Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular
metabolism and pyruvate kinase (PK) activation pioneering therapies
for rare diseases, today announced that the global Phase 3
ENERGIZE-T study of mitapivat in adults with transfusion-dependent
(TD) alpha- or beta-thalassemia achieved its primary endpoint of
transfusion reduction response. Statistical significance was also
achieved for all key secondary endpoints evaluating additional
measures of reduction of transfusion burden compared to placebo.
“Building on the compelling data generated in the Phase 3
ENERGIZE study of mitapivat in adults with
non-transfusion-dependent alpha- or beta- thalassemia announced
earlier this year, today’s results underscore the potential of
mitapivat, with its unique mechanism of action improving red blood
cell health, to be a meaningful oral treatment option for all
thalassemia patients, regardless of transfusion needs,” said Sarah
Gheuens, M.D., Ph.D., chief medical officer and head of R&D at
Agios. “We are grateful to all patients who participated in this
trial, as well as our collaborators, study investigators, and
advisors in the patient and clinical communities for their
partnership. We look forward to submitting a marketing application
in the U.S. encompassing data from ENERGIZE and ENERGIZE-T by the
end of this year.”
“Taken together with the positive data from the ENERGIZE study,
the data from the ENERGIZE-T study have the potential to be
transformative for thalassemia patient care. Treatment options for
patients with transfusion-dependent thalassemia are limited, and
transfusions carry significant risks for patients, such as iron
overload and immune reactions. There is a tremendous need for
alternative ways to manage this chronic disease,” said Maria
Domenica Cappellini, M.D., Professor, Internal Medicine, University
of Milan, Italy. “Based on these data demonstrating that treatment
with mitapivat significantly reduces transfusion burden across
alpha- and beta-thalassemia patients, along with its convenient
oral formulation, mitapivat has the potential to become a novel
advancement in care for thalassemia patients.”
With the positive data generated in the Phase 3 ENERGIZE-T and
ENERGIZE studies of mitapivat in patients with alpha- or beta-
thalassemia regardless of transfusion needs, the company intends to
submit a marketing application for PYRUKYND® (mitapivat) in the
U.S. by the end of 2024 based on all available data from both
studies. The company also plans to submit marketing applications in
Europe and the Gulf Cooperation Council (GCC) countries.
Topline results for the Phase 3 ENERGIZE-T study were as
follows:
- A total of 258 patients were
enrolled in the study, with 171 randomized to mitapivat 100 mg
twice-daily (BID) and 87 randomized to matched placebo. 155
patients (90.6%) in the mitapivat arm and 83 patients (95.4%) in
the placebo arm completed the 48-week double-blind period of the
study.
- The study met its primary endpoint
of transfusion reduction response (TRR, defined as a ≥50% reduction
in transfused red blood cell (RBC) units with a reduction of ≥2
units of transfused RBCs in any consecutive 12-week period through
Week 48 compared with baseline).
- Treatment with mitapivat demonstrated a statistically
significant reduction in transfusion burden compared to placebo, as
measured by the TRR endpoint. In the mitapivat arm, 30.4% of
patients achieved a transfusion reduction response, compared to
12.6% of patients in the placebo arm (2-sided p=0.0003).
- Treatment with mitapivat also
demonstrated a statistically significant reduction in additional
measures of transfusion reduction response compared to placebo as
assessed by the three key secondary endpoints:
- ≥50% reduction in transfused RBC
units in any consecutive 24-week period through week 48 compared
with baseline.
- ≥33% reduction in transfused RBC
units from week 13 through week 48 compared with baseline.
- ≥50% reduction in transfused RBC
units from week 13 through week 48 compared with baseline.
- In addition, a higher proportion of
patients in the mitapivat arm (9.9%) compared to the placebo arm
(1.1%) achieved the secondary endpoint of transfusion independence
(transfusion-free for ≥8 consecutive weeks through week 48).
- Overall, during the 48-week
double-blind period, incidence of adverse events (AEs) was similar
across mitapivat and placebo arms. In the mitapivat arm, 5.8% of
the patients experienced an AE leading to discontinuation, compared
to 1.2% of patients in the placebo arm.
- Based on the safety and efficacy
data observed in ENERGIZE-T, the company will proceed with the
previously stated plans of U.S. regulatory submission by end of
this year.
Agios plans to present a more detailed analysis of the Phase 3
ENERGIZE-T data at an upcoming medical meeting. Data from the Phase
3 ENERGIZE study of mitapivat in non-transfusion-dependent
thalassemia will be presented at the European Hematology
Association 2024 Hybrid Congress in a plenary session on June 15,
2024, and in a poster session on June 14, 2024.
Conference Call Information
Agios will host a webcast investor event today at 8:00 a.m. ET
to review the ENERGIZE-T Phase 3 data and next steps for the
mitapivat development program in thalassemia. 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 INFORMATION
Acute 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 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, 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 mitapivat; Agios’ plans for the future
clinical development of mitapivat in alpha-and-beta thalassemia;
Agios’ plans for future regulatory submissions; 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 Agios’ cash and cash equivalents;
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
Budwick1ABdan@1abmedia.com
Agios Pharmaceuticals (NASDAQ:AGIO)
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