Alpha Cognition Inc. Announces Proposed Public Offering of Common Shares




Alpha Cognition Inc. Announces Proposed Public Offering of Common Shares

VANCOUVER, British Columbia & DALLAS–(BUSINESS WIRE)–Alpha Cognition Inc. (Nasdaq: ACOG) (the “Company”), a commercial-stage biopharmaceutical company dedicated to developing innovative treatments for neurodegenerative diseases, today announced that it is proposing to offer and sell its common shares and, in lieu of common shares to certain investors, pre-funded warrants to purchase its common shares, in an underwritten public offering. In addition, the Company intends to grant the underwriter a 30-day option to purchase up to a number of additional common shares equal to 15% of the total number of common shares (and common shares underlying pre-funded warrants) sold in the proposed public offering, on the same terms and conditions. The proposed public offering is subject to market and other conditions, and there can be no assurance as to whether or when the proposed public offering may be completed, or as to the actual size or terms of the proposed public offering. All securities to be sold in the proposed public offering will be offered by the Company.

The Company plans to allocate the net proceeds for the acceleration of commercial launch, with an emphasis on sales expansion, marketing investment, and enhancing payer coverage and reimbursement infrastructure. These investments are designed to maximize near-term adoption while laying the foundation for long-term revenue growth and a sustainable commercial presence in the Alzheimer’s treatment landscape.

Titan Partners Group, a division of American Capital Partners, is acting as sole bookrunner for this proposed public offering.

The proposed public offering is being made pursuant to a shelf registration statement on Form S-3 (File No. 333-289792) filed with the Securities and Exchange Commission (“SEC”) on August 22, 2025, and declared effective by the SEC on August 29, 2025. A preliminary prospectus supplement and accompanying prospectus relating to the proposed public offering will be filed with the SEC and will be available for free on the SEC’s website at www.sec.gov. Copies of the preliminary prospectus supplement and accompanying prospectus relating to the proposed public offering, when available, may also be obtained by contacting Titan Partners Group LLC, a division of American Capital Partners, LLC, 4 World Trade Center, 29th Floor, New York, NY 10007, by phone at (929) 833-1246 or by email at prospectus@titanpartnersgrp.com.

This proposed public offering will be made only by means of the prospectus supplement and accompanying prospectus forming a part of the effective registration statement. This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

About Alpha Cognition Inc.

Alpha Cognition Inc. is a commercial stage, biopharmaceutical company dedicated to developing treatments for patients suffering from neurodegenerative diseases, such as Alzheimer’s Disease and Cognitive Impairment with mild Traumatic Brain Injury (“mTBI”), for which there are currently no approved treatment options.

ZUNVEYL is a patented drug approved as a new generation acetylcholinesterase inhibitor for the treatment of Alzheimer’s disease, with expected minimal gastrointestinal side effects. ZUNVEYL’s active metabolite is differentiated from donepezil and rivastigmine in that it binds neuronal nicotinic receptors, most notably the alpha-7 subtype, which is known to have a positive effect on cognition. ALPHA-1062 is also being developed in combination with memantine to treat moderate to severe Alzheimer’s dementia, and as an intranasal formulation for Cognitive Impairment with mTBI.

Forward-looking Statements

This news release includes forward-looking statements within the meaning of the “safe harbor” provisions of the United States Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding the completion, timing and size of the Company’s proposed public offering, the grant to the underwriter of an option to purchase additional securities, the satisfaction of customary closing conditions, and the intended use of proceeds therefrom. Except for statements of historical fact, any information contained in this news release may be a forward-looking statement that reflects the Company’s current views about future events and are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause the actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. In some cases, you can identify forward-looking statements by the words “may,” “might,” “will,” “could,” “would,” “should,” “expect,” “intend,” “plan,” “objective,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “target,” “seek,” “contemplate,” “continue” and “ongoing,” or the negative of these terms, or other comparable terminology intended to identify statements about the future. Although the Company believes to have a reasonable basis for each forward-looking statement, we caution you that these statements are based on a combination of facts and factors currently known by us and our expectations of the future, about which we cannot be certain. The Company cannot assure that the actual results will be consistent with these forward-looking statements. These forward-looking statements are subject to certain risks, including risks regarding our ability to raise sufficient capital to implement our plans to commercialize ZUNVEYL, risks regarding the efficacy and tolerability of ZUNVEYL, risks related to ongoing regulatory oversight on the safety of ZUNVEYL, risk related to market adoption of ZUNVEYL, risks related to the Company’s intellectual property in relation to ZUNVEYL, risks related to the commercial manufacturing, distribution, marketing and sale of ZUNVEYL, risks related to product liability and other risks as described in the Company’s filings with the SEC, including those risk factors under the heading “Risk Factors” in the Company’s most recent Annual Report on Form 10-K filed with the SEC on March 31, 2025 and our periodic reports on Form 10-Q and Form 8-K filed with the SEC available at www.sec.gov. These forward‐looking statements speak only as of the date of this news release and the Company undertakes no obligation to revise or update any forward‐looking statements for any reason, even if new information becomes available in the future, except as required by law.

Contacts

For further information:

Investor Relations

IR@alphacognition.com
https://www.alphacognition.com/

VectorBuilder Wins Two IMAPAC Awards, Cementing Leadership in Gene Therapy and CDMO Innovation




VectorBuilder Wins Two IMAPAC Awards, Cementing Leadership in Gene Therapy and CDMO Innovation

CHICAGO–(BUSINESS WIRE)–#BioTechNews—VectorBuilder, a global leader in the gene delivery space, has won two prestigious awards at the Asia-Pacific Cell and Gene Therapy Excellence Awards 2025 (APCGTEA) and the Asia-Pacific Biologics CDMO Excellence Awards 2025 (APBCEA), hosted by IMAPAC.

• Best Cell & Gene Therapy Supplier Award – AAV Vector Manufacturing
• Best Gene Therapy CDMO – Asia-Pacific

VectorBuilder pioneers cutting-edge innovation for AAV gene delivery, with a one-stop solution supporting programs from research to early discovery to clinical development. Its proprietary technology platforms have enabled robust production of tens of thousands of custom AAV vectors for researchers and drug developers worldwide. Leveraging its expansive suite of AAV preclinical services, including a comprehensive capsid evolution platform, biodistribution profiling, and the bespoke cliniVec^TM consultation service, VectorBuilder empowers its partners to accelerate and streamline their cell and gene therapy development. The company’s innovative AAV IP portfolio features novel AAV capsids, miniVec^TM and MuteFree^TM AAV backbones, and novel promoters, positioning VectorBuilder at the forefront of next-generation AAV innovations.

Offering a variety of systems that meet diverse needs, VectorBuilder is a full-service CDMO offering end-to-end solutions from development to production, having developed extensive expertise in process and analytical development for the manufacturing of GMP-grade gene therapy vectors. Operating several state-of-the-art facilities, VectorBuilder has supported many customers with the GMP manufacturing of plasmid DNA, viral vectors, IVT RNA, and LNP encapsulation. The highly experienced team has successfully provided plasmids, lentiviral vectors, and AAV vectors for their customers’ IND and IIT studies in the US and Asia. These recognitions add to a growing list of accolades for VectorBuilder, including the Bio-Industrial Innovation of the Year Award from BioTech Breakthrough (November 2024) and the CDMO Leadership Award from Outsourced Pharma and Life Science Connect (May 2025). Together, these honors reinforce VectorBuilder’s impact on the global CGT industry.

“We are honored to receive two IMAPAC awards at the same time, affirming the value of our commitment to innovation,” said Dr. Bruce Lahn, founder and Chief Scientist of VectorBuilder. “We will continue to advance gene delivery technologies and collaborate globally to enable breakthrough therapies and contribute to better human health.”

For more information, please visit www.vectorbuilder.com.

About VectorBuilder

VectorBuilder is a global leader in gene delivery technologies. As a trusted partner in thousands of labs and biotech/pharma companies around the world, VectorBuilder is a one-stop shop for the design, development, and optimization of gene delivery solutions from basic research to clinical applications. Its award-winning Vector Design Studio is a transformative innovation that allows researchers to easily design and order custom vectors online, freeing them from the tedious work of cloning and packaging vectors in the lab. The global company boasts high-throughput vector production capacity, vast vector and component inventories, one-on-one CRO solutions that include advanced AAV capsid engineering capabilities, and state-of-the-art GMP manufacturing facilities. With leading R&D and CDMO capacity, the VectorBuilder team strives to provide the most effective gene delivery solutions and develop new innovative tools for life sciences research and genetic medicine.

Contacts

Media Inquiries
Sarah Shkargi

sarah@tnsmediacomms.com

Tagomics Publishes a New Approach to Genome-Wide Epigenomic Profiling




Tagomics Publishes a New Approach to Genome-Wide Epigenomic Profiling

• Study published in ‘Cell Reports Methods’ demonstrates the strength of Tagomics’ platform for epigenomic biomarker discovery and applications in liquid biopsy
• Activace platform enables targeted, genome-wide profiling of unmethylated DNA regions, providing a comprehensive view of the epigenome that can be scaled to the study of large patient cohorts

CAMBRIDGE, England–(BUSINESS WIRE)–Tagomics Ltd., a pioneering biomarker discovery and diagnostics company, today announced the publication of a peer-reviewed study in Cell Reports Methods, underpinning its epigenomic profiling technology, Active-Seq, the basis of Tagomics’ Activace™ platform. The paper, titled ‘Genome-wide profiling of unmodified DNA using methyltransferase-directed tagging and enrichment’, built on research from the University of Birmingham, describes Tagomics’ enzymatic approach to epigenomic profiling that targets unmethylated DNA for enrichment, and its application to biomarker identification and disease profiling in colorectal cancer patients.

Current gold standard approaches for assessing genome-wide DNA methylation levels are poorly suited to the challenge of working in liquid biopsy (blood) samples, limiting the performance of cell-free DNA based diagnostic tests. Addressing these limitations, the paper describes Active-Seq (Azide Click Tagging for In Vitro Epigenomic sequencing), a base conversion-free methodology that does not alter the underlying DNA sequence and provides a scalable and comprehensive solution for the discovery of novel biomarkers in liquid biopsy samples. Activace, built on the Active-Seq technology, is a streamlined workflow that incorporates sequencing library preparation with enrichment of unmethylated DNA. The workflow is compatible with low DNA input quantities, down to one nanogram, and has been optimised for the analysis of DNA methylation in cfDNA derived from liquid biopsy samples.

The published paper demonstrates application of Active-Seq for the detection of abnormal DNA methylation signals in a cohort of colorectal cancer patients, identifying thousands of hypomethylated and hypermethylated regions in tumour-derived tissue, both of which are associated with cancer. The study lays the groundwork for application of the approach to the deconvolution of the tissue of origin of cell-free DNA in blood, improving detection and characterisation of disease.

Dr Robert Neely, CSO and co-founder of Tagomics said: “This paper is an important milestone for Tagomics, demonstrating the groundbreaking technology that underpins our Activace and Interlace platforms. We show that our platform enables the sensitive detection of unmethylated genomic regions, which are key markers for DNA tissue of origin. We’re excited about the insight this is providing into the biology of cell-free DNA and are looking forward to seeing new applications for the platform in cancer diagnostics and patient safety monitoring for therapeutics.”

For more information about Tagomics, please visit: https://tagomics.com/

Contacts

Media Contact:
Francesca Wallace

Email: francesca.wallace@zymecommunications.com

WINREVAIR™ (sotatercept-csrk) Reduced the Risk of Clinical Worsening Events by 76% Compared to Placebo in Patients Recently Diagnosed With PAH on Background Therapy in Phase 3 HYPERION Trial




WINREVAIR™ (sotatercept-csrk) Reduced the Risk of Clinical Worsening Events by 76% Compared to Placebo in Patients Recently Diagnosed With PAH on Background Therapy in Phase 3 HYPERION Trial

Results showed the benefits of early initiation of WINREVAIR within the first year after PAH diagnosis

HYPERION results were presented today at ERS 2025 and simultaneously published in the New England Journal of Medicine

RAHWAY, N.J.–(BUSINESS WIRE)–Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced positive results from the Phase 3 HYPERION trial evaluating WINREVAIR™ (sotatercept-csrk) versus placebo (both in combination with background therapy) in recently diagnosed adults with pulmonary arterial hypertension (PAH, WHO* Group 1) functional class (FC) II or III at intermediate or high risk of disease progression. In the study, WINREVAIR reduced the risk of clinical worsening events by 76% (hazard ratio [HR] 0.24 [95% confidence interval [CI], 0.14 to 0.41]; p<0.0001) as measured by a composite endpoint of all-cause death, the need for non-planned PAH-related hospitalization ≥24 hours, atrial septostomy, lung transplantation or PAH deterioration. HYPERION included participants who were within their first year of diagnosis (median seven months and as early as one month), with over 70% of trial participants on double background therapy. In the pivotal Phase 3 study, STELLAR, participants were WHO Group 1, FC II or III at baseline and had an average disease duration of 8.8 years from PAH diagnosis to screening. The safety profile of WINREVAIR was generally consistent with that observed in previous trials. Results from the study were presented today at the 2025 European Respiratory Society (ERS) Congress and simultaneously published in the New England Journal of Medicine.

In HYPERION, there was an early and sustained separation in the Kaplan-Meier curves with treatment benefit observed within six weeks of randomization. Patients on placebo plus background standard of care therapy experienced accumulation of clinical worsening events. Results showed that 10.6% (n=17/160) of patients treated with WINREVAIR compared to 36.9% (n=59/160) in the placebo group experienced at least one clinical worsening event. The treatment effect was consistent across all prespecified subgroups treated with WINREVAIR, including patients with idiopathic PAH, those with connective tissue disease, those on double background therapy, those on triple background therapy and those at intermediate or intermediate-low risk by REVEAL Lite 2 and COMPERA 2.0 risk tools, respectively.

“PAH is a rare condition that can progress quickly making diagnosis and early treatment critically important,” said Dr. Vallerie McLaughlin**, Kim A Eagle MD Endowed Professor of Cardiovascular Medicine and Director, Pulmonary Hypertension Program, University of Michigan in Ann Arbor. “The patients with PAH enrolled in HYPERION were early in their treatment journey, had co-morbidities and were older, which reflects the type of patients we are diagnosing in a contemporary real-world setting. I am encouraged by the compelling results of the HYPERION study demonstrating that initiation of WINREVAIR on top of background therapy within the first year of diagnosis significantly reduces the risk of clinical worsening events compared to placebo.”

“These positive results from HYPERION expand on the body of clinical evidence for WINREVAIR, now including PAH patients within their first year of diagnosis, including those earlier in their treatment journey,” said Dr. Joerg Koglin, senior vice president, head of general and specialty medicine, global clinical development, Merck Research Laboratories. “The totality of WINREVAIR data to date continues to reinforce our confidence in its practice-changing potential. We thank the study participants and investigators for their contributions to this important study.”

The safety profile of WINREVAIR in HYPERION was generally consistent with that observed in previous studies. The median duration of follow-up was longer in those receiving WINREVAIR (14.6 months) compared with those receiving placebo (11.5 months). Adverse events occurred in 89.4% versus 90.0% and serious adverse events in 24.4% versus 28.1% of participants in the WINREVAIR and placebo groups, respectively.

WINREVAIR demonstrated statistically significant improvements in two secondary endpoints, including multicomponent improvement and maintenance or achievement of a low REVEAL Lite 2 score. Results showed that 29.4% of patients treated with WINREVAIR met all three criteria of multicomponent improvement (improvement in 6MWD, improvement or maintenance/achievement of NT-proBNP, and improvement in WHO FC or maintenance of WHO FC II) versus 14.6% treated with placebo. An additional secondary endpoint demonstrated 60.1% of patients treated with WINREVAIR maintained or achieved a low REVEAL LITE 2 score (≤5) relative to baseline at Week 24 compared to 47.9% treated with placebo. WINREVAIR did not show statistical significance in achieving or maintaining low risk for a simplified French risk score (SFRS). Subsequent secondary endpoints showed numerical improvements in the WINREVAIR arm (including NT-proBNP, WHO class and 6MWD), but were not formally tested due to the prespecified hierarchical testing strategy.

Earlier this year, the HYPERION trial was stopped early based on a review of the totality of data from the WINREVAIR clinical program at that time, and all patients were offered the opportunity to receive WINREVAIR through the SOTERIA open-label extension study. HYPERION is the third Phase 3 study of WINREVAIR to demonstrate significant efficacy in adults with PAH. The first was the Phase 3 STELLAR study previously presented at ACC.23, followed by the Phase 3 ZENITH study presented at ACC.25. Results from HYPERION will be submitted to regulatory authorities around the world. WINREVAIR is currently approved in more than 54 countries based on the results from the STELLAR study.

*World Health Organization

**Dr. McLaughlin is a member of the adult sotatercept steering committee, an investigator in the ZENITH and HYPERION studies and a paid consultant to Merck.

About HYPERION

The HYPERION study (NCT04811092) is a global, double-blind, placebo-controlled clinical trial to evaluate WINREVAIR when added to background PAH therapy in newly diagnosed intermediate or high-risk PAH patients. Participants who enrolled in the study had a diagnosis of symptomatic PAH (WHO Group 1, classified as FC II [21.3%; 68/320 participants] or III [78.8%; 252/320 participants] within 12 months of study screening. Two patients had a protocol deviation with a time since the diagnosis of PAH of more than 1 year (442 days in one patient in the sotatercept group; 397 days in one patient in the placebo group). Eligible participants had a confirmed diagnosis of PAH in any of the following subtypes: idiopathic PAH (59.4%; 190/320), heritable PAH (5.9%; 19/320), PAH associated with connective tissue diseases (CTD) (30.3%; 97/320), drug- or toxin-induced PAH (2.5%; 8/320), or PAH associated with simple, congenital systemic-to-pulmonary shunts at least 1 year following repair (1.9%; 6/320), and those at intermediate or intermediate-low risk by REVEAL Lite 2 and COMPERA 2.0 risk tools, respectively. The study excluded patients with PAH Group 1 subtypes: human immunodeficiency virus (HIV)-associated PAH and PAH associated with portal hypertension, schistosomiasis-associated PAH, pulmonary veno occlusive disease, and pulmonary capillary hemangiomatosis.

The study enrolled 320 study participants over the age of 18, who were randomized in a 1:1 ratio to receive either WINREVAIR or placebo, both on top of background therapy. Participants were at an intermediate to high risk of disease progression and on stable doses of double (72.2%; 231/320 participants) or triple (27.8%; 89/320 participants) background PAH therapies for at least 90 days prior to screening. A majority (83.4%; 267/320 participants) were not on prostacyclin-infusion therapy.

The primary composite outcome measure is TTCW as measured by first confirmed morbidity or mortality event. Clinical worsening events are defined as all-cause death, non-planned PAH worsening-related hospitalization of ≥ 24 hours, atrial septostomy, lung transplantation, and deterioration in six-minute walk test from baseline combined with at least one of the following changes: worsening of WHO FC from baseline, signs/symptoms of increased right heart failure, addition of a background PAH therapy or change in the background PAH therapy delivery route to parenteral.

Secondary outcome measures were assessed relative to baseline at Week 24: proportion of participants achieving multicomponent improvement (consisting of improvement in 6MWD, improvement in N-terminal pro-B-type natriuretic peptide (NT-proBNP) level and improvement in WHO FC or maintenance of WHO FC II) as well as additional measures.

About WINREVAIR™ (sotatercept-csrk) for injection, for subcutaneous use, 45 mg, 60 mg

WINREVAIR is FDA-approved for the treatment of adults with pulmonary arterial hypertension (PAH, WHO Group 1) to increase exercise capacity, improve WHO functional class (FC) and reduce the risk of clinical worsening events. WINREVAIR is the first activin signaling inhibitor therapy approved to treat PAH. WINREVAIR improves the balance between pro-proliferative and anti-proliferative signaling to modulate vascular proliferation. In preclinical models, WINREVAIR induced cellular changes that were associated with thinner vessel walls, partial reversal of right ventricular remodeling, and improved hemodynamics.

WINREVAIR is the subject of a licensing agreement with Bristol Myers Squibb.

Selected Safety Information for WINREVAIR in the U.S.

WINREVAIR may increase hemoglobin (Hgb). Severe erythrocytosis may increase the risk of thromboembolic events or hyperviscosity syndrome. Monitor Hgb before each dose for the first 5 doses, or longer if values are unstable, and periodically thereafter, to determine if dose adjustments are required.

WINREVAIR may decrease platelet count. Severe thrombocytopenia may increase the risk of bleeding. Thrombocytopenia occurred more frequently in patients also receiving prostacyclin infusion. Do not initiate treatment if platelet count is <50,000/mm³. Monitor platelets before each dose for the first 5 doses, or longer if values are unstable, and periodically thereafter to determine whether dose adjustments are required.

In clinical studies, serious bleeding (eg, gastrointestinal, intracranial hemorrhage) was reported in 4% of patients taking WINREVAIR and 1% of patients taking placebo. Patients with serious bleeding were more likely to be on prostacyclin background therapy and/or antithrombotic agents, or have low platelet counts. Advise patients about signs and symptoms of blood loss. Do not administer WINREVAIR if the patient is experiencing serious bleeding.

WINREVAIR may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use an effective method of contraception during treatment with WINREVAIR and for at least 4 months after the final dose. Pregnancy testing is recommended for females of reproductive potential before starting WINREVAIR treatment.

Based on findings in animals, WINREVAIR may impair female and male fertility. Advise patients on the potential effects on fertility.

The most common adverse reactions occurring in the phase 3 clinical trial (≥10% for WINREVAIR and at least 5% more than placebo) were headache (24.5% vs 17.5%), epistaxis (22.1% vs 1.9%), rash (20.2% vs 8.1%), telangiectasia (16.6% vs 4.4%), diarrhea (15.3% vs 10.0%), dizziness (14.7% vs 6.2%), and erythema (13.5% vs 3.1%).

Because of the potential for serious adverse reactions in the breastfed child, advise patients that breastfeeding is not recommended during treatment with WINREVAIR, and for 4 months after the final dose.

About PAH

Pulmonary arterial hypertension (PAH) is a rare, progressive and life-threatening blood vessel disorder characterized by the constriction of small pulmonary arteries and elevated blood pressure in the pulmonary circulation. Approximately 90,000 people worldwide are living with PAH. The disease progresses rapidly for many patients. PAH results in significant strain on the heart, leading to limited physical activity, heart failure and reduced life expectancy. The five-year mortality rate for patients with PAH is approximately 43%.

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).

Please see Prescribing Information for WINREVAIR (sotatercept-csrk) at http://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_pi.pdf, Patient Information for WINREVAIR at http://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_ppi.pdf, and Instructions for Use for WINREVAIR (1-vial kit, 2-vial kit) at https://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_ifu_1-vial_2-vial_kits.pdf.

Contacts

Media Contacts:

Julie Cunningham

(617) 519-6264

Courtney Ronaldo

(908) 442-5695

Investor Contacts:

Peter Dannenbaum

(732) 594-1579

Ayn Wisler

(917) 691-6218

Endeavor BioMedicines Announces Publication of Positive Data from Phase 2a Trial in The Lancet Respiratory Medicine Evaluating taladegib (ENV-101) in Individuals with Idiopathic Pulmonary Fibrosis




Endeavor BioMedicines Announces Publication of Positive Data from Phase 2a Trial in The Lancet Respiratory Medicine Evaluating taladegib (ENV-101) in Individuals with Idiopathic Pulmonary Fibrosis

Treatment with taladegib demonstrated improved lung function from baseline, increased total lung capacity and reversed key measures of lung fibrosis in patients with idiopathic pulmonary fibrosis

Data also presented today in ALERT session at European Respiratory Society (ERS) Congress 2025

SAN DIEGO–(BUSINESS WIRE)–Endeavor BioMedicines (“Endeavor”), a clinical-stage biotechnology company developing medicines with the potential to deliver transformational clinical benefits to patients with life-threatening diseases, today announced that The Lancet Respiratory Medicine has published results from a Phase 2a trial evaluating the safety and efficacy of taladegib (ENV-101) in patients with idiopathic pulmonary fibrosis (IPF). Data from the Phase 2a trial were also featured in an ALERT (Abstracts Leading to Evolution in Respiratory Medicine Trials) session at the 2025 ERS Congress in Amsterdam — a special forum highlighting high-impact, practice-changing clinical trials in respiratory medicine.

The proof-of-concept clinical trial demonstrated that treatment with taladegib improved lung function from baseline, increased total lung capacity and reversed key measures of lung fibrosis in patients with idiopathic pulmonary fibrosis. Taladegib also demonstrated a favorable safety and tolerability profile. There were no treatment-related serious adverse events, treatment-related ≥grade 3 adverse events, or clinically meaningful safety findings.

“Patients living with idiopathic pulmonary fibrosis face a relentless and life-altering disease that affects not only their health but every aspect of daily life,” said Toby M. Maher, M.D., Ph.D., Professor of Medicine and Director of Interstitial Lung Disease at Keck School of Medicine, University of Southern California, Los Angeles. “We are encouraged by the totality of the clinical data for taladegib, as we observed not only significant improvements from baseline in FVC measured by spirometry, but also a significant increase in total lung capacity and reductions from baseline in key measures of fibrosis as measured by CT imaging. These encouraging results, published in The Lancet Respiratory Medicine and presented today during the ERS ALERT session, represent meaningful progress toward the goal of delivering an effective treatment for patients living with IPF.”

The randomized, double-blind, placebo-controlled Phase 2a clinical trial evaluated the safety and efficacy of taladegib vs. placebo in 41 patients with confirmed IPF who were treated for 12 weeks. The trial was conducted at 16 clinical sites in Australia, Canada, Malaysia, Mexico and South Korea for patients with IPF older than 40 years of age who were not on background standard of care. Patients were randomized to 200 mg of taladegib or placebo administered once daily orally for 12 weeks with a 6-week follow-up. The primary endpoint was safety as assessed by the incidence and severity of clinical laboratory abnormalities, change from baseline in vital sign measurements, oxygen saturation, frequency and severity of adverse events, and number of hospitalizations during the study. Secondary endpoints included change from baseline in forced vital capacity (FVC), time to progression (defined as either an absolute decline of ≥10% in percent predicted FVC from baseline to week 12 or death) and dyspnea score on the UCSD SOBQ. Exploratory endpoints included measures of fibrosis assessed by high-resolution computed tomography (HRCT).

Key published findings from the taladegib Phase 2a clinical trial include:

• There were no serious adverse events or ≥grade 3 adverse events considered related to taladegib, and no deaths occurred during the trial.
• The most common taladegib-related adverse events were Grade 1 or Grade 2 and included alterations in taste, hair loss/thinning and muscle spasms.
• Patients who received taladegib experienced a statistically significant improvement in lung function assessed via spirometry through the 12 weeks of treatment.
  • The between-group difference from baseline to week 12 in percent predicted forced vital capacity (FVC) significantly favored taladegib (3.95%; 95% confidence interval [CI], 0.31% to 7.60%; p=0.035) with a mean change from baseline of 1.9% in the taladegib arm vs -1.3% for placebo.
  • By week 6, both percent predicted FVC and mean FVC (mL) exhibited an increase from baseline in the taladegib treatment group and a decrease in the placebo group that continued until the study drug was discontinued at week 12.
• Patients who received taladegib experienced improvement across several measures assessed by quantitative analysis of lung fibrosis on High Resolution Computed Tomography (HRCT) including in total lung capacity (TLC), percent quantitative total interstitial lung disease (%QILD), percent quantitative lung fibrosis (%QLF), and percent quantitative ground glass (%QGG).
  • Improvements were seen in TLC, %QILD, %QLF and %QGG for the taladegib treatment group, while these measures of disease remained stable or worsened for the placebo group.
  • The mean change from baseline to week 12 in TLC improved significantly for the taladegib group (206.67 mL; 95% CI, 82.63 to 330.70 mL) but decreased for the placebo group (–55.58 mL; 95% CI, –170.71 to 59.55 mL) resulting in a significant between-group change from baseline to week 12 in TLC of 257.0 mL (95% CI, 86.8 to 427.2 mL; p=0.004).
  • An improvement from baseline to week 12 was also seen for %QILD for the taladegib group but not for the placebo group, and there was a significant between-group difference in change from baseline to week 12 (p=0.047) with mean change from baseline of -9.4% and 1.1% in the taladegib and placebo groups, respectively.

“We are honored that the results of our Phase 2a trial have been published in The Lancet Respiratory Medicine, one of the world’s most respected and top-tier medical journals, and also selected as an ALERT presentation at ERS. This recognition is a testament to the dedication of our scientific and clinical teams, the investigators, and most importantly, the patients who participated in the trial,” said John Hood, Ph.D., Co-Founder, CEO and Chairman, Endeavor BioMedicines. “Our team remains committed to advancing taladegib with our current Phase 2b WHISTLE-PF trial, which is on track and expected to be completed in 2026.”

About Idiopathic Pulmonary Fibrosis

IPF is a chronic, progressive lung disease that affects more than 150,000 adults in the United States. Although the exact cause of IPF is unknown, various environmental factors can deliver repeated injuries to lung cells that trigger abnormal wound-healing processes and life-threatening lung scarring. IPF is a chronic disease with limited treatment options and a very poor prognosis: the average life expectancy is only three to five years after diagnosis.

About Taladegib

Endeavor BioMedicines’ investigational medicine taladegib (ENV-101) is a Hedgehog signaling pathway inhibitor. By binding to and inhibiting a key receptor in the Hedgehog pathway, taladegib eliminates the myofibroblasts that cause fibrosis. This may resolve the excessive wound-healing process seen in pulmonary fibrosis, improving lung volume and function.

About Endeavor BioMedicines

Endeavor BioMedicines is a clinical-stage biotechnology company developing medicines with the potential to deliver transformational clinical benefits to patients with life-threatening diseases. Endeavor’s lead candidate, taladegib (ENV-101), is an inhibitor of the Hedgehog signaling pathway in development for fibrotic lung diseases, including idiopathic pulmonary fibrosis (IPF). More information is available at www.endeavorbiomedicines.com and on LinkedIn or X.

Contacts

Media:
Audra Friis
Sam Brown, Inc.

917-519-9577

audrafriis@sambrown.com

Aurinia Responds to Now Retracted LinkedIn Post




Aurinia Responds to Now Retracted LinkedIn Post

ROCKVILLE, Md. & EDMONTON, Alberta–(BUSINESS WIRE)–Aurinia Pharmaceuticals Inc. (NASDAQ: AUPH) today responded to a now retracted LinkedIn post referencing voclosporin by an FDA official.

Aurinia stands behind the favorable benefit/risk profile of LUPKYNIS^® (voclosporin). LUPKYNIS received full approval from the FDA in January 2021 based on a large, randomized 52-week clinical study known as AURORA 1. Furthermore, the FDA approved a supplementary new drug application for the long-term use of LUPKYNIS in April 2024 based on the results of AURORA 2, which demonstrated sustained efficacy of LUPKYNIS over a three-year period, with safety comparable to AURORA 1.

Please see Prescribing Information, including Boxed Warning, for LUPKYNIS.

About Aurinia

Aurinia is a biopharmaceutical company focused on delivering therapies to people living with autoimmune diseases with high unmet medical needs. In January 2021, the Company introduced LUPKYNIS^® (voclosporin), the first FDA-approved oral therapy for the treatment of adult patients with active lupus nephritis. Aurinia is also developing aritinercept (AUR200), a dual inhibitor of B cell-activating factor (BAFF) and a proliferation-inducing ligand (APRIL) for the potential treatment of autoimmune diseases.

Contacts

General Inquiries
ir@auriniapharma.com

Bio-Rad Laboratories, Inc. Co-Founder and Director Emeritus Alice Schwartz Passes Away




Bio-Rad Laboratories, Inc. Co-Founder and Director Emeritus Alice Schwartz Passes Away

HERCULES, Calif.–(BUSINESS WIRE)–Bio-Rad Laboratories, Inc. (NYSE: BIO and BIO.B), a global leader in life science research and clinical diagnostics products, today announced that Alice N. Schwartz, Bio-Rad’s co-founder and Director Emeritus, passed away on September 25, 2025, at the age of 99.

Mrs. Schwartz co-founded Bio-Rad in 1952 in Berkeley, California, together with her husband David Schwartz, shortly after graduating with a biochemistry degree from the University of California, Berkeley.

The company began by developing specialty chemicals for life science research and later expanded into clinical diagnostics. Alice Schwartz was instrumental in the development of Bio-Rad’s first test kit for thyroid function in the 1960’s, which was based on separation techniques and materials developed for life science research. This innovation marked Bio-Rad’s entry into the field of clinical diagnostics, and set the stage for the company’s future growth and success.

Mrs. Schwartz played a key role in Bio-Rad’s achievements and remained an active Board member until 2022. Her leadership and passion for scientific discovery created a legacy that continues to inspire the company and the broader scientific community.

About Bio-Rad

Bio-Rad Laboratories, Inc. (NYSE: BIO and BIO.B) is a leader in developing, manufacturing, and marketing a broad range of products for the life science research and clinical diagnostics markets. Based in Hercules, California, Bio-Rad operates a global network of research, development, manufacturing, and sales operations with approximately 7,500 employees, and $2.6 billion in revenues in 2024. Our customers include universities, research institutions, hospitals, and biopharmaceutical companies, as well as clinical, food safety and environmental quality laboratories. Together, we develop innovative, high-quality products that advance science and save lives. To learn more, visit bio-rad.com.

Contacts

Investor Contact:
Edward Chung, Investor Relations

510-741-6104

ir@bio-rad.com

Media Contact:
Anna Gralinska, Corporate Communications

510-741-6643

cc@bio-rad.com

Enanta Pharmaceuticals Reports Positive Topline Results from its Phase 2b Study of Zelicapavir for the Treatment of Respiratory Syncytial Virus (RSV) in High-Risk Adults




Enanta Pharmaceuticals Reports Positive Topline Results from its Phase 2b Study of Zelicapavir for the Treatment of Respiratory Syncytial Virus (RSV) in High-Risk Adults

• 6.7-Day Improvement in Time to Complete Resolution of All RSV Symptoms for Patients with Chronic Obstructive Pulmonary Disease (COPD), Congestive Heart Failure (CHF), or Age ≥75
• Statistically Significant Improvement in Patient Global Impression of Severity Score
• Lower Hospitalization Rate for Patients Treated with Zelicapavir (1.7%) vs. Placebo (5%)
• 4- to 5-Day Faster Median Time to Undetectable Viral Load with Zelicapavir vs. Placebo
• Management to Host Conference Call and Webcast Today at 8:30 a.m. ET

WATERTOWN, Mass.–(BUSINESS WIRE)–Enanta Pharmaceuticals, Inc. (NASDAQ: ENTA), a clinical-stage biotechnology company dedicated to creating small molecule drugs for viral infections and immunological diseases, today announced positive topline data from RSVHR, a Phase 2b, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of zelicapavir in outpatient adults with acute RSV infection who are at high risk of complications including the elderly and/or those with congestive heart failure (CHF), chronic obstructive pulmonary disease (COPD) or asthma. Zelicapavir, which received Fast Track designation from the U.S. Food and Drug Administration (FDA), is a novel N-protein inhibitor in development as a once-daily oral treatment for RSV. This proof-of-concept study was designed to understand the antiviral treatment effect on symptom endpoints measured using the Respiratory Infection Intensity and Impact Questionnaire (RiiQ^TM) patient reported outcome tool, as well as other clinically meaningful endpoints, in a broad patient population.

A clinically meaningful improvement in time to complete resolution of all 13 RSV symptoms was observed for zelicapavir compared to placebo, with a benefit of 2.2 days for the overall efficacy population and 6.7 days for patients with CHF, COPD or age ≥75, termed the HR3 population, which comprised the majority (81%) of the efficacy population. Zelicapavir also showed an improvement in time to complete resolution on the 29-parameter total RiiQ™ symptom scale of 3.6 days for the efficacy population and 7.2 days for the HR3 population compared to placebo. Additionally, there was a 3.0-day faster time to complete resolution of lower respiratory tract disease (LRTD) symptoms in the HR3 population; however, no effect was observed on the time to resolution of the LRTD subset of four symptoms to mild, which was the primary endpoint. The study met the secondary endpoint of time to improvement in the Patient Global Impression of Severity (PGI-S) score, with a statistically significant 2-day faster resolution with zelicapavir compared to placebo. Importantly, a lower hospitalization rate was observed for patients treated with zelicapavir compared to placebo. The study met key secondary virology endpoints showing a robust antiviral effect. The study also showed that zelicapavir demonstrated a favorable safety profile and was well-tolerated.

“We are highly encouraged by these results from our Phase 2b trial of zelicapavir in high-risk adults infected with RSV. This represents the first time an RSV antiviral treatment has demonstrated a clinically meaningful benefit in these high-risk adult outpatients. These data demonstrate the potential for zelicapavir to reduce the duration of RSV symptoms in high-risk adults who face an increased risk of hospitalization or death from this virus,” said Scott T. Rottinghaus, M.D., Chief Medical Officer of Enanta Pharmaceuticals. “Building on the previously reported antiviral activity and favorable safety from our first-in-pediatrics study, we believe these findings continue to validate zelicapavir’s mechanism of action and reinforce its potential as a broadly effective, first-in-class RSV treatment. We believe the totality of these data provides strong rationale for further clinical advancement of zelicapavir. Importantly, we identified multiple potential registrational endpoints for a Phase 3 trial. We wish to thank the patients, family members and staff from all the sites who participated in the study. These results would not have been possible without their trust and involvement.”

“The patients enrolled in this study are particularly vulnerable to complications of RSV infection, often facing prolonged symptoms and heightened risk of hospitalization. Currently, there are no safe and effective antiviral RSV treatments available,” said Mohamed Fayed, M.D., UCSF School of Medicine Regional Campus at Fresno (UCSF Fresno), a Principal Investigator in the study. “The RSV symptom benefit observed in this study is compelling and could significantly improve outcomes for high-risk adults.”

Zelicapavir RSVHR Phase 2b Study Topline Results

RSVHR was a Phase 2b, randomized, double-blind, placebo-controlled study of RSV infection in non-hospitalized adults who are at high risk of complications, including the elderly and/or those with CHF, COPD or asthma. The proportion of patients aged 65-74 years or those with asthma was capped at 20% of the total population. Patients were enrolled within 72 hours of symptom onset and received 800mg of zelicapavir or placebo once daily for 5 days. The goal of this proof-of-concept, signal finding study was to inform the design of a Phase 3 trial, including populations and endpoints, as well as give an indication of a treatment effect on symptoms that could be confirmed in a larger registrational study. Symptoms were measured using the RiiQ^TM scale, which evaluates a total of 29 parameters, including 13 RSV symptoms, four of which are LRTD symptoms, and three other impact of disease components (daily activities, emotions, and social relationships). The primary endpoint evaluated the time to resolution of the LRTD subset of four symptoms to mild. Predefined analyses of complete resolution, defined by all symptoms absent, were also conducted. Multiple secondary endpoints, including all 13 RSV symptoms, total RiiQ^TM score, additional patient reported outcomes (e.g.; PGI-S), virology, safety, and hospitalization rate, were assessed.

A total of 186 subjects received 800mg of zelicapavir (n=121) or placebo (n=65) orally, once daily for 5 days and were evaluated for 28 days thereafter (safety population). An efficacy population of 175 patients was further defined as those who were PCR positive for RSV at a central laboratory. An HR3 population was defined as those who had CHF, COPD, or age >75 (81% of the efficacy population). Demographics and baseline characteristics were balanced across treatment groups, with the majority of patients being enrolled within 48 hours of symptom onset.

Zelicapavir demonstrated a favorable safety profile over the initial 5-day dosing period and through 28 days of follow-up, with adverse events (AEs) being similar between zelicapavir and placebo. No AEs led to treatment discontinuation or study withdrawal in the zelicapavir group. The majority of AEs were mild with diarrhea and asthma being the most common AEs on zelicapavir at 3.3% and 2.5%, respectively.

A clinically meaningful improvement in time to complete resolution (defined as all symptoms absent) of all 13 RSV symptoms and in the total RiiQ™ was observed for zelicapavir compared to placebo in both the efficacy and HR3 populations. There was also a faster time to complete resolution of the subset of four LRTD symptoms in the HR3 population.

Improvement in Time to Complete Resolution of Symptoms for Zelicapavir Compared to Placebo

No effect was observed on the time to resolution of symptoms (defined as mild), including the primary endpoint of time to resolution of the LRTD subset of four symptoms in the efficacy population.

Additionally, a statistically significant improvement in RiiQ^TM RSV 13-symptom score in the HR3 population at Days 9 (p=0.0403) and 14 (p=0.0247) was observed in a post-hoc analysis for zelicapavir compared to placebo.

Furthermore, the study met a key secondary endpoint with zelicapavir treatment resulting in a statistically significant 2-day faster improvement in a Patient Global Impression of Severity (PGI-S) score compared to placebo in both the efficacy population (p=0.0446) and the HR3 population (p=0.0465).

Importantly, a lower hospitalization rate was observed for patients treated with zelicapavir (1.7%) compared to placebo (5.0%). Blinded attribution by investigators judged none (0%) of the hospitalizations on zelicapavir and all (5.0%) of the hospitalization on placebo to be related to RSV. Post-hoc attribution suggested RSV-relatedness of 0.9% for the patients on zelicapavir compared to 5.0% on placebo.

The study met key secondary virology endpoints showing a robust antiviral effect, with a statistically significantly greater proportion of zelicapavir patients having an undetectable viral load at the end of treatment compared with placebo. In the efficacy population undetectable viral load at the end of treatment was 23.5% vs. 10.0% in placebo (p=0.0198), and in the HR3 population was 23.9% vs. 10.0% in placebo (p=0.0292). Treatment with zelicapavir resulted in a 4- or 5-day faster median time to undetectable viral load and a 0.6 or 0.7 log decline in viral load at the end of treatment compared to placebo for the efficacy and HR3 populations, respectively.

Full data from the study will be presented at a future medical conference or in a peer-reviewed publication.

Conference Call and Webcast Information

Enanta will host a conference call and webcast today at 8:30 a.m. ET. The live webcast can be accessed under “Events & Presentations” in the investors section of Enanta’s website. To participate by phone, please register for the call here. It is recommended that participants register a minimum of 15 minutes before the call. Once registered, participants will receive an email with the dial-in information. The archived webcast will be available on Enanta’s website for approximately 30 days following the event.

About Zelicapavir

Zelicapavir, Enanta’s lead N-protein inhibitor, is being developed for the treatment of RSV infection, and has been granted Fast Track designation by the U.S. Food and Drug Administration. Zelicapavir is a nanomolar inhibitor of both RSV-A and RSV-B activity. Zelicapavir is differentiated from RSV fusion inhibitors as the N-protein inhibitor targets the virus’ replication machinery and has demonstrated a high barrier to resistance in vitro. In preclinical studies, zelicapavir maintained antiviral potency across all clinical isolates tested and was active against viral variants resistant to other mechanisms. In a Phase 2 challenge study, zelicapavir achieved highly statistically significant (p<0.001) reductions in RSV viral load and clinical symptoms compared to placebo and was safe and well-tolerated, with infrequent adverse events. In a Phase 2 randomized, double-blind, placebo-controlled study of pediatric RSV patients aged 28 days to 3 years old, an antiviral effect was observed for the primary and secondary virology endpoints in the overall pooled efficacy population. The primary endpoint in Part 2 of the study, which focused on virology, showed a pronounced antiviral effect with a 1.4 log decline in viral load at Day 5 compared to placebo. Additionally, a rapid and robust virologic effect was observed in a prespecified subset of patients who were randomized within 3 days of symptom onset, with a 1.2 log decline in viral load at Day 5 compared to placebo. Zelicapavir has a favorable and consistent safety profile in over 600 people exposed to date.

About Respiratory Syncytial Virus in Older Adults

RSV is a common respiratory virus that infects the lungs and respiratory tract. Older adults are at significantly increased risk of severe illness due to the natural weakening of the immune system with age. This risk is even greater for individuals with underlying health conditions such as chronic obstructive pulmonary disease (COPD), asthma, or chronic heart failure. Those conditions can also be exacerbated by RSV, potentially lead to serious complications such as pneumonia, hospitalization, or even death. For adults aged 65 years and older, annually there are approximately 1.7 million medically attended visits, with 120K emergency room visits and between 160K-177K hospitalizations.^1,2

About Enanta Pharmaceuticals, Inc.

Enanta is using its robust, chemistry-driven approach and drug discovery capabilities to become a leader in the discovery and development of small molecule drugs with an emphasis on indications in virology and immunology. Enanta’s clinical programs are currently focused on respiratory syncytial virus (RSV) and its earlier-stage immunology pipeline aims to develop treatments for inflammatory diseases by targeting key drivers of the type 2 immune response, including KIT and STAT6 inhibition.

Glecaprevir, a protease inhibitor discovered by Enanta, is part of one of the leading treatment regimens for curing chronic and acute hepatitis C virus (HCV) infection and is sold by AbbVie in numerous countries under the tradenames MAVYRET® (U.S.) and MAVIRET® (ex-U.S.) (glecaprevir/pibrentasvir). A portion of Enanta’s royalties from HCV products developed under its collaboration with AbbVie contribute ongoing funding to Enanta’s operations. Please visit www.enanta.com for more information.

Forward Looking Statements

This press release contains forward-looking statements, including with respect to the prospects for further development and advancement of zelicapavir for the treatment of RSV. Statements that are not historical facts are based on management’s current expectations, estimates, forecasts and projections about Enanta’s business and the industry in which it operates and management’s beliefs and assumptions. The statements contained in this release are not guarantees of future performance and involve certain risks, uncertainties and assumptions, which are difficult to predict. Therefore, actual outcomes and results may differ materially from what is expressed in such forward-looking statements. Important factors and risks that may affect actual results include: the development risks of early stage discovery efforts in the disease areas in Enanta’s research and development pipeline, such as RSV; the impact of development, regulatory and marketing efforts of others with respect to competitive treatments for RSV; Enanta’s limited clinical development experience; Enanta’s need to attract and retain senior management and key scientific personnel; Enanta’s need to obtain and maintain patent protection for its product candidates and avoid potential infringement of the intellectual property rights of others; and other risk factors described or referred to in “Risk Factors” in Enanta’s most recent Annual Report on Form 10-K for the fiscal year ended September 30, 2024 and other periodic reports filed more recently with the Securities and Exchange Commission. Enanta cautions investors not to place undue reliance on the forward-looking statements contained in this release. All forward-looking statements contained in this release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Enanta undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

1. McLaughlin, John M et al. “Rates of Medically Attended RSV Among US Adults: A Systematic Review and Meta-analysis.” Open forum infectious diseases vol. 9,7 ofac300. 17 Jun. 2022, doi:10.1093/ofid/ofac300
2. Falsey, Ann R et al. “Respiratory Syncytial Virus Infection in Elderly and High-Risk Adults.” The New England journal of medicine vol. 352,17 (2005): 1749-59. doi:10.1056/NEJMoa043951

Contacts

Media and Investors Contact
Jennifer Viera

617-744-3848

jviera@enanta.com

International Day of Older Persons: AOP Health Shines a Light on the Silent Burden of Venous Leg Ulcers




International Day of Older Persons: AOP Health Shines a Light on the Silent Burden of Venous Leg Ulcers

VIENNA–(BUSINESS WIRE)–#AOPHealth—On the United Nations’ International Day of Older Persons, AOP Health draws attention to the often-invisible impact that Chronic Venous Ulcer (CVU) wounds have on older people. CVU are painful, slow-healing wounds that occur on the lower limb and can deprive people of mobility, independence, and dignity. Given the serious consequences, it’s vital to watch for early symptoms and speak to a doctor. Coordinated, multidisciplinary care can support recovery and independence even with established ulcers and slow healing.

“Many older people live with leg wounds that stay hidden—behind long pants, behind stigma, behind a lack of awareness,” says Melissa Fellner, Vice President Global Therapeutic Areas, AOP Health. “Among them are patients suffering from therapy-resistant Chronic Venous Ulcer (CVU) leg wounds, a painful condition assumed to affect several hundred thousand patients in Europe¹²³⁴⁵. The burden is high despite low public recognition, and we are determined to help end the sometimes-shameful silence around CVU.”

“Know the early signs and act,” adds Alessandra Antonello, Senior Director Global Medical Affairs, AOP Health. “If you notice leg swelling, skin changes, or a sore that does not heal, talk to your doctor promptly. Early assessment and guideline-based care can prevent ulcers or stop them from becoming chronic⁶.”

CVU is a serious problem – especially for older people

CVU develop and persist because diseased veins cannot return blood effectively to the heart⁷, damaging skin and tissue. If left untreated, they can lead to infection, prolonged pain, and disability⁷.

As populations age, chronic venous disease becomes more common, progressing from heaviness and swelling in the legs to skin changes and, in some cases, ulceration. These wounds can limit movement, reduce social participation, and increase the risk of complications and recurrence, making day-to-day life particularly hard for older adults. Currently, European vascular guidelines⁸ stress timely diagnosis and compression-based care pathways, which, with proper assessment and specialist-led management, can support healing and help prevent recurrence.

Who is at risk?

Well-known risk factors⁹ include advanced age, higher body weight, and physical inactivity – often in combination with varicose veins and chronic venous insufficiency (CVI).

Addressing Hard-to-Heal Venous Ulcers Together

Against this backdrop, AOP Health reaffirms its commitment to addressing high unmet medical needs – including therapy-resistant CVU – raising disease awareness, and by partnering with research-focused companies such as RHEACELL, a Germany-based biotech enterprise.

About AOP Health

AOP Health is a global enterprise group with roots in Austria, where the headquarters of AOP Orphan Pharmaceuticals GmbH (“AOP Health”) is located. Since 1996, the AOP Health Group has been dedicated to developing innovative solutions to address unmet medical needs, particularly in the fields of rare diseases and intensive care medicine. The group has established itself internationally as a pioneer in integrated therapy solutions and operates worldwide through subsidiaries, representations, and a strong network of partners. With the claim “Needs. Science. Trust.” the AOP Health Group emphasizes its commitment to research and development, as well as the importance of building relationships with physicians and patient advocacy groups to ensure that the needs of these stakeholders are reflected in all aspects of the company’s actions. (aop-health.com)

About RHEACELL

RHEACELL is a leading integrative biopharmaceutical stem cell company with over 20 years of experience based in Heidelberg, Germany. We focus on the development of innovative stem cell therapies for patients suffering from severe immune- and inflammation-related diseases, who have a very high level of suffering and for whom there are currently no adequate treatment options. Our products are based on ABCB5+ mesenchymal stromal cells as a pure active ingredient with a unique mechanism of action – applied topically or systemically, depending on the clinical picture – which enables the targeted control of inflammation and the restoration of normal physiological wound healing.

Contacts

Further inquiry
DI Isolde Fally

Isolde.Fally@aop-health.com
+43-676-500 4048

https://www.aop-health.com

ENHERTU® Demonstrated Highly Statistically Significant and Clinically Meaningful Improvement in Invasive Disease-Free Survival Versus T-DM1 in DESTINY-Breast05 Phase 3 Trial in Patients with High-Risk Early Breast Cancer Following Neoadjuvant Therapy




ENHERTU® Demonstrated Highly Statistically Significant and Clinically Meaningful Improvement in Invasive Disease-Free Survival Versus T-DM1 in DESTINY-Breast05 Phase 3 Trial in Patients with High-Risk Early Breast Cancer Following Neoadjuvant Therapy

• Second positive phase 3 trial of Daiichi Sankyo and AstraZeneca’s ENHERTU in HER2 positive early breast cancer reinforces its potential to become a foundational treatment option in curative-intent setting
• Results from the DESTINY-Breast05 and DESTINY-Breast11 trials will be presented at ESMO 2025 in a Presidential Symposium
• Plans for regulatory submissions are underway

TOKYO & BASKING RIDGE, N.J.–(BUSINESS WIRE)–Positive topline results from a planned interim analysis of the DESTINY-Breast05 phase 3 trial showed ENHERTU^® (trastuzumab deruxtecan) demonstrated a highly statistically significant and clinically meaningful improvement in invasive disease-free survival (IDFS) versus trastuzumab emtansine (T-DM1) in patients with HER2 positive early breast cancer with residual invasive disease in the breast or axillary lymph nodes after neoadjuvant treatment and high risk of disease recurrence. This is the second positive phase 3 trial of ENHERTU in the HER2 positive early breast cancer setting following positive results from the DESTINY-Breast11 phase 3 neoadjuvant trial earlier this year.

Overall survival (OS) was not mature at the time of this planned interim analysis and will be assessed at a subsequent analysis.

ENHERTU is a specifically engineered HER2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca (LSE/STO/Nasdaq: AZN).

Currently, approximately half of patients with HER2 positive early breast cancer have residual disease following neoadjuvant treatment, putting them at an increased risk of disease recurrence.^1-7 Despite receiving additional treatment in the post-neoadjuvant setting, some patients still ultimately experience tumor progression to metastatic disease. ^8,9 New treatment options are needed in the early breast cancer setting to help reduce the likelihood of disease progression and improve long-term outcomes for more patients.^10,11

“In patients with early breast cancer with residual disease following neoadjuvant treatment, it is critical to optimize treatment as this represents the last opportunity to prevent progression to metastatic disease,” said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. “The results of DESTINY-Breast05 demonstrate that treatment with ENHERTU following surgery increases the length of time patients are able to live free of invasive disease compared to the existing standard of care, potentially offering patients with HER2 positive early breast cancer a new treatment approach in this curative-intent setting.”

“This landmark trial is the first to directly compare ENHERTU and T-DM1 in early breast cancer and the results clearly show that ENHERTU delivers superior outcomes, indicating that it may be a better option for patients with high risk HER2 positive early breast cancer in the post-neoadjuvant setting,” said Susan Galbraith, MBBChir, PhD, Executive Vice President, Oncology Hematology R&D, AstraZeneca. “These results from DESTINY-Breast05, coupled with DESTINY-Breast11, underscore our commitment to moving ENHERTU into early-stage HER2 positive breast cancer where patients can achieve sustained long-term outcomes, increasing the opportunity for cure.”

The safety profile of ENHERTU observed in DESTINY-Breast05 was consistent with its known profile with no new safety concerns identified.

Data from DESTINY-Breast05 (Abstract #LBA1) and DESTINY-Breast11 (Abstract #291O) will be presented during Presidential Symposium I on Saturday, October 18 at the upcoming 2025 European Society for Medical Oncology (#ESMO25) Congress. The DESTINY-Breast05 data also will be shared with global regulatory authorities.

DESTINY-Breast05 was conducted in collaboration with the National Surgical Adjuvant Breast and Bowel Project Foundation (NSABP), the German Breast Group (GBG), Arbeitsgemeinschaft Gynäkologische Onkologie (AGO-B) and SOLTI Breast Cancer Research Group.

About DESTINY-Breast05

DESTINY-Breast05 is a global, multicenter, randomized, open-label, phase 3 trial evaluating the efficacy and safety of ENHERTU (5.4 mg/kg) versus T-DM1 in patients with HER2 positive primary breast cancer with residual invasive disease in breast or axillary lymph nodes following neoadjuvant therapy and a high risk of recurrence. High risk of recurrence was defined as presentation with inoperable cancer (prior to neoadjuvant therapy) or pathologically positive axillary lymph nodes following neoadjuvant therapy.

The primary endpoint of DESTINY-Breast05 is investigator-assessed IDFS. IDFS is defined as the time from randomization until first recurrence, distant recurrence or death from any cause. The key secondary endpoint is investigator-assessed disease-free survival. Other secondary endpoints include OS, distant recurrence-free interval, brain metastases-free interval and safety.

DESTINY-Breast05 enrolled 1,635 patients in Asia, Europe, North America, Oceania and South America. For more information about the trial, visit ClinicalTrials.gov.

About HER2 Positive Early Breast Cancer

Breast cancer is the second most common cancer and one of the leading causes of cancer-related deaths worldwide.¹² More than two million breast cancer cases were diagnosed in 2022, with more than 665,000 deaths globally.¹²

HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumors including breast cancer.¹³ HER2 protein overexpression may occur as a result of HER2 gene amplification and is often associated with aggressive disease and poor prognosis in breast cancer.¹⁴ Approximately one in five cases of breast cancer are considered HER2 positive.¹⁵

Currently, approximately half of patients with HER2 positive early breast cancer have residual disease following neoadjuvant treatment, putting them at an increased risk of disease recurrence.^1-7 Despite receiving additional treatment in the post-neoadjuvant setting, some patients still ultimately experience tumor progression to metastatic disease.^8,9 Once patients are diagnosed with metastatic disease, the five-year survival rate drops from nearly 90% to approximately 30%.¹⁶ New treatment options are needed in the early breast cancer setting to help reduce the likelihood of disease progression and improve long-term outcomes for more patients.^10,11

About ENHERTU

ENHERTU (trastuzumab deruxtecan; fam-trastuzumab deruxtecan-nxki in the U.S. only) is a HER2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, ENHERTU is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced program in AstraZeneca’s ADC scientific platform. ENHERTU consists of a HER2 monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

ENHERTU (5.4 mg/kg) is approved in more than 85 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2 positive (immunohistochemistry [IHC] 3+ or in-situ hybridization (ISH)+) breast cancer who have received a prior anti-HER2-based regimen, either in the metastatic setting or in the neoadjuvant or adjuvant setting, and have developed disease recurrence during or within six months of completing therapy based on the results from the DESTINY-Breast03 trial.

ENHERTU (5.4 mg/kg) is approved in more than 85 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2 low (IHC 1+ or IHC 2+/ISH-) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy based on the results from the DESTINY-Breast04 trial.

ENHERTU (5.4 mg/kg) is approved in more than 45 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic hormone receptor (HR) positive, HER2 low (IHC 1+ or IHC 2+ / ISH-) or HER2 ultralow (IHC 0 with membrane staining) breast cancer, as determined by a locally or regionally approved test, that has progressed on one or more endocrine therapies in the metastatic setting based on the results from the DESTINY-Breast06 trial.

ENHERTU (5.4 mg/kg) is approved in more than 60 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic NSCLC whose tumors have activating HER2 (ERBB2) mutations, as detected by a locally or regionally approved test, and who have received a prior systemic therapy based on the results from the DESTINY-Lung02 and/or DESTINY-Lung05 trials. Continued approval in China and the U.S. for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

ENHERTU (6.4 mg/kg) is approved in more than 70 countries/regions worldwide for the treatment of adult patients with locally advanced or metastatic HER2 positive (IHC 3+ or IHC 2+/ISH+) gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01, DESTINY-Gastric02 and/or DESTINY-Gastric06 trials. Continued approval in China for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

ENHERTU (5.4 mg/kg) is approved in more than 10 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2 positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options based on efficacy results from the DESTINY-PanTumor02, DESTINY-Lung01 and DESTINY-CRC02 trials. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

About the ENHERTU Clinical Development Program

A comprehensive global clinical development program is underway evaluating the efficacy and safety of ENHERTU as a monotherapy or in combination or sequentially with other cancer medicines across multiple HER2 targetable cancers.

About the Daiichi Sankyo and AstraZeneca Collaboration

Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize ENHERTU in March 2019 and DATROWAY^® in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of ENHERTU and DATROWAY.

About the ADC Portfolio of Daiichi Sankyo

The Daiichi Sankyo ADC portfolio consists of seven ADCs in clinical development crafted from two distinct ADC technology platforms discovered in-house by Daiichi Sankyo.

The ADC platform furthest in clinical development is Daiichi Sankyo’s DXd ADC Technology where each ADC consists of a monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. The DXd ADC portfolio currently consists of ENHERTU, a HER2 directed ADC, and DATROWAY, a TROP2 directed ADC, which are being jointly developed and commercialized globally with AstraZeneca. Patritumab deruxtecan (HER3-DXd), a HER3 directed ADC, ifinatamab deruxtecan (I-DXd), a B7-H3 directed ADC, and raludotatug deruxtecan (R-DXd), a CDH6 directed ADC, are being jointly developed and commercialized globally with Merck & Co., Inc, Rahway, NJ, USA. DS-3939, a TA-MUC1 directed ADC, is being developed by Daiichi Sankyo.

The second Daiichi Sankyo ADC platform consists of a monoclonal antibody attached to a modified pyrrolobenzodiazepine (PBD) payload. DS-9606, a CLDN6 directed PBD ADC, is the first of several planned ADCs in clinical development utilizing this platform.

Ifinatamab deruxtecan, patritumab deruxtecan, raludotatug deruxtecan, DS-3939 and DS-9606 are investigational medicines that have not been approved for any indication in any country. Safety and efficacy have not been established.

ENHERTU U.S. Important Safety Information

Indications

ENHERTU is a HER2-directed antibody and topoisomerase inhibitor conjugate indicated for the treatment of adult patients with:

• Unresectable or metastatic HER2-positive (IHC 3+ or ISH positive) breast cancer who have received a prior anti-HER2-based regimen either:
  • In the metastatic setting, or
  • In the neoadjuvant or adjuvant setting and have developed disease recurrence during or within six months of completing therapy

• Unresectable or metastatic:
  • Hormone receptor (HR)-positive, HER2-low (IHC 1+ or IHC 2+/ISH-) or HER2-ultralow (IHC 0 with membrane staining) breast cancer, as determined by an FDA-approved test, that has progressed on one or more endocrine therapies in the metastatic setting
  • HER2-low (IHC 1+ or IHC 2+/ISH-) breast cancer, as determined by an FDA-approved test, who have received a prior chemotherapy in the metastatic setting or developed disease recurrence during or within 6 months of completing adjuvant chemotherapy

• Unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy

  This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
  

• Locally advanced or metastatic HER2-positive (IHC 3+ or IHC 2+/ISH positive) gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen

• Unresectable or metastatic HER2-positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options

  This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Contraindications

None.

Warnings and Precautions

Interstitial Lung Disease / Pneumonitis

Severe, life-threatening, or fatal interstitial lung disease (ILD), including pneumonitis, can occur in patients treated with ENHERTU. A higher incidence of Grade 1 and 2 ILD/pneumonitis has been observed in patients with moderate renal impairment. Advise patients to immediately report cough, dyspnea, fever, and/or any new or worsening respiratory symptoms. Monitor patients for signs and symptoms of ILD. Promptly investigate evidence of ILD. Evaluate patients with suspected ILD by radiographic imaging. Consider consultation with a pulmonologist. For asymptomatic ILD/pneumonitis (Grade 1), interrupt ENHERTU until resolved to Grade 0, then if resolved in ≤28 days from date of onset, maintain dose. If resolved in >28 days from date of onset, reduce dose 1 level. Consider corticosteroid treatment as soon as ILD/pneumonitis is suspected (e.g., ≥0.5 mg/kg/day prednisolone or equivalent). For symptomatic ILD/pneumonitis (Grade 2 or greater), permanently discontinue ENHERTU. Promptly initiate systemic corticosteroid treatment as soon as ILD/pneumonitis is suspected (e.g., ≥1 mg/kg/day prednisolone or equivalent) and continue for at least 14 days followed by gradual taper for at least 4 weeks.

HER2-Positive, HER2-Low, and HER2-Ultralow Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

In patients with metastatic breast cancer, HER2-mutant NSCLC, and other solid tumors treated with ENHERTU 5.4 mg/kg, ILD occurred in 12% of patients. Median time to first onset was 5.5 months (range: 0.9 to 31.5). Fatal outcomes due to ILD and/or pneumonitis occurred in 0.9% of patients treated with ENHERTU.

HER2-Positive Locally Advanced or Metastatic Gastric Cancer (6.4 mg/kg)

In patients with locally advanced or metastatic HER2-positive gastric or GEJ adenocarcinoma treated with ENHERTU 6.4 mg/kg, ILD occurred in 10% of patients. Median time to first onset was 2.8 months (range: 1.2 to 21).

Neutropenia

Severe neutropenia, including febrile neutropenia, can occur in patients treated with ENHERTU. Monitor complete blood counts prior to initiation of ENHERTU and prior to each dose, and as clinically indicated. For Grade 3 neutropenia (Absolute Neutrophil Count [ANC] <1.0 to 0.5 x 10⁹/L), interrupt ENHERTU until resolved to Grade 2 or less, then maintain dose. For Grade 4 neutropenia (ANC <0.5 x 10⁹/L), interrupt ENHERTU until resolved to Grade 2 or less, then reduce dose by 1 level. For febrile neutropenia (ANC <1.0 x 10⁹/L and temperature >38.3º C or a sustained temperature of ≥38º C for more than 1 hour), interrupt ENHERTU until resolved, then reduce dose by 1 level.

HER2-Positive, HER2-Low, and HER2-Ultralow Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

In patients with metastatic breast cancer, HER2-mutant NSCLC, and other solid tumors treated with ENHERTU 5.4 mg/kg, a decrease in neutrophil count was reported in 65% of patients. Nineteen percent had Grade 3 or 4 decreased neutrophil count. Median time to first onset of decreased neutrophil count was 22 days (range: 2 to 939). Febrile neutropenia was reported in 1.2% of patients.

HER2-Positive Locally Advanced or Metastatic Gastric Cancer (6.4 mg/kg)

In patients with locally advanced or metastatic HER2-positive gastric or GEJ adenocarcinoma treated with ENHERTU 6.4 mg/kg, a decrease in neutrophil count was reported in 72% of patients. Fifty-one percent had Grade 3 or 4 decreased neutrophil count. Median time to first onset of decreased neutrophil count was 16 days (range: 4 to 187). Febrile neutropenia was reported in 4.8% of patients.

Left Ventricular Dysfunction

Patients treated with ENHERTU may be at increased risk of developing left ventricular dysfunction. Left ventricular ejection fraction (LVEF) decrease has been observed with anti-HER2 therapies, including ENHERTU. Assess LVEF prior to initiation of ENHERTU and at regular intervals during treatment as clinically indicated. Manage LVEF decrease through treatment interruption. When LVEF is >45% and absolute decrease from baseline is 10-20%, continue treatment with ENHERTU. When LVEF is 40-45% and absolute decrease from baseline is 20%, interrupt ENHERTU and repeat LVEF assessment within 3 weeks. If LVEF of 20% is confirmed, permanently discontinue ENHERTU. Permanently discontinue ENHERTU in patients with symptomatic congestive heart failure. Treatment with ENHERTU has not been studied in patients with a history of clinically significant cardiac disease or LVEF <50% prior to initiation of treatment.

HER2-Positive, HER2-Low, and HER2-Ultralow Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

In patients with metastatic breast cancer, HER2-mutant NSCLC, and other solid tumors treated with ENHERTU 5.4 mg/kg, LVEF decrease was reported in 4.6% of patients, of which 0.6% were Grade 3 or 4.

HER2-Positive Locally Advanced or Metastatic Gastric Cancer (6.4 mg/kg)

In patients with locally advanced or metastatic HER2-positive gastric or GEJ adenocarcinoma treated with ENHERTU 6.4 mg/kg, no clinical adverse events of heart failure were reported; however, on echocardiography, 8% were found to have asymptomatic Grade 2 decrease in LVEF.

Embryo-Fetal Toxicity

ENHERTU can cause fetal harm when administered to a pregnant woman. Advise patients of the potential risks to a fetus. Verify the pregnancy status of females of reproductive potential prior to the initiation of ENHERTU. Advise females of reproductive potential to use effective contraception during treatment and for 7 months after the last dose of ENHERTU. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ENHERTU and for 4 months after the last dose of ENHERTU.

Additional Dose Modifications

Thrombocytopenia
For Grade 3 thrombocytopenia (platelets <50 to 25 x 10⁹/L) interrupt ENHERTU until resolved to Grade 1 or less, then maintain dose. For Grade 4 thrombocytopenia (platelets <25 x 10⁹/L) interrupt ENHERTU until resolved to Grade 1 or less, then reduce dose by 1 level.

Adverse Reactions

HER2-Positive, HER2-Low, and HER2-Ultralow Metastatic Breast Cancer, HER2-Mutant NSCLC, and Solid Tumors (Including IHC 3+) (5.4 mg/kg)

The pooled safety population reflects exposure to ENHERTU 5.4 mg/kg intravenously every 3 weeks in 2233 patients in Study DS8201-A-J101 (NCT02564900), DESTINY-Breast01, DESTINY-Breast02, DESTINY-Breast03, DESTINY-Breast04, DESTINY-Breast06, DESTINY-Lung01, DESTINY-Lung02, DESTINY-CRC02, and DESTINY-PanTumor02. Among these patients, 67% were exposed for >6 months and 38% were exposed for >1 year. In this pooled safety population, the most common (≥20%) adverse reactions, including laboratory abnormalities, were decreased white blood cell count (73%), nausea (72%), decreased hemoglobin (67%), decreased neutrophil count (65%), decreased lymphocyte count (60%), fatigue (55%), decreased platelet count (48%), increased aspartate aminotransferase (46%), increased alanine aminotransferase (44%), increased blood alkaline phosphatase (39%), vomiting (38%), alopecia (37%), constipation (32%), decreased blood potassium (32%), decreased appetite (31%), diarrhea (30%), and musculoskeletal pain (24%).

HER2-Positive Metastatic Breast Cancer

DESTINY-Breast03
The safety of ENHERTU was evaluated in 257 patients with unresectable or metastatic HER2-positive breast cancer who received at least 1 dose of ENHERTU 5.4 mg/kg intravenously once every 3 weeks in DESTINY-Breast03. The median duration of treatment was 14 months (range: 0.7 to 30) for patients who received ENHERTU.

Serious adverse reactions occurred in 19% of patients receiving ENHERTU. Serious adverse reactions in >1% of patients who received ENHERTU were vomiting, ILD, pneumonia, pyrexia, and urinary tract infection. Fatalities due to adverse reactions occurred in 0.8% of patients including COVID-19 and sudden death (1 patient each).

ENHERTU was permanently discontinued in 14% of patients, of which ILD/pneumonitis accounted for 8%. Dose interruptions due to adverse reactions occurred in 44% of patients treated with ENHERTU. The most frequent adverse reactions (>2%) associated with dose interruption were neutropenia, leukopenia, anemia, thrombocytopenia, pneumonia, nausea, fatigue, and ILD/pneumonitis. Dose reductions occurred in 21% of patients treated with ENHERTU. The most frequent adverse reactions (>2%) associated with dose reduction were nausea, neutropenia, and fatigue.

The most common (≥20%) adverse reactions, including laboratory abnormalities, were nausea (76%), decreased white blood cell count (74%), decreased neutrophil count (70%), increased aspartate aminotransferase (67%), decreased hemoglobin (64%), decreased lymphocyte count (55%), increased alanine aminotransferase (53%), decreased platelet count (52%), fatigue (49%), vomiting (49%), increased blood alkaline phosphatase (49%), alopecia (37%), decreased blood potassium (35%), constipation (34%), musculoskeletal pain (31%), diarrhea (29%), decreased appetite (29%), headache (22%), respiratory infection (22%), abdominal pain (21%), increased blood bilirubin (20%), and stomatitis (20%).

Contacts

Media Contacts:

Global/US:
Jennifer Brennan

Daiichi Sankyo

jennifer.brennan@daiichisankyo.com
+1 908 900 3183 (mobile)

Japan:
Daiichi Sankyo Co., Ltd.

DS-PR_jp@daiichisankyo.com

Investor Relations Contact:
DaiichiSankyoIR_jp@daiichisankyo.com

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