Nuevocor Announces FDA Clearance of IND for NVC-001 for LMNA-Related Dilated Cardiomyopathy

• LMNA-related dilated cardiomyopathy (LMNA DCM) is one of the most aggressive forms of DCM, affecting approximately 100,000 individuals in the United States and Europe, who progress rapidly to end-stage heart failure.
• NVC-001 demonstrated significant benefits, including survival and cardiac function in preclinical models.
• IND clearance enables initiation of first-in-human, ascending-dose Phase 1/2 clinical trial in early 2026.

SINGAPORE and PHILADELPHIA, June 10, 2025 /PRNewswire/ -- Nuevocor, a Singapore-headquartered biotechnology company developing cures for cardiomyopathies driven by aberrant mechanobiology, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for NVC-001. NVC-001 is an adeno-associated virus (AAV)-based gene therapy designed to treat LMNA-related dilated cardiomyopathy (LMNA DCM). Nuevocor plans to initiate an open-label, ascending-dose Phase 1/2 clinical trial of NVC-001 in patients with LMNA DCM.

"This IND clearance marks a significant milestone in our mission to develop transformative therapies for patients with genetic cardiomyopathies by leveraging unique insights from our proprietary PrOSIA mechanobiology platform," said Dr. Yann Chong Tan, co-founder and CEO of Nuevocor. "NVC-001 is the first disease-modifying therapy for LMNA DCM designed to address the underlying mechanobiological root cause of disease. We look forward to initiating our clinical trial to bring this potentially life-changing therapy to patients."

LMNA DCM is a serious genetic heart condition caused by mutations in the LMNA gene, which encodes lamin A/C – a protein essential for maintaining nuclear envelope integrity and regulating gene expression in cardiac cells. Mutations in LMNA lead to a weakening of the heart muscle, arrhythmias, and progression to end-stage heart failure. LMNA DCM is estimated to affect approximately 100,000 patients in the U.S. and Europe, representing a significant unmet medical need.

NVC-001 is engineered to address the functional mechanical root cause of disease by reducing aberrant mechanical stress on the nucleus, thereby restoring nuclear envelope integrity. Nuclear envelope disruption is a hallmark of LMNA DCM. In preclinical studies, treatment with NVC-001 demonstrated significant benefits, including survival and cardiac function.

The planned Phase 1/2 clinical trial is a first-in-human, 52-week open-label, multicenter, ascending-dose study designed to evaluate the safety, tolerability, and preliminary efficacy of NVC-001 in adult subjects with LMNA-DCM. NVC-001 will be administered as a one-time intravenous infusion to patients in ascending-dose cohorts.

About Nuevocor

Nuevocor is biotechnology company developing novel therapies for genetic cardiomyopathies driven by aberrant mechanobiology, headquartered in Singapore with an office in the U.S. and expansion to Europe. Our unique approach, enabled by our proprietary PrOSIA mechanobiology platform, surpasses the limitations of traditional gene replacement therapy – which treats individual gene mutations – to treating defects in shared disease pathways across multiple cardiomyopathies by addressing the functional root cause of disease. Nuevocor is first-in-disease by addressing genetic cardiomyopathies that are not amendable to gene replacement therapy and have no effective treatment options. ()