of the reference biologic.
Upon licensure by the FDA, an interchangeable biosimilar may be substituted for the reference product without the intervention of the health care provider who prescribed the reference product.
7 unchanged sentences
As a result, the ultimate impact, implementation and meaning of the BPCIA is subject to significant uncertainty.
−Removed: Orphan Drug Designation and Exclusivity
−Removed: Orphan drug designation in the United States is designed to encourage sponsors to develop products intended for the treatment of rare diseases or conditions.
−Removed: In the United States, a rare disease or condition is statutorily defined as a condition that affects fewer than 200,000 individuals in the United States or that affects more than 200,000 individuals in the United States and for which there is no reasonable expectation that the cost of developing and making the product available for the disease or condition will be recovered from sales of the product in the United States.
−Removed: Orphan drug designation qualifies a company for certain tax credits.
−Removed: In addition, if a product candidate that has orphan drug designation subsequently receives the first FDA approval for that drug for the disease for which it has such designation, the product is entitled to orphan drug exclusivity, which means that the FDA may not approve any other applications to market the same drug for the same indication for seven years following product approval unless the subsequent product candidate is demonstrated to be clinically superior.
−Removed: Absent a showing of clinical superiority, the FDA cannot approve the same product made by another manufacturer for the same indication during the market exclusivity period unless it has the consent of the sponsor or the sponsor is unable to provide sufficient quantities.
−Removed: A sponsor may request orphan drug designation of a previously unapproved product or new orphan indication for an already marketed product.
−Removed: More than one sponsor may receive orphan drug designation for the same product for the same rare disease or condition, but each sponsor seeking orphan drug designation must file a complete orphan disease designation application.
−Removed: To qualify for orphan exclusivity, however, the drug must be clinically superior to the previously approved product that is the same drug for the same condition.
−Removed: If a product designated as an orphan drug ultimately receives marketing approval for an indication broader than what was designated in its orphan drug application, it may not be entitled to exclusivity.
−Removed: RLYB212 has been granted orphan drug designation by the FDA for the prevention of FNAIT.
−Removed: Rare Pediatric Disease Designation and Priority Review Vouchers
−Removed: In 2012, Congress enacted the Food and Drug Administration Safety and Innovation Act requiring the FDA to award priority review vouchers to sponsors of certain rare pediatric disease product applications.
−Removed: This program is designed to encourage development of new drug and biological products for prevention and treatment of “rare pediatric diseases” by, upon initial approval of an application meeting certain specified criteria, providing companies with a voucher that can be redeemed to receive a priority review of a subsequent marketing
−Removed: application for a different product.
−Removed: The sponsor of a rare pediatric disease drug product receiving a priority review voucher may sell or otherwise transfer the voucher to another company.
−Removed: The voucher may be further transferred any number of times before the voucher is used, as long as the sponsor making the transfer has not yet submitted an application relying on the priority review voucher.
−Removed: The FDA may also revoke any rare pediatric disease priority review voucher if the rare pediatric disease product for which the voucher was awarded is not marketed in the United States within one year following the date of approval.
−Removed: In order to receive a rare pediatric disease priority review voucher upon BLA or NDA approval, the product must receive designation from the FDA as a drug for a rare pediatric disease prior to submission of the marketing application.
−Removed: A “rare pediatric disease” is a disease that is serious or life-threatening, in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years and affects fewer than 200,000 people in the United States, or affects more than 200,000 people in the United States but there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from sales in the United States of such drug.
−Removed: In addition to receiving rare pediatric disease designation, in order to receive a rare pediatric disease priority review voucher, the NDA or BLA must be given priority review, rely on clinical data derived from studies examining a pediatric population and dosages of the drug intended for that population, not seek approval for a different adult indication in the original rare pediatric disease product application and be for a drug that does not include a previously approved active ingredient.
−Removed: In addition, under current statutory sunset provisions, even if a marketing application meets all of these requirements, FDA may only award a voucher prior to September 30, 2026 and only if the approved product received rare pediatric disease drug product designation prior to December 20, 2024.
−Removed: RLYB211 and RLYB212 have each been granted rare pediatric disease designation by the FDA.
−Removed: FDA Approval or Clearance of Companion Diagnostics
−Removed: Under the FDCA, in vitro diagnostics, including companion diagnostics, are regulated as medical devices.
−Removed: In the United States, the FDCA and its implementing regulations, and other federal and state statutes and regulations govern, among other things, medical device design and development, preclinical and clinical testing, premarket clearance or approval, registration and listing, manufacturing, labeling, storage, advertising and promotion, sales and distribution, export and import, and post-market surveillance.
−Removed: Unless an exemption applies, diagnostic tests require marketing clearance via 510(k) notification or approval via Premarket Approval (“PMA”) application from the FDA prior to commercial distribution.
−Removed: For novel drugs and biologics, a companion diagnostic device and its corresponding therapeutic must be approved or cleared contemporaneously by the FDA for the use indicated in the therapeutic product’s labeling.
−Removed: The FDA previously has required in vitro companion diagnostics intended to select the patients who will respond to a product candidate to obtain PMA simultaneously with approval of the therapeutic product candidate.
−Removed: The PMA process, including the gathering of clinical and preclinical data and the submission to and review by the FDA, can take several years or longer.
−Removed: It involves a rigorous premarket review during which the applicant must prepare and provide the FDA with reasonable assurance of the device’s safety and effectiveness and information about the device and its components regarding, among other things, device design, manufacturing, and labeling.
−Removed: PMA applications are subject to an application fee, which for fiscal year 2025 is approximately $540,000.
−Removed: A clinical trial is typically required for a PMA application and, in some cases, the FDA may require a clinical study in support of a 510(k) submission.
−Removed: A manufacturer that wishes to conduct a clinical study involving the device is subject to the FDA’s investigational device exemption ("IDE") regulation.
−Removed: The IDE regulations distinguish between significant and non-significant risk device studies and the procedures for obtaining approval to begin the study differ accordingly.
−Removed: Also, some types of studies are exempt from the IDE regulations.
−Removed: A significant risk device presents a potential for serious risk to the health, safety, or welfare of a subject.
−Removed: Significant risk devices are devices that are substantially important in diagnosing, curing, mitigating, or treating disease or in preventing impairment to human health.
−Removed: Studies of devices that pose a significant risk require both FDA and an IRB approval prior to initiation of a clinical study.
−Removed: Many companion diagnostics are considered significant risk devices due to their role in diagnosing a disease or condition.
−Removed: Non-significant risk devices are devices that do not pose a significant risk to the human subjects.
−Removed: A non-significant risk device study requires only IRB approval prior to initiation of a clinical study.
−Removed: After a device is placed on the market, it remains subject to significant regulatory requirements.
−Removed: Medical devices may be marketed only for the uses and indications for which they are cleared or approved.
−Removed: manufacturers must also maintain establishment registration and device listings with the FDA.
−Removed: In addition, device manufacturers are subject to FDA’s medical device reporting regulations, which require that a manufacturer report certain adverse events and device malfunctions to the FDA, and FDA’s correction and removal reporting regulations, which require that manufacturers report to the FDA corrections or removals if undertaken to reduce a risk to health posed by the device or to remedy a violation of the FDCA that may present a risk to health.
−Removed: A medical device manufacturer’s manufacturing processes and those of its suppliers are required to comply with the applicable portions of the QSR, which covers the methods and documentation of the design, testing, production, processes, controls, quality assurance, labeling, packaging, and shipping of medical devices.
−Removed: Device manufacturers are also subject to inspection by the FDA.
Regulation Outside of the United States
3 unchanged sentences
From January 1, 2025, in accordance with the “Windsor Framework,” all new medicines for the UK market, including Northern Ireland, will be authorized by the MHRA and UK packaging must carry a clearly legible ‘UK only’ to be allowed onto the UK market.
−Removed: The International Recognition Procedure provides the framework for the MHRA to take into account assessments of medicinal products conducted by trusted regulatory authorities in Australia, Canada, Switzerland, Singapore, Japan, the United States and the EU when assessing applications for marketing authorization.
+Added: The International Recognition Procedure ("IRP") provides the framework for the MHRA to take into account assessments of medicinal products conducted by trusted regulatory authorities in Australia, Canada, Switzerland, Singapore, Japan, the United States and the EU when assessing applications for marketing authorization.
With the exception of the EU EEA applying harmonized regulatory rules for medicinal products, the approval process and requirements governing the conduct of clinical trials, product licensing, and pricing and reimbursement vary greatly between countries and jurisdictions and can involve additional testing and additional administrative review periods.
6 unchanged sentences
In the EU, clinical trials are primarily regulated by Regulation (EU) No 536/2014 (the “CTR”).
−Removed: The CTR requires sponsors to submit one clinical trial authorization (“CTA”) application via the Clinical Trials Information System, which will then be reviewed by the competent regulatory agency selected by the sponsor to lead the validation and evaluation of the application.
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+Added: The CTR requires sponsors to submit one clinical trial authorization (“CTA”) application via the Clinical Trials Information System, which will then be reviewed by the competent regulatory agency selected by the sponsor as the reporting Member State to lead the validation and evaluation of the application.
If successful, the resulting CTA would cover all EU Member States concerned by the application.
6 unchanged sentences
In particular, to the extent the device component has already been conformity assessed and a European Conformity (“CE”) mark affixed, the certificate of conformity issued by the Notified Body must be provided in the MA application dossier.
−Removed: Where the medical device component has not already been CE marked, the MA application dossier must include an opinion issued by a notified body on the conformity of the device
−Removed: component against the relevant general safety and performance requirements set out in Annex I of Regulation (EU) 2017/745.
−Removed: EU regulatory regime contemplates that MAs can be granted either centrally or nationally, albeit through mutual recognition or decentralized procedure.
+Added: Where the medical device component has not already been CE marked, the MA application dossier must include an opinion issued by a notified body on the conformity of the device component against the relevant general safety and performance requirements set out in Annex I of Regulation (EU) 2017/745.
+Added: EU regulatory regime contemplates that MAs can be granted either centrally or nationally, albeit through mutual recognition or decentralized procedure, depending on the type of product and the therapeutic indications for which approval is being sought.
Centralized Procedure
20 unchanged sentences
Under the decentralized procedure, an applicant submits an application, accompanied by a dossier, containing the requisite scientific data, and related materials to the reference member state and concerned member state(s).
−Removed: The reference member state prepares a draft assessment and drafts of the related materials within 120 days of the receipt of a valid application.
+Added: The reference member state prepares a draft assessment and
+Added: T a b le of Contents
+Added: drafts of the related materials within 120 days of the receipt of a valid application.
Within 90 days of receiving the reference member state’s positive assessment report, each concerned member state must approve the assessment report and related materials, unless they identify a potential serious risk to public health.
7 unchanged sentences
Such conditional approvals may be granted for product candidates (including medicines designated as orphan medicinal products) if (1) the product candidate is intended for the treatment, prevention or medical diagnosis of seriously debilitating or life-threatening diseases;
−Removed: (2) the drug candidate is intended to meet unmet medical
−Removed: needs of patients;
+Added: (2) the drug candidate is intended to meet unmet medical needs of patients;
(3) a MA may be granted prior to submission of comprehensive clinical data provided that the benefit of the immediate availability on the market of the medicinal product concerned outweighs the risk inherent in the fact that additional data are still required;
1 unchanged sentence
A conditional MA requires specific obligations to be fulfilled by the MA holder, including obligations with respect to the completion of ongoing or new studies and with respect to the collection of pharmacovigilance data.
−Removed: Conditional MAs are valid for one year, and can be renewed annually, if the risk-benefit balance remains positive, and after a satisfactory assessment of benefit:
+Added: Conditional MAs are valid for one year, and can be renewed annually, if the risk-benefit balance remains positive, and after a satisfactory re-assessment of benefit:
risk balance and progress in fulfilling the specific obligations.
+Added: Failure to submit the required comprehensive data may result in regulatory action against the conditional marketing authorization, including the possibility of its revocation.
The timelines for the centralized procedure described above also apply with respect to the review by the CHMP of applications for a conditional MA.
9 unchanged sentences
European Union Regulatory Data Exclusivity
−Removed: In the EU, new products containing a new active substance (so called “reference medicinal products”) qualify for eight years of data exclusivity and an additional two years of marketing exclusivity upon grants of a MA.
+Added: In the EU, new products containing a new active substance are considered as “reference medicinal products,” and accordingly qualify for eight years of data exclusivity and an additional two years of marketing exclusivity upon granting of a MA.
The data exclusivity period prevents generic or biosimilar applicants from relying on the preclinical and clinical trial data contained in the dossier of the reference medicinal product when applying for a generic or biosimilar MA in the EU.
The marketing exclusivity period prevents a successful generic or biosimilar MA applicant from commercializing its product in the EU until ten years have elapsed from the initial authorization of the reference medicinal product in the EU.
−Removed: The ten-year market exclusivity period can be extended to a maximum of eleven years if, during the first eight years of those ten years, the MA holder obtains an authorization for one or more new therapeutic indications which, during the scientific evaluation prior to their authorization, are held to bring a significant clinical benefit in comparison with existing therapies.
+Added: The ten-year market exclusivity period can be extended to a maximum of eleven years if, during the first eight years of those ten years, the MA holder obtains
+Added: T a b le of Contents
+Added: an authorization for one or more new therapeutic indications which, during the scientific evaluation prior to their authorization, are held to bring a significant clinical benefit in comparison with existing therapies.
European Union Orphan Designation and Exclusivity
1 unchanged sentence
Under Article 3 of Regulation (EC) 141/2000, a medicinal product may be designated as orphan if it is intended for the diagnosis, prevention or treatment of a life- threatening or chronically debilitating condition, which either (a) affects not more than five in 10,000 persons in the EU when the application is made, or (b) , without the benefits derived from orphan status, would not generate sufficient return in the EU to justify investment.
−Removed: In each case, there can exist no satisfactory method of diagnosis, prevention or treatment of such condition authorized in the EU, or if such a method exists, the product will be of significant benefit to those affected by the condition.
+Added: In each case, there exists no satisfactory method of diagnosis, prevention or treatment of such condition authorized in the EU, or if such a method exists, the product will be of significant benefit to those affected by the condition.
The term ‘significant benefit’ is defined in Regulation (EC) 847/2000 to mean a clinically relevant advantage or a major contribution to patient care.
11 unchanged sentences
An equivalent regime is reflected in domestic law in the UK.
−Removed: Under the UK regime, however, orphan designations are not granted and instead a decision is made at the point of marketing authorization grant.
+Added: Under the UK regime, however, orphan designations are not granted and instead a decision is made at the point of MA grant.
RLYB212 has been granted orphan drug designation by the EMA for the prevention of FNAIT.
7 unchanged sentences
To this end, the MA holder must provide the EMA or the competent authority with a consolidated version of the file in respect of the medicinal product’s quality, safety and efficacy, including all variations introduced since the MA was granted, at least nine months before the MA ceases to be valid.
−Removed: Once renewed, the MA is generally valid for an unlimited period, unless the European Commission or the competent authority decides, on justified grounds relating to pharmacovigilance, to proceed with one additional five-year renewal.
−Removed: In accordance with the sunset clause, any authorization which is not followed by the actual placing of the drug on the EU market (in case of centralized procedure) or on the market of the authorizing member state (in the case of the national, decentralized and mutual recognition procedures) within three years after the MA is granted will cease to be valid.
+Added: Once renewed, the MA is generally valid for an unlimited period, unless the EC or the competent authority decides, on justified grounds relating to pharmacovigilance, to proceed with one additional five-year renewal.
+Added: In accordance with the sunset clause, any authorization which is not followed by the actual placing of the drug on the EU market (in case of centralized procedure) or on the market of the authorizing member state (in the
+Added: T a b le of Contents
+Added: case of the national, decentralized and mutual recognition procedures) within three years after the MA is granted will cease to be valid.
+Added: The EU is currently revising its general pharmaceutical legislation, known as the ‘Pharma Package’.
+Added: This initiative is designed to ensure fair access to safe, effective, and affordable medicines throughout the EU.
+Added: It also aims to boost the competitiveness of the pharmaceutical industry by reducing regulatory burdens and enhancing supply chain security to better prevent and manage shortages.
+Added: The legislative process began with the European Commission’s proposal in April 2023, followed by the development of positions by the European Parliament and the Council.
+Added: Trilogue negotiations concluded with a political agreement in December 2025.
+Added: The agreed text now awaits formal adoption by both the Parliament and Council, after which it will be published in the Official Journal of the EU.
+Added: Key measures include a new exclusivity framework, enhanced incentives for orphan drugs and antibiotics, an expanded Bolar exemption, and a shortened regulatory assessment timeframe.
+Added: The reforms also introduce stricter controls on product availability and supply shortages.
+Added: It is anticipated that the new EU pharmaceutical legislation will be fully applicable in 2028, following a two-year transition period.
+Added: Additionally, in December 2025, the EC proposed the Biotech Act.
+Added: This legislation is intended to strengthen the competitiveness of the biotechnology sector and facilitate the development and timely market entry of biotechnology innovations, while maintaining high standards for the protection of human health.
+Added: The Biotech Act introduces a 12-month extension to the Supplementary Protection Certificate ("SPC") for advanced therapy medicinal products such as gene and cell therapies, and innovative biotech medicines.
+Added: This extension aims to incentivize EU-based manufacturing and R&D by rewarding novel, highly effective medicines that conduct clinical trials across multiple EU Member States, addressing the time lost in regulatory approval.
Brexit and the Regulatory Framework in the United Kingdom
6 unchanged sentences
However, the TCA does not foresee wholesale mutual recognition of UK and EU pharmaceutical regulations.
+Added: To be used or sold in the UK, a medicinal product must have a valid MA granted through the national application process.
+Added: National applications are governed by the Human Medicines Regulations (SI 2012/1916).
+Added: Applications are made electronically through the MHRA Submissions Portal.
+Added: The MHRA operates fixed submission and assessment timetables for innovative medicines applications to facilitate consultation with its statutory advisory committee, the Commission on Human Medicines (“CHM”).
+Added: The MHRA assessment procedure for a MA application involves an initial evaluation, including orphan designation if applicable, and consultation with expert advisory groups as needed.
+Added: By Day 90, applicants receive a consolidated request for information (“RFI”), which pauses the review clock until a complete response is submitted electronically.
+Added: Responses are assessed by Day 150, with further RFIs issued for minor issues or a CHM letter for major objections.
+Added: Each subsequent RFI requires a complete response within three months, and the clock is restarted upon submission.
+Added: Applicants may make written or oral representations to the CHM if major objections remain.
+Added: Final compliance checks are conducted once all issues are resolved, and the MHRA issues a grant or refusal letter specifying any conditions and the MA expiry date.
+Added: The entire assessment process is designed to be completed within 210 calendar days, excluding any procedural clock-stops for additional information or representations.
+Added: The innovative medicines timetable allows for a positive decision within 150 clock-on days if all issues are resolved following one round of questions.
+Added: Where there are outstanding issues at Day 150, we will come to a final decision as soon as possible and within 210 clock-on days.
+Added: The innovative medicines timetable allows for a positive decision within 150 clock-on days if all issues are resolved following one round of questions.
+Added: Where there are outstanding issues at Day 150, we will come to a final decision as soon as possible and within 210 clock-on days.
+Added: T a b le of Contents
+Added: In addition, the MHRA 150-day accelerated review is a specialized, fast-track national MA procedure designed for innovative medicines, new active substances, and biosimilars, aiming for a decision in 150 "clock-on" days rather than the standard 210.
+Added: It requires high-quality applications, typically involving one round of questions and a 60-day cool-down period for responses.
+Added: On August 30, 2023, the MHRA published detailed guidance on its recently announced new IRP for MAAs.
+Added: The IRP applies from January 1, 2024, and replaces existing EU decision reliance procedure.
+Added: to apply for authorizations from seven international regulators (e.g.
+Added: Health Canada, Swiss Medic, FDA, EMA, among others).
+Added: The IRP allows the MHRA to take into account the assessment and decision-making of the ‘Reference Regulators” to perform a targeted assessment of IRP applications but retain the authority to reject applications if the evidence provided is considered insufficiently robust.
+Added: There exist two recognition timetables for new IRP MAAs:
+Added: IRP Route A (60 days) and IRP Route B (110 days), both starting from validation.
+Added: Eligibility is determined via an applicant-completed form six weeks before submission.
+Added: Recognition A applies to applications with Reference Regulator approval within the past two years, with no clock stop, but may revert to Recognition B if major objections arise.
+Added: IRP Route B covers Reference Regulator approvals within the past ten years (or exceptionally older) and applies if specific criteria, such as conditional approvals, manufacturing changes, or UK-specific requirements, are met.
+Added: IRP Route B allows for consultation with the CHM and aligns with CHM dates for new active substances.
+Added: Applications not eligible for either timetable may be submitted as full national applications if MHRA requirements are met.
+Added: IRP can be used for post-authorization measures including line extensions, variations, and renewal application.
Rest of the World Regulation
18 unchanged sentences
Some countries provide that drug products may be marketed only after a reimbursement price has been agreed.
−Removed: Some countries may require the completion of additional studies that compare the cost-effectiveness of our product candidate to currently available therapies (so called health technology assessment ("HTA") in order to obtain reimbursement or pricing approval).
+Added: Some countries may require the completion of additional studies that compare the cost-effectiveness of our product candidate to currently available therapies (so called health technology assessment ("HTA") in order to obtain reimbursement
+Added: T a b le of Contents
+Added: or pricing approval).
For example, subject to the requirements set out in Directive 89/105/EEC relating to the transparency of measures regulating the pricing of medicinal products for human use and their inclusion in the scope of national health insurance systems, EU Member States have the legal competence to set national measures of an economic nature on the marketing of medicinal products in order to control public health expenditure on such products.
13 unchanged sentences
From January 13, 2030, all new medicines will come under the scope of the HTA Regulation.
−Removed: The HTA Regulation established the Coordination Group on
−Removed: HTA (the “HTACG”) consisting of representatives of EU member states, mainly from HTA authorities or bodies.
+Added: The HTA Regulation established the Coordination Group on HTA (the “HTACG”) consisting of representatives of EU member states, mainly from HTA authorities or bodies.
The HTACG’s remit is to coordinate and adopt the joint HTA work carried out by its subgroups within the scope of the HTA Regulation and to adopt methodological and procedural guidance documents for joint work, including JCAs.
6 unchanged sentences
Health Care Laws and Regulations
−Removed: In the United States, biopharmaceutical manufacturers and their products are subject to extensive regulation at the federal and state level, such as laws intended to prevent fraud and abuse in the healthcare industry.
+Added: In the United States, biopharmaceutical manufacturers and their products are subject to extensive regulation at the federal and state level, such as laws intended to prevent fraud and abuse in the healthcare industry, which may constrain their business operations.
These laws, some of which will apply only if and when we have an approved product, include:
2 unchanged sentences
• the federal Health Insurance Portability and Accountability Act of 1996 ("HIPAA") which, in addition to privacy protections applicable to healthcare providers and other entities, prohibits executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters;
+Added: T a b le of Contents
• FDCA, which among other things, strictly regulates drug marketing, prohibits manufacturers from marketing such products prior to approval or for off-label use and regulates the distribution of samples;
6 unchanged sentences
state laws requiring pharmaceutical companies to comply with specific compliance standards, restrict financial interactions between pharmaceutical companies and healthcare providers or report information related to payments to health care providers, marketing expenditures or drug prices;
+Added: state or local laws requiring the registration of pharmaceutical sales representatives;
state laws regulating the manufacture and distribution of biopharmaceutical products;
10 unchanged sentences
In particular, government authorities and other third-party payors have attempted to control costs by limiting coverage and the amount of reimbursement for particular medical products and services.
−Removed: Health care reform, specifically reform addressing pricing and payment for drugs, has been an ongoing focus and is likely to continue under the Trump Administration.
+Added: Health care reform, specifically reform addressing pricing and payment for drugs, has been an ongoing focus and is likely to continue under the current presidential administration.
A number of healthcare reforms involving drugs have been successfully implemented, including reforms related to Medicare payment for drugs and manufacturer rebate obligations under the Medicaid Drug Rebate Program.
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We cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative or executive action, either in the United States or abroad.
−Removed: We expect that additional federal and state health care reform measures will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for health care products and services.
+Added: We expect that additional federal
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+Added: and state health care reform measures will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for health care products and services.
For a more detailed discussion of health care reform in the U.S., see “Risk Factors—Risks Related to Healthcare Laws and Other Legal Compliance Matters.”
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There are numerous U.S.
−Removed: federal and state laws and regulations related to the privacy and security of personal information, including laws requiring the safeguarding of personal information and laws requiring notification to governmental authorities and data subjects as well as remediation in the event of a data breach.
+Added: federal and state laws and regulations related to the privacy and security of personal information, including laws requiring the safeguarding of personal information, laws requiring notification to governmental authorities and data subjects as well as remediation in the event of a data breach, and laws that afford individuals numerous rights with respect to their personal information.
There have been several developments in recent years with respect to U.S.
state data privacy laws.
−Removed: For example, the California Consumer Privacy Act of 2018, as amended and supplemented by the California Privacy Rights Act (collectively, “CCPA”) imposed many requirements for the collection, processing, and sharing of personal information of California residents.
+Added: For example, the California Consumer Privacy Act of 2018, as amended by the California Privacy Rights Act (collectively, “CCPA”) imposes many requirements for the collection, processing, and sharing of personal information of California residents.
The CCPA contains significant penalties for companies that violate its requirements and provides California residents a private right of action, including the ability to seek statutory damages, in the event of a breach involving their personal information.
−Removed: Similar omnibus privacy laws have been proposed or passed in more than half of the states in the United States and in the U.S.
−Removed: Congress, reflecting a trend toward more stringent privacy legislation in the United States.
−Removed: In addition, Washington state enacted the My Health, My Data Act, a health-focused consumer privacy law, which took effect in March 2024.
−Removed: This law imposes obligations related to the collection and sharing of certain health-related information that is not subject to HIPAA and that does not fall within certain other exceptions in the law.
−Removed: Other states have enacted, or are in the process of enacting, similar health-focused consumer privacy laws.
+Added: Several states have proposed or passed comprehensive privacy laws, including several laws imposing obligations similar to those of the CCPA.
+Added: In addition, several states have proposed or enacted health-focused consumer privacy laws, such as Washington state's My Health, My Data Act, which took effect in March 2024, and imposes obligations related to the collection and sharing of certain health-related information that is not subject to HIPAA and that does not fall within certain other exceptions in the law.
Also of note, in June 2024, the Protecting Americans’ Data from Foreign Adversaries Act of 2024 took effect.
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Additionally, in January 2025, the U.S.
−Removed: Department of Justice published a final rule implementing President Biden’s Executive Order 14117, “Preventing Access to Americans’ Bulk Sensitive Personal Data and United States Government-Related Data by Countries of Concern.” This final rule prohibits certain data brokerage transactions and transactions involving certain bulk human ‘omic data, including human genomic data and
−Removed: biospecimens from which such data can be derived, with restricted persons and jurisdictions, such as the PRC.
+Added: Department of Justice published a final rule implementing President Biden’s Executive Order 14117, “Preventing Access to Americans’ Bulk Sensitive Personal Data and United States Government-Related Data by Countries of Concern,” which became effective in April 2025.
+Added: This final rule prohibits certain data brokerage transactions and transactions involving certain bulk human ‘omic data and biospecimens from which such data can be derived with restricted persons and jurisdictions, such as the PRC, and “covered persons” that have certain ties to such restricted jurisdictions.
The final rule also places restrictions on certain vendor, employment and investment agreements with such jurisdictions.
−Removed: Most provisions of the final rule are scheduled to take effect in April 2025.
−Removed: These restrictions may affect our ability to engage in collaborations or license agreements with entities in restricted countries or with a nexus to such countries going forward.
−Removed: Compliance with evolving U.S.
−Removed: privacy and security laws, requirements and regulations may result in cost increases due to necessary systems changes, new limitations or constraints on our business models and the development of new administrative processes.
General Data Protection Regulation
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and includes parallel obligations to those set forth by GDPR.
−Removed: These laws and similar privacy laws enacted by other countries may further increase our costs of compliance and expose us to greater legal risk.
Employees and Human Capital Resources
−Removed: Our employees are driven by our mission to identify and accelerate the development of transformative therapies for patients with rare disorders.
−Removed: We believe that our deep commitment to high ethical and professional standards is fundamental to our mission, and we are determined to build a culture that empowers a skilled and experienced workforce to perform at the highest levels.
−Removed: We commit our resources and make investments, including through recruiting, training and collaboration, to promote the culture that we desire, and we expect our employees to embrace the Company’s values and culture in all that we do.
As of December 31, 2025, we employed 14 full-time employees.
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We consider our relationship with our employees to be good.
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Corporate Information
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On June 30, 2021, Rallybio IPD, LLC was converted into a Delaware corporation and changed its name to Rallybio Corporation.
−Removed: Our principal executive offices are located at 234 Church Street, Suite 1020, New Haven, CT 06510 and our telephone number is (203) 859-3820.
+Added: Our principal executive offices are located at 234 Church Street, New Haven, CT 06510 and our telephone number is (203) 859-3820.
Our corporate website address is https://www.rallybio.com.
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Our Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form 8-K, including exhibits, proxy and information statements and amendments to those reports filed or furnished pursuant to Sections 13(a), 14, and 15(d) of the Securities Exchange Act of 1934, as amended (the "Exchange Act”) are available through the “Investors” portion of our website free of charge as soon as reasonably practicable after we electronically file such material with, or furnish it to, the Securities and Exchange Commission ("SEC").
−Removed: In addition, our filings with the SEC may be accessed through the SEC’s
−Removed: Interactive Data Electronic Applications system at https://www.sec.gov.
+Added: In addition, our filings with the SEC may be accessed through the SEC’s Interactive Data Electronic Applications system at https://www.sec.gov.
All statements made in any of our securities filings, including all forward-looking statements or information, are made as of the date of the document in which the statement is included, and we do not assume or undertake any obligation to update any of those statements or documents unless we are required to do so by law.
Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.