FDA OKs Phase 2 Trial of Oral Therapy NMD670 for SMA Type 3

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by Lindsey Shapiro PhD |

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The U.S. Food and Drug Administration (FDA) has given NMD Pharma the green light to conduct a Phase 2 trial of its oral therapy NMD670 in people with spinal muscular atrophy (SMA) type 3.

The upcoming clinical trial aims to evaluate the efficacy, safety, and tolerability of NMD670 against a placebo in adults with SMA type 3 who are able to walk. Dosing is expected to begin at sites in North America and Europe in the first months of 2023.

“Receiving IND [investigational new drug] clearance to start our first clinical trial in SMA patients with NMD670 reflects the progress of NMD Pharma’s clinical development into new indications and geographies,” Thomas Holm Pedersen, CEO of NMD Pharma, said in a company press release.

“SMA is a rare disease and despite recent treatment advances, there is still a substantial unmet medical need to alleviate weakness and fatigue in these patients,” Pedersen added.

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New trial will focus on SMA type 3

SMA comprises a group of neuromuscular disorders characterized by the progressive loss of motor neurons, the nerves that communicate with muscle cells to control voluntary muscle movement. Consequently, muscles are unable to contract properly, leading to muscle weakness and atrophy.

Patients with SMA type 3, which is a milder form of SMA, are typically able to stand and walk independently, although some individuals may lose the ability to walk as the disease progresses.

NMD670 is a first-in-class small molecule that works to increase muscle contractions by inhibiting CIC-1, a chloride channel that causes muscles to relax. In doing so, the treatment is thought to increase muscle strength and function.

NMD Pharma is developing the oral treatment for neuromuscular diseases including SMA and myasthenia gravis (MG), an autoimmune condition similarly marked by muscle weakness and wasting.

Preclinical studies have demonstrated that NMD670 enhances neuromuscular communication and improves skeletal muscle function.

According to the company, the benefits of the treatment have been observed in animal models of MG, SMA, and a range of other neuromuscular disorders.

Results from a small Phase 1/2a clinical trial (CHDR1948) also demonstrated that single or multiple doses of the treatment were safe and well-tolerated among both healthy adults and MG patients. Moreover, signs of clinical efficacy were observed in the patients, including gains in muscle function and strength.

Based on those findings, NMD believes the treatment would also benefit SMA patients. The upcoming randomized, placebo-controlled Phase 2 trial will be the first conducted in this population.

While details of the study have not been released, NMD noted it will be a 2-way crossover study, meaning the trial will consist of two different treatment arms, and all participants will receive both treatments, in random order.

The study also will be double-blinded, meaning that neither participants nor trial investigators will be aware of who receives treatment and who is given the placebo while the trial is ongoing.

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