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This discussion and analysis should be read in conjunction with our financial statements and the accompanying notes included in this report and the audited financial statements and accompanying notes included in our Annual Report on Form 10-K for the year ended December 31, 2024 filed with the SEC on March 4, 2025.
−Removed: Our financial results for the three and nine months ended September 30, 2024 are not necessarily indicative of results that may occur in future interim periods or for the full fiscal year.
+Added: Our financial results for the three months ended March 31, 2025 are not necessarily indicative of results that may occur in future interim periods or for the full fiscal year.
This Quarterly Report on Form 10-Q contains statements indicating expectations about future performance and other forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (Securities Act) and Section 21E of the Securities Exchange Act of 1934, as amended (Exchange Act), and the Private Securities Litigation Reform Act of 1995, that involve risks and uncertainties.
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the availability and sufficiency of our cash and capital resources and the need for additional capital;
−Removed: our ability to successfully identify and acquire or in-license products or companies, and to successfully transition assets to operate acquisitions;
+Added: our ability to successfully identify and acquire or in-license products or companies;
our operations and legal risks;
−Removed: the effectiveness of our cybersecurity risk management process;
−Removed: and our acquisition of certain assets comprising rights to GAVRETO (pralsetinib) in the US.
+Added: and the effectiveness of our cybersecurity risk management process.
You should not place undue reliance on these forward-looking statements.
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We have strategic development collaborations with MDACC to expand our evaluation of olutasidenib in AML and other hematologic cancers with IDH1 mutations, and with CONNECT to conduct a Phase 2 clinical trial to evaluate olutasidenib in combination with temozolomide in patients with HGG harboring an IDH1 mutation.
−Removed: We have a RIPK1 inhibitor program in clinical development with our partner Lilly.
−Removed: We also have product candidates in clinical development with partners BerGenBio and Daiichi.
−Removed: Reverse Stock Split
−Removed: We filed with the Secretary of State of the State of Delaware a certificate of amendment to our Amended and Restated Certificate of Incorporation, to effect a 1-for-10 reverse stock split, effective June 27, 2024.
−Removed: As a result of the reverse stock split, every ten issued and outstanding shares of our common stock were automatically combined into one issued and outstanding share of common stock.
−Removed: Accordingly, an amount equal to the par value of the decreased shares resulting from the reverse stock split was reclassified from common stock to additional paid-in capital on the condensed balance sheet and statement of changes in stockholders’ deficit.
−Removed: No fractional shares were issued in connection with the reverse stock split.
−Removed: Stockholders who otherwise would be entitled to receive fractional shares of common stock were entitled to receive the cash value equal to the fraction to which the stockholder would otherwise be entitled, multiplied by the closing price of the common stock as reported by Nasdaq on the last trading day prior to the effective date of the split.
−Removed: As a result of the reverse stock split, proportionate adjustments were made to the number of shares underlying (and as applicable, the exercise or conversion prices of) our outstanding equity awards and to the number of shares of common stock issuable under our equity incentive plans.
−Removed: The reverse stock split did not change the par value of our common stock, which remains $0.001, or the authorized number of shares of our common stock.
−Removed: All share amounts and per share amounts disclosed in this Quarterly Report on Form 10-Q have been adjusted to reflect the reverse stock split on a retroactive basis for all periods presented.
+Added: We also have a RIPK1 inhibitor program in clinical development with our partner Lilly.
Business Updates
TAVALISSE IN ITP
−Removed: For the nine months ended September 30, 2024, net product sales of TAVALISSE were $73.8 million, increased by $5.7 million or 8% compared to $68.1 million net product sales in the same period in 2023.
−Removed: The increase was primarily due to increased quantities sold, as well as increased price per bottle, partially offset by higher revenue reserves driven by increased government and private payor rebates.
+Added: For the three months ended March 31, 2025, net product sales of TAVALISSE were $28.5 million, increased by $7.4 million or 35% compared to $21.1 million net product sales in the same period in 2024.
+Added: The increase was primarily due to increased quantities sold and higher price per bottle, and partially due to lower revenue reserves rate.
REZLIDHIA in R/R AML with mIDH1
−Removed: For the nine months ended September 30, 2024, net product sales of REZLIDHIA were $15.6 million, increased by $8.9 million or 133% compared to $6.7 million net product sales in the same period in 2023.
−Removed: The increase was primarily due to increased quantities sold primarily driven by increased number of patients under therapy, partially offset by higher revenue reserves primarily due to increased government rebates.
+Added: For the three months ended March 31, 2025, net product sales of REZLIDHIA were $6.1 million, increased by $1.2 million or 25% compared to $4.9 million net product sales in the same period in 2024.
+Added: The increase was primarily due to increased quantities sold as well as higher price per bottle, partially offset by higher revenue reserves rate.
GAVRETO in metastatic RET fusion-positive NSCLC and advanced thyroid cancers
We began our commercialization and started recognizing revenue from product sales of GAVRETO in June 2024.
−Removed: For the nine months ended September 30, 2024, we recognized $9.0 million net product sales of GAVRETO.
−Removed: We believe GAVRETO is highly synergistic with our current product portfolio, and we expect to continue to leverage our existing commercial infrastructure to ensure current and newly prescribed GAVRETO patients have continued access to this important treatment option.
−Removed: We distribute and market GAVRETO for approved indications in RET fusion-positive NSCLC and advanced thyroid cancers.
−Removed: We acquired GAVRETO from Blueprint pursuant to an Asset Purchase Agreement entered into on February 22, 2024.
−Removed: Pursuant to the Asset Purchase Agreement, we purchased certain assets comprising the right to research, develop, manufacture and commercialize GAVRETO in the US from Blueprint.
−Removed: Under the terms of the agreement, we agreed to pay Blueprint a purchase price of $15.0 million, of which, $10.0 million was paid in July 2024 following our first commercial sale of GAVRETO at the end of June 2024, and an additional $5.0 million is payable on the first anniversary of the closing date of the agreement, subject to certain conditions.
−Removed: Blueprint is also eligible to receive up to $97.5 million in future commercial milestone payments and up to $5.0 million in future regulatory milestone payments, in addition to tiered royalties ranging from 10% to 30%.
−Removed: Simultaneously and in conjunction with entering into the Asset Purchase Agreement, we also entered into certain supporting agreements, including a customary transition agreement, pursuant to which, during the transition period, Blueprint will transition regulatory and distribution responsibility for GAVRETO to us.
−Removed: We also agreed to purchase certain drug product inventories from Blueprint under a Material Transfer Agreement, and received such inventories amounting to approximately $6.5 million during the nine months ended September 30, 2024.
−Removed: In October 2024, we issued a Dear Healthcare Provider Letter for GAVRETO related to a new safety signal identified in an ongoing Phase 3 clinical trial of pralsetinib in first-line treatment of RET fusion-positive, metastatic NSCLC patients, being conducted by Roche.
−Removed: The letter advises healthcare providers to apply certain measures to protect patient safety, including enhanced ongoing monitoring for signs and symptoms of infection as well as guidance for withholding treatment to patients in the presence of active infection.
−Removed: R289, an Oral IRAK 1/4 Inhibitor for LR-MDS
+Added: For the three months ended March 31, 2025, we recognized $9.0 million net product sales of GAVRETO.
+Added: We expect to continue to leverage our existing commercial infrastructure to ensure current and newly prescribed GAVRETO patients have continued access to this important treatment option.
+Added: R289, an Oral IRAK 1/4 Inhibitor for Lower-Risk MDS
We advanced the development of our dual IRAK 1/4 inhibitor program, following evaluation of single and multiple ascending doses of R289 in healthy subjects.
−Removed: The Phase 1b open-label, multicenter study evaluates the safety, tolerability and preliminary efficacy of R289 in patients with R/R lower-risk MDS.
−Removed: This Phase 1b study is expected to enroll approximately 40 patients (up to 30 participants in the dose escalation phase, and up to 10 participants in the dose expansion phase).
+Added: The ongoing Phase 1b open-label, multicenter study evaluates the safety, tolerability and preliminary efficacy of R289 in patients with R/R lower-risk MDS.
+Added: This Phase 1b study is expected to enroll approximately 86 patients (up to 36 patients in the dose escalation phase, up to 40 patients in the dose expansion phase, and 10 less heavily pre-treated patients in an exploratory cohort).
The primary objective of the study is safety, with secondary and exploratory objectives to assess preliminary efficacy and characterize the pharmacokinetic and pharmacodynamic profile of R289.
The safety and efficacy data from this Phase 1b study is intended to inform the recommended dose of R289 for further clinical evaluation in lower-risk MDS.
−Removed: Enrollment in the fifth dose level (500 mg / 250mg split dose) is underway.
−Removed: The initial data from the ongoing Phase 1b study will be presented at the 66 th American Society of Hematology (ASH) Annual
−Removed: Meeting and Exposition.
−Removed: Initial data indicate that R289 was generally well tolerated in a heavily pretreated LR-MDS patient population, the majority of whom were high transfusion burden at study entry.
−Removed: As of the data cutoff date (July 15, 2024), 14 of 19 patients were evaluable for efficacy;
−Removed: 4/11 patients receiving R289 doses ≥500 mg/daily achieved transfusion independence/hematologic improvement (HI-E) responses.
+Added: In December 2024, initial data from the dose escalation part of the Phase 1b study was presented at the 66 th American Society of Hematology (ASH) Annual Meeting and Exposition.
+Added: In summary, R289 was generally well tolerated with preliminary signs of efficacy in a heavily pretreated lower-risk MDS patient population, the majority of whom were high transfusion burden (HTB) at baseline.
+Added: Red blood cell (RBC)-transfusion independence (RBC-TI) ≥8 weeks was achieved by three patients (1 at 500 mg QD and 2 at 750 mg QD);
+Added: two HTB patients achieved RBC-TI >24 weeks.
+Added: The median duration of RBC-TI was 29 weeks (range 12.7-51.9 weeks).
+Added: One HTB patient receiving 500 mg QD achieved a minor hematologic improvement-erythroid (HI-E) response, with a 64% reduction in RBC transfusions compared to baseline.
+Added: The three patients that achieved RBC-TI had peak hemoglobin increases exceeding 2.0 g/dL compared to baseline.
+Added: To date, enrollment in the sixth dose level (500 mg twice daily) is ongoing.
+Added: The FDA granted R289 Orphan Drug designation for the treatment of myelodysplastic syndromes in January 2025 and Fast Track designation for the treatment of previously-treated transfusion dependent lower-risk myelodysplastic syndrome in November 2024.
Olutasidenib in AML, Other Hematologic Cancers and HGG
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Under the Strategic Collaboration Agreement, we will jointly lead the clinical development efforts with MDACC to evaluate the potential of olutasidenib to treat newly diagnosed and R/R patients with AML, higher-risk MDS, and advanced myeloproliferative neoplasms, in combination with other agents.
−Removed: The collaboration will also support the evaluation of olutasidenib as monotherapy in patients with IDH1 mutated clonal cytopenia of undermined significance (CCUS) and lower-risk MDS, as well as maintenance therapy following hematopoietic stem cell transplant.
+Added: The collaboration will also support the evaluation of olutasidenib as monotherapy in patients with IDH1 mutated clonal cytopenia of undetermined significance (CCUS) and lower-risk MDS, as well as maintenance therapy following hematopoietic stem cell transplant.
Under the Strategic Collaboration Agreement, we will provide MDACC the study materials and $15.0 million in time-based milestone payments as compensation for services to be provided for the studies, over the five-year collaboration term, unless terminated earlier as provided for in the agreement.
−Removed: Through September 30, 2024, we provided $2.0 million funding to MDACC.
−Removed: In early August 2024, MDACC opened enrollment for a Phase 1b/2 triplet therapy trial of decitabine and venetoclax in combination with olutasidenib in patients with IDH1-mutated AML.
−Removed: In September 2024, we announced the first patient was enrolled.
−Removed: This is the first trial in our multi-year strategic development collaboration with MDACC.
−Removed: The Phase 1b part of the trial seeks to determine the safety and tolerability and recommended Phase 2 dose of decitabine and venetoclax in combination with olutasidenib.
−Removed: The primary objective of the Phase 2 part of the trial is to determine the complete remission rate in both newly diagnosed and R/R patients.
−Removed: In January 2024, we announced our collaboration with Collaborative Network for Neuro-Oncology Clinical Trials (CONNECT), an international collaborative network of pediatric cancer centers, to conduct a Phase 2 clinical trial to evaluate olutasidenib in combination with temozolomide in patients with HGG harboring an IDH1 mutation (TarGet-D).
−Removed: Under the collaboration, CONNECT will include the olutasidenib treatment arm (TarGet-D) within CONNECT’s TarGet study, a molecularly guided Phase 2 umbrella clinical trial for HGG.
−Removed: In our sponsored arm, adolescents and young adult patients (<39 years old) with newly-diagnosed IDH1-mutation positive HGG will receive maintenance therapy with olutasidenib in combination with temozolomide for the first year after radiotherapy, followed by olutasidenib monotherapy for the second year.
−Removed: Under the collaboration, we will provide CONNECT with a funding up to $3.0 million and study material over the four-year collaboration.
−Removed: Collaboration and License Agreement with Kissei
−Removed: In September 2024, we announced the expansion of our relationship with Kissei, granting exclusive rights to develop and commercialize olutasidenib in all human diseases in Japan, Korea and Taiwan, pursuant to a collaboration and license agreement.
−Removed: Under the terms of the agreement, we received a one-time, non-creditable upfront cash payment of $10.0 million from Kissei, with the potential for up to an additional $152.5 million in development, regulatory and commercial milestone payments, and will receive mid twenty to lower thirty percent, tiered, escalated net sales-based payments for the supply of olutasidenib, subject to certain customary reductions and offsets.
−Removed: Pursuant to the agreement, Kissei is responsible for companion diagnostic development in Japan, for which we will share fifty percent of the costs incurred by Kissei, up to $3.0 million, which are creditable against future milestones and transfer price payments owed to us.
−Removed: We remain responsible for the manufacture and supply of olutasidenib for all development and commercialization activities under the agreement.
−Removed: Pursuant to the concurrently executed supply agreement, we will supply Kissei with bulk drug product for use under the collaboration and license agreement.
−Removed: W e in-licensed olutasidenib from Forma with exclusive, worldwide rights for its development, manufacturing and commercialization.
−Removed: Under the agreement with Forma, Forma is entitled to a certain portion of sublicensing revenue, which include, but are not limited to, upfront payments, milestone payments and royalties, that we receive from a third party sublicensee.
−Removed: Following the collaboration and license agreement with Kissei, Forma is entitled to a portion of the sublicensing revenue from Kissei , including $2.3 million upon our receipt of the $10.0 million upfront cash payment which we expect to pay in the fourth quarter of 2024.
+Added: Through March 31, 2025, we provided $5.3 million funding to MDACC.
+Added: T he four studies outlined in the multi-year strategic development alliance are open for enrollment.
+Added: In January 2024, we announced our collaboration with CONNECT, an international collaborative network of pediatric cancer centers, to conduct a Phase 2 clinical trial to evaluate olutasidenib in combination with temozolomide in patients with HGG harboring an IDH1 mutation.
+Added: Under the collaboration, CONNECT will include the olutasidenib treatment arm within CONNECT’s TarGet study, a molecularly guided Phase 2 umbrella clinical trial for HGG.
+Added: In our sponsored arm, TarGet-D, adolescents and young adult patients (<39 years old) with newly-diagnosed IDH1-mutation positive HGG will receive maintenance therapy with olutasidenib in combination with temozolomide for the first year after radiotherapy, followed by olutasidenib monotherapy for the second year.
+Added: Under the collaboration, we will provide CONNECT with funding up to $3.0 million and study material over the four-year collaboration.
+Added: The TarGet-D study is now open for enrollment.
+Added: Incrementally, we plan on initiating a Phase 2 clinical study in recurrent glioma in 2025.
+Added: This, in combination with our strategic collaborations with MDACC and CONNECT, are aimed to expand our olutasidenib pipeline development programs.
Global Strategic Partnership with Lilly
Lilly is continuing to advance ocadusertib (previously R552), an investigational, potent and selective RIPK1 inhibitor.
−Removed: Lilly has initiated the Phase 2a trial studying ocadusertib in adult patients with moderately to severely active rheumatoid arthritis.
−Removed: The Phase 2a enrollment of approximately 100 patients is advancing well, with preliminary analysis of the Phase 2a results anticipated in the first half of 2025.
+Added: Lilly has initiated the Phase 2 trial studying ocadusertib in adult patients with moderately to severely active rheumatoid arthritis.
+Added: The Phase 2a enrollment is advancing well, with preliminary analysis of results in April 2025.
RIPK1 is implicated in a broad range of key inflammatory cellular processes and plays a key role in tumor necrosis factor signaling, especially in the induction of pro-inflammatory necroptosis.
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Under the Lilly Agreement, we have the right to opt-out of co-funding the ocadusertib development activities in the US, Europe and Japan at two different specified times and as a result receive lesser royalties from sales.
−Removed: In September 2023, we provided the first opt-out notice to Lilly and our funding commitment was capped at a specified amount through April 1, 2024, as provided for in the Lilly Agreement, as amended in September 2023 .
−Removed: We provided $21.4 million funding to Lilly throughout the periods for our share for ocadusertib development costs incurred through April 1, 2024.
+Added: Following us providing the first opt-out notice to Lilly in September 2023, our cost share obligation for ocadusetib development ended on April 1, 2024.
+Added: We paid Lilly a total of $21.4 million for our share of development costs incurred through April 1, 2024.
Under the Lilly Agreement as amended, we have the right to opt-in to co-funding of ocadusertib development, upon us providing notice to Lilly within 30 days of certain events, as specified in the Lilly Agreement.
−Removed: If we decide to exercise our opt-in right, we will be required to continue to share in global development costs, and if we later exercise our second opt-out right (no later than April 1, 2025), our share in global development costs will be up to a specified cap through December 31, 2025, as provided for in the Lilly Agreement.
+Added: On April 30, 2025, we provided notice to Lilly of our decision not to exercise our opt-in right following our evaluation of certain events specified in the Lilly Agreement.
+Added: Following this notification, we are no longer obligated to share in any future global development costs, which resulted in the release of the $40.0 million remaining cost share liability currently on our condensed balance sheet.
Patent Infringement Lawsuit
−Removed: In June 2022, we received a notice letter regarding an Abbreviated New Drug Application (ANDA) submitted to the FDA by Annora Pharma Private Limited (Annora) requesting approval to market a generic version of TAVALISSE.
−Removed: In July 2022, we filed a lawsuit in the US District Court for the District of New Jersey against Annora and its subsidiaries for infringement of certain of our US patents.
−Removed: Litigation continues, and no trial date is currently set.
+Added: In March 2025, we entered into a settlement agreement with Annora Pharma Private Ltd., Hetero Labs Ltd., and Hetero USA, Inc.
+Added: (collectively, Annora), resolving patent litigation related to our product TAVALISSE.
+Added: The litigation resulted from submission by Annora of an Abbreviated New Drug Application (ANDA) to the FDA seeking approval to market a generic version of TAVALISSE in the US.
+Added: Under the terms of the settlement agreement, Annora will have a license to sell its generic product in the second quarter of 2032 or earlier under certain circumstances.
+Added: In accordance with the settlement agreement, the parties terminated all ongoing litigation between us and Annora regarding TAVALISSE patents pending in New Jersey.
For a more detailed discussion of this litigation matter, see Part II, Item 1, “Legal Proceedings” of this Quarterly Report on Form 10-Q.
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TAVALISSE/Fostamatinib in ITP
+Added: TAVALISSE overview
Chronic ITP affects an estimated 81,300 adult patients in the US.
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The study showed that 18% of patients receiving fostamatinib achieved a stable platelet response compared to none receiving a placebo control.
−Removed: In October 2016, we
−Removed: announced the results of the second FIT study, reporting that the response rate (16% in the treatment group, versus 4% in the placebo group) was consistent with the first study, although the difference was not statistically significant.
−Removed: In the ITP double-blind studies, the most commonly reported adverse reactions occurring in at least 5% of patients treated with TAVALISSE were diarrhea, hypertension, nausea, dizziness, increased alanine aminotransferase, increased aspartate aminotransferase, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.
+Added: In October 2016, we announced the results of the second FIT study, reporting that the response rate (16% in the treatment group, versus 4% in the placebo group) was consistent with the first study, although the difference was not statistically significant.
+Added: double-blind studies, the most commonly reported adverse reactions occurring in at least 5% of patients treated with TAVALISSE were diarrhea, hypertension, nausea, dizziness, increased alanine aminotransferase, increased aspartate aminotransferase, respiratory infection, rash, abdominal pain, fatigue, chest pain, and neutropenia.
Serious adverse drug reactions occurring in at least 1% of patients treated with TAVALISSE in the ITP double-blind studies were febrile neutropenia, diarrhea, pneumonia, and hypertensive crisis.
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In addition, a report describing the long-term safety and durable efficacy of TAVALISSE with up to five years of treatment was published in Therapeutic Advances in Hematology in 2021.
−Removed: The FDA granted our request for orphan drug designation for fostamatinib for the treatment of ITP in August 2015.
+Added: The FDA granted orphan drug designation for fostamatinib for the treatment of ITP in August 2015.
TAVALISSE was approved by the FDA in April 2018 for the treatment of ITP in adult patients who have had an insufficient response to a previous treatment, and successfully launched in the US in May 2018.
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Other products in the US that are approved by the FDA to increase platelet production through binding to TPO receptors on megakaryocyte precursors include PROMACTA ® (Novartis International AG), Nplate ® (Amgen, Inc.), DOPTELET ® (Swedish Orphan Biovitrum AB) and ALVAIZ TM ( Teva Pharmaceutical Industries Ltd .).
−Removed: In the longer term, we may eventually face competition from potential manufacturers of generic versions of our marketed products, including the proposed generic version of TAVALISSE that is the subject of an ANDA submitted to the FDA by Annora, which, if approved and allowed to enter the market, it could result in significant decreases in the revenue derived from sale of TAVALISSE and thereby materially harm our business and financial condition.
−Removed: Commercial activities, including sales and marketing
+Added: In the longer term, we may eventually face competition from potential manufacturers of generic versions of our marketed products, including the proposed generic version of TAVALISSE, if approved and allowed to enter the market, it could result in significant decreases in the revenue derived from sale of TAVALISSE and thereby materially harm our business and financial condition.
+Added: TAVALISSE Commercial activities, including sales and marketing
Our marketing and sales efforts are focused on hematologists and hematologist-oncologists in the US who manage chronic adult ITP patients.
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Our products are sold initially through third-party wholesale distribution and specialty pharmacy channels and group purchasing organizations before being ultimately prescribed to patients.
−Removed: facilitate our commercial activities in the US, we also enter into arrangements with various third parties, including advertising agencies, market research firms and other sales-support-related services as needed.
−Removed: We believe that our commercial team and distribution practices are adequate to ensure that our marketing efforts reach relevant customers and deliver our products to patients in a timely and compliant fashion.
+Added: To facilitate our commercial activities in the US, we also enter into arrangements with various third parties, including advertising agencies, market research firms and other sales-support-related services as needed.
+Added: We believe that our
+Added: commercial team and distribution practices are adequate to ensure that our marketing efforts reach relevant customers and deliver our products to patients in a timely and compliant fashion.
Also, to help ensure that all eligible patients in the US have appropriate access to our products, we have established a reimbursement and patient support program called Rigel OneCare ® (ROC).
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We have entered into various license and commercial agreements to commercialize fostamatinib globally as discussed below, but we retain the global rights to fostamatinib outside of the respective territories under such license and commercial agreements.
−Removed: Fostamatinib in EU and in the UK
+Added: Fostamatinib outside of the US
We have a commercialization license agreement with Grifols for exclusive rights to commercialize fostamatinib for human diseases, and non-exclusive rights to develop, fostamatinib in their territory.
Grifols territory includes EU, the UK, Turkey, the Middle East, North Africa and Russia (including Commonwealth of Independent States).
−Removed: In January 2020, the European Commission (EC) granted a centralized MA for fostamatinib (TAVLESSE) valid throughout the Europe and in the UK, after the departure of the UK from the EU, for the treatment of chronic ITP in adult patients who are refractory to other treatments.
+Added: In January 2020, the European Commission (EC) granted a centralized MA for fostamatinib (TAVLESSE) valid throughout the EU and which has been grandfathered in the UK, after the departure of the UK from the EU, for the treatment of chronic ITP in adult patients who are refractory to other treatments.
Grifols has launched TAVLESSE in the UK and certain countries in EU including Germany, France, Italy and Spain, and continues a phased rollout across the rest of EU.
−Removed: Fostamatinib in Asia
We have an exclusive license and supply agreement with Kissei to develop and commercialize fostamatinib in all current and potential indications in Japan, China, Taiwan and Korea.
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Kissei was granted orphan drug designation from the Japanese Ministry of Health, Labor and Welfare for R788 (fostamatinib) in chronic ITP in February 2020.
−Removed: In December 2022, Japan’s PMDA approved TAVALISSE for the treatment of chronic ITP, and in April 2023, Kissei launched TAVALISSE for chronic ITP in Japan.
−Removed: Fostamatinib in Canada/Israel
+Added: In December 2022, Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) approved TAVALISSE for the treatment of chronic ITP, and in April 2023, Kissei launched TAVALISSE for chronic ITP in Japan.
+Added: In January 2025, Kissei announced the Korean Ministry of Food and Drug Safety approved TAVALISSE for the treatment of thrombocytopenia in adult patients with chronic idiopathic thrombocytopenic purpura who have had an insufficient response to a previous treatment.
We have exclusive commercial and license agreements with Medison to commercialize fostamatinib in all potential indications in Canada and Israel.
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In August 2021, Medison Israel received the licenses for registrational approval from the Ministry of Health.
−Removed: Medison launched TAVALISSE in Canada and Israel.
−Removed: Fostamatinib in Latin America
−Removed: We have a commercial license agreement with Knight to commercialize fostamatinib for approved indications in Latin America, consisting of Mexico, Central and South America, and the Caribbean.
+Added: TAVALISSE is commercially available in Canada and Israel.
+Added: We have a commercial license agreement with Knight to exclusively commercialize fostamatinib for approved indications in Latin America, consisting of Mexico, Central and South America, and the Caribbean.
We are also responsible for the exclusive manufacture and supply of fostamatinib for all future development and commercialization activities under a commercial and supply agreement.
In August 2023, Knight submitted the MAA for regulatory approval in Mexico, Colombia and Brazil for fostamatinib for the treatment of adult patients with ITP who had insufficient response to a previous treatment.
−Removed: REZLIDHIA in R/R AML with mIDH1
+Added: In December 2024, Knight announced the approval of TAVALISSE in Mexico for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to a previous treatment.
+Added: REZLIDHIA/Olutasidenib in R/R AML with mIDH1
+Added: REZLIDHIA overview
mIDH1 alterations are seen in AML, MDS, glioma, chondrosarcoma, and intrahepatic cholangiocarcinoma.
−Removed: It is estimated that there are approximately 1,000 adult patients, a well-identified patient population, with mIDH1 R/R AML, part of an AML market estimated to have an incidence of approximately 20,000 cases in the US and an estimated 120,000 cases globally.
+Added: It is estimated that there are approximately 1,000 adult patients, a well-identified patient population, with mIDH1 R/R AML, part of an AML market estimated to have an incidence of approximately 22,000 cases in the US in 2025, and an estimated 120,000 cases globally.
Despite having approved treatment options for R/R AML patients who are mIDH1 positive, an unmet need remains.
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When mutated, IDH1 activity can promote blood malignancies and solid tumors.
−Removed: Olutasidenib was designated by the FDA as an orphan drug for the treatment of AML, which provides orphan drug market exclusivity from the time of marketing approval on December 1, 2022.
+Added: Olutasidenib was granted orphan drug designation by the FDA for the treatment of AML, which provides orphan drug market exclusivity from the time of marketing approval on December 1, 2022.
REZLIDHIA is designed to bind to and inhibit mIDH1 to reduce 2-hydroxyglutarate levels and restore normal cellular differentiation of myeloid cells.
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In 2022, certain milestones were met which entitled Forma to receive a $17.5 million milestone payments.
−Removed: No new milestone was met in 2023 and during the nine months ended September 30, 2024.
In addition, subject to the terms and conditions of the license and transition services agreement, Forma would be entitled to tiered royalty payments on net sales of licensed products at percentages ranging from low-teens to mid-thirties, as well as certain portions of our sublicensing revenue, subject to certain standard reductions and offsets.
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The most frequently reported treatment emergent adverse events were nausea, constipation, increased white blood cell count, decreased red blood cell count, pyrexia, febrile neutropenia, and fatigue.
−Removed: In November 2022, we announced the presentation of an updated interim analysis from the Phase 2 registrational trial of olutasidenib in patients with R/R AML demonstrated robust efficacy and safety results.
−Removed: The registrational cohort of the Phase 2 trial enrolled 153 patients with mIDH1 R/R AML who received olutasidenib monotherapy 150 mg twice daily.
−Removed: The efficacy evaluable population was 147 patients who received their first dose at least six months prior to the interim analysis cutoff date of June 18, 2021.
−Removed: The primary endpoint was a CR/CRh defined
−Removed: as less than 5% blasts in the bone marrow, no evidence of disease, and partial recovery of peripheral blood counts (platelets >50,000/microliter and absolute neutrophil count >500/microliter).
−Removed: The results from the updated interim analysis of patients with mIDH1 R/R AML demonstrated a 35% CR+CRh rate with a median duration of 25.9 months.
−Removed: The ORR a secondary end point, was 48%, and was defined as the rate of CR, CRh, CR with incomplete blood count recovery (Cri), partial remission (which required recovery of neutrophil and platelet counts consistent with a CR), or MLFS.
−Removed: Olutasidenib was effective in a broad range of patients including those with prior high-intensity chemotherapy and/or post-venetoclax.
−Removed: The abstract concluded that the observed activity is clinically meaningful and represents a therapeutic advance in the treatment of this patient population.
−Removed: In this pivotal cohort, olutasidenib was well tolerated with an adverse event profile largely characteristic of symptoms or conditions experienced by patients undergoing treatment for AML or of the underlying disease itself.
In January 2023, we announced that REZLIDHIA has been added by the National Comprehensive Cancer Network (NCCN) to the latest NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for AML.
7 unchanged sentences
Further, the available data support the use of REZLIDHIA as monotherapy in R/R AML patients who have failed intensive chemotherapy or venetoclax plus hypomethylating agents combination therapy.
−Removed: In June 2023, we announced presentation of data from an analysis from the Phase 2 study of REZLIDHIA in patients with mIDH1 AML who were previously treated with venetoclax.
−Removed: Data was featured in a poster presentation at the European Hematology Association (EHA) 2023 Hybrid Congress.
−Removed: The data support REZLIDHIA induced durable remissions in patients with mIDH1 AML in this poor-prognosis patient population who were R/R to venetoclax-based treatment.
In April 2024, we announced a peer-reviewed publication in Leukemia & Lymphoma on data from an analysis of the Phase 2 study evaluating REZLIDHIA in patients with mIDH1 AML who are R/R to prior venetoclax-based regimens.
6 unchanged sentences
REZLIDHIA was also effective in achieving remission in patients with mIDH1 R/R AML and served as a bridging strategy towards potentially curative allogeneic transplantation in a substantial subset of these previously ineligible patients.
−Removed: Additionally, REZLIDHIA was well tolerated in a subset of patients with post-myeloproliferative neoplasms (MPN) mlDH1 AML, a patient population often associated with poor responses to available therapies.
+Added: Additionally, REZLIDHIA was well tolerated in a subset of patients with myeloproliferative neoplasms mlDH1 AML, a patient population often associated with poor responses to available therapies.
Competitive landscape for REZLIDHIA
There is currently one other product approved in the US for patients with IDH1 mutation.
−Removed: The FDA granted approval to TIBSOVO ® (ivosidenib), an oral targeted IDH1 mutation inhibitor, (i) in July 2018, for adult patients with R/R AML with a susceptible IDH1 mutation, (ii) in May 2019, for newly diagnosed AML with a susceptible IDH1
−Removed: mutation who are at least 75 years old or who have comorbidities that preclude use of intensive induction chemotherapy, (iii) in August 2021, for adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation as detected by an FDA-approved test, (iv) in May 2022, in combination with azacitidine (azacitidine for injection) for newly diagnosed AML with a susceptible IDH1 mutation, as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy, and (v) in October 2023, for adult patients with R/R MDS with a susceptible IDH1 mutation, as detected by an FDA-approved test.
+Added: The FDA granted approval to TIBSOVO ® (ivosidenib), an oral targeted IDH1 mutation inhibitor, (i) in July 2018, for adult patients with R/R AML with a susceptible IDH1 mutation, (ii) in May 2019, for newly diagnosed AML with a susceptible IDH1 mutation who are at least 75 years old or who have comorbidities that preclude use of intensive induction chemotherapy, (iii) in August 2021, for adult patients with previously treated, locally advanced or metastatic cholangiocarcinoma with an IDH1 mutation as detected by an FDA-approved test, (iv) in May 2022, in combination with azacitidine (azacitidine for injection) for newly diagnosed AML with a susceptible IDH1 mutation, as detected by an FDA-approved test in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy, and (v) in October 2023, for adult patients with R/R MDS with a susceptible IDH1 mutation, as detected by an FDA-approved test.
In addition, some clinicians may utilize non-targeted treatments for patients with mIDH1 R/R AML, including use of venetoclax combinations, hypomethylating agents, other chemotherapy regimens, or investigational agents that may be available to them.
−Removed: Commercial activities, including sales and marketing
+Added: REZLIDHIA commercial activities, including sales and marketing
We believe REZLIDHIA is highly synergistic with our existing hematology-oncology focused commercial and medical affairs infrastructure.
Our commercial effort focuses on growing awareness of REZLIDHIA within key institutions, and among targeted HCPs who manage patients with R/R AML with mIDH1.
−Removed: We retain the global rights, excluding Asian countries as discussed below, to develop and commercialize olutasidenib for all indications, and we are currently exploring other ex-US partnership opportunities.
−Removed: Olutasidenib in Asia
−Removed: In September 2024, we entered into a collaboration and license agreement with Kissei, pursuant to which Kissei was granted exclusive rights to develop and commercialize olutasidenib in all human diseases in Japan, Korea and Taiwan.
+Added: We retain the global rights, excluding certain geographies as discussed below, to develop and commercialize olutasidenib for all indications, and we are currently exploring other ex-US partnership opportunities.
+Added: Olutasidenib outside of the US
+Added: In September 2024, we entered into a collaboration and license agreement with Kissei, pursuant to which Kissei was granted exclusive rights to develop and commercialize olutasidenib in all human diseases in Japan, Korea and
Kissei will initially seek approval for REZLIDHIA in Japan for R/R mIDH1 AML and will be responsible for conducting clinical studies as required by the Japanese PMDA.
We remain responsible for the manufacture and supply of olutasidenib for all development and commercialization activities and will supply Kissei with bulk drug product for use under the license and supply agreements.
−Removed: Under the license and services agreement with Forma as discussed in “Note 5, In-licensing and Acquisition”, Forma is entitled to a certain portion of sublicensing revenue, which include, but are not limited to upfront payment, milestone payments and royalties, that we receive from a third party sublicensee.
−Removed: Following the license agreement with Kissei as discussed above, Forma is entitled to a portion of the sublicensing revenue we receive from Kissei.
−Removed: GAVRETO in metastatic RET fusion-positive NSCLC and advanced thyroid cancers
+Added: In November 2024, we entered into a commercial license agreement with Dr.
+Added: Reddy’s for an exclusive license to develop and commercialize olutasidenib in Dr.
+Added: Reddy’s territory which includes Latin America, South Africa, India, certain countries in the CIS, Southeast Asia region and North Africa, Australia, and New Zealand.
+Added: We are responsible for the exclusive manufacture and supply of olutasidenib for all future development and commercialization activities under a supply agreement.
+Added: Under the license and services agreement with Forma, Forma is entitled to a certain portion of sublicensing revenue, which include, but are not limited to upfront payment, milestone payments and royalties, that we receive from a third party sublicensee.
+Added: Following the license agreements with Kissei and Dr.
+Added: Reddy’s as discussed above, Forma is entitled to a portion of the sublicensing revenue we receive from Kissei and Dr.
+Added: GAVRETO/Pralsetinib in metastatic RET fusion-positive NSCLC and advanced thyroid cancers
+Added: GAVRETO overview
RET is a receptor tyrosine kinase that activates multiple downstream pathways involved in cell proliferation and survival.
13 unchanged sentences
Discussions with the FDA regarding confirmatory requirements are ongoing.
−Removed: On June 24, 2024, we announced the completion of the transfer to us of the NDA for GAVRETO, and GAVRETO is commercially available from us in the US by prescription beginning June 27, 2024.
−Removed: GAVRETO was co-marketed by Blueprint and Genentech, a member of Roche Group (Roche), to patients in the US since September 2020 pursuant to a collaboration agreement between Blueprint and Roche, which was terminated effective in February 2024.
−Removed: The patent portfolio covering pralsetinib contains patents and patent applications directed to compositions of matter for pralsetinib, including solid forms, formulations, and methods of use and manufacture.
−Removed: Pralsetinib is covered as a composition of matter in a US issued patent that has an expiration date in November 2036 and subject to potential extensions.
−Removed: Patents that have been issued or are expected to be issued covering pralsetinib will have statutory expiration dates between 2036 and 2041.
+Added: In June 2024, we announced the completion of the transfer to us of the NDA for GAVRETO, and GAVRETO became commercially available from us in the US by prescription.
+Added: GAVRETO was co-marketed by Blueprint and Genentech, a member of Roche Group (Roche), to patients in the US since September 2020 pursuant to a collaboration agreement between Blueprint and Roche, which agreement was terminated effective in February 2024.
The FDA granted GAVRETO new chemical entity exclusivity until September 2025 and orphan drug exclusivity until September 2027 with respect to the approval for treatment of adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA-approved test.
1 unchanged sentence
Competitive landscape for GAVRETO
−Removed: GAVRETO faces competition for RET fusion-positive NSCLC and advanced thyroid cancers from Lilly’s selpercatinib.
+Added: GAVRETO faces competition for RET fusion-positive NSCLC and advanced thyroid cancers from Lilly’s selpercatinib (Retevmo®) .
In addition, other commercially available therapies used to treat RET fusion-positive NSCLC include cabozantanib and platinum-based chemotherapy regimens with or without pembrolizumab, atezolizumab, nivolumab/ipilumumab, cemiplimab or tremelimumab-durvalumab.
−Removed: Pralsetinib may also face competition from other drug candidates in development for RET-altered cancers, as well as multi-kinase inhibitors with RET activity being evaluated in clinical trials.
−Removed: Commercial activities, including sales and marketing
+Added: GAVRETO may also face competition from other drug candidates in development for RET-altered cancers, as well as multi-kinase inhibitors with RET activity being evaluated in clinical trials.
+Added: GAVRETO commercial activities, including sales and marketing
We began our commercialization and started recognizing revenue from product sales of GAVRETO in June 2024.
12 unchanged sentences
We advanced the development of our IRAK 1/4 inhibitor program, following evaluation of single and multiple ascending doses of R289, a new pro-drug formulation of R835, in healthy subjects.
−Removed: In January 2022, we received clearance from the FDA to initiate a Phase 1b open-label, multicenter study to evaluate the safety, tolerability and preliminary efficacy of R289 in patients with R/R lower-risk MDS.
+Added: In January 2022, we initiated a Phase 1b open-label, multicenter study to evaluate the safety, tolerability and preliminary efficacy of R289 in patients with R/R lower-risk MDS.
In December 2022, we announced the dosing of the first patient.
−Removed: This Phase 1b study is expected to enroll approximately 40 patients (up to 30 participants in the dose escalation phase, and up to 10 participants in the dose expansion phase).
+Added: This Phase 1b study is expected to enroll approximately 86 patients (up to 36 patients in the dose escalation phase, up to 40 patients in the dose expansion phase, and 10 less heavily pre-treated lower-risk-MDS patients in an exploratory cohort).
The primary objective of the study is safety, with secondary and exploratory objectives to assess preliminary efficacy and characterize the pharmacokinetic and pharmacodynamic profile of R289.
The safety and efficacy data from this Phase 1b study is intended to inform the recommended dose of R289 for further clinical evaluation in lower-risk MDS.
−Removed: Enrollment in the fifth dose level (500 mg / 250mg split dose) is underway.
−Removed: The initial data from the ongoing Phase 1b study will be presented at the 66 th ASH Annual Meeting and Exposition.
−Removed: Initial data indicate that R289 was generally well tolerated in a heavily pretreated LR-MDS patient population, the majority of whom were high transfusion burden at study entry.
−Removed: As of the data cutoff date (July 15, 2024), 14 of 19 patients were evaluable for efficacy;
−Removed: 4/11 patients receiving R289 doses ≥500 mg/daily achieved transfusion independence/hematologic improvement (HI-E) responses.
−Removed: Olutasidenib for mIDH1 AML
+Added: In December 2024, initial data from the dose escalation part of the Phase 1b study was presented at the 66 th ASH Annual Meeting and Exposition.
+Added: In summary, R289 was generally well tolerated with preliminary signs of efficacy in this heavily pretreated lower-risk MDS patient population, the majority of whom were HTB at baseline.
+Added: RBC-TI ≥8 weeks was achieved by three patients (1 at 500 mg QD and 2 at 750 mg QD);
+Added: two HTB patients achieved RBC-TI >24 weeks.
+Added: The median duration of RBC-TI was 29 weeks (range 12.7-51.9 weeks).
+Added: One HTB patient receiving 500 mg QD achieved a minor HI-E response, with a 64% reduction in RBC transfusions compared to baseline.
+Added: The three patients that achieved RBC-TI had peak hemoglobin increases exceeding 2.0 g/dL compared to baseline.
+Added: To date, the enrollment in the sixth dose level (500 mg twice daily) is ongoing.
+Added: R289 was granted Fast Track designation by the FDA for the treatment of patients with previously-treated transfusion dependent lower-risk MDS in November 2024.
+Added: In January 2025, the FDA granted R289 orphan drug designation for the treatment of myelodysplastic syndromes .
+Added: Olutasidenib for mIDH1 AML , Other Hematologic Cancers and HGG
We have a strategic collaboration agreement with MDACC to expand our evaluation of olutasidenib in AML and other hematologic cancers with IDH1 mutations.
Under such collaboration agreement, we will jointly lead the clinical development efforts with MDACC to evaluate the potential of olutasidenib to treat newly diagnosed and R/R patients with AML, higher-risk MDS, and advanced myeloproliferative neoplasms, in combination with other agents.
−Removed: The collaboration will also support the evaluation of olutasidenib as monotherapy in patients with IDH1 mutated clonal cytopenia of undermined significance and lower-risk MDS, as well as maintenance therapy following hematopoietic stem cell transplant.
−Removed: In early August 2024, MDACC opened enrollment for a Phase 1b/2 triplet therapy trial of decitabine and venetoclax in combination with olutasidenib in patients with IDH1-mutated AML.
−Removed: In September 2024, we announced the first patient was enrolled.
−Removed: This is the first trial in our multi-year strategic development collaboration with MDACC.
+Added: The collaboration will also support the evaluation of olutasidenib as monotherapy in patients with IDH1 mutated CCUS and lower-risk MDS, as well as maintenance therapy following hematopoietic stem cell transplant.
+Added: The four studies outlined in the multi-year strategic development alliance are open for enrollment.
+Added: The four studies include, (i) a Phase 1b/2 triplet therapy trial of decitabine and venetoclax in combination with olutasidenib in patients with mIDH1 AML.
The Phase 1b part of the trial seeks to determine the safety and tolerability and recommended Phase 2 dose of decitabine and venetoclax in combination with olutasidenib.
The primary objective of the Phase 2 part of the trial is to determine the complete remission rate in both newly diagnosed and R/R patients;
+Added: (ii) a Phase 2 study in patients with IDH1-mutated CCUS, lower-risk MDS and chronic myelomonocytic leukemia (CMML);
+Added: (iii) a Phase 1/2 study of olutasidenib maintenance therapy following an allogeneic stem cell transplant for patients with IDH1-mutated myeloid malignancies;
+Added: and (iv) a Phase 2 study of olutasidenib in combination with hypomethylating agents (HMA) in patients with mIDH1 higher-risk myelodysplastic syndrome (HR-MDS)/ CMML or advanced myeloproliferative neoplasms .
+Added: We also have a collaboration with CONNECT to conduct a Phase 2 clinical trial to evaluate olutasidenib in combination with temozolomide in patients with HGG harboring an IDH1 mutation.
+Added: Under the collaboration, CONNECT will include the olutasidenib treatment arm within CONNECT’s TarGet study, a molecularly guided Phase 2 umbrella clinical trial for HGG.
+Added: In our sponsored arm, TarGet-D, adolescents and young adult patients (<39 years old) with newly-diagnosed IDH1-mutation positive HGG will receive maintenance therapy with olutasidenib in combination with temozolomide for the first year after radiotherapy, followed by olutasidenib monotherapy for the second year.
+Added: The TarGet-D study is now opened for enrollment.
Partnered Clinical Programs
Ocadusertib – Lilly
−Removed: Lilly is continuing to advance ocadusertib (previously R552) and has initiated the Phase 2a trial studying ocadusertib in adult patients with moderately to severely active rheumatoid arthritis.
−Removed: The Phase 2a enrollment of approximately 100 patients is advancing well, with preliminary analysis of the Phase 2a results anticipated in the first half of 2025.
+Added: Lilly is continuing to advance ocadusertib (previously R552) and has initiated the Phase 2 trial studying ocadusertib in adult patients with moderately to severely active rheumatoid arthritis.
+Added: The Phase 2a enrollment is advancing well, with preliminary analysis of results in April 2025.
RIPK1 is implicated in a broad range of key inflammatory cellular processes and plays a key role in tumor necrosis factor signaling, especially in the induction of pro-inflammatory necroptosis.
The program also includes RIPK1 compounds that cross the blood-brain barrier (CNS-penetrants) to address neurodegenerative diseases such as Alzheimer’s disease and amyotrophic lateral sclerosis.
−Removed: Bemcentinib – BerGenBio
−Removed: We have an exclusive, worldwide research, development and commercialization agreement with BerGenBio for our investigational AXL receptor tyrosine kinase inhibitor, R428 (now referred to as bemcentinib (BGB324)).
−Removed: In February 2023, BerGenBio announced positive data from the Phase 2 trial of bemcentinib in combination with pembrolizumab in patients with second-line NSCLC.
−Removed: The treatment with bemcentinib in combination with pembrolizumab demonstrated long survival benefit and sustained disease control, particularly in patients with AXL TPS > 5, substantiating the relevance of AXL as a target and bemcentinib’s selective inhibition capabilities in NSCLC.
−Removed: Also in March 2023, BerGenBio announced its first patient dosed in a Phase 1b/2a trial evaluating bemcentinib in first-line NSCLC patients harboring STK11 mutations .
−Removed: In March 2024, BerGenBio announced initiation of the Phase 2a portion of the study following a positive decision by the Data and Safety Monitoring Board (DSMB) following review of the Phase 1b safety data.
−Removed: In July 2024, BerGenBio announced that the DSMB confirmed acceptable safety at the highest dose tested in Phase 1b and recommended that under the study protocol, no additional patients will be required for Phase
−Removed: In October 2024, BerGenBio announced the preliminary safety data from dose escalation Phase 1b in first-line NSCLC patients.
−Removed: Milademetan – Daiichi
−Removed: DS-3032 is an investigational oral selective inhibitor of the MDM2 protein investigated by Daiichi in three Phase 1 clinical trials for solid and hematological malignancies including AML, acute lymphocytic leukemia, chronic myeloid leukemia in blast phase, lymphoma and MDS.
−Removed: Preliminary safety and efficacy data from a Phase 1 trial of DS-3032 suggests that DS-3032 may be a promising treatment for hematological malignancies including R/R AML and high-risk MDS.
−Removed: In September 2020, worldwide rights to DS-3032 (milademetan) were out-licensed from Daiichi to Rain Oncology Inc.
−Removed: In January 2024, Pathos Al, Inc.
−Removed: (Pathos) completed the acquisition of Rain.
−Removed: Pathos indicated that it has continued interest in further developing milademetan for cancer patients using its propriety PathOS Platform.
+Added: Other Partnered Programs
+Added: We also have product candidates in clinical development with BerGenBio for the development and commercialization of AXL receptor tyrosine kinase inhibitor, R428 (now referred to as bemcentinib (BGB324)), and with Daiichi to pursue research related to MDM2 inhibitor, DS-3032 (now referred as melademetan).
+Added: The worldwide rights to melademetan were out-licensed from Daiichi to Rain Oncology Inc.
+Added: (Rain), which is now Pathos Al, Inc.
+Added: (Pathos) after Pathos completed the acquisition of Rain in January 2024.
Research, Preclinical and Clinical Development Programs
−Removed: We have retained selected experts in drug discovery and preclinical development to leverage our existing proprietary collection of inhibitors, small-molecule compound libraries and large database of associated phenotypic and biochemical assay results of therapeutic interest.
−Removed: We maintain leading expertise on specific areas of operation such as inhibition of SYK, IRAK 1/4, RIPK1 and mIDH1 kinases to assist clinical development and commercial affairs, as well as to expand and explore additional opportunities for such inhibitors in the clinical space.
+Added: We maintain expertise in drug development to leverage our existing proprietary collection of inhibitors, small-molecule compound libraries and large database of associated phenotypic and biochemical assay results of therapeutic interest.
+Added: We also maintain leading expertise on specific areas of operation such as inhibition of SYK, IRAK 1/4 and RIPK1 kinases and mIDH1 to assist clinical development and commercial affairs, as well as to expand and explore additional opportunities for such inhibitors in the clinical space.
Our preclinical operations involve collaborations with clinical research organizations, leading investigators from universities and research organizations around the world, and strategic collaborations with other pharmaceutical companies.
−Removed: We have experts in drug development to design and implement clinical trials and to analyze the data derived from these trials.
+Added: We have experts in clinical development to design and implement clinical trials and to analyze the data derived from these trials.
The clinical development group possesses expertise in project management and regulatory affairs.
−Removed: We work with external clinical research organizations with expertise in managing clinical trials, drug formulation, and the manufacture of clinical trial supplies to support our drug development efforts.
+Added: We work with external clinical research organizations with expertise in managing clinical trials, drug formulation, and the manufacture of clinical trial supplies to support our clinical development efforts.
We also have strategic development collaborations with MDACC and CONNECT to conduct evaluation of olutasidenib in AML, other hematologic cancers and glioma.
+Added: Incrementally, we plan on initiating a Phase 2 clinical study in recurrent glioma in 2025.
+Added: This, in combination with our strategic collaborations with MDACC and CONNECT, are aimed to expand our olutasidenib pipeline development programs.
Commercialization and Sponsored Research and License Agreements
1 unchanged sentence
Results of Operations
−Removed: Three Months Ended September 30,
−Removed: Nine Months Ended September 30,
+Added: Three Months Ended March 31,
(in thousands)
1 unchanged sentence
Contract revenues from collaborations
−Removed: Government contracts
Total revenues
The following table summarizes the percentages of revenues from each of our customers who individually accounted for 10% or more of the total net product sales and revenues from collaborations:
−Removed: Three Months Ended September 30,
−Removed: Nine Months Ended September 30,
+Added: Three Months Ended March 31,
McKesson Corporation
(formerly ASD Healthcare)
+Added: Optime Care, Inc.
Cardinal Health, Inc.
2 unchanged sentences
Typically, our first quarter net sales are impacted by the first quarter reimbursement issues such as the resetting of co-pays and the Medicare donut hole.
−Removed: TAVALISSE net product sales for the three and nine months ended September 30, 2024 were $26.3 million and $73.8 million, respectively, increased by 8% for each period, compared to $24.5 million and $68.1 million net product sales for the three and nine months ended September 30, 2023, respectively.
−Removed: The increase was primarily due to increased quantities sold, as well as increased price per bottle, partially offset by higher revenue reserves driven by increased government and private payor rebates.
−Removed: REZLIDHIA net product sales in the three and nine months ended September 30, 2024 were $5.5 million and $15.6 million, respectively, increased by 107% and 133% compared to $2.7 million and $6.7 million net product sales for the three and nine months ended September 30, 2023, respectively.
−Removed: The increase was primarily due to increased quantities sold primarily driven by increased number of patients under therapy, partially offset by the higher revenue reserves primarily due to increased government rebates.
+Added: TAVALISSE net product sales for the three months ended March 31, 2025 were $28.5 million, increased by 35% compared to $21.1 million for the same period in 2024.
+Added: The increase was primarily due to increased quantities sold and higher price per bottle, and partially due to lower revenue reserves rate.
+Added: REZLIDHIA net product sales in the three months ended March 31, 2025 were $6.1 million, increased by 25% compared to $4.9 million for the same period in 2024.
+Added: The increase was primarily due to increased quantities sold as well as higher price per bottle, partially offset by higher revenue reserves rate.
Following the commercialization of GAVRETO in June 2024, we started recognizing revenue from shipments to our distributors.
−Removed: For the three and nine months ended September 30, 2024, we recognized $7.1 million and $9.0 million, respectively, of GAVRETO net product sales.
−Removed: Contract revenues from collaborations in the three and nine months ended September 30, 2024 consisted primarily of revenue from Kissei of $13.0 million and $17.5 million, respectively, $10.0 million of which was the upfront fee we received from sublicensing olutasidenib, and the remainder was related to the delivery of drug supplies.
−Removed: In addition, we recognized revenue from Grifols of $3.3 million and $5.5 million in the three and nine months ended September 30, 2024, respectively, related to earned royalty and delivery of drug supplies.
−Removed: Contract revenues from collaborations in the three and nine months ended September 30, 2023 consisted primarily of revenue from Grifols of $0.8 million and $5.1 million, respectively, related to earned royalty and delivery of drug supplies.
−Removed: No government contract revenue was recognized during the three and nine months ended September 30, 2024, and three months ended September 30, 2023.
−Removed: Government contract revenue in the nine months ended September 30, 2023 was related to the income we recognized upon achievement of certain milestones from the award granted to us by the DOD.
+Added: For the three months ended March 31, 2025, we recognized $9.0 million of GAVRETO net product sales.
+Added: Contract revenues from collaborations in the three months ended March 31, 2025 comprised primarily of revenue from Grifols of $4.7 million related to earned royalty and delivery of drug supplies, as well as revenue from Kissei of $4.6 million related to a milestone payment and delivery of drug supplies.
+Added: Contract revenues from collaborations in the three months ended March 31, 2024 comprised primarily of revenue from Kissei of $2.3 million related to the delivery of drug supplies, and revenue from Grifols of $1.1 million related to earned royalty.
We expect that our future revenues to include product sales of our existing commercial products and product sales from new commercial products we may have in the future.
1 unchanged sentence
In addition, our future revenues may include payments from our existing and new collaboration partners and government grants.
−Removed: As of September 30, 2024, we had $1.4 million of deferred revenue relating to our collaboration agreement with Kissei which we will recognize as revenue upon satisfaction of our remaining performance obligations.
+Added: As of March 31, 2025, we had $1.4 million of deferred revenue relating to our collaboration agreement with Kissei which we will recognize as revenue upon satisfaction of our remaining performance obligations.
Cost of Product Sales
−Removed: Three Months Ended September 30,
−Removed: Nine Months Ended September 30,
+Added: Three Months Ended March 31,
(in thousands)
1 unchanged sentence
The cost of product sales includes the cost of inventories sold to our customers and to our collaborative partners.
−Removed: Inventories sold for the periods presented include inventory quantities acquired or produced prior to the FDA approval of the product, and do not reflect the full cost of the inventories sold, since such costs incurred prior to FDA approval were previously expensed and charged to research and development expense.
−Removed: In particular, we still utilize active pharmaceutical ingredients with zero cost for our TAVALISSE inventories, which we expect to make use of for the next 1 to 2 years.
+Added: Certain inventories sold for the periods presented include inventory quantities acquired or produced prior to the FDA approval of the product, and do not reflect the full cost of the inventories sold, since such costs incurred prior to FDA approval were previously expensed and charged to research and development expense.
+Added: In particular, we still utilize API with zero cost for our TAVALISSE inventories, which we expect to make use of for the next 12 months.
As such, we recognize lower cost of product sales in the periods where we sell inventory quantities acquired or produced prior to the FDA approval of the product.
As we acquire or produce more FDA approved inventory quantities in the future, our inventory cost in the balance sheet and cost of product sales will reflect the full cost of acquiring or producing such products.
−Removed: Cost of product sales also includes amortization of intangible assets acquired from in-licensing or acquisition of commercialized products, as well as sublicensing revenue fees and royalty expense.
+Added: We rely and will continue to rely on certain third parties, including those located outside the US to manufacture our products.
+Added: The imposition or threat of imposition of trade policies, tariffs (including retaliatory tariffs), taxes and other cross boarder operations could result in higher cost of product sales.
Cost of product sales may also include reserves for potential excess, dated or obsolete inventories, estimated based upon assumptions about future demand and market conditions as well as product shelf lives.
−Removed: The increase in cost of product sales in the three and nine months ended September 30, 2024 compared to the same periods in 2023 was primarily due to increased royalty expense and sublicensing revenue fee of $3.4 million and $4.5 million, respectively, and increased amortization of intangible assets of $0.3 million and $0.7 million, respectively.
−Removed: Further, the increase in cost of product sales was also partly due to increased product sales, and increased delivery of drug supplies pursuant to our supply agreements with our collaborative partners.
+Added: Cost of product sales also includes amortization of intangible assets acquired from in-licensing or acquisition of commercialized products, as well as sublicensing revenue fees and royalty expense.
+Added: The increase in cost of product sales in the three months ended March 31, 2025 compared to the same period in 2024 was primarily due to increased royalties and amortization expense of $1.2 million.
+Added: In addition, cost of product sales also increased by $1.2 million due to the increase in product sales and delivery of drug supplies to our collaboration partners.
Research and Development Expense
−Removed: Three Months Ended September 30,
−Removed: Nine Months Ended September 30,
+Added: Three Months Ended March 31,
(in thousands)
1 unchanged sentence
Stock-based compensation expense included in research and development expense
−Removed: Research and development expense in the three months ended September 30, 2024 decreased compared to the same period in 2023.
−Removed: The decrease in research and development expense primarily due to timing of progress of trial activities of our IRAK 1/4 inhibitor program of $1.7 million and in other various research and development costs of $0.2 million, were partially offset by the increase in our research and development expense related to our ongoing clinical development programs for olutasidenib of approximately $1.6 million.
−Removed: The decrease in research and development expense in the nine months ended September 30, 2024 compared to the same period in 2023 was partly due to decreased clinical trial related expenses of $2.8 million due to the progress of trial activities of our IRAK 1/4 inhibitor program, as well as $1.8 million due to timing of trial activities of our completed Phase 3 clinical trials of fostamatinib in patients with COVID-19 and wAIHA.
−Removed: Other research and development expense including allocated facilities and laboratory costs also decreased by $1.0 million.
−Removed: These decreases
−Removed: were partially offset by the increase in our research and development activities of $2.0 million related to our ongoing clinical development programs for olutasidenib .
+Added: The increase in research and development expense in the three months ended March 31, 2025 compared to the same period in 2024 was primarily driven by the increase in clinical trial related expenses of $1.9 million due to the timing of study progress activities on our ongoing IRAK 1/4 inhibitor program, and clinical development programs for olutasidenib.
+Added: In addition, other various research and development expenses also increased by $0.6 million.
Our research and development expenditures include costs related to preclinical and clinical trials, scientific personnel, supplies, equipment, consultants, sponsored research, stock-based compensation, and allocated facility costs.
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We do not track fully burdened research and development costs separately for each of our drug candidates.
−Removed: We review our research and development expense by focusing on three categories:
−Removed: research, development, and other.
Our research team is focused on identifying and evaluating product candidates in our focused range of therapeutic indications that can be developed into small molecule therapeutics in our own proprietary programs or with potential collaborative partners.
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Our development group leads the implementation of our clinical and regulatory strategies and prioritizes disease indications in which our compounds may be studied in clinical trials.
−Removed: “Development” expenses relate primarily to clinical trials, personnel expenses, costs related to our regulatory filings, lab supplies and fees to third-party research consultants.
+Added: “Development” expenses relate primarily to clinical trials, personnel expenses, costs related to our regulatory filings, lab supplies and fees to third-
+Added: party research consultants.
“Other” expenses primarily consist of allocated facilities costs and allocated stock-based compensation expense relating to personnel in research and development groups.
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The following table presents our total research and development expense by category (in thousands).
−Removed: Three Months Ended September 30,
−Removed: Nine Months Ended September 30,
+Added: Three Months Ended March 31,
From January 1, 2007*
−Removed: to September 30, 2024
+Added: to March 31, 2025
* We started tracking research and development expense by category on January 1, 2007.
−Removed: “Other” expenses in the three months ended September 30, 2024 and 2023 consisted of allocated facilities costs of $0.1 million for each of the periods, and allocated stock-based compensation expense of $0.3 million and $0.4 million, respectively.
−Removed: For the nine months ended September 30, 2024 and 2023, allocated facilities costs was $0.4 million and $0.9 million, respectively, and allocated stock-based compensation expense was $1.2 million and $1.7 million, respectively.
+Added: “Other” expenses in the three months ended March 31, 2025 and 2024 consisted of allocated facilities costs of $0.1 million and $0.1 million, respectively, and allocated stock-based compensation expense of $0.9 million and $0.7 million, respectively.
Selling, General and Administrative Expense
−Removed: Three Months Ended September 30,
−Removed: Nine Months Ended September 30,
+Added: Three Months Ended March 31,
(in thousands)
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Stock-based compensation expense included in selling, general and administrative expense
−Removed: The increase in selling, general and administrative expense in the three months ended September 30, 2024 compared to the same period in 2023 was primarily due to increases in commercial related expenses of $1.9 million and in personnel-related costs and stock-based compensation expense of $1.5 million, partially offset by the decrease in other various sales, general and administrative costs of $1.2 million.
−Removed: The increase in selling, general and administrative expense in the nine months ended September 30, 2024 compared to the same period in 2023 was primarily due to increases of $5.9 million in personnel-related costs and stock-based compensation expense, and in commercial related expenses of $1.7 million, partially offset by the decrease in other various sales, general and administrative costs primarily due to lower facilities cost of $3.0 million.
−Removed: We expect to incur significant selling, general and administrative expenses, as we expect our commercial related expenses to increase as we continue to expand our commercial activities for TAVALISSE, REZLIDHIA, and GAVRETO.
+Added: The decrease in selling, general and administrative expense in the three months ended March 31, 2025 compared to the same period in 2024 was primarily due to decreased stock-based compensation expense of $2.0 million primarily from our performance-based stock awards, and decreased other various sales, general and administrative expenses of $0.4 million.
+Added: These decreases were partially offset by increased personnel-related costs of $1.5 million and increased commercial related expenses of $0.2 million.
+Added: We expect to incur significant selling, general and administrative expenses, and expect our commercial related expenses to increase as we continue to expand our commercial activities.
We continue to deploy resources to enable our field-based employees to engage with healthcare providers.
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Interest Income and Interest Expense
−Removed: Three Months Ended September 30,
−Removed: September 30,
+Added: Three Months Ended March 31,
(in thousands)
1 unchanged sentence
Interest expense
−Removed: Interest income is related to our interest-bearing cash and investment balances.
−Removed: The decrease in interest income in the three and nine months ended September 30, 2024 compared to the same periods in 2023 was primarily driven by lower investment balances throughout the respective periods, partially offset by higher interest rates.
−Removed: Interest expense was comprised primarily of interest on the outstanding term loan with MidCap.
−Removed: Increased interest expense in the three and nine months ended September 30, 2024 compared to the same periods in 2023 was primarily due to higher interest on our term loan with Midcap.
−Removed: Also contributing to higher interest expense for the nine months ended September 30, 2024 compared to the same period in 2023 was the higher outstanding principal balance of the term loan throughout the respective periods as the Tranche 5 ($20.0 million) term loan was funded in March 2023.
+Added: Interest income comprised interest on our cash and investment balances.
+Added: Interest expense comprised interest on our outstanding term loans with MidCap.
+Added: Provision for income Tax
+Added: Three Months Ended March 31,
+Added: (in thousands)
+Added: Provision for income taxes
+Added: The quarterly provision for or benefit from income taxes is based on applying the estimated annual effective tax rate to the year-to-date pre-tax income (loss), adjusted for any discrete items.
+Added: We update our estimate of our annual effective tax rate at the end of each quarterly period.
+Added: For the three months ended March 31, 2025, we recorded $0.1 million of provision for income tax primarily related to estimated state taxes.
+Added: We do not expect to owe federal income tax due to sufficient net operating loss carryforwards that were generated prior to the enactment of the Tax Cuts and Jobs Act, as well as significant research and development credit carryforwards.
+Added: We continue to record a full valuation allowance on our deferred tax assets considering our cumulative losses in prior years.
+Added: For the three months ended March 31, 2024, we did not record a provision for income taxes due to our pre-tax book loss.
Critical Accounting Policies and Use of Estimates
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Our critical accounting estimates and significant accounting policies are described in “Note 1 – Description of Business and Summary of Significant Accounting Policies” to our “Notes to Financial Statements” contained in Part II, Item 8, “Financial Statements and Supplementary Data” of our Annual Report on Form 10-K for the year ended December 31, 2024.
−Removed: There have been no material changes to these accounting policies except for the accounting consideration related to the Asset Purchase Agreement with Blueprint as discussed in “Note 5 – In-licensing and Acquisition” to our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q.
+Added: There have been no material changes to the accounting policies described in our Annual Report on Form 10-K for the year ended December 31, 2024.
Recent Accounting Pronouncements
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Liquidity and Capital Resources
−Removed: As of September 30, 2024 and December 31, 2023, we had approximately $61.1 million and $56.9 million, respectively, in cash, cash equivalents and short-term investments.
+Added: As of March 31, 2025 and December 31, 2024, we had approximately $77.1 million and $77.3 million, respectively, in cash, cash equivalents and short-term investments.
We continue to maintain investment portfolios primarily in money market funds, US treasury bills, government-sponsored enterprise securities, corporate bonds and commercial paper.
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Following summarizes our cash flow activity for the periods presented:
−Removed: Nine Months Ended September 30,
+Added: Three Months Ended March 31,
(in thousands)
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Financing activities
−Removed: Net increase in cash and cash equivalents
−Removed: Net cash provided by operating activities for the nine months ended September 30, 2024 was primarily due the proceeds from sales of our products, and cash received from our collaboration partners including the $10.0 million upfront payment from Kissei pursuant to the collaboration and license agreement, partially offset by the payments of operating expenses.
−Removed: Net cash provided by operating activities for the nine months ended September 30, 2023 was primarily due to the proceeds from sales of our products, cash received from our collaboration partners including the $20.0 million regulatory milestone payment from Kissei received in January 2023, as well as cash received from government grants, partially offset by payments of operating expenses.
−Removed: Net cash provided by investing activities for the nine months ended September 30, 2024 comprised primarily of net maturities of short-term investments of $15.4 million, partially offset by payments for acquisition of intangible assets of $0.4 million.
−Removed: Net cash provided by investing activities for the nine months ended September 30, 2023 comprised net maturities of short-term investments of $17.4 million and proceeds from sale of property and equipment of $0.1 million, partially offset by the payment of milestone obligations to Forma recorded as intangible assets of $15.0 million.
−Removed: Net cash used in financing activities for the nine months ended September 30, 2024 comprised payment of the closing purchase price to Blueprint of $10.0 million and cost share payments to a collaboration partner of $3.6 million, partially offset by the net proceeds from issuance of common stock upon exercise of stock options and participation in the Purchase Plan of $0.5 million.
−Removed: Net cash provided by financing activities for the nine months ended September 30, 2023 comprised net cash proceeds from term loan financing of $20.0 million (Tranche 5) and proceeds from exercise of stock options and participation in the Purchase Plan of $0.6 million, partially offset by our cost share payments to collaboration partner of $2.6 million.
+Added: Net decrease in cash, cash equivalents and restricted cash
+Added: Net cash used in operating activities for the three months ended March 31, 2025 comprised net cash outflows from changes in assets and liabilities of $16.1 million, partially offset by net cash inflows from net income adjusted for non-cash items of $15.2 million.
+Added: Cash outflows from changes in assets and liabilities during the three months ended March 31, 2025 were primarily due to timing of advance payments to our contract manufacturers and strategic development collaboration partner, as well as timing of payments of salaries and annual cash bonuses.
+Added: Net cash used in operating activities for the three months ended March 31, 2024 comprised net cash outflows from changes in assets and liabilities of $2.1 million, and cash outflows from net loss adjusted for non-cash items of $2.9 million.
+Added: Cash outflows from changes in assets and liabilities during the three months ended March 31, 2024 were primarily due to timing of inventory build-up and advance payments to our contract manufacturers, as well as timing of payments of salaries and annual cash bonuses .
+Added: Net cash used in investing activities for the three months ended March 31, 2025 comprised net purchases of short-term investments of $10.6 million.
+Added: Net cash provided by investing activities for the three months ended March 31, 2024 comprised primarily of net maturities of short-term investments of $0.5 million, partially offset by payment for acquisition of intangible assets of $0.1 million .
+Added: Net cash provided by financing activities for the three months ended March 31, 2025 comprised proceeds from issuance of common stock upon exercise of stock options of $0.5 million.
+Added: Net cash used in operating activities for the three months ended March 31, 2024 comprised cost share payments to a collaboration partner of $2.6 million, partially offset by the net proceeds from issuance of common stock upon exercise of stock options of $0.1 million.
We believe that our existing capital resources will be sufficient to support our current and projected funding requirements, including the continued commercialization of our products, through at least the next 12 months from this Form 10-Q filing date.
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Under our existing collaboration agreements that we entered in the ordinary course of business, we received or may be entitled to receive upfront cash payments, payments contingent upon specified events achieved by such partners and royalties on any net sales of products sold by such partners under the agreements.
−Removed: As of September 30, 2024, total future contingent payments to us under our existing agreements could exceed $1.4 billion if all potential product candidates achieved all of the payment triggering events under all of our current agreements.
+Added: As of March 31, 2025, total future contingent payments to us under our existing agreements with our collaboration partners was approximately $1.5 billion, if all potential product candidates achieved all of the payment triggering events under all of our current agreements.
This estimated future contingent amount does not include any estimated royalties that could be due to us if the partners successfully commercialize any of the licensed products.
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Pursuant to such Open Market Sale Agreement, we may sell from time to time, through Jefferies, shares of our common stock in sales deemed to be “at-the-market offerings” as defined in Rule 415 under the Securities Act, subject to conditions specified in the Open Market Sale Agreement, including maintaining an effective registration statement covering the sale of shares under the Open Market Sale Agreement.
−Removed: As of September 30, 2024, we have not sold any shares of common stock under such Open Market Sale Agreement.
−Removed: We had a shelf registration statement (the Prior Registration Statement) filed with the SEC that expired on August 3, 2024.
−Removed: The Prior Registration Statement included a base prospectus registering the offering, issuance, and sale by us of up to $250.0 million in the aggregate of the securities identified from time to time in one or more offerings, including the $100.0 million of shares of our common stock that may be offered, issued and sold under the Open Market Sale Agreement.
−Removed: On August 2, 2024, we filed a new shelf registration statement (the New Registration Statement) with the SEC to replace the Prior Registration Statement.
−Removed: The New Registration Statement was declared effective on August 9, 2024 by the SEC.
−Removed: The New Registration Statement includes a base prospectus to register the offering, issuance and sale by us of up to $250.0 million in the aggregate of securities identified from time to time in one or more offerings, including up to $100.0 million of shares of our common stock that may be offered, issued and sold under the Open Market Sale Agreement.
−Removed: We have a Credit Agreement with MidCap that provides for $60.0 million term loan credit facility, which was fully funded as of September 30, 2024.
+Added: We have an active Registration Statement filed with the SEC, which registered, among other securities, a base prospectus which covers the offering, issuance, and sale by us of up to $250.0 million in the aggregate of the securities identified from time to time in one or more offerings, which include the $100.0 million of shares of our common stock that may be offered, issued and sold under the Open Market Sale Agreement.
+Added: As of March 31, 2025, we have not sold any shares of common stock under such Open Market Sale Agreement.
+Added: We have a Credit Agreement with MidCap that provides for $60.0 million term loan credit facility, which was fully funded as of March 31, 2025.
Our operations will require significant additional funding in the foreseeable future.
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Material Cash Requirements
−Removed: We conduct our commercial activities and research and development programs internally and through third parties that include, among others, arrangements with vendors, consultants, contract research organizations (CROs) and universities.
−Removed: We have contractual arrangements with these parties, however our contracts with them are cancelable generally on reasonable notice within one year and our obligations under these contracts are primarily based on services performed.
−Removed: We do not have any purchase commitments under any collaboration arrangements.
−Removed: We have agreements with certain clinical research organizations to conduct our clinical trials including our strategic development collaborations with MDACC and CONNECT, as well as with third parties relative to our commercialization of our products.
−Removed: The timing of payments for any amounts owed under the respective agreements will depend on various factors including, but not limited to, patient enrollment and other progress of the clinical trials, and various activities related to commercialization.
−Removed: We expect that we will continue to enter into contracts in the normal course of business with various third parties who support our clinical trials, support our preclinical research studies, and provide other services related to our operating purposes as well as our commercialization of our products.
+Added: We conduct our commercial activities and research and development programs internally and with third parties that include, among others, arrangements with vendors, consultants, contract research organizations (CROs) and universities.
+Added: Our contract arrangements with these third parties are generally cancellable on reasonable notice, and our obligations under such arrangements are generally based on services performed.
+Added: We have agreements with certain clinical research organizations to conduct our clinical trials including our strategic development collaborations with MDACC and CONNECT.
+Added: The timing of payments for any amounts owed under the respective agreements depends on various factors including, but not limited to, patient enrollment and other progress of the clinical trials.
We can terminate these agreements at any time, and if terminated, we would not be liable for the full amount of the respective agreements.
1 unchanged sentence
In addition, these agreements may, from time to time, be subjected to amendments as a result of any change orders executed by the parties.
−Removed: As discussed in detail in “Note 4 – Sponsored Research, License Agreements and Government Contracts” of our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q, pursuant to the amended Lilly Agreement, and us providing the first opt-out notice to Lilly on September 29, 2023, we were responsible for funding the development costs for ocadusertib (previously R552) in the US, Europe, and Japan, through April 1, 2024, capped at a specified amount.
−Removed: Lilly billed us $21.4 million of the funding development costs
−Removed: incurred through April 1, 2024 and the amount was fully paid as of September 30, 2024.
−Removed: Although currently we are no longer obligated to pay Lilly for our share in the ocadusertib development cost incurred subsequent to April 1, 2024, under the Lilly Agreement, we have the right to opt-in to co-funding the ocadusertib development, upon us providing notice to Lilly within 30 days of certain events, as specified in the Lilly Agreement.
−Removed: If we decide to exercise our opt-in right, we will be required to continue to share in global development costs, and if we later exercise our second opt-out right (no later than April 1, 2025), our share in global development costs will be up to a specified cap through December 31, 2025, as provided for in the Lilly Agreement.
−Removed: As discussed in detail in “Note 5 – In-licensing and Acquisition” of our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q, pursuant to an Asset Purchase Agreement with Blueprint entered in February 2024, we agreed to pay Blueprint a purchase price of $15.0 million, of which, $10.0 million was paid in July 2024, and an additional $5.0 million is payable on the first anniversary of the closing date of the agreement, subject to certain conditions.
−Removed: Blueprint is also eligible to receive up to $97.5 million in future commercial milestone payments, up to $5.0 million in future regulatory milestone payments, and tiered royalty payments ranging from 10% to 30%.
−Removed: Additionally, as discussed in detail in “Note 5 – In-licensing and Acquisition” of our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q, pursuant to our license and transition services agreement, Forma is entitled to potential development and regulatory milestone payments of up to $67.5 million, commercial milestone payments of up to $165.5 million, and tiered royalty payments on net sales as well as certain portion of sublicensing revenue.
−Removed: Certain milestones were met in 2022 which entitled Forma to receive $17.5 million milestone payments that was paid in the fourth quarter of 2022 and first quarter of 2023.
−Removed: No additional milestone was met through September 30, 2024.
−Removed: As discussed in “Note 4 – Sponsored Research, License Agreements and Government Contracts” of our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q, Forma is entitled to a portion of the sublicensing revenue we receive from Kissei related to our collaboration and license agreement of olutasidenib.
−Removed: With the receipt of the upfront payment of $10.0 million from Kissei in September 2024, Forma is entitled to $2.3 million sublicense revenue fee, which we expect to pay in the fourth quarter of 2024.
−Removed: As of September 30, 2024, we have a contractual commitment related to our leased facilities of $0.5 million and the amount is payable within 12 months.
−Removed: See “Note 11 – Leases” to our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q for further discussions of our leases.
−Removed: As discussed above, we have a contractual commitment with respect to our credit facility with MidCap, and as of September 30, 2024, the outstanding principal amount of the loan was $60.0 million.
−Removed: As discussed in detail in “Note 10 – Debt” to our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q, the term loans mature on September 1, 2027, and the interest-only period is through October 1, 2025.
−Removed: The term loans bear interest equal to the sum of one-month SOFR plus an adjustment of 0.11448%, subject to a 4.00% applicable floor, plus applicable margin of 6.50%.
−Removed: A final payment fee of 4.25% of principal is due at maturity date.
−Removed: As of September 30, 2024, no principal payments are due within 12 months.
−Removed: As of September 30, 2024, future interest calculated using the base interest rate as per the amended Credit Agreement, and the final fee payments associated with the credit facility amounted to $16.0 million, of which, approximately $6.6 million is payable within 12 months.
+Added: We expect to continue entering into contracts in the normal course of business with various third parties to support our commercial activities and research and development programs.
+Added: In the ordinary course of business, we enter into agreements with contract manufacturers to manufacture our inventory products.
+Added: Although the agreements generally provide a termination clause with or without cause, we may still be subjected to payment of cancellation fees.
+Added: The level of cancellation fees is generally dependent on the timing of the written notice in relation to the commencement of work, with the maximum cancellation fees equal to the full price of
+Added: the work order.
+Added: In October 2024, we entered into an agreement with a third-party contract manufacturer to manufacture TAVALISSE that is expected to be delivered starting in 2026 through 2029.
+Added: As of March 31, 2025, the contractual obligation not included in our financial statements related an agreement that may potentially be subjected to cancellation fees amounted to approximately $20.8 million, with approximately $2.8 million due in the remainder of 2025 and $9.7 million due in 2026 and 2027.
+Added: As of March 31, 2025, we have not incurred any cancellation fees under our agreements with contract manufacturers.
+Added: As discussed in detail in “Note 4 – Sponsored Research, License Agreements and Government Contracts” of our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q, under the Lilly Agreement, although our co-funding obligation for development of ocadusertib (previously R552) in the US, Europe, and Japan ended on April 1, 2024, we have the right to opt-in to co-funding, upon us providing notice to Lilly within 30 days of certain events, as specified in the agreement.
+Added: On April 30, 2025, we provided notice to Lilly of our decision not to exercise our opt-in right following our evaluation of certain events specified in the Lilly Agreement.
+Added: Following this notification, we are no longer obligated to share in any future global development costs, which resulted in the release of the $40.0 million remaining cost share liability currently on our condensed balance sheet.
+Added: Also, as discussed in detail in “Note 4 – Sponsored Research, License Agreements and Government Contracts” and “Note 5 – In-licensing and Acquisition” of our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q, pursuant to our license and transition services agreement with Forma, Forma is entitled to potential development and regulatory milestone payments and tiered royalty payments on net sales as well as certain portion of sublicensing revenue.
+Added: Further, following our olutasidenib sublicensing agreements with Kissei and Dr.
+Added: Reddy’s, Forma is entitled to a portion of the sublicensing revenue we receive from Kissei and Dr.
+Added: Reddy’s under such respective agreements.
+Added: Additionally, as discussed in detail in “Note 5 – In-licensing and Acquisition” of our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q, pursuant to an Asset Purchase Agreement with Blueprint, in addition to unpaid purchase price consideration, Blueprint is entitled to potential commercial and regulatory milestone payments, as well as tiered royalty payments.
+Added: We have a contractual commitment with respect to our credit facility with MidCap.
+Added: Under the amended Credit Agreement, the term loans mature on September 1, 2027, and the interest-only period is through October 1, 2025.
+Added: As of March 31, 2025, the outstanding principal amount of the loan was $60.0 million, of which $15.0 million principal payments are due within 12 months.
+Added: As of March 31, 2025, future interest calculated using the base interest rate as per the amended Credit Agreement, and the final fee payments associated with the credit facility amounted to $12.7 million, with approximately $6.2 million payable within 12 months.
+Added: As of March 31, 2025, we have a contractual commitment related to our sublease agreement with Atara which sublease will expire in May 2025.
+Added: In February 2025, we entered into a lease agreement with 611 Gateway to lease the same office space currently subleased from Atara.
+Added: As of March 31, 2025, our contractual commitment related to the lease agreements was $1.5 million, of which $0.6 million is payable in the next 12 months.
We are also subject to claims related to the patent protection of certain of our technologies, as well as purported securities class action lawsuit, other litigations, and other contractual agreements.
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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.