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Expert Interview

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

Alnylam’s AMVUTTRA (Vutrisiran) in ATTR Amyloidosis: Cardiomyopathy & Polyneuropathy Market Outlook

Ticker(s): ALNY

Who's the expert?

A geneticist with experience in helping people with hATTR Amyloidosis.

Interview Questions
Q1.

  • Describe your clinical experience with ATTR amyloidosis. How many patients with hATTR-PN and ATTR-CM do you treat annually?
  • What are the most common challenges in diagnosing and managing ATTR amyloidosis in real-world practice?
  • How do the disease burdens differ between hATTR and wild-type ATTR-CM patients?
  • What are the current standard treatments for ATTR-CM and hATTR-PN, and how effective are they?
  • Added By: slingshot_insights
    Q2.

  • What percentage of your patients see meaningful improvement or stabilization with standard of care?
  • What limitations do you see with therapies like tafamidis, patisiran, and others currently on the market?
  • Which treatments perform best across measures like quality of life, neuropathy impairment, NT-proBNP, 6MWT, and troponin I?
  • Have you had any long-term responders (>10 years)? If so, what factors contributed to those outcomes?
  • What is your impression of the HELIOS-B Phase 3 data, particularly on mortality and cardiovascular event reductions?
  • Added By: slingshot_insights
    Q3.

  • How clinically meaningful are the biomarker improvements seen with vutrisiran (NT-proBNP, troponin I)?
  • How does vutrisiran compare with tafamidis or patisiran in terms of clinical efficacy, dosing convenience, and tolerability?
  • How impactful is the quarterly subcutaneous dosing in your decision-making and patient compliance?
  • What is your view on vutrisiran being the only FDA-approved therapy for both cardiomyopathy and polyneuropathy in ATTR?
  • Would you consider AMVUTTRA as a first-line treatment in either hATTR-PN or ATTR-CM?
  • Do you foresee any access or reimbursement issues with AMVUTTRA in the cardiomyopathy setting?
  • Added By: slingshot_insights
    Q4.

    5% of the patients with primary amyloidosis survive beyond 10 years. Have you had such success stories in your patients, and what would you attribute that to? What percent of patients can go into remission with drug therapy?

    Added By: slingshot_insights
    Q5.

    Can you talk to us more about the potential of RNAi therapeutics like Vutrisiran, where do you see them standing in the near future? What is the likelihood of reducing deposition and cleaning of TTR amyloid deposits in tissues?

    Added By: slingshot_insights
    Q6.

    Alnylam boasts about increased potency and high metabolic stability, that would allow for infrequent subcutaneous injections. How much would that weigh in the balance when deciding on a treatment course for your patients?

    Added By: slingshot_insights
    Q7.

    Could you please discuss the HELIOS-A 18-Month Study Results, posted on Jan 21, treported cardiac improvement when taking vutrisiran?

    Added By: slingshot_insights
    Q8.

    • How important is the promise that most patients will pay $0 out-of-pocket, with support from Alnylam Assist?
    • Which patient types would you prioritize for treatment with AMVUTTRA?
    • What gaps or unmet needs still exist in ATTR care, even with vutrisiran now approved for both indications?

    Added By: slingshot_insights

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