# NMD Pharma presents SYNAPSE‑CMT Phase 2a data at 2026 PNS Annual

**Published:** 2026-06-18T02:23:38.318Z  
**Topic:** Synapse  
**Sentiment:** neutral  
**Publisher:** TrendWatcher — https://www.trendwatcher.in/article/59a11e15-ae82-403c-b4dd-54b8e2b528c1

NMD Pharma’s Phase 2a ignaceclant trial in Charcot‑Marie‑Tooth shows functional gains and safety, presented June 14 2026 at the PNS meeting.

NMD Pharma announced that its Phase 2a SYNAPSE‑CMT study of ignaceclant demonstrated consistent functional improvements and was well tolerated, with data presented in a late‑breaking oral session at the 2026 Peripheral Neuroscience Association Annual Meeting in Maastricht【2】. The findings matter because there are currently no FDA‑approved therapies for Charcot‑Marie‑Tooth (CMT), a hereditary neuropathy affecting muscle strength and daily function.  

| At a glance | |
|---|---|
| Study size | 81 adult CMT patients |
| Treatment duration | 21 days (follow‑up to day 28) |
| Primary endpoint | No difference on 6‑minute walk test |
| Secondary endpoints | Improvements in CMT‑FOM, hand‑grip strength, 9‑hole peg test, patient‑reported outcomes |
| Safety | All adverse events mild/moderate; no serious events or discontinuations |

## Clinical signals across secondary endpoints  
The double‑blind, placebo‑controlled trial enrolled genetically confirmed CMT1 or CMT2 patients across the US and Europe. While the pre‑specified primary endpoint—change in the 6‑minute walk test at day 21—was not met, secondary measures showed statistically significant gains. Hand‑grip strength improved by day 21 (p = 0.02) and further by day 28 (p < 0.01) versus placebo, and the composite CMT‑Functional Outcome Measure (CMT‑FOM) showed separation from placebo as early as day 7, persisting through day 28【2】. Trends toward better performance were also observed in the 9‑hole peg test and the 10‑meter walk/run test.

## Safety profile and regulatory context  
Ignaceclant was reported as safe and well tolerated; all adverse events were mild or moderate, with no serious events or treatment discontinuations【2】. The safety observations align with earlier Phase 2a data in generalized myasthenia gravis, where ignaceclant also demonstrated a favorable profile. In January 2025, the U.S. FDA granted orphan‑drug designation for ignaceclant in CMT, underscoring regulatory interest in this unmet‑need area【1】.

## What to watch  
- **Further clinical read‑outs** – NMD Pharma expects Phase 2 data for its SMA and gMG programs in 2026, which could inform the broader applicability of ClC‑1 inhibition.  
- **Regulatory milestones** – Any FDA or EMA submissions following the Phase 2a results will be key indicators of the pathway toward approval.  
- **Peer‑reviewed publication** – Full study details slated for journal publication later in 2026 will provide deeper insight into efficacy signals and durability.  

The SYNAPSE‑CMT data suggest that short‑term inhibition of the skeletal muscle chloride channel can yield measurable functional benefits in CMT patients, a disease with no approved treatments. Whether longer‑duration studies will amplify these gains and translate into regulatory approval remains the next critical question.

## Sources
1. Nmdpharma — [NMD Pharma announces topline results from its Phase 2a study of](https://www.nmdpharma.com/news/topline-results)
2. FinanzNachrichten.de — [NMD Pharma: SYNAPSE-CMT Phase 2a Clinical Study Data Presented in Late-Breaking Oral Presentation at 2026 PNS Annual Meeting](https://www.finanznachrichten.de/nachrichten-2026-06/68786392-nmd-pharma-synapse-cmt-phase-2a-clinical-study-data-presented-in-late-breaking-oral-presentation-at-2026-pns-annual-meeting-399.htm)

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Cite as: TrendWatcher, "NMD Pharma presents SYNAPSE‑CMT Phase 2a data at 2026 PNS Annual", https://www.trendwatcher.in/article/59a11e15-ae82-403c-b4dd-54b8e2b528c1