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Goldman Sachs 42nd Annual Global Healthcare Conference

Jun 9, 2021

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Okay. We will continue with the next session. Hi, everyone. I'm Paul Choi, Mid-Cap Biotechnology Analyst here at Goldman Sachs. Thank you for joining us for our 42nd Annual Global Healthcare Conference. Our next session will be with UroGen. We're pleased to have CEO Liz Barrett and Jeff Bova, Head of Commercial, joining us here. What we'll do is turn it over to Liz for some opening comments and then go into Q&A. As with prior sessions, if investors have questions along the way, please feel free to submit them via the webcast portal. Alternatively, you can email them to me. I'll read them out loud, time permitting. With that, I'll turn it over to Liz.

Liz Barrett
CEO, UroGen

Thanks, Paul, and thanks for having us. I asked Jeff to join us because I know a lot of the interest is around what's happening with the commercial launch. It's an exciting time for UroGen. I always say that I say this every time, this is a pivotal year for us, but it is a pivotal year for us. We were laughing because someone asked us, we talked about launching June 1st, and somebody said, of this year? It's like no, last year. We launched our first new medicine June 1 of 2020, right in the middle of the pandemic. I know that Jeff had spent the last couple of years prior to that preparing for launch, only to have everything switch at the last minute. I'm really pleased with the way things are going. It's an exciting time for us.

We're coming out of the pandemic, and Jeff will talk a little bit about that in Q&A, how things are going and what we're seeing out there. It's really an exciting time for us. We were able to bring this medicine to the market and to patients with a single-arm study because the FDA recognized the high unmet need in this area of low-grade upper tract urothelial cancer, where these patients, 70%-80% of these patients actually lose their kidney. When you're talking about a patient population in their 70s and 80s, that's actually a really big deal. There had been no medicines actually approved by the FDA for this disease, and so we're really pleased to be the first ones to bring it.

In addition to the commercial launch, which I talk about 2021 is all about the commercial launch, we have a lot of other exciting things happening in our portfolio. We read out last year around our UGN-102, which is for low-grade non-muscle invasive bladder cancer, a portion of that patient called intermediate risk. Again, a patient population with a high unmet need because these patients end up getting repetitive TURBTs because the TURBT just doesn't work for them. We're really pleased again to be able to bring this new medicine forward. We're in our phase III study right now, enrolling patients around the world. That's going well, the enrollment. We were actually really happy with May. We hit our enrollment numbers, which is a big deal, as you know, particularly again, as we're coming out of the pandemic.

It's been a challenging time for us, but an exciting time, and we're pleased with the results and what we've been able to do to advance our portfolio. Lastly, we have our immuno-oncology assets in early development, preclinical right now, working with our TLR7 agonist in combination with CTLA-4, local delivery with our proprietary RTGel. Again, a lot happening, keeping it going. We did some financing, as everyone knows, this year. We're in a great financial position and just looking forward to continuing to advance not only the commercialization, bring this new medicine to patients, but also in our portfolio. I think I'll just stop there and go to Q&A, and we can go from there.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Great. Thanks for that overview, Liz. A lot going on with the company this year. You did, of course, sort of reference probably what is top of mind with investors, which is the launch. Like a lot of recently commercial-stage companies, you did highlight that you did launch last year into a COVID environment. I guess maybe to help us understand or maybe mark to market, can you or Jeff alternatively sort of provide an assessment of what the landscape is like and sort of what are the current trends or updated trends with respect to patients returning to offices and seeing their physicians? And just kind of what are the nature of the conversations patients are having? Maybe just sort of an update on what's happening in the market with physicians.

Liz Barrett
CEO, UroGen

Sure. I'll talk more broadly, and then I'll turn it over to Jeff to talk more specifically about uro-oncology. The reason I was talking more broadly about it is you read kind of every day. First of all, when a lot of companies read out their Q1 and their 2021, I mean, the 2020 results, everyone talked about the reduction in new patients. That was really unfortunate, particularly when you're talking about a cancer diagnosis. It was down 40% in 2020. As you talk about what's happening now, and I was just reading yesterday that things are starting to come back to normal. Everybody expects normal to be back in 2022, but we are seeing more patients comfortable coming in, not just, again, for us at UroGen, but overall in the industry, which is something we need to do.

It's really unfortunate because a lot of these patients, what I'm hearing just anecdotally in talking to physicians, they're saying when patients come in now, they're just further along in their disease. It's like because patients were afraid to come in, we have to make sure that patients understand it's safe now with everything that's happening in the vaccines. Thank you to all the companies that developed the vaccines. I think that's really important. I think we're going to see it get back to normal, but I think it will still take some time. You may see a bolus of patients, I think, as you commented about it, but I don't think it's sort of like a huge bolus. I think patients are trickling in over the month and have been.

With that, I'll just turn it over to Jeff, and he can talk more specifically about what he's seeing specifically in our area. Jeff?

Jeff Bova
Head of Commercial, UroGen

Sure. Thanks, Liz. The one thing that I think we're past is the prioritization of getting a vaccine in our age population. If you're 65 and over and you haven't been vaccinated, that's a choice that you're making, and you're probably coming in and getting your cancer treated. The one thing that Liz alluded to, and it still varies throughout the country, are offices opening up to seeing representatives. I will say this, it's getting better. Every state's a little bit different, every institution's a little bit different, but it is improving. I've been able to be out in the field and sit in lunches where 20 to 25 folks will gather in the lunchroom, and it's relatively back to normal. I'll travel to the next state, and there's still some restrictions with regards to access. I look for that to continually improve.

I always say the importance of face-to-face interaction, especially in orphan drug, Paul, because the reps are the face of JELMYTO. They are the reminder. They are the, oh, that's right, I've got a patient that I can consider for JELMYTO. That face-to-face access continues to get better. As it does, we're going to continue, as we have in the last two, three months, to increase the number of patient enrollment forms and increase the number of patients that see JELMYTO.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Okay, thanks for that, both Jeff and Liz. I guess, as you think about the launch progress to date, how would you maybe frame it versus your expectations? Maybe I guess the follow-up question here is, are your expectations starting to rise now as things are starting to open up here? You're getting more face-to-face meetings with physicians and potential prescribers and group practices. I guess first, how's it tracking, and then are you starting to become a little more optimistic here?

Liz Barrett
CEO, UroGen

Yeah, I'll just make a comment and then turn it over to Jeff. I laugh because I get often told that I'm conservative. You're really conservative in your comments. Look, I'm really pleased with what we've done so far and where we've come so far. I think Jeff and I often talk, what would it have been like, even at launch, had we not launched in a pandemic? We met expectations, exceed expectations in 2020. We talked about the challenges in January and February of this year of patients getting vaccinated. We saw that happen, and we've seen the difference now. We've seen it, as Jeff commented, March, April, and May. I'm going to say I'm cautiously optimistic. I think those are actually also Jeff's words that he uses.

The good news about our therapy is it makes it more challenging because it's a treatment, right? It's a therapy. It's not a simple pill or an infusion. Because of that, patients actually have to enroll. Jeff Bova commented about a patient enrollment form, that actually gives us kind of an early indicator of patients, because you don't have a patient enrollment form unless you have a patient. We continue to see strong patient enrollment forms. Yes, we continue to be very optimistic, very confident, and it's given us a lot of confidence in our ability to deliver on 2021. Even more importantly, the research that Jeff Bova and his team has done really shows that there's a lot of interest in using this medicine with patients.

We expect as we get over and integrate the therapy into the patient's office and get all of the formulary and get the logistics taken care of, we really expect this to be the new standard of care. Jeff, I don't know if you want to add anything to that.

Jeff Bova
Head of Commercial, UroGen

Yeah, I think what helps too is the time it takes to set up an account. When you're limited to emails or virtual only, it adds a little bit of time. This drug, even without the pandemic, you've got to get it through sometimes a formal formulary process. You've got to be able to go in and develop champions. You've got to be able to go in and train on the mixing on the first or second dose. As things open up, you'll start to see account setup time decrease. I'm already starting to see it because you can do so much more when you can walk down the hall and talk to the nurse, when you can walk down the hall and talk to reimbursement and coding versus sort of doing everything virtually.

I'm very happy with where we are with regards to the pandemic. There's all sorts of data that talk about smaller biotechs not launching successfully.

We've done that in an environment where we've had limited access to face-to-face interactions. We were able to do that virtually. We're able to continue that. As things open up, I'm optimistic that we're going to continue this growth. What I said to the representatives is that, and a lot of them, actually, they said it to me, is like, this is our face-to-face launch in a lot of these territories. This is the face-to-face launch, and they're excited, and they're motivated because now I always say it's a fair fight. We can actually go out there, make sure that we're seeing the right physicians, get around the offices, have lunches, and we're able to do what we would've done last year at this time.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Yeah.

Jeff Bova
Head of Commercial, UroGen

That's why I'm optimistic about the success moving forward.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Great. Could you maybe provide us a little color on where you're perhaps getting the most traction? Is it among academic centers where they have great familiarity with your data and probably follow AUA very carefully? Is it more among community practices or LUGPA? Can you maybe just give us a sense for how and where, how much adoption is going on in each particular setting?

Jeff Bova
Head of Commercial, UroGen

Sure. It's a good question. I can divide it up into two parts. Last year we saw a lot of the business from the academic or the hospital setting. The community is much more sensitive, as you know, to the financial aspects of a buy and bill drug. Whether they told me this or not, they want to see a J-code, a permanent J-code. It's not surprising at all that we had that permanent J-code in January. Now you're starting to see more uptake in the community. You're starting to see community urologists use this either through a nephrostomy tube and administer it in a clinic or administer it in their surgery center. We are starting, in fact, every day.

In fact, actually just yesterday morning, I got a text that a big community account in Michigan received payment. When it's a J-code, it's automated. You should be paid in 30 days. They got paid in 30 days. They got paid accurately. When you start to see that reimbursement confidence go up in the community, I expect that to grow as well. Predominantly out of the gates, Paul, it was more in the hospital, and you're starting to see that transition into the community.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Great. Can you maybe talk us through a little bit more about the process of getting a new prescriber up and running here? How many calls has it been taking, either virtually or in person now to get that physician on board? Has there been any debate on sort of hard... You referenced a little bit earlier about setting up offices for mixing drugs and so forth. Can you maybe walk us through the prescriber setup process here?

Jeff Bova
Head of Commercial, UroGen

Sure. As far as the early adopters go, it takes one to two calls. When we do our market research, what's nice, we research with about 100 urologists. They all have a place for it. It's the first time ever I've done research, and there hasn't been a handful that say, I'm not going to use it. There's no place for it. They all have a place for it. The early adopters understand the need to preserve kidney. They pushed either a formulary review, or they pushed their administrator to get this up and running. They pushed pharmacy to get trained. They led the charge. The second group that we're seeing now are those peer-to-peer influencers. They're in a group of 10, 12 urologists, one or two have used, and then that starts to spread to the other urologists. Where are they using?

Out of the gates, it was a recurrent pool. You had patients that had had some sort of endoscopic resection, and now they know their cancer's back. They know what it feels like. The anxiety starts to increase, and the physicians wanted another option. They had already done some sort of resection. It was clear that they thought they got it all. Because of the nature of this disease, it's hard to see, it's hard to get everything, and that's why it recurs early and often. We're starting to see now more newly diagnosed. You see physicians that have used it and hopefully seen success clinically. Now they're starting to sort of open up to our full indication, which, as you know, isn't just the recurrent pool. It's both recurrent and the newly diagnosed. It's both resectable and unresectable.

They understand how hard, which is why so many patients get an RNU. They understand how hard endoscopic resection can be in this patient population. That was the evolution and what we will continue to see. We'll always push to get those later adopters earlier. We've started to see that, whether that's solitary kidney or whether that's they've just seen their peers using it and having success. With regards to, Paul, you asked about the mixing. As everyone knows, we do have a mixing partner. We heard early on, well before launch, that some of these practices didn't have the capability to mix. We didn't want to force them to go to somewhere where they're not as comfortable, which is the hospital.

We want to be able to provide the product in a setting they're comfortable, integrate into what they normally do. The mixing partner has been great in the sense that they'll mix in the morning and courier it over, and the physicians will administer it in their clinic or in the surgery center. That's been something that's helped us not lose the part of the urologists that really don't want to mix or they can't mix.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Okay, great. Thanks for that. I guess, you've given us some metrics on reordering and increased utilization, I guess. What does it typically take? Does it take just one or two patients for a physician to be comfortable with JELMYTO and typically, I guess, to this point, what is your sort of, I guess, reorder rate or any update on progress there?

Jeff Bova
Head of Commercial, UroGen

Yeah, I think we're up to around 40 accounts that have treated more than one patient. It's actually growing on more physicians that have treated more than one patient. That's good too, because that tells me that accounts, it's just not the same physician in an account. You're starting to broaden the depth in that account. You're starting to get more physicians in there. I'm going to keep an eye on that number because that tells me what we've put in place from the training, from the reimbursement guidance and assistance we've been on filling out the form correctly, from the clinical efficacy, that they're starting to see that in their patients. Yeah, I'll keep an eye on that number. The latest that we reported in the last earnings call were 40. It's actually growing. It's higher than that.

That number is important to me because if we didn't put the logistics and everything in place to make it a smooth process, they wouldn't be looking for other patients. They are looking for other patients. That's good to see.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Great. Liz, maybe just you in the past have talked to the importance of the J-code in this application, which is a big driver for utilization among your potential prescriber base. On the coverage piece, can you maybe remind us, are there any sort of either government or private pay stakeholders that are still out there that might represent a chunk of the opportunity that has still yet to come on board from a payer perspective?

Liz Barrett
CEO, UroGen

Actually, no. The good news is that we're out there, the commercial payers are paying. The majority of our patients, the 70 %+ of the patients are actually in Medicare. That's actually also very helpful. As Jeff said, the nice thing about the J-code is that it's automated. I do think that to Jeff's point, some physicians are going to offices say, let me treat a patient, let me see the payment come in correctly, and then I'll look for more patients. There is a bit of a time lag that we talked about, but we are seeing that, and we're seeing that work. We're really pleased with where we are with both reimbursement and coverage. I think it's a pretty easy value proposition for payers.

They look at it and they say, okay, they can do this treatment, or they can go to a kidney removal. Everybody knows that it's not just the kidney removal that's the problem, right? It's the downstream impact of losing a kidney.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

That makes sense. Liz, maybe also for Jeff, as you think about the environment here as we're exiting the pandemic, and we've been talking about things opening up. You've presumably been targeting the low-hanging fruit or the high decile for prescribers as part of your initial launch effort. I guess for you, Liz, how do you think about stepping on the gas and targeting that next tranche of potential prescribers? Where are you in terms of your target accounts and rank ordering them and just when does that process for the next group of or the next opportunity of prescribers open up for you, and when do you plan to more actively promote there?

Liz Barrett
CEO, UroGen

Yeah, it's interesting, as Jeff mentioned, I'll let Jeff comment as well. In the beginning, actually, a lot of academic centers and the hospitals were using it. The reality of it is the top deciles are your community doctors. We actually haven't reached that first tranche, right? We haven't really gotten to all of those physicians to adopt because the patients typically see, and this happens also with oncology as well, you hear more about the academic centers. You hear more from the KOLs and opinion leaders. The reality of it is that patients get treated in the community. Over 70% of the patients get treated in the community, and a big part of it is in these LUGPA that you mentioned earlier, the large group practices, and that's where the patients are going in.

We still, as Jeff talked about before, in the beginning, it's really actually been more the institutions. What we really have to do is penetrate and continue to work on the community practices and the large group practices. We target about 1,400 of those accounts, but only about 500 of them have the majority of the patients. We've still a long way to go. The good news is, we've still a long way to go in even penetrating that first group. Having said that, the representatives are out there seeing everybody, right? We see everybody. It's just the frequency and the prioritization, as you can imagine, is in these large group practices, and that's where we need to see the penetration grow and the adoption grow.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Right. Maybe turning to your development and clinical efforts here. You've talked about your development plans for UGN-102 and the ongoing ATLAS trial. Where are you roughly, I guess, with regard to enrollment here? I had a couple more questions on it.

Liz Barrett
CEO, UroGen

Yeah. We haven't actually given guidance on the actual enrollment. As I mentioned before, we actually met our May enrollment. We have monthly enrollments as we hit that was really important. That was important because more centers are continuing to come on board. As you know, anytime you do a clinical study, I have never done a clinical study that actually in the beginning is where you want it to be, it is a hockey stick. Unlike revenue, maybe more of a slow and steady, hopefully not slow, fast and steady. Enrollment in clinical studies, especially big global clinical studies like this, do become a hockey stick. As you get more and more centers on board, we actually have high expectations for some of our Eastern European countries.

Those are coming on board faster, and we're starting to see that, and we are starting to see enrollment. The reason is because they tend to enroll more patients, even than the U.S. centers. We have more centers in the U.S., but we actually expect that we'll have higher enrollment outside of the U.S. It's an exciting time for us. We will start to share more specifics around numbers in the second half of the year. Suffices to say, we feel like we've hit a good stride, and we're continuing to bring on centers and look forward to that study enrolling.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Great. It sounds like since you mentioned earlier you hit your May target, that you're tracking broadly in line with your guidance for completing by around year-end or so.

Liz Barrett
CEO, UroGen

Correct. Yep.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Okay, great. I guess, for investors who may be unfamiliar with the non-muscle invasive space here, could you maybe just remind us what is the current treatment paradigm with regard to TURBT and what are its pros and cons here for patients in this population?

Liz Barrett
CEO, UroGen

Yeah, sure. I want to take a step back and talk about, like you said, bladder cancer, non-muscle invasive bladder cancer in total. I think that there is sometimes a myth that we're kind of misunderstood. People ask us a lot about BCG. Our drug, our initial UGN-102 medicine, is for low-grade non-muscle invasive bladder cancer. If you think about non-muscle invasive bladder cancer, there's about 700,000 people living with non-muscle invasive bladder cancer in the U.S. Actually, the majority of those are low grade or what they call superficial bladder cancer. The others are high grade. High grade is where you're seeing.

That's where BCG gets used, and that's where you're seeing all of the new entrants coming in. The KEYTRUDA of the world, some of the others that have had files but haven't yet gotten approval.

A lot of the studies you see are in high-grade, in BCG refractory or BCG unresponsive. What's important is UGN-102, the low-grade patient, there's about 20% of those patients are what we call intermediate-risk. Well, what's an intermediate-risk low-grade patient? It's somebody who has multifocal disease, they have larger tumors, and they recur often. These patients, and about, I would say, 70% of the patient population is actually recurrent. They recur. The average time to recurrence is really around six months. These patients get what we call a transurethral resection of the bladder tumor. Now everybody says, okay, no big deal. It is a big deal, right? Again, these patients are in their 70s and 80s. They're coming in for repetitive surgery.

They have to go under general anesthesia, and the physician goes in, the surgeon goes in and has to cut out the tumors. Jeff Bova mentioned earlier, he talked about what you can see and what you can't see. Mark Schoenberg, our Chief Medical Officer, often talks about it like a carpet. It's like, okay, you can see certain tumors, and you can cut out the tumors that you can see. There are tumors that are there in the wall that you don't see. What our drug allows them to do is actually treat the entire area.

These patients go through these multiple TURBTs, and there's some research that's been done that says 50% of patients would want a new option, but not even knowing what that option is, to going through TURBT again, because they've had multiple TURBTs.

Over about 68% of them had two or more in our study. Over 25% have had five or more. Again, this patient population. These patients today, again, standard of care is the TURBT surgery, going in under general anesthesia, having it cut out, and then waiting again, frankly, for it to recur. We're offering with UGN-102 an option. In our study, 65% of the patients actually had a complete response. When Kaplan-Meier analysis, 72% of those were still durable at 12 months. We're excited about the opportunity. We think it's another opportunity similar to JELMYTO, because again, you're talking about the upper tract, and you're talking about the lower tract.

The genetic and molecular makeup of the tract is very similar with the upper tract and lower tract, which gives us a lot of confidence, frankly, in the efficacy and safety of the medicine.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

You've designed ATLAS as a non-inferiority trial to the surgical comparator. Could you maybe talk about what your KOL feedback or physician feedback has been on that, using a non-inferiority endpoint, you potentially have the option to demonstrate superiority as part of your trial plan. Can you maybe just provide some context on how physicians view that?

Liz Barrett
CEO, UroGen

Sure. The trial is a superiority and non-inferiority study. It's not simply non-inferiority. Frankly, it's powered for superiority. We powered the study for superiority. We also know that physicians want an alternative. As I said, patients want an alternative, physicians want an alternative, particularly for this patient population that has these multiple recurrences and continuous recurrences. They call them surgical failures because the TURBT surgery just doesn't work. If they have an alternative, and these patients, unfortunately, there's not a lot of data that has followed this intermediate-risk patient for recurrence. One of the reasons that we want to make sure we have a superiority and non-inferiority is because there's a big, broad range of the patients, right?

Patients might be considered intermediate-risk, but maybe they're on the lower end, or you have some that'll be very severe. It's really important for patients and for physicians that they have an alternative. Look, we've been asked a lot about doing an adjuvant study, and we may embark on an adjuvant study. The reality of it is the biggest benefit to patients and physicians is if they can avoid having the surgery at all. They can avoid going under general anesthesia because we know that just going under general anesthesia multiple times, forget about the surgery, is an independent marker for increased mortality. If we can give an alternative, so physicians are very.

Jeff actually just did some market research that shows the interest in UGN-102 as an alternative for bladder cancer is significant among physicians. That's with a lot of people not really being so familiar with our medicine.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Great. You did speak to enrolling the trial globally here. Could you maybe speak to, first, what is the regulatory alignments between the U.S. and other key geographies? What is utilization of TURBT, I guess, on an international basis, and just how do ex-U.S. physicians think about the approach of using a non-surgical intervention versus the current standard of care?

Liz Barrett
CEO, UroGen

That's a great question. We actually are meeting with both Japan and European authorities in the next couple of months, we don't have the feedback from them on our current study. We want to understand today what are they looking for. We've been really focused on the U.S. with UGN-102 and making sure we've worked very closely with the FDA to design the study. To be honest with you, from our standpoint, we feel like we shouldn't have to go head-to-head against the surgery. We're probably the only company that's ever had to demonstrate the efficacy versus surgery. We're doing this study because it's what the FDA wants us to do, we have a lot of high confidence in that study. We expect to be able to use that globally.

We don't have our official feedback yet, but we will work with them. Having standard of care is the same. That's the good news. It's really the same around the world. Mark, again, has often talked about it's the technique of the surgeon, but the reality of it is it's the same around the world, and that's why we can do a global study, and that's why we can enroll as quickly as we can because we're able to do a global study, particularly, again, as I mentioned in Eastern Europe, where there are a lot of centers. They tend to enroll patients in clinical studies much faster and at a higher rate than they do in the U.S.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

We have a few minutes left. Maybe turning to the other parts of your pipeline. You are also having early-stage efforts in immuno-oncology. Can you maybe talk about where you are with regard to developing your CTLA-4 TLR agonist product and just when could we potentially expect a meaningful update there?

Liz Barrett
CEO, UroGen

No, it's a great question. We're excited about that. Our TLR7/8 agonist was actually something that UroGen acquired quite a few years ago from Telormedix. It's something that we did some pre-clinical work, not much had been done. When I came in, it's like, wait, we have this opportunity to look at our TLR7/8 agonist that was becoming a very interesting target for others. There's always been this sort of story about immuno-oncology, right? Can you take an agonist, an antagonist, and put them together, right? We often talk about take your foot off the brake and put your foot on the accelerator. Do the two work well together? There have been some studies that haven't really worked in this type of agonist/ antagonist.

We have found that in this case, the TLR7 agonist and coupled particularly with CTLA-4, we've talked about checkpoint inhibitors. We've tried checkpoint inhibitors, and we saw in our pre-clinical work that in the combination of the two, they actually work synergistically. We're excited about the opportunity to take the CTLA-4 that we licensed from Agenus for the local delivery. We put the CTLA-4 in our gel, and so what you do is you go in, you give the TLR7 in a water-based solution, and it kind of primes the pump, and then you're able to deliver the CTLA-4 locally. I think a lot of physicians are excited about delivering CTLA-4 locally because as you know, some of the systemic side effects have caused dose-limiting toxicities. The ability to do that locally we're pretty excited about.

We are working very closely with MD Anderson, advancing the two individually because we have to get the optimization of the dose, and we have to get all the tox work done individually before we put them together. That's the work that's being done now, and so we'll be able to deliver and move into the combination studies in 2022. I think that likely we won't be giving any efficacy updates anytime soon in the next few months. Hopefully, by the beginning of next year, we'll be able to show where we are individually and then what our plan is, and we're trying to accelerate as quickly as possible the combination. The good news, Well, I say good news, bad news is we're seeing some competition in this area, others doing some combination.

I think what that tells us is that it really lends credibility to our approach. We were the first ones to embark on this, so we're excited about this area.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Great. Maybe in our last minute here, Liz, you can talk a little bit about subsequent development and leveraging your RTGel platform here. You've had some external partnerships, and I guess you have this technology that could potentially be used in multiple settings, including outside of oncology. Just how do you think about BD and partnering here and leveraging your assets that you have right now?

Liz Barrett
CEO, UroGen

That's a great question. The RTGel technology, which is a reverse thermal gel that allows the delivery of a medicine to a cavity in the body and then the disintegration over time. Of course, sustained release, and it's voided out naturally, can be used actually in many areas. As you alluded to, we are doing a study with Johns Hopkins in GBM, excited about that area. It was one of the first ideas that Mark had when I joined the company. It's about putting the gel into the brain, and I think that's something that this is an area of high unmet need. You're right, in other areas outside of oncology, particularly in urology, we've had people, physicians, academic centers, other companies looking at it.

I think we are prioritizing, obviously, the areas in which we've announced already, but there are others coming in. Our approach will be outside of oncology and uro-oncology, we would partner that. We just have to prioritize where we want to be. We want to be a leading uro-oncology company. We want to work in specialty oncology. We will always prioritize those areas, and we'll work with partners in areas outside of that. Something we have a lot of interest in, we'll be able to share that and talk about that, I think, over the coming months.

Paul Choi
Mid-Cap Biotechnology Analyst, Goldman Sachs

Okay, great. We'll have to end it on that note. My thanks to Liz and Jeff and UroGen for joining us for this session. Thank you very much.

Liz Barrett
CEO, UroGen

Thanks. Thanks, Paul. Thanks for having us.