Medera Inc. Presents Positive Interim Data for AAV Gene Therapy in HFpEF at Heart Failure 2025

Boston, MA – May 19, 2025 – Medera Inc. announced encouraging interim results from its MUSIC-HFpEF Phase 1/2a clinical trial of SRD-002, a one-time adeno-associated type 1 virus (AAV1) gene therapy for heart failure with preserved ejection fraction (HFpEF). The data were presented at the Heart Failure 2025 Congress in Belgrade, Serbia.

SRD-002 delivers the cardiac isoform of the sarcoplasmic reticulum calcium ATPase pump (SERCA2a) via a minimally invasive intracoronary infusion to target the underlying molecular pathways of HFpEF by enhancing myocardial relaxation and reducing stiffness. The trial includes HFpEF patients with exercise-induced diastolic dysfunction and symptomatic HFpEF.

Interim data from six patients (five at a low dose and one at a higher dose) showed no gene therapy-related serious adverse events, with follow-up ranging from 4 to 16 months. Four of five patients in the low-dose group demonstrated improvements in NYHA heart failure classification at 6 months, along with clinically meaningful improvements in the 6-minute walk test and decreases/stabilization in NT-Pro-BNP and high-sensitivity troponin in some patients. Enrollment at the higher dose is ongoing.

Dr. Marat Fudim of Duke University Medical Center highlighted the significant unmet need in HFpEF and noted that these early results suggest SRD-002, an AAV-based therapy, may offer a transformative approach by directly addressing the pathophysiology of impaired myocardial relaxation.

Medera CEO Dr. Ronald Li expressed pleasure with the safety profile and early clinical signals of this first-in-human AAV gene therapy, noting the optimized dosages based on their bioengineered human mini-heart HFpEF models. He emphasized this as an important step in developing innovative therapies for cardiovascular diseases with limited treatment options.

The Heart Failure Congress is a leading global event covering the spectrum of heart failure. Medera’s MUSIC-HFpEF trial (NCT06061549) is ongoing. Medera also announced a merger agreement with Keen Vision Acquisition Corporation in September 2024. HFpEF is a prevalent and growing condition with limited effective treatments beyond symptom management, underscoring the need for disease-modifying therapies like Medera’s AAV gene therapy.