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Expert Interview

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

Discussing Alnylam’s Lumasiran in primary hyperoxaluria type 1

Ticker(s): ALNY

Who's the expert?

A nephrologist with experience in treating PH1

Interview Questions
Q1.

Please describe your clinical practice. How many patients with PH1 do you see on a yearly basis? What is the standard of care, and how well do patients respond to it?

Added By: catalin_admin
Q2.

Can you please walk us through the rationale behind targeting hydroxyacid oxidase 1 HAO1 as a pathway to treat PH1?

Added By: catalin_admin
Q3.

Could you please discuss the results of Lumasiran in reducing plasma oxalate POx? 33% and 42% mean reductions in POx from baseline to Month 6. Treatment with lumasiran led to 35 micromol/L and 48 micromol/L mean absolute reductions in POx from baseline to Month 6.

Added By: catalin_admin
Q4.

Could you comment on the separate analysis presented at ERA-EDTA?

Added By: catalin_admin
Q5.

Can you tell us more about the RNAi asset nedosiran, considering its mixed bag of data in PH3, but approval for PH1 since 2019? How does Lumasiran compare to Dicerna's (DRNA) Nedosiran? How well do patients respond to Nedosiran, is there an unmet need still for PH1 patients, and do you think Lumasiran can fill the gap?

Added By: catalin_admin
Q6.

How likely are you to switch patients to Lumasiran?

Added By: catalin_admin

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