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

HighRes Biosolutions and Novo Nordisk partner to transform drug discovery in automated Lab of the Future24.1.2020 17:00:00 EETPress release

HighRes Biosolutions and Novo Nordisk A/S today announced that they will collaborate to design a state-of-the-art robotic platform for high-throughput biologics engineering and characterisation. The integration of a multi-system robotics platform with sophisticated software control architecture will enable a digital transformation and accelerated discovery for Novo Nordisk’s peptide and large molecule therapeutics research. “In order to maintain our leadership within biologics engineering we are investing in a fully automated and digital design platform. This investment is linked to our build-up in AI and advanced analytics and demonstrates Novo Nordisk’s commitment as a world leading biologics research company,” said Lars Fogh Iversen, senior vice president, Global Research Technologies, Novo Nordisk. The platform will comprise several highly automated and integrated cells for production, characterisation and functional analysis of thousands of biologic compounds every month. Its scop

Conquest Invests in 5 Greenfield Onshore Wind Parks Totalizing 43MW in the Flanders Region24.1.2020 15:37:00 EETPress release

Conquest, acting through the Conquest Sustainable Infrastructure fund, announced the transaction was closed after an exclusive agreement had been reached with ENGIE Electrabel. Conquest acquires 51% of a portfolio expected to provide 111 GWh per annum of renewable power representing the annual electricity consumption of about 32,000 Belgian homes from early 2021 onwards. The projects benefit from 15 to 20-year green certificates local remuneration schemes, and thus generate yearly secured revenues. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200124005221/en/ Credit: Electrabel While the energy and digital transitions continue to drive the infrastructure sector, Conquest keeps building its diversified sustainable real asset portfolio in Western European markets. The success lies in years of collaboration and long-lasting partnerships with fellow industry experts who can notably facilitate access to new growth opportunities

Fluence Expands EMEA Footprint with Horticulture Partners in Papenburg, Germany24.1.2020 14:00:00 EETPress release

Fluence by OSRAM (Fluence), a leading global provider of energy-efficient LED lighting solutions for commercial cannabis and agriculture production, announced today it is expanding its presence in the Emsland region of Germany through partnerships with growers and other horticulture specialists operating out of Papenburg. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200124005109/en/ Friedrich Schulz’s 5.5 hectare farm—which grows herbs year-round in addition to lettuce, peppers and chives—utilizing VYPR 2p fixtures. (Photo: Business Wire) Given Papenburg’s decades-long history of attracting horticulture experts, Fluence’s innovative lighting solutions for horticulture are a natural fit for the region’s tomato, cucumber, leafy greens and herb growers. Forging connections with Papenburg’s horticultural leaders Elektro Evers, an engineering firm specializing in greenhouse technology, is Fluence’s exclusive systems integrator

HCL Technologies Honors Global Goodwill Champions at the 2020 World Economic Forum in Davos24.1.2020 12:47:00 EETPress release

HCL Technologies (HCL), a leading global technology company, today presented its Wave Maker Awards 2020, commemorating global goodwill champions from around the world. The award ceremony, which was held at the HCL Pavilion in Davos, celebrated organizations and individuals passionately committed to such noble causes as diversity & inclusion, education & community upliftment, environment & sustainability, and technology for change. Special guest at the awards was Australian cricket legend Glenn McGrath, who is also founder of the McGrath Foundation, which provides support for Australian women diagnosed with breast cancer and their families. The award recipients were; Lois Auta, Founder and Executive Director of Cedar Seed Foundation, which supports persons with disabilities to participate in all aspects of life; Chido Govera, Founder and Director of The Future of Hope Foundation (TFoHF), which supports marginal and vulnerable members of society through entrepreneurial and self-developme

Ipsen’s palovarotene clinical program in fibrodysplasia ossificans progressiva reaches prespecified interim analysis futility criteria24.1.2020 09:00:00 EETPress release

Regulatory News: Ipsen (Euronext: IPN; ADR: IPSEY) today announced the decision to pause dosing patients in the global Phase III (PVO-1A-301) study designed to evaluate the efficacy and safety of palovarotene in patients with fibrodysplasia ossificans progressiva (FOP), as well as the ongoing Phase II (PVO-1A-202/204) extension studies. In both the Phase III and Phase II extension studies, palovarotene is dosed both chronically (daily) and episodically (during flare-ups). The decision to pause dosing patients in the trial is based on results of a futility analysis reviewed by the Independent Data Monitoring Committee (IDMC) as part of the prespecified interim analysis. The results of a futility analysis indicated that the Phase III FOP trial was unlikely to meet its primary efficacy endpoint (annualized change in new HO volume as compared with Natural History Studyi) upon completion. Despite the results of the prespecified interim analysis, signals of encouraging therapeutic activity w

Amazentis Announces the Launch of Timeline™ Cellular Nutrition at the World Economic Forum in Davos23.1.2020 23:56:00 EETPress release

Amazentis announces the debut of Timeline, a next generation nutrition product designed to promote healthy aging, as part of the Swiss Food & Nutrition Valley in the House of Switzerland at the World Economic Forum in Davos, Switzerland. Given the increase in the aging population globally -- according to a 2019 Deutsche Bank study, there are more people on Earth older than sixty five than younger than five for the first time -- it is imperative for consumers to take proactive measures to maintain a high quality of life, for as long as they live. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200123005820/en/ (Photo: Business Wire) Timeline contains the proprietary nutrient Mitopure™, a highly pure form of Urolithin A. After a decade of rigorous research by leading scientists around the world, Mitopure has been shown to help counter age-associated cellular and muscular decline by revitalizing the mitochondria, the powerplants

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