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  PHARNEXT company press release from 14/03/2023

  14/03/2023 - 08:30

Pharnext supports and participates in two congresses dedicated to Charcot-Marie-Tooth diseases in Marseille, France

  • Thursday, March 17th: "Innovative therapies in CMT", Thematic Day organized by FILNEMUS, the Rare Diseases Network dedicated to neuromuscular diseases.
  • Friday, March 18th: 32nd annual meeting of CMT-France, the French CMT patient advocacy group.

PARIS, France, March 14th, 2023, 08:30 am CET – Pharnext SCA (FR001400BV89 - ALPHA) (the “Company”), an advanced late-clinical stage biopharmaceutical company developing novel therapeutics for neurodegenerative diseases with high unmet medical need, today announces its support to the organization and participation to both events dedicated to Charcot-Marie-Tooth (CMT) diseases that will be held in Marseille, France: The FILNEMUS Thematic Day on "Innovative therapies in CMT" on Thursday, March 17th, 2023 followed by the annual congress of CMT-France, the French CMT patient advocacy group, on March 18th, 2023.

The FILNEMUS Network's Thematic Days aim at bringing together all healthcare professionals involved in diagnosis, care and research, as well as patient advocacy groups involved in rare diseases. This year, members of the FILNEMUS network dedicated to CMT will discuss innovative therapies.

This CMT thematic day will be held on March 17th, 2023 at Parc Chanot in Marseille, France. More information on this medical education event is available at

The annual Congress of CMT-France, the French CMT patient advocacy group is an informative meeting that brings together all those impacted by Charcot-Marie-Tooth diseases around medical specialists.

It will be held on March 18th, 2023 at Parc Chanot in Marseille. More information on this annual event is available on

These two congresses are intended for patients and healthcare professionals involved in Charcot-Marie-Tooth diseases management, including the most common subtype 1A (CMT1A), an indication in which Pharnext's lead candidate, PXT3003, is in pivotal Phase III clinical development. These meetings will provide an opportunity to combine the clinical updates, the latest scientific information and CMT patients testimonies.

In order to strengthen its ties with patients and the medical community involved in the management of CMT1A in France, Pharnext has decided to support and participate in these two events.

About Pharnext

Pharnext is an advanced clinical-stage biopharmaceutical company developing novel therapies for neurodegenerative diseases currently without satisfactory therapeutic solutions. Pharnext has a first-in-class drug candidate, PXT3003, in development for Charcot-Marie-Tooth disease type 1A (CMT1A), a rare, debilitating, inherited peripheral neuropathy. PXT3003 benefits from orphan drug status in Europe and the United States. In 2018, PXT3003 completed a Phase III clinical trial, the PLEO-CMT trial, with encouraging topline results. This trial was followed by an open-label extension study, the PLEO-CMT-FU trial, with 120 patients continuing treatment with PXT3003. Long-term data suggest a sustained benefit, safety, and efficacy, after 5 years of total trial time. An international pivotal Phase III study of PXT3003, the PREMIER trial, is currently ongoing with 387 CMT1A patients enrolled. PREMIER topline results are expected in Q4 2023. PXT3003 originated from the Pleotherapy™ R&D approach. Pharnext draws the attention of investors to the financial and other risk factors detailed in its financial reports. More information can be found at

Pharnext is listed on the Euronext Growth market in Paris (ISIN code: FR001400BV89).


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  Original Source: PHARNEXT