We are sharing an update from NMD Pharma regarding topline results from their Phase 2a clinical study called SYNAPSE-CMT. This clinical study explored whether the new, investigational drug ignaseclant (formerly NMD670), could provide benefit adults with Charcot-Marie-Tooth disease (CMT) type 1 and type 2. The CMTRF is optimistic about what ignaseclant and the results of the clinical trail could mean for the CMT patient community.
NMD Pharma reported that ignaseclant was generally well tolerated in the clinical trial and that encouraging trends were observed in measures of muscle function and strength compared with placebo. As a Phase 2a trial, SYNAPSE-CMT was designed to assess safety, tolerability, and exploratory outcomes, and the results are preliminary and investigational.
This clinical study was informed by prior work from NMD Pharma, including the ESTABLISH observational study, which identified neuromuscular junction transmission deficits in people with CMT and helped establish the scientific rationale for evaluating ignaseclant in patients.
NMD Pharma also shared a letter to the CMT community expressing their appreciation for the patients and caregivers whose participation made this study possible, enabling completion ahead of schedule, and reaffirming their commitment to transparency and thoughtful, data-driven development.
Additional studies will be required to further evaluate the safety and potential clinical benefit of ignaseclant. Please see the links below for NMD Pharma’s full topline results summary, a letter to the patient community, and link to register for a free CMTRF-hosted live discussion with Daniel Brennan, NMD Pharma’s SVP of Corporate and Commercial Strategy.
NMD Pharma Topline Study Results
Join us for a webinar next Thursday to hear directly from the NMD team on this important study. This is a webinar you should not miss.
Register: https://us02web.zoom.us/webinar/register/WN_W2epqkaDRxu38L4bYETSpw
