CMTA Strategy To Accelerate Research (CMTA-STAR) Alliance Partner NMD Pharma A/S (NMD) shared new data from its Phase 2a SYNAPSE-CMT clinical trial evaluating its investigational drug ignaseclant during a late-breaking presentation on March 10th at the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.
According to NMD Pharma, the study evaluated twice-daily oral ignaseclant in adults with genetically confirmed CMT1 or CMT2. The trial enrolled 81 participants across clinical sites in the United States and Europe.
NMD Pharma reported that although the study did not demonstrate a treatment difference on the primary outcome measure, the 6-minute walk test (6MWT) at 21 days, improvements were observed across several measures of muscle strength, dexterity, and functional performance. The company reported improvements in handgrip strength and fine motor skills, including performance on the 9-hole peg test, along with changes in patient-reported outcomes such as the CMT-Health Index (CMT-HI).
“What is particularly encouraging about this data is the consistency of the effects with ignaseclant treatment observed across multiple measures of muscle strength and function in such a short study,” W. David Arnold, M.D., Executive Director of the NextGen Precision Health Initiative and Professor of Physical Medicine and Rehabilitation at the University of Missouri School of Medicine said in a company press release. “Taken together, the totality of the data provides a strong rationale to further investigate ignaseclant as a potential new treatment approach for people living with Charcot-Marie-Tooth disease.”
The company also reported that ignaseclant was generally well tolerated during the study, with no serious adverse events.
During the presentation at the MDA Clinical & Scientific Conference, study principal investigator W. David Arnold, MD, said the results showed consistent effects across multiple measures of muscle strength and function over the short study period, supporting continued evaluation of ignaseclant as a potential therapeutic approach for people living with CMT.
Patients as Partners in Research Accelerated SYNAPSE-CMT Trial
CMTA supported awareness, outreach, and recruitment for the SYNAPSE-CMT trial through the Patients as Partners in Research program, helping connect members of the CMT community with this clinical research opportunity. These efforts contributed to the trial completing approximately five months ahead of schedule and reflect how the community continues to shape how CMT research is designed and carried out.
Participation from individuals and families living with CMT continues to play an essential role in advancing research and supporting the development of potential new therapies for the disease.
Read NMD’s full announcement here.
