Acute Multiple Sclerosis - H.P. Acthar® Gel Reduces Shows Promise In Acute Exacerbations

A presentation at the American Society of Nephrology 44th Annual Meeting, showed study results of H.P. Acthar® Gel, a natural adrenocorticotropic hormone (ACTH) designed for treatment of acute exacerbations of multiple sclerosis in adults, and as mono-therapy for the treatment of IS in infants and children under 2 years of age.

Acthar® may induce a remission of proteinuria in patients with idiopathic membranous nephropathy (iMN), a common cause of nephrotic syndrome in adults. The remission is induced by suppressing production of antibodies to the phospholipase A2 receptor (PLA2R). It has been established that Nephrotic syndrome is a risk factor for progression to end-stage renal disease (ESRD).
Nephrotic syndrome is a nonspecific disorder that can be caused by numerous underlying diseases and disorders, such as iMN, primary focal segmental glomerular sclerosis (FSGS) and other conditions.

It is caused by damage of the kidney glomeruli in which tiny blood vessels filter the blood's waste and excess water into the urine, causing large amounts of protein to leak from the blood into the urine, a condition known as proteinuria.

Questcor estimates, based on epidemiology data and extensive market research with nephrologists, that about 8,000 patients in the US suffer from nephrotic syndrome caused by iMN, and a similar number due to primary FSGS but there are also several other less common causes. If not appropriately treated or if non-responsive to treatment, patients with these types of idiopathic nephrotic syndrome often progress to ESRD, which according to ASN estimations affects over 500,000 patients in the US. This figure is projected to increase by 50% over the next 20 years.

ESRD can be caused by several kidney disorders, and in the U.S., each year over 100,000 new ESRD cases are diagnosed. The only therapy available for ESRD patients is dialysis and kidney transplant.

Dr. Laurence H Beck Jr., M.D., Ph.D. Boston University Assistant Professor of Medicine commented:

"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.

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."


Researchers examined a total of 14 patients with iMN from 2 pilot studies at the Mayo Clinic and Columbia University Medical Center who received Acthar for 6 months. At baseline 12 of 14 patients (86%) were anti-PLA2R positive and at 6 months, researchers observed a reduction in anti-PLA2R in all of these 12 patients, with a complete disappearance of anti-PLA2R in 5 patients, whilst 5 of them experienced a partial remission of proteinuria. They also observed a clinical remission of proteinuria in two additional patients with undetectable baseline anti-PLA2R serum levels.

Steve Cartt, Executive Vice President and Chief Business Officer of Questcor Pharmaceuticals, Inc. commented:

"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. 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."


The ASN accepted a brief abstract containing a summary of the study findings, which is currently available at: www.asn-online.org.

H.P. Acthar® Gel is designed to offer an extended release after intramuscular or subcutaneous injection. Acthar, a natural adrenocorticotropic hormone (ACTH), is indicated to treat acute exacerbations of multiple sclerosis in adults, and as monotherapy for the treatment of IS in infants and children below the age of 2 years. It has also been developed to induce a diuresis or remission of proteinuria in nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus, as well as for the treating various other diseases and disorders.