We can get started. Hi, everyone. I'm Tara Bancroft. I'm one of the senior biotech analysts here at TD Cowen, I wanna thank you all for coming to TD Cowen's 46th Annual Healthcare Conference. For our next session, we have a fireside chat with UroGen. From UroGen, we have the CEO, Liz Barrett.
Hi.
Thank you so much for joining us. It is such a privilege to have you here.
Well, thank you. I really appreciate the opportunity.
Before I get started, I wanna say to anyone who's listening in the audience that you can raise your hand, stand up, shout at me, anything you want. If you wanna get heard, I will make sure that it happens. In the meantime, Liz, I wanted to see if you had any high-level thoughts you wanted to share, an overview, recent highlights from your earnings.
Yeah, sure.
Anything you'd like.
Well, great timing, right?
Yeah.
As we were scheduling our earnings call, one of the dates was yesterday. It's like, well, that makes a lot of sense, right? Because we're going to the conference the day after, so we'll be able to answer a lot of questions that next day. It was interesting because it was our year-end, obviously, so it's a, I mean, a bigger amount of time. Look, we have great results. We feel really good about where we are. UroGen's in a very unique position, both from our own life cycle of a company, but then also as a biotech company. The reason I say that is there's not very many biotech companies that have two commercial products and the opportunity to really build a long-term sustainable growth company.
For us in UroGen, now that I've been here seven years, which is a little bit scary, we are finally at the point of changing our company. It's a transformational year for us any way you look at it because we had got JELMYTO approved for bladder cancer. That was actually the impetus of the company being founded many, many years ago before my time, and we're here. What we talked about a lot on the earnings call was obviously our financial results and where we were, but what are we seeing now that we got our permanent J-code on January 1st? We did some financing, some debt refinancing, we're in a great position from a financial standpoint. Also our head of chief medical officer is here.
We're doing a lot around clinical development. Where are we going from here? With the goal of really finally realizing our strategic vision for our company.
Great place to start. I feel like a good way to go through this, obviously, we'll talk about the JELMYTO launch in the beginning may by timeline. We'll start with kind of Q4 details.
Mm-hmm.
You know, you guys posted a great Q4 above expectations, and maybe you could talk a little bit about the dynamics that you saw there and why or why not it met or didn't your expectations.
Right. Yeah, absolutely. We had been messaging coming, you know, up through Q4 about what we expected. We did something very unique in our Q3 earnings, and that's we shared October. The reason we did that was very purposeful, and that is because our launch, when we launched this medicine, this is very unique in the sense of it's not a traditional pill or infusion, it's a procedure. So there's a lot of time lapse between the time you identify a patient that you wanna treat and actually getting to treat that patient. Account set up because it's a buy and bill drug and because the drug has to be mixed with the gel, there's time associated with that. Because of that is the only reason we shared sort of October numbers.
We had October about $4.5 million, you know, everybody's looking at us like, "Okay, well, for Q4, do you expect then linear growth month-over-month?" We cautioned around that, I think from, you know, consensus perspective, they were right on understanding that as we get into November and December, not only do you have the holidays, but now all of a sudden you're coming closer to the new year when we will have a permanent J-code. Why is a permanent J-code so important? The permanent J-code for physician offices gives them confidence that they're gonna get reimbursed and that they're going to get reimbursed in a timely fashion. You can imagine a lot of these, physician practices, you know, they have to outlay money. They consider these to be expensive medicines.
We're the only one in low grade. Other medicines being introduced early. The permanent J-code was very important to them. Interesting, I spent the last 30 years in oncology, oncologists could care less about a J-code, you know, the permanent J-code, miscellaneous J-code. That's because they've for so many their entire careers have done buy and bill drugs. Their office is set up for it. They have the infrastructure. That's not the case in urology. In urology, buy and bill drugs are fairly new phenomenon for them. The thought of having to outlay the money and then, you know, all they need to do is lose 1 patient or, w e can't guarantee reimbursement. Wish we could. We can't. Because of that, they're taking a risk. The permanent J-code was really important.
As I was out in the field after launch, the doc, "Come see me when you get the J-code. Love the drug, can't wait to use it. Got several patients I wanna use it on. Come see me when you get the J-code." We did see that phenomenon happen. That's why we cautioned around Q4, we were able to, you know, to meet consensus and, you know, to your point, beat a little bit. For the year, we had the $14 million and the $1.8 million that we had in Q3 for JELMYTO. We're excited about kind of where we are. We met our goals exactly where we expected. In Q1, we're even more excited about the acceleration.
What we thought we would see, what everybody was kind of anticipating, we saw an acceleration after the J-code. We're still early, right? We're two months in, still early, but we've tried to message as much as we can that we are seeing that acceleration happening in Q1. Q4, great. We feel like we were right on track with where we needed to be, both with ZUSDURI and JELMYTO. Good coming out of the year with some good momentum with Patient Enrollment Forms, you know, identified accounts and physicians who wanna use the medicine.
Okay. Let's see. You mentioned Patient Enrollment Forms. I'm thinking, you know, a good thing for us to understand would be what kind of data do you guys have eyes on?
Yeah.
I know that you guys have this hub, which has just the plethora of patient-level data. Can you tell us what kind of data do you guys have in-hand that you can assess and learn from as this drug launches?
Yeah, no, it's a very unique sort of situation in the sense that about 80%- 90% of our patients go through the hub. Not everybody does. Hospitals might, you know, just order directly from the specialty pharmas, a specialty distributor, and you won't ever sort of see that. Most of them go through, and they fill out what we call a Patient Enrollment Form. You don't get a ton of patient-level data from that, but it is a script. We know when you get the script, we know when those scripts get filled.
Not only is the hub, do they take the script, but they do the benefits verification, then they also are the ones who coordinate and triage the actual shipment of the drug from the specialty distributor, either to a specialty pharmacy or directly to the account. An account can order it not mixed, they would just get it directly from the specialty distributor, or they order it mixed. Most community practices want it mixed because they don't. One, they don't have a hood, 'cause it came out therapeutic, and two, they don't want the hassle. They don't have the manpower. Most hospitals actually don't want it mixed because the pharmacy and the hospital, really, it's more around a quality control issue. They wanna maintain that quality of quality control. They typically don't order it pre-mixed.
We do know who these patients are, so we know what, you know, that they have low-grade, intermediate-risk non-muscle invasive bladder cancer. So we have all of that information, so we get that patient-level information. A PEF is our first indication of demand, and there we have had great success. When I say one of our challenges, we're getting there, but it's taking 45- 60 days. One of the greatest things we'll be able to do is shrink that time from a Patient Enrollment Form to a new patient start, and that's when you actually start to generate revenue. Importantly, that's when a patient gets treated, and you want these patients to get treated.
A Patient Enrollment Form only comes when you have a patient identified and you know the doctor wants to use the drug on that patient.
Okay, got it. Part of the reason why I'm asking is, you know, 'cause especially leading up to the Q4 report, one of the biggest questions that we all had was, well, what kind of metrics are-?
Right
are they gonna share? It begged the question of excuse me, of well, what patient do they even have?
Right.
maybe could you explain a little bit considering you have PEFs, you have new patient start information, so maybe the rationale for why you provide the metrics that you do, like the sites that are activated.
Yeah
H ow many, you know, who is prescribing
Doctors using it. Yeah.
W ho's repeat prescriber, et cetera.
Yeah. The reason that we don't share the Patient Enrollment Forms is really because it's not consistent. You have this time period, you know, you have this sort of up to 60-day time period where the patient doesn't get dosed. They fall out. Like in steady state for JELMYTO, you've got a 70%-80% conversion. What I'm, Our fear of sharing that is it's not sustainable and too much will be read into it. That's why we like to talk about accounts activated. What does an account activation mean? It means that they can order the drug. They're not going to go through the process of filling out a new account form and all of that to get to a specialty distributor if they don't have interest in the
They clearly have interest in using our medicine, or they wouldn't go through that. Now we've got 800 of those accounts set up. That's great, right? That they're set up. That doesn't mean that they've actually written though, and that's been 1 of our focus in Q1 is all of those accounts that are activated, get them to generate a prescription. You forward to how many doctors have actually used it, right? You've got 100 doctors that use it. How many of those are repeat prescribers? That's important because you know if they're a repeat prescriber that they've had a good experience and that they plan to use it on more than one. We talk often you know, about, you know, crawl, walk, run.
You know, a lot of doctors will use it on one patient before they go to the next patient, and once they get experience, then they're going to use it in a broader patient population. That's kind of where we are now. We actually do have some examples where not everybody did crawl, walk, run. They ran right away, and they knew, and they were, they're very much champions of the medicine.
That repeat prescriber number is really important for us. It's great to have accounts activated and that what our strategy coming into the year has been fish where the fish are. We know if account activated that they're interested. We know if a physician has used it, that they're interested, right? We know a lot of the physicians said to us, as I've mentioned before. I wanna use it.
Come back when you have a J-code. We knew who they were. That's been our priority, is go back where we know that there is interest in using it. How do we build the breadth and the depth in those accounts? Because you might have one doctor in an account that wants to use it, but how do you go from one doctor to two to three to four? That's what we're focused on right now, and we're seeing more and more of that.
How do you think you go from one to two to three to four?
The one thing that I've been really pleased about seeing so far is that the patients are there. I think that's one of the questions in the bladder cancer market more broadly is because it's a new area for most drug companies, there weren't very many or weren't really any, except generic BCG and generic some of the other ones being used in these spaces. Is that really a 5 billion dollar? Is there really those patients? What I'm learning since our launch is those patients exist. The fact that we have one doctor in New York City who's treated 15 patients since launch, that tells me their patients are there. When we talk to doctors, it's not, "Oh, yeah, one patient or two patients." They're talking about the several patients. I think that's how you go from there.
They have to buy into the approach. Do they buy into chemoablation? Do they buy into using these medicines instead? That's our job, right? Our job is to obviously give them the data that they need and educate them on why this is a better way to treat patients. These patients haven't had a new option for a long time, you know, a TURBT to a urologist who does TURBT, you know, all the time, no big deal. To a patient, it is a really big deal. It's not only about avoiding the surgery, but the data that we have, I mean, in my lifetime experience in oncology, I've never seen an 80% complete response rate and 80% of those patients still in response at 12 months.
That in and of itself is the best. The best thing we can do for patients is give them recurrent free, but then also treatment-free living. Like, they don't have to worry about it because you hear more and more about, "I'm always wondering if it's going to come back.
Mm-hmm. Yeah, it's hard to do better than 80% CR, right?
Yes, it is. I think it is. Especially without surgery, right?
Exactly.
Mm-hmm.
All right. Maybe you could tell us more about the practices that these champions are at. Like, what kind of patterns are you noticing? Not just in the champions, but of sites that are activated, starting a prescription, becoming a repeat prescriber, and then becoming a champion. What is the difference between all of those?
Yeah.
not necessarily describe each of the spectrum.
Right
Who are these champions that you probably don't need to be promoting with, right?
Right.
Also curious on the same breadth of who are the sites that are activated that are not prescribing it?
Right. That a great, really great question, particularly I think we will follow a more traditional adoption curve where you absolutely have your physicians who say, "I am not gonna really use that drug. I'm only gonna use it in a very small percentage of patients," to the others that say, "I'm gonna use that in every one." We do have already both of those sides. Most people are in the middle, right? Most people are in the middle. The champions are those who probably, even before we launched our clinical study, bought into that this is a good way to treat patients.
One, you know, doctor, and I hope she doesn't mind me mentioning her name, Jennifer Linehan in California, I remember when the data came out, she had always been a proponent of ZUSDURI even before we had the data. Once we got the data, the question was, "Well, who am I not gonna use it in?" It used to be, you know, "It will be a great tool in my armamentarium, so who will I use it in?" Now, it's more given the data was as strong as it was, that becomes a champion. This person that I talked about, physician in New York, who's used it, he buys into the fact that this is a better approach to treating these patients because of the disease.
You know, the disease is, it's a non-muscle invasive. It's what you can see and it's what you can't see that's lying. They understand the fact that this drug can get to those areas, can kill those tumors. You know, we had one doctor that talked about, actually, they were a non-responder in our study, but he said, "I looked in, and there was like 70 tumors. And there was no way I was going to get that. And I gave the drug, and it got rid of almost all of it. But there were still some, so it was a non-responder." The thought that going into there, that they could actually see that type of response, those are the ones that become champions. I feel like as they use the drug, that's the one.
When they see it on their own patients, that's what we hear over and over again. They actually see it happen. They talk about the tumors melting away. I think that's how you get champions or those that probably already bought into it. When they see it happen on a patient, like somebody who they find difficult, like, I mean, we hear stories of patients having three times a year, having a TURBT, and then all of a sudden not needing. Those are the patients that it's right for. I think that's how you build champions.
Okay. Great answer. I wanna make sure we get enough time to discuss 2026.
Yes.
You know, I know you mentioned that you're seeing an acceleration, which is great to give us this kind of qualitative insight into what you're actually seeing. I'm curious, you know, it's very early stages, we do understand. What would it take for you to gain enough comfort to provide guidance?
No. I think once we need two quarters. That's my, I think Chris and I both agree that we need two quarters. We need to see Q1 and Q2. I think after that, you know, we will make a decision as to whether we provide more guidance. What we have said from a guidance standpoint are a couple things. We haven't been completely. We've said we're comfortable with consensus, you know. That's some guidance, right? We're comfortable in Q1, we're comfortable for the year with guidance. I think that's saying something. I think the other thing that I shared on the call, on the earnings call yesterday and shared today in some of our meetings, is what was really exciting to me was the week.
'Cause we get these weekly numbers, right, of PEFs, NPSs and doses, and I was, like, writing them down. JELMYTO, ZUSDURI, JELMYTO, ZUSDURI. At the time that I saw consistently that ZUSDURI surpassed JELMYTO in all those metrics. PEFs were easy. That happened early on. We were seeing more Patient Enrollment Forms, again, showing you that patients are there. The second being new patient starts and the third being the doses. When I was able to see several weeks of a consistent ZUSDURI having more doses than JELMYTO. What does that tell you? Okay, JELMYTO's about a $100 million drug. In February, the number of doses for ZUSDURI was higher than the number of doses for JELMYTO. You know, you can look at the run rate and say, "That's why we're comfortable with consensus." There, there's.
It's not like we're not telling anybody anything. That gives us a lot of confidence, and that's exciting to sort of see that. Again, because you're talking about a patient population that's much greater than JELMYTO. That tells me the patients are there, physicians are starting to buy into it, and we're seeing that activation go from a Patient Enrollment Form to actual getting the patients dosed.
Okay. Yes, definitely appreciate that. Considering that you're now exceeding JELMYTO's run rate, you know, in the past, you had talked about once the J-code goes into effect in the first half, you know, types of growth that we can look to is more like JELMYTO or Activa.
Mm
W hich are very different first half
Yes
G rowth rates, right? Maybe we can look more towards that Activa type percentage growth?
Well, no, absolutely. What we've said was, and even in Activa, we'll talk a little bit about that, but with the second six months versus the first six months, and that was kind of the
Mm-hmm
P re J-code, J-code era, where you saw that 220% growth in Activa. What we said we're comfortable, that our first six months with a J-code will have a similar growth rate to that. That's good. What we've seen so far, and I was looking just at the Activa number for the second half of the year, maybe the growth wasn't as great in Activa. We are expecting better growth than they've seen.
Yeah
I n the second half of the year versus the first half of the year. Yeah, I can gladly say that, 'cause at that point in time, we didn't have that information. There aren't any. The problem is there's not great analogs out there any way you look at it. But we do like the Activa six-month post J-code, six-month pre J-code, and JELMYTO, again, that timeframe, but not the actual numbers. We believe with ZUSDURI, because the patients are there, much greater patient population, and a much easier to use than JELMYTO, that we'll see that continue to grow.
Okay, great. I do wanna spend some time on a couple of things in the pipeline. Before we do that.
Yeah
I feel like a good thing to mention would be the refinancing that you guys.
Yes
M entioned yesterday. What I think we're wondering is, does this imply at all that you're changing how you're thinking about a path to profitability at all?
Yeah. Not at all. It's one of the things I think may have been confusing with our announcements yesterday. Yesterday we announced the refinancing of our debt. Think about it in the context that we are a very different company, as we said now, than we were when we initially did the debt. Our ability to finance the company is much easier now, and we have a better profile. We actually ended up with reverse inquiries, companies coming to us saying, "Hey, I'd love to refinance your debt." Cowen supported us in a process in which we actually did that. You know, we went out and talked to. We were able to get very favorable terms from our wonderful partner, Pharmakon, so thank you very much. They're great partners, always have been.
Love working with them, and they too understand that we are a different company than we used to be, and that the risk profile of our company today is very different than it was when we did the initial. Look, we took the opportunity to take additional debt, to additional money to add. It did not mean that our path to profitability was different. What it does for us though, it gives us more headroom, it gives us more flexibility. One of the things that, and at that point in time we didn't have our 103 data. Now that we've seen the response rate from UGN-103, we have patent protection to 2042. That allows us, in speaking about the pipeline, to really fund the ability to have life cycle management and other areas with UGN-103.
While we did have some of that built into our current assumptions, this gives us more flexibility to maybe accelerate some of those things in R&D. UGN-501, we wanna make sure that we take advantage of that as quickly as we can and get that not only into bladder cancer, but potentially into other cancers as well. It does give us a lot of flexibility from that standpoint. It doesn't change our trajectory that the current cash that we had could get us to profitability. It's just a matter of how much headroom, how much more can we spend to really build our company for the long term.
Okay. Perfect. Let's do 103 first.
Okay.
There's a lot going on with that. Can you give us just the cadence of milestones?
Sure
T hat you're expecting now? What's next, and then after that and after that?
Absolutely. Well, UGN-103 is our next generation formulation. It was about ensuring supply. We wanna make sure that we're able to supply the patients. What we found when we went looking with Medac is that they have a proprietary mitomycin, which they had patent protection to 2035. In talking to the FDA, they said, "No, it's a different mitomycin, so you have to do a study." Kinda good news, bad news, right, for us in the sense of t he good news is because of that, we were able to get additional patent. We have patent protection on UGN-103 to 2042, so that allows us to have the time to be able to grow through life cycle management. Look, there's not
We expect the clinical data to be very similar, but there are some cost of goods, manufacturing, timing, again, supply, as well as it's more soluble, which makes it easier and quicker to administer, and so there's administration benefits to it. The expectation right now is we got our CR rate, and it was almost identical to ZUSDURI. Now we're waiting on durability. That should be about midyear we'll have some durability. In our discussions with the FDA, what they're allowing us to do is file without everybody at 12 months. We can start to file early, and then we'll update the file with everybody at 12 months. Durability is really what we're waiting on. Midyear you'll hear about durability, and then we'll file and then hopefully get an approval in 2027.
What we'll do is we'll wait for the J-code because we know how important the J-code is before we would launch. We'll launch, and we'll have a switch strategy with 103. Again, even prior to that, we're starting additional studies in other areas of bladder cancer.
Like high-grade.
Like high-grade, yes. I mean, the reality of it is there's no reason to believe that it won't work, you know, in this, in high-grade. It will be treated differently 'cause it will be an adjuvant. In high-grade, you definitely all the medicines are adjuvant. We will have longer duration of treatment because we'll wanna do, you know, we'll wanna do maintenance therapy there as well because it is high-grade, you know, cancer. From that perspective, we have that opportunity. We also wanna do adjuvant in low-grade intermediate-risk, particularly with a newly diagnosed patient. We know from our own ATLAS data that we can be successful there. We expect to do that as well.
With that, how does 501 play into that? Would you be competing with ZUSDURI at all?
Maybe. You know, but maybe, but that's okay, right? I'm a big believer in the best thing we could do is obsolete ourselves before someone else does. You know, UGN-501 is early. It's an oncolytic virus. We believe it's a best-in-class differentiated asset. It not only has a direct tumor kill, obviously it elicits the immune response. You have this sort of dual action virus to sort of work with. The other good news about is 501 works outside the bladder. Obviously, our initial one is in the bladder. We also think we can differentiate by potentially putting it in the gel. Can the longer exposure of the virus help at least from a dosing standpoint or something so that you don't.
You can dose less frequency maybe. You won't need the maintenance that, you know, that others have. From that area, we believe there's a lot of opportunity. The preclinical work that we're doing and the experiments, and we'll start to publish that in the second half of this year, are showing us that it's a very potent virus. We believe because of that, again, we can be best in class.
Okay. We are very excited to see that. I wanna end on a broader, more general question.
Yeah
A nd ask you, I love to ask this every year, what do you think is the most underappreciated aspect of UroGen right now?
Oh my goodness.
choose one.
D o we have an hour? No, as, you know, I ZUSDURI. I can't. I mean, it has to be ZUSDURI because if everybody really believed what we believe, and that is that we will be able to easily treat 20% of these patients, but we think that's being very conservative, That's a $1.2 billion opportunity for us. The valuation of our company barely takes any of that into consideration. I think that has to be the most undervalued, you know, part of our story right now.
Perfect.
You know what? We'll prove it. That's all I can say.
I love it. Yes, I agree. All right. We are out of time now.
Okay, great.
Liz, thank you so much for your time.
Oh, thank you, Oliver. It's always a pleasure.
Thank you to the team for being here, everyone for listening.
Thanks.