PKR

US$5 million PanCAN grant provides important support for the fifth cohort of the GOBLET study Study of modified FOLFIRINOX/pelareorep/atezolizumab (Tecentriq®) combination expands existing pancreatic cancer program First patient expected to be enrolled in Q2 2024 SAN DIEGO and CALGARY, AB, May 9, 2024 /PRNewswire/ -- Oncolytics Biotech® Inc. (NASDAQ: ONCY) (TSX: ONC), a leading clinical-stage company specializing in immunotherapy for oncology, will commence enrollment into a new GOBLET study pancreatic cancer cohort following both German regulatory and ethics approvals. This cohort will evaluate pelareorep in combination with modified FOLFIRINOX (mFOLFIRINOX) with or without the PD-L1 immune checkpoint inhibitor atezolizumab (Tecentriq®) in newly diagnosed patients with pancreatic ductal adenocarcinoma (PDAC). It is supported by a US$5M Therapeutic Accelerator Award from the Pancreatic Cancer Action Network (PanCAN), an innovative program established to accelerate the development of new treatments for pancreatic cancer. The chemotherapy regimens of mFOLFIRINOX or gemcitabine + nab-paclitaxel are the two most common standards of care for pancreatic cancer.1 Oncolytics has already reported data with the combination of gemcitabine and nab-paclitaxel (link to the PR, link to the poster) that surpassed historical outcomes.2-5 Positive results from a combination with mFOLFIRINOX could greatly enhance pelareorep's potential in addressing pancreatic cancer. "Oncolytics is pleased to announce receipt of regulatory clearance to initiate the mFOLFIRINOX cohort in patients with newly diagnosed metastatic PDAC. We appreciate the opportunity to collaborate with PanCAN, Roche, and AIO on this cohort, which is expected to initiate enrollment in the second quarter," said Dr. Matt Coffey, President and Chief Executive Officer of Oncolytics. "We believe that working with PanCAN will help to further enrich Oncolytics' clinical relationships with the pancreatic cancer community. We are also grateful for PanCAN's Therapeutic Accelerator Award, which is enabling the evaluation of this combination therapy." "The Therapeutic Accelerator Award program has been an important part of PanCAN's approach to advancing innovative treatments for pancreatic cancer. We incorporated input from leading scientists and clinicians in the field of pancreatic cancer to select Oncolytics as a recipient of this award," said Anna Berkenblit, MD, MMSc, Chief Scientific and Medical Officer at PanCAN. "Increasing patient access to clinical trials is vital to developing improved treatment options, so we are pleased that Oncolytics has received regulatory clearance for the pelareorep/mFOLFIRINOX combination and is poised to enroll the first patient in this cohort. We hope that the results from this study lead to improved outcomes for patients with pancreatic cancer." Dirk Arnold, M.D., Ph.D., Director of Asklepios Tumorzentrum Hamburg and primary investigator of the GOBLET trial, commented, "Oncolytics has taken a very strategic approach to the development of pelareorep in pancreatic cancer by focusing its clinical studies on combinations with the most widely used treatment regimens. My experience to date with the GOBLET study, including the positive metastatic PDAC and encouraging anal cancer data reported last year, makes me enthusiastic to initiate enrollment in the mFOLFIRINOX cohort." "We previously reported very encouraging results in pancreatic cancer patients for the combination of pelareorep, gemcitabine/nab-paclitaxel, and atezolizumab, and we plan to begin a registration-enabling study of this regimen later this year. The new pelareorep/mFOLFIRINOX cohort offers the opportunity to expand pelareorep's role in pancreatic cancer. If the mFOLFIRINOX combination shows a compelling efficacy signal, this therapeutic approach could also be advanced to a registration-enabling study, providing two opportunities for pelareorep-based treatment to benefit pancreatic cancer patients," said Thomas Heineman, M.D., Ph.D., Chief Medical Officer at Oncolytics. "In addition, translational research studies planned for this cohort will help to further elucidate pelareorep's mechanism of action, including its ability to shape the tumor microenvironment (TME). Notably, we will evaluate the correlation between tumor responses and the expansion of tumor-infiltrating lymphocytes (TILs) in the blood, an effect that was observed in earlier pancreatic cancer studies. We look forward to initiating enrollment into the mFOLFIRINOX/pelareorep study cohort in the second quarter of this year." References Botta G, et al. SWI/SNF complex alterations as a biomarker of immunotherapy efficacy in pancreatic cancer. JCI Insight. 2021;6(18):e150453. Von Hoff D et al. N Engl J Med 2013; 369:1691-1703 DOI: 10.1056/NEJMoa1304369 O'Reilly et al. Eur J Cancer. 2020 June; 132: 112–121. DOI:10.1016/j.ejca.2020.03.005 Karasic et al. JAMA Oncol. 2019 Jul 1; 5(7):993-998. DOI: 10.1001/jamaoncol.2019.0684 Tempero et al. Ann Oncol. 2021 May; 32(5):600-608. DOI: 10.1016/j.annonc.2021.01.070 About GOBLET Cohort 5 The mFOLFIRINOX cohort of the Phase 1/2 GOBLET study is designed to evaluate newly diagnosed PDAC patients treated with pelareorep + mFOLFIRINOX with or without atezolizumab. There will be a three-patient safety run-in to evaluate the tolerability of each treatment arm: pelareorep + mFOLFIRINOX + atezolizumab and pelareorep + mFOLFIRINOX. A total of fifteen evaluable patients will be randomized to each arm in Stage 1 of this Simon-two stage study. The co-primary endpoints are objective response rate and safety. If Stage 1 success criteria are met, one or both treatment arms may be expanded to Stage 2 in which 17 additional evaluable patients per arm will be enrolled. Blood and tumor samples will also be collected for translational evaluations. About GOBLET The GOBLET ( Gastrointestinal tum Ors exploring the treatment com Binations with the oncolytic reovirus pe Lar Eorep and an Ti-PD-L1) study is a phase 1/2 multiple indication study in advanced or metastatic gastrointestinal tumors. The study is being conducted at 12 centers in Germany and is being managed by AIO-Studien-gGmbH. The co-primary endpoints of the study are objective response rate (ORR) and/or disease control rate assessed at week 16 and safety. Key secondary and exploratory endpoints include additional efficacy assessments and evaluation of potential biomarkers (T cell clonality and CEACAM6). The study employs a Simon two-stage design with Stage 1 comprising five treatment groups: Pelareorep in combination with atezolizumab, gemcitabine, and nab-paclitaxel in 1st line advanced/metastatic pancreatic cancer patients; Pelareorep in combination with atezolizumab in 1st line MSI (microsatellite instability)-high metastatic colorectal cancer patients; Pelareorep in combination with atezolizumab and TAS-102 in 3rd line metastatic colorectal cancer patients; Pelareorep in combination with atezolizumab in 2nd line advanced and unresectable anal cancer patients; and Pelareorep in combination with mFOLFIRINOX with and without atezolizumab in newly diagnosed metastatic PDAC patients. Any cohort meeting pre-specified efficacy criteria in Stage 1 may be advanced to Stage 2 and enroll additional patients. About AIO AIO-Studien-gGmbH (AIO) emerged from the study center of the medical oncology working group within the German Cancer Society (DKG). AIO operates with a non-profit purpose of promoting science and research with a focus on medical oncology. Since its foundation, AIO has become a successful sponsor and study management company and has established itself both nationally and internationally. About Oncolytics Biotech Inc. Oncolytics is a clinical-stage biotechnology company developing pelareorep, an intravenously delivered immunotherapeutic agent. Pelareorep has demonstrated promising results in two randomized Phase 2 studies in metastatic breast cancer and Phase 1 and 2 studies in pancreatic cancer. It acts by inducing anti-cancer immune responses and promotes an inflamed tumor phenotype -- turning "cold" tumors "hot" -- through innate and adaptive immune responses to treat a variety of cancers. Pelareorep has demonstrated synergies with multiple approved oncology treatments. Oncolytics is currently conducting and planning combination clinical trials with pelareorep in solid and hematological malignancies as it advances towards registrational studies in metastatic breast cancer and pancreatic cancer, both of which have received Fast Track designation from the FDA. For further information, please visit: or follow the company on social media on LinkedIn and on X @oncolytics. This press release contains forward-looking statements, within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended and forward-looking information under applicable Canadian securities laws (such forward-looking statements and forward-looking information are collectively referred to herein as "forward-looking statements"). Forward-looking statements contained in this press release include Oncolytics' belief as to the potential, mechanism of action and benefits of pelareorep as a cancer therapeutic; our stated goals, objectives and mission; the commencement of enrollment into our new GOBLET study pancreatic cancer cohort and the timing thereof; our belief that positive results from a combination of pelareorep and mFOLFIRINOX could greatly enhance pelareorep's potential in addressing pancreatic cancer, including potentially advancing the approach to a registration-enabling study; our plans to advance towards registrational studies in metastatic breast cancer and pancreatic cancer; and other statements related to anticipated developments in Oncolytics' business and technologies. In any forward-looking statement in which Oncolytics expresses an expectation or belief as to future results, such expectations or beliefs are expressed in good faith and are believed to have a reasonable basis, but there can be no assurance that the statement or expectation or belief will be achieved. Such forward-looking statements involve known and unknown risks and uncertainties, which could cause Oncolytics' actual results to differ materially from those in the forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue research and development projects, the efficacy of pelareorep as a cancer treatment, the success and timely completion of clinical studies and trials, Oncolytics' ability to successfully commercialize pelareorep, uncertainties related to the research and development of pharmaceuticals, uncertainties related to the regulatory process and general changes to the economic environment. In particular, we may be impacted by business interruptions resulting from COVID-19 coronavirus, including operating, manufacturing supply chain, clinical trial and project development delays and disruptions, labour shortages, travel and shipping disruption, and shutdowns (including as a result of government regulation and prevention measures). We may incur expenses or delays relating to such events outside of our control, which could have a material adverse impact on our business, operating results and financial condition. Investors should consult Oncolytics' quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties relating to the forward-looking statements. Investors are cautioned against placing undue reliance on forward-looking statements. The Company does not undertake any obligation to update these forward-looking statements, except as required by applicable laws. Logo: SOURCE Oncolytics Biotech® Inc.

Company developing potent and selective small molecule inhibitors of XRN1, a compelling oncology target to address tumors with high interferon signaling Presentations at Triple Meeting also demonstrate progress for novel DHX9 and potentially first-in-class ADAR1 programs LEXINGTON, Mass., Oct. 10, 2023 /PRNewswire/ -- Accent Therapeutics, a biopharmaceutical company pioneering a new class of small molecule precision cancer therapies, today announced it will present data unveiling a novel, previously undisclosed program in two poster presentations at the 35th AACR-NCI-EORTC Symposium held October 11-15, 2023 in Boston, Massachusetts. The company will also present data supporting development of its ADAR1 and DHX9 programs in two additional poster presentations. 5′→3′ exoribonuclease 1 (XRN1) is an enzyme that degrades single- and double-stranded RNA, playing roles in extracellular mRNA turnover and innate immunity through prevention of dsRNA accumulation and subsequent MDA5 and PKR-mediated immune signaling. Accent has identified XRN1 as a key vulnerability for therapeutic targeting of tumors with elevated type I interferon signaling and is developing small molecule inhibitors of this compelling oncology target, for which there are none currently disclosed. Two of the company's presentations at the meeting describe the preclinical validation of XRN1 as an oncology target and the subsequent characterization of selective inhibitors of XRN1. XRN1 knockout resulted in cell death due to accumulation of dsRNA and downstream activation of the MDA5 and PKR innate immune pathways in cells with intrinsically elevated type I interferon signaling, but not in those with low interferon signaling. Accent has identified novel, potent, and selective allosteric inhibitors that bind XRN1 with nanomolar target affinity without binding to XRN2. "We are excited to share our progress advancing our portfolio and to introduce our XRN1 inhibitor program as a potentially impactful approach to helping cancer patients," said Robert Copeland, Ph.D., President and Chief Scientific Officer of Accent Therapeutics. "These data demonstrate the versatility and promise of pursuing critical intracellular dependences of cancer biology to identify novel oncology targets with the potential to benefit broad patient populations." ADAR1 has emerged as a promising, yet challenging-to-drug, oncology target for which inhibition has been shown to induce downstream immune activation and subsequent cell death in cancer cells with high intrinsic type 1 interferon signaling—a trait that 15-30% of primary tumors share. Accent will also present its innovative suite of in vitro and cellular assays that enable identification of small molecule inhibitors of ADAR1. The resulting compounds are shown to potently inhibit cell growth in a cancer cell line with high interferon type 1 signaling. The company's final presentation at the meeting will highlight its progress developing small molecule inhibitors of the novel oncology target DHX9, an RNA helicase that has been reported to play important roles in the maintenance of genomic stability in multiple cancer types. The presentation will demonstrate that DHX9 inhibition in microsatellite instable tumors exhibiting defective mismatch repair induces replication stress and subsequent cell death. The poster presentations will be archived on the Accent website following the meeting. Details for the presentations are as follows: Title: Exoribonuclease XRN1 is a Therapeutic Vulnerability in Tumors with Intrinsically Elevated Type I Interferon Signaling Abstract Number: A163 Session & Location: Poster Session A – Exhibit Hall D Session Date & Time: Thursday, October 12, 12:30 pm – 4:00 pm ET Presenter: Maureen M. Lynes, Ph.D. Title: Discovery of Small Molecule Inhibitors of ADAR1 Abstract Number: A170 Session & Location: Poster Session A – Exhibit Hall D Session Date & Time: Thursday, October 12, 12:30 pm – 4:00 pm ET Presenter: Shane M. Buker, Ph.D. Title: Characterization of Selective, Allosteric Inhibitors of Human XRN1 Abstract Number: B073 Session & Location: Poster Session B – Exhibit Hall D Session Date & Time: Friday, October 13, 12:30 pm – 4:00 pm ET Presenter: Gordon J. Lockbaum, Ph.D. Title: DHX9 Inhibition as a Novel Therapeutic Modality in Microsatellite Instable Colorectal Cancer Abstract Number: C087 Session & Location: Poster Session C – Exhibit Hall D Session Date & Time: Saturday, October 14, 12:30 pm – 4:00 pm ET Presenter: Jennifer Castro About XRN1 XRN1 is an RNA exonuclease that plays important roles in innate immunity by preventing double-stranded RNA (dsRNA) accumulation. Tumors with intrinsic type I interferon signaling are acutely sensitive to XRN1 loss, making it a strong oncology target. Inhibitors of XRN1 have potential for use as monotherapy and in combination with immuno-oncology therapeutics. Accent is applying structure-based design approaches to develop potent and selective small molecule inhibitors of XRN1 to address cancer indications with high unmet medical need. About ADAR1 Adenosine deaminase acting on RNA 1 (ADAR1) is an enzyme that edits double-stranded RNA (dsRNA) to prevent recognition and downstream immune signaling. In tumor cells with high interferon signaling, small molecule inhibitors of ADAR1 have been shown to induce selective cell killing and immune sensitization, making it a highly attractive target for monotherapy and combination approaches with immunotherapies, such as checkpoint inhibitors. Accent is developing ADAR1 targeted therapies to address many solid tumor indications with significant unmet need, including non-small cell lung cancer, triple-negative breast cancer, ovarian cancer, and head and neck squamous cell carcinomas. About DHX9 Accent's lead program is a first-in-class DHX9 inhibitor with the potential to address high unmet need indications that lack targeted therapies; these include colorectal, endometrial, gastric, and other high microsatellite instable (MSI-H) cancers. Several additional undisclosed cancer types representing large patient populations are also being explored based on their sensitivities to DHX9 inhibition. DHX9 is a DNA/RNA helicase that has been reported to play important roles in replication, transcription, translation, RNA splicing, RNA processing, and maintenance of genomic stability. Hence, this enzyme represents a compelling novel oncology target as inhibition of DHX9 exploits key tumor vulnerabilities, resulting in cancer-specific death. Accent is currently conducting IND-enabling studies evaluating its DHX9 inhibitor. About Accent Therapeutics Accent Therapeutics is pioneering a new class of small molecule precision cancer therapies targeting critical intracellular dependencies that span multiple types of cancer. Building upon industry-leading expertise in RNA-modifying proteins (RMPs) and the systematic mapping of both the RMP space and adjacent high-value areas for drug discovery, the company is building a flexible model that allows for a diversity of approaches to developing potentially transformative biomarker-driven cancer medicines. Accent's therapies are designed for both novel and known, but suboptimally-addressed, high-impact oncogenic targets with the potential to benefit large patient populations with significant unmet need. For more information on Accent's mission to translate extraordinary science into life-changing therapeutics for patients living with cancer, visit or follow us on LinkedIn. SOURCE Accent Therapeutics, Inc.

– Agios to Present New Analyses from ACTIVATE, ACTIVATE-T and Long-Term Extension Studies in Adults with PK Deficiency Reinforcing Clinical Benefit of PYRUKYND® (mitapivat) on Hemoglobin, Hemolysis, Iron Overload and Patient-Reported Outcomes – – Agios to Present Updated Real-World Data on the Burden of Disease in Pyruvate Kinase (PK) Deficiency and Alpha- and Beta-Thalassemia – – Updated Clinical Data to Be Presented from Investigator-Sponsored ESTIMATE Phase 2 Study of PYRUKYND® in Sickle Cell Disease – CAMBRIDGE, Mass., May 11, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that the company and its collaborators will present a broad range of clinical and translational data at the European Hematology Association (EHA) Hybrid Congress, hosted virtually and in person in Frankfurt, Germany, June 8-11, 2023, and continuing virtually on the Congress platform June 14-15, 2023. The accepted abstracts are listed below and are available online on the EHA meeting library website. All posters will be presented during the poster session on Friday, June 9 at 16:30-17:45 CEST. All presentations can be accessed on demand by registered meeting attendees on the EHA Virtual Congress platform until Aug. 15, 2023. Agios-led Abstracts: Poster Presentations Title: Healthcare Resource Use, Economic Burden and In-Patient Mortality in Patients with Alpha- and Beta-Thalassemia Compared to Matched Controls in the Real-World SettingAbstract: P1463Presenting Author: Louise Lombard, M Nutr, Agios Pharmaceuticals, Cambridge, MA, United States Title: Clinically Relevant Hemoglobin Response in Adults with Pyruvate Kinase Deficiency Treated with Mitapivat – A Sub-Analysis of the ACTIVATE TrialAbstract: P1473Presenting Author: Hanny Al-Samkari, M.D., Division of Hematology, Massachusetts General Hospital, Harvard Medical School, Boston, MA, United States Title: The Clinical Characteristics and Overall Survival of Pyruvate Kinase Deficiency Patients in the UK: A Real-World StudyAbstract: P1476Presenting Author: Sarah Higa, PharmD, M.S., Agios Pharmaceuticals, Cambridge, MA, United States Title: Mitapivat Efficacy in Adults with Pyruvate Kinase Deficiency and Baseline Hemoglobin Levels >10 g/dLAbstract: P1477Presenting Author: Rachael F. Grace, M.D., Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA, United States Title: Comorbidities and Complications in Adults with Pyruvate Kinase Deficiency According to Hemoglobin Strata – A Descriptive Analysis from the Peak RegistryAbstract: P1479Presenting Author: Dagmar Pospíšilová, M.D., Ph.D., Department of Pediatrics, Palacky University and University Hospital, Olomouc, Czech Republic Title: PKM And PKR Expression During Hematopoiesis and ErythropoiesisAbstract: P1485Presenting Author: Erin Tsai, M.S., Agios Pharmaceuticals, Cambridge, MA, United States Title: Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency Who Are Regularly TransfusedAbstract: P1497Presenting Author: Eduard J. van Beers, M.D., Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands Publication Only Title: Estimating Utility Values for Health States in Pyruvate Kinase DeficiencyAbstract: PB2547Lead Author: Sarah Higa, PharmD, M.S., Agios Pharmaceuticals, Cambridge, MA, United States Collaborator-led Abstracts: Poster Presentations Title: AG946, A Pyruvate Kinase (PK) Activator Improves PK Properties and Red Blood Cell (RBC) Metabolism Upon Ex Vivo Treatment of RBCs from Patients with Myelodysplastic SyndromesAbstract: P717Presenting Author: Jonathan de Wilde, MSc, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands Title: One-Year Follow-Up of a Phase 2 Study of Mitapivat, an Oral Pyruvate Kinase Activator, for the Treatment of Sickle Cell DiseaseAbstract: P1424Presenting Author: Myrthe J. van Dijk, Ph.D., Division Laboratories, Pharmacy and Biomedical Genetics, CDL en Van Creveldkliniek , University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands Title: Rare Anaemia Disorders European Epidemiological Platform (RADeep): Distribution of Patients Affected by RADs in EuropeAbstract: P1427Presenting Author: Maria Del Mar Mañú Pereira, Ph.D., Vall d'Hebron University Hospital, Group of Translational Research in Cancer and Blood disorders in Children, Barcelona, Spain Title: Mitapivat Ameliorates In Vitro Human β Thalassemic Erythroid Maturation Index and Modulates the Expression of Peroxiredoxin-2Abstract: P1458Presenting Author: Lucia De Franceschi, M.D., Department of Medicine, University of Verona, and Azienda Ospedaliera Universitaria Verona, Policlinico GB Rossi, Verona, Italy Publication Only Title: Glycolytic Activity and Effect of Ex-Vivo Treatment with the Pyruvate Kinase (PK) Activator AG-946 in Red Blood Cells (RBC) From Low-Risk Myelodysplastic Syndromes (LR-MDS) Patients: A Proof-Of-Concept StudyAbstract: PB1990Lead Author: Bruno Fattizzo, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, University of Milan, Milan, Italy Title: A Pilot Study of the International Hemoglobinopathy Research Network (INHERENT)Abstract: PB2515Lead Author: Petros Kountouris, Ph.D., The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus About AgiosAgios is a biopharmaceutical company that is fueled by connections. The Agios team cultivates strong bonds with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver therapies for 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 leadership in the field of cellular metabolism, 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 has a PAH stabilizer in preclinical development as a potential treatment for phenylketonuria (PKU) and deep scientific expertise in classical hematology. For more information, please visit the company’s website at . Cautionary Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of mitapivat and AG-946; Agios’ plans regarding future data presentations; and the potential benefit of its strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” 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. 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; competitive factors; 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; the failure of Agios to receive milestone or royalty payments related to the sale of its oncology business, the uncertainty of the timing of any receipt of any such payments, and the uncertainty of the results and effectiveness of the use of proceeds from the transaction with Servier; 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 ContactCecilia Jones, Chief Financial OfficerAgios PharmaceuticalsIR@agios.com Media Contact Dan Budwick1AB Media dan@1abmedia.com