- A pilot study run by Mallinckrodt (NYSE:MNK) assessing H.P. Acthar Gel in patients with persistently active Systemic Lupus Erythematosus (SLE) on corticosteroid therapy failed to meet its primary endpoint of a clinical response defined by complete resolution of skin or joint activity as measured by the hybrid SLE Disease Activity Index (hSLEDAI) with no new organ system disease as measured by the British Isles Lupus Assessment Group-2004 (BILAG). The data were presented at poster session during the American College of Rheumatology/Association of Rheumatology Health Professionals Annual Meeting in San Francisco.
- The company-sponsored study, "Repository Corticotropin Injection (H.P. Acthar Gel) Attenuates Disease Activity in Patients with Persistently Active Systemic Lupus Erythematosus (SLE) Requiring Corticosteroids" was an eight-week, double-blind, randomized placebo-controlled trial that assessed the clinical efficacy of repository corticotropin injection (RCI) in 38 patients with persistently active SLE involving skin and/or joints despite moderate dose corticosteroids. Subjects were randomized 2:1 to receive RCI 40 units subcutaneously every day, RCI 80 units subcutaneously every other day or volume-matched placebo gel.
- Although the study failed to achieve its primary efficacy endpoint, treatment with RCI did show improvement in secondary endpoints versus placebo, including reduction in total hSLEDAI, BILAG and CLASI (Cutaneous Lupus Erythematosus Disease Area and Severity Index) Activity scores, decreased tender and swollen joint count and increased proportion of subjects achieving response as defined by a composite index called SRI (SLE responder index).
- Most patients in the study were on multiple therapies so the clinical outcomes may not be solely attributable to Acthar, which has not been formally studied in combination with other commonly used SLE treatments.
- SLE, affecting more than 1.5M Americans, is an autoimmune disease in which the immune system produces antibodies to cells within the body that leads to widespread inflammation and tissue damage.
- H.P. Acthar Gel is currently FDA-approved for 19 indications but generates almost all of its sales in five areas: proteinuria in nephrotic syndrome, multiple sclerosis, infantile spasms, exacerbation or maintenance treatment of SLE and systemic dermatomyositis.
- Mallinckrodt obtained the rights to Acthar Gel via its $5.6B acquisition of Questcor Pharmaceuticals in August 2014.