Octapharma's continuing dedication to improving the lives of people with rare bleeding disorders to be showcased at ISTH 2024

• Octapharma to present new clinical and scientific findings for wilate^® and Nuwiq^® at the upcoming ISTH 2024 Congress
• New data and clinical study developments to be showcased in seven oral presentations, two posters and two Supported Symposia

LACHEN, Switzerland, June 10, 2024 -- The most recent developments from Octapharma's Haematology portfolio will be presented at the 32^nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) on June 22–26, 2024, in Bangkok, Thailand. The data will be featured in seven oral presentations, two poster presentations and two Supported Symposia during this key international meeting. Octapharma is a proud Silver Supporter of the ISTH 2024 Congress. Octapharma will also present the offer in modern solutions for acute bleeding and coagulation management in critical and intensive care.

 

Bleeding disorders, such as haemophilia A and von Willebrand disease (VWD), carry the risk of recurrent and prolonged bleeds, which can not only be life-threatening but also take a heavy toll on patients' quality of life and mental health.

The presentations at ISTH 2024 reflect Octapharma's long-term commitment to improving patient care and addressing unmet clinical needs.

"Octapharma is excited to present our latest findings and study results from across our haematology and critical care portfolios at ISTH 2024." said Olaf Walter, Board Member at Octapharma. "Bleeding and coagulation disorder patients continue to face many challenges in their day-to-day care and bleeding management. Here at Octapharma we remain dedicated to frontline research towards improving patients' lives."

Oral Presentations

Although prophylaxis is the standard of care for severe haemophilia A, it remains underutilised in VWD. WIL-31, the largest study in VWD prophylaxis, demonstrated the safety and efficacy of prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate (wilate^®) in patients with VWD. The lead investigator, Dr Robert F. Sidonio Jr., will present sub-analyses of data from the study which explore differences in bleed sites across VWD types and age groups. Prophylaxis was well-tolerated across all age groups in the 22 VWD type 3 patients who were included in the analysis population.

MOTIVATE is an ongoing, international, investigator-initiated study supported through funding from Octapharma AG, evaluating the management of haemophilia A patients with inhibitors. Dr Carmen Escuriola-Ettingshausen, one of the study's coordinating investigators, will present an interim analysis of the study. 

Haemophilic arthropathy is a serious complication of haemophilia A and early diagnosis is critical to minimise its long-term impact. A proof-of-concept study has identified a signature of several circulating small non-coding microRNAs, which may be used as biomarkers to assist in the diagnosis of haemophilic arthropathy. Future work will investigate microRNAs associated with earlier stages of the condition in a sub-study of the PROVE clinical trial.

Altered microvascular endothelial functionality has been observed in haemophilia A patients. Indeed, impaired tubulogenesis, migration and permeability of endothelial cells from haemophilia A patients has been observed, compared to those from healthy patients. New data will be presented showing that Nuwiq^® can bind to endothelial cell surfaces, improving their functionality.

FVIII physically interacts with platelets following vascular injury to ensure efficient clot formation. Different FVIII modifications may impact platelet binding and downstream signalling. Data will be presented showing that Nuwiq^® exhibits significantly higher binding to platelets compared to comparator products, and that these differences in platelet binding may impact the efficacy of different rFVIII products in the treatment of haemophilia A.

The oral presentations are scheduled at the following times:

Sunday June 23

• OC 27.5: Impact of differential binding of recombinant factor VIII concentrates to platelets on platelet clotting functionality.
  
  Presenting author: Viola Vogel. 15:45–16:00 ICT, Room 210 A-D

Monday June 24

• OC 35.1: Factor VIII regulates extracellular matrix proteins to stimulate angiogenesis and vessel stability.
  
  Presenting author: Alessia Cucci. 9:30–9:45 ICT, Room 110 A-C
• OC 36.4: miRNA expression profiles as a potential biomarker of joint and bone health in haemophilia A.
  
  Presenting author: Yesim Dargaud. 10:15–10:30 ICT, Room 111 A-C
• OC 36.5: Factor VIII is a regulator of angiogenesis and a promoter of endothelial barrier stability.
  
  Presenting author: Antonia Follenzi. 10:30–10:45 ICT, Room 111 A-C

Tuesday June 25

• OC 63.4: Bleeding Sites in von Willebrand Disease Patients on Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate – A Sub-Analysis of Data from the WIL-31 Study.
  
  Presenting author: Robert F Sidonio Jr. 15:30–15:45 ICT, Ballroom B3
• OC 61.4: Immune tolerance induction (ITI) strategies in haemophilia A patients with inhibitors – Interim analysis from the MOTIVATE study.
  
  Presenting author: Carmen Escuriola-Ettingshausen. 15:45–16:00 ICT, Ballroom B1

Wednesday June 26

• OC 73.5: Efficacy and Safety of Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Type 3 Patients with von Willebrand Disease – A Sub-Analysis of Data from the WIL-31 Study.
  
  Presenting author: Robert F Sidonio Jr. 11:15–11:30 ICT, Ballroom B4

Poster Presentations 

Poster presentations are scheduled at the following times:

Sunday June 23

• PB 0251 - Indirect comparison of prophylaxis efficacy between simoctocog alfa and efanesoctocog alfa in severe hemophilia A and their cost in the United States.
  
  Presenting author: Craig M. Kessler. 13:45–14:45 ICT, Exhibition Hall

Tuesday June 25

• PB 1102 - Covalently linked complexes of coagulation factor VIII and von Willebrand factor fragment display increased stability and half-life in animal models and retain their function.
  
  Presenting author: Barbara Solecka-Witulska. 13:45–14:45 ICT, Exhibition Hall

Supported Symposia 

Two Supported Symposia during the Congress will share new clinical and scientific data, providing updates on ongoing and new studies in the fields of haemophilia A and VWD.  

• Learn About Tomorrow's Advances Today – Breakthrough in All-Round Bleed Protection 
  
  Sunday June 23, 12:15–13:30 ICT, Room 208 
  
  Chair: Guy Young, Children's Hospital Los Angeles and Keck School of Medicine, Los Angeles, California, USA

Octapharma's Symposium on haemophilia A this year is dedicated to cutting-edge research committed to bleed protection. Speakers will present the first results from the MOTIVATE study interim analysis and introduce the PROVE study investigating bone and joint health. This symposium will also explore the critical role of FVIII binding to platelets and endothelial cells in haemophilia A.

• Next Level Insights on Prophylaxis in von Willebrand Disease – Are You Addressing Your Patient's Needs?
  
  Monday June 24, 12:15–13:30 ICT, Room 210 
  
  Chair: Craig Kessler, Georgetown University Medical Center, Washington DC, USA 

This symposium will explore the critical role of prophylaxis in severe VWD. Topics presented will include the importance of prophylaxis and how to identify patients who may benefit from prophylactic treatment. Key lessons from the WIL-31 VWD prophylaxis study will be shared as well as the important role for prophylaxis in the management of heavy menstrual bleeding in women with VWD. Optimising care during pregnancy will also be discussed and the VIP and EMPOWER studies will be introduced.

Both symposia will be open to congress participants in Bangkok and will be live streamed for participants not directly attending the Congress.  An opportunity to ask questions will be available at both symposia. 

Larisa Belyanskaya, SVP and Head of IBU Haematology at Octapharma commented:

"We are excited to present new insights from the WIL-31 data, and  will take a deep dive into the importance of prophylaxis for specific patient groups, as well as discussing important clinical and scientific questions with the goal of improving the quality of life for people with von Willebrand disease and haemophilia A."

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 nine^{1 2 3} completed clinical trials which included 201 previously treated patients (190 individuals)¹ and 108 previously untreated patients² with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1,000 IU, 1,500 IU, 2,000 IU, 2,500 IU, 3,000 IU and 4,000 IU presentations⁴. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups⁴.

About wilate®

wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production¹.

No albumin is added as a stabiliser⁵. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma¹. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma¹. wilate® is exclusively derived from large pools of human plasma collected in approved plasma donation centres⁶. wilate® is available in 500 IU and 1000 IU presentations. wilate® is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)⁶.

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 nearly 12,000 employees worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology and Critical Care.

Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 195 plasma donation centers across Europe and the US.

"Octapharma press releases are specifically for health specialist/medical media and are not for consumer press."

[1] Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471.

[2] Liesner RJ et al. Thromb Haemost 2021; 121:1400–8.

[3] Octapharma AG, data on file.

[4] Nuwiq® Summary of Product Characteristics

[5] Stadler M et al. Biologicals 2006; 34:281-8.

[6] wilate® Summary of Product Characteristics.

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