Eftrenonacog alfa

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

Eliem to focus on advancing TNT119, an anti-CD19 antibody designed to treat a broad range of autoimmune diseases, including systemic lupus erythematosus, immune thrombocytopenia and membranous nephropathy Post-close cash and cash equivalents of $220 million expected to fund operations into 2027, to enable the potential attainment of key clinical and development milestones for TNT119 Announces the appointments of Dr. Aoife Brennan as President, Chief Executive Officer and Director, and Dr. Jan Hillson as Senior Clinical Advisor; Dr. Stephen Thomas, former Tenet CEO, appointed to Eliem’s Board of Directors SEATTLE and CAMBRIDGE, United Kingdom, June 27, 2024 (GLOBE NEWSWIRE) -- Eliem Therapeutics, Inc. (Nasdaq: ELYM) (“Eliem”), today announced the closing of its acquisition with Tenet Medicines (“Tenet”). The transaction closed on June 27, 2024. Following the closing, Eliem will focus on developing therapeutics for autoimmune-driven inflammatory diseases, including advancing TNT119, an anti-CD19 antibody designed for a broad range of autoimmune diseases, including systemic lupus erythematosus, immune thrombocytopenia and membranous nephropathy. Concurrent with the closing of the acquisition, Eliem completed a $120 million private placement of its common stock with a syndicate of new and existing institutional life science investors, including RA Capital Management, Deep Track Capital, Boxer Capital, Janus Henderson Investors, Pontifax and Samsara Biocapital. Following the close of the acquisition and the private placement, Eliem has total cash and cash equivalents of approximately $220 million. Eliem expects this will be sufficient to fund the combined company’s planned operations into 2027 and to enable the potential attainment of key clinical and development milestones for TNT119. Eliem’s stockholders approved the issuance of shares of Eliem common stock in the transactions on June 26, 2024, along with the other proposals presented at the meeting. “Today marks a transformative milestone for Eliem Therapeutics as we strive to become a leading immunology and inflammation company focused on developing novel treatments for a broad range of autoimmune diseases,” stated Andrew Levin, Executive Chairman of Eliem. “With the concurrent close of our $120 million private placement, we now have a robust balance sheet with approximately $220 million in cash and cash equivalents and are poised to advance the development of TNT119, our lead anti-CD19 antibody, through multiple milestones and for several autoimmune diseases. We look forward to initiating Phase 2 clinical trials of TNT119 later this year.” Levin continued: “We are also thrilled to welcome Aoife and Jan to Eliem Therapeutics. Throughout our comprehensive search to expand our leadership team, it became evident that Aoife and Jan were the ideal candidates, each with exceptional experiences and impeccable track records to guide Eliem following its successful acquisition of Tenet Medicines. I would also like to welcome Stephen Thomas, the former CEO of Tenet, as a new member of our Board. We all look forward to working with Stephen.” “I am thrilled to take the role of CEO at Eliem at this exciting time in the Company’s history,” said Dr. Aoife Brennan, CEO of Eliem Therapeutics. “The successful acquisition of Tenet Medicines and our strong balance sheet position us well to maximize the potential of TNT119, our lead anti-CD19 antibody, and to develop novel treatments for a broad range of autoimmune diseases. With a team of dedicated professionals and the support of our investors, I am confident that we can make significant strides in advancing our clinical and development milestones. I look forward to leading Eliem Therapeutics into its next phase of growth and innovation, and most importantly, making a difference in the lives of patients suffering from autoimmune diseases.” TNT119 is an anti-CD19 antibody designed to achieve broad and deep depletion of pathogenic B-cells with a favorable tolerability pro convenient dosing regimen with the potential for subcutaneous administration. Following the closing of the acquisition, Eliem’s strategy will be to develop TNT119 for a range of autoimmune-mediated diseases, where it believes CD19-targeted approaches have clear biological rationale, where it can potentially achieve clinical proof-of-concept, and where it can introduce product candidates that can be meaningfully differentiated in the market. TNT119’s lead indication is in systemic lupus erythematosus, the most common type of lupus and an autoimmune disease in which the immune system attacks its own tissue, causing widespread inflammation and tissue damage in affected organs including joints, skin, brain, lungs, kidneys and blood vessels. In systemic lupus erythematosus, the underlying pathology involves the production of autoantibodies by autoreactive B cells and the formation of immune complexes that contribute to inflammation and tissue damage. CD19 is a protein expressed on the surface of these B cells, and it plays a role in B cell activation, proliferation and survival. TNT119 is designed to target and deplete CD19-expressing B cells known to produce autoantibodies, thereby providing a novel approach to the potential treatment of systemic lupus erythematosus. Eliem expects to initiate Phase 2 clinical trials of TNT119 later this year. Leadership Team and Board of Director Updates In connection with the closing of the acquisition, Eliem is announcing additions to its executive leadership team with the appointment of Aoife Brennan, M.B., Ch.B., as President and Chief Executive Officer and Jan Hillson, M.D., as Senior Clinical Advisor. Dr. Aoife Brennan and Dr. Stephen Thomas will also join Eliem’s Board of Directors. Aoife Brennan, M.B., Ch.B.: Dr. Brennan brings to Eliem over 20 years of experience leading drug development organizations across a range of stages and therapeutic areas having most recently served as the President and Chief Executive Officer of Synlogic, a clinical stage biotechnology company developing treatments for rare metabolic diseases based on synthetic biology. In that role, she led the organization from early-stage private company to a late-phase public company with internal GMP manufacturing capabilities, pioneering new regulatory pathways for bacterial therapeutics. She joined Synlogic as Chief Medical Officer in 2016 and was promoted to CEO in October 2018. Prior to Synlogic, Dr. Brennan served as Vice President and Head of the Rare Disease Innovation Unit at Biogen, Inc., where she led the global marketing approvals of ALPROLIX®, ELOCTATE® and SPINRAZA® as well as other early-stage programs. She currently serves as a director of Fibrogen Inc., Cerevance, LLC and Xilio Therapeutics, and previously served as a director of Synlogic from October 2018 to March 2024, and as a director of Ra Pharmaceuticals, Inc. from September 2018 through its acquisition in April 2020. Dr. Brennan holds a medical degree from Trinity College Dublin, Ireland and completed her post-graduate training in internal medicine, endocrinology and metabolism at the Royal College of Physicians in Ireland. She also completed post-doctoral training in clinical research and metabolism at the Beth Israel Deaconess Medical Center in Boston and is a graduate of the Harvard Medical School Scholars in Clinical Science Program. Jan Hillson, M.D.: Dr. Hillson is a rheumatologist and clinical immunologist with 20 years of experience in academic research, patient care and teaching, and more than 15 years of experience in the biotech industry spanning translational, preclinical, early and late clinical development. Prior to Eliem, Dr. Hillson was a Partner at Cascadia Drug Development Group providing strategic advisory support, including indication prioritization, investment opportunity diligence, and planning and oversight of therapeutic development with a focus in immunology. Dr. Hillson’s experience in the biotechnology industry includes senior clinical development and leadership roles at ZymoGenetics (acquired by Bristol Myers Squibb), Momenta (acquired by Johnson & Johnson), Chemocentryx (acquired by Amgen), Alpine Immune Sciences, and Provention Bio (acquired by Sanofi), where she was responsible for the design and execution of clinical development plans and trials for multiple therapeutic candidates in autoimmune diseases and immunovirology. Dr. Hillson is currently serving on the Board of Directors for Eledon Pharmaceuticals. Dr. Hillson received her M.D. from Stanford School of Medicine, an M.S. from the California Institute of Technology, an M.S. in Marine Chemistry from Scripps Institute of Oceanography, and a B.S. from Michigan State University. Advisors Leerink Partners served as the exclusive financial advisor and Wilmer Cutler Pickering Hale and Dorr LLP served as legal counsel to Eliem. Cooley LLP served as legal counsel to Tenet. About Eliem Therapeutics, Inc. Eliem Therapeutics, following the close of the acquisition, will be focused on developing therapeutics for autoimmune-driven inflammatory diseases, including advancing TNT119, an anti-CD19 antibody designed for a broad range of autoimmune diseases, including systemic lupus erythematosus, immune thrombocytopenia and membranous nephropathy. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including without limitation statements regarding: future expectations, plans and prospects for Eliem following the consummation of the acquisition of Tenet by Eliem; the anticipated benefits of the acquisition; the strategy, anticipated milestones and key inflection points of the combined company; the anticipated use of proceeds of the private placement; the anticipated cash runway of the combined company; expectations regarding TNT119’s therapeutic benefits, clinical potential and clinical development, and anticipated timelines for initiating clinical trials of TNT119, including initiating Phase 2 clinical trials for the treatment of SLE and ITP in the second half of 2024; and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “will,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. Eliem may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. These risks and uncertainties include, but are not limited to, important risks and uncertainties associated with: the ability of Eliem to timely and successfully achieve or recognize the anticipated benefits of the acquisition; the outcome of any legal proceedings that are instituted against Eliem or Tenet relating to the acquisition and related transactions; costs related to the acquisition, including unexpected costs, charges or expenses resulting from the acquisition; changes in applicable laws or regulation; the possibility that the combined company may be adversely affected by other economic, business and/or competitive factors; competitive responses to the transactions; Eliem’s ability to advance TNT119 and/or its other product candidates on the timelines expected or at all and to obtain and maintain necessary approvals from the U.S. Food and Drug Administration and other regulatory authorities; obtaining and maintaining the necessary approvals from investigational review boards at clinical trial sites and independent data safety monitoring board; replicating in clinical trials positive results found in early-stage clinical trials of TNT119; competing successfully with other companies that are seeking to develop treatments for systemic lupus erythematosus, immune thrombocytopenia, membranous nephropathy and other autoimmune driven inflammatory diseases; maintaining or protecting intellectual property rights related to TNT119 and/or its other product candidates; managing expenses; raising the substantial additional capital needed, on the timeline necessary, to continue development of TNT119 and other product candidates Eliem may develop; and achieving Eliem’s other business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Eliem’s actual results to differ materially from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in Eliem’s most recent filings with the SEC. In addition, the forward-looking statements included in this press release represent Eliem’s views as of the date hereof and should not be relied upon as representing Eliem’s views as of any date subsequent to the date hereof. Eliem anticipates that subsequent events and developments will cause Eliem’s views to change. However, while Eliem may elect to update these forward-looking statements at some point in the future, Eliem specifically disclaims any obligation to do so. Investors Chris Brinzey ICR Westwicke chris.brinzey@westwicke.com 339-970-2843