Vertex at Wells Fargo Conference: Strategic Growth and Challenges

Vertex Pharmaceuticals (NASDAQ:VRTX) presented a strategic overview at the Wells Fargo 20th Annual Healthcare Conference 2025 on Wednesday, 03 September 2025. The company highlighted both its commercial successes and regulatory challenges. Vertex is expanding its market presence with new product launches, while navigating complex regulatory pathways.

Key Takeaways

• Vertex is focusing on commercial diversification with new product launches like Elliptrak, KASJEVY, and Gernavix.
• The company is addressing regulatory challenges in the peripheral neuropathic pain (PNP) market, focusing on diabetic peripheral neuropathy (DPN).
• Vertex is expanding its reach in cystic fibrosis (CF) treatments and kidney disease programs, with promising data expected in the coming years.
• The company is actively working to mitigate risks from the Most Favored Nation (MFN) clause, advocating in Washington, D.C.
• Vertex remains optimistic about its growth strategy and commitment to addressing unmet medical needs.

Financial Results

• Vertex reported strong performance in the second quarter, driven by growing revenues from CF products, KASJEVY, and Gernavix.
• The company emphasized volume-driven growth for Gernavix, with 150 million lives covered and 85 million without prior authorization as of the Q2 call.
• Despite stock fluctuations, Vertex highlighted its solid financial execution.

Operational Updates

• The Gernavix launch is progressing well, with faster-than-expected payer uptake and contracts with two of the three largest pharmacy benefit managers (PBMs).
• Vertex is expanding its sales force for Gernavix as coverage increases.
• The CF business is expected to grow through new approvals and geographic expansion.
• Kidney disease programs are advancing with ongoing Phase 3 studies for IgAN and APOL1-mediated kidney disease.

Future Outlook

• Vertex plans to expand CF treatment to new patient groups and geographies.
• KASJEVY is seeing increased uptake and expansion into the Middle East.
• The company aims for full reimbursed coverage of Gernavix in the U.S.
• Promising data is anticipated from kidney programs, with potential regulatory filings for type 1 diabetes.

Q&A Highlights

• Regulatory discussions on PNP focus on a DPN indication, with two Phase 3 studies planned.
• Vertex is using well-trained clinical trial sites to mitigate placebo effects in DPN trials.
• The company aims to differentiate its IgAN treatment with best-in-class clinical data and patient-centric factors.
• Vertex is addressing the impact of the MFN clause through active advocacy efforts.

For more detailed insights, please refer to the full transcript below.

Full transcript - Wells Fargo 20th Annual Healthcare Conference 2025:

Operator: Great. Thank you very much for joining us today. It’s right before the lunch session

Mohit Bansal, Biopharma Analyst, Wells Fargo: probably. My name is Mohit Bansal. I’m one of the biopharma analysts here at Wells Fargo, and I’m joined by Vertex team today. We have Suzy Lisa, Head of Investor Relations, and we have Manisha Pai. She’s Executive Director, Investor Relations at Vertex.

Thank you very much for being here.

Suzy Lisa, Head of Investor Relations, Vertex: Thanks for having us. Super happy.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Awesome. So maybe, like, before we get started, I mean, there’s a lot to talk here. Can you talk a little bit about the like, set the tone with, like, you know, what what is what is the discussion topics right now, and what what you are excited about for next six months or six to twelve months which investors should be focusing on?

Suzy Lisa, Head of Investor Relations, Vertex: Sure. So I think that it feels like it’s always an exciting time at Vertex, to Right be honest, now, we are executing very well, I think, on three ongoing launches with Elliptrak, our fifth marketed CF medicine with KASJEVY for sickle cell disease and beta thalassemia, where we continue to make very nice, steady progress And then the early days of the Gernavix launch, the first non opioid for pain, the first new therapy for pain acute pain in over twenty years, really exciting in terms of the progress we made, and I’m sure we’ll get into that into more detail. So it’s this new era of commercial diversification and commercial execution, which I think is off to a very good start, that combines with our historical excellence, I think, in terms of R and D execution and pipeline execution, right? So as I mentioned, fifth CF medicine now diversified into three additional disease areas. And we have four ongoing phase three studies, as well as a fifth to start soon.

So expanding into kidney diseases with two ongoing Phase III’s for in enaxaplan for APOL1 mediated kidney disease with POV in immunoglobulin A nephropathy. We also are close to completing the enrollment and dosing in our Phase III study for Zamyla cell, which is the old VX-eight eighty for type I diabetes, with truly outstanding effect. And then we have our ongoing DPN Phase III studies for expanding into peripheral neuropathic pain indication. Then there’s a pipeline behind that with products soon to be, or currently initiating Phase II study, proof of concept studies in ADPKD, autosomal dominant poly polycystic kidney disease, as well as ongoing work in myotonic dystrophy type one in patients in the clinic, and then more behind that. So I think combining those two areas of excellent execution, both in terms of R and D and our differentiated strategy, focusing on sandbox diseases where we understand the causal biology, and hopefully leads to greater rates of success, as well as what is the early days of commercial and revenue diversification.

It’s a really exciting time.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Awesome. So maybe look, I mean, there’s a lot going on, so I can go in any direction. But let’s just begin with the second quarter call here. Sure. So there were two different topics which kind of made people a little bit nervous, and we saw that in the stock reaction as well.

Can you talk a little bit about the regulatory discussions where, I mean, it was like, again, you were discussing with the FDA that there is a is there a path there? But can you talk a little bit more about, for the investors, like what you were discussing and how the discussions unfolded there?

Suzy Lisa, Head of Investor Relations, Vertex: I would say a big dislocation in the stock after the Q2 call, I think, regardless really of the very good early data on our launches commercially and a really good financial quarter execution wise. But I think the main disappointment that investors heard from the call was the fact that our next steps in terms of reaching the market commercially in The US for peripheral neuropathic pain will be through a DPN, diabetic peripheral neuropathy indication, and that we’re starting imminently a second phase three study there. There are ten million patients, ten million plus peripheral neuropathic pain patients in The US. And our goal is still ultimately a broad neuropathic pain label. But at our end of phase two meeting at the July, the FDA had invited us in to talk about that potential.

They have only ever previously granted single indications in peripheral neuropathic pain. We came in and had an excellent, robust discussion. But it was clear that they weren’t there in terms of granting a broad label with our proposal to do one DPN phase three and one lumbosacral radiculopathy, or LSR, study. That would not lead to a broad PNP label. They are still thinking more in terms of etiology, right?

There are many different causes of peripheral neuropathic pain toxins, cancer, metabolic diseases, etcetera. And so although we feel strongly mechanism of action addresses all these different causes, and hence our goal of a broad label, the FDA is not there yet. And so what was clear from that discussion is that doing, as per their past history, two studies in one indication will lead to a label with indication. And then subsequent follow on studies, single study, could lead to add ons to that label. So hence, our decision to not proceed next with a phase three in LSR, but instead do a second phase three in DPN to get that indication as quickly as possible, and then add on from there.

We don’t yet know the next steps. The FDA may be open, pending additional considerations, to smaller basket studies, maybe polyneuropathies or mononeuropathy. Or it may just make sense for us to go and do a single study in the next indication. That’s the work that we’re still ongoing. But we ultimately do see potential for the whole ten million patient population, but very near term focused on the two million plus that have diabetic peripheral neuropathy, and executing on those studies, which are well understood, well trained clinical sites, understand the treatment effect, how to manage placebo effect, etcetera.

And what we said is that we, our plan is to complete enrollment in both of those phase 3s in DPN next year, in 2026.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. So the path for the broad indication is not closed yet, but you need the DPN as an anchor indication, and then just build on that. Is that

Suzy Lisa, Head of Investor Relations, Vertex: That’s exactly right. And with high visibility on the pathway to DPN, which I think is some of what got lost, people felt that the feedback had been anti Suzetra gene or something. And that is in no way the case. And we have high visibility on the pathway in DPN, and are going right about executing that as quickly as possible, with a goal still for the broader population over time, pathways still to be determined.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. So again, so that brings us to the second question. So how do you think about the, like what are you doing to make sure that you have the highest probability of success in DPN trials, given that, you know, if you look at Lyrica’s history, it was hit or miss because, you know, the problem with these subjective trials is that placebo can be So strange what are you doing to make sure that, you know, you you have the highest probability?

Suzy Lisa, Head of Investor Relations, Vertex: Yep, good question. I think DPN is much better understood than, say, LSR, which had never been a phase three study in LSR, for example. There have been multiple phase three studies in DPN. There are clinical trial sites that are well trained in the condition, as well as how to manage placebo. There’s very well understood magnitude of treatment effect for the active agent, as well as the placebo arm.

And by using these well trained sites and established protocols and confidence in our mechanism of action, that’s what leads us to high confidence in the probability of success for our studies.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. So you have one trial which has active control, but the other one is just placebo control, right?

Suzy Lisa, Head of Investor Relations, Vertex: That’s correct. The second phase III will be a little bit smaller, because it will just have the placebo arm as per FDA protocol and what’s required for the indication.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. So can I talk a little bit about the timelines of this? Because one trial you have started earlier, then second one you are starting now. And I think we chatted before that, I mean, there’s this trial conduct issue, because you don’t want a subjective trial to read, one trial to read out fast versus others. So how should we think about the timeline at this point?

Can you just make them together versus

Suzy Lisa, Head of Investor Relations, Vertex: Sure. I don’t have a full answer for you yet. All we have said is that we do expect to complete enrollment in both DPN phase 3s next year. The first one, you’re correct, as you said, actually began in December ’4 and has been enrolling at a very fast pace. We’re pleased with that.

The second one will be slightly smaller. But we will be using to your prior question, we will be using the same well trained sites to ensure probability of success. So at some point it becomes a decision as to which trial to allocate patients to. We may decide to accelerate and complete one. We may decide to manage them both so that they end on a similar timeframe so you don’t have to worry about ultimate results impacting the second study.

But it’s still to be determined, so I don’t have an answer for you beyond completing enrollment in both next year.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. Got it. But, yeah, like infecting the other trial state results because of the first trial readout could be a consideration.

Suzy Lisa, Head of Investor Relations, Vertex: That’s right. That’s right.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Very helpful. And then another topic is the September trial. I don’t know if that was the main reason for the stock weakness, but again, that trial in the acute pain did not show benefit. So it’s a potent drug. So what is the takeaway internally from that trial, and how does this impact the peripheral neuropathy trial, or DPN trial that you’re running with nine ninety three at this point?

Suzy Lisa, Head of Investor Relations, Vertex: Sure. So I think that what perhaps why there was what felt to us like a disproportionate fell off, I think it was primarily related to concerns about the ultimate peripheral neuropathic pain potential. But then in VX993, a next gen that was being studied in bunionectomy in acute pain, we did see a similar sized treatment effect, but had hoped for more essentially based on what we had seen in terms of the potency and efficacy. But what we learned, and I think probably in hindsight on IR we should have framed better, is that going higher up the curve did not get you greater treatment effect. Essentially, we are getting all we can hope to get out of Suzetra gene, out of Gernavix, by inhibiting the Nav1.8 channel.

And although is VX993 certainly remains a viable option in acute pain IV, because it can be formulated as IV, as well as potential combination use with a future Nav1.7 inhibitor, as can Jirnavix, that we won’t be advancing it in acute pain as monotherapy, because we are getting what we need to get out of, or what we can get from inhibiting that channel with Jernavix. But the ongoing phase two in DPN, we’ll run that study out with VX993, see what we learn about dose response curve. There is some accumulation and slight differences, and add it to the body of work. But I think perhaps investors have seen what we’ve done in CF and thought perhaps serial innovation is very, very easy, and there’s always an obvious next step. And this was more to validate our belief in terms of what we’re getting by inhibiting 1.8, and the potential to now move to inhibiting 1.7, which actually triggers the pain sensory reaction, and then potentially combo use by inhibiting both the trigger and propagator, oneeight, that you could ideally have a synergistic effect.

So that’s the direction that serial innovation will take from here.

Mohit Bansal, Biopharma Analyst, Wells Fargo: That’s what you have done with CF as well, starting with Klaydeco and then adding on top of that. Right? So that makes sense, actually. Let’s just talk about, you know, like, Genovix a little bit. You know, you were you have been open about, you know, first half is going to be, like, volume driven and sales will come sales will follow the volume growth.

Considering all that, I think second quarter was pretty good, actually, at least in our opinion. How are you thinking about I mean, do you agree with that, with the progress you have made at this point? And then talk a little bit about the sales effort that you are increasing for Generics launch at this point. I mean, was that already planned? Or is this a reflection of what you are seeing in the marketplace at this point?

Suzy Lisa, Head of Investor Relations, Vertex: Yeah. So thank you for that. We have been pleased with the early results. If you go back prior to launch, I think there were many, many who said not you, Mohammad many who said, you will never no one will ever pay for this when you can get generic opioids for pennies on the day. And I think what we have proven is that, yes, Vertex does know what a PBM is and how to talk with them, as well as the fact, more seriously, that there is a true unmet need for additional mechanisms of managing pain in the marketplace, and a non opioid option that doesn’t have the side effects nor the addictive potential.

And so it is a game that we’re in for the long run here. And I think we’ve been pleased today with slightly faster than expected potentially progress with payers. So we have two of the three largest PBMs under contract, and we continue to expect we have high visibility on adding the third before the end of the year. We have the two largest hospital group purchasing organizations with contracts. And as of the Q2 call in early August, we had said we have 16 state Medicaid plans, and continue to make progress there as well.

So I think from a reimbursed access standpoint, it has been very good progress. The other important barometer is with respect to getting on hospital formulary, right, and going through the P and T committee process, which can take anywhere from six to eighteen months. We’ve tried to accelerate that process where we can, and are pleased there that we’re on about a very good portion of hospitals either have us on formulary or discharge order sets or are ordering Gernavix as of this time. Then it is the field force reach, our virtual sales team reach, and then our targeted media outreach. And there too, I think that we are looking for the long run success here and trying to build as broad a foundation as possible.

So we’re trying to go very broad, consistent with the labeling in terms of physician type, setting of care, pain type, etcetera. So we’ve been pleased with the breadth of physician prescribers that we’ve seen, anesthesiologists, pain specialists, general surgery, ER docs, orthopedic surgeons, etcetera. We’ve seen nice uptake from dentists, if you will. So really broad range there, and seeing good rates it’s still early, but good rates of repeat prescriptions as well. And that’s where we are seeing things being promotionally sensitive.

And now that we have gotten to a point in time where we do see broader access in terms of reimbursed coverage, as well as increasing formulary adoption, now is when it makes sense to add more from both a marketing and a field force standpoint. You want to think about the initial 150 person field force rep size, that they’ll continue their focus on the top 150 or so IDNs that we’re focused on. But in terms of physicians, some of those in sort of they’re covering tier one. Beneath that was the virtual sales force. They’ll move up more of them, and the new team will cover those folks.

And then another bucket will come into the virtual Salesforce team.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it.

Suzy Lisa, Head of Investor Relations, Vertex: But to go out with that biggest Salesforce initially when you didn’t have fully reimbursed coverage and you weren’t on formularies, that probably would have been too soon. So we think this timing is what makes the most sense. But pleased with the breadth, with the physician reaction, with the patient anecdotes that we’ve heard, I think it’s off to a really strong and encouraging start.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Great. Are you seeing usage outside of the hospital setting or surgical setting as well at this point, or mostly

Suzy Lisa, Head of Investor Relations, Vertex: Well, maybe I don’t understand your question, but about The

Mohit Bansal, Biopharma Analyst, Wells Fargo: label is broad, right? But I mean, your target is in the post surgical setting at this point. But are you seeing usage outside of that as well at this point?

Suzy Lisa, Head of Investor Relations, Vertex: So the mix of the scripts is about sixty five percent in that discharge at home setting, and about thirty five percent in the hospital or ASC setting, if I’m getting that. But then you do see, as I mentioned, the dental setting and some other things, if that’s your question. But very strongly, I think given the concerns about opioids and pain management, we see a big role for this in at home post surgical care.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Do you see a role for DTC campaign, things like that, for these kind of medicines?

Suzy Lisa, Head of Investor Relations, Vertex: I think in a very targeted, more direct to patient way. Today, if you are in an ER or you’re looking online for information about a hip replacement, you’re likely to hear from us about, have you thought about your pain management strategy too? And we have non branded celebrity campaigns there with Alex Smith, a former professional quarterback who had a horrible leg injury, look for more on that front from us, potentially in a branded capacity, and more and more targeted ads through micro targeting, think streaming services, etcetera, as opposed to not a Super Bowl ad, that type of thing, but trying to be savvy and efficient with our spend. But doing more and more of that in addition to, as I mentioned the as we mentioned on the q two call, the expanded sales force.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. Very helpful. Thank you for that. So maybe we will again, anyone, if you have questions on the call, please feel free to ask those questions. So let’s just move to CF, the small business you have.

So I think the business is strong, and I mean, you have a long tail as well. How how should we think about the next leg of growth here? I mean, Elefrec conversion is one. I mean, you did like like, that was also a missed thing from second quarter called that conversion actually accelerated for ElefTrak as well on second quarter. But there is a ElefTrak, but, again, do you like, you are well penetrated in those spaces.

So where is the next set of growth coming from? And how should we think about that effect conversion going forward?

Manisha Pai, Executive Director, Investor Relations, Vertex: Sure. Thank you for the CF So I think we do see continued growth ahead for CF overall, while we don’t give long term guidance. Some of the drivers of that growth include new approvals and reimbursements, particularly in younger patients. So TRIKAFTA is approved down to two years old now, AlifTrac down to six years old. We’re working toward one to two for both of them, I believe.

So getting down to younger patients approvals and reimbursements. Uptake still in our core markets, as well as in some of the newer geographies that we started talking about this year. Those are countries where we have relatively recently secured formal reimbursement, where previously it was named patient programs or things like that. And those are countries, let’s say, like Brazil. And then, of course, the Elliptrex global launch.

So we’re approved for six years and older in The U. S, Europe, U. K. And Canada. OUS, we are still working through that reimbursement process, but that’s to be expected.

We have reimbursement in England, Ireland, Denmark, and Germany, I believe, for AlifTrex in Europe. And that’s sort of how we see it. But also the overall market is growing as patients are living a lot longer than they used to, partially thanks to CFTR modulators.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. Got it. That’s very helpful. And then with ElifTrac, I mean, so how should we think about the conversion? Because at one end, have TRIKAFTA, which actually converted very quickly.

Mean, ElifTrac is not on that trajectory, but obviously the label update is probably one part of it. But how are you thinking about longer term? Will we get to a TRIKAFTA level? I mean, tell me how you think about that. Because it is going up against a really good drug, though.

Manisha Pai, Executive Director, Investor Relations, Vertex: Yeah, yeah, absolutely. And so I think one thing to keep in mind is that when Trikafta was approved, our first triple combination, we went from having medicines that could treat about fifty percent of patients to all of a sudden having one that could treat around ninety percent of patients So at that there was this large initial bolus, whereas the populations the patient populations for Elephtrac and Trikafta largely overlap. There are some additional mutations that respond to elyphthrex that don’t respond to Tricaphtha. And some of the most rapid uptake, in fact, that we’ve seen in The U. S.

Has been among those patients for whom they’re newly eligible for a medicine now with the approval of elyphthrex. And then in terms of conversion, we do expect the majority of patients to switch from Trikafta to a LiftRec over time. In The US, we do have that additional liver monitoring requirement in the label, which is for the first six months treatment, a patient has to have monthly liver monitoring. It’s a simple blood test. You can get it done at your local Quest labs or wherever.

You don’t have to go to the hospital. But it is a logistical consideration that patients are working through with their physicians and how to manage that with their day to day lives. In the OUS markets, Canada, UK, and Europe, there is no additional liver monitoring requirement in the label. So it’s quarterly for the first year and annually thereafter.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. Very helpful.

Manisha Pai, Executive Director, Investor Relations, Vertex: Oh, and one thing to add there. On the Q2 call, we talked about that uptake that we’ve seen to date. And as I mentioned, the most rapid uptake has been among those rare mutations that did not have access to a therapy before, followed by patients who discontinued a previous CFTR modulator and were no longer on treatment. Makes sense that these two populations would be in the front of the line there. But the vast majority of patients who are on Elliptrex as of the 2Q call are patients who switch from Trikafta because that is a much bigger pool to draw from.

So we are pleased with the progress we’re seeing on all three of those groups.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. For a new patient, there is no reason to not go on Elephantrex right now, probably. For a new CF patient, is there?

Manisha Pai, Executive Director, Investor Relations, Vertex: Yes. Yeah. I mean, we do believe that ElefTrek is the best CFTR modulator for eligible patients based on the data in the phase three studies on CFTR function, as well as the once daily dosing. So yes, I would imagine that that

Operator: And

Mohit Bansal, Biopharma Analyst, Wells Fargo: would be the another question. We get a lot, and I’m sure you have received a lot. I mean, the MFN part of it. I mean, you were not among the companies which got the latter here. And you are catering to a rare disease here, even though you have a decent Medicaid exposure.

So what is your internal thinking about this? There’s not a lot of detail here, but again, would love to know what you’re saying.

Suzy Lisa, Head of Investor Relations, Vertex: Yeah, I think a lot of advocacy work on our part behind the scenes, trying to stay as up to date and understand and being planful in advance, which can be challenging given lack of details. But we were not one of the initial 17 that received a letter. I think fundamentally, part of our view is that by focusing on serious diseases and trying to deliver transformative effect, that that ultimately can position you better overall in terms of any type of reimbursement or pricing pressure, if you will. And that would continue to be our hope. But it really is a stay tuned, but we remain very active in DC, as well as very active planning for whatever potentially might happen.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. Very helpful. Thank you for that. So maybe let’s just move on to the other exciting pipeline, the kidney space, right? I mean, you have made good strides there, two major trials are reading out next year.

So let’s just start with PoV in IgA nephropathy, you have like so far with early data look best in class, but at the same time it is crowded market. So how are you thinking about your drug? Would you be able to differentiate on the proteinuria alone? Or how should we think about when we see the data and your confidence level in terms of positioning of this drug?

Suzy Lisa, Head of Investor Relations, Vertex: Yeah, I think that we as I mentioned, we have this very rigorous sandbox disease approach and understanding the causal biology. I think as the science developed around B cell mediated diseases, that we did a very thorough survey of the landscape and understanding the data and the candidates, and I think felt strongly that Alpine Immune Sciences and POV offered the best opportunity for differentiated B cell control, and hence the acquisition in April. And with respect to IGAN, I think also an understanding that in this type of market where patients can switch, right, that it isn’t always first entrant that ends up with dominant share. And our hope is to be best in class, if you will, on two significant buckets. One is the clinical data and the differentiation, and secondly are the patient centric factors.

And so in terms of the clinical data, we think the dual inhibition of BAF and APRIL and the targeted design of POV, the data preclinically and clinically, yes, we’re still early in terms of showing only phase II data, But we have seen differentiation in terms of depth of response with greater reductions in ePCR, and we would hope to be able to sustain that in our phase three study. We’ve been pleased with the pace of enrollment. We said that we had completed the interim analysis cohort enrollment on our May call. And then with thirty six weeks of follow-up from that, that would position us to release data on the interim analysis in the ’6. And on the Q2 call, we said that we expect to complete enrollment of the full study by the end of this year.

So I think seeing good receptivity from physicians and that can always, I think, be an encouraging biomarker of underlying optimism or demand for the product. So we’d hope to continue to sustain that depth of response and best in class profile of optimal B cell control. And on the patient centric factors, they were also differentiated with lower dose, lower dose sub q auto injector at home only every four weeks. And so having the combination of those factors plus the best B cell control, we hope, would lead us to a differentiated profile. We also hope to have multiple indications so nephrologists wouldn’t have to learn different meds for different indications and go from there.

I think a strong understanding internally of this type of market, as well as the kidney space, is what led us to the Alpine acquisition. And we continue to only be more and more encouraged by that deal as time goes on.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. That’s very helpful. Thank you for this. Maybe, like, can you please set the tone for the other phase three readout? I don’t know if it is next year, but again, you plan to finish enrollment this year, so it’s like one year for the inaxiplan data.

So can you help us understand, like, what to expect when we see the data next year, or maybe early ’seventy seven?

Suzy Lisa, Head of Investor Relations, Vertex: For inaxiplan? Yeah. Sure. So inaxiplan is our ongoing phase three study in APOL1 mediated kidney disease. And this, I should have mentioned in IGAM, right, a significant patient population.

I think it’s north of 300,000 in US and Europe, north of a million globally. And we partner with Zai Lab in China and area regions, and then Ono in Japan and associated regions there. So, and are seeing good uptake there. With IgAN, with APOL1 mediated kidney disease, excuse me, about two hundred and fifty thousand patients, both in sort of primary AMKD, if you will, and then those with comorbidities. And that study is on track to complete its interim analysis cohort enrollment by the end of this year.

And once that is completed, then it is forty eight weeks of follow-up before we can have the interim analysis. So depending on timing, we’d be hopeful you could see data next year, but still need to determine when that final patient in the interim analysis cohort is enrolled. And really no competition here, nothing that’s going after the causal biology of this disease. We saw very compelling phase two data and would hope to continue to see that. I think, too, we’ve been encouraged by the uptick in the pace of enrollment, which led us to giving this milestone to complete the IA cohort enrollment.

Because of all of our great work in terms of developing this market and awareness and trust with this community, given that it’s the disease of those of African descent. So working with Alonzo Mourning, the NBA All Star who had the disease and needed a kidney transplant, but working with community groups, church groups, etcetera, and that, and as well as physician awareness and opening additional trial sites has led to the accelerated enrollment.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. Very, very helpful. And then you have some of the proof of concept trials as well for some of the assets, like 4,407, the $6.70, and DM1. So can you talk a little bit about the timing of those, if you have disclosed that?

Manisha Pai, Executive Director, Investor Relations, Vertex: Yeah, sure. So VX-six seventy, we’re studying it, as you said, in myotonic dystrophy type one, or DM1. That’s in a phase onetwo single ascending dose, multiple ascending dose study in patients. We are in the MAD portion now, and we expect to complete enrollment and dosing in the first half of twenty twenty six. And then for VX407 in a subset of ADPKD patients, we are in the process of initiating the phase II proof of concept study.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it.

Suzy Lisa, Head of Investor Relations, Vertex: If I could also go back to Povey for a second, Mohit, that we’ve talked about primary membranous nephropathy. We’ll begin a phase three study in the second half of this year. And then on the Q2 call, we said that we have identified additional indications, one in Waha, and then also in generalized myasthenia gravis. And so different phases there. We’ll let the full set of Ruby four data mature through the end of the year before next steps in Waha, but are excited about that in warm Autoimmune.

Autoimmune. Let’s just leave it at Waha, sorry. And that’s probably about thirty five thousand patients here in The US and Europe, so an interesting opportunity. And then in generalized myasthenia gravis, next steps will be go to FDA and talk to them about what the next steps need to be there.

Mohit Bansal, Biopharma Analyst, Wells Fargo: I feel like a PoV analyst day is coming with like a big slide deck of 50 slides probably at this point.

Suzy Lisa, Head of Investor Relations, Vertex: I think that we’re excited for the American Society of Nephrology in November, and stay tuned.

Mohit Bansal, Biopharma Analyst, Wells Fargo: Got it. Very helpful. Okay, so last question. I ask this question to everyone. So fast forward one year.

I hope you are sitting here. I hope I am sitting here and asking this question. What would make you look back at the year and say it was a great year for us?

Suzy Lisa, Head of Investor Relations, Vertex: I think that it will be continued growth and expansion in new patient groups in CF, that you will see continued patient uptake in KASJEVY, where we’ve been pleased there, and expansion into The Middle East, as you have seen. Jernavix with full reimbursed coverage in The US, right? We mentioned one hundred and fifty million lives on the Q2 call in The US. Eighty five million of those with no prior authorization and no step edit. So continuing to grow that number and getting closer to full coverage or being there, and having been able to pull back the patient support programs that we’re currently offering.

So recognizing significant revenue from that, from all three of those disease areas, and then having great data in hand, essentially from POVY and IGAN initially, or getting close to data release, if you will, in AMKD, and having regulatory filings in T1D. And I think that’s

Operator: get us started

Suzy Lisa, Head of Investor Relations, Vertex: a lot of really exciting milestones between over the next twelve to eighteen months at Vertex, and looking for continued excellent commercialization execution.

Mohit Bansal, Biopharma Analyst, Wells Fargo: On that high note, thank you very much for coming Okay.

Suzy Lisa, Head of Investor Relations, Vertex: Thank you. Thank you again.

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