Ixico Regulatory News (IXI)

21 May 2008 07:00



21 May 2008

Phytopharm plc

Myogane granted European Union Orphan Medicinal Product Designation for ALS

GODMANCHESTER, Cambridgeshire, U.K. (21 May 2008) - Phytopharm plc (PYM: London Stock Exchange) ("Phytopharm" or the "Company") announces today that the European Agency for the Evaluation of Medicinal Products ("EMEA") has awarded Orphan Medicinal Product Designation for Myogane™, a novel orally active neurotrophic factor inducer as a treatment for amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease), the most prevalent form of motor neurone disease. The Orphan Medicinal Product Designation provides Phytopharm with market exclusivity in the European Union for 10 years following Myogane™'s market authorisation.

The EMEA grants the Orphan Medicinal Product Designation for products that diagnose, prevent or treat life-threatening or very serious conditions affecting no more than five out of every 10,000 people in the European Union.  ALS is a fatal neurodegenerative disease characterised by progressive degeneration of both upper and lower motor neurones which lead to severe muscle weakness and wasting followed by paralysis. Approximately 350,000 patients suffer from ALS worldwide, of which 50% die within 18 months of diagnosis. This condition has a high unmet medical need (source: Datamonitor). 

In addition to the 10 year market exclusivity, incentives relating to the designation include protocol assistance from the EMEA to maximise the chance of success in achieving market authorisation, fee reductions relating to the application for marketing authorisation, and eligibility for grants from the European Union and member states supporting research and development. 

Myogane™ has previously been granted Orphan Drug status and Fast Track Designation for the treatment of ALS by the United States Food and Drug Administration (FDA).

In July 2007, Phytopharm successfully completed a Phase Ib study with Myogane™, conducted in the UK under a clinical trial authorisation (CTA) from the Medicines and Healthcare Products Regulatory Agency (MHRA). The study used a new liquid formulation suitable for ALS patients and demonstrated a good safety, tolerability and pharmacokinetic profile following single oral doses escalated across groups of healthy adult subjects.

Commenting on today's announcement, Dr Daryl Rees, Chief Executive Officer of Phytopharm, said: "The European Orphan Medicinal Product Designation represents another important regulatory achievement for Myogane™ as a treatment for ALS. Orphan status will facilitate the most efficient and rapid clinical progress for this devastating condition. Myogane™ readily enters the central nervous system and has the potential to induce a person's own neurotrophic factor activity and alter disease progression. Our pre-clinical studies have been highly encouraging in both quality of life and survival parameters and we look forward to further clinical progress in ALS patients later this year, subject to additional grant funding."

-Ends-

Notes to Editors

Phytopharm plc

Phytopharm is a pharmaceutical development and functional food company. Our products are developed from medicinal plants, thereby reducing the development risk, cost and time to market. As a virtual company, Phytopharm's model is centred on a lean cash burn with all laboratory, manufacturing and clinical work out-sourced to specialists, while core competencies such as strategy and management are maintained in-house. Close collaboration with charitable organisations enhances our interaction with worldwide specialists and accelerates our development programmes increasing their value. 

Myogane™

Myogane™ is a novel non-peptide, orally bioavailable neurotrophic factor inducer that readily crosses the blood brain barrier. In pre-clinical studies, Myogane™ stimulates the release of neuronal growth factors, increases neurite outgrowth and protects against oxidative and glutamate neuronal damage. Myogane™ also reverses the decrease of neuronal growth factors, reverses oxidative and glutamate damage and reverses neuronal degeneration observed in motor neurones in vitro. When administered orally to pre-clinical models of ALS, Myogane™ delays the loss of muscle strength and extends survival time.

The neuroprotective and neurotrophic actions of Myogane™ suggest potential beneficial effects in other orphan neurodegenerative diseases including Friedrich's ataxia, progressive supranuclear palsy, Huntington's disease and multiple system atrophy.

Motor Neurone Disease

Amyotrophic lateral sclerosis (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 brainstem) and upper (cerebral cortex) motor neurones, which leads to severe muscle weakness and wasting, followed by paralysis and death, generally caused by respiratory failure. 

ALS is considered an orphan disease (i.e. the condition is rare) as it affects fewer than 200,000 in the US (US definition) and affects no more than 5 in 10,000 people (EU definition). Approximately 350,000 patients suffer from ALS worldwide, of which 50% die within 18 months of diagnosis. In recent years, there is evidence that the incidence of motor neurone disease is increasing although this may be due to more accurate testing and diagnosis (source Motor Neurone Disease Association). The financial cost to families of patients is exceedingly high, and it is estimated that in the advanced stage, care can cost an average of $200,000 per year (source: International Alliance of ALS Associations).

The precise causes of motor neurone degeneration in ALS patients remain unknown. Approximately 5-10% of cases appear to be of familial origin and possible mechanisms include loss of neurotrophic factors coupled with oxidative and glutamate mediated damage of nerve cells. Neurotrophic factors are essential for the survival and maintenance of nerve cells and provide protection against toxic insults, however as proteins, their utility as pharmacological treatments are limited. Riluzole (Rilutek™), a glutamate modulator, is the only agent indicated for the treatment of this condition and increases average survival by only a few months (source Datamonitor). There is an urgent need for the development of new approaches to this devastating condition and non-peptide orally bioavailable neurotrophic factor inducers that readily cross the blood brain barrier represent an important therapeutic approach.

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 release. The Company undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events of 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.

For further information about Phytopharm please see our website at http://www.phytopharm.com