FightMND Grant to Fund Phase 2 Study of Investigational Therapy for ALS

Continuing its support for a potential therapy for amyotrophic lateral sclerosis (ALS), Australia-based organization FightMND has granted about $700,000 to biopharmaceutical company Collaborative Medicinal Development.

The money will fund a randomized, double-blind, placebo-controlled study of CuATSM, one of CMD’s lead investigational therapies. According to a news release, the Phase 2 study will begin later this year. Research details will soon be available at clinicaltrials.gov. The overall goal is to gain U.S. Food and Drug Administration (FDA) approval for the therapy.

“FightMND’s support for CMD to perform a Phase 2 study of CuATSM is consistent with our goals,”  said Rebecca Sheean, PhD, research manager for FightMND. “We are excited that our support for CMD has allowed CuATSM to advance toward the next phase of clinical testing.”

Established in 2014 to find treatments and a cure for motor neuron disease (MND), the nonprofit FightMND backed the medication’s Phase 1 trial three years ago.

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The three-part trial (NCT02870634) showed that the once-daily oral medication may slow disease progression and improve patients’ respiratory and cognitive function. Studied in patients with sporadic and familial ALS, the investigation identified a Phase 2 recommended dose, examined CuATSM’s pharmacokinetic properties, confirmed treatment tolerability, and assessed the medication’s efficacy.

Data showed that after 24 weeks of CuATSM treatment at the recommended Phase 2 dose, ALS progression slowed markedly in those with sporadic ALS. Results also showed enhanced respiratory and cognitive ability. This is in line with preclinical studies in mouse models of ALS, which demonstrated that CuATSM performed dose-dependently when administered to animals.

The therapy is a small artificial molecule that can transport copper to cells containing damaged mitochondria, the cellular compartments that produce energy. Damaged mitochondria are widely considered a hallmark for ALS and other neurodegenerative diseases.

It is estimated that at least 30,000 people in the United States and 450,000 worldwide are living with ALS.

CMD, with bases in the United States and Australia, develops innovative therapies for neurodegenerative diseases.