Vandria has announced positive top line data from its first-in-human Phase I clinical trial evaluating VNA-318, a central nervous system (CNS) compound.
The trial is designed to evaluate the compound’s pharmacokinetic, safety, and tolerability profile in 92 healthy male participants, supporting further clinical development in Alzheimer’s disease.
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VNA-318 is an orally bioavailable, brain-penetrant small molecule with a dual mode of action.
It is initially being developed for Alzheimer’s, targeting cognitive decline and functional loss. The compound’s mechanism of action is also expected to address other CNS disorders.
The VNA-318 target exhibits genetic associations with several human diseases, including Alzheimer’s. In pre-clinical models, the compound demonstrated immediate improvements in cognition and memory, as well as long-term reductions in toxic protein aggregation and neuroinflammation, and enhanced mitochondrial function.
The double-blind, randomised, single and multiple ascending dose trial reported no severe or serious adverse events and no discontinuations due to adverse effects.
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By GlobalDataPharmacokinetic results indicate a low variability, long half-life, and dose-linear exposure suitable for once-daily dosing.
Vandria CEO Klaus Dugi 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 I 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 II clinical development strategy.
“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.”
Vandria has plans for a Series B round in 2026 for financing proof-of-concept Phase II trials. The company will also use these funds for its pre-clinical pipeline targeting liver, muscle, and lung diseases.
