Mallinckrodt Completes Enrollment in Phase 4 Trial Testing Acthar Gel in SLE Patients

Mallinckrodt Pharmaceuticals has completed enrollment for its Phase 4 clinical trial evaluating the safety and effectiveness of H.P. Acthar Gel (repository corticotropin injection) in patients with persistently active systemic lupus erythematosus (SLE).

The company expects the first results to be available by early 2020.

“I am delighted that enrollment is completed, and look forward to evaluating the data as it becomes available,” Anca Askanase, MD, said in a press release. Askanase is director of the Columbia University Lupus Center in New York City and the trial’s principal investigator.

“Lupus patients are a challenging population to manage medically. We hope that the knowledge from this study will help guide treating physicians and can benefit lupus patients,” Askanase said.

Acthar is a prolonged-release, injectable formulation, of a porcine  adrenocorticotropic hormone (ACTH) analogue, designed to control the pro-inflammatory immune response in lupus and several other inflammatory diseases.

Marketed by Mallinckrodt Pharmaceuticals, the therapy is approved in the U.S. for multiple indications, including for use during an exacerbation or as maintenance therapy in selected cases of SLE.

The ongoing Phase 4 trial (NCT02953821) is evaluating the real-life effects of Acthar in SLE patients with persistently active disease. Following a screening period of up to 28 days, patients will be randomized to receive 1 mL (80 Units) of Acthar or the equivalent dose of a matching placebo. Treatment will continue for 24 weeks.

During the first four weeks, treatment will be administered subcutaneously (under the skin) every other day, after which it will be administered twice a week.

The study’s main goal is to measure the proportion of patients who responded to treatment, assessed by the SLE Responder Index (SRI), at week 16.

Efficacy also will be assessed with other measures of disease activity, including the SLE Disease Activity Index 2000 (SLEDAI-2K), British Isles Lupus Assessment Group (BILAG), Physician Global Assessment (PGA), Cutaneous Lupus Erythematosus Disease Area and Severity Index (CLASI), and 28 Joint Count.

The trial has enrolled 172 patients across 74 clinical sites in the United States, Argentina, Chile, Colombia, Mexico and Peru.

Data from a previous trial showed that treatment with Acthar significantly reduced disease activity scores in SLE patients whose disease was persistently active despite treatment with corticosteroid therapy.

“This is another exciting milestone in clinical evidence generation for Acthar. The data from our prior lupus pilot clinical study support the use of Acthar to treat lupus patients who have clinically significant disease activity despite receiving corticosteroids,” said Steven Romano, MD, chief scientific officer and executive vice president at Mallinckrodt.

“We look forward to completing the Phase 4 clinical trial later this year with top-line results anticipated by early 2020, providing additional data to help prescribers better understand how Acthar may be utilized in the management of these more difficult-to-manage patients with SLE,” Romano added.