Ignaseclant
An investigational treatment for Charcot-Marie-Tooth Disease.
The same compound appears under different names depending on the context. Here is how to identify Ignaseclant wherever you encounter it, plus the key facts at a glance.
- Generic name
- Ignaseclant
- Development codes
- NMD670, NMD-670
- Drug class
- ClC-1 inhibitor (skeletal muscle, oral)
- Manufacturer
- NMD Pharma
- How it's taken
- Taken by mouth as a twice-daily oral dose.
An investigational oral medication being developed by NMD Pharma to improve muscle strength and function in CMT Type 1 and Type 2. Ignaseclant (formerly NMD670) does not target the underlying nerve damage; instead, it boosts how muscle fibers respond to the weakened nerve signals they receive. The Phase 2a SYNAPSE-CMT trial (NCT06482437) enrolled 81 patients with CMT Type 1 or Type 2; NMD Pharma announced in February 2026 that the trial missed its primary endpoint, the 6-minute walk test, though some secondary measures such as grip strength favored the drug. The FDA granted ignaseclant Orphan Drug Designation for CMT.
Where Ignaseclant fits
Investigational, not approved. Distinct from PMP22-targeted programs because it works on the muscle response rather than on the genetic cause of nerve damage. Could potentially be combined with disease-modifying therapies in the future. Currently being evaluated for CMT Type 1 and Type 2 collectively, which is unusual since most CMT trials are subtype-specific.
How Ignaseclant works
Most CMT trials target the underlying genetic cause of the nerve damage. Ignaseclant takes a different approach. It works on the muscle, not the nerve.
In healthy muscle, when a nerve fires, the muscle fiber responds with a strong contraction. In CMT, the peripheral nerves are damaged and send weaker, less-coordinated signals, so the muscles do not contract as strongly. ClC-1 (chloride ion channel 1) is a protein on muscle fibers that normally limits how excitable the muscle is — it acts like a damper. By blocking ClC-1, ignaseclant turns down that damper, which makes the muscle fiber more responsive to whatever signal the nerve does manage to deliver.
The practical effect, if the trials confirm benefit, is that a CMT patient's muscles produce more force from the same impaired nerve input. This is symptomatic improvement, not disease modification. The underlying nerve damage is still progressing, but muscle function may be measurably better while the patient is on treatment. Ignaseclant is the first ClC-1 inhibitor of its kind in clinical development.
Mechanism: First-in-class oral skeletal muscle ClC-1 (chloride ion channel) inhibitor. Blocking ClC-1 makes muscle fibers more responsive to weak signals from the nerve, partially compensating for the impaired nerve-to-muscle transmission that produces weakness in CMT.
Side effects and safety
Because ignaseclant is in Phase 2 trials for CMT, the safety profile is still being characterized. Earlier-phase studies in healthy volunteers and in myasthenia gravis patients have generally shown the drug to be well tolerated with manageable side effects. Detailed Phase 2 safety data from the SYNAPSE-CMT trial were presented at the 2026 MDA Clinical & Scientific Conference. The trial team will share complete safety information through peer-reviewed publication.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Ignaseclant
Taken by mouth as a twice-daily oral dose. The Phase 2a SYNAPSE-CMT trial used a 21-day twice-daily dosing protocol.
Clinical trial results
[SYNAPSE-CMT (NCT06482437)](https://clinicaltrials.gov/study/NCT06482437) is the Phase 2a randomized, double-blind, placebo-controlled trial of ignaseclant in patients with CMT Type 1 and Type 2. The trial enrolled 81 patients across global sites and evaluated efficacy, safety, and tolerability over a 21-day twice-daily dosing period. Topline results announced on February 3, 2026 showed the trial missed its primary endpoint, change in the 6-minute walk test at Day 21. Some secondary measures, including grip strength and fine motor skills, moved in the drug's favor.
Development history
NMD Pharma was founded to develop medicines that target ClC-1 in skeletal muscle. Ignaseclant (formerly NMD670) was first studied in myasthenia gravis. After preclinical and translational work supported the rationale for use in peripheral neuropathies, NMD Pharma cleared an FDA IND in June 2024 and dosed the first CMT patient. The Phase 2a SYNAPSE-CMT trial completed enrollment of 81 patients across CMT Type 1 and Type 2 globally. The FDA granted ignaseclant Orphan Drug Designation for CMT.
Explore Charcot-Marie-Tooth Disease trials
Other Charcot-Marie-Tooth Disease treatments
Common questions about Ignaseclant
▸What is Ignaseclant?
An investigational oral medication being developed by NMD Pharma to improve muscle strength and function in CMT Type 1 and Type 2. Ignaseclant (formerly NMD670) does not target the underlying nerve damage; instead, it boosts how muscle fibers respond to the weakened nerve signals they receive. The Phase 2a SYNAPSE-CMT trial (NCT06482437) enrolled 81 patients with CMT Type 1 or Type 2; NMD Pharma announced in February 2026 that the trial missed its primary endpoint, the 6-minute walk test, though some secondary measures such as grip strength favored the drug. The FDA granted ignaseclant Orphan Drug Designation for CMT.
▸How does Ignaseclant work?
Most CMT trials target the underlying genetic cause of the nerve damage. Ignaseclant takes a different approach. It works on the muscle, not the nerve.
In healthy muscle, when a nerve fires, the muscle fiber responds with a strong contraction. In CMT, the peripheral nerves are damaged and send weaker, less-coordinated signals, so the muscles do not contract as strongly. ClC-1 (chloride ion channel 1) is a protein on muscle fibers that normally limits how excitable the muscle is — it acts like a damper. By blocking ClC-1, ignaseclant turns down that damper, which makes the muscle fiber more responsive to whatever signal the nerve does manage to deliver.
The practical effect, if the trials confirm benefit, is that a CMT patient's muscles produce more force from the same impaired nerve input. This is symptomatic improvement, not disease modification. The underlying nerve damage is still progressing, but muscle function may be measurably better while the patient is on treatment. Ignaseclant is the first ClC-1 inhibitor of its kind in clinical development.
▸What are the side effects of Ignaseclant?
Because ignaseclant is in Phase 2 trials for CMT, the safety profile is still being characterized. Earlier-phase studies in healthy volunteers and in myasthenia gravis patients have generally shown the drug to be well tolerated with manageable side effects. Detailed Phase 2 safety data from the SYNAPSE-CMT trial were presented at the 2026 MDA Clinical & Scientific Conference. The trial team will share complete safety information through peer-reviewed publication.
▸How is Ignaseclant taken?
Taken by mouth as a twice-daily oral dose. The Phase 2a SYNAPSE-CMT trial used a 21-day twice-daily dosing protocol.
▸Is Ignaseclant FDA approved?
Ignaseclant is currently in phase 2 clinical trials for Charcot-Marie-Tooth Disease. It has not yet received FDA approval.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- NMD Pharma · February 12, 2026. NMD Pharma results from Phase 2a study of ignaseclant in Charcot-Marie-Tooth disease Types 1 and 2 selected for late-breaking oral presentation at the MDA Clinical & Scientific Conference. https://www.globenewswire.com/news-release/2026/02/12/3236843/0/en/NMD-Pharma-results-from-Phase-2a-study-of-ignaseclant-in-Charcot-Marie-Tooth-disease-Types-1-and-2-selected-for-late-breaking-oral-presentation-at-the-Muscular-Dystrophy-Associatio.html
- NMD Pharma · November 18, 2024. NMD Pharma Initiates Phase 2 Study of NMD670 in Patients with CMT Type 1 and 2. https://www.nmdpharma.com/news/nmd-pharma-initiates-phase-2-study-of-nmd670-in-patients-with-cmt-type-1-and-2
- U.S. National Library of Medicine — ClinicalTrials.gov. Study of NMD670 in CMT Disease Type 1 and 2 (SYNAPSE-CMT). https://clinicaltrials.gov/study/NCT06482437
- Pharmaceutical Technology. FDA grants Orphan Drug Designation to NMD Pharma's CMT therapy NMD670. https://www.pharmaceutical-technology.com/news/fda-nmd-charcot-marie-tooth/
- NMD Pharma · 2026-02-03. NMD Pharma announces topline results from its Phase 2a study of ignaseclant in Charcot-Marie-Tooth disease Types 1 and 2. https://www.globenewswire.com/news-release/2026/02/03/3230721/0/en/NMD-Pharma-announces-topline-results-from-its-Phase-2a-study-of-ignaseclant-in-Charcot-Marie-Tooth-disease-Types-1-and-2.html