Medera Announces Completion of Cohort A and Initiation of Dosing in Cohort B of MUSIC-HFpEF, a Phase 1/2a Clinical Trial Evaluating First-In-Human Gene Therapy SRD-002 for Heart Failure with Preserved Ejection Fraction
Medera announced completion of Cohort A and initiation of Cohort B dosing in its MUSIC-HFpEF Phase 1/2a clinical trial, evaluating SRD-002, a first-in-human gene therapy for Heart Failure with Preserved Ejection Fraction (HFpEF). The trial shows promising efficacy results at six months in Cohort A (low-dose 3x10^13 vg), with no unexpected events or toxicities. Following DSMB recommendation, Cohort B (high-dose 4.5x10^13 vg) enrollment has begun. The company expects to complete Cohort B enrollment in Q1 2025, with interim data expected in H1 2025. HFpEF affects nearly half of 64.3 million heart failure cases worldwide, with treatment options available.
Medera ha annunciato il completamento della Coorte A e l'inizio della somministrazione della Coorte B nel suo trial clinico MUSIC-HFpEF di Fase 1/2a, che valuta SRD-002, una terapia genica prima in umani per l'insufficienza cardiaca con frazione d'eiezione preservata (HFpEF). Il trial mostra risultati promettenti di efficacia a sei mesi nella Coorte A (low-dose 3x10^13 vg), senza eventi imprevisti o tossicità. A seguito della raccomandazione del DSMB, è iniziato il reclutamento della Coorte B (high-dose 4.5x10^13 vg). L'azienda prevede di completare il reclutamento della Coorte B nel primo trimestre del 2025, con dati intermedi attesi nel primo semestre del 2025. L'HFpEF colpisce quasi la metà dei 64,3 milioni di casi di insufficienza cardiaca nel mondo, con opzioni di trattamento disponibili.
Medera anunció la finalización del Cohorte A y el inicio de la dosificación del Cohorte B en su ensayo clínico MUSIC-HFpEF de Fase 1/2a, que evalúa SRD-002, una terapia génica de primera en humanos para la insuficiencia cardíaca con fracción de eyección preservada (HFpEF). El ensayo muestra resultados prometedores de eficacia a los seis meses en el Cohorte A (bajo dosis 3x10^13 vg), sin eventos o toxicidades inesperadas. Siguiendo la recomendación del DSMB, ha comenzado la inscripción del Cohorte B (alta dosis 4.5x10^13 vg). La compañía espera completar la inscripción del Cohorte B en el primer trimestre de 2025, con datos interinos esperados en el primer semestre de 2025. La HFpEF afecta a casi la mitad de los 64.3 millones de casos de insuficiencia cardíaca en todo el mundo, con opciones de tratamiento disponibles.
메데라는 SRD-002의 인체 임상 시험인 MUSIC-HFpEF 1/2a 단계 임상 시험에서 Cohort A의 완료와 Cohort B의 투여 시작을 발표했습니다. 이 치료는 이완 기능이 보존된 심부전(HFpEF)을 위한 최초의 유전자 치료입니다. 이 시험은 Cohort A(저용량 3x10^13 vg)에서 6개월 후 유망한 효능 결과를 보여주고 있으며, 예기치 않은 사건이나 독성이 없습니다. DSMB의 권고에 따라 Cohort B(고용량 4.5x10^13 vg) 모집이 시작되었습니다. 회사는 2025년 1분기 내에 Cohort B 모집을 완료할 것으로 예상하고 있으며, 2025년 상반기에 중간 데이터를 기대하고 있습니다. HFpEF는 전 세계 6,430만 심부전 사례의 거의 절반에 영향을 미치며, 치료 옵션이 제공됩니다.
Medera a annoncé l'achèvement de la Cohorte A et le début de l'administration de la Cohorte B dans son essai clinique de Phase 1/2a MUSIC-HFpEF, qui évalue SRD-002, une thérapie génique innovante pour l'insuffisance cardiaque avec fraction d'éjection préservée (HFpEF). L'essai montre des résultats d'efficacité prometteurs après six mois dans la Cohorte A (faible dose 3x10^13 vg), sans événements ni toxicités inattendues. Suite à la recommandation du DSMB, le recrutement de la Cohorte B (haute dose 4.5x10^13 vg) a commencé. L'entreprise s'attend à achever le recrutement de la Cohorte B au premier trimestre 2025, avec des données intermédiaires attendues au premier semestre 2025. L'HFpEF affecte près de la moitié des 64,3 millions de cas d'insuffisance cardiaque dans le monde, avec des options de traitement disponibles.
Medera hat den Abschluss der Kohorte A und den Beginn der Dosierung der Kohorte B in seiner klinischen Phase 1/2a-Studie MUSIC-HFpEF angekündigt, die SRD-002 bewertet, eine erstmals am Menschen getestete Gentherapie für Herzinsuffizienz mit erhaltener Ejektionsfraktion (HFpEF). Die Studie zeigt vielversprechende Wirksamkeitsergebnisse nach sechs Monaten in der Kohorte A (Niedrigdosis 3x10^13 vg), ohne unerwartete Ereignisse oder Toxizität. Nach der Empfehlung des DSMB hat die Rekrutierung für Kohorte B (Hochdosis 4.5x10^13 vg) begonnen. Das Unternehmen erwartet, die Rekrutierung von Kohorte B im ersten Quartal 2025 abzuschließen, mit vorläufigen Daten, die im ersten Halbjahr 2025 erwartet werden. HFpEF betrifft fast die Hälfte der 64,3 Millionen Fälle von Herzinsuffizienz weltweit, wobei Behandlungsoptionen verfügbar sind.
- Promising efficacy results observed in Cohort A at six months
- SRD-002 well tolerated with no unexpected events or toxicities
- DSMB approved progression to higher dose Cohort B
- FDA Fast Track Designation obtained for MUSIC-HFpEF trial
- None.
Insights
The clinical trial progress for SRD-002 represents a significant milestone in addressing the
Several key aspects make this particularly noteworthy:
- The novel gene therapy approach targeting SERCA2a for HFpEF, which currently lacks effective treatments despite representing nearly
50% of heart failure cases - Validation through human mini-Heart models developed with AstraZeneca, providing strong preclinical evidence
- FDA Fast Track Designation, which could accelerate the approval timeline
The anticipated interim data readout in H1 2025 could be a major catalyst for Medera as it approaches its merger with KVAC. The positive safety profile and early efficacy signals in Cohort A reduce development risk for this first-in-human therapy.
This clinical progress strengthens Medera's position ahead of its planned merger with KVAC. The addressable market is substantial, with 64.3 million global heart failure cases and treatment options for HFpEF patients. The successful completion of Cohort A with positive preliminary data significantly de-risks the development program.
Key value drivers include:
- Proprietary delivery system for gene therapy
- Strategic partnership with AstraZeneca for preclinical validation
- Fast Track Designation providing potential accelerated pathway to market
- Heart failure is a global pandemic with an estimated 64.3 million cases worldwide, costing over
US per year$100B - 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
"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.
About HFpEF
Heart failure (HF) is a global pandemic with an estimated 64.3 million cases worldwide and a rising prevalence trend. Accounting for
About Medera
Medera (www.medera.bio) is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat or currently incurable diseases with significant unmet needs, utilizing next-generation gene and cell-based approaches in combination with bioengineered human-based technology (including the mini-Heart platform). Medera operates via the 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.
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-induced 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.
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. EF Hutton LLC and Brookline Capital Markets, a division of Arcadia Securities, LLC, are serving as Capital Markets Advisors for KVAC.
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FAQ
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