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Expert Interview

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

Exploring the Breakthroughs of Mitapivat in Non-Transfusion-Dependent Alpha- or Beta-Thalassemia: Insights from the Phase 3 ENERGIZE Study

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 Phase 3 ENERGIZE study demonstrated a significant increase in hemoglobin response with Mitapivat. Could you explain the clinical relevance of this finding for patients with non-transfusion-dependent alpha- or beta-thalassemia?

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Q2.

Mitapivat showed a notable improvement in FACIT-Fatigue scores. How does this translate into real-world benefits for patients, considering the chronic nature of thalassemia?

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Q3.

How does Mitapivat's performance in increasing hemoglobin levels and reducing fatigue compare to current standard treatments for non-transfusion-dependent thalassemia?

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Q4.

The ENERGIZE study indicated positive results across all subgroups. What does this suggest about the potential of Mitapivat to treat a diverse range of thalassemia patients?

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Q5.

Given the similar incidence of adverse events between Mitapivat and placebo groups, what are the implications for the long-term safety and tolerability of Mitapivat in clinical practice?

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Q6.

Considering the promising results, where do you see Mitapivat fitting into the current treatment paradigm for non-transfusion-dependent thalassemia?

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Q7.

With the ongoing ENERGIZE-T study for transfusion-dependent thalassemia, how might the results of the ENERGIZE study influence expectations and strategies for this patient subset?

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Q8.

Following the anticipated regulatory approval of Mitapivat for thalassemia by the end of 2024, what future directions do you foresee for research and clinical application in rare hematologic diseases?

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