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Preclinical Results for Motoneurone Disease

In the three independent trials conducted to date using the G93A SOD-1 mouse mutant model, NRP's lead compound has shown remarkable survival time-enhancing results.

The therapeutic model was used where the test drug is applied at the start of visible pathological motor symptoms.

Subsequent daily peripheral injections of NRPs increased the overall survival time of the mice from disease onset to death on average from 37-60%.

In comparison, the only existing approved drug, Riluzole, normally achieves survival increase rates of only maximal 30% in this particular model.

One of the studies was conducted by Professor John Crow at the J. Thomas May Center for Amyotrophic Lateral Sclerosis Research and Translational Medicine.

Prof. Crow has dedicated his research career to find effective therapies and the cause of ALS, has indicated that, in a subset of mSOD1 mice treated at symptom onset, the NRPs have broken through a “wall” in terms of the survival time after disease onset.

The best known gene therapy-derived therapeutic treatment achieved 75% increase in survival time.  This therapeutic effect derives from  a small interfering RNA (siRNA) molecule directed specifically against the mutated form of the SOD-1 messenger RNA and is silencing gene transcription of this particular pathological gene.

However, in our view this gene therapy approach is not viable for human drug development because the genetic fingerprints of Motoneurone Disease sufferers are too diverse to develop an efficiently targeted gene therapy drug.

Therefore, to our knowledge, our NRP drug development effort currently represents one of the best pre-clinical drugs available.

About Motoneurone Disease

Amyotrophic lateral sclerosis (ALS) is also known as:active healthy neurone

  1. Lou Gehrig’s disease in the US
  2. Maladie de Charcot in France
  3. Motor Neuron Disease (MND) in the UK

ALS is a progressive neuromuscular degenerative disease. 

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

This degeneration can affect:

  1. Upper motor neurons in the brain
  2. Lower motor neurons 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.  

Incidence Rate and Market Size

  • CNSMotorneurone Disease (ALS) is a rare disease with an incidence rate (new cases) of 2 to 2.5 in 100,000 persons in Australia with a prevalence rate of 1 to 15,000 (people living with ALS).
  • The statistics in the US show a mortality rate of 1.7 per 100,000 people in 1998, with a trend showing the incidence rate rising. 
  • The market size is approximately $150 million US per year.

Treatments Available

  • There are no highly effective treatments for ALS.
  • So far, the only approved drug is Riluzole, which prolongs life span on average by only 2-3 months.  
  • Riluzole generated global revenue of over $153 million US in 2006. 

Science of ALS & NRPs Benefits

Science behind ALSCausalities or strong hypotheses for the susceptibility to develop Motoneurone Disease are mainly unknown and only 2% of the patient population has inherited familial ALS with mutations/polymorphisms within their superoxide dismutase (SOD-1) gene region.

This enzyme is one of the major anti-oxidant players in the human body. The 98% patient group with unfamilial ALS have no current known genetic risk factors.

Animal Models

  • The best validated animal model mimicking familial ALS is the G93A gain of function SOD-1 mouse mutant model.

The only FDA-approved drug Riluzole shows significant survival-promoting effects in this mouse model.

New drug approval applications are looking for combinational effects of new drugs showing synergistic effects compared to Riluzole alone.

For Motoneurone Disease:Microscope

  • NRPs dosages within a range of factor 100 apart are efficacious in this model.
  • NRPs dosages of ng/kg to μg/kg range are effective in the SOD-1 mouse model
  • Beneficial combinational effect of NRPs 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.
  • Read more about Orphan Drug Status