Medera Announces First Patient Dosed in Phase 2 Portion of MUSIC-HFrEF Phase 1b/2 Trial Evaluating SRD-001 Gene Therapy for Heart Failure with Reduced Ejection Fraction
BOSTON, Oct. 27, 2025 (GLOBE NEWSWIRE) -- Medera Inc. ("Medera"), a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing next-generation therapeutics, today announced that the first patient has been dosed in the Phase 2 portion of the MUSIC-HFrEF Phase 1b/2 clinical trial evaluating the gene therapy candidate SRD-001 in patients with heart failure with reduced ejection fraction (HFrEF). The patient was successfully treated on September 5, 2025, and additional patients are scheduled for treatment.
HFrEF is a prevalent form of heart disease that accounts for half of an estimated 64.3 million heart failure cases worldwide and is currently considered a mass market incurable disease.
The Phase 2 portion is a randomized, double-blind, placebo-controlled study with a 1:1 randomization (placebo versus 4.5x1013vg injected dose per patient) and a total sample size of 50 patients to further evaluate the efficacy and safety of SRD-001 in a larger patient population. The initiation of Phase 2 follows the independent Data and Safety Monitoring Board's (DSMB) recommendation in March 2025 to advance the trial based on its review of data from all nine patients in the Phase 1b portion.
The patient was treated using Medera's proprietary minimally invasive intracoronary infusion methodology and tolerated the procedure well.
"The initiation of the placebo-controlled Phase 2 portion marks an important step forward in evaluating the efficacy and safety of SRD-001 in a larger patient population with advanced heart failure," said Brian Jaski, M.D., Principal Investigator of MUSIC-HFrEF and Scientific Director of Research at the San Diego Cardiac Center. "We are encouraged by the progress of this cardiac gene therapy trial for patients with heart failure with reduced ejection fraction."
"We are pleased to announce this important milestone for the MUSIC-HFrEF program," said Ronald Li, Ph.D., CEO and co-founder of Medera. "The Phase 2 portion will further evaluate SRD-001 and advance our clinical program as we work toward developing next-generation therapeutics for HFrEF patients."
For more information on the MUSIC-HFrEF trial, please visit clinicaltrials.gov/study/NCT04703842.
About SRD-001
SRD-001 is an investigational gene therapy candidate that contains an adeno-associated virus serotype 1 (AAV1) vector expressing the transgene for sarco(endo)plasmic reticulum Ca2+ ATPase 2a isoform (SERCA2a), in anti-AAV1 neutralizing antibody (NAb) negative subjects with ischemic or non-ischemic cardiomyopathy and New York Heart Association (NYHA) class III/IV symptoms of heart failure with reduced ejection fraction (HFrEF).
About Heart Failure with reduced Ejection Fraction (HFrEF)
HFrEF is a complex cardiovascular pathophysiological syndrome that impairs normal cardiac function and results in the inability of the heart to pump a sufficient supply of blood to meet the body demand. Once established, HFrEF is generally progressive, irreversible, associated with debilitating symptoms, frequent re-hospitalizations and high mortality rates. An urgent need exists for disease modifying therapies, such as SRD-001, which aim to reverse the pathophysiology of HFrEF.
About Medera Inc.
Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases by developing a range of next-generation therapeutics. Medera operates via its two preclinical and clinical business units, Novoheart and Sardocor, respectively.
Novoheart capitalizes on the world’s first and award-winning “mini-Heart” Technology for revolutionary disease modelling and drug discovery, uniquely enabling the modelling of human-specific diseases and discovery of therapeutic candidates free from species-specific differences in accordance to the FDA Modernization Act 2.0. Novoheart's versatile technology platform provides a range of state-of-the-art automation hardware and software as well as screening services, for human-specific disease modelling, therapeutic target discovery and validation, drug toxicity and efficacy screening, and dosage optimization carried out in the context of healthy and/or diseased human heart chambers and tissues. Global pharmaceutical and academic leaders are using Novoheart's technology platform for their drug discovery and development purposes. The Novoheart platform has facilitated and accelerated the development of Sardocor's lead therapeutic candidates that are currently in clinical trials.
Sardocor is dedicated to the clinical development of novel next-generation therapies for Medera. Leveraging Novoheart’s human-based drug discovery and validation platforms, Sardocor aims to expedite drug development and regulatory timelines for its gene and cell therapy pipeline. Sardocor has received Investigational New Drug (IND) clearances from the FDA for three ongoing AAV-based cardiac gene therapy clinical trials targeting Heart Failure with Reduced Ejection Fraction (HFrEF), Heart Failure with Preserved Ejection Fraction (HFpEF) with the Fast Track Designation, and Duchenne Muscular Dystrophy-associated Cardiomyopathy (DMD-CM) with the Orphan Drug Designation. Additionally, Sardocor's pipeline includes four preclinical gene therapy and three preclinical small molecule candidates targeting various cardiac, pulmonary, and vascular diseases.
For more information, please visit www.medera.bio.
Contacts
Ally Stubin
Public Relations
ICR Healthcare
Ally.stubin@icrhealthcare.com
646.667.1861
Stephanie Carrington
Investor Relations
ICR Healthcare
Stephanie.carrington@icrhealthcare.com
646.277.1282
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