In phase 2 trials, the drug reduced ‘OFF time’ which refers to periods of the day when Parkinson’s symptoms recur despite medication

Cerevance has announced positive phase 2 trial results for the company’s first-in-class, once-a-day, orally-adminstered compound treating Parkinson’s disease.

The company is a private, clinical-stage drug discovery and development company, focused on central nervous system diseases.

Beyond meeting safety objectives, the drug – CVN424 – also achieved significant and meaningful reduction of periods in which Parkinson’s symptoms recur despite medication. These periods are referred to as ‘OFF time’. At a high dose, CVN424 demonstrated a 1.3-hour improvement in OFF time compared to the placebo at four weeks.

“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, Cerevance’s chief executive officer. “We look forward to rapidly advancing CVN424 into several larger clinical studies aimed at obtaining regulatory approval.”

The drug also had an encouraging side-effect profile, with adverse reactions of nausea, vomiting and headache only occurring in two subjects (4%), both at the higher dose.

Mark Carlton, chief scientific officer at 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. The data also increases the confidence we have in our pipeline of additional programmes against novel targets identified by our approach.”

CVN424 was evaluated in a randomised double-blind, placebo-controlled, multicentre phase 2 study, at two dose levels in Parkinson’s disease patients with motor fluctuations.

“For more than 50 years, physicians have relied on therapeutics that work by directly increasing dopaminergic signalling,” concluded Karl Kieburtz, president of Clintrex, which specialises in development pathways. “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.”