On January 13, 2025 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported it will share corporate progress and highlights from the Company’s broad and diverse investigational pipeline while presenting at the annual J.P. Morgan Healthcare Conference (Press release, Regeneron, JAN 13, 2025, View Source [SID1234649674]). The presentation is scheduled for 2:15 p.m. Pacific Time (5:15 p.m. Eastern Time) and may be accessed from the "Investors & Media" page of Regeneron’s website.
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"The Regeneron name is synonymous with innovation, brought to life through proprietary technologies and world-class science that produce medicines that make a meaningful impact on patients’ lives," said Leonard S. Schleifer, M.D., Ph.D., Board co-Chair, President and Chief Executive Officer of Regeneron. "Thanks to our long-term and consistent R&D investment, we have – in addition to our four blockbuster medicines – one of the industry’s largest, most promising and most diverse clinical pipelines. Our therapeutic candidates tackle a myriad of diseases, with the most advanced programs addressing an aggregate commercial market opportunity expected to exceed $220 billion by 2030. We are well positioned for future growth and more confident than ever in the power of Regeneron’s science."
Marketed Products
Dupixent Updates
Dupixent (dupilumab) is now used to treat over a million patients globally. The recent approval and launch in chronic obstructive pulmonary disease (COPD) has had a successful start, with coverage secured from the top commercial and Medicare payers and Dupixent now well positioned to address approximately 300,000 patients in the U.S.
There is continued growth potential in existing and additional indications for diseases in which type 2 inflammation may play a role, including chronic spontaneous urticaria (CSU) with an expected U.S. Food and Drug Administration (FDA) decision by April 18, 2025, and bullous pemphigoid, for which a supplemental Biologics License Application (sBLA) was submitted in the fourth quarter of 2024.
EYLEA HD and EYLEA Updates
On a combined basis, EYLEA HD (aflibercept) Injection 8 mg and EYLEA (aflibercept) Injection 2 mg remained the U.S. anti-VEGF category leader in 2024. Based on preliminary (unaudited) results, the products achieved 1% year-over-year growth by reaching $6 billion in aggregate U.S. net product sales for the year and $1.5 billion in aggregate U.S. net product sales for the fourth quarter of 2024, despite increasing competition. EYLEA HD U.S. net product sales were $305 million in the fourth quarter of 2024. EYLEA U.S. net product sales were $1.19 billion in the fourth quarter of 2024.
Combined EYLEA HD and EYLEA U.S. net product sales for the fourth quarter of 2024 were favorably impacted by approximately $85 million as a result of higher wholesaler inventory levels for EYLEA, partially offset by lower wholesaler inventory levels for EYLEA HD.
The Company filed an application with the FDA for use of the EYLEA HD pre-filled syringe (PFS) with U.S. approval and launch expected by mid-2025.
Longer term data in wet age-related macular degeneration (wAMD) and diabetic macular edema (DME) are under FDA review with a PDUFA date of April 20, 2025 to potentially extend dosing intervals for EYLEA HD up to every-24 weeks.
The Company plans to submit a sBLA for EYLEA HD for every four-week dosing and for retinal vein occlusion (RVO) in the first quarter of 2025 to potentially maximize dosing flexibility and address more retinal diseases.
Libtayo Updates
Libtayo (cemiplimab) exceeded $1 billion in sales for 2024 and remains foundational to Regeneron’s oncology portfolio.
As announced this morning, a Phase 3 study demonstrated that Libtayo is the only immunotherapy to show a statistically significant and clinically meaningful benefit in high-risk cutaneous squamous cell carcinoma (CSCC) in the adjuvant setting; a recent Phase 3 trial with Keytruda failed in the same setting.1 Specifically, adjuvant Libtayo demonstrated a 68% reduction in the risk of disease recurrence or death, compared to placebo (hazard ratio: 0.32; 95% confidence interval: 0.20-0.51; p<0.0001). Grade ≥3 adverse events occurred in 24% (n = 49 of 205) and 14% (n = 29 of 204) of patients in the Libtayo arm and the placebo arm, respectively. Detailed results will be presented at an upcoming medical meeting and will be shared with regulatory authorities with a plan for FDA submission in the first half of 2025.
Phase 3 and Other Major Pipeline Opportunities
Regeneron is progressing numerous promising drug candidates across diverse disease states, with advanced programs that together have a total addressable commercial market expected to exceed $220 billion by 2030. Some near-term highlights include:
Itepekimab (IL-33) for COPD: Based on genetic data linking IL-33 with increased risk of COPD and Phase 2 results, Regeneron’s next innovation in COPD offers potential for benefit in a broader population, including former smokers, non-cystic fibrosis bronchiectasis and other indications. Results are expected from the Phase 3 AERIFY study in the second half of 2025, with a potential BLA submission to follow.
Fianlimab (LAG3) for melanoma: Combining fianlimab and Libtayo, two potentially best-in-class checkpoint inhibitors, has the potential for differentiated efficacy and safety versus the current standard-of-care. Results from the first Phase 3 study in first-line metastatic melanoma are expected in the second half of 2025, with a potential BLA submission to follow.
Linvoseltamab (BCMAxCD3) for multiple myeloma: Linvoseltamab has potential to be the best-in-class BCMAxCD3 bispecific with its differentiated clinical profile, dosing regimen and administration method. The linvoseltamab BLA has been resubmitted following resolution of third-party manufacturing issues, with launch anticipated in mid-2025. Phase 3 programs in earlier lines of therapy using linvoseltamab monotherapy and novel combinations are also underway.
Odronextamab (CD20xCD3) for lymphoma: Ordspono (odronextamab) has been approved in the European Union for relapsed/refractory follicular lymphoma (FL) and diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy, and enrollment is underway for a confirmatory study to support resubmission of the BLA for FL to the FDA in the first quarter of 2025. A broad and differentiated Phase 3 program is also underway to investigate odronextamab in earlier lines of FL and DLBCL. As reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting, odronextamab monotherapy showed complete responses in 12 out of 12 evaluable patients with first-line FL in the safety lead-in portion of the Phase 3 program.
Factor XI for anticoagulation: Regeneron’s two-pronged approach to anticoagulation is being evaluated for its potential to control thombosis while minimizing bleeding risk in a variety of patient populations and clinical settings. Two Factor XI antibodies, REGN7508 (catalytic domain) and REGN9933 (A2 domain), will advance to pivotal trials in 2025 on the basis of positive proof-of-concept data announced in December 2024. Current standards of care for thrombosis disorders have challenges including elevated risk of bleeding resulting in underutilization, presenting an unmet need for more specific inhibition of the intrinsic coagulation pathway.
Multiple approaches to obesity: Regeneron is studying various combinations with GLP-based therapies to potentially improve quality of weight loss by preserving lean muscle, as well as improve maintenance of weight loss following GLP-1/GIP discontinuations. A Phase 2 study of trevogrumab and semaglutide with and without garetosmab is now fully enrolled and a Phase 2 study testing combinations of tirzepatide and mibavademab is ongoing, with initial data expected from both in the second half of 2025.
BCMAxCD3/Dupixent in severe allergy: Combining linvoseltamab and Dupixent has the potential to eliminate immunoglobulin E (IgE), the key driver of allergic reactions, and thus potentially reverse severe allergies. A trial in patients with severe food allergies is ongoing, with initial clinical data shared in today’s presentation showing profound reduction of IgE in the first patient treated with this two-drug approach.
C5 Combo (pozelimab and cemdisiran) in complement-mediated diseases: Regeneron’s differentiated siRNA and antibody combination approach has the potential to address multiple complement-mediated diseases, such as generalized myasthenia gravis (Phase 3 results expected in the second half of 2025), paroyxsmal noctural hemoglobinuria (Phase 3 registrational data expected in 2026+) and geographic atrophy, an advanced form of dry AMD (Phase 3 pivotal program underway).
DNA Sequence-Linked Healthcare Database
Regeneron continues to grow its leadership in genetics-driven drug discovery and is building the world’s largest DNA sequence-linked healthcare database, designed to unlock profound insights into how genetics impact health and aid in the development new genetic-based therapies and optimized healthcare services.
The Regeneron Genetics Center has sequenced nearly three million people to date, all with deidentified linked healthcare records.
A newly announced strategic collaboration with Truveta, Inc. is expected to dramatically expand the size of this database, with sequencing and linked Electronic Health Records for up to 10 million additional individuals from Truveta’s network of leading U.S. health systems.
On the basis of its industry-leading capabilities, Regeneron Genetics Center was selected by UK BioBank consortium members to complete proteomic assay data generation for the recently announced UK Biobank Pharma Proteomics Project.
"Regeneron continues to diversify our commercial, clinical and research portfolios by relentlessly pushing the boundaries of innovation and technology," said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer of Regeneron. "In 2025, we will progress dozens of promising new assets and expand the reach of our important established medicines to help even more patients in need. We remain at the forefront of biotechnology’s most remarkable era of drug discovery, striving to change the practice of medicine with approaches spanning antibodies, bispecifics, gene editing, gene silencing, gene therapy and cell therapy supported by DNA sequence- and proteomics-linked healthcare database."
The unapproved uses of EYLEA, EYLEA HD, Dupixent, Libtayo and pozelimab noted here are investigational and have not been fully evaluated by any regulatory authority. Cemdisiran, itepekimab, fianlimab, linvoseltamab, REGN7508, REGN9933, trevogrumab and garetosmab are investigational and have also not been fully evaluated by any regulatory authority. Odronextamab is approved in the European Union as Ordspono to treat R/R FL or DLBCL after two or more lines of systemic therapy, but the safety and efficacy of odronextamab has not been fully evaluated by any other regulatory authority.