Clinical Trial Tests H.P. Acthar Gel as Potential ALS Treatment

Joana Fernandes, PhD avatar

by Joana Fernandes, PhD |

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Mallinckrodt has launched the Phase 2b PENNANT trial (NCT03068754) to investigate the safety and effectiveness of H.P. Acthar Gel (repository corticotropin injection) as a treatment for amyotrophic lateral sclerosis (ALS).

The H.P. Acthar Gel is an experimental injectable drug that the U.S. Food and Drug Administration (FDA) has granted fast track designation and orphan drug status for the treatment of ALS patients. The gel contains a highly-purified preparation of adrenocorticotropic hormone (ACTH). ACTH triggers the body’s own corticosteroids — cortisol, corticosterone, and aldosterone — and also may affect immune cells.

“We are pleased to announce the first patient in this important study of Acthar in ALS patients,” Steven Romano, MD and Mallinckrodt’s chief science officer, said in a press release. “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.”

The PENNANT trial will assess the safety and effectiveness of H.P. Acthar Gel treatment in nearly 195 ALS patients aged 18 to 75 who developed the initial symptoms (first muscle weakness and difficulty in articulating words) up to two years before enrollment. Patients will be assigned to receive either daily subcutaneous treatment with H.P. Acthar Gel (0.2 mL, 16 units), or a placebo for 36 weeks.

Researchers will measure functional decline after 36 weeks of treatment using the ALS Functional Rating Scale-Revised.

“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 professor of neurology at Kansas University. “The community welcomes new research aimed at further understanding the disease and potential new treatments for ALS.”

The H.P. Acthar Gel already is used as a therapy for other conditions, including lupus, exacerbations of multiple sclerosis (MS), sarcoidosis, and certain inflammatory processes, such as keratitis and polymyositis.

ALS is characterized by the loss of motor neurons, located in the spinal cord, that control muscle activity, but exactly what factors trigger the development of the disease remains elusive. One possible explanation is that the person’s own immune system reacts against these neurons by sending antibodies to destroy them, leading to the development of this disease.