Background Although there are many types of treatment for rheumatoid arthritis (RA) currently available, some patients have disease that is refractory to treatment and thus fail to achieve remission. Acthar Gel may have a potential role in enabling refractory RA patients to reach their treatment goals.

Objectives To assess the efficacy and safety of subcutaneous injections of Acthar Gel as adjunctive therapy in patients with active RA.

Methods This is an ongoing, 17-week, prospective, single-center, open-label study with planned enrollment of 10 patients (≥18 years) with RA, as confirmed by a full count of 68 joints, who are currently not responding sufficiently to a biological agent and have previously inadequately responded to ≥2 biologic agents with different modes of action. Patients are administered 80 U Acthar Gel subcutaneously every 72 hours for 12 weeks, with assessments conducted at screening (Week -1), baseline (Week 0), and Weeks 2, 6, 12, 14, and 16. Primary outcome measures are tender joint count and swollen joint count (ACR Core Data Set 66/68-joint count) and 20-item Health Assessment Questionnaire (HAQ). Secondary outcome measures are acute erythrocyte sedimentation rate (ESR), C-reactive protein (CRP) levels, Patient Global Visual Analogue Scale (VAS), and Physician Global VAS. Disease Activity Score for RA (DAS28) is also being calculated.

Results Six patients (aged 54-80; 9-39 years since diagnosis of RA) have completed the trial so far. At end of treatment (Week 12), all had reductions from baseline in tender and swollen joint counts; 3 showed improvement in HAQ and 3 showed no change. Improvements in DAS28 (6 of 6 patients), ESR (4/6), CRP (4/6), and Patient (5/6) and Physician (6/6) Global VAS were also seen at end of treatment, with improvements in all measures generally receding 4 weeks post-treatment (Week 16). One patient experienced a mild skin reaction at the injection site, and 1 had hypoglycemic episodes. A third patient was hospitalized for hip pain unrelated to Acthar Gel. All resolved and no patients withdrew from treatment due to adverse events.

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Conclusions It is extremely difficult to provide effective treatment for patients with active RA who have not responded adequately to biological agents with 3 different mechanisms of action. This interim report of an ongoing trial indicates that 80 U Acthar Gel administered subcutaneously every 72 hours for 12 weeks may provide a treatment option for such patients.

Acknowledgements This study is supported by an investigator-initiated research grant from Autoimmune and Rare Diseases Business (formerly Questcor) Mallinckrodt Pharmaceuticals.

Disclosure of Interest T. Gillis Grant/research support from: received study drug and study funding from Autoimmune and Rare Diseases Business (formerly Questcor) Mallinckrodt Pharmaceuticals, M. Crane Grant/research support from: received study drug and study funding from Autoimmune and Rare Diseases Business (formerly Questcor) Mallinckrodt Pharmaceuticals, C. Hinkle Grant/research support from: received study drug and study funding from Autoimmune and Rare Diseases Business (formerly Questcor) Mallinckrodt Pharmaceuticals, N. Wei Grant/research support from: received study drug and study funding from Autoimmune and Rare Diseases Business (formerly Questcor) Mallinckrodt Pharmaceuticals