<img src="https://ml-eu.globenewswire.com/Resource/Download/06da731a-67a1-4258-939f-6ef0531ee3bf?size=2" nborder="0" align="left" hspace="5" vspace="5" title="Sanofi new_logo.jpg" alt="Sanofi Winthrop Industrie"/>Sanofi’s Nexviazyme met all primary and secondary endpoints in infantile-onset Pompe disease phase 3 study <ul type="disc"><li style="text-align:justify;">Nexviazyme met its primary endpoint, participants alive and free of invasive ventilation, in treatment-naïve infants zero to six months of age in the Baby-COMET phase 3 study </li><li style="text-align:justify;">Sanofi intends to submit the data to support a regulatory application in the US for the treatment of infantile-onset Pompe disease</li></ul> Paris, June 30, 2026. Positive results from the Baby-COMET phase 3, single-arm, open-label study (clinical study identifier: <a href="https://clinicaltrials.gov/study/NCT04910776?intr=avalglucosidase%20alfa&amp;rank=3#study-plan" rel="nofollow" target="_blank" title="NCT04910776">NCT04910776</a>), demonstrated that Nexviazyme (avalglucosidase alfa) met its primary endpoint: proportion of treatment-naïve pediatric participants six months of age and younger with infantile-onset Pompe disease (IOPD) alive and free of invasive ventilation at 52 weeks of treatment. In addition, the study met all secondary endpoints, including proportion of participants alive and free of invasive ventilation at 12 and 18 months of age, and numerical improvements in other metrics of disease progression at 52 weeks. The results will be shared on July 8, 2026, at the 19th International Congress on Neuromuscular Diseases in Florence, Italy. In addition, the data will support a regulatory submission for a label extension in the US, anticipated in the second half of 2026. Pompe disease is a rare, inherited/genetic, progressive neuromuscular disease caused by a deficiency of the acid alpha-glucosidase (GAA) enzyme that affects muscle function throughout the body. IOPD constitutes the most aggressive variant of this disease, manifesting with swift symptom progression during the first months of life. Without therapeutic intervention, IOPD results in severe and potentially fatal complications affecting the heart, breathing, and movement. Nexviazyme is being evaluated as a potential treatment option for IOPD, designed to help enter cells and improve uptake of the essential GAA enzyme. This approach may help clear away excess glycogen, which builds up in muscle cells and can cause damage to skeletal and cardiac muscles. In the Baby-COMET study, Nexviazyme was well tolerated and safety was consistent with the established profile of avalglucosidase alfa, with no serious treatment-related treatment-emergent adverse events, deaths, or discontinuations, and manageable infusion-associated reactions in 29.4% of participants. “Infantile-onset Pompe disease is a devastating, rapidly progressive condition that presents within the first days or weeks of life, making early intervention critical to help improve invasive ventilator-free survival beyond one year,” said Priya S. Kishnani, MD, C.L. and Su Chen Professor of Pediatrics; Medical Director, YT and Alice Chen Pediatrics Genetics and Genomics Center; and Division Chief, Medical Genetics, Duke University Medical Center, NC, US. “The Baby-COMET study shows the potential of avalglucosidase alfa to support ventilator-free survival in infants, alongside encouraging cardiac and motor outcomes, offering important insights that may help advance the treatment landscape for these patients.” “These positive results offer the potential to expand access of Nexviazyme to more patients and families facing a condition with limited treatment options in the earliest months of life," said Christopher Corsico, Global Head of Development at Sanofi. “The Baby-COMET findings are consistent with previous studies and reflect years of our scientific research aimed at translating deep biological understanding into clinical advances for the Pompe community.” Nexviazyme is approved in multiple countries for the treatment of people living with Pompe disease, with specific indications varying by country. In the US, Nexviazyme was approved in 2021 for the treatment of late-onset Pompe disease (LOPD) in patients one year of age and older. In Europe, where the medicine is available under the name Nexviadyme, it received approval for the long-term enzyme replacement therapy of patients with Pompe disease (LOPD and IOPD) in 2022. Nexviazyme in IOPD is currently under clinical investigation in the US, and its safety and efficacy in this indication have not been evaluated by the FDA. About Pompe disease People living with Pompe disease have low levels of the GAA enzyme, which results in build-up of glycogen in muscle cells throughout the body, leading to potentially irreversible damage to skeletal and cardiac muscles. Pompe disease can present as either IOPD, the most severe form of the disease with early onset of symptoms in infancy that progress rapidly, or LOPD, which progressively damages muscles over time. Left untreated, IOPD can lead to heart failure and death within the first year of life, while people living with LOPD may require mechanical ventilation to help with breathing or a wheelchair to assist with mobility as the disease progresses. About Nexviazyme Nexviazyme (avalglucosidase alfa) is an ERT designed with high-binding affinity to target the mannose-6-phosphate (M6P) receptor, the key pathway for uptake and transport of ERT. Nexviazyme aims to help improve uptake and enhance glycogen clearance in target tissues with an approximately 15-fold higher level of M6P moieties compared with Myozyme/Lumizyme (alglucosidase alfa). About the Baby-COMET study The Baby-COMET phase 3 study is a single-arm, open-label, international, multicenter study evaluating Nexviazyme in treatment-naïve pediatric participants with IOPD 12 months of age and younger. Seventeen participants received intravenous Nexviazyme 40 mg/kg every other week. After a four-week screening period, participants received treatment for 52 weeks, followed by continued treatment for an additional 52 weeks and up to 104 additional weeks, with a four-week follow-up. The primary endpoint is the proportion of participants who are alive and free of invasive ventilation at week 52 of treatment. Key secondary endpoints included proportion of participants alive and free of invasive ventilation at 12 and 18 months of age, as well as change from baseline to week 52 in left ventricular mass Z-score, Alberta Infant Motor Scale score and urinary glucose tetrasaccharide.  About Sanofi Sanofi is an R&amp;D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Media Relations Sandrine Guendoul | +33 6 25 09 14 25 | <a href="mailto:sandrine.guendoul@sanofi.com" rel="nofollow" target="_blank" title="">sandrine.guendoul@sanofi.com</a> Evan Berland | +1 215 432 0234 | <a href="mailto:evan.berland@sanofi.com" rel="nofollow" target="_blank" title="">evan.berland@sanofi.com</a> Léo Le Bourhis | +33 6 75 06 43 81 | <a href="mailto:leo.lebourhis@sanofi.com" rel="nofollow" target="_blank" title="">leo.lebourhis@sanofi.com</a> Victor Rouault | +1 617 356 4751 | <a href="mailto:victor.rouault@sanofi.com" rel="nofollow" target="_blank" title="">victor.rouault@sanofi.com</a> Timothy Gilbert | +1 516 521 2929 | <a href="mailto:timothy.gilbert@sanofi.com" rel="nofollow" target="_blank" title="">timothy.gilbert@sanofi.com</a> Léa Ubaldi | +33 6 30 19 66 46 | <a href="mailto:lea.ubaldi@sanofi.com" rel="nofollow" target="_blank" title="">lea.ubaldi@sanofi.com</a> Ekaterina Pesheva | +1 410 926 6780 | <a href="mailto:ekaterina.pesheva@sanofi.com" rel="nofollow" target="_blank" title="ekaterina.pesheva@sanofi.com">ekaterina.pesheva@sanofi.com</a> Laura Romby | +33 6 74 16 74 29 | <a href="mailto:laura.romby@sanofi.com" rel="nofollow" target="_blank" title="">laura.romby@sanofi.com</a> Investor Relations Thomas Kudsk Larsen |+ 44 7545 513 693 | <a href="mailto:thomas.larsen@sanofi.com" rel="nofollow" target="_blank" title="">thomas.larsen@sanofi.com</a> Alizé Kaisserian | + 33 6 47 04 12 11 | <a href="mailto:alize.kaisserian@sanofi.com" rel="nofollow" target="_blank" title="">alize.kaisserian@sanofi.com</a> Keita Browne | + 1 781 249 1766 | <a href="mailto:keita.browne@sanofi.com" rel="nofollow" target="_blank" title="">keita.browne@sanofi.com</a> Nathalie Pham | + 33 7 85 93 30 17 | <a href="mailto:nathalie.pham@sanofi.com" rel="nofollow" target="_blank" title="">nathalie.pham@sanofi.com</a> Nina Goworek | +1 908 569 7086 | <a href="mailto:nina.goworek@sanofi.com" rel="nofollow" target="_blank" title="">nina.goworek@sanofi.com</a> Thibaud Châtelet | + 33 6 80 80 89 90 | <a href="mailto:thibaud.chatelet@sanofi.com" rel="nofollow" target="_blank" title="">thibaud.chatelet@sanofi.com</a> Yun Li | +33 6 84 00 90 72 | <a href="mailto:yun.li3@sanofi.com" rel="nofollow" target="_blank" title="">yun.li3@sanofi.com</a> Sanofi forward looking statement This press release contains forward-looking statements within the meaning of applicable securities laws, including 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 and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future events and economic performance. Words such as “expect,” “anticipate,” “believe,” “intend,” “estimate,” “plan,” “can,” “contemplate,” “could,” “is designed to,” “may,” “might,” “potential,” “objective,” "attempt," “target,” “project,” "strategy," "strive," "desire," “predict,” “forecast,” “ambition,” “guideline,” "seek," “should,” “will,” "goal," or the negative of these, and similar expressions are intended to identify forward-looking statements. 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, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the U.S Food and Drug Administration or the European Medicines Agency, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates; the fact that product candidates if approved may not be commercially successful; unexpected regulatory actions or delays, or government regulation generally; authorities’ decisions regarding whether and when to approve a product candidate; political pressure in the United States to mandate lower drug prices including “most favored nation” pricing for State Medicaid programs; the future approval and commercial success of therapeutic alternatives; Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, 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 pending or future litigation and the ultimate outcome of such litigation; trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that 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 French Markest Authority (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, 2025, or contained in our periodic reports on Form 6-K. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. In light of these risks, uncertainties, and assumptions, you should not place undue reliance on any forward-looking statements contained herein. All trademarks mentioned in this press release are the property of the Sanofi group.   
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Press Release: Sanofi’s Nexviazyme met all primary and secondary endpoints in infantile-onset Pompe disease phase 3 study

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