Cerevance Reports Positive Phase 2 Clinical Trial Results with CVN424, a Parkinson’s Disease Drug Working Through a New Mechanism

in Portfolio News

Demonstrates the Power of Cerevance’s Deep Understanding of Specific Human Brain Cell Types

Boston, MA – March 31, 2022 – Cerevance, a private, clinical-stage drug discovery and development company focused on central nervous system diseases, today announced the completion of its Phase 2 clinical trial of CVN424, the company’s first-in-class, once-a-day, orally-delivered compound in development for the treatment of Parkinson’s disease. Beyond meeting safety objectives, the drug achieved a significant and meaningful, dose-dependent reduction of “OFF time,” which refers to periods of the day when Parkinson’s symptoms recur despite medication. It also had an encouraging side-effect profile.

CVN424 was evaluated in a randomized, double-blind, placebo-controlled multicenter Phase 2 study at two dose levels in Parkinson’s disease patients with motor fluctuations. Approximately 135 subjects with Parkinson’s disease, on a stable dosage of levodopa and other Parkinson’s medications but with at least two hours or more per day of average OFF time, were enrolled. Following baseline safety and efficacy assessments, subjects were randomized to receive once-daily doses of low-dose CVN424, high-dose CVN424 or matching placebo for four weeks.

At the high dose, CVN424 showed a 1.3-hour improvement in OFF time compared to placebo (p=0.042) at four weeks. This was accompanied by an increase in ON time without Troublesome Dyskinesia, without a meaningful worsening of ON time with Troublesome Dyskinesia. Efficacy improved at four weeks versus at two weeks, and daytime sleepiness as measured by the Epworth Sleepiness Scale was reduced compared to placebo, differentiating it from most Parkinson’s disease drugs used as adjuncts to levodopa. At the lower dose, CVN424 also demonstrated a meaningful improvement in OFF time and ON time without Troublesome Dyskinesia compared to placebo. The most common adverse reactions were nausea, vomiting and headache, occurring in two subjects (4%) each at the higher dose. All other adverse reactions occurred in one subject or less.

“We are delighted to report these results which we believe demonstrate that CVN424 can provide a significant improvement for patients, with little exacerbation of dopaminergic side effects,” said Brad Margus, chief executive officer of Cerevance. “We look forward to rapidly advancing CVN424 into several larger clinical studies aimed at obtaining regulatory approval.”

Mark Carlton, Ph.D., chief scientific officer of Cerevance, added, “CVN424’s positive results demonstrate the power of the deep, cell-type-specific transcriptional and epigenetic data we are generating by applying our NETSseq platform technology to thousands of post-mortem human brain tissue samples. These data also increase the confidence we have in our pipeline of additional programs against novel targets identified by our approach.”

“For more than 50 years, physicians have relied on therapeutics that work by directly increasing dopaminergic signaling,” said Karl Kieburtz, M.D., President of Clintrex, a group of clinical development and regulatory experts who have collectively led more than 75 international multicenter clinical trials of treatments for movement disorders and neurodegenerative diseases involving tens of thousands of patients. “This new mechanism holds great promise for treating the motor fluctuations eventually experienced by all Parkinson’s disease patients, as well as potential for treating patients in the earlier stages of the disease.”

Cerevance intends to pursue additional Phase 2/3 clinical studies with CVN424 to establish a clear path to regulatory approval of the drug for adjunctive therapy. In parallel, the company will evaluate the drug’s promise as stand-alone treatment for recently diagnosed patients not yet treated with levodopa.

About Cerevance

Cerevance, a private, clinical-stage pharmaceutical company focused on brain diseases, is applying a new technology called NETSseq to reveal transcriptional and epigenetic differences between specific cell types in post-mortem human brain tissue acquired from donors whose ages varied from eight to 104 years old. NETSseq profiles neuronal and glial cell populations at depths not possible with other approaches, generating unprecedented data sets and insights. The company has thus far partnered with more than 20 brain banks around the world to assemble a growing collection of more than 10,000 clinically annotated samples from healthy and diseased donors. By applying NETSseq to specific cell types critical to circuits disrupted by disease and comparing vulnerable and resilient cell populations, Cerevance’s scientists have begun identifying targets and advancing a pipeline of novel therapeutics that modulate them to treat CNS diseases.


Robert Middlebrook, +1-408-220-5722

Andrew Mielach, amielach@lifescicomms.com, +1-646-876-5868

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