Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q2 2023 Earnings Call Transcript

And we’ve said all along that we think a drug like this for indications like hypertension and heart failure are best suited with a partner to reach as many patients globally around the world. And we haven’t provided any details on the timing when we’re going to partner the program, nor — of course but stay tuned to that down the road. Eugene, could you talk a little bit about the Luca’s question about our stats plan for CARDIO-TTRansform.

Eugene Schneider : Sure. So if I understood, it was related to the analysis of the primary endpoint. Is that right, Luca?

Luca Issi : That’s correct. Yes, that’s correct.

Eugene Schneider : Yes. So as we said, the methodology that we’re utilizing or planning to utilize for that analysis is a pretty tried and true using Andersen-Gill method to look at both CV mortality and CV events, recurrent events that are listed in our SAP. So again, there’s nothing — I think fancy about this method has been tried and done in multiple outcome studies. It’s a fairly classic way of what these events.

Operator: The next question is from Yale Jen with Laidlaw & Company.

Yale Jen : The first question is for eplontersen that if you get it approved later or to the end of this year in neuropathy, do you anticipate the drug will also be used for treating the mixed type patients? Then I have another follow-up.

Brett Monia : Absolutely, we do have provided they have deductible polyneuropathy, yes. This drug would be suitable for pure PN patients as well as mixed phenotype patients.

Yale Jen : Okay. And my another question is for olezarsen that in terms of the SHTG enrollment, can you provide any color in terms of the status? And do you anticipate complete enrollment later this year?

Eugene Schneider : I missed the specific…

Brett Monia : How we are on SHTG enrollment?

Eugene Schneider : Enrollment is going very, very well. And either very late this year or very early next year, it’s kind of the guidance that we’ve been giving.

Operator: The last question today comes from Joseph Stringer with Needham & Company.

Joseph Stringer : Just a quick one on olezarsen Phase 3 FCS later this year. Can you just frame expectations on what type of outcome you need to see to be successful commercially? What type of key metrics should we be looking at beyond the primary endpoint from — that would be important from a physician or a payer perspective?

Brett Monia : You want to take that, Eugene? I mean obviously, the primary endpoint is triglyceride lowering — for triglyceride lowering compared to placebo, and that’s important. And we expect what will really drive this market is substantial TG lowering in this patient population. But Eugene and then on Onaiza, maybe to talk about the commercial also what additional endpoints we’re looking to see.

Eugene Schneider : Sure. Happy to. We’re obviously going to also look at responder — a variety of responder analysis. So looking at proportions of patients that get below particular clinically meaningful thresholds, thresholds like 80 for instance, [is the classic 400, 500]. So again, achieving those reductions that are thought to be meaningful in terms of their risk for having acute pancreatitis events is something that we’re — and future prescribers will be very keen to see. We’re also looking at quality of life measures. But again, we’ll indicate whether these patients are actually feeling better, they’re able to function better and those, again, are included as well.