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Expert Interview

Slingshot members are talking to an expert! The topic is:

Exploring Agios Pharma's Phase 3 ENERGIZE Study of Mitapivat for Non-Transfusion-Dependent Thalassemia

Ticker(s): AGIO

Who's the expert?

Institution: Howard University

  • Professor of Medicine and Microbiology/Immunology & Director or the Center for Sickle Cell Disease at Howard University (previously held positions at the NIH and Johns Hopkins).
  • Manages 355 patients with Sickle Cell Disease, 2 active Beta-Thalassemia patients and 1 HbH disease patient.
  • Research interests include the use of genetic tools to understand and treat sickle cell disease and complications of transfusion therapy.

Interview Questions
Q1.

The ENERGIZE study is the first to demonstrate the efficacy of an oral treatment for non-transfusion-dependent thalassemia. Can you elaborate on the significance of this milestone for patients with alpha- and beta-thalassemia?

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Q2.

Mitapivat achieved a statistically significant increase in hemoglobin response rate compared to placebo. How do these results compare to existing treatments, and what advantages does an oral therapy offer to patients?

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Q3.

The study reported improvements in fatigue and exercise capacity among patients treated with mitapivat. How do these improvements impact the daily lives and overall well-being of patients with NTDT?

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Q4.

Hemoglobin response rates were higher in the mitapivat arm across all pre-specified subgroups. Can you discuss the importance of these findings and their implications for a diverse patient population?

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Q5.

The study observed statistically significant improvements in the FACIT-Fatigue Score and hemoglobin concentration. How do these secondary endpoints contribute to understanding the overall efficacy of mitapivat?

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Q6.

The six-minute walk test showed a significant increase in distance walked by patients treated with mitapivat. What does this improvement indicate about the functional benefits of the therapy?

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Q7.

Mitapivat was well-tolerated with no serious treatment-related adverse events reported. How does this safety profile influence the potential for mitapivat to become a widely used treatment for NTDT?

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Q8.

With plans to file for regulatory approval by the end of 2024, what are the next steps for mitapivat, and how might this therapy change the treatment landscape for thalassemia patients worldwide?

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