motor neurone disease (ALS) 

About

Amyotrophic lateral sclerosis (ALS) is also known as Lou Gehrig’s disease in the US, Maladie de Charcot in France and Motor neuron Disease (MND) in the UK.

ALS is a progressive neuromuscular degenerative disease with strong brain stem and spinal cord involvement where motoneurons are located.

The disease is characterised by the progressive degeneration of motor nerve cells in the central nervous system.

This degeneration can affect: 1) Upper motoneurons in the brain stem and 2) Lower motoneurons in the spinal cord.

These are very severe conditions causing a dramatic loss of quality of life and most patients with the disease die between 2-5 years after diagnosis depending on first pathological involvement of upper (bulbar) or lower motoneurons.

ALS is an orphan drug status indication. Read more about orphan drug status...

Prevalence and Market Size

• At any one time 15,000 – 30,000 ALS patients live in the US.
• 2.2 - 2.7 per 100,000 person-years is the incidence rate in Europe.
• Approximately 400,000 prevalent cases worldwide.
• The global market size (2018) was approximately $540 million USD per year and is rapidly growing.
• Novel therapies with potentially significant effects on overall life-span extension during disease course will result in multibillion dollar market size.

Treatments Available

There are no highly effective treatments for ALS.

So far, the only approved drugs are riluzole and edaravone, which prolong life-span on average by a couple of months or possibly longer when taken very early during disease process (edaravone).

The Science of ALS

Causalities or strong hypotheses for the susceptibility to develop Motor neurone Disease are mainly unknown and only 2-10 % of the patient population has inherited familial ALS with mutations/polymorphisms within superoxide dismutase (SOD-1), TDP-43 gene regions or within C9orf72 related DNA regions.

The 90 % patient group with unfamilial ALS have no current known genetic risk factors.

NRP effects:

• NRP2945 shows a broad efficacious dosage range in the preclinical SOD-1 G93A gain-of-function mouse model when administered late during the disease profile (day 90-95).
• NRPs dosages of ng/kg to μg/kg range are effective in the SOD-1 mouse model.
• Beneficial combinational effect of NRP2945 in the presence of Riluzole.

NRPs are likely to be eligible for Orphan Drug Status from the FDA which would significantly shorten the clinical trial time and associated costs.