But phase II data of the small molecule inhibitor for Charcot-Marie-Tooth disease Types 1 and 2 did show it meeting secondary endpoints for functional improvements.
NMD Pharma has pledged to accelerate the development of its treatment for the rare condition Charcot-Marie-Tooth disease, despite ignaseclant missing its primary endpoint in the phase II trial.
Topline phase II findings from the SYNAPSE-CMT study showed the investigational potentially first-in-class small molecule inhibitor failed to demonstrate a treatment difference on the six-minute walk test (6MWT) at 21 days.
However, it did provide durable, secondary endpoint benefits in Charcot-Marie-Tooth (CMT) disease types 1 or 2. Treatment with the skeletal muscle-specific chloride ion channel 1 (CIC-1) inhibitor enabled participants with the rare neuromuscular disease to see gains in muscle strength and motor performance.
These phase IIa clinical study results provide encouraging evidence that ignaseclant positively impacts skeletal muscle strength and function within weeks of treatment initiation, with effects that appear to persist beyond the dosing period"
Dr Ana de Vera, Chief Medical Officer at NMD Pharma, said: “These phase IIa clinical study results provide encouraging evidence that ignaseclant positively impacts skeletal muscle strength and function within weeks of treatment initiation, with effects that appear to persist beyond the dosing period.
“Importantly, we did not observe a plateau at the end of the treatment window, suggesting the potential for further benefit with longer-duration therapy.”
Thomas Holm Pedersen, PhD, Chief Executive Officer of NMD Pharma, said: “In a neuromuscular disease where patients typically experience a progressive loss of strength and function, observing consistent functional improvements over such a short timeframe is highly encouraging.
“We believe these results strongly support continued development of ignaseclant as a potential therapy to deliver meaningful functional benefit for patients living with CMT and will be accelerating the clinical development in this indication.”
NMD Pharma plans to present topline data from SYNAPSE-CMT in H1 of 2026 and publish the full study results in a journal.
