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Vandria Begins Clinical Trials for Promising Mitophagy Inducer

Published on: December 18, 2024 | Last updated: December 18, 2024

A Swiss-based leader in mitochondrial therapeutics, Vandria, has initiated its first-in-human clinical trial for VNA-318, a novel small molecule designed to induce mitophagy. The trial represents a significant step forward in addressing neurodegenerative and potentially other age-related diseases through mitochondrial health.

Longevity.Technology: Mitophagy is the process of selectively removing damaged or dysfunctional mitochondria from cells to maintain cellular health and energy production. Mitochondrial dysfunction is a hallmark of aging and is implicated in various degenerative diseases, including Alzheimer’s and Parkinson’s. By promoting mitophagy, VNA-318 aims to rejuvenate cells and restore normal function, offering a therapeutic avenue to combat the effects of aging and disease.

The randomized, double-blind Phase 1 trial is designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VNA-318 in healthy male participants. Vandria says early data from preclinical studies have shown VNA-318’s dual mode of action – immediate cognitive benefits and long-term disease modification. The compound improves memory and learning while reducing neuroinflammation, toxic protein aggregation, and mitochondrial dysfunction in models of Alzheimer’s and Parkinson’s disease; the trial readout is anticipated by the summer of 2025.

“VNA-318 has the potential to address unmet medical needs like mild cognitive impairment associated with Alzheimer’s disease, major depressive disorder, and other diseases of the CNS, and potentially diseases beyond the CNS.” – Dr. Klaus Dugi, CEO of Vandria

Broader Implications of Mitophagy Induction

VNA-318 is not solely designed for CNS disorders but may have broader implications for mitochondrial therapeutics. Vandria is exploring the application of mitophagy-inducing compounds in non-CNS conditions, such as muscle, liver, and lung diseases. The company’s strategy includes progressing these indications following Phase 2 trials for VNA-318.

Dr. Dugi highlighted that impaired mitophagy and mitochondrial dysfunction have been implicated in numerous diseases, and Vandria is investigating connections between its molecular targets and age-related diseases outside the central nervous system. The company has already generated preclinical efficacy data across various diseases.

Summary of Efficacy Data from Preclinical Studies

Preclinical Study Focus Key Findings
Neurodegenerative Diseases Consistent, dose-dependent efficacy reducing neuroinflammation and mitochondrial dysfunction.
Safety Profile Wide safety margin proven in various animal models.
Intellectual Benefits Notable improvements in memory and learning demonstrated in studies.

Diversity in Clinical Trials

While this Phase 1 trial focuses on healthy male subjects, future trials will aim to diversify the participant pool to ensure the compound’s efficacy and safety across broader demographics. Such inclusivity is critical for determining VNA-318’s applicability in various stages of neurodegenerative diseases and in patient groups that may metabolize or respond differently to the therapy.

“Subject to positive data from this short Phase I trial, we are planning for two to three parallel Phase 2a studies in patient populations that exhibit cognitive impairment,” Dugi mentioned. “The underlying disease areas could be Alzheimer’s Disease, major depressive disorder, or others. In these studies, it is planned to include a wider demographic to ensure broader applicability.”

Market Potential and Next Steps

The global market for Alzheimer’s therapies, estimated at $6 billion in 2024, is projected to grow at a compound annual growth rate (CAGR) of 12% by 2035, driven by aging populations and enhanced diagnostics. Vandria’s innovative approach to mitochondrial health could position it to capture a significant share of this growing market.

With $32 million raised in Series A funding, Vandria plans to advance into efficacy-focused Phase 1b/2a trials in early 2026. This, combined with the company’s aim to expand its pipeline to include compounds targeting non-CNS indications, strengthens its position as a leader in mitochondrial health innovation. The biotech community will be closely monitoring the trial outcomes to assess the broader impact of mitophagy-based therapeutics.

Conclusion

The initiation of Vandria's clinical trial for VNA-318 marks a pivotal development in the field of mitochondrial therapeutics, signaling a potential shift in how age-related diseases may be tackled in the future. As research unfolds, the efficacy of this approach will become clearer, promising a hopeful avenue for numerous patients suffering from debilitating conditions linked to aging and mitochondrial dysfunction.

References: Lifespan.io