Medera's Gene Therapy Shows Promise in Early Trial for Heart Failure with Preserved Ejection Fraction
May 19, 2025
Interim results from the trial indicate a favorable safety profile, with no serious adverse events reported among the five patients treated with a low dose and one patient at a higher dose.
The trial continues to enroll patients at the higher dose of 4.5x10^13 viral genomes per patient, with ongoing monitoring for safety and efficacy.
The presentation, titled "Gene therapy for heart failure with preserved ejection fraction," was delivered by Dr. Marat Fudim, highlighting the innovative approach to treating this condition.
The trial focuses on patients with heart failure with preserved ejection fraction (HFpEF), which affects around half of heart failure patients globally and currently lacks effective disease-modifying treatments.
SRD-002 utilizes an adeno-associated type 1 virus vector to deliver the cardiac isoform of the sarcoplasmic reticulum calcium ATPase pump (SERCA2a), aiming to enhance myocardial relaxation and reduce stiffness.
Medera's subsidiary, Sardocor, has received FDA clearance for three AAV-based cardiac gene therapy clinical trials targeting heart failure and Duchenne Muscular Dystrophy-associated cardiomyopathy.
Notably, four out of five patients in the low-dose group showed improvements in heart failure symptoms after six months, including better performance in the 6-minute walk test and stabilization in NT-Pro-BNP levels.
The Heart Failure Congress serves as a significant platform for showcasing advances in heart failure research and treatment options, underscoring the importance of ongoing innovation in this field.
Medera Inc. recently presented positive interim data from its Phase 1/2a MUSIC-HFpEF clinical trial for the gene therapy SRD-002 at the Heart Failure 2025 Congress in Belgrade, Serbia.
HFpEF is associated with significant morbidity and mortality, yet has seen limited success in interventional trials, making the need for new therapies critical.
Medera's CEO, Dr. Ronald Li, expressed optimism about the early clinical signals from the trial and emphasized the importance of advancing treatments for HFpEF.
As patient enrollment continues, the trial aims to address the significant unmet medical need represented by HFpEF, which currently has limited treatment options.
Summary based on 3 sources
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The Manila Times • May 12, 2025
Medera to Present Late-Breaking Phase 1/2a Clinical Trial Results at Heart Failure 2025 Congress