Vertex Pharmaceuticals Inc 12th Annual Cantor Fitzgerald Global Healthcare Conference

Great. Thanks, Carter. I appreciate your having us and a great question to start with. I think as many people know, the Vertex story from an investor standpoint sometimes is more difficult to tell because there isn't a clear therapeutic category or a platform. Instead, we adhere very strictly to our R&D strategy, which has these set criteria. A few of those criteria include things like understanding the human causal biology. Are there validated biomarkers? Is it a specialty market? Are there streamlined pathways in terms of regulatory and clinical development? We apply those criteria so strictly to any disease area we pursue internally, as well as any acquisition we consider externally.

Now, if something fits in what we call these sandbox disease areas, and it can help accelerate one of our existing programs, we go after it like Entrada and DM1 is a good recent example. Or if we don't have a presence in a disease area that we've identified and we see an asset that can launch our presence into that space with a differentiated product like Alpine or like Crinetics, then we move forward aggressively on it. I think that we executed this deal from a position of strength, right? We're expanding our leadership in CF. You're seeing good commercial uptake on CASGEVY and JOURNAVX. We're on target for $500 million-plus in revenue from both of those products this year. The renal pipeline, as you mentioned, is really exciting.

But this rare specialty endocrinology space, where what you measure in phase II is what you measure in phase III, right? It feels very de-risked to us, and we love the call point and think a lot of our learnings from our CF commercial expertise can be deployed for Crinetics and that, like we did with Alpine, we can hopefully accelerate some of the launch timings or expand the global footprint, et cetera, and bring our expertise. So when we see something that we identify, we move aggressively to get it before. We don't feel like with Alpine, if you recall, there were a lot of assets. We were confident in our due diligence and moved forward the one that we thought was potentially best in class. With Crinetics, it's the same sort of story.

Yeah. So we are excited to be now with the closing Crinetics last week, right? We talk about these disease area pillars, so commercialized in obviously CF as well as hematology with CASGEVY and acute pain. Now with PALSONIFY's launch underway in acromegaly in rare endocrine diseases. Then we look very much forward to a PDUFA date of November 30th for povetacicept in IgAN. So that'll be the first launch in our fifth pillar, if you will, in renal. With three programs behind that, we're excited for that. So the Povi PDUFA date is a key milestone, November 30th. Before that, though, I would say you're likely to get the phase II proof of concept data in essentially a patient population expansion study for inaxaplin. That's the AMPLIFIED phase II study. It's a phase II study of about 50 patients. All patients have two APOL1 alleles.

About half of them have more modest proteinuria than you see in the pivotal study, AMPLITUDE, and about half of them have a heavier proteinuria but type 2 diabetes. We see this as separate and above from the AMPLITUDE pivotal study. That catalyst technically slides into 2027, but you will get that interim analysis of the phase III study in early 2027. That is about 150,000 patients. This study, separate and above, would be an incremental 100,000 patients if you were to see positive results from AMPLIFIED. We are excited for that, and that is probably the nearest term catalyst, then the Povi IgAN that I mentioned. Also we have promised phase II proof of concept data in our myotonic dystrophy type 1 program before the end of the year, as well as a look at the first of our next gen 3.0 CF portfolio of products.

That is VX-828. I think those are the key milestones before the end of the year. Also we will complete enrollment in our two phase III studies in diabetic peripheral neuropathy in our chronic pain franchise. Those studies are both 12-week studies, and so you could look at data, say maybe by mid-2027. Happy then to go into the outlook in type 1 diabetes and some of the other areas that we have too. But those are the key near-term catalysts as well as continued commercial execution.

Yeah, I think that's a really good insight. I think sometimes investors think, sometimes we even joke CF sells itself.