Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q3 2023 Earnings Call Transcript

So it just adds to our confidence in our leading neurology platform. As far as IgAN FB-LRx to be presented at Kidney Week coming up. This will be an additional data cut that was presented at Kidney Week last year in patients with IgAN nephropathy just showing more data, more patients, longer treatment, showing reductions in proteinuria in that study. As far as the competitive landscape, it’s crowded. There’s no question and we’re glad Roche has taken a lead on this because they have a tremendous global might, global strength, global presence get the drug to successful – to bring it to as many patients as possible. But I really think that that’s to be determined once we see the Phase 3 data and to see how this positions itself. But really, we couldn’t be more pleased to have.

When you’re a competitive market like this with a high unmet need, it’s great to have the global strength of a partner like Roche.

Operator: The next question will come from Yanan Zhu with Wells Fargo. Please go ahead.

Yanan Zhu: Great. Thanks for taking the questions. Three questions if I may. On Eplontersen, what launch metrics would you provide so that we can have an understanding of how the drug is performing in the market in the early launch quarters? On the tau program, can you talk about how you see the visibility of the Inotersen [ph] route of the administration in this very large indication. On the –lastly on the Angelman syndrome program, following the recent updated data from Ultragenyx, any insights you could share regarding how your program could be differentiated from that program as we look forward to data in mid-2024? Thank you.

Brett Monia: Thanks Yanan. And maybe we’ll start with launch metrics?

Onaiza Cadoret: Sure. So Yanan, I think the way to think about this market, as I said, it’s a growth market and we have a lot of patients to get diagnosed and get treated on eplontersen. And I think the phenomenon that you might be seeing now with switches is very temporary. And I think we will have — they will have worked through that. We’re squarely focused on growth and growth mindset on newly diagnosed patients. As such our launch success will be measured on getting eplontersen as a preferred choice for newly diagnosed patients new to silencers.

Yanan Zhu: Got it. Thanks.

Brett Monia: So next year, we’ll be providing some metrics and we’ll see how that — so stay tuned for that Yanan. Regarding the Tau program, so Alzheimer’s disease, despite the really remarkable progress, that’s been made recently on delivering medicines to the market to patients for AD, this is still a very severe neurodegenerative disease with a very high unmet medical need. And like I touched on earlier, we think Tau is the best target for treating this disease broadly. And with that comes the need for treatments and we don’t — we believe that intrathecal delivery will be well accepted if the drug is as efficacious as we expect it to be. With that said, the Tau program is an example of the great progress we’re making in further optimizing and advancing our neurological disease drug discovery capabilities with homedicinal chemistry and just experience.

In the Tau program, we actually have a treatment arm that we expect will be efficacious with twice a year dosing, so we’ve moved from monthly to every three months for many programs and now we’ve been moving some programs to twice per year dosing intrathecal. That is part of the Tau program that’s in Phase 2 development today. In addition as we highlighted at Innovation Day, a few weeks ago, we’re making great progress from our research organization in overcoming the blood-brain barrier as an obstacle for delivery of our treatments, our drugs for using subcutaneous or intravenous administration. And obviously, programs like Tau are on our radar as our other programs. So we also think that that could be in the future for a large population chronic disease indication like Alzheimer’s disease.

So, no promises there yet but we’re very pleased with the progress we’re making there. Regarding Angelman syndrome, I prefer not to comment on other people’s programs other companies’ programs. What I will say is that we are fully enrolled in our Angelman’s program. We actually overenrolled the study a bit. We expect data midyear next year. We expect data on efficacy as well as, of course, safety and biomarker data from that trial. And that trial is designed to set up a potential Phase 3 decision based on all that. And then what I’ll also say is that — and it’s kind of related to the earlier question that I got that I tried to address, which is we have a vast amount of experience with our platform in neurological diseases thousands and thousands and thousands of patients have been treated for very extended periods of time with our chemical platform, with our know-how with everything.

And we think that that bodes very well for the Angelman’s patient community, because they’re going to be able to take — they’re going to benefit from the vast experience that we have at Ionis with delivering neuro drugs for the treatment of severe neurological diseases like Angelman syndrome. So stay tuned for all that. We’re very much looking forward to the data next year.

Yanan Zhu: Great. Very helpful. Thank you.

Operator: The next question will come from Debjit Chattopadhyay with Guggenheim. Please go ahead.

Unidentified Analyst: Hey. Good morning, team. This is Robert on for Debjit. Thanks for taking our questions. Two from us this morning. First, could you share any pricing thoughts on eplontersen. Specifically would you anticipate launch price in line with current PN treatments on the high end or CM treatments in the low end. And for the Alexander program, what pace of enrollment would you hope to see in the Phase 3? And subsequently when would you potentially anticipate a Phase 3 readout based on those time lines? Thank you.

Brett Monia : Onaiza, would you want to take the launch price PN and CM?