Agios to Present Clinical and Translational Data at the European Hematology Association Annual Congress
– Agios to Present New Patient-Reported Outcomes (PRO) Data from ACTIVATE Phase 3 Study of PYRUKYND® (mitapivat) in Adults with PK Deficiency Who Do Not Receive Regular Transfusions –
– Agios to Present New Clinical Data Demonstrating the Normalization of Hemoglobin Levels with Long-term Treatment of PYRUKYND® in Adults with PK Deficiency –
– New Clinical Data from Investigator-Sponsored ESTIMATE Phase 2 Study of PYRUKYND® in Sickle Cell Disease to Be Presented –
– Agios to Host Investor Webcast on
The accepted abstracts are listed below and are available online on the EHA meeting library website. All presentations can be accessed on demand by registered meeting attendees on the
Agios-led Abstracts:
Poster Presentations
Title: Long-term Efficacy and Safety of the Oral Pyruvate Kinase Activator Mitapivat in Adults With Non–transfusion-dependent Alpha- or Beta-thalassemia
Session date and time:
Abstract: P1522
Presenting Author:
Title: Comorbidities and Complications Across Genotypes in Adult Patients With Pyruvate Kinase Deficiency: Analysis From the Peak Registry
Session date and time:
Abstract: P1542
Presenting Author: Andreas Glenthøj, M.D.,
Title: Mitapivat Decreases the Need for Transfusions Secondary to Poorly Tolerated Anemia and Acute Events Compared to Placebo in Patients With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused
Session date and time:
Abstract: P1543
Presenting Author:
Title: Bone Mineral Density Remains Stable in Pyruvate Kinase Deficiency Patients Receiving Long-term Treatment With Mitapivat
Session date and time:
Abstract: P1544
Presenting Author:
Title: Durability of Hemoglobin Response and Reduction in Transfusion Burden Is Maintained Over Time in Patients With Pyruvate Kinase Deficiency Treated With Mitapivat in a Long-term Extension Study
Session date and time:
Abstract: P1545
Presenting Author:
Title: ACTIVATE-kidsT: Mitapivat in Children With Pyruvate Kinase Deficiency Who Are Regularly Transfused
Session date and time:
Abstract: P1546
Presenting Author:
Title: ACTIVATE-kids: Mitapivat in Children With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused
Session date and time:
Abstract: P1547
Presenting Author:
Title: Long-term Treatment With Oral Mitapivat Is Associated With Normalization of Hemoglobin Levels in Patients With Pyruvate Kinase Deficiency
Session date and time:
Abstract: P1548
Presenting Author: Wilma Barcellini, M.D., Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico,
Title: Characterizing Iron Overload by Age in Patients Diagnosed with Pyruvate Kinase Deficiency – a Descriptive Analysis from the Peak Registry
Session date and time:
Abstract: P1562
Presenting Author:
Title: Mitapivat Improves Ineffective Erythropoiesis and Reduces Iron Overload in Patients With Pyruvate Kinase Deficiency
Session date and time:
Abstract: P1565
Presenting Author:
Title: Improvements in Patient-Reported Outcomes in Mitapivat-treated Patients With Pyruvate Kinase Deficiency: A Descriptive Analysis From the Phase 3 ACTIVATE Trial
Session date and time:
Abstract: P1735
Presenting Author:
Publication Only
Title: Systematic Literature Review of Health-related Quality of Life Burden in Patients Across the Spectrum of Thalassemia
Abstract: PB2233
Lead Author:
Title: Pharmacokinetic Modeling and Simulation to Support Mitapivat Dose Selection Used in Pediatric Phase III Studies
Abstract: PB2247
Lead Author:
Title: Healthcare Resource Utilization of Adult Patients With Pyruvate Kinase Deficiency: A Real World Study Using US Veterans Health Administration Data
Abstract: PB2359
Lead Author: Erin Zagadailov, Pharm.D.,
Collaborator-led Abstracts:
Oral Presentations
Title: Evidence of Noninferiority of Mitapivat Versus Splenectomy in Murine Hereditary Spherocytosis
Session Date and Time:
Abstract: S273
Presenter: Alessandro Mattè, Ph.D.,
Poster Presentations
Title: Pharmacodynamic Effects of AG-946, a Highly Potent Novel Activator of Pyruvate Kinase, In Ex
Session date and time:
Abstract: P1492
Lead Author: Minke A.E. Rab, M.D., Ph.D.,
Title: Follow-up Results of a Phase 2 Study Assessing the Safety and Efficacy of Mitapivat Treatment, an Oral Pyruvate Kinase Activator, for up to 60 Weeks in Subjects With Sickle Cell Disease
Session date and time:
Abstract: P1501
Presenting Author:
Publication Only
Title: Adapting Patient Educational Approaches in Pyruvate Kinase Deficiency:
Abstract: PB2345
Presenting Author: Wilma Barcellini, M.D., Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico,
Conference Call Information
Agios will host a virtual investor event on
About Agios
Agios 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 genetically defined diseases. In the
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 mitapivat; 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 or its collaborators 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 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; 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
Investors:
Senior Director, Investor Relations
Holly.Manning@agios.com
Media:
Director, Corporate Communications
Jessica.Rennekamp@agios.com
Source: Agios Pharmaceuticals, Inc.
