Pharnext's pleodrug PXT-864 demonstrates positive effects in Parkinson's disease

Published: Thursday 12 November 2015

acamprosate

baclofen

 SAS has announced the publication of positive preclinical results in Nature's Scientific Reports for its pleodrug PXT-864 in Parkinson's disease. After promising Phase 2 clinical data in Charcot-Marie-Tooth with PXT-3003 and preliminary positive clinical data in Alzheimer's disease with PXT-864, the data provide further validation of the company's pleotherapy R&D approach. It is based on a proprietary network pharmacology platform that allows the development of pleodrugs, synergistic low dose combinations of repositioned drugs with established safety profiles.

In the article titled, "Combination of acamprosate and baclofen as a promising therapeutic approach for Parkinson's disease," authors Rodolphe Hajj, et al., report that PXT-864, a combination of baclofen and acamprosate, improved multiple endpoints associated with the development and progression of Parkinson's disease. In cellular and in rodent relevant models of Parkinson's disease, the authors specifically found that PXT-864:

• Synergistically protected neuronal cells against oxidative stress in vitro, a hallmark of Parkinson's disease pathology
• Synergistically protected dopaminergic neurons in vitro in an induced toxicity model
• Protected motor function from 6-OHDA lesions in rats
• Protected substantia nigra pars compacta (SNc) dopaminergic neuronal cell bodies and striatal nerve terminals in vivo
• Restored symptomatically motor function in 6-OHDA pre-lesioned rats
• Did not negatively interact with L-Dopa

PXT-864 may represent an efficient and valuable strategy to slow or stop the progression of Parkinson's disease. In addition, PXT-864 could also improve symptoms of patients without inconvenience related to L-Dopa treatment.

Anthony Schapira, M.D., FMedSci., Head of the Department of Clinical Neuroscience at the UCL Institute of Neurology , London, said, "The data presented provide compelling evidence of PXT-864's potential in the treatment of Parkinson's disease. Today there are no treatments capable of altering the progressive course of the disease by slowing the destruction of dopaminergic neurons. I look forward to the clinical development of PXT-864 to see if these effects can be translated to patients with Parkinson's disease."

Ilya Chumakov, Ph.D., D.Sc., co-founder of Pharnext and scientific advisory board chairman, said, "These data validate and demonstrate the potential of Pharnext's pleotherapy approach. It builds upon the recently published positive results of PXT-864 in Alzheimer's disease and Phase 2 clinical data of lead pleodrug, PXT-3003, in Charcot-Marie-Tooth disease Type 1A (CMT 1A)."

Daniel Cohen, M.D., Ph.D., chairman, chief executive officer and co-founder of Pharnext, said, "The diseases we are targeting such as Alzheimer's, Parkinson's and CMT 1A with our lead pleodrugs are exceedingly difficult for patients in that they are all progressive in nature with worsening symptoms over time. Patients have little to no acceptable treatment options and medical unmet needs remain high. As such, the published results of this multitarget approach are very encouraging for the future."

http://www.medicalnewstoday.com/releases/302538.php?tw