Phytopharm plc announces that the United States Food & Drug Administration (US FDA) has granted Orphan Drug status to Cogane (PYM50028) for the treatment of Amyotrophic Lateral Sclerosis (ALS).

ALS, also known as Lou Gehrig's disease, is the most prevalent form of motor neurone disease, which generally strikes people between 40 and 60 years of age. It is characterised by progressive loss of both lower (spinal cord and brain stem) and upper (cerebral cortex) motor neurones, which leads to severe muscle weakness and wasting, followed by paralysis and death, generally caused by respiratory failure. There is an urgent need for the development of new approaches to this devastating condition. There are estimated to be 20-30,000 people with ALS in the United States with approximately 5,600 new cases diagnosed each year. For a product to gain orphan drug status in the United States, the condition must affect less than 200,000 people in the United States.

As reported in the Company’s Interim Results, Cogane has previously shown promising activity in preclinical in vitro and in vivo models of ALS. A study of Cogane in the genetic “gold standard” in vivo model of ALS is ongoing. This study has the support of the Motor Neurone Disease Association, a UK based charitable organisation, which has provided a grant to cover the costs of the study. Results from the study are expected in  Q4 2011 and, if positive, offers the potential to support a rapid progression into clinical trials of ALS.

Phytopharm is focused on the development of pharmaceutical products in areas of high unmet health needs. As a virtual company, the company’s business model is centred on a lean cash burn with all laboratory, manufacturing and clinical work outsourced to specialists, while core competencies such as strategy and management are maintained in-house. Close collaboration with charitable organisations and leading scientists and clinicians help guide the company’s research and development activities.

Phytopharm’s lead development candidate is Cogane, a member of the sapogenin class of compounds. It is an orally bioavailable neurotrophic factor inducer that readily crosses the blood-brain barrier. Cogane has demonstrated neuroprotective effects in a range of preclinical models of neurodegenerative diseases. Specifically, Cogane has been shown to induce and modulate the production of neurotrophic factors. The neuroprotective and neurotrophic actions of Cogane suggest potential beneficial effects in a range of neurodegenerative diseases, including Parkinson’s disease and orphan diseases such as ALS.

Cogane has completed long term toxicology studies, has been formulated as a once daily, orally administered therapy and has completed phase I studies demonstrating a good bioavailability and safety profile. It is currently being studied in a phase II trial of early stage Parkinson’s disease as well as in preclinical models of ALS.

ALS, also known as Lou Gehrig's disease, is the most prevalent form of motor neurone disease, which generally strikes people between 40 and 60 years of age. It is characterised by progressive loss of both lower (spinal cord and brain stem) and upper (cerebral cortex) motor neurones, which leads to severe muscle weakness and wasting, followed by paralysis and death, generally caused by respiratory failure. The worldwide prevalence is  estimated to be 0.5-0.9 per 10,000. There are estimated to be 20-30,000 people with ALS in the United States with approximately 5,600 new cases diagnosed each year.

Phytopharm is a development stage pharmaceutical company developing novel treatments targeting diseases with high levels of unmet need.