What changed in this filing

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Item 2. Management’s Discussion and Analysis

2026-05-05 compared with 2025-11-04 · 244 added, 296 removed, 335 unchanged (62% of the section changed)

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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, 2025 filed with the SEC on March 3, 2026.

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.

Our financial results for the three months ended March 31, 2026 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 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.

We usually use words such as “may,” “will,” “would,” “should,” “could,” “expect,” “plan,” “anticipate,” “might,” “believe,” “estimate,” “predict,” “intend,” or the negative of these terms or similar expressions to identify these forward-looking statements.

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our business and scientific strategies;

risks and uncertainties associated with the commercialization, distribution and marketing of our products in the US and outside the US;

risks and uncertainties associated with the commercialization, distribution, marketing, and payment for our products in the US and outside the US;

risks that the FDA, EMA, the Medicines and Health Products Regulatory Agency (MHRA) or other regulatory authorities may make adverse decisions regarding our products;

the impact of the US federal government shutdown;

the impact of the US federal government shutdowns or agency funding disruptions;

the progress of our and our collaborators’ product development programs, including clinical testing, and the timing of results thereof;

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our expectations with respect to timing of recognizing product sales;

our expectations with respect to the volume of product sales;

our expectations with respect to regulatory submissions and approvals;

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We focus on products that address signaling pathways that are critical to disease mechanisms.

TAVALISSE (fostamatinib disodium hexahydrate) is our first product approved by the FDA.

TAVALISSE is the only approved oral SYK inhibitor for the treatment of adult patients with chronic ITP who have had an insufficient response to a previous treatment.

The product is also commercially available in Europe and the UK (as TAVLESSE), and in Canada, Israel, Japan and Korea (as TAVALISSE) for the treatment of chronic ITP in adult patients.

REZLIDHIA (olutasidenib) is o ur second FDA-approved product.

REZLIDHIA capsules are indicated for the treatment of adult patients with R/R AML with a susceptible IDH1 mutation as detected by an FDA-approved test.

W e in-licensed REZLIDHIA from Forma with exclusive, worldwide rights for its development, manufacturing and commercialization.

GAVRETO (pralsetinib) is our third FDA-approved product which we began commercializing on June 27, 2024.

TAVALISSE (fostamatinib disodium hexahydrate) is our first FDA-approved product and is the only approved oral SYK inhibitor for the treatment of adult patients with chronic ITP who have had an insufficient response to a previous treatment.

The product is also commercially available in Europe and the UK (as TAVLESSE), and in Japan, Korea, Canada and Israel (as TAVALISSE) for the treatment of chronic ITP in adult patients.

REZLIDHIA (olutasidenib) is our second FDA-approved product indicated for the treatment of adult patients with R/R AML with a susceptible IDH1 mutation as detected by an FDA-approved test.

W e in-licensed REZLIDHIA from Forma with exclusive, worldwide rights for its development, manufacturing and commercialization, pursuant to a license and transition services agreement entered in July 2022.

GAVRETO (pralsetinib) is our third FDA-approved product which we began commercializing in June 2024.

GAVRETO is a once daily, small molecule, oral, kinase inhibitor of wild-type RET and oncogenic RET fusions.

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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.

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.

We also have a RIPK1 inhibitor program in clinical development with our partner Lilly.

Our development pipeline includes R289, our dual IRAK1/4 inhibitor program, which is being advanced 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.

To expand our evaluation of olutasidenib in other disease areas with IDH1 mutations, we have strategic development collaborations with MDACC and with CONNECT.

Business Updates

TAVALISSE IN ITP

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.

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

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.

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

We began our commercialization and started recognizing revenue from product sales of GAVRETO in late June 2024.

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.

Commercial Products

TAVALISSE net product sales for the three months ended March 31, 2026 were $37.3 million, an increase of $8.8 million, or 31%, compared to $28.5 million for the same period in 2025.

The increase was primarily driven by higher volumes and higher price per bottle, as well as a favorable impact from lower revenue reserves.

REZLIDHIA net product sales for the three months ended March 31, 2026 were $8.0 million , an increase of $1.9 million, or 31%, compared to $6.1 million for the same period in 2025 .

The increase was primarily driven by higher volumes and higher price per bottle, partially offset by higher revenue reserves.

GAVRETO net product sales for the three months ended March 31, 2026 were $9.6 million , an increase of $0.6 million, or 7%, compared to $9.0 million for the same period in 2025.

The increase was primarily driven by higher price per bottle and, to a lesser extent, higher volumes.

R289 , an Oral IRAK1/4 Inhibitor for Lower-Risk MDS

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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.

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).

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 patients in an exploratory cohort evaluating post- or ineligible erythropoiesis-stimulating agent (ESA), treatment naïve patients).

The primary objective of the study is safety, with secondary and exploratory objectives to assess prel iminary efficacy and characterize the pharmacokinetic and pharmacodynamic profile of R289.

Enrollment in the dose escalation part of the study was completed in July 2025.

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.

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.

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.

The median duration of RBC-TI was 29 weeks (range 12.7-51.9 weeks).

The three patients that achieved RBC-TI had peak hemoglobin increases exceeding 2.0 g/dL compared to baseline.

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.

Accordingly, this patient was subsequently determined to be a non-responder.

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.

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.

R289 continues to be generally well tolerated and demonstrate preliminary signs of efficacy in doses equal to or above 500 mg daily.

As of the July 15, 2025 data cutoff, 33 patients were enrolled in the dose escalation part of the study.

Patients had a median age of 75 with a median of 3 prior therapies and 61% were high transfusion burden at baseline.

Patients received R289 at doses ranging from 250 mg once daily to 500 mg twice daily.

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.

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.

1 dose limiting toxicity (DLT) (Grade 4 aspartate aminotransferase (AST) increase/Grade 3 ALT increase) was reported in the 750 mg dose group.

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]).

Duration of RBC-TI was >16 weeks in 3 patients, >24 weeks in 2 patients, and >12 months in 1 patient.

The median time to onset of RBC-TI was 2.2 months, and the median duration of RBC-TI was 24.3 weeks.

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).

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.

In October 2025, we announced enrollment of the first patient in the dose expansion part of the study, where up to 40 patients will be randomized to receive either 500 mg once daily or twice daily to determine the recommended Phase 2 dose for future clinical studies.

Enrollment is ongoing and we expect to

complete enrollment of the dose expansion phase of the Phase 1b study and select the recommended Phase 2 dose for future clinical studies in the second half of 2026.

We anticipate sharing preliminary data from the dose expansion phase of the study by the end of 2026.

Olutasidenib in AML, Other Hematologic Cancers and HGG

In December 2023, we entered into a Strategic Collaboration Agreement with MDACC, a comprehensive cancer research, treatment, and prevention center.

The collaboration will expand our evaluation of olutasidenib in AML and other hematologic cancers with IDH1 mutations.

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.

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.

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.

We have a Strategic Collaboration Agreement with MDACC, a comprehensive cancer research, treatment, and prevention center.

The collaboration expanded our evaluation of olutasidenib in AML and other hematologic cancers with IDH1 mutations.

Under the Strategic Collaboration Agreement, we 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.

The collaboration also supports 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.

Further, this collaboration also supports the evaluation of olutasidenib in combination with co-targeted therapies in patients with R/R IDH1 -mutated myeloid malignancies harboring activated signaling pathway mutations.

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.

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.

Through March 31, 2026 , we provided $5.3 million funding to MDACC.

We also have a 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.

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Under the collaboration, we will provide CONNECT with funding up to $3.0 million and study material over the four-year collaboration.

The first patient was enrolled in the Phase 2 TarGet-D study in October 2025.

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.

Enrollment in the Phase 2 TarGet-D study is ongoing.

Global Strategic Partnership with Lilly

Lilly is continuing to advance ocadusertib (previously R552), an investigational, potent and selective RIPK1 inhibitor.

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 .

We entered into a global exclusive and strategic collaboration with Lilly in February 2021 to develop and commercialize ocadusertib (previously R552), an investigational, potent and selective RIPK1 inhibitor, for the treatment of non-CNS diseases, and additional RIPK1 inhibitors for the treatment of CNS diseases.

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.

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.

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.

Following us providing the first opt-out notice to Lilly in September 2023, our cost share obligation for ocadusertib development ended on April 1, 2024.

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 had 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.

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.

As such, we released the $40.0 million remaining cost share liability and recognized the amount as contract revenues from collaboration in the second quarter of 2025.

Patent Infringement Lawsuit

In March 2025, we entered into a settlement agreement with Annora Pharma Private Ltd., Hetero Labs Ltd., and Hetero USA, Inc.

(collectively, Annora), resolving patent litigation related to our product TAVALISSE.

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.

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.

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.

On April 16, 2026, we received written notice from Lilly of its decision to terminate the Lilly Agreement, which will become effective June 15, 2026.

Following termination of the Lilly Agreement, including the prior termination of the CNS disease program effective in November 2025, we do not expect to receive future milestones or royalties under the Lilly Agreement.

Pursuant to such termination, the Lilly Agreement will terminate in accordance with its terms, including the cessation of Lilly’s rights to the licensed compounds, subject to any applicable transition provisions.

We expect to regain full rights to the licensed compounds and related programs upon termination.

Credit Agreement with MidCap

On May 5, 2026, we terminated our Credit Agreement with MidCap, which provided for a $60.0 million term loan facility, under which $45.0 million was outstanding a s of March 31, 2026, and repaid all outstanding borrowings thereunder, including applicable fees and expenses.

Concurrently, we entered into a new Credit Agreement with MidCap, which provides for a revolving credit facility with an initial borrowing capacity of $40.0 million and an option to increase to $60.0 million, subject to customary conditions.

Availability under the revolving credit facility is subject to a borrowing base based primarily on eligible accounts receivable and inventory.

The revolving credit facility under the new Credit Agreement has a five-year term and bears interest at a rate equal to one-month SOFR, subject to a 2.00% floor, plus an applicable margin of 4.00%.

The obligations under the revolving credit facility are secured by a first-priority security interest in substantially all of our assets, including our intellectual property.

The revolving credit facility includes customary fees, including an unused commitment fee, administrative fee and prepayment premiums during the initial period.

As of the date of this filing, we had an outstanding borrowing of $8.0 million under the revolving credit facility.

Our Product Portfolio

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The results of our Phase 2 clinical trial, in which fostamatinib was orally administered to 16 adults with chronic ITP, published in Blood , showed that fostamatinib significantly increased the platelet counts of certain ITP patients, including those who had failed other currently available agents.

Our Fostamatinib for Immune Thrombocytopenia (FIT) Phase 3 clinical program had a total of 150 ITP patients which were randomized into two identical multicenter, double-blind, placebo-controlled clinical trials.

Our Fostamatinib for Immune Thrombocytopenia (FIT) Phase 3 clinical program had a total of 150 ITP patients who were randomized into two identical multicenter, double-blind, placebo-controlled clinical trials.

The patients were diagnosed with persistent or chronic ITP, and had blood platelet counts consistently below 30,000 per microliter of blood.

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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.

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.

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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The FDA recently approved the product WAYRILZ TM (Sanofi SA) for the treatment of adults with persistent or chronic ITP.

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.

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 .).

In addition, the availability of generic versions of TPO receptor agonists may further intensify competition and adversely affect the market.

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, which, if approved and allowed to enter the market, could result in significant decreases in the revenue derived from the sale of TAVALISSE and thereby materially harm our business and financial condition.

TAVALISSE Commercial activities, including sales and marketing

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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.

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.

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).

Through ROC, we provide co-pay assistance to qualified, commercially insured patients to help minimize out-of-pocket costs and provide free product to uninsured or under-insured patients who meet certain established clinical and financial eligibility criteria.

Through ROC, we provide co-pay assistance to qualified, commercially insured patients to help minimize out-of-pocket costs and provide free products to uninsured or under-insured patients who meet certain established clinical and financial eligibility criteria.

In addition, ROC is designed to provide reimbursement support, such as information related to prior authorizations, benefits investigations and appeals.

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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 CIS).

Grifols territory includes European Union (EU), the UK, Turkey, the Middle East, North Africa and Russia (including CIS).

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.

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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.

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.

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.

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TAVALISSE is commercially available in Canada and Israel.

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.

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.

In September 2025, Knight received a rejection from Brazilian Health Regulatory Agency (ANVISA) regarding its marketing authorization application for fostamatinib.

Knight filed an appeal which may take up to fourteen months.

We have a commercial license agreement with Knight under which Knight has exclusivity rights to commercialize fostamatinib for approved indications in Latin America, consisting of Mexico, Central and South America, and the Caribbean, and we are responsible for the exclusive manufacture and supply of fostamatinib for all development and commercialization activities under a related supply agreement.

Knight submitted Marketing Authorization Applications (MAAs) in Mexico, Colombia, Brazil, Argentina and Paraguay for fostamatinib for the treatment of adult patients with chronic ITP who had insufficient response to a previous treatment.

In December 2024, Knight announced that TAVALISSE was approved in Mexico for this indication.

REZLIDHIA/Olutasidenib in R/R AML with mIDH1

REZLIDHIA overview

mIDH1 alterations are seen in AML, MDS, glioma, chondrosarcoma, and intrahepatic cholangiocarcinoma.

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.

Olutasidenib, an oral, small molecule drug designed to selectively bind to and inhibit mIDH1, is a treatment option with durable remissions, reduced QTc potential, and a stable pharmacokinetics profile that enables a consistent drug exposure over time.

m IDH1 alterations are seen in AML, MDS, glioma, chondrosarcoma, and intrahepatic cholangiocarcinoma.

It is estimated that there are approximately 1,000 adult patients, a well-identified patient population, with m IDH1 R/R AML, part of an AML market estimated to have an incidence of approximately 22,720 cases in the US in 2026, and an estimated 120,000 cases globally.

Despite having approved treatment options for R/R AML patients who are m IDH1 positive, an unmet need remains.

Olutasidenib, an oral, small molecule drug designed to selectively bind to and inhibit mIDH1, is a treatment option with durable remissions, reduced QTc potential (referring to a lower observed impact on the heart rate–corrected QT interval on electrocardiogram), and a stable pharmacokinetics profile that enables a consistent drug exposure over time.

This targeted agent has the potential to provide therapeutic benefit by reducing 2-hydroxyglutarate levels and restoring normal cellular differentiation.

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The recommended dosage of REZLIDHIA is 150 mg taken orally twice daily until disease progression or unacceptable toxicity.

The FDA approval was based on the NDA for olutasidenib for the treatment of m1DH1 R/R AML submitted by Forma , that had a PDUFA action date for the application of February 15, 2023.

The NDA application was supported with a Phase 2 registrational trial for olutasidenib in mIDH1 R/R AML.

The FDA approval was based on the New Drug Application ( NDA) for olutasidenib for the treatment of m IDH1 R/R AML submitted by Forma , that had a PDUFA action date for the application of February 15, 2023.

The NDA was supported with a Phase 2 registrational trial for olutasidenib in m IDH1 R/R AML.

Interim results from the Phase 2 registrational trial were reported at the American Society of Clinical Oncology (ASCO) annual meeting in June 2021.

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.

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 .

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 m IDH1 , 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.

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 .

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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REZLIDHIA is now included as a recommended targeted therapy for adult patients with R/R AML with IDH1 mutation.

In February 2023, we announced peer-reviewed publication data in Blood Advances , which summarize clinical results from the Phase 2 registrational trial of REZLIDHIA in patients with mIDH1 R/R AML.

In February 2023, we announced peer-reviewed publication data in Blood Advances , which summarize clinical results from the Phase 2 registrational trial of REZLIDHIA in patients with m IDH1 R/R AML.

The published data demonstrate that REZLIDHIA induced durable remissions and transfusion independence with a well-characterized safety profile.

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REZLIDHIA demonstrated both a high rate of response and an extended median duration of complete response of 28.1 months, which is more than a year longer than what is reported with the standard of care.

In June 2023, we announced the second REZLIDHIA publication in Blood Advances , a review article examining the preclinical and clinical development, and the positioning of REZLIDHIA in the mIDH1 AML treatment landscape.

The review concluded that the approval of REZLIDHIA is a critical addition to the mIDH1 AML treatment landscape.

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.

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.

The review concluded that the approval of REZLIDHIA is a critical addition to the m IDH1 AML treatment landscape.

Further, the available data supports the use of REZLIDHIA as monotherapy in R/R AML patients who have failed intensive chemotherapy or venetoclax plus hypomethylating agents combination therapy.

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 m IDH1 AML who are R/R to prior venetoclax-based regimens.

The findings from these analyses suggest that REZLIDHIA alone or in combination with azacitidine demonstrated potential efficacy in patients with AML following failure of venetoclax combination therapy.

In May 2024, we announced the presentation of the five-year results from the registrational Phase 2 trial of REZLIDHIA in R/R mIDH1 AML patients at the 2024 ASCO Annual Meeting and EHA 2024 Hybrid Congress.

The data published reinforces REZLIDHIA’s efficacy in heavily pretreated patients with mIDH1 AML, including those R/R to prior venetoclax.

In May 2024, we announced the presentation of the registrational Phase 2 trial of REZLIDHIA in R/R m IDH1 AML patients at the 2024 ASCO Annual Meeting and EHA 2024 Hybrid Congress.

The data presented reinforces REZLIDHIA’s efficacy in heavily pretreated patients with m IDH1 AML, including those R/R to prior venetoclax.

The safety profile was consistent with what was previously reported.

Further, REZLIDHIA was generally well tolerated in elderly patients with R/R mIDH1 AML and induced durable remissions.

Further, REZLIDHIA was generally well tolerated in elderly patients with R/R m IDH1 AML and induced remissions.

Despite the challenges of treating elderly patients who had already failed prior AML treatment, the results suggest that elderly patients can benefit from therapy with REZLIDHIA.

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.

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.

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 .

REZLIDHIA was also effective in achieving remission in patients with m IDH1 R/R AML and served as a bridging strategy towards potentially curative allogeneic transplantation in a substantial subset of these previously ineligible patients.

Additionally, REZLIDHIA was well tolerated in a subset of patients with myeloproliferative neoplasms m IDH1 AML, a patient population often associated with poor responses to available therapies.

In October 2025, we announced the publication of the final five-year data for REZLIDHIA in patients with R/R m IDH1 AML in the Journal of Hematology and Oncology .

The publication reports the final follow-up analysis of the registrational Phase 2 trial, with an additional two years of efficacy and safety data.

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There is currently one other product approved in the US for patients with IDH1 mutation.

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.

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 m IDH1 R/R AML, including use of venetoclax combinations, hypomethylating agents, other chemotherapy regimens, or investigational agents that may be available to them.

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.

Our commercial effort focuses on growing awareness of REZLIDHIA within key institutions, and among targeted HCPs who manage patients with R/R AML with m IDH1 .

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.

Olutasidenib outside of the US

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.

We have a collaboration and license agreement with Kissei for an exclusive right to develop and commercialize olutasidenib in all human diseases in Japan, Korea and Taiwan.

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.

In November 2024, we entered into a commercial license agreement with Dr.

We also have a commercial license agreement with Dr.

Reddy’s for an exclusive license to develop and commercialize olutasidenib in Dr.

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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).

This indication was approved by the FDA under accelerated approval

based on overall response rate and duration of response.

This indication was approved by the FDA under accelerated approval 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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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.

On December 22, 2025, the FDA notified us of the approval of a Prior Approval supplemental NDA for GAVRETO, which updated the US Prescribing Information to add a boxed warning regarding serious infections, including opportunistic infections.

We previously communicated this risk information to healthcare providers via a Dear Healthcare Provider letter in October 2024.

The FDA also notified us that we have met our postmarketing commitment for GAVRETO from our September 2020 accelerated approval to submit the final report for the AcceleRET-Lung study.

In January 2026, initial data from the TAPISTRY study was presented in a poster presentation at the ASCO Gastrointestinal Cancers Symposium.

TAPISTRY is a Phase 2, global, open-label, multicohort study evaluating the efficacy and safety of pralsetinib in patients with RET fusion-positive solid tumors, including pancreatic, colorectal, and hepatobiliary cancers.

The reported analysis included results from an efficacy-evaluable population of 39 patients, in which pralsetinib demonstrated an overall response rate of 67%.

These results support continued evaluation of pralsetinib in RET fusion-positive tumors beyond lung cancer;

however, further clinical investigation is ongoing, and there can be no assurance that these results will be confirmed in future studies or lead to regulatory approval for additional indications.

In March 2026, we announced the publication of the final data from the registrational trial evaluating pralsetinib for the treatment of patients with metastatic RET fusion-positive NSCLC in the Journal of Clinical Oncology .

The final data, which includes an additional 42 months of follow-up from data previously published, further supports the robust, durable responses with a manageable safety profile and no new safety signals identified in treatment-naïve and previously treated patients with RET fusion-positive NSCLC and advanced or metastatic thyroid carcinoma.

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.

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 granted GAVRE TO 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.

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.

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.

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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In December 2022, we announced the dosing of the first patient.

Enrollment in the dose escalation part of the study was completed in July 2025.

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.

Enrollment in the dose escalation part of the study was completed in July 2025.

Enrollment is ongoing and we expect to complete enrollment of the dose expansion phase of the Phase 1b study and select the recommended Phase 2 dose for future clinical studies in the second half of 2026.

We anticipate sharing preliminary data from the dose expansion phase of the study by the end of 2026.

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.

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 HTB at baseline.

RBC-TI ≥8 weeks was achieved by three patients (1 at 500 mg once daily and 2 at 750 mg once daily);

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Accordingly, this patient was subsequently determined to be a non-responder.

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.

R289 continues to be generally well tolerated and demonstrate preliminary signs of efficacy in doses equal to or above 500 mg daily.

As of the July 15, 2025 data cutoff, 33 patients were enrolled in the dose escalation part of the study.

Patients had a median age of 75 with a median of 3 prior therapies and 61% were high transfusion burden at baseline.

Patients received R289 at doses ranging from 250 mg once daily to 500 mg twice daily.

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.

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.

1 DLT (Grade 4 AST increase/Grade 3 ALT increase) was reported in the 750 mg dose group.

Duration of RBC-TI was >16 weeks in 3 patients, >24 weeks in 2 patients, and >12 months in 1 patient.

In December 2025, we presented the updated data from the dose escalation phase of our ongoing Phase 1b study evaluating R289 in patients with R/R lower-risk MDS, at the 67th ASH Annual Meeting and Exposition.

R289 continued to be generally well tolerated in a heavily pretreated R/R lower-risk MDS patient population, the majority of whom were HTB at baseline.

As of the October 28, 2025 data cutoff, 33 patients were enrolled in the dose escalation part of the study.

Patients had a median age of 75.

The median number of prior therapies was 3 (range:

76% (25) of patients had received luspatercept, 73% (24) had received an ESA, 67% (22) had received an HMA and 6% (2) had received imetelstat.

61% (20) of patients were HTB at baseline.

67% (22) of patients were ring sideroblast negative.

Median duration of treatment was 5.5 months (range:

0.9 - 27.7 months).

R289 was generally well tolerated across all dose groups in this heavily pre-treated lower-risk MDS patient population, the majority of whom were HTB at baseline.

The most common Grade 1/2 treatment-emergent adverse events were diarrhea, constipation and fatigue, increased creatinine, and cough.

The most frequent Grade 3/4 treatment-emergent adverse events were anemia, decreased neutrophil count and pneumonia, and increased ALT and AST.

One dose limiting toxicity (Grade 4 AST increased/Grade 3 ALT increased) was reported in the 750 mg dose group.

For evaluable transfusion dependent patients (≥16 weeks follow up) at dose levels of at least 500 mg once daily and higher, 6/18 (33%) patients achieved durable RBC-TI of >8 weeks (500 mg once daily (1/3), 750 mg once daily (2/5), 500/250 mg once daily (1/5), 500 mg twice daily (2/5)).

Duration of RBC-TI was >16 weeks in 4 patients and >24 weeks in 3 patients.

The median time to onset of RBC-TI was 1.9 months and the median duration of RBC-TI was 22.9 weeks.

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.

In January 2025, the FDA granted R289 orphan drug designation for the treatment of myelodysplastic syndromes .

Peak hemoglobin increases of 2.9 to 6.1 g/dL compared to baseline occurred in patients achieving RBC-TI.

Of the 6 patients achieving RBC-TI, 5 had received an HMA.

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.

We have a strategic collaboration agreement with MDACC entered in December 2023, 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.

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.

The five studies outlined in the multi-year strategic development alliance are open for enrollment.

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.

Five studies in the multi-year strategic development alliance are open for enrollment.

The five studies include, (i) a Phase 1b/2 triplet therapy trial of decitabine and venetoclax in combination with olutasidenib in patients with m IDH1 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;

(ii) a Phase 2 study in patients with IDH1 -mutated CCUS, lower-risk MDS and chronic myelomonocytic leukemia (CMML);

(iii) a Phase 1/2 study of olutasidenib maintenance therapy following an allogeneic stem cell transplant for patients with IDH1 -mutated myeloid malignancies;

(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;

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.

(iv) a Phase 2 study of olutasidenib in combination with hypomethylating agents (HMA) in patients with m IDH1 higher-risk myelodysplastic syndrome (HR-MDS)/ CMML or advanced myeloproliferative neoplasms;

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.

In January 2024, we announced our 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.

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.

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.

The The first patient was enrolled in the Phase 2 TarGet-D study in October 2025.

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.

The first patient was enrolled in the Phase 2 TarGet-D study in October 2025.

Partnered Clinical Programs

Ocadusertib – Lilly

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.

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.

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

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 milademetan).

The worldwide rights to milademetan were out-licensed from Daiichi to Rain Oncology Inc.

(Rain), which is now Pathos Al, Inc.

(Pathos) after Pathos completed the acquisition of Rain in January 2024.

We 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 milademetan).

The worldwide rights to milademetan were out-licensed from Daiichi to Rain Oncology Inc., now Pathos AI, Inc.

Research, Preclinical and Clinical Development Programs

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.

We also maintain leading expertise on specific areas of operation such as inhibition of SYK, IRAK1/4 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.

We also maintain leading expertise on specific areas of operation such as inhibition of SYK, IRAK1/4 and RIPK1 kinases and m IDH1 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.

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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.

We also have strategic development collaborations with MDACC and CONNECT to conduct evaluation of olutasidenib in other diseases areas with IDH1 mutations.

Commercialization and Sponsored Research and License Agreements

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Results of Operations

Three Months Ended September 30,

Nine Months Ended September 30,

(in thousands)

The following table summarizes revenues for the periods presented (in thousands):

Three Months Ended March 31,

2026 2025 Change

Product sales, net $ 54,923 $ 43,550 $ 11,373

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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:

Three Months Ended September 30,

Nine Months Ended September 30,

Three Months Ended March 31,

McKesson Corporation 48 % 42 %

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Cardinal Health, Inc.

______________________________________________________________________

* Denotes less than 10%

Decrease for Optime Care, Inc.

was due to change in distribution channel

Revenue from product sales is related to our sale of our products in the US, net of chargebacks, discounts and fees, government and other rebates and returns.

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.

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.

The increase was primarily due to increased quantities sold and higher price per bottle, and partly due to benefit from lower revenue reserves.

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.

The increase was primarily due to increased quantities sold and higher price per bottle, partially offset by higher revenue reserves.

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.

We started recognizing GAVRETO net product sales

following the commercialization in late June 2024.

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.

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;

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;

and revenue from Medison of $0.2 million and $0.8 million, respectively, related to earned royalty and delivery of drug supply.

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.

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;

revenue from Grifols of $3.3 million and $5.5 million, respectively, related to earned royalty and delivery of drug supply;

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.

Our net product sales may be impacted by the demand from our customers, changes to government and private payor rebate programs, chargeback and discount programs, co-payment assistance programs, and any other rebate and discount programs we may enter in the future.

Typically, our first quarter net sales are impacted by the first quarter reimbursement issues such as the resetting of deductibles, co-pays, and other access delays for Medicare patients with plan changes that take effect in January.

Consistent with this pattern, our first quarter 2026 net product sales reflected the impact of these seasonal factors, which primarily affected January and February volumes.

We observed improving demands through the quarter, with March showing stronger prescription volumes across our product portfolio.

TAVALISSE net product sales for the three months ended March 31, 2026 were $37.3 million, an increase of 31%, compared to $28.5 million for the three months ended March 31, 2025.

The increase was primarily driven by higher volumes and higher price per bottle, as well as a favorable impact from lower revenue reserves.

REZLIDHIA net product sales for the three months ended March 31, 2026 were $8.0 million, an increase of 31%, compared to $6.1 million for the three months ended March 31, 2025.

The increase was primarily driven by higher volumes and higher price per bottle, partially offset by higher revenue reserves.

GAVRETO net product sales for the three months ended March 31, 2026 were $9.6 million, an increase of 7%, compared to $9.0 million for the three months ended March 31, 2025.

The increase was primarily driven by higher price per bottle and, to a lesser extent, higher volumes

Contract revenues from collaborations for the three months ended March 31, 2026 and 2025 was primarily of revenue from Grifols, Kissei and Medison.

Revenue from Grifols was $1.8 million and $4.7 million for the three months ended March 31, 2026 and 2025, respectively, consisting entirely of royalties in 2026 and, in 2025, both royalties and delivery of drug supplies.

Revenue from Kissei was $1.8 million and $4.6 million, respectively for the three months ended March 31, 2026 and 2025, respectively, consisting entirely of delivery of drug supplies in 2026 and, in 2025, both delivery of drug supply and a $3.0 million milestone payment recognized in connection with the approval of fostamatinib in Korea.

Revenue from Medison was $0.3 million and $0.4 million for the three months ended March 31, 2026 and 2025, respectively, consisting of royalties and delivery of drug supplies.

We expect that revenue from product sales to increase in the coming quarters due to moving past the seasonal reimbursement issues, as well as continued execution of our commercial strategy.

However, net product sales may be impacted by the demand from our customers, changes to government and private payor rebate programs, chargeback and discount programs, co-payment assistance programs, and any other rebate and discount programs we may enter in the future.

In addition, our future revenues may include payments from our existing and new collaboration partners and government grants.

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.

As of March 31, 2026, 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

Three Months Ended September 30,

Nine Months Ended September 30,

(in thousands)

The following table summarizes cost of product sales for the periods presented (in thousands):

Three Months Ended March 31,

2026 2025 Change

Cost of product sales $ 4,606 $ 4,409 $ 197

The cost of product sales includes the cost of inventories sold to our customers and to our collaborative partners.

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.

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.

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.

Certain inventories sold in prior periods were acquired or produced before FDA approval and therefore did not reflect full production costs, as pre-approval manufacturing costs were previously expensed to research and development.

Specifically, we utilized zero-cost API inventory for TAVALISSE, which reduced cost of product sales in those periods.

As post-approval inventory is acquired or produced, inventory and cost of product sales reflect the full manufacturing cost.

We rely and will continue to rely on certain third parties, including those located outside the US to manufacture our products.

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Cost of product sales also includes amortization of intangible assets and royalties.

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.

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.

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

olutasidenib to Kissei.

Additionally, amortization expense increased by $0.2 million.

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.

The increase in cost of product sales for the three months ended March 31, 2026, compared to the same period in 2025, was primarily driven by higher royalties of $0.5 million.

This increase was partially offset by a $0.3 million decrease in product costs, primarily due to the timing of drug supply deliveries to collaboration partners, partially offset by higher product costs associated with increased product sales.

Research and Development Expense

Three Months Ended September 30,

Nine Months Ended September 30,

(in thousands)

The following table summarizes research and development expense for the periods presented (in thousands):

Three Months Ended March 31,

2026 2025 Change

Research and development expense $ 11,676 $ 8,436 $ 3,240

Stock-based compensation expense included in research and development expense $ 441 $ 872 $ (431)

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.

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.

The increase in research and development expense in the three months ended March 31, 2026 compared to the same period in 2025, was primarily due to increased clinical trial related expenses of $2.6 million resulting from the timing of clinical development programs, including the progress activities on our ongoing IRAK1/4 inhibitor program, and increase in other various research and development expenses of $0.7 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.

W e expect to continue to incur significant research and development expense as we continue our activities in our clinical studies including IRAK1/4 inhibitor program;

our collaborative partnerships with MDACC and CONNECT to evaluate olutasidenib in AML, other hematologic cancers and glioma;

our collaborative partnerships with MDACC and CONNECT to conduct evaluation of olutasidenib in other diseases areas with IDH1 mutations;

and any other clinical programs we may pursue in the future.

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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.

Significant delays in clinical testing could materially impact our product development

costs and timing of completion of the clinical trials.

Significant delays in clinical testing could materially impact our product development 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.

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The following table presents our total research and development expense by category (in thousands).

Three Months Ended September 30,

Nine Months Ended September 30,

From January 1, 2007*

to September 30, 2025

Three Months Ended March 31, From January 1, 2007*

2026 2025 to March 31, 2026

Research $ 30 $ 733 $ 271,148

Development 11,109 6,715 623,651

Other 537 988 282,333

$ 11,676 $ 8,436 $ 1,177,132

______________________________________________________________________

* We started tracking research and development expense by category on January 1, 2007.

“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.

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.

“Other” expenses in the three months ended March 31, 2026 and 2025 consisted of allocated facilities costs of $0.1 million and $0.1 million, respectively, and stock-based compensation expense of $0.4 million and $0.9 million , respectively.

Selling, General and Administrative Expense

Three Months Ended September 30,

Nine Months Ended September 30,

(in thousands)

The following table summarizes selling, general and administrative expense for the periods presented (in thousands):

Three Months Ended March 31,

2026 2025 Change

Selling, general and administrative expense $ 30,651 $ 27,715 $ 2,936

Stock-based compensation expense included in selling, general and administrative expense $ 3,015 $ 2,452 $ 563

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.

These increases were partially offset by decrease in other various sales, general and administrative expenses of $0.5 million.

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 .

The increase in selling, general and administrative expense in the three months ended March 31, 2026 compared to the same period in 2025 was primarily due to a $1.6 million increase in commercial related expenses and a $1.7 million increase in personnel-related costs.

These increases were partially offset by decrease in vari ous sales, general and administrative expenses of $0.4 million.

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.

These engagements have enabled our field team to cover existing prescribers, as well as develop relationships with new prescribers to identify appropriate patients for our products.

Interest Income and Interest Expense

Three Months Ended September 30,

September 30,

(in thousands)

The following table summarizes interest income and expense for the periods presented (in thousands):

Three Months Ended March 31,

2026 2025 Change

Interest income $ 1,205 $ 591 $ 614

Interest expense $ (1,433) $ (1,853) $ (420)

Interest income comprised interest on our cash and investment balances.

Interest expense comprised interest on our outstanding term loans with MidCap.

Three Months Ended September 30,

Nine Months Ended September 30,

(in thousands)

(Benefit from) provision for income taxes

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.

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.

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.

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.

In July 2025, the OBBBA was signed into law.

The OBBBA introduces a wide range of provisions affecting business entities, including the establishment of certain permanent business tax measures.

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.

In accordance with ASC 740, Income Taxes , the effects of changes in tax laws are recognized in the period of enactment.

Accordingly, we accounted for the estimated impact of the OBBBA in our current period tax provision.

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.

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.

This assessment requires significant judgment.

In making this determination, all available evidence, both positive and negative, is considered to determine whether, based on the weight of that evidence, a valuation allowance for deferred tax assets is needed.

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.

Interest income reflects returns earned on our cash and investment holdings, while interest expense relates to borrowing costs on our outstanding term loans with MidCap.

The increase in interest income for the three months ended March 31, 2026, compared to the same period in 2025, was primarily driven by higher average investment balances, partially offset by lower interest rates.

The decrease in interest expense for the three months ended March 31, 2026, compared to the same period in 2025, was primarily due to scheduled principal payments that reduced our outstanding debt balance, as well as impact of lower interest rates.

The following table summarizes income tax for the periods presented (in thousands):

Three Months Ended March 31,

2026 2025 Change

Provision for income taxes $ 3,003 $ 65 $ 2,938

The quarterly provision for income taxes is determined by applying the estimated annual effective tax rate to the year-to-date pre-tax income, adjusted for any discrete items.

The estimated annual effective tax rate is updated at the end of each reporting period.

The provision for income taxes for the three months ended March 31, 2026 primarily consisted of federal income tax expense of $2.4 million and estimated state income taxes of $0.6 million .

Prior to the fourth quarter of 2025, we maintained a full valuation allowance against our deferred tax assets.

Although we do not expect to incur federal cash income taxes due to sufficient NOL and research and development credit carryforwards, we recognized federal income tax expense based on the estimated impact of utilizing the deferred tax assets associated with such carryforwards.

The total tax expense differs from the amount computed at the federal statutory rate primarily due to certain non-deductible expenses and state income taxes.

For the three months ended March 31, 2025, the provision for income taxes primarily consisted of estimated state income taxes.

The tax expense differs from the amount computed at the federal statutory rate primarily due to the impact of the valuation allowance and state taxes.

Critical Accounting Policies and Use of Estimates

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Liquidity and Capital Resources

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.

As of March 31, 2026 and December 31, 2025, we had approximately $146.7 million and $155.0 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.

Cash in excess of immediate requirements is invested with regard to liquidity and capital preservation.

Cash in excess of immediate requirements is invested with a focus to liquidity and capital preservation.

We view our investments portfolio as available-for-sale and are available for use in current operations.

Wherever possible, we seek to minimize the potential effects of concentration and degrees of risk.

We continue to monitor the impact of the changes in the conditions of the credit and financial markets to our investment portfolio and assess if future changes in our investment strategy are necessary.

The following table summarizes our cash flow activity for the periods presented:

Nine Months Ended September 30,

(in thousands)

We continue to monitor the impact of the changes in the

conditions of the credit and financial markets on our investment portfolio and assess if future changes in our investment strategy are necessary.

The following table summarizes our cash flow activity for the periods presented (in thousands):

Three Months Ended March 31,

Net cash provided by (used in):

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Financing activities (10,919) 484

Net (decrease) increase in cash, cash equivalents and restricted cash

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.

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.

These were partially offset by increased liabilities driven by the timing of payments.

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.

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.

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.

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 .

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.

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.

Net decrease in cash, cash equivalents and restricted cash $ (16,169) $ (10,961)

Net cash provided by operating activities for the three months ended March 31, 2026 reflected net income, adjusted for non-cash items, partially offset by net cash outflows from changes in working capital.

These outflows were primarily driven by decreases in liabilities due to the timing of payments, increases in prepaid and other current assets mainly due to the timing of advance payments to contract manufacturers and higher inventory levels due to the timing of production build-up, partially offset by lower accounts receivable due to the timing of collections.

In comparison, net cash used in operating activities for the three months ended March 31, 2025 reflected net income, adjusted for non-cash items, offset by net cash outflows from changes in working capital.

These outflows were primarily driven by similar factors, including increases in prepaid and other current assets and decreases in liabilities, partially offset by lower inventory levels.

Net cash used in investing activities for the three months ended March 31, 2026 and 2025 consisted of net purchases of short-term investments of $8.0 million and $10.6 million , respectively.

Net cash used in financing activities for the three months ended March 31, 2026 consisted primarily of $7.5 million of principal payments of term loans and $3.7 million of repurchases of common stock in connection with employee tax withholding on RSU vesting, partially offset by $0.3 million of net proceeds from the issuance of common stock under equity plans.

Net cash provided by financing activities for the three months ended March 31, 2025 consisted of $0.5 million of net proceeds from issuance of common stock under equity plans .

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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We finance our operations primarily through sales of our products, and contract payments under our collaboration agreements, as well as through equity securities and debt financing.

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.

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.

Under our existing collaboration agreements entered in the ordinary course of business, we have received, and may in the future receive upfront cash payments, payments contingent upon the achievement of specified events by our partners, and royalties on net sales of products sold by such partners under the agreements.

The total potential future contingent payments due to us under these agreements are approximately $657.1 million.

This amount accounts for terminated programs and Lilly’s April 2026 notice to terminate the Lilly Agreement and assumes achievement of all applicable milestones under the existing agreements.

The estimate excludes any potential royalties that may be payable to us if our partners successfully commercialize licensed products.

Potential future milestone payments under these agreements are contingent solely upon our partners’ future efforts and the achievement of specified development, regulatory, and commercial milestones.

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.

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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.

As of September 30, 2025, we have not sold any shares of common stock under such Open Market Sale Agreement.

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.

As of March 31, 2026 , we have not sold any shares of common stock under such Open Market Sale Agreement.

We have a Credit Agreement with MidCap that provided for $60.0 million term loan credit facility, under which $45.0 million was outstanding as of March 31, 2026 .

On May 5, 2026, we terminated the Credit Agreement and repaid all outstanding borrowings thereunder, including applicable prepayment premiums, accrued interest and final payment fees, using cash on hand.

Concurrently, we entered into a new Credit Agreement with MidCap, which provides for a revolving credit facility with a maximum borrowing capacity of $40.0 million, with an option to increase to $60.0 million, subject to customary conditions.

Availability under the revolving credit facility is subject to a borrowing base based primarily on eligible accounts receivable and inventory.

While the revolving credit facility enhances our financial flexibility to support operations and working capital needs, our liquidity is dependent on the level of borrowing base availability.

As of the date of this filing, we had an outstanding borrowing of $8.0 million under the revolving credit facility.

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.

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

However, certain external factors such as global geopolitical tensions, political and economic legislations, lingering economic effects of the global pandemic, and other factors may continue to rapidly evolve which could significantly disrupt the global financial markets.

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.

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We have agreements with certain clinical research organizations to conduct our clinical trials including our strategic development collaborations with MDACC and CONNECT.

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.

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.

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In the ordinary course of business, we enter into agreements with contract manufacturers to manufacture our inventory products.

Although the agreements generally provide a termination clause with or without cause, we may still be subjected to payment of cancellation fees.

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 the work order.

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.

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.

As of September 30, 2025, we have not incurred any cancellation fees under our agreements with contract manufacturers.

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.

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.

These agreements generally include termination provisions that may require us to pay cancellation fees, which vary depending on the timing of termination and may equal up to the full value of the work order.

In October 2024, we entered into an agreement with a third-party contract manufacturer to manufacture TAVALISSE, with deliveries expected from 2026 through 2029.

As of March 31, 2026, the contractual obligation not included in our financial statements related to an agreement that may potentially be subjected to cancellation fees were approximately $19.2 million, of which, $5.4 million is expected to be due in the remainder of 2026, and $9.5 million is expected to be due in 2027 and 2028.

As of March 31, 2026, we have not incurred any cancellation fees under our agreements with contract manufacturers.

Also, 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 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.

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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.

We have a contractual commitment with respect to our credit facility with MidCap.

Under the amended Credit Agreement, the term loans mature on September 1, 2027, and the interest-only period is through October 1, 2025.

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.

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.

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.

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.

As discussed above, in connection with the termination of our Credit Agreement with MidCap and entry into the new Credit Agreement, we repaid all outstanding amounts under the term loan facility on May 5, 2026, which was scheduled to mature on September 1, 2027 and had an outstanding principal balance of $45.0 million as of March 31, 2026;

such repayment included all outstanding principal, together with applicable prepayment premiums, accrued interest and final payment fees.

The revolving credit facility under the new Credit Agreement has a five-year term and bears interest at a rate equal to one-month SOFR, subject to a 2.00% floor, plus an applicable margin of 4.00%.

The obligations under the revolving facility is secured by substantially all of our assets, including our intellectual property.

As of March 31, 2026, we have a contractual commitment related to our leased facility, which lease will expire in July 2027.

As of March 31, 2026, our contractual commitment related to the lease agreements was $0.9 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, 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.

determination of the amount of reserves required, if any, for these contingencies is made after careful analysis of each individual matter.

A 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.

What changed 10-Q

Item 2. Management’s Discussion and Analysis