Medera Announces Independent Data Monitoring Committee (DMC) Recommends Continued Advancement of Lead HFpEF Gene Therapy Program

Independent DMC recommends continued conduct of the MUSIC-HFpEF study without protocol modification following scheduled review of accumulated clinical data; randomized HFrEF Phase 2 study also reviewed with no safety concerns requiring changes to study conduct

BOSTON, June 25, 2026 (GLOBE NEWSWIRE) -- Medera Inc. (“Medera”), a clinical-stage biotechnology company developing disease-modifying gene therapies for cardiovascular disease, today announced that the independent Data Monitoring Committee (DMC) has completed its scheduled review of the Company's ongoing MUSIC-HFpEF clinical trial evaluating SRD-002, Medera's investigational AAV1/SERCA2a gene therapy for heart failure with preserved ejection fraction (HFpEF).

Following review of accumulated clinical data, the independent DMC recommended that the MUSIC-HFpEF study continue without protocol modification. The committee identified no safety concerns requiring changes to study conduct, supporting continued advancement of Medera's lead clinical program as the Company prepares for discussions with the U.S. Food and Drug Administration (FDA) regarding the next randomized stage of development.

The DMC recommendation followed review of accumulated safety and clinical data, including long-term follow-up from both dose cohorts.

“We are very encouraged by the outcome of this independent DMC review,” said Ronald Li, PhD, Chief Executive Officer and Founder of Medera. “Independent review by the DMC represents an important milestone for our lead HFpEF program. The review reflected the safety data collected to date and consistency in the physiological and clinical observations across hemodynamic and functional measures. We are particularly encouraged that an independent committee of experienced heart failure specialists and clinical trial experts recommended continued conduct of the study without modification. We believe this outcome further supports the favorable safety profile observed to date and provides additional confidence as we prepare for the next stage of randomized study.”

SRD-002 is Medera's investigational AAV1/SERCA2a gene therapy designed to restore SERCA2a expression and normalize impaired calcium handling within cardiomyocytes, a fundamental biological abnormality underlying HFpEF. Unlike currently approved therapies that primarily improve symptoms or reduce cardiovascular risk, SRD-002 is being developed as a potential disease-modifying therapy targeting the underlying myocardial pathobiology responsible for impaired cardiac relaxation and elevated cardiac filling pressures.

The program utilizes Medera's proprietary targeted intracoronary delivery platform, enabling localized cardiac gene transfer through a minimally invasive catheter-based outpatient procedure while minimizing systemic vector exposure and substantially reducing vector dose requirements compared with conventional systemic intravenous administration.

As of the latest DMC review, all ten patients have been successfully treated across two dose cohorts (Cohort A: 3.0 × 10¹³ vg; Cohort B: 4.5 × 10¹³ vg). All patients in Cohort A have completed 12-month follow-up, while patients in Cohort B continue scheduled long-term follow-up.

The DMC reviewed comprehensive safety information, including adverse events, laboratory assessments, hepatic function, immune monitoring, and cardiovascular safety data. No gene therapy-related serious adverse events have been reported across the HFpEF clinical program to date.

As previously reported at the American Heart Association Scientific Sessions 2025 as a Late-Breaking Clinical Trial Presentation, our Cohort A patients have shown improvements in New York Heart Association (NYHA) functional class, Kansas City Cardiomyopathy Questionnaire (KCCQ) scores, and hemodynamic parameters, including pulmonary capillary wedge pressure (PCWP), a direct measure of cardiac filling pressure and a central physiological hallmark of HFpEF, at rest and during exercise. The company expects to report 12-month data from Cohort B patients in the last quarter of 2026.

“HFpEF remains one of the largest unmet needs in cardiovascular medicine and one of the most challenging diseases to study because of its marked biological and clinical heterogeneity,” added Dr. Li. “We believe objective physiological measurements provide a rigorous framework for assessing therapeutic effects in this setting. This independent DMC recommendation represents an important clinical and regulatory de-risking milestone as we continue preparing for randomized development and future FDA interactions.”

The independent DMC also completed its scheduled review of Medera’s MUSIC-HFrEF Phase 1/2a clinical trial data, with no safety concerns identified. The randomized MUSIC-HFrEF Phase 2b trial is currently ongoing.

“The independent DMC reviews across both our HFpEF and randomized HFrEF programs provide encouraging support for the safety profile observed to date with our cardiovascular gene therapy platform,” said Roger J. Hajjar, MD, President and Chief Medical Officer of Medera. “We remain focused on completing long-term follow-up, generating robust clinical evidence, and advancing both programs through their next stages of clinical and regulatory development.”

Medera expects to provide additional updates regarding its HFpEF and HFrEF clinical programs, including future data presentations, regulatory interactions, and development milestones, as they become available.

About Heart Failure with Preserved Ejection Fraction (HFpEF)

Heart failure affects more than 64 million people worldwide, with HFpEF accounting for approximately half of all heart failure cases. Patients with HFpEF experience morbidity and mortality comparable to those with heart failure with reduced ejection fraction (HFrEF). Despite its growing prevalence, currently approved therapies primarily improve symptoms and reduce cardiovascular risk rather than directly targeting the underlying myocardial abnormalities responsible for the disease. Consequently, HFpEF remains one of the largest unmet needs in cardiovascular medicine and an area of active therapeutic development.

About Medera Inc.

Medera is a clinical-stage biotechnology company developing next-generation gene therapies for cardiovascular and other serious diseases. Its pipeline includes multiple AAV-based gene therapy programs targeting heart failure and related cardiovascular disorders.

The Company operates through two complementary business units: Novoheart, which provides advanced human-specific cardiac disease modeling and drug discovery platforms, and Sardocor, which leads clinical development of Medera's gene therapy pipeline. Together, these capabilities create an integrated translational platform spanning discovery, preclinical validation, manufacturing, and clinical development.

For more information, please visit www.medera.bio.

Forward-Looking Statements

This press release contains forward-looking statements regarding Medera's investigational products, anticipated regulatory interactions, clinical development plans, future clinical data, and potential therapeutic benefits. These statements are based on current expectations and involve risks and uncertainties that may cause actual results to differ materially from those expressed or implied.

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

Legal Disclaimer:

EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.