Ocular Sarcoidosis Open Label Trial of ACTHAR Gel

The initial treatment of ocular sarcoidosis usually relies on a combination of topical
glucocorticoids and oral glucocorticoids, both of which are associated with significant
ocular and systemic toxicities. Steroid-sparing therapies are limited by variable and
unpredictable efficacy, prolonged time until clinical response, medication intolerance, and
difficulties obtaining payor approval. As a result, it is not uncommon that treating
physicians must choose between excessive glucocorticoid toxicity versus poor control of
ocular inflammation. Ongoing ocular inflammation, in turn, leads to eventual visual loss and
occasionally blindness.

There is a need for a more reliable, expeditious therapy that can be used as an alternative
to glucocorticoids in sarcoidosis uveitis. Adrenocorticotropic hormone, through activation of
melanocortin receptors on leukocytes, can dampen immune responses through non-glucocorticoid
dependent mechanisms. The proposed study will aim to define whether there is effectiveness
for ACTHAR gel in these patients, delineate an effect dosing regimen, and provide information
about the safety of this approach for moderate to severe ocular sarcoidosis.

ACTHAR is a 39-amino acid peptide natural form of adrenocorticotropin hormone (ACTH) that was
initially approved in 1952 by the FDA. It has since been approved for 19 indications
including respiratory sarcoidosis, multiple sclerosis, and infantile spasms.

Inclusion Criteria:

- Patient with sarcoidosis as defined by ATS/ERS/WASOG guidelines (American Thoracic
Society/European Respiratory Society /World Association of Sarcoidosis and Other
Granulomatous Diseases)

- Any posterior, intermediate or panuveitis of sufficient severity to warrant therapy,
in the opinion of the treating physician --OR-- Anterior uveitis requiring 4 or more
daily applications of topical corticosteroids to maintain control of inflammation, or
uncontrolled with topical therapy

- Persistent disease activity (active uveitis) at the time of screening

Exclusion Criteria:

- Other cause for ocular inflammation

- Uncontrolled diabetes, hypertension, or other contra-indication to increased dosage of
glucocorticoids

- Recent (less than 4 weeks) intra-ocular or intra-orbital steroid injection

- Escalation of immunosuppressive medications between screening and initiation of the
study medication

- Severe extra-ocular sarcoidosis likely to require additional therapy (in the opinion
of the investigator)

- Administration of an investigational medication for sarcoidosis within 3 months, or 5
half-lives, whichever is longer

- Have a history of any opportunistic infection within 6 months prior to screening

- Have any history of malignancy, except fully resected cutaneous squamous cell cancer
or cutaneous basal cell cancer, or cervical carcinoma in-situ with a minimum of 5
years period without recurrence

- Severe other organ disease felt to be likely to lead to death within the next six
months

- Unable to follow the study protocol, including the requisite travel and follow-up
ocular testing

- Women of childbearing potential must be using adequate birth control measures
(abstinence, hormonal contraceptives, intrauterine device, barrier method with
spermicide, or surgical sterilization) and must agree to continue such precautions,
and not become pregnant or plan a pregnancy for 6 months after receiving their last
treatment with study agent. Women of childbearing potential must test negative on a
serum pregnancy test at screening.

- Breastfeeding women are excluded from participation