Octapharma announces the presentation of the final results from the NuProtect study of Nuwiq® (simoctocog alfa) in previously untreated patients (PUPs) at ASH 2019

Octapharma has announced that the final results of the NuProtect study will be presented by Dr Ri Liesner (Great Ormond Street Hospital for Children, London, UK) as an oral presentation at the 61^st Annual Meeting of the American Society of Hematology (ASH) 2019 in Orlando, Florida.

Dr Liesner commented that "I am excited to be representing the NuProtect study group to share these data. The data should help to inform clinical decision-making and provide valuable evidence to reassure clinicians and patients that Nuwiq^® is an effective treatment option for PUPs".

The NuProtect study is the largest prospective study of a single FVIII concentrate in true PUPs with haemophilia A. The study assessed the immunogenicity, efficacy and safety of the human cell line-derived recombinant FVIII Nuwiq^® (simoctocog alfa) in 108 PUPs. Final results on the immunogenicity, efficacy and safety of Nuwiq^® will be shared for the first time at an oral presentation on Monday 9^th December.

"We are proud to present these important data in PUPs, which showcase the clinical features of Nuwiq^® founded on its human cell line origin" said Larisa Belyanskaya, Head of IBU Haematology at Octapharma. Olaf Walter, Board Member at Octapharma, added that "The NuProtect study data add to the growing portfolio of clinical experience with Nuwiq^® and support our goal at Octapharma to improve the lives of people with bleeding disorders".

About Nuwiq^®
Nuwiq^® (simoctocog alfa) is a 4^th generation recombinant factor VIII (rFVIII) protein, produced in a human cell line without chemical modification or fusion with any other protein¹. It is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for von Willebrand factor¹. Nuwiq^® treatment has been assessed in seven completed clinical trials which included 201 previously treated patients (PTPs; 190 individuals) with severe haemophilia A, including 59 children¹. Treatment of previously untreated patients (PUPs) with Nuwiq^® was assessed in the NuProtect study. In an interim analysis of 66 patients, the cumulative incidence of high-titre inhibitors was 12.8%, and the cumulative incidence of all inhibitors was 20.8%². Nuwiq^® is available in 250 IU, 500 IU, 1000 IU, 2000 IU, 2500 IU, 3000 IU and 4000 IU presentations³. Nuwiq^® is approved in 59 countries for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups³.

About the NuProtect study
The NuProtect study (NCT01712438) was a prospective, multicentre, multinational, open-label, non-controlled phase III study to assess the immunogenicity, efficacy and safety of Nuwiq^® in patients with severe haemophilia A and without any previous exposure to FVIII concentrates or blood products containing FVIII. Patients were treated with Nuwiq^® for 100 exposure days or up to 5 years. The study was conducted at 38 sites in 17 countries. A total of 110 patients were enrolled. Of these, 108 patients were treated with Nuwiq^®.

About Haemophilia A
Haemophilia A is an X-linked hereditary bleeding disorder caused by a deficiency of factor VIII (FVIII) which, if left untreated, may lead to haemorrhages in muscles and joints and consequently to arthropathy and severe morbidity. The disorder affects around one in every 10,000 males worldwide. Prophylaxis with replacement FVIII therapy reduces the number of bleeding episodes and the risk of permanent joint damage.

About Octapharma
The vision of Octapharma is: "Our passion drives us to provide new health solutions advancing human life". 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. Our company values are Ownership, Integrity, Leadership, Sustainability and Entrepreneurship.

In 2018, the Group achieved ?1.8 billion in revenue, an operating income of ?346 million and invested ?240 million into R&D and in capital expenditures in order to ensure future prosperity. Octapharma employs 8,314 people worldwide to support the treatment of patients in 115 countries with products across three therapeutic areas:

• Haematology (coagulation disorders)
• Immunotherapy (immune disorders)
• Critical care (bleeding management and functional volume replacement)

Octapharma has seven R&D sites and six state-of-the-art manufacturing facilities in Austria, France, Germany, Mexico and Sweden.

1. Lissitchkov T et al. Ther Adv Hematol 2019; doi: 10.1177/2040620719858471.
2. Liesner R et al. Haemophilia 2018; 24:211-20.
3. Nuwiq^® Summary of Product Characteristics.