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Funded Studies

Development of a mGluR4 Therapeutic for Parkinson's Disease

Study Rationale: 
One of the major objectives for the development of new treatments for Parkinson’s disease is to find alternatives to the stimulation of the dopaminergic system through levodopa and dopamine agonists, as long-term use of these treatments are responsible for severe side effects. Stimulation of the glutamate receptor mGluR4, which bypasses the dopamine system, leads to functional improvement in pre-clinical models of PD. Prexton has developed a novel series of molecules (PAM) that increase mGluR4 activity and has selected a compound that can be tested in the most predictive model of clinical efficacy.

The study aims to validate that administration of Prexton’s mGluR4 PAM will provide symptomatic relief either as a stand-alone and/or as an add-on therapy to levodopa.

Study Design:
Prexton’s compound will be examined in pre-clinical models to assess anti-parkinsonian efficacy as well as levodopa-sparing potential and anti-dyskinesia activity.

Impact on Diagnosis/Treatment of Parkinson’s Disease:
The primary impact would be to offer a treatment to patients for fluctuating motor symptoms (ON/OFF). Then the benefits will be a significant optimization and reduction of the use of dopaminergic drugs and the possibility to be treated with more efficient and safer therapeutic strategies.

Next Steps for Development:
If the compound shows anti-parkinsonian efficacy in this study, it will then be progressed into pre-clinical toxicity studies to start clinical trials as soon as possible.

Final Outcome

This study tested Prexton's lead compound (PXT002331) in a pre-clinical Parkinson's model. The compound showed anti-parkinsonian efficacy both as a standalone treatment and in combination with levodopa. None of the treatments had a negative effect on cognitive performance. This potential treatment represents a levodopa sparking strategy and may reduce the occurrence of side effects induced by high doses of levodopa.

Based on this set of data, Prexton Therapeutics has decided to pursue pre-clinical development of PXT002331.

September 2015


  • Francois Conquet, PhD

    Geneva Switzerland

  • Delphine Charvin Ayad, PhD

    Geneva Switzerland

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