Vandria SA has announced encouraging topline results from its first-in-human clinical trial of its lead Central Nervous System compound VNA-318.
The findings were presented at the 18th Clinical Trials in Alzheimer’s Disease meeting in San Diego, held from 1–4 December. VNA-318 is described as a first-in-class, orally bioavailable and brain-penetrant small molecule with a dual mode of action.
It is initially being developed to address cognitive impairment and loss of function in patients with Alzheimer’s disease, though Vandria notes its potential to treat other CNS conditions.
The phase 1 trial, VNA-318-01, was a randomized, double-blind study involving 92 healthy male subjects. Interim results showed the drug was well tolerated across all dose levels, with no severe or serious adverse events and no discontinuations.
Pharmacokinetic data demonstrated a long half-life supportive of once-daily dosing and a predictable, dose-linear increase of exposure. Importantly, single doses produced a statistically significant and dose-dependent change in a key plasma target engagement biomarker.
Klaus Dugi M.D., CEO of Vandria, said: “We are very excited about the results of our first-in-human trial of VNA-318, which ticks all the boxes for a Phase 1 trial – and more. The statistically significant dose-dependent change in a key target engagement biomarker is a very important finding and will be valuable for our Phase 2 clinical development strategy.”
He added: “This, coupled with safety, tolerability and demonstrated brain penetration, as well as pre-clinical data strongly support VNA-318’s advancement in Alzheimer’s disease. We believe that VNA-318 has the potential to address unmet medical needs like mild cognitive impairment associated with Alzheimer’s and Major Depressive Disorder, as well as other CNS disorders.”
Steven Arnold M.D., Professor of Neurology at Harvard Medical School, explained: “VNA-318 modulates a novel target with genetic associations with Alzheimer’s disease and related neurodegenerative diseases.
It is very exciting to see the compelling data from Vandria’s pre-clinical and clinical studies, and the progress VNA-318 is making as it gets closer to being tested in patients.”
Vandria plans to raise a Series B in 2026 to fund phase 2 proof-of-concept trials and expand its pipeline into non-CNS indications such as muscle, lung and liver diseases.
