On January 13, 2025 Syndax Pharmaceuticals (Nasdaq: SNDX) reported that it will present at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14th at 10:30 a.m. PT/1:30 p.m. ET (Press release, Syndax, JAN 13, 2025, View Source [SID1234649678]). Ahead of the presentation, Syndax highlighted its recent accomplishments and anticipated 2025 milestones.

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"Building on a transformative 2024 with the FDA approvals of Revuforj and Niktimvo, we are focused on executing two outstanding U.S. launches for these first-in-class, practice-changing medicines," said Michael A. Metzger, Chief Executive Officer. "Syndax is well-positioned for continued success and long-term growth with two approved drugs launching into multi-billion-dollar markets, a clear strategy to expand into additional indications, and a strong cash position expected to fund operations through profitability."

2024 Key Accomplishments

Revumenib:

Launched Revuforj (revumenib), the first and only U.S. Food and Drug Administration (FDA) approved menin inhibitor, in late November 2024. Revuforj was approved by the FDA under the Agency’s Real-Time Oncology Review (RTOR) program for the treatment of relapsed or refractory (R/R) acute leukemia with a KMT2A translocation in adult and pediatric patients one year and older.
Revumenib was added to the latest NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) as a category 2A recommendation for R/R acute leukemia with a KMT2A rearrangement.1
Announced that the primary endpoint was met in the protocol-defined efficacy population of 64 adults with R/R mNPM1 AML in the Phase 2 cohort of the AUGMENT-101 trial of revumenib.
Reported additional positive results from a post-hoc efficacy analysis of all 77 R/R mNPM1 AML patients who met the efficacy evaluable criteria in the Phase 2 cohort of AUGMENT-101.
Published data from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib in adult and pediatric patients with R/R KMT2A-rearranged (KMT2Ar) acute leukemia in the Journal of Clinical Oncology.
Presented a larger data set with longer follow-up from the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib in R/R KMT2Ar acute leukemia at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.
Presented data from multiple ongoing trials evaluating revumenib in mNPM1 and KMT2Ar acute leukemia across the treatment landscape. These trials include:
BEAT AML: A Phase 1 trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar AML patients. The trial is being conducted as part of the Leukemia & Lymphoma Society’s Beat AML Master Clinical Trial. Updated data from the trial showed an overall response rate (ORR)2 of 100% (37/37) and a composite complete remission (CRc) rate of 95% (35/37).
SAVE: A Phase 1/2 trial evaluating an all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in pediatric and adult patients with R/R AML or mixed-lineage acute leukemia (MPAL) harboring either mNPM1, KMT2Ar, or NUP98r alterations. The trial is being conducted by investigators from MD Anderson Cancer Center. Updated data that showed an ORR of 82% (27/33) and a CR/CRh rate of 48% (16/33) were presented at the 66th ASH (Free ASH Whitepaper) Annual Meeting.
Axatilimab:

Received U.S. Food and Drug Administration (FDA) approval for Niktimvo (axatilimab-csfr) for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).
Announced axatilimab-csfr was added to the latest NCCN Guidelines as a category 2A recommendation for the treatment of GVHD after the failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg.
Published results from the pivotal Phase 2 AGAVE-201 trial of axatilimab in adult and pediatric patients with recurrent/refractory active chronic GVHD in the New England Journal of Medicine.
Presented a secondary analysis of overall and organ-specific responses from the pivotal Phase 2 AGAVE-201 trial of axatilimab in adult and pediatric patients with recurrent/refractory active chronic GVHD who had received at least two prior lines of systemic therapy at the 66th ASH (Free ASH Whitepaper) Annual Meeting.
Initiated enrollment in the MAXPIRe trial, a 26-week randomized, double-blinded, placebo-controlled Phase 2 trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF).
The Company’s partner, Incyte, initiated a Phase 2, open-label, randomized, multicenter trial of axatilimab in combination with ruxolitinib in patients ≥12 years of age with newly diagnosed chronic GVHD.
The Company’s partner, Incyte, initiated a Phase 3, randomized, double-blind, placebo-controlled, multi-center trial that will investigate the use of axatilimab in combination with corticosteroids as initial treatment for chronic GVHD.
Corporate:

Announced a $350 million royalty funding agreement with Royalty Pharma based on U.S. net sales of Niktimvo. Syndax expects that its cash, cash equivalents and marketable securities, together with the $350 million from the royalty funding agreement and anticipated product revenue and interest income, will enable the company to reach profitability.
Expected 2025 Key Milestones

Revumenib:

Maximize U.S. adoption of Revuforj as the preferred menin inhibitor, leveraging our first mover advantage and robust clinical data.
Submit a supplemental NDA (sNDA) filing for revumenib in R/R mNPM1 AML in the first half of 2025, followed by a potential FDA approval around year-end 2025.
Publish pivotal data from AUGMENT-101 trial in R/R mNPM1 AML in the first half of 2025.
Initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients unfit to receive intensive chemotherapy in the first quarter of 2025.
Report Phase 1 data from a trial evaluating the combination of revumenib with intensive chemotherapy (7+3) followed by revumenib maintenance treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias in the second half of 2025.
Initiate multiple frontline trials evaluating revumenib in combination with intensive chemotherapy, starting in 2025.
Present additional data at medical congresses from ongoing trials of revumenib in combination with standard-of-care agents.
Axatilimab:

Launch Niktimvo in the U.S. in early first quarter of 2025. In the U.S., Niktimvo will be co-commercialized by Syndax and Incyte.
Complete enrollment in MAXPIRe Phase 2 IPF trial in 2025 with topline data expected in 2026.
Presentation at the 43rd Annual J.P. Morgan Healthcare Conference

Syndax will webcast its presentation from the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2025 at 10:30 a.m. PT (1:30 p.m. ET). A live webcast of the fireside chat can be accessed from the Investor section of the Company’s website at www.syndax.com, where a replay of the event will also be available for a limited time.

On January 13, 2025 SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, reported its preliminary fourth quarter and full year 2024 U.S. net product revenue for OGSIVEO (nirogacestat) and provided additional company updates ahead of its presentation at the 43rd Annual J.P. Morgan Healthcare conference (Press release, SpringWorks Therapeutics, JAN 13, 2025, View Source [SID1234649677]).

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Preliminary Fourth Quarter and Full Year 2024 Financial Results* and Recent Business Highlights

Preliminary fourth quarter and full-year 2024 U.S. net product revenue for OGSIVEO were $61.5 million and $172.0 million, respectively.
As of December 31, 2024, total preliminary cash, cash equivalents, and marketable securities was $461.9 million. SpringWorks expects its cash position to fund operations through profitability, which the Company anticipates achieving in the first half of 2026.
Presented long-term follow-up data from the Phase 3 DeFi trial of nirogacestat in adults with progressing desmoid tumors in the fourth quarter of 2024. These results showed that longer-term treatment with nirogacestat (median duration of treatment: 34 months) was associated with further reductions in tumor size, increase in objective response rate with additional partial responses and complete responses, sustained improvements in desmoid tumor symptoms including pain, and a consistent safety profile.
Obtained an exclusive, global license from Rappta Therapeutics Oy for a first-in-class molecular glue of specific Protein Phosphatase 2A (PP2A) complexes. PP2A mutations represent a class of targetable oncogenic drivers in molecularly defined subsets of uterine cancer patients with high unmet need. In preclinical models of PP2A mutant uterine cancer, SW-3431 (formerly RPT04402) showed rapid, deep and durable tumor regressions as a monotherapy. In exchange for the license, Rappta received a $13 million upfront payment and is eligible to receive further clinical, regulatory and commercial milestone payments, and tiered single digit royalties on net sales.
* The preliminary fourth quarter and full year 2024 financial results are unaudited and do not present all information necessary for an understanding of the Company’s results of operations and financial position for the fourth quarter and full year 2024. Such financial results are subject to adjustment and could differ from the Company’s announcement of complete financial results in February 2025.

2025 Priorities and Anticipated Milestones

OGSIVEO (nirogacestat)

Continue strong commercial execution of the OGSIVEO launch in the U.S.
Secure regulatory approval for OGSIVEO in the European Union (EU) and launch OGSIVEO following reimbursement authorization in individual EU countries, beginning with Germany in mid-2025.
Publish long-term follow-up data from the Phase 3 DeFi trial of nirogacestat in adults with desmoid tumors in a peer-reviewed journal by the end of 2025.
Report initial data from the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with ovarian granulosa cell tumors in the first half of 2025.
Continue to support several industry and academic collaborator studies evaluating nirogacestat as part of B-cell maturation antigen (BCMA) combination therapy regimens across treatment lines in patients with multiple myeloma.
Mirdametinib (NF1-PN)

Secure FDA approval in adults and children with NF1-associated plexiform neurofibromas, or NF1-PN (PDUFA: February 28, 2025), and launch in the U.S.
Obtain regulatory approval in the EU for mirdametinib for the treatment of adults and children with NF1-PN and begin initial launch in 2025.
Emerging Pipeline

SpringWorks expects additional data to be presented by MapKure from the brimarafenib monotherapy trial in the second half of 2025.
Continue enrolling patients in Phase 1 trial of SW-682 in Hippo-mutant solid tumors.
File an Investigational New Drug (IND) application for SW-3431 by the end of 2025.
"2025 is set up to be another transformative year for SpringWorks. With the potential launch of our second medicine and global expansion to serve patients with two devastating diseases, we are energized by the opportunity to continue delivering on the commitments we have made to the patient communities we are dedicated to serving," said Saqib Islam, Chief Executive Officer of SpringWorks. "In parallel with our commercial launches, we are advancing our pipeline of oncology programs for heavily underserved patient populations and are confident that our strong foundation will drive our long-term growth and success."

Presentation at the 43rd Annual J.P. Morgan Healthcare Conference

SpringWorks will webcast its presentation from the 43rd Annual J.P. Morgan Healthcare Conference today, Monday, January 13, 2025 at 11:15 a.m. PT (2:15 p.m. ET). To access the live webcast, please visit the Events & Presentations page within the Investors & Media section of the company’s website at View Source A replay of the webcast will be available on SpringWorks’ website for a limited time following the conference.

On January 13, 2025 Sana Biotechnology presented its corporate presentation (Presentation, Sana Biotechnology, JAN 13, 2025, View Source [SID1234649676]).

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On January 13, 2025 Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, reported a business update including preliminary total revenue and net product sales for the fourth quarter of 2024, ongoing activity from the commercial business and development pipeline, and its financial outlook for 2025 (Press release, Rigel, JAN 13, 2025, View Source [SID1234649675]).

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"2024 was a transformational year for Rigel as we successfully executed on our corporate strategy to grow our hematology and oncology focused organization. We generated record sales of both TAVALISSE and REZLIDHIA and welcomed a third product to our commercial portfolio, GAVRETO, which made a substantial contribution to our sales in the latter half of the year. This commercial success, combined with our commitment to financial discipline, enabled Rigel to reach financial breakeven, a key milestone for the company," said Raul Rodriguez, Rigel’s president and CEO. "In addition, we advanced our development pipeline, with the Phase 1b study of R289 in lower-risk MDS continuing to enroll patients and publishing promising initial safety and efficacy data. Building on this progress, we will continue to implement effective strategies that further grow and advance our portfolio in 2025, thereby generating significant value for Rigel and our shareholders."

Preliminary 2024 Financial Results and Business Update

Preliminary Financial Results

While Rigel is still determining final results for the fourth quarter of 2024, the company expects to report fourth quarter total revenue of $57.6 million, compared to $35.8 million for the same period of 2023.
Rigel expects to report fourth quarter net product sales of $46.5 million, compared to $29.5 million for the same period of 2023, including:
TAVALISSE (fostamatinib disodium hexahydrate) net product sales of $31.0 million compared to $25.7 million for the same period of 2023.
REZLIDHIA (olutasidenib) net product sales of $7.4 million compared to $3.9 million for the same period of 2023.
GAVRETO (pralsetinib) net product sales of $8.1 million. GAVRETO became commercially available from Rigel in June 2024.
The following table summarizes total bottles shipped for the fourth quarter:

TAVALISSE

REZLIDHIA

GAVRETO*

Bottles shipped to patients and clinics

2,855

503

874

Change in bottles remaining in distribution channel

317

62

64

Total bottles shipped

3,172

565

938

*GAVRETO bottle count represents 60-count bottle equivalent

Contract revenues from collaborations for the fourth quarter of 2024 is expected to be approximately $11.1 million, including a $4.0 million upfront cash payment from Dr. Reddy’s Laboratories Ltd. (Dr. Reddy’s); $3.6 million of revenue from Grifols S.A. related to delivery of drug supplies and earned royalties; $2.9 million of revenue from Kissei Pharmaceutical Co., Ltd. (Kissei) related to delivery of drug supplies; and $0.3 million of revenue from Medison Pharma Trading AG related to delivery of drug supplies and earned royalties.
For the full year, Rigel expects to report total revenue of $179.3 million, including net product sales of $144.9 million and contract revenues from collaborations of $34.4 million, compared to total revenue of $116.9 million in 2023, which included net product sales of $104.3 million, contract revenues from collaborations of $11.5 million and government contract revenue of $1.1 million.
Rigel expects to report cash, cash equivalents, and short-term investments of approximately $77.3 million as of December 31, 2024, compared to $56.9 million as of December 31, 2023.
The above information is preliminary, has not been audited, and is subject to change upon the audit of Rigel’s financial statements for the year ended December 31, 2024. Rigel expects to provide complete fourth quarter and full year 2024 financial results in March 2025.

Commercial Update

TAVALISSE surpassed $100 million in net product sales in 2024, reporting $104.8 million in net product sales.
Rigel entered into an exclusive license agreement with Dr. Reddy’s in November to develop and commercialize REZLIDHIA in all potential indications throughout Dr. Reddy’s territory, which includes Latin America, South Africa, certain countries in the Commonwealth of Independent States (CIS), India, certain countries in Southeast Asia and North Africa, Australia and New Zealand. Rigel is entitled to receive an upfront cash payment of $4.0 million with the potential for up to $36.0 million in future regulatory and commercial milestone payments.
In December, Rigel’s partner Knight Therapeutics announced Mexico’s Comisión Federal para la Protección contra Riesgos Sanitarios approved TAVALISSE for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.
Clinical and Development Update

R2891, a novel and selective dual IRAK1/4 inhibitor, has been granted Fast Track designation for the treatment of previously-treated transfusion dependent lower-risk MDS and Orphan Drug designation for the treatment of MDS by the U.S. Food and Drug Administration (FDA).
Rigel continues to advance its Phase 1b clinical study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of R289 in patients with relapsed or refractory (R/R) lower-risk myelodysplastic syndrome (MDS). Enrollment in the fifth dose level (500mg / 250mg split dose) is ongoing.
Rigel presented initial data from the ongoing Phase 1b clinical study of R289 at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in December, demonstrating that R289 was generally well tolerated in this heavily pretreated R/R lower-risk MDS patient population, the majority of whom were high transfusion burden (HTB) at baseline.
In an ad-hoc analysis of the R289 Phase 1b initial data, responding patients (those achieving transfusion independence) appeared to have a greater increase in hemoglobin level over time compared to non-responding patients.
Also at the ASH (Free ASH Whitepaper) Annual Meeting, four posters were presented on olutasidenib, which included data that adds to the growing body of evidence supporting the benefits of its use in patients with mIDH1 AML.
As part of a multi-year strategic development alliance, Rigel and The University of Texas MD Anderson Cancer Center (MD Anderson), opened enrollment for two trials in December. The trials are a Phase 2 study in patients with IDH1-mutated clonal cytopenia of undetermined significance (CCUS), lower-risk MDS and chronic myelomonocytic leukemia (CMML), and a Phase 1/2 study of olutasidenib maintenance therapy following an allogeneic stem cell transplant for patients with IDH1-mutated myeloid malignancies. The Phase 1b/2 triplet therapy trial of decitabine and venetoclax in combination with olutasidenib in patients with mIDH1 AML is ongoing.
In December, in a paper titled "Olutasidenib demonstrates significant clinical activity in mutated IDH1 acute myeloid leukaemia arising from a prior myeloproliferative neoplasm", was published by Stéphane de Botton, M.D., Ph.D., head of translational research in hematology, Institut Gustave Roussy, France, in the British Journal of Haematology.
In November, the National Comprehensive Cancer Network (NCCN) added olutasidenib to the latest NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Myelodysplastic Syndromes. Olutasidenib was added as a recommended option to the following treatment algorithms: Management of Lower-Risk Disease, Management of Lower-Risk Disease – Evaluation of Related Anemia and Management of Higher-Risk Disease, and was recommended as NCCN Category 2B in all circumstances. If mIDH1 positive, olutasidenib was either recommended as a single agent, in combination with azacitidine, or both.*
*NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.

2025 Outlook

Rigel anticipates 2025 total revenue of approximately $200 to $210 million, including:

Net product sales of approximately $185 to $192 million
Contract revenues from collaborations of approximately $15 to $18 million.
The company anticipates it will report positive net income for the full year 2025, while funding existing and new clinical development programs.

In addition, Rigel plans to initiate a Phase 2 clinical study in recurrent glioma in 2025.

Additional information is included in Rigel’s corporate presentation, which can be found in the Investor Relations section of the company’s website at www.rigel.com.

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.