Pilot Clinical Trial Meets Primary Endpoint With Allon Therapeutics, Inc.'s Davunetide

VANCOUVER, BRITISH COLUMBIA--(Marketwire - October 08, 2010) - Allon Therapeutics Inc. (TSX: NPC) announced today that a pilot clinical trial successfully met its primary endpoint of safety and tolerability, with the Company’s lead neuroprotective drug candidate davunetide, in patients with progressive supranuclear palsy (PSP) and other types of frontotemporal dementia (FTD) like corticobasal syndrome, and progressive non-fluent aphasia. FTD is a group of rapidly progressive and fatal degenerative brain diseases, often misdiagnosed as Parkinson’s or Alzheimer’s disease.

The new clinical data are being presented today by Adam Boxer, MD, PhD, the principal investigator of the trial, to the International Conference on Frontotemporal Dementias in Indianapolis, Indiana. The conference is being attended by approximately 500 scientists, physicians, and caregivers from around the world. Dr. Boxer, one of the world’s leading authorities on FTD, is an Associate Professor of Neurology at the University of California San Francisco and director of the Alzheimer disease and frontotemporal dementia clinical trials program at the UCSF Memory and Aging Center.

“In this study, davunetide met the primary endpoint of safety over 12 weeks of treatment,” Dr. Boxer said. “This pilot study was small and enrolled three different clinical syndromes of frontotemporal dementia, including progressive supranuclear palsy.”

In addition to the positive safety data, Dr. Boxer reported that the trial also provided the perspectives of PSP patients and their caregivers on the battery of tests proposed for Allon’s efficacy trial. This feedback from patients and caregivers will help Allon and its clinical collaborators conduct the efficacy trial. “The results on the secondary outcome measures, some of which will be used in the Phase 2/3 efficacy trial in progressive supranuclear palsy, are consistent with our expectations,” he said.

Dr. Bruce Morimoto, Allon’s Vice President of Drug Development, said that despite the size and duration of the pilot clinical trial, the investigators noted a positive trend on a secondary outcome, the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS), a cognitive measure of memory, language, and attention.

“The results from this pilot study are quite encouraging, and support the rationale, design, and duration of our larger Phase 2/3 clinical trial in progressive supranuclear palsy,” Dr. Morimoto said.

Allon intends to develop davunetide as the first approved treatment for PSP, a type of frontotemporal dementia (FTD). The new data confirm earlier clinical safety results in patients with other neurodegenerative diseases and supports the Company’s plans to begin a robust efficacy trial with PSP patients in the near future.

The pilot clinical trial was a 12-week randomized, double-blind, placebo-controlled study of davunetide in 12 patients. During the trial, patients were given 15 milligrams of intranasal davunetide or placebo twice daily.

Allon’s progress thus far in 2010 in its PSP program includes:

 -- Orphan Drug designation for davunetide for a treatment for PSP in the United States and the European Union, the world’s two largest pharmaceutical markets; -- Fast Track status in the U.S. for davunetide for the treatment of PSP; -- Completion of a Phase 1 clinical trial that began enrolling patients January 28, 2010. The resulting Phase 1 data expanded the demonstrated safety range and pharmacokinetic profile of davunetide at dosage levels higher than previously used in the Company’s clinical trials; and -- Completion of this pilot study at University of California, San Francisco to validate the trial design for the efficacy clinical trial. 

About davunetide

Davunetide is derived from a naturally occurring neuroprotective brain protein known as activity dependent neuroprotective protein (ADNP). Allon’s laboratory and animal studies have shown that davunetide improves cognition in a number of disease models through a mechanism believed to involve effects on microtubules, structures in the brain critical to communication between cells.

About Allon’s neuroprotective platforms

Allon’s two neuroprotective 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 associated with schizophrenia, and progressive supranuclear palsy (PSP). AL-309 is being developed for the treatment of peripheral neuropathies and is administered orally or subcutaneously.

About Allon

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. Allon has Phase 2 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. The Company is listed on the Toronto Stock Exchange under the trading symbol “NPC” (Neuro Protection Company™) and based in Vancouver. For additional information please visit the Company’s website: www.allontherapeutics.com.

About Progressive Supranuclear Palsy

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. Approximately 20,000 and 50,000 persons in the U.S. and EU respectively have PSP.

Forward Looking Statements

Statements contained herein, other than those which are strictly statements of historical fact may include forward-looking information. Such statements will typically contain words such as “believes”, “may”, “plans”, “will”, “estimate”, “continue”, “anticipates”, “intends”, “expects”, and similar expressions. While forward-looking statements represent management’s outlook based on assumptions that management believes are reasonable, forward-looking statements by their nature are subject to known and unknown risks, uncertainties and other factors that may cause the actual results, events or developments to be materially different from any future results, events or developments expressed or implied by them. Such factors include, among others, the inherent uncertainty involved in scientific research and drug development, Allon’s early stage of development, lack of product revenues, its additional capital requirements, the risks associated with successful completion of clinical trials and the long lead-times and high costs associated with obtaining regulatory approval to market any product which Allon may eventually develop. Other risk factors include the limited protections afforded by intellectual property rights, rapid technology and product obsolescence in a highly competitive environment and Allon’s dependence on collaborative partners and contract research organizations. These factors can be reviewed in Allon’s public filings at www.sedar.com and should be considered carefully. Readers are cautioned not to place undue reliance on such forward-looking statements.