Business Wire

Final results from the NuProtect study demonstrating efficacy of Nuwiq® (simoctocog alfa) in previously untreated patients (PUPs) presented at ASH 2019

Share

Octapharma has announced today that the final results from the NuProtect study, the largest prospective study of a single factor VIII (FVIII) product in true PUPs with severe haemophilia A, were presented today in an oral presentation at the American Society of Hematology (ASH) 2019 Annual Meeting in Orlando, Florida.

The NuProtect study was a prospective, multinational, open-label, non-controlled phase III study to assess the immunogenicity, efficacy and safety of Nuwiq® (simoctocog alfa), a human cell line-derived recombinant FVIII, in PUPs with severe haemophilia A. Dr Ri Liesner from Great Ormond Street Hospital for Children, London, UK, presented for the first time the final data on the immunogenicity, efficacy and safety of Nuwiq® (simoctocog alfa) in PUPs followed for up to 100 exposure days.

Dr Liesner, the Principal Investigator of the study, commented that “the NuProtect study has made a valuable contribution to our understanding of factors affecting inhibitor development in haemophilia A and should help to inform treatment decisions in PUPs.

When treating PUPs with severe haemophilia A, the development of neutralising antibodies against FVIII is the most significant concern. In 105 patients in the NuProtect study, the cumulative incidence of high-titre inhibitors was 17.6% and the cumulative incidence of all inhibitors was 27.9%. Dr Liesner discussed these results in the context of data from the SIPPET study, in which the cumulative incidence of high-titre inhibitors was 28.4% in patients treated with recombinant FVIII products derived from hamster cell lines, and 18.6% in those treated with plasma-derived FVIII products.

Interestingly, an analysis of the underlying F8 gene mutation demonstrated that no patients with non-null mutations developed inhibitors to Nuwiq® in the NuProtect study.

The data suggest that Nuwiq® has the potential for low immunogenicity in PUPs, with a profile more similar to plasma-derived FVIII products than to recombinant FVIII products derived from hamster cells”, commented Dr Liesner. “This relatively large clinical data set provides support for the use of Nuwiq® in PUPs”.

The data also demonstrated the efficacy and safety profiles of Nuwiq® for the prevention and treatment of bleeding in PUPs. Efficacy in the prevention of spontaneous bleeding events was rated as “excellent” in all 50 patients receiving continuous prophylaxis with Nuwiq®. On-demand treatment was rated as “excellent” or “good” for 93% of bleeding episodes, and efficacy as surgical prophylaxis was rated as “excellent” or “good” for 95% of 19 rated surgeries. Nuwiq® was well-tolerated, with only one possibly treatment-related serious adverse event (a mild rash requiring hospitalisation) reported by investigators, which resolved and treatment was continued.

Dr Larisa Belyanskaya, Head of IBU Haematology at Octapharma, said that “we are proud to be able to communicate such exciting data for Nuwiq® in this challenging patient group. This landmark study adds to the broad clinical experience with Nuwiq® and represents an important step in demonstrating the value of Nuwiq® for patients with haemophilia A at all clinical stages, from the very first treatment to long-term protection”.

Olaf Walter, Board Member at Octapharma, added that “We believe that the immunogenicity profile of Nuwiq® reflects its human cell line origin, and more broadly the foundations of Octapharma’s approach to using human proteins to improve the lives of patients. These data demonstrate the value of our strategy and show Octapharma’s ongoing commitment to the bleeding disorders community”.

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 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. 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 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.

Contact information

Octapharma AG:

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

About Business Wire

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Former HSBC Group COO Andy Maguire Joins Thought Machine as New Chair23.9.2020 11:00:00 EESTPress release

Thought Machine, the cloud native core banking technology company today announces the appointment of Andy Maguire as Chair. Maguire, former Group Chief Operating Officer of HSBC, joins Thought Machine effective immediately. At HSBC, Maguire led the bank’s IT, business operations, change support, procurement and real estate services. Prior to HSBC, Maguire was Boston Consulting Group’s managing partner for the UK and Ireland, and a member of their global executive committee. Maguire’s appointment comes at a time of significant momentum for Thought Machine, which recently closed its series B round at $125m, the fourth largest fundraising round in UK fintech this year. Furthermore, Thought Machine has added more than 150 employees in 2020 and has made a number of key senior hires in Q2 including Nicole Jude as Chief People Officer. Nicole recently led HR and people functions at Diligent Corporation and Bloomberg. The appointment of Maguire happens against the backdrop of banks around the

Arthur D. Little Announces New Board of Directors23.9.2020 11:00:00 EESTPress release

Following the completion of its ninth Partners’ and Shareholders’ Meeting since becoming a private partnership again in 2011, Arthur D. Little (ADL) today announces the new members of its Board of Directors. The Shareholders’ Meeting also re-elected Ignacio García Alves to serve a new tenure of three years as Chief Executive Officer. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200923005336/en/ Arthur D. Little Board of Directors (Photo: Business Wire) The results of the 2020 digital elections for the Board of Directors, with each member to serve a tenure of three years, were as follows: Ignacio García Alves (Chairman) Rodolfo Guzman Yusuke Harada Thomas Kuruvilla Bernd Schreiber Ulrica Sehlstedt Nick White. Held this year as a digital event that included over 100 participants, the Partners’ and Shareholders’ Meeting was an opportunity to celebrate the company’s ongoing success and resilience in the face of an exceptionall

ABP Publishing, a European Market Leader Audiobook Publisher, Rose Superior to Coronacrisis and Increased Sales 65%23.9.2020 11:00:00 EESTPress release

ABP Publishing shares the results of the second quarter. They show a significant rise in popularity and interest to audiobooks. Based on the financial reports for the second quarter, the sales increased 65% compared with the same period of the previous year. The most impressive results have been achieved in the French market with doubled sales. Noteworthy, during the quarantine, the structure of the listeners' interests has changed. For instance, the audiobooks in the category Relationships and parenting were bought by 50% more often than before the lockdown. The listeners who had to spend more time with their families were searching for ways of comfortable and mutually respected communication with their significant others. As judged by reviews, ABP Publishing audiobooks were immensely helpful in such an unusual situation. Speaking of listeners' reviews, the average merit of ABP Publishing audiobooks at Audible is more than 4.5 out of 5.0. Moreover, the number of users have completed l

Servify Raises $23 Million in Series C, Led by Iron Pillar23.9.2020 10:45:00 EESTPress release

Servify , the device lifecycle management platform that integrates multiple OEM Brands and their sales and service ecosystem to deliver great after-sales service experience today announced that they have raised $23M in Series C financing. This round was led by its existing investor Iron Pillar, an India focused venture growth investor from its recently concluded Top Up Fund. In addition to all the existing investors such as Blume, Beenext and Tetrao SPF, this funding round also saw participation from funds managed by Global Alternatives Investor 57 Stars, multiple strategic investors like Sparkle Fund (an investment arm of DMI Finance), SF Roofdeck Capital LLC (Investment arm of Silicon Valley Investment Bank FT Partners), Go Ventures (investment arm of Go PLC, a Maltese integrated telecommunications company) and Madhu Kela Family Office. Servify’s existing Venture Debt provider Trifecta Capital also participated in the round. Sreevathsa Prabhakar, Founder, Servify said, “It is very sa

ARTIFICIAL INTELLIGENCE and CLOUD: The First Edition of the Second Level Master’s From Politecnico di Torino and Reply Will Begin in January 202123.9.2020 10:30:00 EESTPress release

Enrolment for the Politecnico di Torino Master’s in "Artificial Intelligence & Cloud: Hands-on Innovation", created by Reply, is now open. The unique educational programme has been developed to offer highly qualified students a professional specialization in the IT sector through its most innovative technologies: artificial intelligence and cloud. Starting in January 2021, the Master’s will last 12 months. Modules in the first semester - which will be taught on campus at Politecnico di Torino - will alternate between professors from Politecnico di Torino and Reply specialists, in a combination of theoretical lessons and development experiences. In the second semester students will carry out a 6-month work project at Reply, where they will be able to put into practice the knowledge acquired in the classroom by working within teams on live projects. The entire course is in English and open to international entrants. The first edition of the Master’s will be open to students with a Master

Solvay Again Wins Patent Case in Germany23.9.2020 10:00:00 EESTPress release

Solvay has won another patent infringement case in Germany against Neo Chemicals & Oxides Ltd.. In a judgment released on September 3, 2020, the Düsseldorf Regional Court concluded that Neo’s commercialization of certain Cerium/Zirconium based mixed oxides had infringed Solvay’s patent EP 1527018. The infringement judgment is still subject to Neo’s right to appeal, but enforceable immediately under the condition of the provision of a security while the European Patent Office has confirmed the validity of the patent in two instances. The Court ordered Neo to stop the infringement, meaning that Neo must not commercialize its products covered by the Solvay patent in Germany any longer. Neo has also been ordered to recall the infringing products which are still kept by its customers. Solvay has taken steps to enforce the judgment. Neo was previously held liable for numerous violations of Solvay’s patents in the rare earth field, including unappealable judgments handed down by superior cour

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom