Parkinson’s disease: Prexton Therapeutics completes phase 1 clinical trial


Study shows mGluR4 positive allosteric modulator is safe and well tolerated at doses well above those that produce robust effects in Parkinson’s disease animal models

Geneva, Switzerland, September 19, 2016 - Prexton Therapeutics, a biopharmaceutical company developing novel therapeutics for the treatment of CNS conditions, today announces the successful completion of the phase 1 clinical trial of PXT002331, their lead compound. A total of 64 healthy volunteers were enrolled to evaluate several doses of PXT002331, which has proved safe and well tolerated. A phase 2 trial for Parkinson’s disease patients is due in the first half of 2017.

This study was the first in man clinical trial for an mGluR4 positive allosteric modulator. The randomized, double-blind, placebo controlled single and multiple ascending dose studies were designed to assess safety and tolerability of PXT002331 dosed orally. It was completed on time and showed that PXT002331 is safe and well tolerated at doses well above those that produce robust effects in Parkinson’s disease animal models.

"We are very happy with the completion of the first clinical trial with our lead compound PXT002331, which is the first mGluR4 PAM ever to enter a clinical phase. This is an important step for Prexton,” said Francois Conquet, CEO of Prexton Therapeutics. “This achievement ensures Prexton is in a solid position for a successful clinical development of PXT002331 as a novel treatment for Parkinson’s disease and we look forward to moving in that direction.”

Prexton’s innovative approach in the treatment of Parkinson’s is unique as it stimulates a compensatory neuronal system that is not impacted by the disease. Competitors in this indication mostly target the dopaminergic system, which does not address all symptoms and is accompanied by a number of adverse effects. Prexton’s compound activates a specific target of the glutamatergic system, with the goal of providing a robust therapeutic effect without these adverse events. Prexton’s first in class treatment has the potential to offer a better benefit/risk ratio for Parkinson’s patients than existing therapies.

The company aims to develop a group of first-in-class molecules that target the metabotropic Glutamate Receptor 4 (mGluR4), a protein belonging to the metabotropic glutamate receptor family. Preclinical data shows compelling evidence of the efficacy of Prexton’s molecule. It has the potential to alleviate motor complications by modulating glutamate over activity in the central nervous system of Parkinson patients.

A phase 2 clinical trial in Parkinson’s disease patients will take place in the first half of 2017, with the aim of demonstrating the effectiveness of the compound.

About Parkinson’s disease
Parkinson's disease is a chronic and progressive neurological disorder characterized by a number of symptoms including tremors, limb stiffness, slowness of movement and difficulties with posture and balance. Global figures show that 10 million people are affected by Parkinson’s disease with 200,000 being diagnosed each year. The incidence of the disease is expected to rise as the average age of the population increases. Today, the worldwide market for Parkinson’s disease should be around $3.1bn (€2.75bn) in 2016 (Global Business Insight, 2015. It is dominated by matured dopaminergic treatments, which frequently induce negative side effects. There is an overall consensus in the field supporting the development of more efficient approaches, while limiting or even abolishing the occurrence of adverse effects.

About Prexton Therapeutics
Prexton Therapeutics is a Swiss-based biopharmaceutical company, founded in 2012 by Francois Conquet and M Ventures, the corporate venture capital fund of Merck KGaA, Darmstadt, Germany. Prexton Therapeutics applies a new scientific approach that fully integrates molecular, behavioral and chemistry technologies to address Parkinson’s disease and other brain disorders. Prexton Therapeutics uses its powerful discovery platform to target specific novel compounds focused on the treatment of Parkinson’s disease.