Agios Announces Key Anticipated 2024 Milestones Across Rare Disease Portfolio
08 January 2024 - 11:00PM
Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field
of cellular metabolism pioneering therapies for rare diseases,
today announced its anticipated 2024 milestones and value-driving
catalysts through 2026 that support the company’s mission to
transform patient outcomes in rare diseases. Agios will present at
the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday,
January 10, 2024, at 7:30 a.m. PT, and a live webcast will be
available at investor.agios.com.
“We were pleased to announce positive topline data from the
Phase 3 study of our lead PK activator, mitapivat, in
non-transfusion-dependent alpha- or beta-thalassemia last week, a
segment of the population with no currently approved therapeutic
options in the U.S. We look forward to data readouts from four
additional Phase 3 studies across our industry-leading PK activator
franchise by the end of 2025,” said Brian Goff, chief executive
officer at Agios. “This robust series of near-term catalysts
positions Agios for potential launches of a first- and
best-in-class therapy in thalassemia in 2025 and in sickle cell
disease in 2026, and we look forward to maximizing the commercial
opportunities ahead of us. Supported by our strong cash position,
Agios is poised for significant progress in the next 12-24 months,
and we look forward to the opportunity to deliver a novel oral
treatment option for two additional hematologic diseases with high
unmet need.”
2023 Highlights
- Thalassemia: Completed enrollment in
the Phase 3 ENERGIZE and ENERGIZE-T studies of mitapivat in
non-transfusion-dependent and transfusion-dependent thalassemia,
respectively
- Sickle Cell Disease: Announced
positive data from the Phase 2 portion of the RISE UP study of
mitapivat and dosed the first patients in the Phase 3 portion
- Pediatric PK Deficiency: Completed
enrollment in the Phase 3 ACTIVATE kids-T study of mitapivat in
children with PK deficiency who are regularly transfused. Enrolled
more than half of patients in the Phase 3 ACTIVATE-kids study of
mitapivat in children with pediatric PK deficiency who are not
regularly transfused
- Lower-risk Myelodysplastic Syndromes
(LR-MDS): Announced clinical proof-of-concept in Phase 2a study of
AG-946, supporting continued development in Phase 2b
- Earlier-stage Pipeline: Filed an
Investigational New Drug Application (IND) for PAH stabilizer for
the treatment of phenylketonuria (PKU)
- Business Development: Announced
exclusive worldwide license agreement with Alnylam for novel siRNA
targeting TMPRSS6 for the potential treatment of polycythemia vera
(PV)
- Data Presentations: Presented broad
set of clinical and translational data at the 65th American Society
of Hematology (ASH) Annual Meeting & Exposition, including
positive data from the Phase 2 portion of the RISE UP study of
mitapivat in sickle cell disease
Anticipated 2024 Milestones
- Thalassemia: Following the
announcement of topline data from the Phase 3 ENERGIZE study last
week, Agios plans to report topline data from the Phase 3
ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia
(mid-year) and submit a New Drug Application (NDA) for mitapivat in
thalassemia (year-end)
- Sickle Cell Disease: Complete
enrollment in the Phase 3 portion of the RISE UP study of mitapivat
(year-end)
- Pediatric PK Deficiency: Complete
enrollment in the Phase 3 ACTIVATE-kids study of mitapivat
(mid-2024). Report topline data from Phase 3 ACTIVATE kids-T study
(year-end)
- Lower-risk Myelodysplastic Syndromes
(LR-MDS): Dose first patient in Phase 2b study of AG-946
(mid-year)
- Earlier-stage Pipeline: Dose the
first patient in the Phase 1 study of PAH stabilizer for the
treatment of PKU (H1 2024)
Four Additional Phase 3 Readouts and Two Potential New
Indication Approvals Expected by End of 2026
2024
- Data readout from Phase 3 ENERGIZE
study of mitapivat in non-transfusion-dependent thalassemia
(announced January 3, 2024)
- Data readout from Phase 3 ENERGIZE-T
study of mitapivat in transfusion-dependent thalassemia
(mid-year)
- Data readout from Phase 3 ACTIVATE
kids-T study of mitapivat in pediatric PK deficiency
(year-end)
2025
- Data readout from Phase 3 portion of
the RISE UP study of mitapivat in sickle cell disease
- Data readout from Phase 3 ACTIVATE
kids study of mitapivat in pediatric PK deficiency
- Potential FDA approval for mitapivat
in thalassemia
2026
- Potential FDA approval for mitapivat
in sickle cell disease
- Potential FDA approval for mitapivat
in pediatric PK deficiency
Presentation at 42nd Annual J.P. Morgan Healthcare
Conference
Agios will webcast its corporate presentation from the 42nd
Annual J.P. Morgan Healthcare Conference on Wednesday, January 10
at 7:30 a.m. PT. A live webcast of the presentation can be accessed
under “Events & Presentations” in the Investors section of the
company’s website at agios.com. A replay of the webcast will be
archived on the Agios website for at least two weeks following the
presentation.
About Agios
Agios 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 (PV),
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), AG-946, Agios’s
PAH stabilizer and its novel siRNA Targeting TMPRSS6; Agios’ plans,
strategies and expectations for its preclinical, clinical and
commercial advancement of its drug development, including
PYRUKYND®, AG-946, its PAH stabilizer and its novel siRNA Targeting
TMPRSS6; Agios’ strategic vision and goals, including its key
milestones for 2024 and potential catalysts through 2026; and the
potential benefits of Agios’s strategic plans and focus. The words
“anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,”
“plan,” “predict,” “project,” “would,” “could,” “potential,”
“possible,” “hope,” “strategy” and “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
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 pandemics 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 establish and
maintain key collaborations; uncertainty regarding any milestone or
royalty payments related to the sale of Agios’ 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 Contact
Chris Taylor, VP Investor Relations and Corporate
Communications
Agios Pharmaceuticals
IR@agios.com
Media Contact
Dan Budwick
1AB
dan@1abmedia.com
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