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Clinical Trials
9
8 active
Approvals
0
Total approvals
Agencies
Regulatory bodies
Founded
N/A
Active, not recruiting
8
88.9%
Completed
1
11.1%
No approval data available
- 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.