Vertex Pharmaceuticals Inc American Society of Nephrology (ASN) Kidney Week 2026
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We're going to get started. Thank you very much to everyone here in the room for finding us despite locked doors and escalators and challenging room locations. We really appreciate it. Obviously, a very, very exciting ASN meeting overall, and I think particularly for Vertex Pharmaceuticals. So very, very happy that you all can be with us tonight for our kidney program updates at the American Society of Nephrology Kidney Week 2026. I do just want to go through the agenda briefly. Reshma will kick us off with some remarks about our R&D strategy and our programs, give a quick portfolio overview. Then I have the pleasure of introducing to many of you, I think for the first time, Dr. Akinlolu Ojo from the University of Kansas, who will discuss the recent AMPLIFIED data and our AMKD program with inaxaplin.
Then we'll hand it over to Dr. Richard Lafayette, whom you all know well, and he will discuss the data released this morning in the New England Journal of Medicine about povetacicept in IgAN. Then we'll have time for your questions. We'll try and keep it really punchy. We do have a hard stop right at 7:00 P.M. Again, thank you for joining us. Please note that we will be making forward-looking statements, and we ask you to consult all of our SEC filings to discuss the risk factors. With that, Reshma. Thank you so much.
Susie, I appreciate it. It is very surprising for me to see so many of you at an ASN. It is a real pleasure to see all of you here for the Vertex event. Believe it or not, when I was an actual practicing nephrologist and would come to ASN as a doctor, there were no real analyst meetings. There were no investors here. There were hardly any people here. So the field has come a long way. Interestingly enough, while Vertex started as a CF company, we are quite the renal company now. This slide talks to you about, on the left side, our strategy, which has not changed. It's a disease-first strategy. We are not about platforms. We are not about franchises.
It simply turns out to be the case that when you think about unmet need, causal human biology, the ability to have validated targets, usually genetic, but pharmacologic is just fine, biomarkers that translate and efficient regulatory and development pathways in a way where we can do our work with a low SG&A spend. It turns out that there are few renal diseases that meet every mark. What you see there are the four renal diseases that are in mid and late-stage development for us. APOL1-mediated kidney disease, Dr. Ojo will talk about that. IgAN, Dr. Lafayette will talk about that one. Membranous is another povetacicept indication. You'll remember when we did the Alpine acquisition, we talked about povetacicept as a pipeline and a product. While we're not going to touch on it today, watch this space for ADPKD. We are excited about a molecule.
It's a small molecule that properly folds a misfolded protein. If that sounds like something you've heard from Vertex before, you have. That's how our CF medicines work. There are some reasons for us to be optimistic there. When you think about these diseases, they are actually rare diseases, many of them in the U.S. or in Europe, but they are big rare diseases. We're talking about 100,000 to 300,000. When you think about IgAN, it's well over 1 million because it's a particularly prevalent disease in Asia. You'll remember for Asia, we have partnerships with Zai Lab. They will be marketing for us in China and other regions. We have another partnership with Ono, who will be bringing this medicine to Japan and other regions. It's an interesting area of rare, but big, rare diseases. The other interesting point to note is the $13 billion.
Let's just round and think about CF as a $10 billion franchise. Many people ask me what I think could be another CF-like franchise, and there are a few, but renal is amongst them. I think when you put these diseases together, the four that I've mentioned, and remember, the ADPKD disease that I'm mentioning to you is only 10% of the overall ADPKD. ADPKD is about 350,000 people worldwide. Our first approach will be appropriate for about 35,000. Just in those four diseases, I see it being about a $10 billion franchise. This is renal in a box, and if you wanted to have a quick reference guide, this is the one to look for. There are some B-cell-driven diseases. That's where povetacicept plays for IgAN and membranous.
Not on the side because it's not in renal, but we also have a trial for povetacicept in myasthenia gravis because that is another one of these very B-cell-mediated diseases. APOL1-mediated kidney disease in the middle. The phase III program is AMPLITUDE, but you would never know that because people are so interested in AMPLIFIED, so Dr. Ojo is going to cover both for you today. The ADPKD program is in phase II proof of concept. I want to just do one slide before I turn over the stage to the stars of the show, Dr. Ojo and Dr. Lafayette. These are all of the results, the latest available from the various APRIL/BAFFs. They're not meant to be there for comparison because these were not head-to-head studies. It's meant as a compilation of information, so everything is in one place at one time.
The thing I want to focus on is povetacicept in the middle, and you'll hear a lot about it. We are excited about the fact that the PDUFA date is right around the corner, November 30th. All of the things that you would expect to be going on between the company and FDA are going on, and we are really excited about bringing povetacicept as our first renal medicine. The other thing I want to point out is sort of towards the top of the slide and towards the bottom of the slide. Pre-clinically, povetacicept was specifically engineered to have some characteristics. We wanted those characteristics so that we could get broad tissue penetration, really good binding affinity, as well as potency, and they manifest themselves in the bottom of the slide. This is the ability to have a once-monthly dose with a small volume in an auto-injector.
These are not separate, that sort of, kind of happened to come together by good luck. This is how we engineered this medicine to behave. With that, I am going to turn it over to Dr. Ojo. It is a real pleasure to introduce him. He is an MD PhD scientist. He has worked in multiple disease areas, but you will hear from him why he has a passion for AMKD. We know Dr. Ojo because he sits on the steering committee. The good news on AMPLITUDE, the phase III study, it is fully enrolled. It did take a long time, as we predicted, but it is fully enrolled. The other piece of good news is we are fully on track to be able to share the interim analysis results early next year.
Dr. Ojo. Thank you very much, Reshma.
Good evening to all of you. I am a practicing nephrologist, and I also do research, and the focus of my clinical work, as well as my research programs, has been on kidney disease in Black populations for the last three decades. What is represented to you on this slide is the summary of the result of the AMPLIFIED study. The AMPLIFIED study is a study of individuals who have AMKD, but were not studied in the AMPLITUDE trial for a number of very cogent reasons. There are three points to note on these summary results. One is that the two cohorts there, that is cohort 1, individuals who have very modest amount of excess urinary protein excretion, low level of proteinuria, as well as cohort 2, individuals who have moderate amount of proteinuria, but also have evidence of diabetic kidney disease or diabetic nephropathy.
Those two cohorts represent the largest fraction of individuals with chronic kidney disease in the U.S. and in the rest of the world. In the U.S. alone, these two cohorts represent individuals that numbers in several hundred thousand. The second thing to note is that the participants in the AMPLIFIED study are already on the most contemporary and the most comprehensive background treatment for both chronic kidney disease and diabetic nephropathy were relevant, that is in cohort 2. So these are individuals who, before entering the trial, are getting the best treatment for their conditions that we have. The third point to note on this result is the reduction in urinary protein excretion in both cohorts. In both cohorts, the reduction in urinary protein excretion is substantial and clinically meaningful.
You will note that in cohort 2, the reduction in urinary protein excretion is between 17% and 25%, and the confidence interval crosses one. That does not detract from the significance of these findings. Because if you go back and look at registration trials for RAS inhibition therapy, the RENAAL study that led to the approval of losartan, for example, at three months after randomization, the reduction in urinary protein excretion was 25%, exactly what you see in the cohort 2 here. At the end of four years of follow-up, reduction in proteinuria continues, and that translated into a 44% reduction in renal outcome and a 30% reduction in cardiovascular outcome.
The numbers that you see here, even though the study population, the study sample is small, is demonstrative of significant clinical benefit on the long run when this study is followed for a longer period of time or if a larger study is conducted in this population. The AMPLITUDE study is a phase III study of inaxaplin in individuals with AMKD. This clinical trial is monumental for a number of reasons, including the fact that it is the first phase III study of a treatment, a disease-modifying treatment in a population that had not had any disease-modifying therapy ever since we have known about the excess burden of kidney disease in people of African ancestry.
As you know, people of African ancestry, they carry a fourfold excess risk of kidney failure compared to any other racial ethnic group, and for the last 50 years, we have not found a treatment that will address their core problem. We have had a variety of ameliorative therapies that are non-specific and not targeted to the underlying mechanism of disease. That is why AMPLITUDE is different. As Reshma said, this study is now fully enrolled. It included 66 patients who were carried over from phase IIb study of inaxaplin, along with 456 study individuals who are newly enrolled into phase III for a total of 522. As Reshma pointed out, the interim analysis is forthcoming beginning of next year.
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