Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field
of cellular metabolism to treat rare and genetically defined
diseases, today announced that a broad set of clinical and
translational data from its programs in hemolytic anemias,
including PK deficiency, thalassemia and sickle cell disease, will
be presented at the 64th American Society of Hematology (ASH)
Annual Meeting & Exposition, to be held Dec. 10-13, 2022, in
New Orleans.
In total, 22 abstracts led by Agios and external collaborators
will be presented. 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® in adults with PK
deficiency is associated with sustained clinical benefits,
including improvements in hemoglobin, iron overload, transfusion
burden and patient-reported outcomes. In addition, Agios is
presenting data supporting its pediatric PK deficiency program,
including characterization of disease complications and
co-morbidities in pediatric patients.
Oral Presentation:
Title: Long-term Improvements in
Patient-reported Outcomes in Patients with Pyruvate Kinase
Deficiency Treated with MitapivatPresentation
Time: Sunday, Dec. 11, 2022, at 9:45 a.m. CT Oral
Abstract Session: 904. Outcomes Research—Non-malignant
Conditions: Classical Hematology: From Horses to
ZebrasAbstract: 506Presenter:
Kevin H. M. Kuo, M.D., Division of Hematology, University of
Toronto, Toronto, Canada
Poster Presentations:
Title: Next Generation Sequencing for the
Diagnosis of Hereditary Hemolytic Anemias Including Pyruvate Kinase
Deficiency: Report from a No-cost Diagnostic ProgramPoster
Session: 101. Red Cells and Erythropoiesis, Excluding
Iron: Poster ISession Date and Time: Saturday,
Dec. 10, 2022, 5:30-7:30 p.m. CTAbstract:
1010Lead Author: Jorune Balciuniene, PerkinElmer
Genomics
Title: Mitapivat Improves Iron Overload in
Patients with Pyruvate Kinase DeficiencyPoster
Session: 102. Iron Homeostasis and Biology: Poster
ISession Date and Time: Saturday, Dec. 10, 2022,
5:30-7:30 p.m. CTAbstract: 1021Lead
Author: Kevin H. M. Kuo, M.D., Division of Hematology,
University of Toronto, Toronto, Canada
Title: Long-term Hemoglobin Response and
Reduction in Transfusion Burden Are Maintained in Patients with
Pyruvate Kinase Deficiency Treated with MitapivatPoster
Session: 101. Red Cells and Erythropoiesis, Excluding
Iron: Poster IISession Date and Time: Sunday, Dec.
11, 2022, 6-8 p.m. CTAbstract: 2328Lead
Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston
Children's Cancer and Blood Disorders Center
Title: Comorbidities and Complications in
Pediatric Patients with Pyruvate Kinase Deficiency Enrolled in the
Peak Registry / Peak Pediatric ComorbiditiesPoster
Session: 101. Red Cells and Erythropoiesis, Excluding
Iron: Poster IISession Date and Time: Sunday, Dec.
11, 2022, 6-8 p.m. CTAbstract: 2329Lead
Author: Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston
Children's Cancer and Blood Disorders Center
Title: ACTIVATE-KidsT: Mitapivat in Children
with Pyruvate Kinase Deficiency Who Are Regularly
TransfusedPoster Session: 101. Red Cells and
Erythropoiesis, Excluding Iron: Poster IISession Date and
Time: Sunday, Dec. 11, 2022, 6-8 p.m.
CTAbstract: 2330Lead Author:
Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children's Cancer
and Blood Disorders Center
Title: Age of Onset of Complications in
Patients with Pyruvate Kinase Deficiency: Analysis from the Peak
RegistryPoster Session: 101. Red Cells and
Erythropoiesis, Excluding Iron: Poster IISession Date and
Time: Sunday, Dec. 11, 2022, 6-8 p.m.
CTAbstract: 2332Lead Author:
Andreas Glenthøj, M.D., Ph.D., Department of Haematology,
Copenhagen University Hospital - Rigshospitalet, Copenhagen,
DenmarkTitle: ACTIVATE-Kids: Mitapivat in Children
with Pyruvate Kinase Deficiency Who Are Not Regularly
TransfusedPoster Session: 101. Red Cells and
Erythropoiesis, Excluding Iron: Poster IISession Date and
Time: Sunday, Dec. 11, 2022, 6-8 p.m.
CTAbstract: 2335Lead Author:
Rachael F. Grace, M.D., MMSc, Dana-Farber/Boston Children's Cancer
and Blood Disorders Center
Title: Mitapivat Improves Markers of Hemolysis
and Erythropoiesis in Patients with Pyruvate Kinase Deficiency
Irrespective of Hemoglobin ResponsePoster Session:
101. Red Cells and Erythropoiesis, Excluding Iron: Poster
IIISession Date and Time: Monday, Dec. 12, 2022,
6-8 p.m. CTAbstract: 3644Lead
Author: Hanny Al-Samkari, M.D., Division of Hematology,
Massachusetts General Hospital, Harvard Medical School, Boston, MA,
United States
Publication Only:
Title: The Launch of Two Sub-studies of the
Peak Registry, A Global, Longitudinal Study of Pyruvate Kinase
DeficiencyAbstract: 4960Lead
Author: Carl Lander, Thrive with Pyruvate Kinase
Deficiency Patient Organization
ThalassemiaLong-term Phase 2 data for PYRUKYND®
in adults with alpha- or beta-thalassemia treated for up to 72
weeks show sustained hemoglobin response and improvements in
hemolysis and ineffective erythropoiesis. Additional data
underscore the significant disease burden across both alpha- and
beta-thalassemia.
Poster Presentations:
Title: Mitapivat Improves Markers of
Erythropoietic Activity in Long-term Study of Adults with Alpha- or
Beta-non-transfusion-dependent ThalassemiaPoster
Session: 112. Thalassemia and Globin Gene Regulation:
Poster ISession Date and Time: Saturday, Dec. 10,
2022, 5:30-7:30 p.m. CTAbstract: 1030Lead
Author: Kevin H. M. Kuo, M.D., Division of Hematology,
University of Toronto, Toronto, Canada
Title: Characterizing the Clinical,
Health-related Quality of Life and Economic Burden of
Alpha-thalassemia: A Systematic Literature Review and Evidence Gaps
AssessmentPoster Session: 112. Thalassemia and
Globin Gene Regulation: Poster ISession Date and
Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m.
CTAbstract: 1036Lead Author:
Khaled M. Musallam, M.D., Ph.D., Thalassemia Center, Burjeel
Medical City, Abu Dhabi, United Arab Emirates
Title: Clinical Burden of Alpha- and
Beta-thalassemia Compared to Matched Controls in the Real-world
SettingPoster Session: 112. Thalassemia and Globin
Gene Regulation: Poster IISession Date and Time:
Sunday, Dec. 11, 2022, 6-8 p.m. CTAbstract:
2351Lead Author: Arielle L. Langer, M.D., MPH,
Division of Hematology, Brigham & Women’s Hospital
Sickle Cell DiseaseData to be presented
continue to highlight that PK activation may be a promising
therapeutic approach for patients with sickle cell disease.
Oral Presentations:
Title: Effects of Pyruvate Kinase Activators on
Red Blood Cell Rheology, Sickling and Senescence in Sickle Cell
DiseasePresentation Time: Saturday, Dec. 10, 2022,
at 9:45 a.m. CTOral Abstract Session: 114.
Hemoglobinopathies, Excluding Thalassemia: Clinical and
Epidemiological: Novel TherapiesAbstract:
8Presenter: Philippe Joly, Ph.D., Laboratory of
Biochemistry and Molecular Biology, UF Biochemistry of Red Blood
Cell Diseases, Est Center of Biology and Pathology, Hospices Civils
de Lyon, Lyon France
Title: Untargeted Metabolomics in Dried Blood
Spots of Patients with Sickle Cell Disease Treated with the
Pyruvate Kinase Activator MitapivatPresentation
Time: Saturday, Dec. 10, 2022, at 10 a.m. CTOral
Abstract Session: 114. Hemoglobinopathies, Excluding
Thalassemia: Clinical and Epidemiological: Novel
TherapiesAbstract: 9Presenter:
Myrthe J. van Dijk, Van Creveldkliniek, University Medical Center
Utrecht, Utrecht University, Utrecht, The Netherlands
Title: AG-946 Normalizes Glycolysis and
Improves Red Cell Indices in a Humanized Sickle Cell Mouse
ModelPresentation Time: Sunday, Dec. 11, 2022, at
9:30 a.m. CTOral Abstract Session: 113.
Hemoglobinopathies, Excluding Thalassemia: Basic and Translational:
Targeting the Red Blood Cell: Novel Therapeutic Approaches in
Sickle Cell DiseaseAbstract: 391Lead
Author: Rohitash Jamwal, Ph.D., Agios Pharmaceuticals
Title: PKLR Variants Associated with Acute Pain
in Sickle Cell Disease Influence ATP Concentrations in Red Blood
CellsPresentation Time: Sunday, Dec. 11, 2022, at
10 a.m. CTOral Abstract Session: 113.
Hemoglobinopathies, Excluding Thalassemia: Basic and Translational:
Targeting the Red Blood Cell: Novel Therapeutic Approaches in
Sickle Cell DiseaseAbstract:
393Presenter: Xunde Wang, Ph.D., Sickle Cell
Branch, National Heart Lung and Blood Institute, National
Institutes of Health
Poster Presentations:
Title: The Pyruvate Kinase Activator Mitapivat
Improves Red Blood Cell Deformability and Sickling Kinetics in
Adult Patients with Sickle Cell DiseasePoster
Session: 113. Hemoglobinopathies, Excluding Thalassemia:
Basic and Translational: Poster ISession Date and
Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1044Lead Author:
Maureen Lundt, NHLBI / NIH, Bethesda, Maryland, United States
Title: Feasibility of Near-infrared
Spectroscopy for Monitoring Hemodynamic Changes in Sickle Cell
Disease Patients Treated with MitapivatPoster
Session: 113. Hemoglobinopathies, Excluding Thalassemia:
Basic and Translational: Poster ISession Date and
Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m. CT
Abstract: 1053 Lead Author:
Timothy Quang, National Institute of Child Health and Human
Development
Title: Activating Pyruvate Kinase Improves Red
Blood Cell Integrity by Reducing Band3 Tyrosine
PhosphorylationPoster Session: 113.
Hemoglobinopathies, Excluding Thalassemia: Basic and Translational:
Poster IISession Date and Time: Sunday, Dec. 11,
2022, 6-8 p.m. CT Abstract: 2367Lead
Author: Kang Le, Ph.D., National Institutes of Health
AG-946New clinical data from the Phase 1
healthy volunteers study of Agios’ novel PK activator, AG-946,
support the initiation of a Phase 2a/2b study in lower-risk
myelodysplastic syndromes.
Poster Presentations:
Title: A Phase 2a/2b Multicenter Study of
AG-946 in Patients with Anemia Due to Lower-risk Myelodysplastic
SyndromesPoster Session: 637. Myelodysplastic
Syndromes – Clinical and Epidemiological: Poster ISession
Date and Time: Saturday, Dec. 10, 2022, 5:30-7:30 p.m.
CTAbstract: 1773Lead Author:
Hanny Al-Samkari, M.D., Division of Hematology, Massachusetts
General Hospital, Harvard Medical School, Boston, MA, United
States
Title: Results from the Single and Multiple
Ascending Dose Study to Assess the Safety, Tolerability,
Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy
VolunteersPoster Session: 114. Hemoglobinopathies,
Excluding Thalassemia: Clinical and Epidemiological: Poster
IISession Date and Time: Saturday, Dec. 10, 2022,
5:30-7:30 p.m. CTAbstract: 2383Lead
Author: Xiaoshu Dai, Ph.D., Agios Pharmaceuticals
Conference Call InformationAgios will host a
live investor event on Dec. 12, 2022, at 7:00 a.m.
ET in New Orleans 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 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
and genetically defined 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 multiple investigational therapies
in preclinical development and an industry-leading research team
with unmatched expertise in cellular metabolism and genetics. For
more information, please visit the company’s website at
www.agios.com.
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,” “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; 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 ;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:Holly Manning, 617-844-6630Senior
Director, Investor RelationsHolly.Manning@agios.com
Media:Jessica Rennekamp, 857-209-3286Senior
Director, Corporate
CommunicationsJessica.Rennekamp@agios.com
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