LIB Therapeutics Announces Results from Late Breaking Science Presentation at 2025 American Heart Association Meeting in New Orleans November 9, 2025




LIB Therapeutics Announces Results from Late Breaking Science Presentation at 2025 American Heart Association Meeting in New Orleans November 9, 2025

CINCINNATI–(BUSINESS WIRE)–LIB Therapeutics Inc. (LIB), a privately-held, late-stage biopharmaceutical company advancing Lerodalcibep (Lerochol®), a novel, monthly, small dose third-generation PCSK9 inhibitor today announced results from Late Breaking Science presentations at the November 7-10th 2025 American Heart Association meeting in New Orleans.

• Long-term Efficacy and Safety of Lerodalcibep in the Open-label 72-week Extension Study of Subjects Previously on Inclisiran or Lerodalcibep in the LIBerate-VI Trial – Dr Evan Stein MD PhD November 9th

• Lerodalcibep demonstrated consistent long-term efficacy in those continued on the drug and was well tolerated, with no treatment related serious adverse events.
• 72-week primary endpoint; mean & absolute LDL-C reductions of 59.2% & 66.6 mg/dL respectively.
• Mean ApoB was reduced by 45.3% and median Lp(a) by 35.5%.
• Subjects switched from Inclisiran to Lerodalcibep had:

• Substantial additional LDL-C reductions from the end of the base trial of ~40% through week 48 and 30% lower through week 72.
• Additional large reductions in Apo B and Lp(a) and
• Significant increase in subjects achieving guideline LDL-C targets.

“The study is the first to assess a plasma binding PCSK9 inhibitor, Lerodalcibep, in patients switched from a hepatic PCSK9 synthesis inhibitor and which showed synergistic efficacy, further reducing LDL-C, Apo B and Lp(a) and increased patients ability to achieve the new more stringent LDL-C guideline targets,” said Dr. Evan Stein MD PhD, Chief Executive and Scientific Officer of LIB Therapeutics and continued. “The results are consistent with combined inhibition of circulating free PCSK9 from non-hepatic and reduced hepatic synthesis and provides an additional pathway to achieving treatment lipid goals in the many patients who cannot achieve them with current therapies. Significant additional studies are required to determine the optimal dosing frequency and safety of combining these therapies.”

About Lerodalcibep

Lerodalcibep is a novel, small protein-binding, third-generation PCSK9 inhibitor, and has been developed as a more convenient, once-monthly, single small-volume, subcutaneous injection that will not require refrigeration at home or in travel. These features make Lerodalcibep a unique alternative to approved PCSK9 inhibitors. The anti-clockwise binding domain of Lerodalcibep is an 11-kDa polypeptide called an accenting, engineered for high-affinity subnanomolar binding to human PCSK9 and fused to human serum albumin to enhance plasma half-life.

In clinical trials, Lerodalcibep has demonstrated sustained LDL-C reductions of ≥60% in patients with, or at very-high or high risk of, cardiovascular disease (CVD) and ≥50% in those with heterozygous familial hypercholesterolemia (FH) who have more severe LDL-C elevations, and is expected to expand treatment options for the millions of patients around the world with CVD, including the 30 million individuals with VHF.

The global Phase 3 LIBerate program enrolled a diverse population of over 2,900 patients with CVD, without CVD at very high and high risk for CVD, including heterozygous and homozygous familial hypercholesterolemia. Lerodalcibep was dosed once monthly for up to 52 weeks in these key registration-enabling, placebo-controlled trials, and over 2,400 patients have continued in the 72-week open-label extension trial.

LIB submitted a Biologic License Application (BLA) to the U.S. Food and Drug Administration (FDA) in December 2024, and received formal filing by FDA in February with an anticipated PDUFA date on target for mid December this year. LIB is seeking approval of Lerodalcibep (LEROCHOL®) as an adjunct to diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including heterozygous familial hypercholesterolemia (HeFH). Marketing Authorization Application was submitted to, and accepted by the European Medicines Agency in May with anticipated approval in June 2026.

About LIB Therapeutics Inc.

LIB Therapeutics is a privately-held, late-stage biopharmaceutical company dedicated to bringing Lerodalcibep to the millions of patients with cardiovascular disease and to the 30 million individuals with familial hypercholesterolemia (FH) around the world, who require additional large reductions in LDL-C, despite maximally tolerated stains and other lipid lowering agents, to achieve LDL-C goals.

For more information, please visit: www.libtherapeutics.com.

Contacts

Kate Caldwell

kcaldwell@libtherapeutics.com

U.S. Hemp Roundtable Strongly Opposes Proposed Senate Language to Recriminalize Hemp




U.S. Hemp Roundtable Strongly Opposes Proposed Senate Language to Recriminalize Hemp

WASHINGTON–(BUSINESS WIRE)–#HempHelps–The U.S. Hemp Roundtable issues the following statement regarding the proposed Senate language to recriminalize hemp products, condemning the harmful decision by Congress that threatens to eliminate America’s $28.4 billion hemp industry and jeopardizes more than 300,000 American jobs. If passed, this legislation would wipe out 95% of the industry, shuttering small businesses and American farms while costing states $1.5 billion in lost tax revenue.

Despite misleading claims this language protects non-intoxicating CBD products, the reality is that more than 90% of non-intoxicating hemp-derived products contain levels of THC that are greater than the proposed cap of .4 mg per container. As a result, seniors, veterans, and many other consumers who depend on hemp for their health and well-being would be violating federal law to purchase these products, disrupting their care and leaving them scrambling for potentially harmful alternatives.

While Congress pushes forward with this hemp-killing provision, the fight is far from over. Senator Rand Paul (R-KY) is submitting an emergency amendment to strike the hemp ban from the proposed legislation, a move fully supported by the hemp industry. His leadership offers a critical lifeline to thousands of American farmers, entrepreneurs, and consumers, and the industry stands united behind his efforts.

If the language passes, as-is, the hemp industry is committed to continuing the fight. During the one-year proposed moratorium, U.S. Hemp Roundtable will work closely with lawmakers to reverse the ban and replace it with responsible, science-based regulations that crackdown on misleading and purely synthetic products, create restrictions that keep products out of the hands of children, and promote standard manufacturing practices. Unlike these regulations, the current proposal fails to protect consumers and risks fueling a dangerous black market.

“Our industry is being used as a pawn as leaders work to reopen the government. Recriminalizing hemp will force American farms and businesses to close and disrupt the wellbeing of countless Americans who depend on hemp,” said Jonathan Miller, U.S. Hemp Roundtable General Counsel. “We support Senator Rand Paul’s efforts to push back on this language and will continue to fight alongside him for a regulated, safe, and robust hemp industry.”

# # #

About the U.S. Hemp Roundtable:

The U.S. Hemp Roundtable is a coalition of dozens of leading companies and organizations committed to safe hemp and CBD products. We proudly represent the industry’s major national grassroots organizations and are leading the way forward for hemp and CBD products through education and action.

Contacts

Media Contact
news@hempsupporter.com

Hepta Unveils First Multi-Omic Atlas of MASH, Linking Liver Pathway Biology to cfDNA Methylation in Blood




Hepta Unveils First Multi-Omic Atlas of MASH, Linking Liver Pathway Biology to cfDNA Methylation in Blood

Hepta’s liquid biopsy-native AI model links coordinated methylation and gene expression changes to fibrosis progression, showing that liver biology can be measured non-invasively from blood

FOSTER CITY, Calif.–(BUSINESS WIRE)–Hepta, a biotechnology company using transformer-based AI to decode the cell-free DNA (cfDNA) epigenome and detect organ-specific signals of chronic disease, today announced the creation of an unparalleled multi-omic atlas of metabolic dysfunction–associated steatohepatitis (MASH) in collaboration with Duke University. This work builds on Hepta’s expanded cfDNA studies with Mainz University and Akero Therapeutics’ Phase 3 clinical trial program, further strengthening the company’s partnerships across academic and clinical research.

The atlas integrates single-cell and bulk data across hundreds of liver samples, including gene expression, chromatin accessibility, and DNA methylation, paired with cfDNA methylation from matched blood plasma. Presented this week at AASLD’s The Liver Meeting, the atlas reveals how coordinated methylation and transcriptional programs drive inflammation and fibrosis and demonstrates that these same molecular signals are detectable in blood using Hepta’s liquid biopsy-native AI platform.

“In liver tissue, we observe pathway activity, gene expression, and cell type composition that are mediated by methylation across disease severity,” said Anna Mae Diehl, M.D., Florence McAlister Distinguished Professor of Medicine at Duke University. “Critically, those same signatures are measurable in plasma cfDNA, indicating that cfDNA methylation can serve as a faithful mirror of liver biology. That concordance supports cfDNA methylation as a plausible, non-invasive readout of pathway activities in liver disease, offering insights far deeper than traditional biomarkers.”

The MASH atlas maps how methylation governs pathway-level activity across hepatocytes, cholangiocytes, and stromal cells in the liver. The analysis shows the role of methylation in a broad dysregulation of metabolic, inflammatory, fibrogenic, and bile-acid pathways that track with fibrosis severity, including mechanisms targeted by emerging therapeutic classes such as GLP-1, FGF-21, and THR-β agonists. The MASH atlas also demonstrates that the same pathway signatures observed in liver tissue are reproducibly detectable in cfDNA circulating in blood, creating a molecular bridge between liver-tissue biology and liquid-biopsy measurement.

This foundational work, in addition to Hepta’s expanded cfDNA results spanning across multiple independent cohorts — including Duke University, Mainz University, and Akero Therapeutics’ phase 3 clinical trial program — was featured in Dr. Jörn Schattenberg’s presentation at the Precision Liver Symposium.

“Across independent cohorts, we see strong, consistent diagnostic performance from Hepta’s cfDNA methylation platform,” said Jörn Schattenberg, M.D., Director of the Metabolic Liver Research Center at Saarland University. “What’s striking is that the same readout also delivers deep biological insights. It’s not just a yes-or-no test; these signals reveal how repair and fibrosis programs evolve, which opens the door to earlier intervention and therapy guidance.”

Hepta’s transformer-based, liquid biopsy–native AI was specifically designed to resolve the complexity of cfDNA. The model interprets billions of cfDNA fragments in context, detecting subtle, distributed patterns that correspond to tissue-level changes. By decoding the entire cfDNA epigenetic landscape, the platform enables tissue-level biological interpretation from a standard blood draw.

While traditional panel biomarkers infer fibrosis indirectly, cfDNA methylation directly reads the gene-regulatory programs driving those changes. Together with the multi-omic atlas, these data represent the largest multi-cohort validation to date of cfDNA methylation as a biomarker in MASH and demonstrate how deep multi-omics can inform clinical translation.

“Our data demonstrate that cfDNA captures the molecular fingerprint of disrupted repair,” said Hamed Amini, Ph.D., Co-founder and CEO of Hepta. “The atlas establishes the biological foundation for our technology, showing that the same pathways we observe in tissue are encoded in blood. This work lays a foundation for biology-driven detection and for future therapeutic strategies that address the mechanisms behind liver damage and fibrosis.”

Hepta’s approach extends the frontier of liquid biopsy beyond oncology, positioning cfDNA methylation as a scalable molecular window into chronic disease biology and, potentially, a snapshot of full body health. The company is expanding its clinical studies to further validate cfDNA-based biomarkers for early detection, informing treatment strategies, and longitudinal monitoring across the care continuum.

About Hepta

Hepta detects chronic disease early by using cfDNA epigenetic analysis and AI to deliver accurate, non-invasive diagnostics at population scale. Founded by former Illumina, Grail and Google scientists, the company is backed by Felicis Ventures, Illumina Ventures, and SeaX Ventures. Hepta is proving that blood-based epigenetic biomarkers can replace invasive biopsies, enabling earlier and more accessible detection of diseases like MASH and laying the foundation for future applications across chronic diseases. For more information, visit www.hepta.bio.

Contacts

Media Contact
Patrick Schmidt

Consort Partners for Hepta

pr@hepta.bio

Medincell to Join MSCI World Small Cap Index, a Leading Global Benchmark




Medincell to Join MSCI World Small Cap Index, a Leading Global Benchmark

MONTPELLIER, France–(BUSINESS WIRE)–Medincell has been selected for inclusion in the Morgan Stanley Capital International (MSCI) World Small Cap Index, which encompasses the most liquid and high-performing small-cap companies across 23 developed markets.

Joining the MSCI World Small Cap Index reflects the strength of Medincell’s business model, its growth potential, and its commitment to innovation and social responsibility.

The inclusion will enhance Medincell’s visibility among institutional investors and index-tracking funds that follow MSCI indices.

The entry will become effective at the opening of trading on 24 November 2025.

About the MSCI World Small Cap Index

• The MSCI World Small Cap Index captures small-cap representation across 23 developed market countries. With approximately 3,840 constituents, the index covers about 14% of the free float-adjusted market capitalization in each country.
• For more information: https://www.msci.com/documents/10199/255599/msci-world-small-cap-index.pdf

About Medincell

Medincell is a clinical- and commercial-stage biopharmaceutical licensing company developing long-acting injectable treatments across multiple therapeutic areas. Our innovative treatments are designed to ensure adherence to medical prescriptions, enhance the effectiveness and accessibility of medicines, and reduce their environmental impact.

These treatments combine active pharmaceutical ingredients with our proprietary BEPO^® technology, which enables controlled drug delivery at therapeutic levels for several days, weeks, or months following a subcutaneous or local injection of a small, fully bioresorbable deposit.

The first treatment based on BEPO^® technology was approved for schizophrenia by the FDA in April 2023 and is now marketed in the United States by Teva under the name UZEDY^® (BEPO^® technology is licensed to Teva under the name SteadyTeq™).

Our investigational pipeline includes numerous innovative therapeutic candidates in various stages of development, from formulation to Phase 3 clinical trials. We collaborate with leading pharmaceutical companies and foundations to advance global health through new treatment options.

Headquartered in Montpellier, France, Medincell employs over 140 people representing more than 25 nationalities.

UZEDY^® and SteadyTeq™ are trademarks of Teva Pharmaceuticals.

www.medincell.com

Contacts

David Heuzé
Head of Corporate and Financial Communications, and ESG

david.heuze@Medincell.com / +33 (0)6 83 25 21 86

Grace Kim
Chief Strategy Officer, U.S. Finance

grace.kim@medincell.com / +1 (646) 991-4023

Nicolas Mérigeau / Arthur Rouillé
Media Relations

Medincell@newcap.eu / +33 (0)1 44 71 94 94

Louis-Victor Delouvrier / Alban Dufumier
Investor Relations France

Medincell@newcap.eu / +33 (0)1 44 71 94 94

Elevance Health Foundation Invites Applications for $5 Million Patient Safety Prize Through New Community Action Leadership Initiative




Elevance Health Foundation Invites Applications for $5 Million Patient Safety Prize Through New Community Action Leadership Initiative

Applications open December 9 for innovative solutions to improve patient safety in three key areas: medication safety, fall prevention, and health literacy.

INDIANAPOLIS–(BUSINESS WIRE)–Elevance Health Foundation today announced that applications will open December 9, 2025, for its inaugural $5 million Patient Safety Prize. With a focus on improving health literacy, eliminating medication errors, and preventing patient falls, the Prize invites changemakers to submit transformational solutions that improve safety, advance quality of care, and enhance outcomes for vulnerable populations.

As part of the Community Action Leadership (CAL) initiative, the inaugural Patient Safety Prize advances the Foundation’s mission to drive community-based innovation that addresses critical health challenges. Unlike traditional grantmaking, this unique take on philanthropy will cultivate collaboration among external thought leaders while continuing to cement the Foundation’s role as a catalyst for positive change in the community.

A diverse array of nonprofits, healthcare institutions, technology companies, patient advocacy groups, and thought leaders are encouraged to apply for a share of the $5 million prize pool, which will recognize and accelerate their initiatives.

“Patient safety is one of the most pressing challenges facing our healthcare system today. Too often, preventable harm impacts already disadvantaged communities,” said Shantanu Agrawal, M.D., Chief Health Officer, Elevance Health. “Through the Community Action Leadership Patient Safety Prize initiative, we are creating a platform for bold, innovative solutions that can reduce harm, improve outcomes, and make healthcare safer, more accessible, and more responsive to the needs of every patient. We look forward to seeing the transformational solutions that emerge from this inaugural challenge.”

Patient safety events disproportionately impact socially vulnerable communities and are often preventable. The Centers for Disease Control and Prevention (CDC) reports that more than 1.5 million Americans visit emergency departments each year due to adverse drug events, while the Agency for Healthcare Research and Quality (AHRQ) estimates 700,000 to 1 million patient falls occur in U.S. hospitals annually. The Center for Health Care Strategies (CHCS) also finds that nearly nine in ten adults struggle with health literacy, increasing the risk of preventable harm.

The Patient Safety Prize initiative was announced at the National Academy of Medicine (NAM)’s November Meeting, “The Future of Patient Safety: A New Paradigm.” With support from the NAM, Elevance Health Foundation will be convening a distinguished panel of experts to evaluate Prize submissions. Building on the foundation of the NAM’s landmark work, including “To Err is Human,” the Foundation aims to advance innovative, evidence-based solutions that expand access to safer, higher-quality care and improve health care experiences for all communities.

To learn more about the Patient Safety Prize and access the RFP when it goes live on December 9, 2025, please visit www.patientsafetyprize.org. Applicants must complete their registration by March 17, 2026, and submit their proposal by April 7, 2026, to be considered.

About Elevance Health Foundation

Elevance Health Foundation is the philanthropic arm of Elevance Health Inc. The Foundation works to improve the health of the socially vulnerable through partnerships and programs in our communities with an emphasis on maternal-infant health; behavioral health; and food as medicine. Through its key areas of focus, the Foundation also strategically aligns with Elevance Health’s focus on community health and becoming a lifetime, trusted health partner that is fueled by its purpose to improve the health of humanity. To learn more about Elevance Health Foundation, please visit www.elevancehealth.foundation or follow us @ElevanceFND on X and Elevance Health Foundation on Facebook.

Contacts

Media Contact
Shelby Kaiser

Elevance Health Foundation

shelby.kaiser@elevancehealth.com

Abselion Launches His-tagged Protein Quantification Kit and Sensors for Amperia at PEGS Europe 2025




Abselion Launches His-tagged Protein Quantification Kit and Sensors for Amperia at PEGS Europe 2025

• Ready-to-use solution, developed in collaboration with GenScript, offers rapid and reliable quantification of His-tagged proteins
• Broadens Amperia’s applications across recombinant protein research, screening, and development
• Abselion to showcase kit alongside Amperia platform at PEGS Europe in Lisbon, Portugal from 11–13 November

CAMBRIDGE, England–(BUSINESS WIRE)–#Abselion–Abselion, a pioneering life sciences technology company focused on simplifying biomolecule quantification, has expanded its Amperia™ assay portfolio with the launch of its Tagged Protein Quantification Kit | His-tag and Tagged Protein Sensor | His-tag Quantification. The novel assay, developed with GenScript’s THE™ His Tag Monoclonal Antibodies, provides researchers with a ready-to-use solution for rapid, reliable measurement of His-tagged proteins. This launch marks a significant milestone in extending Amperia, beyond antibody and AAV assays, into the wider field of recombinant protein analysis, enabling researchers to generate high-quality data more quickly and dependably across expression, optimisation, and development workflows.

His-tags are among the most widely used affinity tags, supporting purification and detection of recombinant proteins expressed in bacterial, insect, and mammalian systems. Quantification often involves ELISA, gels, or spectrophotometric methods, which can be time-consuming, variable, or less effective, particularly when working with crude samples. Abselion’s Tagged Protein Quantification Kit provides a fast, reproducible, and ready-to-use alternative for drug discovery, structural biology, therapeutic research, and bioprocess development workflows.

Abselion’s Amperia benchtop platform is a simple-to-use solution that uses redox electrochemical detection for rapid and accurate automated quantification of a range of biomolecules, without optics or fluidics. The new assay runs in a premix competition format, within Amperia’s intuitive, software-controlled workflow, to quantify His-tagged proteins directly from crude or purified samples without specialist training.

Each kit includes pre-coated sensor strips, together with assay plates, detection reagents, and buffers in a complete, ready-to-use package. At the core of the new kit are electrochemical sensor strips pre-coated with GenScript’s validated antibodies, immobilised on the sensor surface. These high affinity antibodies offer broad reactivity across common His-tag variants and expression systems for enhanced assay robustness. Incorporating these directly onto the strip reduces manual handling steps and the need for additional assay development or optimisation, improving usability and supporting more consistent performance.

In parallel to the complete Tagged Protein Quantification Kit | His-tag for guided, automated workflows, Abselion has also launched the Tagged Protein Sensor | His-tag Quantification as a standalone consumable pack. This provides direct access to the antibody-coated sensor strips, offering additional flexibility for a wide range of His-tag assays on the Amperia platform.

Dr Ruizhi Wang, CEO and Founder, Abselion, said: “The His-tag launch is one of the first affinity-based, electrochemical, automated kits for crude samples and marks an important step in extending Amperia into one of the most widely used areas of protein science. By combining GenScript’s trusted antibody technology with our ready-to-use design, we are giving researchers faster, more dependable ways to quantify His-tagged proteins directly from complex samples. Together with the flexibility of a standalone sensor option, this reflects Abselion’s mission to make high-quality protein quantification more practical and accessible to meet the needs of scientists across research, development and bioprocessing.”

Higgins Qin, Senior Director, Protein & Antibody R&D, GenScript, added: “Our THE His Tag Monoclonal Antibodies are recognised for their high affinity and broad applicability across recombinant protein workflows. Incorporating these into Abselion’s Amperia system provides researchers with a solution that simplifies His-tag quantification and supports consistent measurement from crude through to purified samples. We are pleased to see GenScript’s trusted antibody technology applied in an innovative format that makes protein analysis more reliable and accessible.”

For further information about Abselion’s Tagged Protein Quantification Kit | His-tag and Tagged Protein Sensor | His-tag, please visit: https://www.abselion.com/assay-kits and https://www.abselion.com/sensors/. To download the application notes “Enabling His-tagged Protein Analysis in Expression Workflows” and “Use Case: His-tag Protein Expression Screening from Crude Lysates” or case study “VIB Nanobody Core validates Amperia™ for rapid his-tag nanobody quantification”, please visit: https://www.abselion.com/resources/.

Meet the Abselion team at PEGS Europe from 11–13 November in Lisbon, Portugal (Booth #416) to learn more.

Please contact Codon Communications for high-resolution images.

Contacts

Codon Communications
Dr Michelle Ricketts

Tel: +44 7789 053885

Email: michelle.ricketts@codoncommunications.com

Vir Biotechnology Announces AASLD The Liver Meeting® Presentation & New England Journal of Medicine Publication of Phase 2 Data Demonstrating Tobevibart & Elebsiran Combination Deliver High Rates of Undetectable HDV RNA with Favorable Safety Profile




Vir Biotechnology Announces AASLD The Liver Meeting® Presentation & New England Journal of Medicine Publication of Phase 2 Data Demonstrating Tobevibart & Elebsiran Combination Deliver High Rates of Undetectable HDV RNA with Favorable Safety Profile

• SOLSTICE trial data demonstrate that 66% of chronic hepatitis delta participants receiving a monthly dose of tobevibart and elebsiran achieved undetectable HDV RNA at Week 48
• Combination well-tolerated: No grade 3 or higher treatment-related adverse events and no treatment-related discontinuations
• ECLIPSE registrational program evaluating the combination of tobevibart and elebsiran for chronic hepatitis delta fully underway, with topline data expected in the first quarter of 2027
• Data presented at AASLD The Liver Meeting® and simultaneously published in the New England Journal of Medicine

SAN FRANCISCO–(BUSINESS WIRE)–Vir Biotechnology, Inc. (Nasdaq: VIR) today announced that Week 48 endpoint analysis from the Company’s Phase 2 SOLSTICE trial for chronic hepatitis delta (CHD) demonstrated that participants receiving a monthly dose of the combination of tobevibart and elebsiran achieved robust and sustained rates of hepatitis delta virus (HDV) RNA target not detected (TND), including those participants with cirrhosis and high baseline HDV RNA. The combination also showed alanine aminotransferase (ALT) reductions over time and a favorable safety profile. These data were presented in an oral session at the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting ^®, in Washington, D.C., and simultaneously published in the New England Journal of Medicine.

“Achieving undetectable HDV RNA is a key endpoint in clinical trials, and HDV RNA undetectability is associated with better outcomes for people living with chronic hepatitis delta,” said Tarik Asselah, M.D., Ph.D., Professor of Hepatology at the Hôpital Beaujon, APHP, Clichy, France, and at the University of Paris-Cité, and Head of the unit Viral Hepatitis UMR1149 at INSERM, France. “The combination of tobevibart and elebsiran has consistently demonstrated impressive hepatitis delta virologic suppression in the SOLSTICE Phase 2 trial, and these 48-week data are encouraging as they continue to support its potential to deliver meaningful patient benefit.”

Data demonstrate that 66% (21/32) of participants with CHD receiving a monthly dose of the combination of tobevibart and elebsiran achieved and sustained HDV RNA TND at 48 weeks. Additionally, approximately 90% of participants achieved reduction in hepatitis B surface antigen (HBsAg) to values <10 IU/mL by Week 48. HBsAg reduction indicates suppression of the fundamental biologic mechanisms that HDV requires for viral replication. ALT was normalized in 56% (18/32) of participants by Week 48. The combination was well-tolerated, with no grade 3 or higher treatment-related adverse events and no treatment-related discontinuations. Most treatment-related adverse events were generally mild to moderate and transient.

The combination of tobevibart and elebsiran is currently being evaluated in Vir Biotechnology’s ECLIPSE registrational program for the treatment of CHD, which includes three randomized, controlled trials. ECLIPSE 1 has completed enrollment ahead of the Company’s expectations. Topline results from ECLIPSE 1, 2, and 3 are expected in the first quarter of 2027.

“The 48-week data from the SOLSTICE Phase 2 trial presented at AASLD’s The Liver Meeting® reinforce our confidence that a monthly dose of the combination of tobevibart and elebsiran can deliver meaningful patient benefit with convenient dosing, and I am proud to see the caliber of our data recognized by our publication in the prestigious New England Journal of Medicine,” said Marianne De Backer, M.Sc., Ph.D., MBA, Chief Executive Officer, Vir Biotechnology. “We are committed to advancing our registrational ECLIPSE program efficiently with the goal of addressing the critical unmet needs of the hepatitis delta community.”

CHD is the most severe form of chronic viral hepatitis,¹ recently classified as carcinogenic by the International Agency for Research on Cancer.² People living with the disease rapidly progress to cirrhosis, liver failure³ and liver-related death.¹ There are currently no approved treatments in the U.S., and options are limited in the European Union and globally. The objective is to eliminate the virus, and tobevibart in combination with elebsiran offers the potential to achieve this by tackling the viral lifecycle through multiple mechanisms.

The significant unmet need in CHD and the potential for the combination of tobevibart and elebsiran to provide a much-needed treatment option has been recognized by the U.S. Food and Drug Administration (FDA) with Breakthrough Therapy and Fast Track designations, and by the European Medicines Agency (EMA) with Priority Medicines (PRIME) and orphan drug designations.

About the ECLIPSE Registrational Program

ECLIPSE is a registrational program to evaluate the safety and efficacy of tobevibart in combination with elebsiran in patients with chronic hepatitis delta (CHD). ECLIPSE includes three randomized, controlled trials designed to evaluate the combination therapy in comparison to deferred treatment or bulevirtide. ECLIPSE 1 (NCT06903338) is a Phase 3 trial evaluating the safety and efficacy of tobevibart in combination with elebsiran compared to deferred treatment in the U.S. or other regions where bulevirtide use is limited. ECLIPSE 2 (NCT07128550) is a Phase 3 trial that will evaluate the efficacy and safety of switching to tobevibart and elebsiran in people with CHD who have not achieved viral suppression with bulevirtide therapy. ECLIPSE 1 and 2 are designed to provide the registrational efficacy and safety data needed for potential submission to global regulatory agencies. ECLIPSE 3 (NCT07142811) is a Phase 2b head-to-head trial to evaluate tobevibart and elebsiran compared with bulevirtide in bulevirtide-naïve patients, and it is designed to provide important supportive data to help establish access and reimbursement in key markets.

About Tobevibart and Elebsiran

Tobevibart is an investigational broadly neutralizing monoclonal antibody targeting the hepatitis B surface antigen (HBsAg). It is designed to inhibit the entry of hepatitis B and hepatitis delta viruses into hepatocytes and to reduce the level of circulating viral and subviral particles in the blood. Tobevibart was identified using Vir Biotechnology’s proprietary monoclonal antibody discovery platform. The Fc domain has been engineered to increase immune engagement and clearance of HBsAg immune complexes and incorporates Xencor’s Xtend™ technology to extend half-life. Tobevibart is administered subcutaneously, and it is currently in clinical development for the treatment of patients with chronic hepatitis delta.

Elebsiran is an investigational hepatitis B virus-targeting small interfering ribonucleic acid (siRNA) discovered by Alnylam Pharmaceuticals, Inc. It is designed to degrade hepatitis B virus RNA transcripts and limit the production of hepatitis B surface antigen. Current data indicate that it has the potential to have direct antiviral activity against hepatitis B virus and hepatitis delta virus. Elebsiran is administered subcutaneously, and it is currently in clinical development for the treatment of patients with chronic hepatitis delta.

About Vir Biotechnology, Inc.

Vir Biotechnology, Inc. is a clinical-stage biopharmaceutical company focused on powering the immune system to transform lives by discovering and developing medicines for serious infectious diseases and cancer. Its clinical-stage portfolio includes programs for chronic hepatitis delta and multiple dual-masked T-cell engagers across validated targets in solid tumor indications. Vir Biotechnology also has a preclinical portfolio of programs across a range of infectious diseases and oncologic malignancies. Vir Biotechnology routinely posts information that may be important to investors on its website.

References:

¹ NIH National Institute of Diabetes and Digestive and Kidney Diseases Hepatitis D – NIDDK (nih.gov), accessed September 2025

² Karagas, Margaret R et al., Carcinogenicity of hepatitis D virus, human cytomegalovirus, and Merkel cell polyomavirus, The Lancet Oncology, Volume 26, Issue 8, 994 – 995.

³ CDC What is Hepatitis D – FAQ | CDC, accessed September 2025

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “should,” “could,” “may,” “might,” “will,” “plan,” “potential,” “aim,” “expect,” “anticipate,” “promising” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements regarding: the therapeutic potential of the combination of tobevibart and elebsiran to treat CHD and Vir Biotechnology’s belief that monthly dosing of the combination can deliver meaningful patient benefit and address the critical unmet needs of the hepatitis delta community; Vir Biotechnology’s clinical development plans and expectations for the ECLIPSE Phase 3 registrational program, including protocols for and enrollment into ongoing and planned clinical trials, target endpoints and data readouts (including the expectation of topline data for all three trials in the first quarter of 2027); Vir Biotechnology’s strategy and plans; and any assumptions underlying any of the foregoing. Many factors may cause differences between current expectations and actual results, including, without limitation: unexpected safety or efficacy data or results observed during clinical studies or in data readouts, including the occurrence of adverse safety events; risks of unexpected costs, delays or other unexpected hurdles; challenges in accessing manufacturing capacity; clinical site activation rates or clinical enrollment rates that are lower than expected; the timing and outcome of Vir Biotechnology’s planned interactions with regulatory authorities, as well as general difficulties in obtaining any necessary regulatory approvals; successful development and/or commercialization of alternative product candidates by Vir Biotechnology’s competitors, as well as changes in expected or existing competition; geopolitical changes or other external factors; and unexpected litigation or other disputes. In light of these risks and uncertainties, the events or circumstances referred to in the forward-looking statements may not occur. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical studies may not be indicative of full results or results from later stage or larger scale clinical studies and do not ensure regulatory approval. The actual results may vary from the anticipated results, and the variations may be material. You are cautioned not to place undue reliance on any scientific data presented or these forward-looking statements, which are based on Vir Biotechnology’s available information, expectations and assumptions as of the date of this press release. Other factors that may cause Vir Biotechnology’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Vir Biotechnology’s filings with the U.S. Securities and Exchange Commission, including the section titled “Risk Factors” contained therein. Except as required by law, Vir Biotechnology assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.

Contacts

Media Contact
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Virion Therapeutics Reports Progress Towards HBV Functional Cure with Sustained and Continued HBsAg Declines up to One Year After a Single VRON-0200 Dose From its Phase 1b Study at AASLD’s The Liver Meeting® 2025




Virion Therapeutics Reports Progress Towards HBV Functional Cure with Sustained and Continued HBsAg Declines up to One Year After a Single VRON-0200 Dose From its Phase 1b Study at AASLD’s The Liver Meeting® 2025

Highlights from the Data Presentation

• VRON-0200 was safe and well tolerated, with no serious treatment-related adverse events, treatment discontinuations, or treatment-related clinical laboratory abnormalities reported
• In the majority of patients (83%; 19/23), a single intramuscular VRON-0200 dose, added to standard of care antiviral therapy, induced anti-HBV immune activation and restoration, with HBsAg declines beginning at Day 28; these HBsAg declines were sustained and/or continued to decline up to one year after VRON-0200 treatment, with 47% of patients (9/19) achieving greater than a 50% reduction (4 patients had a 1 log₁₀ IU/mL or greater decline) at Day 360
• A single VRON-0200 prime dose, followed by the addition of monthly doses of investigational antivirals initiated 28 days later, produced rapid and profound HBsAg declines (<2 IU/mL) in all patients (n=7); at Week 20, three of six patients achieved complete HBsAg loss, one within 7 days of the first combination dose
• The “Spark and Fan” model where an upfront VRON-0200 “spark” dose “primes” an anti-HBV immune response which is then “fanned” (aka boosted) by an antiviral regimen that removes the virus (e.g., HBsAg), could make VRON-0200 the foundational backbone agent to a wide range of future Functional Cure treatments

PHILADELPHIA–(BUSINESS WIRE)–Virion Therapeutics, LLC, a clinical-stage biotechnology company, developing novel T cell-based immunotherapies that utilize checkpoint modifiers, today announced at AASLD’s The Liver Meeting^®, in Washington DC, that a single intramuscular dose of VRON-0200, its novel, first-in-class, immunotherapy for HBV Functional Cure, induced HBV-specific immune activation, restoration, and HBsAg declines, that were sustained and/or continued up to one-year post dosing, in the majority of chronically HBV-infected treated patients. The data, presented as an oral presentation, by Dr. Grace Wong, M.D., from the Chinese University of Hong Kong, also highlighted VRON-0200’s ongoing favorable safety and tolerability profile, and rapid and profound HBsAg declines when VRON-0200 was combined with an investigational antiviral regimen.

Professor Grace Wong, M.D., from the Chinese University of Hong Kong, and one of the study investigators commented: “Current and investigational HBV Functional Cure treatments have been limited by their inability to restore a patient’s own immune responses against the virus. As a result, once treatment is discontinued, and the antiviral agents are no longer present, viral rebound typically occurs. What makes these data so exciting is that not only was a single, well tolerated VRON-0200 dose, alone, able to restore broad anti-HBV immune responses in the majority of chronically HBV-infected patients, but these responses were sustained, and/or improved, up to one year after end of treatment. VRON-0200’s ability to restore a patient’s own anti-HBV responses, with the potential for sustained viral control after antiviral treatment ends, is a significant advance for the field and opens up a wide range of possible future Functional Cure treatment options.”

“These new VRON-0200 clinical study data highlight its potential as a key component in future HBV Functional Cure regimens,” said Dr. Sue Currie, COO of Virion, and one of the study authors. “Off treatment viral rebound has been the “Achilles heel” for every HBV Functional Cure regimen to date. A single VRON-0200 dose alone was able to “Spark” a new and sustained anti-HBV response that could offer a solution to treatment rebound. This novel and exciting approach, where a patient’s own immune response is “sparked” (i.e. primed) with VRON-0200, and then “fanned” (aka boosted) by the removal of the HBV virus, position VRON-0200 to be the foundational backbone agent for new combination therapies that could produce meaningful Functional Cure rates for the almost 260 million persons living with chronic HBV worldwide. A Phase 2b SPARK-B trial for HBV Functional Cure is in development and will use the “Spark and Fan” approach to evaluate VRON-0200 in combination with an investigational antiviral.”

Professor Ed Gane, M.D., from the University of Auckland, and one of the study investigators, noted: “Sustained viral control after finite treatment is difficult to achieve with current HBV Functional Cure regimens, including those containing pegylated interferon. What is particularly exciting about these VRON-0200 data are that, in the majority of patients, most infected at birth, VRON-0200 was not only able to activate and restore an HBV-specific immune response, but also, these HBsAg responses were sustained and further reduced even one year after end of VRON-0200 dosing. These sustained anti-HBV immune responses, after finite treatment, could provide a solution for long-term prevention of viral rebound and ultimately get us closer to meaningful Functional Cure rates.”

The presentation is available for download at www.VirionTx.com and more details of this study can be found at ClinicalTrials.gov (Identifier: NCT06070051).

About Chronic Hepatitis B

Despite a preventative vaccine, cases of chronic hepatitis B (CHB) continue to rise, with an estimated 254 million persons infected worldwide and 1.1 million deaths per year from HBV-related liver complications. Chronic HBV remains a global health issue with a high unmet medical need, since there is no cure available. The current standard of care requires lifelong antiviral therapy to maintain control of the virus.

About VRON-0200

VRON-0200 is an investigational therapeutic immunotherapy designed with the goal of providing a functional cure for chronic HBV infection. Clinical data from an ongoing Phase 1b trial have shown VRON-0200 to be safe and well tolerated, and, when given as a single intramuscular dose, was immunogenic, and able to “Spark” anti-HBV activity in chronically HBV-infected patients on nucleos(t)ide therapy alone, and, also, when administered first and given with combination antiviral therapies. These results suggest the potential of VRON-0200 to be the key backbone agent, in combination HBV functional cure regimens.

About Virion Therapeutics (Virion)

Virion Therapeutics, LLC is a clinical-stage company developing novel immunotherapies that utilize proprietary checkpoint modifiers to enhance/restore, broaden, and elicit sustained immune responses, with the goal to cure cancer and chronic infectious diseases. Virion has since developed a robust pipeline, including its lead VRON-0200 clinical program, and several additional IND-enabling programs, such as its VRON-0300 oncology program for advanced solid tumors, leveraging its proprietary platform technologies.

To learn more, visit www.VirionTx.com

Contacts

Virion Therapeutics, LLC, Dr. Sue Currie, Chief Operating Officer

scurrie@viriontx.com
1-800-841-9303

Merck’s Enlicitide Decanoate, an Investigational Oral PCSK9 Inhibitor, Significantly Reduced LDL-C in Adults with Heterozygous Familial Hypercholesterolemia (HeFH) in Phase 3 CORALreef HeFH Trial




Merck’s Enlicitide Decanoate, an Investigational Oral PCSK9 Inhibitor, Significantly Reduced LDL-C in Adults with Heterozygous Familial Hypercholesterolemia (HeFH) in Phase 3 CORALreef HeFH Trial

Enlicitide has the potential to be the first approved oral PCSK9 inhibitor designed to deliver antibody-like efficacy and help address critical unmet needs for patients with HeFH to help combat the ongoing CV epidemic

Results were presented today at AHA Scientific Sessions 2025 and simultaneously published in the Journal of the American Medical Association

RAHWAY, N.J.–(BUSINESS WIRE)–Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced the first presentation of results from the pivotal Phase 3 CORALreef HeFH trial demonstrating that treatment with enlicitide decanoate, an investigational, once-daily oral proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor, resulted in a statistically significant and clinically meaningful reduction in low-density lipoprotein cholesterol (LDL-C) of 59.4% compared to placebo at week 24 (95% CI: -65.6, -53.2; p<0.001) in adults with heterozygous familial hypercholesterolemia (HeFH). The effect size and safety profile was comparable to that observed in the pivotal Phase 3 CORALreef Lipids study. These late-breaking data will be presented for the first time today at the American Heart Association (AHA) Scientific Sessions 2025 (Abstract #4391641) and published simultaneously in the Journal of the American Medical Association.

In CORALreef HeFH, enlicitide demonstrated statistically significant and clinically meaningful reductions in LDL-C at week 24 (primary endpoint) and statistically significant reductions in secondary endpoints including LDL-C at one year (week 52), and non-high-density lipoprotein cholesterol (non-HDL-C), apolipoprotein B (ApoB), and lipoprotein(a) (Lp(a)) at week 24, in adults with HeFH receiving stable background lipid-lowering therapy including at least moderate or high intensity statin therapy. The overall safety profile was comparable to placebo. High adherence with study intervention (97%) and dosing instructions (96%) were observed across treatment groups.

“Data from CORALreef HeFH demonstrate the potential for enlicitide to help address critical unmet needs for adults with heterozygous familial hypercholesterolemia are at risk for premature atherosclerotic cardiovascular events yet a significant portion of patients do not achieve guideline-recommended LDL-C level despite available lipid-lowering therapies,” said Dr. Christie M. Ballantyne, a lead author of the CORALreef HeFH study and Professor of Medicine at Baylor College of Medicine. “As the potentially first approved oral PCSK9 inhibitor, enlicitide was designed to provide efficacy similar to anti-PCSK9 monoclonal antibodies and may be an important new treatment option to help adults with heterozygous familial hypercholesterolemia reach their guideline-recommended LDL-C goal. Lowering elevated LDL-C levels helps reduce the risk of atherosclerotic cardiovascular disease.”

“Results from the CORALreef HeFH study demonstrated statistically significant and sustained reductions in LDL-C, ApoB, non-HDL-C, and Lp(a) over one year in a diverse population of adults with heterozygous familial hypercholesterolemia receiving stable background lipid-lowering therapies,” said Dr. Dean Y. Li, president, Merck Research Laboratories. “We look forward to sharing the totality of the results from the CORALreef program presented at AHA with regulatory authorities and progressing enlicitide’s ongoing clinical development program to bring forward the potential first approved oral PCSK9 inhibitor to help address the growing CV epidemic.”

In CORALreef HeFH, LDL-C reductions were observed as early as week 4 and maintained through one year. Treatment with enlicitide resulted in a sustained statistically significant reduction in LDL-C of 61.5% compared to placebo (95% CI: -69.4, -53.7, p<0.001) at one year. At week 24, enlicitide demonstrated statistically significant reductions in non-HDL-C of 53.0% (95% CI: -58.5, -47.4, p<0.001), ApoB of 49.1% (95% CI: -54.0, -44.3, p<0.001) and Lp(a) of 27.5% (-95% CI: -34.3, -20.6, p<0.001) compared to placebo. The study also showed that 67.3% of patients treated with enlicitide achieved at least a 50% reduction in LDL-C along with an LDL-C <55 mg/dL (1.42 mmol/L) compared to 1.0% in the placebo arm at week 24.

Enlicitide had a safety profile similar to placebo. The incidence of adverse events (AEs), serious AEs and discontinuations due to AEs were similar between groups. Discontinuations due to AEs were low and similar between enlicitide (2.0%) and placebo (3.0%).

Merck plans to share data from this trial, along with data from CORALreef Lipids and CORALreef AddOn with regulatory authorities worldwide.

About CORALreef HeFH

CORALreef HeFH (NCT05952869) is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of enlicitide compared to placebo in adults with HeFH who had a history of or were at risk for a major ASCVD event and received stable background lipid-lowering therapy including at least moderate or high intensity statins. The study enrolled 303 participants who were randomized 2:1 to receive either 20 mg of enlicitide orally once daily or placebo. The primary endpoints were mean percent change in LDL-C from baseline at week 24 versus placebo, number of participants with one or more AEs, and number of participants who discontinued study drug due to an AE. Key secondary multiplicity-controlled efficacy endpoints included change in LDL-C at one year (week 52) and changes in non-HDL-C, ApoB and Lp(a) at week 24. Non-multiplicity-controlled secondary endpoints included LDL-C goal attainment of at least a 50% reduction in LDL-C and an LDL-C <70 mg/dL (1.81 mmol/L) and at least 50% reduction in LDL-C and an LDL-C <55 mg/dL (1.42 mmol/L).

About enlicitide and PCSK9

Enlicitide has the potential to be the first FDA approved oral PCSK9 inhibitor. It is designed to lower LDL-C via the same biological mechanism as currently approved monoclonal antibody, injectable PCSK9 inhibitors but in a daily pill form. Enlicitide is a novel small molecule macrocyclic peptide candidate that binds to PCSK9 and inhibits the interaction of PCSK9 with LDL receptors.

PCSK9 plays a key role in cholesterol homeostasis by regulating levels of the LDL receptor, which is responsible for the uptake of cholesterol into cells. Inhibition of PCSK9 is designed to prevent the interaction of PCSK9 with LDL receptors. This results in greater numbers of LDL receptors available on the cell surface to remove LDL cholesterol from the blood.

About CORALreef Clinical Trial Program

The efficacy and safety profile of enlicitide is being evaluated through the comprehensive CORALreef Clinical Trial program evaluating over 19,000 participants who have hypercholesterolemia. As previously announced, enlicitide demonstrated statistically significant and clinically meaningful reductions in LDL-C in three pivotal Phase 3 studies: CORALreef Lipids (NCT05952856), CORALreef HeFH (NCT05952869) and CORALreef AddOn (NCT06450366). Enlicitide is continuing to be evaluated in the large cardiovascular outcomes trial, CORALreef Outcomes (NCT06008756), which has completed enrollment with over 14,500 participants. Additional CORALreef clinical trials include CORALreef Extension (NCT06492291), CORALreef Pediatric (NCT07058077) and CORALreef Combination (NCT07216482).

About heterozygous familial hypercholesterolemia (HeFH)

Heterozygous familial hypercholesterolemia (HeFH) is a common genetic disorder that affects approximately 1 in 250 individuals and is characterized by elevated levels of LDL-C. Patients with HeFH typically present with substantially elevated LDL-C levels and face an increased risk of premature atherosclerotic cardiovascular disease (ASCVD) due to cumulative lifetime exposure to LDL-C. HeFH cannot be managed through lifestyle and diet changes alone, and cholesterol-lowering medication is typically needed for patients to manage this condition. A large proportion of patients with HeFH fail to achieve guideline-recommended LDL-C goals despite available therapies and have a 13-fold higher risk for coronary artery disease compared with the general population.

About the CV epidemic and atherosclerotic cardiovascular disease

The silent CV epidemic is the leading cause of deaths globally, contributing to the majority of heart attacks and strokes, and deaths related to CV continue to rise. ASCVD accounts for 85% of CV deaths. It is caused by the buildup of plaque within the arteries, leading to narrowed or blocked blood vessels that can result in serious CV events such as heart attacks and strokes as well as coronary artery disease, peripheral artery disease and cerebrovascular disease.

Merck’s focus on cardiovascular disease

Merck has a long history of developing treatments for cardiovascular disease. Nearly 70 years ago, we introduced our first cardiovascular therapy—and our scientific efforts to understand and treat cardiovascular-related disorders have continued. Cardiovascular disease continues to be one of the most serious health challenges of the 21st century and is the leading cause of death worldwide. Approximately 18 million people across the globe die from cardiovascular disease every year; in the United States, one person dies every 36 seconds from cardiovascular disease.

At Merck, we strive for scientific excellence and innovation in all stages of research, from discovery through approval and life cycle management. We work with experts throughout the cardiovascular and pulmonary community to advance research that can help improve the lives of patients globally.

Information for other currently enrolling cardiovascular studies can be found by visiting: https://www.merckclinicaltrials.com/cardiovascular.

About Merck

At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn.

Forward-Looking Statement of Merck & Co., Inc., Rahway, N.J., USA

This news release of Merck & Co., Inc., Rahway, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions.

The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s Annual Report on Form 10-K for the year ended December 31, 2024 and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site (www.sec.gov).

Contacts

Media Contacts:

Julie Cunningham

(617) 519-6264

Justine Moore

(347) 281-3754

Investor Contacts:

Peter Dannenbaum

(732) 594-1579

Ayn Wisler

(917) 691-6218

Baxdrostat demonstrated a statistically significant and highly clinically meaningful placebo-adjusted reduction of 14.0 mmHg in 24-hour ambulatory systolic blood pressure in patients with resistant hypertension in the Bax24 Phase III trial




Baxdrostat demonstrated a statistically significant and highly clinically meaningful placebo-adjusted reduction of 14.0 mmHg in 24-hour ambulatory systolic blood pressure in patients with resistant hypertension in the Bax24 Phase III trial

Baxdrostat demonstrated a statistically significant placebo-adjusted reduction of 13.9 mmHg in night-time ambulatory systolic blood pressure at 12 weeks with a safety profile consistent with the BaxHTN trial

Full results presented at the American Heart Association Scientific Sessions 2025

WILMINGTON, Del.–(BUSINESS WIRE)–Positive full results from the Bax24 Phase III trial showed baxdrostat demonstrated a statistically significant and highly clinically meaningful reduction in ambulatory 24-hour average systolic blood pressure (SBP) compared with placebo at 12 weeks. Patients with treatment-resistant hypertension (rHTN) received baxdrostat 2mg or placebo on top of standard of care.¹ Efficacy was observed throughout the 24-hour period, including early morning, when patients with hypertension are at a higher risk of cardiovascular events.^2-4

Baxdrostat met the primary endpoint in the Bax24 Phase III trial, delivering clinically meaningful and consistent blood pressure reductions in patients with treatment-resistant hypertension. At 12 weeks, the placebo-adjusted reduction in ambulatory 24-hour average SBP was 14.0 mmHg (95% confidence interval [CI] -17.2, -10.8; p<0.0001).¹ Baxdrostat was generally well tolerated, with a safety profile consistent with the BaxHTN trial.^1,5

Baxdrostat demonstrated statistically significant and clinically meaningful reductions in key secondary endpoints, including ambulatory night-time average SBP (13.9 mmHg placebo-adjusted [95% CI -17.5, -10.3; p<0.0001]) and seated SBP (10.3 mmHg placebo-adjusted [95% CI -14.9, -5.6; p<0.0001]) consistent with data from the BaxHTN trial.^1,5 Significantly more patients treated with baxdrostat (71%) achieved ambulatory 24-hour average SBP of less than 130 mmHg compared with patients receiving placebo (17%) (Odds ratio 15.2 [95% CI 6.6, 35.2; p<0.0001]).¹

Dr. Bryan Williams, Chair of Medicine at University College London, primary investigator, said: “The landmark results from Bax24 Phase III trial demonstrate that patients with the hardest-to-control hypertension treated with baxdrostat achieved a highly clinically meaningful 14 mmHg placebo-adjusted reduction in 24-hour systolic blood pressure, which could transform treatment practice. It’s remarkable to see this magnitude of reduction coupled with the fact that just over 70% of baxdrostat patients achieved guideline targets, consistently over 24 hours.”

Sharon Barr, Executive Vice President, BioPharmaceuticals R&D, said: “The Bax24 data demonstrate the significant impact that baxdrostat’s long half-life and highly selective inhibition of aldosterone synthase can have in improving 24-hour and overnight blood pressure for patients with resistant hypertension. Patients with elevated night-time blood pressure are especially vulnerable to cardiovascular events, including heart attack and stroke. Together with the results from BaxHTN, these findings demonstrate the potential of baxdrostat to redefine what is possible for the millions of patients whose hypertension remains uncontrolled despite current therapies.”

There are 1.4 billion people worldwide living with hypertension.⁶ In the US, approximately 50% of patients living with hypertension on multiple treatments do not have their blood pressure under control.⁷ Consistent 24-hour blood pressure control is an important clinical outcome in patients with hard-to-control hypertension.^8-10 Multiple studies have demonstrated that 24-hour blood pressure is a more powerful predictor of cardiovascular events than a clinic-based measurement.^4,11 When 24-hour average systolic blood pressure rises by 9.5 mmHg, the risk of all-cause mortality increases by 30%.⁴

Full results from the Bax24 trial were presented today at the Emerging Opportunities for Managing Cardiometabolic Syndrome late breaker session at the American Heart Association (AHA) Scientific Sessions 2025. These data will be shared with regulatory authorities around the world.

Baxdrostat is designed to lower blood pressure by specifically inhibiting aldosterone, a key hormone that raises blood pressure and increases the risk of heart and kidney problems. Phase I studies show baxdrostat reached peak levels in the blood within 2 to 4 hours and had a half-life of about 26 to 30 hours.^12,13 Baxdrostat is currently being investigated as a monotherapy for hypertension^13-16 and primary aldosteronism,¹⁷ and in combination with dapagliflozin for chronic kidney disease^18,19 and the prevention of heart failure in high-risk patients.²⁰

Primary and secondary endpoints¹

*The main analyses of ambulatory BP endpoints include patients with valid ambulatory blood pressure monitoring at both baseline and Week 12, without imputation of missing data.

LS, least squares; n Number of subjects in analysis; N Number of subjects per treatment group

Notes

Hard-to-control hypertension

Hypertension is a medical condition characterized by consistently high blood pressure levels, affecting an estimated 1.4 billion people worldwide.^6,21,22 Over time, this can damage blood vessels and vital organs, increasing the risk of serious health problems such as heart attack, stroke, heart failure and kidney disease.^20,21 An observational study of nearly 60,000 patients studied over a median of 9.7 years showed that a 9.5 mmHg increase in 24-hour ambulatory SBP was associated with a 30% increase in risk of all-cause mortality and 41% increase in risk of cardiovascular death.⁴ Studies have shown that increased night-time blood pressure is associated with higher cardiovascular risk,^8,11 and patients with hypertension have a higher risk of cardiovascular events like heart attack, stroke and death around the time of their morning blood pressure surge.^2,3

Hard-to-control (uncontrolled and resistant) hypertension remains a major public health challenge.²³ Despite lifestyle changes and the use of multiple medications, approximately 50% of patients in the US who are being treated for hypertension still do not have their blood pressure under control.⁷ Uncontrolled hypertension refers to persistently elevated blood pressure despite the use of two or more medications, while resistant hypertension, a more severe form, remains elevated despite treatment with three or more medications.^7,21 Guidelines currently recommend that in patients with hypertension, treated BP values should be targeted to 130/80 mmHg or lower in most patients.^21,22

A key contributor to hard-to-control hypertension is aldosterone, a hormone that raises blood pressure by promoting sodium and water retention.^24,25 Elevated aldosterone levels, along with factors such as obesity, high salt intake, and various genetic or secondary conditions,²⁶ are strongly associated with poor blood pressure control. When left untreated, hypertension significantly increases the risk of cardiovascular and kidney-related complications.^21,22

Bax24 trial

The Phase III Bax24 trial¹⁶ is a randomized, double-blind, placebo-controlled, parallel group study to evaluate the effects of 2mg baxdrostat versus placebo, administered once a day (QD) orally, on the reduction of ambulatory SBP, as well as safety and tolerability in participants with resistant hypertension. A total of 218 patients were randomized in a 1:1 ratio to receive baxdrostat 2mg or placebo once daily during a 12-week double blind period. The primary efficacy endpoint was the change from baseline in ambulatory 24-hour average SBP at Week 12.

Additional secondary endpoints include the effect of baxdrostat versus placebo on change from baseline in ambulatory night-time average SBP, change from baseline in ambulatory daytime average SBP, change from baseline in seated SBP, the number of participants achieving ambulatory 24-hour average SBP of less than 130 mmHg , change from baseline in ambulatory 24-hour average diastolic blood pressure (DBP), change from baseline in ambulatory night-time average DBP, change from baseline in the average ambulatory daytime average DBP, change from baseline on seated DBP and the number of participants achieving a nocturnal SBP dipping of greater than or equal to 10%, all measured at Week 12. Occurrence of adverse events was evaluated during the 12-week treatment period as well as during a 2-week safety follow-up period.

Baxdrostat

Baxdrostat is a potential first-in-class, highly selective and potent, oral, small molecule that inhibits aldosterone synthase,¹² an enzyme encoded by the CYP11B2 gene, which is responsible for the synthesis of aldosterone in the adrenal gland.²⁴ In clinical trials, baxdrostat was observed to significantly lower aldosterone levels without affecting cortisol levels across a wide range of doses.^13,27 Baxdrostat is currently being investigated in clinical trials as a monotherapy for hypertension^13-16 and primary aldosteronism,¹⁷ and in combination with dapagliflozin for chronic kidney disease and hypertension,^18,19 and the prevention of heart failure in high-risk patients.²⁰

AstraZeneca acquired baxdrostat through its purchase of CinCor Pharma, Inc. in February 2023.²⁸

AstraZeneca in CVRM

Cardiovascular, Renal and Metabolism (CVRM), part of BioPharmaceuticals, forms one of AstraZeneca’s main disease areas and is a key growth driver for the Company. By following the science to understand more clearly the underlying links between the heart, kidneys, liver and pancreas, AstraZeneca is investing in a portfolio of medicines for organ protection by slowing or stopping disease progression, and ultimately paving the way towards regenerative therapies. The Company’s ambition is to improve and save the lives of millions of people, by better understanding the interconnections between CVRM diseases and targeting the mechanisms that drive them, so we can detect, diagnose and treat people earlier and more effectively.

AstraZeneca

AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines in Oncology, Rare Diseases and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit www.astrazeneca-us.com and follow the Company on social media @AstraZeneca.

References

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2. Renna NF, et al. Morning blood pressure surge as a predictor of cardiovascular events in patients with hypertension. Blood Press Monit. 2023;28(3):149-157
3. Kario K et al. Morning hypertension: the strongest independent risk factor for stroke in elderly hypertensive patients. Hypertens Res. 2006;29(8):581-7.
4. Staplin N, et al. Relationship between clinic and ambulatory blood pressure and mortality: an observational cohort study in 59 124 patients. Lancet. 2023;401(10393):2041-2050.
5. Flack JM, et al. Efficacy and Safety of Baxdrostat in Uncontrolled and Resistant Hypertension. N Engl J Med. 2025. Aug 30:10.1056/NEJMoa2507109. doi: 10.1056/NEJMoa2507109.
6. World Health Organization. Global report on hypertension 2025: high stakes: turning evidence into action. 2025. https://iris.who.int/handle/10665/382841. Accessed September 2025.
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9. Kario K, et al. Nighttime Blood Pressure Phenotype and Cardiovascular Prognosis. Circulation. 2020;142(19):1810-1820
10. Williams B, et al. 2018 ESC/ESH Guidelines for the management of arterial hypertension: The Task Force for the management of arterial hypertension of the European Society of Cardiology (ESC) and the European Society of Hypertension (ESH). European Heart Journal. 2018;39(33):3021-3104.
11. Niiranen TJ, Mäki J, Puukka P, Karanko H, Jula AM. Office, home, and ambulatory blood pressures as predictors of cardiovascular risk. Hypertension. 2014 Aug;64(2):281-6.
12. Bogman K, et al. Preclinical and early clinical profile of a highly selective and potent oral inhibitor of aldosterone synthase (CYP11B2). Hypertension. 2017;69(1):189-196.
13. Freeman MW, et al. Results from a phase 1, randomized, double-blind, multiple ascending dose study characterizing the pharmacokinetics and demonstrating the safety and selectivity of the aldosterone synthase inhibitor baxdrostat in healthy volunteers. Hypertens Res. 2023;46(1):108-118.
14. ClinicalTrials.gov.A Study to Investigate the Efficacy and Safety of Baxdrostat in Participants With Uncontrolled Hypertension on Two or More Medications Including Participants With Resistant Hypertension (BaxHTN). Available at: https://clinicaltrials.gov/study/NCT06034743. Accessed October 2025.
15. ClinicalTrials.gov. A Study to Investigate the Efficacy and Safety of Baxdrostat in Participants With Uncontrolled Hypertension on Two or More Medications Including Participants With Resistant Hypertension (BaxAsia). Available at: https://clinicaltrials.gov/study/NCT06344104. Accessed October 2025.
16. ClinicalTrials.gov. A Study to Investigate the Effect of Baxdrostat on Ambulatory Blood Pressure in Participants With Resistant Hypertension (Bax24). Available at: https://clinicaltrials.gov/study/NCT06168409. Accessed October 2025.
17. ClinicalTrials.gov. A Study to Assess Efficacy and Safety of Baxdrostat in Participants With Primary Aldosteronism (BaxPA). Available at: https://clinicaltrials.gov/study/NCT07007793. Accessed October 2025.
18. ClinicalTrials.gov. A Phase III Study to Investigate the Efficacy and Safety of Baxdrostat in Combination With Dapagliflozin on CKD Progression in Participants With CKD and High Blood Pressure. Available at: https://clinicaltrials.gov/study/NCT06268873. Accessed October 2025.
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