Agios Recognizes 14th Annual Rare Disease Day and Raises Awareness of Unmet Needs in Rare Hemolytic Anemias
– Company to Host Facebook Live Discussion with Pyruvate Kinase (PK) Deficiency Patient, Caregiver and Key Opinion Leader Today at
“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
Throughout the month of February, the Agios team raised funds for the
“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
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 Watsonand 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 Michiganand 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.
Agios 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 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 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
Director, Investor Relations
Source: Agios Pharmaceuticals, Inc.