BOSTON--(BUSINESS WIRE)--Cerevel Therapeutics, a company dedicated to unraveling the mysteries of the brain to treat neuroscience diseases, announced today positive results from a Phase 2 clinical trial evaluating oral tavapadon (formerly PF-06649751) in patients with early-stage Parkinson’s disease. The study met its primary endpoint, demonstrating a statistically significant improvement from baseline in motor symptoms as evaluated by the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score at 15 weeks in patients treated with tavapadon compared with those treated with placebo.
The study results are being presented today at the 2019 International Congress of Parkinson’s Disease and Movement Disorders®, in Nice, France, by David Gray, Ph.D., vice president of chemistry at Cerevel Therapeutics and Phase 2 study team leader, during a poster presentation session from 1:45 to 3:15 p.m. CEST (7:45 to 9:15 a.m. EDT).
“The positive Phase 2 efficacy and safety data presented today reinforce the potential of tavapadon as a new treatment option for patients with Parkinson’s disease,” said Raymond Sanchez, M.D., chief medical officer of Cerevel Therapeutics. “Given the favorable profile of tavapadon in clinical studies to date, we see potential for this novel therapy in a variety of treatment settings for Parkinson’s disease, both as a monotherapy for patients with early-stage disease and as an adjunct to levodopa for patients with late-stage disease. Over the course of 2020, we plan to initiate a robust Phase 3 development program to fully characterize the utility of tavapadon in patients with early- and late-stage Parkinson’s.”
The randomized, double-blind, placebo-controlled, flexible-dose Phase 2 study enrolled 57 patients, age 45 to 80 years, with early-stage Parkinson’s disease (disease severity ranging from Stage I to III on the Hoehn & Yahr scale). The 15-week treatment period included nine weeks of dose optimization followed by six weeks of stable dosing. The primary efficacy endpoint was the change from baseline in the MDS-UPDRS Part III total score at week 15, evaluating motor symptoms. The study also included the Patient Global Impression of Change (PGI-C) and The Epworth Sleepiness Scale as secondary endpoints. Treatment compliance was high in both groups and 82 percent of patients who received study medication completed the trial.
Results demonstrated the following:
- The mean change from baseline at Week 15 in the MDS-UPDRS Part III score was -9.0 for tavapadon and -4.3 for placebo, with a least squares mean improvement over placebo of -4.8 in favor of the tavapadon group (p=0.0407).
- At Week 15, 50% of subjects treated with tavapadon reported being “much improved” or “very much improved” on the PGI-C, compared with 25% in the placebo group.
- No statistically significant effects of tavapadon compared with placebo were observed on daytime sleepiness at Weeks 9 and 15, as measured by the Epworth Sleepiness Scale.
- Tavapadon demonstrated a favorable safety and tolerability profile, with the majority of adverse events reported as mild or moderate. The most frequently reported adverse events in patients treated with tavapadon were nausea, headache, dry mouth, somnolence, and tremor.
“Current approaches and treatment options for patients with Parkinson’s disease are often associated with significant complications, troublesome side effects or limited efficacy,” said Dr. Gray. “I’m encouraged by these results, which demonstrated that tavapadon was significantly more effective than placebo in improving motor symptoms and was well tolerated. It has the potential to be a promising new treatment option for people with Parkinson’s disease.”
Tavapadon is a potent, orally-administered, selective partial agonist of the dopamine D1 and D5 receptors being evaluated for the once-daily symptomatic treatment of Parkinson’s disease.
About Parkinson’s Disease
More than 10 million people worldwide are living with Parkinson’s disease, according to the Parkinson’s Foundation. The disease is characterized by a progressive degeneration of dopaminergic neurons (the main source of dopamine) leading to a loss of critical motor and non-motor functions. Symptom severity and disease progression differ between individuals but typically include slowness of movement (bradykinesia), trembling in the extremities (tremors), stiffness (rigidity), cognitive or behavioral abnormalities, sleep disturbances, and sensory dysfunction.1 There is no laboratory or blood test for Parkinson’s disease, so a diagnosis is made based on clinical observation,2 which may contribute to an underestimation of the incidence.
About Cerevel Therapeutics
Cerevel Therapeutics is dedicated to unraveling the mysteries of the brain to treat neuroscience diseases. The company is unlocking new treatment opportunities by understanding the neurocircuitry of the symptoms. Cerevel Therapeutics has a diversified pipeline comprising four clinical-stage investigational therapies and several pre-clinical compounds with the potential to treat a range of neuroscience diseases, including Parkinson’s, epilepsy, schizophrenia and addiction. Headquartered in Boston, Cerevel Therapeutics is advancing its current research and development programs while exploring new modalities through internal research efforts, external collaborations or potential acquisitions. For more information, visit www.cerevel.com.
1 J Neurol Neurosurg Psychiatry. 2008;79:368-376. doi:10.1136/jnnp.2007.131045.
2 Cold Spring Harb Perspect Med. 2012;2:a008870.