Meningococcal vaccine groups A C Y W-135 conjugate (second generation)(Sanofi)

Taylor Tieden for BioSpace On the FDA’s docket this month are two expansion bids, one for GSK’s asthma drug Nucala into COPD and another for Merck’s oral cancer drug for a pair of rare tumors. Amid the growing macro-level headwinds in the vaccine space, the FDA this month is expected to decide on Moderna’s mRNA COVID-19 shot, alongside a handful of other big verdicts. Read below for more. GSK Asks FDA to Approve Asthma Injection Nucala for COPD GSK is proposing its subcutaneous biologic Nucala to treat adult patients with chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype. The FDA’s decision is due on May 7. A pulmonary disease, COPD is characterized by inflammation in the lungs, manifesting as breathlessness, coughs and airflow obstruction. According to GSK, some 40% of patients suffer from disease exacerbations caused by type 2 inflammation, which in turn is linked to high eosinophil counts in the blood. Nucala , a monoclonal antibody, targets and blocks the IL-5 cytokine, which is crucial to maintain the activity of eosinophils. GSK is supporting Nucala’s COPD application with data from the Phase III MATINEE trial. The pharma released a topline readout from the study in September 2024, noting that Nucala elicited a “statistically significant and clinically meaningful reduction” in yearly rates of moderate or severe disease exacerbations as opposed to placebo. Nucala is currently approved for eosinophilic granulomatosis with polyangiitis and hypereosinophilic syndrome, and as an add-on maintenance treatment for asthma and chronic rhinosinusitis with nasal polyps. Arcutis Seeks Zoryve Expansion into Body and Scalp Psoriasis By May 22, the FDA will release its verdict on Arcutis Biotherapeutics’ proposed expansion for Zoryve into body and scalp psoriasis. Arcutis is backing its expansion bid with data from the Phase III ARRECTOR trial, which found that 66.4% of Zoryve-treated patients achieved clear or almost-clear skin, as opposed to 27.8% in placebo patients. Results also showed that 65.3% of patients in the Zoryve arm saw significant improvements in itch, versus 30.3% in the placebo group. Arcutis also filed findings from a Phase IIb study as well as long-term efficacy and safety data for Zoryve in plaque psoriasis. Zoryve is a topical phosphodiesterase 4 inhibitor that works by helping to suppress inflammation in the skin. The drug won FDA approval in 2011 for the treatment of plaque psoriasis in patients aged 12 years and up. If approved for body and scalp psoriasis, Zoryve would be a “truly meaningful innovation” for patients who suffer from inadequately controlled symptoms, Arcutis CEO Frank Watanabe said in a September 2024 release. Sanofi Eyes Extension of Meningococcal Disease Shot to Infants, Toddlers Sanofi’s meningococcal disease vaccine MenQuadfi is currently approved for the primary immunization of children aged two years and up. As a booster, it can be used in those 13 years and older who continue to be at risk of meningococcal disease. Sanofi is seeking to expand MenQuadfi’s label to also include infants and toddlers from six weeks to 23 months of age. The FDA is currently reviewing this proposal and is due to release its decision on May 23. If approved, MenQuadfi could protect this younger population from the A, C, W and Y serogroups of Neisseria meningitidis . In an April presentation before the CDC’s Advisory Committee for Immunization Practices, Sanofi presented data from several trials, including the Phase III MET42 study. Results showed that MenQuadfi was safe to use in infants and toddlers, with most side effects being mild or moderate in severity. Efficacy-wise, MenQuadfi was able to match a commercially approved vaccine in immune response and seroprotection. Liquidia Awaits Final Yutrepia Approval in Interstitial Lung Disease On May 23, Liquidia is expecting to win final approval of its inhalation powder Yutrepia for the treatment of patients with pulmonary arterial hypertension associated with interstitial lung disease (PH-ILD). The FDA gave the drug tentative approval in August 2024, indicating that it had cleared all efficacy and safety hurdles and satisfied regulatory standards of product quality. However, Liquidia still needed to wait out regulatory exclusivity for a competing product. Yutrepia is Liquidia’s inhalable version of treprostinil, a vasodilator that has known activity against pulmonary hypertension. Yutrepia is backed by data from the Phase III INSPIRE study, which found that the drug was both safe and well-tolerated in patients who were naïve to treprostinil or who were switching to Yutrepia from nebulized treprostinil. The INSPIRE trial found that patients treated with Yutrepia saw symptoms stabilize or improve over one year of dosing. Merck Hopes to Tack Two Rare Cancers Onto Welireg’s Label Merck is hoping to expand its oral cancer drug Welireg into advanced pheochromocytoma and paraganglioma (PPGL). The FDA’s decision deadline is May 26. PPGL refers to two types of rare tumors of the adrenal gland. Diagnosed in some 2,000 patients per year, they are typically caused by specific genetic mutations. Symptoms include high blood pressure, palpitations and headaches. According to Merck, up to 25% of PPGL cases are already at the metastatic stage at diagnosis. Merck is supporting Welireg’s PPGL bid with data from Cohort A1 of the Phase II LITESPARK-015 study. The pharma did not provide specific data in a January news release , revealing only that the application is based on objective response rate and duration of response in PPGL patients after Welireg treatment. The FDA has granted the drug priority review in this indication. Welireg is an orally available inhibitor of the HIF-2α transcription factor, which is involved in oxygen sensing and in the body’s response to hypoxia. The drug is indicated for patients with renal cell carcinoma (RCC) and those with von Hippel-Lindau disease—a rare genetic disease involving the formation of tumors across various organs—who also require treatment for associated RCC. If approved, Welireg will become the only drug for advanced PPGL in the U.S., Merck said. After Delay, Eton Nears Approval for Oral Hydrocortisone By May 28, the FDA will release its verdict on Eton Pharmaceuticals’ ET-400, an oral formulation of hydrocortisone, for the treatment of adrenal insufficiency in infants. This new target action date comes after the FDA in February announced that it needed more time to review Eton’s data package for ET-400. Eton submitted supplemental information in December last year, in response to a request from the regulator. ET-400 is a proprietary oral formulation of hydrocortisone that, according to the biotech, is stable at room temperature. If approved, ET-400 will follow in the footsteps of Eton’s Alkindi Sprinkle, which also has hydrocortisone as an active ingredient, but is formulated as oral granules. Alkindi Sprinkle, approved in 2020 , is designed to rapidly deliver glucocorticoids to children with adrenocortical insufficiency. In a July 2024 announcement , Eton CEO Sean Brynjelsen said that the approval of ET-400 would allow the biotech to “capture a greater percentage of the oral hydrocortisone market” and, alongside Alkindi Sprinkle, would open up a peak opportunity of “more than $50 million annually.” Moderna Seeks Approval for Next-Generation COVID-19 Shot The FDA’s last big milestone for the month belongs to Moderna and its investigational next-generation COVID-19 vaccine mRNA-1283. The regulator is expected to release its decision on May 31. In June 2024, the company released Phase III data for the experimental shot, touting better efficacy than Spikevax, its currently approved COVID-19 vaccine. Moderna did not provide specific data, only claiming that mRNA-1283 elicited a “higher immune response” against SARS-CoV-2 than Spikevax. Of note, Moderna emphasized at the time that mRNA-1283 could induce robust responses against Omicron and original strains of the virus—a benefit the company claimed was most evident in seniors over 65 years of age. The CDC’s vaccine advisors were supposed to convene in late February to discuss various vaccines, including mRNA-1283, before the meeting was postponed . The panel finally convened last month.

Vaccines InvestorEvent: Sanofireiterates confidence in strong growth outlook and showcases pipeline of innovative vaccine candidates Sanofi confirms Vaccines mid-term guidance of mid-to-high single-digit sales growth1, ambition to more than double Vaccines sales by the end of the decade2 Broad pipeline of innovative vaccines, with a total of 10 candidates planned to advance into the clinic by 2025, six of which will leverage the recently established mRNA Center of Excellence Hybrid Vaccines Investor Event will take place from 2 p.m. to 5 p.m. CET / 8 a.m. to 11 a.m. EST (webcast, in-person meeting at Sanofi Paris Headquarters) PARIS–December 1, 2021 – Today, Sanofi hosts a hybrid Vaccines Investor Event with key members of the Vaccines leadership team to discuss how the company applies the global Play to Win strategy to pursue growth opportunities and to build an industry-leading vaccines pipeline. Sanofi confirms its mid-term sales guidance announced at the 2019 Capital Markets Day of mid-to-high single-digit growth1 for its Vaccines business. Sustained growth will be driven by four core franchises of influenza, meningitis, PPH3 & Boosters, as well as the planned launch of nirsevimab, a first-in-class monoclonal antibody for all infant protection against Respiratory Syncytial Virus. These core franchises are well positioned to capture market growth, such as in the global influenza market which Sanofi expects to exceed €15 billion by 20304. “The pandemic has elevated the crucial role of vaccines in public health, as evidenced by the strong demand for our differentiated flu vaccines which provide protection beyond flu by reducing the risk of severe consequences of influenza” said Thomas Triomphe, Executive Vice President, Sanofi Pasteur. “As a world leader in vaccines, we are uniquely positioned to unlock the true potential of vaccines by moving fast towards next-generation mRNA and other technologies applicable to the prevention of disease. We are delightedto share more details about the rapid advances of ourindustry-leadingvaccine programs.” Sanofi has made significant progress in the field of mRNA technology with the recently established Center of Excellence and the integration of Translate Bio. Results from a first influenza mRNA clinical trial will be shared as well as initial successes in mRNA technology improvements. Of the 10 new vaccine candidates planned to enter the clinic by 2025, six will use mRNA technologies to target diseases with high unmet needs and disease burden such as chlamydia and acne. “Sanofi owns multiple vaccine platforms including mRNA andbuilds on years of experience inthe science of immunology, structural biology, andmachine learning-based antigen design. Equipped with this set of expertise, we are best positioned to deliver innovative vaccines designed to combat endemic and epidemic infectious diseases. I am excited to share that Sanofi is also opening a new chapter of its history by pioneering the use of vaccines to tackle chronic diseases like acne,” said Jean-François Toussaint, Head of Vaccines R&D. Sanofi will present the following assets in its growing R&D pipeline: A pneumococcal vaccine candidate covering 21 serotypes, with Phase 1/2 clinical trials underway in three target populations; results are expected by end of 2022. A combination of MenQuadfi and a new meningitis B vaccine candidate to create a best-in-class pentavalent meningococcal vaccine with broader strain coverage; Phase 2 meningitis B read-out is expected by H2 2022 and the initiation of a Phase 1 trial for the pentavalent candidate is planned in 2023. RSV vaccine candidates for toddlers and older adults to address a high unmet disease burden; Phase 1/2 trial results in toddlers are anticipated in 2022; in parallel, an mRNA vaccine candidate for older adults is planned to enter a Phase 1/2 study in H2 2022. Next generation influenza programs to deliver differentiated flu vaccines that set a new standard of care; the influenza mRNA QIV program will enter Phase 1/2 trial in 2022 and a Phase 3 trial is planned to start in 2023; Sanofi also deploys proprietary machine-learning to explore the potential for better strain selection in the future. Opening new growth areas: Chlamydia: Sanofi has initiated a program to prevent chlamydia infection, a silent cause of infertility in women; a Phase 1 trial of an mRNA-based candidate is planned to start in 2023. Acne: Sanofi strives to leverage its vaccine technology to develop the first immunotherapy against acne and to enter the clinic in 2023. Vaccines Investor Eventdetails For background slides and webcast, please refer to the following link: 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. Media Relations Contacts Sandrine Guendoul Tel.: +33 (0)6 25 09 14 25 Sandrine.Guendoul@sanofi.com Sally Bain Tel.: +1 (617) 834-6026 Sally.Bain@sanofi.com Nicolas Obrist Tel.: +33 (0)6 77 21 27 55 Nicolas.Obrist@sanofi.com Victor Rouault Tel.: +33 (0)6 70 93 71 40 Victor.Rouault@sanofi.com Investor Relations Contacts Paris Eva Schaefer-Jansen Arnaud Delepine Nathalie Pham Investor Relations Contacts North America Felix Lauscher Tel.: +33 (0)1 53 77 45 45 investor.relations@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 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, 2020. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. 1 Compound Annual Growth Rate from 2018 to 2025 2 From 2018 3 PPH: Polio Pertussis Hib 4 Source: Sanofi internal forecast Attachment 211201_Press_release_Vaccines_Day_EN