University of Verona

LUND, Sweden, Oct. 7, 2024 /PRNewswire/ -- Immunovia (IMMNOV: Nasdaq Stockholm), the pancreatic cancer diagnostics company, today announces that the company has acquired all blood samples required to clinically validate its next-generation test for pancreatic cancer. Concluding the sample collection effort marks a key milestone toward successfully completing the clinical validation study. The study seeks to confirm the sensitivity and specificity reported previously in the company's model-development study of its new test. The trial will be a case-control study evaluating the accuracy of the next-generation test in differentiating blood samples from people with early-stage pancreatic cancer from individuals without it. Immunovia has secured over 1,000 blood samples, including over 200 from patients with stage 1 and 2 pancreatic cancer and more than 800 blood samples from high-risk individuals without cancer. The study will be one of the largest clinical validation studies of a pancreatic cancer biomarker test conducted to date. Immunovia leveraged long-standing relationships with researchers at pancreatic cancer centers in Europe and North America to acquire the blood samples. Key collaborators who contributed an especially large number of samples included Dr. Randall Brand, University of Pittsburgh Department of Medicine; Dr. Rosie Sears, Oregon Health and Science University; and Dr. Salvatore Paiella, University of Verona.  This study marks Immunovia's first research collaboration with the Pancreatic Cancer Early Detection (PRECEDE) Consortium. The PRECEDE Consortium, led by Dr. Diane Simeone, is an international, multi-institutional collaborative group of experts working to improve early detection of pancreatic cancer through a novel model of collaboration and data sharing. The PRECEDE Consortium provided nearly 500 samples from its ongoing study of people at high-risk for hereditary cancer.  "We are excited to announce that we have secured all samples required to clinically validate our next-generation test. This study represents a pivotal step in our mission to bring early detection of pancreatic cancer to high-risk individuals," says Jeff Borcherding, CEO and President of Immunovia. As communicated previously, Immunovia expects to complete the clinical validation study in December 2024 in preparation for launching its next-generation test for early detection of pancreatic cancer later in 2025. For more information, please contact: Jeff Borcherding CEO and President [email protected] Karin Almqvist Liwendahl Chief Financial Officer [email protected] +46 709 11 56 08 Immunovia in brief Immunovia AB is a diagnostic company whose mission is to increase survival rates for patients with pancreatic cancer through early detection. Immunovia is focused on the development and commercialization of simple blood-based testing to detect proteins and antibodies that indicate a high-risk individual has developed pancreatic cancer. Immunovia collaborates and engages with healthcare providers, leading experts and patient advocacy groups to make its test available to individuals at increased risk for pancreatic cancer. USA is the world's largest market for detection of pancreatic cancer. The company estimates that in the USA, 1.8 million individuals are at high-risk for pancreatic cancer and could benefit from annual surveillance testing. Immunovia's shares (IMMNOV) are listed on Nasdaq Stockholm. For more information, please visit  This information was brought to you by Cision . The following files are available for download: SOURCE Immunovia AB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

World-renowned scientists and clinicians to provide expert insight and advice to support advancement of the company’s lead Phase 3 asset, new indications, and pipeline expansion SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Quince Therapeutics, Inc. (Nasdaq: QNCX), a late-stage biotechnology company developing an innovative drug delivery technology that leverages a patient’s own biology to deliver rare disease therapeutics, today announced the launch of a Scientific Advisory Board (SAB) comprised of leading experts in biochemistry, neurology, immunology, hematology, pharmacology, and clinical practice. “We are pleased to announce the formation of our Scientific Advisory Board to support Quince in our scientific and medical programs,” said Dirk Thye, M.D., Quince’s Chief Executive Officer and Chief Medical Officer. “We have assembled a highly distinguished group of scientific and clinical thought leaders who are uniquely positioned to provide deep insights and advice, including to support the advancement of our Phase 3 lead asset, EryDex, for the potential treatment of a rare neurodegenerative disease, Ataxia-Telangiectasia.” Dr. Mauro Magnani, Ph.D., Chair of Quince’s Scientific Advisory Board, said, “Quince’s AIDE technology is designed to harness the power of a patient’s own red blood cells to optimize the pharmacokinetics and biodistribution of a therapeutic and deliver sustained treatment. I look forward to collaborating with the renowned members of Quince’s management team, Board of Directors, and Scientific Advisory Board to support the company’s mission to bring its innovative drug delivery technology to patients living with rare diseases.” Quince’s newly formed SAB includes the following seven founding members: Mauro Magnani, Ph.D., Chair of the SAB, is a Professor of Biochemistry at the University of Urbino, Italy and is a co-founder of Quince’s proprietary Autologous Intracellular Drug Encapsulation (AIDE) technology platform. Dr. Magnani developed and patented a method for using autologous red blood cells that allow for the slow delivery of different drugs in circulation to treat patients in need. His areas of interest and current research include red blood cells as drug delivery systems and as circulating bioreactors, development and delivery of biologics, and nanomaterials in drug delivery and imaging. Dr. Magnani has authored more than 500 papers published in international peer-reviewed scientific journals, holds 16 patents, and has co-edited three books, in addition to serving as an editorial board member for several biomedical journals and referee for different international scientific institutions. He is a member of the National Committee for Biotechnology, Presidency of the Italian Government, and an Italian delegate at the Organization for Economic Cooperation and Development and at the European Commission. Dr. Magnani received a Ph.D. in Biochemistry from the University of Urbino, Italy. Carlo Brugnara, M.D., is a Professor of Pathology at Harvard Medical School and Director of the Hematology Laboratory at Boston Children’s Hospital. Dr. Brugnara’s clinical laboratory-based research has focused on the use of hematological parameters to assess the balance between iron availability and erythropoiesis. He has described the appearance of functional iron deficiency in normal subjects treated with recombinant human erythropoietin (r-HuEPO) based on the particular flow cytometric characteristics of erythrocytes and reticulocytes. Dr. Brugnara also has shown the clinical value of reticulocyte parameters in the setting of r-HuEPO use and in the diagnosis of iron deficiency in children. He is a fellow of the American Society of Clinical Investigation, the Association of American Physicians, and the Academy of Clinical Laboratory Physicians and Scientists, in addition to serving as the Editor-in-Chief of the American Journal of Hematology. He received his M.D. from the University of Verona, Italy and completed his postdoctoral fellowship at the laboratory of Dr. Daniel Tosteson in the Department of Physiology at Harvard Medical School. Dr. Brugnara also trained in Clinical Pathology and Transfusion Medicine at Brigham and Women’s Hospital in Boston and holds board certifications in both specialties. William Jusko, Ph.D., is a State University of New York Distinguished Professor of Pharmaceutical Sciences at the Buffalo University Jacobs School of Medicine and Biomedical Sciences. Dr. Jusko’s research interests are in theoretical, basic, and clinical aspects of the pharmacokinetics and pharmacodynamics of various immunosuppressive agents, including corticosteroids, as well as drugs used to treat diabetes, inflammation, and cancer. His research expertise includes the diverse effects of corticosteroids and he has evolved advanced mathematical models of receptor/gene-mediated responses. Dr. Jusko has developed mechanism-based pharmacokinetic, pharmacodynamic, and disease progression models and computational methods describing the action of various drugs and utilizes mathematical models of drug action to determine optimal dosage regimens. He has authored more than 600 publications, is a fellow of several societies, serves on the editorial boards of seven journals, and is the former Editor-in-Chief of the Journal of Pharmacokinetics & Pharmacodynamics. Dr. Jusko received a Ph.D. in Pharmaceutical Sciences from the State University of New York at Buffalo. Howard Lederman, M.D., Ph.D., is a Professor of Pediatrics at the Johns Hopkins University School of Medicine and Director of the Immunodeficiency Clinic, the Pediatric Immunology Laboratory, and the Ataxia-Telangiectasia (A-T) Clinical Center at Johns Hopkins Medical Center. Dr. Lederman specializes in the evaluation, diagnostic testing, and long-term management of patients of all ages who have known or suspected primary immunodeficiency diseases. Considered one of the world’s leading experts in the rare neurodegenerative disease A-T, Dr. Lederman’s current laboratory and clinical research focuses on better understanding and treating A-T patients, and will participate as an investigator in Quince’s upcoming Phase 3 clinical trial of its lead asset, EryDex. He is a member of numerous professional societies, including the American Association of Immunologists, the American Society for Microbiology, and the Clinical Immunology Society. Dr. Lederman received a M.D. and Ph.D. from the University of Michigan Medical School and completed a residency in pediatrics at Johns Hopkins Children’s Center and a fellowship in immunology at the Hospital for Sick Children in Toronto. He is board certified by the American Board of Pediatrics. Vladimir Muzykantov, M.D., Ph.D., is a Founders Professor of Nanoparticle Research at the University of Pennsylvania, Philadelphia (UPENN), Professor and Vice-Chair of the Department of Systems Pharmacology and Translational Therapeutics at the Perelman School of Medicine, UPENN, and Founding Director of the Center for Translational Targeted Therapeutics and Nanomedicine, UPENN. Dr. Muzykantov conducts research in drug/gene targeting in the vascular system, including devising drug delivery systems for precise molecular interventions in the lungs, heart, brain, spleen, and other organs and blood components, including host defense agents. Since the early 1980s, he has explored red blood cells as natural carriers for prolonged circulation and site-specific delivery of drugs aimed at regulation of bleeding, clotting, thrombolysis, inflammation, and complement. He also investigates the mechanisms controlling drug delivery at the level of whole organism to nanoscale regulation of intracellular targeting/trafficking of drugs. He holds a portfolio of intellectual property, including about 40 patents and disclosures, and has authored more than 280 publications. Dr. Muzykantov was elected as a co-Chair of the Gordon Research Conference on Drug Carriers and was a recipient of the Established Investigator Award and Bugher Stroke Award from the American Heart Association. He received a M.D. in Internal Medicine from the First Moscow Medical School and a Ph.D. in Biochemistry from the Russian National Cardiology Research Center in Moscow. Susan Perlman, M.D., is a Clinical Professor in the Department of Neurology at the David Geffen School of Medicine at the University of California, Los Angeles and Director of the UCLA Ataxia and Neurogenetics Clinical Trials Programs and Post-Polio Program. Leveraging three decades as a clinical professor of Neurology, Dr. Perlman is an expert in building subspecialty clinics that diagnose and treat patients living with rare, progressive, and incurable disorders, including Friedreich’s ataxia, Huntington’s disease, and all types of genetic and non-genetic cerebellar ataxias, such as A-T. She has long been a site primary investigator for Friedreich's ataxia trials, sits on the Medical Advisory Board of the National Ataxia Foundation, and will participate as an investigator in Quince’s upcoming Phase 3 clinical trial of its lead asset, EryDex. Dr. Perlman currently oversees four natural history studies and serves as an investigator for numerous clinical trials as she works to spearhead the development of disease-modifying therapies to address the significant unmet needs in rare disease. She began her specialty work in chronic diseases of the neuromuscular system, including muscular dystrophy, spinal cord diseases, and cerebral palsy. Dr. Perlman was awarded the Sherman M. Mellinkoff Faculty Award at the Hippocratic Oath ceremony of the graduating class of 2008, which is considered the highest faculty distinction at the David Geffen School of Medicine. She received a M.D. from the Renaissance School of Medicine at Stony Brook University and completed a residency in Neurology and a two-year Muscular Dystrophy Association fellowship in Neurology at the University of California, Los Angeles. James Spudich, Ph.D., is the Douglass M. and Nola Leishman Professor of Cardiovascular Disease in the Department of Biochemistry at the Stanford University School of Medicine. Over the last five decades, the Spudich laboratory studied the structure and function of the myosin family of molecular motors in vitro and in vivo, and they developed multiple new tools, including in vitro motility assays taken to the single molecule level using laser traps. That work led to his laboratory’s current focus at Stanford on the human cardiac sarcomere and the molecular basis of hypertrophic and dilated cardiomyopathy. Dr. Spudich postulated in 2015 that a majority of hypertrophic cardiomyopathy mutations are likely to be shifting beta-cardiac myosin heads from a sequestered off-state to an active on-state for interaction with actin, resulting in the hyper-contractility seen clinically in hypertrophic cardiomyopathy (HMC) patients. This unifying hypothesis is different from earlier prevailing views, and this viewing an old disease in a new light has become the favored view in the field of the molecular basis of hypercontractility caused by HCM mutations. Dr. Spudich has given more than 50 named lectureships and keynote addresses and has received many honors, including election to the National Academy of Sciences and recipient of the Albert Lasker Basic Medical Research Award. He is credited with co-founding MyoKardia Inc, which was acquired by Bristol Myers Squibb for $13.1 billion in 2020, and Cytokinetics, Inc., a late-stage, specialty cardiovascular biopharmaceutical company. Dr. Spudich received a Ph.D. in Biochemistry from Stanford University, in addition to completing his postdoctoral work in Genetics at Stanford University and in Structural Biology at the MRC Laboratory at Cambridge University. He is a fellow of the American Academy of Arts and Sciences and a member of the National Academy of Sciences. Quince’s SAB will provide expert insight and advice to support the advancement of the company’s lead asset, EryDex, targeting A-T, which is expected to begin enrollment for its pivotal Phase 3 clinical trial in the second quarter of 2024. The SAB also will be a valuable resource as Quince looks to strategically expand its development pipeline to include additional potential rare disease indications for EryDex, as well as additional potential applications of its AIDE technology platform targeting rare and debilitating disease programs. About Quince Therapeutics Quince Therapeutics (Nasdaq: QNCX) is a late-stage biotechnology company dedicated to unlocking the potential of a patient’s own biology to deliver innovative and life-changing therapeutics to those living with rare diseases. For more information on the company and its latest news, visit and follow Quince Therapeutics on social media platforms LinkedIn, Facebook, and Twitter/X. Forward-looking Statements Statements in this news release contain “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 as contained in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the “safe harbor” created by those sections. All statements, other than statements of historical facts, may be forward-looking statements. Forward-looking statements contained in this news release may be identified by the use of words such as “believe,” “may,” “should,” “expect,” “anticipate,” “plan,” “believe,” “estimated,” “potential,” “intend,” “will,” “can,” “seek,” or other similar words. Examples of forward-looking statements include, among others, statements relating to current and future clinical development of EryDex, expansion of the company’s proprietary autologous intracellular drug encapsulation (AIDE) technology for treatment of other rare diseases, and benefits and impact of Quince’s Scientific Advisory Board. Forward-looking statements are based on Quince’s current expectations and are subject to inherent uncertainties, risks, and assumptions that are difficult to predict and could cause actual results to differ materially from what the company expects. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. Factors that could cause actual results to differ include, but are not limited to, the risks and uncertainties described in the section titled “Risk Factors” in the company’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 14, 2023, and other reports as filed with the SEC. Forward-looking statements contained in this news release are made as of this date, and Quince undertakes no duty to update such information except as required under applicable law.

– Both Mitapivat Dose Arms Achieved Statistically Significant Hemoglobin Response in Phase 2 Portion of RISE UP Pivotal Study in Sickle Cell Disease, Compared to the Placebo Arm; Data Support Advancement to Phase 3 Portion of RISE UP – – Agios to Highlight Additional Data on the Burden of Disease in Pyruvate Kinase (PK) Deficiency and Alpha- and Beta-Thalassemia – – New Preclinical Data for Novel PK Activator AG-946 Suggest Improvements in Ineffective Erythropoiesis in Model of Myelodysplastic Syndromes (MDS) – – Agios to Host Live and Webcast Investor Event on Dec. 11, 2023, at 7:00 a.m. Pacific Time – CAMBRIDGE, Mass., Nov. 02, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that a broad set of clinical and translational data from its programs will be presented at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition, to be held Dec. 9-12, 2023, in San Diego. The presentations will focus on rare blood disorders, including PK deficiency, thalassemia, sickle cell disease and anemia associated with lower-risk myelodysplastic syndromes (LR-MDS). In total, 23 abstracts led by Agios and external collaborators will be presented or published. The accepted abstracts are listed below and are available online on the ASH conference website at https://www.hematology.org/meetings/annual-meeting/abstracts. PK DeficiencyData to be presented highlight that long-term treatment with PYRUKYND® (mitapivat) in adults with PK deficiency is associated with sustained clinical benefits, including improvements in hemoglobin, iron overload, and decreased burden of disease on work and school activities. Poster Presentations: Title: Mitapivat Treatment Reduces Levels of Interference in Work/School Activity for Adult Patients with Pyruvate Kinase DeficiencyPoster Session: 904. Outcomes Research – Non-Malignant Conditions: Poster ISession Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PTAbstract: 2365 Lead Author: Jennifer A. Rothman, MD, Duke University Medical Center, Durham, NC Title: Understanding the Physical and Psychosocial Impacts of Pyruvate Kinase Deficiency: Patient-Led Development of the Pyruvate Kinase Deficiency Life Phase ModelPoster Session: 901. Health Services and Quality Improvement - Non-Malignant Conditions: Poster II Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PTAbstract: 3691 Lead Author: Rachael F. Grace, MD, MMSc, Dana-Farber/Boston Children’s Cancer and Blood Disorder Center, Harvard Medical School, Boston, MA Publication Only: Title: Improvements in Markers of Hemolysis and Liver Iron Concentration in Mitapivat-Treated Adult Patients with a Delayed Hemoglobin ResponseAbstract: 5266Lead Author: Eduard J. van Beers, MD, PhD; Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands Title: Regional Genetic Heterogeneity Among Patients with Pyruvate Kinase DeficiencyAbstract: 5203Lead Author: Paola Bianchi, BSc, PhD; Hematology Unit, Pathophysiology of Anemia Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy ThalassemiaData for PYRUKYND® in adults with beta-thalassemia show evidence of reduced oxidative stress in red blood cells, as well as enhanced PK activity and metabolic reprogramming. Additionally, qualitative data demonstrate the equally common burden of disease across alpha- and beta-thalassemia patients, regardless of transfusion dependency. Poster Presentations: Title: Mitapivat Treatment Increases β-thalassemic Erythroblasts Energy Production and Responsiveness to Oxidative Stress Poster Session: 112. Thalassemia and Globin Gene Regulation: Poster IIISession Date and Time: Monday, Dec. 11, 2023, 6:00-8:00 p.m. PTAbstract: 3850Lead Author: Alessandro Matte, PhD; Department of Medicine, University of Verona and AOUI Verona, Verona, Italy Publication Only: Title: Association of Hemoglobin Levels With Healthcare Resource Utilization and Costs in Non–Transfusion-Dependent α- and β-Thalassemia: A Retrospective Observational Study Using Real-World DataAbstract: 5244Lead Author: Arielle L. Langer, MD; MPH, Division of Hematology, Brigham & Women’s Hospital, Harvard Medical School, Boston MA Title: Burden of Illness of Alpha- and Beta-Thalassemia: A Qualitative StudyAbstract: 7329Lead Author: Sujit Sheth, MD; Joan and Sanford I Weill Medical College of Cornell University, New York, NY Title: Investigating Health Literacy in Thalassemia: Founding a Patient-Led Research Approach Abstract: 5251Lead Author: Sujit Sheth, MD; Joan and Sanford I Weill Medical College of Cornell University, New York, NY Sickle Cell DiseaseResults from the Phase 2 portion of Agios’ RISE UP study will highlight improvements in hemoglobin response rates, markers of hemolysis and erythropoiesis and reductions in annualized rates of pain crises for mitapivat compared to placebo, supporting advancement into the Phase 3 portion of RISE UP. Agios will showcase its patient-centric approach to sickle cell disease clinic trials, including the incorporation of patient insights and decision making into the clinical trial design and campaign. Oral Presentations: Title: A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study of Mitapivat in Patients With Sickle Cell Disease: RISE UP Phase 2 ResultsPresentation Time: Saturday, Dec. 9, 2023, at 4:00 p.m. PTOral Abstract Session: 114. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Clinical and Epidemiological: Building on Momentum in Disease-Modifying Therapeutics for Sickle Cell DiseaseAbstract: 271Presenter: Modupe Idowu, MD; McGovern Medical School, UT Health, Houston, TX Title: Long-term safety and Efficacy of Mitapivat, an Oral Pyruvate Kinase Activator, in Adults with Sickle Cell Disease: Extension of a Phase 1 Dose Escalation StudyPresentation Time: Saturday, Dec. 9, 2023, at 4:30 p.m. PTOral Abstract Session: 114. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Clinical and Epidemiological: Building on Momentum in Disease-Modifying Therapeutics for Sickle Cell DiseaseAbstract: 273Presenter: Swee Lay Thein, MBBS, DSc, FRCP, FRCPath, MRCP, MRCPath; Sickle Cell Branch, National Heart, Lung & Blood Institute, NIH, Bethesda, MD Title: Pyruvate Kinase Thermostability Is Associated with Red Blood Cell Adhesion, Deformability and Oxygen Affinity in Patients with Sickle Cell DiseasePresentation Time: Sunday, Dec. 10, 2023, at 5:00 p.m. PTOral Abstract Session: 113. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Pathophysiology of Sickle Hemoglobinopathies: From Mice to HumansAbstract: 561Lead Author: Marissa J.M. Traets, PhD Candidate; Department of Central Diagnostic Laboratory - Research, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands Poster Presentations: Title: A Patient-Centric Approach to Sickle Cell Disease Clinical Trials: Integrating Patient Perspectives in the RISE UP Phase 2/3 Trial of Mitapivat for Informed Protocol Design and Associated Patient Community BenefitPoster Session: 904. Outcomes Research – Non-Malignant Conditions: Poster I Session Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PT Abstract: 2376Lead Author: Charles Jonassaint, PhD, MHS; School of Medicine, University of Pittsburgh, Pittsburgh, PA Title: One-Year Safety and Efficacy of Mitapivat in Sickle Cell Disease: Follow-Up Results of a Phase 2, Open-Label StudyPoster Session: 114. Sickle cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Clinical and Epidemiological: Poster IISession Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PTAbstract: 2515Lead Author: Myrthe J. van Dijk, PhD; Division Laboratories, Pharmacy and Biomedical Genetics, CDL en Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands Title: Functional and Multi-omics Signatures of Mitapivat Efficacy Upon Activation of Pyruvate Kinase in Red Blood Cells from Patients with Sickle Cell DiseasePoster Session: 113. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Poster IISession Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT Abstract: 2485Lead Author: Angelo D’Alessandro, PhD; Biochemistry and Molecular Genetics, University of Colorado Anschutz Medical Campus, Aurora, CO Title: Longitudinal Characterization of Hemodynamic Changes with Multimodal Optical Techniques in Patients with Sickle Cell Disease Treated with MitapivatPoster Session: 113. Sickle Cell Disease, Sickle Cell Trait and Other Hemoglobinopathies, Excluding Thalassemias: Basic and Translational: Poster IISession Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PT Abstract: 2492Lead Author: Timothy Quang, PhD; Section on Biomedical Optics, National Institute of Child Health and Human Development, NIH, Bethesda, MD Publication Only: Title: Outpatient Costs of Patients with Sickle Cell Disease With or Without Hydroxyurea at an Institution in Rio de Janeiro, BrazilAbstract: 7325Lead Author: Tarun Aurora, MD; Department of Global Pediatric Medicine, St Jude Children’s Research Hospital, Memphis, TN AG-946New data from preclinical studies of Agios’ AG-946 continue to support its novel mechanism of action and PK activation as a promising potential treatment option for anemia associated with lower-risk myelodysplastic syndromes. Poster Presentations: Title: AG-946, An Activator of Pyruvate Kinase, Improves Ineffective Erythropoiesis in the Bone Marrow of Mouse Models of Myelodysplastic SyndromesPoster Session: 636. Myelodysplastic Syndromes – Basic and Translational: Poster ISession Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PTAbstract: 1854 Lead Author: Megan Wind-Rotolo, PhD, Agios Pharmaceuticals, Cambridge, MA Title: The Pyruvate Kinase (PK) Activator AG-946 Improves PK Properties and Red Blood Cell (RBC) Characteristics upon Ex Vivo Treatment of RBCs from Patients with Myelodysplastic SyndromesPoster Session: 636. Myelodysplastic Syndromes—Basic and Translational: Poster II Session Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PTAbstract: 3222Lead Author: Jonathan R.A. de Wilde, PhD Candidate; Red Blood Cell Research Group, Central Diagnostic Laboratory-Research, University Medical Center Utrecht, Utrecht University, Utrecht, the Netherlands Publication Only: Title: Biochemical and Metabolomic Analysis of Glycolytic Activity in Red Blood Cells (RBC) from Low-risk Myelodysplastic Syndromes (LR-MDS) Patients and In-vitro Effect of the Pyruvate Kinase (PK) Activator AG-946Abstract: 6456Lead Author: Bruno Fattizzo, MD; Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy Patient AdvocacyAgios is fueled by connections, with patient and KOL engagement at the core of our mission. Information presented provides an overview of the formation and vision of the Red Cell Revolution – a multi-stakeholder, patient-advocacy data collection approach to understanding the unmet needs of the patients, caregivers and healthcare professionals for PK deficiency, sickle cell disease and thalassemia. Poster Presentation: Title: Cross-community Collaboration and Data Collection to Optimize Patient Care in Hemolytic AnemiasPoster Session: 901. Health Services and Quality Improvement - Non-Malignant Conditions: Poster IISession Date and Time: Sunday, Dec. 10, 2023, 6:00-8:00 p.m. PTAbstract: 3692Lead Author: Biree Andemariam, MD; New England Sickle Cell Institute, University of Connecticut Health, Farmington, CT Publication Only: Title: Setting Industry Standards for Patient Engagement, Partnership, Allyship and Care: The Patient Vision Project Abstract: 7233Lead Author: Biree Andemariam, MD; New England Sickle Cell Institute, University of Connecticut Health, Farmington, CT OtherAgios’ collaborators present new data evaluating mitapivat as a potential treatment for other rare blood diseases. Poster Presentation: Title: Ex Vivo Treatment by Mitapivat, an Allosteric Pyruvate Kinase Activator, Reduced Oxidative Stress and Promoted Terminal Erythropoiesis in a Severe Hemolytic Anemia Patients Due to Krϋppel-like Factor 1 MutationsPoster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster ISession Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PTAbstract: 1071Lead Author: Thidarat Suksangpleng, PhD.; Siriraj-Thalassemia Center, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand Title: Safety and Efficacy of Mitapivat Sulfate in Adult Patients with Erythrocyte Membranopathies (SATISFY)Poster Session: 101. Red Cells and Erythropoiesis, Excluding Iron: Poster ISession Date and Time: Saturday, Dec. 9, 2023, 5:30-7:30 p.m. PTAbstract: 1085Lead Author: Andreas Glenthøj, MD, PhD; Department of Haematology, Copenhagen University Hospital - Rigshospitalet, Copenhagen, Denmark Conference Call InformationAgios will host a live investor event on Dec. 11, 2023, at 7:00 a.m. PT in San Diego to review the key clinical oral and poster presentations from this year’s ASH meeting. The event will be webcast live and can be accessed under “Events & Presentations” in the Investors and Media section of the company's website at www.agios.com. The archived webcast will be available on the company's website beginning approximately two hours after the event. About AgiosAgios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company's deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency and MDS-associated anemia. In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera, and a preclinical PAH stabilizer as a potential treatment for phenylketonuria (PKU). For more information, please visit the company’s website at www.agios.com. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND® (mitapivat) and AG-946; Agios’ plans regarding future data presentations; and the potential benefit of its strategic plans and focus. The words “anticipate,” “expect,” “intend,” “potential,” “milestone,” “goal,” “will,” “on track,” “upcoming,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios' current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios' product candidates will successfully continue. Moreover, there can be no guarantee that any medicines ultimately commercialized by Agios will receive commercial acceptance. There can be no guarantee that any positive developments in Agios' business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of the COVID-19 pandemic or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to maintain key collaborations; uncertainty regarding any milestone or royalty payments related to the sale of its oncology business or its in-licensing of TMPRSS6 siRNA, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of proceeds from the transaction with Servier; competitive factors; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Contacts: Investor ContactChris Taylor, VP, Investor Relations and Corporate CommunicationsAgios PharmaceuticalsIR@agios.com Media ContactDan Budwick1AB Mediadan@1abmedia.com