Octapharma to present new clinical data and studies at ASH 2022 to demonstrate its continued mission to meet the challenges faced by people with bleeding disorders

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Dec 05, 2022, 07:13 ET

LACHEN, Switzerland, Dec. 5, 2022 /PRNewswire/ -- New clinical data from Octapharma's haematology portfolio, including the results of the phase 3 WIL-31 study, will be presented during the Scientific Program at the 64^th American Society of Hematology (ASH) Meeting on December 10–13, 2022.

Prophylaxis with wilate^® significantly reduced bleeding rates in children and adults with any type of von Willebrand disease (VWD) in the phase 3 WIL-31 study.
Significantly higher zero bleed rates were observed in haemophilia A patients undergoing pharmacokinetic-personalised prophylaxis with Nuwiq^® versus comparator products based on matching-adjusted indirect comparison (MAIC) analyses.
The NuPOWER study will investigate the efficacy and safety of Nuwiq^® for the management of bleeding during major surgery in patients with haemophilia A on emicizumab prophylaxis.
The ATN-06 study will investigate the efficacy, safety and single-dose pharmacokinetics of Atenativ^® in patients with congenital antithrombin deficiency undergoing surgery or childbirth.

Olaf Walter, Board Member and Head of International Business Units at Octapharma, stated, "We look forward to highlighting our continued efforts in improving the care for people with bleeding disorders." Larisa Belyanskaya, Head of IBU Haematology, added: "The positive results of the WIL-31 study support the use of regular wilate^® prophylaxis in people with von Willebrand disease of all ages and all types. The MAIC analysis strengthens the use of personalised prophylaxis with Nuwiq^® for bleed management in patients with haemophilia A. The NuPOWER study will give important insight into the use of FVIII in haemophilia A patients on emicizumab during surgery, while the ATN-06 study will provide insights into the prevention of thrombosis in people with congenital antithrombin deficiency during surgery."

Aiming for all-round bleed protection in people with haemophilia A and von Willebrand disease and prevention of thrombosis in people with congenital antithrombin deficiency:

The phase 3 WIL-31 study investigated the efficacy and safety of wilate^® prophylaxis over 12 months in people of all ages with VWD of any type. The primary endpoint was achieved, with an 84% reduction in annualized bleeding rate (ABR) compared with on-demand treatment during the prior run-in study. The median spontaneous ABR decreased by 95%. No serious drug-related adverse events or thrombotic events occurred during prophylaxis with wilate^®. The findings indicate that prophylaxis with wilate^® is effective in children and adults with VWD of all types.
In rare diseases, such as haemophilia A, it is difficult to perform head-to-head studies directly comparing different products. Instead, indirect comparison methods, such as MAIC, can be used to assess the relative treatment effects of different products across studies. A series of MAIC analyses was performed comparing the efficacy of pharmacokinetic-guided, personalised prophylaxis with Nuwiq^® with that of personalised prophylaxis regimens with three other FVIII products. The results showed that personalised prophylaxis with Nuwiq^® can lead to decreased ABRs and significantly higher rates of patients with zero bleeds compared with comparator products.
People with haemophilia A who are receiving emicizumab prophylaxis and need to undergo surgery often require additional haemostatic therapy to control bleeding during and after the invasive procedure. However, there are limited data on the safety and efficacy of using emicizumab and concomitant therapy during surgery. The NuPOWER study will therefore investigate the perioperative efficacy and safety of Nuwiq^® in patients receiving emicizumab prophylaxis who undergo major surgery. The study is planned to start in 2023.
People with congenital antithrombin deficiency have an increased risk for thrombosis, particularly during high-risk periods such as surgery or childbirth. Anticoagulation therapy (e.g., with an antithrombin concentrate) is therefore advisable in such settings. The ATN-06 study is investigating the use of Atenativ^®in people with congenital antithrombin deficiency undergoing surgery or childbirth. Site initiation has begun in 8 countries and 15 sites, and patient enrolment has started.

Therapy	Title	Poster number Date and time	Presenter
Nuwiq^®	Matching-adjusted indirect comparisons of personalised prophylaxis with simoctocog alfa versus efmoroctocog alfa, damoctocog alfa pegol and rurioctocog alfa pegol in patients with haemophilia A	#1167 Saturday, Dec 10, 2022 5:30–7:30pm	Craig Kessler
Nuwiq^®	The NuPOWER study: Nuwiq^® for perioperative management of patients with haemophilia A on emicizumab regular prophylaxis	#2481 Sunday, Dec 11, 2022 6:00–8:00pm	Sylvia Werner
wilate^®	Efficacy and safety of prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate in previously treated patients with von Willebrand disease	#3782 Monday, Dec 12, 2022 6:00–8:00pm	Robert F. Sidonio, Jr.
Atenativ^®	Atenativ, a Plasma-Derived Antithrombin Concentrate, Used in Patients with Congenital Antithrombin Deficiency Undergoing Surgery or Parturition: Study Description (NCT04918173)	#3835 Monday, Dec 12, 2022 6:00–8:00pm	Craig Kessler

"We are excited to provide updates to the bleeding disorder community at the 64^th ASH Meeting," continued Olaf Walter, "The ASH Meeting is an important platform to further demonstrate Octapharma's constant commitment to reducing the burden of living with a bleeding disorder and improving patients' lives".

About haemophilia A and VWD

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.

VWD is the most common inheritable bleeding disorder caused by deficient or defective clotting protein von Willebrand factor (VWF). Currently, recommended guidelines on the use of prophylaxis are limited. VWD affects males and females equally but is diagnosed more frequently in women due to heavy menstrual bleeding and childbirth. Guidelines for managing these issues are also inadequate.

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 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 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 factor². Nuwiq^® treatment has been assessed in nine^2-4 completed clinical trials which included 201 previously treated patients (190 individuals)² and 108 previously untreated patients³ with severe haemophilia A. Nuwiq^® is available in 250 IU, 500 IU, 1000 IU, 1500 IU, 2000 IU, 2500 IU, 3000 IU and 4000 IU presentations⁵. Nuwiq^® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups⁵.

About wilate®

wilate^® is a high-purity human VWF/FVIII concentrate, that undergoes two virus inactivation steps during its production⁶. No albumin is added as a stabiliser⁶. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma⁶. wilate^® contains a VWF triplet structure and content of large high molecular weight multimers like normal human plasma⁶. 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 VWD, when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency)⁶.