New Research Suggests That Acthar Reduces Proteinuria in Idiopathic Membranous Nephropathy In Part Through Suppression of Anti-PLA2R Antibodies

PHILADELPHIA, Nov. 10, 2011 /PRNewswire/ -- On November 10 at the American Society of Nephrology 44th Annual Meeting, Boston University Assistant Professor of Medicine Laurence H. Beck, Jr., M.D., Ph.D. will present results from a study which found that H.P. Acthar® Gel may induce a remission of proteinuria in patients with idiopathic membranous nephropathy (iMN) by suppressing production of antibodies to the phospholipase A2 receptor (PLA2R).  iMN is a common cause of nephrotic syndrome in adults.  Nephrotic syndrome is a known risk factor for progression to end-stage renal disease (ESRD).

"A high proportion of patients with idiopathic membranous nephropathy have circulating anti-PLA2R antibodies.  These antibodies attack a key protein located on the podocyte cells, which are found within the kidney and are directly involved in renal filtration," said Dr. Beck.  "We believe that anti-PLA2R antibodies are an important therapeutic target in the treatment of this form of chronic kidney disease.  The results of this study indicate that there appears to be a direct beneficial effect of Acthar on the immune system in patients with idiopathic membranous nephropathy."

Overall, the study included 14 patients with iMN from 2 pilot studies (Mayo Clinic and Columbia University Medical Center).  Patients were treated with Acthar for 6 months. In the study, 12 of 14 patients (86%) were anti-PLA2R positive at baseline.  All 12 patients with a baseline anti-PLA2R level experienced a reduction in anti-PLA2R by 6 months, with the complete disappearance of anti-PLA2R in 5 patients.  There were 5 patients with partial remission of proteinuria at 6 months.  Two additional patients with undetectable baseline anti-PLA2R serum levels did achieve clinical remission of proteinuria.  

"These findings provide a major step forward in the understanding of how patients with nephrotic syndrome due to idiopathic membranous nephropathy may benefit from Acthar treatment," said Steve Cartt, Executive Vice President and Chief Business Officer of Questcor Pharmaceuticals, Inc. "These data indicate that Acthar appears to work, at least in part, through an immune-modulating effect that can help restore kidney function in these patients.  We look forward to supporting further research in this area."

Nephrotic syndrome results from damage to the kidney glomeruli, tiny blood vessels that filter wastes and excess water from the blood and send them to the bladder as urine. It is characterized by excessive loss of protein in the urine, a condition known as proteinuria. This can be caused by a number of underlying diseases and disorders, including iMN, primary focal segmental glomerular sclerosis (FSGS) and other conditions. Based on epidemiology data and extensive market research with nephrologists, Questcor estimates that there are approximately 8,000 patients in the US with nephrotic syndrome due to iMN, and a similar number due to primary FSGS. There are also a number of other less common causes as well. Patients suffering from these types of idiopathic nephrotic syndrome often progress to ESRD if their kidney disease is not appropriately treated or if they do not respond adequately to treatment. According to ASN estimates, more than 500,000 patients in the US suffer from ESRD and this figure is expected to grow by 50% over the next 20 years. There are a number of disorders of the kidney that can result in ESRD and more than 100,000 new ESRD cases are diagnosed annually in the US.  Dialysis and kidney transplant are the only treatment alternatives for patients with ESRD.

A brief abstract containing a summary of the study findings was submitted and accepted by ASN and is currently available for viewing at: www.asn-online.org.  Dr. Beck's oral presentation on November 10 will provide an expanded set of data compared to that published in the abstract.

About H.P. Acthar® Gel

H.P. Acthar® Gel is a natural adrenocorticotropic hormone (ACTH) designed to provide a prolonged release after intramuscular or subcutaneous injection. Acthar is indicated for the treatment of acute exacerbations of multiple sclerosis in adults, and as monotherapy for the treatment of IS in infants and children under 2 years of age. It is also indicated to induce a diuresis or a remission of proteinuria in the nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus, and for the treatment of several other diseases and disorders. For more information, please visit www.acthar.com.

About Questcor

Questcor Pharmaceuticals, Inc. is a biopharmaceutical company whose products help patients with serious, difficult-to-treat medical conditions. Questcor markets H.P. Acthar® Gel (repository corticotropin injection), which is indicated for the treatment of acute exacerbations of multiple sclerosis in adults, and as monotherapy for the treatment of IS in infants and children under 2 years of age. It is also indicated to induce a diuresis or a remission of proteinuria in the nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus, and for the treatment of several other diseases and disorders.  The Company also markets Doral® (quazepam), which is indicated for the treatment of insomnia characterized by difficulty in falling asleep, frequent nocturnal awakenings, and/or early morning awakenings. For more information, please visit www.questcor.com.

SOURCE Questcor Pharmaceuticals, Inc.