FDA Approves PharmaTher's Phase 2 Trial For Ketamine ALS Treatment

PharmaTher Holdings Ltd PHRRF PHRM, is a Canadian company focused on the development and commercialization of medicines derived from ketamine. PharmaTher announced on Wednesday that the U.S. Food and Drug Administration (FDA) has accepted an application to proceed with a Phase 2 clinical trial for an ALS treatment. 

According to the press release, “The FDA has accepted an investigator-initiated investigational new drug ("IND") application to proceed with a Phase 2 clinical trial (the “Study”) evaluating ketamine in the treatment of Amyotrophic Lateral Sclerosis (“ALS”), also known as Lou Gehrig's disease.”

This designation follows an earlier one that granted PharmaTher’s ketamine therapy for ALS as an orphan drug designation. Orphan drug designations grant pharma companies special funding, as the addressable markets are typically very small and not profitable for orphan drugs. 

If the phase 2 trial goes well, PharmaTher will ask the FDA to allow the company to proceed with a phase 3 trial. While it is a long road ahead, the current move is a step in the right direction for the development of new drugs and therapies for rare diseases such as ALS. 

“We are very pleased to have supported Dr. Barohn and his team by providing information for the IND that achieved FDA acceptance to conduct the Phase 2 clinical study,” PharmaTher CEO Fabio Chianelli said. “ALS is a devastating neurodegenerative disease with limited treatment options and ketamine, based on preclinical research, has the potential to have a positive impact on ALS patients.”

In the phase 2 trial, 36 participants with ALS will be separated into three 12-person groups. The different groups will receive varying amounts of PharmaTher’s ketamine treatment. 

“The Phase 2 clinical trial is a prospective, double-blind, randomized controlled multiple ascending dose study of ketamine, which will enroll a total of 36 participants with ALS in 3 sequential cohorts, with 12 participants in each cohort,” according to the press release. 

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