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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 six months ended June 30, 2025 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 and nine months ended September 30, 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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risks that the FDA, EMA, the Medicines and Health Products Regulatory Agency (MHRA) or other regulatory authorities may make adverse decisions regarding our products;
+Added: the impact of the US federal government shutdown;
the progress of our and our collaborators’ product development programs, including clinical testing, and the timing of results thereof;
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We acquired the rights to research, develop, manufacture and commercialize GAVRETO in the US from Blueprint pursuant to an Asset Purchase Agreement entered in February 2024.
−Removed: We continue to advance the development of R289, our dual IRAK 1/4 inhibitor program, in an open-label, Phase 1b study to determine the tolerability and preliminary efficacy of the drug in patients with lower-risk MDS who are relapsed, refractory or resistant to prior therapies.
+Added: We continue to advance the development of R289, our dual IRAK1/4 inhibitor program, in an open-label, Phase 1b study to determine the safety, tolerability and preliminary efficacy of the drug in patients with lower-risk MDS who are relapsed, refractory or resistant to prior therapies.
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.
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TAVALISSE IN ITP
−Removed: For the six months ended June 30, 2025, net product sales of TAVALISSE were $68.5 million, increased by $21.1 million or 44% compared to $47.5 million net product sales in the same period in 2024.
−Removed: The increase was primarily due to increased quantities sold and higher price per bottle, and partly due to lower revenue reserves rate.
+Added: For the nine months ended September 30, 2025, net product sales of TAVALISSE were $113.3 million, increased by $39.5 million or 54% compared to $73.8 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 partly due to benefit from lower revenue reserves.
REZLIDHIA in R/R AML with mIDH1
−Removed: For the six months ended June 30, 2025, net product sales of REZLIDHIA were $13.1 million, increased by $3.1 million or 31% compared to $10.0 million net product sales in the same period in 2024.
−Removed: The increase was primarily due to increased quantities sold and higher price per bottle, which were partially offset by higher revenue reserves rate.
+Added: For the nine months ended September 30, 2025, net product sales of REZLIDHIA were $21.4 million, increased by $5.9 million or 38% compared to $15.6 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, partially offset by higher revenue reserves.
GAVRETO in metastatic RET fusion-positive NSCLC and advanced thyroid cancers
−Removed: We began our commercialization and started recognizing revenue from product sales of GAVRETO in June 2024.
−Removed: For the six months ended June 30, 2025, net product sales of GAVRETO were $20.8 million, compared to $1.9 million in the same period in 2024.
+Added: We began our commercialization and started recognizing revenue from product sales of GAVRETO in late June 2024.
+Added: For the nine months ended September 30, 2025, net product sales of GAVRETO were $31.9 million, compared to $9.0 million in the same period in 2024.
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.
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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).
+Added: Enrollment in the dose escalation part of the study was completed in July 2025.
+Added: In October 2025, we announced that the first patient was enrolled in the dose expansion part of the study, in which patients will be randomized to receive either 500 mg once daily or 500 mg twice daily, with the goal to determine the recommended phase 2 dose for future clinical studies.
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.
−Removed: 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.
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.
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.
−Removed: 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: Red blood cell (RBC)-transfusion independence (RBC-TI) ≥8 weeks was achieved by three patients (1 at 500 mg once daily and 2 at 750 mg once daily);
two HTB patients achieved RBC-TI >24 weeks.
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The three patients that achieved RBC-TI had peak hemoglobin increases exceeding 2.0 g/dL compared to baseline.
−Removed: We also reported that 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: We also reported that one HTB patient receiving 500 mg once daily achieved a minor hematologic improvement-erythroid (HI-E) response, with a 64% reduction in RBC transfusions compared to baseline;
however, in the July 15, 2025 data cut, we determined that this patient had received blood transfusions that were not captured in the database at the time of the initial data analysis.
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As the study is ongoing, interim results represent information at the time of the data cut, and final study results will be available after the database lock at the end of the study.
−Removed: Enrollment in the dose escalation part of the study was completed in July 2025.
−Removed: We expect to share updated data from the study and plan to initiate the dose expansion part of the study in the second half of 2025.
+Added: On November 3, 2025, we announced that we will present updated data from the dose escalation phase of our ongoing Phase 1b study evaluating R289 in patients with R/R lower-risk MDS, in an oral presentation at the upcoming 67th ASH Annual Meeting and Exposition to be held December 6-9, 2025, in Orlando, Florida, and virtually.
+Added: R289 continues to be generally well tolerated and demonstrate preliminary signs of efficacy in doses equal to or above 500 mg daily.
+Added: As of the July 15, 2025 data cutoff, 33 patients were enrolled in the dose escalation part of the study.
+Added: Patients had a median age of 75 with a median of 3 prior therapies and 61% were high transfusion burden at baseline.
+Added: Patients received R289 at doses ranging from 250 mg once daily to 500 mg twice daily.
+Added: For the 500 mg twice daily dose group, five patients were not yet evaluable (<16 weeks follow up) for determination of hematologic responses and one patient withdrew consent.
+Added: The most frequent treatment emergent adverse events (≥20%) were diarrhea (28.1%), constipation/fatigue (25% each), and creatinine/alanine aminotransferase (ALT) increased (21.9% each), the majority being Grade 1/2.
+Added: 1 dose limiting toxicity (DLT) (Grade 4 aspartate aminotransferase (AST) increase/Grade 3 ALT increase) was reported in the 750 mg dose group.
+Added: For evaluable transfusion dependent patients (≥16 weeks follow up) receiving doses of at least 500 mg once daily and higher, 4/13 patients (31%) achieved durable RBC-TI for > 8 weeks (500 mg once daily [1/3], 750 mg once daily [2/5], 500/250 mg once daily [1/5]).
+Added: Duration of RBC-TI was >16 weeks in 3 patients, >24 weeks in 2 patients, and >12 months in 1 patient.
+Added: The median time to onset of RBC-TI was 2.2 months, and the median duration of RBC-TI was 24.3 weeks.
+Added: All responding patients had R835 plasma concentrations similar to those at which ≥50% LPS-induced inhibition of cytokine release was observed in healthy volunteers, indicating a potential threshold for dose response (≥500 mg once daily).
+Added: Updated data as of a October 28, 2025 data cutoff will be presented during the oral presentation.
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.
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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.
+Added: Further, this collaboration will also support the evaluation of olutasidenib in combination with co-targeted therapies in patients with R/R IDH1-mutated myeloid malignancies harboring activated signaling pathway mutations.
+Added: T here are five studies open for enrollment associated with the multi-year strategic development alliance .
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 June 30, 2025, we provided $5.3 million funding to MDACC.
−Removed: T he four studies outlined in the multi-year strategic development alliance are open for enrollment.
+Added: Through September 30, 2025, we provided $5.3 million funding to MDACC.
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.
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.
−Removed: 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: In our sponsored arm, TarGet-D, adolescents and young adult patients (ages 12 to 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.
Under the collaboration, we will provide CONNECT with funding up to $3.0 million and study material over the four-year collaboration.
−Removed: The TarGet-D study is open for enrollment.
−Removed: Incrementally, we plan on initiating a Phase 2 clinical study in recurrent glioma in 2025.
−Removed: This, in combination with our strategic collaborations with MDACC and CONNECT, are aimed to expand our olutasidenib pipeline development programs.
+Added: The first patient was enrolled in the Phase 2 TarGet-D study in October 2025.
+Added: Incrementally, we plan to expand the evaluation of olutasidenib through additional strategic collaborations and potential Rigel-led studies, complementing our existing partnerships with MDACC and CONNECT.
Global Strategic Partnership with Lilly
Lilly is continuing to advance ocadusertib (previously R552), an investigational, potent and selective RIPK1 inhibitor.
−Removed: Enrollment in Lilly’s Phase 2a clinical trial studying ocadusertib in adult patients with moderately to severely active rheumatoid arthritis is ongoing, with preliminary analysis of results in April 2025.
+Added: Enrollment in Lilly’s Phase 2a clinical trial studying ocadusertib in adult patients with moderately to severely active rheumatoid arthritis is ongoing, with a preliminary analysis of results made available 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.
−Removed: 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.
+Added: On October 1, 2025, we received a notice from Lilly of its intent to terminate the CNS disease program under the Lilly Agreement, which will become effective sixty (60) days following notification.
Under the Lilly Agreement, we were responsible for 20% of the development costs for ocadusertib in the US, Europe, and Japan, up to a specified cap, and Lilly is responsible for funding the remainder of all development activities for ocadusertib and other non-CNS disease development candidates.
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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.
−Removed: 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 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.
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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Fostamatinib is a potent and relatively selective SYK inhibitor, and its inhibition of Fc receptors and B-cell receptors of signaling pathways make it a potentially broad immunomodulatory agent.
−Removed: 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 .).
+Added: The FDA recently approved the product WAYRILZ TM (Sanofi SA) for the treatment of adults with persistent or chronic ITP.
+Added: 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.),
+Added: DOPTELET ® (Swedish Orphan Biovitrum AB) and ALVAIZ TM ( Teva Pharmaceutical Industries Ltd .).
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.
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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.
−Removed: We believe that our
−Removed: 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: 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.
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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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 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.
−Removed: 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.
+Added: In December 2022, Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) approved TAVALISSE for the treatment of persistent and chronic ITP, and in April 2023, Kissei launched TAVALISSE 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 ITP who have had an insufficient response to a previous treatment.
In July 2025, Kissei announced that its licensing partner, JW Pharmaceutical Corporation, commercially launched TAVALISSE in Korea.
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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: In September 2025, Knight received a rejection from Brazilian Health Regulatory Agency (ANVISA) regarding its marketing authorization application for fostamatinib.
+Added: Knight filed an appeal which may take up to fourteen months.
REZLIDHIA/Olutasidenib in R/R AML with mIDH1
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The interim results of this trial of 153 patients showed that olutasidenib demonstrated a favorable tolerability profile as a monotherapy in patients with R/R AML who have a susceptible mIDH1, and achieved a complete remission (CR) plus CR with partial hematologic recovery (CRh) rate of 33.3% (30% CR and 3% CRh), the primary efficacy endpoint.
−Removed: While a median duration of CR/CRh was not yet reached, a sensitivity analysis (with a hematopoietic stem cell transplant, as the end of a response) indicated the median duration of CR/CRh was 13.8 months .
+Added: While a median duration of CR/CRh was not yet reached, a sensitivity analysis (with a hematopoietic
+Added: stem cell transplant, as the end of a response) indicated the median duration of CR/CRh was 13.8 months .
The overall response rate, comprised CR, CRh, Cri, partial response, and morphologic leukemia-free state (MLFS), was 46% and the median duration of overall response rate (ORR) was 11.7 months.
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Additionally, REZLIDHIA was well tolerated in a subset of patients with myeloproliferative neoplasms mIDH1 AML, a patient population often associated with poor responses to available therapies.
+Added: In October 2025, we announced the publication of the final five-year data for REZLIDHIA in patients with R/R MIDH1 AML in the Journal of Hematology and Oncology .
+Added: The publication reports the final follow-up analysis of the registrational Phase 2 trial, with an additional two years of efficacy and safety data.
+Added: These five-year data further support the durable responses and manageable safety profile observed with olutasidenib in patients with R/R m IDH1 AML, including those R/R to prior venetoclax.
+Added: The safety profile remained consistent with what was previously reported, with no new safety signals identified.
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 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
+Added: 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.
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GAVRETO is also approved for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).
−Removed: This indication was approved by the FDA under accelerated approval based on overall response rate and duration of response.
+Added: This indication was approved by the FDA under accelerated approval
+Added: based on overall response rate and duration of response.
Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial.
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The NCCN Guidelines for NSCLC recommends pralsetinib as a preferred first-line treatment option for RET+ patients, including for patients identified during first-line treatment with systemic therapy.
−Removed: 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-
−Removed: positive NSCLC as detected by an FDA-approved test.
+Added: 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.
The FDA also granted GAVRETO two orphan drug exclusivities until December 2027 with respect to FDA approval for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate), and for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid carcinoma who require systemic therapy.
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In October 2019, we announced results from a Phase 1 randomized, placebo-controlled, double-blind clinical study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of R835 in 91 healthy adult subjects.
−Removed: The Phase 1 study showed that R835 had a favorable safety, tolerability and PK profile and established proof-of-mechanism by demonstrating the inhibition of inflammatory cytokine production in response to a lipopolysaccharide (LPS) challenge.
+Added: The Phase 1 study showed that R835 had a favorable safety, tolerability and PK profile and established proof-
+Added: of-mechanism by demonstrating the inhibition of inflammatory cytokine production in response to a lipopolysaccharide (LPS) challenge.
We advanced the development of our IRAK1/4 inhibitor program, following evaluation of single and multiple ascending doses of R289, a new pro-drug formulation of R835, in healthy subjects.
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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).
+Added: Enrollment in the dose escalation part of the study was completed in July 2025.
+Added: In October 2025, we announced that the first patient was enrolled in the dose expansion part of the study, in which patients will be randomized to receive either 500 mg once daily or 500 mg twice daily, with the goal to determine the recommended phase 2 dose for future clinical studies.
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.
−Removed: 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.
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.
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.
−Removed: RBC-TI ≥8 weeks was achieved by
−Removed: three patients (1 at 500 mg QD and 2 at 750 mg QD);
+Added: RBC-TI ≥8 weeks was achieved by three patients (1 at 500 mg once daily and 2 at 750 mg once daily);
two HTB patients achieved RBC-TI >24 weeks.
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The three patients that achieved RBC-TI had peak hemoglobin increases exceeding 2.0 g/dL compared to baseline.
−Removed: We also reported that one HTB patient receiving 500 mg QD achieved a minor HI-E response, with a 64% reduction in RBC transfusions compared to baseline;
+Added: We also reported that one HTB patient receiving 500 mg once daily achieved a minor HI-E response, with a 64% reduction in RBC transfusions compared to baseline;
however, in the July 15, 2025 data cut, we determined that this patient had received blood transfusions that were not captured in the database at the time of the initial data analysis.
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As the study is ongoing, interim results represent information at the time of the data cut, and final study results will be available after the database lock at the end of the study.
−Removed: Enrollment in the dose escalation part of the study was completed in July 2025.
−Removed: We expect to share updated data from the study and plan to initiate the dose expansion part of the study in the second half of 2025.
+Added: On November 3, 2025, we announced that we will present updated data from the dose escalation phase of our ongoing Phase 1b study evaluating R289 in patients with R/R lower-risk MDS, in an oral presentation at the upcoming 67th ASH Annual Meeting and Exposition to be held December 6-9, 2025, in Orlando, Florida, and virtually.
+Added: R289 continues to be generally well tolerated and demonstrate preliminary signs of efficacy in doses equal to or above 500 mg daily.
+Added: As of the July 15, 2025 data cutoff, 33 patients were enrolled in the dose escalation part of the study.
+Added: Patients had a median age of 75 with a median of 3 prior therapies and 61% were high transfusion burden at baseline.
+Added: Patients received R289 at doses ranging from 250 mg once daily to 500 mg twice daily.
+Added: For the 500 mg twice daily dose group, five patients were not yet evaluable (<16 weeks follow up) for determination of hematologic responses and one patient withdrew consent.
+Added: The most frequent treatment emergent adverse events (≥20%) were diarrhea (28.1%), constipation/fatigue (25% each), and creatinine/ALT increased (21.9% each), the majority being Grade 1/2.
+Added: 1 DLT (Grade 4 AST increase/Grade 3 ALT increase) was reported in the 750 mg dose group.
+Added: For evaluable transfusion dependent patients (≥16 weeks follow up) receiving doses of at least 500 mg once daily and higher, 4/13 patients (31%) achieved durable RBC-TI for > 8 weeks (500 mg once daily [1/3], 750 mg once daily [2/5], 500/250 mg once daily [1/5]).
+Added: Duration of RBC-TI was >16 weeks in 3 patients, >24 weeks in 2 patients, and >12 months in 1 patient.
+Added: The median time to onset of RBC-TI was 2.2 months, and the median duration of RBC-TI was 24.3 weeks.
+Added: All responding patients had R835 plasma concentrations similar to those at which ≥50% LPS-induced inhibition of cytokine release was observed in healthy volunteers, indicating a potential threshold for dose response (≥500 mg once daily).
+Added: Updated data as of a October 28, 2025 data cutoff will be presented during the oral presentation.
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.
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We have a strategic collaboration agreement with MDACC to expand our evaluation of olutasidenib in AML and other hematologic cancers with IDH1 mutations.
−Removed: 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.
+Added: 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
+Added: patients with AML, higher-risk MDS, and advanced myeloproliferative neoplasms, in combination with other agents.
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.
−Removed: The four studies outlined in the multi-year strategic development alliance are open for enrollment.
−Removed: 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.
+Added: The five studies outlined in the multi-year strategic development alliance are open for enrollment.
+Added: The five 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.
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(iii) a Phase 1/2 study of olutasidenib maintenance therapy following an allogeneic stem cell transplant for patients with IDH1-mutated myeloid malignancies;
−Removed: 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: (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: and (v) a P hase 2 multi-arm, multi-center, open-label, non-randomized clinical study will evaluate olutasidenib in combination with co-targeted therapies in patients with R/R IDH1-mutated myeloid malignancies harboring activated signaling pathway mutations The primary objectives of the study are to evaluate safety and the composite complete remission rate.
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.
1 unchanged sentence
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.
−Removed: The TarGet-D study is open for enrollment.
+Added: The The first patient was enrolled in the Phase 2 TarGet-D study in October 2025.
Partnered Clinical Programs
1 unchanged sentence
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.
−Removed: Enrollment in Lilly’s Phase 2a clinical trial studying ocadusertib in adult patients with moderately to severely active rheumatoid arthritis is ongoing, with preliminary analysis of results in April 2025.
+Added: Enrollment in Lilly’s Phase 2a clinical trial studying ocadusertib in adult patients with moderately to severely active rheumatoid arthritis is ongoing, with a preliminary analysis of results made available 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.
−Removed: 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.
+Added: On October 1, 2025, we received a notice from Lilly of its intent to terminate the CNS disease program under the Lilly Agreement, which will become effective sixty (60) days following notification.
Other Partnered Programs
11 unchanged sentences
We also have strategic development collaborations with MDACC and CONNECT to conduct evaluation of olutasidenib in AML, other hematologic cancers and glioma.
−Removed: Incrementally, we plan on initiating a Phase 2 clinical study in recurrent glioma in 2025.
−Removed: This, in combination with our strategic collaborations with MDACC and CONNECT, are aimed to expand our olutasidenib pipeline development programs.
+Added: Incrementally, we plan to expand the evaluation of olutasidenib through additional strategic collaborations and potential Rigel-led studies, complementing our existing partnerships with MDACC and CONNECT.
Commercialization and Sponsored Research and License Agreements
−Removed: See “Note 4 – Sponsored Research, License Agreements and Government Contracts” and “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 for related discussions.
+Added: See “Note 4 – Sponsored Research and License Agreements” and “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 for related discussions.
Results of Operations
−Removed: Three Months Ended June 30,
−Removed: Six Months Ended June 30,
+Added: Three Months Ended September 30,
+Added: Nine Months Ended September 30,
(in thousands)
3 unchanged sentences
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 June 30,
−Removed: Six Months Ended June 30,
+Added: Three Months Ended September 30,
+Added: Nine Months Ended September 30,
McKesson Corporation
Cencora, Inc.
+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 six months ended June 30, 2025 were $40.1 million and $68.5 million, respectively, increased by 52% and 44%, respectively, compared to $26.4 million and $47.5 million for the three and six months ended June 30, 2024, respectively.
−Removed: The increase was primarily due to increased quantities sold and higher price per bottle, and partly due to lower revenue reserves rate.
−Removed: REZLIDHIA net product sales for the three and six months ended June 30, 2025 were $7.0 million and $13.1 million, respectively, increased by 36% and 31%, respectively, compared to $5.2 million and $10.0 million for the three and six months ended June 30, 2024, respectively.
−Removed: The increase was primarily due to increased quantities sold and higher price per bottle, which were partially offset by higher revenue reserves rate.
−Removed: Following the commercialization of GAVRETO in June 2024, we started recognizing revenue from shipments to our distributors.
−Removed: For the three and six months ended June 30, 2025, we recognized $11.8 million and $20.8 million of GAVRETO net product sales, compared to $1.9 million net product sales for the three and six months ended June 30, 2024.
−Removed: Contract revenues from collaborations for the three and six months ended June 30, 2025 primarily consisted of $40.0 million of non-cash revenue related to the release of cost share liability from our collaboration with Lilly.
−Removed: In addition, for the three and six months ended June 30, 2025, contract revenues from collaborations includes revenue from Grifols of $2.0 million and $6.7 million, respectively, related to earned royalty and delivery of drug supplies, as well as revenue from Kissei of $0.4 million and $5.1 million, respectively, related to delivery of drug supplies and a milestone payment in the first quarter of 2025.
−Removed: Contract revenues from collaborations in the three and six months ended June 30, 2024 consisted primarily of revenue from Kissei of $2.2 million and $4.5 million, respectively, related to the delivery of drug supplies, and revenue from Grifols of $1.1 million and $2.2 million, respectively, related to earned royalty.
+Added: TAVALISSE net product sales for the three and nine months ended September 30, 2025 were $44.7 million and $113.3 million, respectively, increased by 70% and 54%, respectively, compared to $26.3 million and $73.8 million for the three and nine months ended September 30, 2024, respectively.
+Added: The increase was primarily due to increased quantities sold and higher price per bottle, and partly due to benefit from lower revenue reserves.
+Added: REZLIDHIA net product sales for the three and nine months ended September 30, 2025 were $8.3 million and $21.4 million, respectively, increased by 50% and 38%, respectively, compared to $5.5 million and $15.6 million for the three and nine months ended September 30, 2024, respectively.
+Added: The increase was primarily due to increased quantities sold and higher price per bottle, partially offset by higher revenue reserves.
+Added: GAVRETO net product sales for the three and nine months ended September 30, 2025 were $11.0 million and $31.9 million, respectively, compared to $7.1 million and $9.0 million for the three and nine months ended September 30, 2024, respectively.
+Added: We started recognizing GAVRETO net product sales
+Added: following the commercialization in late June 2024.
+Added: The increase in GAVRETO net product sales for the three months ended September 30, 2025 compared to the same period in 2024 was primarily due to increased quantities sold, and partly due to higher price per bottle.
+Added: Contract revenues from collaborations for the three and nine months ended September 30, 2025 comprised primarily of revenue from Grifols of $3.1 million and $9.9 million, respectively, related to earned royalty and delivery of drug supply;
+Added: revenue from Kissei of $1.8 million and $6.9 million, respectively, related to delivery of drug supply and a milestone payment in the first quarter of 2025;
+Added: and revenue from Medison of $0.2 million and $0.8 million, respectively, related to earned royalty and delivery of drug supply.
+Added: In addition, contract revenues from collaborations for the nine months ended September 30, 2025 include a $40.0 million non-cash revenue related to the release of cost share liability from our collaboration with Lilly.
+Added: Contract revenues from collaborations in the three and nine months ended September 30, 2024 comprised primarily of revenue from Kissei of $13.0 million and $17.5 million, respectively, related to an upfront fee from sublicensing olutasidenib and delivery of drug supplies;
+Added: revenue from Grifols of $3.3 million and $5.5 million, respectively, related to earned royalty and delivery of drug supply;
+Added: and revenue from Medison of $0.1 million and $0.2 million, respectively, related to earned royalty and delivery of drug supply.
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 June 30, 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.
+Added: As of September 30, 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 June 30,
−Removed: Six Months Ended June 30,
+Added: Three Months Ended September 30,
+Added: Nine Months Ended September 30,
(in thousands)
2 unchanged sentences
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.
−Removed: 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.
+Added: Specifically, we have been utilizing zero-cost API for TAVALISSE, with remaining inventories expected to be consumed within 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.
−Removed: 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.
+Added: As we acquire or produce more FDA approved inventory quantities, our inventory cost in the balance sheet and cost of product sales will reflect the full cost of acquiring or producing such products.
We rely and will continue to rely on certain third parties, including those located outside the US to manufacture our products.
1 unchanged sentence
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: 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.
−Removed: Cost of product sales increased in the three and six months ended June 30, 2025 compared to the same periods in 2024, primarily due to increased royalties of $1.3 million and $2.2 million, respectively, as well as increased cost of products and other increases of $0.4 million and $1.9 million, respectively, generally due to the increase in product sales and delivery of drug supplies to our collaboration partners.
+Added: Cost of product sales also includes amortization of intangible assets and royalties.
+Added: The decrease in cost of product sales for the three months ended September 30, 2025, compared to the same period in 2024, was primarily driven by a $2.1 million decrease in product costs due to the timing of drug supply deliveries to our collaboration partners, partially offset by higher product costs resulting from increased product sales.
+Added: Additionally, royalties decreased by $1.1 million due to a sublicensing revenue fee recognized in the third quarter of 2024 related to the sublicensing of olutasidenib to Kissei, partially offset by increased royalties from higher sublicensed product sales.
+Added: The increase in cost of product sales for the nine months ended September 30, 2025, compared to the same period in 2024, was primarily driven by $1.1 million in higher royalties resulting from increased sublicensed product sales, partially offset by a sublicensing revenue fee recognized in the third quarter of 2024 related to the sublicensing of
+Added: olutasidenib to Kissei.
+Added: Additionally, amortization expense increased by $0.2 million.
+Added: These increases were partially offset by decreased product costs of $0.5 million primarily due to the timing of drug supply deliveries to collaboration partners, partially offset by higher product costs resulting from increased product sales.
Research and Development Expense
−Removed: Three Months Ended June 30,
−Removed: Six Months Ended June 30,
+Added: Three Months Ended September 30,
+Added: Nine Months Ended September 30,
(in thousands)
1 unchanged sentence
Stock-based compensation expense included in research and development expense
−Removed: The increase in research and development expense in the three months ended June 30, 2025 compared to the same period in 2024 was primarily driven by the increase in clinical trial related expenses of $0.9 million due to timing of clinical development programs for olutasidenib study, and the progress activities on our ongoing IRAK 1/4 inhibitor program.
+Added: The increase in research and development expense in the three months ended September 30, 2025 compared to the same period in 2024 was primarily due to a $0.5 million increase in personnel-related costs, and a $0.5 million increase in clinical trial related expenses resulting from the timing of clinical development programs including the progress activities on our ongoing IRAK1/4 inhibitor program.
In addition, other various research and development expenses increased by $0.2 million.
−Removed: The increase in research and development expense in the six months ended June 30, 2025 compared to the same period in 2024 was primarily driven by the increase in clinical trial related expenses of $2.7 million due to timing of clinical development programs for olutasidenib study, and the progress activities on our ongoing IRAK 1/4 inhibitor program.
+Added: The increase in research and development expense in the nine months ended September 30, 2025 compared to the same period in 2024 was primarily due to a $3.2 million increase in clinical trial related expenses resulting from the timing of clinical development programs for olutasidenib study, and the progress activities on our ongoing IRAK1/4 inhibitor program, and a $1.4 million increase in personnel-related costs.
In addition, other various research and development expenses increased by $0.3 million.
13 unchanged sentences
In general, biopharmaceutical development involves a series of steps, beginning with identification of a potential target and including, among others, proof of concept in animals and Phase 1, 2 and 3 clinical trials in humans.
−Removed: Significant delays in clinical testing could materially impact our product development costs and timing of completion of the clinical trials.
+Added: Significant delays in clinical testing could materially impact our product development
+Added: costs and timing of completion of the clinical trials.
We do not know whether planned clinical trials will begin on time, will need to be halted or revamped or will be completed on schedule, or at all.
5 unchanged sentences
The following table presents our total research and development expense by category (in thousands).
−Removed: Three Months Ended June 30,
−Removed: Six Months Ended June 30,
+Added: Three Months Ended September 30,
+Added: Nine Months Ended September 30,
From January 1, 2007*
−Removed: to June 30, 2025
+Added: to September 30, 2025
* We started tracking research and development expense by category on January 1, 2007.
−Removed: “Other” expenses in the three months ended June 30, 2025 and 2024 consisted of allocated facilities costs of $0.1 million and $0.2 million, respectively, and allocated stock-based compensation expense of $0.5 million and $0.3 million, respectively.
−Removed: For the six months ended June 30, 2025 and 2024, allocated facilities costs were $0.2 million and $0.3 million, respectively, and allocated stock-based compensation expense was $1.4 million and $1.0 million, respectively.
+Added: “Other” expenses in the three months ended September 30, 2025 and 2024 consisted of allocated facilities costs of $0.1 million for each periods, and allocated stock-based compensation expense of $0.4 million and $0.3 million, respectively.
+Added: For the nine months ended September 30, 2025 and 2024, allocated facilities costs were $0.3 million and $0.4 million, respectively, and allocated stock-based compensation expense was $1.8 million and $1.2 million, respectively.
Selling, General and Administrative Expense
−Removed: Three Months Ended June 30,
−Removed: Six Months Ended June 30,
+Added: Three Months Ended September 30,
+Added: Nine Months Ended September 30,
(in thousands)
1 unchanged sentence
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 June 30, 2025 compared to the same period in 2024 was primarily due to increased personnel-related costs and stock-based compensation expense of $1.5 million, and increased other various sales, general and administrative expenses of $0.6 million.
+Added: The increase in selling, general and administrative expense in the three months ended September 30, 2025 compared to the same period in 2024 was primarily due to a $1.6 million increase in personnel-related costs, and a $0.8 million increase in consulting and third-party services.
+Added: These increases were partially offset by decrease in other various sales, general and administrative expenses of $0.5 million.
+Added: The increase in selling, general and administrative expense in the nine months ended September 30, 2025 compared to the same period in 2024 was primarily due to a $2.6 million increase in personnel-related costs, and a $0.4 million increase in other various sales, general and administrative expenses.
These increases were partially offset by decreased consulting and third-party services of $0.7 million .
−Removed: The increase in selling, general and administrative expense in the six months ended June 30, 2025 compared to the same period in 2024 was primarily due to increased personnel-related costs of $2.5 million and increased other various sales, general and administrative expenses of $1.0 million.
−Removed: These increases were partially offset by decreased consulting and third-party services of $1.5 million, and decreased stock-based compensation expense of $1.5 million primarily related to our performance-based stock awards.
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.
2 unchanged sentences
Interest Income and Interest Expense
−Removed: Three Months Ended June 30,
+Added: Three Months Ended September 30,
+Added: September 30,
(in thousands)
3 unchanged sentences
Interest expense comprised interest on our outstanding term loans with MidCap.
−Removed: Provision for income Taxes
−Removed: Three Months Ended June 30,
−Removed: Six Months Ended June 30,
+Added: Three Months Ended September 30,
+Added: Nine Months Ended September 30,
(in thousands)
−Removed: Provision for income taxes
−Removed: 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: (Benefit from) 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, adjusted for any discrete items.
We update our estimate of our annual effective tax rate at the end of each quarterly period.
−Removed: The provision for income taxes for the three and six months ended June 30, 2025 was primarily related to estimated state taxes.
−Removed: For the three and six months ended June 30, 2024, we did not record a provision for income taxes due to our pre-tax book loss.
−Removed: 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 TCJA, as well as significant research and development credit carryforwards.
−Removed: As of June 30, 2025, we continue to record a full valuation allowance on our deferred tax assets.
+Added: The benefit from or provision for income taxes for the three and nine months ended September 30, 2025 was primarily related to estimated state income taxes.
+Added: For the three and nine months ended September 30, 2024, we did not record a provision for income taxes based on the forecasted pre-tax book loss.
+Added: We do not expect to owe federal income tax due to sufficient net operating loss carryforwards, as well as significant research and development credit carryforwards.
+Added: In July 2025, the OBBBA was signed into law.
+Added: The OBBBA introduces a wide range of provisions affecting business entities, including the establishment of certain permanent business tax measures.
+Added: Key provisions include a permanent and immediate deduction for domestic research and development expenditures, the restoration and permanent extension of 100% expensing for qualified equipment purchases, and restores the ability to add back depreciation and amortization expense when determining the limitation on interest deductions.
+Added: In accordance with ASC 740, Income Taxes , the effects of changes in tax laws are recognized in the period of enactment.
+Added: Accordingly, we accounted for the estimated impact of the OBBBA in our current period tax provision.
+Added: The enactment of the OBBBA did not have a material impact on our condensed financial statements for the three and nine months ended September 30, 2025.
+Added: As of September 30, 2025, we continue to record a full valuation allowance on our deferred tax assets.
The realization of deferred tax assets is dependent upon demonstrating sufficient positive evidence to conclude that it is more-likely-than-not that our deferred tax assets will be realized.
2 unchanged sentences
If sufficient positive evidence becomes available to allow us to reach a conclusion that a portion of the valuation allowance against the deferred tax assets may be reversed, the reversal would result in an income tax benefit for the quarterly and annual fiscal period in which we determine to release such valuation allowance.
−Removed: In July 2025, the OBBBA was enacted, which includes a broad array of measures affecting corporations and other business entities, including locking in a key set of business tax provisions.
−Removed: These include incentives designed to promote innovation-driven investment, such as a permanent and immediate deduction for domestic research and development costs.
−Removed: The legislation also locks in 100% expensing for qualified equipment purchases and makes permanent the deduction of up to 20% for qualified business income.
−Removed: ASC 740, “ Income Taxes ”, requires the effects of changes in tax rates and laws on deferred tax balances to be recognized in the period in which the legislation is enacted.
−Removed: Consequently, we are evaluating the impact of the OBBBA on our financial statements and an estimate of the financial impact cannot be made at this time.
Critical Accounting Policies and Use of Estimates
9 unchanged sentences
Liquidity and Capital Resources
−Removed: As of June 30, 2025 and December 31, 2024, we had approximately $108.4 million and $77.3 million, respectively, in cash, cash equivalents and short-term investments.
+Added: As of September 30, 2025 and December 31, 2024, we had approximately $137.1 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.
4 unchanged sentences
The following table summarizes our cash flow activity for the periods presented:
−Removed: Six Months Ended June 30,
+Added: Nine Months Ended September 30,
(in thousands)
4 unchanged sentences
Net (decrease) increase in cash, cash equivalents and restricted cash
−Removed: Net cash provided by operating activities for the six months ended June 30, 2025 comprised net cash inflows from net income adjusted for non-cash items, partially offset by net cash outflows from changes in assets and liabilities.
−Removed: Net cash used in operating activities for the six months ended June 30, 2024 comprised net cash outflows from net loss adjusted for non-cash items, and net cash outflows from changes in assets and liabilities.
−Removed: Net cash flows from changes in assets and liabilities for the six months ended June 30, 2025 and 2024 were primarily due to timing of inventory build-up, timing of advance payments to our contract manufacturers and strategic development collaboration partners, timing of payment of liabilities, and timing of collection of accounts receivable.
−Removed: Net cash used in investing activities for the six months ended June 30, 2025 primarily comprised net purchases of short-term investments of $33.8 million.
−Removed: Net cash provided by investing activities for the six months ended June 30, 2024 comprised primarily of net maturities of short-term investments of $11.6 million, partially offset by payments for acquisition of intangible assets of $0.4 million .
−Removed: Net cash provided by financing activities for the six months ended June 30, 2025 comprised net proceeds from issuance of common stock from equity plans of $0.9 million.
−Removed: Net cash used in operating activities for the six months ended June 30, 2024 comprised cost share payments to a collaboration partner of $3.6 million, partially offset by the net proceeds from issuance of common stock from equity plans of $0.3 million.
+Added: Net cash provided by operating activities for the nine months ended September 30, 2025, reflected net income adjusted for non-cash items, partially offset by net cash outflows from changes in working capital.
+Added: The working capital outflows were primarily driven by increases in prepaid and other current assets due to the timing of advance payments to contract manufacturers and strategic development partners, higher inventory levels due to the timing of production build-up, and increased accounts receivable resulting from timing of collection.
+Added: These were partially offset by increased liabilities driven by the timing of payments.
+Added: In comparison, net cash provided by operating activities for the nine months ended September 30, 2024, included net income adjusted for non-cash items and net cash inflows from changes in working capital.
+Added: The working capital inflows were primarily the result of higher liabilities driven by the timing of payments, partially offset by increases in prepaid and other current assets due to the timing of advance payments to contract manufacturers, and higher inventory levels due to the timing of production build-up.
+Added: Net cash used in investing activities for the nine months ended September 30, 2025 primarily comprised net purchases of short-term investments of $67.0 million.
+Added: 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 .
+Added: Net cash provided by financing activities for the nine months ended September 30, 2025 comprised net proceeds from issuance of common stock from equity plans of $5.2 million.
+Added: 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 from equity plans of $0.5 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.
4 unchanged sentences
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 June 30, 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.
+Added: The total potential future contingent payments due to us under all existing collaboration agreements are approximately $1.1 billion, which amount reflects the impact of Lilly’s notice of intent to terminate the CNS disease program in October 2025, and assumes that all potential product candidates achieve every payment-triggering milestone under 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.
Future events that may trigger payments to us under the agreements are based solely on our partners’ future efforts and achievements of specified development, regulatory and/or commercial events.
−Removed: See further discussion in “Note 4 – Sponsored Research, License Agreements and Government Contracts” to our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q.
+Added: See further discussion in “Note 4 – Sponsored Research and License Agreements” to our “Notes to Condensed Financial Statements” contained in Part I, Item 1 of this Quarterly Report on Form 10-Q.
We have an Open Market Sale Agreement with Jefferies LLC (Jefferies), as a sole agent, entered on August 4, 2020, and amended and restated on August 2, 2024.
1 unchanged sentence
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.
−Removed: As of June 30, 2025, we have not sold any shares of common stock under such 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 June 30, 2025.
−Removed: Our operations will require significant additional funding in the foreseeable future.
−Removed: Unless and until we can generate sufficient cash from our operating activities, we may choose to raise additional funds through public and/or private offerings of equity securities, debt financings, or from other sources.
−Removed: However, certain external factors such as global pandemics, the global tensions arising from the Russia-Ukraine war and Hamas-Israel war, political and economic legislations, and other factors may continue to rapidly evolve which could significantly disrupt the global financial markets.
+Added: As of September 30, 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 September 30, 2025.
+Added: We may from time to time consider raising additional funds through public and/or private offerings of equity securities, debt financings, or from other sources, in order to fund ongoing operations, to strengthen our long-term financial profile or to pursue opportunistic corporate development activities.
+Added: However, certain external factors such as global pandemics, the global tensions arising from the Russia-Ukraine war and Hamas-Israel war, political and economic legislations, and other factors may continue to rapidly evolve which could significantly disrupt the global financial
Our ability to raise additional funds may be adversely impacted by potential worsening of global economic conditions and volatility in the credit and financial markets in the US and worldwide.
25 unchanged sentences
We have agreements with certain clinical research organizations to conduct our clinical trials including our strategic development collaborations with MDACC and CONNECT.
−Removed: 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.
+Added: The timing of payments for any amounts owed under the respective agreements depends on
+Added: 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.
6 unchanged sentences
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.
−Removed: As of June 30, 2025, the contractual obligation not included in our financial statements related to an agreement that may potentially be subjected to cancellation fees amounting to approximately $22.5 million, with approximately $3.0 million due in the remainder of 2025 and $10.5 million due in 2026 and 2027.
−Removed: As of June 30, 2025, we have not incurred any cancellation fees under our agreements with contract manufacturers.
−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, 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: As of September 30, 2025, the contractual obligation not included in our financial statements related to an agreement that may potentially be subjected to cancellation fees amounting to approximately $22.6 million, with approximately $3.0 million due in the remainder of 2025 and $10.5 million due in 2026 and 2027.
+Added: As of September 30, 2025, we have not incurred any cancellation fees under our agreements with contract manufacturers.
+Added: As discussed in detail in “Note 4 – Sponsored Research and License Agreements” 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.
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.
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.
−Removed: 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: Also, as discussed in detail in “Note 4 – Sponsored Research and License Agreements” 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.
Further, following our olutasidenib sublicensing agreements with Kissei and Dr.
4 unchanged sentences
Under the amended Credit Agreement, the term loans mature on September 1, 2027, and the interest-only period is through October 1, 2025.
−Removed: As of June 30, 2025, the outstanding principal amount of the loan was $60.0 million, of which $22.5 million principal payments are due within 12 months.
−Removed: As of June 30, 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 $11.0 million, with approximately $5.8 million payable within 12 months.
−Removed: As of June 30, 2025, we have a contractual commitment related to our lease agreement with 611 Gateway which lease will expire in July 2027.
−Removed: As of June 30, 2025, our contractual commitment related to the lease agreements was $1.4 million, of which $0.6 million is payable in the next 12 months.
+Added: As of September 30, 2025, the outstanding principal amount of the loan was $60.0 million, of which $30.0 million principal payments are due within 12 months.
+Added: As of September 30, 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 $9.4 million, with approximately $5.1 million payable within 12 months.
+Added: As of September 30, 2025, we have a contractual commitment related to our lease agreement with 611 Gateway which lease will expire in July 2027.
+Added: As of September 30, 2025, our contractual commitment related to the lease agreements was $1.3 million, of which $0.7 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.
We are required to assess the likelihood of any adverse judgments or outcomes to these matters as well as potential ranges of probable losses.
−Removed: A determination of the amount of reserves required, if any, for these contingencies is made after careful analysis of each individual matter.
+Added: determination of the amount of reserves required, if any, for these contingencies is made after careful analysis of each individual matter.
We do not have other material contractual commitments with respect to matters discussed above.
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.