Single administration of CIDEB<\/strong>siRNA<\/strong><\/sup> resulted in rapid, deep and durable reductions in liver fat by up to 80% that were sustained through 36 weeks<\/strong><\/p>\n Data from first-in-human trial were shared in oral presentation at AASLD The Liver Meeting<\/strong>\u00ae <\/strong><\/sup>and simultaneously published in <\/strong>The New England Journal of Medicine<\/em><\/strong><\/p>\n Regeneron to host virtual \u201cRegeneron Roundtable\u201d investor event to discuss its MASH pipeline programs on Tuesday, November 10 at 8:30 a.m. ET<\/strong><\/p>\n TARRYTOWN, N.Y., Nov. 08, 2026 (GLOBE NEWSWIRE) — Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the first results from a first-in-human trial that demonstrated a single subcutaneous dose of CIDEBsiRNA<\/sup> rapidly normalized liver fat and improved liver injury markers in patients with metabolic dysfunction-associated steatotic liver disease (MASLD) with effects that were sustained through week 36. CIDEBsiRNA<\/sup> is an investigational, first-in-class, liver-targeted small interfering RNA (siRNA) therapeutic that mimics the protective effect of rare mutations in the CIDEB <\/em>gene, which were discovered<\/a> by the Regeneron Genetics Center\u00ae<\/sup>. The results were published<\/a> by The New England Journal of Medicine <\/em>and simultaneously presented in an oral session at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting\u00ae<\/sup> 2026.<\/p>\n \u201cPrior advances in steatotic liver disease have begun to make a difference, but there is so much more progress to be made to help patients normalize liver fat and reverse liver damage,\u201d said J\u00f6rg T\u00e4ubel, M.D., F.F.P.M., F.E.S.C., Executive Chairman of Richmond Pharmacology, Visiting Professor at King\u2019s College London, Honorary Senior Research Fellow at St. George\u2019s University in London, President of The Association for Applied Human Pharmacology and a lead investigator for the trial. \u201cThese results show CIDEBsiRNA<\/sup> resulted in rapid, deep and sustained reductions in liver fat with improvements in markers of liver injury. With effects out to 36 weeks that support the potential for twice-yearly dosing, alongside the safety profile, the findings highlight CIDEBsiRNA<\/sup> as a promising new therapeutic for MASH and other steatotic liver diseases.\u201d<\/p>\n MASLD occurs when excess fat accumulates in the liver and encompasses a spectrum of conditions ranging from simple fatty liver to the more serious disease of metabolic dysfunction-associated steatohepatitis (MASH). MASH is a progressive, fibrotic liver disease that affects approximately 13 million U.S. adults, which is characterized by inflammation and cell damage that can lead to cirrhosis, liver transplant, liver cancer and death if not properly managed. Historical clinical trial data report that currently approved MASH therapies have reached mean liver fat reductions of 47-57% at approximately one year.1,2<\/sup><\/p>\n A first-in-human, Phase 1\/2 trial evaluated a single subcutaneous dose of CIDEBsiRNA<\/sup> at four dose levels (n=30 for CIDEBsiRNA<\/sup> groups) versus placebo (n=10) in adults with MASLD. The primary goal of the trial was to evaluate safety and tolerability, with liver fat and markers of liver injury evaluated as secondary endpoints. Meta-analyses of clinical trials of patients with MASH indicate that the depth of liver fat reduction correlates with liver biopsy response (e.g., MASH resolution and fibrosis improvement), supporting changes in liver fat as an informative, early pharmacodynamic readout.<\/p>\n Results for CIDEBsiRNA<\/sup> recipients showed:<\/p>\n Overall rates of adverse events (AEs) were generally similar between the CIDEBsiRNA<\/sup> and placebo groups. All AEs were mild or moderate in severity, except for a severe, serious event of hyperglycemia in a CIDEBsiRNA<\/sup>-treated participant that was not attributed to study drug (instead later attributed to a diagnosis of adult-onset Type 1 diabetes). There were no trial discontinuations and no nausea or vomiting AEs reported in the CIDEBsiRNA<\/sup> groups, addressing a key tolerability limitation associated with other therapies.<\/p>\n Longer-term safety and histologic outcomes are being evaluated in multiple, ongoing trials, including Phase 2a<\/a>, Phase 2b<\/a> and Phase 3<\/a> trials that are now recruiting. Regeneron is also investigating additional drug candidates informed by human genetics for the treatment of steatotic liver diseases.<\/p>\n The potential uses of CIDEBsiRNA<\/sup> described above are investigational, and its safety and efficacy have not been evaluated by any regulatory authority.<\/p>\n About the \u201cRegeneron Roundtable\u201d Investor Event<\/strong> Links to the webcast and to register via telephone may be accessed from the \u2018Investors and Media\u2019 page of Regeneron\u2019s website at https:\/\/investor.regeneron.com\/events-and-presentations<\/a>. 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\u2019s website for at least 30 days.<\/p>\n About CIDEB<\/strong>siRNA<\/strong><\/sup>, the trial and the Regeneron MASLD\/MASH Program<\/strong> The ongoing Phase 1\/2 randomized, double-blind, placebo-controlled trial is evaluating the safety and tolerability of CIDEBsiRNA<\/sup> in adult participants with MASLD and MASH. The primary goal of the trial is to evaluate safety and tolerability with efficacy evaluated as a secondary endpoint through measures such as change in liver fat fraction by MRI-PDFF.<\/p>\n Scientists at the Regeneron Genetics Center\u00ae<\/sup>\u202f(RGC) analyzed genetic data to discover and\/or validate genetic variants in the CIDEB <\/em>and other genes that are associated with liver disease risk. Building on these findings, Regeneron collaborated with Alnylam to generate an siRNA targeting CIDEB<\/em> and has the exclusive rights for the development of and commercialization of CIDEBsiRNA<\/sup>; Alnylam receives milestone and royalty payments.<\/p>\n About Regeneron<\/strong> Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite<\/em>\u00ae<\/em><\/sup>, which produces optimized fully human antibodies and new classes of bispecific antibodies.\u202fWe are shaping the next frontier\u00a0of medicine with data-powered insights from the\u202fRegeneron Genetics Center\u00ae<\/sup>\u202fand pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases.<\/p>\n For more information, please visit\u00a0www.Regeneron.com<\/a>\u00a0or follow Regeneron on LinkedIn<\/a>, Instagram<\/a>, Facebook<\/a> or X<\/a>.<\/p>\n Forward-Looking Statements and Use of Digital Media<\/strong> 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 (<\/em>https:\/\/investor.regeneron.com<\/em><\/a>) and its LinkedIn page (<\/em>https:\/\/www.linkedin.com\/company\/regeneron-pharmaceuticals<\/em><\/a>).<\/em><\/p>\n 1<\/sup> Harrison SA, Bedossa P, Guy CD, et al. A Phase 3, Randomized, Controlled Trial of Resmetirom in NASH with Liver Fibrosis. N Engl J Med<\/em>. 2024;390(6):497-509. doi:10.1056\/NEJMoa2309000. Single administration of CIDEBsiRNA resulted in rapid, deep and durable reductions in liver fat by up to 80% that were sustained through 36 weeks Data from first-in-human trial were shared in oral presentation at AASLD The Liver Meeting\u00ae and simultaneously published in The New England Journal of Medicine Regeneron to host virtual \u201cRegeneron Roundtable\u201d investor […]<\/p>\n","protected":false},"author":3,"featured_media":434680,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"site-sidebar-layout":"default","site-content-layout":"","ast-site-content-layout":"default","site-content-style":"default","site-sidebar-style":"default","ast-global-header-display":"","ast-banner-title-visibility":"","ast-main-header-display":"","ast-hfb-above-header-display":"","ast-hfb-below-header-display":"","ast-hfb-mobile-header-display":"","site-post-title":"","ast-breadcrumbs-content":"","ast-featured-img":"","footer-sml-layout":"","ast-disable-related-posts":"","theme-transparent-header-meta":"","adv-header-id-meta":"","stick-header-meta":"","header-above-stick-meta":"","header-main-stick-meta":"","header-below-stick-meta":"","astra-migrate-meta-layouts":"default","ast-page-background-enabled":"default","ast-page-background-meta":{"desktop":{"background-color":"var(--ast-global-color-4)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"ast-content-background-meta":{"desktop":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"tablet":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""},"mobile":{"background-color":"var(--ast-global-color-5)","background-image":"","background-repeat":"repeat","background-position":"center center","background-size":"auto","background-attachment":"scroll","background-type":"","background-media":"","overlay-type":"","overlay-color":"","overlay-opacity":"","overlay-gradient":""}},"fifu_image_url":"https:\/\/ml.globenewswire.com\/Resource\/Download\/add5ebc7-88c9-49dc-8834-abbb07c622d1","fifu_image_alt":"","footnotes":""},"categories":[57166],"tags":[],"class_list":["post-434679","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-globenewswire"],"acf":[],"_links":{"self":[{"href":"https:\/\/dailybanglanewspapers.com\/news\/wp-json\/wp\/v2\/posts\/434679","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dailybanglanewspapers.com\/news\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dailybanglanewspapers.com\/news\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dailybanglanewspapers.com\/news\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/dailybanglanewspapers.com\/news\/wp-json\/wp\/v2\/comments?post=434679"}],"version-history":[{"count":0,"href":"https:\/\/dailybanglanewspapers.com\/news\/wp-json\/wp\/v2\/posts\/434679\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dailybanglanewspapers.com\/news\/wp-json\/wp\/v2\/media\/434680"}],"wp:attachment":[{"href":"https:\/\/dailybanglanewspapers.com\/news\/wp-json\/wp\/v2\/media?parent=434679"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dailybanglanewspapers.com\/news\/wp-json\/wp\/v2\/categories?post=434679"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dailybanglanewspapers.com\/news\/wp-json\/wp\/v2\/tags?post=434679"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}\n
Regeneron will host a virtual investor event to discuss its cardiometabolic and MASH pipeline programs on Tuesday, November 10 at 8:30 a.m. ET. This is the next webcast in an investor event series called the \u201cRegeneron Roundtable,\u201d intended to highlight programs from the company\u2019s innovative investigational pipeline.<\/p>\n
CIDEBsiRNA<\/sup> is an investigational, first-in-class, liver-targeted siRNA therapeutic that targets a receptor exclusively expressed in the liver to mimic the protective effect of naturally occurring loss-of-function and missense CIDEB<\/em> mutations in a broad population of people with MASLD and MASH.<\/p>\n
Regeneron\u00a0(NASDAQ: REGN) is a leading biotechnology company that invents,\u00a0develops\u00a0and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to\u00a0repeatedly and consistently translate science\u00a0into medicine has led to numerous approved treatments and\u202fproduct candidates in development, most 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, neurological diseases, hematologic conditions, infectious\u00a0diseases, and rare diseases.\u00a0<\/p>\n
This press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (\u201cRegeneron\u201d or the \u201cCompany\u201d), and actual events or results may differ materially from these forward-looking statements. Words such as \u201canticipate,\u201d \u201cexpect,\u201d \u201cintend,\u201d \u201cplan,\u201d \u201cbelieve,\u201d \u201cseek,\u201d \u201cestimate,\u201d 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, \u201cRegeneron\u2019s Products\u201d) and product candidates being developed by Regeneron and\/or its collaborators or licensees (collectively, \u201cRegeneron\u2019s Product Candidates\u201d) and research and clinical programs now underway or planned, <\/em>including <\/em>without limitation<\/em> CIDEB<\/em>siRNA<\/em><\/sup> (also known as ALN-CIDEB)<\/em>; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron\u2019s Product Candidates and new indications for Regeneron\u2019s Products, such as <\/em>CIDEB<\/em>siRNA<\/em><\/sup> for the treatment of metabolic dysfunction-associated steatotic liver disease as discussed in this press release as well as the other clinical programs for the treatment of metabolic dysfunction-associated steatohepatitis discussed or referenced in this press release; uncertainty of the utilization, market acceptance, and\/or commercial success of Regeneron\u2019s Products and Regeneron\u2019s 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\u2019s Products and Regeneron\u2019s Product Candidates (such as <\/em>CIDEB<\/em>siRNA<\/em><\/sup>); the ability of Regeneron\u2019s collaborators, licensees, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron\u2019s Products and Regeneron\u2019s Product Candidates; the ability of Regeneron to manage supply chains for multiple products and product candidates and risks associated with tariffs and other trade restrictions; safety issues resulting from the administration of Regeneron\u2019s Products and Regeneron\u2019s Product Candidates (such as <\/em>CIDEB<\/em>siRNA<\/em><\/sup>) <\/em>i<\/em>n patients, including serious complications or side effects in connection with the use of Regeneron\u2019s Products and Regeneron\u2019s Product Candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron\u2019s ability to continue to develop or commercialize Regeneron\u2019s Products and Regeneron\u2019s Product Candidates; ongoing regulatory obligations and oversight impacting Regeneron\u2019s Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement or copay assistance for Regeneron\u2019s 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\u2019s pricing strategy, including in connection with Regeneron\u2019s April 2026 agreements with the U.S. government; other changes in laws, regulations, and policies affecting the healthcare industry; competing products and product candidates (including biosimilar products) that may be superior to, or more cost effective than, Regeneron\u2019s Products and Regeneron\u2019s 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\u2019s agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable) as well as Regeneron’s license agreement with Alnylam Pharmaceuticals, Inc. referenced in this press release, 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 the Company or other parties and pending or future litigation or patent office proceedings relating thereto (including without limitation patent litigation and other related proceedings relating to Regeneron\u2019s Products), the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron\u2019s business, prospects, operating results, and financial condition. A more complete description of these and other material risks can be found in Regeneron\u2019s filings with the U.S. Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2025 and its Form 10-Q for the quarterly period ended September 30, 2026. Any forward-looking statements are made based on management\u2019s 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.<\/em><\/p>\n
2 <\/sup>Flint A, Andersen G, Hockings P, et al. Randomised clinical trial: semaglutide versus placebo reduced liver steatosis but not liver stiffness in subjects with non-alcoholic fatty liver disease assessed by magnetic resonance imaging. Aliment Pharmacol Ther<\/em>. 2021;54(9):1150-1161. doi:10.1111\/apt.16608.<\/p>\n\n <\/div>\n","protected":false},"excerpt":{"rendered":"