Efficacy and Safety of H.P. Acthar Gel for the Treatment of Refractory Cutaneous Manifestations of Dermatomyositis

Adult and juvenile dermatomyositis (DM) are systemic immune-mediated inflammatory diseases
most commonly affecting the skin and musculoskeletal system. Amyopathic dermatomyositis is a
subtype of dermatomyositis that affects only the skin and lacks the characteristic muscle
involvement. Treatment of these conditions, in particular the cutaneous manifestations, is
challenging and currently no universally effective single treatment exists. Many patients
have cutaneous manifestations that are refractory to numerous medications.

H.P. Acthar gel (adrenocorticotropic hormone gel) received FDA approval for treatment of a
variety of diseases, including dermatomyositis, in 1952. Despite this there is a paucity of
clinical data concerning the efficacy of H.P. Acthar gel for treating dermatomyositis.
Recently a small, retrospective case series describing significant improvement in both
cutaneous and musculoskeletal symptoms in 5 patients with refractory dermatomyositis treated
with H.P. Acthar gel was reported and has resulted in renewed interest in use of this
medication in dermatomyositis patient (reference below). The proposed efficacy of H.P. Acthar
gel has been attributed to its unique ability to induce production of endogenous cortisol,
corticosterone, aldosterone, and to bind melanocortin receptors on lymphocytes and other
cells to modulate immunologic responses.

Inclusion Criteria:

- Must be 18 years of age or older with refractory cutaneous symptoms related to either
classic dermatomyositis (CD), juvenile dermatomyositis (JD), or amyopathic
dermatomyositis(AD). Diagnosis will be based on either Bohan and Peter criteria (CD
and JD) or Sontheimer's criteria (AD)

- Must have had a skin biopsy with histologic features consistent with dermatomyositis
and current cutaneous manifestations consistent with dermatomyositis.

- Although not mandatory, patients with evidence of current or previous active myositis
will be eligible for enrollment. Patients will be considered to have refractory
disease if cutaneous manifestations exist despite treatment with steroids and at least
one steroid-sparing systemic treatment commonly found to be useful in patients with
dermatomyositis. These may include azathioprine, cyclosporine, mycophenolate mofetil,
IVIG, methotrexate, cyclophosphamide, chlorambucil, sirolimus, adalimumab, infliximab
and rituximab.

- Use of topical medications and sunscreen currently and in past will be noted but not
weighed for assessment of refractory cutaneous disease.

Exclusion Criteria:

- Patients with dermatomyositis who have minimal-to-no active cutaneous features (focal
involvement with less than 1% total body surface area involved or minimal modified
CDASI activity score).

- Patients whose cutaneous findings are not consistent with dermatomyositis and/or have
previous biopsy results suggestive of an alternative diagnosis

- Patients with inflammatory myositis other than dermatomyositis, such as polymyositis
or inclusion body myositis.

- Patients with malignancy-associated dermatomyositis

- Patients with clear features of an overlap myositis

- Patients younger than 18 years old

- Patients with acutely active or chronic infections.

- Patients with uncontrolled diabetes, hypertension, cardiovascular, hepatic, or renal
disease

- Pregnant or lactating females.

- Patients with any medical condition that is felt by the primary investigator to place
the patient at unreasonable risk for adverse effects during treatment with H.P.
Acthar.

- Hypersensitivity to H.P. Acthar, any of its components (allergy to pig-derived
proteins)

- Patients with osteoporosis

- Patients who have had surgery within 8 weeks of screening

- Patients with a history of or current gastric ulcers

- Patients taking daily doses of systemic corticosteroids greater than the equivalent of
40mg prednisone.