Cerevance Achieves Key Endpoints in Phase 1 Clinical Trial of Novel Parkinson’s Disease Drug CVN424

Results Demonstrate Safety of CVN424 and Enable Initiation of
Phase 2 Study in Patients

BOSTON–(BUSINESS WIRE)–lt;a href="https://twitter.com/hashtag/CNS?src=hash" target="_blank"gt;#CNSlt;/agt;–Cerevance, a clinical stage biopharmaceutical company advancing new
medicines for brain diseases, has successfully completed its Phase 1
clinical trial of CVN424, the company’s first-in-class, orally-delivered
compound in development for the treatment of Parkinson’s disease. The
clinical study successfully met its primary endpoint of safety in
healthy volunteers.

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.

About Cerevance

Cerevance is a private pharmaceutical company focused on central nervous
system diseases. The company’s strengths include its powerful
NETSseq target discovery platform, a large collection of human brain
tissue samples, a pipeline of novel discovery-stage and clinical-stage
compounds and a team with a proven track record. Its scientists believe
that they are well positioned to deliver life-changing therapeutics for
patients who have brain-related disorders.

Contacts

Cerevance Contact:
Robert Middlebrook, +1.408.220.5722

Media Contact:
Cait Williamson, Ph.D., [email protected],
+1.646.751.4366