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Sanofi (SAN.PA)

Paris - Paris Delayed price. Currency in EUR
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82.94+0.20 (+0.24%)
At close: 5:39PM CEST
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Previous close82.74
Open82.38
Bid0.00 x 0
Ask0.00 x 0
Day's range81.99 - 83.92
52-week range67.65 - 95.82
Volume1,224,867
Avg. volume1,744,196
Market cap104.201B
Beta (5Y monthly)0.37
PE ratio (TTM)9.46
EPS (TTM)N/A
Earnings dateN/A
Forward dividend & yield3.15 (3.80%)
Ex-dividend date04 May 2020
1y target estN/A
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  • Globe Newswire

    CHMP recommends approval of Dupixent® (dupilumab) for children aged 6 to 11 years with severe atopic dermatitis

    CHMP recommends approval of Dupixent® (dupilumab) for children aged 6 to 11 years with severe atopic dermatitis Recommendation based on pivotal trial that showed Dupixent plus topical corticosteroids (TCS) significantly improved measures of overall disease severity, skin clearance, itch and health-related quality of life measures, compared to TCS aloneData further reinforce the well-established safety profile of Dupixent in adult and adolescent atopic dermatitis patientsDupixent would be the first biologic medicine available in the EU to treat this patient group and remains the only biologic medicine approved in moderate-to-severe atopic dermatitis for adolescents and adults PARIS and TARRYTOWN, NY – October 16, 2020 – The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Dupixent® (dupilumab), recommending to extend the approval in the European Union (EU) to include children aged 6 to 11 years with severe atopic dermatitis who are candidates for systemic therapy. Dupixent is the first and only biologic approved for the treatment of uncontrolled moderate-to-severe atopic dermatitis for ages 12+ in the EU and ages 6+ in the U.S. Dupixent is also approved in the EU for certain patients with severe asthma and severe chronic rhinosinusitis with nasal polyps, two other type 2 inflammatory diseases. The European Commission is expected to announce a final decision on the Dupixent application in the coming months. The positive CHMP opinion is supported by data that include pivotal Phase 3 results on the efficacy and safety of Dupixent combined with TCS in children aged 6 to 11 years with severe atopic dermatitis that is uncontrolled on topical prescription therapies. In the trial, children treated with Dupixent and TCS experienced significantly improved measures of overall disease severity (Eczema Area and Severity Index), skin clearance, itch and health-related quality of life measures, compared to TCS alone. Adverse events more commonly observed with Dupixent included conjunctivitis, nasopharyngitis and injection site reactions. These data are consistent with the well-established efficacy and safety profile of Dupixent observed across adult and adolescent atopic dermatitis trials. The use of Dupixent in children aged 6 to 11 years is investigational and its efficacy and safety has not yet been fully evaluated in the EU. Atopic dermatitis is a chronic inflammatory disease of the skin that can be debilitating. The current standard of care for children with severe atopic dermatitis in Europe is limited to TCS leaving those with poorly controlled disease to cope with intense, unrelenting itch and skin lesions that cover much of the body resulting in skin cracking, redness or darkening, crusting and oozing. In addition, uncontrolled severe atopic dermatitis can have a significant emotional and psychosocial impact causing sleep disturbance, symptoms of anxiety and depression, and feelings of isolation for children and their families. Dupixent is a fully-human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) proteins, and is not an immunosuppressant. Data from Dupixent clinical trials have shown that IL-4 and IL-13 are key drivers of the type 2 inflammation that plays a major role in atopic dermatitis, asthma and CRSwNP. Dupixent is currently approved in more than 60 countries, and more than 170,000 patients have been treated globally. About Dupixent Dupixent is currently approved in the EU for use in adults and adolescents 12 years and older with moderate-to-severe atopic dermatitis who are candidates for systemic therapy. It is also approved in the EU for adults and adolescents 12 years and older as an add-on maintenance treatment for severe asthma with type 2 inflammation characterized by raised blood eosinophils and/or raised fractional exhaled nitric oxide (FeNO), who are inadequately controlled with high dose inhaled corticosteroid (ICS) plus another medicinal product for maintenance treatment. Dupixent is also approved in the EU for adults with severe CRSwNP for whom therapy with systemic corticosteroids and/or surgery do not provide adequate disease control. Outside of the EU, Dupixent is approved for use in specific patients with moderate-to-severe atopic dermatitis and certain patients with asthma in a number of other countries around the world, including the U.S. and Japan. Dupixent is also approved in the U.S. and Japan to treat certain adults with severe CRSwNP. Dupilumab Development Program To date, dupilumab has been studied in more than 10,000 patients ages 6 and up across 50 clinical trials in various chronic diseases driven by type 2 inflammation. In addition to the currently approved indications, Sanofi and Regeneron are also studying dupilumab in a broad range of clinical development programs for diseases driven by allergic and other type 2 inflammation, including pediatric asthma (6 to 11 years of age, Phase 3), pediatric atopic dermatitis (6 months to 5 years of age, Phase 2/3), eosinophilic esophagitis (Phase 3), chronic obstructive pulmonary disease (Phase 3), bullous pemphigoid (Phase 3), prurigo nodularis (Phase 3), chronic spontaneous urticaria (Phase 3), and food and environmental allergies (Phase 2). These potential uses are investigational, and the safety and efficacy have not been evaluated by any regulatory authority. Dupilumab is being jointly developed by Sanofi and Regeneron under a global collaboration agreement. About RegeneronRegeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to eight FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, pain, infectious diseases and rare diseases. Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® technologies, such as VelocImmune®, which uses unique genetically-humanized mice to produce optimized fully-human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world. For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.  About Sanofi Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on huan health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.  With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.  Sanofi, Empowering Life   Sanofi Media Relations Contact Sally BainTel.: +1 (781) 264 1091Sally.Bain@sanofi.com             Regeneron Media Relations ContactSharon ChenTel.: +1 914 847 1546Sharon.Chen@regeneron.com Sanofi Investor Relations – Contacts ParisEva Schaefer-JansenArnaud DelepineYvonne Naughton Sanofi Investor Relations – Contacts North AmericaFelix LauscherFara BerkowitzSuzanne Greco Sanofi IR main line:Tel.: +33 (0)1 53 77 45 45ir@sanofi.comhttps://www.sanofi.com/en/investors/contact Regeneron Investor Relations ContactMark HudsonTel.: +1 (914) 847 3482Mark.Hudson@regeneron.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 COVID-19 will 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.  Any material effect of COVID-19 on any of the foregoing could also adversely impact us. This situation is changing rapidly and additional impacts may arise of which we are not currently aware and may exacerbate other previously identified risks. 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, 2019. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. Regeneron Forward-Looking Statements and Use of Digital Media This press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and actual events or results may differ materially from these forward-looking statements. Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words.  These statements concern, and these risks and uncertainties include, among others, the impact of SARS-CoV-2 (the virus that has caused the COVID-19 pandemic) on Regeneron’s business and its employees, collaborators, and suppliers and other third parties on which Regeneron relies, Regeneron’s and its collaborators’ ability to continue to conduct research and clinical programs, Regeneron’s ability to manage its supply chain, net product sales of products marketed by Regeneron and/or its collaborators (collectively, “Regeneron’s Products”), and the global economy; the nature, timing, and possible success and therapeutic applications of Regeneron’s Products and Regeneron’s product candidates and research and clinical programs now underway or planned, including without limitation Dupixent® (dupilumab); the impact of the opinion adopted by the European Medicines Agency's Committee for Medicinal Products for Human Use discussed in this press release on the European Commission's decision regarding the application to extend Dupixent’s approval in the European Union to include children aged 6 to 11 years with severe atopic dermatitis who are candidates for systemic therapy; uncertainty of market acceptance and commercial success of Regeneron’s Products and product candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary) on the commercial success of Regeneron's Products (such as Dupixent) and product candidates; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron’s product candidates and new indications for Regeneron’s Products, including possible regulatory approval of Dupixent for children aged 6 to 11 years with severe atopic dermatitis who are candidates for systemic therapy in the European Union discussed in this press release and possible regulatory approval of Dupixent in other jurisdictions and indications (such as for the treatment of pediatric asthma, pediatric atopic dermatitis, eosinophilic esophagitis, chronic obstructive pulmonary disease, bullous pemphigoid, prurigo nodularis, chronic spontaneous urticaria, food and environmental allergies, and other potential indications); safety issues resulting from the administration of Regeneron’s Products (such as Dupixent) and product candidates in patients, including serious complications or side effects in connection with the use of Regeneron’s Products and product candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron’s ability to continue to develop or commercialize Regeneron’s Products (such as Dupixent) and product candidates; ongoing regulatory obligations and oversight impacting Regeneron’s Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement of Regeneron’s Products from third-party payers, including private payer healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payers and new policies and procedures adopted by such payers; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron’s Products and product candidates; the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; the ability of Regeneron to manufacture and manage supply chains for multiple products and product candidates; the ability of Regeneron’s collaborators, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron’s Products and product candidates; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the potential for any license or collaboration agreement, including Regeneron's agreements with Sanofi, Bayer, and Teva Pharmaceutical Industries Ltd. (or their respective affiliated companies, as applicable), to be cancelled or terminated; and risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA® (aflibercept) Injection, Dupixent, and Praluent® (alirocumab)), other litigation and other proceedings and government investigations relating to the Company and/or its operations, the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron's business, prospects, operating results, and financial condition. A more complete description of these and other material risks can be found in Regeneron’s filings with the U.S. Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2019 and its Form 10-Q for the quarterly period ended June 30, 2020.  Any forward-looking statements are made based on management’s current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron. Regeneron does not undertake any obligation to update publicly any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise. Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron's media and investor relations website (http://newsroom.regeneron.com) and its Twitter feed (http://twitter.com/regeneron).  Attachment Press release

  • Globe Newswire

    Sanofi and Translate Bio mRNA COVID-19 vaccine candidate induced high antibody levels in preclinical studies

    Sanofi and Translate Bio mRNA COVID-19 vaccine candidate induced high antibody levels in preclinical studies mRNA-based vaccine candidate MRT5500 induced potent neutralizing antibodies against SARS-CoV-2 in preclinical studiesTwo doses of MRT5500 induced neutralizing antibody levels significantly higher than those observed in COVID-19 patientsPhase 1/2 clinical trial anticipated to begin in the fourth quarter of 2020 PARIS and LEXINGTON, MASS. – October 15, 2020 - Sanofi Pasteur, the vaccines global business unit of Sanofi, and Translate Bio (NASDAQ: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company, today announced the preclinical results for MRT5500, a mRNA-based vaccine candidate against SARS-CoV-2, the virus that causes COVID-19 disease. Preclinical evaluation of MRT5500, importantly, demonstrated a favorable immune response profile against SARS-CoV-2. These data support the selection of MRT5500 for clinical development. A Phase 1/2 clinical trial is anticipated to begin in the fourth quarter of 2020. Full results are available here. MRT5500 is being developed under a collaboration agreement between Sanofi Pasteur and Translate Bio. “To tackle this global pandemic, we must look to both the strong knowledge we have from years of infectious disease expertise and the promise of new, innovative technologies,” said Thomas Triomphe, Executive Vice President and Global Head of Sanofi Pasteur. “Today’s presentation of these positive results is another development milestone for providing a safe and effective potential vaccine against SARS-CoV2 and shows how promising this technology is. We are looking forward to working on next steps with our partner Translate Bio to bring this technology to people worldwide.” “The rapid development of effective vaccines to address the COVID-19 pandemic continues to be an urgent global public health need and I am encouraged by the progress we’ve made to date with our partner Sanofi Pasteur toward the development of a promising mRNA vaccine candidate,” said Ronald Renaud, Chief Executive Officer at Translate Bio. “The preclinical results we report in this paper demonstrate the ability of MRT5500 to elicit a favorable immune response in both mice and non-human primates. Importantly, these results provide additional support for using our mRNA platform to potentially expedite the development of alternative approaches to traditional vaccines.” Key preclinical findingsThe main findings of the preclinical studies demonstrate the potential of MRT5500 to elicit neutralizing antibodies against SARS-CoV-2. In mice, four dose levels were assessed at 0.2, 1, 5 and 10 µg per dose using a two-dose vaccination schedule, administered three weeks apart. MRT5500 induced dose-dependent levels of binding antibodies and neutralizing antibodies specific to the SARS-CoV-2 spike protein. 100% seroconversion was observed at all dose levels after one administration, and a further increase in titers was observed following a second administration. Neutralizing antibody titers were observed across all dose levels after receiving the two-dose-administration regimen. In the higher dose groups (5 µg, 10 µg), titers were detected after one administration of MRT5500 and were more pronounced after the second administration. In non-human primates (NHPs), three dose levels were assessed at 15, 45 and 135 µg per dose using a two-administration vaccination schedule, three weeks apart. The potency of MRT5500 was assessed by two types of neutralization assays: pseudovirus neutralization and micro-neutralization. After the first administration, the majority of NHPs developed neutralizing antibodies reactive to the SARS-CoV-2 spike protein and those antibody titers were further enhanced after a second administration with 100% of NHPs reaching levels significantly higher than those from human convalescent sera by day 35. It was also demonstrated that MRT5500-immunized mice and non-human primates exhibited a Th1-biased T cell response against SARS-CoV-2. The preprint publication “Immunogenicity of novel mRNA COVID-19 vaccine MRT5500 in mice and non-human primates,” is available here. Shots on goal in the fight against COVID-19In addition to the mRNA vaccine candidate in collaboration with Translate Bio, Sanofi is collaborating with GSK on a COVID-19 vaccine candidate using the same recombinant protein-based manufacturing technology as one of Sanofi’s seasonal influenza vaccines, combined with GSK’s established pandemic adjuvant platform. The Companies announced the start of the Phase 1/2 clinical trial for their adjuvanted recombinant COVID-19 vaccine candidate in September and anticipate first results in early December 2020, to support the initiation of a pivotal Phase 3 study before the end of the year. About mRNA vaccines  Vaccines work by mimicking disease agents to stimulate the immune system, building up a defense mechanism that remains active in the body to fight future infections. mRNA vaccines offer an innovative approach by delivering a nucleotide sequence encoding the antigen or antigens selected for their high potential to induce a protective immune response. mRNA vaccines also represent a potentially innovative alternative to conventional vaccine approaches for several reasons - their high potency, ability to initiate protein production without the need for nuclear entry, capacity for rapid development and potential for low-cost manufacture and safe administration using non-viral delivery. This approach potentially enables the development of vaccines for disease areas where vaccination is not a viable option today. Additionally, a desired antigen or multiple antigens can be expressed from mRNA without the need to adjust the production process, offering maximum flexibility and efficiency in development. About the Sanofi Pasteur and Translate Bio collaborationIn 2018, Translate Bio entered into a collaboration and exclusive license agreement with Sanofi Pasteur Inc., the vaccines global business unit of Sanofi, to develop mRNA vaccines for up to five infectious disease pathogens. The agreement was first expanded in March 2020 to include development of a novel mRNA vaccine for COVID-19. In June 2020, the two Companies built upon the existing collaboration to pursue novel mRNA vaccines to broadly address current and future infectious diseases. This collaboration brings together Sanofi Pasteur’s leadership in vaccines and Translate Bio’s mRNA research and development expertise. Under the agreement, the Companies are jointly conducting research and development activities to advance mRNA vaccines and mRNA vaccine platform development during a research term of at least four years after the original signing in 2018. About Translate BioTranslate Bio is a clinical-stage mRNA therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, or to prevent infectious diseases by generating protective immunity. Translate Bio is primarily focused on applying its technology to treat pulmonary diseases caused by insufficient protein production or where the reduction of proteins can modify disease. Translate Bio’s lead pulmonary candidate is being evaluated as an inhaled treatment for cystic fibrosis (CF) in a Phase 1/2 clinical trial. Additional pulmonary diseases are being evaluated in discovery-stage research programs that utilize a proprietary lung delivery platform. Translate Bio believes that mRNA can be delivered to target tissues via multiple routes of administration and, consequently, its technology may apply broadly to a wide range of diseases, including diseases that affect the liver. Translate Bio is also pursuing the development of mRNA vaccines for infectious diseases under a collaboration with Sanofi Pasteur.   About Sanofi   Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.   With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.   Sanofi, Empowering Life   Sanofi Media Relations Nicolas Kressmann Tel.: +1 (732) 532 53-18 Nicolas.Kressmann@sanofi.com   Translate Bio Media Relations Maura GavaghanTel: +1 (617) 233-1154 mgavaghan@translate.bio Sanofi Investor Relations - Paris Eva Schaefer-Jansen Arnaud DelepineYvonne Naughton   Sanofi Investor Relations – North America Felix LauscherFara BerkowitzSuzanne Greco   IR Main Line:Tel.: +33 (0)1 53 77 45 45ir@sanofi.com   Translate Bio Investor RelationsTeri Dahlman Tel: +1 (617) 817-8655 tdahlman@translate.bio 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 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 COVID-19 will 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.  Any material effect of COVID-19 on any of the foregoing could also adversely impact us. This situation is changing rapidly, and additional impacts may arise of which we are not currently aware and may exacerbate other previously identified risks. 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, 2019. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. Translate Bio Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding:  the goal to initiate a Phase 1/2 clinical trial of MRT5500 in the fourth quarter of 2020; the potential for MRT5500 to be a promising candidate for clinical development; the development of an mRNA vaccine candidate; the potential for MRT5500 to elicit a robust immune response; Translate Bio’s beliefs regarding the broad applicability of its MRT platform; and Translate Bio’s plans, strategies and prospects for its business, including its lead development programs and continued development of mRNA vaccines for the treatment of infectious diseases. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “forward,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from current expectations and beliefs, including but not limited to: the current and potential future impacts of the COVID-19 pandemic on Translate Bio’s business, financial condition, operations and liquidity; Translate Bio’s ability to advance the development of its platform and programs, including without limitation its vaccine development program generally and MRT5500 specifically, under the timelines it projects, demonstrate the requisite safety and efficacy of its product candidates and replicate in clinical trials any positive findings from preclinical studies; the successful advancement of the collaboration agreement between Translate Bio and Sanofi; uncertainties relating to the discovery and development of vaccine candidates based on mRNA, and specifically as it relates to the novel coronavirus, COVID-19; the content and timing of decisions made by the U.S. Food and Drug Administration, other regulatory authorities and investigational review boards at clinical trial sites, including decisions as it relates to ongoing and planned clinical trials; Translate Bio’s ability to obtain, maintain and enforce necessary patent and other intellectual property protection; the availability of significant cash required to fund operations; competitive factors; general economic and market conditions and other important risk factors set forth under the caption “Risk Factors” in Translate Bio’s Quarterly  Report on Form 10-Q for the fiscal quarter ended June 30, 2020 filed with the Securities and Exchange Commission on August 6, 2020 and in any other subsequent filings made by Translate Bio. Any forward-looking statements contained in this press release speak only as of the date hereof, and Translate Bio specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Attachment PDF

  • Globe Newswire

    EMA accepts regulatory submission for avalglucosidase alfa, a potentially new standard of care enzyme replacement therapy for Pompe disease

    EMA accepts regulatory submission for avalglucosidase alfa, a potentially new standard of care enzyme replacement therapy for Pompe disease Avalglucosidase alfa, an investigational enzyme replacement therapy for patients with Pompe disease, reaches its first important regulatory milestonePompe disease affects an estimated 50,000 people worldwideSubmission based on positive data from two trials including both infantile-onset and late-onset Pompe disease patientsRegulatory approval in Europe anticipated in the second half of 2021Milestone builds on company’s 20+ year commitment to the Pompe disease communityAvalglucosidase alfa receives Promising Innovative Medicine designation in UK, adding to U.S. Breakthrough Therapy designation received earlier this year PARIS – October 2, 2020 –The European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for avalglucosidase alfa, for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency). Avalglucosidase alfa is an investigational enzyme replacement therapy, which, if approved, would offer a potential new standard of care for patients with Pompe disease.    Pompe disease is a rare, degenerative muscle disorder that can impact an individual’s ability to move and breathe. It affects an estimated 50,000 people worldwide and can manifest at any age from infancy to late adulthood. The MAA is based on positive data from two trials: Phase 3, double-blind, comparator-controlled, pivotal COMET trial, which evaluated the safety and efficacy of avalglucosidase alfa compared to alglucosidase alfa (standard of care) in patients with late-onset Pompe disease. Results from this trial were presented during a Sanofi-hosted virtual scientific session in June 2020.Phase 2 mini-COMET trial, which evaluated the safety and exploratory efficacy of avalglucosidase alfa in patients with infantile-onset Pompe disease previously treated with alglucosidase alfa. Results from this trial were presented at the WORLDSymposium, in February 2020. Pompe disease is caused by a genetic deficiency or dysfunction of the lysosomal enzyme acid alpha-glucosidase (GAA), which results in build-up of complex sugars (glycogen) in muscle cells throughout the body. The accumulation of glycogen leads to irreversible damage to the muscles, including respiratory muscles, such as the diaphragm muscle that supports the lungs, and other skeletal muscles that affect mobility. Avalglucosidase alfa is designed to improve the delivery of GAA enzyme into the lysosomes of muscle cells to breakdown glycogen and help address respiratory impairment, as well as decreased muscle strength and function (i.e. mobility), which are critical manifestations of Pompe disease. The Medicines and Healthcare Products Regulatory Agency in the UK has granted Promising Innovative Medicine designation for avalglucosidase alfa, an early indication that the investigational therapy is a promising candidate for the Early Access to Medicines Scheme in the UK.i The U.S. Food and Drug Administration has granted Breakthrough Therapy and Fast Track designations to avalglucosidase alfa. Delivery of GAA to Clear Glycogen To reduce the glycogen accumulation caused by Pompe disease, the GAA enzyme must be delivered into the lysosomes within muscle cells. Research led by Sanofi has focused on ways to enhance the delivery of GAA into the lysosomes of muscle cells by targeting the mannose-6-phosphate (M6P) receptor that plays a key role in the transport of GAA. Avalglucosidase alfa is designed with approximately 15-fold increase in M6P content, compared to alglucosidase alfa, and aims to help improve cellular enzyme uptake and enhance glycogen clearance in target tissues.ii The clinical relevance of this difference has not been confirmed. Avalglucosidase alfa is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority worldwide.  About Sanofi Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions. With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe. Sanofi, Empowering Life  Media Relations Contact Sally Bain Tel.: +1 781 264 1091 sally.bain@sanofi.com Sanofi Investor Relations Contacts Paris Eva Schaefer-Jansen Arnaud DelepineYvonne Naughton Sanofi Investor Relations Contacts North America Felix LauscherFara BerkowitzSuzanne Greco IR main line:Tel.: +33 (0)1 53 77 45 45 ir@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 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 COVID-19 will 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.  Any material effect of COVID-19 on any of the foregoing could also adversely impact us. This situation is changing rapidly and additional impacts may arise of which we are not currently aware and may exacerbate other previously identified risks. 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, 2019. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. i Medicines and Healthcare Products Regulatory Agency. Promising Innovative Medicine (PIM) Designation - Step I of Early Access to Medicines Scheme (EAMS). Gov.UK. https://assets.publishing.service.gov.uk/government/uploads/system/uploads/attachment_data/file/375327/PIM_designation_guidance.pdf. Published April 2014. Accessed September 24, 2020. ii Zhou Q. Bioconjug Chem. 2011 Apr 20;22(4):741-51 Attachment PDF