Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q4 2022 Earnings Call Transcript

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Yale Jen : Okay. Great. And congrats for all the progresses.

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

Joseph Stringer: Just a quick one from us on the broad pipeline prioritization here, just given the company’s pipeline breadth, do you consider the current pipeline size and R&D spend is sort of rightsized at this time? And is this an evolving outlook as you go forward?

Brett Monia: Thanks, Joey. Since I moved into the CEO role, one of my key objectives as well as my team here has been to really focus organization on what is going to bring the greatest value to the company. That has been really important is if you think about it, prior to our evolution to full integration, we partnered all of our programs. So prioritization and focus was less important. It’s vastly more important as we make investments to bring products through Phase III into the market ourselves. So although I cannot say whether or not the size of our pipeline today will be the same size 5 years from now. What I can tell you is that we will focus the pipeline as we have done over the last couple of years when we announced and we said we’re moving away from indications like oncology for example, which we moved away from because we don’t think that that’s the best use of our resources.

And we’ll continue to do that. I don’t know what that magical number will be that size, but we are focusing and prioritizing. And I wouldn’t be surprised if the pipeline was proactively reduced in size based on a number of drugs and maybe even for those drugs, we will expand the indications as an example, to really maximize the value of each asset. Do you want to add anything to that, Onaiza.

Onaiza Cadoret: Just again, I think I spoke about this briefly in the last question. I do — I think to emphasize some of Brent’s vision and how we’re putting it into a process to give you a little bit more color is that we took on a more rigorous approach to prioritization last year, and it’s continuing this year as well. And what I really like about the processes that included really a large portion of the Ionis team from research development and commercialization, manufacturing talks all the way through to really think through not just the quantitative aspects of each of our programs, but also strategically and qualitatively, what’s most important to bring forward and how do we bring it forward. So we have some really nice ways to think about what we want to keep all the way to market.

We also have some really good distinctions on where we think some programs will again belong with partners and realize their full potential. So stay tuned. We continue to kind of bring in the focus that Brett talked about, and you’ll see more of it in our pipeline.

Brett Monia: And Joe, you had another part to that question, which I forget.

Joseph Stringer: No, that was it.

Operator: The next question comes from Yaron Werber with Cowen.

Yaron Werber: Great. Brett, I just got 2. The first one is just maybe just to follow up on the Factor B question. The I believe you guys ran a study that was started in 2018, 120 patients, obviously smaller than the current study, which is 330. That started and then I think that study was terminated. Just give us a sense kind of what happened there. I think the primary endpoint was also different. That was a factor B levels, whereas in the current study is obviously looking at change in GA? And then second, just your thoughts in the nuts preliminary on the IRA. Eplontersen potentially will have 2 indications in the future, hopefully, what does that mean from a negotiation standpoint or can you ultimately get a broad TTR label?

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