Medera Announces Completion of Cohort A and Initiation of Dosing in Cohort B of MUSIC-HFpEF

• Heart failure is a global pandemic with an estimated 64.3 million cases worldwide, costing over US$100B per year
• Heart failure with preserved ejection fraction (HFpEF) accounts for nearly half of all heart failure cases, but has limited disease-modifying therapeutics
• Promising efficacy results in cardiovascular performance observed in Cohort A (low-dose SRD-002) at six months with additional data being collected
• Initiated dosing patients in Cohort B (high-dose SRD-002) following recommendation of the DSMB

SUMMIT, N.J. and BOSTON, Oct. 24, 2024 /PRNewswire/ -- Medera Inc. today announced that its clinical-stage gene therapy subsidiary, Sardocor Corp., has successfully completed its Cohort A (low-dose). Following the recommendation of the Data Safety Monitoring Board (DSMB), dosing has begun for patients in Cohort B (high-dose). This Phase 1/2a clinical trial evaluates the first-in-human cardiac gene therapy candidate SRD-002 for the treatment of Heart Failure with Preserved Ejection Fraction (HFpEF).

"New treatment approaches, like Medera's gene therapy product, are critical for patients and caregivers faced with this devastating disease," stated Marat Fudim, MD, MHS, Advanced Heart Failure Specialist and Associate Professor at Duke University Medical Centre and site principal investigator. "We look forward to continuing this critical research in the second cohort."

"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," said Roger Hajjar, MD, President and co-founder of Medera. "SRD-002 has been well tolerated with no unexpected events or toxicities observed and promising efficacy results observed in some patients in Cohort A at the six-month mark, with additional data being collected and analysed. We expect to complete enrolment in the second cohort in the first quarter 2025, with an interim data readout anticipated in the first half of 2025."

MUSIC-HFpEF is an open-label, dose-escalation, 52-week clinical trial designed to evaluate the safety and tolerability, as well as preliminary efficacy, of SRD-002 in patients diagnosed with HFpEF. SRD-002, a gene therapy candidate utilizing an adeno-associated virus (AAV) vector carrying the cardiac isoform of the sarcoplasmic reticulum calcium ATPase pump (SERCA2a), is administered via Sardocor's proprietary one-time intracoronary infusion methodology. The role of SERCA2a, in relaxation has been demonstrated by Novoheart (a subsidiary of Medera which develops cardiac tissue engineering technology for disease modelling and drug discovery) in their first-of-a-kind bioengineered human mini-Heart HFpEF models, co-developed with AstraZeneca as previously announced. Delivery of SRD-002 in the human mini-Heart HFpEF models leads to improved relaxation defects as published. Human mini-Heart models have also been used to optimise dosages. These data were used to support our Fast Track Designation (FTD) for MUSIC-HFpEF obtained from the FDA, in accordance to the Modernization Act 2.0.

In Cohort A, five patients were infused with SRD-002 at the low dose of 3x10^13 vg per patient. Following the DSMB's recommendation to proceed, investigators are currently enrolling the next five patients in Cohort B, evaluating SRD-002 at the high dose of 4.5x10^13 vg per patient.

For additional information about the MUSIC-HEpEF trial, visit ClinicalTrials.gov using the study identifier NCT06061549.

On September 5, 2024, Medera and Keen Vision Acquisition Corporation ("KVAC") (Nasdaq: KVAC, KVACW) announced they had entered into a definitive merger agreement.