Ascension Healthcare Announces Positive Data from Phase 2 Study of SelectAte in Severe Haemophilia A Patients with inhibitors
Results showed co-administration of PEGLip with factor VIII led to an extended dosing interval and a significant reduction in the number of bleeding events
Demonstrates PEGLip’s potential as a prophylactic treatment for severe haemophilia A patients with a history of inhibitor development to factor VIII or with low-titre inhibitors
Data presented at the 64th American Society of Hematology Annual Meeting
LONDON, 12 December 2022 – Ascension Healthcare plc (“Ascension” or the “Company”), a clinical-stage biopharmaceutical company focused on developing and commercialising innovative therapies for haemophilia and osteoarthritis, today announces positive data from a Phase 2 open-label, multicenter trial of SelectAte, in 24 severe haemophilia A patients both with (n=5) or prone to generate (n=8) inhibitors to factor VIII and without inhibitors (n=11) as control. SelectAte is a proprietary pre-mixed combination of a recombinant factor VIII clotting protein and PEGylated liposomes (PEGLip), co-injected intravenously.
Haemophilia A patients lack sufficient factor VIII clotting factors which leaves them at risk of uncontrolled bleeding and approximately 30% develop antibodies which prevent factor VIII (FVIII) replacement treatments from working. This trial was designed to demonstrate whether FVIII, when combined with PEGLip, can be repeatedly administered to severe haemophilia A patients who are either inhibitor prone or who have low titre antibody inhibitors to FVIII, to provide coagulation correction, without generating or increasing the level of inhibitors.
Results from the study demonstrated that the clotting improvements conferred by PEGLip co-injection with standard half-life FVIII led to an extended dosing interval of, on average, once every 5.2 days for a 30IU/kg dose, compared to the normal prophylactic dosing interval for this FVIII (and similar standard half-life FVIII products), at this dose, of once every other day or 2-4 times a week for long-term prophylaxis. Current extended half-life FVIII products approved by the FDA recommend dosing between 2-3 times weekly or every four to five days but at a higher dose of 45IU/kg (average). These results were achieved in both non-inhibitor patients and also those that were both inhibitor prone and presenting with low levels of inhibitors. Despite the lower dose rate, the number of bleeding episodes was significantly reduced in all three patient groups (non-inhibitor, inhibitor-prone and inhibitor-presenting) over the six-week trial period, compared to the 6 month history preceding the study.
Professor Edward Tuddenham, Emeritus Professor of Haemophilia at University College London and member of Ascension’s Medical and Scientific Advisory Board said: “The risk of bleeding episodes still remains a significant concern for haemophilia A patients. These promising early clinical findings for SelectAte show the potential of using our PEGLip technology in conjunction with factor VIII to both extend dosing intervals and reduce the risk of bleeding.”
Biresh Roy, Chief Executive Officer at Ascension added: “These clinical results are a significant milestone for Ascension Healthcare and highlight the potential of our PEGLip technology with FVIII to provide prophylactic benefits for haemophilia A patients, including those with inhibitors.. We look forward to progressing SelectAte into late-stage clinical trials.”
Further information on the trial can be found at www.clinicaltrials.gov/ct2/show/NCT04592692.
Full details of the poster presentation:
Prophylactic Treatment of Severe Haemophilia A Patients with Inhibitors to FVIII with PEGLip-FVIII
Publication No: 2477
322. Disorders of Coagulation or Fibrinolysis: Clinical and Epidemiological: Poster II
Session Date: Sunday, December 11, 2022
Presentation Time: 6:00 PM – 8:00 PM
Location: Ernest N. Morial Convention Center, Hall D
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About Haemophilia A
Haemophilia A (“HA”) is a congenital bleeding disorder which leaves a patient at risk of uncontrolled bleeding due to a lack of blood clotting Factor VIII (“FVIII”). HA is the most common form of haemophilia, with a prevalence rate estimated at1.7 – 2.5 in 10,000 male births and can be categorised from mild (5-40% of normal circulating FVIII) to moderate (1-5% of normal), to severe (<1% of normal); of these, severe HA is prevalent in circa 0.6 to 1 in 10,000 males (World Federation of Haemophilia Global Survey 2021. The global market for haemophilia A treatments was estimated to be worth approximately $11 billion in 2021 of which it is estimated circa $2.5bn is spent on direct treatments for inhibitor patients (excluding Immune Tolerance Therapy) (Ascension Healthcare research).
SelectAte is being developed to treat severe HA patients who have developed inhibitors (neutralising antibodies) to FVIII. Approximately 30% of severe HA patients develop inhibitors to prophylactic FVIII replacement therapy, rendering such therapy ineffective. Current options for these patients are (i) Immune Tolerance Induction Therapy (ITI) to eradicate inhibitors, which is costly, lengthy and sometimes unsuccessful, and unpleasant; (ii) bypassing blood factor agents to treat or prevent bleeds on demand or (iii) non-factor agents prophylactically which can be very costly; some of these agents have an FDA “black box” safety warning (risk of blood clots and death). This is a large market that is, at present, not served by a safe, convenient, inexpensive prophylactic replacement factor treatment.
About Ascension Healthcare plc
Ascension Healthcare plc is a clinical-stage biopharmaceutical company focused on developing and commercialising innovative therapies for the treatment of haemophilia and osteoarthritis. The Company has three products in clinical development for the treatment of haemophilia A and also a range of internationally marketed products for osteoarthritis sufferers. For more information please visit: www.ascension.co.uk/
For more information contact
Ascension Healthcare plc
Biresh Roy, Chief Executive Officer
[email protected] / +44 (0)20 7291 5400
Consilium Strategic Communications
(European Media and Investor Enquiries)
Lindsey Neville, Ashley Tapp
Tel: +44 (0)20 3709 5700 / [email protected]