Agios Announces Clinical Proof-of-Concept in Phase 2a Trial of AG-946 for the Treatment of Anemia in Lower-Risk Myelodysplastic Syndromes
20 November 2023 - 11:00PM
Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field
of cellular metabolism pioneering therapies for rare diseases,
today announced that clinical proof-of-concept has been achieved in
the Phase 2a portion of a study of investigational pyruvate kinase
(PK) activator AG-946 as a potential treatment for anemia in adults
with lower-risk myelodysplastic syndromes (LR-MDS). Four of the 10
patients with low transfusion burden (LTB) achieved the transfusion
independence endpoint, and one of the 22 patients treated in the
study achieved the hemoglobin response endpoint in the 16-week
treatment (core) period. The safety profile observed was consistent
with data reported in the healthy volunteer study. Based on the
favorable efficacy data and positive benefit-risk profile in the
Phase 2a core period, Agios intends to advance its clinical program
evaluating AG-946 in LR-MDS by initiating the placebo-controlled
Phase 2b portion of the study in mid-2024.
“We are pleased with the results of the Phase 2a study, which
underscore the potential of AG-946 to be a first-in-class, oral,
safe and effective option for the treatment of anemia in adults
with LR-MDS by improving red blood cell health through its unique
mechanism of action. Regular blood transfusions are burdensome for
patients. A meaningful reduction in transfusions allows patients to
potentially decrease visits to the clinic and experience improved
quality of life. 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, and we look forward to
advancing the clinical program to Phase 2b,” said Sarah Gheuens,
M.D., Ph.D., chief medical officer and head of R&D at Agios.
“Agios is proud to be the leader in PK activation with two distinct
PK activators – AG-946 and mitapivat – that have the potential to
be meaningful treatment options for a wide range of blood disorders
and positively impact many patients’ lives.”
A total of 22 patients enrolled in the Phase 2a portion of the
study, including 12 classified as non-transfused (NTD) and 10
classified as LTB. Of the 22 patients enrolled in Phase 2a, 19
patients elected to continue to the extension period. Agios intends
to complete a full evaluation of the Phase 2a data and assess the
impact on the Phase 2b portion of the protocol, and present results
of the Phase 2a portion of the study at a medical meeting in
2024.
Phase 2a Design The open-label,
proof-of-concept, Phase 2a portion of the study was designed to
evaluate the safety and efficacy of AG-946 in adults with anemia
due to lower-risk MDS. Patients with a documented diagnosis of MDS
were eligible for the study if they met the Revised International
Prognostic Scoring System (IPSS-R) classification of lower-risk
disease (risk score: ≤3.5) and <5% blasts, as determined by the
participant’s bone marrow biopsy/aspirate during the screening
period, and if their hemoglobin concentration measured <11.0
g/dL during the 4-week screening period. Participants were
classified as non-transfused (NTD) if they had received <3 red
blood cell (RBC) units in the 16-week period before administration
of the first dose of study drug and no transfusions in the 8-week
period before administration of the first dose of study drug, or
low transfusion burden (LTB) if they had received 3 to 7 RBC units
in the 16-week period before administration of the first dose of
study drug and <4 RBC units in the 8-week period before
administration of the first dose of study drug. Participants with a
high transfusion burden were not eligible for the Phase 2a portion
of the study.
Participants received 5mg of oral AG-946 once daily for up to 16
weeks in the core period. At the discretion of the investigator,
participants who completed the core period of the study are
eligible to receive the same dose in an extension period for up to
156 weeks.
The two primary endpoints of the study were transfusion
independence (for participants classified as LTB), defined as
transfusion-free for ≥8 consecutive weeks during the core period,
and hemoglobin response, defined as a ≥1.5 g/dL increase from
baseline in the average hemoglobin concentration from Week 8
through Week 16.
Next month, Agios and its collaborators are presenting
preclinical data supporting its MDS clinical development program –
as well as additional data across the company’s additional
therapeutic areas of focus, including PK deficiency, thalassemia
and sickle cell disease – at the American Society of Hematology
(ASH) Annual Meeting, which is being held December 9-12, 2023, in
San Diego.
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.
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 AG-946; Agios’ plans for the future clinical
development of AG-946 in LR-MDS; 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 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 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 maintain key
collaborations; uncertainty regarding any milestone or royalty
payments related to the sale of its 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
Budwick1ABdan@1abmedia.com
Agios Pharmaceuticals (NASDAQ:AGIO)
Historical Stock Chart
From Apr 2024 to May 2024
Agios Pharmaceuticals (NASDAQ:AGIO)
Historical Stock Chart
From May 2023 to May 2024