Welcome to this next in a series of fireside chats here at the Wainwright BioConnect Conference. My name is Ram Selvaraju, and I'm a Managing Director and Senior Healthcare Equity Research Analyst here at H.C. Wainwright. I'm joined by the next company on our roster, which is UroGen Pharma, traded under the ticker symbol URGN on the Nasdaq. We cover UroGen Pharma with a buy rating and 12-month price target of $45 per share. I'm joined here on the stage by Liz Barrett, Chief Executive Officer, and Mark Schoenberg, Chief Medical Officer. It's a pleasure to have you with us.
Pleasure, pleasure is ours. Thank you.
Thank you.
You know, for those who don't know, UroGen has two commercial stage products currently on the market. One of these was brought to market years ago, during the pandemic actually, for treatment of upper tract urothelial carcinoma, and the second, which was launched much more recently last year, is called ZUSDURI for treatment of low-grade intermediate-risk non-muscle invasive bladder cancer, and I think it's there that we really should start. You know, both these assets are based on your proprietary technology platform. I think, Liz, it would be helpful if you could just tell the audience a little bit about that, the reversed thermostable gel, and utilize a very well characterized and widely known chemoablative agent called mitomycin C.
I think also it would be really helpful to spotlight the non-muscle invasive bladder cancer opportunity just in terms of the total patient population and the unmet need.
Sure. I'm actually going to ask Mark to talk about the technology and then I'll come back on the market size.
Well, thanks. The proprietary technology RTGel is a mixture of inert polymers, a proprietary mixture that when cooled forms a liquid. It's sort of counterintuitive. It's, at a cool temperature it's a liquid, and when the gel or the polymers are instilled into a body cavity like the urinary bladder or the interior of the kidney and the solution rises to body temperature, it forms a soft gel that then stays in residence for four to six hours, slowly disintegrating, and it is by this mechanism that we're able to mix this proprietary set of polymers with drugs like mitomycin C to deliver them in a depot form and that forms the basis for the therapeutic effects that we see in our patients who are treated with these products.
When you think about the non-muscle invasive bladder cancer space, it's actually pretty complex, and in the U.S. alone there's about 800,000 people living with non-muscle invasive bladder cancer. The space where ZUSDURI is in low-grade intermediate-risk non-muscle invasive bladder cancer. There's about 60 recurrent, in the recurrent setting about 20,000 newly diagnosed that are considered low-grade IR. The pool of patients that are the recurrent patients get pulled from about 400,000 patients, and again, this is in the U.S. alone. Actually, low-grade is the more common. You would sometimes think that probably high-grade would be. We'll talk a little bit about both high-grade and low-grade, and Mark can describe the differences between that.
It's important particularly because our medicine that we just got approved, ZUSDURI, is for low-grade intermediate-risk non-muscle invasive bladder cancer, and there's a lot of characteristics that make it that, again, low-grade, and that's a disease of recurrence. It's not a disease of progression, and it's not a disease where there's a risk of mortality. There's a very, very, very low percentage of patients. What you're managing is the recurrence, and these patients unfortunately have a lot of recurrences over their lifetime.
You know, pertinent to the recurrences, historically before the advent of a drug like ZUSDURI, how were low-grade patients treated? You know, I think in particular, our audience would benefit from a description of the nature of the surgical procedure that was typically applied and, you know, to what extent is ZUSDURI capable of supplanting or replacing this type of invasive procedure for these patients?
Yeah. Mark, can you explain and describe a TURBT?
I'm gonna try. Endoscopic surgery for this particular malignancy originates in New York actually, at the beginning of the 20th century. Doctors to this day have put scopes with lenses inside the urinary tract and have applied various forms, excuse me, of energy to these tumors to ablate them and remove them. It's a surgical procedure done under anesthesia. It can be extremely complicated to do. It actually can take some time to do, and because the patient population being operated on is older and has comorbidities, it can be complex to recover those patients from these interventions. Nonetheless, until the advent of ZUSDURI, this was the standard of care for the management of recurrent disease in the population Liz has described. Patients would undergo with some regularity regular maintenance surgical procedures to remove recurrent disease.
In the context of ZUSDURI's, let's call it pharmacoeconomic as well as therapeutic impact on the lives of patients with NMIBC, Liz, as you said earlier, you know, this is a disease that people live with for a very long time. If historically recurrences had to be managed with TURBT, you know, approximately, you know, how many TURBT procedures might a drug like ZUSDURI, which, you know, you can please feel free to elaborate on the long-term clinical data.
Yes.
That's recently been presented at AUA, on ZUSDURI's long-term benefit, particularly in terms of complete response, right, which is putting people into remission. How many TURBT procedures theoretically could ZUSDURI save a patient from going through?
Yeah, it's a great question. You know, I also wanna go back to, you know, if you talk to a doctor, they think of a TURBT as being a fairly benign, you know, procedure, but you talk to patients, it is not a benign, you know, procedure. Trust me, the first time that I went with Mark into surgery to see a TURBT, I was like, "Oh my goodness." Recently, to your point, we're very excited about last week, we shared our 36-month data from our ENVISION study. What it showed was that 64% of patients that had got an initial CR were still in CR at 36 months. Why is this so critical? It's critical for a lot of reasons. One, first to say that our median still hasn't been reached.
Excited about the fact that what we know then is that the median is more than 36 months. Our internal projections is that it will likely be similar to JELMYTO, which is at 48 months. These patients, 23% of patients will have five or more recurrences. 68% will have two or more recurrences. To your point, it really depends obviously on the patient, but they would hopefully forego many TURBTs during their lifetime. The other thing about ZUSDURI, now we need to generate data on retreatment, but we do expect, assuming a patient gets a good response, that they would be able to be retreated, and that's something that we'll be looking to generate data. The good news, bad news is until patients recur, you can't really generate that data.
We will be looking to do that, but we're happy about the fact that the durability, which is the most important thing, you know, it's great our CR is about 80% and then about 80% of patients still in CR at 12 months, but to see this longer term data, very compelling.
You know, I think it would also be helpful to understand the competitive advantage conferred by the convenience of ZUSDURI. Maybe you could talk through, you know, the frequency of administration that is required with ZUSDURI and how that compares to both other already approved therapeutics as well as some late-stage agents that have been talked about extensively, you know, aimed at non-muscle invasive bladder cancer.
Sure. No, absolutely. One of the things that we'd love to start talking about now is not only recurrence-free, but treatment-free living. What do I mean by that? Our drug is you don't have to have surgery. That's the first and most important thing, especially for these patients. You come in once a week for six weeks, and then you're done. A lot of people, you know, asked us, "Well, why didn't you do maintenance?" One of the reasons was the burden of administration for patients. We wanted to see how the drug would do with just six weeks, and given the response that we've seen so far, we're really thrilled that we did that.
When you start to look at the other agents, and Mark can comment on this as well, the first thing is that they're an adjuvant setting, they are in addition to. When you're looking at the data, you have to take that into consideration. The data that they're sharing is a surgery plus the treatment. In almost all the cases, not only do they have the six weeks of, quote-unquote, "induction," but they allow a reinduction, and then there's maintenance beyond that. That's in both the high-grade and those that are being studied now in low-grade IR, that they are re-requiring both a surgery and maintenance beyond. We really think that we have a competitive advantage, and we're the most patient-friendly, you know, therapy in this space. You know, I think that's an exciting thing for patients.
I don't know if you have anything to add to that.
No, the only thing I'd add, and I think you've said it well, is that in our field right now, there's a huge emphasis on trying to examine possibilities for deintensifying cure, particularly for this population of patients. As Liz points out, ZUSDURI is six weeks and you're done. Everything else is surgery and then a year or two of additional therapy with multiple visits, potential installations of various devices for drug administration, much more burdensome. That actually figures importantly into how we're thinking into the future about how to cure for this population.
I think it would also be helpful to recap the commercial performance of the enterprise. You know, JELMYTO has now been on the market for several years. You know, I think it would be helpful to talk through the kind of revenue base you've been able to build there. More importantly, how ZUSDURI has done since launch, and particularly since the J-code came into effect at the beginning of this year, and the degree to which, you know, that is streamlining and facilitating access.
Oh, absolutely. As you mentioned earlier, we launched JELMYTO into the pandemic, about seven years ago, about six years ago, I'm sorry, for low-grade upper tract urothelial carcinoma. It's really important to understand that JELMYTO, because it's the upper tract, the installation is very different. You have to have fluoroscopy. You either have to go up and manipulate the upper tract, or you have to have a nephrostomy tube inserted. The patient population are only about 6,000 patients in the U.S., about 3,000 newly diagnosed and about 3,000 recurrent patients. We are around a $100 million drug a year. We've been growing at low single digits, we expect to continue to grow at that low single digit. There's still patients that need to get JELMYTO, very similar to what you see in bladder.
Every year, a certain number of patients will recur and have the opportunity potentially to see JELMYTO. What, you know, JELMYTO also allows is for these patients not to lose their kidney, and we are talking about an elderly patient population. When we think about, again, JELMYTO, it's about $100 million. If you compare that to ZUSDURI, one, the market is about 10 times the size. You know, we often talk about ZUSDURI being a $1 billion plus, you know, peak revenue medicine. That's easy when you say, "Well, we're already with JELMYTO at about $100 million, 10 times the size," you sort of get there pretty easily. There are a lot of important factors that go into that. One, it's actually a much simpler product to give.
While they're similar, it's much easier because in the physician office, particularly in the community practice, it's very easy to insert the catheter into the bladder, deliver the medicine. It's very similar to what they're used to doing with other intravesical therapy, it reaches the market. What we shared in 2025 is that in Q4, there was about $14 million in revenue. This was pre-J-Code. We received our J-Code on January 1st, we've reported $29.2 million of revenue in Q1. What we've also said is that we're comfortable with where consensus is, both for the quarters and for the year, we expect linear growth. We do expect to continue to see quarter-over-quarter growth.
I think the other thing we shared when you try to compare it with JELMYTO was in February, already in February, the second month on the market, we saw that our the early indicators that we look at, patient enrollment forms, new patient starts, and obviously number of doses, surpassed JELMYTO in February. We expect that to continue to grow. Look, it's not rocket science to say if you're at $100 million of JELMYTO revenue and you already surpassed them in February, Now, of course, the pricing is a little different. ZUSDURI's a little bit less costly than JELMYTO , but it's easy to get to seeing where we are from a run rate perspective.
I think it's also important for our audience to reflect on two key things. Both JELMYTO and ZUSDURI are proprietary products.
Yes.
that UroGen sells. The fact that you had all of this commercial experience accumulated with JELMYTO , the fact that you had to launch it into an adverse market environment, means that this is a very battle-tested commercial organization, and the two products are highly synergistic.
Yes.
The prescriber base is clearly heavily overlapping.
Absolutely.
Because of the presence of JELMYTO and the revenue base that you have already built, the sales force effectively pays for itself, the sales force that you are using.
Yes.
to sell ZUSDURI.
Absolutely. We did increase the number of our customer-facing, you know, people, employees when we launched ZUSDURI because, you know, for you had about 5,000 targets to move to about 8,000 targets. To your point, again, JELMYTO , there's only 6,000 patients, but they're being seen by everybody in urology. The difference for ZUSDURI is everybody's seeing these patients, but they're seeing multiples of these patients. While, you know, every doctor who uses JELMYTO uses ZUSDURI, not every ZUSDURI doctor is a JELMYTO doctor. We are seeing even some what I call reverse halo. You go in to talk about ZUSDURI and you also talk about JELMYTO .
Where a physician may not have been willing to use JELMYTO in the past, for one or two of their patients, if they start to adopt this type of therapy for ZUSDURI, they may be more likely to adopt it for upper tract as well.
With respect to how you expect overall the customer mix to evolve for ZUSDURI, you know, talk a little bit about how ZUSDURI is priced, how you expect that to compare to existing marketed as well as late-stage therapeutics in the NMIBC context, particularly for low-grade patients.
Right.
How this may affect the degree to which ZUSDURI winds up being ported through the community hospital channel versus the academic center channel, and what implications that has ultimately for volume.
Yeah. It's important to note that most of these patients get seen in the community. About 70% of these patients will initially be in the community. You know, Mark works at an institution, and they typically see the worst of the patients, right? Either after a patient has recurred so many times, but the majority of the particularly low-grade patient is seen in the community. In the beginning of the launch, though, most of our usage was in the institutions, and it was there driven by reimbursement. To your point, one of the dynamics that occur are community practice are much more concerned about the financials. They wanna make sure that they get reimbursed because it is their private practice, and so if they lose on one patient, they're very concerned about that, right? The revenue that they lose.
We do a lot to support the community doctor, and they wanna make sure they get to keep those patients. While everybody talks about TURBT being the bread and butter of urologists, frankly, being able to do intravesical therapy in the office is much more, from a practice economic perspective, it's actually more beneficial to the doctor. While right now we were at 60/40, in 2025 we're already at 50/50, we expect that to flip, and you'll probably get about 65% of the revenue coming from community. Not only is it because that's where the patients are seen initially, but doctors figure out pretty quickly that it fits into their workflow and that it can be beneficial for them and their practice.
I think it's important to note that, you know, many times in the community setting, the deployment of an extremely expensive drug is extremely difficult.
Yes.
You know? Some of you may have heard about 340B and the impact of that. Because ZUSDURI is priced the way it is.
Yes.
That is not an issue, and it will not be an issue in the future.
Right. Yeah, when you compare it, to your point, against some of the other medicines that are on the market, first of all, they're starting in high-grade, and so their pricing is priced for high-grade. We start in low-grade and our pricing is for low-grade. You know, you're not going to use a million dollar drug in a low-grade patient that you know doesn't have the risk of mortality or, you know, of moving. It's gonna be interesting to see how things play out when these expensive high-grade drugs move into the low-grade space. Unfortunately, the intensity of the amount of drug they're getting is actually still the same even though it's in low-grade, but the way that they are, the way that their clinical studies are and the way their dosing is they're still getting the same amount.
It's gonna be interesting, and I do believe urologists are much more price sensitive than, say, oncologists are. How are they going to position themselves in the low-grade space from a pricing standpoint will be very interesting. I think, you know, it's gonna be not only a physician decision, but a payer decision down the road as well.
I think it would also be important to, A, highlight the complex nature of the formulation, both in the case of JELMYTO as well as in the case of ZUSDURI. You know, those in the audience who know some of the work that my group has historically done in the specialty biopharmaceutical space know that I tend to gravitate towards situations where the product, for want of a better term, is not readily genericizable.
Right.
Presents myriad challenges to a potential copycat entrant. I think, you know, that's an appropriate place to start when thinking not only about how difficult JELMYTO and ZUSDURI might be for generic companies to copy and even enter the marketplace, but also the life cycle management strategy that you already have. Certainly, I think the centerpiece of this is the recent data that was generated.
Yes.
from the UTOPIA trial with the follow-on, the successor product to ZUSDURI, especially now that you have six months CR data.
Absolutely. I'll ask Mark just to talk, one, about sort of our products and why they're unique and why our technology is unique, and then our life cycle for moving, and then I'll sort of chime in on the sort of commercial part of that.
Because of IP considerations, although actually because we were looking for a secondary supplier of mitomycin, we came upon an opportunity to create successor molecules to JELMYTO and ZUSDURI using a proprietary version of mitomycin made by a company called medac in Germany. In partnering with them, what we realized was, and I'll defer to Liz to talk about the IP implications of this, an opportunity to pair this specific mitomycin with our gel technology to create two successor molecules, which are currently named UGN-103 for ZUSDURI successor molecule, and UGN-104 for JELMYTO, which are in the final phases of phase III evaluation. 104 will be submitted as a new NDA in the third quarter of this year with the expectation through a 10-month review process of approval in 2027.
Liz will tell you a little bit about the launch strategy and withdrawal of ZUSDURI that will follow that. 104 follows all of that by about a year. It is expected that we will complete enrollment of the 104 trial this year and approval will be in 2029. We have two additional drugs that will follow the path of JELMYTO and ZUSDURI. In addition to those opportunities that you'll hear about in a second from an IP perspective, the complexity of the gel, making the gel, has been alluded to already. We've had large pharmaceutical companies approach us for help in trying to figure out what we're doing. Liz may wanna comment further on that.
The gel itself is exceedingly difficult to make, in large part because there are some very subtle changes associated with achieving the parameters required in order to deliver this to the urinary tract. In any event, we have two successor molecules that will be available in the market long before our IP lapses on the existing drugs that are currently being marketed. I don't know if you wanna comment further.
Yeah, no, absolutely. As we roll out You know, it was interesting because to Mark's point, when we originally went to the FDA and said, "Well, what can we do in our new molecule?" They said, "They're different, you're going to have to run a clinical study." Since then what they've done is develop what we call product-specific guidance, that says that anyone coming in is going to have to demonstrate that they are identical. There's no such thing, they say, there's no biosimilar, there's no similarities, there's no equivalence. You must demonstrate you're identical, if you can't do that, you're gonna have to run a clinical study. Where we are with, I think everybody knows that Teva filed an, you know, an ANDA.
They still have not received conditional approval from that, and so that's telling for us. They also narrowed their claims, in the lawsuit because it used to be non-infringement or invalidity. Now they're saying, "Well, we infringe." There's just an invalidity claim, which we know is a higher bar. We feel very strongly about our IP. We feel very strongly about the know-how, you know, based on what Mark was saying. In addition to that, the expectation is that as we roll out UGN-103 and UGN-104, we will pull ZUSDURI and JELMYTO off of the market. Even if someone post-2031 figures out a way to design it, then they're going to enter into a market where there's generic substitution. There's not interchangeability.
They would have to launch it as more of a branded, you know, product. We feel, again, really good about where our patents are. UGN-103 and 104 go through 2042. That allows us to launch the new studies that Mark was talking about. We have a long time to be able to study and hopefully launch in areas around urothelial cancers. To your question the other day, absolutely expect to be able to show ZUSDURI and JELMYTO's use in across the urothelial cancer landscape.
I think, you know, just some parting thoughts. Firstly, our valuation does not give you credit for 103 or 104. You know, we are effectively modeling gradual erosion of both JELMYTO and ZUSDURI sales starting in 2031. Clearly, if you are successful with the LCM strategy involving 103 and 104, the market exclusivity period could be extended by more than a decade. Secondly, you know, you alluded to this a little bit, you are looking at the possible applicability of your technology of mitomycin as a chemoablative agent, you know, not just in low-grade, but also in high-grade. You also have a whole pipeline beyond all of this, you know, including an oncolytic virus candidate, which we haven't had time to talk about today, which aren't included in our valuation assessment either.
I think those are important things for our audience to kind of reflect on. That may be, I think, where we're gonna have to leave it. Thank you so-
Although I think you have to raise your price target is what you're saying. Thank you.
No comment. Thank you so much for being with us.
Thank you.
Really appreciate your telling us and telling the audience about UroGen Pharma. Please keep an eye on this company. To our audience, thank you so much for your attention.
Thanks. Thanks, Ram.
Thank you.
Appreciate it.