We are a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta, or TGF-ß, family of proteins.
−Removed: We are a leader in understanding the role of the TGF-ß family of proteins, which are master regulators of the growth, repair and maintenance of a number of tissues, including blood, bone, skeletal muscle, adipose and heart tissue.
+Added: We are a leader in understanding the role of the TGF-ß family of proteins, which are master regulators of the growth, repair and maintenance of a number of tissues, including skeletal muscle, bone, adipose, heart tissue and blood.
By leveraging this understanding, we have discovered and are developing protein therapeutics that have the potential to provide meaningful and potentially disease-modifying benefit to patients.
−Removed: One of o ur product candidates, cibotercept (KER-012), is being developed for the treatment of pulmonary arterial hypertension, or PAH, and for the treatment of cardiovascular disorders .
−Removed: Our second product candidate, KER-065, is being developed for the treatment of neuromuscular diseases.
+Added: Our lead product candidate, rinvatercept (KER-065), is being developed for the treatment of neuromuscular diseases.
Our most advanced product candidate, elritercept (KER-050), is being developed for the treatment of low blood cell counts, or cytopenias, including anemia and thrombocytopenia, in patients with myelodysplastic syndromes, or MDS, and in patients with myelofibrosis.
In December 2024, we entered into an exclusive license agreement with Takeda Pharmaceuticals U.S.A., Inc., or Takeda, to further develop, manufacture and commercialize elritercept worldwide outside of mainland China, Hong Kong and Macau, which became effective on January 16, 2025.
−Removed: Cibotercept is designed to bind to and inhibit the signaling of TGF-ß ligands that stimulate the proliferation of vascular endothelial and smooth muscle cells and fibroblasts, including activin A, activin B and myostatin (GDF8).
−Removed: We believe that cibotercept has the potential to increase the signaling of bone morphogenic protein, or BMP, pathways through this inhibition of activin A and activin B signaling, and consequently treat diseases such as PAH that are associated with reduced BMP signaling, including inactivating mutations in the BMP receptors.
−Removed: We are developing cibotercept for the treatment of PAH and for the treatment of cardiovascular disorders.
−Removed: We expect to present topline data from our Phase 2 clinical trial evaluating cibotercept in patients with PAH, which we refer to as the TROPOS trial, in the second quarter of 2025.
−Removed: We announced the early termination of the TROPOS trial in January 2025, based on an ongoing safety review due to the unanticipated observation of pericardial effusion adverse events in the trial.
−Removed: Following completion of the TROPOS trial, we plan to evaluate the appropriate development strategy for cibotercept, including in PAH and other potential indications.
−Removed: KER-065 is designed to bind to and inhibit TGF-ß ligands, including myostatin (GDF8) and activin A, which are negative regulators of muscle and bone mass and strength.
−Removed: Through inhibition of these TGF-ß ligands, we believe that KER-065 has the potential to increase skeletal muscle regeneration, increase muscle size and strength, reduce body fat, reduce fibrosis of the skeletal muscle and increase bone strength.
−Removed: We are developing KER-065 for the treatment of neuromuscular disorders, with an initial focus on Duchenne muscular dystrophy, or DMD.
−Removed: Glucocorticoids, the standard of care in DMD, can have significant side effects when used long-term, including catabolism of muscle, increased fat and accelerated bone loss.
−Removed: We have commenced a Phase 1 clinical trial of KER-065 in a healthy volunteer adult population, and expect to announce initial data from this trial in the first quarter of 2025.
+Added: Rinvatercept is designed to bind to and inhibit TGF-ß ligands, including myostatin (GDF8) and activin A, which are negative regulators of muscle and bone mass and strength.
+Added: Through inhibition of these TGF-ß ligands, we believe that rinvatercept has the potential to increase skeletal muscle regeneration, increase muscle size and strength, reduce body fat, reduce fibrosis of the skeletal muscle and increase bone strength.
+Added: We are developing rinvatercept for the treatment of Duchenne muscular dystrophy, or DMD and for the treatment of amyotrophic lateral sclerosis, or ALS.
+Added: In March 2025, we announced initial topline results from the Phase 1 clinical trial of rinvatercept in healthy adult volunteers.
+Added: We expect to commence a Phase 2 clinical trial of rinvatercept in patients with DMD in the second quarter of 2026.
+Added: We also plan to engage regulators on the design of a Phase 2 clinical trial of rinvatercept in patients with ALS in the second half of 2026.
Elritercept is an engineered ligand trap comprised of a modified ligand-binding domain of the TGF-ß receptor known as activin receptor type IIA, or ActRIIA, that is fused to the portion of the human antibody known as the Fc domain.
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We believe elritercept has the potential to provide benefit to patients suffering from red blood cell and platelet differentiation and maturation defects occurring across the spectrum from early through terminal stages of hematopoiesis, and consequently may be effective for many patients that have limited treatment options or are refractory to available therapies.
−Removed: In December 2024, we announced additional data from our ongoing Phase 2 clinical trial evaluating elritercept for the treatment of anemia and thrombocytopenia in patients with very low-, low-, or intermediate-risk MDS, which we refer to as lower-risk MDS, and initiated our placebo-controlled Phase 3 clinical trial in patients with lower-risk MDS.
−Removed: Additionally, in December 2024, we announced additional data from our ongoing Phase 2 clinical trial evaluating elritercept for the treatment of patients with myelofibrosis-associated cytopenias, which we refer to as the RESTORE trial.
−Removed: Our strategy focuses on the role of members of the TGF-ß family of proteins in the development of a number of tissues, including skeletal muscle, bone, blood, adipose and heart tissue.
+Added: In July 2025, we announced that the first patient was dosed in the placebo-controlled Phase 3 RENEW clinical trial in patients with very low-, low-, or intermediate-risk MDS, which we refer to as lower-risk MDS.
+Added: The dosing of the first patient triggered a $10 million milestone payment to us under the license agreement with Takeda.
+Added: Our strategy focuses on the role of members of the TGF-ß family of proteins in the development of a number of tissues, including skeletal muscle, bone, adipose, heart tissue and blood.
Aged and damaged cells are routinely replaced by new cells in normally functioning organs.
−Removed: These new cells are derived from stem cells that have the ability to differentiate into cells with specialized function when appropriate signals are provided to maintain the homeostatic state of the tissue.
−Removed: Members of the TGF-ß family of proteins, including activins and BMPs, provide the necessary signals for this process of self-renewal and repair.
+Added: These new cells are derived from stem cells that have the ability to differentiate into cells with specialized functions when appropriate signals are provided to maintain the homeostatic state of the tissue.
+Added: Members of the TGF-ß family of proteins, including activins and bone morphogenic proteins (BMPs), provide the necessary signals for this process of self-renewal and repair.
We seek to address the limitations of current therapeutic approaches to treating diseases whose manifestations are linked to dysfunction of TGF-ß signaling pathways by:
• leveraging our comprehensive insights into the TGF-ß signaling pathways to discover therapeutics to treat disorders that are linked to dysfunctional TGF-ß signaling;
−Removed: ▪ expanding our library of proprietary molecules that are engineered to induce desired biological effects, such as increased muscle mass and strength, improved muscle quality and reduced intramuscular fat, improved bone mineral density and modulated blood cell production;
+Added: • expanding our library of proprietary molecules that are engineered to induce desired biological effects, such as increased muscle mass and strength, increased muscle regeneration, improved muscle quality and reduced tissue fat, improved bone mineral density, reduced inflammation, reduced fibrosis and modulated blood cell production;
• engineering proprietary molecules to selectively target specific proteins in the TGF-ß signaling pathways to provide therapeutic benefit while potentially minimizing safety risks;
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• targeting the TGF-ß family of proteins, which are highly conserved throughout evolution, permitting the use of animal models to potentially predict with high confidence the therapeutic benefit in patients.
−Removed: We are led by a highly experienced management team and scientific advisory board who have significant experience and expertise researching and developing therapeutics in the TGF-ß family of proteins.
−Removed: Our team has collectively worked on marketed therapeutics such as Reblozyl, Takhzyro and Winrevair, and led drug discovery and clinical development at companies including Acceleron Pharma Inc.
+Added: We are led by a highly experienced management team and scientific advisory board who have significant experience and expertise researching and developing therapeutics in targeting the TGF-ß family of proteins.
+Added: Our team has collectively worked on marketed therapeutics such as Reblozyl and Winrevair, and led drug discovery at companies including Acceleron Pharma Inc.
(which was acquired by Merck & Co.
−Removed: in November 2021), Dyax Corp, Scholar Rock Holding Corporation, Tourmaline Bio, Inc.
−Removed: and Wyeth Pharmaceuticals Inc.
+Added: in November 2021) and Wyeth Pharmaceuticals Inc.
The following table sets forth our product candidates and their current development stages:
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The key elements of our strategy include:
−Removed: ▪ Rapidly advance the clinical development of cibotercept, KER-065 and elritercept.
−Removed: We expect to present topline data from our Phase 2 clinical trial evaluating cibotercept in patients with PAH in the second quarter of 2025.
−Removed: We also expect to announce initial data from our ongoing Phase 1 clinical trial of KER-065 in a healthy volunteer adult population in the first quarter of 2025.
−Removed: We initiated our Phase 3 clinical trial of elritercept in patients with lower-risk MDS in December 2024.
+Added: • Rapidly advance the clinical development of rinvatercept .
+Added: In March 2025, we announced initial topline results from the Phase 1 clinical trial of rinvatercept in healthy adult volunteers.
+Added: We expect to commence a Phase 2 clinical trial of rinvatercept in patients with DMD in the second quarter of 2026.
+Added: We also plan to engage regulators on the design of a Phase 2 clinical trial of rinvatercept in patients with ALS in the second half of 2026.
• Pursue development and, if approved, commercialization of our product candidates in indications and regions where we believe we can maximize their value independently or through strategic collaborations .
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Our discovery efforts are focused on expanding our pipeline of wholly-owned assets for the treatment of disorders that are linked to dysfunctional TGF-ß signaling .
−Removed: Accordingly, we intend to identify and develop product candidates to treat diseases where targeting the TGF-ß signaling pathways has clinical validation or biological rationale.
+Added: Accordingly, we intend to identify and develop product candidates to treat diseases where targeting the TGF-ß signaling pathways has clinical validation or biological rationale, including neuromuscular and neurodegenerative disorders, rare bone and fibrosis diseases, and obesity and frailty indications.
• Maintain a dynamic, data-driven operating model .
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Our extensive knowledge of our assets and the process of drug development informs our decision-making process regarding when to advance the science and clinical path to pursue demonstrating proof-of-concept, balanced with the imperative of maintaining an efficient timeframe and cost-effective budget.
−Removed: Our Pulmonary and Cardiovascular Franchise
−Removed: Cibotercept is a ligand trap comprised of a modified ligand-binding domain of activin receptor type IIB, or ActRIIB, that is fused to the portion of the human antibody known as the Fc domain.
−Removed: Cibotercept is designed to normalize blood vessel thickness and heart function by binding to and inhibiting the signaling of select TGF-ß ligands, including activin A, activin B and myostatin (GDF8), that stimulate the proliferation of vascular endothelial and smooth muscle cells and fibroblasts, without a dose-limiting increase in red blood cells.
−Removed: We believe that cibotercept has the potential to increase the signaling of BMP pathways through this inhibition of activin A and activin B signaling, and consequently treat diseases such as PAH that are associated with reduced BMP signaling due to inactivating mutations in the BMP receptors.
−Removed: We are developing cibotercept for the treatment of PAH and for the treatment of cardiovascular disorders.
−Removed: Following completion of the TROPOS trial, we plan to evaluate the appropriate development strategy for cibotercept, including in PAH and other potential indications.
−Removed: Pulmonary Arterial Hypertension
−Removed: PAH is a debilitating disorder characterized by elevated pulmonary vascular resistance due to the progressive narrowing and obliteration of precapillary pulmonary arteries.
−Removed: This increase in pulmonary vascular resistance results in severe elevation in pulmonary artery pressure, leading to right ventricular hypertrophy and ultimately, death from right heart failure.
−Removed: Patients with PAH develop shortness of breath, fatigue, fainting, chest pain, palpitations and swelling of extremities and abdomen.
−Removed: We estimate that there are approximately 40,000 addressable patients in the United States living with this condition.
−Removed: Despite current treatment options, survival with PAH remains only slightly above 60% at five years, with mortality typically resulting from right ventricle failure.
−Removed: Loss-of-function mutations in the gene encoding the BMP type II receptor, or BMPR2, are present in over 70% of cases of heritable PAH, or HPAH, while loss-of-function mutations in certain BMPR2 co-receptors are present in other cases of HPAH and idiopathic PAH.
−Removed: Histology and gene expression studies from the lungs of human and experimental PAH showed diminished BMPR2 expression and BMP signaling even in the absence of loss-of-function mutations, as well as enhanced TGF-ß signaling.
−Removed: Consistent with an imbalance in the signaling of these families of ligands, it was recently found that PAH due to cirrhosis and portal hypertension is marked by a severe deficiency of circulating BMP9, while circulating TGF-ß, activin and growth differentiation factor, or GDF, ligands were found to be increased in PAH, even in the absence of causative mutations.
−Removed: Multiple experimental third-party models also demonstrated the efficacy of augmenting BMP signaling or suppressing TGF-ß, activin or GDF signaling, which we believe supports the notion that imbalanced homeostatic BMP and pathogenic TGF-ß, activin and GDF signaling drive the development and progression of pulmonary vascular disease.
−Removed: Limitations of Current Treatment Options for PAH
−Removed: All of the currently-approved therapies for PAH are vasodilators, which are medications that dilate blood vessels.
−Removed: These vasodilators fall into one of three categories:
−Removed: (i) prostanoids, which are agonists of the prostacyclin signaling pathway;
−Removed: (ii) endothelin receptor antagonists, or ERAs;
−Removed: or (iii) therapies that stimulate the nitric oxide-soluble guanylate cyclase-cyclic guanosine monophosphate axis, such as (a) phosphodiesterase 5 inhibitors, or PDE5i, or (b) soluble guanylate cyclase activators, which augment cGMP signaling, a key mediator in pulmonary arterial vasodilation.
−Removed: One common approach to treating early-stage or mild PAH is an oral combination therapy using ERA and PDE5i medications.
−Removed: More severe PAH generally requires the addition of prostanoid, via oral or inhaled administration, while advanced PAH typically requires continuous parenteral administration.
−Removed: Each of these individual therapies may modestly improve a patient’s functional status and in some cases survival, but is limited by systemic hypotension, systemic side effects and tachyphylaxis, which is an acute, sudden decrease in response to a product after its administration.
−Removed: Additionally, combination therapy is limited by the combined side effect profiles.
−Removed: Although existing treatments may modestly slow the progression of PAH, none appear to fully halt or reverse the disease’s progression.
−Removed: The key pathologic features of PAH include an unchecked proliferation of different vascular cells in the pulmonary arterial wall, including smooth muscle cells, endothelial cells and fibroblasts, and an exaggerated perivascular infiltration of inflammatory cells leading to a marked narrowing of small to medium sized pulmonary arteries.
−Removed: However, most currently approved therapies lower pulmonary vascular resistance through vasodilatation and do not fully target the obliterative pulmonary vascular remodeling.
−Removed: Accordingly, we believe there is a significant unmet need for a treatment that primarily targets the proliferative pathological processes and can be used alone or in combination with other PAH therapies.
−Removed: We believe that potent therapies that do not exhibit tachyphylaxis, are orally bioavailable or do not require continuous infusion therapy would have advantages over the currently available treatments for PAH.
−Removed: Therapies that delay or reverse the obliterative pulmonary vascular remodeling could have a long-term clinical stabilizing effect in PAH.
−Removed: We believe that cibotercept has the potential to increase the signaling of BMP pathways through the inhibition of activin A and activin B signaling, and consequently treat diseases such as PAH that are associated with reduced BMP signaling due to inactivating mutations in the BMP receptors.
−Removed: Phase 2 Clinical Trial in Patients with Pulmonary Arterial Hypertension
−Removed: We conducted a randomized, double-blind, placebo-controlled Phase 2 clinical trial to evaluate cibotercept in combination with background therapy in adult patients with PAH, which we refer to as the TROPOS trial.
−Removed: The primary objective of this trial
−Removed: was to evaluate the effect of cibotercept on hemodynamics compared to placebo in patients on background PAH therapy, and the primary endpoint was change from baseline in pulmonary vascular resistance at Week 24.
−Removed: The key secondary objective of this trial was to evaluate the effect of cibotercept on exercise capacity compared to placebo in patients on background PAH therapy, and the key secondary endpoint was change from baseline in 6-minute walk distance at Week 24.
−Removed: Additionally secondary objectives of this trial included evaluating the safety and tolerability of cibotercept, the effects of cibotercept on N-terminal pro B-type natriuretic peptide, or NT-proBNP, a biomarker of myocardial stress, and the improvement in functional class of cibotercept compared to placebo.
−Removed: The original trial design is summarized in the figure below.
−Removed: In December 2024, we announced that we voluntarily halted dosing in the 3.0 mg/kg and 4.5 mg/kg treatment arms in the fully enrolled TROPOS trial based on a safety review due to the unanticipated observation of pericardial effusion adverse events at those dose levels.
−Removed: Subsequently, we announced in January 2025 that we voluntarily halted all dosing in the TROPOS trial, including the 1.5 mg/kg and placebo treatment arms, based on the ongoing safety review due to new observations of pericardial effusion adverse events.
−Removed: The TROPOS trial is being terminated early, and patients are expected to be monitored through the end-of-trial visits.
−Removed: We expect to present topline data from all treatment arms in this trial in the second quarter of 2025.
−Removed: Completed Phase 1 Clinical Trial in Healthy Volunteers
−Removed: In September 2022, we completed a randomized, double-blind, placebo-controlled, two-part Phase 1 clinical trial to evaluate single and multiple ascending doses of cibotercept in healthy volunteers.
−Removed: The primary objectives of this trial were safety, tolerability and pharmacokinetics.
−Removed: The trial design is summarized in the figure below.
−Removed: Phase 1 Clinical Trial Design
−Removed: Observed tolerability data
−Removed: Cibotercept was generally well tolerated in Part 1 of this trial at dose levels up to 5 mg/kg, the highest dose level tested, when administered as a single dose, and multiple doses of 0.75 mg/kg, 1.5 mg/kg and 4.5 mg/kg.
−Removed: In Part 1 of this trial, one subject withdrew consent after receiving a single 1.5 mg/kg dose of cibotercept and did not complete the safety follow-up.
−Removed: In Part 2 of this trial, one subject discontinued after receiving two doses of placebo due to a serious adverse event unrelated to treatment and another subject withdrew consent after receiving two 1.5 mg/kg doses of cibotercept.
−Removed: None of the discontinuations in this trial were due to treatment-related adverse events.
−Removed: No serious adverse events were reported in Part 1 of this trial.
−Removed: Additionally, the majority of the adverse events that were observed in this trial were mild in severity and resolved.
−Removed: Trend for increased whole-body bone mineral density observed
−Removed: Bone mineral density, or BMD, was assessed temporally by dual-energy x-ray absorptiometry in Part 2 of this trial at baseline and at Day 113 of the trial.
−Removed: A trend for increased whole-body BMD was observed after multiple doses of cibotercept.
−Removed: Part 2 of the Trial:
−Removed: BMD Change from Baseline
−Removed: Observed changes in pharmacodynamic markers were consistent with increased BMP signaling in the bone
−Removed: We observed dose-dependent increases in serum bone specific alkaline phosphatase, or BSAP, a marker of osteoblast activity, with a maximal increase observed at the highest doses evaluated in this trial.
−Removed: Cibotercept is designed to inhibit activins and growth differentiation factor ligands in bone, which potentially results in reduced SMAD 2/3 signaling and increased signaling of the BMP pathway (SMAD 1/5/9).
−Removed: The increased BMP signaling potentially promotes bone formation through a dual mechanism of activation and recruitment of bone forming osteoblasts and repression of osteoclasts, as demonstrated in our preclinical studies.
−Removed: In Part 2 of this trial, we observed increases in BSAP after each dose, which is supportive of activation of osteoblasts after each dose.
−Removed: Part 2 of the Trial:
−Removed: BSAP Percent Change from Baseline
−Removed: Multiple doses of cibotercept did not elicit changes in erythropoiesis
−Removed: Administration of cibotercept did not elicit clinically meaningful changes in hemoglobin or red blood cells in this trial, and no changes in red blood cells were observed after the second or third dose.
−Removed: Observed Mean Hemoglobin Change
−Removed: Observed Mean Red Blood Cell Change
−Removed: The observed lack of effect on erythropoiesis in this trial is consistent with the lack of effect observed in our multiple preclinical models.
−Removed: Preclinical Data
−Removed: We have generated preclinical data that we believe demonstrated proof-of-mechanism of cibotercept for the treatment of PAH and for the treatment of cardiovascular disorders.
−Removed: Specifically, in preclinical studies, cibotercept:
−Removed: • Demonstrated effects on bone, including:
−Removed: ◦ Exhibited high affinity for, and potent inhibition of, ligands involved in the regulation of bone homeostasis;
−Removed: ◦ Increased bone mineral density and trabecular bone volume in wild-type mice and mice with established osteoporosis;
−Removed: ◦ Rats receiving a rodent version of cibotercept, or RKER-012, were protected from hypoxia-associated bone loss.
−Removed: • Demonstrated potential for reduced bleeding risk, including:
−Removed: ◦ No inhibition of retinal neovascularization observed in healthy newborn mice.
−Removed: • Demonstrated benefit in a model of cardiovascular disease, including:
−Removed: ◦ In a mouse model of pulmonary arterial banding, or PAB, RKER-012 was observed to protect against both the PAB-related cardiac dysfunction and remodeling.
−Removed: Cibotercept targeted ligands that signal through ActRIIA and ActRIIB in preclinical studies
−Removed: Cibotercept is a modified ActRIIB ligand trap that contains sequences from both wild-type ActRIIB and wild-type ActRIIA.
−Removed: In preclinical studies, cibotercept bound to and inhibited multiple ligands that signal through these cell surface receptors, including activin A, activin B and growth differentiation factor 11.
−Removed: These ligands are key regulators of bone remodeling that act to suppress bone growth.
−Removed: BMP9 is a ligand capable of signaling through the ActRIIB and bone morphogenetic receptor II.
−Removed: Inhibition of BMP9 results in disruption of vascular remodeling, which can lead to the development of epistaxis and telangiectasias.
−Removed: Cibotercept did not bind BMP9 or inhibit BMP9 signaling in preclinical studies.
−Removed: Consequently, we believe cibotercept has the potential to avoid bleeding.
−Removed: Treatment with cibotercept increased bone mineral density
−Removed: In preclinical studies conducted in wild-type mice, twice weekly intraperitoneal 20 mg/kg dosing of cibotercept increased bone mineral density compared to vehicle-treated mice 31 days post-treatment.
−Removed: Additionally, we observed that treatment with
−Removed: cibotercept statistically significantly increased trabecular bone formation and mineral apposition rate, which we believe is consistent with an anabolic effect on bone.
−Removed: Bone Mineral Density in Mice and Representative Microct Scans
−Removed: *** P value <0.001
−Removed: In a separate preclinical study, we observed that treatment with cibotercept increased the ratio of osteoblasts, which are bone forming cells, to osteoclasts, which are bone resorbing cells, which further supports that cibotercept acts via an anabolic effect on bone.
−Removed: We also observed in preclinical studies conducted in mice with established osteoporosis that twice weekly intraperitoneal 20 mg/kg dosing of cibotercept increased bone mass compared to vehicle-treated mice 46 days post-treatment.
−Removed: Osteoblast-to-Osteoclast Ratio in Mice
−Removed: ** P value <0.01
−Removed: Treatment with RKER-012 prevented bone loss from hypoxia in the rat model of PAH
−Removed: In the rat model of PAH, chronic hypoxia induced a catabolic state that resulted in wasting of tissue, including bone and muscle.
−Removed: Treatment with a subcutaneous 10 mg/kg dose of RKER-012 was observed to prevent bone loss in the rat model of PAH.
−Removed: Bone Volume Changes as a Result of Hypoxia in the Rat Model of PAH
−Removed: * P value <0.05;
−Removed: ** P value <0.01
−Removed: Treatment with RKER-012 did not inhibit retinal neovascularization in healthy newborn mice
−Removed: In the mouse model of retinal vascularization, which is an established model to study vascular growth and remodeling during development and disease, inhibition of BMP signaling leads to premature termination and increased density of blood vessels.
−Removed: Newborn mice were treated on postnatal day 1 and 3, and on day 8, retinas were dissected and stained to visualize the vasculature and measure vascular plexus.
−Removed: Administration of 10 mpk of ALK1-Fc (a potent inhibitor of BMP9 (which is required for normal vascular remodeling) and BMP10) significantly reduced retinal neovascularization.
−Removed: Additionally, administration of 20 mpk of a research form of sotatercept, RAP-011, which bound BMP9 with higher affinity than RKER-012, showed a dose-related inhibition of retinal vessel outgrowth.
−Removed: However, administration of 10 mpk and 20 mpk of RKER-012 did not inhibit retinal neovascularization.
−Removed: This lack of observed perturbation of retinal blood vessels of newborn mice treated with RKER-012 supports the potential for reduced bleeding risk with cibotercept.
−Removed: Quantification of Vascular Outgrowth
−Removed: **P value <0.01;
−Removed: ***P value <0.001;
−Removed: ****P value <0.0001;
−Removed: ns = not significant
−Removed: Treatment with RKER-012 prevented cardiac dysfunction and remodeling in a mouse PAB model
−Removed: We used mechanical restriction of the pulmonary artery in mice to increase pressure in the right ventricular of the heart.
−Removed: In this model, increased ventricular pressure resulted in cardiac dysfunction, as demonstrated by increased end systolic pressure-volume relationship, or ESPVR, and increased myocardial performance index, or MPI.
−Removed: The increased ventricular pressure also results in cardiac remodeling, as evidenced by an increase in the Fulton index, an increase in the right ventricular free wall thickness, or RVFWT, and increased fibrosis in the heart.
−Removed: Treatment with twice weekly subcutaneous 10 mg/kg dosing of RKER-012 was observed to protect against both the PAB-related cardiac dysfunction and remodeling, which we believe demonstrates that cibotercept has the potential to have a cardioprotective effect that could potentially provide benefit in diseases such as PAH and other cardiovascular diseases in patients.
−Removed: ESPVR, MPI, Fulton Index, RVFWT and Cardiac Fibrosis in a Mouse PAB Model
−Removed: *** P value <0.001;
−Removed: **** P value <0.0001 vs.
−Removed: ## P value <0.01, #### P value <0.0001 vs.
Our Neuromuscular Franchise
−Removed: KER-065 is a novel ligand trap comprised of a modified ligand-binding domain derived from ActRIIA and ActRIIB that is fused to the portion of the human antibody known as the Fc domain.
−Removed: KER-065 is designed to act as a ligand trap and inhibit the biological effects of myostatin and activin A, two ligands that signal through activin receptors, increase skeletal muscle regeneration, increase muscle size and strength, reduce body fat, reduce fibrosis of the skeletal muscle and increase bone strength.
−Removed: We are developing KER-065 for the treatment of neuromuscular diseases, with an initial focus on DMD.
−Removed: Neuromuscular Disease, including Duchenne Muscular Dystrophy
+Added: Rinvatercept is a novel ligand trap comprised of a modified ligand-binding domain derived from ActRIIA and ActRIIB that is fused to the portion of the human antibody known as the Fc domain.
+Added: Rinvatercept is designed to act as a ligand trap and inhibit the biological effects of myostatin and activin A, which are negative regulators of muscle and bone mass and strength, to improve skeletal muscle regeneration, increase muscle size and strength, inhibit and reduce fibrosis, inhibit inflammation, reduce fat accumulation and improve bone health through bone anabolic mechanisms.
+Added: We are developing rinvatercept for the treatment of DMD and for the treatment of ALS.
+Added: We expect to commence a Phase 2 clinical trial of rinvatercept in patients with DMD in the second quarter of 2026.
+Added: We also plan to engage regulators on the design of a Phase 2 clinical trial of rinvatercept in patients with ALS in the second half of 2026.
+Added: Neuromuscular Disease
Neuromuscular disease is a broad term that encompasses many diseases that either directly (via intrinsic muscle pathology) or indirectly (via nerve pathology) impair the functioning of muscles.
Symptoms of neuromuscular disease include increasing generalized weakness and fatigue, dysphagia, dyspnea on exertion and at rest, sleepiness, morning headache, difficulties with concentration and mood changes.
−Removed: Most neuromuscular diseases are characterized by progressive muscular impairment leading to loss of muscle function and can lead to loss of ambulation, being wheelchair-bound, swallowing difficulties, respiratory muscle weakness and death.
+Added: Most neuromuscular diseases are characterized by progressive muscular impairment
+Added: leading to loss of muscle function and can lead to loss of ambulation, being wheelchair-bound, swallowing difficulties, respiratory muscle weakness and death.
Neuromuscular disorders can progress rapidly or slowly.
Decline in muscle mass can also be associated with secondary osteoporosis and obesity.
+Added: Duchenne Muscular Dystrophy
One example of a rapidly progressive condition is Duchenne muscular dystrophy, or DMD, which is the most common form of muscular dystrophy and results in muscle degeneration and premature death.
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This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD.
−Removed: Organization for Rare Disorders estimates that approximately one in every 3,500 male births is affected by DMD worldwide.
+Added: The National Organization for Rare Disorders estimates that approximately one in every 3,500 male births is affected by DMD worldwide.
The symptoms of DMD typically manifest in the first few years of life.
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Additionally, fracture can lead to premature loss of ambulation, which can have a detrimental effect on independent mobility and quality of life.
−Removed: Decreased mobility along with the use of glucocorticoids are associated with increased risk of obesity and the associated negative health consequences, including type 2 diabetes and cardiovascular disease, in DMD patients.
+Added: Decreased mobility, along with the use of glucocorticoids, is associated with increased risk of obesity and the associated negative health consequences, including type 2 diabetes and cardiovascular disease, in DMD patients.
Limitations of Current Treatment Options for DMD
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This product was approved using the accelerated approval pathway on the basis of expression of ELEVIDYS micro-dystrophin in patients treated with ELEVIDYS.
−Removed: Continued approval for this indication in non-ambulatory individuals aged four years and older may be contingent upon verification and description of clinical benefit in confirmatory clinical trials.
+Added: Following reports of fatal liver injury, the FDA removed the non-ambulatory indication from the official label in November 2025.
+Added: In the European Union, neither ELEVIDYS nor any exon-skipping therapies currently hold marketing authorization for the treatment of Duchenne muscular dystrophy.
In March 2024, Italfarmaco S.p.A.
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Low platelet counts resulted in Duvyzat dose reduction in 28% of DMD patients in a randomized, double-blind, placebo-controlled 18-month trial.
−Removed: Based on our preclinical data, we believe that KER-065 has the potential to treat multiple pathophysiologies of DMD by improving muscle and bone strength and reducing fat mass and cardiac fibrosis.
−Removed: Ongoing Phase 1 Clinical Trial in Healthy Volunteers
−Removed: We have initiated a randomized, double-blind, placebo-controlled, two-part Phase 1 clinical trial to evaluate single and multiple ascending doses of KER-065 in healthy volunteers.
−Removed: The primary objectives of this trial are to assess safety, tolerability and pharmacokinetics of KER-065.
−Removed: Exploratory endpoints include assessments of the pharmacodynamic effect on bone, adipose, muscle, cardiac tissue and fibrosis.
−Removed: To aid in the assessment of adipose tissue, volunteers will be required to have a BMI ≥ 27 to ≤ 35 kg/m 2 to be enrolled in Part 2 of this trial.
+Added: Based on our preclinical data, we believe that rinvatercept has the potential to treat multiple pathophysiologies of DMD by improving muscle and bone strength and reducing fat mass and cardiac fibrosis.
+Added: Amyotrophic Lateral Sclerosis
+Added: Amyotrophic lateral sclerosis, or ALS, is a fatal, progressive, neurodegenerative disease that specifically targets motor neurons in the spinal cord and brain.These are specialized cells that are responsible for controlling voluntary muscle movement.
+Added: Each muscle is made up of multiple motor units, which consists of a motor neuron that is connected and activates each muscle fiber via a neuromuscular junction.
+Added: In ALS, the loss of motor neurons and neuromuscular junctions leads to atrophy and a subsequent loss of connected muscle fibers.
+Added: However, healthy, connected adjacent muscle fibers and motor units remain.
+Added: Early symptoms of ALS include muscle twitching and weakness in a limb.
+Added: As the disease progresses, patients with ALS lose the ability to move, speak, eat and breathe.
+Added: This progressive loss of motor neurons leads to muscle weakness, loss of function and eventually paralysis.
+Added: The majority of patients with ALS die from respiratory failure within three to five years of first symptom onset, and the patient’s quality of life is typically poor.
+Added: In the United States alone, ALS has a prevalence of approximately 9.9 per 100,000 people, with approximately 33,000 people estimated to have been living with ALS in 2022.
+Added: The biological mechanisms that cause ALS are only partially understood.
+Added: While 10% of ALS cases are familial, linked to mutations in certain genes, the vast majority of ALS cases (approximately 90%) are sporadic with no known family history.
+Added: Limitations of Current Treatment Options for ALS
+Added: There is currently no cure for ALS, and currently approved therapies offer limited benefits, aiming to provide temporary relief of symptoms without treating underlying disease progression for the majority of patients with ALS.
+Added: There are three therapies approved by FDA to treat ALS and its symptoms:
+Added: riluzole, an anti-glutamatergic agent;
+Added: edaravone, a free-radical scavenger;
+Added: and tofersen, an antisense oligonucleotide treatment for ALS associated with superoxide dismutase 1 (SOD1) gene mutation.
+Added: One additional therapeutic option, sodium phenylbutyrate and taurursodiol, was approved under an accelerated pathway by the FDA but subsequently withdrawn from the market following negative results from a confirmatory Phase 3 trial.
+Added: These treatments have demonstrated only modest clinical efficacy.
+Added: Based on our preclinical data, we believe that rinvatercept has the potential to maximize the functional strength of healthy, connected muscle fibers, allowing them to compensate for atrophied fibers and motor units.
+Added: This has the potential to lead to overall preservation of muscle strength and function and improved quality of life for patients with ALS.
+Added: Phase 2 Clinical Trial in Patients with DMD
+Added: We expect to commence an open-label, multi-cohort Phase 2 basket clinical trial to evaluate rinvatercept in patients with DMD in the second quarter of 2026.
+Added: The primary objective of this trial is to assess the safety and tolerability of rinvatercept in late-ambulatory and early non-ambulatory patients with DMD.
+Added: The key secondary objectives of this trial are to evaluate pharmacokinetics, anti-drug antibodies, body composition and comprehensive functional improvements (skeletal muscle, motor, cardiac and pulmonary), plus exploratory serum biomarkers.
The trial design is summarized in the figure below.
Phase 2 Clinical Trial Design
−Removed: We expect to report initial data from this trial in the first quarter of 2025.
−Removed: We believe this trial has the potential to inform the development of KER-065 in neuromuscular indications, such as DMD.
+Added: Rinvatercept is administered to patients subcutaneously once every four weeks, starting at a dose of 0.5 mg/kg.
+Added: Based on individual titration rules, the dose may be escalated in 0.5 mg/kg increments after two doses, up to a maximum dose of 2.0 mg/kg.
+Added: Completed Phase 1 Clinical Trial in Healthy Adult Volunteers
+Added: We completed a randomized, double-blind, placebo-controlled, two-part Phase 1 clinical trial to evaluate single and multiple ascending doses of rinvatercept in healthy adult male volunteers.
+Added: The primary objectives of this trial were to assess safety, tolerability and pharmacokinetics of rinvatercept.
+Added: Exploratory endpoints included assessments of the pharmacodynamic effect on bone, adipose, muscle, cardiac tissue and fibrosis.
+Added: To aid in the assessment of adipose tissue, volunteers were required to have a BMI ≥ 27 to ≤ 35 kg/m 2 to be enrolled in Part 2 of this trial.
+Added: The trial design is summarized in the figure below.
+Added: Phase 1 Clinical Trial Design
+Added: Rinvatercept was generally well-tolerated, with no major safety signals observed in the trial.
+Added: No serious adverse events or dose-limiting toxicities were reported.
+Added: Additionally, we observed evidence for activin inhibition across tissues of interest, as rinvatercept elicited:
+Added: • Increased lean muscle mass and thigh muscle volume;
+Added: • Increases in bone specific alkaline phosphate, a biomarker of bone formation, demonstrating mobilization of osteoblasts;
+Added: • Decreases in C-Terminal Telopeptide, a biomarker that reflects the rate of bone resorption;
+Added: • Increases in whole body bone mineral density that was durable and sustained, suggesting a balance of osteoblast and osteoclast activity;
+Added: • Increases in adiponectin, a biomarker of fat mobilization, and decreases in leptin, a biomarker of fat mass;
+Added: • Corresponding decreases in fat mass, both whole body and visceral fat mass.
Preclinical Data
−Removed: We have generated preclinical data that we believe demonstrated proof-of-mechanism of KER-065 for the treatment of neuromuscular diseases, such as DMD.
+Added: We have generated preclinical data that we believe demonstrated proof-of-mechanism of rinvatercept for the treatment of DMD and for the treatment of ALS.
Specifically, in preclinical studies:
−Removed: • KER-065 showed high affinity for and potent inhibition of ligands involved in the regulation of muscle and bone homeostasis;
−Removed: • RKER-065 increased utrophin expression and muscle strength in a mouse model of DMD;
+Added: • Rinvatercept showed high affinity for and potent inhibition of ligands involved in the regulation of muscle and bone homeostasis;
+Added: • RKER-065, the research form of rinvatercept, increased utrophin expression and muscle strength in a mouse model of DMD;
• Co-treatment with prednisolone and RKER-065 increased both muscle mass and strength and trabecular bone and strength;
+Added: • Co-treatment with an HDAC inhibitor and RKER-065 increased muscle mass and strength and reduced fibrosis;
• RKER-065 increased satellite cells in skeletal muscle;
• Co-treatment with phosphorodiamidate morpholino oligomer, or PMO, therapy and RKER-065 improved grip strength and the efficiency of exon skipping;
+Added: • RKER-065 treatment reduced muscle wasting and preserved muscle function in a mouse model of ALS.
KER-065 targeted ligands that signal through ActRIIA and ActRIIB to increase skeletal muscle and bone in preclinical studies
4 unchanged sentences
Treatment with RKER-065 increased utrophin expression and muscle strength in a mouse model of DMD
−Removed: In preclinical studies conducted in the MDX mouse model of DMD, twice weekly, intraperitoneal 10 mg/kg dosing of RKER-065 increased expression of utrophin in muscle fibers, potentially contributing to the observed increased strength.
+Added: In preclinical studies conducted in the X-linked muscular dystrophy, or MDX, mouse model of DMD, twice weekly, intraperitoneal 10 mg/kg dosing of RKER-065 increased expression of utrophin in muscle fibers, potentially contributing to the observed increased strength.
Evoked Force Maximum Gastrocnemius
1 unchanged sentence
Co-treatment with prednisolone and RKER-065 increased both muscle mass and strength and trabecular bone and strength
−Removed: MDX mice were treated with vehicle or 2-prednisolone, or co-treated with 10 mg/kg of prednisolone and RKER-065 twice weekly.
+Added: MDX mice were treated with vehicle (Veh) or 2-prednisolone (Pred), or co-treated with 10 mg/kg of prednisolone and RKER-065 twice weekly (Pred+065).
Prednisolone-treated MDX mice had less muscle mass and strength than vehicle-treated mice, while co-treatment with prednisolone and RKER-065 increased both muscle mass and strength and trabecular bone and strength.
24 unchanged sentences
***P value <0.001
+Added: Co-treatment with an HDAC inhibitor and RKER-065 increased both muscle mass and strength and reduced fibrosis
+Added: MDX mice were treated with vehicle or an HDAC inhibitor, or HDACi, or co-treated with 10 mg/kg HDACi daily and 3 mg/kg RKER-065 twice weekly.
+Added: HDACi-treated MDX mice had less muscle mass and strength than vehicle-treated mice, while co-treatment with HDACi and RKER-065 increased both muscle mass and strength and reduced markers of fibrosis in the muscle tissue.
+Added: **P value <0.01;
+Added: **** P value < 0.0001
+Added: RKER-065 treatment reduced muscle wasting and preserved muscle function in a mouse model of ALS
+Added: In a mouse model of ALS, ALS mice were treated with vehicle or 10 mg/kg RKER-065 twice weekly.
+Added: RKER-065 treatment preserved both muscle mass and strength and neuromuscular transmission.
+Added: *P value ≤0.05;
+Added: **P value <0.01;
+Added: ****P value <0.0001
Our Hematology Franchise
8 unchanged sentences
These cells progress through a series of intermediate stages before becoming a mature cell with a specialized function.
−Removed: At any given time, pools of each progenitor cell are maintained and primed to rapidly respond to a
−Removed: reduction of red blood cells, white blood cells and platelets.
−Removed: The graphic below depicts the stages of hematopoiesis for red blood cells and platelets.
−Removed: Stages of Hematopoiesis
+Added: At any given time, pools of each progenitor cell are maintained and primed to rapidly respond to a reduction of red blood cells, white blood cells and platelets.
TGF-ß signaling pathways involving activins prevent differentiation in order to maintain progenitor cells in a quiescent state while others involving BMPs promote differentiation of progenitor cells.
1 unchanged sentence
In many hematological disorders, there is abnormal proliferation and differentiation of the progenitor cells for red blood cells, platelets and neutrophils.
−Removed: This failure to produce fully mature cells is termed ineffective hematopoiesis, and may be due to single or multiple defects that can lead to a hyperproliferation or a shortage of progenitor cells.
+Added: This failure to produce fully mature cells is termed ineffective hematopoiesis, and may be due to single or multiple defects that can lead to hyperproliferation or a shortage of progenitor cells.
These changes have clinical consequences:
1 unchanged sentence
The failure of progenitor cells to differentiate can also lead to a build-up of these cells, resulting in bone marrow failure and fibrotic disease.
−Removed: The graphic below provides an illustration of the difference in the number of progenitor cells and mature bloods cells that are produced in normal hematopoiesis and in ineffective hematopoiesis.
−Removed: Another critical component in red blood cell development is the production of hemoglobin, an iron-containing protein that delivers oxygen to cells and removes carbon dioxide.
−Removed: The synthesis of hemoglobin requires that sufficient levels of iron are present in the bone marrow and if iron levels are too low, it can result in a failure to produce sufficient numbers of red blood cells.
−Removed: Anemia is a common consequence of diseases where normal iron mobilization is hindered.
For the Treatment of Ineffective Hematopoiesis to Address Cytopenias
−Removed: We are developing elritercept, our lead product candidate, for the treatment of cytopenias that occur due to ineffective hematopoiesis, including anemia and thrombocytopenia, in patients with MDS and in patients with myelofibrosis.
+Added: Elritercept, our most advanced product candidate, is being developed for the treatment of cytopenias that occur due to ineffective hematopoiesis, including anemia and thrombocytopenia, in patients with MDS and in patients with myelofibrosis.
Elritercept is designed to benefit patients suffering from defects in red blood cell and platelet differentiation and maturation across the spectrum from early through terminal stages of hematopoiesis.
29 unchanged sentences
While ESAs have been shown to alleviate anemia in a subset of patients with MDS, patients that have elevated endogenous erythropoietin levels are unlikely to respond.
−Removed: In two controlled Phase 3 clinical trials evaluating darbepoetin alfa (Aranesp) and epoetin alpha (Epogen/Procrit) for the treatment of MDS-associated anemia, 15% to 31% of patients responded, respectively.
+Added: In two controlled Phase 3 clinical trials evaluating darbepoetin alfa (Aranesp) and epoetin alpha (Epogen/Procrit) for the treatment of MDS-associated anemia, 15% to 31% of
+Added: patients responded, respectively.
However, this response was limited to patients with low endogenous erythropoietin levels at baseline and to patients who had a low transfusion burden at baseline.
34 unchanged sentences
Currently approved products for the treatment of myelofibrosis, including JAK inhibitors ruxolitinib (Jakafi), fedratinib (Inrebic) and pacritinib (Vonjo), have been observed to exacerbate myelofibrosis-associated cytopenias.
−Removed: In a third-party Phase 3 clinical trial of Jakafi and a third-party Phase 3 clinical trial of Inrebic, treatment led to significant reductions in spleen volume and improvement in total symptom scores.
+Added: In a third-party Phase 3 clinical trial of Jakafi and a third-party Phase 3 clinical trial of Inrebic, treatment led to significant reductions in spleen volume
+Added: and improvement in total symptom scores.
However, JAK inhibitors interfere with normal hematopoiesis and treatment with Jakafi and Inrebic also resulted in clinically significant anemia and thrombocytopenia in these Phase 3 trials.
16 unchanged sentences
Mechanism of Action of Elritercept
−Removed: Consistent with our preclinical studies, which showed improvement in bone health, we observed an increase in b one-specific alkaline phosphatase, a biomarker of bone remodeling , in our Phase 1 clinical trial in healthy volunteers and our ongoing Phase 2 clinical trial in patients with MDS, following administration of elritercept.
+Added: Consistent with our preclinical studies, which showed improvement in bone health, we observed an increase in b one-specific alkaline phosphatase, a biomarker of bone remodeling , in our Phase 1 clinical trial in healthy volunteers and our ongoing
+Added: Phase 2 clinical trial in patients with MDS, following administration of elritercept.
Based on these data, we believe elritercept also has the potential to regenerate a healthy bone marrow and slow disease progression.
17 unchanged sentences
We observed an increase in platelets 12 hours after administration of RKER-050, which we believe supports that elritercept can potentially promote production of platelets by blocking inhibitory TGF-ß ligands so that megakaryocytes can fully differentiate.
−Removed: Additionally, bone marrow analysis performed 24 hours post-dose demonstrated that administration of RKER-050 increased the megakaryocyte precursor population, and that these
−Removed: cells had increased ploidy, compared to vehicle.
+Added: Additionally, bone marrow analysis performed 24 hours post-dose demonstrated that administration of RKER-050 increased the megakaryocyte precursor population, and that these cells had increased ploidy, compared to vehicle.
These data suggest that RKER-050 promoted the maturation of early megakaryocyte populations and primed megakaryocytes for proplatelet production.
12 unchanged sentences
In December 2024, we entered into an exclusive license agreement with Takeda to further develop, manufacture and commercialize elritercept worldwide outside of mainland China, Hong Kong and Macau, which became effective on January 16, 2025.
−Removed: See the section titled “Business—Collaborations and License Agreement—2024 License Agreement with Takeda Pharmaceuticals U.S.A., Inc.” set forth in Part I, Item 1 of this Annual Report on Form 10-K for additional information regarding our license agreement with Takeda.
+Added: See the section titled “Business—Collaborations and License Agreement—2024 License Agreement with Takeda
+Added: Pharmaceuticals U.S.A., Inc.” set forth in Part I, Item 1 of this Annual Report on Form 10-K for additional information regarding our license agreement with Takeda.
Ongoing Phase 3 Clinical Trial in Patients with Myelodysplastic Syndromes
4 unchanged sentences
Phase 3 Clinical Trial Design
−Removed: Ongoing Phase 2 Clinical Trial in Patients with Myelodysplastic Syndromes
−Removed: We are conducting an open label, two-part, multiple ascending dose Phase 2 clinical trial to evaluate elritercept in patients with lower-risk MDS who either have or have not previously received treatment with an ESA.
−Removed: The primary objective of this trial is to assess the safety and tolerability of elritercept in patients with MDS that either have ring sideroblasts, or RS positive, or do not have ring sideroblasts, or non-RS.
−Removed: The primary objective of Part 2 of this trial is confirmation of the safety and tolerability of the selected dose levels.
−Removed: The secondary objectives of this trial are to evaluate the pharmacokinetics, pharmacodynamics and efficacy of elritercept.
−Removed: The trial design is summarized in the figure below.
−Removed: Phase 2 Clinical Trial Design
−Removed: chronic myelomonocytic leukemia
−Removed: Elritercept is being administered to patients subcutaneously once every four weeks.
−Removed: In Part 2, the dose confirmation portion of the trial, an identical dosing schedule was followed, and patients initiated treatment at a starting dose of 3.75 mg/kg, the recommended Part 2 dose, or RP2D, with the opportunity to dose escalate to 5.0 mg/kg or to down-titrate based on individual titration rules.
−Removed: Following completion of Part 1, eligible patients were given the opportunity to escalate up to the RP2D and receive long-term treatment with elritercept for up to an additional 20 cycles, which we refer to as the Part 1 Extension.
−Removed: In December 2024, we presented additional data from this ongoing trial at the 66th American Society of Hematology, or ASH, Annual Meeting and Exposition.
−Removed: As of August 30, 2024, which was the data cut-off date, 95 patients had received at least one dose of elritercept at RP2D, which we refer to as the safety population.
−Removed: 87 of these patients had completed at least 24 weeks of treatment or discontinued as of the data cut-off date, which we refer to as the mITT 24 patients.
−Removed: Data for hematological response and markers of hematopoiesis were presented from exploratory analyses of these mITT 24 patients.
−Removed: Of the 95 patients in the safety population, 60.0% (n=57) had high transfusion burden, or HTB, while 24.2% (n=23) had low transfusion burden, or LTB, and 15.8% (n=15) were non-transfused, or NT.
−Removed: Elritercept was generally well tolerated as of the data cut-off date.
−Removed: There were four cases of fatal treatment-emergent adverse events, or TEAEs, in the trial that were all determined to be unrelated to treatment.
−Removed: The most commonly reported TEAEs (in ≥15% of patients) were diarrhea, fatigue, COVID-19, dyspnea, dizziness, anemia, nausea and epistaxis.
−Removed: One patient had progressed to acute myeloid leukemia as of the data cutoff date.
−Removed: As of the data cut-off date, 55.2% (n=48/87) of the mITT 24 patients achieved an overall erythroid response over the first 24 weeks of treatment, which is defined as meeting either modified International Working Group 2006 Hematological improvement-erythroid, or HI-E, or transfusion independence, or TI, for at least eight weeks in transfusion-dependent patients who required ≥ 2 red blood cell units transfused at baseline.
−Removed: The median duration of transfusion independence was 134.1 weeks.
−Removed: Due to ongoing TI responses as of the data cutoff date, the median duration of TI is expected to change as data continues to accumulate.
−Removed: 48.1% (n=13/27) of patients with a TI response had ongoing TI as of the data cutoff date, of which 92.3% (n=12/13) had ongoing TI for greater than 52 weeks.
−Removed: Additional data from the mITT 24 patients, as of the data cut-off date, include:
−Removed: • 39.1% (n=27/69) of the TI-evaluable patients achieved TI for at least eight weeks over the first 24 weeks of treatment.
−Removed: • Of the patients with HTB, 31.4% (n=16/51) achieved TI for at least eight weeks during the first 24 weeks of treatment.
−Removed: Eight of those 16 patients (50.0%) achieved TI for at least 24 weeks over the first 48 weeks of treatment.
−Removed: Studies in mainly lower-risk-MDS patients suggest that the majority (~90%) of patients have serum erythropoietin levels less than 500 U/L.
−Removed: Additionally, erythropoietin levels of ≥ 500 U/L are associated with lower erythroid response rates across
−Removed: multiple treatments.
−Removed: Accordingly, we evaluated a subset of transfusion-dependent mITT 24 patients with a baseline erythropoietin level less than 500 U/L (n=55), and observed the following, as of the data cut-off date:
−Removed: • 47.3% (n=26/55) achieved TI for at least eight weeks over the first 24 weeks of treatment.
−Removed: • Of the mITT 24 patients with baseline erythropoietin level less than 500 U/L and HTB, 38.5% (n=15/39) achieved TI for at least eight weeks over the first 24 weeks of treatment.
−Removed: The FACIT-Fatigue scale, a measure of self-reported fatigue and its impact upon daily activities and function, was utilized to assess health-related quality of life, including in a subgroup of patients (n=17) achieving TI for at least 24 weeks over the first 48 weeks of treatment.
−Removed: Patients in this subgroup showed clinically meaningful improvements in quality of life, and meaningful improvements in FACIT-Fatigue were observed early and generally continued to improve over time in patients with more durable TI responses.
−Removed: The majority of patients enrolled in this ongoing trial had HTB and/or multi-lineage dysplasia, indicating a difficult-to-treat trial population.
−Removed: Durable TI responses continued to be observed in a broad range of patients with lower-risk MDS, including in those with HTB, which support the potential for elritercept to ameliorate ineffective hematopoiesis across multiple lineages in patients with MDS.
−Removed: In a subgroup analysis of patients that were NT at baseline, treatment with elritercept showed:
−Removed: • Robust hematological responses observed with 93.3% (n=14/15) of NT patients having an increase greater than 1.0 g/dL and 86.7% (n=13/15) having an HI-E response.
−Removed: • Durable HI-E responses observed with elritercept treatment with 100% (n=13/13) achieving a continuous response duration of greater than 24 weeks and 76.9% (n=10/13) achieving a cumulative response duration greater than 52 weeks.
−Removed: • Sustained and durable increases in hemoglobin and soluble transferrin receptor, a marker of erythropoietic activity, were observed in NT participants.
−Removed: • Overall improvement in mean platelet and neutrophil counts along with decreases in mean ferritin and hepcidin were observed after only one dose and were generally maintained through 48 weeks, demonstrating that elritercept has the potential to address ineffective hematopoiesis across multiple lineages and improve iron utilization and reduce inflammation.
−Removed: • NT patients achieved meaningful improvements in FACIT-Fatigue scores, with improvements seen early, generally within the first two treatment cycles.
−Removed: Ongoing Phase 2 Clinical Trial in Patients with Myelofibrosis-Associated Cytopenias
−Removed: We are conducting an open label, two-part, multiple ascending dose Phase 2 clinical trial to evaluate elritercept as a monotherapy and in combination with ruxolitinib in patients with myelofibrosis-associated cytopenias.
−Removed: The primary objective of this trial is to assess the safety and tolerability of elritercept in patients with myelofibrosis-associated cytopenias.
−Removed: The primary objective of Part 2 of this trial is confirmation of the safety and tolerability of the selected dose levels.
−Removed: The secondary objectives of this trial are to evaluate the pharmacokinetics, pharmacodynamics and efficacy of elritercept administered with or without ruxolitinib.
−Removed: The trial design is summarized in the figure below.
−Removed: Phase 2 Clinical Trial Design
−Removed: In December 2024, we presented additional data from this ongoing trial at the 66th ASH Annual Meeting and Exposition.
−Removed: Safety data were presented for all patients that received at least one dose of elritercept (n=73) as of the August 30, 2024 data cutoff date.
−Removed: Evaluations of markers of hematopoiesis and anemia over 12 weeks, along with measurements of spleen volume and symptom scores (by the Myelofibrosis-Symptom Assessment form-Total Symptom Score, or MF-SAF-TSS) over 24 weeks, were presented for dose levels 1 through 4 in Part 1 and the RP2D, ranging from 0.75 mg/kg to 5.0 mg/kg, which we refer to as the efficacy evaluable patients.
−Removed: Enrollment of Part 1 of the trial, the dose escalation portion, is complete.
−Removed: Part 2, the dose expansion portion, is enrolling with an RP2D of 3.75 mg/kg with the option to up-titrate to 5.0 mg/kg.
−Removed: Elritercept was generally well tolerated by the safety population as of the data cut-off date.
−Removed: There were six cases of fatal TEAEs in the trial that were each deemed unrelated to treatment.
−Removed: The most commonly reported TEAEs (in ≥15% of patients) were thrombocytopenia and diarrhea.
−Removed: The majority of treatment-related TEAEs were mild to moderate, with 12 patients experiencing Grade 3 or higher treatment-related TEAEs of thrombocytopenia.
−Removed: 93.3% (n=14/15) of patients with a TEAE of thrombocytopenia had baseline platelets below 150 x 10 9 /L.
−Removed: Additional data from the efficacy evaluable patients as of the data cut-off date include:
−Removed: • Increases in hemoglobin were observed in 82.8% (n=24/29) of evaluable non-transfusion dependent patients in both arms over a 12-week period within the first 24 weeks, suggesting that elritercept has the potential to address anemia due to MF and ruxolitinib-associated anemia.
−Removed: • 63.4% (n=26/41) of patients that received at least three red blood cell units per 12 weeks at baseline in both arms and all dose levels tested showed reductions in transfusion burden over 12 weeks within the first 24 weeks.
−Removed: 24.4% (n=10/41) of the patients who showed reductions in transfusion burdens achieved TI.
−Removed: ◦ Additionally, within the subgroup of these patients in the combination arm who received a starting dose of 3.0 mg/kg of elritercept or higher, 62.5% (n=10/16) had reductions of 50% or greater, and 37.5% (n=6/16) achieved TI.
−Removed: • At week 24, reduction in spleen volume was observed in 40% (n=8/20) of patients with baseline spleen size ≥ 450 cm 3 and a week 24 spleen assessment, including three patients who had reductions of 35% or greater.
−Removed: Reductions in spleen volume in the combination arm generally occurred without an increase in ruxolitinib dose.
−Removed: ◦ For evaluable patients in the combination arm with a starting dose of 3.0 mg/kg of elritercept or higher, 88% (n=7/8) had some reduction in spleen size at week 24.
−Removed: • At week 24, reduction in disease symptoms was observed in 66.7% (n=18/27) of patients with at least two symptoms with an average score ≥ 3 or an average total score of ≥ 10 on the MF-SAF-TSS questionnaire at baseline and a week 24 MF-SAF-TSS assessment.
−Removed: Five patients had reductions of at least 50%, including three in the monotherapy arm and two in the combination arm.
−Removed: The data support the potential of elritercept to ameliorate ineffective hematopoiesis and address cytopenias due to myelofibrosis and associated with ruxolitinib, and provide broader clinical benefit in patients, as supported by the observed reduction in spleen volume and improvement in total symptom scores.
−Removed: Completed Phase 1 Clinical Trial
−Removed: In January 2020, we completed a randomized, double-blind, placebo-controlled, two-part, dose-escalation Phase 1 clinical trial of elritercept in 48 healthy post-menopausal women.
−Removed: The primary objectives of this trial were safety, tolerability and pharmacokinetics.
−Removed: We also investigated changes in hematology and bone biomarkers in this clinical trial.
−Removed: In Part 1 of this trial, 30 subjects received a single dose of elritercept and eight subjects received a single dose of placebo, each administered subcutaneously with a 12-week safety follow-up.
−Removed: The subjects were enrolled in sequential single-ascending dose escalation cohorts of up to ten subjects each.
−Removed: In Part 2 of this trial, eight subjects received elritercept and two received placebo, administered subcutaneously, on two occasions 28 days apart, with a 12-week safety follow-up after the second dose.
−Removed: In Part 2 of this trial, only one dose level was evaluated, as it was deemed to provide the necessary data, in addition to that from Part 1 of the trial, to inform the design of the Phase 2 clinical trials of elritercept in patients with MDS and in patients with myelofibrosis.
−Removed: The trial design is summarized in the figure below.
−Removed: Observed tolerability data
−Removed: Elritercept was well tolerated in this Phase 1 clinical trial at dose levels up to 4.5 mg/kg, the highest dose level tested, and multiple doses of 0.75 mg/kg.
−Removed: While one subject in the placebo group withdrew consent, there were no discontinuations due to treatment-related adverse events.
−Removed: No treatment-related serious adverse events were reported.
−Removed: The most common adverse events observed in subjects in this trial were nausea, gastroenteritis, injection site erythema and, consistent with the mechanism of action of elritercept, increased hemoglobin and hypertension.
−Removed: The reversible, mild hypertension events were observed in subjects with an approximately 3 g/dL increase in hemoglobin.
−Removed: Long half-life observed, potentially supporting monthly or less frequent dosing
−Removed: We observed that elritercept drug levels were dose proportional in Part 1 of this trial, with a mean half-life of approximately ten to 12 days.
−Removed: The half-life coupled with the pharmacodynamic effect observed in the hematologic parameters support the potential for administration of monthly or less frequent dosing, which we believe will decrease the burden on patients and improve compliance.
−Removed: Rapid and sustained increases in mean reticulocyte counts, hemoglobin, red blood cell counts and platelet counts observed
−Removed: In Part 1 of this trial, we observed rapid and sustained increases in mean reticulocyte counts, hemoglobin, red blood cell counts and platelet counts.
−Removed: Consistent with the underlying biology, increases in reticulocytes were observed early with increases of hemoglobin following thereafter.
−Removed: Increases in reticulocytes were observed as early as Day 2 and reached a peak
−Removed: around Day 15.
−Removed: Increases in hemoglobin concentration were also observed as early as Day 2, reached a peak around Day 29 and remained elevated for several weeks.
−Removed: We also observed a dose-dependent increase in the proportion of subjects with hemoglobin increases of at least 1.5 g/dL.
−Removed: We believe a 1.5 g/dL increase would be considered clinically meaningful in patients with low red blood cell counts.
−Removed: In addition to the changes in erythroid parameters, robust, dose-dependent increases in platelet count were observed after a single dose of elritercept.
−Removed: All subjects who received a 4.5 mg/kg dose of elritercept, the highest dose evaluated, demonstrated an increase of 30 x 10 9 cells/L or greater at any one point in the trial, which we believe would be considered clinically meaningful in patients with low platelet counts.
−Removed: We believe the rapid onset and durability of increased hemoglobin and platelet count observed in our Phase 1 clinical trial supports the potential for a dual effect of elritercept on both early-stage differentiation and terminal maturation.
−Removed: Additionally, we observed reductions in follicle-stimulating hormone, a biomarker of activin inhibition, following administration of elritercept, which we believe is indicative of target engagement and activin inhibition.
−Removed: We also observed an increase in bone-specific alkaline phosphatase, a biomarker of bone remodeling, which we believe demonstrates that elritercept has the potential to increase bone mass.
−Removed: We believe that the findings from this Phase 1 clinical trial demonstrate the translation of biological action from rodents to humans.
−Removed: We also believe that data from our preclinical studies and clinical trials support that treatment with elritercept has the potential to address ineffective hematopoiesis in diseases where multiple cytopenias arise from the blockage in progression of progenitor cells to mature blood cells, such as in MDS and myelofibrosis.
Our Preclinical Pipeline
Our Proprietary Discovery Approach
−Removed: We believe, based on our previous experience with ActRII ligand traps using the endogenous and wild-type sequences, that observations in preclinical rodent models have the potential to translate to humans in the clinic.
+Added: We believe, based on our previous experience with ActRII ligand traps using endogenous and wild-type sequences, that observations in preclinical rodent models have the potential to translate to humans in the clinic.
Specifically:
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▪ Molecules designed to increase muscle and bone mass with reduced BMP9 binding without impacting vascular remodeling that leads to weak blood vessels observed with the wild-type ActRIIB-Fc.
−Removed: Our discovery approach has built on these initial observations to generate product candidates designed to target ActRII receptors without certain downsides observed in third-party preclinical studies and clinical trials of ActRIIA-Fc and ActRIIB-Fc.
−Removed: We believe that we are well positioned to advance our product candidates and pursue the commercial opportunities in diseases where muscle and bone loss result in a debilitating impact on survival and quality of life, if our product candidates are successfully developed and approved.
−Removed: Our deep knowledge and expertise of the TGF-ß family of proteins provides a streamlined approach to screen and develop novel product candidates for hematological, pulmonary and cardiovascular disorders.
+Added: Our discovery approach has built on these initial observations to generate product candidates designed to target ActRII receptors that address certain downsides observed in third-party preclinical studies and clinical trials of ActRIIA-Fc and ActRIIB-Fc.
+Added: We believe that we are well positioned to advance our product candidates and pursue commercial opportunities in diseases where muscle and bone loss result in a debilitating impact on survival and quality of life, if our product candidates are successfully developed and approved.
+Added: Our deep knowledge and expertise of the TGF-ß family of proteins provides a streamlined approach to screen and develop novel product candidates for a broad range of indications, including neuromuscular and neurodegenerative disorders, rare bone and fibrosis diseases and obesity/frailty indication.
Manufacturing
1 unchanged sentence
We require that our CMOs produce bulk drug substances and finished drug products in accordance with current Good Manufacturing Practices, or cGMPs, and all other applicable laws and regulations.
−Removed: We maintain agreements with our manufacturers that include confidentiality and intellectual property provisions to protect our proprietary rights related to our product candidates.
+Added: We have quality agreements in place with our key CMOs.
+Added: We have also established an internal quality management organization, which audits and qualifies our CMOs.
+Added: Additionally, we maintain agreements with our manufacturers that include confidentiality and intellectual property provisions to protect our proprietary rights related to our product candidates.
We have engaged CMOs to manufacture supply for preclinical and clinical use.
8 unchanged sentences
There are many other companies, including large biotechnology and pharmaceutical companies, that have commercialized and/or are developing therapies for the same therapeutic areas that our product candidates target.
−Removed: For example, in March 2024, Merck & Co.
−Removed: Inc., or Merck, received FDA approval of its product, sotatercept (WINREVAIR), for the treatment of adults with PAH.
−Removed: In August 2024, Merck announced that the European Commission approved sotatercept for the treatment of adults with PAH.
−Removed: All of the other currently-approved therapies for PAH are vasodilators, which are medications that dilate blood vessels.
−Removed: Gossamer Bio, Inc.
−Removed: is developing seralutinib for the treatment of PAH.
Currently, patients with DMD are treated with corticosteroids to manage the inflammatory component of the disease.
EMFLAZA (deflazacort) is an FDA-approved corticosteroid marketed by PTC Therapeutics, Inc.
−Removed: In October 2023, the FDA granted Agamree (vamorolone) approval in patients with DMD aged two years and older and Catalyst Pharmaceuticals, Inc.
−Removed: announced commercialization of this product in the United States in March 2024 following its North America exclusive license deal with Santhera Pharmaceuticals Holding AG.
+Added: and Agamree (vamorolone) is an FDA-approved corticosteroid marketed by Catalyst Pharmaceuticals in the United States.
In addition, there are four FDA-approved exon skipping drugs:
2 unchanged sentences
In June 2024, the FDA granted ELEVIDYS full approval for the treatment of ambulatory individuals aged four years and older, and accelerated approval for the treatment of non-ambulatory individuals aged four years and older.
+Added: In July 2025, Sarepta announced its decision to voluntarily and temporarily pause all shipments of ELEVIDYS for patients with DMD in the United States, following reports of fatal liver injury.
+Added: Subsequently, in July 2025, Sarepta announced that the FDA notified Sarepta that it may lift its voluntary pause on shipments of ELEVIDYS for ambulatory patients with DMD, and Sarepta resumed shipping to such patients immediately.
In March 2024, Italfarmaco S.p.A.
announced that the FDA approved Duvyzat (givinostat), a histone deacetylase inhibitor for the treatment of DMD in patients aged six years and older.
−Removed: In addition, several companies are developing gene therapies to treat DMD, including Pfizer Inc., Audentes Therapeutics, Inc.
+Added: In addition, several companies are developing gene therapies to treat DMD, including REGENXBIO Inc.
and Solid Biosciences Inc.
−Removed: Gene editing treatments that are in preclinical development are also being pursued by Vertex Pharmaceuticals, Inc.
−Removed: Additionally, Santhera Pharmaceuticals, in collaboration with ReveraGen Biopharma, Inc, is developing a steroid therapy for DMD, and Italfarmaco is developing a histone deacetylase (HDAC) inhibitor for DMD.
−Removed: FibroGen Inc.
−Removed: and Astellas Pharma Inc.
−Removed: are developing product candidates for the treatment of anemia, and Merck, Bristol-Myers Squibb Company and Disc Medicine are developing product candidates targeting diseases associated with MDS and myelofibrosis, including chronic anemia.
+Added: RNA-targeted treatments that are in clinical and preclinical development are also being pursued by several companies, including Avidity Biosciences, Inc., Wave Life Sciences Ltd., Dyne Therapeutics, Inc.
+Added: Edgewise Therapeutics, Inc.
+Added: is developing sevasemten, a myosin ATPase inhibitor, for both DMD and Becker muscular dystrophy.
+Added: Satellos Bioscience Inc.
+Added: is developing SAT-3277, an AAK1 inhibitor, for the treatment of DMD.
+Added: There are three therapies approved by FDA to treat ALS and its symptoms:
+Added: riluzole, an anti-glutamatergic agent;
+Added: edaravone, a free-radical scavenger;
+Added: and tofersen, an antisense oligonucleotide treatment of SOD1-ALS.
+Added: One additional therapeutic, sodium phenylbutyrate and taurursodiol, was approved under an accelerated pathway by the FDA but subsequently withdrawn from the market following negative results from a confirmatory Phase 3 trial.
+Added: To the best of our knowledge, there are no other companies developing product candidates to treat ALS by targeting skeletal muscle.
+Added: However, several companies are developing product candidates to treat ALS through other mechanisms, including Otsuka Pharmaceutical Co., Ltd., Novartis AG and Genervon Biopharmaceuticals LLC.
+Added: Kyntra Bio Inc.
+Added: (formerly FibroGen Inc.) and Astellas Pharma Inc.
+Added: are developing product candidates for the treatment of anemia, and Merck & Co.
+Added: Inc., or Merck, Bristol-Myers Squibb Company and Disc Medicine are developing product candidates targeting diseases associated with MDS and myelofibrosis, including chronic anemia.
Additionally, in April 2020, Merck and Bristol-Myers Squibb Company received FDA approval of its product, Reblozyl, for the treatment of anemia failing an erythropoiesis stimulating agent and requiring two or more red blood cell units over eight weeks in adult patients with very low- to intermediate-risk MDS with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis.
In June 2020, Merck further announced that the European Commission approved Reblozyl for the treatment of transfusion-dependent anemia in adult patients with MDS or beta thalassemia and in September 2020, Merck announced that Health Canada approved Reblozyl for the treatment of adult patients with red blood cell transfusion-dependent anemia associated with beta thalassemia.
−Removed: 2023, Bristol-Myers Squibb Company announced that the FDA approved Reblozyl for the treatment of anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk MDS who may require regular red blood cell transfusions.
+Added: In August 2023, Bristol-Myers Squibb Company announced that the FDA approved Reblozyl for the treatment of anemia without previous erythropoiesis stimulating agent use (ESA-naïve) in adult patients with very low- to intermediate-risk MDS who may require regular red blood cell transfusions.
In April 2024, Bristol-Myers Squibb Company further announced that the European Commission expanded approval of Reblozyl to include treatment of adult patients with and without ring sideroblasts with transfusion-dependent anemia due to lower-risk MDS.
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Pursuant to the terms of the Takeda Agreement, we received a $200.0 million upfront payment in February 2025.
−Removed: In addition to the upfront payment, we are entitled to receive up to an aggregate of (i) $370.0 million upon the achievement of specified development and commercial milestones and (ii) $740.0 million upon the achievement of specified sales milestones.
+Added: In July 2025, we announced that the first patient was dosed in the Phase 3 RENEW clinical trial of elritercept, which triggered a one-time $10.0 million milestone payment to us under the Takeda Agreement.
+Added: We received the $10.0 million payment for the achievement of this development milestone in August 2025.
+Added: In addition to the upfront payment and milestone payment, we are entitled to receive up to an aggregate of (i) $80.0 million upon the achievement of specified development milestones, (ii) $280.0 million upon the achievement of specified commercial milestone and (iii) $740.0 million upon the achievement of specified sales milestones.
If a licensed product is approved for marketing in the Takeda Territory, we will be entitled to receive royalty payments based on tiered increments of annual net sales in the Takeda Territory, with such percentage ranging from the low double-digits to high teens, subject to specified potential royalty reductions.
3 unchanged sentences
We may terminate the Takeda Agreement in its entirety in the event that Takeda or its affiliates bring a patent challenge.
−Removed: Either party may terminate the Agreement in its entirety (i) if the other party materially breaches the Takeda Agreement and fails to cure such breach;
+Added: Either party may terminate the Takeda Agreement in its entirety (i) if the other party materially breaches the Takeda Agreement and fails to cure such breach;
or (ii) upon the bankruptcy of the other party.
4 unchanged sentences
In addition, Hansoh will use commercially reasonable efforts to develop, obtain regulatory approval for, and commercialize licensed products in any region in the Hansoh Territory.
−Removed: Pursuant to the terms of the Hansoh Agreement, we received an upfront payment in 2022.
+Added: Pursuant to the terms of the Hansoh Agreement, we received a net $18.0 million upfront payment in January 2022.
In addition to the upfront payment and development milestones achieved to date, we are entitled to receive up to an aggregate of (i) $23.5 million upon the achievement of specified development milestones and (ii) $144.0 million upon the achievement of specified net sales thresholds for all licensed products in the Hansoh Territory.
If a licensed product is approved for marketing in the Hansoh Territory, we will be entitled to receive royalty payments based on a tiered percentage of annual net sales in each region within the Hansoh Territory, with such percentage ranging from the low double digit to high teens, subject to specified potential royalty reductions.
+Added: We recognized $3.0 million as revenue and $0.3 million in withholding tax upon the achievement of a development milestone related to the Hansoh Agreement on our consolidated statement of operations for the year ended December 31, 2024, and a receivable, net of withholding tax, on our consolidated balance sheet as of December 31, 2024.
+Added: Payment was received during the year ended December 31, 2025.
+Added: No milestones were achieved during the year ended December 31, 2025.
Hansoh’s obligation to pay royalties for a given licensed product in a given region in the Hansoh Territory will begin on the date of the first commercial sale for such licensed product in such region and continue until the latest of (i) ten years from the date of the first commercial sale for such licensed product in such region, (ii) the expiration of the last valid claim of certain licensed patents or joint patents, and (iii) expiration of regulatory exclusivity in such region.
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Additionally, we are required to pay a low-five digit to mid-five digit annual maintenance fee prior to the first commercial sale of our first product or process, a mid-five digit annual maintenance fee after the first commercial sale of our first product or process that is creditable against royalties, certain clinical and regulatory milestone payments for the first three products or indications to achieve such milestones, which milestone payments are $8.6 million in the aggregate, and certain commercial milestone payments for the first three products or indications to achieve such milestones, which milestone payments are $18.0 million in the aggregate.
−Removed: We made payments of $50,000 and $300,000 in 2020 and 2021, respectively, for the achievement of the clinical and regulatory milestones of (i) filing of an IND in the first country and (ii) the completion of a Phase 1 clinical trial, respectively.
+Added: We made payments of $50,000 and $300,000 in 2020 and 2021, respectively, for the achievement of the clinical and regulatory milestones of (i) filing of an IND in the first
+Added: country and (ii) the completion of a Phase 1 clinical trial, respectively.
We are also obligated to pay tiered royalties on net sales of licensed products ranging in the low-single digits to mid-single digits.
18 unchanged sentences
patents and 102 pending ex-U.S.
−Removed: applications, with expected expiry dates not earlier than between March 13, 2029 and January 9, 2046.
−Removed: Of these, 18 issued patents and 104 patent applications relate to cibotercept, KER-065 and elritercept, and 19 issued patents and 36 patent applications relate to other technologies, in each case as described in more detail below.
+Added: applications, with expected expiry dates not earlier than between March 13, 2029 and October 29, 2046.
+Added: Of these, 28 issued patents and 83 patent applications relate to rinvatercept and elritercept, and 40 issued patents and 46 patent applications relate to other technologies, in each case as described in more detail below.
Each of our pending international patent applications has been filed under the Patent Cooperation Treaty and has not yet entered any national jurisdictions.
4 unchanged sentences
Patent applications directed to our most advanced programs are summarized below.
−Removed: Cibotercept is a modified ActRIIB ligand trap that is designed to bind to different TGF-ß ligands that signal through a TGF-ß signaling pathway.
+Added: Rinvatercept is a ligand trap comprised of a modified ligand-binding domain derived from ActRIIA and ActRIIB that is designed to bind to different TGF-ß ligands that signal through a TGF-ß signaling pathway.
We own one issued U.S.
patent, 11 pending U.S.
−Removed: patent applications, two issued ex-U.S.
−Removed: patents and 29 pending ex-U.S.
−Removed: applications that contain claims or supporting disclosure directed to ActRIIB ligand traps and use thereof to treat muscle disease, bone disease, anemia, fibrosis, pulmonary hypertension, metabolic disease, thrombocytopenia, and neutropenia, among others.
−Removed: Any patents issuing from these applications will have expiration dates between January 11, 2039 and February 27, 2045, absent any patent term adjustments or extensions.
−Removed: KER-065 is a ligand trap comprised of a modified ligand-binding domain derived from ActRIIA and ActRIIB that is designed to bind to different TGF-ß ligands that signal through a TGF-ß signaling pathway.
−Removed: We own 15 pending U.S.
patent applications, and 26 pending ex-U.S.
applications that contain claims or supporting disclosure directed to ligand traps comprised of a modified ligand-binding domain derived from ActRIIA and ActRIIB and use thereof to treat muscle disease, bone disease, anemia, fibrosis, pulmonary hypertension, metabolic disease, thrombocytopenia, and neutropenia, among others.
−Removed: Any patents issuing from these applications will have expiration dates between March 19, 2041 and February 27, 2045, absent any patent term adjustments or extensions.
+Added: Any patents issuing from these applications will have expiration dates between March 19, 2041 and May 8, 2046, absent any patent term adjustments or extensions.
Elritercept is a modified ActRIIA ligand trap that is designed to bind to different TGF-ß ligands that signal through a TGF-ß signaling pathway.
−Removed: We own four issued U.S.
+Added: We own six issued U.S.
patents, 21 issued ex-U.S.
4 unchanged sentences
We plan to seek United States and international patent protection for a variety of additional technologies.
−Removed: We own two issued U.S.
−Removed: patents, four issued ex-U.S.
+Added: We own nine issued U.S.
+Added: patents, 18 issued ex-U.S.
patents, 21 pending U.S.
patent applications, and 46 pending ex-U.S.
−Removed: applications that contain claims or supporting disclosure directed to GDNF fusion polypeptides, ALK2 antibodies, crystal forms of an ALK2 inhibitor, ActRII chimera ligand traps, and uses of small molecule ALK2 inhibitors.
−Removed: Any patents issuing from these applications will have expiration dates between November 9, 2037 and January 9, 2046, absent any patent term adjustments or extensions.
+Added: applications that contain claims or supporting disclosure directed to cibotercept, GDNF fusion polypeptides, ALK2 antibodies, crystal forms of an ALK2 inhibitor, ActRII chimera ligand traps, and uses of small molecule ALK2 inhibitors.
+Added: Any patents issuing from these applications will have expiration dates between November 9, 2037 and November 9, 2043, absent any patent term adjustments or extensions.
Intellectual Property Protection
95 unchanged sentences
Under the goals and policies agreed to by the FDA under PDUFA, the FDA aims to review standard applications within ten months from the filing date, during which it will complete its initial review of a new molecular entity NDA or original BLA and respond to the applicant, or within six months from the filing date of a new molecular entity NDA or original BLA designated for priority review.
−Removed: In both standard and priority reviews, the FDA does not always meet its PDUFA goal dates, and the review process is
−Removed: often significantly extended by FDA requests for additional information or clarification.
+Added: In both standard and priority reviews, the FDA does not always meet its PDUFA goal dates, and the review process is often significantly extended by FDA requests for additional information or clarification.
The FDA reviews the application to determine, among other things, whether a product is safe and effective, or for a biologic, safe, pure and potent for its intended use, and whether the facility in which it is manufactured, processed, packed or held meets standards designed to assure and preserve the product’s identity, safety, strength, quality, potency and purity.
25 unchanged sentences
The orphan drug designation does not convey any advantage in, or shorten the duration of, the regulatory review or approval process.
−Removed: If a product that has orphan drug designation subsequently receives the first FDA approval 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, including a full NDA or BLA, to market the same product for the same indication for seven years, except in limited circumstances, such as a showing of clinical superiority to the product with orphan drug exclusivity or if the FDA finds that the holder of the orphan drug exclusivity has not shown that it can assure the availability of sufficient quantities of the orphan drug to meet the needs of patients with the disease or condition for which the drug was designated.
−Removed: Orphan drug exclusivity does not prevent the FDA from approving a different drug or biologic for the same disease or condition, or the
−Removed: same drug or biologic for a different disease or condition.
+Added: If a product that has orphan drug designation subsequently receives the first FDA approval 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, including a full NDA or BLA, to market the same product for the same indication for seven years, except in
+Added: limited circumstances, such as a showing of clinical superiority to the product with orphan drug exclusivity or if the FDA finds that the holder of the orphan drug exclusivity has not shown that it can assure the availability of sufficient quantities of the orphan drug to meet the needs of patients with the disease or condition for which the drug was designated.
+Added: Orphan drug exclusivity does not prevent the FDA from approving a different drug or biologic for the same disease or condition, or the same drug or biologic for a different disease or condition.
Among the other benefits of orphan drug designation are tax credits for certain research and a waiver of the application fee.
32 unchanged sentences
Such off-label uses are common across medical specialties.
−Removed: Physicians may believe that such off-label uses are the best treatment for many patients in varied circumstances.
+Added: Physicians may believe that such off-label uses are the best treatment for many patients
+Added: in varied circumstances.
The FDA does not regulate the behavior of physicians in their choice of treatments.
33 unchanged sentences
In all cases, clinical trials must be conducted in accordance with GCP and the applicable regulatory requirements and the ethical principles that have their origin in the Declaration of Helsinki.
−Removed: Medicines used in clinical trials, including ATMPs, must
−Removed: be manufactured in accordance with the guidelines on cGMP and in a GMP licensed facility, which can be subject to GMP inspections.
+Added: Medicines used in clinical trials, including ATMPs, must be manufactured in accordance with the guidelines on cGMP and in a GMP licensed facility, which can be subject to GMP inspections.
EU Review and Approval Process
26 unchanged sentences
Any authorization which is not followed by the actual placing of the medicinal product on the EU market (for a centralized MA) or on the market of the authorizing EU Member State within three years after authorization ceases to be valid (the so-called sunset clause).
+Added: Accelerated and Alternative Marketing Authorization Mechanisms
Innovative products that target an unmet medical need and are expected to be of major public health interest may be eligible for a number of expedited development and review programs, such as the Priority Medicines, or PRIME, scheme, which provides incentives similar to the breakthrough therapy designation in the U.S.
PRIME is a voluntary scheme aimed at enhancing the EMA’s support for the development of medicinal products that target unmet medical needs.
−Removed: Eligible products must target conditions for which there is an unmet medical need (there is no satisfactory method of diagnosis, prevention or
−Removed: treatment in the EU or, if there is, the new medicinal product will bring a major therapeutic advantage) and they must demonstrate the potential to address the unmet medical need by introducing new methods of therapy or improving existing ones.
+Added: Eligible products must target conditions for which there is an unmet medical need (there is no satisfactory method of diagnosis, prevention or treatment in the EU or, if there is, the new medicinal product will bring a major therapeutic advantage) and they must demonstrate the potential to address the unmet medical need by introducing new methods of therapy or improving existing ones.
Benefits accrue to sponsors of product candidates with PRIME designation, including but not limited to, early and proactive regulatory dialogue with the EMA, frequent discussions on clinical trial designs and other development program elements, and potentially accelerated MAA assessment once a dossier has been submitted.
30 unchanged sentences
Data exclusivity, if granted, prevents regulatory authorities in the EU from referencing the innovator’s data to assess a generic application or biosimilar application for eight years from the date of authorization of the innovative product, after which a generic or biosimilar MAA can be submitted, and the innovator’s data may be referenced.
−Removed: The market exclusivity period prevents a successful generic or biosimilar applicant from commercializing its product in the EU until 10 years have elapsed from the
−Removed: initial MA of the reference product in the EU.
+Added: The market exclusivity period prevents a successful generic or biosimilar applicant from commercializing its product in the EU until 10 years have elapsed from the initial MA of the reference product in the EU.
The overall ten-year period may, occasionally, be extended for a further year to a maximum of 11 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.
32 unchanged sentences
Such risk- minimization measures or post-authorization obligations may include additional safety monitoring, more frequent submission of PSURs, or the conduct of additional clinical trials or post-authorization safety studies.
+Added: Other EU Compliance Requirements
In the EU, the advertising and promotion of medicinal products are subject to both EU and EU Member States’ laws governing promotion of medicinal products, interactions with physicians and other healthcare professionals, misleading and comparative advertising and unfair commercial practices.
28 unchanged sentences
In addition, HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act, or HITECH, and their implementing regulations, impose certain requirements on HIPAA covered entities, which include certain healthcare providers, healthcare clearing houses and health plans, and individuals and entities that provide services on their behalf that involve individually identifiable health information, known as business associates, as well as their covered subcontractors, relating to the privacy, security and transmission of individually identifiable health information.
−Removed: federal Physician Payments Sunshine Act requires certain manufacturers of drugs, devices, biologics and medical supplies that are reimbursable under Medicare, Medicaid or the Children’s Health Insurance Program, with specific exceptions, to report annually to the Centers for Medicare & Medicaid Services, or CMS, information related to certain payments and other transfers of value made in the prior year to physicians (defined to include doctors, dentists, optometrists, podiatrists, and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners), and teaching hospitals, as well as ownership and investment interests held by such physicians and their immediate family members.
+Added: federal Physician Payments Sunshine Act requires certain manufacturers of drugs, devices, biologics and medical supplies that are reimbursable under Medicare, Medicaid or the Children’s Health Insurance Program, with specific
+Added: exceptions, to report annually to the Centers for Medicare & Medicaid Services, or CMS, information related to certain payments and other transfers of value made in the prior year to physicians (defined to include doctors, dentists, optometrists, podiatrists, and chiropractors), other healthcare professionals (such as physician assistants and nurse practitioners), and teaching hospitals, as well as ownership and investment interests held by such physicians and their immediate family members.
We are also subject to additional similar U.S.
−Removed: state and foreign law equivalents of each of the above federal laws, such as anti-kickback and false claims laws which may apply to sales or marketing arrangements and claims involving healthcare
−Removed: items or services reimbursed by non-governmental third-party payors, including private insurers, or that apply regardless of payor, state laws which require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the federal government, state and local laws which require pharmaceutical companies to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures, state laws which require the reporting of information related to drug pricing, state and local laws requiring the registration of pharmaceutical sales representatives, and state and foreign laws governing the privacy and security of health information which, in some cases, differ from each other in significant ways, and may not have the same effect, thus complicating compliance efforts.
+Added: state and foreign law equivalents of each of the above federal laws, such as anti-kickback and false claims laws which may apply to sales or marketing arrangements and claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers, or that apply regardless of payor, state and foreign laws which require pharmaceutical companies to comply with the pharmaceutical industry’s voluntary compliance guidelines and the relevant compliance guidance promulgated by the government, state and local laws which require pharmaceutical companies to report information related to payments and other transfers of value to physicians and other healthcare providers or marketing expenditures, state laws which require the reporting of information related to drug pricing, state and local laws requiring the registration of pharmaceutical sales representatives, and state and foreign laws governing the privacy and security of health information which, in some cases, differ from each other in significant ways, and may not have the same effect, thus complicating compliance efforts.
If our operations are found to be in violation of any of such laws or any other governmental regulations that apply, we may be subject to penalties, including, without limitation, significant civil, criminal and administrative penalties, damages, fines, exclusion from government-funded healthcare programs, such as Medicare and Medicaid or similar programs in other countries or jurisdictions, integrity oversight and reporting obligations to resolve allegations of non-compliance, disgorgement, imprisonment, contractual damages, reputational harm, diminished profits and the curtailment or restructuring of our operations.
13 unchanged sentences
government, state legislatures and foreign governments have continued implementing cost-containment programs, including price controls, restrictions on coverage and reimbursement and requirements for substitution of generic products.
+Added: For example, the U.S.
+Added: Department of Health and Human Services, or HHS, imposes rebates on many Medicare Part B and Medicare Part D products to penalize price increases that outpace inflation on an annual basis.
+Added: HHS has also been empowered to negotiate the price of certain single-source drugs that have been on the market for at least seven years covered under Medicare as part of the Medicare Drug Price Negotiation Program.
+Added: Each year up to 20 products will be selected by HHS for the Medicare Drug Price Negotiation Program.
+Added: Products subject to the Medicare Drug Price Negotiation Program are expected to experience a significant reduction in reimbursement from the Medicare program on a per unit basis.
Third-party payors are increasingly challenging the prices charged for medical products and services, examining the medical necessity and reviewing the cost effectiveness of pharmaceutical or biological products, medical devices and medical services, in addition to questioning safety and efficacy.
3 unchanged sentences
In the EU, pricing and reimbursement schemes vary widely from country to country.
−Removed: Some EU Member States may approve a specific price for a product, or they may instead adopt a system of direct or indirect controls on the profitability of the company placing the product on the market.
+Added: Some EU Member States may approve a specific price for a product, or they may instead adopt a system of direct or indirect controls on the profitability of the
+Added: company placing the product on the market.
Other EU Member States allow companies to fix their own prices for products but monitor and control prescription volumes and issue guidance to physicians to limit prescriptions.
4 unchanged sentences
The extent to which pricing and reimbursement decisions are influenced by the HTA of the specific medicinal product currently varies between EU Member States.
−Removed: In December 2021, Regulation No 2021/2282 on HTA, was adopted in the EU.
−Removed: This Regulation, which entered into application on January 12, 2025 and has a phased implementation, is intended to boost cooperation among EU Member States in assessing health technologies, including new medicinal products, and providing the
−Removed: basis for cooperation at EU level for joint clinical assessments in these areas.
−Removed: The Regulation permits EU Member States to use common HTA tools, methodologies, and procedures across the EU, working together in four main areas, including joint clinical assessment of the innovative health technologies with the most potential impact for patients, joint scientific consultations whereby developers can seek advice from HTA authorities, identification of emerging health technologies to identify promising technologies early, and continuing voluntary cooperation in other areas.
+Added: On January 12, 2025, Regulation No 2021/2282 on HTA entered into application through a phased implementation.
+Added: The Regulation initially applies to new active substances for oncology and ATMPs.
+Added: It will be expanded to orphan medicinal products in January 2028, and to all centrally authorized medicinal products as of 2030.
+Added: Select high-risk medical devices also came into scope in 2026.
+Added: The Regulation initially applies to new active substances for oncology and ATMPs.
+Added: It will be expanded to orphan medicinal products in January 2028, and to all centrally authorized medicinal products as of 2030.
+Added: Select high-risk medical devices also came into scope in 2026.
+Added: The HTA Regulation is intended to boost cooperation among EU Member States in assessing health technologies, including new medicinal products.
+Added: The Regulation establishes a framework for EU-level joint clinical assessments, joint scientific consultations and the early identification of emerging health technologies.
+Added: The Regulation permits EU Member States to use common HTA tools, methodologies, and procedures across the EU and requires them to rely on EU‑level joint clinical assessment reports for the clinical components of their national HTA evaluations.
Individual EU Member States continue to be responsible for assessing non-clinical (e.g., economic, social, ethical) aspects of health technologies, and making decisions on pricing and reimbursement.
5 unchanged sentences
Since its enactment, there have been judicial, Congressional and executive branch challenges and amendments to certain aspects of the ACA.
−Removed: For example, on August 16, 2022, the Inflation Reduction Act of 2022, or the IRA, was signed into law, which among other things, extends enhanced subsidies for individuals purchasing health insurance coverage in ACA marketplaces through plan year 2025.
−Removed: The IRA also eliminates the “donut hole” under the Medicare Part D program beginning in 2025 by significantly lowering the beneficiary maximum out-of-pocket cost and creating a new manufacturer discount program.
−Removed: It is unclear how any such challenges and additional healthcare reform measures of the second Trump administration will impact the ACA and our business.
+Added: For example, on July 4, 2025, the One Big Beautiful Bill Act, or the OBBBA, was signed into law, which narrowed access to ACA marketplace exchange enrollment and declined to extend the ACA enhanced advanced premium tax credits that expired at the end of 2025, which, among other provisions in the law, are anticipated to reduce the number of Americans with health insurance.
+Added: The OBBBA also is expected to reduce Medicaid spending and enrollment by implementing work requirements for some beneficiaries, capping state-directed payments, reducing federal funding and limiting provider taxes used to fund the program.
+Added: Congress is considering proposed legislation intended to further reduce healthcare costs with alternatives to replace the expired ACA subsidies.
+Added: We expect that additional U.S.
+Added: federal healthcare reform measures will be adopted in the future, any of which could limit the amounts that the U.S.
+Added: federal government will pay for healthcare products and services, which could result in reduced demand for our product candidates or additional pricing pressures.
Other legislative changes have been proposed and adopted since the ACA was enacted, including aggregate reductions of Medicare payments to providers of 2% per fiscal year and reduced payments to several types of Medicare providers.
−Removed: These reductions went into effect in April 2013 and, due to subsequent legislative amendments to the statute, will remain in effect through 2032, unless additional action is taken by Congress.
−Removed: Additionally, on March 11, 2021, the American Rescue Plan Act of 2021 was signed into law, which eliminates the statutory Medicaid drug rebate cap, currently set at 100% of a drug’s average manufacturer price, for single source and innovator multiple source drugs, beginning January 1, 2024.
−Removed: In addition, Congress is considering additional health reform measures as part of the other reform measures.
−Removed: Moreover, there has recently been heightened governmental scrutiny over the manner in which manufacturers set prices for their marketed products, which has resulted in several Congressional inquiries and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drug products.
−Removed: At the federal level, the IRA, among other things, (i) directs the U.S.
−Removed: Department of Health and Human Services, or HHS, to negotiate the price of certain high-expenditure, single-source drugs that have been on the market for at least 7 years and biologics that have been on the market for at least 7 years covered under Medicare, and subject drug manufacturers to civil monetary penalties and a potential excise tax by offering a price that is not equal to or less than the negotiated “maximum fair price” for such drugs and biologics under the law, or the“Medicare Drug Price Negotiation Program, and (ii) imposes rebates with respect to certain drugs and biologics covered under Medicare Part B or Medicare Part D to penalize price increases that outpace inflation.
−Removed: The IRA permits HHS to implement many of these provisions through guidance, as opposed to regulation, for the initial years.
−Removed: These provisions began to take effect progressively in fiscal year 2023, although they may be subject to legal challenges.
−Removed: On August 15, 2024, HHS announced the list agreed-upon price of the first ten drugs that will be were subject to price negotiations, although the Medicare Drug Price Negotiation Program is currently subject to legal challenges.
−Removed: On January 17, 2025, HHS selected fifteen additional products covered under Part D for price negotiation in 2025.
−Removed: Each year thereafter more Part B and Part D products will become subject to the Medicare Drug Price Negotiation Program.
+Added: These reductions went into effect in April 2013 and, due to subsequent legislative amendments to the statute, will remain in effect through 2032, unless additional action is taken by Congress In addition, Congress is considering additional health reform measures as part of the other reform measures.
+Added: The current administration is pursuing policies to reduce regulations and expenditures across government agencies including at HHS, the FDA, CMS and related agencies.
+Added: These actions, presently directed by executive orders or memoranda from the Office of Management and Budget, may propose policy changes that create additional uncertainty for our business.
+Added: For example, the current administration has announced agreements with several pharmaceutical companies that require the drug manufacturers to offer, through a direct-to-consumer platform, U.S.
+Added: patients and Medicaid programs prescription drug Most-Favored Nation pricing equal to or lower than those paid in other developed nations, with additional mandates for direct-to-patient discounts and repatriation of foreign revenues.
+Added: Other recent actions, for example, include (1) directing agencies to reduce agency workforce and cut programs;
+Added: (2) directing HHS and other agencies to lower prescription drug costs through a variety of initiatives, including by improving upon the Medicare Drug Price Negotiation Program and establishing Most-Favored-Nation pricing for pharmaceutical products;
+Added: (3) imposing tariffs on imported pharmaceutical products;
+Added: and (4) as part of the Make America Healthy Again Commission’s Strategy Report released in September 2025, working across government agencies to increase enforcement on direct-to-consumer pharmaceutical advertising.
+Added: Additionally, the current administration
+Added: recently called on Congress to enact "The Great Healthcare Plan," to codify and expand Most-Favored Nation pricing, lower government subsidies to private insurance companies, increase healthcare price transparency, expand pharmaceutical drugs available for over-the-counter purchase, and enact restrictions on pharmacy benefit manager payment methodologies, among other things.
+Added: These actions and policies may significantly reduce U.S.
+Added: drug prices, potentially impacting manufacturers’ global pricing strategies and profitability, while increasing their operational costs and compliance risks.
+Added: In June 2024, in Loper Bright Enterprises v.
+Added: Raimondo, the U.S.
+Added: Supreme Court greatly reduced judicial deference to regulatory agencies, which could increase successful legal challenges to federal regulations affecting our operations.
+Added: Congress may introduce and ultimately pass health care related legislation that could impact the drug approval process and make changes to the Medicare Drug Price Negotiation Program.
At the state level, legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.
+Added: We expect that additional state, federal, and foreign healthcare reform measures will be adopted in the future.
Additional Regulation
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Annual salary increases and incentive bonuses are based on merit and include individual and corporate performance factors.
−Removed: To encourage our employees to think like owners and share in the Company’s success, all employees are granted stock options.
+Added: To encourage our employees to think like owners and share in the Company’s success, all employees are granted stock options and/or restricted stock units.
All employees are eligible for health insurance, paid and unpaid leaves including paid parental leave, retirement plans with an employer contribution match and life and disability/accident coverage.
13 unchanged sentences
Our Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q, current reports on Form 8-K and amendments to those reports filed or furnished pursuant to Sections 13(a) and 15(d) of the Securities Exchange Act of 1934, as amended, or the Exchange Act, are available free of charge on our investor relations website as soon as reasonably practicable after we electronically file such material with, or furnish it to, the Securities and Exchange Commission, or the SEC.
−Removed: The SEC maintains an internet site
−Removed: that contains reports, proxy and information statements and other information.
+Added: The SEC maintains an internet site that contains reports, proxy and information statements and other information.
The address of the SEC’s website is www.sec.gov.
1 unchanged sentence
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.