Agios Recognizes 14th Annual Rare Disease Day and Raises Awareness of Unmet Needs in Rare Hemolytic Anemias
February 26 2021 - 7:00AM
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field
of cellular metabolism to treat cancer and genetically defined
diseases, today announced its support for the 14th annual Rare
Disease Day, a global awareness campaign for the more than 300
million people living with a rare disease worldwide.
“Patients are central to everything we do at Agios, and we are
proud to join with patients, advocates, caregivers, clinicians and
industry colleagues around the world to support Rare Disease Day,”
said Jackie Fouse, Ph.D., chief executive officer at Agios. “We
strive to work closely with patient communities, understanding
their perspectives and cultivating true partnerships. In honor of
Rare Disease Day, we are collaborating with the PK deficiency,
thalassemia and sickle cell disease communities to advance
important initiatives designed to raise awareness and address needs
faced by people living with these hemolytic anemias.”
Throughout the month of February, the Agios team raised funds
for the Sickle Cell Community Consortium (SCCC) COVID-19 relief
fund. Following a series of sickle cell disease community meetings
and surveys organized by the SCCC, patients and caregivers
identified areas where they most need support as a result of the
COVID-19 pandemic. The community identified a critical need for
better educational resources for students with sickle cell disease
who are learning in remote or hybrid environments. The funds raised
by Agios help address this gap by providing tablets, printers,
online tools, access to tutors and other resources for these
students.
“We are living through the first global pandemic in any of our
lifetimes, and every aspect of life has changed, including the way
students are expected to learn. In this new environment, many
students impacted by sickle cell disease are falling behind from
both health concerns and insufficient access to resources and
support. Without equalizing resources and support, the education
system is no longer grading children’s ability to learn, but
instead, is judging their access to resources and grading their
privilege. In the ‘land of plenty,’ this is not acceptable,”
said Lakiea Bailey, Ph.D., executive director of the SCCC. “Our
children are intelligent, hardworking, talented and capable of
great educational achievement. We are grateful to the Agios team
for their support to ensure that children and teens living with
sickle cell disease have equal access to the resources they need to
achieve their educational goals.”
In recognition of Rare Disease Day, Agios is spearheading the
following additional initiatives:
- Today at 2 p.m. ET, Agios is hosting a Facebook Live discussion
on its “Know PK Deficiency” Facebook page. The event will feature
Jenny M. Despotovic, D.O., hematology center director at Texas
Children’s Hospital and associate professor of hematology/oncology
at Baylor College of Medicine, as well as 16-year old PK deficiency
patient Jonathan Watson and his mother Alejandra, discussing the
impact of PK deficiency on daily life and how patients, caregivers
and healthcare professionals can work together in partnership to
best manage the disease.
- Agios sponsored MassBio’s 2021 Rare Disease Day virtual event,
which will focus on strategies to better address health inequity
and racial disparities among the rare disease community, including
improving representation in clinical trials. The event takes place
today at 10 a.m. ET.
- Agios organized an internal, virtual speaker series throughout
February to increase the team’s understanding of the experiences of
patients living with hemolytic anemias. Speakers included:
- Ahmar Zaidi, M.D., hematologist at Children’s Hospital of
Michigan and sickle cell disease advocate, presented on addressing
health inequities in the treatment of sickle cell disease.
- Shamonica Wiggins, sickle cell disease patient and advocate,
and her fiancé shared their personal perspectives on managing
sickle cell disease and the impact it can have on relationships.
Biree Andemariam, M.D., board member and former chief medical
officer of the Sickle Cell Disease Association of America (SCDAA)
and associate professor of medicine at the University of
Connecticut Health, joined them to provide further insights into
clinical management of the disease.
- Sujit Sheth, M.D., pediatric hematologist-oncologist at Weill
Cornell Medicine with a specialty in thalassemia, and one of his
thalassemia patients shared their perspectives on disease
management and the patient/doctor partnership.
Agios is advancing clinical programs in three serious hemolytic
anemias: PK deficiency, thalassemia and sickle cell disease. In
late 2020, Agios announced that it intends to move forward with a
singular focus on accelerating and expanding its genetically
defined disease portfolio, including these clinical programs and a
robust pipeline of therapeutic candidates.
About AgiosAgios is focused on discovering and
developing novel investigational medicines to treat malignant
hematology, solid tumors and genetically defined diseases through
scientific leadership in the field of cellular metabolism. In
addition to an active research and discovery pipeline across these
three therapeutic areas, Agios has two approved oncology precision
medicines and multiple first-in-class investigational therapies in
clinical and/or preclinical development. 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 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. 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 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; 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 maintain key collaborations; 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.
ContactHolly Manning, 617-844-6630Director,
Investor RelationsHolly.Manning@agios.com
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