We estimate there’s around 1,500 patients who are eligible for TRIKAFTA. That’s 6 and over in Brazil, as I mentioned in my prepared remarks, a number of those patients did already have access to TRIKAFTA through named patient sales, but we now have a reimbursement agreement with the national government there, which is going to allow us to launch the medicine and make it available for all of those patients now.
Operator: The next question will come from Olivia Brayer with Cantor Fitzgerald.
Olivia Brayer: What’s your level of confidence that you’ll get priority review and acute pain? And Stuart, I know you’ve talked about the commercial build-out, but what’s your base case for when you’ll start to actually see revenue recognition from that program, and just a quick clarification on CASGEVY. Just wanted to clarify that I heard 5 patients have already finished collection versus just having initiated the cell collection process.
Reshma Kewalramani: Olivia, this is Reshma. Let me take 1 and 3, and then I’ll ask Stuart to take the question on where are we exactly with paying commercialization. On number 3, again, just to set expectations on CASGEVY. We’re thrilled with the number of ATCs, 25 since approval, which has been in just the last few months, and we commented on the cell collection, but we’re not going to comment any further on exactly where each 1 patient is in their journey. On VX-548 and acute pain, 3 things to say. Maybe the most important thing is we’ll know whether or not we’ve received priority review in about after we complete the submission and then it takes some 60 days or so for the FDA to tell us what the final review time lines will be.
However, the leading indicators of whether or not we will get priority review are all quite favorable. We have fast track status. We have breakthrough designation and our conversations with the FDA have shown me that they have high enthusiasm for a medicine that has high efficacy and does not have addictive potential. I’ll turn it over to Stuart for the question about where are we with the acute pain launch and when we’re going to be out there.
Stuart Arbuckle: Yes. So the recruitment of our teams is going very well. Obviously, we are in the middle of our rolling submission here. Obviously, once we’ve completed that, we’ll get an indication from the regulators on when we could expect our PDUFA date to be and we are going to be launch ready in terms of the question around revenue recognition. This is unlike CASGEVY, I would say, which has an extended treatment process where revenue recognition is at infusion. This is a small molecule, and therefore, we’re going to be kind of selling and distributing it in the normal way. And so there really isn’t going to be that kind of lag, I would suggest around revenue recognition that people are aware of with CASHGEVY.
Operator: Next question will come from Debjit Chattopadhyay with Guggenheim Securities.
Debjit Chattopadhyay: I got a couple. First on IgAN. When Vertex is ready to launch in again, it’s likely Otsuka will have GFR data? How are you thinking about navigating this commercially? And then on DM1, with the IND cleared and the Phase I/II underway, do you think myotonia is an approvable endpoint? Or is the agency going to ask for placing correction with strength or force measurements.
Reshma Kewalramani: Let me take both of those. On DM1 or myotonic dystrophy type 1. We actually haven’t had a chance to talk about it extensively. But this is a program that is in Phase I/II in patients. So we are going to have the opportunity in this study to not only assess safety, but to assess efficacy as well. With regard to what the agency might want to see for the endpoint for approval, the real answer is I don’t know yet because we haven’t gotten to that phase in the clinical trial. But your point around is Myotonia possible insofar as this is a disease that is a rare disease, a serious disease and one that doesn’t have any therapies that target the underlying cause of the disease or the very specifically works on the genetic defect, I think that opportunity is there.
And I’ve seen — and we’ve seen a lot of openness for accelerated endpoints in these kinds of rare serious diseases. On IgA nephropathy, so the most important thing to know about IgA nephropathy is that it’s a serious chronic disease, and this is a disease that over time leads to decline in GFR and end-stage renal disease, death or transplantation. The most important thing that I would be looking at as a nephrologist is efficacy because proteinuria is known to translate to GFR and therefore, the decline in renal function. So if we have a medicine that has high reductions in proteinuria. And as I said in my prepared remarks, everything that we’ve seen from Poly preclinically and clinically through Phase II is best-in-class across many dimensions, but certainly including efficacy.
I think that’s the drug that physicians will choose.
Susie Lisa: One last quick question, please, Chuck.
Operator: Yes, ma’am. That will come from Ms. Lissa Bayko with Evercore ISI.
Liisa Bayko: So just two from me. Just a follow-up on IgA nephropathy. Have you thought any more about how you might highlight having bliss because in addition to APRIL, I think that’s one kind of key differentiator of this program. And just wondering how you’re thinking about how you could differentiate on that point. I don’t know if there’s biopsies or some kind of different points that you could really highlight the potential benefits of list. And then just for the for CF and TRIKAFTA for the quarter, I noticed you had your price increase yet sales looked slightly down quarter-over-quarter. Can you kind of just describe in the U.S., what’s going on? Was there some higher gross to net inventory changes, whatnot, may be great for some color there.
Reshma Kewalramani: Let me take the IgAN question first, and then I’ll ask Charlie to comment on CF. On IgAN, You are correct in pointing out that it’s a dual inhibitor. It’s an inhibitor of BAFF as well as APRIL. And this is one of the most attractive features of povetacicept is this dual inhibition. Yes, preclinically, we can certainly share and we have information, and you’ll certainly see all of this with the fullness of time. The inhibition of BAFF and the measurement of that and how we can show that preclinically, we can also do that with APRIL. However, I think the data that’s more interesting is the clinical data, which is already available, and that is with this dual APRIL, BAFF inhibitor on proteinuria, but I’d encourage you to look at the poster from the WCN meeting that Alpine showed, it has proteinuria results.
It has hematuria results. It has GFR results, and it has a composite of remission. And I find those data very, very interesting, particularly the hematuria results clinically because, as you know, hematuria is a hallmark of this disease along with proteinuria. Let me turn it over to Charlie on the question about CF and U.S.
Charles Wagner: Yes, Lisa, on the quarter, I wouldn’t read too much into sequential quarter fluctuations. We saw strong volume growth in the U.S. year-over-year. As we normally do, we see some seasonal gross to net in the first quarter and the benefit of the price increase really isn’t fully reflected in the quarter that comes throughout the balance of the year. So all of those factors affect the comparison. But overall, very, very strong year-over-year growth in the U.S. and outside the U.S. Thanks, Chuck.
Operator: This concludes our question-and-answer session as well as our conference call for today. Thank you for attending today’s presentation. A replay of today’s event will be available shortly after the call concludes by dialing 1-877-344-7529 or 1-412-317-0088 using replay access code 101-869-68, again, that is 101 869-68. Thank you for your time today. You may now disconnect.
