Vandria SA, a company focused on mitochondrial therapeutics, has announced the dosing of the first subjects in a Phase 1 clinical trial of its lead compound, VNA-318. This first-in-human trial marks a significant step in the development of a novel therapeutic approach for central nervous system (CNS) and neurodegenerative disorders.
The Phase 1 trial is a randomized, double-blind, single and multiple ascending dose study designed to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic parameters of VNA-318 in healthy male subjects. The company expects to report initial results from the trial in the summer of 2025.
VNA-318: A Novel Mitophagy Inducer
VNA-318 is an orally available, first-in-class small molecule that induces mitophagy, a process by which cells remove damaged mitochondria. The drug targets a novel mechanism with strong genetic links to human diseases, including Alzheimer's disease. Preclinical studies have demonstrated that VNA-318 has a dual mode of action, providing both immediate improvement in memory, learning, and cognitive function, as well as long-term disease-modifying effects such as reduced neuroinflammation, less toxic protein aggregation, and improved mitochondrial function. These effects have been observed in preclinical models of Alzheimer's and Parkinson's disease. Toxicity studies have also indicated a wide safety window for VNA-318.
Clinical and Market Potential
Vandria is initially developing VNA-318 for patients with early-stage CNS and neurodegenerative disorders, with the goal of improving cognition and limiting memory loss progression. Klaus Dugi M.D., CEO of Vandria, stated that VNA-318 has the potential to address unmet medical needs in conditions such as mild cognitive impairment associated with Alzheimer's disease and major depressive disorder. Professor Li-Huei Tsai from MIT, a member of Vandria's Scientific Advisory Board, expressed excitement about the clinical development of VNA-318, citing the breadth and depth of preclinical data demonstrating consistent, dose-dependent efficacy in several models of neurodegenerative disease.
The global market for Alzheimer's disease is estimated at $6 billion and is projected to grow at a CAGR of 12% to 2035, driven by an aging population, improved diagnosis, and increased awareness of the condition.
Funding and Future Plans
Vandria has secured $32 million in Series A funding to support the Phase 1 trial, its analysis, and the planning of efficacy-focused Phase 1b/2a trials of VNA-318, with the first expected to commence in Q1 2026 or earlier. The company plans to raise a Series B round in 2025 to fund the planned Phase 2 trials and to advance other pipeline compounds in non-CNS indications such as muscle, lung, and liver diseases.
