Efanesoctocog alfa once weekly met primary endpoint in phase 3 study, resulting in clinically meaningful prevention of bleeding episodes (bleed protection)
For the key secondary endpoint, efanesoctocog alfa demonstrated superiority in preventing bleeding episodes and demonstrated a statistically significant and clinically meaningful reduction in annualized bleeding rate compared to prior factor VIII prophylactic therapy
Efanesoctocog alfa is a novel and investigational factor VIII therapy designed to provide near-normal levels of factor activity for most of the week in a once-weekly prophylactic regimen
STOCKHOLM, March 9, 2022 /PRNewswire/ — Swedish Orphan Biovitrum AB (publ) (Sobi®) (STO:SOBI) and Sanofi (EURONEXT: SAN and NASDAQ: SNY) today announced positive top-line results from the pivotal Phase 3 XTEND study 1 evaluating known safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001) in previously treated patients ≥ 12 years of age with severe hemophilia A.
The study met its primary endpoint, demonstrating clinically meaningful prevention of bleeding in patients with severe hemophilia A who received weekly prophylaxis with efanesoctocog alfa for 52 weeks. The median annualized bleeding rate (ABR) was 0 with a mean ABR of 0.71. The key secondary endpoint was also met, showing that once-weekly dosing with efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy, demonstrating a statistically significant reduction in ABR based on within-patient comparison. Efanesoctocog alfa was well tolerated and no factor VIII inhibitor development was detected. The most common treatment-emergent adverse events (~5 percent of participants overall) were headache, arthralgia, falls, and back pain.
“We believe that once-weekly efanesoctocog alfa has the potential to represent a new class of factor VIII therapies designed to provide high, sustained, near-normal levels of factor VIII activity for most of the week,” he said Anders Ullman, MD, PhD, Head of Research and Development and Chief Medical Officer at Sobi. “We look forward to sharing these Phase 3 results, including physical health, pain and joint health data, at future medical meetings.”
The story goes on
Hemophilia A is a rare genetic disorder in which a person’s blood clotting is impaired due to a deficiency of factor VIII. Hemophilia A occurs in about 1 in 5,000 male births each year, less often in women. People with hemophilia can experience bleeding episodes that can cause pain, irreversible joint damage, and life-threatening bleeding.
“While advances have been made in the treatment of hemophilia, there remains an unmet medical need. These positive top-line data, demonstrating a very low annual bleeding rate, reinforce efanesoctocog alfa’s potential to transform hemophilia A therapy. We believe efanesoctocog alfa offers greater protection for longer duration with reduced treatment requirements with once-weekly dosing, and we look forward to working with regulators to bring this therapy to patients as soon as possible,” he said Dietmar BergerMD, PhD, Global Head of Development at Sanofi.
Starting this year, the data will serve as the basis for submission to regulatory authorities around the world. The EU submission is pending availability of data from the ongoing XTEND-Kids pediatric study, which is expected in 2023. Efanesoctocog alfa has been granted orphan drug designation by the US Food and Drug Administration (FDA). August 2017 and the European Commission in June 2019. The US FDA granted Fast Track Designation in February 2021. Efanesoctocog alfa is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory agency.
About the Phase 3 XTEND-1 study
The phase 3 XTEND-1 study (NCT04161495) is an open-label, non-randomized intervention study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa in subjects 12 years of age and older (n=159) with severe hemophilia A who have severe hemophilia A sufferers were previously treated with factor VIII replacement therapy. The study has two parallel treatment arms – the prophylaxis arm, in which subjects received a weekly prophylactic dose of 50 IU/kg efanesoctocog alfa for 52 weeks (Arm A), some of whom were switched to prophylaxis with the currently marketed factor after an observation period VIII replacement therapies and an on-demand arm, in which subjects received 50 IU/kg as needed for 26 weeks, followed by weekly prophylaxis for an additional 26 weeks (Arm B).
The primary efficacy endpoint was annualized bleeding rate (ABR) in Arm A, and the key secondary endpoint was an intra-patient comparison of ABR during the weekly efanesoctocog alfa prophylaxis treatment period to the prior prophylaxis ABR for participants in Arm A who it included study 242HA201/OBS16221, an observational study.
About efanesoctocog alfa (BIVV001)
Efanesoctocog alfa is a novel and investigational recombinant factor VIII therapy designed to prolong protection from bleeding with once-weekly prophylactic dosing in people with hemophilia A. It builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® Polypeptides to increase its circulation time. It is the first investigational factor VIII therapy shown to break the von Willebrand factor ceiling that 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 agency.
About the collaboration between Sobi and Sanofi
Sobi and Sanofi are collaborating on the development and commercialization of Alprolix® and Elocta®/Eloctate®. The companies are also collaborating to develop and commercialize efanesoctocog alfa, an investigational factor VIII therapy with the potential to provide patients with hemophilia A with high sustained levels of factor activity when administered once weekly. Sobi owns the ultimate development and commercialization rights in the Sobi area (essentially Europe, North Africa, Russia and most Middle East markets). Sanofi has ultimate development and commercialization rights in North America and all other regions of the world except Sobi Territory.
Sanofi is an innovative global healthcare company driven by one purpose: to hunt the wonders of science to improve people’s lives. Her team in around 100 countries is dedicated to transforming medical practice by working to make the impossible possible. Sanofi brings potentially life-changing treatment options and life-saving immunization coverage to millions of people worldwide, with sustainability and social responsibility at the heart of its ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY.
Sobi is a specialty international biopharmaceutical company transforming the lives of people living with rare diseases. Sobi offers sustainable access to innovative medicines in the fields of hematology, immunology and special care and employs around 1,600 people Europe, North Americathe middle East and Asia. In 2021, the revenue amounted to 15.5 billion SEK. Sobi (STO:SOBI) stock is listed on Nasdaq Stockholm. Learn more about Sobi at sobi.com, LinkedIn, and YouTube.
This information is information that Sobi is required to disclose under the EU Market Abuse Regulation. The information has been submitted for publication, mediated by the contact persons below 07:00 CET at March 9, 2022.
Thomas Kudsk Larsen
Head of Communications and Investor Relations
+44 7443 191 773
Global Communications Manager
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 XTEN® is a registered trademark of Amunix Pharmaceuticals, Inc
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SOURCE Swedish Orphan Biovitrum AB