Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field
of cellular metabolism to treat genetically defined diseases, today
announced that a broad set of clinical and translational data from
its genetically defined disease programs in chronic hemolytic
anemias, including pyruvate kinase (PK) deficiency, thalassemia and
sickle cell disease, will be presented at the American Society of
Hematology (ASH) Annual Meeting & Exposition, to be held Dec.
11-14, 2021, virtually and in person in Atlanta.
In total, eight abstracts led by Agios will be presented, as
well as five abstracts led by external collaborators. The accepted
abstracts are listed below and are available online on the ASH
conference website:
https://www.hematology.org/meetings/annual-meeting/abstracts.
Presentations by Agios
Oral Presentations:
Title: Long-term Efficacy and Safety of the
Oral Pyruvate Kinase Activator Mitapivat in Adults with
Non-transfusion-dependent Alpha- or Beta-Thalassemia
Presentation Time: Monday, Dec. 13,
2021, at 11:45 a.m. ETOral Abstract
Session: 112. Thalassemia and Globin Gene
RegulationAbstract: 576Presenter:
Kevin H. M. Kuo, M.D., Division of Hematology, University
of Toronto, Toronto, Canada
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 StudyPresentation
Time: Monday, Dec. 13, 2021, at 6:30 p.m.
ETOral Abstract Session: 101. Red Cells and
Erythropoiesis, Excluding Iron: Normal and Perturbed
ErythropoiesisLocation: Georgia World Congress
Center, B401-B402Abstract:
848Presenter: Rachael Grace, M.D., MMSc,
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
Poster Presentations:Title:
Bone Mineral Density Remains Stable in Pyruvate Kinase Deficiency
Patients Receiving Long-term Treatment with Mitapivat
Poster Session: 101. Red Cells and Erythropoiesis,
Excluding Iron: Poster I Session Date and Time:
Saturday, Dec. 11, 2021, from 5:30-7:30 p.m.
ETAbstract: 924Lead
Author: Hanny Al-Samkari, M.D., Division of
Hematology, Massachusetts General Hospital, Harvard
Medical School, Boston, MA, United States
Title: Survey of 275 Patients and Caregivers
Affected by Pyruvate Kinase Deficiency: Impact of Communication
with Hematologists on Mental Health and Quality of
LifePoster Session: 904. Outcomes
Research—Non-Malignant Conditions: Poster I Session Date
and Time: Saturday, Dec. 11, 2021, from 5:30-7:30 p.m.
ETAbstract: 152326 Lead Author:
Rachael Grace, M.D., MMSc, Dana-Farber/Boston Children's Cancer and
Blood Disorders Center
Title: Mitapivat Improves Ineffective
Erythropoiesis and Reduces Iron Overload in Patients with Pyruvate
Kinase DeficiencyPoster Session: 102. Iron
Homeostasis and Biology: Poster II Session Date and
Time: Sunday, Dec. 12, 2021, from 6-8 p.m.
ETAbstract: 2005Lead
Author: Eduard J. van Beers, M.D., Ph.D., Department
of Internal Medicine, University Medical Center Utrecht
Title: Phase 1 Single and Multiple Ascending
Dose Study to Assess the Safety, Tolerability, Pharmacokinetics,
and Pharmacodynamics of AG-946 in Healthy VolunteersPoster
Session: 113. Hemoglobinopathies, Excluding Thalassemia:
Poster IISession Date and Time: Sunday, Dec. 12,
2021, from 6-8 p.m. ETAbstract: 2043Lead
Author: Varsha Iyer, Ph.D., Agios Pharmaceuticals
Title: Characterizing Iron Overload by Age in
Patients Diagnosed with Pyruvate Kinase Deficiency – A Descriptive
Analysis from the PEAK RegistryPoster Session:
102. Iron Homeostasis and Biology: Poster IIISession Date
and Time: Monday, Dec. 13, 2021, from 5:30-7:30 p.m.
ETAbstract: 3074Lead
Author: Paola Bianchi, BSc, Ph.D., UOC
Ematologia, Fondazione IRCCS Ca’ Granda Ospedale Maggiore
Policlinico, Milan, Italy
Title: A Phase 2/3, Randomized, Double-blind,
Placebo-controlled Study of Mitapivat in Patients with Sickle Cell
DiseasePoster Session: 114. Hemoglobinopathies,
Excluding Thalassemia: Clinical and Epidemiological: Poster
IIISession Date and Time: Monday, Dec. 13, 2021,
from 6-8 p.m. ETAbstract: 3109Lead
Author: Joanna Howard, MB BChir, MRCP, Department of
Hematology, Guy’s and St. Thomas’ NHS Foundation Trust, London,
United Kingdom
Publication Only:
Title: Validation of the Pyruvate Kinase
Deficiency Impact Assessment (PKDIA): A Patient-reported Outcome
Measure for Pyruvate Kinase (PK)
DeficiencyAbstract: 4145Lead
Author: Shayna Egan, MPH, Endpoint Outcomes
Title: Validation of the Pyruvate Kinase
Deficiency Diary (PKDD): A Patient-reported Outcome Measure for
Pyruvate Kinase (PK) DeficiencyAbstract:
4144Lead Author: Shayna Egan, MPH, Endpoint
Outcomes
Presentations by External Collaborators
Oral Presentations:
Title: Mitapivat (AG-348) Demonstrates Safety,
Tolerability, and Improvements in Anemia, Hemolysis, Oxygen
Affinity, and Hemoglobin S Polymerization Kinetics in Adults with
Sickle Cell Disease: A Phase 1 Dose Escalation
StudyPresentation Time: Saturday, Dec. 11,
2021, at 10:15 a.m. ETOral Abstract
Session: 114. Hemoglobinopathies, Excluding Thalassemia:
Clinical and Epidemiological: New Therapies for Sickle Cell
DiseaseAbstract:
10Presenter: Julia Z. Xu, M.D., Sickle Cell
Branch, National Heart, Lung, and Blood Institute, National
Institutes of Health
Poster Presentations:
Title: Ex Vivo Evaluation of Erythrocyte
Adhesion and Whole Blood Thrombosis in PKD SubjectsPoster
Session: 101. Red Cells and Erythropoiesis, Excluding
Iron: Poster ISession Date and Time: Saturday,
Dec. 11, 2021, from 5:30-7:30 p.m. ETAbstract:
923Lead Author: Patrick C. Hines, M.D.,
Ph.D., Wayne State University School of Medicine
Title: Safety and Efficacy of Mitapivat
(AG-348), An Oral Activator of Pyruvate Kinase-R, in Subjects with
Sickle Cell Disease: A Phase 2, Open-label Study
(ESTIMATE)Poster Session: 102. Iron Homeostasis
and Biology: Poster IISession Date and Time:
Sunday, Dec. 12, 2021, from 6-8 p.m. ETAbstract:
2005Lead Author: Myrthe J. van Dijk, Van
Creveldkliniek, University Medical Center Utrecht, Utrecht
University, Utrecht, The Netherlands
Title: Pharmacodynamic Effects of AG-946, A
Highly Potent Next-generation Activator of Pyruvate Kinase, In Ex
Vivo Treatment of Red Blood Cells from Sickle Cell Disease
PatientsPoster Session: 113. Hemoglobinopathies,
Excluding Thalassemia: Basic and Translational: Poster
IISession Date and Time: Sunday, Dec. 12, 2021,
from 6-8 p.m. ETAbstract: 2029Lead
Author: Minke A. E. Rab, University Medical Center
Utrecht
Title: Mitapivat Improves Transfusion Burden
and Reduces Iron Overload in Thalassemic MicePoster
Session: 112. Thalassemia and Globin Gene Regulation:
Poster IISession Date and Time: Sunday, Dec. 12,
2021, from 6-8 p.m. ETAbstract: 2016Lead
Author: Alessandro Mattè, Ph.D., University of Verona
and AOUI Verona, Verona, Italy
Conference Call InformationAgios will host a
virtual investor event on Dec. 14, 2021, at 7:30
a.m. ET 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 focused on discovering and
developing novel investigational medicines to treat genetically
defined diseases through scientific leadership in the field of
cellular metabolism. The company’s most advanced drug candidate is
a first-in-class pyruvate kinase R (PKR) activator, mitapivat, that
is currently being evaluated for the treatment of three distinct
hemolytic anemias. In addition to its active late-stage clinical
pipeline, Agios has multiple novel, investigational therapies in
clinical and preclinical development. 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 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.
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 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; the impact of the COVID-19 pandemic on
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
future approved products, and launching, marketing and selling
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, including with respect to the regulatory
submissions for mitapivat, 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 and 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 establish and maintain
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, or SEC, including the risks and
uncertainties set forth under the heading Risk Factors in our
filings with the SEC. While the list of factors presented here is
considered representative, this list should not be considered to be
a complete statement of all potential risks and uncertainties. Any
forward-looking statements contained in this communication are made
only as of the date hereof, and we undertake no obligation to
update forward-looking statements to reflect developments or
information obtained after the date hereof and disclaim any
obligation to do so other than as may be required by law.
Contacts
Investors:Holly Manning, 617-844-6630Senior
Director, Investor RelationsHolly.Manning@agios.com
Media:Jessica Rennekamp, 857-209-3286Director,
Corporate CommunicationsJessica.Rennekamp@agios.com
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