Business Wire

Octapharma present new data on the benefits of Nuwiq® in patients with haemophilia A at the World Federation of Hemophilia 2018 World Congress

Share

Octapharma announced today that new data on the benefits of Nuwiq® in patients with haemophilia A were presented during a symposium at the recent World Federation of Hemophilia (WFH) 2018 World Congress in Glasgow, UK. Nuwiq® is a 4th generation human cell line-derived recombinant FVIII (rFVIII) approved for the prevention and treatment of bleeding episodes in patients of all ages with haemophilia A. The symposium, entitled ‘Going further to meet clinical needs: New data with Nuwiq ® ▼ (simoctocog alfa; human-cl rFVIII) from clinical trials and real-world experience’, focused on the challenges within the current haemophilia treatment landscape and opportunities to improve patient care.

The symposium, chaired by Craig Kessler, addressed the reality that no two haemophilia A patients are the same. An optimal treatment approach should therefore reflect each individual patient’s profile. Striving towards this goal, two approaches to using pharmacokinetic (PK) data to personalise prophylaxis with Nuwiq® were presented in the symposium. John Pasi (The Royal London Hospital, London, UK) shared a summary of the NuPreviq study, which uses an individual’s PK profile to optimise the treatment plan1. This individualised dosing approach enabled over half (57%) of patients in the NuPreviq study to reduce dosing with Nuwiq® to twice weekly or less, whilst maintaining effective bleed protection (median annualised bleeding rate [interquartile range]: 0 [0, 1.9]) for all bleeds). During personalised prophylaxis with Nuwiq®, 83% of patients were spontaneous bleed free. A second approach, using WAPPS (Web Accessible Population Pharmacokinetics Program), was presented by Stacy Croteau (Boston Children’s Hospital, Boston, USA). This approach uses the PK analysis of a group of people to predict the optimal treatment for an individual, and provides a flexible approach to dosing that requires fewer samples. A Nuwiq®-specific WAPPS model is available and is based on PK data from 114 patients, including 26 children, treated with Nuwiq®. These approaches represent promising steps towards achieving personalised haemophilia care.

For previously untreated patients (PUPs), the risk of inhibitor development remains the greatest concern with haemophilia treatment. Ellis Neufeld (St. Jude Children’s Research Hospital, Memphis, USA) presented an overview of interim data from the NuProtect study, which is investigating Nuwiq® in PUPs2. The interim data indicate a cumulative incidence of high-titre inhibitors of only 12.8% in patients treated with Nuwiq®.The presentation also included data from a recent sub-analysis of inhibitor development based on patients’ F8 gene mutation status3. This analysis demonstrated a cumulative inhibitor incidence of 26.7% in patients with high-risk mutations, whilst no patients with low-risk mutations developed inhibitors. Dan Hart (The Royal London Hospital, London, UK) presented a novel genomic approach to further analyse data from the NuProtect study, with the aim of predicting inhibitor risk using transcriptional profiles.

Ri Liesner (Great Ormond Hospital for Children, London, UK) presented real-world data on the use of Nuwiq® for immune tolerance induction (ITI), which remains the only proven strategy to eradicate inhibitors. Three of the five patients in the cohort have achieved eradication of inhibitors, while the other two remain on ITI and show continued decline in inhibitor levels.

Larisa Belyanskaya, Head of Octapharma’s IBU Haematology stated “we at Octapharma are delighted with the excellent data presented at WFH 2018, which build on the growing wealth of positive experience with Nuwiq ® . The broad range of data demonstrate the expanding value of Nuwiq ® in clinical practice and highlight Octapharma’s commitment to ‘going further’ to address the developing demands of haemophilia A patients”. Olaf Walter, Board Member at Octapharma, added “The data presented at the symposium build on a broad portfolio of clinical experience with Nuwiq ® . The WFH congress is a key platform for sharing information in the haemophilia field and Octapharma is proud to present new data on Nuwiq to the international haemophilia community at this meeting”.

About Nuwiq ®

Nuwiq® is a 4th generation rFVIII protein4, produced in a human cell line without chemical modification or fusion with any other protein5. Nuwiq® is cultured without additives of human or animal origin5, is devoid of antigenic non-human protein epitopes6 and a high affinity for the von Willebrand coagulation factor7. Nuwiq® treatment has been assessed in seven completed clinical trials which included 201 PTPs8,9 (190 individuals) with severe haemophilia A, including 59 children10. 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.

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

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 2017, the Group achieved €1.72 billion in revenue, an operating income of €349 million and invested €287 million to ensure future prosperity. Octapharma employs around 7,700 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 has seven R&D sites and six state-of-the-art manufacturing facilities in Austria, France, Germany, Mexico and Sweden.

For more information visit www.octapharma.com

References:

1. Lissitchkov T, et al. Haemophilia 2017;23:697-704.

2. Liesner R, et al. Haemophilia 2018;24:211-220.

3. Liesner R, et al. Blood eLetter 2018; available at http://www.bloodjournal.org/content/early/2017/08/02/blood-2017-06-791756

4. Lieuw K. J Blood Med 2017; 8: 67–73.

5. Casademunt E, et al. Eur J Haematol 2012; 89: 165-76.

6. Kannicht C, et al. Thromb Res. 2013; 131: 78-88.

7. Sandberg H, et al. Thromb Res 2012; 130: 808-17.

8. Valentino LA, et al. Haemophilia 2014; 20(Suppl. 1): 1-9.

9. Lissitchkov T, et al. Haemophilia 2017; 23: 697–704.

10. Klukowska A, et al. Haemophilia 2016; 22, 232-39.

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

About Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

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

The LYCRA Company Launches Annual Fall Denim Co-Marketing Program17.9.2019 09:00:00 CESTPress release

The LYCRA Company, owner of the LYCRA® brand family of fibers, announced today that it has launched a global co-branded marketing program to promote denim jeans made with its LYCRA® dualFX® technology. The marketing program will premiere on Refinery29, a leading global media and entertainment company for young women. Patented LYCRA® dualFX® technology combines two LYCRA® brand stretch fibers to add extra flexibility and bounce back recovery to denim. This means jeans hold their shape all day, every day resisting bag and sag while delivering exceptional comfort and fit. Inspired by the two fibers in LYCRA® dualFX® technology, diverse pairs of real women are featured in some of this year’s custom content campaign created by Refinery29. Whether it is a night on the town, on the dance floor or simply hanging out together, these women demonstrate how jeans with LYCRA® dualFX® technology deliver a comfortable, lasting fit for all body types regardless of their shape or size. The annual fall

Enapter Opens Serial Fabrication Facility for Modular Hydrogen Electrolysers17.9.2019 08:00:00 CESTPress release

Enapter, a manufacturer of modular hydrogen electrolysers, announced today the unveiling of its new serial fabrication facility in Pisa. The revamped manufacturing process will increase Enapter’s production capacity eightfold and reduce electrolyser costs by more than 20 percent. These improvements can make onsite hydrogen generation an affordable reality for a multitude of applications worldwide, including energy storage, backup power, power-to-heat, industrial hydrogen production and hydrogen-based mobility. Hydrogen generation has the potential to play a key role in the clean energy transition but has been limited by cost and scalability. Enapter’s EL 2.0 electrolyser addresses these challenges through its patented Anion Exchange Membrane (AEM) technology. With a simple balance of plant and no noble metals needed, Enapter’s AEM reduces costs and increases lifetime performance of hydrogen generation. Serial fabrication allows Enapter to deploy the current EL 2.0 hydrogen generator to

Seoul Semiconductor: Future Proof Human Centric Office-Lighting With SunLike is Already Here17.9.2019 08:00:00 CESTPress release

At Arlandastad in Stockholm, Sweden, the global technology supplier Schaeffler Group has its Nordic headquarters in a four-storey building being renovated in stages. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190917005268/en/ SunLike luminaire made by LedLuks (Photo: Business Wire) The first floor to be opened after renovation shows carefully designed new lighting environments with the latest LED-technology, SunLike from Seoul Semiconductor. The ergonomically designed LED-luminaires are Plain Spectra from Annell, designed by well-renowned lighting designer Kai Piippo and produced by Ledluks. The lighting solution with the pleasant and healthy light was chosen by the architect for the project, VIRA Architects in Stockholm. Lighting designers were Lena Sjöström Larsson from VIRA Architects in cooperation with lighting supplier Annell. The result is called Human Centric Lighting. SunLike™ offers great possibilities, similar

New nCipher HSM as a Service Delivers High-Assurance Security for Organizations Adopting Cloud-First Strategies17.9.2019 07:01:00 CESTPress release

nCipher Security, an Entrust Datacard company, announces nShield as a Service, a cloud-based hardware security module (HSM) service that allows organizations to protect sensitive data and applications and helps meet compliance mandates – simply and efficiently, using on-demand cryptography. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190917005391/en/ How nCipher Security nShield as a Service works (Graphic: Business Wire) “Organizations embracing cloud-first strategies require cloud-first encryption,” said Peter Galvin, vice president of strategy, nCipher Security. “This means remote, automated management at scale, and flexible access control both in the cloud and onsite. nShield as a Service makes it easy to adopt a secure, multi-cloud encryption strategy using the same nShield HSMs organizations deploy onsite today.” nShield as a Service is ideal for cloud-first strategies, selective cloud migration, or adding HSM capac

Netsam boosts metro network capacity with ADVA technology17.9.2019 07:00:00 CESTPress release

ADVA (FSE: ADV) today announced that Netsam has deployed its FSP 3000 and FSP 150 to dramatically expand its metro network and answer fierce data demand. Featuring the ADVA FSP 3000 open line system (OLS) with micro-ROADMs and ADVA FSP 150 demarcation technology, the new solution gives the Swedish communication service provider (CSP) the power to quickly and easily turn up new high-value services. The flexible ROADM-based photonic layer ensures Netsam’s fiber resources are used with optimal efficiency. With this and the ADVA FSP 150 deployed at customer premises, Netsam is able to provide SLA-based business services with speeds up to 100Gbit/s. ADVA’s partner NetNordic, a leading solutions integrator in Scandinavia, also played a key role in the project. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190917005130/en/ Netsam has deployed the FSP 3000 and FSP 150 to quickly and easily turn up new high-value services (Photo: Bu

Riversand Technologies Appoints New EVP to Lead Growth in Europe and Middle East17.9.2019 05:00:00 CESTPress release

Riversand Technologies, a leading software provider of Cloud-native SaaS Master Data Management (MDM) and Product Information Management (PIM), announces the appointment of Mr. Mikkel Jensen as executive vice president for Europe and the Middle East regions. Mikkel will be responsible for overseeing sales and revenue growth in Europe and the Middle East and managing these regions’ operations. He has more than 18 years of experience in Master Data Management. Previously, he held a variety of roles with increasing responsibility at Stibo Systems, including EVP, and VP of Product Strategy. “We are delighted to bring onboard a leader of the caliber of Mikkel with his depth and breadth of knowledge, to head Riversand’s European and Middle East regions,” says Upen Varanasi, founder and CEO at Riversand. Riversand has steadily increased its footprint in Europe over the past few years, successfully establishing itself as a Product Information Management leader serving top European brands acros