Acthar in Treatment of Refractory Dermatomyositis and Polymyositis

Inclusion Criteria:

• Definite or probable polymyositis (PM) or dermatomyositis (DM) by Bohan and Petercriteria.

• PM patients must either possess a myositis-associated autoantibody or undergoadjudication for confirmation of the PM diagnosis by consensus of two experts toensure non-PM patients are not enrolled. This step is necessary since there arewell-known mimics of PM.

• Age ≥ 18 years.

• Active myositis as defined by baseline Manual Muscle Testing (MMT-8) no greater than 125/150 and at least 2 additional CSM meeting the criteria stipulated below:

1. Patient global with a minimum value of 2.0 cm on a 10 cm visual analog scale(VAS)

2. Physician global with a minimum value of 2.0 cm on a 10 cm VAS scale

3. Health Assessment Questionnaire (HAQ) disability index with a minimum value of 0.25

4. Elevation of at least one of the muscle enzymes [which includes creatine kinase (CK), aldolase, lactate dehydrogenase (LDH), alanine aminotransferase (ALT) andaspartate aminotransferase (AST)] at a minimum level of 1.3 x the upper limit ofnormal.

5. Global extramuscular disease activity score with a minimum value of 1.0 cm on a 10 cm VAS scale [this measure is the physician's composite evaluation and isbased on assessments of activity scores on the constitutional, cutaneous,skeletal, gastrointestinal, pulmonary and cardiac scales of the Myositis DiseaseActivity Assessment Tool (MDAAT)].

• To ensure that we can enroll active DM patients with a severe rash who may not meetthe MMT-8 criterion noted above, we propose additional enrollment criteria such thatthe International Myositis Assessment and Clinical Studies (IMACS) definition ofimprovement (DOI) can potentially be met:

1. Cutaneous VAS score on MDAAT > 3 cm on a 10 cm VAS scale, and

2. At least 3 of the above 5 (a through e under 4.) criteria.

• Refractory myositis is defined by active disease despite an adequate glucocorticoidtrial (> 2 months of usual glucocorticoid therapy or intolerance to such therapy)and/or ≥ 1 conventional immunosuppressive agent (e.g. methotrexate, azathioprine,tacrolimus, cyclosporine, mycophenolate mofetil, IVIG, anti-TNF or rituximab) for areasonable dose and duration (> 3 months or intolerance to therapy). It is recommendedto enroll refractory patients failing (or intolerant to) both glucocorticoids and atleast 1 conventional immunosuppressive agent.

• If the enrolling physician is planning to continue current immunosuppressive agents orglucocorticoids as concomitant therapy with Acthar gel during the trial, then patientmust be on a stable glucocorticoid and/or immunosuppressive dose 2 weeks prior tovisit 1. The patient should have been on that immunosuppressive medication for atleast 8 weeks (and at least 4 weeks for glucocorticoids) prior to visit 1.

• If the enrolling physician is planning to discontinue current immunosuppressive agentor glucocorticoids, then following wash out period is required prior to visit 1.

• If previous concomitant medications were discontinued, the following wash out periodsare required prior to Visit 1

• Methotrexate -4 weeks

• Other IS agent (e.g. azathioprine, cyclosporine, tacrolimus, leflunomide,mycophenolate mofetil) - 4 weeks

• IVIg or cyclophosphamide - 2 months

• rituximab -6 months

• infliximab or adalimumab -8 weeks

• glucocorticoids - 2 weeks

• etanercept -2 weeks

• anakinra -1 week

Exclusion Criteria:

• Juvenile DM or PM, myositis in overlap with another connective tissue disease, cancerassociated myositis, inclusion body myositis, or any other non immune-mediatedmyopathy.

• Hypersensitivity to Acthar

• Severe cardiac or pulmonary involvement

• Severe muscle damage defined as a baseline global muscle damage score on the MDI (Myositis Damage Index) of ≥ 5 cm on a 10 cm VAS.

• Patients with malignancy within 3 years of screening (except basal cell cancer orsquamous cell cancer of skin).

• Uncontrolled diabetes, hepatic or renal disease.

• Ongoing active or chronic infections.

• Pregnant or lactating females.

• For any medical or physical or socio-psychological reasons that PI feels would notallow the subject to complete the study.