Phase 4 of Pulmonary Sarcoidosis Treatment Enrolls First Patient

This morning, it was confirmed that the first patient was enrolled in Mallinckrodt’s Phase 4 multi-center, randomized, double-blind, placebo-controlled pilot study to further assess the efficacy of H.P. Acthar Gel (Repository Corticotropin Injection) as a therapy option in patients with pulmonary sarcoidosis.

Subjects enrolled in the study are required to be receiving prednisone treatment and will be randomly assigned to receive either 1mL (80 units) of H.P. Acthar Gel or 1mL of a matching placebo subcutaneously twice per week.

Pulmonary sarcoidosis is the growth of tiny collections of inflammatory cells, or granulomas, in the lungs. The condition can be triggered by the inhalation of bacteria, viruses, dust or chemicals, and symptoms most commonly include persistent dry cough, shortness of breath, wheezing, and chest pain.

In some cases, untreated pulmonary sarcoidosis can lead to permanent scarring in the lungs, making it difficult to breathe.

"I am pleased that the first patient has been enrolled in this important Phase 4 study, the results of which may potentially provide evidence to further support Acthar as a treatment option in appropriate symptomatic sarcoidosis patients," said Dr. Robert Baughman, M.D., Professor in the Department of Internal Medicine, University of Cincinnati College of Medicine, Cincinnati, Ohio in a press release. "In my experience, a considerable number of sarcoidosis patients can have persistent disease that may not be resolved by first-line treatment."

Study participants who complete the 24-week treatment period will be eligible to continue in an optional 24-week open-label extension. Response will be evaluated by Sarcoidosis Treatment Score (STS) with a time frame of baseline to 24 weeks. STS combines the results of pulmonary function tests, high-resolution computed tomography, quality of life (QOL), and corticosteroid tapering.

"The enrollment of the first patient in this study is an important milestone in our further assessment of Acthar's efficacy as a treatment option for sarcoidosis patients," said Tunde Otulana, M.D., Chief Medical Officer at Mallinckrodt. "We believe this exploratory evaluation of the more challenging patients who may still have disease activity after previous therapies can potentially help physicians better understand which individuals may benefit from the drug as a treatment alternative."

Currently, the injectable formulation of H.P. Acthar Gel is approved by the U.S. FDA for the treatment of 19 indications, including use in patients with systemic lupus erythematosus, rheumatoid arthritis, and nephrotic syndrome with uremia, among others.

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