Ixico Regulatory News (IXI)

28 July 2011

Cogane™ - US FDA Grant Orphan Drug status for treatment of ALS

Phytopharm plc (PYM: London Stock Exchange) ("Phytopharm" or the "Company") today 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.

About Phytopharm

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 is listed on the Official List of the London Stock Exchange. Further information on Phytopharm is available from the Company's website www.phytopharm.com

About Cogane™

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, CoganeTM has been shown to induce and modulate the production of neurotrophic factors. The neuroprotective and neurotrophic actions of CoganeTM 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.

CoganeTM is currently being studied in a Phase II trial of early stage Parkinson's disease as well as in preclinical models of ALS.

About 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. 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.

About Orphan Drug Designation

For a product to gain orphan drug status in the United States, the clinical condition must affect less than 200,000 people in the United States. If a product has been granted orphan drug status, FDA will provide assistance in the design of the preclinical and clinical studies needed to achieve marketing approval for the designated clinical condition. Additionally, there are financial incentives available (such as waiver of the fee for the marketing application, currently over $1.5M) and 7 years' market exclusivity (compared to 5 years available for non‑orphan diseases).

Forward-looking statements

Certain information included in these statements is forward‑looking and involves risk and uncertainties that could cause results to differ materially from those expressed or implied by the forward looking statements.

Forward‑looking statements include, without limitation, projections relating to results of operations and financial conditions, market estimates, the Company's plans and objectives for future operations, including future revenues, financial plans and expected expenditures and divestments. All forward‑looking statements in this report are based upon information known to the Company on the date of this announcement. The Company undertakes no obligation to publicly update or revise any forward‑looking statement, whether as a result of new information, future events or otherwise.

It is not reasonably possible to itemise all of the many factors and specific events that could cause the Company's forward‑looking statements to be incorrect or that could otherwise have a material adverse effect on the future operations or results of the Company.