SWEDISH ORPHAN BIOVITRUM AB Once-weekly efanesoctocog alfa met primary endpoint in phase 3 study, resulting in a clinically meaningful prevention of bleeding episodes (bleed protection)
In the key secondary endpoint, efanesoctocog alfa demonstrated superiority in prevention of bleeding episodes, showing a statistically significant and clinically meaningful reduction in annualised bleeding rate compared to prior factor VIII prophylaxis therapy
Efanesoctocog alfa is a novel and investigational factor VIII therapy designed to provide near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen
The study met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe haemophilia A receiving weekly prophylaxis with efanesoctocog alfa over a period of 52 weeks. The median annualised bleeding rate (ABR) was 0 with a mean ABR of 0.71. The key secondary endpoint was also met, demonstrating once-weekly efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy, showing a statistically significant reduction in ABR based on intra-patient comparison. Efanesoctocog alfa was well-tolerated, and inhibitor development to factor VIII was not detected. The most common treatment-emergent adverse events (˃5 per cent of participants overall) were headache, arthralgia, fall, and back pain.
"We believe once weekly efanesoctocog alfa has the potential to represent a new class of factor VIII therapy designed to provide high sustained factor VIII activity levels near normal for the majority of the week," said
Haemophilia A is a rare, genetic disorder in which the ability of a person's blood to clot is impaired due to a lack of factor VIII. Haemophilia A occurs in about one in 5,000 male births annually, and more rarely in females. People with haemophilia can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages.
"While advances have been made in the treatment of haemophilia, unmet medical needs still exist. These positive top-line data, showing a very low annualised bleeding rate, enhance efanesoctocog alfa's potential to transform haemophilia A therapy. We believe efanesoctocog alfa provides higher protection for longer duration with reduced treatment burden of once-weekly dosing, and we look forward to working with regulators to bring this therapy to patients as soon as possible," said
The data will be the basis for submission to regulatory authorities around the globe beginning this year. Submission in the EU will follow availability of data from the ongoing XTEND-Kids paediatric study, expected in 2023. Efanesoctocog alfa was granted Orphan Drug Designation by the
About phase 3 XTEND-1 study
The phase 3 XTEND-1 study (NCT04161495) is an open-label, non-randomised interventional study assessing the safety, efficacy and pharmacokinetics of efanesoctocog alfa in people 12 years of age or older (n=159) with severe haemophilia A who were previously treated with factor VIII replacement therapy. The study includes two parallel treatment arms - the prophylaxis arm, where study participants received a weekly prophylactic 50 IU/kg dose of efanesoctocog alfa for 52 weeks (Arm A), some of which were enrolled following an observation period on prophylaxis using currently marketed factor VIII replacement therapies, and an on-demand arm, where study participants received 50 IU/kg as needed for 26 weeks followed by weekly prophylaxis for another 26 weeks (Arm B).
The primary efficacy endpoint was the annualised bleeding rate (ABR) in arm A, and the key secondary endpoint was an intra-patient comparison of ABR during the efanesoctocog alfa weekly prophylaxis treatment period versus the prior prophylaxis ABR for participants in arm A who participated in study 242HA201/OBS16221, an observational study.
About efanesoctocog alfa (BIVV001)
Efanesoctocog alfa is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with haemophilia A. It builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN®[1] polypeptides to extend its time in circulation. It is the first investigational factor VIII therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. Efanesoctocog alfa is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
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Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,600 employees across
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https://news.cision.com/swedish-orphan-biovitrum-ab/r/efanesoctocog-alfa-met-primary-and-key-secondary-endpoints-in-pivotal-study-in-haemophilia-a--demons,c3521324
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