ACTHAR Gel for Cutaneous Sarcoidosis: An Open Label Trial

Sarcoidosis is a multisystem granulomatous disease of unknown cause. Although sarcoidosis
most commonly affects the lung3, it may affect any organ. Although corticosteroids are
recognized as the drug of choice for sarcoidosis, ACTH is the only drug that is FDA-approved
for this disorder. However, there is limited data on the efficacy of ACTH for this
condition.

Presently, corticosteroids as considered the drug of choice for the treatment of cutaneous
sarcoidosis. However, ACTHER GEL not only has obvious anti-inflammatory effects by resulting
in corticosteroid production, but it may also activate melanocortin receptors. The
melanocortin system has powerful anti-inflammatory properties that may be beneficial in the
treatment of cutaneous sarcoidosis.

Inclusion Criteria:

1. Patients must have biopsy-proven sarcoidosis.

2. a) Patients with active cutaneous sarcoidosis and two active lesions on stable
therapy or no therapy for at least two months AND/OR b) Patients with active
cutaneous sarcoidosis and lupus pernio lesions on stable therapy or no therapy for at
least two months. A maximum of 5 patients may be enrolled fulfilling criterion b)
above.

3. Both lesions must have a SASI induration score of > 1 and a SASI induration +
erythema score of > 2.

4. If two lesions are present, one must be > 1cm in diameter and the subject must be
willing to have it biopsied. The second lesion must be at least 0.5 cm in diameter.

5. If a subject has only lupus pernio facial lesions, one needs to be at least 0.5 cm in
diameter.

Exclusion Criteria:

1. Previous toxic or allergic reaction to ACTHAR gel

2. The presence of another skin condition in addition to sarcoidosis that would
interfere with the assessment of the sarcoidosis skin lesions.

3. Uncontrolled hypertension.

4. Uncontrolled diabetes.

5. Active infection.

6. A medical condition that, in the opinion of the investigator would place the subject
at significant risk by administering ACTHAR gel.