Octapharma Phase II/III trial for the targeted treatment of relapsing multiple sclerosis enrols first patients18.12.2013
Lachen, Switzerland, December 18th 2013– Octapharma AG, one of the largest global manufacturers of human proteins, today announced the enrolment of the first two patients in its GAM-27 Phase II/III clinical trial for the treatment of relapsing multiple sclerosis (RMS). Through intravenous administration of immunoglobulin (Octagam 5%), the study seeks to support the overall clinical benefit of a lower annualised relapse rate in patients where first-line treatment is not suitable.
This randomised, active-controlled phase II/III study aims to confirm preliminary results that suggested it is possible to identify potential responders to treatment with Octagam 5%. Using a combination of functional genomic and protein expression tests, a special assay panel, Heidelberg Assay Panel (HAP), was developed that could potentially stratify patients into predicted responders or non-responders. These laboratory-based tests require just a simple blood sample from the patient.
A subgroup of patients with RMS experience disabling side effects with current first-line treatments, which can result in an inability to comply with their treatment protocol, or do not benefit from this treatment. By identifying predicted responders it is believed that the known and favourable tolerability profile of Octagam 5% could improve the quality of life for this group of patients. The GAM-27 Steering Committee (Prof. Thomas Berger, Clinical Department of Neurology, Medical University Innsbruck, Austria; Prof. Irina Elovaara, Medical School, Department of Neurology, University of Tampere, Finland; Prof. Hans-Peter Hartung, Neurological Clinic, Heinrich-Heine-Universität, Düsseldorf, Germany) welcomed the enrolment of the first 2 patients into the trial in Bulgaria and anticipates further enrolment in the other participating countries.
“Funding this study is part of our on-going long-term commitment to supporting advances in MS therapies,” commented Dr Wolfgang Frenzel, International Medical Director at Octapharma. “This extensive trial is a critical step in confirming the ability to identify the subset of patients for whom treatment with Octagam 5% has the potential to change the course of the disease and offer an improvement in their quality of life.”
“The potential for this HAP test to accurately identify the estimated 20-30% of these patients with relapsing multiple sclerosis who should benefit from Octagam 5% treatment, could provide a significant step towards personalised therapies for this patient group,” said Stefan Meuer, Professor of Immunology at the Department of Immunology at the Ruprecht Karls-University in Heidelberg, in whose group the HAP test had been developed.
The study will recruit 216 patients with early relapsing MS at approximately 35 European sites in several countries; including Austria, Bulgaria, Germany, Hungary and Russia. The patients will be treated for a 24 month period with expected trial completion by the end of 2016. Full study design was presented at the European Charcot Foundation annual meeting in Baveno, Italy from November 28-30 2013.
Notes to Editors
Multiple sclerosis (MS)
MS is a heterogeneous, life-long inflammatory neurodegenerative disease of the central nervous system presenting with a wide clinical, pathological and genetic spectrum. Affecting approximately 2.5 million people worldwide (twice as many women than men) with the first symptoms starting in most cases before the age of 30, there currently exists no curative therapy.
Treatment of MS patients with intravenous immunoglobulins has been reported for 30 years and is currently recommended in treatment guidelines (e.g. the European Federation of Neurological Sciences), for patients in whom first-line treatments are contraindicated, ineffective, not well tolerated or simply not accepted by the patient for other reasons.
An interesting aspect of the clinical studies of intravenous immunoglobulins in MS is that not all patients benefit from therapy, which suggests that against the background of the heterogeneous pathology and the variable genetic background of this disabling disease there is a sub-population of patients who are responsive to IVIG. However, to date, it has not been possible to predict which patients are likely to respond to immunoglobulin treatment.
Heidelberg Assay Panel (HAP) Score
The HAP score uses functional genomic and protein expression tests and is conducted centrally by Prof. Meuer and Dr. Giese of the Institute of Immunology at the University of Heidelberg, Germany.
Headquartered in Lachen, Switzerland, Octapharma AG is one of the largest human protein products manufacturers in the world and has been committed to patient care and medical innovation for 30 years. Its core business is the development, production and sale of human proteins from human plasma and human cell-lines. Patients in over 100 countries are treated with products in the following therapeutic areas:
- Haematology (coagulation disorders)
- Immunotherapy (immune disorders)
- Intensive Care and Emergency Medicine
Octapharma owns five state-of-the-art production facilities in Austria, France, Germany, Sweden and Mexico.
Octapharma has been involved in MS for over 16 years. The first study conducted on the intravenous immunoglobulin (IVIG) Octagam was run in 1997. Since then Octapharma has continued its research into MS in the belief that alternative treatment options could offer significant improvement to the lives of patients.
Dr Wolfgang Frenzel
International Medical Director
Clinical Research & Development Department
Email address: pressrelease.enquiry @octapharma .com