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

Nuevocor, a biotechnology company based in Singapore, specializing in developing treatments for genetic heart diseases, has announced a significant development in its efforts to tackle one of the more aggressive forms of dilated cardiomyopathy, known as LMNA-related dilated cardiomyopathy (LMNA DCM). This condition affects roughly 100,000 individuals in the United States and Europe, leading them rapidly towards severe heart failure. The company's recent advancements have been recognized by the U.S. Food and Drug Administration (FDA), which has approved their Investigational New Drug (IND) application for their novel therapy, NVC-001.

NVC-001 represents an innovative approach to treating LMNA DCM, using an adeno-associated virus (AAV)-based gene therapy. The therapy aims to mitigate the disease's effects by addressing the core mechanical issues causing the condition. This development is a part of Nuevocor's broader mission to devise transformative solutions for genetic heart conditions using insights derived from their proprietary PrOSIA mechanobiology platform.

The CEO and co-founder of Nuevocor, Dr. Yann Chong Tan, emphasized the importance of this IND clearance. It marks a pivotal step towards bringing NVC-001, which is the first therapy designed to modify the course of LMNA DCM by targeting its mechanobiological roots, into clinical trials. Dr. Tan expressed optimism about the potential impact of this therapy, which could significantly alter the treatment landscape for patients afflicted by this challenging condition.

LMNA DCM is caused by mutations in the LMNA gene, which is crucial for producing lamin A/C. This protein is vital for maintaining the nuclear envelope's integrity and regulating gene expression within cardiac cells. Mutations lead to the weakening of the heart muscle, irregular heartbeats, and eventually escalating to severe heart failure. The condition represents a substantial unmet medical need, due to the lack of effective existing treatments.

The therapeutic approach of NVC-001 involves correcting the mechanical stress experienced by the nucleus, thereby restoring the integrity of the nuclear envelope, which is typically compromised in LMNA DCM. Preclinical studies have shown that NVC-001 can offer significant benefits, improving both survival rates and cardiac function in models.

Following the IND clearance, Nuevocor is set to launch its first human clinical trial in early 2026. This Phase 1/2 trial will be an open-label, multicenter study over a 52-week period, designed to assess the safety, tolerability, and early efficacy of NVC-001. The study will involve adult participants diagnosed with LMNA DCM, who will receive the therapy as a one-time intravenous infusion across different dosage levels.

Nuevocor is committed to pioneering therapies for genetic cardiomyopathies that are tough to tackle with traditional gene replacement techniques. By focusing on the shared pathways of disease across various cardiomyopathies, their innovative platform aims to address the foundational causes of these conditions. With its headquarters in Singapore and facilities in the U.S., Nuevocor is expanding its reach to Europe, addressing genetic heart diseases for which there are currently no viable treatments.

This development represents a promising step forward in the fight against LMNA DCM and other similar genetic heart disorders, potentially offering new hope for patients around the globe facing these challenging conditions.