Orphan Drug Status Granted
Phytopharm PLC
26 July 2004
26 July 2004
Phytopharm plc
Orphan drug designation granted by the FDA for drug candidate to treat ALS
Phytopharm plc (PYM: London Stock Exchange) ('Phytopharm') announces today that
the United States Food and Drug Administration (FDA) has granted orphan drug
designation to its drug candidate PYM50018 for the treatment of amyotrophic
lateral sclerosis (ALS, also known as Lou Gehrig's disease). Phytopharm has
recently registered the trademark Myogane(TM) as a name for this product.
Orphan drug designation can be granted by the FDA for treatments that might
provide significant benefit to patients with serious, life-threatening diseases
that affect less than 200,000 persons in the United States. The Orphan Drug Act
was created by the United States Congress to provide assistance and incentives
for sponsors to develop drugs judged to be of potential benefit for a qualifying
disease. Orphan drug designation qualifies a product for possible financial
incentives, including seven years of marketing exclusivity upon FDA approval,
and the potential of an expedited approval. In addition to market exclusivity,
orphan drug status provides possible tax incentives for a company's investment
in U.S. clinical research.
ALS, the most common motor neurone disease, results from progressive
degeneration of both upper and lower motor neurones and is usually fatal within
five years. PYM50018 is a patented, orally active, neuroprotective and
neuroregenerative compound from the P59 programme. In pre-clinical models,
PYM50018 has been observed to protect against neuronal damage, increase neurite
outgrowth, reverse oxidative damage and reverse neuronal apoptosis in vitro.
When administered orally to a transgenic pre-clinical model of ALS, PYM50018
delays the loss of muscle strength and extends survival time.
In April 2004 Phytopharm announced the successful completion of a phase I
clinical study to evaluate the safety, tolerability and pharmacokinetic profile
of PYM50018. This residential clinical study was conducted under an
investigational new drug (IND) application filed with the FDA. The results of
this clinical study will support the conduct of a repeat dose phase Ib clinical
study to evaluate the safety, tolerability and pharmacokinetic profile of
PYM50018 in healthy volunteers.
Commenting on today's announcement, Richard Dixey, Chief Executive of
Phytopharm, said: 'We are pleased that upon a review of our pre-clinical data
and rationale for developing PYM50018 (Myogane(TM)) as a potential treatment of
ALS, the FDA found cause to grant orphan drug designation at this stage of
development. We will continue to work with them to advance this product to
clinical evaluation in ALS patients as quickly as possible.'
-ENDS-
Enquiries:
Phytopharm plc
Dr Richard Dixey, Chief Executive Tel: 01480 437697
Mobile: 07867 782000
Financial Dynamics
David Yates / Ben Atwell Tel: 0207 831 3113
NOTES TO EDITORS
Phytopharm plc
Phytopharm is a leading company in the development of Botanical pharmaceuticals.
These plant-based medicines, manufactured to pharmaceutical standards, can be
clinically evaluated in chronic and poorly understood diseases. Where novel
modes of action are discovered, such research can form the basis for drug
discovery platforms, which enable the development of new medicines and the
isolation of single chemical entities of clinical importance. Phytopharm has
four drug discovery platforms in full development, for metabolic disease,
neurodegeneration, inflammation and dermatology.
Phytopharm is developing nine programmes based on its four drug discovery
platforms alongside a number of other projects in early evaluation.
ALS is a fatal neurodegenerative disease that most commonly strikes people
between 40 and 60 years of age. The underlying cause of ALS is unknown,
although approximately 5-10% of cases appear to be of familial origin. It is
characterized 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. Phytopharm has developed a large group of patented compounds whose
properties provide a platform for the development of novel therapeutic
approaches for neurodegenerative disorders. PYM50018 has neuroprotective and
neuroregenerative effects demonstrated using various pre-clinical models that
make it a promising treatment for ALS.
For the families of ALS patients, the burden of providing supportive care is
exceedingly high and it is estimated that, in the advanced stage of the disease,
supportive care can cost an average of $200,000 per year (source: International
Alliance of ALS Associations). Treatment with the only drug currently indicated
for ALS typically increases the average survival time by only three months
(source Datamonitor). Thus, there is an urgent need for the development of new
and more effective therapies for this devastating condition.
More information concerning Phytopharm's activities can be found on its web site
at http://www.phytopharm.com
This information is provided by RNS
The company news service from the London Stock Exchange