Vertex Pharmaceuticals Inc Wells Fargo 21st Annual Healthcare Conference

Thanks for having us, Mohit.

Sure. I think it is an exciting time. We have sort of a catalyst-rich period coming up.

I would say most of the conversations relate around that. I will say recent news last month, we had been getting a lot of CF questions heading into competitor data that I think ended up being not as much of a concern as many had thought.

Right. The CF questions have gone back more towards commercial aspects, the ALYFTREK switch, which continues to go very well, and then our next gen 3.0 family of therapies that are in the pipeline.

The majority of questions I think focus on upcoming catalysts, and that is primarily within our new disease area pillar of renal therapies, namely disease-modifying therapies that are addressing unmet need in the renal area where historically they have had basically repurposed cardiovascular medicine. Really, we are calling it a renal renaissance and a very exciting time. The most near-term catalyst would be on our inaxaplin therapy.

which is for APOL1-mediated kidney disease, and that's where we've said that you should expect in the next couple of months our phase II proof of concept study for a patient population expansion study called AMPLITUDE Right which is looking at patients with two APOL1 alleles and then two cohorts, one that has more modest level of proteinuria, and the other cohort has the two APOL1 alleles and type 2 diabetes.

You'll get that proof of concept data sometime in the coming months. The next catalyst that's super exciting will be our November 30th PDUFA date for povetacicept in IgAN. This will be our first commercial launch in the renal area. We're very excited for that. We are launch-ready. Our sales force is hired. We have very high degrees of nephrology experience in that sales force, given the depth of the pipeline, the clinical differentiation of pove, the safety profile, and the patient administration benefits. We're very excited for that.

Then the next, you'll get a couple other data sets before the end of the year, likely one in myotonic dystrophy type 1, which is certainly timely right now. We can go into more detail there. In our CF therapies, VX-522 is the first of the next gen 3.0. You could see data before the end of the year. Then we'll complete enrollment in our two phase III diabetic peripheral neuropathy studies, and that's in the chronic pain space. We'll complete enrollment before the end of the year. Those are 12-week studies that would put us on pace for data sometime in the first half of 2027, likely.

Then very importantly is you'll get the phase III interim analysis in the first quarter of 2027 on inaxaplin in the sort of primary or pivotal study there of primary AMKD, which is patients with two APOL1 alleles and heavy proteinuria burden. So we're very excited for that. That's been a long time coming and likely would be our second launch commercially in the renal space.

Awesome. I would just add too, Crinetics just closed last week.