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Item 2. Management’s Discussion and Analysis

2025-10-28 compared with 2025-07-29 · 227 added, 464 removed, 392 unchanged (64% of the section changed)

Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following discussion of our financial condition and results of operations as of and for the three and six months ended June 30, 2025 should be read in conjunction with the unaudited condensed consolidated financial statements and notes to those statements included elsewhere in this Quarterly Report on Form 10-Q and our audited consolidated financial statements as of and for the year ended December 31, 2024 included in our Annual Report on Form 10-K for the year ended December 31, 2024 previously filed with the SEC.

The following discussion of our financial condition and results of operations as of and for the three and nine months ended September 30, 2025 should be read in conjunction with the unaudited condensed consolidated financial statements and notes to those statements included elsewhere in this Quarterly Report on Form 10-Q and our audited consolidated financial statements as of and for the year ended December 31, 2024 included in our Annual Report on Form 10-K for the year ended December 31, 2024 previously filed with the SEC.

Forward-Looking Statements

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Often, these statements include the words “believe,” “expect,” “target,” “anticipate,” “intend,” “plan,” “seek,” “estimate,” “potential,” or words of similar meaning, or future or conditional verbs such as “will,” “would,” “should,” “could,” “might,” or “may,” or the negative of these terms, and other similar expressions.

These forward-looking statements include statements as to:

These forward-looking statements include, among other things, statements as to:

• the discovery, development, formulation, manufacturing and commercialization of our compounds, our drug candidates and JAKAFI ® /JAKAVI ® (ruxolitinib), PEMAZYRE ® (pemigatinib), ICLUSIG ® (ponatinib), MONJUVI ® (tafasitamab-cxix) / MINJUVI ® (tafasitamab), OPZELURA ® (ruxolitinib) cream, ZYNYZ ® (retifanlimab-dlwr) and NIKTIMVO TM (axatilimab);

• our plans to further develop our operations outside of the United States;

• conducting clinical trials internally, with collaborators, or with clinical research organizations;

• our collaboration and strategic relationship strategy, and anticipated benefits and disadvantages of entering into collaboration agreements;

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• the safety, effectiveness and potential benefits and indications of our drug candidates and other compounds under development;

• the timing and size of our clinical trials;

• the timing, structure and size of our clinical trials;

the compounds expected to enter clinical trials;

timing of clinical trial results;

the timing of clinical trial results;

• our ability to manage expansion of our drug discovery and development operations;

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• plans to develop and commercialize products on our own;

• plans to use third-party manufacturers;

• plans for our manufacturing operations;

• plans for our manufacturing operations, including plans to use third-party manufacturers;

• expected expenses and expenditure levels;

expected uses of cash;

expected revenues and sources of revenues, including milestone payments;

expectations with respect to the need or ability to raise additional capital;

expected revenues and sources of revenues;

expectations with respect to inventory;

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the adequacy of our capital resources to continue operations;

• the need to raise additional capital;

• the costs and other financial impacts associated with resolving matters in litigation and governmental proceedings;

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These risks and uncertainties could cause actual results to differ materially from those projected and include, but are not limited to:

• our ability to successfully commercialize our drug products and drug candidates;

• our ability to discover, develop, formulate, manufacture and successfully commercialize our drug products and drug candidates;

• our ability to obtain, or maintain at anticipated levels, coverage and reimbursement for our products from government health administration authorities, private health insurers and other organizations;

• risks relating to changes in pricing and reimbursement in the markets in which we compete;

• our ability to establish and maintain effective sales, marketing and distribution capabilities;

• the risk of reliance on other parties to manufacture our products, which could result in a short supply of our products, increased costs, and withdrawal of regulatory approvals;

• our ability to maintain regulatory approvals to market our products;

• our ability to obtain and maintain regulatory approvals to market our products;

• our ability to achieve a significant market share in order to achieve or maintain profitability;

• the risk of civil or criminal penalties if we market our products in a manner that violates health care fraud and abuse and other applicable laws, rules and regulations;

• our ability to discover, develop, formulate, manufacture and commercialize our drug candidates;

• the risk of unanticipated delays in, or discontinuations of, research and development efforts;

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• the risk that results of our clinical trials do not support submission of a marketing approval application for our drug candidates;

• the risk of significant delays or costs in obtaining regulatory approvals;

• risks relating to our reliance on third-party manufacturers, collaborators, and clinical research organizations;

• risks relating to the development of new products and their use by us and our current and potential collaborators;

• risks relating to our inability to control the development of out-licensed compounds or drug candidates;

• risks relating to our collaborators’ ability to develop and commercialize drug products and the drug candidates licensed from us;

• costs associated with prosecuting, maintaining, defending and enforcing patent claims and other intellectual property rights;

• our ability to maintain or obtain adequate product liability and other insurance coverage;

• the risk that our drug candidates may not obtain or maintain regulatory approval;

• the impact of technological advances and competition, including potential generic competition;

• our ability to compete against third parties with greater resources than ours;

• risks relating to changes in pricing and reimbursement in the markets in which we may compete;

• risks relating to governmental healthcare reform efforts, including efforts to control, set or cap pricing for our commercial drugs in the U.S.

• competition to develop and commercialize similar drug products;

• our ability to obtain and maintain patent protection and freedom to operate for our discoveries and to continue to be effective in expanding our patent coverage;

• the impact of technological advances and competition to develop and commercialize similar drug products, including potential generic competition;

• our ability to obtain and maintain patent protection and freedom to operate for our discoveries and to continue to be effective in prosecuting, maintaining, defending and enforcing patent claims and other intellectual property rights;

• the impact of changing laws on our patent portfolio;

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• risks related to public health pandemics such as the COVID-19 pandemic, natural disasters, or geopolitical events such as the Russian invasion of Ukraine and conflicts in the Middle East;

• the risks set forth under “Risk Factors.”

• the risks set forth under “Risk Factors” in Item 1A of this Quarterly Report on Form 10-Q.

Given these risks and uncertainties, you should not place undue reliance on these forward-looking statements.

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You should carefully consider all of the information in this report and, in particular, the following principal risks and all of the other specific factors described in Item 1A.

of this report, “Risk Factors,” before deciding whether to invest in our company.

“Risk Factors” of this report before deciding whether to invest in our company.

• We depend heavily on JAKAFI/JAKAVI (ruxolitinib), and if we are not able to maintain revenues from JAKAFI/JAKAVI or those revenues decrease, our business may be materially harmed.

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One therapeutic area is Hematology/Oncology, which comprises Myeloproliferative Neoplasms (“MPNs”), Graft-Versus-Host Disease (“GVHD”), solid tumors and hematologic malignancies.

The other therapeutic area is Inflammation and Autoimmunity (IAI), which includes our Dermatology commercial franchise.

The other therapeutic area is Inflammation and Autoimmunity (“IAI”), which includes our Dermatology franchise.

We are also eligible to receive milestones and royalties on molecules discovered by us and licensed to third parties.

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Our hematology and oncology franchise comprises six approved products, which are JAKAFI (ruxolitinib), MONJUVI (tafasitamab-cxix)/MINJUVI (tafasitamab), PEMAZYRE (pemigatinib), ICLUSIG (ponatinib), ZYNYZ (retifanlimab-dlwr), and NIKTIMVO (axatilimab-csfr), as well as numerous clinical development programs.

Approved Products

JAKAFI (ruxolitinib)

JAKAFI (ruxolitinib) is our first product to be approved for sale in the United States.

JAKAFI is the most advanced compound in our janus associated kinase (“JAK”) program and is an oral JAK1 and JAK2 inhibitor.

It was approved by the U.S.

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in December 2014 for the treatment of adults with polycythemia vera (“PV”) who have had an inadequate response to or are intolerant of hydroxyurea;

in May 2019 for the treatment of steroid-refractory acute graft-versus-host disease (GVHD) in adult and pediatric patients 12 years and older;

in May 2019 for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older;

and in September 2021 for the treatment of chronic GVHD after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older .

MF and PV are both myeloproliferative neoplasms (MPNs), a type of rare blood cancer, and GVHD is an adverse immune response to an allogeneic hematopoietic stem cell transplant (HSCT).

Under our collaboration agreement with our collaboration partner Novartis Pharmaceutical International Ltd., Novartis received exclusive development and commercialization rights to ruxolitinib outside of the United States for all hematologic and oncologic indications and sells ruxolitinib outside of the United States under the name JAKAVI.

In 2003, we initiated a research and development program to explore the inhibition of enzymes called janus associated kinases (JAK).

The JAK family is composed of four tyrosine kinases—JAK1, JAK2, JAK3 and Tyk2—that are involved in the signaling of a number of cytokines and growth factors.

JAKs are central to a number of biologic processes, including the formation and development of blood cells and the regulation of immune functions.

Dysregulation of the JAK-STAT signaling pathway has been associated with a number of diseases, including myeloproliferative neoplasms, other hematological malignancies, rheumatoid arthritis and other chronic inflammatory diseases.

We have discovered multiple potent, selective and orally bioavailable JAK inhibitors that are selective for JAK1 or JAK1 and JAK2.

JAKAFI is the most advanced compound in our JAK program.

It is an oral JAK1 and JAK2 inhibitor.

JAKAFI is marketed in the United States through our own specialty sales force and commercial team.

MF and PV are both MPNs, a group of rare blood cancers, and GVHD is an adverse immune response to an allogeneic hematopoietic stem cell transplant.

Under our collaboration agreement with our collaboration partner Novartis Pharmaceutical International Ltd.

(“Novartis”), Novartis received exclusive development and commercialization rights to ruxolitinib outside of the United States for all hematologic and oncologic indications and sells ruxolitinib outside of the United States under the name JAKAVI.

JAKAFI was the first FDA-approved JAK inhibitor for any indication, was the first FDA-approved product in MF, PV and steroid-refractory acute GVHD, and was recently approved in steroid-refractory chronic GVHD.

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The FDA has granted JAKAFI orphan drug status for MF, PV and GVHD.

In addition, ruxolitinib phosphate qualifies for the Small Biotech Exception from the Centers for Medicare and Medicaid Services (“CMS”) under the Inflation Reduction Act.

JAKAFI is distributed primarily through a network of specialty pharmacy providers and wholesalers that allow for efficient delivery of the medication by mail directly to patients or direct delivery to the patient's pharmacy.

Our distribution process uses a model that is well established and familiar to physicians who practice within the oncology field.

To further support appropriate use and future development of JAKAFI, our U.S.

Medical Affairs department is responsible for providing appropriate scientific and medical education and information to physicians, preparing scientific presentations and publications, and overseeing the process for supporting investigator sponsored trials.

In September 2023, we were notified by the Centers for Medicare and Medicaid Services (CMS) that ruxolitinib phosphate qualified for the Small Biotech Exception.

Myelofibrosis.

MF is a rare, life-threatening condition.

MF, considered the most serious of the myeloproliferative neoplasms, can occur either as primary MF, or as secondary MF that develops in some patients who previously had polycythemia vera or essential thrombocythemia.

Based on the modern prognostic scoring systems referred to as International Prognostic Scoring System and Dynamic International Prognostic Scoring System, we believe intermediate and high-risk patients represent 80% to 90% of all patients with MF in the United States and encompass patients over the age of 65, or patients who have or have ever had any of the following:

anemia, constitutional symptoms, elevated white blood cell or blast counts, or platelet counts less than 100,000 per microliter of blood.

Most MF patients have enlarged spleens and many suffer from debilitating symptoms, including abdominal discomfort, pruritus (itching), night sweats and cachexia (involuntary weight loss).

There were no FDA approved therapies for MF until the approval of JAKAFI.

The FDA approval was based on results from two randomized Phase 3 trials (COMFORT-I and COMFORT-II), which demonstrated that patients treated with JAKAFI experienced significant reductions in splenomegaly (enlarged spleen).

COMFORT-I also demonstrated improvements in symptoms.

The most common hematologic adverse reactions in both trials were thrombocytopenia and anemia.

These events rarely led to discontinuation of JAKAFI treatment.

The most common non-hematologic adverse reactions were bruising, dizziness and headache.

In August 2014, the FDA approved supplemental labeling for JAKAFI to include Kaplan-Meier overall survival curves as well as additional safety and dosing information.

The overall survival information is based on three-year data from COMFORT-I and II and shows that at three years the probability of survival for patients treated with JAKAFI in COMFORT-I was 70% and for those patients originally randomized to placebo it was 61%.

In COMFORT-II, at three years the probability of survival for patients treated with JAKAFI was 79% and for patients originally randomized to best available therapy it was 59%.

In December 2016, we announced an exploratory pooled analysis of data from the five-year follow-up of the COMFORT-I and COMFORT-II trials of patients treated with JAKAFI, which further supported previously published overall survival findings.

In September 2016, we announced that JAKAFI had been included as a recommended treatment in the latest National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for myelofibrosis, underscoring the important and long term clinical benefits seen in patients treated with JAKAFI.

In October 2017, the FDA approved updated labeling for JAKAFI to include the addition of new patient-reported outcome (PRO) data from the COMFORT-I study, as well as updating the warning related to progressive multifocal leukoencephalopathy.

An exploratory analysis of PRO data of patients with myelofibrosis receiving JAKAFI showed improvement in fatigue-related symptoms at Week 24.

Fatigue response (defined as a reduction of 4.5 points or more from baseline in the PROMIS ® Fatigue total score) was reported in 35% of patients treated with JAKAFI versus 14% of the patients treated with placebo.

Polycythemia Vera.

PV is a myeloproliferative neoplasm typically characterized by elevated hematocrit, the volume percentage of red blood cells in whole blood, which can lead to a thickening of the blood and an increased risk of blood clots, as well as an elevated white blood cell and platelet count.

In December 2014, the FDA approved JAKAFI for the treatment of patients with PV who have had an inadequate response to or are intolerant of hydroxyurea.

The approval of JAKAFI for PV was based on data from the pivotal Phase 3 RESPONSE trial.

In this trial, patients treated with JAKAFI demonstrated superior hematocrit control and reductions in spleen volume compared to best available therapy.

In addition, a greater proportion of patients treated with JAKAFI achieved complete hematologic remission—which was defined as achieving hematocrit control and lowering platelet and white blood cell counts.

In the RESPONSE trial, the most common hematologic adverse reactions (incidence > 20%) were thrombocytopenia and anemia.

The most common non-hematologic adverse events (incidence >10%) were headache, abdominal pain, diarrhea, dizziness, fatigue, pruritus, dyspnea and muscle spasms.

In March 2016, the FDA approved supplemental labeling for JAKAFI to include additional safety data as well as efficacy analyses from the RESPONSE trial to assess the durability of response in JAKAFI treated patients after 80 weeks.

At this time, 83% patients were still on treatment, and 76% of the responders at 32 weeks maintained their response through 80 weeks.

In June 2016, we announced data from the Phase 3 RESPONSE-2 study of JAKAFI in patients with inadequately controlled PV that was resistant to or intolerant of hydroxyurea who did not have an enlarged spleen.

These data showed that JAKAFI was superior to best available therapy in maintaining hematocrit control (62.2% vs.

18.7%, respectively;

P<0.0001) without the need for phlebotomy.

In August 2017, we announced that JAKAFI had been included as a recommended treatment in the latest NCCN Guidelines for patients with polycythemia vera who have had an inadequate response to first-line therapies, such as hydroxyurea.

Graft-versus-host disease.

GVHD is a condition that can occur after an allogeneic HSCT (the transfer of genetically dissimilar stem cells or tissue).

In GVHD, the donated bone marrow or peripheral blood stem cells view the recipient’s body as foreign and attack various tissues.

In June 2016, we announced that the FDA granted Breakthrough Therapy designation for ruxolitinib in patients with acute GVHD.

In May 2019, the FDA approved JAKAFI for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.

The approval was based on data from REACH1, an open-label, single-arm, multicenter study of JAKAFI in combination with corticosteroids in patients with steroid-refractory grade II-IV acute GVHD.

The overall response rate (ORR) in patients refractory to steroids alone was 57% with a complete response (CR) rate of 31%.

The most frequently reported adverse reactions among all study participants were infections (55%) and edema (51%), and the most common laboratory abnormalities were anemia (75%), thrombocytopenia (75%) and neutropenia (58%).

In September 2021, the FDA approved JAKAFI for the treatment of chronic GVHD after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older.

This approval was based on data from REACH3, a Phase 3, randomized, open-label, multicenter study of JAKAFI in comparison to best available therapy for treatment of steroid-refractory chronic GVHD after allogeneic stem cell transplantation.

The overall response rate through Cycle 7 Day 1 was 70% for JAKAFI compared to 57% for best available therapy.

The most common hematologic adverse reactions (incidence > 35%) were anemia and thrombocytopenia.

The most common non-hematologic adverse reactions (incidence ≥ 20%) were infections (pathogen not specified) and viral infection.

In addition, the FDA updated labeling for JAKAFI to include warnings of increased risk of major adverse cardiovascular events, thrombosis, and secondary malignancies related to another JAK-inhibitor treating rheumatoid arthritis, a condition for which JAKAFI is not indicated.

In patients with MF and PV treated with JAKAFI in clinical trials, the rates of thromboembolic events were similar in JAKAFI and control treated patients.

We have retained all development and commercialization rights to JAKAFI in the United States and are eligible to receive development and sales milestones as well as royalties from product sales outside the United States.

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MONJUVI (tafasitamab-cxix) / MINJUVI (tafasitamab)

In January 2020, we and MorphoSys AG entered into a collaboration and license agreement to further develop and commercialize MorphoSys’ proprietary anti-CD19 antibody tafasitamab (MOR208) globally.

Tafasitamab is an Fc-engineered antibody against CD19 currently in clinical development for the treatment of B cell malignancies.

In January 2020, we and MorphoSys AG (“MorphoSys”) entered into a collaboration and license agreement to further develop and commercialize MorphoSys’ proprietary anti-CD19 antibody tafasitamab (MOR208), an Fc-engineered antibody against CD19.

Under the terms of the collaboration and license agreement, we received rights to co-commercialize tafasitamab in the United States with MorphoSys, and exclusive development and commercialization rights outside of the United States.

As more fully described in Note 6 of Notes to the Condensed Consolidated Financial Statements, in February 2024, we entered into a purchase agreement with MorphoSys, and as a result, we now hold exclusive global rights for tafasitamab, and the collaboration and license agreement was terminated.

In July 2020, we and MorphoSys announced that the FDA had approved MONJUVI (tafasitamab-cxix), which is indicated in combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT).

MONJUVI was approved under accelerated approval based on overall response rate from the MorphoSys-sponsored Phase 2 L-MIND study, an open label, multicenter, single arm trial of MONJUVI in combination with lenalidomide as a treatment for adult patients with r/r DLBCL.

Results from the study showed an objective response rate of 55% (39 out of 71 patients;

primary endpoint) and a complete response rate of 37% (26 out of 71 patients).

The median duration of response was 21.7 months.

The most frequent serious adverse reactions were infections (26%), including pneumonia (7%) and febrile neutropenia (6%).

Updated three-year data from L-MIND were presented at the American Society of Clinical Oncology (ASCO) 2021 and final five-year data were presented at the American Association for Cancer Research (AACR) 2023, which showed that the MONJUVI plus lenalidomide regimen followed by MONJUVI monotherapy provided prolonged, durable responses in adult patients with r/r DLBCL.

In August 2020, we and MorphoSys announced that MONJUVI in combination with lenalidomide had been included in the latest NCCN Clinical Practice Guidelines in Oncology for B-cell Lymphomas.

In August 2021, we and MorphoSys announced that the European Commission had granted conditional marketing authorization for MINJUVI (tafasitamab) in combination with lenalidomide, followed by MINJUVI monotherapy, for the treatment of adult patients with relapsed or refractory DLBCL who are not eligible for ASCT.

The conditional approval was based on the three-year results from the L-MIND study evaluating the safety and efficacy of MINJUVI in combination with lenalidomide as a treatment for patients with r/r DLBCL who are not eligible for ASCT.

The results showed best objective response rate of 56.8% (primary endpoint), including a complete response rate of 39.5% and a partial response rate of 17.3%, as assessed by an independent review committee.

The median duration of response was 43.9 months after a minimum follow up of 35 months (secondary endpoint).

MINJUVI together with lenalidomide was shown to provide a clinically meaningful response and the side effects were manageable.

Warnings and precautions for MINJUVI include infusion-related reactions, myelosuppression, including neutropenia and thrombocytopenia, infections and tumour lysis syndrome.

In June 2025, MONJUVI (tafasitamab-cxix) was approved by the FDA for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) in combination with rituximab and lenalidomide.

The FDA approval was based on data from the pivotal Phase 3 inMIND trial.

In July 2020, the FDA approved MONJUVI (tafasitamab-cxix), in combination with lenalidomide, for the treatment of adult patients with relapsed or refractory (“r/r”) diffuse large B-cell lymphoma (“DLBCL”) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (“ASCT”).

In August 2021, the European Commission granted conditional marketing authorization for MINJUVI (tafasitamab), in combination with lenalidomide, followed by MINJUVI monotherapy, for the treatment of adult patients with r/r DLBCL who are not eligible for ASCT.

In June 2025, MONJUVI (tafasitamab-cxix) was approved by the FDA for the treatment of adult patients with r/r follicular lymphoma (“FL”) in combination with rituximab and lenalidomide.

PEMAZYRE (pemigatinib)

PEMAZYRE is the first internally discovered product to be internationally commercialized by us.

In April 2020, we announced that the FDA had approved PEMAZYRE (pemigatinib), a selective fibroblast growth factor receptor (FGFR) kinase inhibitor, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or other rearrangement as detected by an FDA-approved test.

In April 2020, the FDA approved PEMAZYRE (pemigatinib), a selective fibroblast growth factor receptor (FGFR) kinase inhibitor, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or other rearrangement as detected by an FDA-approved test.

PEMAZYRE is the first FDA-approved treatment for this indication, which was approved under accelerated approval based on overall response rate and duration of response.

In March 2021, PEMAZYRE was approved by the Japanese Ministry of Health, Labour and Welfare (MHLW) for the treatment of patients with unresectable biliary tract cancer (BTC) with an FGFR2 fusion gene, worsening after cancer chemotherapy.

Also in March 2021, PEMAZYRE was approved by the European Commission for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement that has progressed after at least one prior line of systemic therapy.

In March 2021, PEMAZYRE was approved by the Japanese Ministry of Health, Labour and Welfare (“MHLW”) for the treatment of patients with unresectable biliary tract cancer with an FGFR2 fusion gene, worsening after cancer chemotherapy, and was approved by the European Commission for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement that has progressed after at least one prior line of systemic therapy.

In July 2021, the UK’s National Institute for Health and Care Excellence (“NICE”) recommended PEMAZYRE for patients with cholangiocarcinoma with a FGFR2 fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

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In March 2022, PEMAZYRE was approved by the National Medical Products Administration of the People’s Republic of China for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with a FGFR2 fusion or rearrangement as confirmed by a validated diagnostic test that has progressed after at least one prior line of systemic therapy.

Cholangiocarcinoma is a rare cancer that arises from the cells within the bile ducts.

It is often diagnosed late (stages III and IV) and the prognosis is poor.

The incidence of cholangiocarcinoma with FGFR2 fusions or rearrangements is increasing, and it is currently estimated that there are 2,000-3,000 patients in the United States, Europe and Japan.

The approval of PEMAZYRE was based on data from FIGHT-202, a multi-center, open-label, single-arm study evaluating PEMAZYRE as a treatment for adults with cholangiocarcinoma.

In FIGHT-202, and in patients harboring FGFR2 fusions or rearrangements (Cohort A), PEMAZYRE monotherapy resulted in an overall response rate of 36% (primary endpoint), and median DOR of 9.1 months (secondary endpoint).

FIGHT-302, a Phase 3 trial of pemigatinib for the first-line treatment of patients with cholangiocarcinoma and FGFR2 fusions or rearrangements, is ongoing.

In August 2022, PEMAZYRE was approved by the FDA as the first and only targeted treatment for myeloid/lymphoid neoplasms (“MLNs”) with FGFR1 rearrangement.

MLNs with FGFR1 rearrangement are extremely rare and aggressive blood cancers.

In March 2023, PEMAZYRE was approved by the MHLW for the treatment of MLNs with FGFR1 fusion.

ICLUSIG (ponatinib)

In June 2016, we acquired the European operations of ARIAD Pharmaceuticals, Inc., and obtained an exclusive license to develop and commercialize ICLUSIG (ponatinib) in Europe and other select countries.

ICLUSIG is a kinase inhibitor.

In June 2016, we acquired the European operations of ARIAD Pharmaceuticals, Inc., and obtained an exclusive license to develop and commercialize ICLUSIG (ponatinib), a kinase inhibitor, in Europe and other select countries.

The primary target for ICLUSIG is BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia (“CML”) and Philadelphia-chromosome positive acute lymphoblastic leukemia (“Ph+ ALL”).

In the European Union, ICLUSIG is approved for the treatment of adult patients with chronic phase, accelerated phase or blast phase CML who are resistant to dasatinib or nilotinib;

who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate;

In the European Union, ICLUSIG is approved for the treatment of adult patients with chronic phase, accelerated phase or blast phase CML who are resistant to dasatinib or nilotinib, who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate;

or who have the T315I mutation, or the treatment of adult patients with Ph+ ALL who are resistant to dasatinib;

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ZYNYZ (retifanlimab-dlwr)

In October 2017, we and MacroGenics, Inc., announced an exclusive global collaboration and license agreement for MacroGenics’ retifanlimab (formerly INCMGA0012), a humanized monoclonal antibody targeting programmed death receptor-1 (PD-1).

In October 2017, we and MacroGenics, Inc.

(“MacroGenics”), announced an exclusive global collaboration and license agreement for MacroGenics’ retifanlimab (formerly INCMGA0012), a humanized monoclonal antibody targeting programmed death receptor-1 (PD-1).

Under this collaboration, we obtained exclusive worldwide rights for the development and commercialization of retifanlimab in all indications.

The molecule currently is being evaluated both as monotherapy and in combination therapy across various tumor types.

Two Phase 3 trials evaluating retifanlimab in squamous cell anal cancer (SCAC) and non-small cell lung cancer (NSCLC) are ongoing.

In March 2023, we announced that the FDA had approved ZYNYZ (retifanlimab-dlwr) under accelerated approval, for the treatment of adults with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC).

This represents the first regulatory approval for our PD-1 inhibitor.

In April 2024, the European Commission approved ZYNYZ (retifanlimab) as monotherapy for the first-line treatment of adult patients with metastatic or recurrent locally advanced MCC not amenable to curative surgery or radiation therapy following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP).

In September 2024, we announced positive results from the Phase 3 POD1UM-303/InterAACT2 trial of ZYNYZ (retifanlimab) in combination with platinum-based chemotherapy (carboplatin–paclitaxel) for the treatment of adults with inoperable locally recurrent or metastatic SCAC.

In May 2025, ZYNYZ (retifanlimab-dlwr) was approved by the FDA for the treatment of adult patients with advanced SCAC in combination with chemotherapy and as a single agent.

The FDA approval was based on data from two trials:

the Phase 3 POD1UM-303/InterAACT2 and the Phase 2 POD1UM-202 trial.

We have submitted a Type II variation Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) and a Japanese New Drug Application (J-NDA) for retifanlimab in advanced SCAC.

In March 2023, the FDA approved ZYNYZ (retifanlimab-dlwr) under accelerated approval for the treatment of adults with metastatic or recurrent locally advanced Merkel cell carcinoma (“MCC”).

In April 2024, the European Commission approved ZYNYZ (retifanlimab) as a monotherapy for the first-line treatment of adult patients with metastatic or recurrent locally advanced MCC not amenable to curative surgery or radiation therapy.

In May 2025, the FDA approved ZYNYZ (retifanlimab-dlwr) for the treatment of adult patients with advanced squamous cell anal cancer (“SCAC”) in combination with chemotherapy and as a single agent.

We have submitted a Type II variation Marketing Authorization Application (“MAA”) to the European Medicines Agency (“EMA”) and a Japanese New Drug Application for retifanlimab in advanced SCAC.

NIKTIMVO (axatilimab-csfr)

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announced an exclusive worldwide collaboration and license agreement to develop and commercialize axatilimab, Syndax’s anti-CSF-1R monoclonal antibody.

Together, we are developing axatilimab as a therapy for patients with chronic GVHD where CSF-1R-dependent monocytes and macrophages are believed to contribute to organ fibrosis.

In December 2021, updated positive data were presented at ASH from the Phase 1/2 trial evaluating axatilimab as a monotherapy in patients with recurrent or refractory chronic GVHD after two or more prior lines of therapy.

A 68% overall response rate and broad clinical benefit across multiple organs were observed at doses being assessed in AGAVE-201, a global pivotal trial evaluating axatilimab monotherapy in patients with chronic GVHD in the third line setting.

In May 2022, Syndax announced that axatilimab had been granted fast-track designation by the FDA for the treatment of patients with chronic GVHD after failure of two or more lines of systemic therapy.

In July 2023, we and Syndax announced that AGAVE-201 had met its primary endpoint across all cohorts with an overall response rate (ORR) of 74% at the dose of 0.3 mg/kg administered every two weeks.

The data highlight the durable response seen at the 0.3 mg/kg dose with 60% of patients who responded to axatilimab still responding at one year.

In December 2023, a Biologics License Application (BLA) was submitted to the FDA for axatilimab for the treatment of patients with chronic GVHD after failure of two or more lines of systemic therapy and accepted for Priority Review in February 2024.

We have initiated two combination trials with axatilimab in cGVHD in 2024, including a randomized Phase 2 combination trial with ruxolitinib and a randomized Phase 3 combination trial with steroids, both directed at treating patients with cGVHD in earlier lines of therapy.

In August 2024, we and Syndax announced the FDA approval of NIKTIMVO (axatilimab-csfr) for the treatment of chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients.

In August 2024, the FDA approved NIKTIMVO (axatilimab-csfr) for the treatment of chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients.

NIKTIMVO is the first approved anti-CSF-1R antibody targeting the drivers of inflammation and fibrosis seen in chronic GVHD.

In September, we and Syndax announced the New England Journal of Medicine publication of data from the pivotal AGAVE-201 trial of NIKTIMVO in chronic GVHD and the addition of NIKTIMVO to the NCCN Clinical Practice Guidelines in Oncology for the treatment of chronic GVHD.

In January 2025, the FDA approved two smaller vial sizes (9mg and 22mg) of NIKTIMVO to facilitate patient dosing and limit product waste.

commercial launch of NIKTIMVO commenced at th e end of January 2025.

commercial launch of NIKTIMVO commenced in January 2025.

Also in January 2025, the FDA approved two smaller vial sizes (9mg and 22mg) of NIKTIMVO to facilitate patient dosing and limit product waste.

Clinical Programs in Hematology and Oncology

We are evaluating combinations of ruxolitinib with other therapeutic modalities, as well as developing a once-a-day formulation of ruxolitinib for potential use as monotherapy and in combinations.

Ruxolitinib XR

We are developing a once-a-day formulation of ruxolitinib for potential use as monotherapy and in combinations.

Bioavailability and bioequivalence data were published for ruxolitinib’s once-daily (“QD”) extended release (“XR”) formulation at the European Hematology Association (“EHA”) Virtual Congress in June 2021.

In March 2023, the FDA issued a complete response letter (CRL) for ruxolitinib extended-release (XR) tablets for once-daily (QD) use in the treatment of certain types of MF, PV and GVHD.

In March 2023, the FDA issued a complete response letter (“CRL”) for ruxolitinib XR tablets for QD use in the treatment of certain types of MF, PV and GVHD.

In December 2023, we received FDA feedback and agreed on the requirements to address the CRL.

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These data are anticipated to be submitted to the FDA, in response to the CRL, by year-end 2025.

Phase 2 trials combining ruxolitinib with investigational agents from our portfolio such as INCB57643 (BET) in patients with MF are ongoing with data presented demonstrating early signals of clinical activity in monotherapy and in combination with ruxolitinib.

INCB57643 (BET inhibitor)

INCB057643 is a small-molecule inhibitor of BET that was being evaluated as monotherapy and in combination with ruxolitinib in patients with advanced malignancies.

In October 2025, development for INCB57643 was discontinued.

INCA033989 (mutCALR)

In December 2022, new research detailing the development and mechanism of action of INCA033989, an Incyte-discovered, investigational novel anti-mutant calreticulin (CALR)-targeted monoclonal antibody, was featured in the Plenary Scientific Session at the 64th American Society of Hematology (ASH) Annual Meeting.

INCA033989 binds with high affinity to mutant CALR and inhibits oncogenesis, the process of cells becoming cancerous, in cells expressing this oncoprotein.

CALR mutations are responsible for disease development in approximately 25-35% of patients with MF and ET.

In July 2023, a Phase 1 study evaluating INCA033989 was initiated.

In June 2025, data from the Phase 1 study evaluating INCA033989 in mutCALR positive patients with essential thrombocythemia were presented during a late-breaking session at the 2025 European Hematology Association (EHA) Congress in Milan, Italy.

The data showed rapid and durable normalization of platelet counts across all dose levels and importantly, a reduction in peripheral blood mutCALR variant allele frequency (VAF) correlating with hematologic response.

In June 2025, data from our Phase 1 study evaluating INCA033989, an Incyte-discovered, investigational novel anti-mutant calreticulin (“CALR”)-targeted monoclonal antibody, in mutCALR positive patients with essential thrombocythemia were presented during a late-breaking session at the 2025 EHA Congress in Milan, Italy.

The data showed rapid and durable normalization of platelet counts across all dose levels and importantly, a reduction in peripheral blood mutCALR variant allele frequency correlating with hematologic response.

INCA033989 was well tolerated with a favorable safety profile with no dose limiting toxicities reported.

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The Phase 1 data in patients with MF as monotherapy and in combination with ruxolitinib are anticipated in the second half of 2025.

In October 2025, we announced a strategic partnership with Enable Injections, Inc.

(“Enable”) to develop and commercialize specific assets in our portfolio, including INCA033989, with Enable’s enFuse ® on-body delivery system.

Under the terms of the agreement, we will obtain a worldwide, exclusive license to use the enFuse technology with INCA033989 in essential thrombocythemia and MF, with the potential to expand to additional assets and indications.

Other Clinical Programs

INCB160058 (JAK2V617Fi)

In December 2023, new research detailing the development and mechanism of action of INCB160058, an Incyte-discovered, investigational novel potent and selective JAK2 pseudokinase domain binder with potential to be a disease modifying therapeutic was disclosed at the 65th ASH Annual Meeting.

Pseudokinase binding offers a new mechanism of action for selective inhibition of JAK2V617F, with potential to eradicate mutant clones.

We initiated a Phase 1 study of INCB160058, an Incyte-discovered, investigational novel potent and selective JAK2 pseudokinase domain binder with potential to be a disease modifying therapeutic, in the first quarter of 2024.

In preclinical studies, INCB160058 inhibited cytokine independent activity of JAK2V617F while sparing WT JAK2.

The JAK2V617F mutation is found in 55% of primary myelofibrosis, 95% of polycythemia vera and 60% of essential thrombocythemia patients.

A Phase 1 study of INCB160058 was initiated in the first quarter of 2024.

Tafasitamab is an anti-CD19 antibody and is being investigated as a therapeutic option in B cell malignancies in a number of ongoing and planned combination trials.

An open-label Phase 2 combination trial (L-MIND) is investigating the safety and efficacy of tafasitamab in combination with lenalidomide in patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL), and the ongoing Phase 3 B-MIND trial is assessing the combination of tafasitamab and bendamustine versus rituximab and bendamustine in r/r DLBCL.

firstMIND is a Phase 1b safety trial of tafasitamab as a first-line therapy for patients with DLBCL, and frontMIND, a placebo-controlled Phase 3 trial evaluating tafasitamab in combination with lenalidomide added to rituximab plus chemotherapy (R-CHOP) as a first-line therapy for patients with DLBCL, is ongoing.

In January 2021, the FDA granted orphan drug designation to tafasitamab as a treatment for patients with follicular lymphoma.

In December 2024, we announced the full results from the pivotal Phase 3 inMIND trial evaluating treatment with tafasitamab in combination with lenalidomide and rituximab compared with placebo plus lenalidomide and rituximab in patients with relapsed or refractory follicular lymphoma (FL).

The data showed that the study met its primary endpoint by demonstrating a statistically significant and clinically meaningful improvement in progression-free survival (PFS) by investigator assessment in 548 patients with FL.

Tafasitamab was generally well-tolerated, and safety was consistent with other CD19 and immunotherapy combination regimens.

In July 2024, we announced positive topline results from both the two Phase 3 clinical studies evaluating retifanlimab, a humanized monoclonal antibody targeting programmed cell death receptor-1 (PD-1), in SCAC and NSCLC.

The phase 3 study in SCAC met its primary endpoint of progression free survival while the Phase 3 study in NSCLC meet its primary endpoint of overall survival.

The safety analysis from both studies showed retifanlimab was generally well-tolerated with no new safety signals observed.

POD1UM-303 is a Phase 3, global, multicenter, randomized, double-blind study evaluating carboplatin-paclitaxel with retifanlimab or placebo in patients with inoperable locally recurrent or metastatic SCAC who have not previously been treated with chemotherapy.

The open-label Phase 2 combination trial (L-MIND) is investigating the safety and efficacy of tafasitamab in combination with lenalidomide in patients with r/r DLBCL and the ongoing Phase 3 B-MIND trial is assessing the combination of tafasitamab and bendamustine versus rituximab and bendamustine in r/r DLBCL.

firstMIND is a Phase 1b safety trial of tafasitamab as a first-line therapy for patients with DLBCL, and frontMIND is an ongoing placebo-controlled Phase 3 trial evaluating tafasitamab in combination with lenalidomide added to rituximab plus chemotherapy as a first-line therapy for patients with DLBCL.

We are conducting two Phase 3 clinical studies evaluating retifanlimab, a humanized monoclonal antibody targeting PD-1, in SCAC and non-small cell lung cancer (“NSCLC”).

POD1UM-303/InterAACT2 is a Phase 3, global, multicenter, randomized, double-blind study evaluating carboplatin-paclitaxel with retifanlimab or placebo in patients with inoperable locally recurrent or metastatic SCAC who have not previously been treated with chemotherapy.

POD1UM-304 is a Phase 3, global, multicenter, randomized, double-blind study evaluating platinum-based chemotherapy with retifanlimab or placebo in patients with first-line, metastatic squamous or nonsquamous NSCLC.

In September 2024, we presented late-breaking Phase 3 results for retifanlimab that were featured during the 2024 European Society for Medical Oncology (ESMO) Presidential Symposium.

The Phase 3 POD1UM-303/InterAACT2 trial for retifanlimab met the primary endpoint of PFS and demonstrated improvement across key secondary endpoints in patients with SCAC receiving retifanlimab in combination with platinum-based chemotherapy (carboplatin-paclitaxel).

In July 2024, we announced that both trials met their respective primary endpoints.

In September 2024, we presented late-breaking Phase 3 results showing that the Phase 3 POD1UM-303/InterAACT2 trial for retifanlimab met the primary endpoint of progression free survival and demonstrated improvement across key secondary endpoints in patients with SCAC receiving retifanlimab in combination with platinum-based chemotherapy (carboplatin-paclitaxel).

INCB123667 (CDK2)

In the cell cycle, the serine threonine kinase, CDK2, regulates the transition from the G1 phase (cell growth) to the S-phase (DNA replication).

INCB123667 is a novel, potent and selective oral small molecule inhibitor of CDK2 which has been shown to suppress tumor growth as monotherapy and in combination with standard of care, in Cyclin E amplified tumor models, in vivo.

In April 2023, we presented data at the AACR Annual Meeting, demonstrating that INCB123667 exhibited significant single-agent activity in vivo, in CCNE1 high breast cancer xenograft and patient-derived xenograft models.

INCB123667 currently is being evaluated in a Phase 1 clinical trial in patients with advanced malignancies including CCNE1 high TNBC and HR+HER2- tumors post-CDK4/6 inhibitors.

In January 2024, we disclosed that early clinical activity was observed in patients with amplification/over expression of CCNE1 in a Phase 1 clinical trial, with significant tumor shrinkage observed.

Several patients achieved partial responses across multiple tumor types including ovarian cancer patients with CCNE1 amplification and/or over expression.

The safety data seen during this disclosure aligns with CDK2 mechanism of action.

Additional data from this trial is anticipated in 2025.

In September 2024, we presented initial data from the Phase 1 CDK2 inhibitor program at the 2024 ESMO Congress.

INCB123667 is a novel, potent and selective oral small molecule inhibitor of serine threonine kinase (“CDK2”) which has been shown to suppress tumor growth as monotherapy and in combination with standard of care, in Cyclin E amplified tumor models, in vivo.

We are evaluating INCB123667 in a Phase 1 clinical trial in patients with advanced malignancies including CCNE1 high TNBC and HR+HER2- tumors post-CDK4/6 inhibitors.

In September 2024, we presented initial data from the Phase 1 CDK2 inhibitor program at the 2024 European Society of Medical Oncology (“ESMO”) Congress.

Phase 1 data of INCB123667 were presented demonstrating single-agent antitumor activity across a range of doses and regimens, notably in patients with ovarian cancer and endometrial cancer whose tumors overexpress Cyclin E1.

The Phase 1 trial is ongoing with INCB123667 in combination with other agents.

We currently anticipate initiating a pivotal trial in ovarian cancer in 2025.

MPN, GVHD and Oncology Programs Indication and Phase

Ruxolitinib XR (QD)

(JAK1/JAK2) Myelofibrosis, polycythemia vera and GVHD

Ruxolitinib + INCB57643

(JAK1/JAK2 + BETi) Myelofibrosis:

Ruxolitinib + axatilimab 1

(JAK1/JAK2 + anti-CSF-1R)

Chronic GVHD:

Steroids + axatilimab 1

(Steroids + anti-CSF-1R)

Chronic GVHD:

(mutCALR) Myelofibrosis, essential thrombocythemia:

(JAK2V617Fi) Myeloproliferative Neoplasms (MPNs):

Tafasitamab (MONJUVI/MINJUVI)

(CD19) Relapsed or refractory diffuse large B-cell lymphoma (DLBCL):

Phase 3 (B-MIND)

First-line DLBCL:

Phase 3 ( front MIND)

Retifanlimab (ZYNYZ) 2

Non-small cell lung cancer (NSCLC):

Phase 3 (POD1UM-304)

MSI-high endometrial cancer:

Phase 2 (POD1UM-101, POD1UM-204)

(CDK2i) Solid tumors with CCNE1 amplification/Cyclin E overexpression:

(KRASG12D) Advanced metastatic solid tumors with a KRASG12D mutation:

(TGFßR2×PD-1) 3

Advanced or metastatic solid tumors:

Clinical development of axatilimab in GVHD conducted in collaboration with Syndax Pharmaceuticals.

Retifanlimab licensed from MacroGenics.

Development collaboration with Merus.

Earlier-Stage Development Programs in Hematology and Oncology

In September 2025, a Phase 2 single-arm study of INCB123667 (CDK2i) in patients with platinum-resistant ovarian cancer (PROC) with Cyclin E1 overexpression was initiated.

A Phase 3, randomized, open-label study of INCB123667 versus investigator’s choice chemotherapy in patients with PROC with Cyclin E1 overexpression is planned to initiate by year-end 2025.

Select Earlier-Stage Development Programs in Hematology and Oncology

INCB161734 (KRAS G12D)

A Phase 1 study evaluating INCB161734 (KRASG12D) was initiated in the first quarter of 2024.

INCB161734 is a potent, selective and orally bioavailable KRAS G12D inhibitor and, as highlighted at AACR in April 2024, has shown excellent efficacy in several preclinical models.

With no currently approved G12D-targeting agents, INCB161734 could address an important patient need as the KRASG12D mutation is found in 40% of pancreatic ductal adenocarcinoma, 15% of colorectal cancers, and 5% of non-small cell lung cancers.

Data from the ongoing Phase 1 study is expected in 2025.

INCB161734 is a potent, selective and orally bioavailable KRAS G12D inhibitor that is currently being evaluated in a Phase 1 study in patients with locally advanced or metastatic solid tumor with KRASG12D mutation.

In October 2025, preliminary data from the ongoing Phase 1 study were presented at the 2025 European Society of Medical Oncology (ESMO) Congress.

In the study, INCB161734 demonstrated a manageable safety profile and clinical efficacy in heavily pretreated pancreatic ductal adenocarcinoma (PDAC) patients with a KRASG12D mutation.

Based upon these results, we intend to continue further development of INCB161734.

INCA33890 (TGFβR2xPD-1)

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INCA33890 has a 10-fold higher binding affinity for PD-1 relative to TGFβR2, and specifically blocks TGFβ signaling in cells co-expressing PD-1.

In April 2023, we presented preclinical data at AACR that showed that INCA33890 inhibits tumor growth in PD-1-resistant mouse models.

In July 2023, we initiated a Phase 1 study evaluating INCA33890 in patients with select advanced solid tumors.

Data from the ongoing Phase 1 study is expected in 2025.

INCB186748 (Next-generation KRAS G12D)

INCB186748 is a next-generation KRAS G12D inhibitor that demonstrated high potency, selectivity and oral bioavailability in preclinical studies.

INCB186748 is being evaluated in KRAS G12D mutant tumors.

Inflammation and AutoImmunity (IAI)

Incyte Dermatology launched its first approved product, OPZELURA (ruxolitinib) cream, in October 2021, following FDA approval for atopic dermatitis in September 2021.

In July 2023, we initiated a Phase 1 study evaluating INCA33890 in patients with select advanced/metastatic solid tumors.

In October 2025, data from the ongoing Phase 1 study evaluating were presented at the 2025 ESMO Congress.

In the study, INCA33890 demonstrated clinical efficacy across multiple tumors, including microsatellite stable colorectal cancer (MSS CRC) in patients with and without active liver metastases.

INCA33890 was generally well tolerated and evaluation of INCA33890 in combination with standard of care (SoC) treatments in patients with metastatic CRC is ongoing.

Based on these initial findings, we plan to initiate a registrational program evaluating INCA33890 in MSS CRC in 2026.

Inflammation and AutoImmunity

Incyte Dermatology launched its first approved product, OPZELURA (ruxolitinib) cream, in October 2021.

OPZELURA subsequently was approved by the FDA and European Commission for vitiligo in July 2022 and April 2023, respectively.

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Atopic Dermatitis .

In September 2021, we announced that the FDA approved OPZELURA (ruxolitinib) cream, a novel cream formulation of Incyte’s selective JAK1/JAK2 inhibitor ruxolitinib, for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (AD) in non-immunocompromised patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable.

In September 2021, the FDA approved OPZELURA (ruxolitinib) cream, a novel cream formulation of Incyte’s selective JAK1/JAK2 inhibitor ruxolitinib, for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (“AD”) in non-immunocompromised patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable.

AD is a skin disorder that causes long term inflammation of the skin resulting in itchy, red, swollen and cracked skin.

Onset can occur at any age but is more common in infants and children.

In the United States, we estimate that there are approximately 10 million diagnosed adolescent and adult patients with AD.

The approval of OPZELURA was based on data from two randomized, double-blind, vehicle-controlled Phase 3 studies (TRuE-AD1 and TRuE-AD 2) evaluating the safety and efficacy of OPZELURA in adolescents and adults with mild to moderate AD.

Significantly more patients treated with OPZELURA achieved Investigator’s Global Assessment (IGA) Treatment Success at Week 8 (defined as an IGA score of 0 or 1 with at least a 2-point improvement from baseline, the primary endpoint:

53.8% in TRuE-AD1 and 51.3% in TRuE-AD2, compared to vehicle (15.1% in TRuE-AD1, 7.6% in TRuE-AD2;

Significantly more patients treated with OPZELURA experienced a clinically meaningful reduction in itch from baseline at Week 8, as measured by a ≥4-point reduction in the itch Numerical Rating Scale (itch NRS4):

52.2% in TRuE-AD1 and 50.7% in TRuE-AD2, compared to vehicle (15.4% in TRuE-AD1, 16.3% in TRuE-AD2;

P<0.0001), among patients with an NRS score of at least 4 at baseline.

The most common (≥1%) treatment-emergent adverse reactions in patients treated with OPZELURA were nasopharyngitis, diarrhea, bronchitis, ear infection, eosinophil count increased, urticaria, folliculitis, tonsillitis and rhinorrhea.

1 In collaboration with Merus.

In July 2022, we announced that the FDA approved OPZELURA for the topical treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older.

In September 2025, the FDA approved the supplemental New Drug Application (“sNDA”) for OPZELURA for the short-term and non-continuous chronic treatment of mild to moderate AD in non-immunocompromised children two years of age and older whose disease is not well controlled with topical prescription therapies, or when those therapies are not recommended.

In July 2022, the FDA approved OPZELURA for the topical treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older.

OPZELURA was approved for continuous use and no limits to duration as a treatment for nonsegmental vitiligo.

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OPZELURA is the first and only FDA approved treatment for repigmentation of vitiligo lesions.

The approval of OPZELURA in vitiligo was based on two randomized, double-blind, vehicle-controlled Phase 3 studies (TRuE-V1 and TRuE-V2) evaluating the safety and efficacy of OPZELURA in adolescents and adults with nonsegmental vitiligo.

Treatment with 1.5% ruxolitinib cream twice daily (BID) resulted in greater improvement versus vehicle for the primary and all key secondary endpoints in both the TRuE-V1 and TRuE-V2 studies.

Results, which were consistent across both studies, showed that 29.9% of patients applying ruxolitinib cream achieved >75% improvement from baseline in the facial Vitiligo Area Scoring Index (F-VASI75) at Week 24, the primary endpoint.

At Week 52, approximately 50% of patients achieved F-VASI75.

The most common (>1%) treatment-emergent adverse reactions in patients treated with OPZELURA were application site acne, application site pruritus, nasopharyngitis, headache, urinary tract infection, application site erythema and pyrexia.

In March 2023, long-term 104-week safety and efficacy data for ruxolitinib cream in vitiligo were presented at the American Academy of Dermatology (AAD) conference, demonstrating that patients who achieved a high level of facial repigmentation (≥F-VASI90) at Week 52 maintained durable response one year following withdrawal of treatment and that those patients who continued treatment with OPZELURA for up to two years demonstrated sustained facial repigmentation and further improvements in facial and total body repigmentation.

In April 2023, we announced that the European Commission had approved OPZELURA for the topical treatment of nonsegmental vitiligo with facial involvement in adults and adolescents 12 years and older following a positive opinion from the CHMP.

In October 2023, new results of a pooled analysis of long-term extension (LTE) data from the pivotal Phase 3 TRuE-V program assessing OPZELURA cream 1.5% in patients 12 years of age and older with nonsegmental vitiligo who previously experienced limited or no response to treatment at Week 24 were presented at the European Academy of Dermatology and Venereology (EADV) Congress 2023 as a late-breaking oral presentation.

These results showed that patients who initially experienced limited or no facial or total body repigmentation at six months achieved improved repigmentation after continued treatment with OPZELURA for up to two years.

In April 2023, the European Commission approved OPZELURA for the topical treatment of nonsegmental vitiligo with facial involvement in adults and adolescents 12 years and older following a positive opinion from the Committee for Medicinal Products for Human Use (“CHMP”).

In October 2024, OPZELURA cream 1.5% was granted a Notice of Compliance by Health Canada for the topical treatment of both mild to moderate AD and nonsegmental vitiligo in patients 12 years of age and older.

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Atopic Dermatitis.

In July 2023, we announced that the Phase 3 trial (TRuE-AD3) evaluating ruxolitinib cream in pediatric AD patients (age > 2 and <12) had met its primary endpoint.

The study showed that significantly more patients treated with ruxolitinib cream 0.75% and 1.5% achieved Investigator's Global Assessment Treatment Success (IGA-TS) than patients treated with vehicle control.

In October 2023, the expanded results from the pivotal Phase 3 TRuE-AD3 were presented at EADV.

Again, significantly more patients treated with ruxolitinib cream (0.75% and 1.5%) achieved Investigator’s Global Assessment Treatment Success (IGA-TS) than patients treated with vehicle control (non-medicated cream).

In October 2024, the supplemental New Drug Application (sNDA) submission for ruxolitinib cream in pediatric atopic dermatitis was filed with the FDA with approval anticipated in the second half of 2025.

In June 2025, the FDA extended the review period for the sNDA for ruxolitinib cream for the treatment of children 2-11 years old with mild to moderate AD.

The Prescription Drug User Fee Act (PDUFA) action date was extended by three months to September 19, 2025.

In July 2025, we announced positive topline results from the Phase 3 (TRuE-AD4) study evaluating ruxolitinib cream in adult patients with moderate atopic dermatitis.

The study met the co-primary endpoints at Week 8, with a statistically significant proportion of patients achieving both Investigator's Global Assessment Treatment Success (IGA-TS) and EASI75, which is defined as a 75% or greater improvement in the Eczema Area Severity Index score from baseline.

The study met the co-primary endpoints at Week 8, with a statistically significant proportion of patients achieving both Investigator’s Global Assessment Treatment Success and EASI75, which is defined as a 75% or greater improvement in the Eczema Area Severity Index score from baseline.

In addition, the study met all key secondary endpoints.

Ruxolitinib cream was well tolerated with no new safety signals.

The full dataset will be presented at an upcoming medical conference.

Hidradenitis Suppurativa.

In January 2024, we announced positive topline results from a randomized controlled Phase 2 study evaluating ruxolitinib cream in HS.

Ruxolitinib 1.5% cream BID met the primary efficacy endpoint as measured by a change from baseline in abscess and nodule count at Week 16 versus placebo in patients with mild to moderate HS.

Ruxolitinib 1.5% cream twice daily met the primary efficacy endpoint as measured by a change from baseline in abscess and nodule count at Week 16 versus placebo in patients with mild to moderate HS.

Ruxolitinib cream was well tolerated and consistent with its known safety profile.

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In March 2025, results from two Phase 3 studies (TRuE-PN1 and TRuE-PN2) evaluating ruxolitinib cream in patients with PN were presented in a late-breaking oral session at the American Academy of Dermatology annual meeting.

The TRuE-PN1 study met the primary endpoint of a > 4-point improvement from baseline in Worst-Itch Numeric Rating Scale (WI-NRS4) at Week 12 and all key secondary endpoints.

The TRuE-PN1 study met the primary endpoint of a > 4-point improvement from baseline in Worst-Itch Numeric Rating Scale at Week 12 and all key secondary endpoints.

The TRuE-PN2 study did not reach statistical significance for the primary endpoint, resulting in the key secondary endpoints with nominal p-values.

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We also are developing povorcitinib, which is an oral small molecule selective JAK1 inhibitor.

Povorcitinib is undergoing evaluation in patients with hidradenitis suppurativa, nonsegmental vitiligo, prurigo nodularis, asthma and chronic spontaneous urticaria (CSU).

Povorcitinib is undergoing evaluation in patients with HS, nonsegmental vitiligo, PN, asthma and chronic spontaneous urticaria (“CSU”).

Hidradenitis Suppurativa.

HS is a chronic skin condition where lesions develop as a result of inflammation and infection of the sweat glands.

In October 2020, initial results from the clinical program were presented and a randomized Phase 2b trial of povorcitinib was initiated in patients with HS.

In August 2022, we presented positive results from the Phase 2 trial of povorcitinib in HS.

In December 2022, we initiated two Phase 3 trials (STOP-HS1 and STOP-HS2) in moderate to severe HS.

In February 2023, 52-week results from the Phase 2 study evaluating povorcitinib in HS were presented as an oral presentation at the European Hidradenitis Suppurativa Foundation (EHSF) Annual Meeting.

The data demonstrated that longer-term treatment with povorcitinib 75 mg resulted in sustained and durable efficacy across all treatment arms and that importantly, 22-29% of patients achieved HiSCR100, which is defined as a 100% reduction from baseline in total AN count with no increase from baseline in abscess or draining tunnel count.

In March 2025, positive results from two Phase 3 studies (STOP-HS1 and STOP-HS2) evaluating povorcitinib in patients with HS were presented and demonstrated that both studies met their primary endpoint of Hidradenitis Suppurativa Clinical Response (HiSCR) at Week 12 and at both tested doses (45mg and 75mg).

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Furthermore, povorcitinib demonstrated rapid onset of response, including rapid skin pain reduction.

These data support the planned regulatory submission of povorcitinib for the treatment of HS worldwide.

In September 2025, additional data from the STOP-HS1 and STOP-HS2 studies were presented at the European Association of Dermatology and Venereology (EADV) Annual Meeting.

In the studies, povorcitinib demonstrated sustained improvements in symptoms for patients with active moderate to severe hidradenitis suppurativa (HS) through 24 weeks.

These data support the planned regulatory submissions of povorcitinib for the treatment of HS in 2025 and 2026.

Nonsegmental Vitiligo.

In March 2023, 36-week results from the Phase 2b study evaluating povorcitinib in patients with extensive nonsegmental vitiligo were presented as an oral late-breaking presentation at the American Academy of Dermatology (AAD) Annual Meeting.

The data demonstrated that treatment with oral povorcitinib was associated with substantial total body repigmentation in patients with extensive nonsegmental vitiligo, as measured by total Vitiligo Area Scoring Index (T-VASI) scores.

Specifically, the study met its primary endpoint, and patients receiving povorcitinib experienced statistically superior improvements in T-VASI at Week 24 compared to placebo.

In October 2023, positive 52-week data from a Phase 2b clinical trial evaluating the safety and efficacy of povorcitinib in adult patients with extensive nonsegmental vitiligo were presented at EADV as a late-breaking oral presentation.

Results showed that treatment with oral povorcitinib was associated with substantial total body and facial repigmentation across all treatment groups at Week 52 and further reinforces the efficacy profile and potential of povorcitinib as an oral treatment for patients with extensive nonsegmental vitiligo.

In March and October 2023, we presented results from the Phase 2b clinical trial evaluating povorcitinib in patients with extensive nonsegmental vitiligo which demonstrated that treatment with oral povorcitinib was associated with substantial total body and facial repigmentation, as measured by total Vitiligo Area Scoring Index.

Prurigo Nodularis.

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In October 2024, following the positive Phase 2 results, two Phase 3 studies in patients with PN were initiated.

Asthma and Chronic Spontaneous Urticaria.

In July 2023, we initiated two Phase 2 trials evaluating povorcitinib in patients with moderate to severe uncontrolled asthma and in chronic spontaneous urticaria.

Data for CSU are anticipated in the first half of 2025 and data in asthma are anticipated in the second half of 2025.

In April 2025, we announced positive topline results from the Phase 2 study evaluating povorcitinib in patients with chronic spontaneous urticaria.

The study met the primary endpoint at Week 12 of change from baseline in the Urticaria Activity Score summed over 7 days (UAS7).

Asthma and Chronic Spontaneous Urticaria (CSU).

In July 2023, we initiated a Phase 2 trial evaluating povorcitinib in patients with moderate to severe uncontrolled asthma.

Data from this proof-of-concept study is anticipated in 2026.

In April 2025, we announced positive topline results from the Phase 2 study evaluating povorcitinib in patients with CSU.

The study met the primary endpoint at Week 12 of change from baseline in the Urticaria Activity Score summed over 7 days.

Povorcitinib was well tolerated with no new safety signals observed.

These data will support planned discussions with regulatory agencies and will be presented at an upcoming medical conference.

INCB000262 (MRGPRX2) & INCB000547 (MRGPRX4)

As more fully described in Note 6 of Notes to the Condensed Consolidated Financial Statements, in May 2024, we acquired Escient Pharmaceuticals, Inc.

Escient is a clinical-stage drug discovery and development company advancing novel small molecule therapeutics for systemic immune and neuro-immune disorders.

Escient’s clinical development portfolio includes INCB000262 , a potent, highly selective, once-daily small molecule antagonist of Mas-related G protein-coupled receptor (MRGPRX2) and INCB000547 , an oral MRGPRX4 antagonist.

In November 2024, we announced that enrollment was paused in the ongoing Phase 2 study of MRGPRX2 (INCB000262) in CSU following the observation of certain in vivo preclinical toxicology findings.

Subsequently, we made the decision to not pursue further development.

In November 2024, we announced that data from the Phase 2 study evaluating MRGPRX4 (INCB000547) in cholestatic pruritus did not support further development.

IAI and Dermatology Programs Indication and Phase

Ruxolitinib cream (OPZELURA) 1

Atopic dermatitis:

Phase 3 pediatric study (TRuE-AD3);

sNDA under review in the U.S.

Hidradenitis suppurativa:

Phase 3 (TRuE-HS1, TRuE-HS2)

Prurigo nodularis:

Phase 3 (TRuE-PN1, TRuE-PN2)

(JAK1) Hidradenitis suppurativa:

Phase 3 (STOP-HS1, STOP-HS2)

Phase 3 (STOP-V1, STOP-V2)

Prurigo nodularis:

Phase 3 (STOP-PN1, STOP-PN2)

Chronic spontaneous urticaria:

(anti-CD122) Vitiligo:

Novartis’ rights for ruxolitinib outside of the United States under our Collaboration and License Agreement with Novartis do not include topical administration.

Earlier-Stage Development Programs in Dermatology

In October 2025, we decided not to pursue further development of povorcitinib in CSU to prioritize other programs.

Other Clinical Programs

INCA034460 (anti-CD122)

In November 2022, we acquired Villaris Therapeutics, Inc., an asset-centric biopharmaceutical company focused on the development of novel antibody therapeutics for vitiligo.

INCA034460 is a novel, humanized anti-IL-15Rβ monoclonal antibody designed to target and deplete autoreactive tissue resident memory T cells (TRM) that has demonstrated efficacy as a treatment for vitiligo in preclinical models.

In July 2023, INCA034460 received Investigational New Drug application (IND) clearance and in October 2023, we announced the first patient had been dosed.

Other Clinical Programs

INCA034460 is a novel, humanized anti-IL-15Rβ monoclonal antibody designed to target and deplete autoreactive tissue resident memory T cells that has demonstrated efficacy as a treatment for vitiligo in preclinical model s.

In October 2025, we paused further development of INCA034460.

INCB00928 (zilurgisertib)

In May 2022, we initiated a Phase 2 trial evaluating zilurgisertib (INCB00928) in patients with fibrodysplasia ossificans progressiva (“FOP”), a disorder in which muscle tissue and connective tissue are gradually replaced by bone.

The FDA has granted Fast Track designation and orphan drug designation to zilurgisertib as a treatment for patients with FOP.

Other Clinical Program Indication and Phase

Zilurgisertib

(ALK2) Fibrodysplasia ossificans progressiva:

Pivotal Phase 2

Collaborative Partnered Programs

As described below under “License Agreements and Business Relationships,” we are eligible for milestone payments and royalties on certain products that we licensed to third parties.

These include OLUMIANT (baricitinib), which is licensed to our collaborative partner Eli Lilly and Company, and JAKAVI (ruxolitinib) and TABRECTA (capmatinib), which are licensed to Novartis.

These include OLUMIANT (baricitinib), which is licensed to our collaborative partner Eli Lilly and Company (“Lilly”), and JAKAVI (ruxolitinib) and TABRECTA (capmatinib), which are licensed to Novartis.

We have a second JAK1 and JAK2 inhibitor, baricitinib, which is subject to our collaboration agreement with Lilly, in which Lilly received exclusive worldwide development and commercialization rights to the compound for inflammatory and autoimmune diseases.

Rheumatoid Arthritis.

Rheumatoid arthritis is an autoimmune disease characterized by aberrant or abnormal immune mechanisms that lead to joint inflammation and swelling and, in some patients, the progressive destruction of joints.

Rheumatoid arthritis also can affect connective tissue in the skin and organs of the body.

Current rheumatoid arthritis treatments include the use of non-steroidal anti-inflammatory drugs, disease-modifying anti-rheumatic drugs such as methotrexate, and the newer biological response modifiers that target pro-inflammatory cytokines, such as tumor necrosis factor, implicated in the pathogenesis of rheumatoid arthritis.

None of these approaches to treatment is curative;

therefore, there remains an unmet need for safe and effective treatment options for these patients.

Rheumatoid arthritis is estimated to affect about 1% of the world’s population.

The Phase 3 program of baricitinib in patients with rheumatoid arthritis incorporated all three rheumatoid arthritis populations (methotrexate naïve, biologic naïve, and tumor necrosis factor (TNF) inhibitor inadequate responders);

used event rates to fully power the baricitinib program for structural comparison and non-inferiority vs.

and evaluated patient-reported outcomes.

All four Phase 3 trials met their respective primary endpoints.

In January 2016, Lilly submitted a New Drug Application (NDA) to the FDA and a Marketing Authorization Application to the European Medicines Agency (EMA) for baricitinib as treatment for rheumatoid arthritis.

In February 2017, we and Lilly announced that the European Commission approved baricitinib as OLUMIANT for the treatment of moderate-to-severe rheumatoid arthritis in adult patients who have responded inadequately to, or who are intolerant to, one or more disease-modifying antirheumatic drugs (DMARDs).

In February 2017, the European Commission approved baricitinib as OLUMIANT for the treatment of moderate-to-severe rheumatoid arthritis in adult patients who have responded inadequately to, or who are intolerant to, one or more disease-modifying antirheumatic drugs.

In July 2017, the MHLW granted marketing approval for OLUMIANT for the treatment of rheumatoid arthritis (including the prevention of structural injury of joints) in patients with inadequate response to standard-of-care therapies.

In June 2018, the FDA approved the 2mg dose of OLUMIANT for the treatment of adults with moderately-to-severely active rheumatoid arthritis (RA) who have had an inadequate response to one or more tumor necrosis factor (TNF) inhibitor therapies.

In June 2018, the FDA approved the 2mg dose of OLUMIANT for the treatment of adults with moderately-to-severely active rheumatoid arthritis who have had an inadequate response to one or more TNF inhibitor therapies.

Atopic Dermatitis.

Lilly has conducted a Phase 2a trial and a Phase 3 program to evaluate the safety and efficacy of baricitinib in patients with moderate-to-severe atopic dermatitis.

The JAK-STAT pathway has been shown to play an essential role in the dysregulation of immune responses in atopic dermatitis.

Therefore, we believe that inhibiting cytokine pathways dependent on JAK1 and JAK2 may lead to positive clinical outcomes in AD.

In February 2019, we and Lilly announced that baricitinib met the primary endpoint in BREEZE-AD1 and BREEZE-AD2, two Phase 3 studies evaluating the efficacy and safety of baricitinib monotherapy for the treatment of adult patients with moderate-to-severe AD and, in August 2019, we and Lilly announced that baricitinib met the primary endpoint in BREEZE-AD7, a Phase 3 study evaluating the efficacy and safety of baricitinib in combination with standard-of-care topical corticosteroids in patients with moderate-to-severe AD.

In January 2020, we and Lilly announced that baricitinib met the primary endpoint in both BREEZE-AD4 and BREEZE-AD5, the results of which completed the placebo-controlled data program intended to support global registrations.

A sNDA for baricitinib was submitted by Lilly for the treatment of patients with moderate to severe AD.

In April 2021, we and Lilly announced the FDA extended the review period for the sNDA for baricitinib for the treatment of moderate to severe AD by three months to allow time for additional data analyses.

In July 2021, we and Lilly announced that the FDA will not meet the PDUFA action date for the sNDA for baricitinib for the treatment of adults with moderate to severe AD due to the FDA's ongoing assessment of JAK inhibitors.

In January 2022, Lilly provided a regulatory update on the sNDA based on ongoing discussions with the FDA.

Lilly announced that alignment with the FDA on the indicated population had not yet been reached and given the FDA’s position, there would be the possibility of a Complete Response Letter.

In January 2020, Lilly announced that baricitinib had been submitted for regulatory review in Europe as a treatment for patients with moderate-to-severe AD.

In October 2020, Lilly announced that the European Commission approved baricitinib as OLUMIANT for the treatment of moderate-to-severe AD in adult patients who are candidates for systemic therapy.

Lilly has conducted a Phase 2a trial and a Phase 3 program to evaluate the safety and efficacy of baricitinib in patients with moderate-to-severe AD.

In October 2020, the European Commission approved baricitinib as OLUMIANT for the treatment of moderate-to-severe AD in adult patients who are candidates for systemic therapy.

In December 2020, baricitinib was approved by the MHLW for the treatment of patients with moderate-to-severe AD.

Alopecia Areata .

Alopecia areata is an autoimmune disorder in which the immune system attacks the hair follicles, causing hair loss in patches.

In March 2020, Lilly announced that baricitinib received Breakthrough Therapy designation for the treatment of alopecia areata, based on the positive Phase 2 results of Lilly’s adaptive Phase 2/3 study BRAVE-AA1.

In March 2021, we and Lilly announced positive results from BRAVE-AA2, the Phase 3 trial evaluating the efficacy and safety of once-daily baricitinib in adults with severe alopecia areata.

In April 2021, we and Lilly announced positive results from the Phase 3 portion of BRAVE-AA1.

In September 2021, we and Lilly announced detailed results from BRAVE-AA1 and BRAVE-AA2 at the European Academy of Dermatology and Venereology Congress (EADV).

The two studies showed statistically significant improvement in scalp hair regrowth across both baricitinib dosing groups when compared to placebo.

In March 2022, we and Lilly announced positive 52 week results from BRAVE-AA1 and BRAVE-AA2 at the American Academy of Dermatology (AAD) annual meeting showing 40% of adults saw at least 80% scalp coverage.

In June 2022, the FDA approved 2mg, and 4mg doses of OLUMIANT for the treatment of adults with severe alopecia areata, becoming the first and only systemic treatment in the indication.

In June 2022, the FDA approved 2mg, and 4mg doses of OLUMIANT for the treatment of adults with severe alopecia areata, an autoimmune disorder in which the immune system attacks the hair follicles causing hair loss in patches, becoming the first and only systemic treatment in the indication.

In June 2022, OLUMIANT was approved as a treatment for alopecia areata in Europe and Japan.

Systemic Lupus Erythematosus.

Systemic lupus erythematosus (SLE) is a chronic disease that causes inflammation.

In addition to affecting the skin and joints, it can affect other organs in the body such as the kidneys, the tissue lining the lungs and heart, and the brain.

Lilly has conducted a Phase 2 trial to evaluate the safety and efficacy of baricitinib in patients with SLE.

Baricitinib’s activity profile suggests that it inhibits cytokines implicated in SLE such as type I interferon (IFN), type II IFN-γ, IL-6, and IL-23 as well as other cytokines that may have a role in SLE, including granulocyte macrophage colony stimulating factor (GM-CSF) and IL-12.

In January 2022, Lilly announced the discontinuation of the Phase 3 development program for baricitinib in SLE based on top-line efficacy results from two pivotal Phase 3 trials (SLE-BRAVE-I and –II).

The primary endpoint of SRI-4 response was reached in SLE-BRAVE-I but was not reached in SLE-BRAVE-II and key secondary endpoints were not met in either study.

In May 2020, we amended our agreement with Lilly to enable Lilly to commercialize baricitinib for the treatment of COVID-19.

In November 2020, we and Lilly announced that the FDA issued an Emergency Use Authorization (EUA) for the distribution and emergency use of baricitinib to be used in combination with remdesivir in hospitalized adult and pediatric patients two years of age or older with suspected or laboratory confirmed COVID-19 who require supplemental oxygen, invasive mechanical ventilation, or extracorporeal membrane oxygenation.

In December 2020, we and Lilly announced that data from ACTT-2 supportive of the EUA were published in the New England Journal of Medicine.

In July 2021, we and Lilly announced that the FDA broadened the EUA for baricitinib to allow for treatment with or without remdesivir.

The EUA now provides for the use of baricitinib for treatment of COVID-19 in hospitalized adults and pediatric patients two years of age or older requiring supplemental oxygen, non-invasive or invasive mechanical ventilation or extracorporeal membrane oxygenation (ECMO).

In June 2022, we and Lilly announced the FDA approved baricitinib as OLUMIANT for the treatment of COVID-19 in hospitalized adults requiring supplemental oxygen, non-invasive or invasive mechanical ventilation or ECMO.

Capmatinib is a potent and highly selective MET inhibitor.

The FDA’s Emergence Use Authorization provides for the use of baricitinib for the treatment of COVID-19 in hospitalized adults and pediatric patients two years of age or older requiring supplemental oxygen, non-invasive or invasive mechanical ventilation or extracorporeal membrane oxygenation (“ECMO”).

In June 2022, the FDA approved baricitinib as OLUMIANT for the treatment of COVID-19 in hospitalized adults requiring supplemental oxygen, non-invasive or invasive mechanical ventilation or ECMO.

Type 1 Diabetes .

In October 2025, we amended our agreement with Lilly to enable Lilly to commercialize baricitinib for the treatment of Type 1 diabetes mellitus.

Capmatinib is a potent and highly selective mesenchymal-epithelial-transition factor gene (“MET”) inhibitor.

The investigational compound has demonstrated inhibitory activity in cell-based biochemical and functional assays that measure MET signaling and MET dependent cell proliferation, survival and migration.

Under our agreement, Novartis received worldwide exclusive development and commercialization rights to capmatinib and certain back-up compounds in all indications.

Capmatinib is being evaluated in patients with hepatocellular carcinoma, non-small cell lung cancer and other solid tumors, and may have potential utility as a combination agent.

MET is a clinically validated receptor kinase cancer target.

Abnormal MET activation in cancer correlates with poor prognosis.

Dysregulation of the MET pathway triggers tumor growth, formation of new blood vessels that supply the tumor with nutrients and causes cancer to spread to other organs.

Dysregulation of the MET pathway is seen in many types of cancers, including lung, kidney, liver, stomach, breast and brain.

In May 2020, we and Novartis announced the FDA approval of capmatinib as TABRECTA for the treatment of adult patients with metastatic NSCLC whose tumors have a mutation that leads to MET exon 14 skipping (METex14) as detected by an FDA-approved test.

Capmatinib is being evaluated in patients with hepatocellular carcinoma, NSCLC and other solid tumors, and may have potential utility as a combination agent.

In May 2020, the FDA approved capmatinib as TABRECTA for the treatment of adult patients with metastatic NSCLC whose tumors have a mutation that leads to MET exon 14 (“METex14”) skipping as detected by an FDA-approved test.

TABRECTA is the first and only treatment approved to specifically target NSCLC with this driver mutation and is approved for first-line and previously treated patients regardless of prior treatment type.

The FDA approval of TABRECTA was based on results from the pivotal GEOMETRY mono-1 study.

In the METex14 population (n=97), the confirmed overall response rate was 68% and 41% among treatment-naive (n=28) and previously treated patients (n=69), respectively, based on the Blinded Independent Review Committee (BIRC) assessment per RECIST v1.1.

In patients taking TABRECTA, the study also demonstrated a median duration of response of 12.6 months in treatment-naive patients (19 responders) and 9.7 months in previously treated patients (28 responders).

The most common treatment-related adverse events (AEs) (incidence ≥20%) are peripheral edema, nausea, fatigue, vomiting, dyspnea, and decreased appetite.

In September 2020, we and Novartis announced that GEOMETRY mono-1 results were published in The New England Journal of Medicine.

In June 2020, we and Novartis announced that the MHLW approved TABRECTA for METex14 mutation-positive advanced and/or recurrent unresectable NSCLC.

In April 2022, we and Novartis announced a positive opinion from the CHMP based on data from the Phase 2 GEOMETRY mono-1 study showing an overall response rate (ORR) of 51.6% in a cohort evaluating second-line patients only and 44% in all previously-treated patients with advanced NSCLC harboring alterations leading to MET exon 14 skipping.

In June 2022, we and Novartis announced the European Commission approval of capmatinib as TABRECTA as monotherapy treatment of adults with advanced NSCLC harboring alterations leading to mesenchymal-epithelial-transition factor gene (MET) exon 14 (METex14) skipping who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy.

NSCLC is the most common type of lung cancer, impacting more than 2 million people per year globally.

Approximately 3-4 percent of all patients with NSCLC have tumors with a mutation that leads to MET exon 14 skipping.

Though rare, this mutation is an indicator of especially poor prognosis and poor responses to standard therapies, including immunotherapy.

In June 2020, the MHLW approved TABRECTA for METex14 mutation-positive advanced and/or recurrent unresectable NSCLC.

In April 2022, we and Novartis announced a positive opinion from the CHMP based on data from the Phase 2 GEOMETRY mono-1 study.

In June 2022, the European Commission approved capmatinib as TABRECTA as a monotherapy treatment of adults with advanced NSCLC harboring alterations leading to METex14 skipping who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy.

Graft-versus-host disease.

In March 2022, we and Novartis announced a positive opinion from the CHMP for ruxolitinib in acute and chronic GVHD, based on data from the Phase 3 REACH2 and REACH3 trials.

GVHD is a life-threatening complication of stem cell transplants, with no established standard of care in Europe for patients who do not adequately respond to first-line steroid treatment.

In May 2022, we and Novartis announced the European Commission approval of ruxolitinib as JAKAVI for the treatment of acute or chronic GVHD in patients aged 12 years and older who have inadequate response to corticosteroids or other systemic therapies.

In August 2023, Novartis announced that JAKAVI had been approved in Japan for use in graft-versus-host disease after hematopoietic stem cell transplant.

Partnered Programs Indication and Phase

Ruxolitinib (JAKAVI) 1

Acute and chronic GVHD:

Approved in Europe and Japan

Baricitinib (OLUMIANT) 2

Approved in Europe and Japan

Severe alopecia areata (AA):

Approved in the U.S., Europe and Japan

Capmatinib (TABRECTA) 3

NSCLC (with MET exon 14 skipping mutations):

Approved in the U.S., Europe and Japan

Ruxolitinib (JAKAVI) licensed to Novartis outside of the United States for use in hematology and oncology excluding topical administration.

Baricitinib (OLUMIANT) licensed to Lilly.

Capmatinib (TABRECTA) licensed to Novartis.

In May 2022, the European Commission approved ruxolitinib as JAKAVI for the treatment of acute or chronic GVHD in patients aged 12 years and older who have inadequate response to corticosteroids or other systemic therapies.

In August 2023, Novartis announced that JAKAVI had been approved in Japan for use in GVHD after hematopoietic stem cell transplant.

License Agreements and Business Relationships

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In March 2016, we entered into an amendment to the agreement with Lilly that allows us to engage in the development and commercialization of ruxolitinib in the GVHD field.

In May 2020, we amended our agreement with Lilly to enable Lilly to commercialize baricitinib for the treatment of COVID-19.

In May 2020, we amended our agreement with Lilly to enable Lilly to commercialize baricitinib for the treatment of COVID-19 and in October 2025, we further amended the agreement to enable Lilly to commercialize baricitinib for the treatment of Type 1 diabetes mellitus.

We will receive an upfront payment of $100.0 million in connection with the 2025 amendment, which amendment also restructured the royalty obligations on net sales of baricitinib, certain developmental and regulatory milestones associated with baricitinib, and the marketing and sales support obligations of Lilly.

On baricitinib sales for any indication, we are now eligible to receive either a fixed royalty amount or tiered royalties based on a defined level of quarterly global net sales, with the tiered royalties up to a rate in the mid-teens.

Additionally, for the treatment of COVID-19, we still receive a premium on royalties.

In-License Agreements

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Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Annual Report on Form 10-K for the year ended December 31, 2024.

There have been no significant changes to our critical accounting policies or estimates during the six months ended June 30, 2025.

There have been no significant changes to our critical accounting policies or estimates during the nine months ended September 30, 2025.

Recent Accounting Pronouncements and Regulatory Updates

In December 2023, the FASB issued ASU No.

In December 2023, the Financial Accounting Standards Board (FASB) issued ASU No.

2023-09, “ Income Taxes (Topic 740):

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We are currently evaluating the impact that ASU No.

2023-09 will have on our condensed consolidated financial statements.

2023-09 will have on our income tax disclosures and the method of adoption.

2023-09 does not affect our results of operations, financial condition or cash flows.

In November 2024, the FASB issued ASU No.

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2024-03 will have on our condensed consolidated financial statements and related disclosures.

In July 2025, the FASB issued ASU No.

2025-05, “ Financial Instruments - Credit Losses (Topic 326):

Measurement of Credit Losses for Accounts Receivable and Contract Assets.

” This amended guidance applies to all entities and aims to simplify the estimation of expected credit losses for current accounts receivable and contract assets by providing a practical expedient for all companies and an accounting policy election for non-public companies.

The amendments are effective for annual reporting periods beginning after December 15, 2025 and interim reporting periods within those annual periods.

If electing the practical expedient or accounting policy election (if applicable), entities should apply the amendments in this update prospectively.

We are currently evaluating the impact ASU No.

2025-05 will have on our consolidated financial statements and related disclosures.

In September 2025, the FASB issued ASU No.

2025-06, “ Intangibles - Goodwill and Other - Internal-Use (Subtopic 350-40):

Targeted Improvements to the Accounting for Internal-Use Software.

” This amended guidance applies to all entities and serves to modernize the accounting for software costs that are accounted for under Subtopic 305-40, Intangibles - Goodwill and Other - Internal-Use Software (referred to as "internal-use software").

The amendments in this update are effective for all entities for annual reporting periods beginning after December 15, 2027, and interim reporting periods within those annual reporting periods.

Early adoption is permitted as of the beginning of an annual reporting period.

Entities may adopt the new guidance using a prospective, modified, or retrospective transition approach.

We are currently evaluating the impact ASU No.

2025-06 will have on our consolidated financial statements and related disclosures.

In September 2025, the FASB issued ASU No.

2025-07, “ Derivatives and Hedging (Topic 815) and Revenue from Contracts with Customers (Topic 606):

Derivatives Scope Refinements and Scope Clarification for Share-Based Noncash Consideration from a Customer in a Revenue Contract.

” This amended guidance applies to all entities and it refines the scope of derivative accounting and clarifies rules for share-based noncash consideration in revenue contracts.

Specifically, this update is intended to address concerns about the application of derivative accounting to contracts that have features based on the operations or activities of one of the parties to the contract and to reduce diversity in the accounting for share-based payments in revenue contracts.

The amendments in this update are effective for all entities for annual reporting periods beginning after December 15, 2026, and interim reporting periods within those annual reporting periods.

Early adoption is permitted.

Entities may adopt the new guidance prospectively, or on a modified retrospective basis.

We are currently evaluating the impact ASU No.

2025-07 will have on our consolidated financial statements and related disclosures.

Results of Operations

We recorded net income of $405.0 million and basic net income per share of $2.09 and diluted net income per share of $2.04 for the three months ended June 30, 2025, as compared to net loss of $444.6 million and basic and diluted net loss per share of $2.04 in the corresponding period in 2024.

We recorded net income of $563.2 million and basic net income per share of $2.91 and diluted net income per share of $2.84 for the six months ended June 30, 2025, as compared to net loss of $275.1 million and basic and diluted net loss per share of $1.24 in the corresponding period in 2024.

We recorded net income of $424.2 million and basic net income per share of $2.17 and diluted net income per share of $2.11 for the three months ended September 30, 2025, as compared to net income of $106.5 million and basic net income per share of $0.55 and diluted net income per share of $0.54 in the corresponding period in 2024.

We recorded net income of $987.4 million and basic net income per share of $5.08 and diluted net income per share of $4.95 for the nine months ended September 30, 2025, as compared to net loss of $168.6 million and basic and diluted net loss per share of $0.80 in the corresponding period in 2024.

Three Months Ended

June 30, Six Months Ended

September 30, Nine Months Ended

September 30,

2025 2024 2025 2024

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Total revenues $ 1,366.0 $ 1,137.9 $ 3,634.4 $ 3,062.5

The increase in JAKAFI for the three and six months ended June 30, 2025 as compared to the corresponding periods in 2024 was primarily driven by an increase in paid demand reflecting continued demand growth in all indications.

JAKAFI inventory levels were within normal range at the end of the second quarter of 2025.

The increase in OPZELURA net product revenues for the three and six months ended June 30, 2025 as compared to the corresponding periods in 2024 was primarily due to increased patient demand and refills in the U.S.

The increase in JAKAFI for the three and nine months ended September 30, 2025 as compared to the corresponding periods in 2024 was primarily driven by an increase in paid demand reflecting continued demand growth in all indications.

JAKAFI inventory levels were within normal range at the end of the third quarter of 2025.

The increase in OPZELURA net product revenues for the three and nine months ended September 30, 2025 as compared to the corresponding periods in 2024 was primarily due to increased patient demand and refills in the U.S.

in both atopic dermatitis and vitiligo.

Additionally, $32.3 million of net product revenues during the second quarter of 2025 were from outside of the U.S., driven by continued uptake in France, as well as growth from the recent launches in Italy and Spain.

OPZELURA inventory levels were within normal range at the end of the second quarter of 2025.

NIKTIMVO net product revenues for the three and six months ended June 30, 2025 reflects continued strong uptake of the product following its commercial launch during the first quarter of 2025.

The increase in ZYNYZ net product revenue for the three and six months ended June 30, 2025 was primarily driven by the approval of the product in squamous cell anal carcinoma in the second quarter of 2025.

The increase in total royalty revenues for the three and six months ended June 30, 2025 as compared to the corresponding periods in 2024 was primarily driven by growth in JAKAVI royalty revenue.

Additionally, $44.4 million of net product revenues during the third quarter of 2025 were from outside of the U.S., driven by continued uptake in France and Italy in vitiligo.

OPZELURA inventory levels were within normal range at the end of the third quarter of 2025.

NIKTIMVO net product revenues for the three and nine months ended September 30, 2025 reflects continued strong uptake of the product following its commercial launch during the first quarter of 2025.

The increase in ZYNYZ net product revenue for the three and nine months ended September 30, 2025 was primarily driven by the approval of the product in squamous cell anal carcinoma in the second quarter of 2025.

The increase in total royalty revenues for the three and nine months ended September 30, 2025 as compared to the corresponding periods in 2024 was primarily driven by growth in JAKAVI royalty revenue.

Our product revenues may fluctuate from quarter to quarter due to our customers’ purchasing patterns over the course of the year, including as a result of increased inventory building by customers in advance of expected or announced price increases.

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The following table provides a summary of activity with respect to our sales allowances and accruals (in thousands):

Six Months Ended June 30, 2025 Discounts and

Nine Months Ended September 30, 2025 Discounts and

Fees Commercial & Government

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Credits/payments for prior period sales (21,144) (137,935) (8,081) (7,539) (174,699)

Balance at June 30, 2025 $ 29,207 $ 480,767 $ 12,559 $ 26,531 $ 549,064

Balance at September 30, 2025 $ 39,900 $ 528,846 $ 9,640 $ 28,807 $ 607,193

government rebates and chargebacks are the most significant component of our sales allowances.

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We brought a lawsuit against the U.S.

Centers for Medicare and Medicaid Services (“CMS”) alleging that a recent regulation issued by CMS on the definition of “line extension” for purposes of the Medicaid rebate program is too broad and has the unintended consequence of treating OPZELURA as a “line extension” of JAKAFI under this program.

Centers for Medicare and Medicaid Services (“CMS”) alleging that a regulation issued by CMS on the definition of “line extension” for purposes of the Medicaid rebate program is too broad and has the unintended consequence of treating OPZELURA as a “line extension” of JAKAFI under this program.

We believe that such a reading would violate CMS’s statutory authority and be arbitrary and capricious given that OPZELURA, among other differentiators, is indicated to treat entirely different medical conditions and entirely different patient populations than JAKAFI.

As of June 30, 2025, we have accrued approximately $165.2 million within accrued and other current liabilities on the condensed consolidated balance sheet, relating to the incremental rebates that would be owed were OPZELURA considered a line extension of JAKAFI.

The impact on OPZELURA gross to net deductions for the quarter ending June 30, 2025 is approximately 6.6%.

As of September 30, 2025, we have accrued approximately $188.9 million within accrued and other current liabilities on the condensed consolidated balance sheet, relating to the incremental rebates that would be owed were OPZELURA considered a line extension of JAKAFI.

The impact on OPZELURA gross to net deductions for the quarter ending September 30, 2025 is approximately 6.8%.

If OPZELURA is not treated as a line extension of JAKAFI, this would result in a reversal of our accrual and a lower future gross to net deduction for OPZELURA.

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Product royalty revenues on commercial sales of OLUMIANT by Lilly are based on net sales of licensed products in licensed territories as provided by Lilly.

Our milestone and contract revenues for the three and six months ended June 30, 2025, was derived from a $5.0 million development milestone upon approval of tafasitamab in treating follicular lymphoma.

Our milestone and contract revenues for the six months ended June 30, 2024, was derived from a $25.0 million upfront payment received during the first quarter of 2024 upon our transfer of functional intellectual property to CMSHL.

Our milestone and contract revenues for the three and nine months ended September 30, 2025 and September 30, 2024 were derived from a combination of upfront payments received from our third party collaborators for the transfer of functional intellectual property, as well as developmental milestones received from our third party collaborators.

Cost of Product Revenues

Three Months Ended

June 30, Six Months Ended

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2025 2024 2025 2024

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Cost of product revenues includes all product related costs, reserves for obsolescence, employee personnel costs, including stock compensation, for those employees dedicated to the production of our commercial products, royalties and profit sharing under our collaborative agreements and amortization of our licensed intellectual property rights for ICLUSIG and capitalized milestone payments .

The increase in cost of product revenues for the three and six months ended June 30, 2025 as compared to the same periods in 2024 was driven by growth in net product revenues, the NIKTIMVO profit share and increased manufacturing related costs, partially offset by the impact of the contract dispute settlement with Novartis.

The increase in cost of product revenues for the three and nine months ended September 30, 2025 as compared to the same periods in 2024 was driven by growth in net product revenues, the NIKTIMVO profit share and increased manufacturing related costs, partially offset by the impact of the contract dispute settlement with Novartis.

Contract Dispute Settlement

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Under the settlement agreement, we paid Novartis $280.0 million as the settlement of disputed royalties on net sales of JAKAFI in the United States through December 31, 2024, and agreed to reduce by 50% the royalty rate payable by us on future net sales of JAKAFI in the United States beginning January 1, 2025.

The reduced royalty paid for the quarter ending March 31, 2025, was approximately $14.9 million.

The difference of $242.2 million between the total accrued royalties and the total amount paid by us to Novartis as disclosed above was recorded in contract dispute settlement on our condensed consolidated statement of operations for the three and six months ended June 30, 2025.

The reduced royalty paid for the quarter ended March 31, 2025, was approximately $14.9 million.

Operating Expenses

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Three Months Ended

June 30, Six Months Ended

September 30, Nine Months Ended

September 30,

2025 2024 2025 2024

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We account for research and development costs by natural expense line and not costs by project.

The increase in salary and benefits related expense for the three and six months ended June 30, 2025 as compared to the corresponding period in 2024 was due primarily to increased headcount to sustain our development pipeline.

The increase in salary and benefits related expense for the three and nine months ended September 30, 2025 as compared to the corresponding period in 2024 was due primarily to increased headcount to sustain our development pipeline.

Stock compensation expense may fluctuate from period to period based on the number of awards granted, stock price volatility and expected award lives, as well as expected award forfeiture rates which are used to value equity-based compensation.

Additionally, as described in Note 6 to the Condensed Consolidated Financial Statements, as part of the Escient acquisition, during the three and six months ended June 30, 2024, we recognized compensation expense in research and development of $11.3 million associated with the accelerated vesting for certain Escient stock awards in connection with the acquisition on our condensed consolidated statements of operations.

Research and development expenses for the three and six months ended June 30, 2024 also include the $679.4 million of expense related to the acquired in-process research and development assets as part of the Escient acquisition, as described in Note 6 to the Condensed Consolidated Financial Statements.

The increase in clinical research and outside services expense for the three months ended June 30, 2025 as compared to the corresponding period in 2024, was primarily due to continued investment in our late stage development assets.

Research and development expenses include upfront and milestone expenses related to our collaborative agreements of $12.6 million and $28.1 million, respectively, for the three and six months ended June 30, 2025.

Research and development expenses include upfront and milestone expenses related to our collaborative agreements of $0.4 million and $1.4 million, respectively, for the three and six months ended June 30, 2024.

Research and development expenses for the three and six months ended June 30, 2025 and 2024 were net of $3.9 million, $6.5 million, $4.2 million and $21.3 million, respectively, of costs reimbursed by our collaborative partners.

Additionally, as described in Note 6 of Notes to the Condensed Consolidated Financial Statements, as part of the Escient acquisition, during the second quarter of 2024, we recognized compensation expense in research and development of $11.3 million associated with the accelerated vesting for certain Escient stock awards in connection with the acquisition on our condensed consolidated statements of operations.

Research and development expenses for the nine months ended September 30, 2024 also include the $679.4 million of expense related to the acquired in-process research and development assets as part of the Escient acquisition, as described in Note 6 of Notes to the Condensed Consolidated Financial Statements.

The decrease in clinical research and outside services expense for the three months ended September 30, 2025 as compared to the corresponding period in 2024, was primarily due to the $100.0 million milestone payment made to MacroGenics during the third quarter of 2024.

Research and development expenses include upfront and milestone expenses related to our collaborative agreements of $0.1 million and $28.2 million, respectively, for the three and nine months ended September 30, 2025.

Research and development expenses include upfront and milestone expenses related to our collaborative agreements of $100.0 million and $101.4 million, respectively, for the three and nine months ended September 30, 2024.

Research and development expenses for the three and nine months ended September 30, 2025 and 2024 were net of $4.3 million, $10.8 million, $3.8 million and $25.1 million, respectively, of costs reimbursed by our collaborative partners.

In addition to one-time expenses resulting from upfront fees in connection with the entry into any new or amended collaboration agreements and payment of milestones under those agreements, research and development expenses may fluctuate from period to period depending upon the stage of certain projects and the level of preclinical and clinical trial related activities.

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Three Months Ended

June 30, Six Months Ended

September 30, Nine Months Ended

September 30,

2025 2024 2025 2024

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Total selling, general and administrative expenses $ 329.1 $ 309.2 $ 985.8 $ 915.4

The increase in salary and benefits related expense for the three and six months ended June 30, 2025 as compared to the corresponding period in 2024 was due primarily to increased headcount.

The increase in salary and benefits related expense for the three and nine months ended September 30, 2025 as compared to the corresponding period in 2024 was due primarily to increased headcount.

Additionally, as described in Note 6 to the Condensed Consolidated Financial Statements, as part of the Escient acquisition, during the three months ended June 30, 2024, we recognized compensation expense in selling, general and administrative expenses of $20.2 million associated with the accelerated vesting for certain Escient stock awards in connection with the acquisition on our condensed consolidated statements of operations.

The increase in other contract services and outside costs for the three months ended June 30, 2025, as compared to the corresponding period in 2024, was primarily due to increased legal costs relating to the Novartis contract dispute settlement and other matters and timing of consumer marketing activities.

Loss on change in fair value of acquisition-related contingent consideration

Additionally, as described in Note 6 of Notes to the Condensed Consolidated Financial Statements, as part of the Escient acquisition, during the second quarter of 2024, we recognized compensation expense in selling, general and administrative expenses of $20.2 million associated with the accelerated vesting for certain Escient stock awards in connection with the acquisition on our condensed consolidated statements of operations.

The increase in other contract services and outside costs for the three months ended September 30, 2025, as compared to the corresponding period in 2024, was primarily due to international marketing activities to support product launches.

(Gain) loss on change in fair value of acquisition-related contingent consideration

Acquisition-related contingent consideration, which consists of our future royalty obligations to ARIAD/Takeda, was recorded on the acquisition date, June 1, 2016, at the estimated fair value of the obligation, in accordance with the acquisition method of accounting.

The change in fair value of the acquisition-related contingent consideration for the three and six months ended June 30, 2025 was a loss of $22.8 million and $34.3 million, respectively, which is recorded in loss on change in fair value of acquisition-related contingent consideration on the condensed consolidated statements of operations.

The change in fair value of the acquisition-related contingent consideration for the three and six months ended June 30, 2024 was a loss of $0.9 million and loss of $0.4 million, respectively, which is recorded in loss on change in fair value of acquisition-related contingent consideration on the condensed consolidated statements of operations.

The change in fair value of the contingent consideration during the three and six months ended June 30, 2025 and 2024 was primarily due to fluctuations in foreign currency exchange rates impacting future revenue projections of ICLUSIG and the passage of time.

Profit sharing from co-commercialization activities

Under the former collaboration and license agreement with MorphoSys, which was executed in March 2020 and continued through February 5, 2024 as described further in Note 6 of Notes to the Condensed Consolidated Financial Statements, we and MorphoSys were both responsible for the commercialization efforts of tafasitamab in the United States and shared equally the profits and losses from the co-commercialization efforts.

For the period from January 1, 2024 through February 5, 2024, our 50% share of the profits for tafasitamab was $1.0 million, as recorded in (Profit) and loss sharing under collaboration agreements on the condensed consolidated statement of operations.

Under the collaboration agreement with Syndax, as described further in Note 8 of the Notes to the Condensed Consolidated Financial Statements, we and Syndax are both responsible for the co-commercialization of axatilimab in the United States and share equally in the profits and losses from those efforts.

We are the principal in the U.S.

axatilimab co-commercialization efforts and record 100% of all product revenues and associated costs in accordance with our profit sharing from co-commercialization activities accounting policy outlined in our Annual Report on Form 10-K for the year ended December 31, 2024.

For the three and six months ended June 30, 2025, we recorded revenues of $36.2 million and $49.8 million, respectively, from sales of NIKTIMVO (axatilimab), and total associated costs of $15.2 million and $28.9 million respectively, from co-commercialization activities.

The change in fair value of the acquisition-related contingent consideration for the three and nine months ended September 30, 2025 was a gain of $12.2 million and loss of $22.1 million, respectively, which is recorded in (gain) loss on change in fair value of acquisition-related contingent consideration on the condensed consolidated statements of operations.

The change in fair value of the acquisition-related contingent consideration for the three and nine months ended September 30, 2024 was a loss of $23.4 million and $23.8 million, respectively, which is recorded in (gain) loss on change in fair value of acquisition-related contingent consideration on the condensed consolidated statements of operations.

The change in fair value of the contingent consideration during the three and nine months ended September 30, 2025 and 2024 was due primarily to updated projections of future net revenues of Iclusig, including the impacts from fluctuations in foreign currency exchange rates, and the passage of time.

Non-operating Income and Expenses

Interest income

Interest income for the three and six months ended June 30, 2025 was $25.1 million and $48.1 million, respectively.

Interest income for the three and six months ended June 30, 2024 was $41.5 million and $88.2 million, respectively.

The decrease in Interest income for the three and six months ended June 30, 2025 is primarily due to lower interest rates and cash and cash equivalent balances in 2025 as compared to the corresponding periods in 2024.

(Loss) gain on equity investments

Losses and gains on equity investments will fluctuate from period to period, based on sales of securities and the change in fair value of the securities we hold in our publicly held collaboration partners.

The following table provides a summary of those (losses) gains:

Interest income for the three and nine months ended September 30, 2025 was $26.8 million and $74.8 million, respectively.

Interest income for the three and nine months ended September 30, 2024 was $19.3 million and $107.5 million, respectively.

The increase in Interest income for the three months ended September 30, 2025 is primarily due to higher cash and cash equivalent balances in the third quarter of 2025 as compared to the corresponding period in 2024, and the decrease in Interest income for the nine months ended September 30, 2025 is primarily due to lower interest rates in 2025 and to a lesser extent, lower average cash balances as compared to the corresponding periods in 2024.

Gain (loss) on equity investments

Gains and losses on equity investments will fluctuate from period to period, based on sales of securities and the change in fair value of the securities we hold in our publicly held collaboration partners.

The following table provides a summary of those gains (losses):

Three Months Ended

June 30, Six Months Ended

September 30, Nine Months Ended

September 30,

2025 2024 2025 2024

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Other — (0.2) — (0.5)

Total (loss) gain on equity investments $ (4.2) $ 39.2 $ (5.5) $ 139.2

Total gain (loss) on equity investments $ 8.6 $ (13.0) $ 3.1 $ 126.2

Provision for income taxes

The provision for income taxes for the three and six months ended June 30, 2025 was $153.0 million and $229.0 million, respectively.

The provision for income taxes for the three and six months ended June 30, 2024 was $54.8 million and $121.4 million, respectively.

Our effective tax rate for the three and six months ended June 30, 2025 was higher than the U.S.

The provision for income taxes for the three and nine months ended September 30, 2025 was $58.1 million and $287.1 million, respectively.

The provision for income taxes for the three and nine months ended September 30, 2024 was $50.1 million and $171.5 million, respectively.

Our effective tax rate for the three months ended September 30, 2025 is lower than the U.S.

statutory rate primarily due to a net decrease in our valuation allowance against certain U.S.

federal deferred tax assets, resulting from the recently enacted U.S.

tax law changes, as more fully described in Note 14 of Notes to the Condensed Consolidated Financial Statements.

Our effective tax rate for the nine months ended September 30, 2025 was higher than the U.S.

statutory rate primarily due to an increase in our valuation allowance against certain U.S.

federal and state deferred tax assets.

This was partially offset by tax rate benefits associated with research and development and orphan drug tax credit generations, the foreign derived intangible income deduction and a decrease in a prior year valuation allowance against certain U.S.

federal deferred tax assets, resulting from the recently enacted U.S.

tax law changes.

Our effective tax rate for the three months ended September 30, 2024 was higher than the U.S.

statutory rate primarily due to foreign losses with no associated tax benefit (i.e., full valuation allowance) and an increase in our valuation allowance against certain U.S.

federal and state deferred tax assets.

This was partially offset by tax rate benefits associated with research and development and orphan drug tax credit generations and the foreign derived intangible income deduction.

Our effective tax rate for the three and six months ended June 30, 2024 was higher than the U.S.

statutory rate primarily due to a non-deductible charge of $710.9 million associated with the Escient acquisition.

Our effective tax rate for the nine months ended September 30, 2024 was higher than the U.S.

statutory rate primarily due to non-deductible charges of $710.9 million associated with the Escient acquisition.

Liquidity and Capital Resources

At June 30, 2025, we had available cash, cash equivalents and marketable securities of $2.4 billion.

At September 30, 2025, we had available cash, cash equivalents and marketable securities of $2.9 billion.

Our cash and marketable securities balances are primarily held in a variety of interest-bearing instruments, including money market accounts and U.S.

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Available cash is invested in accordance with our investment policy’s primary objectives of liquidity, safety of principal and diversity of investments.

Net cash provided by operating activities for the six months ended June 30, 2025 was $310.8 million and net cash used in operating activities for the six months ended June 30, 2024 was $356.8 million.

The increase in cash provided by operating activities was due primarily to the changes in net income as a result of the contract dispute settlement during the second quarter of 2025 and the Escient acquisition during the second quarter of 2024.

Net cash provided by operating activities for the nine months ended September 30, 2025 was $870.2 million and net cash used in operating activities for the nine months ended September 30, 2024 was $45.9 million.

Our investing activities, other than purchases, sales and maturities of marketable securities, have consisted predominantly of capital expenditures and sales of long term investments.

Net cash used in investing activities was $17.8 million for the six months ended June 30, 2025, which primarily represented by purchases of marketable securities of $97.3 million and capital expenditures of $22.2 million, offset in part by maturities of marketable securities of $101.8 million.

Net cash provided by investing activities was $140.7 million for the six months ended June 30, 2024, which primarily represented sales of equity investments of $227.3 million and maturities of marketable securities of $182.6 million, offset in part by purchases of marketable securities of $204.1 million, and capital expenditures of $63.7 million.

Net cash used in investing activities was $65.2 million for the nine months ended September 30, 2025, which primarily represented purchases of marketable securities of $212.9 million, capital expenditures of $37.0 million and payments for intangible assets of $25.0 million, offset in part by maturities of marketable securities of $209.6 million.

Net cash provided by investing activities was $179.0 million for the nine months ended September 30, 2024, which primarily represented sales of equity investments of $282.9 million and maturities of marketable securities of $207.9 million, offset in part by purchases of marketable securities of $229.0 million, capital expenditures of $68.9 million and payments for intangible assets of $13.9 million.

In the future, net cash used by investing activities may fluctuate significantly from period to period due to the timing of strategic equity investments, acquisitions, and capital expenditures and maturities/sales and purchases of marketable securities.

Net cash used in financing activities was $23.9 million for the six months ended June 30, 2025, primarily representing the $19.1 million paid for excise taxes relating to the June 2024 share repurchase, cash paid for tax withholdings related to restricted and performance share vesting and cash paid to ARIAD/Takeda for contingent consideration, partially offset by proceeds from issuance of common stock under our stock plans.

Net cash used in financing activities was $2.01 billion for the six months ended June 30, 2024 and was primarily driven by expenditures associated with the share repurchase of $2.00 billion.

Net cash used in financing activities was $36.7 million for the nine months ended September 30, 2025, primarily representing the $19.1 million paid for excise taxes relating to the June 2024 share repurchase, cash paid for tax withholdings related to restricted and performance share vesting and cash paid to ARIAD/Takeda for contingent consideration, partially offset by proceeds from issuance of common stock under our stock plans.

Net cash used in financing activities was $2.04 billion for the nine months ended September 30, 2024 and was primarily driven by expenditures associated with the share repurchase of $2.00 billion.

In August 2021, we entered into a $500.0 million, senior unsecured revolving credit facility, which was subsequently amended in May 2023 and June 2024 (as amended, the “Credit Agreement”).

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We may increase the maximum revolving commitments or add one or more incremental term loan facilities, subject to obtaining commitments from any participating lenders and certain other conditions, in an amount not to exceed $250.0 million plus a contingent additional amount that is dependent on our pro forma consolidated leverage ratio.

As of June 30, 2025, we had no outstanding borrowings and were in compliance with all covenants under this facility.

As of September 30, 2025, we had no outstanding borrowings and were in compliance with all covenants under this facility.

The Credit Agreement is described further in Note 17 of Notes to the Condensed Consolidated Financial Statements.

As of June 30, 2025, our U.S.

tax liabilities continue to reflect the adverse impacts of the mandatory capitalization and amortization of research and development costs as required under the Tax Cuts and Jobs Act of 2017, which eliminated the immediate expensing of such costs.

The enactment of the One Big Beautiful Bill Act on July 4, 2025 modified key provisions of the Tax Cuts and Jobs Act of 2017 that may have an impact on our U.S.

tax liabilities.

We are evaluating the potential impacts of the modified provisions.

The enactment of the One Big Beautiful Bill Act on July 4, 2025 modified key provisions of the Tax Cuts and Jobs Act of 2017.

The change related to the expensing of domestic research costs will materially reduce our U.S.

tax liabilities in 2025 and 2026.

We continue to evaluate the impacts of the modified provisions and have reflected an estimate in our financial statements for the period ending September 30, 2025.

We believe that our cash flow from operations, together with our cash, cash equivalents and marketable securities and funds available under our revolving credit facility, will be adequate to satisfy our capital needs for the foreseeable future.

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Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-Q

Item 2. Management’s Discussion and Analysis