{"id":22279,"date":"2023-06-13T22:42:37","date_gmt":"2023-06-14T02:42:37","guid":{"rendered":"https:\/\/ifintechworld.com\/news\/vertex-pharmaceuticals-incorporated-vrtx-goldman-sachs-44th-annual-global-healthcare-conference-transcript\/"},"modified":"2023-06-13T22:42:37","modified_gmt":"2023-06-14T02:42:37","slug":"vertex-pharmaceuticals-incorporated-vrtx-goldman-sachs-44th-annual-global-healthcare-conference-transcript","status":"publish","type":"post","link":"https:\/\/ifintechworld.com\/?p=22279","title":{"rendered":"Vertex Pharmaceuticals Incorporated (VRTX) Goldman Sachs 44th Annual Global Healthcare Conference (Transcript)"},"content":{"rendered":"
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Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX<\/span>) Goldman Sachs 44th Annual Global Healthcare Conference Call June 13, 2023 5:40 PM ET<\/p>\n

Company Participants<\/strong><\/p>\n

Reshma Kewalramani – President and Chief Executive Officer<\/p>\n

Stuart Arbuckle – Chief Operating Officer<\/p>\n

Conference Call Participants<\/strong><\/p>\n

Salveen Richter – Goldman Sachs<\/p>\n

Salveen Richter<\/strong><\/p>\n

Great. Good afternoon, everyone. Thanks for joining us. I am Salveen Richter, I cover the biotechnology sector at Goldman Sachs. And we are really pleased to have Vertex team with us today. So we have Reshma Kewalramani, who is the CEO and the President, as well as Stuart Arbuckle, who is the Chief Operating Officer of the company. Thank you both for joining us.<\/p>\n

Question-and-Answer Session<\/strong><\/p>\n

Q – Salveen Richter<\/strong><\/p>\n

To start here, maybe we could give a snapshot of the business where it stands today, what we should anticipate in terms of strategy, commercial execution and the pipeline as we look towards the second half of the year and beyond?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Sounds great. Well, first of all, it\u2019s very nice to see all of you in person as we are still all getting back to in-person meetings. Let me say a few words about where we are at the highest levels, and I\u2019ll ask Stuart to comment on where we are in terms of the CF franchise. The first thing to say is that we have had a really great start to this year. And as we have been talking about for the last 6 to 18 months, we\u2019re at an inflection point for the company. The CF franchise, which Stuart will talk about, has been going exceptionally well as we reach more and more patients with our medicines. Next in line for CF is the vanzacaftor triple combination, let\u2019s call it the next-generation CFTR modulator regimen, which we expect towards the end of this year, beginning of next. And also in CF, we\u2019re expecting to see results from our mRNA program with our partners at Moderna in the next year, shall we say. That program, VX-522, is in its Phase 1\/2 study in the last 10% of patients with cystic fibrosis. Those are those patients who make no CFTR protein.<\/span><\/span><\/p>\n

On the R&D pipeline side, we now have 8 disease areas in the clinic, 5 of which are beyond Phase 2, and we\u2019ll certainly have time to talk about all of those. But maybe the important point to make is we are now on the brink of launching five disease areas in the next 5 years. So that is sickle cell disease and beta thalassemia with exa-cel as well as vanzacaftor in CF and VX-548 in acute pain. And for the fifth one, it\u2019s a bit of dealer\u2019s choice. You can think about 548 in neuropathic pain, the type 1 diabetes program, AATD, the inaxaplin program in kidney disease as some examples for what that \u2013 what could round out those 5 and 5. And on the finance side, our balance sheet, as you know, is very strong, and we feel really good about how we\u2019ve invested our capital in terms of investing in innovation, something you can expect to continue with modest share buybacks that we have also implemented over the last 18, 24 months. Stuart, I\u2019ll turn it over to you for a little bit more color on CF.<\/p>\n

Stuart Arbuckle<\/span><\/strong><\/p>\n

Yes. Obviously, CF as a franchise has delivered incredible growth for the company over the last decade. As we entered 2023, we updated our estimates for the epidemiology of cystic fibrosis to 88,000 people living with cystic fibrosis in North America, Europe and Australasia. And of those 88,000, about 20,000 or so, as we entered the year, were not being treated with one of our CFTR modulators. They asked the question why? Really, it\u2019s one of four reasons. The first one is that we are regulatorily approved. We also have reimbursement, but we are just early in the launch curve. So I think of countries like Australia and New Zealand. Secondly, there are a diminishingly small number of countries where we don\u2019t yet have reimbursement, which we\u2019re continuing to work on. And thirdly, we\u2019re continuing to expand our labels to younger and younger patient groups in the U.S. We now have approval for TRIKAFTA down to 2 to 5-year-olds or ORKAMBI to 2-year-olds and KALYDECO now amazingly down to children between 1 month and 4 months of age, and we are continuing to get those label expansions around the globe. So those are three reasons why we see continued growth in cystic fibrosis with our existing medicines. The last group of patients in that 20,000, is about 6,000 or so patients around the world who have discontinued one of our CFTR modulators. We think they are going to be great candidates for vanzacaftor, our next-generation triple combination which, as Reshma said, is deep into its Phase 3 program. We expect to see the results end of this year, beginning of next. The last area of growth for our cystic fibrosis franchise is getting to those 7%, about 5,000 or so patients who won\u2019t respond to our CFTR modulators, because they produce no CFTR protein at all. They are going to be good candidates for our mRNA therapy, which has just begun its SAD program. This is the VX-522 program in partnership with mRNA. So we\u2019ve seen incredible growth in CF over the last decade. We expect to see continued growth for our CF franchise for many years to come.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

Maybe diving into vanzacaftor here, so we are going to see data, I believe by year end. What do you need to see to know that this is \u2013 or why do you believe that this is superior to what you\u2019re seeing with TRIKAFTA and where do you think this will be then positioned within the marketplace?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Yes, let\u2019s tag team that one. Let me tell you why we believe that vanzacaftor has the chance to be even better than TRIKAFTA. And I know that\u2019s a tall order, and that\u2019s a big statement. There are really two lines of evidence that make us think that, that is possible. The first is our HBE assays. These are the human bronchial epithelial cell assays that have been predictive not only qualitatively, but quantitatively so for KALYDECO, ORKAMBI, SYMDEKO, TRIKAFTA and the other medicines that we advanced through late stages of development but chose not to commercialize because we selected TRIKAFTA. Those HBE assays tell us that the vanzacaftor triple is even better than TRIKAFTA. So that\u2019s one line of evidence. And the second line of evidence comes from our Phase 2 program. We haven\u2019t studied every combination that you need to, to make direct comparisons. But if you look at the totality of our Phase 2 data, in het\/min patients or the FMF patients, as they\u2019re called, or the FF patients and you look across those, some studies which had a TRIKAFTA arm, the vanza triple is better than TRIKAFTA consistently so in terms of sweat chloride, and that\u2019s important because that\u2019s the direct readout of CFTR function. In certain studies, you can even see hints of it in terms of ppFEV1, but that\u2019s harder because the sample size in Phase 2 is small and the variability of ppFEV1 is high. But those are really the two lines of evidence that give us confidence that vanzacaftor has the potential to be even better than TRIKAFTA. Stuart, a little bit on commercial potential?<\/p>\n

Stuart Arbuckle<\/span><\/strong><\/p>\n

Yes. From a commercial potential, there\u2019s really kind of two separate populations. There is those patients who were already established on a CFTR modulator, the vast majority of which now are on TRIKAFTA. If we have a product that has the clinical profile that Reshma is just described that there is a sort of upgrade even on TRIKAFTA and it\u2019s also once a day, which is going to be more convenient for the patients, we think there\u2019s going to be a lot of interest from patients and physicians to switch from TRIKAFTA to vanzacaftor and then as importantly is that discontinued population. These are patients who have already demonstrated they would like to be on a CFTR modulator, but for a variety of reasons, have not been able to stay on a CFTR modulator. As I said, there is about 6,000 of those patients today. And we think they are going to be very good candidates for vanzacaftor, because it will be a new potential treatment option for them. So we think it\u2019s going to have great potential in both of those segments of the market.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

When we think about the future of your portfolio in the context of competition, are the assays at this point just providing this, I guess, this level of understanding as well as allowing you to get \u2013 given your portfolio these sweat chloride levels that it\u2019s going to be hard for any competitor to kind of step in and show superiority over. I mean, how should we think about that?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Yes. The assays, these HBE assays are certainly one of the absolute key to our success, but it\u2019s not the only thing. So when you think about cystic fibrosis today, it is a world of triple and the standard of care is TRIKAFTA with a fairly significant improvement in ppFEV1 in the clinical trial. That\u2019s the regulatory enabling end point, a really good-looking benefit-risk profile. And if you are going to compete with TRIKAFTA, you have to go head-to-head versus TRIKAFTA, which as we\u2019ve already discussed is a tall order. Now if you work backwards from there, if you say, okay, understood that in the clinical trial scenario, you have to go head-to-head versus TRIKAFTA, you have to be able to compete there. How do you do that? Well, first, it\u2019s a world of triples. To actually get two correctors and a potentiator that work together and have the right drug-like properties, that\u2019s a tough, tough thing to do. Then there is the assays where you can study these in a high throughput fashion, so you can pick the right combination to bring into the clinic. That\u2019s a hard thing to do. And the last thing I would say is for patients who are on CFTR modulators, as Stuart expressed, we are now down to age 2 plus in the U.S. with TRIKAFTA. These are patients who are now taking medicines in the instance of KALYDECO from the age of 1 month, in the case of TRIKAFTA since the age of 2. It\u2019s very, very difficult to believe that you could simply come in with a new medicine and have all of these patients move over. The way you do the drug development in this disease state is you start at 12 plus. So all the 1-month-old and the 2-year-olds and the 5-year-olds, they\u2019re going to be on the medicines like TRIKAFTA or vanza and moving all of those patients to another medicine. That\u2019s a tall order. And the HBE assays are absolutely critical, but there is all of this additional context to be had as well.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

Okay. And when you look at the mRNA partner program you have with Moderna, what would proof-of-concept look like there when you finally see that data set in those ineligible patients?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Yes. So for the last 10%, who simply don\u2019t make protein, what we\u2019re doing is a Phase 1\/2 study, so it\u2019s already in CF patients. The readout is not going to be sweat chloride because this is a nebulized therapy. So it\u2019s a direct delivery to the lung. We expect to see changes in ppFEV1 very similar to what we\u2019ve done with our CFTR modulators. I expect that, that\u2019s going to happen when we get to the MAD portion of the study. We\u2019re only in the single ascending dose portion now. And I expect that the readout will be changes in ppFEV1 very, very similar to what you\u2019ve seen across the CFTR modulator portfolio.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

Great. So maybe switching to an upcoming potential launch by year end so exa-cel, which is your CRISPR-gene editing or edited product for beta thal and sickle cell. You have recently gotten your PDUFA dates. Do you expect a panel here as you look to both beta thal and sickle cell?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Yes. Whether \u2013 we are thrilled to have the filings accepted. And now what this means is in Europe, in the UK and in the U.S., we are under active review for both sickle cell disease and beta thalassemia. That is a huge milestone in science and medicine. It\u2019s the first CRISPR\/Cas9 based therapeutic to get to this point. Whether or not we have an AdCom in the U.S. is the discretion of the FDA, and they don\u2019t have to tell us until they\u2019re ready to do so. If you ask me whether I thought it was more likely or less likely, I would say it is more likely that we have an AdCom simply because of the novel nature of this therapeutic, it is the first CRISPR\/Cas9 base treatment. But we don\u2019t get to find out until they\u2019re ready to say.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

And you\u2019re going to have authorized treatment centers ready to go at launch and then you\u2019ll probably add some over time. Help us understand how many will be ready to go globally and what proportion of these patients will be addressed through these centers.<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Stuart?<\/p>\n

Stuart Arbuckle<\/span><\/strong><\/p>\n

Yes. So let\u2019s start with the patients first. So there\u2019s about 150,000 patients with sickle cell and thalassemia between the U.S. and the EU. For exa-cel and it\u2019s kind of current iteration. So with the current busulfan based conditioning regimen, we think about 32,000 or so of those patients are likely to be thought of as good candidates given the severity of their disease. They\u2019re going to be patients like we\u2019ve seen in our clinical trials. They\u2019re having multiple transfusions up to 1 every 3 or 4 weeks, and in sickle cell disease, they\u2019re having multiple of these vaso-occlusive crisis, these very severe pain crisis, which lead to them being hospitalized. So about 32,000, about 25,000 sickle cells, 7,000 thalassemia. So when you think about the concentration of those patients in the United States, about 90% of those patients are concentrated in 25 states. And we\u2019ve said that we are planning to have 50 authorized treatment centers up and running approximately by the time that we launch, obviously largely concentrated in and around those states where the majority of patients are.<\/p>\n

In Europe, over 75% of patients are concentrated in the UK, Germany, France and in particular, for thalassemia in Italy. And we said that we\u2019re targeting about 25 treatment centers in total across those four countries. We\u2019ve been having dialogue with those treatment centers over the last few months, a lot of enthusiasm to get involved in exa-cel. I\u2019m anticipating we\u2019ll have close to those numbers, if not exactly those numbers by the time we get our approvals.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

And there has always been a debate about whether there truly is demand here for therapy or gene therapy or gene editing asset for this population. What gives you the confidence that these patients are willing to get on therapy?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

I\u2019ll let Stuart go into the details, but the one thing that Stuart has taught me is how quickly a study enrolls and the demand for a study is good, as Stuart says, biomarker for demand. And I\u2019ll tell you that both the sickle cell and the beta thalassemia studies were oversubscribed. Stuart?<\/p>\n

Stuart Arbuckle<\/span><\/strong><\/p>\n

Yes. No, I mean I do think that is a great biomarker of the unmet need and how excited people are about your technology is how quickly your clinical trials ramp up and went incredibly quickly, which is fantastic. In addition to that, as you can imagine, we\u2019ve done numerous rounds of research with physicians and patients as lately as EHA, where we presented new data, updated data on our exa-cel program. Enthusiasm across the physician community is high. And indeed, awareness and enthusiasm in the patient community is high. And up to 25% of sickle cell in TDT patients now say that they think a genetic therapy might be the best approach for them. So we think there\u2019s going to be significant demand for exa-cel, and we\u2019re excited to get ready to launch it, approvals willing.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

So bluebird has a gene therapy on the market right now. Maybe you could just speak to the learnings that you\u2019re seeing there, be it pricing, reimbursement, their version of these access centers and demand and how that applies to you globally, but also how you think that demand might be divided between the two assets.<\/p>\n

Stuart Arbuckle<\/span><\/strong><\/p>\n

Yes. So it\u2019s kind of a tale of two cities in some ways. So obviously, there\u2019s their experience in Europe where they decided after a few months to kind of pull out of the European market and withdraw their licenses. We\u2019ve obviously sought to learn from their experience. I think we\u2019ve learned a lot about what we need to do to be able to demonstrate the level of unmet need and the value of these types of technologies is one thing. And that supports pricing. The other one is around payment models. And I think one of the experiences we\u2019ve had in cystic fibrosis and also in our discussions about payment models for exa-cel is one size does not fit all. And that different governments are going to want different types of payment models based on their individual situations and what their individual needs are. And I do think bluebird have been very public. That was one of their learnings that they had a kind of one-size-fits-all approach in taking their medicine to Europe from a payment model point of view, and that didn\u2019t seem to get much traction. So that\u2019s one thing.<\/p>\n

In the U.S., I would say our learnings from their experience with their current therapy in TDT is very limited because they\u2019ve really only been on the market for a brief period of time. So I\u2019m not sure we can really glean many learnings from that. Certainly, from our own work in the U.S. We\u2019ve had great conversations with payers, both commercial and importantly, government, in particular, Medicaid. The payer mix for exa-cel is going to be sort of 60-40 government, commercial. And Medicaid is the primary payer. We\u2019ve had great conversations with many, many states, including all 25 of the states where the vast majority of patients are. Those discussions have been very, very productive. They\u2019re very well aware of the unmet need in sickle cell disease, the fact that this is an underrepresented and underserved population and they\u2019re excited about the profile of exa-cel. And similarly, with commercial payers in the U.S., our conversations have been very productive with them, and we are trying to work with them to get as close as we possibly can to kind of getting day 1 access as soon as we\u2019re approved, we want to be able to try and get this medicine to patients in need.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

Great. You\u2019re also looking at potentially a second launch via your acute pain program that\u2019s in Phase 3 and data is coming shortly. Help us understand what that optimal profile is that you\u2019re looking for, for this to be a success? And why you have confidence based on the Phase 2 datasets?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Yes. So I think it\u2019s going to be a race for what\u2019s next faster exa-cel in terms of launch because there\u2019s the vanza data that are going to come out end of this year, beginning of next, and it\u2019s the VX-548 acute pain data around the same time frame. But focusing on VX-548, so this is a NAV1.8 inhibitor. And both NAV1.7 and NAV1.8 have been called the holy grail of targets for the pain indication because these are genetically validated targets for which we can see people who have mutations can have a high tolerance of pain in the case of 1.7 and a constant sensation of pain in the case of a gain of function mutation in NAV1.8. We have studied this particular target for longer than we\u2019ve studied cystic fibrosis at the same site right here in San Diego. The predecessor molecule, VX-150 has already shown effectiveness in both acute pain and neuropathic pain. And it also showed effectiveness in musculoskeletal let\u2019s call it, osteoarthritis body pains of that kind. We see acute pain and neuropathic pain as fully Vertexian in terms of developing and commercializing.<\/p>\n

And in terms of acute pain for VX-548, not only did the predecessor molecule demonstrate success, but VX-548 itself demonstrated success in abdominoplasty and in bunionectomy in Phase 2. In the Phase 2 trial, we also had an opioid arm for context. It wasn\u2019t there for statistical comparison, but you can see the numbers there. And the VX-548 molecule performed very well in terms of efficacy and also in terms of benefit\/risk profile. For Phase 3, we do expect the results tail end of this year, beginning of next. It\u2019s the same exact pain conditions, bunionectomy and abdominoplasty. It\u2019s the same dose, the high dose from Phase 2. It\u2019s the same treatment duration, 48 hours, and it\u2019s the same exact endpoint. So because of those similarities, we have high confidence in the Phase 3 program. Maybe the last thing to share is at the end of Phase 2 meeting with the regulators, we discussed what the program needs to look like in order to get the label that we seek. And the label we seek is not a post-bunionectomy or post-abdominoplasty or even a postsurgical label, it\u2019s a broad label for acute pain. And so we have a third study in our Phase 3 program that\u2019s a single-arm, safety and effectiveness study that allows patients of all kinds of pain conditions, surgical and non-surgical, so that we can get this broad label.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

And Stuart, you\u2019ve talked about this being a $4 billion opportunity. What gets you there? And how are these hospitals and physicians incentivized to work with you to get access to this drug? Maybe just help us understand the logistics around that?<\/p>\n

Stuart Arbuckle<\/span><\/strong><\/p>\n

Sure. So it\u2019s a $4 billion market today. Acute pain despite 90% of the prescriptions being generic because there\u2019s been no innovation really in the pain market for literally decades, 90% of prescriptions are generic, and despite that, it\u2019s a $4 billion market today. So we don\u2019t need to get a very big share of that very big market at a branded price to have a multibillion-dollar asset on our hands, and that\u2019s what we believe we have. But why we think is going to be successful, I\u2019ll give you a couple of reasons. The first one is what is the level of unmet need? Because of the very, very well-known issues with opioids in acute pain, including their contribution to the opioid epidemic, just about every state, every hospital institution in the country has policies in place that restrict who can prescribe opioids, for whom, for how long and in what settings. What has that done? That has created a gap between prescription strength NSAIDs and people who are able to get access to opioids. It\u2019s created an incredible unmet need in the market for better pain treatment. Then there\u2019s the profile. You go to somebody and say, we\u2019ve got something that even is opioid like in terms of its efficacy, but without all of the baggage and including the addictive potential, is that something you\u2019re interested, overwhelmingly that is something that people are interested in.<\/p>\n

So then the challenge becomes what about pricing? You\u2019re going to price this as a branded medicine, and they can prescribe generic opioids in the hospital and that\u2019s going to be \u2013 those are really, really cheap, and they\u2019re all going to do that to maximize. I think that is a true criticism. Until you see some of the policy changes, which are being made, which I think are being made to provide a tailwind to the use of non-opioid pain medicines. And I\u2019ll point you to something that was signed into law by the Biden administration in December of last year. It was called the NOPAIN Act. The NOPAIN Act understands that there is essentially a financial disincentive to prescribe a better branded medicine because of the DRGs that govern hospital reimbursement. And so the NOPAIN Act instructs CMS to provide an add-on payment in the outpatient and in the ambulatory surgical center setting specifically over and above the DRG to compensate people for non-opioid pain medicines. I think that is the first of many such policy tailwinds that we will see instead of policy headwinds, which opioids have faced for good reasons. I think we are going to see the kind of the winds change and provide policy tailwinds to try and encourage and\/or remove the financial barriers to people using better, branded non-opioid pain medicines. So, we are really excited about VX-548 in acute pain and hopefully subsequently in neuropathic pain as well.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

And remind us, where you stand right now with the neuropathic pain program, but also your outlook towards chronic pain?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Yes. So, as Stuart outlined, we really see pain as three distinct categories acute pain, neuropathic pain and then the musculoskeletal pain, osteoarthritis and such. The latter two often people call chronic pain. We divide it in these a third because we see acute and neuropathic pain as fully Vertexian where we would commercialize ourselves. Just like with 548 in acute pain, 548 in neuropathic pain has the predecessor molecule, VX-150, which already demonstrated success in neuropathic pain. For VX-548, we are currently in Phase 2. The trial is well underway. I expect results towards the tail end of this year or beginning of next for neuropathic pain as well. And pending positive fingers-crossed results, the next steps from there would be to proceed on to Phase 3. And again there, we are looking for a broad peripheral neuropathic pain label.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

Great. So, you have a pretty deep pipeline beyond that, and we are going to get some data at the upcoming diabetes meeting your naked cell, stem cell program, regenerative stem cell program in type 1 diabetes. Help us understand how we should think about where this program sits right now in your whole vertical from Semma and then the steps towards getting to address the whole population. And is the science ready at this point? Like it seems like it\u2019s an optimization question, but are we here?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Yes. When we talk about this inflection point for Vertex in \u201823 being such an important year, think about it, we are now at the point where in our CF program, we are down to 2-year-olds with TRIKAFTA. The next, next gen is going to deliver results any moment. We are already in CF patients with the mRNA program, the first ever CRISPR\/Cas9 program for sickle cell and beta thal has been accepted by the FDA. And by the way, we have our cell-based regenerative medicine for type 1 diabetes and those results are going to come out in the next week or so at ADA. So, it really is a really important and very special year for the company. On type 1 diabetes in particular, we have three distinct programs under the type 1 diabetes umbrella. The first, as Salveen, you mentioned is, let\u2019s call it, the naked cell program. It\u2019s the cells alone and you need to use off-the-shelf immunosuppressive to protect the cells from the immune system. Those are the data that are going to be presented at ADA. We have Part A, and we have already shared those data from the first two patients dosed at half dose. So, the first patient is insulin independent. That is a huge, huge accomplishment, and it\u2019s never been done in an allogeneic sense before. The second patient also derived benefit. And based on those two patients worth of data, we declared proof-of-concept. We have completed Part A, that\u2019s those two patients. We have also completed Part B, which is full dose with a stagger. And now we are on to Part C full dose without a stagger. At ADA, you will see the Part A patients, so longer duration of follow-up. And you will also see all the patients in Part B, so more patients. The second program, the same exact cells where we have already shown proof-of-concept enveloped in a device, no immunosuppressions necessary. The CTA has cleared in Canada. The IND is cleared here and in the coming weeks and months, you should expect to see us dosing those patients. And the third program is those same exact cells that we make certain edits to, so that those edits protect the cells from the immune system, they make them, let\u2019s call them, hypoimmune or immuno-evasive cells. That third program is still in the labs, in the research stage. So, that\u2019s what it looks like in terms of the program where you can expect at ADA. And just to close on it in terms of, is the science ready, absolutely yes. We are actively dosing patients, and you will see the results for yourself in a couple of weeks. Whether or not the best format for patients is cells alone, cells plus device, edit itself, we are going to have all of it to look at. And depending on the patient population, they can make the right choice for the benefit risk profile that\u2019s best for them.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

And with the third program, the edited program, how are you contrasting that with your ViaCyte acquisition and the programs you got through that?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Yes. So, the \u2013 once we saw the first two patients\u2019 data and we are able to declare proof-of-concept, once we had that leadership position, we wanted to make sure we covered the waterfront as it were. ViaCyte has a very similar goal that they were trying to achieve as we do, which is to cure type 1 diabetes. Their approach is slightly different. What we gained from the ViaCyte acquisition was people, tools, capabilities, manufacturing. As an example, they had already \u2013 ViaCyte had already worked with CRISPR on editing cells, albeit with a different starting material. And through this acquisition, we get access to tools, technologies, IP, cell banks, but also the program that\u2019s already in the clinic using the ViaCyte cells and CRISPR edited cells, so we can explore those as well. You put it all together, and we feel really great about the position we now have, including access to already edited cells that are already in patients.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

Great. And remind us, you have a few other programs in the pipeline that are in the clinic. When might we see data from those programs?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Right. So, if we move to some of the other programs that are in the mid-stage, let\u2019s say, let\u2019s maybe go through those, VX-548 in type 2 \u2013 in peripheral neuropathic pain that should be end of this year, beginning of next. Inaxaplin previously known as VX-147 in a particular kind of genetically mediated kidney disease. It\u2019s a Phase 2\/3 adaptive design program that also has a pathway to accelerated approval in the U.S. that we have already agreed to with the agency. The part \u2013 the Phase 2 part of the 2\/3 program should also be ready sometime towards the end of this year. We have talked about the diabetes program, maybe a quick word on AATD. We have two programs running there. One is the first-generation program. It\u2019s called VX-864 that is being run in a longer-term longer term study, so we can assess both the liver and the functional AAT levels. And a next-generation program called VX-634, which we are running through Phase 1. It\u2019s a more potent molecule. At some point next year, we will have access to all of that data, so that we can make our appropriate decisions for next steps. I should say for AATD, this disease is important to understand in that it has a liver manifestation and a lung manifestation of disease. And there is no other program out there that\u2019s targeting both simultaneously. That\u2019s why we think this is so important in terms of the Vertex approach.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

Great. And maybe just a last question here. You have over $11 billion on cash on your balance sheet. How are you thinking about capital allocation in terms of whether you would consider doing M&A at this point even though you have a deep pipeline or even dividends?<\/p>\n

Reshma Kewalramani<\/span><\/strong><\/p>\n

Yes. I am going to sound like a broken record on this. The primary use of our capital is going to be in innovation, both internal and external. And you have seen us do the kind of deals that just fit perfectly in our strategy. It\u2019s not a matter of size. It\u2019s not a matter of whether it\u2019s cheap or not. It only matters whether it fits our strategy. You have seen us do modest share buybacks. You should expect that to continue. And as I look at my crystal ball, I don\u2019t see dividends in the near future.<\/p>\n

Salveen Richter<\/span><\/strong><\/p>\n

Great. Well, perfect. Thank you so much Reshma and Stuart. Appreciate it.<\/p>\n

Stuart Arbuckle<\/strong><\/p>\n

Welcome. Thank you.<\/p>\n

Reshma Kewalramani<\/strong><\/p>\n

Thank you so much.<\/p>\n<\/div>\n

Read the full article here<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Goldman Sachs 44th Annual Global Healthcare Conference Call June 13, 2023 5:40 PM ET Company Participants Reshma Kewalramani – President and Chief Executive Officer Stuart Arbuckle – Chief Operating Officer Conference Call Participants Salveen Richter – Goldman Sachs Salveen Richter Great. Good afternoon, everyone. Thanks for joining us. I am Salveen […]<\/p>\n","protected":false},"author":1,"featured_media":613,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"gallery","meta":{"footnotes":""},"categories":[236],"tags":[83],"class_list":["post-22279","post","type-post","status-publish","format-gallery","has-post-thumbnail","hentry","category-news","tag-featured","post_format-post-format-gallery"],"yoast_head":"\nVertex Pharmaceuticals Incorporated (VRTX) Goldman Sachs 44th Annual Global Healthcare Conference (Transcript) | iFintechWorld<\/title>\n<meta name=\"description\" content=\"Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Goldman Sachs 44th Annual Global Healthcare Conference Call June 13, 2023 5:40 PM ET Company\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/ifintechworld.com\/?p=22279\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta 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