Final Data From the NuProtect Study Published on the Immunogenicity of Nuwiq ® in Previously Untreated Patients With Severe Haemophilia A

Octapharma announced today that the final results from the NuProtect study on the immunogenicity of Nuwiq^® in previously untreated patients (PUPs) with severe haemophilia A have been published in the leading medical journal Thrombosis and Haemostasis (Liesner RJ et al. “Simoctocog Alfa (Nuwiq^®) in Previously Untreated Patients with Severe Haemophilia A: Final Results of the NuProtect Study” https://www.thieme-connect.com/products/ejournals/abstract/10.1055/s-0040-1722623).

The NuProtect study (NCT01712438; EudraCT 2012-002554-23) was a prospective, multinational, open-label, non-controlled phase III study initiated in March 2013 to assess the immunogenicity, efficacy and safety of Nuwiq^®. The study recruited patients of any age and ethnicity at 38 sites in 17 countries and followed patients for 100 exposure days or up to 5 years. With enrolment of 110 patients, the NuProtect study was the largest clinical study to investigate a single product in true PUPs.

Of 105 evaluable PUPs with severe haemophilia A who received Nuwiq^® for the prevention and treatment of bleeding:

• 16.2% (17/105) of patients developed high-titre inhibitors
• 10.5% (11/105) of patients developed low-titre inhibitors, five of which were transient
• 26.7% (28/105) of patients developed any inhibitor

“The NuProtect study showed that there was a low risk of inhibitor development in PUPs starting treatment with Nuwiq^®,” commented Dr Ri Liesner, coordinating investigator of the NuProtect study and clinician at Great Ormond Street Hospital for Children in London, UK. “These data indicate Nuwiq^® may be an attractive option for people newly diagnosed with severe haemophilia A, who are young children and represent a vulnerable patient subset.”

The development of inhibitors to replacement factor VIII (FVIII) is a concern for doctors, as well as for patients and their families, particularly when first starting treatment. Inhibitors render FVIII therapy ineffective and limit treatment options.

These data complement the wealth of clinical experience in patients with haemophilia A receiving Nuwiq^® for the treatment and prevention of bleeding.

“Nuwiq^® was developed in a human cell line with the aim of minimising inhibitor risk in PUPs,” said Larisa Belyanskaya, Head of Octapharma’s IBU Haematology. “We are pleased to be able to share these positive data, which reflect this aim and which we hope will contribute to addressing this key treatment challenge.”

“We know that haemophilia A patients face a lifetime of treatment decisions,” commented Olaf Walter, Board Member at Octapharma. “These data highlight the potential of Nuwiq^® to reassure PUPs and their families as they start their treatment. This publication brings us closer to Octapharma’s goal of enabling all patients to lead healthy lives.”

Earlier this year the FDA approved the inclusion of the immunogenicity data from the NuProtect study in the Nuwiq^® Prescribing Information. Further publications on other analyses from the NuProtect study are planned.

Professor Anthony Chan, a co-author of the publication and Professor of Paediatrics at McMaster University, Canada, said: “Congratulations and thanks to Octapharma for developing Nuwiq, sponsoring the trial and providing a very good therapy for patients with haemophilia A.”

Octapharma would also like to extend thanks to all participating centres, as well as patients and their caregivers, for their contribution to the study.

About Nuwiq^®

Nuwiq^® (simoctocog alfa) is a 4th 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. Nuwiq^® is available in 250 IU, 500 IU, 1000 IU, 2000 IU, 2500 IU, 3000 IU and 4000 IU presentations². Nuwiq^® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups².

1. Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471.
2. Nuwiq^® Summary of Product Characteristics.

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

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.

Octapharma employs more than 9,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Haematology, Immunotherapy, and Critical Care.

Octapharma has seven R&D sites and six state-of-the-art manufacturing facilities in Austria, France, Germany, Mexico and Sweden, and operates more than 160 plasma donation centres across Europe and the USA.

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Contact information

Octapharma AG Contacts:

Octapharma AG International Business Unit - Haematology
Olaf Walter
Olaf.Walter@octapharma.com

Larisa Belyanskaya
Larisa.Belyanskaya@octapharma.com
Tel: +41 55 4512121

Ivana Spotakova
Communications Manager
ivana.spotakova@octapharma.com
Tel.: +41793474607