Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field
of cellular metabolism pioneering therapies for rare and
genetically defined diseases, today announced that the European
Commission (EC) has granted marketing authorization for PYRUKYND®
for the treatment of PK deficiency in adult patients. PYRUKYND® is
a first-in-class, oral PK activator and the first approved
disease-modifying therapy for patients in the EU with this rare,
debilitating, lifelong hemolytic anemia.
“People with PK deficiency suffer from a lifetime of chronic
anemia, associated complications and symptoms that can be
detrimental to their work, family and social lives,” said Andreas
Glenthøj, hematologist and head of the Danish Center for
Hemoglobinopathies at Rigshospitalet, and associate professor at
the University of Copenhagen. “PYRUKYND® offers new hope for this
community, and I am honored to have contributed to the research
efforts that enabled the approval of the first therapy for adults
with PK deficiency in the EU.”
“With today’s EU approval, we are proud to expand the positive
impact of PYRUKYND® for more patients with PK deficiency around the
globe,” said Brian Goff, chief executive officer at Agios. “We are
dedicated to continued innovation on behalf of people with rare and
genetically defined diseases, and are working to further expand the
impact of PYRUKYND® through our ongoing investigational pivotal
programs in pediatric PK deficiency, thalassemia and sickle cell
disease.”
Agios is providing access to PYRUKYND® for the treatment of PK
deficiency in adults receiving care in the EU through a global
managed access program. More details about this program can be
found on Agios.com.
PYRUKYND® was previously granted orphan drug designation by the
EMA, which is maintained at the time of EU marketing authorization.
Agios has also applied for a marketing authorization for PYRUKYND®
as a treatment for PK deficiency in adult patients in Great Britain
under the European Commission Decision Reliance Procedure (ECDRP)
with the Medicines and Healthcare Products Regulatory Agency
(MHRA).
PYRUKYND® was approved by the U.S. Food and Drug Administration
(FDA) in February 2022 for the treatment of hemolytic anemia in
adults with PK deficiency.
PYRUKYND® Safety and
Efficacy DataThe EU marketing authorization was based on
results from two pivotal studies, ACTIVATE and ACTIVATE-T,
conducted in not regularly transfused and regularly transfused
adults with PK deficiency, respectively.
- The Phase 3 ACTIVATE trial of mitapivat achieved its primary
endpoint. PYRUKYND® demonstrated a statistically significant
increase in hemoglobin in patients with PK deficiency who are not
regularly transfused.
- 40 percent (n=16) of patients randomized to PYRUKYND® achieved
a hemoglobin response, compared to 0 patients randomized to placebo
(2-sided p<0.0001).
- Statistically significant improvements compared to placebo were
also demonstrated for all pre-specified secondary endpoints,
including markers of hemolysis and ineffective erythropoiesis.
- The Phase 3 ACTIVATE-T trial of mitapivat achieved its primary
endpoint. Mitapivat demonstrated a statistically significant and
clinically meaningful reduction in transfusion burden for patients
who are regularly transfused.
- 37 percent (n=10) of patients achieved a transfusion reduction
response, defined as a ≥33% reduction in transfusion burden in the
24-week fixed dose period compared with individual historical
transfusion burden standardized to 24 weeks.
- 22 percent (n=6) of patients were transfusion-free during the
fixed-dose period.
- The most common adverse reaction across both studies was
insomnia (19.4%), and the most common laboratory abnormalities
observed were oestrone decreased (males) (43.5%) and oestradiol
decreased (males) (8.7%).
A full analysis of these data was presented at
the 2021 European Hematology Association (EHA) Virtual
Congress. Results from ACTIVATE were published in the New England
Journal of Medicine, and results from ACTIVATE-T were published in
The Lancet Haematology. An ongoing extension study for adults with
PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is
designed to evaluate the long-term safety, tolerability and
efficacy of treatment with mitapivat; initial results from the
extension study were presented at the 2021 American Society of
Hematology (ASH) Annual Meeting and Exposition.
The company is enrolling pediatric PK deficiency patients in two
pivotal studies – ACTIVATE-kids and ACTIVATE-kidsT – in patients
who are not regularly transfused and who are regularly transfused,
respectively. Agios also continues to advance its Phase 3 ENERGIZE
and ENERGIZE-T studies in non-transfusion-dependent and
transfusion-dependent adults with thalassemia, respectively, as
well as its Phase 2/3 RISE UP study in sickle cell disease.
About PK Deficiency Pyruvate kinase (PK)
deficiency is a rare, inherited disease that presents as chronic
hemolytic anemia, which is the accelerated destruction of red blood
cells. The inherited mutation in the PKLR gene can cause a deficit
in energy within the red blood cell, as evidenced by lower PK
enzyme activity, a decline in adenosine triphosphate (ATP) levels
and a build-up of upstream metabolites, including 2,3-DPG
(2,3-diphosphoglycerate).
PK deficiency is associated with serious complications,
including gallstones, pulmonary hypertension, extramedullary
hematopoiesis, osteoporosis and iron overload and its sequelae,
which can occur regardless of the degree of anemia or transfusion
burden. PK deficiency can also cause quality of life problems,
including challenges with work and school activities, social life
and emotional health. Current management strategies for PK
deficiency, including red blood cell transfusions and splenectomy,
are associated with both short- and long-term risks. For more
information, please visit www.knowpkdeficiency.com.
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 a biopharmaceutical company
that is fueled by connections. The Agios team cultivates strong
bonds with patient communities, healthcare professionals, partners
and colleagues to discover, develop and deliver therapies for rare
and genetically defined 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
leadership in the field of cellular metabolism, 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 has multiple investigational therapies
in preclinical development and an industry-leading research team
with unmatched expertise in cellular metabolism and genetics. 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 Agios' products, including
PYRUKYND® (mitapivat), and 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 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, 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; 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; 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. 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 press release 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, 857-209-3286Senior
Director, Corporate
CommunicationsJessica.Rennekamp@agios.com
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