Cynapsus Therapeutics has enrolled the final patient in its pivotal Phase 3 clinical study investigating the clinical effects of APL-130277, a fast-acting sublingual thin film for the treatment of debilitating OFF states in Parkinson’s disease (PD). Dose-titration phase results and a final evaluation of the study are expected to be released by the end of 2016.
OFF periods are a complication of the disease associated with motor symptoms, such as rigidity, tremor at rest, dysfunctional movement, significant non-motor symptoms like cognitive impairment, and mood dysfunctions. From 25 to 50 percent of patients whose symptoms are managed with current drug treatments experience OFF states a minimum of once a day and a maximum of six times a day, with a duration of between 30 minutes and two hours.
The CTH-300 clinical study is a placebo-controlled, double-blind, parallel-design trial involving participants who have at least one OFF episode every 24 hours and a total OFF time of at least two hours per day. The objective of the trial is to estimate the safety and efficacy of APL-130277 compared to a placebo in people suffering from Parkinson’s. Participants will be followed for 12 weeks.
The trial’s primary endpoint is the mean change in the Unified Parkinson’s Disease Rating Scale Part III score, determined by the Movement Disorder Society, at 30 minutes after clinical treatment at week 12. The key secondary endpoint is the rate of participants who turn from the OFF episodes to the ON state at or before 30 minutes of treatment with APL-130277 at the last visit.
Cynapsus Therapeutics, based in Toronto, Canada, has successfully completed a Phase 2 clinical study for APL-130277 (apomorphine, apomorphine hydrochloride). At this point, only apomorphine is approved as a treatment for acute OFF episodes in advanced Parkinson’s patients. It has only been approved in the U.S. to be administered via subcutaneous injection.
APL-130277 can convert patients from OFF episodes to ON states without the issues linked with subcutaneous administration of apomorphine. It is intended to convert even morning OFF states, which are considered the most problematic to manage. Cynapsus plans to submit a New Drug Application (NDA) to the U.S. FDA by the end of this year or in early 2017.