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Expert Interview

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

Discussing Agios’ Mitapivat (pyruvate kinase R activator) in Adult PK Deficiency following June 2022 Phase 3 ACTIVATE analysis

Ticker(s): AGIO

Who's the expert?

To understand the needs of PK Deficiency patients, the treatment protocols, and to assess Mitapivat’s potential.

Interview Questions

Please tell us about your clinical experience. How many patients with argininemia do you seeon a yearly basis? What is the standard of care and what treatments are available? Can you tell us more about the spectrum of disease severity for PKD patients?

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How often do patients complain of symptoms, such as tiredness, fatigue, increased need for sleep, weakness, lightheadedness, dizziness, irritability, headaches, pale skin color, difficulty breathing (dyspnea), shortness of breath, and cardiac symptoms, and which ones are the most worrisome?

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How much of a need is there for novel treatments in this space ? Where do you see Mitapivat fitting, compared to standard of care? What other upcoming therapies are you excited about?

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How often do affected individuals also develop an enlarged spleen (splenomegaly)?

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Could you please discuss the Phase 3 data reported at EHA in June 2021, where:-40 percent (n=16) of patients randomized to mitapivat achieved a hemoglobin response, compared to 0 patients randomized to placebo (2-sided p<0.0001).-37 percent (n=10) of patients achieved a transfusion reduction response, defined as a ≥33% reduction in transfusion burden in the 24-week fixed dose period compared with individual historical transfusion burden standardized to 24 weeks (1-sided p = 0.0002); 9 responders achieved a ≥50% reduction.-22 percent (n=6) of patients were transfusion-free during the fixed-dose period.-11 percent (n=3) of patients achieved hemoglobin concentrations in the normal range at least once, eight weeks or more after a transfusion, during the fixed dose period.

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The most frequently reported adverse events in patients receiving mitapivat included alanine aminotransferase increase (n=10; 37%), headache (n=10; 37%), aspartate aminotransferase increase (n=5; 18.5%), fatigue (n=5; 18.5%) and nausea (n=5; 18.5%).

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Based on the available knowledge on Mitapivat, what’s your conclusion about its safety and efficacy profile? How likely would you be to prescribe it?

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