Pharnext Pharmaceuticals, a clinical-stage biopharmaceutical company, partnered with HNF in 2012 to support the development of PXT3003.
PXT3003 is a novel combination drug of baclofen, naltrexone, and sorbitol designed to downregulate the overexpression of PMP22, the protein impacted in CMT1A. This leads to the improvement of neuronal signaling in dysfunctional peripheral nerves.
Results of their pivotal phase III are expected by Q4, 2023.
“GRIN data has been essential in clinical trial protocol design, site selection, and efficient patient recruitment for the pivotal Phase III clinical study of our lead asset PXT3003, the PREMIER trial, in development for the most common type of CMT, CMT1A. In addition, the robust data collected in GRIN, together with the ongoing CMT&Me digital survey, will enable a better understanding of the burden of disease, comorbidities, and critical patient demographics as we move forward to a potential commercialization of PXT3003 if approved by the FDA. Topline data of the PREMIER trial are expected in the last quarter of 2023.” Xavier Paoli M.Sc. Pharnext, Chief Operating Officer
Learn more about HNF’s history with Pharnext.
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Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A
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