Denmark-based clinical-stage biotech company NMD Pharma A/S announced on Tuesday that it has secured US Food and Drug Administration (FDA) clearance for its Investigational New Drug (IND) application to initiate a Phase 2 trial (SYNAPSE-CMT) of NMD670, a first-in-class muscle-targeted therapy, in patients with Charcot-Marie-Tooth (CMT) type 1 and 2.
The randomised, double-blind, placebo-controlled trial will assess the safety, efficacy and tolerability of NMD670 in around 80 adult patients across the United States and Europe. Expected to begin enrolment soon, the 21-day trial will evaluate endpoints including 6-minute walk test and timed-up-and-go test.
This follows NMD Pharma's announcement of positive results from the ESTABLISH study in June 2023, highlighting neuromuscular junction dysfunction as a characteristic in CMT patients.
SYNAPSE-CMT joins two ongoing Phase 2 trials of NMD670 for spinal muscular atrophy (SMA) type 3 and generalised myasthenia gravis, solidifying NMD Pharma's focus on rare neuromuscular diseases.
Sanofi receives FDA orphan drug designation for riliprubart in transplant rejection
Hoth Therapeutics reports positive interim results for HT-001 topical therapy
Breckenridge Pharmaceutical's generic for Ablysinol granted final FDA approval
Hemab Therapeutics presents bleeding disorder clinical and preclinical data at ISTH 2025 Congress
Cumberland Pharmaceuticals reports ifetroban Phase 2 DMD heart disease trial results
Sarclisa recommended for EU approval in newly diagnosed transplant-eligible multiple myeloma
FDA approves Dupixent as first targeted therapy for rare skin disease bullous pemphigoid