What changed in this filing

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Item 1. Financial Statements

2025-08-05 compared with 2025-05-06 · 157 added, 127 removed, 483 unchanged (37% of the section changed)

3 unchanged sentences

(In thousands)

March 31, 2025

June 30, 2025

December 31, 2024 (1)

13 unchanged sentences

Accrued research and development

Acquisition-related liabilities

Revenue reserves and refund liability

11 unchanged sentences

Additional paid-in capital

Accumulated other comprehensive (loss) income

Accumulated other comprehensive income

Accumulated deficit

8 unchanged sentences

(In thousands, except per share amounts)

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

Product sales, net

14 unchanged sentences

Weighted average shares used in computing net income (loss) per share

(1) Share and per share amounts have been restated to reflect the 1-for-10 reverse stock split effected on June 27, 2024 on a retroactive basis for prior period presented.

See Accompanying Notes to Condensed Financial Statements

2 unchanged sentences

(In thousands)

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

Net income (loss)

Other comprehensive loss:

Net unrealized loss on short-term investments

Other comprehensive income (loss):

Net unrealized income (loss) on short-term investments

Comprehensive income (loss)

15 unchanged sentences

( 1,378,619 )

Net change in unrealized gain on short-term investments

Issuance of common stock upon exercise of options, net of shares withheld, and participation in Purchase Plan

Issuance of common stock upon vesting of RSUs

Stock-based compensation expense

Balance as of June 30, 2025

( 1,319,006 )

Accumulated Other

Common Stock (1)

Comprehensive

9 unchanged sentences

( 1,415,797 )

(1) All share amounts in this column, including appropriate reclassifications between common stock and additional paid-in capital, have been restated to reflect the 1-for-10 reverse stock split effected on June 27, 2024 on a retroactive basis.

Net change in unrealized loss on short-term investments

Issuance of common stock upon exercise of options and participation in Purchase Plan

Issuance of common stock upon vesting of RSUs

Stock-based compensation expense

Balance as of June 30, 2024

( 1,416,827 )

See Accompanying Notes to Condensed Financial Statements

2 unchanged sentences

(In thousands)

Three Months Ended March 31,

Six Months Ended June 30,

Operating activities

Net income (loss)

Adjustments to reconcile net income (loss) to net cash used in operating activities:

Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities:

Stock-based compensation expense

Depreciation and amortization

Release of cost share liability

Net amortization of discount on short-term investments and term loans

9 unchanged sentences

Lease liability

Net cash used in operating activities

Net cash provided by (used in) operating activities

Investing activities

7 unchanged sentences

Net cash provided by (used in) financing activities

Net decrease in cash, cash equivalents and restricted cash

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

Cash and cash equivalents at beginning of period

14 unchanged sentences

TAVALISSE is the only approved oral spleen tyrosine kinase (SYK) inhibitor for the treatment of adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

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

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

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

5 unchanged sentences

GAVRETO is also approved under accelerated approval based on overall response rate and duration response rate, 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).

We acquired the rights to research, develop, manufacture and commercialize GAVRETO in the US from Blueprint Medicines Corporation (Blueprint) pursuant to an Asset Purchase Agreement entered in February 2024.

We acquired the rights to research, develop, manufacture and commercialize GAVRETO in the US from Blueprint Medicines Corporation, now a Sanofi SA company (Blueprint), pursuant to an Asset Purchase Agreement entered in February 2024.

We continue to advance the development of R289, our dual interleukin receptor-associated kinases 1 and 4 (IRAK 1/4) inhibitor program, in an open-label, Phase 1b study to determine the tolerability and preliminary efficacy of the drug in patients with lower-risk myelodysplastic syndrome (MDS) who are relapsed, refractory or resistant to prior therapies.

14 unchanged sentences

Our significant accounting policies are described in “Note 1 – Description of Business and Summary of Significant Accounting Policies” to our “Notes to Financial Statements” contained in Part II, Item 8, “Financial Statements and Supplementary Data” of our Annual Report on Form 10-K for the year ended December 31, 2024.

As of March 31, 2025, we had approximately $ 77.1 million in cash, cash equivalents and short-term investments.

As of June 30, 2025, we had approximately $ 108.4 million in cash, cash equivalents and short-term investments.

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.

13 unchanged sentences

The following table sets forth the computation of basic and diluted earnings per share (in thousands except per share amounts):

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

EPS Numerator:

8 unchanged sentences

The potential shares of common stock that were excluded from the computation of diluted net income (loss) per share for the periods presented because including them would have been antidilutive are as follows (in thousands):

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

Stock options

Shares under Purchase Plan

Revenues disaggregated by category were as follows (in thousands):

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

Product sales:

3 unchanged sentences

Revenues from collaborations:

Release of cost share liability

Milestone revenue

5 unchanged sentences

Our net product sales include gross product sales, net of chargebacks, discounts and fees, government and other rebates and returns.

Of the total discounts and allowances from gross product sales for the three months ended March 31, 2025 and 2024, $ 16.1 million and $ 12.3 million, respectively, was accounted for as additions to revenue reserves and refund liability, and $ 0.5 million and $ 0.1 million, respectively, as reductions in accounts receivable (as it relates to allowance for prompt pay discount) and prepaid and other current assets (as it relates to certain chargebacks and other fees that were prepaid) in the condensed balance sheet.

Of the total discounts and allowances from gross product sales for the six months ended June 30, 2025 and 2024, $ 36.5 million and $ 27.6 million, respectively, was accounted for as additions to revenue reserves and refund liability, and $ 0.9 million and $ 0.4 million, respectively, as reductions in accounts receivable (as it relates to allowance for prompt pay discount) and prepaid and other current assets (as it relates to certain chargebacks and other fees that were prepaid) in the condensed balance sheet.

The following tables summarize the activities in chargebacks, discounts and fees, government and other rebates and returns that were accounted for within revenue reserves and refund liability, for each of the periods presented (in thousands):

4 unchanged sentences

Credit or payments made during the period

Balance as of March 31, 2025

Balance as of June 30, 2025

Discounts and

2 unchanged sentences

Credit or payments made during the period

Balance as of March 31, 2024

Balance as of June 30, 2024

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 March 31,

Three Months Ended June 30,

Six Months Ended June 30,

McKesson Corporation

(formerly ASD Healthcare)

Optime Care, Inc.

Cencora, Inc.

Cardinal Health, Inc.

3 unchanged sentences

We conduct research and development programs independently and in connection with our corporate collaborators.

As of March 31, 2025, we are a party to collaboration agreements with Lilly to develop and commercialize ocadusertib (previously R552), a RIPK1 inhibitor, for the treatment of non-central nervous system (non-CNS) diseases and collaboration aimed at developing additional RIPK1 inhibitors for the treatment of central nervous system (CNS) diseases;

As of June 30, 2025, we are a party to collaboration agreements with Lilly to develop and commercialize ocadusertib (previously R552), a RIPK1 inhibitor, for the treatment of non-central nervous system (non-CNS) diseases and collaboration aimed at developing additional RIPK1 inhibitors for the treatment of central nervous system (CNS) diseases;

with Grifols S.A.

(Grifols) to commercialize fostamatinib for human diseases in all indications in Grifols territory which includes Europe, the UK, Turkey, the Middle East, North Africa and Russia (including Commonwealth of Independent States);

with Kissei Pharmaceutical Co., Ltd.

(Kissei) to develop and commercialize fostamatinib in Japan, China, Taiwan and the Republic of Korea (Korea), and olutasidenib in Japan, Korea and Taiwan;

(Kissei) to develop and commercialize fostamatinib in Japan, China, Taiwan and Korea, and olutasidenib in Japan, Korea and Taiwan;

with Medison Pharma Trading AG (Medison Canada) and Medison Pharma Ltd.

(Medison Israel and, together with Medison Canada, Medison) to commercialize fostamatinib in all indications, in Medison territory which includes Canada and Israel;

with Knight Therapeutics International SA (Knight) to commercialize fostamatinib in all indications, in Knight territory

which includes Latin America, consisting of Mexico, Central and South America, and the Caribbean;

with Knight Therapeutics International SA (Knight) to commercialize fostamatinib in all indications, in Knight territory which

includes Latin America, consisting of Mexico, Central and South America, and the Caribbean;

Reddy’s Laboratories (Dr.

1 unchanged sentence

Reddy’s territory which includes Latin America, South Africa, India, Australia, New Zealand, and certain countries in the CIS, Southeast Asia region and North Africa.

Further, we are also a party to collaboration agreements, but do not have ongoing performance obligations with BerGenBio ASA (BerGenBio) for the development and commercialization of AXL receptor tyrosine kinase i nhibitor, R428 (now referred to as bemcentinib (BGB324)), and with Daiichi Sankyo (Daiichi) to pursue research related to murine double minute 2 ( MDM2) inhibitor, DS-3032 (now referred as melademetan).

Under the above existing agreements that we entered into 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.

As of March 31, 2025, total future contingent payments to us under all of the above existing agreements was approximately $ 1.5 billion, if all potential product candidates achieved all of the payment triggering events under all of our current agreements.

As of June 30, 2025, total future contingent payments to us under all of the above existing agreements was approximately $ 1.5 billion, if all potential product candidates achieved all of the payment triggering events under all of our current agreements.

Of this amount, $ 279.5 million relates to the achievement of development events, $ 310.6 million relates to the achievement of regulatory events and $ 902.0 million relates to the achievement of certain commercial events.

21 unchanged sentences

Pursuant to the terms of 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.

Under the Lilly Agreement, if we exercised our first opt-out right prior to September 30, 2023, we were required to fund our share of the ocadusertib development activities up to a maximum funding commitment of $ 65.0 million through April 1, 2024.

Under the Lilly Agreement, if the first opt-out right was exercised prior to September 30, 2023, we were required to fund our share of the ocadusertib development activities up to a maximum funding commitment of $ 65.0 million through April 1, 2024.

We accounted for this agreement under ASC 606 and identified the following distinct performance obligations at inception of the agreement:

6 unchanged sentences

Interest expense was accreted on such liability over the expected commitment period, adjusted for timing of expected cost share payments.

No interest was accreted during the three months ended March 31, 2025 and 2024.

At the inception, we allocated the net transaction price of $ 67.1 million to each performance obligation based on our best estimate of its relative standalone selling price using the adjusted market assessment approach.

1 unchanged sentence

The transaction price allocated to the CNS penetrant IP of $ 6.7 million was recognized as revenue from the effective date of the Lilly Agreement through the eventual acceptance by Lilly in June 2022 using the input method.

There was no outstanding deferred revenue related to Lilly Agreement as of March 31, 2025 and December 31, 2024.

There was no outstanding deferred revenue related to Lilly Agreement as of June 30, 2025 and December 31, 2024.

In September 2023, we provided our first-opt out notice to Lilly, and concurrently entered into a second amendment to the Lilly Agreement.

The amended Lilly Agreement provided, among others that if we exercise our first opt-out right, we have the right to opt-in to the co-funding of ocadusertib development, upon us providing notice to Lilly within 30 days of certain events as specified in the Lilly Agreement, and as a result receive greater royalties from sales.

If we decide to exercise our opt-in right, we will be required to continue to share in global development costs, capped at a specified amount and for a specified period if we exercise our second opt-out right.

If we exercised our opt-in right, we would be required to continue to share in global development costs, capped at a specified amount and for a specified period if the second opt-out right was exercised.

As discussed above, following us providing the first opt-out notice to Lilly, 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.

As of March 31, 2025 and December 31, 2024, the outstanding liability to Lilly reported within other long-term liabilities in the condensed balance sheets amounted to $ 40.0 million.

Although our cost share obligation for the ocadusertib development ended on April 1, 2024, the outstanding liability reported in our condensed balance sheet as of December 31, 2024 and March 31, 2025 amounting to $ 40.0 million has not been recognized as revenue during the respective periods, because we cannot conclude that it is probable that a significant reversal of the amount of revenue, if recognized, will not occur until the likelihood of us exercising our opt-in right becomes remote, or when the opt-in right period lapses.

As of December 31, 2024, the outstanding liability to Lilly reported within other long-term liabilities in the condensed balance sheets amounted to $ 40.0 million.

Although our cost share obligation for the ocadusertib development ended on April 1, 2024, the remaining cost share liability was not released in the periods prior to us providing the notice to Lilly not to exercise our opt-in right because we were not able to conclude that it was probable that a significant reversal of the amount of revenue, if recognized, would not occur until the likelihood of us exercising our opt-in right became remote, or when the opt-in right period lapsed.

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 currently on our condensed balance sheet.

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 three and six months ended June 30, 2025.

Grifols License Agreement

5 unchanged sentences

We accounted for this agreement under ASC 606, and recognized the corresponding revenue in the period we satisfied the performance obligations.

There was no outstanding deferred revenue related to the Grifols license agreement as of March 31, 2025 and December 31, 2024.

There was no outstanding deferred revenue related to the Grifols license agreement as of June 30, 2025 and December 31, 2024.

We have a commercial supply agreement with Grifols entered in October 2020 to supply and sell our drug product priced at a certain markup specified in the agreement, in quantities Grifols order from us pursuant to and in accordance with the agreement.

For the three months ended March 31, 2025, we recognized $ 3.3 million of revenue related to the delivery of drug supply to Grifols.

No revenue was recognized related to delivery of drug supplies to Grifols during the three months ended March 31, 2024.

For the three months ended March 31, 2025, and 2024, we recognized royalty revenue from Grifols of $ 1.4 million and $ 1.1 million, respectively.

Kissei License Agreement – Olutasidenib

We entered into a collaboration and license agreement with Kissei in September 2024 to grant exclusive rights to Kissei to develop and commercialize olutasidenib in all human diseases in Japan, Korea and Taiwan.

For the three and six months ended June 30, 2025, we recognized revenue of $ 0.4 million and $ 3.7 million, respectively, related to the delivery of drug supply to Grifols.

No revenue was recognized related to delivery of drug supplies to Grifols during the three and six months ended June 30, 2024.

We recognize royalty revenue from Grifols included within contract revenues from collaboration.

Royalty revenue recognized for the three months ended June 30, 2025 and 2024 was $ 1.6 million and $ 1.1 million, respectively, and for the six months ended June 30, 2025 and 2024 was $ 3.1 million and $ 2.2 million, respectively.

Kissei License Agreements

We have a collaboration and license agreement with Kissei entered in September 2024 to grant exclusive rights to Kissei to develop and commercialize olutasidenib in all human diseases in Japan, Korea and Taiwan.

Kissei is responsible for performing and funding the development activities for olutasidenib in the Kissei territory and we retained the co-exclusive right to conduct development activities in the Kissei territory solely for the purpose of supporting and obtaining regulatory approval of and commercializing olutasidenib in the world outside the Kissei territory.

5 unchanged sentences

Accordingly, we recognized the upfront payment as revenue during the third quarter of 2024.

No revenue was recognized during the three months ended March 31, 2025 related to this collaboration and license agreement.

Kissei License Agreement – Fostamatinib

We have an exclusive license and supply agreement with Kissei entered in October 2018, amended in November 2022, October 2023, August 2024, September 2024 and October 2024, to develop and commercialize fostamatinib in all current and potential indications in Japan, China, Taiwan and Korea.

We also have an exclusive license and supply agreement with Kissei entered in October 2018, amended in November 2022, October 2023, August 2024, September 2024 and October 2024, to develop and commercialize fostamatinib in all current and potential indications in Japan, China, Taiwan and Korea.

Kissei is responsible for performing and funding all development activities for fostamatinib in the above-mentioned territories.

1 unchanged sentence

Further, the agreement provides for up to $ 115.0 million in potential development, regulatory and commercial milestone payments, and mid- to upper twenty percent, tiered, escalated net sales-based payments for the supply of fostamatinib.

Under the agreement, we granted

Kissei the license rights to fostamatinib in Kissei’s territory and are obligated to supply Kissei with drug product for use in clinical trials and pre-commercialization activities.

We are also responsible for the manufacture and supply of fostamatinib for all future development and commercialization activities.

In April 2022, Kissei announced that an NDA was submitted to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for fostamatinib in chronic ITP which entitled us to receive a $ 5.0 million non-refundable and non-creditable milestone payment.

In December 2022, Kissei announced that Japan’s PMDA approved the NDA for fostamatinib in chronic ITP, which entitled us to receive a $ 20.0 million non-refundable and non-creditable milestone payment.

Under the agreement, we granted Kissei the license rights to fostamatinib in Kissei’s territory and are obligated to supply Kissei with drug product for use in clinical trials and pre-commercialization activities.

We are also responsible for the manufacture and supply of

fostamatinib for all future development and commercialization activities.

We accounted for this agreement under ASC 606, and recognized the corresponding revenue in the period we satisfied the performance obligations.

As of March 31, 2025 and December 31, 2024, the remaining deferred revenue was related to the material right associated with discounted fostamatinib supply which amounted to $ 1.4 million .

No revenue was recognized during the three months ended March 31, 2025 and 2024 associated with the remaining performance obligation, respectively.

In January 2025, Kissei announced the Korean Ministry of Food and Drug Safety approved fostamatinib for the treatment of chronic ITP, which entitled us to receive a $ 3.0 million non-refundable and non-creditable milestone payment that we recognized as revenue in the three months ended March 31, 2025.

For the three months ended March 31, 2025 and 2024, we recognized revenue from Kissei of $ 1.6 million and $ 2.3 million, respectively, related to the delivery of fostamatinib drug supply for commercial use.

As of June 30, 2025 and December 31, 2024, the remaining deferred revenue was related to the material right associated with discounted fostamatinib supply which amounted to $ 1.4 million .

No revenue was recognized during the three and six months ended June 30, 2025 and 2024 associated with the remaining performance obligation.

In April 2022, Kissei announced that an NDA for fostamatinib in chronic ITP was submitted to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), and in December 2022, Kissei announced that Japan’s PMDA approved the NDA.

Following such milestones, we were entitled to receive a total of $ 25.0 million non-refundable and non-creditable milestone payments that we recognized as revenue in 2022.

Revenue recognized related to the delivery of drug supply to Kissei for the three months ended June 30, 2025 and 2024 was $ 0.4 million and $ 2.2 million, respectively, and for the six months ended June 30, 2025 and 2024 was $ 2.0 million and $ 4.5 million, respectively.

Medison Commercial and License Agreements

6 unchanged sentences

As such, in accordance with ASC 606, we relieved the outstanding financing liability which included the upfront payment and accreted interest, and recognized such amount as revenue in 2022.

There was no outstanding deferred revenue related to Medison commercial and license agreement as of March 31, 2025 and December 31, 2024.

For the three months ended March 31, 2025 and 2024, we recognized revenue from Medison of $ 0.4 million and $ 0.1 million, respectively, related to the delivery of drug supply and earned royalties.

There was no outstanding deferred revenue related to Medison commercial and license agreement as of June 30, 2025 and December 31, 2024.

Revenue recognized from Medison related to delivery of drug supply and earned royalties for the three months ended June 30, 2025 and 2024 was $ 0.2 million and $ 0.1 million, respectively, and for the six months ended June 30, 2025 and 2024 was $ 0.6 million and $ 0.2 million, respectively.

Knight Commercial License and Supply Agreement

4 unchanged sentences

We are also responsible for the exclusive manufacture and supply of fostamatinib for all future development and commercialization activities under the agreement.

No revenue was recognized during the three months ended March 31, 2025 and 2024 from Knight.

No revenue was recognized during the three and six months ended June 30, 2025 and 2024 from Knight.

Reddy’s Commercial License Agreement

11 unchanged sentences

Reddy’s, which revenue was recognized during the fourth quarter of 2024.

No revenue was recognized during the three months ended March 31, 2025 related to the commercial license agreement with Dr.

No revenue was recognized during the three and six months ended June 30, 2025 related to the commercial license agreement with Dr.

Government Contract

In August 2023, we were awarded up to $ 0.8 million by Biomedical Advanced Research and Development (BARDA), part of the Office of the Assistant Secretary for the Preparedness and Response at the US Department of Health and Human Services (DHHS), for our evaluation of fostamatinib in mitigating the impact of long-term respiratory distress.

No revenue was recognized during the three months ended March 31, 2025 and 2024 from this grant.

Through March 31, 2025, we have received $ 0.1 million of the award .

No revenue was recognized during the three and six months ended June 30, 2025 and 2024 from this grant.

Through June 30, 2025, we have received $ 0.1 million of the award .

Strategic Development Collaborations with MDACC and CONNECT

2 unchanged sentences

Under the collaboration, 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 March 31, 2025, we provided $ 5.3 million funding to MDACC.

Through June 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 glioma.

4 unchanged sentences

We acquired the US rights to research, develop, manufacture and commercialize GAVRETO from Blueprint pursuant to an Asset Purchase Agreement entered in February 2024.

The acquired assets include, among other things, applicable intellectual property related to pralsetinib in the US, including patents, copyrights and trademarks, as well as clinical regulatory and commercial data and records.

The acquired assets from Blueprint include, among other things, applicable intellectual property related to pralsetinib in the US, including patents, copyrights and trademarks, as well as clinical regulatory and commercial data and records.

Pursuant to the Asset Purchase Agreement, we agreed to pay a purchase price of $ 15.0 million, of which, $ 10.0 million was payable upon our first commercial sale of GAVRETO and an additional $ 5.0 million is payable on the first anniversary of the closing date of the agreement, subject to certain conditions.

Blueprint is also eligible to receive up to $ 97.5 million in future commercial milestone payments and up to $ 5.0 million in future regulatory milestone payments.

The potential regulatory milestones include full regulatory approval of pralsetinib (or related compounds) for the treatment of adult RET-fusion positive thyroid cancer, and maintenance of the current regulatory approval of pralsetinib for the treatment of adult RET-fusion positive thyroid cancer during the period beginning on February 22, 2024 and ending on the third anniversary of the first commercial sale of pralsetinib subject to certain conditions.

The potential regulatory milestones include

full regulatory approval of pralsetinib (or related compounds) for the treatment of adult RET-fusion positive thyroid cancer, and maintenance of the current regulatory approval of pralsetinib for the treatment of adult RET-fusion positive thyroid cancer during the period beginning on February 22, 2024 and ending on the third anniversary of the first commercial sale of pralsetinib subject to certain conditions.

Subject to the terms and conditions of the Asset Purchase Agreement, Blueprint would be entitled to tiered royalty payments on net sales of products containing pralsetinib (or related compounds) ranging from 10 % to 30 %, subject to certain reductions and offsets.

3 unchanged sentences

Of the total closing purchase price, $ 10.0 million was paid in July 2024.

The remaining $ 5.0 million was outstanding and presented as acquisition-related liabilities in the condensed balance sheet as of March 31, 2025.

As of March 31, 2025 and December 31, 2024, we classified the outstanding acquisition-related liabilities as non-current considering the amount is expected to be payable in a period longer than one year as of balance sheet date.

The remaining $ 5.0 million was outstanding and presented as acquisition-related liabilities in the condensed balance sheet as of June 30, 2025 and December 31, 2024.

We classify the outstanding acquisition-related liabilities as current or non-current liabilities based on the period the amount is expected to be due as of the balance sheet date.

In accordance with the guidance, we classify payments of the closing purchase price under financing activity in the condensed statements of cash flows, considering that the payments are not made soon after the acquisition date.

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In accordance with the guidance, in a transaction accounted for as an asset acquisition, any acquired in-process research and development (IPR&D) that does not have alternative future use is charged to expense at the acquisition date.

acquisition date, the acquired license asset was accounted for as IPR&D, and we anticipated no other economic benefit to be derived from such acquired licensed asset other than the primary indications.

At the acquisition date, the acquired license asset was accounted for as IPR&D, and we anticipated no other economic benefit to be derived from such acquired licensed asset other than the primary indications.

As such, we accounted for the upfront fee of $ 2.0 million as IPR&D and recorded such cost within research and development expense in the statements of operations in 2022.

3 unchanged sentences

Prior to the FDA approval of REZLIDHIA in December 2022, a certain regulatory milestone was met which entitled Forma to receive a $ 2.5 million milestone payment.

Because such milestone payment obligation was incurred prior to a regulatory approval of an indication associated with the acquired licensed asset, we recorded such amount as research and development expense in the fourth quarter of 2022.

Because such milestone payment obligation was incurred prior to a regulatory approval of an

indication associated with the acquired licensed asset, we recorded such amount as research and development expense in the fourth quarter of 2022.

On December 1, 2022, the FDA approved REZLIDHIA capsules for the treatment of adult patients with R/R AML with susceptible IDH1 mutations as detected by an FDA-approved test.

6 unchanged sentences

Stock-based compensation for the periods presented was as follows (in thousands):

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

Selling, general and administrative

1 unchanged sentence

Total stock-based compensation expense

During the three months ended March 31, 2025, we granted stock options to purchase 589,877 shares of common stock with weighted-average grant-date fair value of $ 17.09 per share, and 126,226 stock options were exercised.

The stock options granted during the three months ended March 31, 2025 generally vest over 3 years .

As of March 31, 2025, there were 3,965,254 stock options outstanding, of which, 185,814 are outstanding performance-based stock options wherein the achievement of the corresponding corporate-based milestones were assessed not probable as of March 31, 2025.

Accordingly, none of the $ 3.5 million grant date fair value for these awards has been recognized as stock-based compensation expense as of March 31, 2025.

During the six months ended June 30, 2025, we granted stock options to purchase 619,572 shares of common stock with weighted-average grant-date fair value of $ 16.95 per share, and the grants generally vest over 3 years .

During the six months ended June 30, 2025, 140,126 stock options were exercised.

As of June 30, 2025, there were 3,846,358 stock options outstanding, of which, 168,564 are outstanding performance-based stock options wherein the achievement of the corresponding corporate-based milestones were assessed not probable as of June 30, 2025.

Accordingly, none of the $ 3.2 million grant date fair value for these awards has been recognized as stock-based compensation expense as of June 30, 2025.

The fair value of each option award is estimated on the date of grant using the Black-Scholes option pricing model.

The following table summarizes the weighted-average assumptions relating to options granted pursuant to our Equity Incentive Plans (our 2018 Equity Incentive Plan and Inducement Plan, as amended) for the periods presented:

Three Months Ended March 31,

The following table summarizes the weighted-average assumptions relating to options granted pursuant to our 2018 Equity Incentive Plan (2018 Plan) and our Inducement Plan, as amended (Inducement Plan, and together with 2018 Plan, the Equity Incentive Plans) for the periods presented:

Three Months Ended June 30,

Six Months Ended June 30,

Risk-free interest rate

2 unchanged sentences

Expected volatility

During the three months ended March 31, 2025, we granted 318,262 RSUs with a grant-date weighted-average fair value of $ 22.49 per share, and 125,783 RSUs were released.

The RSUs granted during the three months ended March 31, 2025 generally vest over 3 years .

As of March 31, 2025, there were 568,024 RSUs outstanding.

As of March 31, 2025, there was approximately $ 23.2 million of unrecognized stock-based compensation cost which is expected to be recognized over a remaining weighted-average period of 2.38 years, related to time-based stock options, performance-based stock options wherein achievement of the corresponding corporate-based milestones was considered as probable, and RSUs.

As of March 31, 2025, there were 605,514 shares of common stock available for future grant under our Equity Incentive Plans.

During the six months ended June 30, 2025, we granted 340,817 RSUs with a grant-date weighted-average fair value of $ 19.27 per share, and the grants generally vest over 3 years .

During the six months ended June 30, 2025, 143,691 RSUs were released.

As of June 30, 2025, there were 565,846 RSUs outstanding.

As of June 30, 2025, there was approximately $ 20.5 million of unrecognized stock-based compensation cost which is expected to be recognized over a remaining weighted-average period of 2.19 years, related to time-based stock options, performance-based stock options wherein achievement of the corresponding corporate-based milestones was considered as probable, and RSUs.

During the six months ended June 30, 2025, our Board of Directors approved an additional 23,350 shares of common stock reserved for issuance under our Inducement Plan.

In May 2025, our stockholders approved an amendment to our 2018 Plan to, among other items, add an additional 700,000 shares to the number of shares of common stock authorized for issuance under our 2018 Plan.

As of June 30, 2025, there were 1,396,042 shares of common stock available for future grant under our Equity Incentive Plans.

Employee Stock Purchase Plan

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The fair value of awards under our Purchase Plan is estimated on the date of our new offering period using the Black-Scholes option pricing model, which is being amortized over the requisite service periods.

As of March 31, 2025, there was approximately $ 0.4 million of unrecognized stock-based compensation cost which is expected to be recognized over a remaining weighted-average period of 1.02 years, related to our Purchase Plan.

As of March 31, 2025, there were 184,174 shares reserved for future issuance under the Purchase Plan.

As of June 30, 2025, there was approximately $ 0.2 million of unrecognized stock-based compensation cost which is expected to be recognized over a remaining weighted-average period of 0.81 years, related to our Purchase Plan.

During the six months ended June 30, 2025, there were 39,801 shares purchased under the Purchase Plan.

As of June 30, 2025, there were 144,373 shares reserved for future issuance under the Purchase Plan.

Other Balance Sheet Components

Inventories for the periods presented consist of the following (in thousands):

March 31, 2025

June 30, 2025

December 31, 2024

7 unchanged sentences

Prepaid and other current assets for the periods presented consist of the following (in thousands):

March 31, 2025

June 30, 2025

December 31, 2024

4 unchanged sentences

Intangible assets consist of the following (in thousands):

March 31, 2025

June 30, 2025

December 31, 2024

2 unchanged sentences

Intangible assets, net

Amortization expense recorded within cost of product sales in the condensed statements of operations for the three months ended March 31, 2025 and 2024 was $ 0.6 million and $ 0.4 million, respectively.

The following table presents the estimated future amortization expense of intangible assets as of March 31, 2025 (in thousands):

Amortization expense recorded within cost of product sales in the condensed statements of operations for the three months ended June 30, 2025 and 2024 was $ 0.6 million and $ 0.6 million, respectively, and for the six months ended June 30, 2025 and 2024 was $ 1.2 million and $ 1.0 million, respectively.

The following table presents the estimated future amortization expense of intangible assets as of June 30, 2025 (in thousands):

Remainder of 2025

1 unchanged sentence

Cash, cash equivalents, restricted cash, and short-term investments for the periods presented consist of the following (in thousands):

March 31, 2025

June 30, 2025

December 31, 2024

8 unchanged sentences

Cash equivalents and short-term investments include the following securities with gross unrealized gains and losses (in thousands):

As of March 31, 2025

As of June 30, 2025

US treasury bills

5 unchanged sentences

Corporate bonds and commercial paper

As of March 31, 2025 and December 31, 2024, our cash equivalents and short-term investments had a weighted-average time to maturity of approximately 98 days and 69 days , respectively.

As of June 30, 2025 and December 31, 2024, our cash equivalents and short-term investments had a weighted-average time to maturity of approximately 117 days and 69 days , respectively.

Our short-term investments are classified as available-for-sale securities.

Accordingly, we have classified these securities as short-term investments on our condensed balance sheets as they are available for use in the current operations.

As of March 31, 2025, a total of 19 individual securities had been in an unrealized loss position for 12 months or less, and the losses were determined to be temporary.

As of June 30, 2025, a total of 40 individual securities had been in an unrealized loss position for 12 months or less, and the losses were determined to be temporary.

We regularly review the securities in an unrealized loss position and evaluate the current expected credit loss by considering factors such as historical experience, market data, issuer-specific factors, and current economic conditions.

We have no t recognized any credit losses as of March 31, 2025 and December 31, 2024.

We have no t recognized any credit losses as of June 30, 2025 and December 31, 2024.

The following table shows the fair value and gross unrealized losses of our investments in individual securities that are in an unrealized loss position, aggregated by investment category (in thousands):

As of March 31, 2025

As of June 30, 2025

Gross Unrealized Losses

US treasury bills

Government-sponsored enterprise securities

1 unchanged sentence

The table below summarizes the fair value of our cash equivalents and short-term investments measured at fair value on a recurring basis, and are categorized based upon the lowest level of significant input to the valuations (in thousands):

Assets at Fair Value as of March 31, 2025

Assets at Fair Value as of June 30, 2025

Money market funds

8 unchanged sentences

The following table summarizes loans payable, net (in thousands):

March 31, 2025

June 30, 2025

December 31, 2024

5 unchanged sentences

The outstanding loans payable as of the periods presented was related to our Credit and Security Agreement (Credit Agreement) with MidCap Financial Trust (MidCap) entered into on September 27, 2019 (Closing Date) and amended on March 29, 2021 (First Amendment), February 11, 2022 (Second Amendment), July 27, 2022 (Third Amendment), and on April 11, 2024 (Fourth Amendment).

The Credit Agreement provides for a $ 60.0 million term loan credit facility, which was fully funded as of March 31, 2025 and December 31, 2024.

The Credit Agreement provides for a $ 60.0 million term loan credit facility, which was fully funded as of June 30, 2025 and December 31, 2024.

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

The term loans bear interest equal to the sum of one-month Secured Overnight

Financing Rate (SOFR) plus an adjustment of 0.11448% , subject to a 4.00 % applicable floor, plus applicable margin of 6.50 % .

The term loans bear interest equal to the sum of one-month Secured Overnight Financing Rate (SOFR) plus an adjustment of 0.11448% , subject to a 4.00 % applicable floor, plus applicable margin of 6.50 % .

A final payment fee of 4.25 % of principal is due at maturity date.

2 unchanged sentences

The obligations under the amended Credit Agreement are secured by a perfected security interest in all of our assets including our intellectual property.

Interest expense, including amortization of the debt discount and accretion of the final fees related to the Credit Agreement for the three months ended March 31, 2025 and 2024 was $ 1.9 million and $ 1.9 million, respectively.

Accrued interest of $ 2.2 million was included within other accrued liabilities in the condensed balance sheet as of March 31, 2025.

The following table presents the future minimum principal payments of the outstanding loan as of March 31, 2025 (in thousands):

Interest expense, including amortization of the debt discount and accretion of the final fees related to the Credit Agreement for the three months ended June 30, 2025 and 2024 was $ 1.9 million and $ 2.0 million, respectively, and for the six months ended June 30, 2025 and 2024 was $ 3.7 million and $ 3.9 million, respectively.

Accrued interest of $ 2.3 million was included within other accrued liabilities in the condensed balance sheet as of June 30, 2025.

The following table presents the future minimum principal payments of the outstanding loan as of June 30, 2025 (in thousands):

Remainder of 2025

1 unchanged sentence

The amended Credit Agreement contains certain covenants which, among others, require us to deliver financial reports at designated times of the year and maintain minimum unrestricted cash and trailing net revenues.

As of March 31, 2025, we were not in violation of any covenants.

As of June 30, 2025, we were not in violation of any covenants.

Commitments and Contingencies

Operating Leases

We have a sublease agreement with Atara Biotherapeutics, Inc.

(Atara) entered in October 2022 to sublease an office space currently used as headquarters located in South San Francisco, California.

Subject to the terms of the sublease agreement, the lease term commenced in November 2022 and expire in May 2025.

In February 2025, we entered into a lease agreement with 611 Gateway Center LP (611 Gateway) to lease the same office space currently subleased from Atara.

Subject to the terms of the lease agreement, the lease term shall commence following the expiration of the sublease with Atara and shall expire in July 2027.

In accordance with ASC 842, Leases , at lease measurement date, we recognized the operating lease right-of-use asset and lease liability of approximately $ 1.2 million.

The amount recognized as operating right-of-use lease asset and lease liability represents the present value of the future minimum lease payments over the term of the lease, measured using our incremental borrowing rate.

Our current headquarters located in South San Francisco, California was previously subleased from Atara Biotherapeutics, Inc.

(Atara) pursuant to a sublease agreement entered in October 2022, which lease term commenced in November 2022 and expired in May 2025.

In February 2025, we entered into a lease agreement with 611 Gateway Center LP (611 Gateway) to lease the same office space, which lease term commenced following the expiration of the sublease with Atara and will expire in July 2027.

Following our lease agreement with 611 Gateway, i n accordance with ASC 842, Leases , at lease measurement date, we recognized operating lease right-of-use asset and lease liability of approximately $ 1.2 million, which amount represents the present value of the future minimum lease payments over the term of the lease measured using our incremental borrowing rate.

The components of our operating lease expense were as follows (in thousands):

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

Fixed operating lease expense

1 unchanged sentence

Total operating lease expense

For the three months ended March 31, 2025 and 2024, cash payments included in the measurement of operating lease liabilities amounted to $ 0.2 million and $ 0.2 million, respectively.

The weighted average remaining term of our leases as of March 31, 2025 was 2.33 years.

The following table presents the future lease payments as of March 31, 2025 (in thousands):

Cash payments included in the measurement of operating lease liabilities for the three months ended June 30, 2025 and 2024 was $ 0.1 million and $ 0.2 million, respectively, and for the six months ended June 30, 2025 and 2024 was $ 0.3 million and $ 0.4 million, respectively.

The weighted average remaining term of our leases as of June 30, 2025 was 2.08 years.

The following table presents the future lease payments as of June 30, 2025 (in thousands):

Remainder of 2025

5 unchanged sentences

In October 2024, we entered into an agreement with a third-party contract manufacturer to manufacture TAVALISSE that is expected to be delivered starting in 2026 through 2029.

As of March 31, 2025, the contractual obligation not included in our financial statements related to an agreement that may potentially be subjected to cancellation fees of approximately $ 20.8 million, of which, $ 2.8 million is expected to be due in the remainder of 2025, and $ 9.7 million is expected to be due in 2026 and 2027.

As of March 31, 2025, we have no t incurred any cancellation fees under our agreements with contract manufacturers.

As of June 30, 2025, the contractual obligation not included in our financial statements related to an agreement that may potentially be subjected to cancellation fees of approximately $ 22.5 million, of which, $ 3.0 million is expected to be due in the remainder of 2025, and $ 10.5 million is expected to be due in 2026 and 2027.

As of June 30, 2025, we have no t incurred any cancellation fees under our agreements with contract manufacturers.

Legal Contingencies

4 unchanged sentences

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.

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.

2 unchanged sentences

We update our estimate of our annual effective tax rate at the end of each quarterly period.

For the three months ended March 31, 2025, we recorded $ 0.1 million of provision for income tax primarily related to estimated state taxes.

We do not expect to owe federal income tax due to sufficient net operating loss carryforwards that were generated prior to the enactment of the Tax Cuts and Jobs Act, as well as significant research and development credit carryforwards.

For the three months ended March 31, 2024, we did no t record a provision for income taxes due to our pre-tax book loss.

We continue to record a full valuation allowance on our deferred tax assets considering our cumulative losses in prior years.

The realization of deferred tax assets is dependent if there are sufficient positive evidences that exists to conclude that it is more-likely-than-not that our deferred tax assets will be realized.

The provision for income taxes for the three and six months ended June 30, 2025 was primarily related to state income taxes.

We do not expect to owe federal income tax due to sufficient net operating loss carryforwards that were generated prior to the enactment of the Tax Cuts and Jobs Act (TCJA), as well as significant research and development credit carryforwards.

For the three and six months ended June 30, 2024, we did no t record a provision for income taxes due to our pre-tax book loss.

As of June 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, shall be considered to determine whether, based on the weight of that evidence, a valuation allowance for deferred tax assets is needed.

If sufficient positive evidence may become 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.

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.

Segment Information

1 unchanged sentence

The following table presents segment information for the periods presented:

Three Months Ended March 31,

Three Months Ended June 30,

Six Months Ended June 30,

(in thousands)

Total Revenues

13 unchanged sentences

For details of stock-based compensation expense, see “Note 6 – Stock-Based Compensation.” Other segment items for the periods presented primarily comprised travel related expenses, business insurance, taxes and licenses, and facility related expenses.

Subsequent Event

In July 2025, the US Congress approved, and President Trump signed into law, the One Big Beautiful Bill Act (OBBBA), formally titled “An Act to provide for reconciliation pursuant to title II of H.

14.” The OBBBA includes a broad array of measures affecting corporations and other business entities, including locking in a key set of business tax provisions.

These include incentives designed to promote innovation-driven investment, such as a permanent and immediate deduction for domestic research and development costs.

The legislation also locks in 100% expensing for qualified equipment purchases and makes permanent the deduction of up to 20% for qualified business income.

ASC 740, “ Income Taxes ”, requires the effects of changes in tax rates and laws on deferred tax balances to be recognized in the period in which the legislation is enacted.

Consequently, we are evaluating the impact of the OBBBA on our financial statements and an estimate of the financial impact cannot be made at this time.

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 1. Financial Statements