Thanks, thanks for joining us, everybody. I'm Terence Flynn, the U.S. Biopharm analyst here at Morgan Stanley. Before we get started, for important disclosures, please see the Morgan Stanley Research Disclosure website at www.morganstanley.com/researchdisclosures. Very pleased to be hosting Vertex this morning or this afternoon. Joining us from the company, we have the company's President and CEO, Reshma Kewalramani, and Charlie Wagner, the company's CFO. Thank you both so much for being here. Really appreciate you taking time out of your busy schedules this close to the end of summer, but really looking forward to the conversation. So thank you.
Reshma, maybe I'll turn it over to you just for some opening remarks to set the stage, and then I obviously have a lot of questions we can run through, but I'll turn it over to you to start.
Sure. Well, Terence, thank you so much for hosting us. It's nice to see everyone in person. Maybe three things to start with. First, in our cystic fibrosis franchise, we continue to reach more patients than ever before as we go out to more geographies, get regulatory and reimbursement agreements for the lower and lower age groups. And we are very excited about the vanzacaftor NDA, which is also in for review with the European regulators. That brings... And I know it's hard to believe, but it brings what looks like even more efficacy than TRIKAFTA. That PDUFA date is January of next year. Also, in CF, we're very excited about the VX-522 program, which is an mRNA program with our partners at Moderna, which aims to treat the last 10%.
There's about 5,000-7,000 people with cystic fibrosis who simply do not make any protein, so the small molecule approach just won't work for them. For those last 10%, the VX-522 program is making its way through its phase I/II study in patients. The SAD single-ascending dose is complete, and we're working through the MAD. The other exciting news to share, which we haven't been able to do in the past when we talk about our commercialized assets, is our newest franchise in hematology, the CASGEVY program in sickle cell disease and beta thalassemia. The regulatory approval was historic, as was the clinical trial and the results, and now I'm really pleased to see that translate to real-world benefit for patients. You'll hear from Charlie and me talk about the early launch, which is going very well, and the metrics-
... that we're following and the patients that are getting to benefit from this in the real world. Moving then to the mid and late-stage pipeline. In the past, we could cover at least most of the pipeline, if not all of the pipeline, in a quick one-minute summary. That won't be possible today, so I'll just pick a couple of programs. In phase III, already submitted for regulatory approval is the suzetrigine program, previously known as VX-548, a NaV 1.8 inhibitor for acute pain. There is several programs behind that, but I'll just call out the program in diabetic peripheral neuropathy, which is in phase III, and the program in LSR, a different kind of peripheral neuropathic pain, which is in phase II.
That one is accelerated in terms of enrollment and dosing, and we now expect results from that phase II program sometime this year. There's a phase III program with Povetacicept in IgA nephropathy and a whole host of potential indications behind that in phase II. There's a phase III program in a particular kind of kidney disease called AMKD, and there are phase I, II programs in type 1 diabetes, a fabulously interesting, innovative program with regenerative medicine and using allogeneic stem cells to basically restore pancreatic function, and another program in patients in something called DM1, myotonic dystrophy type 1, and then lastly, if I think about the earlier stage pipeline, there's a lot going on there in our way of doing R&D, and I'm happy to talk a little bit about that.
And the punchline is we are a disease-first company. We don't work by platform or modalities, nor do we work by therapeutic areas. So there's a whole host of diseases that fit our very strict criteria for what we believe will yield disproportionate success. And I'll just mention in the early stages, there's a NaV1.7 program in late preclinical development that I'm super excited about because of its potential as monotherapy. But more interesting to me is possible combination therapy with our NaV1.8 molecules. There's a very neat program in improved conditioning, which might make CASGEVY available to even more patients, and I'll pause there.
Great. Well, no, I appreciate the breadth of the pipeline. As you said, it used to be you'd talk about CF for a whole session, but now you guys are in so many different areas, so the diversification strategy is really, you know, playing out here. I guess first, you know, again, Reshma, you started with vanzacaftor. You have the PDUFA date coming up in January. Maybe just help us think about kind of the early launch dynamics as we go into 2025. It's gonna be another product cycle. You guys know that market and all the patients phenomenally well. Maybe just level set us in terms of where you're gonna be focused in terms of that early opportunity, in terms of the launch next year.
Definitely. I'll ask Charlie to talk about that. The only thing I'll say is the results that were so impressive to us from phase III, which is exactly what we wanted, is a medicine that matches the ppFEV1 lung function gains of TRIKAFTA, but do even better in terms of sweat chloride, which is a measure of the protein function, and that's the protein that's dysfunctional in CF patients. And our big goal here is to get all of our patients to carrier levels of that particular function. Over to you, Charlie.
Yeah. Maybe just to add on that, you know, our. We're in the fortunate position that our patients and the, and their physicians are incredibly well-informed on our medicines, and there's a lot of anticipation around vanza, and the benefit that it can bring. So I would have you think in terms of two cohorts of folks. You know, one, there are roughly about 6,000 patients who have discontinued one of our prior medicines for a variety of reasons. Those folks would be very eager to try a new therapy, and vanza certainly represents a great opportunity there. To give you a sense, I mean, that discontinuation rate is quite low, but sometimes folks will discontinue because of minor side effects, like a rash, or maybe they've had a life event, that sort of thing.
Makes it very easy for them to come back and try a new medicine, and so that's obviously an incremental opportunity to what we have today. Additionally, we would expect, over time, significant transition from prior medicines to vanza. We're not, at this point, gonna call exactly what that looks like for 2025 specifically, but based on our conversations, based on our market research, we think over time, the majority of patients will switch to vanza. That said, those that choose to stay on Tri, for example, are on a fantastic medicine, and that's a great result as well.
I think in addition to the clinical profile that Reshma described, vanza also has the benefit of once a day dosing, which is meaningful in terms of convenience, in terms of compliance, and then from a company standpoint, it has the benefit of a meaningfully lower royalty burden. We're really excited about the launch, and we'll have more to say on that, you know, as we get into early 2025.
Okay, great. I guess just in the interest of time, I wanna cover a lot here. Moving on to suzetrigine. You're gonna have the phase III data presented in October at the ASA meeting. Maybe just again, high level, I know when we saw the data earlier this year, you gave a pretty in-depth overview of all the data, kind of primary, secondary endpoints, the safety. But any additional analyses or things we should be focused on heading into this presentation?
Yeah. The VX-548 suzetrigine data have been accepted. The RCTs have been accepted as best in ASA, American Society of Anesthesiologists conference, so they'll be presented there. You're right. Some people would say our press releases were novellas, 'cause it was very detailed. I think you know all of the key data from there, but it will be the first presentation in a congress to the medical community, and I think that's always exciting, but I think you know the thrust of the information. We're well into the part where we've submitted the NDA. They've completed their initial review, the filing has been accepted, it's been assigned a priority date, so I think we're well into the process, but it's always exciting to have data presented to physicians in a congress.
Yeah. Okay, great, and I guess on the commercial prep, Charlie, maybe you could just elaborate. I know it sounds like you've been having more recent discussions with folks out there in the channel, and so just what are you and the team doing here to get ready for this launch? Again, you know, you guys will have two launches next year, so again, I'm sure it's all hands on deck.
Yeah. Yeah, you know, Stuart Arbuckle, our Chief Commercial and Operating Officer, and his team are fully into the launch at this point. We've mentioned previously, we've hired roughly a couple hundred folks in the field for pain over the last 18 months, including sales reps, key account reps, MSLs. They are all fully on board, fully trained, and fully in the field. And it's significant because we're having conversations with a wide range of stakeholders. You know, we've commented previously that there are roughly 80 million people a year in the U.S. seek treatment for moderate to severe acute pain. Half of those people have prescriptions written in an institutional setting, so a hospital or a surgical center, and given that concentration of prescribing, that's going to be the initial focus of our commercial organization.
That said, for those prescriptions that are written in institutions, 70% of them are filled in a pharmacy or in a retail setting, so we will focus on the institutions from a prescribing standpoint, but we're also working, and there, we're having conversations with physicians, with hospitals, with IDNs, all the way throughout, to help them understand the profile, the significant benefits of suz relative to, you know, to opioids. Those conversations are going really well. We know that there is post-approval, there is a kind of a well-understood, well-trodden path to go through P&T committees, get on formulary, so we're laying all of the groundwork for that, anticipating that it's gonna take a little bit of time once we get approval.
Simultaneously, we're talking with payers, PBMs, pharmacies, to make sure that the product will be available for patients who wanna get their prescriptions filled at retail. And in fact, we think the uptake, the revenue opportunity might be a little bit quicker there.
Just because we think that once we get through contracting with payers and some of these organizations, the ability will be there for patients to get their prescriptions filled. We're also sort of evaluating patient assistance programs and other things to aid with the momentum in the first year of the launch. So, we're fully in it at this point, you know, really excited and feeling very confident in our readiness for an early 2025 PDUFA date.
Okay, great. What in terms of... I mean, there's a lot of unique features of this launch, but any analogs that you would point us to as we think about the kind of rollout or, again, it is somewhat unique 'cause there's not, as we all know, many branded pain medications that have launched recently. So are there-- what analogs would you encourage us to think about as we think about the pace of ramp?
Maybe I'll answer it in the inverse and maybe Charlie can add. It is really the case where there are no analogs, which makes it challenging to sort of get your head around, What should I expect? But there hasn't actually been development of a new pain class in more than 20 years, so there genuinely isn't something to look at. What would I not look at? I would not look at medicines that have launched in the recent past in the hospitalized environment, but are only used in the surgical setting. So there are certain medicines that you can use intra-op.
There are some medicines that are a gel or a patch or something like that, but they are not really analogs because this is an oral small molecule, and sometimes in thinking about the vastness of it all, we can lose some of the sort of important smaller threads. It's an oral small molecule. You take it for any kind of pain, and our goal here, and we designed the program with the agency, to be able to get a broad, moderate to severe acute pain label, whether it's post-surgical or not post-surgical. I think you'd have to go back a good 20 years for a branded small molecule to sort of have a sense for it.
Okay. What-
Charlie, anything you wanted to add?
Maybe the only thing I would add is, you know, given the conversations we've had in the field, our confidence in this being a multi-billion dollar franchise is greater than ever, and we are building for the long term. You know, not trying to lower expectations for 2025 , but as we enter these contracting conversations, we're thinking about the trade-offs between value and speed, and we're building a franchise for the long term. So I think we're gonna be thoughtful. We're gonna move as quickly as we can, but we wanna set up the business for success over the long term because we see such huge potential here.
Yep. And then how would you consider handling guidance at this point for the pain franchise? I know CF, you kind of have the framework laid out, what we expect. CASGEVY, again, totally different from pain here. Again, very unique, given, you know, the nature of the therapy and the market. But how do you think about expectations? Should we just not expect guidance in the first year for the pain side? Because, again, the analogs aren't there. Again, a lot of moving pieces, or would you consider giving guidance in?
Yeah, I appreciate the question. It's a little early for us to give an answer. So the good news is we're in the fortunate position where we would expect to have revenue from three different franchises next year. Obviously, though, with CF and the stage of that business, it's so much larger.
Yeah.
We are thinking through different permutations on guidance, but we'll have more to say later this year.
Okay, great. The other, you know, question coming up more frequently is just where, you know, you accelerated the phase II LSR data.
Mm.
So that's coming now, the end of this year. It is going versus a placebo. This is a disease that does not have any options. So maybe just help us think about expectations there. I guess one question we get sometimes from patients is just placebo response.
Yeah
... and your confidence that there won't be kind of an outsized placebo response. But again, we know the safety profile from the other studies, so it's more about that kind of effect size-
Yeah
... placebo response. So maybe just walk us through kind of how to think about that ahead of this data set.
Yeah. So maybe three or four things that are important-
As we await the LSR results. So LSR is lumbosacral radiculopathy. It's the impingement of a nerve in the L5 region. That's the pain that you feel. There have been trials that have been done, but there are no medicines that are specifically approved for pain from LSR. And LSR fits into the broader category of peripheral neuropathic pain. So diabetic peripheral neuropathy, the program in which we released phase II results last year, where we're in phase III, that's one kind of peripheral neuropathic pain. DPN, diabetic peripheral neuropathy, is about 20% of the overall peripheral neuropathic pain market. 40% of it is this, LSR, and the rest of it is things like herpetic neuralgia or small fiber neuropathy. So putting DPN together with LSR, that is more than 50% of what is PNP. All right, the phase II trial.
The phase II trial has enrolled and dosed faster than we expected, and we have very ambitious timelines, but it has gone faster, so the data readout will be this year, and we had originally expected it to be next year. The study has taken the high dose from the DPN program, so the 69 milligram dose, which is the same dose that's in phase III in the diabetic peripheral neuropathy study. It is a study that has the arm that is randomized to suzetrigine. The analysis is a change in the pain score from baseline to the end of the study period, and there's a placebo group. Same thing, the change in baseline pain score to the end of the study period. The question you asked, Terence, about what is your confidence level?
It's the same question we got when we were studying acute pain and when we were studying DPN, and my confidence level in those two instances was high, and it is equally high here, and there are three reasons for that. First, the mechanism of action. This is a very specific medicine that targets NaV1.8. NaV is the sodium channel 1.8. That channel is specifically found in C fibers of peripheral nerves only, and the job of that particular channel is to propagate the action potential, which is what leads to the sensation of pain. So it's extremely specific. That specificity has led to a whole suite of positive trials with the predecessor molecule. It was called VX-150, then with a suite of studies in acute pain and DPN with VX-548.
I happen to be a Bayesian in my thinking around probabilities and what's gonna happen next. In so far as all of the trials worked before this one, my confidence level is high. The next part of this to think through is the selection of patients, and we've been very thoughtful about making sure that we have patients who are included in the study who genuinely have this thing called LSR, and their inclusion and exclusion criteria, physical exam findings, et cetera, that allow us to do this. Lastly, you mentioned it, and it's absolutely true, pain studies are hard to do because of the placebo effect. There is a placebo effect. The perception of pain in one person and what they might score it is different than another person.
We've really worked our way through how you should design these studies, and we've done it successfully now in the whole trial program with VX-150 as well as with VX-548. You put all of those things together, and that's why my confidence level is high.
Okay, great. Maybe last one, it's probably more for Charlie before we go on to Pove. You know, then another question we get frequently is just partnering. Obviously, you guys have laid out the strategy for the acute setting. Chronic, I think there's still some debate about, you know, this is a much larger potential opportunity. How do you think about, you know, maybe potentially partnering this for that setting, you know, over the longer term to address-
Yeah
... maybe a wider prescriber base?
I think most importantly, people tend to think of chronic pain as one thing. I think we think of it as two things, which is neuropathic pain and all other chronic pain. Neuropathic pain we see as, kind of something we can serve with a specialty sales force, much like we see with acute. We are going to focus on commercializing in neuropathic pain. We think we can do that exceptionally well. We have seen in some of our trials that the medicines work for other forms of chronic pain, musculoskeletal pain, et cetera, and we do think that there is opportunity there for patients and opportunity for value. We'd like to establish the value of the medicine in acute and neuropathic, and then over time, we think there will be an opportunity in all other chronic.
Given that that's a kind of a primary care call point, it's likely that we would partner for something like that, but first things first.
Have you gotten interest from folks, like, given the profile of the asset?
I mean, I think the profile of the asset is pretty well understood at this point, so, it's... I think it's interesting to a lot of people.
Okay. Okay, great. You know, moving on, Pove, you know, this is a product and a platform and a product here, as you've talked about, Reshma. You know, one thing I think some people are trying to understand is just this asset goes after BAFF and APRIL, and some of the other competitors are focused on CD38.
Mm-hmm.
So why did you guys select this asset in the context of the pathway and, you know, the data you've seen? Maybe we'll start there.
Yeah. So, Povetacicept is exactly as you described, a pipeline and a product, and there are eight indications right now in phase II already. IgA nephropathy was just the lead indication, and on the earnings call, I talked about the fact that we would be in phase III in August. It's now September, so it's a good day. The reason we're interested in APRIL/ BAFF inhibition is the following: there are a host of renal diseases and hematologic diseases and actually others, but I'll focus my comments on renal and heme, that are known to be B-cell driven. So in the basket of renal diseases, which are already in phase II, it's called the RUBY-3 program. There was IgA nephropathy, membranous nephropathy, lupus nephritis, and something called ANCA-associated renal diseases.
These are all B-cell mediated diseases. And in the heme basket, you have things like ITP, cold agglutinin disease, et cetera. The interesting thing about BAFF/ APRIL is its dual inhibition of pathway, and what you get from that is inhibition of maturation, differentiation, and proliferation of B-cells, which is critical in tamping down the immune response, which is the cause of these diseases. So the dual inhibition at early and later stages of B-cell development versus some other approach is what attracted us to this. We also had a chance to see the phase II data in IgAN, and we had real respect for the Alpine team. I think the design of these basket studies was very clever.
So you put it all together and it was a real find for us, and we're delighted with how the acquisition has gone since.
Yeah
... the announcements.
and on the other side of that, the, you know, the risk-benefit, the infection risk, I guess, with depleting B cells, just, you know, walk us through that, that aspect. And again, I mean, this is, there's a lot of history here.
Yeah
Around this, so I'm assuming that's where you got the comfort from.
Yeah. It's a, it's a really excellent question because the trade-off in, in all of the immunological diseases, because the goal of it is to tamp down the immune system, is obviously and clearly, infection and such. So we looked very, very carefully at the IgG levels and the IgA levels, and we looked very, very carefully at the AE profile. The efficacy in this case is world-class, and because of the benefit-risk assessment, we believe that this is the molecule that has best-in-class potential, not just a medicine that looks great on efficacy or on safety. Benefit-risk is gonna be important. It's a biologic. It also has to have additional characteristics. This one is a monthly injection. It is a low volume, and those kinds of features end up being really, really important for chronic biologic administration.
The preclinical science was very, very sound. What we saw in the clinical trials data made us very excited and has these features of what is gonna end up being very important to commercialize.
Okay, great. Maybe we'll just talk about the pivotal trial in IgAN a little bit. So you mentioned the trial is now underway. I think you guys, on the second quarter earnings call, you gave a little bit more of the details about the trial, this 36 week interim analysis-
Yep
-year. Maybe just remind us, like, what-- do you have a buy-in sign off from FDA on kind of what that bar looks like and the endpoint and then, you know, what would be the, you know, traditional approval, like, endpoint-
Yeah
-and what you need to show there, I guess?
Yeah. The short answer is yes. The long answer is, we have multiple programs now in renal medicine by the progress of our pipeline. The in IgA nephropathy, it's been established, and the agency's been very transparent about the fact that they would accept proteinuria as an accelerated approval endpoint, and that's exactly our discussions with them. We've had our end-of-phase II discussions. We've designed our program in consultation with them, and the high-level important points are it's 38 weeks to the accelerated approval analysis for proteinuria. The final analysis is for slope, the change in the slope of GFR, for making sure that the renal function stable is stabilized. And it's a fairly traditional approach for a homogeneous proteinuria kidney disease.
It's a very similar approach we've taken for our other phase III program in AMKD.
Yep. Is 30% the bar for that kind of proteinuria endpoint, is that fair?
Um-
I think there's some historical data out there on that.
Yeah, I think that that's a very fair assessment, but if you ask me what is our data at the last time that Alpine showed data, it was at the World Congress of Nephrology in about April. There. It's a small data set, but at, I don't know, 24 weeks or so, it was like a 67% reduction in proteinuria, and there's only, like, one or two people at the longer endpoint, but it was a 70%+ reduction in proteinuria.
Okay. Okay, great. Maybe just in the interest of time, we'll move over to CASGEVY. You know, you talked about the early momentum you've had here. Maybe just talk us through some of the remaining bottlenecks that you guys are working on here to continue that momentum. Again, I'm assuming part of it is the geographic rollout, but maybe just help us think through kind of, you know, rest of this year into next year.
Definitely. Charlie?
Yeah. I mean, it's exciting 'cause we're commercializing in North America, in Europe, and in the Middle East, all at the same time. We've commented that. I think in terms of authorized treatment centers, we're north of 30-35 treatment centers at this point, on our way to 75 or so total. And we should be well close to that goal by the end of the year. We've had cell collections from more than 20 patients. We've already dosed our first patient. The momentum is really strong, and the cell collections have been, again, across all of those geographies. So interestingly, with the ATCs activated, I think the term you used was bottlenecks. We're not really seeing any bottlenecks.
You know, we've got enough ATCs activated, more and more activated every day. We have no bottlenecks in manufacturing, none in our field organization, and importantly, real support from payers, and that's really significant, so whether it's commercial payers, government payers, inside the U.S., outside the U.S., really strong recognition for the seriousness of the disease, really strong recognition for the value of this therapy, and so we are really happy with the trajectory at this point. We've described 2024 as a foundational year. Again, it's better, in our view, to have an outstanding first year. These patients, given that we can address 80%+ of these patients in 75 ATCs, means that a lot of these patients know each other. They see the same physicians. They see each other in waiting rooms.
We really need to make sure that the first patients have the best experience, and we're all in and focused on that, because we think then momentum will build in a really significant way. So we're thrilled with where we are in 2024. We'll update on those stats again in the next earnings call, but we're well on our way to build a, you know, kind of a pipeline of patients who are advancing and setting us up for a nice year in 2025.
Okay, great. Any reason why, you know, a patient that had cells collected wouldn't ultimately transition to receive the product? I know it's a long process here, but are there any other things we need to think about, or is it, should we just assume that, you know, 100% of these people will ultimately go on to have the infused product?
Yeah. It's a really good question. The timeline is what you have to think about. So if the patient had cells collected, the cells were edited, and they're ready to be shipped back and received, sometimes a patient just decides that the last step, although they thought they wanted to go through it, the last step is the busulfan-
Yep
... conditioning step, and even though they went through the process, they had their cells collected, they thought they wanted to do this, they might decide, "You know what? I don't wanna do this." So I think it's idiosyncratic, patient-based factors, simply based on where they are at that time and their willingness to say, "Yes, I wanna go through the journey.
Yep. Okay. Any, just a financial one, as you think about the transition of this JV with CRISPR to profitability, have you guys given any rough bookends for when that might occur?
We've not yet. Again, just as a reminder, the economics are a 60/40 profit share, with Vertex 60%, CRISPR 40%. Importantly, we'll book 100% of the revenues.
Yep.
And then the profit share happens as you know they will essentially pay their share of R&D costs, and then there's a commercial margin that's calculated, and the profit share is based off of that. So no, we've not given any guidance on that specifically. But you know look it, we're in year one. There are some costs associated with getting a business like this off the ground. We're still conducting clinical trials in you know adolescents. We've invested in manufacturing capacity so that it is not a bottleneck. And as the patient volume ramps, those costs will get absorbed nicely. So again, I think importantly, we see this as a multi-billion dollar business and one that's gonna have attractive profitability as it scales.
Yep. Okay. Okay, great. I want to touch on VX-880. Again, Reshma-
Yeah
... you alluded to this, another, you know, very interesting pipeline program here, the potential in type 1 diabetes. The kind of three-pronged approach that you guys have taken, VX-880 is obviously the cell plus immunosuppressive approach. Sounds like we're gonna get some updated data at EASD.
Yep.
Is that correct?
That's exactly right.
So, maybe just, is this additional patients, additional follow-up? Just kind of remind us-
Yep
... level set, what, what we should be focused on here going into that, update.
Yep. So the VX-880 program is the first of three programs in our type 1 diabetes portfolio. This is allogeneic stem cell-derived pancreatic islet cells for the purpose of replacement and hopefully a curative therapy for these patients. Where we are with VX-880 is that we have fully enrolled and dosed the original cohort of 17 patients that makes up the phase II study as it was originally designed. We have now expanded that. We've spoken to the regulators, and we're thrilled to have expanded that by another 20 patients. The data you're gonna see at EASD. I think that's a September meeting, so later this month-
Yep
... is data on more patients who have longer durations of follow-up than the last cut of the data, which was at ADA in around June, and where we are, what we're looking to do with the VX-880 program now is complete our discussions with the regulators and plan forward for phase III. The next program after that is VX-264. It's those same cells, which we already know work, encapsulated in a device, so as to obviate the need for immunosuppression. And the third program, that program is in phase I/II, it's in the clinic. The third program is a program that's in preclinical development, so it's in the lab, and what we're trying to do there is a second way to avoid immunosuppression.
And what we do is we are working to make certain gene edits so that the cells are hidden, if you will, from the immune system.
What? How do you feel about durability for VX-880 now that we're getting now? I mean, I think this is gonna be, what? Well past 18 months.
Yeah, certainly some patients are, are at that point. This is a first in man study. No one else has been successful, so we, we don't have any analogues to look at in terms of allogeneic stem cells. But what I can tell you is we fashioned our program after what's called CIT-007. It's donor cadaveric donor islets. And when you look at that program, there are people who are out years, five, 10, 15, 20 years, and they're doing really well with their transplant. The problem with cadaveric islets is quantity and quality of cells. Quantity, i.e., we don't have enough cadavers, and quality, because the pancreas, in addition to being an endocrine organ, is also an organ that releases enzymes for digestion. So trying to isolate islets is a difficult thing.
Our goal here is, you know, no doubt about it. Our goal is to have a functional cure for type 1 diabetes. It also turns out to be the case, and we learned this from the cadaveric islet experience, if you need a second dose, it's doable. The problem with cadaverics is they don't have enough dose. You can't even get enough dose for the first dose. In our instance, if you need, I hate the term, but people call it a top off, it's completely fine.
We make these cells in our lab. We have as many as you want.
But our goal is to transform this disease and to deliver a functional cure.
Okay, great. Well, I think we're up against time, but Reshma and Charlie, really appreciate you joining us today. Thank you.
Thank you.
Thank you, Terence.
Thank you.