Medera Advances SRD-001 Gene Therapy to Phase 2 Trial for Heart Failure with Reduced Ejection Fraction
核心洞察
Medera Inc. (搜索) has successfully dosed the first patient in the Phase 2 portion of its MUSIC-HFrEF trial evaluating SRD-001 gene therapy for heart failure with reduced ejection fraction.
The advancement follows a positive recommendation from the independent Data and Safety Monitoring Board based on data from nine patients in the Phase 1b portion.
The Phase 2 study is a randomized, double-blind, placebo-controlled trial with 50 patients using a 1:1 randomization design to evaluate efficacy and safety.
Medera Inc. (搜索), a clinical-stage biopharmaceutical company, has achieved a significant milestone by dosing the first patient in the Phase 2 portion of its MUSIC-HFrEF Phase 1b/2 clinical trial evaluating SRD-001 gene therapy for heart failure with reduced ejection fraction (HFrEF). The patient was successfully treated on September 5, 2025, with additional patients scheduled for treatment.
The advancement to Phase 2 follows a positive recommendation from the independent Data and Safety Monitoring Board (DSMB) in March 2025, which reviewed data from all nine patients in the Phase 1b portion and recommended advancing the trial based on their findings.
Trial Design and Patient Population
The Phase 2 portion is designed as a randomized, double-blind, placebo-controlled study with a 1:1 randomization comparing placebo versus a 4.5x10¹³ viral genome injected dose per patient. The study will enroll a total of 50 patients to further evaluate the efficacy and safety of SRD-001 in a larger patient population with advanced heart failure.
The patient was treated using Medera's proprietary minimally invasive intracoronary infusion methodology and tolerated the procedure well, according to the company's announcement.
Clinical Significance and Market Impact
HFrEF represents a prevalent form of heart disease that accounts for half of an estimated 64.3 million heart failure cases worldwide. The condition is currently considered a mass market incurable disease, highlighting the significant unmet medical need that SRD-001 aims to address.
"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."
Gene Therapy Mechanism
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 (搜索)). The therapy is designed for 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.
Disease Background and Treatment Need
HFrEF is described as a complex cardiovascular pathophysiological syndrome that impairs normal cardiac function and results in the heart's inability to pump sufficient blood to meet the body's demand. Once established, HFrEF is generally progressive and irreversible, associated with debilitating symptoms, frequent re-hospitalizations, and high mortality rates. This creates an urgent need for disease-modifying therapies like SRD-001, which aim to reverse the pathophysiology of HFrEF.
"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."
Company Pipeline and Platform
Medera operates through two business units: Novoheart for preclinical research and Sardocor for clinical development. Sardocor has received Investigational New Drug (IND) clearances from the FDA for three ongoing AAV-based cardiac gene therapy clinical trials, including the current HFrEF trial, a Heart Failure with Preserved Ejection Fraction (HFpEF) trial with Fast Track Designation, and a Duchenne Muscular Dystrophy-associated Cardiomyopathy (DMD-CM) trial with Orphan Drug Designation.