LACHEN, Switzerland--(EON: Enhanced Online News)--On Sunday, 4th December, 2016 Octapharma presented interim data from the ongoing phase 3 study, NuProtect at the 58th ASH Annual Meeting & Exposition in San Diego, California. The presentation was nominated for inclusion in the 2017 North American Highlights of ASH Roadshow meeting with an abstract published in Blood (Raina Liesner et al, Blood 2016 128:327). The NuProtect Study examines the immunogenicity, efficacy and safety of treatment with human cell-line derived recombinant FVIII (Nuwiq®) in previously untreated patients (PUPs) with severe haemophilia A – those at greatest risk of inhibitor development. “We are immensely excited to be able to share these important results in PUPs with the wider Haematology community at the ASH Annual Meeting as part of Octapharma’s commitment to reduce the patient burden of living with haemophilia A,” said Larisa Belyanskaya, Head of IBU Haematology at Octapharma AG.
The well-attended talk, entitled “Inhibitor Development in Previously Untreated Patients with Severe Hemophilia A Treated with Nuwiq®, a New Generation Recombinant FVIII of Human Origin”, was presented by the principal investigator, Professor Raina Liesner (Great Ormond Hospital for Children NHS Trust Haemophilia Centre, London, UK) who shared this new data with Nuwiq®, a 4th generation recombinant factor VIII (rFVIII) produced in human cells without chemical modification or protein fusion. The presentation focused on immunogenicity of Nuwiq® in 66 haemophilia A patients who have received at least 20 days of treatment in the ongoing NuProtect study, with no previous exposure to FVIII concentrates or other blood products. The cumulative incidence (95% confidence interval) of all inhibitors was 20.8% (10.7–31.0); 12.8% (4.5–21.2) for high-titre inhibitors and 8.4% (1.3–15.6) for low-tire inhibitors. These data were reported as part of a pre-planned interim analysis for the NuProtect study, which plans to ultimately evaluate at least 100 PUPs, making it one of the largest studies with a single FVIII concentrate.
“Our goal was to design a rFVIII with reduced immunogenic potential. We are now very proud to present this significant clinical data with Nuwiq® which we believe validates our approach in the most vulnerable patient population, and thank all the patients and investigators for their participation in the study,” said Olaf Walter, Board Member at Octapharma AG.
About GENA-05 (NuProtect)
The GENA-05 clinical study (NuProtect) is a phase 3, open-label, interventional clinical study being conducted across 38 centres, to evaluate at least 100 previously untreated patients (PUPs) with severe haemophilia A of all ages and ethnicities enrolled for study up to 100 exposure days (EDs) or 5 years maximum. Patients treated previously with FVIII concentrates/blood products containing FVIII are excluded. The primary objective is to assess the immunogenicity of Nuwiq® by determining inhibitor activity using the Nijmegen-modified Bethesda assay at a central laboratory. More information on this trial is available at: www.clinicaltrials.gov (registration number NCT01712438).
About Haemophilia A
Haemophilia A is an X-linked hereditary disorder caused by FVIII deficiency which if left untreated leads to haemorrhages in muscles and joints and consequently to arthropathy and severe morbidity. FVIII replacement prophylactic treatment reduces the number of bleeding episodes and the risk of permanent joint damage. This disorder affects one in every 5,000 to 10,000 men worldwide. Globally, 75% of haemophilia cases are left undiagnosed or untreated. The development of neutralising FVIII antibodies (FVIII inhibitors) against infused FVIII represents the most serious treatment complication. The cumulative risk of FVIII inhibitor development is reported to be currently up to 38%.
Headquarted 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 over 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)
- Critical care
Octapharma owns five state-of-the-art production facilities in Austria, France, Germany, Sweden and Mexico.