pp-248 Tablets
Dossier type
EU CTD
Dossier status
Under development
Country of origin
Switzerland
GMP approvals
EU GMP
Comments
Indication: Charcot-Marie-Tooth disease (CMT1, CMT2) / Clinical stage: Phase 2a SYNAPSE-CMT (80 patients) and Phase 2 SMA type 3 ongoing / Modality: Small molecule skeletal muscle ClC-1 chloride channel inhibitor / Mechanism & Target: CLC-1 channel inhibition amplifying neuromuscular transmission / Route & form: Oral, twice-daily dosing / Differentiation: First-in-class CLC-1 inhibitor; dual mechanism enhancing muscle excitability; FDA Orphan Drug Designation for CMT
Manufacturer #39447
Clinical-stage biotech focused on neuromuscular therapeutics targeting chloride ion channel modulation. Develops first-in-class inhibitors for rare inherited and acquired neuromuscular conditions. Advances Phase 2 programs across multiple neuromuscular disease indications.
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