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Press Release: Sanofi's SAR446268 earns US fast track designation for the treatment of non-congenital myotonic dystrophy type 1

1. Sanofi's SAR446268 receives FDA fast track designation for DM1 treatment. 2. SAR446268 targets DMPK expression to improve muscle function. 3. This gene therapy is the only candidate in development for DM1. 4. No approved therapies exist for myotonic dystrophy type 1. 5. Enrollment for SAR446268's clinical trial starts late 2025.

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Why Bullish?

The fast track designation boosts SNY's potential for market entry. Historical cases, such as accelerated approvals by the FDA, often led to significant stock price increases for companies involved.

How important is it?

The article addresses a significant development in a unique therapy, with potential market implications for SNY. The rarity of DM1 and the lack of current treatments enhance its relevance.

Why Long Term?

The ongoing trial and potential market entry will unfold over the coming years. Successful results can lead to substantial revenue growth once the therapy hits the market.

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Sanofi’s SAR446268 earns US fast track designation for the treatment of non-congenital myotonic dystrophy type 1 Designation earned for one-time AAV gene therapy SAR446268, designed to silence DMPK expressionMyotonic dystrophy type 1 (DM1) is a rare, genetic disorder that causes progressive muscle weakness and wasting, with no currently approved medicines Paris, September 23, 2025. The US Food and Drug Administration (FDA) has granted fast track designation to SAR446268, Sanofi's one-time AAV gene therapy for the treatment of non-congenital (juvenile and adult onset) DM1 myotonic dystrophy type 1 (DM1). This designation process aims to facilitate the development and expedite the review of medicines to treat serious conditions and fill an unmet medical need. The FDA created this process to help deliver important new medicine to patients earlier and it covers a broad range of serious illnesses. SAR446268 employs a vectorized RNA interference (RNAi) approach to silence DMPK expression through a single administration. By reducing DMPK transcripts, the gene therapy aims to eliminate the abnormal and toxic RNA foci responsible for splicing defects in muscle tissue, thereby restoring normal splicing and improving muscular function. This approach has the potential to address key symptoms of the disease, including progressive muscle weakness, difficulty relaxing muscles (myotonia), and effects on multiple body systems including heart, lungs, and endocrine functions. SAR446268 is the only investigational therapy in clinical development for this disease, and there are no currently approved therapies for DM1. SAR446268 is currently under investigation in a first-in-human, phase 1-2 study to evaluate the safety, tolerability, and efficacy (clinical study identifier: NCT06844214). The first patient is planned for enrolment in late 2025. Sanofi has already been granted orphan designations for SAR446268 in both the US (July 2024) and EU (October 2024).About myotonic dystrophy type 1 Myotonic dystrophy type 1, also known as Steinert's disease, is an inherited, progressive, rare disorder affecting approximately 1 in 2,300 people worldwide. The condition is caused by mutations in the DMPK gene and is characterized by progressive muscle weakness, difficulty relaxing muscles (myotonia), and effects on multiple body systems including heart, lungs, and endocrine functions. DM1 can manifest at any age with varying severity, from mild adult cases to severe congenital forms, and has a profound impact on quality of life, affecting patients' ability to perform daily activities, maintain independence, and in severe cases, sustain vital functions. There are no currently approved treatments for DM1. About Sanofi Sanofi is an R&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 RelationsSandrine Guendoul | +33 6 25 09 14 25 | sandrine.guendoul@sanofi.comEvan Berland | +1 215 432 0234 | evan.berland@sanofi.comLéo Le Bourhis | +33 6 75 06 43 81 | leo.lebourhis@sanofi.comVictor Rouault | +33 6 70 93 71 40 | victor.rouault@sanofi.comTimothy Gilbert | +1 516 521 2929 | timothy.gilbert@sanofi.comLéa Ubaldi | +33 6 30 19 66 46 | lea.ubaldi@sanofi.com Investor RelationsThomas Kudsk Larsen |+44 7545 513 693 | thomas.larsen@sanofi.comAlizé Kaisserian | +33 6 47 04 12 11 | alize.kaisserian@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.comYun Li | +33 6 84 00 90 72 | yun.li3@sanofi.com Sanofi forward-looking statements This 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 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 performance. 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, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, 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, 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, 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 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, 2024. 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. Press Release

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