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Last update 10 Mar 2026

Cemdisiran

Last update 10 Mar 2026

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

siRNA

Synonyms

AD-62643, ALN-62643, ALN-CC5

Target

Action

inhibitors

Mechanism

C5 inhibitors(Complement C5 inhibitors)

Therapeutic Areas

NeoplasmsImmune System DiseasesNervous System Diseases+ [5]

Active Indication

Inactive Indication

Atypical Hemolytic Uremic Syndrome

Originator Organization

Alnylam Pharmaceuticals, Inc.

Active Organization

Regeneron Pharmaceuticals, Inc.

Curia, Inc

Zai Lab (Shanghai) Co., Ltd.

+ [1]

Inactive Organization

Curiato, Inc.

License Organization

Regeneron Pharmaceuticals, Inc.

Alnylam Pharmaceuticals, Inc.

Drug Highest PhasePhase 3

First Approval Date-

RegulationOrphan Drug (United States), Orphan Drug (European Union)

Structure/Sequence

Boost your research with our RNA technology data.

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Sequence Code 32355856

Source: *****

Sequence Code 1103240105

Source: *****

Clinical Trials associated with Cemdisiran

NCT07154745

/ RecruitingPhase 3

A Single Arm Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Control of Intravascular Hemolysis on Currently Available C5 Inhibitor Therapy

This study is researching a treatment combination with two experimental drugs called pozelimab and cemdisiran referred to as "study drugs". Researchers are looking for a better way to treat Paroxysmal Nocturnal Hemoglobinuria (PNH).
The aim of the study is to see how well the pozelimab and cemdisiran combination works to lower hemolysis in participants whose PNH has been not well controlled even after taking other complement component 5 (C5) inhibitors, eculizumab/eculizumab biosimilar, ravulizumab or crovalimab.
The study is looking at several other research questions, including:
* What side effects may happen from taking the study drugs?
* How much of the study drugs are in the blood at different times?
* Whether the body makes antibodies against the study drug (which could make the study drugs not work as well or could lead to side effects)

Start Date30 Jan 2026

Sponsor / Collaborator

Regeneron Pharmaceuticals, Inc.

KCT0010985

/ Not yet recruitingPhase 3

A SINGLE ARM STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WITH INADEQUATE CONTROL OF INTRAVASCULAR HEMOLYSIS ON CURRENTLY AVAILABLE C5 INHIBITOR THERAPY

Start Date01 Dec 2025

Sponsor / Collaborator-

NCT06541704

/ RecruitingPhase 3

A Multicenter, Randomized, Double-Masked, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Tolerability of Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

This study is researching experimental (study) drugs called pozelimab and cemdisiran. The study is focused on participants who have Geographic Atrophy (GA) caused by Age-related Macular Degeneration (AMD). Geographic atrophy is a medical term that refers to later-stage cases of AMD which is an eye condition affecting central vision (what one sees straight ahead).
The purpose of this study is to evaluate the progression rate of Geographic Atrophy in eyes of patients treated with cemdisiran alone or in combination with pozelimab compared to those treated with placebo.
The study is looking at several other research questions, including:
* What side effects may happen from taking the study drug(s)
* How much study drug(s) are in the blood at different times
* Whether the body makes antibodies against the study drug(s) (which could make the study drug(s) less effective or could lead to side effects)

Start Date30 Oct 2024

Sponsor / Collaborator

Regeneron Pharmaceuticals, Inc.

100 Clinical Results associated with Cemdisiran

100 Translational Medicine associated with Cemdisiran

100 Patents (Medical) associated with Cemdisiran

Literatures (Medical) associated with Cemdisiran

22 Dec 2025·Cureus Journal of Medical Science

Emerging Therapies in IgA Nephropathy: From A Proliferation-Inducing Ligand (APRIL) and B-cell Activating Factor (BAFF) Inhibitors to Precision Medicine

Review

Author: Panta, Raju ; Sharma, Ishwor

IgA nephropathy (IgAN) is the most prevalent primary glomerular disease worldwide and a significant contributor to end-stage kidney disease (ESKD). Traditional management has centered on supportive care and non-specific immunosuppression, but recent advances in the understanding of pathogenic pathways have catalyzed the development of targeted therapies. This review synthesizes current evidence on evolving treatments, with a focus on A Proliferation-Inducing Ligand (APRIL) and B-cell Activating Factor (BAFF) (e.g., sibeprenlimab, atacicept, povetacicept, telitacicept), complement pathway modulators (e.g., iptacopan, cemdisiran, ravulizumab), and novel agents such as felzartamab and sparsentan. It also explores precision medicine strategies, including biomarker-guided therapy, individualized risk stratification, and combination regimens. Supported by high-quality recent clinical trial data and the latest kidney disease outcome guidelines, these innovations represent a paradigm shift toward personalized, disease-modifying treatment in IgAN, offering a new horizon for improved renal outcomes and long-term disease control.

01 Nov 2025·DRUGS

Pharmacological Therapies in Paroxysmal Nocturnal Haemoglobinuria: Focus on Complement Inhibition

Review

Author: Holt, Matthew ; Szer, Jeff ; Kelly, Richard J

Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-rare acquired genetic stem cell disorder based on a mutation in the PIGA gene that results in susceptibility of resulting blood cells to complement-mediated intravascular haemolysis (IVH). In countries where anti-complement therapy is available, pharmacological treatments have transformed this disease from a highly morbid and sometimes lethal disorder. The first treatment developed was the terminal complement (C5) monoclonal antibody inhibitor eculizumab, in 2002. This has been largely supplanted by a longer-acting antibody, ravulizumab, targeting the same binding site on C5. These agents significantly modify the natural history of the disease by reducing the risk of thrombosis, the most lethal complication of PNH, as well as reducing transfusion dependence and improving renal function, quality of life and probably, survival. Other terminal inhibitors available include eculizumab biosimilars, crovalimab, pozelimab and cemdisiran (combination). Despite this, a proportion of patients develop extravascular haemolysis (EVH) based on the accumulation of C3 components on these PNH blood cells, which no longer undergo IVH because of C5 inhibition. This has led to the development of proximal complement inhibitors, which have been generally successful at reducing this iatrogenic complication, improving haemoglobin concentrations, reducing transfusion dependency and improving quality of life. Currently available proximal inhibitors (and their targets) are pegcetacoplan (C3), danicopan (Factor D) and iptacopan (Factor B). While effective, as with all other complement inhibitors, there is a risk of breakthrough IVH with their use and approaches to manage this complication are being developed.

01 Aug 2025·EJHaem

Safety, Efficacy, and Patient‐Reported Outcomes From a Phase 2 Randomized Trial of Pozelimab and Cemdisiran Combination in Patients With Paroxysmal Nocturnal Hemoglobinuria

Article

Author: Pavani, Rodrigo ; Wong, Raymond Siu Ming ; Perlee, Lorah ; Mohan, Kosalai ; Kelly, Richard J. ; Souttou, Amal ; Meagher, Karoline ; Hartford, Christopher ; Sherman, Steven ; Aurand, Lisa ; Rofail, Diana ; Jang, Jun‐Ho ; Nguyen, Quang

ABSTRACT:

Introduction:

Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra‐rare, life‐threatening disease associated with chronic intravascular hemolysis due to uncontrolled complement activation. PNH results in anemia with an increased risk of thrombosis, and often causes severe fatigue, and decreased physical function and health‐related quality of life (QoL). We investigated the efficacy, safety, and patient‐reported outcomes data of the combination of pozelimab (a fully human monoclonal antibody) and cemdisiran (an N‐acetylgalactosamine‐conjugated small interfering ribonucleic acid) from a Phase 2 trial (NCT04811716) in patients with PNH who transitioned from pozelimab monotherapy.

Methods:

In this randomized, open‐label, Phase 2 study, patients were randomized (1:1) to one of two treatment arms; both arms received subcutaneous cemdisiran 200 mg every 4 weeks (Q4W) plus subcutaneous pozelimab 400 mg either Q4W (Arm 1) or every 2 weeks (Arm 2).

Results:

Twenty‐four patients were treated with combination dosing. During the 28‐week open‐label treatment period (OLTP), 20 patients (83.3%) maintained control of lactate dehydrogenase (≤ 1.5 × upper limit of normal) at all timepoints. The majority of patients (92%) did not require a blood transfusion. While most patients (66.7%) experienced treatment‐emergent adverse events, the majority of these events were mild to moderate in severity. No meningococcal infections, thrombotic events, or deaths were reported. The combination therapy maintained improvements in patient‐reported fatigue, physical functioning, and QoL throughout the OLTP.

Conclusion:

Combination treatment maintained adequate hemolysis control and was generally well tolerated. Administration of pozelimab Q2W did not improve disease control as compared to pozelimab Q4W.

Trial Registration:

ClinicalTrials.gov/NCT04811716

News (Medical) associated with Cemdisiran

12 Feb 2026

·www.biospace.com

Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2025 Financial Results and Highlights Recent Period Progress

− Achieved Fourth Quarter and Full Year 2025 Global Net Product Revenues of $995 Million and $2,987 Million, Respectively, Representing 121% and 81% Growth Compared to Same Periods in 2024 – − Attained GAAP and non-GAAP Profitability for Full Year 2025, with Sustainable Growth in Operating Income Expected – − Launched “ Alnylam 2030 ” Strategy Focused on Scaling Alnylam through Durable ATTR Leadership, Long-Term Sustainable Innovation, and Exceptional Financial Results – − Announced 2026 Pipeline Goals, Including 4 Clinical Readouts, 3 Ongoing Pivotal Studies, 3 Phase 2 Study Initiations, and 3+ New IND Filings – − Reiterates Net Product Sales Guidance and Provides Additional 2026 Financial Guidance – CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today reported its consolidated financial results for the fourth quarter and full year ended December 31, 2025 and reviewed recent business highlights. “2025 was a year of key accomplishments for Alnylam, highlighted by the landmark approval of AMVUTTRA for ATTR-CM in the U.S., which drove total net product revenues of nearly $3 billion, or 81% growth year-over-year, and propelled us to profitability. We also achieved great progress across our portfolio, initiating three Phase 3 studies, expanding our pipeline with four proprietary CTAs, and launching a potential best-in-class enzymatic ligation-based RNAi manufacturing platform,” said Yvonne Greenstreet, M.D., Chief Executive Officer of Alnylam. “Further, we are excited to have recently unveiled our new set of five-year aspirational goals, Alnylam 2030 , under which we aim to achieve global TTR leadership with a durable franchise; grow through innovation by delivering therapies that prevent, halt, or reverse disease; and scale with financial discipline and agility. By pursuing these ambitious goals, we believe Alnylam will drive substantial patient impact by addressing serious unmet medical needs around the world and create substantial long-term shareholder value.” Fourth Quarter 2025 and Recent Significant Business Highlights Total TTR: AMVUTTRA ® (vutrisiran) & ONPATTRO ® (patisiran) Achieved global net product revenues for AMVUTTRA and ONPATTRO for the fourth quarter of $827 million and $32 million, respectively, together representing 151% total TTR growth compared to Q4 2024, and full year 2025 revenues of $2,314 million and $173 million, respectively, together representing 103% total TTR growth compared to full year 2024. Presented new analyses from the HELIOS-B Phase 3 clinical trial of vutrisiran in patients with ATTR-CM at the American Heart Association Scientific Sessions 2025 . Cardiovascular magnetic resonance (CMR) and echocardiographic analyses demonstrated that treatment with vutrisiran resulted in significant changes indicating significant improvement on multiple functional and structural cardiac parameters. CMR imaging showed amyloid regression in 22% of vutrisiran treated patients with no regression found in patients who received placebo. Treatment with vutrisiran preserved kidney function in HELIOS-B patients, and reduced risk of death and cardiovascular events in patients with advanced chronic kidney disease. Total Rare: GIVLAARI ® (givosiran) & OXLUMO ® (lumasiran) Achieved global net product revenues for GIVLAARI and OXLUMO for the fourth quarter of $87 million and $50 million, respectively, together representing 26% total Rare growth compared to Q4 2024, and full year 2025 revenues of $308 million and $191 million, respectively, together representing $500 million in revenues and 18% total Rare growth compared to full year 2024. Other Highlights Initiated a Phase 2 clinical trial of ALN-4324 , an investigational RNAi therapeutic targeting GRB14 for type 2 diabetes mellitus. Initiated a Phase 1 clinical trial of ALN-2232 , an investigational RNAi therapeutic targeting ACVR1C in adipose tissue for obesity and weight management. Advanced two new programs ( ALN-4285 and ALN-4915 ) into clinical development in healthy volunteers. Announced the planned expansion of its state-of-the-art manufacturing facility in Norton, Massachusetts. The Company plans to invest $250 million to develop the industry's first fully dedicated, proprietary siRNA enzymatic-ligation manufacturing facility. This new enzymatic-ligation platform, siRELIS™, is expected to meaningfully expand capacity, significantly reduce production costs, and position the Company to support future launches across its growing pipeline of potential new medicines. Additional Business Updates Announced changes to the Company's Board of Directors, including the departures of Mike Bonney and Carolyn Bertozzi, Ph.D., and the appointment of Stuart Arbuckle. Key Upcoming Events The Company will host an investor webinar marking the one-year anniversary of the FDA approval of AMVUTTRA in ATTR-CM on March 24, 2026. The Company will highlight progress in delivering for ATTR-CM patients and the long-term growth and durability of its flagship TTR franchise. In the first half of 2026, Alnylam expects to: Complete enrollment in the cAPPricorn-1 Phase 2 clinical trial of mivelsiran in patients with cerebral amyloid angiopathy. Initiate a Phase 2 clinical trial of mivelsiran in patients with Alzheimer's disease. Initiate a Phase 2 clinical trial of ALN-6400 in a second bleeding disorder. In the second half of 2026, Alnylam expects to announce clinical de-risking data from several pipeline programs, including: Results from Phase 1 and Phase 2 clinical trials of ALN-6400 in healthy volunteers and patients with hereditary hemorrhagic telangiectasia (HHT), respectively. Results from a Phase 1 clinical trial of ALN-HTT02 in patients with Huntington's disease. Results from a Phase 1 clinical trial of ALN-2232 in obesity and weight management. Financial Results for the Fourth Quarter and Full Year 2025 Three Months Ended December 31, Twelve Months Ended December 31, (In thousands, except per share amounts) 2025 2024 2025 2024 Total revenues $ 1,097,033 $ 593,166 $ 3,713,937 $ 2,248,243 GAAP Income (loss) from operations $ 131,718 $ (105,159 ) $ 501,578 $ (176,885 ) Non-GAAP Income (loss) from operations $ 203,350 $ (13,514 ) $ 849,813 $ 95,199 GAAP Net income (loss) $ 111,543 $ (83,763 ) $ 313,747 $ (278,157 ) Non-GAAP Net income (loss) $ 169,753 $ 8,048 $ 683,644 $ (3,051 ) GAAP Net income (loss) per common share — basic $ 0.84 $ (0.65 ) $ 2.39 $ (2.18 ) GAAP Net income (loss) per common share — diluted $ 0.82 $ (0.65 ) $ 2.33 $ (2.18 ) Non-GAAP Net income (loss) per common share — basic $ 1.28 $ 0.06 $ 5.22 $ (0.02 ) Non-GAAP Net income (loss) per share — diluted $ 1.25 $ 0.06 $ 5.08 $ (0.02 ) For an explanation of our use of non-GAAP financial measures, refer to the “Use of Non-GAAP Financial Measures” section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measure, see the tables at the end of this press release. Revenue Summary Three Months Ended December 31, (In thousands, except percentages) 2025 2024 % Change At CER* Net product revenues: AMVUTTRA $ 826,588 $ 286,510 189 % 187 % ONPATTRO 31,687 56,103 (44 )% (45 )% Total TTR net product revenues 858,275 342,613 151 % 149 % GIVLAARI 86,796 64,645 34 % 32 % OXLUMO 49,646 43,573 14 % 10 % Total Rare net product revenues 136,442 108,218 26 % 23 % Total net product revenues 994,717 450,831 121 % 119 % Net revenues from collaborations: Roche 32,954 12,014 174 % 174 % Regeneron Pharmaceuticals 7,834 30,657 (74 )% (74 )% Novartis AG — 60,003 (100 )% (100 )% Other 155 4,274 (96 )% (96 )% Total net revenues from collaborations 40,943 106,948 (62 )% (62 )% Royalty revenue 61,373 35,387 73 % 73 % Total revenues $ 1,097,033 $ 593,166 85 % 83 % * Change at constant exchange rates, or CER, represents percent change calculated as if exchange rates had remained unchanged from those used during the three months ended December 31, 2024. CER is a non-GAAP financial measure. Twelve Months Ended December 31, (In thousands, except percentages) 2025 2024 % Change At CER* Net product revenues: AMVUTTRA $ 2,313,836 $ 970,450 138 % 137 % ONPATTRO 172,789 252,857 (32 )% (32 )% Total TTR net product revenues 2,486,625 1,223,307 103 % 102 % GIVLAARI 308,487 255,871 21 % 20 % OXLUMO 191,437 167,050 15 % 13 % Total Rare net product revenues 499,924 422,921 18 % 17 % Total net product revenues 2,986,549 1,646,228 81 % 80 % Net revenues from collaborations: Roche 394,881 119,489 230 % 230 % Regeneron Pharmaceuticals 113,957 302,798 (62 )% (62 )% Novartis AG — 79,759 (100 )% (100 )% Other 44,528 8,175 445 % 445 % Total net revenues from collaborations 553,366 510,221 8 % 8 % Royalty revenue 174,022 91,794 90 % 90 % Total revenues $ 3,713,937 $ 2,248,243 65 % 64 % * Change at constant exchange rates, or CER, represents growth calculated as if exchange rates had remained unchanged from those used during the twelve months ended December 31, 2024. CER is a non-GAAP financial measure. Total Net Product Revenues Net product revenues increased 121% and 81% at actual currency during the three and twelve months ended December 31, 2025, respectively, compared to the same periods in 2024, and 119% and 80% at CER, respectively. The increases were primarily due to growth from AMVUTTRA revenues driven by increased patient demand, mainly in patients with ATTR-CM in the U.S., which was partially offset by a decreased number of patients on ONPATTRO, and due to growth from an increased number of patients on GIVLAARI and OXLUMO. Net Revenues from Collaborations Net revenues from collaborations decreased $66 million during the three months ended December 31, 2025, as compared to the same period in 2024, primarily due to revenue recognized under our license agreement with Novartis associated with the achievement of a specified Leqvio commercialization milestone during the three months ended December 31, 2024. Net revenues from collaborations increased by $43 million during the twelve months ended December 31, 2025, as compared to the same period in 2024, primarily driven by recognition of $300 million of milestone revenue under our collaboration with Roche in September 2025 associated with the dosing of the first patient in the ZENITH Phase 3 clinical trial of zilebesiran and recognition of a $30 million payment in connection with the amendment to our agreement with Vir Biotechnology in March 2025. In comparison, during 2024, we recognized $185 million in revenues under our collaboration with Regeneron as we modified the collaboration in June 2024 and provided Regeneron with an exclusive license to develop, manufacture and commercialize cemdisiran as a monotherapy, and also recognized $65 million of milestone revenue under our collaboration with Roche in March 2024 associated with the dosing of the first patient in the KARDIA-3 Phase 2 clinical trial of zilebesiran. Royalty Revenue Royalty revenue increased during the three and twelve months ended December 31, 2025, as compared to the same periods in 2024, due to increased volume and rate of royalties earned from global net sales of Leqvio by Novartis. Operating Expense Summary Three Months Ended December 31, Twelve Months Ended December 31, (In thousands, except percentages) 2025 2024 % Change 2025 2024 % Change Cost of goods sold $ 267,723 $ 102,649 161 % $ 677,166 $ 306,513 121 % % of net product revenues 26.9 % 22.8 % 22.7 % 18.6 % Cost of collaborations and royalties $ — $ 168 (100 )% $ 4,705 $ 16,857 (72 )% GAAP Research and development expenses $ 372,218 $ 300,169 24 % $ 1,319,775 $ 1,126,232 17 % Non-GAAP Research and development expenses $ 340,898 $ 259,544 31 % $ 1,166,380 $ 998,483 17 % GAAP Selling, general and administrative expenses $ 325,374 $ 295,339 10 % $ 1,210,713 $ 975,526 24 % Non-GAAP Selling, general and administrative expenses $ 285,062 $ 244,319 17 % $ 1,015,873 $ 831,191 22 % Cost of Goods Sold Cost of goods sold as a percentage of net product revenues increased during the three and twelve months ended December 31, 2025, as compared to the same periods in 2024, primarily as a result of increased sales of AMVUTTRA and an associated increase in the blended royalty rate payable on net sales of AMVUTTRA. Research & Development (R&D) Expenses GAAP and non-GAAP R&D expenses increased during the three and twelve months ended December 31, 2025, as compared to the same periods in 2024, primarily due to increased clinical trial expenses for the ZENITH Phase 3 clinical trial of zilebesiran, the TRITON-CM Phase 3 clinical trial of nucresiran in patients with ATTR-CM and the TRITON-PN Phase 3 clinical trial of nucresiran in patients with hATTR-PN, as well as increased employee compensation and related expenses to support our research and development pipeline and development expenses. Additionally, GAAP R&D expenses increased due to higher stock-based compensation expenses. Selling, General & Administrative (SG&A) Expenses GAAP and non-GAAP SG&A expenses increased during the three and twelve months ended December 31, 2025, as compared to the same periods in 2024, primarily due to higher employee compensation costs, including stock-based compensation, mainly driven by higher headcount, and increased marketing investment associated with the commercial launch of AMVUTTRA in ATTR-CM. Other Financial Highlights Interest expense Interest expense for the three and twelve months ended December 31, 2025 of $65 million and $253 million, respectively, included interest of $38 million and $150 million, respectively, attributed to the liability related to the sale of future Leqvio royalties, and $25 million and $89 million, respectively, attributed to the liabilities related to the vutrisiran and zilebesiran development funding. Benefit from (provision for) income taxes During the three months year ended December 31, 2025, we recorded a benefit from income taxes of $25 million primarily related to the generation of Switzerland deferred tax assets, partially offset by additional U.S state income tax. During the twelve months ended December 31, 2025, we recorded a provision for income taxes of $9 million primarily related to U.S state income taxes, utilization of Switzerland net deferred tax assets, as well as taxable income from jurisdictions in which we are subject to tax. We will continue to utilize deferred tax assets in Switzerland to offset current cash tax liabilities and will continue to monitor the requirement for a valuation allowance against our net deferred tax assets in the U.S. and certain deferred tax assets in Switzerland. Financial position Cash, cash equivalents and marketable securities were $2.91 billion as of December 31, 2025, as compared to $2.69 billion as of December 31, 2024, with the increase primarily due to improved operating performance, proceeds from exercise of stock options, and net proceeds from the issuance of our 0.00% convertible senior notes due 2028, offset in part by cash paid for the partial repurchases of our 1.00% convertible senior notes due 2027. Net cash provided by operating activities for the three and twelve months ended December 31, 2025 included $23 million and $118 million, respectively, of payments associated with the liability related to the sale of future Leqvio royalties recorded to interest expense, as well as $30 million and $85 million, respectively, of payments associated with the liabilities related to vutrisiran and zilebesiran development funding recorded to interest expense. A reconciliation of our GAAP to non-GAAP results for the three and twelve months ended December 31, 2025 and 2024, is included in the tables at the end of this press release. 2026 Financial Guidance Full-year 2026 financial guidance is summarized below: Total TTR net product revenues (AMVUTTRA, ONPATTRO) 1 $4,400 million - $4,700 million Total Rare net product revenues (GIVLAARI, OXLUMO) 1 $500 million - $600 million Total net product revenues 1 $4,900 million - $5,300 million Net product revenues growth vs. 2025 at currency exchange rates as of December 31, 2025 2 64% - 77% Net product revenues growth vs. 2025 at constant exchange rates 2 64% - 77% Net revenues from collaborations and royalties $400 million - $500 million Non-GAAP R&D and SG&A expenses 3 $2,700 million - $2,800 million 1 Full-year 2026 guidance utilizing currency exchange rates as of December 31, 2025: 1 EUR = 1.17 USD and 1 USD = 157 JPY 2 Representing growth calculated as if the exchange rates had remained unchanged from those used in 2025, which is a non-GAAP financial measure 3 Excludes $300 million - $400 million of stock-based compensation expense from estimated GAAP R&D and SG&A expenses Use of Non-GAAP Financial Measures This press release contains non-GAAP financial measures, including expenses adjusted to exclude certain non-cash expenses and non-recurring gains or losses outside the ordinary course of the Company’s business. These measures are not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies. The items included in GAAP presentations but excluded for purposes of determining non-GAAP financial measures for the periods presented in this press release are stock-based compensation expenses, loss related to convertible debt, and realized and unrealized gains or losses on marketable equity securities. The Company has excluded the impact of stock-based compensation expense, which may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards. The Company has excluded the loss related to convertible debt because the Company believes the item is a non-recurring transaction outside the ordinary course of the Company’s business. The Company has excluded the impact of the realized and unrealized gains or losses on marketable equity securities because the Company does not believe these adjustments accurately reflect the performance of the Company’s ongoing operations for the period in which such gains or losses are reported, as their sole purpose is to adjust amounts on the balance sheet. Percentage changes in revenue growth at CER are presented excluding the impact of changes in foreign currency exchange rates for investors to understand the underlying business performance. The current period’s foreign currency revenue values are converted into U.S. dollars using the average exchange rates from the prior period. The Company believes the presentation of non-GAAP financial measures provides useful information to management and investors regarding the Company’s financial condition and results of operations. When GAAP financial measures are viewed in conjunction with non-GAAP financial measures, investors are provided with a more meaningful understanding of the Company’s ongoing operating performance and are better able to compare the Company’s performance between periods. In addition, these non-GAAP financial measures are among those indicators the Company uses as a basis for evaluating performance, allocating resources and planning and forecasting future periods. Non-GAAP financial measures are not intended to be considered in isolation or as a substitute for GAAP financial measures. A reconciliation between GAAP and non-GAAP measures is provided later in this press release. Conference Call Information Management will provide an update on the Company and discuss fourth quarter and full year 2025 results as well as expectations for the future via conference call on Thursday, February 12, 2026 at 8:30 am ET. A live audio webcast of the call will be available on the Investors section of the Company’s website at . An archived webcast will be available on the Alnylam website approximately two hours after the event. About AMVUTTRA ® (vutrisiran) AMVUTTRA ® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection by a healthcare professional, AMVUTTRA is approved and marketed for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. For more information about AMVUTTRA, including the full U.S. Prescribing Information , visit AMVUTTRA.com . About ONPATTRO ® (patisiran) ONPATTRO is an RNAi therapeutic that is approved in the United States and Canada for the treatment of adults with hATTR amyloidosis with polyneuropathy. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby reducing the production of TTR protein before it is made. Reducing the pathogenic protein leads to a reduction in amyloid deposits in tissues. For more information about ONPATTRO, including full Prescribing Information , visit ONPATTRO.com . About GIVLAARI ® (givosiran) GIVLAARI (givosiran) is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved in the United States and Brazil for the treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is also approved in the European Union for the treatment of AHP in adults and adolescents aged 12 years and older. In the pivotal trial, GIVLAARI was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam’s first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare professional. GIVLAARI works by specifically reducing elevated levels of ALAS1 messenger RNA (mRNA), leading to reduction of toxins associated with attacks and other disease manifestations of AHP. For more information about GIVLAARI, including the full U.S. Prescribing Information , visit GIVLAARI.com . About OXLUMO ® (lumasiran) OXLUMO (lumasiran) is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1). HAO1 encodes glycolate oxidase (GO). Thus, by silencing HAO1 and depleting the GO enzyme, OXLUMO inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. OXLUMO utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. Contacts Alnylam Pharmaceuticals, Inc. Christine Akinc (Investors and Media) 617-682-4340 Josh Brodsky (Investors) 617-551-8276 Read full story here

Phase 1Phase 2Drug ApprovalFinancial StatementPhase 3

30 Jan 2026

·www.globenewswire.com

Regeneron Reports Fourth Quarter and Full Year 2025 Financial and Operating Results

Fourth quarter 2025 revenues increased 3% to $3.9 billion versus fourth quarter 2024; full year 2025 revenues increased 1% to $14.3 billion versus 2024Fourth quarter 2025 Dupixent® global net sales (recorded by Sanofi) increased 34% to $4.9 billion versus fourth quarter 2024; full year 2025 Dupixent global net sales increased 26% to $17.8 billion versus 2024Fourth quarter 2025 EYLEA HD® U.S. net sales increased 66% to $506 million and total EYLEA HD and EYLEA® U.S. net sales decreased 28% to $1.1 billion; full year 2025 EYLEA HD U.S. net sales increased 36% to $1.6 billion and total EYLEA HD and EYLEA U.S. net sales decreased 27% to $4.4 billionFourth quarter 2025 GAAP EPS of $7.86 and non-GAAP EPS(a) of $11.44; fourth quarter 2025 includes unfavorable $0.14 impact from acquired IPR&D chargeEYLEA HD approved by FDA for treatment of macular edema following retinal vein occlusion (RVO) and for monthly dosing flexibility across approved indicationsFDA approved new manufacturer to fill vials for EYLEA HD; regulatory application to include new manufacturer for EYLEA HD pre-filled syringe submitted to FDA, with decision expected in second quarter 2026Libtayo® approved by FDA and EC as first and only immunotherapy for high-risk adjuvant cutaneous squamous cell carcinoma (CSCC)Dupixent approved by EC for chronic spontaneous urticaria (CSU) TARRYTOWN, N.Y., Jan. 30, 2026 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced financial results for the fourth quarter and full year 2025 and provided a business update. "Regeneron performed well in 2025, with financial strength driven by our four blockbuster medicines and future growth supported by our exciting late-stage clinical portfolio," said Leonard S. Schleifer, M.D., Ph.D., Board co-Chair, President and Chief Executive Officer of Regeneron. "In the fourth quarter, we secured label expansions and new filler solutions for EYLEA HD, further enhancing its commercial potential. Dupixent received new approvals in Japan and Europe and is currently the most widely used innovative branded antibody medicine, with over 1.4 million active patients worldwide. Libtayo also secured additional approvals and continues to be the leading immunotherapy for non-melanoma skin cancers. Our current success is underscored by decades of investment in innovative science and technology, setting us up for exciting data read-outs and potential approvals in 2026 in a broad range of diseases." Financial Highlights ($ in millions, except per share data) Q4 2025 Q4 2024 % Change FY 2025 FY 2024 % ChangeTotal revenues $3,884 $3,789 3% $14,343 $14,202 1%GAAP net income $845 $918 (8%) $4,505 $4,413 2%GAAP net income per share - diluted $7.86 $8.06 (2%) $41.48 $38.34 8%Non-GAAP net income(a) $1,249 $1,390 (10%) $4,888 $5,319 (8%)Non-GAAP net income per share - diluted(a) $11.44 $12.07 (5%) $44.31 $45.62 (3%) "2025 was another strong year for Regeneron, marked by notable pipeline advances, remarkable commercial execution, and solid financial performance," said Christopher Fenimore, Executive Vice President, Finance and Chief Financial Officer of Regeneron. "As we look ahead to 2026, our focus remains on prioritizing internal investments, evaluating complementary business development opportunities, and enhancing shareholder returns through share repurchases and dividends, positioning the Company to deliver sustainable growth and long-term value to shareholders." Business Highlights Key Pipeline ProgressRegeneron has approximately 45 product candidates in clinical development, including a number of marketed products for which it is investigating additional indications. Updates from the clinical pipeline include: Dupixent (dupilumab) In November 2025, the European Commission (EC) approved Dupixent for the treatment of CSU in adults and adolescents aged 12 years and older who remain symptomatic despite antihistamine treatment. Dupixent is approved for CSU in certain adults and adolescents in several countries, including the United States and Japan. Additionally, regulatory applications are under review for CSU in children aged 2 to 11 years in the United States, European Union (EU), and Japan.In December 2025, the Ministry of Health, Labour and Welfare (MHLW) in Japan approved Dupixent for the treatment of bronchial asthma in children aged 6 to 11 years with severe or refractory disease whose symptoms are inadequately controlled with existing therapy. This expands the previous approval of this indication in Japan for patients aged 12 years and older.In November 2025, the Company and Sanofi announced positive results from the Phase 3 trial in adults and children aged 6 years and older with allergic fungal rhinosinusitis (AFRS). Additionally, the U.S. Food and Drug Administration (FDA) accepted for priority review the supplemental Biologics License Application (sBLA) for this indication, which has a target action date in February 2026. EYLEA HD (aflibercept) 8 mg In November 2025, the FDA approved EYLEA HD for the treatment of patients with RVO with up to every 8-week dosing after an initial monthly dosing period. The FDA also approved an every 4-week (monthly) dosing option across approved indications: wet age-related macular degeneration (wAMD), diabetic macular edema (DME), diabetic retinopathy (DR), and RVO.In January 2026, the EC approved EYLEA 8 mg (known as EYLEA HD in the United States) for the treatment of RVO.In December 2025, the FDA approved the addition of a new manufacturer to fill vials for EYLEA HD.In December 2025, the Company submitted a regulatory application to the FDA to include a new manufacturer to fill pre-filled syringes for EYLEA HD. An FDA decision is expected in the second quarter of 2026. Libtayo (cemiplimab) In October and November of 2025, the FDA and EC, respectively, approved Libtayo as an adjuvant treatment for adults with CSCC at high risk of recurrence after surgery and radiation, making Libtayo the first and only immunotherapy approved in this setting. This expands Libtayo's approved indications in CSCC beyond the metastatic or locally advanced setting. Additionally, in December 2025, a regulatory application was submitted in Japan for the adjuvant treatment of CSCC. Other Programs In December 2025, the Company submitted a BLA to the FDA for DB-OTO (an AAV-based gene therapy) for the treatment of profound genetic hearing loss in children due to variants of the otoferlin (OTOF) gene. An FDA decision is expected in the first half of 2026.In December 2025, the Company submitted U.S. and EU regulatory applications for garetosmab (an Activin A antibody) in adults with fibrodysplasia ossificans progressiva (FOP).A second Phase 3 study was initiated for REGN5713-5715, an investigational treatment for birch allergy. Corporate Updates In October 2025, Dupixent was recognized as the “Best Biotechnology Product” of 2025 by the Galien Foundation, which acknowledges extraordinary scientific innovations that improve the human condition.In January 2026, the Company's collaboration agreement with Tessera Therapeutics, Inc. to develop and commercialize TSRA-196 (Tessera's investigational program for the treatment of alpha-1 antitrypsin deficiency (AATD)) became effective. Tessera will lead the initial first-in-human trial, while Regeneron will lead subsequent global development and commercialization.In addition to the previously announced ongoing and planned domestic investments totaling more than $7 billion, the Company plans to invest approximately $2 billion to develop a state-of-the-art bulk manufacturing facility in Saratoga Springs, New York, expected to create 1,000 high-paying jobs and to significantly expand manufacturing capacity. Select Upcoming 2026 Milestones Programs MilestonesOphthalmology - FDA decision for EYLEA HD pre-filled syringe (second quarter 2026) - Report initial results from lead-in cohort of Phase 3 study for cemdisiran (C5 siRNA therapy) as monotherapy and in combination with pozelimab (C5 antibody) in geographic atrophy (second half 2026)Immunology & Inflammation - EC decision on regulatory submission for Dupixent in bullous pemphigoid (first half 2026); FDA decision on sBLA for Dupixent in AFRS (February 2026) - Initiate long-acting IL-13 antibody clinical program in atopic dermatitis (first half 2026) - Initiate second Phase 3 study for REGN1908-1909 (Fel d 1 multi-antibody) in cat allergy (first half 2026)Cardiovascular & Metabolic Diseases - Initiate Phase 3 program for olatorepatide (GLP-1/GIP receptor agonist) in obesity in patients with and without Type 2 diabetes (2026) - Initiate clinical program for olatorepatide in combination with Praluent® (alirocumab) (2026) - Report additional data from Phase 2 study for semaglutide in combination with trevogrumab (myostatin antibody) with and without garetosmab (Activin A antibody) in obesity (2026)Hematology - Initiate additional Phase 3 studies for Factor XI antibodies (REGN7508 and REGN9933) in anticoagulation (first half 2026) - Report results from Phase 3 study for pozelimab (C5 antibody) in combination with cemdisiran (C5 siRNA therapy) in paroxysmal nocturnal hemoglobinuria (PNH) (fourth quarter 2026/first quarter 2027)Oncology & Hematology-Oncology - Report results from Phase 3 study for fianlimab (LAG-3 antibody), in combination with Libtayo, versus pembrolizumab in first-line metastatic melanoma (first half 2026) - Initiate additional Phase 3 studies for Lynozyfic™ (linvoseltamab) in multiple myeloma and precursor conditions (2026)Neurology & Rare Diseases - FDA decision on BLA for DB-OTO (AAV-based gene therapy) in hearing deficit due to variants of the otoferlin gene (first half 2026) - FDA decision on BLA and EC decision on MAA for garetosmab (Activin A antibody) in FOP (second half 2026) - Submit New Drug Application (NDA) for cemdisiran (C5 siRNA therapy) in myasthenia gravis (first quarter 2026) and FDA decision on NDA (fourth quarter 2026/first quarter 2027) Fourth Quarter and Full Year 2025 Financial Results Revenues ($ in millions) Q4 2025 Q4 2024 % Change FY 2025 FY 2024 % ChangeNet product sales: EYLEA HD - U.S. $506 $305 66% $1,637 $1,201 36%EYLEA - U.S. 577 1,190 (52%) 2,748 4,767 (42%)Total EYLEA HD and EYLEA - U.S. 1,083 1,495 (28%) 4,385 5,968 (27%)Libtayo - U.S. 285 251 14% 945 787 20%Libtayo - ROW* 140 116 21% 508 430 18%Total Libtayo - Global 425 367 16% 1,453 1,217 19%Praluent - U.S. 73 63 16% 262 242 8%Evkeeza®- U.S. 48 38 26% 162 126 29%Inmazeb®- U.S. 37 40 (8%) 37 76 (51%)Other products - Global 9 — ** 10 — ** Total net product sales 1,675 2,003 (16%) 6,309 7,629 (17%) Collaboration revenue: Sanofi 1,640 1,213 35% 5,884 4,531 30%Bayer 319 377 (15%) 1,422 1,499 (5%)Other 12 17 (29%) 25 28 (11%)Other revenue 238 179 33% 703 515 37%Total revenues $3,884 $3,789 3% $14,343 $14,202 1% * Rest of world (ROW)** Percentage not meaningful Net product sales of EYLEA HD increased in the fourth quarter and full year 2025, compared to the same periods of 2024, due to higher sales volumes driven by increased demand, partly offset by a lower net selling price. Net product sales of EYLEA in the fourth quarter and full year 2025, compared to the same periods of 2024, were negatively impacted by (i) lower sales volumes as a result of continued competitive pressures, loss in market share to compounded bevacizumab due to patient affordability constraints, and the continued transition of patients to EYLEA HD, and (ii) a lower net selling price. In addition, as previously reported, EYLEA HD and EYLEA net product sales in the fourth quarter of 2025 were each favorably impacted by approximately $30 million due to higher wholesaler inventory levels at the end of the fourth quarter of 2025 compared to the end of the third quarter of 2025. Sanofi collaboration revenue increased in the fourth quarter and full year 2025, compared to the same periods of 2024, due to an increase in the Company's share of profits from the commercialization of antibodies, which were $1.486 billion and $1.043 billion in the fourth quarter of 2025 and 2024, respectively, and $5.242 billion and $3.924 billion for full year 2025 and 2024, respectively. The change in the Company's share of profits from commercialization of antibodies was driven by higher profits primarily associated with an increase in Dupixent sales. Refer to Table 4 for a summary of collaboration revenue. Other revenue increased in the fourth quarter and full year 2025, compared to the same periods of 2024, primarily due to royalties earned on sales of Ilaris® and share of profits from sales of ARCALYST®, which were $179 million and $112 million in the aggregate for the fourth quarter of 2025 and 2024, respectively, and $506 million and $293 million for full year 2025 and 2024, respectively. Operating Expenses GAAP % Change Non-GAAP(a) % Change($ in millions) Q4 2025 Q4 2024 Q4 2025 Q4 2024 Research and development (R&D) $1,626 $1,412 15% $1,331 $1,224 9%Acquired in-process research and development (IPR&D) $19 $14 36% * * n/a Selling, general, and administrative (SG&A) $775 $792 (2%) $691 $681 1%Cost of goods sold (COGS) $319 $327 (2%) $257 $271 (5%)Gross margin on net product sales(b) 81% 84% 85% 86% Cost of collaboration and contract manufacturing (COCM)(c) $266 $239 11% * * n/a Other operating (income) expense, net $— $16 (100%) * $— ** GAAP % Change Non-GAAP(a) % Change FY 2025 FY 2024 FY 2025 FY 2024 Research and development $5,850 $5,132 14% $5,150 $4,563 13%Acquired in-process research and development $124 $101 23% * * n/a Selling, general, and administrative $2,700 $2,954 (9%) $2,311 $2,544 (9%)Cost of goods sold $1,141 $1,087 5% $924 $898 3%Gross margin on net product sales(b) 82% 86% 85% 88% Cost of collaboration and contract manufacturing(c) $960 $883 9% * * n/a Other operating (income) expense, net $(10) $53 ** * $— ** * GAAP and non-GAAP amounts are equivalent as no non-GAAP adjustments have been recorded** Percentage not meaningful GAAP and non-GAAP R&D expenses increased for full year 2025, compared to the same period in the prior year, driven by the advancement of the Company's late-stage clinical pipeline. GAAP R&D expenses for the fourth quarter and full year 2025 included $155 million related to a previously purchased FDA Rare Pediatric Disease Priority Review Voucher (PRV), which the Company decided in the fourth quarter of 2025 to utilize for a regulatory submission.Acquired IPR&D expenses for full year 2025 included an $80 million up-front payment in connection with the Company's license agreement with Hansoh Pharmaceuticals Group Company Limited. Acquired IPR&D expenses for full year 2024 included a $45 million development milestone in connection with the Company's collaboration agreement with Sonoma Biotherapeutics, Inc.GAAP and non-GAAP SG&A expenses decreased for full year 2025, compared to the same period in the prior year, primarily due to lower charitable contributions to Good Days, an independent non-profit patient assistance organization. GAAP and non-GAAP SG&A expenses in the fourth quarter 2025 included approximately $60 million for matching donations made to Good Days; in July 2025, the Company launched a matching program for donations made to Good Days and committed to matching donations quarterly through the end of 2025. The Company recently committed to matching donations for up to a total of $200 million during 2026.GAAP and non-GAAP gross margin on net product sales decreased in the fourth quarter and full year 2025, compared to the same periods in the prior year, partly due to ongoing investments to support the Company's manufacturing operations and higher inventory write-offs and reserves. In addition, GAAP gross margin on net product sales decreased due to higher amortization expense associated with the Company's Libtayo intangible asset. Other Financial Information GAAP other income (expense), net included the recognition of net losses on marketable and other securities of $22 million in the fourth quarter of 2025, compared to net losses of $213 million in the fourth quarter of 2024. GAAP other income (expense), net included the recognition of net gains on marketable and other securities of $946 million for full year 2025, compared to net gains of $118 million for full year 2024. In the fourth quarter and full year 2025, the Company's GAAP effective tax rate (ETR) was 19.1% and 13.9%, respectively, compared to 4.2% and 7.7% in the fourth quarter and full year 2024, respectively. The GAAP ETR increased in the fourth quarter and full year 2025, compared to the same periods in the prior year, primarily due to lower tax benefits from less stock option exercises and the shortfall related to stock-based compensation. A shortfall occurs when the actual tax deduction a company recognizes from stock-based compensation is less than the deferred tax asset that was previously recorded for financial reporting purposes, resulting in the write-off of the deferred tax asset (and recognition of income tax expense). The GAAP ETR for the fourth quarter of 2025 was also negatively impacted by a lower benefit from income earned in foreign jurisdictions with tax rates lower than the U.S. federal statutory rate. The GAAP ETR for full year 2025 was positively impacted by the release of liabilities for uncertain tax positions recognized upon the settlement of an IRS audit. In the fourth quarter and full year 2025, the non-GAAP ETR was 17.1% and 12.6%, respectively, compared to 9.9% and 9.6% in the fourth quarter and full year 2024, respectively. A reconciliation of the Company's GAAP to non-GAAP results is included in Table 3 of this press release. Capital Allocation During the fourth quarter and full year 2025, the Company repurchased shares of its common stock and recorded the cost of the shares, or $671 million and $3.5 billion, respectively, as Treasury Stock. As of December 31, 2025, $1.5 billion remained available for share repurchases under the Company's share repurchase programs. In January 2026, the Company's board of directors declared a cash dividend of $0.94 per share on the Company's common stock and Class A stock, payable on March 5, 2026 to shareholders of record as of February 20, 2026. 2026 Financial Guidance* The Company's full year 2026 financial guidance consists of the following components: 2026 GuidanceGAAP R&D$6.450–$6.680 billionNon-GAAP R&D(a)$5.900–$6.100 billionGAAP SG&A$2.860–$3.040 billionNon-GAAP SG&A(a)$2.500–$2.650 billionGAAP gross margin on net product sales79%–80%Non-GAAP gross margin on net product sales(a)83%–84%COCM**$940 million–$1.020 billionCapital expenditures**$1.100–$1.300 billionGAAP effective tax rate12%–14%Non-GAAP effective tax rate(a)13%–15% * The Company's 2026 financial guidance does not assume the completion of any business development transactions not completed as of the date of this press release** GAAP and non-GAAP amounts are equivalent as no non-GAAP adjustments are expected to be recorded A reconciliation of full year 2026 GAAP to non-GAAP financial guidance is included below: Projected Range($ in millions) Low HighGAAP R&D $6,450 $6,680 Stock-based compensation expense 550 580 Non-GAAP R&D(a) $5,900 $6,100 GAAP SG&A $2,860 $3,040 Stock-based compensation expense 360 390 Non-GAAP SG&A(a) $2,500 $2,650 GAAP gross margin on net product sales 79% 80% Intangible asset amortization expense 3% 3% Stock-based compensation expense 1% 1% Non-GAAP gross margin on net product sales(a) 83% 84% GAAP ETR 12% 14% Income tax effect of GAAP to non-GAAP reconciling items 1% 1% Non-GAAP ETR(a) 13% 15% (a)This press release uses non-GAAP R&D, non-GAAP SG&A, non-GAAP COGS, non-GAAP gross margin on net product sales, non-GAAP other operating (income) expense, net, non-GAAP other income (expense), net, non-GAAP ETR, non-GAAP net income, non-GAAP net income per share, and free cash flow, which are financial measures that are not calculated in accordance with U.S. Generally Accepted Accounting Principles (GAAP). These non-GAAP financial measures are computed by excluding certain non-cash and/or other items from the related GAAP financial measure. The Company also includes a non-GAAP adjustment for the estimated income tax effect of reconciling items. A reconciliation of the Company's GAAP to non-GAAP results is included in Table 3 of this press release.The Company makes such adjustments for items the Company does not view as useful in evaluating its operating performance. For example, adjustments may be made for items that fluctuate from period to period based on factors that are not within the Company's control (such as the Company's stock price on the dates share-based grants are issued or changes in the fair value of the Company's investments in equity securities) or items that are not associated with normal, recurring operations (such as acquisition and integration costs). Management uses these non-GAAP measures for planning, budgeting, forecasting, assessing historical performance, and making financial and operational decisions, and also provides forecasts to investors on this basis. With respect to free cash flow, the Company believes that this non-GAAP measure provides a further measure of the Company's ability to generate cash flows from its operations. Additionally, the non-GAAP measures presented are intended to provide investors with an enhanced understanding of the financial performance of the Company's core business operations. However, there are limitations in the use of these and other non-GAAP financial measures as they exclude certain expenses that are recurring in nature. Furthermore, the Company's non-GAAP financial measures may not be comparable with non-GAAP information provided by other companies. Any non-GAAP financial measure presented by the Company should be considered supplemental to, and not a substitute for, measures of financial performance prepared in accordance with GAAP. (b)Gross margin on net product sales represents gross profit expressed as a percentage of total net product sales recorded by the Company. Gross profit is calculated as net product sales less cost of goods sold. (c)Corresponding reimbursements from collaborators and others for manufacturing product is recorded within revenues. Conference Call Information Regeneron will host a conference call and simultaneous webcast to discuss its fourth quarter and full year 2025 financial and operating results on Friday, January 30, 2026, at 8:30 AM Eastern Time. Participants may access the conference call live via webcast, or register in advance and participate via telephone, on the "Investors and Media" page of Regeneron's website at www.regeneron.com. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. A replay of the conference call and webcast will be archived on the Company's website for at least 30 days. About Regeneron Regeneron is a leading biotechnology company that invents, develops, and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, Regeneron's unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in Regeneron's laboratories. Regeneron's medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases. Regeneron pushes the boundaries of scientific discovery and accelerates drug development using its proprietary technologies, such as VelociSuite®, which produces optimized fully human antibodies and new classes of bispecific antibodies. Regeneron is shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center® and pioneering genetic medicine platforms, enabling Regeneron to identify innovative targets and complementary approaches to potentially treat or cure diseases. For more information, please visit www.regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook, or X. Forward-Looking Statements and Use of Digital Media This press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. ("Regeneron" or the "Company"), and actual events or results may differ materially from these forward-looking statements. Words such as "anticipate," "expect," "intend," "plan," "believe," "seek," "estimate," variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. These statements concern, and these risks and uncertainties include, among others, competing products and product candidates (including biosimilar products) that may be superior to, or more cost effective than, products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, "Regeneron's Products") and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, "Regeneron's Product Candidates"); uncertainty of the utilization, market acceptance, and commercial success of Regeneron's Products and Regeneron's Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary) or recommendations and guidelines from governmental authorities and other third parties or other factors beyond Regeneron's control on the commercial success of Regeneron's Products and Regeneron's Product Candidates; the nature, timing, and possible success and therapeutic applications of Regeneron's Products and Regeneron's Product Candidates and research and clinical programs now underway or planned, including without limitation EYLEA HD® (aflibercept) Injection 8 mg, EYLEA® (aflibercept) Injection, Dupixent® (dupilumab), Libtayo® (cemiplimab), Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab), Veopoz® (pozelimab), Ordspono™ (odronextamab), Lynozyfic™ (linvoseltamab), other clinical programs discussed in this press release, Regeneron's and its collaborators' earlier-stage programs, and the use of human genetics in Regeneron's research programs; the likelihood and timing of achieving any of the anticipated milestones described in this press release; safety issues resulting from the administration of Regeneron's Products and Regeneron's Product Candidates in patients, including serious complications or side effects in connection with the use of Regeneron’s Products and Regeneron's Product Candidates in clinical trials; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron's Product Candidates and new indications for Regeneron's Products, including those listed above and/or otherwise discussed in this press release; the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; ongoing regulatory obligations and oversight impacting Regeneron's Products, research and clinical programs, and business, including those relating to patient privacy; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron's ability to continue to develop or commercialize Regeneron's Products and Regeneron's Product Candidates; the ability of Regeneron to manufacture and manage supply chains for multiple products and product candidates and risks associated with tariffs and other trade restrictions; the ability of Regeneron’s collaborators, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron’s Products and Regeneron's Product Candidates; the availability and extent of reimbursement or copay assistance for Regeneron’s Products from third-party payors and other third parties, including private payor healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payors and other third parties and new policies and procedures adopted by such payors and other third parties; changes to drug pricing regulations and requirements and Regeneron's drug pricing strategy; other changes in laws, regulations, and policies affecting the healthcare industry; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance, including GAAP and non-GAAP R&D, GAAP and non-GAAP SG&A, GAAP and non-GAAP gross margin on net product sales, COCM, capital expenditures, and GAAP and non-GAAP ETR; the potential for any license or collaboration agreement, including Regeneron's agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable), to be cancelled or terminated; the impact of public health outbreaks, epidemics, or pandemics on Regeneron's business; and risks associated with litigation and other proceedings and government investigations relating to the Company and/or its operations (including the pending civil proceedings initiated or joined by the U.S. Department of Justice and the U.S. Attorney's Office for the District of Massachusetts), risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA), the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron’s business, prospects, operating results, and financial condition. A more complete description of these and other material risks can be found in Regeneron's filings with the U.S. Securities and Exchange Commission. Any forward-looking statements are made based on management's current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron. Regeneron does not undertake any obligation to update (publicly or otherwise) any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise. Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron's media and investor relations website (https://investor.regeneron.com) and its LinkedIn page (https://www.linkedin.com/company/regeneron-pharmaceuticals). Non-GAAP Financial Measures This press release and/or the financial results attached to this press release include amounts that are considered "non-GAAP financial measures" under SEC rules. As required, Regeneron has provided reconciliations of such non-GAAP financial measures. Contact Information: Ryan Crowe Christina ChanInvestor Relations Corporate Affairs914-847-8790 914-847-8827ryan.crowe@regeneron.com christina.chan@regeneron.com TABLE 1 REGENERON PHARMACEUTICALS, INC.CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited)(In millions) December 31, 2025 2024 Assets: Cash and marketable securities $18,865.8 $17,912.6 Accounts receivable, net 5,741.1 6,211.9 Inventories 3,200.8 3,087.3 Property, plant, and equipment, net 5,120.4 4,599.7 Intangible assets, net 1,257.4 1,148.6 Deferred tax assets 4,077.2 3,314.1 Other assets 2,296.0 1,485.2 Total assets $40,558.7 $37,759.4 Liabilities and stockholders' equity: Accounts payable, accrued expenses, and other liabilities $5,834.2 $4,888.0 Finance lease liabilities 720.0 720.0 Deferred revenue 761.7 813.4 Long-term debt 1,985.9 1,984.4 Stockholders' equity 31,256.9 29,353.6 Total liabilities and stockholders' equity $40,558.7 $37,759.4 TABLE 2 REGENERON PHARMACEUTICALS, INC.CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited)(In millions, except per share data) Three Months EndedDecember 31, Year EndedDecember 31, 2025 2024 2025 2024 Revenues: Net product sales $1,674.8 $2,002.9 $6,309.1 $7,629.2 Collaboration revenue 1,970.9 1,606.9 7,331.2 6,057.8 Other revenue 238.6 179.4 702.6 515.0 3,884.3 3,789.2 14,342.9 14,202.0 Expenses: Research and development 1,626.1 1,412.1 5,850.2 5,132.0 Acquired in-process research and development 18.7 13.8 124.1 101.0 Selling, general, and administrative 775.0 792.2 2,700.0 2,954.4 Cost of goods sold 318.7 326.8 1,140.8 1,087.3 Cost of collaboration and contract manufacturing 265.9 238.6 959.9 883.2 Other operating (income) expense, net — 15.5 (10.0) 53.4 3,004.4 2,799.0 10,765.0 10,211.3 Income from operations 879.9 990.2 3,577.9 3,990.7 Other income (expense): Other income (expense), net 176.0 (21.6) 1,696.6 844.4 Interest expense (12.2) (10.5) (43.8) (55.2) 163.8 (32.1) 1,652.8 789.2 Income before income taxes 1,043.7 958.1 5,230.7 4,779.9 Income tax expense 199.1 40.4 725.8 367.3 Net income $844.6 $917.7 $4,504.9 $4,412.6 Net income per share - basic $8.21 $8.53 $43.07 $40.90 Net income per share - diluted $7.86 $8.06 $41.48 $38.34 Weighted average shares outstanding - basic 102.9 107.6 104.6 107.9 Weighted average shares outstanding - diluted 107.5 113.8 108.6 115.1 TABLE 3 REGENERON PHARMACEUTICALS, INC.RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL INFORMATION (Unaudited)(In millions, except per share data) Three Months EndedDecember 31, Year EndedDecember 31, 2025 2024 2025 2024 GAAP R&D $1,626.1 $1,412.1 $5,850.2 $5,132.0 Stock-based compensation expense 140.3 174.7 545.4 543.8 Acquisition and integration costs — 13.8 — 24.9 Priority review voucher 155.0 — 155.0 — Non-GAAP R&D $1,330.8 $1,223.6 $5,149.8 $4,563.3 GAAP SG&A $775.0 $792.2 $2,700.0 $2,954.4 Stock-based compensation expense 83.9 103.1 362.9 355.0 Acquisition and integration costs — 5.5 0.8 42.2 Litigation settlements — 3.0 25.0 13.0 Non-GAAP SG&A $691.1 $680.6 $2,311.3 $2,544.2 GAAP COGS $318.7 $326.8 $1,140.8 $1,087.3 Stock-based compensation expense 25.1 26.6 85.4 84.0 Acquisition and integration costs — 0.3 — 2.0 Intangible asset amortization expense 36.9 29.1 131.7 103.5 Non-GAAP COGS $256.7 $270.8 $923.7 $897.8 GAAP other operating (income) expense, net $— $15.5 $(10.0) $53.4 Change in fair value of contingent consideration — 15.5 — 53.4 Non-GAAP other operating (income) expense, net $— $— $(10.0) $— GAAP other income (expense), net $163.8 $(32.1) $1,652.8 $789.2 Losses (gains) on marketable and other securities, net 21.5 212.9 (946.1) (118.3)Non-GAAP other income (expense), net $185.3 $180.8 $706.7 $670.9 GAAP net income $844.6 $917.7 $4,504.9 $4,412.6 Total of GAAP to non-GAAP reconciling items above 462.7 584.5 360.1 1,103.5 Income tax effect of GAAP to non-GAAP reconciling items (91.4) (112.5) (54.4) (196.9)Income tax expense: Shortfall from stock-based compensation 32.6 — 32.6 — Income tax expense: Charge related to enactment of OBBBA — — 44.5 — Non-GAAP net income $1,248.5 $1,389.7 $4,887.7 $5,319.2 Non-GAAP net income per share - basic $12.13 $12.92 $46.73 $49.30 Non-GAAP net income per share - diluted $11.44 $12.07 $44.31 $45.62 Shares used in calculating: Non-GAAP net income per share - basic 102.9 107.6 104.6 107.9 Non-GAAP net income per share - diluted 109.1 115.1 110.3 116.6 RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL INFORMATION (Unaudited)(continued) Three Months EndedDecember 31, Year EndedDecember 31, 2025 2024 2025 2024Effective tax rate reconciliation: GAAP ETR 19.1% 4.2% 13.9% 7.7%Income tax effect of GAAP to non-GAAP reconciling items 0.2% 5.7% 0.1% 1.9%Income tax expense: Shortfall from stock-based compensation (2.2%) —% (0.6%) —%Income tax expense: Charge related to enactment of OBBBA —% —% (0.8%) —%Non-GAAP ETR 17.1% 9.9% 12.6% 9.6% Gross margin on net product sales reconciliation: GAAP gross margin on net product sales 81% 84% 82% 86%Intangible asset amortization expense 2% 1% 2% 1%Stock-based compensation expense 2% 1% 1% 1%Non-GAAP gross margin on net product sales 85% 86% 85% 88% Year EndedDecember 31, 2025 2024 Free cash flow reconciliation: Net cash provided by operating activities $4,978.9 $4,420.5 Capital expenditures (898.4) (755.9) Free cash flow $4,080.5 $3,664.6 TABLE 4 REGENERON PHARMACEUTICALS, INC.COLLABORATION REVENUE (Unaudited)(In millions) Three Months EndedDecember 31, Year EndedDecember 31, 2025 2024 2025 2024 Sanofi collaboration revenue: Regeneron's share of profits in connection with commercialization of antibodies $1,485.9 $1,042.9 $5,241.6 $3,923.5 Reimbursement for manufacturing of commercial supplies 154.3 169.7 642.4 607.9 Total Sanofi collaboration revenue 1,640.2 1,212.6 5,884.0 4,531.4 Bayer collaboration revenue: Regeneron's share of profits in connection with commercialization of EYLEA 8 mg and EYLEA outside the United States 270.1 348.8 1,282.7 1,403.3 Reimbursement for manufacturing of commercial supplies 48.6 28.3 139.7 95.7 Total Bayer collaboration revenue 318.7 377.1 1,422.4 1,499.0 Other collaboration revenue 12.0 17.2 24.8 27.4 Total collaboration revenue $1,970.9 $1,606.9 $7,331.2 $6,057.8 TABLE 5 REGENERON PHARMACEUTICALS, INC.NET PRODUCT SALES OF REGENERON-DISCOVERED PRODUCTS (Unaudited)(In millions) Three Months EndedDecember 31, 2025 2024 % Change U.S. ROW Total U.S. ROW Total (Total Sales)EYLEA HD(a) $506.3 $312.2 $818.5 $304.6 $90.4 $395.0 107%EYLEA(a) $576.9 $504.8 $1,081.7 $1,190.4 $797.5 $1,987.9 (46%)Total EYLEA HD and EYLEA $1,083.2 $817.0 $1,900.2 $1,495.0 $887.9 $2,382.9 (20%)Dupixent(b) $3,733.8 $1,205.7 $4,939.5 $2,745.8 $951.8 $3,697.6 34%Libtayo(c) $285.3 $140.1 $425.4 $251.2 $115.7 $366.9 16%Praluent(d) $72.2 $153.6 $225.8 $62.7 $117.7 $180.4 25%Kevzara(b) $100.0 $52.0 $152.0 $82.4 $52.4 $134.8 13%Other products(e) $92.9 $31.8 $124.7 $78.5 $24.8 $103.3 21% Year EndedDecember 31, 2025 2024 % Change U.S. ROW Total U.S. ROW Total (Total Sales)EYLEA HD(a) $1,636.9 $932.7 $2,569.6 $1,201.1 $239.9 $1,441.0 78%EYLEA(a) $2,747.8 $2,573.6 $5,321.4 $4,767.1 $3,336.9 $8,104.0 (34%)Total EYLEA HD and EYLEA $4,384.7 $3,506.3 $7,891.0 $5,968.2 $3,576.8 $9,545.0 (17%)Dupixent(b) $13,187.0 $4,619.7 $17,806.7 $10,398.7 $3,749.3 $14,148.0 26%Libtayo(c) $944.7 $507.5 $1,452.2 $787.3 $429.5 $1,216.8 19%Praluent(d) $262.5 $594.3 $856.8 $241.7 $523.3 $765.0 12%Kevzara(b) $371.4 $203.2 $574.6 $270.2 $188.5 $458.7 25%Other products(e) $210.0 $113.3 $323.3 $202.9 $90.0 $292.9 10% Note: The table above includes net product sales of Regeneron-discovered products. Such net product sales are recorded by the Company or others, as further described in the footnotes below.(a) The Company records net product sales of EYLEA HD and EYLEA in the United States, and Bayer records net product sales outside the United States. The Company records its share of profits in connection with sales outside the United States within Collaboration revenue.(b) Sanofi records global net product sales of Dupixent and Kevzara, and the Company records its share of profits in connection with global sales of such products within Collaboration revenue(c) The Company records global net product sales of Libtayo and pays Sanofi a royalty on such sales(d) The Company records net product sales of Praluent in the United States. Sanofi records net product sales of Praluent outside the United States and pays the Company a royalty on such sales, which is recorded within Other revenue.(e) Included in this line item are products which are sold by the Company and others. Refer to "Fourth Quarter and Full Year 2025 Financial Results" section above for a listing of net product sales recorded by the Company. Not included in this line item are net product sales of ARCALYST, which are recorded by Kiniksa.

Phase 3Drug ApprovalAcquisitionLicense out/inClinical Result

12 Dec 2025

·endpoints.news

FDA broadens label for Amgen's Uplizna as it enters rare disease competition

The FDA on Thursday expanded the label for Amgen’s rare disease drug Uplizna, approving it in an autoimmune disorder that’s quickly become a hot — and crowded — area for biopharma. Regulators gave the green light to Uplizna in generalized myasthenia gravis, a chronic condition that causes muscle weakness, particularly in the eyes, face, throat and lungs. It’s the second drug approved for myasthenia gravis this year, and the fifth since 2021. Uplizna’s pricing will remain the same for myasthenia gravis as its other two approved conditions, an Amgen spokesperson said. Currently, Uplizna’s average wholesale acquisition cost is $140,248.50 per dose. In myasthenia gravis, patients take two doses per year, plus an additional “loading dose” in the first year. Uplizna will face competition with both formulations of argenx’s Vyvgart, UCB’s Rystiggo and Zilbrysq, Johnson & Johnson’s Imaavy, and AstraZeneca-Alexion’s Soliris and Ultomiris. Jasper van Grunsven, SVP of Amgen’s rare disease portfolio, told Endpoints News the company hopes Uplizna can differentiate itself because researchers saw the drug provide a double whammy in clinical trials: a long-lasting response with twice-yearly dosing. Based on the data, “we feel we could be competitive in both” first-line and second-line settings, van Grunsven said. Many other drugmakers are testing experimental treatments for myasthenia gravis in the clinic. AstraZeneca’s gefurulimab passed a Phase 3 test in July, and Regeneron’s siRNA drug cemdisiran also succeeded in a pivotal trial in August. Cartesian Therapeutics is also testing a CAR-T therapy for the condition. Amgen acquired Uplizna as part of its $27.8 billion buyout of Horizon Therapeutics. Van Grunsven said that the deal, combined with the 2022 acquisition of ChemoCentryx, has positioned Amgen well in rare diseases going forward. Next for Uplizna are Phase 2 studies for CD19-positive, B cell-mediated autoimmune disease in combination with Blincyto, another of Amgen’s rare disease drugs. “The rare disease franchise looks very good. Our engine is very committed behind further developing that,” van Grunsven said.

Drug ApprovalPhase 3AcquisitionPhase 2

100 Deals associated with Cemdisiran

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Indication	Highest Phase	Country/Location	Organization	Date
Hemolysis	Phase 3	Brazil	Regeneron Pharmaceuticals, Inc.	07 Nov 2025
Hemolysis	Phase 3	Canada	Regeneron Pharmaceuticals, Inc.	07 Nov 2025
Hemolysis	Phase 3	Italy	Regeneron Pharmaceuticals, Inc.	07 Nov 2025
Hemolysis	Phase 3	Poland	Regeneron Pharmaceuticals, Inc.	07 Nov 2025
Hemolysis	Phase 3	South Korea	Regeneron Pharmaceuticals, Inc.	07 Nov 2025
Hemolysis	Phase 3	Spain	Regeneron Pharmaceuticals, Inc.	07 Nov 2025
Hemolysis	Phase 3	Taiwan Province	Regeneron Pharmaceuticals, Inc.	07 Nov 2025
Hemolysis	Phase 3	Turkey	Regeneron Pharmaceuticals, Inc.	07 Nov 2025
Age Related Macular Degeneration	Phase 3	United States	Regeneron Pharmaceuticals, Inc.	30 Oct 2024
Age Related Macular Degeneration	Phase 3	Austria	Regeneron Pharmaceuticals, Inc.	30 Oct 2024

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05744921 (ASH2025)	Phase 3	Hemoglobinuria, Paroxysmal	44	Pozelimab plus cemdisiran	azkvgzcici(aetfnrmeri) = xpartmkski kehkwldwxl (assliplqwr ) View more	Positive	06 Dec 2025
				Pozelimab plus cemdisiran	azkvgzcici(aetfnrmeri) = xpiguwbeot kehkwldwxl (assliplqwr ) View more
NCT05070858 (NIMBLE, Company_Website) Manual	Phase 3	Myasthenia Gravis	190	Cemdisiran（600 mg）	labziesmpm(uftakquhmj) = bbakbetuxu pdlwnnggji (mvctorjlmm ) Met View more	Positive	26 Aug 2025
				Cemdi-poze (cemdisiran 200 mg and pozelimab 200 mg)	labziesmpm(uftakquhmj) = cbzcngmmgc pdlwnnggji (mvctorjlmm ) Met View more
NCT04811716 (Pubmed \| EJHaem)	Phase 2	Hemoglobinuria, Paroxysmal	24	Cemdisiran 200 mg Q4WPozelimab 400 mg Q4W + Cemdisiran 200 mg Q4WPozelimab 400 mg Q4WW	lbbqyjtfwf(lieiowxhip) = While most patients (66.7%) experienced treatment‐emergent adverse events, the majority of these events were mild to moderate in severity. zxymkvumyh (qougukiiwh ) View more	Positive	01 Aug 2025
NCT05133531 (ASH2024) Manual	Phase 3	Hemoglobinuria, Paroxysmal	48	Pozelimab plus Cemdisiran	yphlsjmdbo(ujewbtrhzr) = sowcpyfhyr edhnvzuzgh (qamdebcala ) View more	Positive	07 Dec 2024
				Ravulizumab	yphlsjmdbo(ujewbtrhzr) = fbncbrvouk edhnvzuzgh (qamdebcala ) View more
NCT04811716 (EHA2024) Manual	Phase 2	Hemoglobinuria, Paroxysmal	22	Cemdisiran20Pozelimab+ Pozelimab 400 mg SC Q4W	jgiougsguh(sktkbdnnsp) = CH50 remained fully suppressed in all patients at all post-baseline time points phanubboim (bjuutysuoj ) View more	Positive	14 May 2024
NCT05133531 (EHA2024) Manual	Phase 3	Hemoglobinuria, Paroxysmal First line C5 Mutation	48	Pozelimab + Cemdisiran	vlqnnsrpen(bmahxbrgyl) = ragwsmoacl tbmsuxqito (rawelgadqp ) View more	Positive	14 May 2024
				Ravulizumab	vlqnnsrpen(bmahxbrgyl) = emvpvexlvu tbmsuxqito (rawelgadqp ) View more
NCT03841448 (Phase 2 Study of Cemdisiran, CTgov)	Phase 2	Glomerulonephritis, IGA	31	Placebo	aggthlebsv(dxdtthuamn) = ggimovqyij hrbpgyydil (outqdnxxzm, 0.258) View more	-	08 Dec 2023
NCT04811716 (EHA2023)	Phase 2	Hemoglobinuria, Paroxysmal	24	Pozelimab	xrnjkhmaki(yfhdhabdjs) = ihycamllom mckszjrwkj (hjdgrkmrcz )	-	08 Jun 2023
				Cemdisiran	xrnjkhmaki(yfhdhabdjs) = cpjyrdzkdx mckszjrwkj (hjdgrkmrcz )
NCT04888507 (ASH 12022 \| ASH 22022) Manual	Phase 2	Hemoglobinuria, Paroxysmal	6	Cemdisiran+Pozelimab	rmuzfabwlo(jbypxgkgmc) = sxyoozgzph ojcdblynqj (mjpinubbnn ) View more	Positive	15 Nov 2022
NCT04811716 (ASH 12022 \| ASH 22022) Manual	Phase 2	Hemoglobinuria, Paroxysmal	22	Cemdisiran+Pozelimab (Arm 1 (Q4W))	zjaonfdsnq(tvznujvpqx) = buszvqesws qlxkyamvgg (wahpgrufkr ) View more	Positive	15 Nov 2022
				Cemdisiran+Pozelimab (Arm 2 (Q2W))	zjaonfdsnq(tvznujvpqx) = sgjdorltvg qlxkyamvgg (wahpgrufkr ) View more

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