Regeneron Presentations at ASH Highlight Expanding Clinical Research in Blood Cancers and Disorders

Rhea-AI Summary

Regeneron Pharmaceuticals (NASDAQ: REGN) announced new data to be presented at the 2021 American Society of Hematology (ASH) Annual Meeting from December 11-14. Highlighted is the updated Phase 1 data for REGN5458, targeting heavily pre-treated multiple myeloma, with a Phase 2 trial currently enrolling. Additionally, first data on the combination of pozelimab and cemdisiran will be shared, aimed at treating paroxysmal nocturnal hemoglobinuria. An investor webcast will take place on December 13 to discuss the hematology portfolio.

Positive

• Updated Phase 1 results for REGN5458 show potential for treating heavily pre-treated multiple myeloma.
• Phase 2 trial of REGN5458 currently enrolling with registrational intent.
• First data from the combination of pozelimab and cemdisiran targeting paroxysmal nocturnal hemoglobinuria.

Negative

• None.

New data for REGN5458 (BCMAxCD3 bispecific antibody) in patients with heavily pre-treated multiple myeloma to be highlighted in an oral presentation

First data evaluating a combination therapy approach with pozelimab (C5 antibody) and Alnylam's cemdisiran (siRNA C5 inhibitor) in healthy volunteers

Regeneron will host an investor webcast on Monday, December 13 to provide further updates across its hematology portfolio

TARRYTOWN, N.Y., Nov. 4, 2021 /PRNewswire/ -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that new and updated data from its diverse hematology program in blood cancers and disorders will be presented at the 2021 American Society of Hematology (ASH) Annual Meeting from December 11-14 in Atlanta, GA.

"Our hematology portfolio continues to expand with multiple trials now underway to investigate our bispecific antibodies, RNA-based therapies and gene editing, among other approaches," said L. Andres Sirulnik, M.D., Ph.D., Senior Vice President, Translational and Clinical Sciences, Hematology at Regeneron. "Our diverse pipeline includes investigational medicines across several blood cancers including multiple myeloma and lymphoma, in addition to blood disorders such as paroxysmal nocturnal hemoglobinuria, aplastic anemia, amyloidosis and thrombosis. We continue to make progress across our hematology portfolio with eight assets currently in the clinic."  

Regeneron data at ASH include an oral presentation with updated Phase 1 results from the completed dose escalation for REGN5458, an investigational BCMAxCD3 bispecific antibody, in patients with heavily pre-treated multiple myeloma. REGN5458 has the potential to advance treatment for patients with relapsed refractory disease, and the Phase 2 portion of this trial is currently enrolling with registrational intent. In addition, Regeneron and collaborator Alnylam will share the first data from healthy volunteers for a novel investigational combination of Regeneron's C5 antibody pozelimab and Alnylam's C5-inhibiting siRNA cemdisiran. The combination is planned to be evaluated in patients with the rare blood disorder, paroxysmal nocturnal hemoglobinuria (PNH), and other complement-driven disorders.

Additional presentations will include updated Phase 2 data investigating pozelimab monotherapy in patients with PNH, as well as analyses of real-world quality of life and treatment preferences among patients with diffuse large B-cell lymphoma to inform Regeneron's therapeutic development in this tumor type.

Investor Webcast Information
Regeneron will host a conference call and simultaneous webcast to share updates on the company's hematology portfolio on Monday, December 13 at 4:30 PM ET. To access this call, dial (888) 660-6127 (U.S.) or (973) 890-8355 (International); conference ID 2668896. A link to the webcast may be accessed from the 'Investors and Media' page of Regeneron's website at http://investor.regeneron.com/events.cfm. A replay of the conference call and webcast will be archived on the company's website for at least 30 days.

Regeneron Presentations at ASH:

-- Oral presentation (#160): Early, deep, and durable responses, and low rates of cytokine release syndrome with REGN5458, a BCMAxCD3 bispecific monoclonal antibody, in a Phase 1/2 first-in-human study in patients with relapsed/refractory multiple myeloma (RRMM) (Jeffrey A. Zonder, M.D.: Saturday, December 11, 12:45 PM ET)

-- Other presentations:

• Poster #1128: Pozelimab, a human monoclonal antibody against complement factor C5, provided inhibition of intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (Jun-Ho Jang, M.D., Ph.D: Saturday, December 11, 5:30-7:30 PM ET)
• Poster #1998: Interim analysis of an open-label, ascending-dose, Phase 1 study of the safety, tolerability, pharmacokinetics, and pharmacodynamics of single doses of the subcutaneously administered human monoclonal antibody pozelimab in combination with single doses of the subcutaneously administered siRNA cemdisiran in healthy volunteers (Tavé van Zyl, M.D.: Sunday, December 12, 6:00-8:00 PM ET)
• Poster #4111: Real-world health-related quality of life in patients with diffuse large B-cell lymphoma: Comparisons with reference populations and by line of therapy (Qiufei Ma: Monday, December 13, 6:00-8:00 PM ET)
• Online publication: Treatment preferences among patients with diffuse large B-cell lymphoma: A survey across western Europe and the United States of America (P. Connor Johnson)

The potential uses of REGN5458, pozelimab and cemdisiran described above are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.

About Regeneron in Hematology
At Regeneron, we're translating more than three decades of biology expertise with our proprietary VelociSuite^® technologies to develop potentially paradigm-changing medicines for patients with diverse blood cancers and rare blood disorders.

Our blood cancer research is focused on bispecific antibodies that are being investigated both as monotherapies and in combination with each other and emerging therapeutic modalities. Together, they provide us with unique combinatorial flexibility to develop customized and potentially synergistic cancer treatments.

Our research and collaborations to develop potential treatments for rare blood disorders include explorations in antibody medicine, gene editing using CRISPR and gene-knockout technologies, as well as investigational RNA-approaches that are being investigated for their ability to deplete abnormal proteins or block disease-causing cellular signaling.

For more information, visit https://www.regeneron.com/pipeline.

About Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to nine FDA-approved treatments and numerous product candidates in development, almost all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, pain, hematologic conditions, infectious diseases and rare diseases.

Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite technologies, such as VelocImmune, which uses unique genetically humanized mice to produce optimized fully human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world.

For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.

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, the impact of SARS-CoV-2 (the virus that has caused the COVID-19 pandemic) on Regeneron's business and its employees, collaborators, and suppliers and other third parties on which Regeneron relies, Regeneron's and its collaborators' ability to continue to conduct research and clinical programs, Regeneron's ability to manage its supply chain, net product sales of products marketed or otherwise commercialized by Regeneron and/or its collaborators (collectively, "Regeneron's Products"), and the global economy; the nature, timing, and possible success and therapeutic applications of Regeneron's Products and product candidates being developed by Regeneron and/or its collaborators (collectively, "Regeneron's Product Candidates") and research and clinical programs now underway or planned, including without limitation REGN5458 (a BCMAxCD3 bispecific antibody), pozelimab (a C5 antibody) as monotherapy or in combination with cemdisiran (a siRNA C5 inhibitor being developed by Alnylam Pharmaceuticals, Inc.), and Regeneron's other oncology programs (including its bispecific portfolio); the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron's Product Candidates and new indications for Regeneron's Products, such as REGN5458 in multiple myeloma and pozelimab (as monotherapy or in combination with cemdisiran) in paroxysmal nocturnal hemoglobinuria; 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), including the studies discussed or reference in this press release, on any of the foregoing; 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 ability of Regeneron to manufacture and manage supply chains for multiple products and product candidates; 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; 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; ongoing regulatory obligations and oversight impacting Regeneron's Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement of Regeneron's Products from third-party payers, including private payer 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 payers and new policies and procedures adopted by such payers; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron's Products and Regeneron's Product Candidates; 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; 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; the potential for any license, collaboration, or supply agreement, including Regeneron's agreements with Sanofi, Bayer, and Teva Pharmaceutical Industries Ltd. (or their respective affiliated companies, as applicable), to be cancelled or terminated; and 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^® (aflibercept) Injection, Dupixent^® (dupilumab), Praluent^® (alirocumab), and REGEN-COV^® (casirivimab and imdevimab)), other litigation and other proceedings and government investigations relating to the Company and/or its operations, 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 (http://newsroom.regeneron.com) and its Twitter feed (http://twitter.com/regeneron).

Contacts:   Media Relations Taylor Ramsey Tel: +1 914-409-2381 taylor.ramsey@regeneron.com

Investor Relations Vesna Tosic Tel: +1 914-847-5443 vesna.tosic@regeneron.com

View original content:https://www.prnewswire.com/news-releases/regeneron-presentations-at-ash-highlight-expanding-clinical-research-in-blood-cancers-and-disorders-301415975.html

SOURCE Regeneron Pharmaceuticals, Inc.

FAQ

What is the significance of REGN5458 in multiple myeloma treatment?

REGN5458 is a BCMAxCD3 bispecific antibody showing promise with updated Phase 1 results, indicating potential effectiveness for patients with heavily pre-treated multiple myeloma.

When will Regeneron present data at the ASH Annual Meeting?

Data will be presented from December 11-14 at the 2021 ASH Annual Meeting.

What are the details of the investor webcast on December 13?

Regeneron will host an investor webcast on December 13 at 4:30 PM ET, discussing updates across its hematology portfolio.

What is the combination therapy involving pozelimab and cemdisiran?

The combination therapy involves pozelimab (a C5 antibody) and cemdisiran (a siRNA C5 inhibitor) aimed at treating paroxysmal nocturnal hemoglobinuria.