The United States Food & Drug Administration (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.
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.