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Myogane™ 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 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. 

Mode of action

Myogane^TM is a novel non-peptide, orally bioavailable neurotrophic factor inducer that readily crosses the blood-brain barrier. Myogane^TM induces the body's own production of proteins known as neurotrophic factors. In particular, one of these factors known as brain derived neurotrophic factor (BDNF) has been shown to be particularly effective in re-growing damaged motor neurones. Myogane^TM readily crosses the blood-brain barrier and in preclinical models has been shown to stimulate the release of BDNF in the brain and protect against neuronal damage and when administered orally to preclinical models of ALS, delays the loss of muscle strength and extends survival time.

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

Progress to date

It is our intention to carry out a proof of concept clinical study provided adequate funding was available from non-dilutive sources. Several charitable grants are under discussion and we continue to have constructive dialogue with The Motor Neurone Disease Association.