New Octapharma 10% High Purity Immunoglobulin Enters Phase II/III Study in Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

Octapharma to set new standards in IVIG development in neurology, focussed on improving outcomes for patients and physicians

LACHEN, Switzerland--(BUSINESS WIRE)--Octapharma AG today announced the imminent start of the biggest ever study of an intravenous immunoglobulin preparation (IVIG) in chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).

“Octapharma’s stated aim is not to develop just another IVIG brand but to invest extensive time and preclinical resources to ensure that the new IVIG will offer outstanding features, representing tangible added value for the patient and care giver, such as exceptional tolerability”

The double-blind, placebo-controlled, randomised, multicentre, adaptive, two-stage Phase 2/3 dose-finding study will investigate the efficacy and safety of Octapharma's novel 10% intravenous immunoglobulin in the treatment of CIDP and, together with results from additional on-going and upcoming studies, will support its regulatory filing in Europe and the US.

Commenting on the start of the study, Kim Björnstrup, Vice Chairman of Octapharma Group said, “The development of our novel 10% IVIG is part of our ongoing commitment to invest in the development of protein based immunotherapies and in particular in IgG preparations. For 25 years, our cutting-edge research program has sought to develop new biological entities tailored specifically around the needs of clinicians and patients - delivering improved quality of life for patients and ease of delivery and management for hospitals.”

“Octapharma’s stated aim is not to develop just another IVIG brand but to invest extensive time and preclinical resources to ensure that the new IVIG will offer outstanding features, representing tangible added value for the patient and care giver, such as exceptional tolerability,” added Kim Björnstrup.

The development of this completely new high purity IVIG builds upon Octapharma’s experience in the area of immunoglobulin products. Octapharma’s current leading IVIG brand has been introduced to the market in 1995 and is currently registered in about 60 countries, including the EU and the US.

“Octapharma’s new 10% IVIG will be a step forward in the evolution of IVIG products. Regarding the development of the product, Octapharma has looked to optimise the characteristics of the product for improved patient outcomes, such as high tolerability even at high infusion rates. Pre-clinical studies and data from an on-going clinical trial in primary immunodeficiency (PI) have confirmed that a favourable tolerability profile may be expected” commented Dr Stefan Haag, Head of Octapharma's International Immunotherapy Business Unit.

The Phase II/III study in CIDP represents another element in a series of studies to investigate Octapharma’s new 10% IVIG for a range of neurologic and haematological conditions including immune thrombocytopenic purpura (ITP), Guillain-Barré syndrome (GBS), Kawasaki disease and chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).

About Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

CIDP is a distinct acquired chronic progressive or relapsing and spontaneously remitting

neurological condition characterised by the slow onset of weakness, areflexia and impaired sensation, which progressively increases¹. It is reported that 4% to 17% of CIDP patients die from the disease usually as a consequence of respiratory failure or pulmonary embolism, about 50% require persistent treatment, and 13% are permanently disabled². CIDP is generally considered to be an autoimmune disease caused by the activation of autoimmune mechanisms resulting from exposure to environmental antigens. These antigens may trigger immune responses that cross react with antigens in the myelin sheath of peripheral nerves³. Over the last 20 years, high-dose IVIG has become an effective and safe therapeutic option for CIDP. Because of the ease of its application, IVIG has become the preferred treatment for children with CIDP^4,5. A recently published placebo-controlled study on CIDP patients confirmed this short term benefit and showed sustained benefit from repeated IVIG treatment⁶.

About Octapharma AG

Octapharma, a biopharmaceutical company, was founded in 1983. Its mission is to work for the safe and optimal usage of human proteins. Octapharma products are state-of-the-art within treatment of haemophilia, immune diseases, volume expansion and plasma transfusions. At the present time it has 37 subsidiaries and representative offices and is making sales in over 80 countries. Turnover in 2009 was Euro 1,008 million. Octapharma is a privately held company with headquarters in Lachen, Switzerland, and employs 4,000 people worldwide.

Forward-looking statements

This news release contains forward-looking statements, which include known and unknown risks, uncertainties and other factors not under the company’s control. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments. These factors include results of current or pending research and development activities and actions by regulatory authorities.

References

(1) Hughes RA, Allen D, Makowska A, Gregson NA. Pathogenesis of chronic inflammatory demyelinating polyradiculoneuropathy. J Peripher Nerv Syst 2006; Mar;11(1):30-46.

(2) Hughes RA. Management of chronic inflammatory demyelinating polyradiculoneuropathy. Drugs 2003;63(3):275-87.

(3) Hughes RA, Hadden RD, Gregson NA, Smith KJ. Pathogenesis of Guillain-Barre syndrome. J Neuroimmunol 1999 Dec;100(1-2):74-97.

(4) Simmons Z, Wald JJ, Albers JW. Chronic inflammatory demyelinating polyradiculoneuropathy in children: II. Long-term follow-up, with comparison to adults. Muscle Nerve 1997 Dec;20(12):1569-75.

(5) Simmons Z, Wald JJ, Albers JW. Chronic inflammatory demyelinating polyradiculoneuropathy in children: I. Presentation, electrodiagnostic studies, and initial clinical course, with comparison to adults. Muscle Nerve 1997 Aug;20(8):1008-15.

(6) Hughes RA, Donofrio P, Bril V, Dalakas MC, Deng C, Hanna K, et al. Intravenous immune globulin (10% caprylate-chromatography purified) for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy (ICE study): a randomised placebo-controlled trial. Lancet Neurol 2008 Jan 4;7(2):136-44.