Mallinckrodt, a leading global specialty pharmaceutical company, confirmed enrollment of the first patient in the company's phase 4, multi-center, randomized, double-blind, placebo-controlled pilot study to further assess the efficacy of H.P. Acthar Gel as a therapy option in patients with pulmonary sarcoidosis.

H.P. Acthar Gel is approved by the US Food and Drug Administration (FDA) for the respiratory condition symptomatic sarcoidosis, one of 19 indications.

"I am pleased that the first patient has been enrolled in this important phase 4 study, the results of which may potentially provide evidence to further support Acthar as a treatment option in appropriate symptomatic sarcoidosis patients," said Dr. Robert Baughman, M.D., Professor in the Department of Internal Medicine, University of Cincinnati College of Medicine, Cincinnati, Ohio1. "In my experience, a considerable number of sarcoidosis patients can have persistent disease that may not be resolved by first-line treatment."

"The enrollment of the first patient in this study is an important milestone in our further assessment of Acthar's efficacy as a treatment option for sarcoidosis patients," said Tunde Otulana, M.D., chief medical officer at Mallinckrodt. "We believe this exploratory evaluation of the more challenging patients who may still have disease activity after previous therapies can potentially help physicians better understand which individuals may benefit from the drug as a treatment alternative."

The phase 4 clinical study is titled "Pilot Study to Assess the Efficacy and Safety of H.P. Acthar Gel in Subjects With Pulmonary Sarcoidosis." This is a phase 4, multicenter, randomized, double-blind, placebo-controlled pilot study evaluating the efficacy and safety of H.P. Acthar Gel in the treatment of pulmonary sarcoidosis. All subjects who meet eligibility criteria – which include a requirement to be receiving prednisone treatment – will be randomly assigned to receive either 1mL (80 units) of H.P. Acthar Gel or 1mL of a matching placebo subcutaneously 2 times/week. Subjects will be assigned to treatment in a 1:1 ratio with up to 50 subjects per arm for a total of up to 100 subjects. Subjects who complete the 24-week randomized, double-blind, placebo-controlled treatment period will be eligible to continue in an optional 24-week open-label extension.

Response will be evaluated by Sarcoidosis Treatment Score (STS) with a time frame of baseline to 24 weeks. STS is an exploratory newly developed composite score that combines the results of pulmonary function tests (forced vital capacity; diffusing capacity of the lungs for carbon monoxide), high-resolution computed tomography, quality of life (King's Sarcoidosis Questionnaire; Fatigue Assessment Scale) and corticosteroid tapering.

Sarcoidosis is a challenging, yet treatable, rare multisystem disease. In some cases the symptoms may come and go throughout a lifetime. This is referred to as symptomatic sarcoidosis. In people with sarcoidosis the immune system overreacts, forming clumps of cells called granulomas that result in inflammation to the body's tissues. The disease can impact any organ, but it most often impacts the lungs, lymph nodes, eyes, and skin. Over 90 per cent of people with sarcoidosis will suffer lung problems. Concomitant involvement of organs outside of the lungs is common, occurring in as many as half of all sarcoidosis cases.

H.P. Acthar gel is an injectable drug approved by the FDA for the treatment of 19 indications. Of these, today the majority of Acthar use is in these indications: Treatment during an exacerbation or as maintenance therapy in selected cases of systemic lupus erythematosus; Monotherapy for the treatment of infantile spasms in infants and children under 2 years of age; The treatment of acute exacerbations of multiple sclerosis in adults. Controlled clinical trials have shown H.P. Acthar Gel to be effective in speeding the resolution of acute exacerbations of multiple sclerosis. However, there is no evidence that it affects the ultimate outcome or natural history of the disease; Inducing a diuresis or a remission of proteinuria in nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus; Treatment during an exacerbation or as maintenance therapy in selected cases of systemic dermatomyositis (polymyositis);    The treatment of symptomatic sarcoidosis; Adjunctive therapy for short-term administration (to tide the patient over an acute episode or exacerbation) in rheumatoid arthritis, including juvenile rheumatoid arthritis (selected cases may require low-dose maintenance therapy);   Treatment of severe acute and chronic allergic and inflammatory processes involving the eye and its adnexa such as: keratitis, iritis, iridocyclitis, diffuse posterior uveitis and choroiditis, optic neuritis, chorioretinitis, anterior segment inflammation.