Ixico Regulatory News (IXI)

16 January 2012

Phytopharm plc

Cogane™ demonstrates positive results in Amyotrophic Lateral Sclerosis study

Phytopharm plc (PYM: London Stock Exchange) ("Phytopharm" or the "Company") today announces preliminary data indicating that Cogane™ has demonstrated efficacy in a genetic preclinical model of amyotrophic lateral sclerosis (ALS).

The study was performed in a model that has a mutation in the SOD1 gene (SOD1G93A); mutation of the SOD1 gene is a known cause of ALS in humans. In this study Cogane™ was administered orally for 50 days, commencing after ALS‑type symptoms were manifest. This is therefore considered to be a model of severe, late-stage ALS.

Main findings:

·; Administration of Cogane™ resulted in a 30‑50% improvement in muscle strength in one muscle type compared to both the untreated control group and a group treated with riluzole (currently the only product marketed for the treatment of ALS).

·; Treatment with CoganeTM also resulted in an increase in the number of motor units (a measure of functional motor neurones) compared with both the untreated and riluzole treated control groups.

·; Treatment effects were less clear in a second muscle type which was more severely damaged in the model, though the group treated with Cogane™ again showed an improvement in strength compared to the riluzole treated group.

These are preliminary, headline results and the full results from the study, including histopathology data, will be published in due course.

These results support those reported previously by Phytopharm in which Cogane™ showed benefit in an environmental (toxin-induced) model of ALS, in a progressive motor neuropathy model and in a nerve crush model. Collectively the results from these four different models of ALS provide strong support for the utility of Cogane™ in the treatment of this condition.

ALS, also known as Lou Gehrig's disease, is the most common form of motor neurone disease, a neurodegenerative disease with limited treatment options and poor prognosis. 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.

Phytopharm has obtained orphan drug status in Europe and the US for Cogane™ in ALS.

This study was performed by the group of Professor Linda Greensmith, University College, London, with the financial support of the Motor Neurone Disease Association, a UK based charitable organisation.

Professor Greensmith commented, "The data from this genetic model of ALS are very encouraging and, taken with the data from the other models of ALS in which it has been tested, indicate that Cogane has significant potential as a therapy for ALS and merits further evaluation."

Mr Tim Sharpington, CEO, Phytopharm, said, "These results are very encouraging as we set a difficult challenge in this study by looking for efficacy in a severe, end-stage disease model. We have established an impressive set of data for CoganeTM in a broad range of neurodegenerative disease models, including Parkinson's and Alzheimer's disease as well as ALS. There is a major unmet need and substantial commercial opportunity for new therapies which can delay or halt the progression of these diseases. We look forward to receiving more data on Cogane™ as we complete our ongoing clinical study in Parkinson's disease."

About Phytopharm

Phytopharm is a development stage pharmaceutical company developing novel treatments targeting diseases with high levels of unmet need. Our lead series of compounds, the sapogenins (including Cogane™ and Myogane™), has the potential to be a new class of therapy for neurodegenerative diseases including Parkinson's disease, ALS and glaucoma.

Phytopharm operates as a virtual company ensuring the majority of our financial resources are focused on our pharmaceutical pipeline. We utilise a network of scientific and clinical experts to help guide our development projects with our experienced pharmaceutical managers overseeing operations.

Our commercially focused development projects have the potential to produce significant treatment advances in our target areas of neurodegeneration and inflammatory disease. Our products are single chemical entities with novel mechanisms of action protected by strong patent families.

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 modulator 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 Cogane™ suggest potential beneficial effects in a range of neurodegenerative diseases, including ALS and Parkinson's disease.

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.

In addition to the preclinical programme in ALS, Cogane™ is being studied in an ongoing 28 week Phase II trial of early stage Parkinson's disease (CONFIDENT‑PD).

In 2011 Cogane™ was granted Orphan Drug status by both the European Commission and by the US Food & Drug Administration for development in ALS. Orphan status allows significant access to the regulatory authorities for advice and expedited clinical progression as well as providing financial advantages.

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 estimated that there are over 30,000 patients living with ALS in the seven major markets.

About the SOD1G93A rodent model

SOD1 (superoxide dismutase 1) gene is responsible for generation of the superoxide dismutase‑1 enzyme. This enzyme helps in the control of free radicals. Mutation of this gene can result in the development of ALS. Although a genetic cause has not been identified in many patients with ALS, approximately 5% have a genetic mutation of the SOD1 gene. The SOD1 rodent model is the most widely studied ALS model.

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.