- Results from MUSIC-HFpEF Phase 1/2a clinical trial show favorable safety profile and early clinical benefits with SRD-002 gene therapy
BOSTON, MA, USA I May 19, 2025 I Medera Inc. (“Medera”), a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing a range of next-generation therapeutics, today announced that positive interim data from its ongoing MUSIC-HFpEF Phase 1/2a clinical trial was presented at the Heart Failure 2025 Congress taking place in Belgrade, Serbia. The late-breaking presentation, titled “Gene therapy for heart failure with preserved ejection fraction,” was delivered by Marat Fudim, MD, MHS, Advanced Heart Failure Specialist and Associate Professor at Duke University Medical Center, during the “Late-Breaking Clinical Trials in Chronic Heart Failure” session.
The MUSIC-HFpEF trial is investigating SRD-002, a one-time gene therapy treatment for heart failure with preserved ejection fraction (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 the molecular pathways underlying the core pathology of HFpEF by enhancing myocardial relaxation and reducing stiffness. The trial is evaluating the therapy in HFpEF patients with exercise-induced diastolic dysfunction, including those with LVEF ≥ 50% and symptomatic HFpEF defined by prior hospitalization or New York Heart Association (NYHA) Class II or III symptoms.
The presentation highlighted interim data from the ongoing trial, which, as of the data cutoff date, has treated five patients in Cohort A with a low dose of 3×1013 viral genomes (vg) per patient and one patient in Cohort B at a dose of 4.5×1013 vg per patient. With follow-up ranging from 4 to 16 months, no gene therapy-related serious adverse events have been reported. Four out of five patients in the low-dose group have shown improvements in NYHA heart failure classification at 6 months, with clinically meaningful improvements in 6-minute walk test (6MWT), decreases/stabilization in NT-Pro-BNP, and high-sensitivity troponin observed in some patients. The enrollment of patients at the higher dose of 4.5×1013 vg per patient is ongoing.
“HFpEF represents a significant unmet medical need, affecting approximately half of all heart failure patients worldwide with limited disease-modifying therapeutic options,” said Marat Fudim, MD, MHS. “These early results are encouraging and suggest that SRD-002 may offer a potentially transformative approach for patients with HFpEF by directly addressing the underlying pathophysiology of impaired myocardial relaxation.”
“With our therapeutic dosages optimized by our bioengineered human mini-heart HFpEF models – approximately 100-fold less than that of IV infusion – we are pleased with the safety profile and early clinical signals observed in the MUSIC-HFpEF trial to date,” said Ronald Li, Ph.D., CEO and co-founder of Medera. “This first-in-human gene therapy approach represents an important step forward in our mission to develop innovative therapies for cardiovascular diseases with limited treatment options.”
The Heart Failure Congress is the world’s leading event covering the entire spectrum of heart failure, from prevention to diagnosis and treatment. It is the annual meeting of the Heart Failure Association of the European Society of Cardiology.
For additional information about the MUSIC-HFpEF 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.
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, except for the use of sacubitril-valsartan (Entresto™) and the sodium glucose transporter-2 (SGLT-2) inhibitor empagliflozin (Jardiance™) for reducing cardiovascular mortality and heart failure hospitalization. However, these agents are not disease-modifying, highlighting the critical need for therapeutic interventions targeting the physiological mechanisms involved in HFpEF.
About Medera
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.
About Keen Vision Acquisition Corporation
Keen Vision Acquisition Corp (“KVAC”), listed on Nasdaq, is a blank check company incorporated for the purpose of effecting a merger, share exchange, asset acquisition, share purchase, reorganization or similar business combination with one or more businesses or entities. KVAC is focused on biotechnology, consumer goods or agriculture opportunities, which are also evaluated on their sustainability, environmental, social, and corporate governance (“ESG”) imperatives.
For more information, please visit www.kv-ac.com.
SOURCE: Medera
Post Views: 2,526
- Results from MUSIC-HFpEF Phase 1/2a clinical trial show favorable safety profile and early clinical benefits with SRD-002 gene therapy
BOSTON, MA, USA I May 19, 2025 I Medera Inc. (“Medera”), a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing a range of next-generation therapeutics, today announced that positive interim data from its ongoing MUSIC-HFpEF Phase 1/2a clinical trial was presented at the Heart Failure 2025 Congress taking place in Belgrade, Serbia. The late-breaking presentation, titled “Gene therapy for heart failure with preserved ejection fraction,” was delivered by Marat Fudim, MD, MHS, Advanced Heart Failure Specialist and Associate Professor at Duke University Medical Center, during the “Late-Breaking Clinical Trials in Chronic Heart Failure” session.
The MUSIC-HFpEF trial is investigating SRD-002, a one-time gene therapy treatment for heart failure with preserved ejection fraction (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 the molecular pathways underlying the core pathology of HFpEF by enhancing myocardial relaxation and reducing stiffness. The trial is evaluating the therapy in HFpEF patients with exercise-induced diastolic dysfunction, including those with LVEF ≥ 50% and symptomatic HFpEF defined by prior hospitalization or New York Heart Association (NYHA) Class II or III symptoms.
The presentation highlighted interim data from the ongoing trial, which, as of the data cutoff date, has treated five patients in Cohort A with a low dose of 3×1013 viral genomes (vg) per patient and one patient in Cohort B at a dose of 4.5×1013 vg per patient. With follow-up ranging from 4 to 16 months, no gene therapy-related serious adverse events have been reported. Four out of five patients in the low-dose group have shown improvements in NYHA heart failure classification at 6 months, with clinically meaningful improvements in 6-minute walk test (6MWT), decreases/stabilization in NT-Pro-BNP, and high-sensitivity troponin observed in some patients. The enrollment of patients at the higher dose of 4.5×1013 vg per patient is ongoing.
“HFpEF represents a significant unmet medical need, affecting approximately half of all heart failure patients worldwide with limited disease-modifying therapeutic options,” said Marat Fudim, MD, MHS. “These early results are encouraging and suggest that SRD-002 may offer a potentially transformative approach for patients with HFpEF by directly addressing the underlying pathophysiology of impaired myocardial relaxation.”
“With our therapeutic dosages optimized by our bioengineered human mini-heart HFpEF models – approximately 100-fold less than that of IV infusion – we are pleased with the safety profile and early clinical signals observed in the MUSIC-HFpEF trial to date,” said Ronald Li, Ph.D., CEO and co-founder of Medera. “This first-in-human gene therapy approach represents an important step forward in our mission to develop innovative therapies for cardiovascular diseases with limited treatment options.”
The Heart Failure Congress is the world’s leading event covering the entire spectrum of heart failure, from prevention to diagnosis and treatment. It is the annual meeting of the Heart Failure Association of the European Society of Cardiology.
For additional information about the MUSIC-HFpEF 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.
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, except for the use of sacubitril-valsartan (Entresto™) and the sodium glucose transporter-2 (SGLT-2) inhibitor empagliflozin (Jardiance™) for reducing cardiovascular mortality and heart failure hospitalization. However, these agents are not disease-modifying, highlighting the critical need for therapeutic interventions targeting the physiological mechanisms involved in HFpEF.
About Medera
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.
About Keen Vision Acquisition Corporation
Keen Vision Acquisition Corp (“KVAC”), listed on Nasdaq, is a blank check company incorporated for the purpose of effecting a merger, share exchange, asset acquisition, share purchase, reorganization or similar business combination with one or more businesses or entities. KVAC is focused on biotechnology, consumer goods or agriculture opportunities, which are also evaluated on their sustainability, environmental, social, and corporate governance (“ESG”) imperatives.
For more information, please visit www.kv-ac.com.
SOURCE: Medera
Post Views: 2,526
