Phase 3 Study of Oral Therapy for ‘Off’ Episodes in Parkinson’s Now Enrolling Patients

Cynapsus Therapeutics recently provided an update on APL-130277, its sublingual formulation of apomorphine hydrochloride or apomorphine. Apomorphine injection is the only treatment approved for acute, intermittent “off” episodes in advanced Parkinson’s disease (PD) patients.

Motor symptoms such as tremor at rest, rigidity, and impaired movement or freezing, as well as difficulties with starting to move, characterize Parkinson’s disease. The re-emergence of PD symptoms — either through a treatment’s wearing off or as intermittent episodes — is referred to as an “off” episode. APL-130277 is a “turning on” drug, developed to rapidly, safely and reliably convert a patient from an “off” to an “on” state, while avoiding the side events associated with subcutaneous delivery of apomorphine.

The company has successfully completed a Phase 2 clinical trial for APL-130277, but the product is currently only approved as a subcutaneous injection in the United States. Two Pivotal Phase 3 clinical trials (CTH-300/CTH-301 trials) for APL-130277 are underway and currently recruiting PD patients.

The CTH-300 trial (NCT02469090) is a double-blind placebo-controlled study for PD patients, responsive to standard therapy (levodopa), who have at least one “off” episode every day and a total “off” time of at least two hours per day. Patients will be observed for 12 weeks, with dosing at home and in the clinic, and evaluated every four weeks in the clinic. Following completion of the study, patients in the placebo arm and others may join the CTH-301 trial and receive APL-130277.

The CTH-301 trial will assess the safety and tolerability of APL-130277 in advanced PD patients meeting similar conditions as the trial discussed above, but treatment will continue for six months.

Patients are being invited to participate in the trial, which is taking place at 34 sites across the U.S. and in Ontario, Canada. More information is available through the trial’s clinical trials.gov website.

“2016 is a critical year for Cynapsus as we continue to focus on the management and conclusion of our pivotal Phase 3 clinical program for APL-130277,” Anthony Giovinazzo, company president and CEO, said in a press release. “We expect to announce top-line data from the CTH-300 Phase 3 efficacy trial in the second or third quarter of this year, and we intend to file our NDA with the U.S. FDA [Food and Drug Administration] near the end of 2016 or in early 2017.  We are also … engaging with European regulatory authorities to finalize our clinical development strategy in Europe.”