Pharnext SA, a biopharmaceutical company, has announced a timeline for the release of its PREMIER trial results, which evaluate the efficacy of PXT3003 in treating Charcot-Marie-Tooth disease Type 1A (CMT1A). The company expects to close the trial database on December 1, with initial results anticipated by December 8 and top-level data expected to be disclosed on December 11.
The global PREMIER study has been assessing PXT3003's effectiveness compared to a placebo among individuals with mild-to-moderate CMT1A. The trial used the Overall Neuropathy Limitations Scale (ONLS) to measure functional motor disability over fifteen months and included 387 subjects. Following this phase, participants have been part of the PREMIER-OLE since September 2022 for extended safety evaluation pending market launch, conditional upon FDA and EMA approval.
CMT1A is a condition that affects approximately 150,000 people across Europe and America. It leads to progressive muscle wasting and sensory impairments due to impaired peripheral nerve conduction and axonal loss caused by anomalies in Schwann cells associated with PMP22 gene duplication. Currently managed with physical therapy and aids like leg braces, PXT3003 has received orphan drug status, indicating its potential as a future therapeutic option.
Investors have been advised to be aware of possible fluctuations in Pharnext's share value following dilutive capital initiatives with Global Tech Opportunities 13. The company suggests cautious risk assessment before making financial commitments.
Following the announcement made today, Pharnext's stock saw a nearly 3% increase in value along with significant trading activity on the Euronext Growth Stock Exchange Paris where it is listed (ISIN code: FR001400JXB0). Pharnext continues to focus on its Pleotherapy™ R&D strategy aiming to develop treatments for neurodegenerative diseases that currently have limited medical solutions.
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