Mallinckrodt plc has enrolled the first patient in the company’s Phase 2B study designed to assess the efficacy and safety of HP Acthar Gel (repository corticotropin injection) as a treatment for amyotrophic lateral sclerosis (ALS).
The US FDA previously granted the company’s request for a Fast Track designation and orphan status for its HP Acthar Gel Investigational New Drug application in patients with ALS. The drug is not FDA-approved for the ALS indication.
The study will enroll patients ages 18 to 75 with ALS and symptom onset (defined as first muscle weakness or dysarthria) ? two years prior to the screening visit. Subjects will be randomized on a 2:1 basis to receive subcutaneous (SC) HP Acthar Gel 0.2 mL (16 units) daily or SC matching placebo 0.2 mL daily for 36 weeks.
The efficacy of HP Acthar Gel will be assessed using standard measures of functional decline, including change from baseline in the ALS Functional Rating Scale-Revised, assessed after 36 weeks of therapy. Approximately 195 patients will be enrolled across multiple sites.
“ALS is a rare and incurable disorder that impacts patients from all walks of life,” said Todd Levine, MD, founder and director of the Phoenix Neurological ALS Clinic and adjunct Professor of Neurology at Kansas University. “The community welcomes new research aimed at further understanding the disease and potential new treatments for ALS.”
“We are pleased to announce the first patient in this important study of Acthar in ALS patients,” said Steven Romano, MD, Chief Scientific Officer and Executive Vice President at Mallinckrodt. “This multi-center, double blind, placebo-controlled trial will evaluate the effects of the drug on established measures of disease symptoms and progression, enabling us to assess the potential value Acthar may bring to patients with this devastating disease.”