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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Good News for CMT1A Patients – PXT3003
Three major regulatory agencies in the United Kingdom, Europe and US have recognized PXT3003 as a lead drug candidate to treat CMT1A.
Pharnext Announces PXT3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A has Been Granted Promising Innovative Medicine (PIM) Designation by UK Medicines and Healthcare Products Regulatory Agency
United Kingdom’s Medicine and Healthcare products Regulatory Agency (MHRA) has granted Promising Innovative Medicine (PIM) designation to its lead drug candidate, PXT3003, for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A) in patients 16 years and older.
Pharnext raises € 7.7 million in a private placement
Pharnext SA (FR0011191287 – ALPHA) (the “Company”), a biopharmaceutical company pioneering a new approach to developing innovative drug combinations based on its PLEOTHERAPY artificial intelligence platform harnessing big genomics data and network pharmacology, today announced a capital raise of circa € 7.7 million by way of issuance of 1,799,061 new ordinary shares (the “New Shares”) with one warrant attached each (together with the New Shares, the “ABSA”).
Pharnext’s lead PLEODRUG © for treatment of CMT1
Pharnext completed patient enrollment for the international Phase 3 clinical trial of PXT3003, Pharnext’s lead PLEODRUG ©, for treatment of CMT1A.
Hot Off the Press – Potential Treatment for CMT1A
Two recent publications from Pharnext describe a novel synergistic combination of 3 drugs (baclofen, naltrexone and sorbitol) and its effect on CMT1A both in the lab and in a phase II clinical trial. These 3 drugs already approved but for unrelated conditions, are combined at new optimal lower doses and under a new formulation. This novel potential therapeutic is called PXT-3003.
Pharnext Announces Pleotherapy Proof of Concept in Charcot-Marie-Tooth Disease Type 1A
PARIS, December 18th, 2014 – Pharnext SAS today announced the proof of concept of its pleotherapy research and development approach based on a proprietary network pharmacology platform that identifies synergic combinations of drugs already approved for other diseases. Indeed, Pharnext’s lead pleodrug, PXT-3003, has shown positive results both in preclinical and Phase 2 clinical studies published today in the Orphanet Journal of Rare Diseases.
Building Awareness on CMT and Supporting the Patient Community – HNF Announces Support from Pharnext
HNFpharnext recently entered into a partnership with the French biopharmaceutical company, Pharnext, to help raise awareness of Charcot-Marie-Tooth (CMT) disease and support the CMT patient community through several initiatives. Building awareness is key! Pharnext’s support will assist HNF in distributing HNF’s CMT Update quarterly newsletter, enhancing the Global Registry for Inherited Neuropathies (GRIN), setting up activities for CMT September Awareness Month in the US and strengthening the CMT Inspire Community.
Targeting PMP22 in CMT1A Patients with Gene Duplication
In previous collaborative work with a group of investigators including Dr Rolf Renne from the University of Florida, Dr. Alex Murashov from East Carolina University and Dr. Lynn Hudson from the NIH-NINDS, we validated a microRNA known as miR29a as a reagent that corrected the expression level of PMP22 in rodent Schwann cells.