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
Agios Pharmaceuticals (NASDAQ:AGIO)
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