Medera Inc., via its subsidiary Sardocor Corp., has announced the completion of Cohort A and the initiation of dosing in Cohort B of the MUSIC-HFpEF Phase 1/2a clinical trial. This trial is evaluating SRD-002, a first-in-human cardiac gene therapy, for the treatment of Heart Failure with Preserved Ejection Fraction (HFpEF).
The MUSIC-HFpEF trial (NCT06061549) is an open-label, dose-escalation study designed to assess the safety, tolerability, and preliminary efficacy of SRD-002 over 52 weeks in HFpEF patients. Following positive recommendations from the Data Safety Monitoring Board (DSMB), the trial has advanced to Cohort B, which will evaluate a higher dose of the gene therapy.
SRD-002: A Novel Gene Therapy Approach
SRD-002 utilizes an adeno-associated virus (AAV) vector to deliver the cardiac isoform of the sarcoplasmic reticulum calcium ATPase pump (SERCA2a). SERCA2a plays a crucial role in cardiac muscle relaxation. The therapy is administered via Sardocor's proprietary one-time intracoronary infusion methodology. Preclinical data using bioengineered human mini-hearts, co-developed with AstraZeneca by Medera's Novoheart subsidiary, demonstrated that SRD-002 improved relaxation defects in HFpEF models.
Clinical Progress and Interim Data
In Cohort A, five patients received a low dose of SRD-002 (3x10^13 vg per patient). Preliminary efficacy results at six months showed promising improvements in cardiovascular performance, with no unexpected adverse events or toxicities reported. Based on these findings, the DSMB recommended proceeding to Cohort B, where five additional patients will receive a higher dose of SRD-002 (4.5x10^13 vg per patient). Enrollment in Cohort B is expected to be completed in the first quarter of 2025, with an interim data readout anticipated in the first half of 2025.
The Unmet Need in HFpEF
Heart failure is a global pandemic, affecting an estimated 64.3 million people worldwide and costing over US$100 billion annually. HFpEF accounts for nearly half of all heart failure cases. While treatments like sacubitril-valsartan (Entresto™) and empagliflozin (Jardiance™) have shown some benefit in reducing cardiovascular mortality and heart failure hospitalization, there remains a significant need for disease-modifying therapies that target the underlying physiological mechanisms of HFpEF.
Expert Commentary
"New treatment approaches, like Medera's gene therapy product, are critical for patients and caregivers faced with this devastating disease," said Marat Fudim, MD, MHS, Advanced Heart Failure Specialist and Associate Professor at Duke University Medical Centre and site principal investigator. Roger Hajjar, MD, President and co-founder of Medera, added, "The progression of MUSIC-HFpEF, with dosing underway in the second cohort, is a key milestone in the clinical development of our gene therapy candidate SRD-002 as a potential effective treatment for HFpEF patients, who still have significant unmet needs."
Medera and Keen Vision Acquisition Corporation ("KVAC") (Nasdaq: KVAC, KVACW) previously announced a definitive merger agreement on September 5, 2024.
