Vertex Pharmaceuticals Incorporated (VRTX) 41st Annual J.P. Morgan Healthcare Conference – (Transcript)

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) 41st Annual J.P. Morgan Healthcare Conference Call January 9, 2023 11:15 AM ET

Company Participants

Reshma Kewalramani – Chief Executive Officer and President

Charlie Wagner – Chief Financial Officer

Stuart Arbuckle – Chief Operating Officer

David Altshuler – Executive Vice President, Global Research, and Chief Scientific Officer

Conference Call Participants

Jessica Fye – J.P. Morgan

Jessica Fye

Great. Good morning, everyone. Welcome. I’m Jessica Fye. I’m a Large Cap Biotechnology Analyst at J.P. Morgan, and we’re delighted to be continuing the conference this morning with Vertex. There’s going to be a Q&A session right in this room after the company’s presentation, so no need to switch rooms. There’s going to be mic runners. So, if you have a question, you want to raise your hand, you can do that. Alternatively, you can use the portal to fire me questions to an iPad up here, and I can ask them for you, or you can just listen to my questions.

Without any further ado, let me pass it over to the CEO of Vertex, Reshma Kewalramani for the presentation.

Reshma Kewalramani

Well, good morning, everybody. Just thank you so much and thank you to J.P. Morgan for hosting this. It is nice to be back in San Francisco, in-person, and to do this live. Some housekeeping notes to start with. This slide has our Safe Harbor statement listed. In brief, let me remind you that the forward-looking statements made during this presentation are subject to risks and uncertainties that are discussed in our SEC filings, and I encourage you to read the filings, actual events, and outcomes may differ materially.

With that, let’s get on with it. At its core, Vertex’s strategy is to invest in scientific innovation in order to create transformative medicines for people with serious diseases, with a focus on specialty markets. Underlying this corporate strategy is an R&D approach, which is designed to yield disproportionate success. This approach has fueled our achievements in CF, generated a broad, advanced, and diverse portfolio, delivered multiple programs with near-term launch potential, and built a company with a strong financial profile, talent base, and culture that is going to drive long-term success.

The results of this approach are depicted to the right of the slide. We have successfully launched four medicines in cystic fibrosis and our [2022] [ph] guidance is between $8.8 billion and $8.9 billion. We have near-term commercial opportunities in four disease areas: Exa-cel, previously known as CTX001 in sickle cell disease and beta thalassemia, VX-548 in acute pain, and the vanzacaftor triple combination in cystic fibrosis.

And as a marker for R&D productivity of the six disease areas outside of CF, in which we entered Phase 2 development in the recent past 5 of the 6 programs have yielded positive proof of concept results. This includes sickle cell disease and beta thalassemia, as well as acute pain that I’ve already spoken about, but it also includes APOL1-mediated kidney disease or AMKD, as well as type 1 diabetes with our cell-based program.

We also have a Phase 2 study underway with VX-548 in neuropathic pain and another Phase 2 study with longer duration of treatment with VX-864 in alpha-1 antitrypsin deficiency. In total, this makes 8 programs in mid and late stage development all of which I believe have a high likelihood of success. This slide lists the 8 programs that are in mid and late stage development.

Each asset presents an opportunity for a transformative medicine and each opportunity represents a multibillion dollar market. This vision, this broad potential of our clinical stage pipeline is not a distant vision. This is right in front of us. We have the opportunity to bring new products to 5 disease areas in the next 5 years, our 5 and 5 goal.

Let me start with CF, and let me start with the numbers. We are updating the epidemiology in CF, in the U.S., Europe, Australia, and Canada to 88,000 patients, up from 83,000 patients in 2021. The growth in this population comes really from three places. First, there are additional patients coming forward to receive treatment given the overall improvement in CF care. Second, registries are capturing data better around the globe. And third, and maybe most importantly, CF patients are living longer.

Our medicines are delivering meaningful long-term benefit. For example, we recently showed that there is a 70% plus improvement in lung transplantation and mortality with people who are taking CFTR modulators. Notably, what this means is for a baby born in 2021. The mean predicted survival for this child is 65 years of age. Just a few decades ago, that number was in the mid-30s.

Our goal, to be clear, is to have all patients who could benefit from CFTR modulators on our therapy. And we see continued significant growth in CF as there are more than 20,000 patients who are eligible for CFTR modulators or could benefit, but are not currently on therapy. These patients largely fall into two categories. First, where we have recently secured reimbursement and we are early in the launch curve; and second, younger age groups.

For these younger age group patients, we continue to study our medicines in younger and younger patients. I’m pleased to let you know that our TRIKAFTA, 2 to 5 year old submission, has been accepted for priority review in the U.S. with the PDUFA date of April 28. Our next-in-class, vanzacaftor triple has completed its 52-week, I should say, has completed its enrollment, and the 52-week dosing period continues.

We now project that this study, the vanzacaftor triple combination Phase 3 studies will complete by the end of this year. We have high expectations for this program and we look forward to the data readout, but we’re not done. We have already identified additional potentiators and correctors in the lab, and we are already advancing them into the clinic.

Lastly, there are about 5,000 CF patients who cannot benefit from CFTR modulators because they simply don’t make sufficient protein. For these patients, our mRNA approach or VX-522 may be appropriate. This molecule VX-522 is a result of our partnership with Moderna and it has been cleared to enter the clinic. It was also recently granted Fast Track designation by the FDA. The initiation of this trial, which has already occurred is an enormous milestone. It is our first mRNA therapy to enter clinical trials and brings hope to the remaining CF patients who are waiting for a drug to treat the underlying cause of their disease.

Let me move from CF to the pipeline and let me start with Exa-cel for sickle cell disease and beta thalassemia. This is our next near-term commercial opportunity. Exa-cel holds transformative potential for patients and is poised to become the first approved gene editing therapy for any disease. Sickle cell disease and beta thalassemia are a significant burden to patients, to families, and to the healthcare system.

In the U.S., economic analyses have predicted and modeled that the lifetime costs of sickle cell disease – severe sickle cell disease is between $4 million and $6 million over a lifetime. Exa-cel is a one-time autologous ex-vivo gene editing investigational therapy in which a patient’s own cells are collected. Using CRISPR/Cas9, these cells are precisely and durably edited at the BCL11A locus. And then these edited cells are transfused back into the patient.

The clinical benefits, they are evident from the data that we’ve shown to date. And we’re excited to bring this therapy that holds the potential for a one-time functional cure to patients who have been historically underserved. I’m going to move on to our commercial preparedness, but I do want to give you an important update on our regulatory status with Exa-cel.

I’m pleased to let you know that Exa-cel has been filed in the EU and the UK for both sickle cell disease and beta thalassemia as of last December. You know that we’ve initiated the rolling submission in the U.S. and we remain on-track to complete that submission later this quarter. We are making strong progress in our commercial readiness to launch Exa-cel. Our commercial and medical affairs teams are hired and trained and we have established the infrastructure to support physicians and patients through the treatment journey.

Our initial launch of Exa-cel is going to focus on the 32,000 most severe sickle cell patients and beta thalassemia patients out of the universe of approximately 150,000 sickle cell and beta thal patients in the U.S. and EU. We believe these 32,000 patients can be efficiently served with about 50 authorized treatment centers in the U.S. and about 25 authorized treatment centers in the EU because the geography of these patients both in the U.S. and outside the U.S. are concentrated into specific geographies.

In parallel engaging with the treatment centers, we’ve also been working with payers, as well as policymakers to ensure that these stakeholders understand the significant burden of this disease and to ensure patients have access and reimbursement to this therapy if and when Exa-cel is approved.

Let me move on now from Exa-cel to the pain program and I’ll focus my comments on acute pain with VX-548. There is a staggering unmet need in acute pain. The toolkit for the management of acute pain has remained largely unchanged for decades. VX-548 is a NAV 1.8 inhibitor, and if approved would represent the first new class of medicines with the potential to treat this high unmet need.

It’s important to know VX-548 acts on the peripheral nerves. It blocks the pain signal there. And thus, it may be able to provide effective pain relief without abuse potential. Abuse potential is a central nervous system phenomenon. The NAV 1.8 target is both genetically validated and pharmacologically validated. We ourselves have completed successfully 5 Phase 2 proof of concept studies with VX-150, the predecessor molecule to 548 and with VX-548 itself.

The pivotal development for 548 in acute pain initiated last year. We’re currently enrolling two randomized controlled trials and one single arm study. With a goal of seeking a broad moderate to severe acute pain label. I’m pleased to share that we now anticipate that the VX-548 pivotal development will complete towards the tail end of this year or the beginning of next. And with that, we see the opportunity for a potentially near-term and significant potential to support patients and launch yet another molecule.

Let me talk a little bit more about the treatment gap that exists. The current standard of care for acute pain has NSAIDs and acetaminophen on the one end of the treatment landscape and opioids on the other. While NSAIDs and acetaminophen have pain relief, the relief level is limited, and there are concerns with GI and liver toxicity.

Opioids on the other hand are effective pain medicines, but they have important side effects, including somnolence, dizziness, nausea, and most importantly, abuse potential. This leaves a gap in the treatment landscape for medicines that can be used for effective pain relief with a desirable benefit risk profile without abuse potential. VX-548 showed strong efficacy in the Phase 2 program, an attractive benefit risk profile, and by way of its mechanism of action, that’s to say peripherally acting, lacks abuse potential.

VX-548 has been granted both fast track and breakthrough therapy designation indicative of FDA’s recognition of the high unmet need and the compelling clinical profile to date. Let me put some numbers around this opportunity. Today in the U.S. alone, there are approximately 1.5 billion treatment days of an acute pain medicine prescribed annually. Even though 90% of these prescriptions are generic, currently, this is a $4 billion market.

With respect to the settings of care and where these prescriptions are written, approximately two-thirds or 1 billion of these prescriptions are driven by hospital or institutional prescribing. These are concentrated in about 1,700 hospitals and about 200 IDNs or integrated delivery networks. In other words, it’s concentrated, and we believe we can approach this opportunity with a specialty sales force.

In addition, we see both high demand and a clear path to access and reimbursement. Let me give you some examples for why I say that. Almost all institutions and all 50 states have some guidelines in place regard to opioid prescriptions. Most recently, the NOPAIN Act signed into law on December 29 of last year, directs CMS to make a separate add-on payment to hospital outpatient settings and to ambulatory surgical care settings for non-opioid medicines.

This new law is an example of the growing movement to empower hospitals, patients, providers, to utilize non-opioid treatments. And we are excited for VX-548 to be a potentially near-term effective non-opioid treatment for patients who are waiting.

Let me move from pain to Inaxaplin or VX-147. VX-147 has the potential to treat the underlying cause of APOL1-mediated kidney disease. This genetic disease was just recently defined in 2010. This is a disease that relentlessly and rapidly progresses to dialysis, transplantation, or death, and there are no approved treatments for this disease.

We’ve already shared that in our 13-week Phase 2 study, we demonstrated a 47.6% reduction in proteinuria. That is an unprecedented result in FSGS, let alone in APOL1-mediated kidney disease. We are now in a Phase 2/3 pivotal trial with Inaxaplin with a pathway established for accelerated approval if the data are supportive.

Given that AMKD has only recently been defined, and it is a relatively asymptomatic disease until late in its course, diagnosis rates are low. We are addressing that with multiple initiatives, including one with Alonzo Mourning, the basketball Hall-of-Famer, who himself suffered from AMKD until his transplantation. And our goal here is to improve awareness, diagnosis, as well as screening.

In short, AMKD affects about a 100,000 people in the U.S. and EU. With Inaxaplin, we see the potential of bringing a first-in-class treatment that could transform the underlying cause of this disease. And thereby unlocking a big market opportunity. Let me move from Inaxaplin to Type 1 diabetes. This slide shows you a trio of our programs that are all grounded in the VX-880 cells. These are the naked cells. This VX-880 program has already demonstrated proof of concept with the first two patients dosed at half the target dose.

What I mean by that is, we’ve demonstrated that patients have improvements in hemoglobin A1C, elevations in C-peptide, and decreases in exogenous insulin need. In fact, for patient number 1 treated at half the targeted dose, there was a complete elimination of exogenous insulin. As I mentioned, all three of our programs in this disease area rely on the foundational VX-880 cells.

The VX-880 program itself is now in Part B. That’s where we dose with the full targeted dose. This part of the study is now fully enrolled. And we expect to be able to share more data and longer-term data from these patients this year at upcoming medical congresses. I should note that the next portion of the study Part C, is the part of the study where we are able to dose at full targeted dose without a stagger. Part B has a dosing stagger between each patient.

Moving to the middle panel, the VX-264 program. The VX-264 program is the cells, the same VX-880 cells, this time cloaked in a device so as to evade the immune system. Whereas VX-880 uses standard immunosuppressives, this program does not have a need for immunosuppressants. Last year, we simultaneously filed a CTA in Canada, as well as the IND in the U.S. I’m very pleased to share that the CTA has cleared and we look forward to initiating the enrollment and dosing of the VX-264 trial in Canada in the coming months.

In the U.S., the IND has not cleared and the program is therefore on-hold until we receive the FDA’s questions and satisfactorily address them. On the last panel is our hypoimmune cells. In this program, we’re editing the VX-880 cells, and that will cloak the cells from the immune system. That’s our second approach, the device being the first, to potentially eliminate the need for immunosuppressants. This program continues to make nice progress through preclinical development. This is a top line view of our pipeline with select preclinical assets, as well as our clinical stage program.

I’ll make three points on the clinical stage program. It’s broad, diverse, and advanced. It reflects four modalities in play: MRNA, cell therapy, gene editing, and small molecules. And it also reflects a nice balance between internal and external innovation with 40% of the pipeline coming from BD activities. A couple of comments on the select preclinical assets on this slide. The next wave of innovation is right around the corner. And I’ll call out the DMD program here, which would be our first in vivo gene editing program for which we expect to file the IND later this year.

The improved conditioning program, which would vastly expand the number of patients eligible for Exa-cel and the NaV1.7 program, another pain program that alone or in combination with VX-548 would expand our leadership position there. A moment on our financial profile as I’ve already told you, we guided for 2022 8.8 billion to 8.9 billion in revenue, and we’ll be giving you actuals on February 7 along with 2023 guidance on our earnings call.

In the middle panel, you see a select few of our external innovation investments that have led to this pipeline that I showed you on the previous page. And with our business model, with the lean SG&A spend, large treatment effects, you see that we continue to provide excellent operating margins at the top end of our peer group. I’m going to leave the slide for you to review, but in essence, 2022, we had a very good year across the board that sets ourselves up for a very important 2023 with multiple catalysts.

And I will end with this slide. You could use this as a marker, a report card, for our advancement and progress in the CF business and through the pipeline for 2023 and beyond. We seek to continue our journey in CF, prepare for near-term commercial launches, drive and accelerate our pipeline forward, including our goal of five launches in the next five years and continue to deliver financial performance.

Thank you very much, and I’ll turn it over to Jessica.

Question-and-Answer Session

Q – Jessica Fye

Great. I think some of the other members of the management team are going to come up for Q&A as well. And again, if you have a question, you can raise your hand, otherwise, you can submit it electronically as well, but I’ll start. Let me just cut right to the chase here. So, your company is generating plenty of cash. You’ve got a nice cash balance. How much of a priority is returning cash at this juncture versus reinvesting in the business or anything else?

Reshma Kewalramani

Yeah. Charlie, do you want to take that one?

Charlie Wagner

Yeah, sure. Listen, we know that the greatest way for us to create value is to develop transformative medicines serious diseases. And therefore, innovation continues to be our top priority. For capital allocation, we’ve been active investing both internally and externally over the last several years. And I think we’ve shown that those investments have been wise, you see the advancement in the pipeline, you see the level of success that we’ve had. And importantly on the BD side, a number of the programs 40% have benefited from BD that we’ve done in recent years.

So, looking ahead, investment in innovation both internally and externally will continue to be the priority. We have also maintained a share buyback program over recent years. We’ve purchased about $1.5 billion of shares in the last two years and you can back that we’ll continue to have a repurchase program going forward.

Jessica Fye

Great. There’s one that came in on the portal so far. So, have you begun price negotiations with payers in Europe for Exa-cel or when can that happen? And it’s sort of alluding to bluebird’s negotiations in Germany, I think, how do you expect it to go for Exa-cel?

Reshma Kewalramani

Sure. So, I’m going to ask Stuart to address that. Obviously, we’re at the stage now where we’ve completed the filings for sickle and beta thal in the EU and UK. And Stuart can tell you a little bit about where we are in the international region and in the U.S.

Stuart Arbuckle

Yes, in both regions, we have both our commercial and medical affairs teams up and running and they’re already engaging with payers, policymakers, and indeed potential authorized treatment centers. We haven’t started talking about specific pricing negotiations. It’s much too early to do that, but we have been talking with payers and policymakers about the unmet need, which I have to tell you is well understood in Europe, the unmet need for new products in this particular area.

We’ve also been talking with them about the potential mechanisms and payment models because pricing is one thing, payment model is another thing for these one-time functional [cures] [ph] as this is a – obviously a relatively new development across the industry. What I will tell you about that is, based on our work in CF and our initial discussions with payers there, I think it’s very clear that from a payment model point of view, one size is not going to fit all.

And that is a big part of the discussions that we’re having, what is the needs of the individual countries and therefore, what is the right payment model for them. And I think our experience in CF is going to stand us in good stead there. We’ve been very creative, very flexible in working with countries and that’s led to our success in negotiating many, many reimbursement agreements for CF. And I think that’s a capability that we’re going to be able to utilize to help us be successful in – with Exa-cel as well.

Jessica Fye

Okay. One question in the audience. Could you get a mic over there?

Unidentified Analyst

Hi. I was just curious about the stem cell therapy in diabetes, and what that might cost individual patients? If it’s cost prohibitive, if you could provide any color on that? Thank you.

Reshma Kewalramani

Yes. I’ll ask Stuart to tackle that. I’ll start by sharing that there are islet cell transplants available, cadaveric islet cell transplants, and whole pancreas transplants, and that can give you a sense for [a way to] [ph] start. Stuart?

Stuart Arbuckle

Yes, again, I mean, it’s obviously much too early to be talking about specific pricing ranges. We’re absolutely thrilled with the progress of the program to be able to declare proof of concept for the VX-880 program, the naked cells, just based on two patients is truly amazing. As Reshma said, what we’re looking to do here is develop a one-time functional cure and a one-time functional cure over time that we can cloak from the immune system so that we don’t need immunosuppression as well.

So that is the promise of what we’re doing here. Clearly, the cost of type 1 diabetes is very, very high. These patients are very intensively managed. The cost of cell therapies, as Reshma said, the cost for cadaveric cell transplants are well-known. The issue with those is really not price prohibitive, it really is quantity and quality of cells prohibitive.

And that’s what we are seeking to fix by developing fully mature, fully differentiated, fully functioning, glucose sensing, [insulating, secreting] [ph], islet cells, but being able to do that in industrial quantities at very, very high quality. So, cost prohibition is [not what hold back] [ph] cadaveric islet cell transplant, it’s really quality and quantity of cells and that’s what we’re seeking to fix.

Jessica Fye

On the pivotal program in pain, you talked about that completing as soon as the end of this year potentially. Should we expect to start hearing data by the end of this year? Is that on the table or is that, sort of when the trial completes and then we would hear something maybe in 2024? And then second, there’s multiple trials here, should we expect this to come as sort of one update or could they come sort of in sequence?

Reshma Kewalramani

Yes. So just to ground everyone, the pivotal program for VX-548 in acute pain is comprised of three studies. There’s one RCT, randomized clinical trial in bunionectomy, one in abdominoplasty as a model for soft tissue and a model for hard tissue pain, and then a single-arm study with pain of any variety. So, that’s what comprises the Phase 3 program. The two RCTs are very, very similar to the RCTs we’ve already completed in Phase II. They’re really the same models. They’re the same endpoints, and it’s the same duration of therapy, 48 hours.

We are sharing that we expect the completion of the trial late this year or early next. After that, it takes a little bit of time to bring in the data to analyze it and to share it, but we move pretty fast. And I do expect that the update will be a singular update for the entire program.

Jessica Fye

You also talked in the presentation about some updated [epi data] [ph], can you walk-through, kind of the reasons why you’re able to update it and why you see more patients?

Reshma Kewalramani

Yes, Stuart, can I ask you to take that one?

Stuart Arbuckle

Yes. So, the way we collect the epi is largely working with countries around the world that have very well-established registries. And what we’ve seen over the last few years is increasing numbers of patients reported in those registries and still active and alive in those registries. And really what’s driving that increase is a couple of things. The first one is, more patients coming forward to be looked at in registries, and we believe that is largely as a result of more patients, kind of returning to be treated as a result of highly effective therapies.

In addition to that, the registries have continued to develop and expand and so there’s better data capture in those registries. And thirdly, and perhaps most importantly is the increase in survival that Reshma mentioned that we’ve seen, which has been going on for a number of years now. That increase in survival is something that we expect to continue as more and more people are treated with CFTR modulators over time.

And so, we see this being a trend which is going to continue. And just to dimensionalize it, back in 2021 at J.P. Morgan, we updated our epidemiology from 75,000 patients in North America, Europe, and Australia to 83,000. This year, we’re updating that for those same geographies to 88,000 patients who are living with CF today.

Jessica Fye

For your Phase 2/3 study in kidney disease, can you talk about how enrollments going there? What progress you’re making on sort of identifying the patients to, kind of put into that study?

Reshma Kewalramani

Yeah, this study in comparison to something like the VX-548 acute pain study, we always knew would have a different enrollment trajectory, and it would take a little bit of time because this disease itself was just defined in 2010. There is not widespread screening or diagnosis. And the disease itself is actually rather asymptomatic until the late stages, so you don’t even know you have it.

We are working to ensure that enrollment is efficient by having a concurrently run genotyping study. And if you genotype in, you can then enroll in the randomized clinical trial We’re opening many, many sites, so there are sites close to where our patients are. And we’re embarking on campaigns like the one with Alonzo Mourning to ensure that patients know to look for this disease asked for testing and such. Where we are today is that we are expecting the Phase 2 portion of the Phase 2/3 pivotal trial to complete this year.

Jessica Fye

So, we’re also expecting pivotal data for the new triple coming up. Can you talk about you know, what you want to see from that, you know, it seems like TRIKAFTA sets a pretty high bar here, so maybe also talk about what your level of confidence in and being able to – isn’t being able to beat TRIKAFTA?

Reshma Kewalramani

Yeah. I’ll ask David to take that one. Our confidence in the vanzacaftor triple is very high, and David can speak to why we feel that way. David?

David Altshuler

Yes. No, it’s true that TRIKAFTA has a remarkable profile, and so it is very difficult to do better, but if there’s any medicine we think can do better, it’s the vanzacaftor triple therapy. And the reasons for that are two-fold. One, we have evidence from our HPE assay, human bronchial epithelial assay, running our labs in San Diego. That assay as we run it has proven quantitatively to translate from the lab two levels of CFTR function in patients repeatedly for all four medicines in multiple others in clinical trials. And the vanzacaftor triple at clinical exposures is better in the HPE assay, drives higher levels of chloride transport, then does TRIKAFTA. That’s one laboratory data.

The second is, Phase 2 data. We did a Phase 2 trial of the vanzacaftor triple, and we focus here on sweat chloride because sweat chloride is a direct measure of CFTR function, but also it’s more tightly measured in a small study as you get a more accurate measure in the small Phase 2 study than you can of FEV1, which is a more variable measure. In that Phase 2 data, the sweat chloride we have obtained when compared through PKPD modeling cross-study comparison to previous studies with TRIKAFTA clearly shows higher levels of CFTR function.

So, the combination of laboratory data and our Phase 2 data tells us that the vanzacaftor triple has a high likelihood in Phase 3 of [driving] [ph] higher levels of CFTR function, and we know from natural history and our own clinical trials, it’s higher levels of CFTR function that leads ultimately to patient benefit.

Jessica Fye

Is there anything you can say about how long the IP for the vanzacaftor triple might extend beyond the TRIKAFTA IP?

Reshma Kewalramani

We haven’t shared the IP protection for vanza, but what I will tell you is that the TRIKAFTA protection runs all the way out to 2037, so you could expect it to be longer than that.

Jessica Fye

And I know we’re talking about how hard it is to beat TRIKAFTA, but sometimes we get questions about other products and development for CF. So, can you talk a little bit about what you’re watching for competitively in the CF landscape and the extent to which you think your franchise is defensible?

Reshma Kewalramani

Yes. We look at everything in CF as you would imagine. And as I think about the competitive landscape, the nearest term and most substantial competitor to TRIKAFTA is vanzacaftor and that triple combination, and that study will end by the end of this year.

Unidentified Analyst

Just a follow-up on the 880 program, you said that you’re looking for a cure, so what is the bar for the data for that to be the modality that you move forward as opposed to the other two?

Reshma Kewalramani

Yeah. The three programs are both related to each other. And we have somewhat different goals for them. So, if you [wonder about] [ph] why do you have three programs? The most important thing to understand is that the foundation for each of these programs is the VX880 cells. There are two things you’re trying to do when you’re working with cell therapy for type 1 diabetes. One, have cells that are fully differentiated, insulin producing, self-regulated. You need that. And the second thing you need, and it’s in this order. The second thing you need is to cloak those cells from the immune system.

So, the big breakthrough here is that we already know that our VX880 cells work because at half the dose we were able to show proof of concept. That program will use standard immunosuppressives, and what we’re doing with the device program and the gene editing program is two alternative ways to cloak those cells so that you don’t have to use immunosuppression. But as I think about it, that’s the secondary issue.

The primary issue in the big breakthrough is having cells that are fully differentiated, insulin producing, and can regulate glucose. The data that we’ve already shared and you could look through, it goes through C-peptide and hemoglobin A1C and in exogenous insulin, etcetera, gives you that confidence in those cells.

Okay. And we are out of time, so we’re going to wrap it up there. Thank you, everyone.

Charlie Wagner

Thanks, Jess.

Reshma Kewalramani

Thanks, Jess.