RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan), Orphan Drug (South Korea), Paediatric investigation plan (European Union), Conditional marketing approval (China), Priority Review (China)

Structure/Sequence

Sequence Code 37465

Source: *****

Clinical Trials associated with Luspatercept-AAMT

NCT07338344

/ RecruitingPhase 2IIT

Evaluation of the Clinical Efficacy and Safety of Luspatercept Combined With Low-dose Thalidomide Versus Luspatercept Alone in the Treatment of Adult Patients With Transfusion-dependent β-thalassemia

β-thalassemia is one of the most common inherited hemoglobinopathies worldwide and a major public health issue that severely impacts birth quality, human health, and social progress. Currently, there are limited clinical drugs specifically designed to treat patients with β-thalassemia. This clinical trial aims to evaluate the efficacy and safety of luspatercept combined with low-dose thalidomide compared with luspatercept alone in patients with thalassemia. Key questions to be answered include:
* Does luspatercept combined with low-dose thalidomide reduce the transfusion burden in patients with β-thalassemia major?
* What medical problems may occur when patients receive luspatercept combined with low-dose thalidomide? In this clinical trial, participants were randomly assigned in a 1:1 ratio to either an intervention group (luspatercept combined with low-dose thalidomide) or a control group (luspatercept combined with placebo) using a central randomization system. The clinical efficacy and safety of the two groups were evaluated. The primary outcome measure was the clinical efficacy of luspatercept combined with low-dose thalidomide in reducing the transfusion burden in patients with β-thalassemia major.

Start Date01 Feb 2026

Sponsor / Collaborator

First Affiliated Hospital of Guangxi Medical University

NCT07096297

/ RecruitingPhase 2IIT

A Phase II Study of Luspatercept Plus Darbepoetin Alfa in Non-mutated SF3B1 Lower-risk Myelodysplastic Syndromes

This is a single arm open-label Phase II trial of luspatercept and darbepoetin alfa in non-mutated SF3B1 , lower-risk, RBC transfusion dependent MDS participants with an endogenous erythropoietin (EPO) level < 500 IU/L.

Start Date01 Feb 2026

Sponsor / Collaborator

Yale University

[+1]

NCT07331818

/ Not yet recruitingPhase 2IIT

Luspatercept in Patients Affected With Rare Inherited Anemias

This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias

Start Date15 Jan 2026

Sponsor / Collaborator-

100 Clinical Results associated with Luspatercept-AAMT

100 Translational Medicine associated with Luspatercept-AAMT

100 Patents (Medical) associated with Luspatercept-AAMT

249

Literatures (Medical) associated with Luspatercept-AAMT

02 Jan 2026·Anti-inflammatory & anti-allergy agents in medicinal chemistry

Clinical Applications of Ligand Traps Targeting Activin Type II Receptors

Article

Author: Tsuchida, Kunihiro

This review summarizes recent advances in ligand trap therapies targeting activin type II receptors [ActRIIA/ACVR2A and ActRIIB/ACVR2B], which serve as shared receptors for members of the TGF-β family, including activins, GDF11, and myostatin [MSTN]. These receptors mediate Smad2/3 signaling and play critical roles in hematopoiesis, vascular homeostasis, and muscle regulation. Two peptide-based ligand traps have recently received clinical approval: luspatercept [ActRIIB-Fc], an erythroid maturation agent, and sotatercept [ActRIIA-Fc], a novel therapeutic agent for pulmonary arterial hypertension [PAH]. Luspatercept primarily inhibits activin B and GDF11, thereby promoting late-stage erythropoiesis and demonstrating efficacy in anemia associated with conditions such as myelodysplastic syndromes [MDS] and β-thalassemia. Sotatercept binds activins and GDFs to rebalance Smad2/3 and Smad1/5/8 signaling, thereby improving vascular remodeling in PAH. Although both agents have failed to increase skeletal muscle mass in clinical trials consistently, they represent significant advances in the treatment of hematopoietic and vascular disorders. Future studies should focus on optimal dosing strategies, long-term safety, and potential synergistic effects when combined with other therapeutic modalities.

01 Jan 2026·HemaSphere

Erythropoiesis in health and disease: Distinguishing defective and ineffective erythropoiesis

Review

Author: Lizarralde‐Iragorri, Maria A. ; Hermine, Olivier ; Maciel, Thiago Trovati ; El Hoss, Sara

Abstract:

Erythropoiesis is a finely regulated process ensuring continuous red blood cell production to maintain oxygen delivery. Disruptions in this process give rise to defective erythropoiesis, characterized by impaired lineage commitment and progenitor development, and ineffective erythropoiesis (IE), marked by expansion of erythroid progenitors with intramedullary apoptosis of late precursors. These abnormalities underlie anemia in diverse hematological disorders, including β‐thalassemia, sickle cell disease (SCD), myelodysplastic syndromes (MDSs), and emerging entities such as vacuoles, E1 enzyme, X‐linked, autoinflammatory, somatic (VEXAS). This review synthesizes recent insights into the molecular regulators of erythropoiesis, emphasizing the roles of GATA1, its chaperone HSP70, caspase activity, and extrinsic signals such as growth differentiation factor 11 (GDF11) and inflammatory cytokines. We highlight how globin‐chain imbalance, inflammasome activation, oxidative stress, and clonal mutations converge on common pathways that disrupt erythroid maturation. Advances in therapy now directly target these mechanisms: Luspatercept, a TGF‐β ligand trap, has transformed care in β‐thalassemia and MDS by restoring late‐stage differentiation, while anti‐inflammatory strategies, metabolic modulators, and gene editing approaches are being actively explored. Together, these discoveries reframe IE as a modifiable process rather than an inevitable consequence of disease. By distinguishing defective from IE and mapping their mechanistic underpinnings, this review outlines how novel therapeutic strategies can improve anemia, reduce systemic complications, and ultimately enhance outcomes in patients with erythroid disorders.

01 Jan 2026·BRITISH JOURNAL OF HAEMATOLOGY

Achieving clinically meaningful changes in haemoglobin levels in patients with non‐transfusion‐dependent β‐thalassaemia treated with luspatercept: A post hoc analysis of the phase 2 BEYOND trial

Article

In this post hoc analysis of the phase 2 BEYOND trial, the majority of patients with non-transfusion-dependent β-thalassaemia achieved clinically meaningful haemoglobin levels ≥10.0 g/dL and increases from baseline ≥1.0 g/dL, thresholds associated with reduced risk of morbidity and mortality and recommended as an indication and target for treatment by international management guidelines, respectively.

192

News (Medical) associated with Luspatercept-AAMT

03 Feb 2026

·www.businesswire.com

Merck & Co., Inc., Rahway, N.J., USA Announces Fourth-Quarter and Full-Year 2025 Financial Results; Highlights Progress Advancing Broad, Diverse Pipeline

Fourth-Quarter Worldwide Sales Were $16.4 Billion (5% Growth; 4% Growth ex-FX) Fourth-Quarter GAAP EPS Was $1.19; Non-GAAP EPS Was $2.04; GAAP and Non-GAAP EPS Include a Charge of $0.05 per Share for the Acquisition of MK-8690 Sole Global Rights Full-Year Worldwide Sales Were $65.0 Billion (1% Growth; 2% Growth ex-FX) KEYTRUDA/KEYTRUDA QLEX Sales Were $31.7 Billion (7% Growth Both Nominally and ex-FX); Includes KEYTRUDA QLEX Sales of $40 Million WINREVAIR Sales Were $1.4 Billion CAPVAXIVE Sales Were $759 Million GARDASIL/GARDASIL 9 Sales Were $5.2 Billion (39% Decline Both Nominally and ex-FX) Animal Health Sales Were $6.4 Billion (8% Growth; 9% Growth ex-FX) KEYTRUDA/KEYTRUDA QLEX Sales Were $31.7 Billion (7% Growth Both Nominally and ex-FX); Includes KEYTRUDA QLEX Sales of $40 Million WINREVAIR Sales Were $1.4 Billion CAPVAXIVE Sales Were $759 Million GARDASIL/GARDASIL 9 Sales Were $5.2 Billion (39% Decline Both Nominally and ex-FX) Animal Health Sales Were $6.4 Billion (8% Growth; 9% Growth ex-FX) Full-Year 2025 GAAP EPS Was $7.28; Non-GAAP EPS Was $8.98; GAAP and Non-GAAP EPS Include Charges of $0.20 per Share Related to Certain Business Development Transactions Announced Positive Late-Stage Trial Results From 18 Phase 3 Trials in 2025 Augmented Pipeline and Portfolio Through Acquisitions of Verona Pharma and Cidara Therapeutics and License Agreement With Hengrui Pharma In the Fourth Quarter Received FDA Commissioner’s National Priority Vouchers for Enlicitide and Sacituzumab Tirumotecan (Sac-TMT), Providing an Opportunity To Expedite Potential FDA Review Timelines for These Phase 3 Candidates Presented Positive Results From Phase 3 CORALreef Lipids and HeFH Trials Demonstrating Enlicitide Significantly Reduced LDL-C in Adults Reached Agreement With U.S. Government To Expand Access to Medicines and Lower Costs for Americans Received FDA Commissioner’s National Priority Vouchers for Enlicitide and Sacituzumab Tirumotecan (Sac-TMT), Providing an Opportunity To Expedite Potential FDA Review Timelines for These Phase 3 Candidates Presented Positive Results From Phase 3 CORALreef Lipids and HeFH Trials Demonstrating Enlicitide Significantly Reduced LDL-C in Adults Reached Agreement With U.S. Government To Expand Access to Medicines and Lower Costs for Americans Full-Year 2026 Financial Outlook Anticipates Worldwide Sales To Be Between $65.5 Billion and $67.0 Billion Expects Non-GAAP EPS To Be Between $5.00 and $5.15; Outlook Reflects a One-Time Charge of Approximately $3.65 per Share for the Acquisition of Cidara Anticipates Worldwide Sales To Be Between $65.5 Billion and $67.0 Billion Expects Non-GAAP EPS To Be Between $5.00 and $5.15; Outlook Reflects a One-Time Charge of Approximately $3.65 per Share for the Acquisition of Cidara RAHWAY, N.J.--(BUSINESS WIRE)--Merck & Co., Inc., Rahway, N.J., USA (NYSE: MRK), known as MSD outside the United States and Canada, today announced financial results for the fourth quarter and full year of 2025. "In 2025, we continued to advance leading-edge science to deliver transformative medicines and vaccines that are improving health outcomes for patients around the world,” said Robert M. Davis, chairman and chief executive officer. "Our business benefited from demand for our innovative portfolio, including for KEYTRUDA, increasing contributions from new launches in cardiometabolic and respiratory as well as vaccines, and strong performance of Animal Health. The transformation of our portfolio, bolstered by the acquisitions of Verona Pharma and Cidara Therapeutics, is well underway, and momentum is building as we continue to execute on our strategy. Our progress positions us to continue delivering on our purpose for patients and creating durable value for shareholders.” Financial Summary $ in millions, except EPS amounts Fourth Quarter Year Ended 2025 2024 Change Change Ex-Exchange Dec. 31, 2025 Dec. 31, 2024 Change Change Ex-Exchange Sales $16,400 $15,624 5% 4% $65,011 $64,168 1% 2% GAAP net income1 2,963 3,743 -21% -20% 18,254 17,117 7% 9% Non-GAAP net income that excludes certain items1,2* 5,088 4,372 16% 17% 22,513 19,444 16% 18% GAAP EPS 1.19 1.48 -20% -18% 7.28 6.74 8% 10% Non-GAAP EPS that excludes certain items2* 2.04 1.72 19% 19% 8.98 7.65 17% 19% *Refer to table on page 9. Generally Accepted Accounting Principles (GAAP) earnings per share (EPS) assuming dilution was $1.19 for the fourth quarter and $7.28 for the full year of 2025. Non-GAAP EPS was $2.04 for the fourth quarter and $8.98 for the full year of 2025. GAAP and non-GAAP EPS in the fourth quarter of 2025 include a charge of $0.05 per share related to an agreement with Dr. Falk Pharma GmbH (Falk) pursuant to which the Company secured the sole global rights to MK-8690. GAAP and non-GAAP EPS in the fourth quarter of 2024 include a charge of $0.23 per share related to the execution of licensing agreements with LaNova Medicines Ltd. (acquired by Sino Pharmaceutical Limited) and Hansoh Pharma. GAAP and non-GAAP EPS for the full years of 2025 and 2024 include charges of $0.20 and $1.28 per share, respectively, related to certain licensing agreements and asset acquisitions. Non-GAAP EPS excludes acquisition- and divestiture-related costs, costs related to restructuring programs, and income and losses from investments in equity securities. Non-GAAP EPS in 2025 also excludes a net tax benefit, which reflects a net benefit related to favorable audit reserve adjustments. Non-GAAP EPS in the fourth quarter and full year of 2024 also exclude a benefit due to a reduction in reserves for unrecognized income tax benefits resulting from the expiration of the statute of limitations for assessments related to certain federal tax return years. Fourth-Quarter Sales Performance The following table reflects sales of the Company’s top products and significant performance drivers. Fourth Quarter $ in millions 2025 2024 Change Change Ex-Exchange Commentary Total Sales $16,400 $15,624 5% 4% Pharmaceutical 14,843 14,042 6% 4% Increase primarily driven by growth in oncology as well as cardiometabolic and respiratory, partially offset by a decline in vaccines. KEYTRUDA/ KEYTRUDA QLEX 8,372 7,836 7% 5% Growth driven by strong global uptake in earlier-stage indications, including triple-negative breast cancer (TNBC), non-small cell lung cancer (NSCLC), renal cell carcinoma, cervical and head and neck cancers, as well as continued global demand in metastatic indications, including urothelial, gastric and endometrial cancers. Sales growth was partially offset by timing of purchases in the U.S. Sales of KEYTRUDA QLEX were $35 million. GARDASIL/ GARDASIL 9 1,031 1,550 -34% -35% Decline primarily due to lower demand in China, as well as lower sales in Japan following the national catch-up immunization program, partially offset by higher sales in the U.S. and timing in certain international markets. PROQUAD, M-M-R II and VARIVAX 619 594 4% 3% Increase primarily reflects higher sales of PROQUAD, which largely resulted from both the replenishment of doses borrowed from the U.S. Centers for Disease Control and Prevention Pediatric Vaccine Stockpile and from higher demand in Europe, partially offset by lower demand for M-M-R II in certain international markets and lower demand for VARIVAX in the U.S. JANUVIA/JANUMET 501 487 3% 3% Growth driven by higher net pricing in the U.S., partially offset by lower demand in China as well as in most other international markets due to generic competition. BRIDION 499 449 11% 11% Growth primarily due to higher demand and net pricing in the U.S., partially offset by lower demand in several international markets due to ongoing generic competition. WINREVAIR 467 200 133% 133% Growth primarily reflects continued uptake in the U.S. and early launch uptake in certain international markets, partially offset by lower net pricing in the U.S. largely due to Medicare Part D redesign. Lynparza* 389 365 7% 4% Growth primarily due to higher demand in several international markets. CAPVAXIVE 279 50 N/M N/M Growth largely due to continued uptake in the U.S. PREVYMIS 275 215 28% 26% Increase primarily due to higher demand in the U.S. as well as in most international markets, reflecting in part the launch of new indications. Lenvima* 272 255 7% 6% Increase due to higher sales in the U.S., primarily reflecting higher demand, partially offset by lower pricing. WELIREG 220 160 37% 37% Growth primarily due to higher demand in the U.S. and continued launch uptake in several international markets, partially offset by lower net pricing in the U.S. OHTUVAYRE 178 - - - Represents sales following the Company's Oct. 7, 2025 acquisition of Verona Pharma plc (Verona Pharma). Animal Health 1,505 1,397 8% 6% Growth primarily due to higher demand of livestock products. Livestock 987 889 11% 9% Growth primarily driven by higher demand across all species, as well as improved supply and new product launches. Companion Animal 518 508 2% 0% Growth from new product launches was partially offset by lower demand for other products in portfolio, reflecting a reduction in veterinary visits. Sales of BRAVECTO line of products were $222 million and $209 million in current and prior-year quarters, respectively, which represents an increase of 6%, or 5% excluding impact of foreign exchange. Other Revenues** 52 185 -71% -15% Decline primarily due to unfavorable impact of revenue-hedging activities and lower revenue from third-party manufacturing arrangements. *Alliance revenue for this product represents the Company’s share of profits, which are product sales net of cost of sales and commercialization costs. **Other revenues are comprised primarily of revenues from third-party manufacturing arrangements and miscellaneous corporate revenues, including revenue-hedging activities. N/M - Not meaningful. Full-Year Sales Performance The following table reflects sales of the Company’s top products and significant performance drivers. Year Ended $ in millions Dec. 31, 2025 Dec. 31, 2024 Change Change Ex-Exchange Total Sales $65,011 $64,168 1% 2% Pharmaceutical 58,142 57,400 1% 1% KEYTRUDA/KEYTRUDA QLEX 31,680 29,482 7% 7% GARDASIL/GARDASIL 9 5,233 8,583 -39% -39% JANUVIA/JANUMET 2,544 2,268 12% 13% PROQUAD, M-M-R II and VARIVAX 2,451 2,485 -1% -2% BRIDION 1,841 1,764 4% 4% Lynparza* 1,450 1,311 11% 10% WINREVAIR 1,443 419 N/M N/M Lenvima* 1,053 1,010 4% 4% PREVYMIS 978 785 25% 23% VAXNEUVANCE 825 808 2% 1% CAPVAXIVE 759 97 N/M N/M WELIREG 716 509 41% 41% ROTATEQ 673 711 -5% -5% Reblozyl* 525 371 41% 41% LAGEVRIO 380 964 -61% -61% Simponi** - 543 -100% -100% Animal Health 6,354 5,877 8% 9% Livestock 3,896 3,462 13% 14% Companion Animal 2,458 2,415 2% 2% Other Revenues*** 515 891 -42% -6% *Alliance revenue for Lynparza and Lenvima represent the Company’s share of profits, which are product sales net of cost of sales and commercialization costs. Alliance revenue for Reblozyl represents royalties. **Marketing rights in former territories of the Company reverted to Johnson & Johnson on Oct. 1, 2024. ***Other revenues are comprised primarily of revenues from third-party manufacturing arrangements and miscellaneous corporate revenues, including revenue-hedging activities. N/M - Not meaningful. In addition, Koselugo alliance revenue was $436 million for the full year of 2025 compared with $170 million for the full year of 2024. The increase was due to an amendment to the collaboration agreement with AstraZeneca in 2025, which discontinued the provisions whereby the Company shared revenue and costs with AstraZeneca, and revised the payment structure, resulting in the Company’s recognition of a $150 million upfront payment and $175 million of regulatory milestones. Full-year 2025 Pharmaceutical sales were $58.1 billion, representing growth of 1% both nominally and excluding the impact of foreign exchange. Sales growth was primarily driven by higher sales in oncology, particularly KEYTRUDA and WELIREG, as well as increased alliance revenue from Koselugo (resulting from the amendment to the collaboration agreement noted above), Reblozyl and Lynparza. Also contributing to sales growth were higher sales in the cardiometabolic and respiratory franchise largely attributable to the ongoing launch of WINREVAIR, as well as the inclusion of OHTUVAYRE sales resulting from the acquisition of Verona Pharma, which closed on Oct. 7, 2025. Growth in the diabetes franchise, largely attributable to higher net pricing of JANUVIA in the U.S., also contributed to sales growth. Sales growth in 2025 was partially offset by lower sales in the vaccines franchise reflecting lower sales of GARDASIL/GARDASIL 9, which were offset in part by the ongoing launch of CAPVAXIVE and the U.S. launch of ENFLONSIA. Lower sales in the immunology franchise (due to the return of the marketing rights for Simponi and Remicade in former Company territories to Johnson & Johnson on Oct. 1, 2024) and lower sales in the virology franchise (largely attributable to LAGEVRIO) also offset Pharmaceutical sales growth in 2025. Full-year 2025 Animal Health sales were $6.4 billion, representing growth of 8%, or 9% excluding the impact of foreign exchange. Sales growth was primarily driven by the performance of Livestock products across all species and new product launches in Companion Animal. Sales of the BRAVECTO line of products were $1.1 billion in 2025, representing growth of 1% both nominally and excluding the impact of foreign exchange. Fourth-Quarter and Full-Year Expense and Related Information The table below presents selected expense information. $ in millions GAAP Acquisition- and Divestiture- Related Costs3 Restructuring Costs (Income) Loss From Investments in Equity Securities Non- GAAP2 Fourth Quarter 2025 Cost of sales $5,551 $1,054 $1,173 $- $3,324 Selling, general and administrative 2,898 48 2 - 2,848 Research and development 3,886 5 (111) - 3,992 Restructuring costs 213 - 213 - - Other (income) expense, net 432 - - 206 226 Fourth Quarter 2024 Cost of sales $3,828 $701 $121 $- $3,006 Selling, general and administrative 2,864 29 16 - 2,819 Research and development 4,585 12 (1) - 4,574 Restructuring costs 51 - 51 - - Other (income) expense, net 126 (31) - 152 5 $ in millions GAAP Acquisition- and Divestiture- Related Costs3 Restructuring Costs (Income) Loss From Investments in Equity Securities Non- GAAP2 Year Ended Dec. 31, 2025 Cost of sales $16,382 $2,871 $1,484 $- $12,027 Selling, general and administrative 10,733 120 3 - 10,610 Research and development 15,789 19 175 - 15,595 Restructuring costs 889 - 889 - - Other (income) expense, net 151 (3) - (306) 460 Year Ended Dec. 31, 2024 Cost of sales $15,193 $2,409 $495 $- $12,289 Selling, general and administrative 10,816 117 83 - 10,616 Research and development 17,938 72 1 - 17,865 Restructuring costs 309 - 309 - - Other (income) expense, net (24) (79) - 45 10 GAAP Expense, EPS and Related Information Gross margin was 66.2% for the fourth quarter of 2025 compared with 75.5% for the fourth quarter of 2024. Gross margin was 74.8% for the full year of 2025 compared with 76.3% for the full year of 2024. The gross margin decline in both periods was primarily due to the unfavorable impacts of higher restructuring costs (primarily related to the accelerated depreciation of manufacturing lines at two sites under the 2025 Restructuring Program), inventory write-offs and amortization of intangible assets, as well as the recognition of inventory fair value step-up related to the Verona Pharma acquisition, partially offset by the favorable impact of product mix. Selling, general and administrative (SG&A) expenses were $2.9 billion in the fourth quarter of 2025, an increase of 1% compared with the fourth quarter of 2024. The increase was primarily due to higher administrative costs, partially offset by lower promotional costs. Full-year 2025 SG&A expenses were $10.7 billion, a decrease of 1% compared with the full year of 2024. The decrease was primarily due to lower restructuring and promotional costs, partially offset by increased administrative costs. Research and development (R&D) expenses were $3.9 billion in the fourth quarter of 2025, a decrease of 15% compared with the fourth quarter of 2024. The decrease was primarily due to lower charges for business development activity and a reduction to estimated contractual termination costs associated with restructuring actions, partially offset by higher clinical development costs. R&D expenses were $15.8 billion for the full year of 2025, a decrease of 12% compared with the full year of 2024. The decrease was primarily due to lower charges for business development activity, partially offset by higher clinical development spending and higher restructuring costs. Other (income) expense, net, was $432 million of expense in the fourth quarter of 2025 compared with $126 million of expense in the fourth quarter of 2024 primarily due to higher net interest expense, higher foreign exchange losses and increased net losses from investments in equity securities. Other (income) expense, net, was $151 million of expense in the full year of 2025 compared with $24 million of income in the full year of 2024. The unfavorable year-over-year change primarily reflects $170 million of income in 2024 related to the expansion of an existing development and commercialization agreement with Daiichi Sankyo, as well as higher net interest expense and higher foreign exchange losses in 2025, partially offset by higher net income from investments in equity securities in 2025. The effective tax rate was 13.4% for the fourth quarter of 2025 and 13.3% for the full year of 2025. GAAP EPS was $1.19 for the fourth quarter of 2025 compared with $1.48 for the fourth quarter of 2024. The decrease was primarily driven by higher restructuring costs and amortization of intangible assets, partially offset by favorability from lower charges for business development transactions, as well as operational strength in the business driven in part by the benefits of the previously announced multiyear optimization initiative. GAAP EPS was $7.28 for the full year of 2025 compared with $6.74 for the full year of 2024. The increase was primarily driven by favorability from lower charges for business development transactions and operational strength in the business, partially offset by higher restructuring costs and amortization of intangible assets. Non-GAAP Expense, EPS and Related Information Non-GAAP gross margin was 79.7% for the fourth quarter of 2025 compared with 80.8% for the fourth quarter of 2024. The decrease was primarily due to higher inventory write-offs, partially offset by the favorable impact of product mix. Non-GAAP gross margin was 81.5% for the full year of 2025 compared with 80.8% for the full year of 2024. The increase was primarily due to the favorable impact of product mix, partially offset by higher inventory write-offs. Non-GAAP SG&A expenses were $2.8 billion in the fourth quarter of 2025, an increase of 1% compared with the fourth quarter of 2024. The increase was primarily due to higher administrative costs, partially offset by lower promotional costs. Non-GAAP SG&A expenses were $10.6 billion for the full year of 2025, flat compared with the full year of 2024 as lower promotional costs were largely offset by higher administrative costs. Non-GAAP R&D expenses were $4.0 billion in the fourth quarter of 2025, a decrease of 13% compared with the fourth quarter of 2024. Non-GAAP R&D expenses were $15.6 billion for the full year of 2025, a decrease of 13% compared with the full year of 2024. The decrease in both periods was primarily due to lower charges for business development activity, partially offset by higher clinical development costs. Non-GAAP other (income) expense, net, was $226 million of expense in the fourth quarter of 2025 compared with $5 million of expense in the fourth quarter of 2024 primarily due to higher net interest expense and higher foreign exchange losses. Non-GAAP other (income) expense, net, was $460 million of expense in the full year of 2025 compared with $10 million of expense in the full year of 2024. The unfavorable year-over-year change primarily reflects $170 million of income in 2024 related to the expansion of an existing development and commercialization agreement with Daiichi Sankyo, as well as higher net interest expense and higher foreign exchange losses in 2025. The non-GAAP effective tax rate was 15.4% for the fourth quarter of 2025 and 14.4% for the full year of 2025. Non-GAAP EPS was $2.04 for the fourth quarter of 2025 compared with $1.72 for the fourth quarter of 2024. Non-GAAP EPS was $8.98 for the full year of 2025 compared with $7.65 for the full year of 2024. The increase in both periods was primarily driven by favorability from lower charges for business development transactions, as well as operational strength in the business driven in part by the benefits of the previously announced multiyear optimization initiative. A reconciliation of GAAP to non-GAAP net income and EPS is provided in the table that follows. Fourth Quarter Year Ended $ in millions, except EPS amounts 2025 2024 Dec. 31, 2025 Dec. 31, 2024 EPS GAAP EPS $1.19 $1.48 $7.28 $6.74 Difference 0.85 0.24 1.70 0.91 Non-GAAP EPS that excludes items listed below2 $2.04 $1.72 $8.98 $7.65 Net Income GAAP net income1 $2,963 $3,743 $18,254 $17,117 Difference 2,125 629 4,259 2,327 Non-GAAP net income that excludes items listed below1,2 $5,088 $4,372 $22,513 $19,444 Excluded Items: Acquisition- and divestiture-related costs3 $1,107 $711 $3,007 $2,519 Restructuring costs 1,277 187 2,551 888 Loss (income) from investments in equity securities 206 152 (306) 45 Decrease to net income before taxes 2,590 1,050 5,252 3,452 Estimated income tax (benefit) expense4 (465) (421) (993) (1,125) Decrease to net income $2,125 $629 $4,259 $2,327 Pipeline and Portfolio Highlights In 2025, the Company announced positive late-stage trial results from 18 Phase 3 trials and began enrolling patients in 21 new Phase 3 studies evaluating multiple indications and therapeutic areas, with approximately 80 Phase 3 studies currently underway. Throughout the fourth quarter, the Company made important progress to advance its broad, diverse pipeline, meeting significant regulatory and clinical milestones. Oncology: U.S. Food and Drug Administration (FDA) approved KEYTRUDA and KEYTRUDA QLEX, each in combination with Padcev, for the perioperative treatment of adult patients with muscle-invasive bladder cancer (MIBC) who are ineligible for cisplatin-based chemotherapy based on Phase 3 KEYNOTE-905 trial. Approvals represent the first PD-1 inhibitor plus antibody-drug conjugate (ADC) regimens for this patient population. FDA awarded a priority review voucher under the Commissioner’s National Priority Voucher (CNPV) pilot program for sac-TMT, an investigational anti-TROP2 ADC being developed in collaboration with Kelun-Biotech. European Commission (EC) approved the subcutaneous route of administration and new pharmaceutical formulation of KEYTRUDA for use across all KEYTRUDA indications for adult patients in Europe. FDA accepted two supplemental Biologics License Applications (sBLAs) for KEYTRUDA and KEYTRUDA QLEX, each with Trodelvy, for the first-line treatment of certain patients with PD-L1+ inoperable (unresectable) locally advanced or metastatic TNBC based on Phase 3 KEYNOTE-D19/ASCENT-04 trial. FDA set Prescription Drug User Fee Act (PDUFA) dates in the second half of 2026 for these applications. Announced positive topline results from Phase 3 KEYNOTE-B15 trial in patients with MIBC who are eligible for cisplatin-based chemotherapy showing KEYTRUDA plus Padcev significantly improved event-free survival (EFS), overall survival (OS) and pathologic complete response (pCR) rates versus neoadjuvant chemotherapy and surgery when given before and after surgery. In collaboration with Moderna, Inc. (Moderna), announced median five-year follow-up data from Phase 2b KEYNOTE-942/mRNA-4157-P201 study for intismeran autogene, an investigational mRNA-based individualized neoantigen therapy, in combination with KEYTRUDA in patients with high-risk melanoma (stage III/IV) following complete resection. U.S. Food and Drug Administration (FDA) approved KEYTRUDA and KEYTRUDA QLEX, each in combination with Padcev, for the perioperative treatment of adult patients with muscle-invasive bladder cancer (MIBC) who are ineligible for cisplatin-based chemotherapy based on Phase 3 KEYNOTE-905 trial. Approvals represent the first PD-1 inhibitor plus antibody-drug conjugate (ADC) regimens for this patient population. FDA awarded a priority review voucher under the Commissioner’s National Priority Voucher (CNPV) pilot program for sac-TMT, an investigational anti-TROP2 ADC being developed in collaboration with Kelun-Biotech. European Commission (EC) approved the subcutaneous route of administration and new pharmaceutical formulation of KEYTRUDA for use across all KEYTRUDA indications for adult patients in Europe. FDA accepted two supplemental Biologics License Applications (sBLAs) for KEYTRUDA and KEYTRUDA QLEX, each with Trodelvy, for the first-line treatment of certain patients with PD-L1+ inoperable (unresectable) locally advanced or metastatic TNBC based on Phase 3 KEYNOTE-D19/ASCENT-04 trial. FDA set Prescription Drug User Fee Act (PDUFA) dates in the second half of 2026 for these applications. Announced positive topline results from Phase 3 KEYNOTE-B15 trial in patients with MIBC who are eligible for cisplatin-based chemotherapy showing KEYTRUDA plus Padcev significantly improved event-free survival (EFS), overall survival (OS) and pathologic complete response (pCR) rates versus neoadjuvant chemotherapy and surgery when given before and after surgery. In collaboration with Moderna, Inc. (Moderna), announced median five-year follow-up data from Phase 2b KEYNOTE-942/mRNA-4157-P201 study for intismeran autogene, an investigational mRNA-based individualized neoantigen therapy, in combination with KEYTRUDA in patients with high-risk melanoma (stage III/IV) following complete resection. Infectious Diseases: Announced positive topline results from the Phase 3 trial of the investigational, once-daily, oral, two-drug, single-tablet regimen of doravirine/islatravir (DOR/ISL) for the treatment of adults with HIV-1 infection who had not previously received antiretroviral treatment (treatment-naïve). Announced positive topline results from the Phase 3 trial of the investigational, once-daily, oral, two-drug, single-tablet regimen of doravirine/islatravir (DOR/ISL) for the treatment of adults with HIV-1 infection who had not previously received antiretroviral treatment (treatment-naïve). Cardiometabolic and Respiratory: Presented new data at the American Heart Association Scientific Sessions 2025, including results from the Phase 3 CORALreef Lipids and heterozygous familial hypercholesterolemia (HeFH) trials, demonstrating that enlicitide decanoate, an investigational, oral proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor being evaluated for the treatment of adults with hypercholesterolemia, significantly reduced low-density lipoprotein cholesterol (LDL-C) with a safety profile comparable to placebo. FDA awarded a priority review voucher under the CNPV pilot program for enlicitide decanoate. In January 2026, EC approved an expanded indication for WINREVAIR, in combination with other pulmonary arterial hypertension (PAH) therapies, for the treatment of PAH (Group 1 pulmonary hypertension) in adult patients with World Health Organization (WHO) Functional Class II, III and IV based on Phase 3 ZENITH trial. In February 2026, FDA accepted a new sBLA for WINREVAIR seeking approval to update the U.S. product label based on Phase 3 HYPERION trial. FDA set PDUFA date of September 21, 2026. Announced that Phase 2, proof-of-concept CADENCE study evaluating WINREVAIR in adults for the treatment of combined post- and precapillary pulmonary hypertension (CpcPH) due to heart failure with preserved ejection fraction (HFpEF) met its primary endpoint. Presented new data at the American Heart Association Scientific Sessions 2025, including results from the Phase 3 CORALreef Lipids and heterozygous familial hypercholesterolemia (HeFH) trials, demonstrating that enlicitide decanoate, an investigational, oral proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor being evaluated for the treatment of adults with hypercholesterolemia, significantly reduced low-density lipoprotein cholesterol (LDL-C) with a safety profile comparable to placebo. FDA awarded a priority review voucher under the CNPV pilot program for enlicitide decanoate. In January 2026, EC approved an expanded indication for WINREVAIR, in combination with other pulmonary arterial hypertension (PAH) therapies, for the treatment of PAH (Group 1 pulmonary hypertension) in adult patients with World Health Organization (WHO) Functional Class II, III and IV based on Phase 3 ZENITH trial. In February 2026, FDA accepted a new sBLA for WINREVAIR seeking approval to update the U.S. product label based on Phase 3 HYPERION trial. FDA set PDUFA date of September 21, 2026. Announced that Phase 2, proof-of-concept CADENCE study evaluating WINREVAIR in adults for the treatment of combined post- and precapillary pulmonary hypertension (CpcPH) due to heart failure with preserved ejection fraction (HFpEF) met its primary endpoint. Business Development: In 2026, completed acquisition of Cidara Therapeutics, Inc. (Cidara) for a total transaction value of approximately $9.2 billion. Added MK-1406 (formerly CD388), an investigational long-acting, strain-agnostic antiviral agent designed to prevent influenza infection in individuals at higher risk of complications, to the Company’s portfolio. MK-1406 is currently being evaluated in the Phase 3 ANCHOR study. Entered into strategic financing agreement with Blackstone Life Sciences to partially fund the development of sac-TMT in 2026. Entered into an agreement with Falk for certain development and commercialization rights to MK-8690, an investigational anti-CD30 ligand monoclonal antibody. In 2026, completed acquisition of Cidara Therapeutics, Inc. (Cidara) for a total transaction value of approximately $9.2 billion. Added MK-1406 (formerly CD388), an investigational long-acting, strain-agnostic antiviral agent designed to prevent influenza infection in individuals at higher risk of complications, to the Company’s portfolio. MK-1406 is currently being evaluated in the Phase 3 ANCHOR study. Entered into strategic financing agreement with Blackstone Life Sciences to partially fund the development of sac-TMT in 2026. Entered into an agreement with Falk for certain development and commercialization rights to MK-8690, an investigational anti-CD30 ligand monoclonal antibody. Notable recent news releases on the Company’s pipeline and portfolio are provided in the table that follows. Visit the News Releases section of the Company’s website to read the releases.* Oncology FDA Approved KEYTRUDA and KEYTRUDA QLEX, Each With Padcev, as Perioperative Treatment for Adults With Cisplatin-Ineligible MIBC; Based on Results From Phase 3 KEYNOTE-905 Trial EC Approved Subcutaneous Administration of KEYTRUDA for All Adult Indications Approved in EU; Based on Results From Phase 3 3475A-D77 Trial KEYTRUDA Plus Padcev Significantly Improved EFS, OS and pCR Rates for Cisplatin-Eligible Patients With MIBC When Given Before and After Surgery; Based on Results From Phase 3 KEYNOTE-B15 Trial The Company and Moderna Announced 5-Year Data for Intismeran Autogene in Combination With KEYTRUDA Demonstrated Sustained Improvement in the Primary Endpoint of Recurrence-Free Survival in Patients With High-Risk Stage III/IV Melanoma Following Complete Resection; Based on Follow-up Analysis From Phase 2b KEYNOTE-942/mRNA-4157-P201 Trial The Company Initiated Phase 3 KANDLELIT-007 Trial Evaluating Calderasib (MK-1084), an Investigational Oral KRAS G12C Inhibitor, in Combination With KEYTRUDA QLEX in Certain Patients With Advanced NSCLC The Company Presented Data at the American Society of Hematology Annual Meeting 2025 That Showcased Continued Advancements in Hematology Pipeline and Novel Therapeutic Approaches Vaccines and Infectious Diseases The Company Announced Positive Topline Results From Pivotal Phase 3 Trial Evaluating Investigational, Once-Daily, Oral, Two-Drug, Single-Tablet Regimen of DOR/ISL in Treatment-Naïve Adults With HIV-1 Infection Cardiometabolic and Respiratory Enlicitide Decanoate Significantly Reduced LDL-C in Phase 3 CORALreef Lipids Trial Enlicitide Decanoate Significantly Reduced LDL-C in Adults With HeFH in Phase 3 CORALreef HeFH Trial WINREVAIR Met Primary Endpoint in Phase 2, Proof-Of-Concept CADENCE Study in Adults With CpcPH Due to HFpEF Neuroscience The Company Showcased Data for Alzheimer’s Disease Candidates MK-2214 and MK-1167 at Clinical Trials on Alzheimer’s Disease 2025 Animal Health FDA Conditionally Approved EXZOLT CATTLE-CA1 for Prevention and Treatment of New World Screwworm (Cochliomyia Hominivorax) Larvae (Myiasis) *References to the Company’s name in the above news release titles have been modified for the purpose of this announcement. U.S. Government Agreement The Company reached an agreement with the U.S. government that is intended to lower medicine costs for Americans. This agreement enables the Company to continue its long-standing commitment to advancing breakthrough scientific discoveries for patients and helps ensure Americans can access the medicines they need at lower costs. The voluntary agreement addresses all four components of the President’s July letter. Under the agreement, among other things, the Company plans to provide key products through a direct-to-patient program at affordable prices for eligible patients in the U.S. In addition, the Company reached an understanding with the U.S. Department of Commerce to delay Section 232 tariffs for three years, enabling the Company to make investments in the U.S. to reshore manufacturing for American patients. The Company has committed more than $70 billion in capital and R&D spending to strengthen U.S. production and innovation. Full-Year 2026 Financial Outlook The following table summarizes the Company’s full-year financial outlook. Full Year 2026 Sales* $65.5 billion to $67.0 billion Non-GAAP Gross margin2 Approximately 82% Non-GAAP Operating expenses2** $35.9 billion to $36.9 billion Non-GAAP Other (income) expense, net2 Approximately $1.3 billion expense Non-GAAP Effective tax rate2 23.5% to 24.5% Non-GAAP EPS2*** $5.00 to $5.15 Share count (assuming dilution) Approximately 2.48 billion *The Company does not have any non-GAAP adjustments to sales. **Includes a one-time charge of approximately $9.0 billion associated with the acquisition of Cidara. Outlook does not assume any additional significant potential business development transactions. ***Includes a one-time charge of approximately $3.65 per share associated with the acquisition of Cidara. The Company has not provided a reconciliation of forward-looking non-GAAP gross margin, non-GAAP operating expenses, non-GAAP other (income) expense, net, non-GAAP effective tax rate and non-GAAP EPS to the most directly comparable GAAP measures, given it cannot predict with reasonable certainty the amounts necessary for such a reconciliation, including intangible asset impairment charges, legal settlements, and income and losses from investments in equity securities either owned directly or through ownership interests in investment funds, without unreasonable effort. These items are inherently difficult to forecast and could have a significant impact on the Company’s future GAAP results. The Company anticipates full-year 2026 sales to be between $65.5 billion and $67.0 billion, including a positive impact from foreign exchange of approximately 1% at mid-January 2026 exchange rates. The Company’s full-year non-GAAP effective income tax rate is expected to be between 23.5% and 24.5% including the impact of the non-tax deductible one-time charge for the acquisition of Cidara. The Company expects full-year 2026 non-GAAP EPS to be between $5.00 and $5.15, including a positive impact from foreign exchange of approximately $0.10 per share at mid-January 2026 exchange rates. This range includes a one-time charge of approximately $9.0 billion, or approximately $3.65 per share, as well as approximately $0.30 per share of related financing and operational costs, related to the acquisition of Cidara. In 2025, non-GAAP EPS of $8.98 was negatively impacted by one-time charges of $0.20 per share related to certain business development transactions. Consistent with past practice, the financial outlook does not assume additional significant potential business development transactions. Non-GAAP EPS excludes acquisition- and divestiture-related costs, costs related to restructuring programs, as well as income and losses from investments in equity securities. Earnings Conference Call Investors, journalists and the general public may access a live audio webcast of the call on Tuesday, Feb. 3, at 9 a.m. ET via this weblink. A replay of the webcast, along with the sales and earnings news release, supplemental financial disclosures and slides highlighting the results, will be available on the Company’s website. All participants may join the call by dialing (800) 369-3351 (U.S. and Canada Toll-Free) or (517) 308-9448 and using the access code 9818590. About Our Company At Merck & Co., Inc., Rahway, N.J., USA, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. Forward-Looking Statement of Merck & Co., Inc., Rahway, N.J., USA This news release of Merck & Co., Inc., Rahway, N.J., USA (the “Company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the Company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the Company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the Company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024 and the Company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site (www.sec.gov). Appendix Generic product names are provided below. Pharmaceutical BRIDION (sugammadex) CAPVAXIVE (Pneumococcal 21-valent Conjugate Vaccine) ENFLONSIA (clesrovimab-cfor) GARDASIL (Human Papillomavirus Quadrivalent [Types 6, 11, 16 and 18] Vaccine, Recombinant) GARDASIL 9 (Human Papillomavirus 9-valent Vaccine, Recombinant) JANUMET (sitagliptin and metformin HCl) JANUVIA (sitagliptin) KEYTRUDA (pembrolizumab) KEYTRUDA QLEX (pembrolizumab and berahyaluronidase alfa-pmph) LAGEVRIO (molnupiravir) Lenvima (lenvatinib) Lynparza (olaparib) M-M-R II (Measles, Mumps and Rubella Virus Vaccine Live) OHTUVAYRE (ensifentrine) PREVYMIS (letermovir) PROQUAD (Measles, Mumps, Rubella and Varicella Virus Vaccine Live) Reblozyl (luspatercept-aamt) ROTATEQ (Rotavirus Vaccine, Live, Oral, Pentavalent) Simponi (golimumab) VARIVAX (Varicella Virus Vaccine Live) VAXNEUVANCE (Pneumococcal 15-valent Conjugate Vaccine) WELIREG (belzutifan) WINREVAIR (sotatercept-csrk) Animal Health BRAVECTO (fluralaner) ____________________ 1 Net income attributable to the Company. 2 The Company is providing certain 2025 and 2024 non-GAAP information that excludes certain items because of the nature of these items and the impact they have on the analysis of underlying business performance and trends. Management believes that providing this information enhances investors’ understanding of the Company’s results because management uses non-GAAP results to assess performance. Management uses non-GAAP measures internally for planning and forecasting purposes and to measure the performance of the Company along with other metrics. In addition, annual employee compensation, including senior management’s compensation, is derived in part using a non-GAAP pretax income metric. This information should be considered in addition to, but not as a substitute for or superior to, information prepared in accordance with GAAP. For a description of the non-GAAP adjustments, see Table 2a attached to this release. 3 Reflects expenses related to business combinations, including the amortization of intangible assets, intangible asset impairment charges, and expense or income related to changes in the estimated fair value measurement of liabilities for contingent consideration. Also includes integration, transaction and certain other costs associated with acquisitions and divestitures, as well as amortization of intangible assets related to collaborations, licensing arrangements and asset acquisitions, and recognition of fair value step-up to inventories for asset acquisitions. 4 Includes the estimated tax impacts on the reconciling items based on applying the statutory rate of the originating territory of the non-GAAP adjustments for all periods presented. Amount in the full year of 2025 also includes a $60 million net benefit, which reflects a net benefit related to favorable audit reserve adjustments. Amounts in the fourth quarter and full year of 2024 also include a $260 million benefit and a $519 million benefit, respectively, due to reductions in reserves for unrecognized income tax benefits resulting from the expiration of the statute of limitations for assessments related to certain federal tax return years. The benefit recognized in the fourth quarter of 2024 relates to the 2020 federal tax return year and the benefit for the full year of 2024 relates to both the 2020 and 2019 federal tax return years. 4Q25 4Q24 $ 16,400 $ 15,624 5% $ 65,011 $ 64,168 1% 5,551 3,828 45% 16,382 15,193 8% 2,898 2,864 1% 10,733 10,816 -1% 3,886 4,585 -15% 15,789 17,938 -12% 213 51 889 309 432 126 151 (24 ) 3,420 4,170 -18% 21,067 19,936 6% 458 425 2,804 2,803 2,962 3,745 -21% 18,263 17,133 7% (1 ) 2 9 16 $ 2,963 $ 3,743 -21% $ 18,254 $ 17,117 7% $ 1.19 $ 1.48 -20% $ 7.28 $ 6.74 8% 2,488 2,537 2,507 2,541 13.4 % 10.2 % 13.3 % 14.1 % $ 5,551 1,054 1,173 2,227 $ 3,324 2,898 48 2 50 2,848 3,886 5 (111 ) (106 ) 3,992 213 213 213 – 432 206 206 226 3,420 (1,107 ) (1,277 ) (206 ) (2,590 ) 6,010 458 (187 ) (3) (234 ) (3) (44 ) (3) (465 ) 923 2,962 (920 ) (1,043 ) (162 ) (2,125 ) 5,087 2,963 (920 ) (1,043 ) (162 ) (2,125 ) 5,088 $ 1.19 (0.37 ) (0.42 ) (0.06 ) (0.85 ) $ 2.04 13.4 % 15.4 % $ 16,382 2,871 1,484 4,355 $ 12,027 10,733 120 3 123 10,610 15,789 19 175 194 15,595 889 889 889 – 151 (3 ) (306 ) (309 ) 460 21,067 (3,007 ) (2,551 ) 306 (5,252 ) 26,319 2,804 (525 ) (3) (473 ) (3) 65 (3) (60 ) (4) (993 ) 3,797 18,263 (2,482 ) (2,078 ) 241 60 (4,259 ) 22,522 18,254 (2,482 ) (2,078 ) 241 60 (4,259 ) 22,513 $ 7.28 (0.99 ) (0.83 ) 0.10 0.02 (1.70 ) $ 8.98 13.3 % 14.4 % 2025 2024 4Q Full Year $15,529 $15,806 $17,276 $16,400 $65,011 $15,775 $16,112 $16,657 $15,624 $64,168 5 4 1 2 13,638 14,050 15,611 14,843 58,142 14,006 14,408 14,943 14,042 57,400 6 4 1 1 7,205 7,956 8,142 8,337 31,641 6,947 7,270 7,429 7,836 29,482 6 5 7 7 5 35 40 - - - - 312 370 379 389 1,450 292 317 337 365 1,311 7 4 11 10 258 265 258 272 1,053 255 249 251 255 1,010 7 6 4 4 137 162 196 220 716 85 126 139 160 509 37 37 41 41 119 107 136 164 525 71 90 100 110 371 48 48 41 41 1,327 1,126 1,749 1,031 5,233 2,249 2,478 2,306 1,550 8,583 -34 -35 -39 -39 539 609 684 619 2,451 570 617 703 594 2,485 4 3 -1 -2 230 229 226 140 825 219 189 239 161 808 -13 -16 2 1 107 129 244 279 759 47 50 97 228 121 204 119 673 216 163 193 139 711 -14 -15 -5 -5 41 38 45 42 166 61 59 68 74 263 -43 -44 -37 -37 441 461 439 499 1,841 440 455 420 449 1,764 11 11 4 4 208 228 266 275 978 174 188 208 215 785 28 26 25 23 70 74 81 87 312 56 62 64 70 252 24 23 24 24 83 96 43 25 247 73 92 96 79 340 -68 -68 -27 -27 280 336 360 467 1,443 70 149 200 419 133 133 106 123 112 129 470 98 106 102 109 415 18 18 13 13 68 80 82 83 312 70 72 72 73 287 14 9 9 6 178 178 - - - - 102 83 138 57 380 350 110 383 121 964 -53 -53 -61 -61 90 86 82 67 325 111 89 102 92 394 -27 -28 -18 -18 67 83 77 79 306 56 60 65 69 249 15 9 23 20 45 41 43 42 171 42 39 42 40 163 6 4 5 4 50 40 47 49 186 46 53 78 45 222 8 9 -16 -16 184 172 189 543 -100 -100 39 35 41 114 -100 -100 549 372 382 302 1,604 419 405 278 232 1,334 30 30 20 21 247 251 243 199 940 251 224 204 255 935 -22 -22 1 2 729 584 948 658 2,917 632 618 638 699 2,590 -6 -5 13 13 1,588 1,646 1,615 1,505 6,354 1,511 1,482 1,487 1,397 5,877 8 6 8 9 924 961 1,023 987 3,896 850 837 886 889 3,462 11 9 13 14 664 685 592 518 2,458 661 645 601 508 2,415 2 0 2 2 303 110 50 52 515 258 222 227 185 891 -71 -15 -42 -6

Clinical ResultAcquisitionPhase 3Financial StatementVaccine

14 Jan 2026

·endpoints.news

Q&A: Bristol Myers CEO Chris Boerner on megamergers, Trump in 2026, and brain health as the 'next frontier'

SAN FRANCISCO — In his third year as CEO of Bristol Myers Squibb, Chris Boerner kicked off the JP Morgan Healthcare Conference with plans for six pivotal readouts of new drugs in 2026. This array of potential medicines for everything from heart disease to cancer to neuropsychiatry will be key in replacing revenues from current bestsellers Opdivo and Eliquis, which are expected to potentially face generic competition in 2028. The $114 billion pharma has been on a dealmaking run to bolster its pipeline and portfolio since Boerner took the helm, acquiring RayzeBio for $4.1 billion , Mirati Therapeutics for $4.8 billion , and Karuna Therapeutics for $14 billion . Boerner sat down with Endpoints News on Tuesday, the day after his JPM presentation, for a wide-ranging discussion of the year ahead and his take on the future of Bristol Myers and the pharma industry. The following transcript has been edited for length and clarity. Andrew Dunn: We’ll get to JPM stuff, but I wanted to actually start with your 2002 PhD dissertation on why some pharmas are better at developing drugs than others. Chris Boerner: Oh, wow. That’s going way back. Dunn: I figured that’d be an interesting place to start. What was your answer in 2002, and how does that hold up today in 2026? Boerner: I have to remember first. What prompted me to look at that question was that I read an article that talked about Bristol Myers Squibb being really great at developing oncology drugs. I called somebody who had graduated from Berkeley a few years before, and I said, ‘I don’t understand why BMS is so much better.’ It turns out nobody has done that work. If you boil it down, the core element is that experience matters. It matters a lot. The data suggested that BMS and other companies that were very good at developing drugs in a given therapeutic area, they had a lot of history of developing in that space. What that meant was they knew the science, they had access to physicians who could help them prosecute that science, they knew how to develop the medicines in terms of clinical trials, so the data showed very clearly they were faster and had a higher probability of success. If you’re doing business development, the idea at the time was these virtual pharmaceutical companies. You could have MBAs and PhDs, and they could go select assets and in-license them and create companies. It turns out that’s very difficult to do, because you don’t know which science to pick. Dunn: What do you think is the biggest change from ’02 to ’26 on that topic? Boerner: It begs the question of how do you ever get into something new. The most successful companies, in many respects, have taken two paths. One is they buy infrastructure, and they don’t screw it up. They keep the people and the knowledge base that exists. Or they follow the science down a path, and it leads them into a new area. A good example of that is how Genentech got into ophthalmology. They followed the science for VEGF in oncology and wove their way over to ophthalmology because that’s where the science takes them. Dunn: There’s not a dogmatic approach of “We don’t do that.” Boerner: Exactly. Companies can be overly strategic and just say, well, we’ll just put blinders on that. You’ve got to be careful about that. When we acquired Rayze right after I became CEO, we made the decision to keep that largely separate. As a result, we did the most important thing: We kept all of those people who had deep expertise. Fundamentally, we’ve kept that as a freestanding unit, and that very much ties into one of the insights I had coming out of that Berkeley work. Dunn: My first JPM was 2019, the year of the Bristol-Celgene deal. Boerner: That was a pretty big deal to happen. Dunn: How do you judge the success of that deal? What are the lessons learned, and anything you’d have done differently? Boerner: Bristol Myers Squibb going into that acquisition, we were a company heavily indexed on two medicines: Eliquis and Opdivo. Every earnings call was about those two medicines. What we accomplished with that deal is to broaden the discussion. I highlighted yesterday that within our growth portfolio, we’ve got four medicines young in the lifecycle, all annualizing to over $1 billion in sales: Camzyos, Breyanzi, Opdualag, and Reblozyl, which is actually analyzing to over $2 billion. That diversity in your business is a real strongpoint for us because you’re not beholden to one or two blockbuster medicines as you think about what the growth trajectory of the company is going to be in the long term. Lessons learned for the future: How you integrate a big acquisition like that has to be done very thoughtfully. There are places we probably could have integrated faster. Dunn: Is there an example? Boerner: Three or four years later, we were still surveying the employee base to say were you heritage Celgene or heritage BMS? But at the time we were doing that, the majority of the people at the company had joined after the Celgene acquisition. In some ways, we ended up distracting ourselves with that, which is one big challenge with any big acquisition. Dunn: $30 billion in deals over the past two years. Do you have a target deal size or general M&A capacity for 2026? Boerner: We are somewhat agnostic on size. Our criteria remain the same: One, it has to be an area of science we know. Two, it has to make strategic sense. And finally, perhaps obviously, but in some cases not obviously, it’s got to make financial sense. Dunn: Is the idea of a megamerger on the table in 2026? Boerner: That’s not our primary focus, to be honest with you. Our primary focus is on doing deals more akin to the ones you’ve seen us do over the last three or four years. Dunn: What led to increasing Bristol’s exposure to neurology, particularly with the anti-psychotic Cobenfy, and what do you see as the future of these diseases, given they feel like the hardest to make traction on? Boerner: They are hard, but the nice thing about the history of Bristol Myers Squibb is we focus on hard things. The next frontier in healthcare, I believe, is going to be brain health. We have some mid-stage programs in Alzheimer’s disease. I’m looking forward to seeing them continue to progress. When we made the decision to invest in Karuna, we were making the decision to do two things: first, commit to neuroscience, not just neurodegeneration, but neuropsych. Because of the nature of the asset we were acquiring and the potential to not just focus on schizophrenia but Alzheimer’s disease psychosis, agitation and cognition, it was a bridge between neuropsych and neurodegeneration. We had already made the commitment to be in this space, and this was an opportunity to expand, but also to bridge. Dunn: Biggest potential opportunity and risk in working with the Trump administration in 2026? Boerner: The biggest opportunity — one of the things that has been refreshing with this administration is they have recognized that access and affordability are absolutely critical. Every dollar associated with a branded medicine, about 50% of that dollar goes to somebody who didn’t take any risk in the discovery, development and certainly isn’t involved in the commercialization of that product. There’s a real opportunity for us to find ways to simplify this healthcare system. The risk, and it’s less of a risk associated with working with this administration, but it’s a risk for the United States in terms of the ecosystem that we have here. We have the best ecosystem for biopharmaceuticals in the world, and it is the envy of the world. This conference is an embodiment of a lot of that ecosystem that doesn’t exist in its form like this anywhere else in the world. But it is not a birthright. There are other places, China is notable among them, that have made a stated ambition and are making great progress in replicating that. The challenge we all have in the United States is how do you strengthen this ecosystem? We have a vested interest as a company that’s been in the United States for over 160 years to ensure that the future of the ecosystem here continues to be as attractive as it is today, if not more attractive. That is the fundamental challenge we have as an industry.

Acquisition

15 Dec 2025

·fibrogen.gcs-web.com

Roxadustat Granted Orphan Drug Designation for the Treatment of Myelodysplastic Syndromes by the U.S. Food and Drug Administration

Company is on track to file the Phase 3 protocol in the fourth quarter of 2025 SAN FRANCISCO, Dec. 15, 2025 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted roxadustat Orphan Drug Designation for the treatment of myelodysplastic syndromes (MDS). “The Orphan Drug Designation granted to roxadustat for MDS underscores the significant treatment gap in this indication, and highlights patients’ need for additional convenient treatments that can provide durable response,” said Thane Wettig, Chief Executive Officer of FibroGen. “Roxadustat showed an improvement in transfusion-independence in a subset of patients with high transfusion burden in a post-hoc analysis from the Phase 3 MATTHERHORN trial, which along with its favorable tolerability profile and oral route of administration has the ability to set it apart from current second-line treatments. Our team is finalizing the Phase 3 protocol in this patient population for submission to the FDA in the fourth quarter of 2025.” There are approximately 58,000 patients diagnosed with LR-MDS in the U.S. with 85% of them suffering from anemia. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusions. Transfusion-dependent patients suffer higher rates of complications and decreased quality of life. Current first-line treatments lead to transfusion independence in less than 50% of patients and relief is often temporary with limited options for second line and beyond treatments. In a post-hoc analysis from the Phase 3 MATTERHORN trial, roxadustat demonstrated transfusion independence benefits compared to placebo in patients with high transfusion burden. The FDA Orphan Drug Designation is granted to drugs intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Benefits of the designation may include exemption from certain FDA fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. following drug approval. About Myelodysplastic Syndromes Anemia Myelodysplastic syndromes (MDS) are a group of disorders characterized by dysfunctional progenitor blood cells and stem cells, resulting in chronic anemia in most patients. Annual incidence rates of MDS are estimated to be 4.9/100,000 adults in the U.S, of which 77% are considered lower-risk MDS. Approximately 80% of patients with MDS have anemia at the time of diagnosis, and around 60% of patients with MDS will experience severe anemia (hemoglobin <8 g/dL) at some point during the course of their disease. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusion. Approximately 50% of patients with MDS require regular red blood cell transfusions. Transfusion-dependent MDS patients suffer higher rates of cardiac events, infections, and iron overload with the related complications. In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. Today, patients are routinely treated with erythropoiesis-stimulating agents (ESAs), luspatercept, imetelstat, or lenalidomide in lower-risk MDS with isolated del(5q), and hypomethylating agents (HMAs) in higher-risk disease. Only 35-40% of patients respond to current treatments and the durability of response is short. Moreover, these treatments are challenging to dose-calibrate and can only be administered via subcutaneous injection or through IV infusion. There remains a high unmet need for the treatment of anemia associated with MDS, and new strategies that provide durable response and the convenience of oral administration are highly desired in managing patients with MDS. About Roxadustat Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is approved in Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). FibroGen has the sole rights to roxadustat in the United States, Canada, Mexico, and in all markets not held by AstraZeneca or licensed to Astellas. Astellas and FibroGen are collaborating on the commercialization of roxadustat for the treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa. About FibroGen FibroGen, Inc. is a biopharmaceutical company focused on development of novel therapies at the frontiers of cancer biology and anemia. Roxadustat (爱瑞卓®, EVRENZOTM) is currently approved in Europe, Japan, and numerous other countries for the treatment of anemia in chronic kidney disease (CKD) patients on dialysis and not on dialysis. The Company continues to evaluate the development plan for the Phase 3 trial of roxadustat in anemia associated with lower-risk myelodysplastic syndrome (LR-MDS) in the U.S. FG-3246 (also known as FOR46), a first-in-class antibody-drug conjugate (ADC) targeting CD46, is in Phase 2 development for the treatment of metastatic castration-resistant prostate cancer. This program also includes the development of FG-3180, an associated CD46-targeted PET biomarker. For more information, please visit www.fibrogen.com. Forward-Looking Statements This release contains forward-looking statements regarding FibroGen’s strategy, future plans and prospects, including statements regarding its commercial products and clinical programs and those of it and its collaboration partners Fortis and UCSF. These forward-looking statements include, but are not limited to, statements regarding the efficacy, safety, convenience, and potential clinical or commercial success of FibroGen products and product candidates, statements about regulatory interactions, and statements about FibroGen’s plans and objectives. These forward-looking statements are typically identified by use of terms such as “may,” “will”, “should,” “on track,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue” and similar words, although some forward-looking statements are expressed differently. FibroGen’s actual results may differ materially from those indicated in these forward-looking statements due to risks and uncertainties related to the continued progress and timing of its various programs, including the enrollment and results from ongoing and potential future clinical trials, and other matters that are described in FibroGen’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q, each as filed with the Securities and Exchange Commission (SEC), including the risk factors set forth therein. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and FibroGen undertakes no obligation to update any forward-looking statement in this press release, except as required by law. For Investor Inquiries: David DeLucia, CFA Senior Vice President and Chief Financial Officer ir@fibrogen.com Source: FibroGen, Inc.

Phase 2Phase 3Orphan DrugDrug ApprovalClinical Result

100 Deals associated with Luspatercept-AAMT

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KEGG	Wiki	ATC	Drug Bank
-	Luspatercept-AAMT	B03XA06	DB12281

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Non-transfusion dependent thalassaemia	European Union	Bristol-Myers Squibb Pharma EEIG	20 Mar 2023
Non-transfusion dependent thalassaemia	Iceland	Bristol-Myers Squibb Pharma EEIG	20 Mar 2023
Non-transfusion dependent thalassaemia	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	20 Mar 2023
Non-transfusion dependent thalassaemia	Norway	Bristol-Myers Squibb Pharma EEIG	20 Mar 2023
Anemia	South Korea	Bristol Myers Squibb Co.	09 May 2022
Anemia, Sideroblastic	European Union	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Anemia, Sideroblastic	Iceland	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Anemia, Sideroblastic	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Anemia, Sideroblastic	Norway	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Myelodysplastic Syndromes	European Union	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Myelodysplastic Syndromes	Iceland	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Myelodysplastic Syndromes	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Myelodysplastic Syndromes	Norway	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion dependent anaemia	European Union	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion dependent anaemia	Iceland	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion dependent anaemia	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion dependent anaemia	Norway	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion-dependent Beta Thalassemia	European Union	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion-dependent Beta Thalassemia	Iceland	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020
Transfusion-dependent Beta Thalassemia	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	25 Jun 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Myelofibrosis	Phase 3	China	Celgene Corp.	23 Feb 2022
Polycythemia Vera	Phase 3	China	Celgene Corp.	23 Feb 2022
Thrombocythemia, Essential	Phase 3	China	Celgene Corp.	23 Feb 2022
Post-polycythemia vera myelofibrosis	Phase 3	United States	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	China	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	Japan	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	Argentina	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	Australia	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	Austria	Celgene Corp.	25 Feb 2021
Post-polycythemia vera myelofibrosis	Phase 3	Belgium	Celgene Corp.	25 Feb 2021

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2025	Not Applicable	Non-transfusion dependent thalassaemia	50	Luspatercept 1.0 mg/kg Q3W	uvtseyxwzl(bueunbmlun) = kaebvgqpgk yoqxlfweoh (lliwyrfxem ) View more	Positive	06 Dec 2025
QOL-ONE Phoenix (ASH2025)	Phase 2	Anemia	22	Luspatercept 1.0 mg/kg	hljmldvouv(uyzhbfbnzt) = ojysuewbjn rtikgwtztf (oapxpsxbfr ) View more	Positive	06 Dec 2025
NCT03682536 (COMMANDS, ASH2025)	Phase 3	Myelodysplastic Syndromes	363	Luspatercept	uatxkhlgjv(yxkvrgkokv) = vfgfbhytfp wwefaicqhm (ebjgqlmghy ) View more	Positive	06 Dec 2025
				Erythropoiesis-stimulating agent (ESA) epoetin alfa	uatxkhlgjv(yxkvrgkokv) = prcvuszalw wwefaicqhm (ebjgqlmghy ) View more
ASH2025	Not Applicable	Myelodysplastic Syndromes	1,482	Luspatercept	aimcuslige(cmdadctbgz) = ggshxumtby hsckcrwakl (cabgccttsm ) View more	Positive	06 Dec 2025
				Erythropoiesis-stimulating agents (ESAs)	aimcuslige(cmdadctbgz) = hrupshasdv hsckcrwakl (cabgccttsm ) View more
ASH2025	Not Applicable	Myelodysplastic Syndromes	104	Luspatercept (Non-transfusion dependent (NTD))	nsigfrrwtz(jpkijmicao) = bbvznchnqt yohnyftlws (nwjdrsugzt ) View more	Positive	06 Dec 2025
				Luspatercept (Low TB (LTB))	nsigfrrwtz(jpkijmicao) = ktvokoamzq yohnyftlws (nwjdrsugzt ) View more
NCT02604433 (BELIEVE, ASH2025)	Phase 3	Transfusion-dependent Thalassemia	336	Luspatercept	afowxmxlkv(ovdrxcjoef) = ftidslnrzj tdgvaahqvt (fbbqnthsns ) View more	Positive	06 Dec 2025
				Placebo	afowxmxlkv(ovdrxcjoef) = cnqngtzbxn tdgvaahqvt (fbbqnthsns ) View more
NCT06045689 (MAXILUS, ASH2025)	Phase 3	Myelodysplastic Syndromes	105	Luspatercept 1.75 mg/kg	calqvfpzch(iwuszzqrfz) = dzdamcbuxl eszyghxdys (ssjmddxhcr ) View more	Positive	06 Dec 2025
				Luspatercept 1.75 mg/kg	calqvfpzch(iwuszzqrfz) = iounxtotfm eszyghxdys (ssjmddxhcr ) View more
ASH2025	Not Applicable	Myelodysplastic Syndromes First line	320	Luspatercept 1 mg/kg	klcmnqdgsg(gbafooiuhv) = ffxukmajnu cqmotnwqzz (xhscjnogpf ) View more	Positive	06 Dec 2025
				Luspatercept (TD)	ylqdacogcu(dohkvasigu) = zirsrbjobe rowteqeivb (rjrfcrvuhe )
ASH2025	Not Applicable	Myelodysplastic Syndromes First line	418	Luspatercept	woczfsvmrj(saslureybi) = nzkkcjyfrb fbcnhrakik (tskhslfthd ) View more	Positive	06 Dec 2025
				ESAs	woczfsvmrj(saslureybi) = bopaprzddr fbcnhrakik (tskhslfthd ) View more
ASH2025	Not Applicable	Anemia, Myelophthisic	99	Luspatercept	nqzbktjdnx(zpfbvxrjlk) = xwnmieshuf dzrkyjwbdo (imorhukwdt, 30.4 - NR) View more	Positive	06 Dec 2025

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