Efmoroctocog alfa(Sobi Analytics)

XTEND-Kids is the first assessment of once-weekly ALTUVOCT® prophylaxis in previously treated children Results indicate highly effective bleed protection in children under 12 years of age with severe haemophilia A STOCKHOLM, July 18, 2024 /PRNewswire/ -- Sobi® today announced the publication of full results from the Phase 3 XTEND-Kids study in The New England Journal of Medicine (NEJM) confirming the safety and efficacy profile of ALTUVOCT® (efanesoctocog alfa), in children younger than 12 years old with severe haemophilia A. The study demonstrated that fixed-dose once-weekly prophylaxis with ALTUVOCT, a new class of high-sustained factor VIII therapy, provided highly effective bleed protection in children. "Children represent a population for which it has been historically difficult to achieve effective bleed prevention," said Lynn Malec, MD, Medical Director of Comprehensive Center for Bleeding Disorders and Associate Investigator at The Versiti Blood Research Institute, and Associate Professor of Medicine and Paediatrics at The Medical College of Wisconsin. "The clearance of administered factor concentrates is greater in children than we see in adults, which leads to burdensome treatment regimens involving multiple injections per week. By providing high-sustained factor VIII activity levels consistently early in life with once-weekly dosing, ALTUVOCT has the potential to improve reported outcomes for children living with haemophilia." Published in NEJM, the pivotal XTEND-Kids study shows ALTUVOCT met primary and key secondary endpoints, which included occurrence of factor VIII inhibitors and annualised bleed rates (ABRs). Results revealed no development of factor VIII inhibitors and sustained protection from bleeding events with once-weekly dosing. These findings support the value of sustaining high factor levels with ALTUVOCT in children with haemophilia A. "The recent XTEND-Kids study further underscores the safety and effectiveness of ALTUVOCT, showcasing how high-sustained factor VIII activity can improve clinical outcomes in children," said Lydia Abad-Franch, MBA, Head of Research, Development, and Medical Affairs, and Chief Medical Officer at Sobi. "Maintaining normal factor VIII levels in the non-haemophilia range for a significant part of the week effectively reduces the occurrence of any type of bleed and enables people with haemophilia to lead more active lives. Today's announcement reinforces our enduring commitment to transforming standards of care across all ages within the haemophilia community." For patients treated per protocol (n=73), the estimated mean (95% CI) and median [interquartile range (IQR)] overall ABRs were 0.61 (0.42–0.90) and 0.00 (0.00–1.02), respectively. In 95% of cases bleeding episodes (41/43) resolved with one ALTUVOCT injection. The majority (64%) of patients had zero bleeding episodes, 84% of patients had zero joint bleeds and 88% of patients had zero spontaneous bleeds. ALTUVOCT prophylaxis provided high-sustained factor VIII activity in the non-haemophilia range (above 40%) for three days. In the study, ALTUVOCT was well tolerated. Development of factor VIII inhibitors was not detected (0% [95% confidence interval (CI)] 0–4.9]), and no adverse events led to treatment discontinuation. The most common treatment-emergent adverse events (>10%) were SARS-CoV-2 test positive, upper respiratory tract infection, and fever (pyrexia). No serious allergic reactions, anaphylaxis, or embolic or thrombotic events were reported. About Haemophilia A Haemophilia A is a rare, lifelong genetic condition in which the body does not produce enough, or makes dysfunctional, factor VIII – a protein that is essential for blood clotting. It 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. About XTEND-Kids XTEND-Kids was an open-label, non-randomised interventional, single-arm study. Participants received a weekly prophylactic dose of ALTUVOCT® for 52 weeks. XTEND-Kids evaluates efficacy, safety, and pharmacokinetics in 74 previously treated patients <12 years of age with severe haemophilia A. About ALTUVOCT® ALTUVOCT® (efanesoctocog alfa) [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is the first high-sustained FVIII replacement therapy with the potential to deliver near-normal factor activity levels for a significant part of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. ALTUVOCT builds on the established Fc fusion technology by innovatively adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. The European Commission granted Orphan Drug designation in June 2019. It is approved and marketed as ALTUVOCT by Sobi in Europe. It is approved and marketed as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the United States, Japan, and Taiwan. About the Sanofi and Sobi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of ALTUVOCT® (efanesoctocog alfa), or ALTUVIIIO™ in the US. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia, and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. About Sanofi We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people's lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the centre of our ambitions. Sanofi is listed on EURONEXT: SAN andNASDAQ: SNY Sobi® Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology, and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia, and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download:

NEJM publishes ALTUVIIIO XTEND-Kids phase 3 data supporting its potential to transform the treatment landscape for children with severe hemophilia A ALTUVIIIO provides high-sustained factor levels with once-weekly dosing in children under 12 with hemophilia A XTEND-Kids results show highly effective bleed protection in hemophilia A with no inhibitor development to factor VIII Paris, July 17, 2024 – Full results from the XTEND-Kids phase 3 study published in The New England Journal of Medicine (NEJM) highlights the efficacy, safety, and pharmacokinetic profile of ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein]. ALTUVIIIO (efanesoctocog alfa), a first-in-class, high-sustained factor VIII replacement therapy, is approved for adults and children with hemophilia A for routine prophylaxis and on-demand treatment to control bleeding episodes as well as for perioperative management (surgery). Lynn Malec, MDMedical Director of Comprehensive Center for Bleeding Disorders and Associate Investigator at The Versiti Blood Research Institute, and Associate Professor of Medicine and Pediatrics at The Medical College of Wisconsin “Children represent a population for which it has been historically difficult to achieve effective bleed prevention and these published results demonstrate an important breakthrough as we strive to optimize the standard of care. Achieving high-sustained factor activity with once-weekly dosing helps mitigate the need to make a tradeoff between the treatment burden of factor replacement therapy and efficacy, which we often witness in treating severe hemophilia.” The pivotal XTEND-Kids study published in NEJM shows ALTUVIIIO met primary and secondary endpoints, which included occurrence of factor VIII inhibitors and annualized bleed rates (ABRs). The results show no inhibitor development to factor VIII was detected with ALTUVIIIO (0% [95% confidence interval (CI)] 0–5]). The median annualized bleed rate (ABR) was 0.00 (interquartile range [IQR]: 0.00-1.02), and the estimated mean (95% CI) ABR was 0.61 (0.42–0.90) in the study of 73 patients treated per protocol. In the pediatric population, clearance of administered factor concentrates from the blood is greater than in adults, often meaning injections are needed 2-4 times per week using standard (SHL) or extended half-life (EHL) factor VIII products. Prevention of all joint bleeds is critical to maintain joint health throughout life. Eighty-two percent of the children treated with once-weekly ALTUVIIIO had zero joint bleeds, demonstrating ALTUVIIIO weekly prophylaxis has the potential to provide long-term preservation of joint health. Dietmar Berger, MD, PhDGlobal Head of Development and Chief Medical Officer at Sanofi“The XTEND-Kids data validate the connection between high-sustained factor activity levels and improved health outcomes, including joint health. Offering a treatment option that emphasizes effective bleed protection in children with hemophilia can help give families increased peace of mind when their loved ones participate in everyday activities. The results are a testament to our scientific expertise and commitment to redefine the standard of care for children living with hemophilia through ALTUVIIIO and our broader portfolio of hemophilia therapies.” ALTUVIIIO was well-tolerated in children, and no adverse events led to treatment discontinuation. The most common treatment-emergent adverse events (>10%) were SARS-CoV-2 test positive, upper respiratory tract infection, and fever (pyrexia). No serious allergic reactions, anaphylaxis, or embolic or thrombotic events were reported. About ALTUVIIIOALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] is a first-in-class high-sustained factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for adults and children with hemophilia A. In adults and adolescents, ALTUVIIIO has a 3 to 4-fold longer half-life relative to standard and extended half-life factor VIII products, providing high-sustained factor activity levels within normal to near-normal range, allowing for once-weekly administration. ALTUVIIIO is the first factor VIII therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on earlier generation factor VIII therapies. ALTUVIIIO builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides to extend its time in circulation. ALTUVIIIO is currently approved and marketed in the US, Taiwan, and Japan. On June 17, 2024, it was approved by the European Commission for the treatment and prevention of bleeds and perioperative prophylaxis in hemophilia A under the name ALTUVOCT. ALTUVIIIO is the first factor VIII therapy to receive Breakthrough Therapy Designation by the US Food and Drug Administration in May 2022, Fast Track Designation in February 2021, and Orphan Drug Designation in 2017. The European Commission granted orphan designation in June 2019. About XTEND-KidsThe XTEND-Kids study (NCT04759131) was an open-label, non-randomized interventional study of once-weekly ALTUVIIIO in previously treated patients younger than 12 years of age with severe hemophilia A. Patients (N=74) received once-weekly ALTUVIIIO (50 IU/kg) prophylaxis for 52 weeks. The primary endpoint was the occurrence of factor VIII inhibitors. Secondary endpoints included annualized bleed rates (ABR) of treated bleeds, bleed treatment, joint health, quality of life, perioperative management, pharmacokinetics, and safety. An ongoing extension study, XTEND-ed (NCT04644575) is evaluating the long-term safety and efficacy of ALTUVIIIO in previously treated patients with severe hemophilia A for up to four years. About Hemophilia AHemophilia A is a rare condition in which the ability of a person’s blood to clot properly is impaired, leading to excessive and spontaneous bleeds into joints that can result in joint damage and chronic pain, and potentially impact quality of life. Disease severity is determined by the level of clotting factor activity in a person’s blood, meaning there is a negative correlation between bleeding risk and factor activity levels. About Sanofi and Sobi collaborationSobi and Sanofi collaborate on the development and commercialization of Alprolix and Elocta/Eloctate. The companies also collaborate on the development and commercialization of efanesoctocog alfa, or ALTUVIIIO in the US, Taiwan, and Japan and ALTUVOCT™ in Europe. Sobi has final development and commercialization rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). Sanofi has final development and commercialization rights in North America and all other regions in the world excluding the Sobi territory. About Sobi®Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn . About SanofiWe are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Media RelationsSandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.comEvan Berland | +1 215 432 0234 | evan.berland@sanofi.comNicolas Obrist | + 33 6 77 21 27 55 | nicolas.obrist@sanofi.comVictor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.comTimothy Gilbert | + 1 516 521 2929 | timothy.gilbert@sanofi.com Investor RelationsThomas Kudsk Larsen |+ 44 7545 513 693 | thomas.larsen@sanofi.comAlizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.comArnaud Delépine | + 33 6 73 69 36 93 |arnaud.delepine@sanofi.comFelix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.comKeita Browne | + 1 781 249 1766 | keita.browne@sanofi.comNathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.comTarik Elgoutni | + 1 617 710 3587 | tarik.elgoutni@sanofi.comThibaud Châtelet | + 33 6 80 80 89 90 | thibaud.chatelet@sanofi.com Sanofi Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates regarding the marketing and other potential of the product, or regarding potential future revenues from the product. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, unexpected regulatory actions or delays, or government regulation generally, that could affect the availability or commercial potential of the product, the fact that product may not be commercially successful, the uncertainties inherent in research and development, including future clinical data and analysis of existing clinical data relating to the product, including post marketing, unexpected safety, quality or manufacturing issues, competition in general, risks associated with intellectual property and any related future litigation and the ultimate outcome of such litigation, and volatile economic and market conditions, and the impact that pandemics or other global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2023. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. All trademarks mentioned in this press release are the property of the Sanofi group with the exception of Sobi and ALTUVOCT. Attachment Press Release