Corticotropin Studied for Treatment of Amyotrophic Lateral Sclerosis

Repository corticotropin injection (Acthar gel; Mallinckrodt, Staines-upon-Thames, UKI) is being assessed as an investigational treatment for amyotrophic lateral sclerosis (ALS), and 50% of the intended participants in a phase 2b study (NCT03068754) are now enrolled. 

Participants, age 18 to 75 years, who diagnosed with ALS who had their first symptoms in the previous 2 years will be randomly assigned to receive subcutaneous repository corticotropin or placebo on a 2:1 basis for 36 weeks. The intended enrollment is 210 people. Efficacy will be assessed with the ALS Functional Rating Scale-Revised.

The Food and Drug Administration (FDA) has granted both fast track and orphan status designations to repository corticotropin injection for ALS, which is already approved for 19 other indications, including exacerbations (relapses) of multiple sclerosis, rheumatoid arthritis, systemic lupus erythematosus, and symptomatic sarcoidosis; infantile spasms, polymyositis, and acute and chronic inflammatory eye diseases. Increasing evidence suggests that neuroinflammation plays a part in motor neuron death that occurs in ALS, leading to the hypothesis that corticotropin may also be useful in treating ALS.  

Todd Levine, MD, medical director of Honor Health Neurology and adjunct professor of neurology at Kansas University said, "ALS is a rare and incurable disorder that impacts patients from all walks of life," said. "The community looks forward to learning more from this research aimed at further understanding the disease and potential new treatments for ALS."

"We are very pleased to reach this milestone in our important study of Acthar gel in ALS patients," said Steven Romano, MD, executive vice president and chief scientific officer at Mallinckrodt. "We embarked on this multicenter double-blind placebo-controlled trial to evaluate the effects of the therapy on established measures of disease symptoms and progression and look forward to assessing the potential clinical value Acthar Gel may bring to patients with this devastating disease."