Cerevance has reported positive data from a Phase I clinical trial of its first-in-class, orally-delivered compound in development, CVN424, for the treatment of Parkinson’s disease.

The double-blind, single and multiple-ascending dose study, which included 64 healthy volunteers, successfully met its primary endpoint of safety in healthy volunteers.

As part of the study, the volunteers were given either single doses or seven daily doses of CVN424, ranging from 1mg to 225mg, or placebo. No serious or severe adverse events or clinically significant changes in vital signs were found.

Cerevance Clinical and Translational Medicine senior vice-president David Margolin said: “CVN424’s excellent safety profile helps validate Cerevance’s approach to CNS target selection and drug discovery.

“Our NETSseq technology platform uses human post-mortem brain tissue to identify targets that are selectively expressed in specific cell types.”

“CVN424 acts through a target that is selectively expressed by neurons important in controlling movement and was engineered to achieve excellent CNS penetration with minimal effects on other organs.”

The compound CVN424 acts on a novel, non-dopaminergic target protein which is present specifically in dopamine receptor D2-expressing medium spiny neurons in the indirect pathway of the basal ganglia.

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It modulates the D2-dependent indirect pathway. The selective targeting has been designed to produce the positive therapeutic effects of current treatments while avoiding various side effects.

Cerevance CEO Brad Margus said: “Our NETSseq technology platform uses human post-mortem brain tissue to identify targets that are selectively expressed in specific cell types and circuits or changed in disease states, allowing us to discover new therapeutics for neurodegenerative diseases.”

The company started dosing patients in the Phase I first-in-human clinical trial of CVN424 in September last year.

Based on the positive Phase I data, the company plans to initiate a Phase II study later this year to demonstrate the clinical benefits of CVN424.