Intellia Therapeutics, Inc. (NTLA) September 2026 Conference Transcript

to self-exclude or based on their own priorities to participate as a study. And my expectation is it'll look like that later on as well.

And just remind us, what percentage of people would have that HLA subtype that would be impacted?

It really depends on ethnicity. If you look back in the trial population, it's about 12% of patients that have the allele. that's reflective of a European sort of Caucasian makeup, which is the predominant patient type in the patient population. But as you move increasingly into an Asian ethnicity or into African-Americans, the prevalence really falls to as low as 1% to 2% in African-Americans. So it really depends on the patient type.

Okay, understood. I guess as you think about the competitive landscape, again, a similar question to the HAE discussion we're having is just where do you envision this fitting in the paradigm? Again, it is, you know, I'd say it's not as crowded as HAE. Again, it's not the prophylaxis versus on-demand dynamic different here. You have cardio versus PN. But, again, where do you envision this fitting in that new paradigm?

Well, we know that ATTR amyloidosis, whether it's polyneuropathy or cardiomyopathy, is a progressive disease. And we know that, at least of the currently approved agents, the patient populations progress on those agents. I don't think experts in the field, certainly the ones that we talk to who know all the drugs and participate in all the trials and write all the papers, basically, that any of them believes that we're at the end point here with the pharmacopoeia as it exists in approved therapies today. So what we learned from our study and the alnylam study, which I think has many similarities, is really, I think, going to set the stage for how these drugs are used. Cardiologists don't want to have a succession of drugs where you're chasing failures because as you fail, it becomes increasingly difficult to get the benefit. The cardiologists that we work with want to stop the disease in its tracks, and it's best to do that with the most effective therapy as early as possible. And, again, I think that that's the patient population that we're trying to, you know, make sure we have plenty of examples of to show that, in fact, that it will work that way. And as we think about the pricing dynamic, is it the same framework that we should think about here, multiples of existing therapies given your same idea, you're one and done here or is there a different framework here for some It's certainly a consideration and I think the current silence it's a very extremely highly priced market right now at least from a cardiology point of view we think that that this patient population, which is elderly and very much a Medicare patient population in the United States, and outside the United States, I mean, these are mainly central payer type populations, that that plays to our strong suit from a pharmacoeconomic point of view. So presuming an efficacy that is not surpassed by other agents, and I think that that's the likely outcome here, we think we're going to have a very competitive economic offering that will be very attractive to, you know, that group of payers that serves those patients.

And, Ed, what does the commercial footprint look like here relative to HAE?

We haven't scoped it out to the same extent as you might imagine that we've done with HAE. It's, you know, there's more cardiologists for sure than, you know, physicians who treat HAE. So it's a bigger footprint, and here's where we have other options. We have our colleagues at Regeneron who are part of the mix here. So as we get a little further into that, I'm sure we'll be talking more about it, but it will be a bigger undertaking for sure.

And I'll just add in between HAE and CM is this PN indication that gets lost in the mix. It's a little bit smaller, but it should unfold that PN will be first. we will have the benefit of learning things from HAE in a population that's older but not quite as significantly older as the CM population. And so I think there's some synergies there on the near term. But, yeah, ultimately there's a bigger build-out, and, again, we have that collaboration with Regeneron.

And just remind us how the split works there in terms of economics, and are there any, like, opt-ins where that decision was already made?

Yes, from the onset, it's been 75%, 25% sort of in our favor. In terms of just decision-making, it's Intelia's ultimate decision, and we support 75% of the economics. That will hold true. Commercialization will be essentially a 75%, 25% profit split. There is an opt-in decision. It's sort of non-consequential from an economic perspective. We don't get an opt-in payment, for example, but they do have a right to co-promote in a minority fashion in the U.S. for the TTR asset. And the way to simply think about that is if, you know, for every 100 reps, they'll have some proportion. It's actually for general employees instead of Intelli employees. Net-net, it's still the same cost, still the same split, but that comes roughly about two years before anticipated approval. So that is on the horizon, but, again, not all that consequential from the overall collaboration perspective.

So they have to make that two years before approval. Okay. I guess just you mentioned this, Ed, before, just as you think about the HAE launch and what that means from, you know, a capital perspective for the company and having maybe more flexibility to invest in the broader pipeline. And so maybe just talk through, like, what that would look like and do you guys already have, like, opportunities that you're thinking about, like, rolling out or is that going to be staged? Like, how do you think about capital deployment in the event that you do have a successful HAE launch?

Yeah, so we have a pretty clear five-year plan. I mean, the good thing about running these studies in the case of CM, which really drives a lot of our operating expenses, is you've got good visibility over the next few years. So we've been planning on that for quite some time. If we take a step back, end of the second quarter, we had about $630 million in cash, and we've been very conservative with our guidance where we just exclude Lombosi revenue altogether. We still have cash to run the company into 2028. And that's intentional, right? We want to make sure we not just get approval, but the key question investors have is, are you going to sell it? Can a one-time therapy be successful? We think the answer is yes. We need time for that to play out for the reasons we talked about. Just last week as well, we announced now a debt facility with Orbamed, which gives us $300 million in committed capital. We took down the initial $75 million of that, and we've given disclosures. But in a nutshell, we get another $75 million available to us at Bombo Z approval. There's three distinct $40 million payments that we can get linked to sales performance of Bombo Z, but that gives us even more optionality and flexibility. We've also preserved the opportunity to do converts or synthetic royalties, so we want to make sure we have a lot of options just given everything that's going on at the company. But the thing that's most fun for us to think about, which really transforms if we need capital, how much and when, will be the performance of Lombo Z. So we've given ourselves enough runway to see that through. But in many ways, there's been a lot of stuff that's happened in the TTR landscape, as we just talked about in particular. The goal was always do really well with Lombo Z, but that gives us capital to then redeploy against much larger opportunities, particularly in CM. And so despite some of the noise and the challenges and opportunities that come with the recent data sets, there's now a really big and growing market and probably one less competitor. So just think about the possibilities there. That's what we're really playing for. The other part we'll eventually talk about, not today, will be the pipeline. We do have active research going on. It is a competitive world out there. Given everything else the company has, we're not going to get credit, so we don't talk about that. But we do have very active, viable programs that we will in due course. So we thought about all that on a five-plus year view. We feel really good about where we are, but we really want to execute and deliver on the Lombosy opportunity for good reasons.

Well, thank you so much both for being here. Really appreciate it, and best of luck. Appreciate it.