The study included 64 healthy volunteers who received either single doses or seven daily doses of CVN424, ranging from 1 mg to 225 mg, or placebo. There were no serious or severe adverse events or clinically significant changes in vital signs, ECG or laboratory values. The drug was rapidly absorbed orally and had a half-life that will support once-daily dosing.

“CVN424’s excellent safety profile helps validate Cerevance’s approach to CNS target selection and drug discovery,” said David H. Margolin, MD, PhD, Senior Vice President for Clinical and Translational Medicine at Cerevance. “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.”

CVN424 acts on a novel, non-dopaminergic target protein present specifically in dopamine receptor D2-expressing medium spiny neurons in the indirect pathway of the basal ganglia. The compound modulates the D2-dependent indirect pathway, but not the D1-dependent direct pathway. This selective targeting is designed to produce the positive therapeutic effects of current treatments while avoiding side effects such as dyskinesia associated with dopaminergic therapies.

“We are pleased with the Phase 1 trial results for CVN424 and are eager to advance into Phase 2,” said Brad Margus, Chief Executive Officer of Cerevance. “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. Beyond CVN424, we look forward to advancing a broad pipeline of therapies acting on targets that have not yet been explored for the treatment of CNS disorders.”

Based on the positive Phase 1 results, a Phase 2 study to demonstrate the clinical benefits of CVN424 will be initiated later this year.