BOSTON, Jan. 05, 2026 (GLOBE NEWSWIRE) -- Medera Inc. ("Medera"), a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing next-generation therapeutics, today announced that the final patient has been dosed in Cohort B (high-dose cohort, 4.50×10¹³ viral genomes (vg) per patient) of the ongoing MUSIC-HFpEF Phase 1/2a clinical trial evaluating SRD-002 gene therapy for heart failure with preserved ejection fraction (HFpEF). The patient was successfully treated using Medera's proprietary minimally invasive intracoronary infusion methodology and tolerated the procedure well. Completion of enrollment in Cohort B underscores the clinical feasibility of targeted intracoronary cardiac gene therapy in complex HFpEF with substantial unmet medical need.
Heart failure is a global pandemic with an estimated 64.3 million cases worldwide and a rising prevalence trend. HFpEF accounts for nearly half of all heart failure cases but has limited disease-modifying therapeutics. The MUSIC-HFpEF trial is investigating SRD-002, a one-time gene therapy treatment for HFpEF delivered through a proprietary minimally invasive intracoronary infusion methodology. SRD-002 utilizes an adeno-associated type 1 virus vector carrying the cardiac isoform of the sarcoplasmic reticulum calcium ATPase pump (SERCA2a) to directly target core molecular pathways driving HFpEF, including impaired calcium handling, myocardial stiffness, and diastolic dysfunction.
"The completion of enrollment in Cohort B represents an important milestone in our clinical development program," said Ronald Li, PhD, CEO and Founder of Medera. "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 received substantially higher doses than in prior studies (1011–1013 vg per patient), while remaining well below doses typically required for systemic intravenous (IV) infusion (~1015–1016 vg per patient), reflecting the efficiency of cardiac-targeted intracoronary delivery and reduced systemic exposure. The dosing strategy was rationally designed utilizing the minimally invasive intracoronary infusion methodology and optimized using Medera's proprietary human-based mini-Heart™ technology platform, which enabled rational dose selection and translational confidence through human-relevant HFpEF disease modeling. This HFpEF disease model, co-developed with AstraZeneca, has contributed to a US Food and Drug Administration (FDA) Investigational New Drug (IND) clearance and Fast Track Designation.
The MUSIC-HFpEF trial continues to demonstrate a favorable safety profile. As of the most recent interim data cutoff, no gene therapy-related serious adverse events have been reported across both cohorts. Patients in Cohort A (low-dose cohort, 3.0×10¹³ vg per patient) have completed 12-month follow-up and have shown improvements in New York Heart Association (NYHA) heart failure classification and Kansas City Cardiomyopathy Questionnaire at both 6 and 12 months, with clinically meaningful stabilizations and improvements in pulmonary capillary wedge pressure (PCWP) at rest and peak exercise, assessed through direct invasive hemodynamic measurements known to align with HFpEF severity. These safety and efficacy data were presented recently at the American Heart Association Scientific Sessions 2025 as a Late-Breaking Clinical Trial.
Cohort B patients are currently in early follow-up, with safety and efficacy data continuing to be collected and evaluated in accordance with the study protocol.
"Completing enrollment in Cohort B is a testament to the dedication of our clinical investigators and the commitment of patients participating in this groundbreaking trial," said Marat Fudim, MD, MHS, Advanced Heart Failure Specialist and Associate Professor at Duke University Medical Center. "This milestone positions us well to gather the additional safety and efficacy data needed to support progression toward Phase 2b and potential disease-modification studies."
The trial will continue to follow patients for 24 months post-treatment. Long-term safety and efficacy data are expected to inform the next phase of clinical development.
For additional information about the MUSIC-HFpEF trial, visit ClinicalTrials.gov using the study identifier NCT06061549.
About Heart Failure with Preserved Ejection Fraction (HFpEF)
Heart failure (HF) is a global pandemic with an estimated 64.3 million cases worldwide and a rising prevalence trend. Accounting for 50% or more of the overall HF population, HFpEF is an age-related condition that has become increasingly prevalent in recent years. This surge is partly due to better awareness and identification of the condition and partly due to lifestyle changes affecting cardiac myocytes. Individuals affected by HFpEF experience similar morbidity and mortality to patients with HF with reduced ejection fraction (HFrEF). Despite the growing epidemic of this emerging syndrome, HFpEF-focused interventional trials have had little success, with currently approved therapies largely focused on symptom management and risk reduction rather than targeting the underlying myocardial biology driving HFpEF. However, these agents are not disease-modifying, highlighting the critical need for therapeutic interventions targeting the physiological mechanisms involved in HFpEF.
About Medera Inc.
Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases by developing a range of next-generation therapeutics. Medera operates via its two preclinical and clinical business units, Novoheart and Sardocor, respectively.
Novoheart capitalizes on the world’s first and award-winning “mini-Heart” Technology for revolutionary disease modelling and drug discovery, uniquely enabling the modelling of human-specific diseases and discovery of therapeutic candidates free from species-specific differences in accordance to the FDA Modernization Act 2.0. Novoheart's versatile technology platform provides a range of state-of-the-art automation hardware and software as well as screening services, for human-specific disease modelling, therapeutic target discovery and validation, drug toxicity and efficacy screening, and dosage optimization carried out in the context of healthy and/or diseased human heart chambers and tissues. Global pharmaceutical and academic leaders are using Novoheart's technology platform for their drug discovery and development purposes. The Novoheart platform has facilitated and accelerated the development of Sardocor's lead therapeutic candidates that are currently in clinical trials.
Sardocor is dedicated to the clinical development of novel next-generation therapies for Medera. Leveraging Novoheart’s human-based drug discovery and validation platforms, Sardocor aims to expedite drug development and regulatory timelines for its gene and cell therapy pipeline. Sardocor has received Investigational New Drug (IND) clearances from the FDA for three ongoing AAV-based cardiac gene therapy clinical trials targeting Heart Failure with Reduced Ejection Fraction (HFrEF), Heart Failure with Preserved Ejection Fraction (HFpEF) with the Fast Track Designation, and Duchenne Muscular Dystrophy-associated Cardiomyopathy (DMD-CM) with the Orphan Drug Designation. Additionally, Sardocor's pipeline includes four preclinical gene therapy and three preclinical small molecule candidates targeting various cardiac, pulmonary, and vascular diseases.
For more information, please visit www.medera.bio.
Contacts
Ally Stubin
Public Relations
ICR Healthcare
Ally.stubin@icrhealthcare.com
646.667.1861
Stephanie Carrington
Investor Relations
ICR Healthcare
Stephanie.carrington@icrhealthcare.com
646.277.1282
