Davunetide, an investigative drug for the treatment of Progressive Supranuclear Palsy (PSP), has been granted Orphan Drug Designation by the United States Food and Drug Administration (US FDA), Allon Therapeutics Inc announced.
Davunetide is derived from a naturally occurring neuroprotective brain protein known as activity dependent neuroprotective protein (ADNP).
Davunetide hs been found restoring the function of structures in the brain — known as microtubules — which are critical to communication between brain cells and the structure of individual cells.
In 2008, Allon reported Phase IIa clinical trial results showing that davunetide had a statistically significant positive impact on memory function in patients with amnestic mild cognitive impairment (aMCI), a precursor to Alzheimer’s disease (AD).
On December 7, 2009, Allon reported Phase IIa clinical trial results showing that davunetide improved memory function of schizophrenia patients and had a positive impact on the ability of these patients to carry out important activities in their daily lives. The data was presented at the annual meeting of the American College of Neuropsychopharmacology.
“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,” stated Gordon McCauley, President and CEO of Allon.
Orphan Drug Designation brings additional commercial benefits to Allon’s program to develop davunetide to become the first approved treatment for a disease often mis-diagnosed as Parkinson’s or Alzheimer’s disease, he said.
Orphan drug status is granted by the FDA to encourage biotechnology and pharmaceutical companies to develop drugs that demonstrate promise for the treatment of diseases which affect fewer than 200,000 people in the United States.
Orphan Drug Designation qualifies Allon for an accelerated review process, tax credits, financial assistance for development costs, plus seven years of marketing exclusivity if davunetide is eventually approved by the FDA as a treatment for PSP.
Orphan Drug designation also allows for a possible exemption from the FDA-user fee and assistance in clinical trial protocol design.
Progressive Supranuclear Palsy (PSP) is one of a group of progressive disorders called frontotemporal dementia (FTD), that affect the frontal and temporal lobes of the brain, and for which there are no approved treatments.
Progressive Supranuclear Palsy (PSP) is often characterized by progressive difficulty with balance and walking, eye movement abnormalities, and cognitive and personality changes.
Patients are typically diagnosed when they are between 45 and 65 years of age.
Progressive Supranuclear Palsy (PSP) is associated with progressive disability and death with a median survival of five to 10 years following onset.
The disease is slightly more common in men than women, but there are no known geographical, occupational or racial patterns.
Approximately 200,000 persons in the United States and Canada have been diagnosed with a form of frontotemporal dementia (FTD), including 20,000 diagnosed with Progressive Supranuclear Palsy (PSP).
Approximately half of frontotemporal dementia (FTD)s, including PSP, are tauopathies, which are pathologies that involve impairment of the tau protein in brain cells.
Allon Therapeutics Inc. is a clinical-stage biotechnology company developing treatments for major neurodegenerative conditions.
Allon’s drug davunetide has demonstrated human efficacy in amnestic mild cognitive impairment, a precursor to Alzheimer’s disease, and cognitive impairment associated with schizophrenia.
Based in Vancouver, Allon has Phase II human efficacy programs pursuing large underserved markets, such as Alzheimer’s disease and cognitive impairment associated with schizophrenia, and in orphan markets, such as frontotemporal dementias.
Allon’s two neuroprotetive technology platforms are based on two naturally occurring proteins produced by the brain in response to a range of insults. The platforms are activity-dependent neuroprotective protein (ADNP) and activity-dependent neurotrophic factor (ADNF).
Because the two platforms are based on different proteins, the drugs from each are different molecules with different therapeutic mechanisms and distinct commercial opportunities.
Clinical-stage drugs based on davunetide are derived from ADNP, while preclinical stage drug AL-309 is derived from ADNF.
Davunetide is focused on Alzheimer’s disease, cognitive impairment in schizophrenia, and frontotemporal dementia.
ADNF drug candidate AL-309 is being developed for the treatment of peripheral neuropathies and is administered orally or subcutaneously.
Allon’s clinical program 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.