Cynapsus' Parkinson's Disease Therapy Succeeds In Phase 2 Study
By C. Rajan, contributing writer
Cynapsus Therapeutics has announced positive results from the Phase 2 clinical trial of its lead candidate, APL-130277, for treating OFF motor symptoms of Parkinson’s disease. APL-130277 is a fast-acting formulation of apomorphine, which is designed to help patients quickly gain control of their symptoms.
In the multicenter, open-label Phase 2 study (CTH-105), APL-130277 was evaluated in 16 patients with Parkinson's disease who suffered from extended duration OFF episodes and its debilitating effects daily. The patients were restricted from their therapeutic levodopa doses to induce a severe OFF state (immobility) and then were treated with five escalating doses of APL-130277 until ON state (full functionality) was achieved.
The results showed that APL-130277 was safe and well tolerated, and all five dose strengths caused the patients to convert from OFF to ON state. The improvement in symptoms was seen as early as 10 minutes and lasted up to 90 minutes.
The CTH-105 clinical study was partly funded by a grant of $500,000 from The Michael J. Fox Foundation for Parkinson’s Research, awarded to Cynapsus in July this year.
Based on the positive results of this study, Cynapsus plans to confirm these results in pivotal studies that will last longer and enroll more patients. If the results of the pivotal studies are as expected, then Cynapsus will submit a New Drug Application with the U.S. FDA in 2016.
"The results of this Phase 2 trial are important, as the data show that APL-130277 provided Parkinson's patients with a rapid improvement in motor function during OFF episodes," said Anthony Giovinazzo, President and CEO of Cynapsus.
"APL-130277 is being developed to address a significant unmet need facing people with Parkinson's disease today. The CTH-105 trial results lead us to maintain that APL-130277 may be able to serve the majority of Parkinson's patients seeking to restore movement rapidly, on demand, with an easy to retrieve and to administer form of apomorphine, the only approved and most efficacious drug for this purpose."
According to Cynapsus, approximately one million people in the U.S. suffer from Parkinson's disease, which is a progressive neurodegenerative disease affecting the motor functions. Nearly half of Parkinson’s patients experience a further complication called OFF episodes, which can make them rigid and totally immobile. These episodes can occur multiple times a day, and last up to 2 hours.
Apomorphine is the only approved drug that can help patients manage the OFF episodes and recover their mobility. However, as of now, apomorphine is available only as an injection, which can be difficult to administer for the multiple OFF episodes daily.
Cynapsus’ APL-130277 is the only orally administered form of apomorphine, which is formulated as a thin film strip to be placed under the tongue when an OFF episode is starting. Last month, APL-130277 was recognized as one of the Top Ten Neurology Projects to Watch by a joint selection committee of brain and neurology experts.