Pharnext's CMT1A Drug PXT3003 Fails to Meet Primary Endpoint in Phase 3 PREMIER Trial
• French biotech Pharnext's lead drug PXT3003 for Charcot-Marie-Tooth disease type 1A failed to demonstrate superiority over placebo in Phase 3 PREMIER trial due to unexpected placebo response.
• Despite the setback, Pharnext remains optimistic, citing 10 years of accumulated data and an ongoing Phase 3 trial with Chinese partner Tasly that could support regulatory submission.
• PXT3003, a combination of baclofen, naltrexone, and sorbitol, maintained its favorable safety profile and showed no disease deterioration in treated patients during the study period.
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