Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field
of cellular metabolism and pioneering therapies for rare diseases,
today announced that the global Phase 3 ENERGIZE study of mitapivat
in adults with non-transfusion-dependent (NTD) alpha- or
beta-thalassemia achieved its primary endpoint of hemoglobin
response. Statistical significance was also achieved for both key
secondary endpoints associated with change from baseline in
FACIT-Fatigue Score and hemoglobin concentration.
“The results of the Phase 3 ENERGIZE study underscore the
potential of mitapivat to be a meaningful treatment option for
adults with non-transfusion dependent alpha- or beta-thalassemia.
All subgroup analyses favored the mitapivat treatment arm compared
to placebo,” said Sarah Gheuens, M.D., Ph.D., chief medical officer
and head of R&D at Agios. “We are grateful to all of the
patients who participated in this trial, our collaborators, study
investigators and advisors in the patient and clinical communities
for their partnership in achieving this milestone. These data bring
us one step closer to a treatment for all thalassemia patients, and
we look forward to the ENERGIZE-T readout mid-year.”
“The results of the ENERGIZE study support the potential of
mitapivat to be the first oral therapy for all NTD thalassemia
patients, including those with alpha- or beta-thalassemia,” said
Ali Taher, M.D., Ph.D., Professor of Medicine, Hematology &
Oncology and Director – Naef K. Basile Cancer Institute, American
University of Beirut Medical Center in Beirut, Lebanon. “For NTD
thalassemia patients across the globe, there are currently no
approved oral treatments, and NTD thalassemia has consistently been
associated with morbidity and mortality if left untreated. NTD
thalassemia represents over half of clinically significant forms of
thalassemia, so there is a tremendous unmet need. Based on the data
reported to date, mitapivat has the potential to be a foundational
treatment option for the thalassemia community.”
Agios is also advancing the fully enrolled Phase 3 ENERGIZE-T
study of mitapivat in adults with transfusion-dependent alpha- or
beta-thalassemia and expects to announce topline data from this
48-week study in mid-2024. Following the read-out of ENERGIZE-T,
the company intends to file for regulatory approval of mitapivat as
a treatment for thalassemia by the end of 2024, incorporating all
available data from both studies.
Topline results for the Phase 3 ENERGIZE study were as
follows:
- A total of 194 patients were enrolled in the study, with 130
randomized to mitapivat 100 mg twice-daily (BID) and 64 randomized
to matched placebo. 122 (93.8%) in the mitapivat arm and 62 (96.9%)
in the placebo arm completed the 24-week double-blind period of the
study.
- The study met the primary endpoint of hemoglobin response.
Hemoglobin response was defined as an increase of ≥1 g/dL in
average hemoglobin concentrations from Week 12 through Week 24
compared with baseline.
- Treatment with mitapivat demonstrated a statistically
significant increase compared to placebo.
- 42.3% of patients in the mitapivat arm achieved a hemoglobin
response, compared to 1.6% of patients in the placebo arm (2-sided
p<0.0001).
- Treatment with mitapivat also demonstrated statistically
significant improvements compared to placebo for both key secondary
endpoints:
- Change from baseline in average FACIT-Fatigue (Functional
Assessment of Chronic Illness Therapy-Fatigue) subscale score from
Week 12 to Week 24.
- Change from baseline in average hemoglobin concentration from
Week 12 to Week 24.
- Overall, during the 24-week double-blind period, incidence of
adverse events was similar across mitapivat and placebo arms. Four
(3.1%) subjects in the mitapivat arm experienced adverse events
(AEs) leading to discontinuation; there were no AEs leading to
discontinuation in the placebo arm.
Agios plans to present a more detailed analysis of the Phase 3
ENERGIZE data at an upcoming medical meeting.
Conference Call InformationAgios will host a
webcast investor event today at 8:00 a.m. ET to review the ENERGIZE
Phase 3 data and next steps for the Phase 3 ENERGIZE-T study. The
event 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 PYRUKYND®
(mitapivat)PYRUKYND is a pyruvate kinase activator
indicated for the treatment of hemolytic anemia in adults with
pyruvate kinase (PK) deficiency in the United States, and
for the treatment of PK deficiency in adult patients in
the European Union.
IMPORTANT SAFETY INFORMATIONAcute
Hemolysis: Acute hemolysis with subsequent anemia has been
observed following abrupt interruption or discontinuation of
PYRUKYND in a dose-ranging study. Avoid abruptly discontinuing
PYRUKYND. Gradually taper the dose of PYRUKYND to discontinue
treatment if possible. When discontinuing treatment, monitor
patients for signs of acute hemolysis and anemia including
jaundice, scleral icterus, dark urine, dizziness, confusion,
fatigue, or shortness of breath.
Adverse Reactions: Serious adverse reactions
occurred in 10% of patients receiving PYRUKYND in the ACTIVATE
trial, including atrial fibrillation, gastroenteritis, rib
fracture, and musculoskeletal pain, each of which occurred in 1
patient. In the ACTIVATE trial, the most common adverse reactions
including laboratory abnormalities (≥10%) in patients with PK
deficiency were estrone decreased (males), increased urate, back
pain, estradiol decreased (males), and arthralgia.
Drug Interactions:
- Strong CYP3A Inhibitors and Inducers: Avoid concomitant
use.
- Moderate CYP3A Inhibitors: Do not titrate PYRUKYND beyond 20 mg
twice daily.
- Moderate CYP3A Inducers: Consider alternatives that are not
moderate inducers. If there are no alternatives, adjust PYRUKYND
dosage.
- Sensitive CYP3A, CYP2B6, CYP2C Substrates Including Hormonal
Contraceptives: Avoid concomitant use with substrates that have
narrow therapeutic index.
- UGT1A1 Substrates: Avoid concomitant use with substrates that
have narrow therapeutic index.
- P-gp Substrates: Avoid concomitant use with substrates that
have narrow therapeutic index.
Hepatic Impairment: Avoid use of PYRUKYND in
patients with moderate and severe hepatic impairment.
Please see full Prescribing
Information and Summary of
Product Characteristics for
PYRUKYND.
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
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; Agios’ plans for
the future clinical development of mitapivat in alpha-and-beta
thalassemia; and Agios’ strategic plans and prospects. The words
“anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,”
“plan,” “predict,” “project,” “would,” “could,” “potential,”
“possible,” “hope” 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 ContactChris Taylor, VP Investor
Relations and Corporate CommunicationsAgios
PharmaceuticalsIR@agios.com
Media Contact Dan Budwick1AB
dan@1abmedia.com
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