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Expert Interview

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

Deciphering the HELIOS-B Trial: Discussing Alnylam's Vutrisiran in ATTR-Cardiomyopathy

Ticker(s): ALNY

Who's the expert?

Institution: MUSC

  • Professor of medicine in cardiology & Director of the Cardiovascular Genetics Program at Medical University of South Carolina.
  • Manages around 100 patients with ATTR CM.
  • Extensive experience caring for individuals and families with inherited forms of cardiomyopathy, aortic aneurysm, amyloidosis, and cardiac involvement from other genetic conditions.

Interview Questions
Q1.

Considering the recent modifications to the HELIOS-B trial's duration and endpoints, how might these changes influence the overall interpretation and clinical significance of the trial results for vutrisiran?

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Q2.

With 40% of HELIOS-B trial participants on tafamidis at baseline, how do you anticipate vutrisiran's efficacy will be evaluated against tafamidis, and what implications could this have for its potential use in clinical practice?

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Q3.

How does the delay in the HELIOS-B data readout from early to mid-2024 affect the reliability and anticipation of the trial's outcomes?

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Q4.

Following the changes in the HELIOS-B trial, how should investors and clinicians reassess the risk profile of vutrisiran for ATTR-cardiomyopathy treatment?

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Q5.

Can you elaborate on how the altered primary endpoint and extended trial duration might affect the statistical power and event rates observed in the HELIOS-B trial?

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Q6.

How might the new focus on evaluating vutrisiran as a standalone treatment in the HELIOS-B trial influence its market position and adoption, especially against existing treatments like tafamidis?

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Q7.

Based on the current trajectory of the HELIOS-B trial and the evolving landscape of ATTR-CM treatment, what are your predictions for the future of this disease management?

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Q8.

What are the potential financial and clinical implications for Alnylam Pharmaceuticals if HELIOS-B results in a moderate success or fails to demonstrate superiority over existing treatments like tafamidis?

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