Allon Therapeutics Inc. (NPC:CA)

Drugs that impact neurodegenerative diseases


Allon Therapeutics Inc. is a clinical-stage biotechnology company focused on bringing to market innovative central nervous system therapies. Allon's lead drug davunetide, is proceeding in a pivotal Phase 2/3 clinical trial in an orphan indication, progressive supranuclear palsy (PSP), under an SPA with the FDA.


This pivotal trial is based upon statistically significant human efficacy demonstrated in amnestic mild cognitive impairment, cognitive impairment associated with schizophrenia, and positive biomarker data.

Development opportunities


Allon has opportunities in a number of markets of unmet need: progressive supranuclear palsy (PSP), Alzheimer's disease, cognitive impairment associated with schizophrenia and Parkinson's disease.  All of these diseases impact the protein tau.  Davunetide has shown a positive impact on tauopathies in animal studies and in human clinical trials.  The Company is moving forward towards its first approval for davunetide for the potential treatment of PSP.  In the fourth quarter of 2010, Allon began enrolling patients in a pivotal Phase 2/3 clinical trial for PSP; this pivotal trial is based upon statistically significant human efficacy demonstrated in its Phase 2 Alzheimer's and schizophrenia programs.


Orphan strategy


In 2010, Allon embarked on the strategic path to pursue approval in a smaller “orphan drug” disease called Progressive Supranuclear Palsy (PSP). This market is estimated at $700 million, but with a more direct and less costly pathway than larger market opportunities such as Alzheimer's disease, schizophrenia and Parkinson's. This strategy provides the opportunity to retain more of the value for Allon and its shareholders. PSP is made more attractive by “orphan drug” regulations that streamline the approval process and exclude competition for several years. Many of the world's leading biotech and pharma companies have succeeded with this strategy.
PSP rationale

There is a strong scientific rationale for Allon's strategy: The pathology of PSP involves impairment of the brain protein tau – and Allon's animal research plus Phase 2 clinical trial in pre-Alzheimer's patients demonstrate that davunetide is the most advanced tau therapy in the world.



Special Protocol Assessment (SPA)


In January 2011, the Company announced that the Food and Drug Administration (FDA) and Allon agreed on a Special Protocol Assessment (SPA), for its pivotal Phase 2/3 clinical trial in PSP.


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.


Progress in Allon's PSP program includes:

• Allon began enroling patients in a pivotal Phase 2/3 clinical trial
• FDA and Allon agree on a Special Protocol Assessment
• Allon announced that pilot clinical trial meets primary endpoint with Allon's davunetide
• Allon received Orphan drug designation for davunetide
  
  in the United States (FDA) and the European Union, the world's two largest pharmaceutical markets;
• Allon received Fast track status in the U.S. (FDA)

Allon believes this next trial will not just provide data for a potential approval in PSP, but will also define the opportunity in other tau-related diseases, such as Alzheimer's, schizophrenia, Parkinson's and several types of frontotemporal dementia.


Because orphan drug designation does not restrict a drug from larger diseases, it will have no impact on the Company's strategy to seek approval in these larger markets, such as Alzheimer's, schizophrenia and Parkinson's in collaboration with a major pharma partner.
For more information
Rick Smith

604-742-2543

Director, Investor Relations

rsmith@allontherapeutics.com