Mallinckrodt plc, a leading global specialty biopharmaceutical company, confirmed enrollment of the first patient in the company's phase 4 clinical study assessing the efficacy of H.P. Acthar gel (repository corticotropin injection) for inducing remission of proteinuria due to treatment-resistant or treatment-intolerant idiopathic focal segmental glomerulosclerosis (FSGS). FSGS is a rare disease that can cause nephrotic syndrome, a serious kidney disorder.

"This study is expected to be the largest controlled FSGS-focused clinical trial conducted to date," said lead investigator James A. Tumlin, M.D., founder and medical director of the Southeast Renal Research Institute.

"Patients with the disease who are unable to achieve remission are at risk to eventually experience kidney failure, which may lead some patients to the need for dialysis or a transplant. I am pleased to be working with Mallinckrodt in this important effort to better understand how Acthar may help address this serious unmet need."

Acthar is US Food and Drug Administration (FDA)-approved to induce a diuresis or a remission of proteinuria in nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus. Clinical experience has shown reduction in proteinuria in multiple glomerular nephropathies, including FSGS.

"Mallinckrodt is committed to providing treatment options where few exist," said Steven Romano, M.D., chief scientific officer and senior vice president at Mallinckrodt. "Clinical experience supports the use of Acthar to induce remission of proteinuria in FSGS. We are pleased to enroll the first patient in the PODOCYTE trial to better understand how clinicians may utilize Acthar in the management of proteinuria in this patient population."

The phase 4, multicenter, randomized, double-blind, parallel group, placebo-controlled efficacy study is titled, "Treatment of Proteinuria Due to Treatment Resistant or Treatment Intolerant Idiopathic Focal Segmental Glomerulosclerosis: A 2 Part Prospective Study of H.P. Acthar Gel (PODOCYTE)." The primary endpoint of the study is to measure complete and partial remission of proteinuria at week 24 as compared with patients' baseline. As is typical with sizeable rare disease clinical trials, the study is expected to take a number of years.

Nephrotic syndrome (NS) is a collection of symptoms that occur when the blood vessels in the kidney begin to leak excess protein in the urine, a condition called proteinuria. A variety of diseases and underlying disorders damage the kidneys and cause proteinuria in people with NS. These etiologies can include glomerular diseases such as: idiopathic membranous nephropathy, focal segmental glomerulosclerosis, minimal change disease, membranoproliferative glomerulonephritis, lupus nephritis, and IgA nephropathy. In these and other related disorders, the glomeruli, or small blood vessels that work as the kidney's filtering system, are damaged.

Proteinuria is one of the most important adverse prognostic factors for progression to end stage renal failure in patients with glomerular disease. One of the goals of treating nephrotic syndrome includes reducing or eliminating proteinuria.

HP Acthar Gel (repository corticotropin injection), is an injectable drug approved by the US Food and Drug Administration (FDA) for the treatment of 19 indications. Of these 19 indications, including the following on-label indications which are currently promoted:

Inducing a diuresis or a remission of proteinuria in nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus.

Controlled clinical trials have shown Acthar 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.

As monotherapy for the treatment of infantile spasms in infants and children under 2 years of age.
Use during an exacerbation or as maintenance therapy in selected cases of systemic lupus erythematosus.
Use during an exacerbation or as maintenance therapy in selected cases of systemic dermatomyositis (polymyositis).