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Drug intelligence / Profile preview
PXT 3003
- Development stage
- Phase 3
- Lead developer
- Pharnext
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
PXT 3003 is an oral fixed-dose combination therapy comprising [RS]-baclofen, naltrexone hydrochloride, and D-sorbitol. Developed by Pharnext using its proprietary Pleotherapy R&D platform, the drug is designed to treat Charcot-Marie-Tooth disease type 1A (CMT1A), a rare orphan neuromuscular disease caused by the duplication of the PMP22 gene. PXT 3003 works by synergistically downregulating the overexpression of the PMP22 protein, which is responsible for the degradation of the myelin sheath. The candidate has undergone multiple Phase III clinical trials, including the pivotal PREMIER study, and has received Orphan Drug Designation in both the U.S. and Europe. The combination aims to improve motor and sensory functions in patients by restoring myelin sheath integrity and axonal health.
- Brand names
- SYNGILITY
- Other names
- baclofen + naltrexone + sorbitolbaclofen/naltrexone/sorbitol
02
Targets
GABA-B (Gamma-aminobutyric acid receptor type B)MOR (Mu opioid receptor)CHRM3 (M3)
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