Allon Therapeutics Inc. announced today that it has reached
agreement with the FDA on a
Special Protocol Assessment (SPA) for a pivotal Phase 2/3 clinical trial to
evaluate the company’s lead neuroprotective drug candidate, davunetide, as a potential treatment
for progressive supranuclear palsy (PSP), a rapidly-progressing and fatal
degenerative brain disease.
The SPA is a mechanism through which the FDA and Allon reach
agreement on the design, size, clinical endpoints, and data analysis of a
clinical trial that is intended to support an efficacy claim in a New Drug
Application (NDA) for regulatory approval. The SPA ensures that the agreed
clinical trial design meets the FDA’s expectations for a pivotal study. Allon
believes that if this pivotal study generates statistically significant and
consistent data, davunetide will be considered by FDA for approval in PSP.
(http://www.allontherapeutics.com/leadership.html) , President and CEO of
Allon, said the SPA agreement gives Allon a clear path to develop davunetide
for the treatment of PSP and provides a foundation for further development in
other brain diseases involving tau pathology.
“This SPA is an important part of our regulatory
strategy for davunetide. We believe the pivotal trial agreed to in the SPA will
generate data in about 2 years that will form the basis of approval of
davunetide in PSP,” said McCauley.
“Most important is the opportunity to help thousands of
patients who suffer from a debilitating and fatal disease,” said McCauley.
“PSP patients and their families endure an unspeakable tragedy, often
dying a little more than three years after diagnosis.”
“Our research to date suggests that davunetide will
work in patients with the tau pathology which is a characteristic feature of PSP.
Given these outcomes and that there is a recognized rating scale measuring
clinically relevant outcomes, we believe there is a strong rationale to pursue
approval in PSP.”
The pivotal Phase 2/3 randomized, double-blinded, placebo
controlled trial in PSP will enroll 300 patients in the United States, Canada,
United Kingdom, France, Germany,
Study participants will receive either placebo or 30 mg of davunetide
administered twice daily for a period of 12-months.
The primary outcome measures will be the Progressive
Supranuclear Palsy Rating Scale (PSPRS) and the Schwab and England Activities
of Daily Living (SEADL) scale. Secondary measures will include Clinical Global
Impression (CGI) and brain imaging by magnetic resonance tomography (MRI).
Additional exploratory endpoints include cognitive and executive function as
well as cerebrospinal fluid (CSF) biomarkers.
Allon previously announced that davunetide received orphan
drug designation in the United States (January 12, 2010) and the European Union
(March 17, 2010), Fast Track Status with the FDA (April 6, 2010), and that
davunetide met the primary outcome measure of safety and tolerability, with
potential trends from secondary efficacy endpoints, in a pilot study of 12 patients
suffering PSP and related tau pathology disorders (October 8, 2010).