The CMT community took action and answered the call, helping achieve an important research milestone as CMTA-STAR Alliance Partner NMD Pharma A/S announced reaching full patient enrollment in its Phase 2 SYNAPSE-CMT clinical trial.
The study reached its goal ahead of schedule, enrolling 81 participants with CMT types 1 and 2 across sites in the United States and Europe.
The SYNAPSE-CMT trial is evaluating NMD Pharma’s investigational drug, NMD670, which the company says is designed to target the chloride ion channel (ClC-1) found only in skeletal muscle. This biological pathway is not specific to any single gene, meaning the approach could have potential benefit across many CMT subtypes.
“Enrollment finished about five months ahead of plan,” said Daniel Brennan, NMD Pharma Senior Vice President, “which literally shaves off five months of overall development time to get this drug to those who need it, if results confirm safety and meaningful benefit to patients.”
CMTA supported awareness, outreach, and participant recruitment for the SYNAPSE-CMT study through its Patients as Partners in Research program, connecting community members to this clinical trial opportunity. This effort helped accelerate enrollment and demonstrates the impact patients have on CMT research progress.
“This milestone belongs to the CMT community,” said Sue Bruhn, PhD, CMTA CEO. “It shows what’s possible when participants, families, and researchers move forward together. We’re grateful to NMD Pharma for their continued collaboration and to everyone whose commitment made this progress possible.”
With the final participant now enrolled, NMD Pharma reports that investigators are conducting final in-clinic visits and data analysis. The company expects to share results from the Phase 2 trial in early 2026. If outcomes are positive, NMD Pharma plans to move forward with the next, and potentially final, phase of clinical study, in continued collaboration with CMTA-STAR and Patients as Partners in Research.
CMTA thanks the CMT community members who took part in the SYNAPSE-CMT trial and helped make this milestone possible. We also congratulate NMD Pharma on completing enrollment ahead of schedule. Through CMTA-STAR and Patients as Partners in Research, we’re proud to see the CMT community driving discovery together. CMTA will continue to follow NMD Pharma’s progress and share updates as the study moves forward.
CMT Research Starts With You
Join Patients as Partners in Research today to stay informed about new CMT research opportunities, including updates when NMD Pharma begins recruiting for its planned next clinical trial phase.
Read the full letter from NMD Pharma to the CMT community here.
