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Expert Interview

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

Digging into Alnylam’s recently approved Oxlumo in primary hyperoxaluria

Ticker(s): ALNY

Who's the expert?

An urologist with experience in treating primary hyperoxaluria type 1

Interview Questions
Q1.

Please describe your practice as a clinician,how many patients with primary hyperoxaluria type 1 do you treat on a yearly basis? Could you walk us through the standard of care, and tell us a bit about the most promising upcoming treatments, or interesting clinical trials in this space?

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Q2.

How much of an unmet need is there for PH1? How much use do you see for Oxlumo going forward?

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Q3.

Judging from the Phase 3 results, how likely are you to switch patients to oxlumo?
(For the primary end point, least-squares mean reductions in POx were 33.3% (95% CI, −15.2% to 81.8%) in cohort A (n = 6) and 42.4% (95% CI, 34.2%-50.7%) in cohort B (n = 15).)

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Q4.

 In new exploratory analyses, left ventricular ejection fraction (LVEF) and global longitudinal strain (GLS) were measured to assess the impact of lumasiran on the cardiac manifestations of systemic oxalosis. Among patients with abnormal LVEFi at baseline across Cohort A (N=1) and Cohort B (N=4), the patient in Cohort A and two of four patients in Cohort B experienced a ≥ 5 percent improvement following six months of treatment with lumasiran. Among patients with abnormal GLSii at baseline across Cohort A (N=1) and Cohort B (N=3), all four patients showed a ≥2 percent improvement at Month 6. How important is this endpoint result in assessing the efficacy of the drug in treating PH1?

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