Allon Therapeutics Inc. has announced that its lead neuroprotective drug candidate, davunetide, has been granted Orphan Drug Status in the European Union (EU) for the treatment of Progressive Supranuclear Palsy (PSP), a rapidly-progressing and fatal degenerative brain disease. Allon announced on January 12, 2010 that the U.S. Food and Drug Administration granted davunetide Orphan Drug Status for PSP treatment in the United States.
Gordon McCauley (http://www.allontherapeutics.com/leadership.html) , President and CEO of Allon, said the EU designation extends both the scientific validation and the potential market for davunetide as a treatment for PSP as well as other degenerative brain diseases with similar pathologies.
"Extending davunetide's potential use in the area of PSP is a significant business opportunity for Allon, made even more attractive by achieving Orphan Drug Designation in the world's two largest pharmaceutical markets, the U.S. and the EU," said McCauley. "Allon has demonstrated a strong scientific and clinical rationale for the potential efficacy of davunetide in PSP. The pathology of PSP and Alzheimer's is similar in that both diseases involve impairment of the brain protein tau. Our clinical trial program is focused on confirming davunetide's potential to be the first approved treatment for PSP and other brain diseases involving tau impairment."
Research has shown that davunetide reduced tau impairment and preserved memory in mice bred to replicate Alzheimer's or PSP tau pathology. In addition, Phase 2 clinical trials have shown that davunetide can also improve memory function in patients with amnestic mild cognitive impairment (aMCI), a precursor to Alzheimer's disease in which a primary neuropathology involves tau.
"We are pleased to have reached this important milestone and look forward to working with the European regulators as we plan and begin the European portion of our upcoming PSP clinical trial," said McCauley.
Allon announced January 26, 2010 that the clinical trial program for PSP is commencing with a pilot clinical trial in the U.S. sponsored by the Memory and Aging Center of the University of California, San Francisco (UCSF). This pilot clinical study, enrolling approximately 12 patients, will help Allon and its clinical collaborators validate the trial design and prepare for a larger multinational Phase 2 PSP clinical trial scheduled to begin this year.
Trial investigators are among the leading experts in the field, including Drs. Bruce Miller and Adam Boxer of the UCSF Memory and Aging Center.
On March 11, 2010, Allon announced it had completed a Phase 1 clinical trial that broadened the demonstrated safety and dose range of davunetide. Davunetide has already shown human efficacy and safety in clinical trials with daily doses of 5 mg and 30 mg. The new trial evaluated the drug's safety and pharmacokinetic profile at doses up to 60 mg per day.