Octapharma communicates an update on the development of SubQ-8, a novel subcutaneous recombinant FVIII, at ISTH 2019

Octapharma announced that an update on the clinical development plan and pre-clinical data with SubQ-8 were presented today in a scientific symposium at the 27th International Society on Thrombosis and Haemostasis (ISTH) Congress in Melbourne, Australia. SubQ-8 combines simoctocog alfa, a human cell line-derived recombinant FVIII, with a recombinant VWF fragment dimer produced in a human cell line, in an innovative approach to facilitate uptake of subcutaneous FVIII into the circulation.

Larisa Belyanskaya, Head of Octapharma’s Haematology International Business Unit, said “We are very pleased to share the latest data and clinical development plans for SubQ-8 with the haemophilia community at the ISTH Congress. We believe that subcutaneous administration of FVIII could play an important role in aiding patients to receive a potential ‘state of the art’ treatment for haemophilia A and we are very pleased with the promising preclinical data with SubQ-8 and its reception within the haemophilia community”.

Olaf Walter, Board Member of Octapharma, added that “Octapharma is committed to improving the lives of patients with coagulation disorders and we are proud to be developing a product with such potential to address a major need for people with haemophilia A. The ISTH congress coincides with the enrolment of the first patient in a phase 1/2 study of SubQ-8 in previously treated patients with haemophilia A, a key milestone in the clinical development of this medicinal product”.

The symposium, entitled “The Future of Haemophilia A: Discussing SubQ-8, a New Subcutaneous FVIII Development Based on Simoctocog Alfa”, considered how SubQ-8 might address the long-term needs of people with haemophilia A. Regular intravenous administration of FVIII poses a considerable burden to people with haemophilia A and their families. Alternative routes of administration may help to reduce this burden and improve adherence to prophylaxis.

Andreas Tiede (Hannover Medical School, Hannover, Germany) chaired the symposium and introduced the relevance of SubQ-8 within the rapidly advancing haemophilia A therapeutic field. Robert Sidonio (Emory University School of Medicine and Aflac Cancer and Blood Disorders Center, Atlanta, USA) discussed the use of FVIII therapy as a natural approach to restoring blood clotting in haemophilia A. He reviewed the proven benefits of early FVIII prophylaxis, including protection against the development of inhibitors and intracranial bleeds, reduced risk of long-term joint damage, and the challenges of intravenous administration.

Guido Kohla (Octapharma Research & Development, Berlin, Germany) explained the rationale for the development of SubQ-8 and the challenge of achieving sufficient FVIII bioavailability after subcutaneous infusion. Data in pre-clinical animal models showed high bioavailability of FVIII after subcutaneous administration of SubQ-8, and a greater than 3-fold prolongation of FVIII half-life compared with FVIII administered alone.

Andreas Tiede (Hannover Medical School, Hannover, Germany) addressed the immunogenicity of therapeutics administered subcutaneously or intravenously, concluding that there is no evidence for a difference in risk between administration routes. The immunogenicity of SubQ-8 has been studied in mice, demonstrating slower development of anti-FVIII antibodies after subcutaneous administration of SubQ-8 compared with intravenous administration of FVIII in the pre-clinical setting.

The clinical development programme for SubQ-8 was presented by Sigurd Knaub (Octapharma AG, Lachen, Switzerland). The first patient is about to be enrolled in a phase 1/2 study to assess safety and pharmacokinetics of SubQ-8 in previously treated patients with haemophilia A, and the results of this study will determine the dosing of SubQ-8 in future phase 3 studies in adults and children.

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 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
  
  Octapharma has seven R&D sites and six state-of-the-art manufacturing facilities in Austria, France, Germany, Mexico and Sweden.

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