Publication of Clinical Data Demonstrating Low Immunogenicity and Excellent Efficacy of Nuwiq® in Previously Untreated Patients

Octapharma is delighted to announce the publication of interim data from the NuProtect study (GENA-05, NCT01712438), the first clinical data on treatment with Nuwiq^® in previously untreated patients (PUPs), on August 16^th 2017 as an Early View article in the internationally renowned medical journal Haemophilia.

The NuProtect study was initiated in March 2013 to investigate the immunogenicity, efficacy and safety of Nuwiq^® in PUPs with severe haemophilia A, who are at the greatest risk of developing inhibitors. The ongoing study has enrolled 110 PUPs of any age or ethnicity who will receive Nuwiq^® for up to 100 exposure days (EDs), making it one of the largest studies with a single FVIII product. Patients who previously received any FVIII concentrates or blood products containing FVIII are excluded from the study.

The recently published article describes interim results for 66 PUPs treated for at least 20 EDs, the time by which most inhibitors arise. The cumulative incidence (95% confidence interval) was 20.8% (10.7–31.0) for all inhibitors and 12.8% (4.5–21.2) for high-titre inhibitors. The study reported excellent efficacy of Nuwiq^® in the prevention of bleeds, with median annual bleeding rates of 0 for spontaneous bleeds and 2.40 for all bleeds. Nuwiq^® was also efficacious for the treatment of bleeds (92.4% of bleeds were controlled with one or two infusions) and as surgical prophylaxis (rated as “excellent” or “good” for 89% of surgical procedures). These interim results confirm the excellent haemostatic efficacy reported with Nuwiq^® in previously treated patients.

Larisa Belyanskaya, Head of IBU Haematology, said: “We are very excited by the results of the NuProtect study and pleased that these interim data are now available as a publication in the specialist peer-reviewed journal Haemophilia. This is the first study to report data from PUPs treated with a rFVIII produced in a human cell line, and the results further differentiate Nuwiq ^® from other products on the market.”

Olaf Walter, Board Member at Octapharma, added that “These data demonstrate low immunogenicity of Nuwiq ^® in PUPs, the patient group at the greatest risk of inhibitor development, and confirm the excellent efficacy observed in previously treated patients. This publication is a further important step towards Octapharma’s goal of enabling patients with coagulation disorders to live a normal life.”

Octapharma would like to thank everyone involved in the study, in particular the patients and their families, without whom this research would not be possible.

About the NuProtect (GENA-05) study

The NuProtect clinical study (NCT01712438) 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 any 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. Final data from the NuProtect study are expected to become available in 2019. More information on this trial is available at www.clinicaltrials.gov. An extension study (GENA-15, NCT01992549) will further evaluate the long-term immunogenicity, efficacy and safety of Nuwiq^® in PUPs.

About Nuwiq ^®

Nuwiq^® is a 4^th generation rFVIII protein¹, produced in a human cell line without chemical modification or fusion with any other protein². Nuwiq^® is cultured without additives of human or animal origin², is devoid of antigenic non-human protein epitopes³ and has a mean half-life of 17.1 hours^4,5 and a high affinity for the von Willebrand coagulation factor⁶. Nuwiq^® treatment has been assessed in seven completed clinical trials which included 201 PTPs^7,8 (190 individuals) with severe haemophilia A, including 59 children⁹. Nuwiq^® is approved for use in the treatment and prophylaxis of bleeding across all age groups of PTPs with haemophilia A in the EU, US, Canada, Australia, Latin America and Russia. Further worldwide submissions for Nuwiq^® are planned.

References:
1. FDA Nuwiq^® memorandum (STN 125555\0), 9 October 2014.
2. Casademunt E, et al. Eur J Haematol 2012; 89: 165-76.
3. Kannicht C, et al. Thromb Res. 2013;131:78-88.
4. Nuwiq^® European Public Assessment Report, 22 May 2014.
5. NUWIQ^® US Prescribing Information, September 2015.
6. Sandberg H, et al. Thromb Res 2012; 130: 808-17.
7. Valentino LA, et al. Haemophilia 2014; 20(Suppl. 1): 1-9
8. Lissitchkov T, et al. Haemophilia 2017; e-pub ahead of print; doi: 10.1111/hae.13251
9. Klukowska A, et al. Haemophilia 2016; 22, 232-39.

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 39%.

About Octapharma

Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. As a family-owned company, Octapharma believes in investing to make a difference in people’s lives and has been doing so since 1983; because it’s in our blood.

In 2016, the Group achieved €1.6 billion in revenue, an operating income of €383 million and invested €249 million to ensure future prosperity. Octapharma employs more than 7,100 people worldwide to support the treatment of patients in 113 countries with products across three therapeutic areas:

• Haematology (coagulation disorders)
• Immunotherapy (immune disorders)
• Critical care

Octapharma owns six state-of-the-art production facilities in Austria, France, Germany, Mexico and Sweden.

For more information visit www.octapharma.com

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Contact information

Octapharma AG
International Business Unit - Haematology
Olaf Walter
Olaf.Walter@octapharma.com
or
Larisa Belyanskaya
Larisa.Belyanskaya@octapharma.com
Tel: +41 55 4512121