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Stock exchange listing: Toronto Stock Exchange (TSX)
Stock symbol: NPC
Shares outstanding: 100.6 M
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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 fully enrolled 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 and completed the enrollment objective of 300 patients in October 2011 .  This pivotal trial is based upon statistically significant human efficacy demonstrated in its Phase 2 Alzheimer’s and schizophrenia programs.

Clinical development

Phase 2a data from aMCI stufy provides human proof cof concept for frontetemporal dementias and Alzheimer's disease

Fig 1: Studies have shown that davunetide can repair Tau protien in the brains of mice that are bred to have a Tau impairment, known as Tau huperphosphorylation, common to half of frontotemporal dementias (FTD’s). Other studies have shown that these pathological improvements result in dramatically improved cognitive performance in these mice.

Fig 2: Studies in mice bred to have Alzheimer’s disease, show that Allon’s compound davunetide is the first in the world to have an affect on both pathologies of the disease: amyloid beta plaques and neurofibrillary tangles. Research has established that impairment of the brain protein Tau is involved in tangles.

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.

Fig 1. The Phase 2a clinical trial showed that davunetide improves memory function in aMCI subjects. The delayed match-to-sample test (12 sec delay) focuses on important areas of working, recognition and short-term memory which are important in aMCI and AD.

Fig 2. Davunetide provided improvement in memory function in a second test that is important in both aMCI and AD. This shows the activity of davunetide on memory which requires effective processing of language, speech and sub-vocal parts of the brain.

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 completed enrolling patients in a pivotal Phase 2/3 clinical trial
• FDA and Allon agree on a Special Protocol Assessment
• Allon announced that a 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.