NMD Pharma has secured clearance for an Investigational New Drug (IND) application from the US Food and Drug Administration to commence a Phase IIb clinical trial of muscle-targeted therapy, NMD670, for generalised myasthenia gravis (gMG).
This study will have gMG patients who are positive for anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibodies.
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The dose range-finding, double-blind, placebo-controlled trial will assess the twice-a-day dose of NMD670 in gMG patients.
To be conducted at both US and European study centres, the trial will commence subject enrolment soon.
A muscle-targeted small molecule inhibitor specifically targeting the ClC-1 chloride ion channel in skeletal muscles, NMD670 is being developed to alleviate persistent or fluctuating symptoms in gMG patients who are not fully responsive to existing standard treatments.
Recent Phase I/IIa trial results have shown promising clinical proof-of-mechanism for ClC-1 inhibitors in gMG patients, along with a strong safety and tolerability profile for NMD670.
NMD Pharma announced in November last year that it secured approximately $80m in a Series B financing round.
These funds are earmarked to complete three Phase II studies of NMD670, including trials for gMG, spinal muscular atrophy, and Charcot-Marie-Tooth disease.
NMD Pharma CEO Thomas Holm Pedersen said: “This is an important milestone for NMD Pharma, enabling us to progress our lead development candidate NMD670 into a Phase IIb trial in the US in generalised myasthenia gravis patients with AChR and MuSK positive antibodies.
“It follows positive Phase I/IIa data which provided proof-of-mechanism and where clinically and statistically significant effects were seen in patients.
“We are excited to bring forward a unique and validated muscle-targeted approach with promise to provide improvements in muscle function leading to clinically meaningful improvements in activities of daily living and quality of life.”
