Medera has completed patient dosing in Cohort B of its ongoing MUSIC-HFpEF Phase I/IIa study of SRD-002 for heart failure with preserved ejection fraction (HFpEF).
The last patient was administered treatment through Medera’s intracoronary infusion approach.
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SRD-002 is a single-administration gene therapy delivered via minimally invasive intracoronary infusion.
It uses an adeno-associated type 1 virus vector with the cardiac form of the sarcoplasmic reticulum calcium ATPase pump (SERCA2a), directly targeting pathways linked to HFpEF such as diastolic dysfunction, calcium handling, and myocardial stiffness.
Cohort B received higher doses, significantly above prior studies but below systemic intravenous (IV) dosing levels.
Dose selection was optimised using Medera’s human-based mini-Heart technology platform co-developed with AstraZeneca, supporting US Food and Drug Administration (FDA) investigational new drug (IND) clearance and fast track designation.
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By GlobalDataNo serious adverse events related to gene therapy have been observed in either cohort. Patients in Cohort A (low-dose) have finished 12 months of follow-up, showing enhanced New York Heart Association (NYHA) classification and better scores on the Kansas City Cardiomyopathy Questionnaire.
Additionally, improvements and stabilisation were observed in pulmonary capillary wedge pressure during both rest and peak exercise.
Medera CEO and founder Ronald Li said: “With all five patients in Cohort B now dosed at the higher therapeutic dose, we continue to build a robust safety and efficacy dataset across both cohorts.
“The very encouraging results to date support our confidence in advancing this first-in-human gene therapy approach for patients with HFpEF, a condition affecting approximately half of all heart failure patients worldwide with limited disease-modifying therapeutic options.”
Cohort B patients are currently being monitored for efficacy and safety as per protocol. The study will follow all patients for 24 months post-treatment to generate long-term data guiding further clinical development.
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