LongBench v2 / 66fa7f1dbb02136c067c6e6b / Based on these press releases from Sanofi regarding their pharmaceutical…

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Press Release 
 
 
Dupixent approved in the EU as the first-ever targeted 
therapy for patients with COPD  
 
* 
First-in-world approval of Dupixent for adults with uncontrolled COPD with raised blood 
eosinophils based on two landmark phase 3 studies showing Dupixent significantly 
reduced exacerbations, improved lung function and also improved health-related 
quality of life 
* 
Dupixent is the first new treatment approach for COPD in more than a decade and a 
new option for approximately 220,000 adults in the EU  
* 
Approval represents the sixth approved indication for Dupixent in the EU and seventh 
approved indication globally 
 
Paris and Tarrytown, NY, July 3, 2024. The European Medicines Agency (EMA) has 
approved Dupixent (dupilumab) as an add-on maintenance treatment for adults with 
uncontrolled chronic obstructive pulmonary disease (COPD) characterized by raised blood 
eosinophils. Specifically, the approval covers patients already on a combination of an inhaled 
corticosteroid (ICS), a long-acting beta2-agonist (LABA) and a long-acting muscarinic 
antagonist (LAMA), or on a combination of a LABA and a LAMA if ICS is not appropriate. The 
EMA is the first regulatory authority in the world to approve Dupixent for COPD patients. 
Additional submissions are under review with other regulatory authorities around the world, 
including in the US, China, and Japan. 
 
Tonya Winders  
President & CEO of Global Allergy & Airways Patient Platform  
“As a progressive and devastating disease, COPD leads to suffering from breathlessness that 
limits a person’s ability to conduct everyday activities such as walking up the stairs or to the 
mailbox. Many patients feel marginalized and isolated because of the physical and mental 
toll of the disease. After more than a decade of limited treatment advancements for those 
living with uncontrolled COPD, we are now in a new era of disease management for patients 
and caregivers, and we welcome the addition of innovative, new treatments such as Dupixent 
to help manage this progressive and irreversible disease.”  
 
Paul Hudson 
Chief Executive Officer at Sanofi  
“Patients with uncontrolled COPD have been waiting for a new treatment approach for many 
years, so we are thrilled to bring to market the first biologic to target an underlying cause of 
this devastating disease to reduce COPD exacerbations and improve lung function. With 
today’s approval of Dupixent, we can change the treatment landscape for the more than 
200,000 patients throughout the EU living with uncontrolled COPD with raised blood 
eosinophils. We look forward to working with other regulators around the world as quickly 
as possible to bring this novel treatment approach to patients in more countries.”  
 
The approval is based on results from the landmark phase 3 BOREAS and NOTUS  studies, 
which were separately published in The New England Journal of Medicine and evaluated the 
efficacy and safety of Dupixent in adults with uncontrolled COPD with evidence of type 2 


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inflammation (i.e., blood eosinophils ≥300 cells per µL). All patients were on background 
maximal standard-of-care inhaled therapy (with nearly all on triple therapy). In terms of 
efficacy, Dupixent patients in BOREAS (n=468) and NOTUS (n=470) experienced the 
following, respectively, compared to placebo (BOREAS n=471; NOTUS n=465): 
• 
30% and 34% reduction in the annualized rate of moderate or severe COPD 
exacerbations over 52 weeks, the primary endpoint.  
• 
Improvements in lung function (pre-bronchodilator FEV1) from baseline by 160 mL and 
139 mL at 12 weeks compared to 77 mL and 57 mL. These improvements were 
observed as early as week 2 and 4 and were sustained at 52 weeks in both studies.  
• 
Improvements in health-related quality of life (statistically significant in BOREAS and 
nominally significant in NOTUS), as assessed by the St. George’s Respiratory 
Questionnaire. 
 
Reductions in exacerbations and improvements in lung function for Dupixent versus placebo 
were also observed in patients with higher baseline fractional exhaled nitric oxide (≥20ppb) 
- an airway biomarker of inflammation – and across all pre-defined subgroups including 
smoking status, baseline lung function, and history of exacerbations. 
 
Safety results in both studies were generally consistent with the known safety profile of 
Dupixent in its approved indications. The most common side effects across indications include 
injection site reactions, conjunctivitis, conjunctivitis allergic, arthralgia, oral herpes, and 
eosinophilia. Adverse events more commonly observed with Dupixent (≥5%) compared to 
placebo in either COPD study were back pain, COVID-19, diarrhea, headache and 
nasopharyngitis. Additional adverse reactions of injection site bruising, injection site 
induration, injection site rash and injection site dermatitis were reported in the COPD studies. 
 
George D. Yancopoulos, M.D., Ph.D. 
Board Co-Chair, President and Chief Scientific Officer at Regeneron 
“The approval of Dupixent for COPD is a long-awaited turning point for those who struggle 
to breathe even through the simplest of tasks, while also facing the risk of hospitalization, 
irreversible health decline and feelings of hopelessness. With this approval, we are proud that 
Dupixent has the potential to redefine the treatment landscape in yet another disease, as a 
first-in-class therapy demonstrating unprecedented improvements on exacerbations and 
lung function, as well as improving health-related quality of life across two large phase 3 
trials.” 
 
About COPD 
COPD is a respiratory disease that damages the lungs and causes progressive lung function 
decline and is the fourth leading cause of death worldwide. Symptoms include persistent 
cough, excessive mucus production and shortness of breath that may impair the ability to 
perform routine daily activities, which may lead to sleep disturbances, anxiety, and 
depression. COPD is also associated with a significant health and economic burden due to 
recurrent acute exacerbations that require systemic corticosteroid treatment and/or lead to 
hospitalization. Smoking and exposure to noxious particles are key risk factors for COPD, but 
even individuals who quit smoking can still develop or continue having the disease. There 
have been no new treatment approaches approved for more than a decade. 
 
About the Dupixent COPD phase 3 study program 
BOREAS and NOTUS were replicate, randomized, phase 3, double-blind, placebo-controlled 
studies that evaluated the efficacy and safety of Dupixent in adults who were current or former 


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smokers with moderate-to-severe COPD with evidence of type 2 inflammation, as measured 
by blood eosinophils ≥300 cells per µL. The studies enrolled 1,874 patients who were aged 
40 to 80 years in BOREAS and 40 to 85 years in NOTUS.  
 
During the 52-week treatment period, patients in BOREAS and NOTUS received Dupixent or 
placebo every two weeks added to a maximal standard-of-care inhaled triple therapy of ICS, 
LABA and LAMA. Double maintenance therapy, which included LABA and LAMA, was allowed 
if ICS was not appropriate. 
 
The primary endpoint for BOREAS and NOTUS evaluated the annualized rate of moderate or 
severe COPD exacerbations. Moderate exacerbations were defined as those requiring systemic 
steroids and/or antibiotics. Severe exacerbations were defined as those requiring 
hospitalization; requiring more than a day of observation in an emergency department or 
urgent care facility; or resulting in death. Key secondary endpoints included change from 
baseline in lung function (assessed by pre-bronchodilator forced expiratory volume [FEV1]) at 
12 and 52 weeks, change from baseline at 52 weeks in SGRQ total score compared to placebo, 
and safety. 
 
About Sanofi and Regeneron’s COPD clinical research program 
Sanofi and Regeneron are motivated to transform the treatment paradigm of COPD by 
examining the role different types of inflammation play in the disease progression through 
the investigation of two potentially first-in-class biologics, Dupixent and itepekimab. 
 
Dupixent inhibits the signaling of the interleukin-4 (IL4) and interleukin-13 (IL13) pathways 
and the program focuses on a specific population of people with evidence of type 2 
inflammation. Itepekimab is a fully human monoclonal antibody that binds to and inhibits 
interleukin-33 (IL-33), an initiator and amplifier of broad inflammation in COPD. 
 
Itepekimab is currently under clinical investigation in two phase 3 studies, and its safety and 
efficacy have not been evaluated by any regulatory authority. 
 
About Dupixent 
Dupixent is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 
(IL4) and interleukin-13 (IL13) pathways and is not an immunosuppressant. The Dupixent 
development program has shown significant clinical benefit and a decrease in type 2 
inflammation in phase 3 studies, establishing that IL4 and IL13 are key and central drivers of 
the type 2 inflammation that plays a major role in multiple related and often co-morbid 
diseases. 
 
Dupixent has received regulatory approvals in more than 60 countries in one or more 
indications including certain patients with atopic dermatitis, asthma, chronic rhinosinusitis 
with nasal polyposis, eosinophilic esophagitis, prurigo nodularis, chronic spontaneous 
urticaria, and COPD in different age populations. More than 900,000 patients are being treated 
with Dupixent globally. 
 
Dupilumab Development Program 
Dupilumab is being jointly developed by Sanofi and Regeneron under a global collaboration 
agreement. To date, dupilumab has been studied across more than 60 clinical studies 
involving more than 10,000 patients with various chronic diseases driven in part by type 2 
inflammation. 


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In addition to the currently approved indications, Sanofi and Regeneron are studying 
dupilumab in a broad range of diseases driven by type 2 inflammation or other allergic 
processes in phase 3 studies, including chronic pruritus of unknown origin and bullous 
pemphigoid. These potential uses of dupilumab are currently under clinical investigation, and 
the safety and efficacy in these conditions have not been fully evaluated by any regulatory 
authority. 
 
About Regeneron 
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and 
commercializes life-transforming medicines for people with serious diseases. Founded and led 
by physician-scientists, our unique ability to repeatedly and consistently translate science into 
medicine has led to numerous approved treatments and product candidates in development, 
most 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, neurological diseases, hematologic conditions, infectious diseases, 
and rare diseases. 
 
Regeneron pushes 
the 
boundaries 
of 
scientific 
discovery 
and accelerates 
drug 
development using our proprietary technologies, such as VelociSuite®, which produces 
optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the 
next frontier of medicine with data-powered insights from the Regeneron Genetics 
Center® and pioneering genetic medicine platforms, enabling us to identify innovative targets 
and complementary approaches to potentially treat or cure diseases. 
 
For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn, 
Instagram, Facebook or X. 
 
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 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 
 
Sanofi Media Relations 
Sandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.com 
Evan Berland | + 1 215 432 0234 | evan.berland@sanofi.com 
Victor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.com 
Timothy Gilbert | + 1 516 521 2929 | timothy.gilbert@sanofi.com 
 
Sanofi Investor Relations 
Thomas Kudsk Larsen |+ 44 7545 513 693 | thomas.larsen@sanofi.com 
Alizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.com 
Arnaud Delépine | + 33 6 73 69 36 93 |arnaud.delepine@sanofi.com 
Felix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.com 
Keita Browne | + 1 781 249 1766 | keita.browne@sanofi.com 
Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com 
Tarik Elgoutni | + 1 617 710 3587 | tarik.elgoutni@sanofi.com 
Thibaud Châtelet | + 33 6 80 80 89 90 | thibaud.chatelet@sanofi.com 


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Regeneron Media Relations 
Hannah Kwagh | +1 914-847-6314| hannah.kwagh@regeneron.com 
  
Regeneron Investor Relations 
Vesna Tosic | + 914-847-5443 | vesna.tosic@regeneron.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 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 protected. 
 
 
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 nature, timing, and possible success and therapeutic applications of products marketed or otherwise commercialized by 
Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Products”) and product candidates being developed by 
Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Product Candidates”) and research and clinical programs 
now underway or planned, including without limitation Dupixent® (dupilumab) as an add-on maintenance treatment for adults with 
uncontrolled chronic obstructive pulmonary disease (“COPD”) characterized by raised blood eosinophils on a combination of an inhaled 
corticosteroid (ICS), a long-acting beta2-agonist (LABA), and a long-acting muscarinic antagonist (LAMA), or on a combination of a 
LABA and a LAMA if ICS is not appropriate; uncertainty of the utilization, market acceptance, and commercial success of Regeneron’s 
Products and Regeneron’s Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether 
mandated or voluntary), including the studies discussed or referenced in this press release, on any of the foregoing or any potential 
regulatory approval of Regeneron’s Products (such as Dupixent) and Regeneron’s Product Candidates (such as itepekimab); the 
likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron’s Product Candidates and new 
indications for Regeneron’s Products, such as Dupixent for the treatment of COPD in the United States, China, and other jurisdictions 
as well as Dupixent for the treatment of chronic pruritus of unknown origin, bullous pemphigoid, and other potential indications; the 
ability of Regeneron’s collaborators, licensees, suppliers, or other third parties (as applicable) to perform manufacturing, filling, 
finishing, packaging, labeling, distribution, and other steps related to Regeneron’s Products and Regeneron’s Product Candidates; the 
ability of Regeneron to manage supply chains for multiple products and product candidates; safety issues resulting from the 
administration of Regeneron’s Products (such as Dupixent) and Regeneron’s Product Candidates (such as itepekimab) in patients, 
including serious complications or side effects in connection with the use of Regeneron’s Products and Regeneron’s 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 and Regeneron’s 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 Regeneron’s Product Candidates; the extent to which the results from the research and development 
programs conducted by Regeneron and/or its collaborators or licensees may be replicated in other studies and/or lead to advancement 
of product candidates to clinical trials, therapeutic applications, or regulatory approval; 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, collaboration, or supply agreement, 
including Regeneron’s agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable) to be cancelled or 
terminated; the impact of public health outbreaks, epidemics, or pandemics (such as the COVID-19 pandemic) on Regeneron's 


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business; 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), other litigation and 
other proceedings and government investigations relating to the Company and/or its operations (including the pending civil 
proceedings initiated or joined by the U.S. Department of Justice and the U.S. Attorney's Office for the District of Massachusetts), 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, 
2023 and its Form 10-Q for the quarterly period ended March 31, 2024. 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 or otherwise) 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 (https://investor.regeneron.com) and its 
LinkedIn page (https://www.linkedin.com/company/regeneron-pharmaceuticals). 
 


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Press Release 
 
 
Sarclisa approved in the US as the first anti-CD38 therapy 
in combination with standard-of-care treatment for adult 
patients with newly diagnosed multiple myeloma not 
eligible for transplant 
  
 
• 
Approval based on positive results from the IMROZ phase 3 study demonstrating Sarclisa 
in combination with bortezomib, lenalidomide, and dexamethasone (VRd) significantly 
improved progression-free survival (PFS), compared to standard-of-care in newly 
diagnosed adult patients not eligible for autologous stem cell transplant (ASCT) 
• 
Third indication for Sarclisa, evaluated under FDA Priority Review, underscores Sanofi’s 
commitment to helping close a critical care gap in multiple myeloma (MM)   
 
PARIS, September 21, 2024. The US Food and Drug Administration (FDA) has approved 
Sarclisa (isatuximab) in combination with bortezomib, lenalidomide, and dexamethasone (VRd) 
as a first line treatment option for adult patients with newly diagnosed multiple myeloma (NDMM) 
who are not eligible for autologous stem cell transplant (ASCT). Sarclisa is the first anti-CD38 
therapy in combination with standard-of-care VRd to significantly reduce disease progression or 
death (by 40%) compared to VRd alone for patients with NDMM not eligible for transplant. 
 
Thomas Martin M.D. 
Helen Diller Family Comprehensive Cancer Center Clinical Professor of Medicine at the 
University of California San Francisco 
“Multiple myeloma is most frequently diagnosed in patients 65 years and older, yet the options 
for treatment in this population are limited due to a combination of age, frailty, and co-
morbidities. This has resulted in a longstanding need for new treatment options that can 
potentially improve the standard-of-care. The significant clinical benefit and improvements in 
progression-free survival demonstrated by the IMROZ regimen of isatuximab plus VRd versus 
VRd alone make today’s approval an important moment for this vulnerable patient population 
and the larger multiple myeloma community.”  
 
This decision marks the third approved indication for Sarclisa in the US and the first approved 
indication in newly diagnosed patients. The FDA evaluated Sarclisa for this indication under 
Priority Review, which is reserved for medicines that represent potentially significant 
improvements in efficacy or safety in treating serious conditions. Sarclisa is also currently 
approved in more than 50 countries across two indications for the treatment of people with 
relapsed or refractory disease.
 
Brian Foard 
Executive Vice President, Head of Specialty Care, Sanofi 
“Since first launching in 2020, we have made significant progress towards our ambition of 
establishing Sarclisa as a best-in-class therapy. The FDA’s decision marks another momentous 
milestone toward our goal and expands the reach of this potentially transformative therapy to a 
larger population. With today’s approval, doctors now have an important new option at their 
disposal that’s been shown to slow disease progression for longer compared to the current 
standard-of-care for adults living with newly diagnosed multiple myeloma who are not eligible 
for transplant in the US.” 
 
Results from the IMROZ phase 3 study supporting Sarclisa in NDMM not eligible for ASCT 
 
The FDA approval is based on data from the IMROZ phase 3 study recently presented at the 
American Society of Clinical Oncology (ASCO) 2024 annual meeting and published in The New 
England Journal of Medicine. IMROZ is the first global phase 3 study of an anti-CD38 monoclonal 


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antibody in combination with standard-of-care VRd to significantly improve PFS versus VRd 
alone. 
 
In the IMROZ study, Sarclisa-VRd followed by Sarclisa-Rd met the primary endpoint of PFS, 
significantly reducing the risk of recurrence or death by 40%, compared to VRd followed by Rd, 
in patients with NDMM not eligible for ASCT (HR 0.60; 95% CI: 0.44 to 0.81, p=0.0009). At a 
median follow-up of 59.7 months, the median PFS with the Sarclisa-VRd combination was not 
reached versus 54.3 months with VRd. The estimated PFS-rate at 60 months was 63.2% for 
patients treated with Sarclisa-VRd versus 45.2% for VRd.  
 
Sarclisa-VRd also met several secondary endpoints which demonstrated deep responses in this 
patient population:  
• 
Approximately three-quarters (74.7%) of patients treated with Sarclisa-VRd achieved a 
complete response (CR) or better compared to 64.1% of patients taking VRd (OR 1.7; 
95% CI: 1.097-2.5; p=0.0160). 
• 
More than half (55.5%) of patients treated with Sarclisa-VRd achieved MRD negative CR 
compared to 40.9% of patients taking VRd (OR 1.8; 95% CI: 1.229-2.646; p=0.0026). 
 
The safety and tolerability of Sarclisa observed in this study was consistent with the established 
safety profile of Sarclisa and VRd with no new safety signals observed. The most common 
adverse reactions (≥20%) were upper respiratory tract infections, diarrhea, fatigue, peripheral 
sensory neuropathy, pneumonia, musculoskeletal pain, cataract, constipation, peripheral 
edema, rash, infusion-related reaction, insomnia, and COVID-19. The most common hematologic 
laboratory abnormalities (≥80%) were decreased hemoglobin, decreased leukocytes, decreased 
lymphocytes, decreased platelets, and decreased neutrophils. Serious adverse reactions 
occurred in 71% of patients receiving Sarclisa combination therapy. The most frequent serious 
adverse reaction occurring in more than 5% of patients was pneumonia (30%). Permanent 
discontinuation of treatment due to an adverse reaction occurred in 22.8% of patients treated 
with Sarclisa combination therapy, compared to 26% in the comparator arm.   
 
Advancing Sarclisa in multiple myeloma 
 
Sanofi continues to advance Sarclisa as part of a patient-centric clinical development program, 
which includes several phase 2 and phase 3 studies across the MM treatment continuum 
spanning six potential indications. In addition, the company is evaluating a subcutaneous 
administration method for Sarclisa in clinical studies. The safety and efficacy of Sarclisa has not 
been evaluated by any regulatory authority outside of its approved indications and methods of 
delivery.  
 
In September, isatuximab-irfc (Sarclisa) was also added to the National Comprehensive Cancer 
Network (NCCN®) Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for MM non-
transplant candidates as an NCCN Category 1 Preferred option in combination with VRd for 
patients <80 years old who are not frail. Category 1 is based upon high-level evidence, there is 
uniform NCCN consensus that the intervention is appropriate. Preferred intervention are 
interventions that are based on superior efficacy, safety, and evidence; and, when appropriate, 
affordability. 
 
*NCCN makes no warranties of any kind whatsoever regarding their content, use or application 
and disclaims any responsibility for their application or use in any way. 
 
About Sarclisa 
Sarclisa (isatuximab) is a monoclonal antibody that binds to a specific epitope on the CD38 
receptor on MM cells, inducing distinct antitumor activity. It is designed to work through multiple 
mechanisms 
of 
action 
including 
programmed 
tumor 
cell 
death 
(apoptosis) 
and 
immunomodulatory activity. CD38 is highly and uniformly expressed on the surface of MM cells, 
making it a target for antibody-based therapeutics such as Sarclisa. 
  
Based on the ICARIA-MM phase 3 study, Sarclisa is approved in more than 50 countries, 
including the US and the EU, in combination with pomalidomide and dexamethasone for the 


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treatment of patients with relapsed refractory MM (RRMM) who have received ≥2 prior therapies, 
including lenalidomide and a proteasome inhibitor and who progressed on last therapy. Based 
on the IKEMA phase 3 study, Sarclisa is also approved in 50 countries in combination with 
carfilzomib and dexamethasone, including in the US for the treatment of patients with RRMM 
who have received 1–3 prior lines of therapy and in the EU for patients with MM who have 
received at least one prior therapy. In the US, the non-proprietary name for Sarclisa is 
isatuximab-irfc, with irfc as the suffix designated in accordance with nonproprietary naming of 
biological products guidance for industry issued by the US Food and Drug Administration. 
  
Sarclisa continues to be evaluated in multiple ongoing phase 3 clinical studies in combination 
with current standard treatments across the MM treatment continuum. It is also under 
investigation for the treatment of other hematologic malignancies, and its safety and efficacy 
have not been evaluated by any regulatory authority outside of its approved indication. 
 
Sanofi is committed to pursuing the advancement of Sarclisa through several investigational 
studies across the MM treatment continuum. Various patient-centric clinical development 
programs aim to bring Sarclisa to more patients, intercept the disease earlier in the treatment 
journey, explore potential new combinations and assess subcutaneous administration via a 
proprietary on body device system. The safety and efficacy of Sarclisa has not been evaluated 
by any regulatory authority outside of its approved indications and methods of delivery. 
  
In striving to become the number one immunoscience company globally, Sanofi remains 
committed to advancing oncology innovation. Through focused strategic decisions the company 
has reshaped and prioritized its pipeline, leveraging its expertise in immunoscience to drive 
progress. Efforts are centered on difficult-to-treat cancers such as select hematologic 
malignancies, and solid tumors with critical unmet needs, including multiple myeloma, acute 
myeloid leukemia, certain types of lymphomas, as well as gastrointestinal and lung cancers. 
 
For more information on Sarclisa clinical studies, please visit www.clinicaltrials.gov. 
 
About multiple myeloma 
MM is the second most common hematologic malignancy1, affecting more than 130,000 
patients in the US; approximately 32,000 Americans are diagnosed with MM each year.2 
Despite available treatments, MM remains an incurable malignancy with an estimated 52% 
five-year survival rate for newly diagnosed patients.3 According to physician-based surveys, 
the majority of NDMM patients are not considered eligible for transplant, creating a need for 
new frontline therapeutic options, particularly due to high attrition rates in subsequent lines of 
therapy. 
 
 
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 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 Relations 
Sandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.com 
Evan Berland | +1 215 432 0234 | evan.berland@sanofi.com  
Nicolas Obrist | + 33 6 77 21 27 55 | nicolas.obrist@sanofi.com  
Victor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.com 
Timothy Gilbert | + 1 516 521 2929 | timothy.gilbert@sanofi.com 
 
Investor Relations 
Thomas Kudsk Larsen |+ 44 7545 513 693 | thomas.larsen@sanofi.com  
Alizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.com 
Arnaud Delépine | + 33 6 73 69 36 93 | arnaud.delepine@sanofi.com 


4/4 
 
Felix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.com  
Keita Browne | + 1 781 249 1766 | keita.browne@sanofi.com 
Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com 
Tarik Elgoutni | + 1 617 710 3587 | tarik.elgoutni@sanofi.com  
Thibaud Châtelet | + 33 6 80 80 89 90 | thibaud.chatelet@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 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. 
 
1 Kazandjian. Multiple myeloma epidemiology and survival: A unique malignancy. Semin Oncol. 2016;43(6):676-681. 
doi:10.1053/j/seminoncol.2016.11.004. 
2 National Cancer Institute. Myeloma Cancer Stat Facts. Available at: 
www.seer.cancer.gov/statfacts/html/mulmy.html. Accessed on December 12, 2019. 
3 Fonseca, R., Usmani, S.Z., Mehra, M. et al. Frontline treatment patterns and attrition rates by subsequent lines of 
therapy in patients with newly diagnosed multiple myeloma. BMC Cancer. 2020: 20(1087). 
https://doi.org/10.1186/s12885-020-07503-y. 


1/4 
 
 
 
 
 
Press Release 
 
Tolebrutinib demonstrated a 31% delay in time to onset of 
confirmed disability progression in non-relapsing 
secondary progressive multiple sclerosis phase 3 study 
 
• 
Data presented at ECTRIMS show that tolebrutinib, a brain-penetrant BTK inhibitor, 
addresses disability accumulation that occurs independently from relapse activity 
• 
Global regulatory submissions will begin in H2 2024 
 
Paris, September 20, 2024. Positive results from the HERCULES phase 3 study in people 
with non-relapsing secondary progressive multiple sclerosis (nrSPMS) demonstrated that 
tolebrutinib delayed the time to onset of 6-month confirmed disability progression (CDP) by 
31% compared to placebo (HR 0.69; 95% CI 0.55-0.88; p=0.0026). Further analysis of 
secondary endpoints demonstrated that the number of participants who experienced confirmed 
disability improvement increased by nearly two-fold, 10% with tolebrutinib compared to 5% 
with placebo (HR 1.88; 95% CI 1.10 to 3.21; nominal p=0.021). These results were presented 
today as a late-breaking presentation at the European Committee for Treatment and Research 
in Multiple Sclerosis (ECTRIMS) 2024 conference in Copenhagen, Denmark. 
 
Robert Fox, MD 
Vice Chair of Research at Cleveland Clinic’s Neurological Institute, Cleveland, Ohio and 
Chair of the HERCULES Global Steering Committee 
“Secondary progressive multiple sclerosis is characterized by insidious worsening of disability over 
time, independent of relapses, and represents a critical unmet need because we don’t have effective 
treatments. The results of HERCULES show clearly that tolebrutinib delayed disability progression in 
people with nrSPMS – and some people even improved disability – by uniquely targeting the 
biological processes driving disease progression in the brain.” Dr. Fox is a paid advisor to Sanofi for 
the HERCULES trial. 
 
Based on preliminary analysis of the HERCULES study, there was a slight increase in 
tolebrutinib-treated patients of some adverse events. Liver enzyme elevations (>3xULN) were 
observed in 4.1% of participants receiving tolebrutinib compared with 1.6% in the placebo 
group, a side effect also reported with other BTK inhibitors in MS. A small (0.5%) proportion of 
participants in the tolebrutinib group experienced peak ALT increases of >20xULN, all occurring 
within the first 90 days of treatment. All but one case of liver enzyme elevations resolved 
without further medical intervention. Prior to the implementation of the revised study protocol 
with more stringent monitoring, one participant in the tolebrutinib arm received a liver 
transplant and died due to post-operative complications. To date, the implementation of more 
frequent monitoring has mitigated such serious liver sequelae. Other deaths in the trial were 
assessed as unrelated to treatment by investigator; deaths were even across the placebo and 
tolebrutinib arms at 0.3%.  
 
Adverse events (≥10%*)  
tolebrutinib 
N=752 (%) 
placebo 
N=375 (%) 
COVID-19 infections 
192 (25.5%) 
85 (22.7%) 
Urinary tract infections 
85 (11.3%) 
49 (13.1%) 
*For participants receiving tolebrutinib 
 
Houman Ashrafian, MD, PhD 
Head of Research & Development, Sanofi 
“With no treatment options currently available for the broad population of patients with secondary 
progressive multiple sclerosis, tolebrutinib has demonstrated its ability to delay disability by targeting 
underlying drivers of the disease. We look forward to discussing these results with healthcare 


2/4 
 
 
 
authorities and are eager to see the results of tolebrutinib in primary progressive MS when they 
become available next year. We extend our deepest appreciation to the study participants, their 
families, and the healthcare professionals involved in these trials.” 
 
The GEMINI 1 and 2 phase 3 study results of tolebrutinib compared to Aubagio (teriflunomide), 
a standard-of-care treatment, in participants with relapsing multiple sclerosis (RMS) were also 
presented today as a late-breaking presentation at ECTRIMS. Both studies did not meet their 
primary endpoints of statistically significant improvement in annualized relapse rates (ARR) 
compared to Aubagio. However, in the key secondary endpoint, a pooled analysis of data from 
GEMINI 1 and 2, tolebrutinib delayed the time to onset of 6-month confirmed disability 
worsening (CDW) by 29% (HR 0.71; 95% CI: 0.53-0.95; nominal p=0.023). The results of the 
29% delay in CDW endpoint in participants with RMS are in line with the 31% delay in CDP 
observed in participants with nrSPMS. The significant impact of tolebrutinib on disability 
accumulation versus Aubagio, in the absence of a statistically superior impact on relapses, 
suggests that tolebrutinib may address smoldering neuroinflammation, which manifests as 
progression independent of relapses. 
Furthermore, results showed historically low ARR in the Aubagio arm in both GEMINI 1 and 2, 
and no difference was observed between Aubagio and tolebrutinib in a pooled analysis. These 
relapse rates amount to approximately 1 relapse every 8 years.  
 
 
tolebrutinib ARR 
Aubagio ARR 
GEMINI 1  
(adjusted rate ratio 1.06; 95% CI: 0.80 to 1.39; p=0.67) 
0.13 
0.12 
GEMINI 2  
(adjusted rate ratio 1.00; 95% CI: 0.75 to 1.32; p=0.98) 
0.11 
0.11 
Pooled analysis 
(adjusted rate ratio 1.03; 95% CI: 0.84 to 1.25; p=0.80) 
0.12 
0.12 
 
In preliminary analysis of the GEMINI 1 and 2 pooled safety data, adverse events observed 
between the tolebrutinib and Aubagio arms were generally balanced. Liver enzyme elevations 
(>3x ULN) were observed in 5.6% of participants receiving tolebrutinib compared with 6.3% of 
participants receiving Aubagio, a side effect reported with other BTK inhibitors in MS and 
resolved without further medical intervention. A small (0.5%) proportion of participants in the 
tolebrutinib group experienced peak ALT increases of >20xULN, all occurring within the first 90 
days of treatment. Deaths were balanced across the Aubagio and tolebrutinib arms, at 0.2% 
and 0.1% respectively, and were assessed as unrelated to treatment by investigator.  
 
Adverse events (≥10%*) 
Tolebrutinib  
N=933 (%) 
Aubagio 
N=939 (%) 
COVID-19 infections 
225 (24.1%) 
252 (26.8%) 
Nasopharyngitis 
119 (12.8%) 
105 (11.2%) 
Headache 
117 (12.5%) 
98 (10.4%) 
*For participants receiving tolebrutinib 
 
Study results will form the basis for future discussions with global regulatory authorities with 
submissions starting in H2 2024. Tolebrutinib is currently under clinical investigation, and its 
safety and efficacy have not been evaluated by any regulatory authority.  
 
The PERSEUS phase 3 study in primary progressive MS is currently ongoing with study results 
anticipated in H2 2025. 
 
About Multiple Sclerosis 
Multiple sclerosis is a chronic, immune-mediated, neurodegenerative disease that results in 
accumulation of irreversible disabilities over time. The physical and cognitive disability 
impairments translate into gradual deterioration of health status and lower quality of life, 
impacting patients’ care and life expectancy. Disability accumulation remains the significant 
unmet medical need in MS. To date, the primary target of current therapies has been 
peripheral B and T cells, while innate immunity, which is believed to drive disability 
accumulation, remains largely unaddressed by current therapies. Currently approved, or 


3/4 
 
 
 
medicines being tested for MS mainly target the adaptive immune system and/or do not act 
directly within the central nervous system (CNS) to drive clinical benefit.  
  
RMS refers to people with MS who experience episodes of new or worsening symptoms (known 
as relapses) followed by periods of partial or complete recovery. nrSPMS refers to people with 
MS who have stopped experiencing confirmed relapses but continue to experience 
accumulation of disability, experienced as symptoms such as fatigue, cognition impairment, 
balance and gait impairment, loss of bowel and/or bladder function, sexual disfunction, 
amongst others. 
 
About HERCULES 
HERCULES (NCT04411641) was a double-blind randomized phase 3 clinical study evaluating 
the efficacy and safety of tolebrutinib in participants with nrSPMS. nrSPMS was defined at 
baseline as having a SPMS diagnosis with an expanded disability status scale (EDSS) between 
3.0 and 6.5, no clinical relapses for the previous 24 months and documented evidence of 
disability accumulation in the previous 12 months. Participants were randomized (2:1) to 
receive either an oral daily dose of tolebrutinib or matching placebo for up to approximately 48 
months. 
 
The primary endpoint was 6-month CDP defined as the increase of ≥1.0 point from the 
baseline EDSS score when the baseline score is ≤5.0, or the increase of ≥0.5 point when the 
baseline EDSS score was >5.0. Secondary endpoints included 3-month change in 9 hole peg 
test and T25-FW test, time to onset of 3-month CDP as assessed by EDSS score, total number 
of new or enlarging T2 hyperintense lesions as detected by MRI, change in cognitive function 
at the EOS compared to baseline as assessed by the Symbol Digit Modalities Test and by the 
California Verbal Learning Test as well as the safety and tolerability of tolebrutinib. 
 
About GEMINI 1 and 2 
GEMINI 1 (clinical study identifier: NCT04410978) and GEMINI 2 (clinical study identifier: 
NCT04410991) were double-blind randomized phase 3 clinical studies evaluating the efficacy 
and safety of tolebrutinib compared to Aubagio in participants with relapsing forms of MS. 
Participants were randomized in both studies (1:1) to receive either tolebrutinib and placebo 
daily or 14mg Aubagio and placebo. 
 
The primary endpoint for both studies was the annualized relapse rate for up to approximately 
36 months defined as the number of confirmed adjudicated protocol defined relapses. 
Secondary endpoints included time to onset of CDW, confirmed over at least 6 months, defined 
as an increase of ≥1.5 points from the baseline EDSS score when the baseline score is 0, an 
increase of ≥1.0 point from the baseline EDSS score when the baseline score is 0.5 to ≤5.5 or 
an increase of ≥0.5 point from the baseline EDSS score when the baseline score was >5.5 in 
addition to the total number of new and/or enlarging T2 hyperintense lesions as detected by 
MRI from baseline through the end of study, the total number of Gd-enhancing T1 
hyperintense lesions as detected by MRI from baseline through the end of study and the safety 
and tolerability of tolebrutinib. 
 
About tolebrutinib 
Tolebrutinib is an investigational, oral, brain-penetrant, and bioactive Bruton’s tyrosine kinase 
(BTK) inhibitor that achieves CSF concentrations predicted to modulate B lymphocytes and 
disease-associated microglia. Tolebrutinib is being evaluated in phase 3 clinical studies for the 
treatment of various forms of multiple sclerosis and its safety and efficacy have not been 
evaluated by any regulatory authority worldwide. For more information on tolebrutinib clinical 
studies, please visit www.clinicaltrials.gov. 
 
 
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 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 


4/4 
 
 
 
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 Relations 
Sandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.com 
Evan Berland | +1 215 432 0234 | evan.berland@sanofi.com  
Nicolas Obrist | + 33 6 77 21 27 55 | nicolas.obrist@sanofi.com  
Victor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.com 
Timothy Gilbert | + 1 516 521 2929 | timothy.gilbert@sanofi.com 
 
Investor Relations 
Thomas Kudsk Larsen |+ 44 7545 513 693 | thomas.larsen@sanofi.com  
Alizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.com 
Arnaud Delépine | + 33 6 73 69 36 93 | arnaud.delepine@sanofi.com 
Felix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.com  
Keita Browne | + 1 781 249 1766 | keita.browne@sanofi.com 
Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com 
Tarik Elgoutni | + 1 617 710 3587 | tarik.elgoutni@sanofi.com  
Thibaud Châtelet | + 33 6 80 80 89 90 | thibaud.chatelet@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 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, 
 


1/3 
 
 
Press Release 
 
 
 
 
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, MD 
Medical 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, PhD 
Global 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.”   


2/3 
 
 
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 ALTUVIIIO 
ALTUVIIIO [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-Kids 
The 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 A 
Hemophilia 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 collaboration 
Sobi 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 


3/3 
 
to SEK 22.1 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at 
sobi.com and LinkedIn . 
 
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 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 Relations 
Sandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.com 
Evan Berland | +1 215 432 0234 | evan.berland@sanofi.com  
Nicolas Obrist | + 33 6 77 21 27 55 | nicolas.obrist@sanofi.com  
Victor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.com 
Timothy Gilbert | + 1 516 521 2929 | timothy.gilbert@sanofi.com 
 
Investor Relations 
Thomas Kudsk Larsen |+ 44 7545 513 693 | thomas.larsen@sanofi.com 
Alizé Kaisserian | + 33 6 47 04 12 11 | alize.kaisserian@sanofi.com 
Arnaud Delépine | + 33 6 73 69 36 93 |arnaud.delepine@sanofi.com 
Felix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.com 
Keita Browne | + 1 781 249 1766 | keita.browne@sanofi.com 
Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com 
Tarik Elgoutni | + 1 617 710 3587 | tarik.elgoutni@sanofi.com 
Thibaud Châtelet | + 33 6 80 80 89 90 | thibaud.chatelet@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 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.