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Expert Interview

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

A Second Look: Discussing the phase 2 SEQUOIA trial data on Arrowhead 's fazirsiran in treating A1AT liver disease

Ticker(s): ARWR

Who's the expert?

Institution: NYU Langone

  • Assistant Professor of Gastroenterology and Hepatology at NYU Langone Health.
  • Treats 750 patients with liver disease and 11 patients with A1ATD.
  • Research interests include radiofrequency Ablation in a variety of gastrointestinal conditions.

Interview Questions
Q1.

Can you give an overview of your practice?

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Q2.

Can you give me an idea how many A1AD liver disease patients end up needing a liver transplant, and how long that usually takes?

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Q3.

On a high level, can you discuss the approaches from Arrowhead and Vertex, siRNAs vs correctors?

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Q4.

How do you think patients on existing augmentation therapy would affect efficacy, if at all?

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Q5.

Do you expect any variance in efficacy depending on fibrosis stage?

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Q6.

Given that there are no approved therapies for the A1AD liver disease, what do you envision would be an appropriate phase 3 design, including primary endpoint?

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Q7.

Let’s assume fazirsiran is the 1st drug approved in AATD, either lung or liver, how do you imagine it being used vs the current SoC?

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Q8.

How would you rate your enthusiasm about fazirsiran on a scale of 1-10, and you can you elaborate on why?

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Q9.

Are there any other approaches to A1AD, whether liver or lung disease, that you find interesting and are following?

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Q10.

Are there any opinions on gene therapy for A1ATD?

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