Policy & Regulation
NMD Pharma gets FDA approval for Phase 2 trial of NMD670 in Charcot-Marie-Tooth disease
18 June 2024 -

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.