CMTA Strategy To Accelerate Research (CMTA-STAR) Alliance Partner NMD Pharma A/S (NMD) has announced topline results from its Phase 2a SYNAPSE-CMT clinical trial for its investigational drug ignaseclant (formerly NMD670). According to the company, ignaseclant is a skeletal muscle-specific chloride ion channel 1 (CIC-1) inhibitor designed to improve muscle function in people living with CMT.
NMD Pharma shared that the trial showed consistent improvements across multiple measures of muscle strength, motor function, and patient-reported outcomes. Improvements were most pronounced in upper-limb strength and fine motor skills, including handgrip strength and dexterity, which affect day-to-day activities for people living with CMT. The company also reported that ignaseclant was generally well-tolerated during the study.
The SYNAPSE-CMT trial enrolled 81 adults with genetically confirmed CMT1 or CMT2 across clinical sites in the United States and Europe. The first patient received the first dose from Yessar Hussain, MD, the CMTA Center of Excellence clinic director in Austin, TX. NMD plans to share additional information about the trial at an upcoming conference.
In their announcement, Thomas Holm Pedersen, PhD, NMD Pharma CEO, said the company observed consistent improvements in muscle strength and function over a relatively short timeframe, with findings across objective and patient-reported measures aligning with ignaseclant’s proposed mechanism of action. He added that these results support continued clinical development of ignaseclant in CMT.
We’re pleased to see NMD report on its findings from this study, particularly the improvements in muscle strength and fine motor function that affect daily life for people living with CMT. This work reflects the commitment of the individuals and families who participated in the trial, and we’re grateful to the CMT community for making progress like this possible. CMTA congratulates NMD Pharma on sharing this update and thanks the individuals and families who participated in this study. Through CMTA-STAR and Patients as Partners in Research, CMTA remains committed to supporting rigorous clinical research and keeping the CMT community informed as research efforts continue.
Sue Bruhn, PhD, CMTA CEO
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 nearly six months ahead of schedule and reflect how the community continues to shape how clinical research is designed and conducted in CMT. Participation from people living with CMT is essential to advancing research and bringing potential treatments closer to reality.
Read a letter from NMD Pharma to the CMT Community
Read NMD Pharma’s Full Announcement
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