Company: AGIO
Filing Date: 2025-04-25
Form Type: DEF 14A
Source: 0001193125-25-096719
Chunk: 32

Company: AGIOS PHARMACEUTICALS, INC.
Filing Date: 2025-04-25
Form: DEF 14A
Chunk 32
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 (mitapivat) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia with the U.S., European Union, Kingdom of Saudi Arabia and United Arab Emirates health authorities. Beyond our clinical programs, we have taken a role in helping strengthen the thalassemia community by fostering meaningful connections among physicians, patients, caregivers and industry stakeholders. One key initiative is the formation of the Thalassemia Advocacy Advisory Council, or AAC, which was established to provide strategic insights that ensure Agios’ thalassemia efforts deliver real value to those impacted by the disease. AAC members also spearhead projects that empower both local and global thalassemia communities through education, advocacy and collaboration. In 2024, the AAC launched the Global Thalassemia Health Literacy Survey, which is an initiative aimed at identifying gaps in understanding and uncovering misinformation related to disease management. Insights from this survey are now informing the development of peer-to-peer educational programs that address critical knowledge gaps, strengthen patient empowerment and promote informed decision-making in care.

With our SCD program, our goal is to deliver a novel, oral therapy that improves anemia and reduces vaso-occlusive crises. Our phase 3 RISE UP study is a global, double-blind, randomized, placebo-controlled trial evaluating the efficacy and safety of mitapivat in SCD patients who are 16 years of age or older. We collaborated closely with the SCD community to design and drive awareness of the RISE UP clinical program. As a result, the**

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**phase 3 RISE UP study completed enrollment of over 200 patients worldwide in just over a year, with the topline results from the study expected by the end of 2025. In addition, Agios provides funding for the American Society of Hematology’s Sickle Cell Disease Centers Workshop, a program designed to offer valuable information and support to institutions exploring the development of comprehensive SCD centers. We also sponsor a traveling art exhibit showcasing the work of a prominent sickle cell warrior and advocate.

Recognizing the shared challenges and experiences of these patient communities, Agios launched the Red Cell Revolution — a multi-stakeholder advocacy advisory council focused on hemolytic anemias, including SCD, thalassemia, and PK deficiency. The council comprises 20 individuals, including patients, caregivers, healthcare professionals, and representatives from Agios. United by