Octapharma shares new data at ISTH 2022 in its mission to meet the challenges faced by people with bleeding disorders

LACHEN,Switzerland,July 4,2022 --

Octapharma to present new clinical and scientific data from across Octapharma's haematology and critical care portfolios during the ISTH 2022 Congress

Data to be presented in 11 poster presentations and during two Supported Symposia

New data from the extensive clinical trial programme with Nuwiq® (simoctocog alfa) to be presented,confirming Nuwiq®'s efficacy and safety profile

The latest developments in Octapharma's haematology and critical care portfolios will be presented at the 30th International Society on Thrombosis and Haemostasis (ISTH) Congress on July 9–13,2022,in London,UK. The data will be featured in eleven poster presentations and two Supported Symposia during this major international meeting. Octapharma is proud to be a Gold Supporter of the ISTH 2022 Congress.

Poster presentations at the Congress will include data from the Nuwiq® clinical trial programme,including a pooled analysis of long-term data from clinical trials and the first presentation of the results of the global GENA-99 study of Nuwiq in real-world clinical practice. These studies confirm the proven efficacy and safety profile of Nuwiq® in patients with haemophilia A. The safety and immunogenicity results from the phase I/II study of OCTA101,a recombinant FVIII developed for subcutaneous administration,will also be presented,following the recent decision to terminate the study due to safety concerns. Updates from the clinical development programme with the four-factor prothrombin complex concentrate Octaplex® and the high-purity,double virus inactivated,lyophilized human antithrombin III concentrate Atenativ®will also be provided,demonstrating Octapharma's commitment to clinical research and development in critical care.

The following posters will be presented.

Monday July 11,18:30–19:30

PB0690. Dose optimisation in children with severe haemophilia A on long-term octanate® prophylaxis. Presenting Author: Evgeny Dmitriev

PB0672. Safety and pharmacokinetics of a subcutaneous recombinant FVIII (OCTA101) in adult patients with severe haemophilia A. Presenting Author: Sigurd Knaub

PB0559. LEX-210: a phase 3,randomized,double-blinded study of four-factor prothrombin complex concentrate in patients with acute major bleeding on direct oral anticoagulant therapy with factor Xa inhibitors. Presenting Author: Ravi Sarode

PB0560. Use of heparin-calibrated assays to estimate anti-factor Xa activity of factor Xa inhibitors (FXaI): a literature correlation analysis. Presenting Author: Ravi Sarode

PB0716. Prospective,Phase III Study of the Efficacy,Safety,and Pharmacokinetics of a Human Antithrombin III Concentrate in Congenital Antithrombin Deficiency During Surgery or Childbirth. Presenting Author: Cristina Solomon

PB0541. Freeze-dried plasma development and assessment of biochemical quality. Presenting Author: Andrea Heger

VPB0492. LEX-211 (FARES-II): a phase 3,prospective,active-control randomised study of four-factor prothrombin complex concentrate versus frozen plasma in bleeding adult cardiac surgery patients. Presenting Author: Jeannie Callum

Tuesday July 12,18:30–19:30

PB1149. Pooled analysis of long-term efficacy and safety of simoctocog alfa in previously treated patients with haemophilia A. Presenting Author: Gita Pezeshki,

PB1134. Practical utilisation of Octapharma FVIII concentrates in previously untreated and minimally treated haemophilia A patients entering routine clinical treatment – The Protect-NOW Study. Presenting Author: Susan Halimeh

PB1126. Safety and efficacy of simoctocog alfa in patients with haemophilia A in routine clinical practice (GENA-99). Presenting Author: Claire Berger

VPB0996. FiiRST-2; a prospective,randomized study of clotting factor concentrates versus standard massive haemorrhage protocol in severely bleeding trauma patients. Presenting Author: Jeannie Callum

"We are excited to share our new data and updates at ISTH,"said Olaf Walter,Board Member at Octapharma. "Despite advances in the field,managing emergency bleeding and bleeding disorders still comes with considerable challenges. We are constantly striving to address these challenges head-on and to lessen these patients' burdens to improve their day-to-day lives."

New data will also be shared during two Supported Symposia as part of Octapharma's 'Under the Spotlight' series.

A Key Factor: Aiming for All-Round Bleed Protection in Haemophilia A Saturday July 9,11:30–12:45 BST

Chair: Dr Georgina Hall,John Radcliffe Hospital,Oxford,UK

The symposium will focus on new clinical and scientific insights into the use of Nuwiq® to improve the lives of people with haemophilia A. The topics include the use of matching-adjusted indirect comparisons to assess the relative clinical impact of personalised prophylaxis with different FVIII products,and the use of combination therapy to manage bleeding in people with haemophilia A undergoing major surgery. Also presented will be new in vitro data on the binding of FVIII to platelets,and the use of novel RNA sequencing methods to help predict the risk for inhibitor development in children treated for the first time with FVIII.

Focus on Females: Patient Experience and Novel Treatment Strategies in Bleeding DisordersTuesday July 12,13:15–14:30 BST

Chair: Dr Veronica Flood,Medical College of Wisconsin,Milwaukee,USA

Women with bleeding disorders face unique challenges; many are left untreated or are diagnosed late,which can increase their bleeding risk and impact quality of life. During the symposium,the distinguished panel will discuss the challenges faced by women and girls with von Willebrand disease and haemophilia A. The speakers will illustrate the need forincreased awareness and women-specific clinical data,as well as treatment guidelines to support clinicians in making well-informed treatment decisions for female patients with bleeding disorders. New and ongoing studies with wilate® and Nuwiq® will be presented that aim to improve the management of women with von Willebrand disease or haemophilia A.

"We look forward to continuing our efforts in raising awareness around and improving the care for women with bleeding disorders,with the ultimate goal of ensuring that these patients have the ability to receive safe and effective management for their symptoms," commented Larisa Belyanskaya,Head of IBU Haematology at Octapharma.

About Octapharma

Headquartered in Lachen,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 around 10,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy,Haematology,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 180 plasma donation centres across Europe and the US.

About Nuwiq®

Nuwiq®(simoctocog alfa) is a 4thgeneration 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 280 patients with severe haemophilia A,of which were 190 previously treated patients (PTPs; 190 individuals) and 90 were previously untreated patients (PUPs) 1. Nuwiq® is available in 250 IU,500 IU,1000 IU,2000 IU,2500 IU,3000 IU and 4000 IU presentations1. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups1.

About wilate®

wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate,that undergoes two virus inactivation steps during its production.2No albumin is added as a stabiliser2. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma2. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma2. wilate® is exclusively derived from large pools of human plasma collected in approved plasma donation centres3. 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)3.

Nuwiq® Summary of Product Characteristics.

Stadler M et al.Biologicals2006; 34:281-8.

wilate® Summary of Product Characteristics.

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