Pharnext announces the inclusion of the first patient in the open label extension of the pivotal Phase III study of PXT3003 in Charcot-Marie-Tooth disease type 1A, the PREMIER trial – 09/12/2022 at 08: 30


PARIS, France, September 12, 2022 at 8:30 a.m. (CET) – Pharnext SA (FR0011191287 – ALPHA) (“the Company”), an advanced clinical-stage biopharmaceutical company developing new therapies for neurodegenerative diseases with no satisfactory therapeutic solution, announces today Today Enrollment of the First Patient in the Open Label Extension Study of the PREMIER (‘PREMIER-OLE’ – Open Label Extension) Trial of PXT3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A) in the United States. This patient, who completed the double-blind, placebo-controlled PREMIER study, was enrolled in May 2021. All patients who completed the PREMIER trial will be eligible for inclusion in the PREMIER-OLE study and will receive the dose (DE) of PXT3003 until its commercial availability, if it is finally approved in the United States by the FDA and in Europe by the EMA. PXT3003 is the Company’s most advanced program for the treatment of CMT1A, a disabling disease for which there is currently no approved therapy.



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