Good evening, everybody. Thank you so much for joining us today on day three of the J.P. Morgan Healthcare Conference. My name is Bhavna Balakrishnan. I'm an associate with the Healthcare Investment Banking team. Today we're joined by the management of PureTech. And with us, we have Robert Lyne, Chief Executive Officer, Eric Elenko, Co-founder and President. Over to you, Robert.
Wonderful. Thank you very much, Bhavna, and thank you for everyone who's turned up today and for those who are joining on the webcast. I'm delighted to be here and thank J.P. Morgan for the opportunity to present to you all, so as Bhavna said, I'm Robert Lyne. I'm the CEO here at PureTech Health. I've been in the business two years now. I've previously held a number of senior leadership roles and CEO roles in various London-listed life science businesses and was delighted to step into the CEO role here at PureTech at the end of last year, so as we move through the presentation, there may be a number of forward-looking statements that we'll be making. I'll refer you to our SEC filings in that regard, so as an introduction to PureTech Health, we have a proven hub-and-spoke model for drug development.
What does that mean and how does that differentiate us from other drug development businesses? What we do is we take a de-risked, diversified approach to developing a number of potential new therapeutic treatments for patients. We do this with a hub approach where a centralized, lean, efficient hub looks at novel opportunities to develop dramatically different treatments for patients in areas of high unmet need. We look to spin out those programs into what we term our spokes. Those are standalone businesses that leverage external capital to take those programs forward, hopefully advancing those treatments all the way through to registration so that they can get to market and be available to patients, and also so that they can generate significant value on a financial basis back into PureTech that we can then use to benefit our shareholders.
We see two distinct advantages to this portfolio approach. One is that we have the opportunity to seed a large number of potential companies and therefore advance a greater number of therapies than we could if we were focusing on just one asset alone. But also, from a financial perspective, we have the ability to balance risk here. Everything we do has empirical risk. We're very cognizant of that. And by having a portfolio approach, we can smooth that out and generate a greater chance of getting a hit across the portfolio and also generate a smoother and, we think, overall larger return for our shareholders financially. So, when we look at the track record of the business so far, we're incredibly proud of our remarkable success in clinical trials so far.
Around 80% of our clinical trials that have been run by PureTech or by our founded entities have hit their primary endpoints. For us, that we think is really remarkable, and that is testament to the de-risked approach, which I'll talk about in a moment, which we think has led to that very high probability of success. So far, as a business, we've had three FDA-approved treatments, including very recently Cobenfy, formerly KarXT, which was originally developed by PureTech and spun out into Karuna Therapeutics, which has been one of our most successful founded entity spinouts to date. We've seen this model work previously at PureTech, and we have a strong confidence that we can repeat this performance as we go forward. If we look at our innovative approach to drug development, how is this different to how others might do it?
A key starting point for us is we look at validated pharmacology in the targets that we're working on. So, typically, we'll look at assets that have already been in human trials, that have been either left on the shelf or haven't been advanced for some reason. And what we'll do is we'll look at what was holding that drug back, what was holding that molecule back. And then what we do is apply an innovative approach to that to think, okay, what can we do differently here? What modification could we make? What combination could we make that would allow us to then overcome whatever's been holding that drug back? We think this has distinct advantages. First of all, it gives us a higher probability of success that we'll then be successful when we move that drug through.
We believe that the clinical trial success rate that I previously talked about demonstrates that. Also, from our perspective, we're then leveraging the capital and time that others have already put into that molecule, meaning we're sort of one step ahead than we would otherwise be if we're looking at totally novel mechanisms of action. In addition to that differentiated approach that we have, we also, as with many others, we will look at making sure there's high unmet need in the patient population that we're targeting. We're also very focused to make sure that there's a defined regulatory and commercial path to success for that drug so that it will be able to get to patients and it will be able to generate a material financial return to us.
Again, as I mentioned previously, because we have this portfolio approach, we also, across a number of different potential therapeutic targets, we're looking at making sure that we allocate our capital efficiently there. So, what that means is we will very often look at a potential drug. Eric is very fond of saying, we'll look at something and think, what do we not want to see in a clinical trial, in an early proof of concept study? And then we'll run that and we'll see. And if we see what we didn't want to see, we will move on and we will divert our capital elsewhere where we feel it's more efficient. For us, we think that is a distinct advantage over other vehicles which are maybe following a single asset. We don't have a bias to continue any particular program.
We have a bias to make sure that we are pursuing the ones that have the greatest chance of success. So, what does this mean in terms of patient impact? For us, our mission at PureTech is to deliver meaningful new treatments which will transform the paradigm for the patients that we're targeting. We've already done this with Cobenfy. This is already now being prescribed to patients. Obviously, schizophrenia has been an area of huge unmet need. Cobenfy was the first new novel treatment to be approved in 50 years in this indication. Very large patient population, and we're really excited to now see the sales that have been rolled out by Bristol Myers Squibb, which has acquired Cobenfy and is now bringing that out to patients.
As we look forward, we have a number of other spokes which we're spinning out in the form of Celea Therapeutics and Gallop Oncology. And we also have Seaport Therapeutics, which spun out of PureTech last year. Each of these companies is targeting really meaningful patient populations, and each of them have transformative treatments that we think will make an enormous difference to patients when they come through. So, as we look at what makes up PureTech, and particularly from the components of value, as previously mentioned, we have these spokes that we've spun out. We have Seaport Therapeutics, and I'll talk a little bit more about that later, which is the most advanced in terms of its corporate development. It's an independent entity that's been spun out. It's raised significant financing, $325 million from top-tier investors.
We still, however, retain the largest shareholding in that business at just over 35%. And in addition, we have meaningful non-dilutive economics that attach to those drug assets. And for that, we see that as an important differentiator of the model. Because when we look at these early opportunities, we are typically carrying out novel work on these molecules. We will be developing proprietary IP, which then gives PureTech not only this equity interest, which naturally will be diluted as we spin out these programs, but also these non-dilutive economics that we typically attach in the form of royalties and milestones. So, we have two opportunities to deliver value back into PureTech and to our shareholders from these programs. In addition to Seaport, which has already successfully spun out, we're now in the process of spinning out Celea Therapeutics and Gallop Oncology, each of which I'll talk about later.
In terms of historic success, Cobenfy, we still have these non-dilutive economics which attach to Cobenfy. We've seen an enormous financial return already back into PureTech from the equity stake that we had in that business. However, we continue to have royalties and milestones due to us on sales of Cobenfy, which will produce meaningful income back into PureTech. We also are proud of the balance sheet that we have. It was $320 million at the 2025 half-year. All of that is capital that has been generated by the PureTech model. We haven't had to raise capital in nearly eight years. So, we've demonstrated that we can become a self-funding business with no need to dilute our shareholders and ample capital to move our programs forward.
Finally, a focus of the business is the next wave of innovation, what we're going to be looking at and what we're going to be working on in order to build the new founded entities of the future. At this stage, we've got a few exciting things cooking. We're not ready to share too much of that today. As and when we start to bring some of those programs forward, many of you, I'm sure, will recognize some of the PureTech hallmarks of the PureTech model in terms of these de-risked assets where we already have validated pharmacology, typically some early trial data, but we think we can really transform these drugs and their efficacy and side effect profile to transformative treatments. An overview of some of the founded entities we have. We'll start with Celea Therapeutics.
This is a company that we're spinning out at the moment. Delighted to be joined in the audience by Sven, the CEO of this business, who has been busy at this conference. Celea is an incredibly exciting opportunity for us. It's developing a new treatment here for idiopathic pulmonary fibrosis. For those who don't know, this is a devastating illness where there's currently no cure, and it is at this stage a fatal disease. It affects a large number of patients across Europe and North America. At the moment, because of the paradigm of treatments that are available, three out of four patients who get this disease never even start on the antifibrotics, which are already approved for treatment of this indication. We see this as a huge unmet need and also a huge potential that we can address through the drug that we've been developing, which is deupirfenidone.
This is a novel oral small molecule, and it is a next-generation antifibrotic. How does deupirfenidone differentiate itself? It's a deuterated form of pirfenidone, which is one of the approved existing standard of care treatments for IPF. As I've already mentioned in the PureTech model, this is somewhere where we already have a high degree of confidence as to how we think this can play out and how this can work. What happens here? You'll see on the left hand of the slide here, we have the results from the Elevate phase II-B trial where deupirfenidone was being dosed in IPF patients. Against placebo, it achieved statistical significant response. We see a very meaningful reduction in the lung function decline, which is the measurement of disease progression in this indication.
That progression was reduced to such an extent that it actually approached the lung function decline, which we see in naturally occurring healthy older adults. So, for us, this was an incredibly exciting signal. We were also encouraged from the 26-week data we have from the phase II-B trial to then see the open label extension, which we ran. Around 90% of patients transferred from Part A into the Part B open label extension. Again, we saw that as very, very encouraging. The 52-week data that you'll see here on the right of the slide shows the strong and durable response we saw where this continued arresting of the lung function decline approaching natural healthy older adult lung function decline was demonstrated. I'll now move on to Gallop Oncology. This is the oncology asset which we've spun out into this business, Gallop Oncology.
It's a novel antibody targeting Galectin-9, and it's for the treatment of lung cancers, specifically AML. Again, it has the hallmark of a significant unmet need here. Currently, it's being dosed in the phase I trial in a frontline setting. These are relapsed refractory patients who have a life expectancy at this stage of around two and a half months. So, it is a really desperate need for these patients to have further treatment options. If we look at the data that we've seen from the phase I trial, this has been achieved through a novel dual mechanism that LYT-200 targets. So, not only does the drug directly kill cancer cells, but it also has a second effect of activating the immune system and having a cancer cell-killing effect there.
If we look at the latest cut of the data, and this is still maturing and still improving, but the latest cut of the data we have on the right-hand side here, we see the critical overall survival data that was coming out of the phase I trial. Recent FDA guidance has emphasized the importance of overall survival in this indication as the primary endpoint. We're very encouraged to see that the 13.2 months median overall survival that we're already seeing is well ahead of the nine-month KOL-defined benchmark of success. As with Celea Therapeutics, Gallop is a program that we are actively fundraising for. We've seen a lot of positive engagement from potential investors. These are the two programs that we're looking to spin out of the business with external capital to allow them to take these two important programs forward.
We'll then come on to Seaport Therapeutics, as mentioned at the opening of the presentation. This is the most advanced in the corporate setting of the companies that we have. It was spun out last year, two years ago now nearly. It's raised $325 million from top-tier investors across two rounds. Despite that significant capital raising, we remain the largest shareholder in this business with a very meaningful 35% equity stake here, and this has three therapeutic candidates within its portfolio. Two of these are clinical stage, GlyphAllo, targeting major depressive disorder, and GlyphAgo, targeting generalized anxiety disorder, and again, if one's looking at the hallmarks of PureTech assets, these are programs that are based on validated targets on approved therapies where we believe we can transform their effect through the novel application of the proprietary Glyph platform, so, from a financial perspective, what can this model deliver for us?
We have here an example of the financial returns that we've made here to date so far from Cobenfy. So, as I mentioned, this is the KarXT, as was previously developed by Karuna, which was spun out from PureTech. And you can see here the enormous potential for financial return that we can deliver. We invested $18.5 million into the development work of Cobenfy. That has to date delivered over $1.1 billion back into PureTech on a gross basis. So, an enormous financial return. And that is an example of what we can do by leveraging external capital, carrying out these very early, lean, efficient, de-risking activities that demonstrate the potential of the drug, which then allow us to leverage external capital and allow the program to be taken forward by others while still returning significant value into PureTech.
Because of the model here, because Cobenfy was invented by PureTech, we were the lead inventor here on the patents. He is sitting next to me. We retain these non-dilutive economics, as I've mentioned previously. We've been modeling these on a basis, simply on a simple average of analyst forecasts, and the milestones and royalties that are due to us through 2033, we're valuing at around $300 million based simply on those average of analyst forecasts. That's without any discount for time, but that gives an idea of still the significant value that can come back into PureTech from the success we've had, and also, this is a sort of model that one can expect to see us repeat across our other founded entities as they mature and spin out. So, as we look to 2026, we're pleased to build on the tremendous success that we've had in 2025.
We have operational priorities, which is to secure funding for Gallop Oncology and Celea Therapeutics. We want to make sure that those programs are fully funded through their next trials. And in each case, we believe that that funding can get each of those programs through to pivotal trial readouts, which would be incredibly exciting for us. In addition, at a hub level, as we progress the business and as we mature PureTech, we want to spin those programs out that will allow us to operate a lean, efficient hub model, which will then continue that future innovation on a very capital-efficient basis. As we have that financial development going through the year, we also have clinical progress that we expect from our programs. In the case of Celea Therapeutics, we'll be looking to initiate the phase III trial once we have that funding in place.
From Gallop Oncology, we're expecting important additional efficacy data and that key final overall survival data in the first half of this year. While we rebuild the business so that we can deliver those strategic priorities, we're also making sure that we have an efficient hub that can deliver the next wave of innovation, which will provide the further generation of founded entities that can generate further value for PureTech, but most importantly, a range of important new treatment options for patients. Thank you very much for that. I'm joined now by Eric Elenko, my colleague, Co-founder and President of PureTech for the Q&A.
Thank you so much, Robert. If anybody has questions, please do raise your hands and we'll get you a mic. Rob, you recently stepped into the role of the CEO. Could you tell us more about sort of your vision for the next phase of the company?
Yeah, certainly, so look, I've always been, when I stepped into the CEO role, I was delighted to be asked, and I always admire PureTech as a business. One of the things, just to give you an idea about what attracted me to the company and where I think we can really be transformative, I have a history working in venture capital, and a lot of the VCs do great work, but it's also always struck me the number of VCs that, despite enormous success they have, don't necessarily actually ever get a drug to market, so the track record of PureTech, when I was asked to come and join the business, we'd just seen the positive phase III readout from Karuna. It was very clear to me and obviously many others that that was going to get approved.
That, alongside the other two FDA approvals that the business have had, I found that a hugely exciting prospect. And it was very clear that there's a very special company here. So, from my perspective, it was a hugely attractive opportunity to come and join the business. In terms of the vision going forward, what we want to do is ensure that we have a continuous generation of new ideas. How do we do that? We want to make sure that we have the right people and resources within our hub so that we can be generating exciting new programs, but also from a financial perspective, that we have the right resources to move those programs on.
That means doing the early de-risking experiments that I talked about, but it also means making sure that we can leverage external capital at the right time so that we can then return additional capital in and generate new programs and continue the cycle. So, my vision is that when we get these programs spun out, we're going to be able to have a lean, efficient hub at the business that we've already seen has a great track record of drug development, and we will be able to marry that alongside what should be a very successful financial performance from the successful founded entities that we're spinning out.
Yeah, and that sort of leads into my next question. As we think about the new wave of programs as sort of core to your hub and spoke model, how do you think about your innovation strategy there, and are there any specific therapeutic areas that are of interest?
Eric, do you want to?
Sure, absolutely. One thing that really distinguishes PureTech is how we do innovation. And Rob described this emphasis on validated pharmacology, meaning looking at a drug where there's clear human data that that drug is having a positive effect, but there was something holding back that drug so it wasn't realizing its full potential. And then what we'll do is we'll apply new thinking or technologies to solve whatever problem was holding back that drug. And that's what happened in the case of Karuna, that's Seaport, that's Celea. And what we really see going forward is, given how impactful that strategy has been, to really continue looking at opportunities and innovation based on validated pharmacology. Going forward, we really see the emphasis on small molecules.
And in terms of therapeutic areas, while we're open, we will be spending more time and thinking about those areas where we've historically had experience and had success. So, for example, CNS is an area of high interest for us as we look to new innovation.
Yep, if you could just wait for the mic. Sorry. Thank you.
Hey, thank you. So, when you're taking drugs that have been shelved for any reason, are you doing any actual new chemistry, or is it more finding the right patient population for that drug?
Yeah, it's very case-dependent. And so, typically, we're in a situation where we take a drug that's in the clinic and we actually bring it back to be preclinical, and we're doing something. And that could be something in terms of chemistry, for instance, making a prodrug. It could be something formulation. In the case of Karuna, it was combining one drug with another drug. So, PureTech isn't based on a single technology platform, but it's more about the process of taking a drug, solving the problem, and that's very bespoke to each drug that we're working on. Our strategy is very product-focused and very asset-focused, and that's something that we anticipate going forward.
You talk about Karuna and Cobenfy as one of the validations of your model. What gives you the confidence that it is replicable and there's opportunity for future success?
I think partly what we've seen already in terms of companies that we've been able to spin out. In the case of Seaport Therapeutics, as we say, a number of absolutely top-tier blue-chip investors were very keen to get into these funding rounds. They clearly see the potential and the opportunity in that business. A similar approach, as Eric was saying, was taken there in the broad sense in terms of drug development, as we have with Karuna. We feel we've already had a number of validations going forward. It's not easy. We're very open about that. These opportunities are not just sort of lying around. They take a lot of work and a lot of thought to identify and to execute on properly. We don't shy away from that.
But we feel that we've had a track record of success that is continuing, and we can see a path forward to that, as I say, repeating itself in the future.
Yeah, if you look at Karuna, we would argue it's not happenstance. Now, we can't guarantee that everything we work on will result in the exact same success as Karuna. I think that might have been the fine print that Rob was showing in the disclaimer in the beginning. But if you look, there are some key elements that were present in the case of Karuna, and we really see those going forward with other opportunities. One was there's a very large unmet need, and Rob described that in his presentation. A second is there was clearly a drug that was having an impact on patients. A third was we could clearly identify the problem associated that was causing the adverse events with that drug. Fourth, we could come up with a clear solution that made sense pharmacologically.
And fifth, we had a very clear path and way of testing that solution was going to work. And if you look at all those elements, you see that in the programs that we currently have, and that's really going to be the strategy as we move forward.
You've been self-funded for the last eight years or so. How do you think about capital allocation and financial discipline looking forward?
Yeah, it's a great question. I mean, for us, as I say, because of our portfolio approach, actually, we see that as a real strength of the business. As I mentioned, we don't have any bias to continue an individual program. Our bias is to get the best return from the capital that we're deploying financially and also in terms of patient impact. So, for us, when we're thinking about capital allocation, we have a range of opportunities. We have later-stage assets. We have existing founded entities. We have early innovation programs. We also have the potential to make returns directly to our shareholders. So, we think very carefully about the capital that we have and where it is best used across that spectrum, and for us, we think that's a real advantage to the model.
It means that we're very happy to seize programs where we don't see a future or we don't see that they're going to be as efficient in terms of the return on capital and the impact that can have as elsewhere. So, that healthy competition within the business, we think, is a key advantage in terms of efficient capital allocation for us.
Maybe talking about Celea Therapeutics for a moment, and welcome, Sven. You recently met with the FDA to talk about phase II-B. What more can you tell us about your upcoming phase III?
Yeah, sure, absolutely. So, the phase III design, which is really going to be centered around comparing deupirfenidone to pirfenidone, is based on conversations we had with a number of key stakeholders. So, physicians, patients, of course, key opinion leaders, and very importantly, as you indicated, the FDA. So, we had an end-of-phase II meeting in December of last year. And that resulted in the design, which is centered around the comparison of deupirfenidone to show superiority to pirfenidone. Now, really, if you look at the study that we did previously, Elevate, versus the study we plan on carrying out, it's really what's changing is the arms, right? Those two arms versus in Elevate, we had two different doses of deupirfenidone and a placebo. However, the other core elements are essentially the same. The primary endpoint of forced vital capacity, FVC, the patient population.
Really, the idea is we are going to go and reproduce what we did in Elevate, but in this phase III trial with these two different arms. Given the difference in effect that we saw between deupirfenidone and pirfenidone and Elevate, that really gives us confidence as we move into this phase III trial.
Great, thank you for that color . Wanted to open up for any final questions? If not, Rob, want to turn it over to you for any closing comments that you might have?
Thanks, Bhavna . So, look, as I was saying earlier, for us, 2025 has been a transformative year. We ended last year at the start of the year off the back of the incredibly exciting phase II-B Elevate data in the IPF study. That has provided an enormous boost to the business during the course of the previous year. 2026 for us is going to be pivotal. We're now in a situation where we've got these two major programs that we're spinning out. We're looking to raise that external capital during the course of 2026. That will be transformative to PureTech. It's going to crystallize significant value within the business when those assets are spun out. It will also allow us to have a lean hub that remains after those spinouts, which will then be working on this exciting new wave of innovation.
As ever, our main mission is to bring forward these important treatments to patients so that they can get to market and help people. But also, we see the potential to generate significant value through these programs and through that to generate a positive return for our shareholders.
Great, thank you so much, Rob. Thank you, Eric.
Thank you .