Phase 4 Study of Acthar Gel Enrolls First SLE Patients with Active Disease

Mallinckrodt Pharmaceuticals announced the enrollment of the first patients in the company’s Phase 4 clinical trial of H.P. Acthar Gel (repository corticotropin injection) for the treatment of systemic lupus erythematosus (SLE) patients with persistently active disease.

The randomized and double-blind study (NCT02953821) will assess the effectiveness of the gel against placebo. This trial was designed based on results of a pilot study (NCT01753401), whose results were recently published online in the journal Lupus Science & Medicine, in the study, “Efficacy and tolerability of repository corticotropin injection in patients with persistently active SLE: results of a phase 4, randomised, controlled pilot study.”

“The data generated in our pilot clinical study support the use of Acthar to treat lupus patients — for which it is already indicated — who have clinically significant disease activity despite receiving standard of care therapies,” Steven Romano, MD, Mallinckrodt’s chief scientific officer and executive vice president, said in a press release. “We are pleased to enroll the first patient in this follow-on trial, which will generate additional data to better understand how clinicians may utilize Acthar in the management of these more difficult to manage patients with SLE.”

Currently enrolling patients, the new  study counts as its primary endpoint the proportion of people responding to 16 weeks of treatment, as assessed by the SLE responder index (SRI). The SRI is a combined measure of three different expressions of disease activity in response to therapy.

Target enrollment is about 16o patients. Those interested or wanting more information can access the study through its identification number, given above, or its clinical trials.gov webpage.

In a presentation at the American College of Rheumatology/Association of Rheumatology Health Professionals (ACR/ARHP) recent annual meeting, Mallinckrodt also presented the results of a retrospective, claims-based analysis, suggesting that Acthar Gel is associated with reductions in corticosteroid use among patients with rheumatoid arthritis (RA), SLE, and dermatomyositis and polymyositis (DM/PM).

Specifically, the analysis looked at medication use — corticosteroids like prednisone, non-steroidal anti-inflammatory drugs and disease-modifying anti-rheumatic drugs — after the start of Acthar treatment in 504 RA, 322 SLE, and 222 DM/PM patients. Findings indicated a drop in corticosteroid use in all groups, from 67% to 54% among people with rheumatoid arthritis, from 73% to 58% among SLE patients, and from 76% to 58% among those with DM/PM. In patients using corticosteroids consistently for six months before starting on Acthar, significant dose reductions were seen in RA patients (28%) with a trend toward reductions, though not reaching significance, in SLE and DM/PM patients.

The proportions of patients relying on other medications, like biologics or disease-modifying drugs, was also significantly lower across groups after Acthar initiation, according to a company press release.

H.P. Acthar Gel is approved by the U.S. Food and Drug Administration (FDA) for use during an exacerbation or as maintenance therapy in selected cases of SLE, and as a treatment for other conditions that include infantile spasms, acute exacerbations of multiple sclerosis in adults, and certain cases of rheumatoid arthritis or symptomatic sarcoidosis.