Cemdisiran

Ten abstracts, including three oral presentations, spotlight the expanding body of evidence supporting odronextamab in follicular lymphoma and diffuse large B-cell lymphoma Additional presentations include the first review of primary endpoint results with longer follow-up from the pivotal trial for linvoseltamab in heavily pre-treated patients with multiple myeloma TARRYTOWN, N.Y., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that new and updated data from its hematology pipeline will be shared in 19 abstracts at the American Society of Hematology (ASH) Annual Meeting from December 9 to 12 in San Diego, CA. These include research across six investigational medicines that span eight difficult-to-treat blood cancers and disorders. Together, these presentations showcase the diversity of approaches Regeneron is advancing through its hematology pipeline and its dedication to leading-edge research. “We are fusing our legacy of innovation with our deep scientific expertise in hematology to advance research across multiple modalities as we aim to ultimately make a meaningful impact in patients’ lives. Our data at ASH are a testament to our progress towards this ambition,” said L. Andres Sirulnik, M.D., Ph.D., Senior Vice President, Translational and Clinical Sciences, Hematology at Regeneron. “In addition to new results from our pivotal trials evaluating odronextamab and linvoseltamab, we are presenting findings on our growing blood disorders pipeline. Further, our presentations span emerging measures of disease that contribute to a deeper understanding of these advanced conditions, which in the future could form the basis of response-directed treatment paradigms.” At ASH, 10 abstracts will feature updated data and analyses for Regeneron’s most advanced investigational blood cancer medicine, odronextamab (CD20xCD3 bispecific antibody), in relapsed/refractory (R/R) follicular lymphoma (FL) and R/R diffuse large B-cell lymphoma (DLBCL). Among them are three oral presentations from its pivotal trial (ELM-2), including: the final analysis in R/R DLBCL patients; a comprehensive analysis of minimal residual disease status and circulating tumor DNA profiling in R/R FL and DLBCL patients; and updated analyses and long-term follow-up of efficacy, safety and patient reported outcomes in R/R FL. Furthermore, the company will share long-term survival outcomes and a responder analysis for odronextamab from a Phase 1 trial (ELM-1) in R/R DLBCL patients who have progressed after CAR-T therapy, a patient population who have a particularly dismal prognosis and limited effective treatment options. Odronextamab is currently under regulatory review for the treatment of R/R FL and R/R DLBCL by the U.S. Food and Drug Administration, with a target action date of March 31, 2024, as well as by the European Medicines Agency. Five presentations will highlight data supporting linvoseltamab (BCMAxCD3 bispecific antibody), including the first presentation of primary endpoint results with longer follow-up from the pivotal Phase 2 trial (LINKER-MM1) in heavily pre-treated patients with multiple myeloma. Additionally, two presentations will review the latest results from three Phase 2 studies evaluating pozelimab (C5 antibody) in combination with Alnylam Pharmaceuticals, Inc.’s cemdisiran (siRNA C5 inhibitor) in patients with paroxysmal nocturnal hemoglobinuria, a rare blood disorder. Regeneron presentations at ASH: Abstract title Abstract Presenting/Lead AuthorPresentationdate/time(PT)OdronextamabCirculating Tumor DNA Analysis Associates with Progression-Free Survival (PFS) with Odronextamab Monotherapy in Relapsed/Refractory (R/R) Follicular Lymphoma (FL) and Diffuse Large B-Cell Lymphoma (DLBCL): Identification of Minimal Residual Disease Status and High-Risk Subgroups from the Phase 2 ELM-2 Study#427 Oral PresentationJon E. ArnasonSunday,December 10,9:30 AM Final Analysis of the Phase 2 ELM-2 Study: Odronextamab in Patients with Relapsed/Refractory (R/R) Diffuse Large B-Cell Lymphoma (DLBCL)#436 Oral PresentationSabarish Ram AyyappanSunday,December 10,10:15 AM Maintenance of Moderate to High Levels of Functioning and Quality of Life with Odronextamab Monotherapy in Patients with Relapsed or Refractory Follicular Lymphoma#669 Oral PresentationBenoît TessoulinSunday,December 10,5:00 PM Odronextamab Monotherapy for the Treatment of Relapsed/Refractory (R/R) Follicular Lymphoma (FL) and Diffuse Large B-Cell Lymphoma (DLBCL): Focus on Clinical Pharmacology and Pharmacometrics in the ELM-1 and ELM-2 Studies#1436 Poster PresentationMin ZhuSaturday,December 9,5:30-7:30 PM Results of a Second, Prespecified Analysis of the Phase 2 Study ELM-2 Confirm High Rates of Durable Complete Response with Odronextamab in Patients with Relapsed/Refractory (R/R) Follicular Lymphoma (FL) with Extended Follow-Up#3041 Poster PresentationJose (J.C.) C. Villasboas BisnetoSunday,December 10,6:00-8:00 PM Trial in Progress: Phase 1 Trial Evaluating the Safety and Tolerability of Odronextamab in Combination with Cemiplimab in Relapsed/Refractory Aggressive B-cell Non-Hodgkin Lymphoma#3100 Poster PresentationCecilia CarpioSunday,December 10,6:00-8:00 PM Odronextamab Demonstrates Durable Complete Responses in Patients with Diffuse Large B-Cell Lymphoma (DLBCL) Progressing After CAR-T Therapy: Outcomes from the ELM-1 Study#4461 Poster PresentationJennifer L. CrombieMonday,December 11,6:00-8:00 PM Health-Related Quality of Life and Symptoms in Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma Treated with Odronextamab Monotherapy in the Phase 2 ELM-2 Study#4504 Poster PresentationElżbieta Iskierka-JażdżewskaMonday,December 11,6:00-8:00 PM Key prognostic factors in patients with relapsed/refractory follicular lymphoma: An evidence based systematic literature and medical review#7261 Online publicationAna Jimenéz-UbietoN/A Key prognostic factors in patients with relapsed/refractory diffuse large B-cell lymphoma: An evidence based systematic literature and medical review#7258 Online PublicationBastien von TresckowN/A LinvoseltamabIncidence of Second Primary Malignancies in Medicare-Insured Patients in the US with Triple-Class Exposed Relapsed/Refractory Multiple Myeloma#912 Oral PresentationSikander AilawadhiMonday,December 11,4:00 PM Health-Related Quality of Life (HRQoL) Among Patients with Triple-Class Exposed Relapsed/Refractory Multiple Myeloma (RRMM) Treated with Linvoseltamab in LINKER-MM1: Interim Assessment Up to 36 Weeks of Treatment#3359 Poster PresentationJames E. HoffmanSunday,December 10,6:00-8:00 PM Trial In Progress: A Phase 2 Study of Linvoseltamab for the Treatment of High-Risk Smoldering Multiple Myeloma (LINKER-SMM1)#3393 Poster PresentationPaula Rodriguez-OteroSunday,December 10,6:00-8:00 PM Real-World Study of Patients with Triple-Class Exposed Relapsed/Refractory Multiple Myeloma: Analysis Across a Spectrum of Advanced Disease Stage Medicare Patients in the United States#3773 Poster PresentationQiufei MaSunday,December 10,6:00-8:00 PM Patterns of Response to 200 mg Linvoseltamab in Patients with Relapsed/Refractory Multiple Myeloma: Longer Follow-up of the LINKER-MM1 study#4746 Poster PresentationSundar JagannathMonday,December 11,6:00-8:00 PM Pozelimab + Cemdisiran*Psychometric Evaluation of the PNH Symptom Questionnaire (PNH-SQ) Among Patients With Paroxysmal Nocturnal Hemoglobinuria from Three Phase 2 Clinical Trials With Pozelimab Monotherapy or in Combination With Cemdisiran#3752 Poster PresentationChristopher HartfordSunday,December 10,6:00-8:00 PM 52-Week Open-Label Extension Data from A Phase 2 Study Evaluating the Safety and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Switched from Eculizumab#2716 Poster PresentationRichard J. KellySunday,December 10,6:00-8:00 PM REGN7999 (TMPRSS6 inhibitor)Single Ascending Doses of REGN7999, A Monoclonal Antibody Inhibitor of TMPRSS6, Increase Serum Hepcidin And Cause Deep, Sustained Reductions in Serum Iron in Healthy Human Volunteers#3841 Poster PresentationNikhil SinghMonday,December 11,6:00-8:00 PM REGN7257 (IL2RG antibody)Blockade of Common Gamma Chain Cytokine Signaling with REGN7257, an Interleukin 2 Receptor Gamma (IL2RG) Monoclonal Antibody, in Combination with Costimulatory Blockers Delayed Skin Graft Rejection in Mice#2550 Poster PresentationAudrey Le Floc’hSunday,December 10,6:00-8:00 PM REGV131-LNP1265 (in vivo CRISPR/Cas9-based Factor 9 gene insertion therapy)**Novel Approaches for Gene-Based Therapies: Targeted Gene Insertion of Factor 9 as a Durable Treatment for Hemophilia BInvited TalkLeah SabinSaturday,December 9,9:30-10:45 AM *In collaboration with Alnylam Pharmaceuticals, Inc.**In collaboration with Intellia Therapeutics, Inc. The potential uses of odronextamab, linvoseltamab, pozelimab, cemdisiran, REGN7999, REGN7257, and REGV131-LNP1265 described above are investigational, and their safety and efficacy have not been fully evaluated by any regulatory authority. About Regeneron in HematologyAt Regeneron, we’re applying more than three decades of biology expertise with our proprietary VelociSuite® technologies to develop 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 and gene-knockout technologies, and investigational RNA-approaches focused on depleting abnormal proteins or blocking disease-causing cellular signaling. If you are interested in learning more about our clinical trials, please contact us (clinicaltrials@regeneron.com or 844-734-6643) or visit our clinical trials website. About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led for 35 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous FDA-approved treatments and product candidates in development, almost all of which were homegrown in our laboratories. Regeneron’s medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, hematologic conditions, infectious diseases and rare diseases. Regeneron is accelerating and improving the traditional drug development process through its 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 Regeneron, please visit www.regeneron.com or follow Regeneron on LinkedIn. Forward-Looking Statements and Use of Digital MediaThis 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 nature, timing, and possible success and therapeutic applications of 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”) and research and clinical programs now underway or planned, including without limitation odronextamab (CD20xCD3 bispecific antibody), linvoseltamab (BCMAxCD3 bispecific antibody), pozelimab (C5 antibody) in combination with Alnylam Pharmaceuticals, Inc.’s cemdisiran (siRNA C5 inhibitor), and other of Regeneron’s Product Candidates discussed or referenced in this press release; 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 odronextamab for the treatment of relapsed/refractory (“R/R”) diffuse large B-cell lymphoma and R/R follicular lymphoma, linvoseltamab for the treatment of multiple myeloma, and pozelimab in combination with cemdisiran for the treatment of 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 referenced in this press release, on any of the foregoing or any potential regulatory approval of Regeneron’s Products and Regeneron’s Product Candidates (such as odronextamab, linvoseltamab, and pozelimab in combination with cemdisiran); the ability of Regeneron’s collaborators, licensees, 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 manage supply chains for multiple products and product candidates; safety issues resulting from the administration of Regeneron’s Products and Regeneron’s Product Candidates (such as odronextamab, linvoseltamab, and pozelimab in combination with cemdisiran) 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 or licensees 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 and Bayer (or their respective affiliated companies, as applicable) as well as Regeneron’s agreement with Alnylam Pharmaceuticals, Inc. as referenced in this press release, to be cancelled or terminated; the impact of public health outbreaks, epidemics, or pandemics (such as the COVID-19 pandemic) on Regeneron's business; 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 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, including its Form 10-K for the year ended December 31, 2022 and its Form 10-Q for the quarterly period ended September 30, 2023. 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). Contacts: Media RelationsTammy Allen Tel: +1 914-306-2698tammy.allen@regeneron.comInvestor RelationsVesna TosicTel: +1 914-847-5443vesna.tosic@regeneron.com

CHAPLE is an ultra-rare hereditary disease that can cause potentially life-threatening gastrointestinal and cardiovascular symptoms Approval represents 10th FDA-approved medicine invented by Regeneron With the approval of Veopoz, the pre-approval inspection issues related to the aflibercept 8 mg biologics license application (BLA) have been addressed FDA action on the aflibercept 8 mg BLA is expected in the next few weeks Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the U.S. Food and Drug Administration (FDA) has approved Veopoz™ (pozelimab-bbfg) for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease, also known as CD55-deficient protein-losing enteropathy. Veopoz is the first and only treatment indicated specifically for CHAPLE. With the approval of Veopoz, the pre-approval inspection issues related to the aflibercept 8 mg BLA have been addressed. FDA action on the aflibercept 8 mg BLA is expected in the next few weeks. “Most patients with CHAPLE disease are children who face severely debilitating symptoms and often life-threatening complications that begin in infancy,” said Michael Lenardo, M.D., Chief, Molecular Development of the Immune System Section; Co-Director, Clinical Genomics Program, National Institute of Allergy and Infectious Disease (NIAID), National Institutes of Health (NIH). “As an investigator in this pivotal trial and one of the discoverers of this disease, I saw first-hand the transformational clinical improvement that pozelimab achieves in those suffering from CHAPLE. The approval of pozelimab is a milestone to celebrate, providing a new medicine that can help these long-suffering patients.” CHAPLE is an ultra-rare and life-threatening hereditary immune disease driven by an overactivation of the complement system. In healthy individuals, the complement system is a mechanism for destroying microbes. However, those living with CHAPLE are unable to regulate complement activity due to mutations in their CD55 gene. Without proper CD55 regulation, the complement system may attack normal cells, causing damage to blood and lymph vessels along the upper digestive tract and leading to the loss of circulating proteins. There are fewer than 10 patients with CHAPLE disease identified in the U.S. Veopoz, a fully human monoclonal antibody, is designed to target complement factor C5, a protein involved in complement system activation. “As the first-ever treatment for CHAPLE, Veopoz is a testament to our commitment to uncovering genetic insights and applying them to the development of effective treatments for patients in need – regardless of the prevalence of their disease,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer at Regeneron. “Beyond CHAPLE, we believe Veopoz has promise in a variety of complement-mediated diseases and are driving forward several clinical programs to explore its broader potential.” Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. Meningococcal infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update meningococcal vaccination at least two weeks prior to administering the first dose of Veopoz, unless the risks of delaying therapy outweigh the risks of developing meningococcal infection. If urgent therapy is indicated in a patient who is not up-to-date with both meningococcal vaccines according to Advisory Committee on Immunization Practices (ACIP) recommendations, administer meningococcal vaccines as soon as possible and provide the patient with antibacterial drug prophylaxis. Vaccination reduces, but does not eliminate, the risk of meningococcal infections. Regeneron is committed to helping people with rare diseases access their medications. Assistance for eligible CHAPLE patients, including information on treatment, insurance coverage, and financial support, will be available through Regeneron’s myRARE™ patient support program. Patients can call 1-833-4my-RARE (1-833-469-7273) for more information. Healthcare providers with questions regarding Veopoz access outside the United States can contact Clinigen at medicineaccess@clinigengroup.com and register for an account to request access at https://www.clinigengroup.com/direct/en/invitation-form/. Veopoz was reviewed under Priority Review, and the Company received a Rare Pediatric Disease Priority Review voucher upon approval. Veopoz was previously granted Rare Pediatric Disease designation, Orphan Disease designation and Fast Track designation. About the Pivotal CHAPLE Trial The FDA approval is based on results from a Phase 2/3 open-label trial that investigated the efficacy and safety of pozelimab in 10 patients aged 3 to 19 (median of 8.5 years). Patients were given a single loading dose of pozelimab 30 mg/kg intravenously on day 1, followed by subcutaneous weekly weight-based doses of pozelimab. All ten patients achieved normalization of serum albumin and serum IgG concentrations by week 12 and maintained these concentrations through at least 72 weeks of treatment. Five of the 10 patients received a total of 60 albumin transfusions in the 48 weeks prior to treatment. In the 48 weeks after starting treatment, one patient received one albumin transfusion. Nine of the 10 patients were hospitalized for a total of 268 days in the 48 weeks prior to treatment. In the 48 weeks after starting treatment, two patients were hospitalized for a total of 7 days. The most common adverse reactions occurring in two or more patients included upper respiratory tract infection, fracture, urticaria, and alopecia. About Veopoz Veopoz was invented using Regeneron’s proprietary VelocImmune® technology and is a fully human, monoclonal antibody designed to block the activity of complement factor C5 and prevent diseases mediated by the complement pathway. It is an IgG4 antibody that binds with high affinity to wild-type and variant human C5. As part of its ongoing development program, Veopoz is also being evaluated in combination with Alnylam’s cemdisiran (siRNAi C5 inhibitor) as an investigational combination therapy for the treatment of other complement-mediated disorders including paroxysmal nocturnal hemoglobinuria (PNH) and myasthenia gravis (MG). This combination is currently under clinical development, and its safety and efficacy have not been evaluated by any regulatory authority. About Regeneron's VelocImmune® Technology Regeneron's VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron's co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved fully human monoclonal antibodies currently available. This includes REGEN-COV® (casirivimab and imdevimab), Dupixent® (dupilumab), Libtayo® (cemiplimab-rwlc), Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb) and Inmazeb® (atoltivimab, maftivimab and odesivimab-ebgn). About Regeneron Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led for 35 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous FDA-approved treatments and 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, 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. The content above comes from the network. if any infringement, please contact us to modify.