Medera Completes Cohort A and Starts Dosing Cohort B in MUSIC-HFpEF Gene Therapy Trial for Heart Failure

Heart failure is a significant global health issue, affecting an estimated 64.3 million people worldwide and costing over $100 billion annually. Among the various types of heart failure, Heart Failure with Preserved Ejection Fraction (HFpEF) constitutes nearly half of all cases but has limited treatment options that modify the disease itself. In light of this, Medera Inc. has announced promising progress in its clinical-stage gene therapy subsidiary, Sardocor Corp.

Medera Inc., based in Summit, N.J., and Boston, revealed that Sardocor Corp. has successfully completed dosing patients in Cohort A (low-dose) of its Phase 1/2a clinical trial for their cardiac gene therapy candidate, SRD-002. Following the positive results and recommendations from the Data Safety Monitoring Board (DSMB), the company has begun dosing patients in Cohort B (high-dose).

SRD-002 is a pioneering gene therapy treatment designed to address HFpEF. The clinical trial, named MUSIC-HFpEF, aims to assess the safety, tolerability, and preliminary efficacy of SRD-002, administered via a proprietary one-time intracoronary infusion. This therapy utilizes an adeno-associated virus (AAV) vector carrying the cardiac isoform of the sarcoplasmic reticulum calcium ATPase pump (SERCA2a). The role of SERCA2a in heart muscle relaxation has been demonstrated in bioengineered human mini-Heart HFpEF models developed by Novoheart, a subsidiary of Medera, in collaboration with AstraZeneca.

In the initial phase of the trial (Cohort A), five patients received a low dose of SRD-002, showing promising efficacy in cardiovascular performance at the six-month mark without unexpected events or toxicities. Encouraged by these results, the trial has progressed to Cohort B, where another group of five patients will receive a higher dose of 4.5x10^13 vg per patient.

Dr. Marat Fudim, an Advanced Heart Failure Specialist at Duke University Medical Centre and site principal investigator, emphasized the importance of new treatments like SRD-002 for patients and caregivers dealing with HFpEF. He expressed optimism about the continued research in the second cohort.

Roger Hajjar, President and co-founder of Medera, highlighted the significance of this milestone. He noted that SRD-002 has shown promising efficacy and has been well-tolerated in patients, with ongoing data collection and analysis. The company aims to complete enrollment in the second cohort by the first quarter of 2025, with an interim data readout anticipated in the first half of 2025.

The MUSIC-HFpEF trial is a 52-week open-label, dose-escalation study designed to evaluate the safety and tolerability of SRD-002 in patients diagnosed with HFpEF. The data from Novoheart’s mini-Heart models have been crucial in optimizing dosages and supporting the Fast Track Designation (FTD) granted by the FDA for this trial.

Medera operates through two units: Novoheart and Sardocor. Novoheart focuses on disease modeling and drug discovery using its mini-Heart technology, enabling the study of human-specific diseases and therapeutic candidates. Sardocor is dedicated to developing next-generation therapies, leveraging Novoheart’s platforms to expedite drug development.

Sardocor has received Investigational New Drug (IND) clearances from the FDA for three AAV-based cardiac gene therapy trials targeting different forms of heart failure, including the Fast Track Designation for HFpEF and Orphan Drug Designation for Duchenne Muscular Dystrophy-induced Cardiomyopathy.

In summary, Medera’s advancements in gene therapy for HFpEF offer hope for a condition with limited treatment options. The ongoing clinical trials and promising preliminary results signify a crucial step towards developing effective therapies for heart failure patients worldwide.