The Phase 3 Safety Study and the Ongoing Phase 3 Efficacy Study Will Form the Basis for the Expected NDA Submission Near the End of 2016

TORONTO, Canada I September 2, 2015 I Cynapsus Therapeutics Inc. (NASDAQ:CYNA) (CTH.TO) (the “Company”) today announced enrollment of the first patient in the CTH-301 clinical trial, a pivotal Phase 3 study to examine the safety and tolerability of APL-130277 for the acute treatment of OFF episodes in patients with Parkinson’s disease (PD). CTH-300, a pivotal Phase 3 efficacy study, is also ongoing with data expected from both trials in 2016.

Dr. Albert Agro, Chief Medical Officer of Cynapsus, stated, “Enrolling the first patient in the CTH-301 safety study is a significant milestone for Cynapsus. The design and endpoints for this study were based on an End-of-Phase 2 meeting with the FDA earlier this year. The CTH-301 study, together with the CTH-300 efficacy study, will form the basis for our expected NDA submission near the end of 2016. We believe that apomorphine, the only approved drug to rapidly treat OFF episodes, combined with our sublingual film delivery technology, could provide many patients with a rapid, reliable means of turning ON from the OFF state.”

The CTH-301 trial is a 6-month, open-label, single arm safety study in PD patients who have at least one OFF episode every 24 hours, with total OFF time of at least two hours per day. The primary endpoint for the study is the safety and tolerability of APL-130277 in patients with PD. The secondary endpoints examine efficacy variables including the change in the MDS-UPDRS Part III scores over the 6-months of treatment. Sites will recruit patients over several months, with each patient being evaluated for six months. An estimated 226 patients will be enrolled, including up to 126 who had been enrolled in the CTH-300 efficacy study and rolled over to this study, plus an additional 100 new patients.

For additional information about the CTH-301 trial and to learn more about eligibility, patients can visit

Anthony Giovinazzo, President and Chief Executive Officer of Cynapsus, said, “Approximately 400,000 people with PD in the U.S. experience debilitating OFF episodes. When PD patients experience an OFF episode, they are often unable to perform simple daily tasks such as eating, bathing and dressing, thus becoming increasingly dependent on caregivers. We believe that APL-130277 can, if approved, change the way a large number of patients around the world manage OFF episodes. While we expect to have 12 weeks of safety data from the CTH-300 efficacy study in early to mid 2016, the 6-month data from the CTH-301 safety study will provide a much more robust assessment of safety.”

About Cynapsus

Cynapsus is a specialty Central Nervous System pharmaceutical company developing and preparing to commercialize a fast-acting, easy-to-use, sublingual thin film for the on-demand turning ON of debilitating OFF episodes associated with PD. The Company recently completed a Phase 2 clinical trial for its lead product candidate, APL-130277, a sublingual formulation of apomorphine hydrochloride, or apomorphine. Apomorphine is the only molecule approved for acute, intermittent treatment of OFF episodes for advanced PD patients, but is currently only approved as a subcutaneous injection in the United States. APL-130277 is a “turning ON” medication designed to rapidly, safely and reliably convert a PD patient from the OFF to the ON state while avoiding many of the issues associated with subcutaneous delivery of apomorphine. It is designed to convert all types of OFF episodes, including morning OFF episodes, often considered the most difficult to treat. Cynapsus has initiated its Phase 3 clinical program for APL-130277, relying on the abbreviated Section 505(b)(2) regulatory pathway in the United States, and the Company intends to submit a new drug application in 2016.

SOURCE: Cynapsus Therapeutics