Acute Lung Injury

PRESS RELEASE AB SCIENCE RECEIVES NOTICE OF ALLOWANCE FOR EUROPEAN PATENT COVERING MASITINIB UNTIL 2040 IN THE TREATMENT OF SICKLE CELL DISEASE THIS DECISION FURTHER STRENGTHENS MASITINIB’S INTELLECTUAL PROPERTY ADDING ONE MORE INDICATION WITH LONG TERM PROTECTION MASITINIB IS IN PHASE 2 IN SICKLE CELL DISEASE Paris, October 28, 2024, 5.45pm CET AB Science SA (Euronext - FR0010557264 - AB) today announced that the European Patent Office has issued a Notice of Allowance for a patent relating to methods of treating sickle cell disease (i.e. a medical use patent) with its lead compound masitinib, based on preclinical data. This new European patent provides intellectual property protection for masitinib in this indication until November 2040. Professor Olivier Hermine, President of the AB Science Scientific Committee and member of the French Academy of Sciences and Head of Hematology Department at Necker Hospital commented: “Masitinib represents a promising novel strategy for treating sickle cell disease and its serious complication of acute chest syndrome, which can lead to the development of chronic lung disease and is a common cause of hospitalization or even death. There is an increasing need in sickle cell disease with several new drugs being retrieved from market for lack of efficacy or toxicity; however, unlike masitinib, none of these drugs target mast cells”. Sickle cell disease (SCD) is a group of inherited red blood cell disorders, with masitinib being developed to treat the severest forms of the disease, accounting for about 65% of SCD. Severe SCD represents a major public health challenge and leads to early death. Treatment for SCD can be curative based on gene therapy (targets the HbS mutation), however, such an option remains extremely limited due to scarcity of donors, unresolved safety challenges, and very high costs. Standard treatment for SCD includes ​​red blood cell transfusions and treatment with hydroxyurea to manage complications, but significant unmet need remains. Mast cells, a major target for masitinib, appear to play a critical role for the severe forms of SCD and its complications, such as vaso-occlusive crises (VOC), acute chest syndrome (ACS), and pain [1-2]. Masitinib has demonstrated survival benefit in an SCD mouse model: all control SCD mice experienced VOC and 83% died in the first 3 hours, whereas SCD mice pretreated with masitinib for 4 days, experienced no VOCs and no death. Furthermore, lung histology and immune immunohistochemistry showed that masitinib protects from acute lung injuries and mast cell infiltration in an SCD mouse model. Masitinib clinical development in SCD is being conducted as part of the SICKMAST collaborative program, funded with 9.2 million euros [3], which aims to demonstrate in a phase 2 clinical trial the efficacy of masitinib in the treatment of acute and chronic complications of SCD in patients identified based on biomarkers. The Assistance Publique-Hôpitaux de Paris (AP-HP) will be the promoter of these phase 2 studies. AB Science will mainly be involved in supplying masitinib and monitoring masitinib pharmacovigilance data. AB Science remains free to carry out, as it sees fit, any potential phase 3 development following the success of phase 2. Sickle cell disease Sickle cell disease (SCD) is an autosomal recessive disorder affecting millions of people worldwide. Although life expectancy has increased over the last 20 years, acute and chronic complications still result in comorbidities, high social burden and premature death at around 40 years. Approximately 1.1% of couples worldwide are at risk of having a child with a hemoglobin disorder (sickle cell disease or thalassemia), and 2.3 conceptions per 1,000 are affected by sickle cell disease. Estimates suggest that each year, around 300,000 children are born with sickle cell disease, and this number could reach 400,000 by 2050 [4]. Sickle cell disease affects over 100,000 children and adults in the United States. In France, approximately 26,000 patients are affected (50% children, 50% adults). Rationale for the use of masitinib in this indication Inflammation mediated by innate immune cells and promoting vaso-occlusion has recently been shown to play a major role in sickle cell disease. In particular, our clinical observations and experimental work in mice, have revealed the involvement of mast cells and basophils in complications associated with sickle cell disease: The degree of mast cell activation in patients with sickle cell disease may contribute to the heterogeneity of inflammation and chronic and acute complications.The potential role of basophils in sickle cell disease has not been studied, however, given their role in various diseases and their ability to release substance P and histamine, they could also play important roles in the pathophysiology of sickle cell disease. Masitinib is an inhibitor of KIT, LYN, and FYN, three major kinases involved in the activation of mast cells and basophils. Medical need The classic view of sickle cell disease pathophysiology involves polymerization of mutated hemoglobin (HbS) leading to red blood cell (RBC) sickling with subsequent hemolytic anemia, painful vaso-occlusive crisis (VOC) and acute chest syndrome (ACS). Current treatment options such as hydroxycarbamide, chronic transfusion or anti-P-selectin antibodies, do not fully prevent life-threatening acute and chronic complications of sickle cell disease. Allogeneic stem cell transplantation and gene therapy are available only for a minority of patients, are associated with toxicity and are very expensive, which limits their use. There is a significant medical need to prevent the acute and chronic complications of sickle cell disease. References [1] Allali S, Lionnet F, Mattioni S, et al. Br J Haematol. 2019;186(1):125-129. [2] Allali S, Maciel TT, Hermine O, de Montalembert M. Haematologica. 2020;105(2):273-283. [3] AB Science Press Release, 27th November 2023. https://www.ab-science.com/the-clinical-development-of-masitinib-in-sickle-cell-disease-is-among-the-19-winning-projects-under-the-sixth-call-for-hospital-inuversity-research-in-health-rhu/ [4] Piel FB, Steinberg MH, Rees DC. Sickle Cell Disease. N Engl J Med. 2017;376(16):1561-1573. About AB Science Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment. AB Science has developed a proprietary portfolio of molecules and the Company’s lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, inflammatory diseases and viral diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB). Further information is available on AB Science’s website: www.ab-science.com. Forward-looking Statements - AB Science This press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance. These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents published by AB Science. AB Science disclaims any obligation or undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations. For additional information, please contact: AB Science Financial Communication & Media Relations investors@ab-science.com Attachment Sickle Cell Patent EPO vENG VF

Oct. 14, 2024 -- GEn1E Lifesciences, a clinical-stage Phase 2 company, and a leading innovator in precision therapies for inflammatory and rare diseases, with a strong partnership with the University of Maryland, Baltimore (UMB), today announced the publication of a landmark study in the prestigious Journal of Pharmacology and Experimental Therapeutics (JPET). The study, titled First-in-Class Mitogen-Activated Protein Kinase p38α: MAPK-Activated Protein Kinase-2 (MK2) Dual Signal Modulator with Anti-inflammatory and Endothelial-Stabilizing Properties, marks a significant advancement of next-generation immunomodulatory therapeutics for treating inflammatory and autoimmune diseases. “Our findings represent a new era in kinase pathways,” said Paul Shapiro, PhD, co-author and professor in the Department of Pharmaceutical Sciences at the University of Maryland School of Pharmacy and its associate dean for research. “We have reasoned that the past clinical failure of previous generation p38 catalytic inhibitors reflects an intrinsic flaw in the strategy of inhibiting kinase activity. So, we utilized an alternative and unique strategy based on the p38 MAPK structure and functions enabling us to modulate critical inflammatory pathways while minimizing off-target effects - a breakthrough that could transform the treatment of inflammatory diseases.” Jeff Hasday, MD, the Dr. Herbert Berger Professor of Medicine and former Head of the Pulmonary and Critical Care Medicine Division at the University of Maryland School of Medicine and the study's co-contributing author, emphasized the clinical relevance of the findings. “GEn-1124 offers a targeted way to destabilize the p38:MK2 complex without blocking p38 catalytic activity or ablating downstream signaling. The result is a rebalancing of downstream pro- and anti- inflammatory signaling, yielding anti-inflammatory, endothelial-stabilizing, and lung-protective effects with therapeutic potential in pulmonary diseases where inflammation and endothelial barrier dysfunction play key roles.” Key Points from the Study: Targeted Modulation: GEn-1124 precisely disrupts the p38α:MK2 complex, maintaining downstream signaling essential for cellular function while selectively inhibiting pro-inflammatory responses. Efficacy in Preclinical Models: GEn-1124 demonstrated remarkable efficacy in preclinical studies, improving survival rates by 4-fold in acute lung injury and 5-fold in influenza-induced pneumonia along with the compound’s potential to halt disease progression. Endothelial-Stabilizing Properties: The compound showed robust barrier-stabilizing effects in human pulmonary artery endothelial cells (hPAEC), a key factor in treating diseases characterized by vascular leakage and inflammation. The breakthrough findings are well-timed, as GEn1E Lifesciences, which has an exclusive, worldwide and all fields license from UMB is enrolling patients for a Phase 2 study for ARDS, a devastating disease costing $25B to the US society. “We’re thrilled to see the immense potential of next generation dual signal modulators unfold in this study,” said Ritu Lal, CEO, Co-founder of GEn1E Lifesciences and adjunct Associate Professor at the University of Maryland School of Pharmacy. “This breakthrough underscores our mission to develop precision therapies for inflammatory diseases and improve outcomes for patients suffering from devastating conditions.” GEn1E Lifesciences is a clinical-stage, Phase 2 company accelerating novel, dual signal modulator precision therapies for rare and inflammatory diseases. GEn1E Lifesciences is based in Palo Alto, California, with a laboratory in Mountain View, California and a strong collaboration with the University of Maryland, Baltimore. GEn1E has developed a well-differentiated “Platform-in-a-Mechanism” model that uses AI to span the entire end-to-end drug development cycle. The company has accelerated several milestones with novel Signal Modulators being actively developed as first-in-class therapies for rare and inflammatory diseases with its lead compound in Phase 2 currently enrolling patients in the US. The content above comes from the network. if any infringement, please contact us to modify.

$185+ million in incremental orders for ACAM2000® and VIGIV to be delivered in 2024 and 2025GAITHERSBURG, Md., Sept. 25, 2024 (GLOBE NEWSWIRE) -- Emergent BioSolutions Inc. (NYSE: EBS) today announced it has secured approximately $400 million in orders in 2024 and 2025 associated with its vaccinia, smallpox and mpox product portfolio. This includes the previously disclosed U.S. government contract modification to procure ACAM2000®, (Smallpox and Mpox (Vaccinia) Vaccine, Live), as well as CNJ-016® [Vaccinia Immune Globulin Intravenous (Human)] (VIGIV) contract options exercised in 2023 and 2024. To date in 2024, customer orders of nearly $210 million have been delivered for ACAM2000® and VIGIV. For the remainder of 2024 and into 2025, Emergent is confirmed to deliver an incremental $185+ million in ACAM2000® and VIGIV orders. “Emergent continues to be a trusted partner to supply medical countermeasures for biodefense and global health preparedness, and these incremental orders demonstrate our ongoing leadership to help address serious viral threats like smallpox and mpox,” said Joe Papa, president and CEO of Emergent. “As a leader in public health preparedness for 25 years, we recognize that the world is increasingly dangerous, and we believe our products, services and overall capabilities are critical to safeguarding communities against potential public health outbreaks.” Emergent is actively involved in playing its part in addressing the ongoing mpox outbreak. In August, the U.S. Food and Drug Administration approved an update for ACAM2000® to include prevention of mpox disease in individuals determined to be at high risk for mpox infection in its label, in addition to the previously approved smallpox indication. Shortly after achieving this regulatory milestone, Emergent announced that it submitted an Expression of Interest for ACAM2000® vaccine to be assessed for Emergency Use Listing (EUL) with the World Health Organization (WHO). This was in response to the WHO’s invitation for mpox vaccine manufacturers to submit dossiers for EUL evaluation. Emergent is currently in discussions with the WHO surrounding next steps for a potential EUL or Prequalification submission for ACAM2000®. The EUL/Prequalification process is part of the WHO’s broader efforts to support the mpox outbreak response and is a prerequisite of the recent and related UNICEF emergency tender. In furtherance of its commitment to support mpox response efforts, Emergent has donated 50,000 doses of ACAM2000® for potential deployment across impacted countries across the African continent. About ACAM2000®, (Smallpox and Mpox (Vaccinia) Vaccine, Live)ACAM2000® is indicated in the U.S. for active immunization for the prevention of smallpox and mpox disease in individuals determined to be at high risk for smallpox and mpox infection. The labeling for ACAM2000® contains a contraindication for individuals with severe immunodeficiency. Severe localized or systemic infection with vaccinia (progressive vaccinia) may occur in persons with weakened immune systems. Individuals with severe immunodeficiency who are not expected to benefit from the vaccine should not receive ACAM2000®. The risk for experiencing severe vaccination complications must be weighed against the risk for experiencing a potentially severe or fatal smallpox or mpox infection. Additionally, there are warnings and precautions for myocarditis, pericarditis, encephalitis, encephalomyelitis, encephalopathy, progressive vaccinia, generalized vaccinia, severe vaccinial skin infections, erythema multiforme major (including Stevens-Johnson Syndrome), eczema vaccinatum resulting in permanent sequelae or death, accidental eye infection (ocular vaccinia), which can cause ocular complications that may lead to blindness, and fetal death. These may occur following either primary vaccination or revaccination with live vaccinia virus vaccines, including ACAM2000®. These risks are increased in certain individuals and may result in severe disability, permanent neurological sequalae and/or death. Please see the full Prescribing Information for ACAM2000®, (Smallpox and Mpox (Vaccinia) Vaccine, Live) Vaccine for full Boxed Warning and additional safety information. About CNJ-016® [Vaccinia Immune Globulin Intravenous (Human)] (VIGIV)(See full prescribing information for complete boxed warning) WARNING: INTERACTIONS WITH GLUCOSE MONITORING SYSTEMSBlood glucose measurement in patients receiving Vaccinia Immune Globulin Intravenous (Human) (VIGIV) must be done with a glucose-specific method (monitor and test strips) to avoid interference by maltose contained in VIGIV. Maltose in IGIV products may give falsely high blood glucose levels in certain types of blood glucose testing systems (for example those based on the GDH-PQQ or glucose-dye-oxidoreductase methods) resulting in inappropriate administration of insulin and life-threatening hypoglycemia. Cases of true hypoglycemia may go untreated if the hypoglycemic state is masked by falsely elevated glucose readings. Carefully review the product information of the blood glucose testing system, including that of the test strips, to determine if the system is appropriate for use with maltose-containing parenteral products. VIGIV (vaccinia immune globulin intravenous, human) is an Immune Globulin (Human), 5% Liquid, indicated for the treatment of complications due to vaccinia vaccination. VIGIV is indicated for the treatment, and/or modification of the following conditions: eczema vaccinatum; progressive vaccinia; severe generalized vaccinia; vaccinia infections in individuals who have skin conditions such as burns, impetigo, varicella-zoster, or poison ivy; or in individuals who have eczematous skin lesions because of either the activity or extensiveness of such lesions; and aberrant infections induced by vaccinia virus that include its accidental implantation in eyes (except in cases of isolated keratitis), mouth, or other areas where vaccinia infection would constitute a special hazard. VIGIV is not considered to be effective in the treatment of postvaccinial encephalitis. VIGIV is contraindicated in: isolated vaccinia keratitis; Individuals with a history of anaphylaxis or prior severe systemic reaction associated with the parenteral administration of this or other human immune globulin preparations; IgA-deficient patients with antibodies against IgA and a history of IgA hypersensitivity, as it contains trace amounts of IgA (40 mcg/mL).Warnings and Precautions for VIGIV include: Hypersensitivity to human immune globulin (acute anaphylaxis)Acute renal dysfunction/failure. Use VIGIV with caution in patients with pre-existing renal insufficiency and in patients at increased risk of developing renal insufficiency.Thrombosis may occur with immune globulin products, including VIGIV. For patients at risk of thrombosis, administer VIGIV at the minimum dose and infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity.Hemolysis or hemolytic anemiaAseptic meningitis syndrome (AMS)Noncardiogenic pulmonary edema [Transfusion-Related Acute Lung Injury (TRALI)]Transmission of infectious agents from human plasmaMonitor renal function and urine output in patients at risk of renal failure; check baseline blood viscosity in patients at risk of hyperviscosity; and conduct confirmatory tests if hemolysis or TRALI is suspected.Blood glucose monitoring There is no human or animal data for use of VIGIV during pregnancy. VIGIV should only be given to pregnant and nursing women if the potential benefits outweigh the potential risks. It is not known whether VIGIV is excreted in human milk. The safety and efficacy of VIGIV has not been established in pediatric and geriatric populations. The most frequently reported adverse reactions to VIGIV treatment in clinical trials (>10%) include: headache, nausea, rigors, and dizziness. Please see full Prescribing Information for VIGIV for additional safety information. About Emergent BioSolutionsAt Emergent, our mission is to protect and enhance life. For 25 years, we’ve been at work defending people from things we hope will never happen—so we are prepared just in case they ever do. We provide solutions for complex and urgent public health threats through a portfolio of vaccines and therapeutics that we develop and manufacture for governments and consumers. We also offer a range of integrated contract development and manufacturing services for pharmaceutical and biotechnology customers. To learn more about how we plan to protect or enhance 1 billion lives by 2030, visit our website and follow us on LinkedIn, X, Instagram, Apple Podcasts and Spotify. Safe Harbor StatementThis communication includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical fact, including statements regarding the supply and expected timing for delivery of the ACAM2000® and VIGIV, Emergent’s ability to increase inventories of ACAM2000® and VIGIV to meet requested levels within specified time frames, if needed, the discussions with the WHO regarding Emergent’s EUL or Prequalification submission for ACAM2000®, and the WHO’s assessment of such submission and related UNICEF emergency tender, are forward-looking statements. We generally identify forward-looking statements by using words like “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “forecast,” “future,” “goal,” “intend,” “may,” “plan,” “position,” “possible,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would,” and similar expressions or variations thereof, or the negative thereof, but these terms are not the exclusive means of identifying such statements. Forward-looking statements are based on our current intentions, beliefs and expectations regarding future events based on information that is currently available. We cannot guarantee that any forward-looking statement will be accurate. Readers should realize that if underlying assumptions prove inaccurate or unknown risks or uncertainties materialize, actual results could differ materially from our expectations. Readers are, therefore, cautioned not to place undue reliance on any forward-looking statements. Any forward-looking statement speaks only as of the date of this communication and, except as required by law, we do not undertake any obligation to update any forward-looking statement to reflect new information, events or circumstances. There are a number of important factors that could cause our actual results to differ materially from those indicated by any forward-looking statements. Readers should consider this cautionary statement, as well as the risk factors and other disclosures included in our periodic reports filed with the Securities and Exchange Commission, when evaluating our forward-looking statements. Investor Contact:Richard S. LindahlExecutive Vice President, CFOlindahlr@ebsi.com Media Contact:Assal HellmerVice President, Communicationsmediarelations@ebsi.com