Pharnext's Pleodrug PXT-864 Demonstrates positive Effects in Parkinson's Disease Preclinical Data Published in Nature's Scientific Reports

In Cellular and in Rodent Models of Parkinson's Disease, PXT-864 Protects Dopaminergic Neurons and Restores Motor Function

Preclinical Data Provide Further Validation of Pharnext's Pleotherapy Approach

PARIS, Nov. 10, 2015 /PRNewswire/ -- Pharnext SAS [] today 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.

The paper can be found here: []


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."

About Parkinson's disease

Parkinson's disease is a chronic neurodegenerative disease characterized by progressive loss of dopaminergic neurons in the brain, causing motor disorders including bradykinesia, rigidity, tremors and postural instability. Worsening Parkinson's disease symptoms can lead to incidents that result in death. It affects around 6 million people in the world. Current treatments (L-Dopa, monoamine oxidase inhibitors and dopaminergic agonists) aim at replacing dopamine deficiency. They are only symptomatic, do not address neuronal loss and are often associated with adverse effects (such as abnormal involuntary movements).

About Pharnext

Pharnext is an advanced clinical stage biopharmaceutical company developing novel therapeutics that simultaneously target multiple key disease pathways for severe orphan and common neurological disorders. The proprietary research and development platform of Pharnext, based on network pharmacology, is applicable to a broad spectrum of diseases and allows the rapid development of "pleodrugs", synergistic combinations of repositioned drugs with established safety profiles. The company's two lead pleodrugs are PXT-3003 for the treatment of orphan disease Charcot Marie Tooth type 1A (Phase 2 clinical trial completed) and PXT-864 for Alzheimer's disease (Phase 2 clinical trial ongoing) and other neurologic indications (including Parkinson's disease and amyotrophic lateral sclerosis).

For further information, visit []

Pharnext Contact
Rodolphe Hajj
Experimental Biology Director []

Media Contacts
Tony Russo, Ph.D.
Matt Middleman, M.D.
Russo Partners
T: 212-845-4251
T: 212-845-4272 [] []


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