SYNAPSE-CMT trial: NMD Pharma announces preliminary results

The SYNAPSE-CMT Phase IIa clinical trial evaluated NMD670 (ignaseclant) administered orally twice daily, compared to placeboLe placebo est un produit dont la présentation est identique à celle d’un médicament, mais qui ne contient pas de principe actif. Dans un essai clinique, un placebo est utilisé pour mesurer l’action réelle du médicament testé, en comparant les effets du médicament testé contenant le principe actif et ceux du placebo., in 81 adults with Charcot-Marie-Tooth disease (CMT1 and CMT2) over 21 days in five countries, including France. Initial results from the trial were shared in a press release:

• the primary endpoint, corresponding to improvement in distance walked in the 6-minute walk test, was not achieved by the treated group compared to placebo.
• However, signs of efficacy were observed for several secondary endpoints, including grip strength, fine motor skills of the hands and certain clinical scores (CMT-FOM and CMT-HI).

The laboratory considers that the data support the continued development of the product in a longer trial involving a larger group of patients.

NMD Pharma announces topline results from its Phase 2a study of ignaseclant in Charcot-Marie-Tooth disease Types 1 and 2. NMD pharma. Communiqué de presse du 3 février 2026