Allon Therapeutics Inc announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s lead neuroprotective drug candidate davunetide for the treatment of Progressive Supranuclear Palsy (PSP), a rapidly-progressing and fatal degenerative brain disease.
Gordon McCauley, president and CEO of Allon, said Orphan Drug Designation brings additional commercial benefits to the company’s programme to develop davunetide to become the first approved treatment for a disease often mis-diagnosed as Parkinson’s or Alzheimer’s disease.
“PSP is devastating for thousands of middle-age patients and their families,” said McCauley. “Meeting the desperate need for a treatment is a significant business opportunity for Allon made more attractive by the US FDA’s Orphan Drug Designation for davunetide.”
McCauley said 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 — and we have shown that davunetide is the most advanced tau therapy in the world.”
McCauley said davunetide’s potential as a treatment for PSP was confirmed in both animal studies and phase-II human clinical trials with patients with amnestic mild cognitive impairment (aMCI), a precursor to Alzheimer’s.
“Our research has shown that davunetide reduced tau impairment and preserved memory in mice bred to replicate Alzheimer’s or PSP tau pathology. In addition, our phase-II clinical trials have shown that davunetide can also improve memory function in aMCI patients.”
Allon’s clinical programme in this area is expected to begin shortly with a Pilot trial treating a small number of patients with tau impairments, including PSP. This trial will help validate the trial design and prepare for a larger Phase II clinical trial that the Company intends to initiate in 2010. Trial investigators are among the leading experts in the field, including Dr Bruce Miller and Dr Adam Boxer of the Memory and Aging Center of the University of California, San Francisco.
Davunetide is derived from a naturally occurring neuroprotective brain protein known as activity dependent neuroprotective protein (ADNP).
Allon’s two neuroprotetive technology platforms are based on two naturally occurring proteins produced by the brain in response to a range of insults.
Allon Therapeutics is a clinical-stage biotechnology company developing treatments for major neurodegenerative conditions.