First ALS Patient Enrolled in Phase 2/3 Trial of CuATSM, CMD Announces

Collaborative Medicinal Development (CMD) has enrolled the first patient in its Phase 2/3 clinical trial assessing the safety and efficacy of CuATSM, an investigational therapy for amyotrophic lateral sclerosis (ALS).

The trial (NCT04082832) is aimed at evaluating the efficacy and safety of CuATSM. The multicenter, randomized, double-blinded, placebo-controlled study is planning to recruit up to 80 ALS patients at four clinical sites in Australia.

“We are excited to have enrolled the first patient in this study. Working together with our patients and the CMD team, we are optimistic that this may be the first effective disease-modifying treatment for ALS,” Dominic Rowe, MD, professor of neurology at Macquarie University and coordinating principal investigator of the study, said in a press release.

CuATSM is a small artificial molecule able to cross the blood-brain barrier and deliver copper to cells containing damaged mitochondria, the cell compartments responsible for the production of energy. Damaged mitochondria are considered a hallmark of several neurodegenerative disorders, including ALS, Parkinson’s disease, Huntington’s disease, and Alzheimer’s disease.

Since CuATSM delivers copper only to damaged cells, leaving healthy cells unharmed, the idea is to use this molecule to target only unhealthy cells and reduce the damage caused by the disease.

Following enrollment in the Phase 2/3 trial, study participants will be randomly assigned to receive an oral suspension of CuATSM, or a placebo, for six months (six cycles of 28 days).

Primary objectives will include assessing the effects of treatment on disease progression and severity, which will be measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised, and on participants’ cognitive decline, which will be measured by the Edinburgh Cognitive and Behavioral ALS Screen.

Secondary goals will include assessing the effects of treatment on participants’ lung function, which will be measured by seated slow vital capacity (SVC), and the incidence of adverse events.

CMD anticipates announcing the first results of the trial within 18 months.

A previous open-label Phase 1 trial (NCT02870634) in patients with sporadic and familial ALS who received CuATSM for six months showed that the potential therapy slowed disease progression, as well as participants’ cognitive and lung function decline.

“CuATSM is the first product candidate from CMD’s pipeline and highlights our commitment to developing innovative medicines for neurological diseases with high unmet need,” said Craig Rosenfeld, MD, CEO of CMD.

CuATSM has already received orphan drug designation from the U.S. Food and Drug Administration for the treatment of ALS.