Welcome everyone to the very last session of day two of the Cantor Conference. I think you'll all be well rewarded by our fireside chat with UroGen Pharmaceuticals. My name is Eric Schmidt. I'm one of the analysts at the company, and it's my distinct pleasure to welcome Liz Barrett, who's the company's President and CEO. Hi, Eric. As well as Mark Schoenberg, the company's Chief Medical Officer. We have Christopher Degnan, the company's CFO, and Vincent Perrone, who's the Senior Director of IR as well, here with us. Thanks for the whole team coming out today. Really, I guess, exciting last few months. Maybe less exciting for you and more exciting for those of us who get to watch from the front row. Liz, why don't you just quickly recap what all this excitement has been about and the fortunate position you are right now to commercialize your second of two drugs? Yeah, we're just getting Mark out of therapy, so he's doing fine after everything that happened at the ODAC. Look, an exciting time for us, as you said. You know, we got our approval. It was bumpier than we expected, although some people said it was a gift, so they were able to get in a little bit early and low on the stock. Good for those who understood what was going to happen. Exciting time for us. This has been, I would say, the culmination of the company. When I talked to all of the team that was around when the company started, it was always about bladder cancer. It was always around this indication. We were able to get Jelmyto earlier because of high unmet need with patients losing their kidney. Bladder was always the, you know, sort of the place, the gold ring that everybody was looking for. To be able to see this milestone finally happen from the company for so many people in our company and our investors and the physicians and patients, this is a very exciting time for us. Liz, just let's step back and take a higher level for those of us who are less familiar with the company. What are the products that we're talking about? The technology enables them both. Sure. We'll talk a bit about Jelmyto before we get into the more exciting bladder update. No, absolutely. Next, I'm going to ask Mark to talk about the history of the company and our technology. Obviously, I'll probably add in there. Mark, you know. Sure. Okay, so you know, start out with a urology joke. A urologist walks into a cocktail party, and that's literally the origin of this company because apparently in 2004, a urologist ran into a material science chemist at a cocktail party in Israel and asked the question, can you make drugs stay in the bladder longer? The genesis of a very interesting research program that led to the development of our core technology, which is RTGel, the reverse-thermal hydrogel that permits us to put a variety of chemicals and drugs of various molecular sizes and proportions into the bladder and keep them there in the urinary tract for extended periods of time, up to six hours. That really is the finesse technological advance that makes it possible for us to treat a variety of urothelial cancers, which is the core focus of our year two effort to date. Okay, great. First product approved, Jelmyto. That's for Low-Grade Upper Tract Urothelial Carcinoma. Mark, just quickly, what is this disease about in terms of the pathology, the alternative treatment paradigm, and the number of patients who present with this condition? For upper tract urothelial cancer, which is what Jelmyto was developed for, this is a disease that affects a total of 7,000 new patients in the U.S. each year. Jelmyto was proved to be useful in the treatment of patients who have what's called low-grade histology, representing about 40% of that 7,000. About 3,500 new patients a year historically have had their kidneys removed for the treatment of this disease, assuming they had another functional kidney. A rather draconian intervention for which Jelmyto is really a tremendously useful alternative. Historically, urologists periodically were able to put scopes up in the upper tract and burn out small tumors, but that's an unsuccessful approach that led to recurrence in less than a year in over 90% of patients. Jelmyto addresses upper tract urothelial cancer in a way that is minimally invasive and preserving of the kidney with a high rate of complete response with minimal morbidity. Great. Let's do the same thing with Zosduri, UGN-102. Is it Zosduri? Zosduri, perfect. That's perfect. That's low-grade, intermediate-risk NMIBC. What is this disease? Obviously, it's a larger opportunity, but walk us right through that. Sure. Low-grade, for starters, bladder cancer, 80,000 new patients diagnosed a year, 75% of those patients have what's referred to as non-invasive disease. Over 50 or 60% of those people have what's called low-grade histology. It's a chronic relapsing disease in those patients, very low rates of progression, very high rates of recurrence, particularly in the group we've identified, not by us exclusively, but have been identified in the urologic literature as having intermediate risk disease, namely multifocality, high rate of recurrence, early recurrence. We estimate that based on CIR data, there are about 60,000 new patients annually who have recurrent low-grade, intermediate risk disease, and that's the focus of the Zosduri product in the treatment of that group. Okay, so if I did the math right, at least a tenfold larger opportunity for Zosduri relative to Jelmyto. Correct. Correct. Yeah. Jelmyto, been on the market now several years, growing steadily. Liz, just give us an update on what the sales projections are and what the potential ceiling is for this product. Sure. So with Jelmyto, we actually have it guided to $94 million to $98 million this year. We still feel really good about that number. We're on track to hit that number. To your point, we expect to see sort of continued single-digit growth for Jelmyto. I actually am very anxious to see now that we have an expanded sales team, an expanded presence out in the marketplace, that we may even be able to see more growth out of Jelmyto. You know, we haven't talked a lot about peak, but we expect, again, it to continue to grow, probably peak around $150 million for Jelmyto. You know, as Mark said, there's about 6,000 patients a year. Half of those are newly diagnosed, half of those are recurrent. Most of those patients will ultimately get a, you know, lose a kidney. Ultimately, what's important about it is that it's a rare disease that gets treated by a large number of physicians. One of the biggest challenges with Jelmyto is actually finding the patients because any one doctor may only see one or two of these a year. That's why the expanded sales team may unlock some opportunity for Jelmyto. We're excited, obviously, about Zosduri, but also the leverage and synergy that we could see with Jelmyto as well. Okay. I know that you're already working on the next generation formulation of Jelmyto, but what is the patent life of the first gen and the transition strategy to the next gen? Sure. I'll actually talk about both of those together because our patent expiry, which is in January 2031, is actually for our gel technology. Both Jelmyto and Zosduri, the active, you know, that we mix it with is mitomycin. It's a generic mitomycin. It's the same, and therefore, the patent for both of those expires in 2031. The good news is on Zosduri, we actually have some incremental opportunity for additional patent protection built around that 2031. That's the timing of it. What we've done and actually started looking for a secondary supplier as we were looking to ensure supply because, as you said, Zosduri is a much bigger market. We needed to make sure that from a manufacturing standpoint, we had the supply and the capacity. As we were searching for that, we found a company in Germany called Medac. They actually have a proprietary mitomycin, and they are already patent protected. They have until 2035. Not only do we get incremental patent, but it's easier to manufacture. We have, you know, lower manufacturing costs. Also importantly, it's easier for preparation, so it takes a shorter amount of time at the pharmacy. While we expect the clinical benefit to be very similar, we see there are some other manufacturing and supply and preparation benefits to the second, you know, to the second generation. In the conversations with the FDA, what was interesting about that is they acknowledge that it's a different mitomycin. Because of that, they said, you need to do a clinical study. That shows us, and we were able to get incremental patent, which proves, you know, that it's a different, it's a different drug. Because of that, we actually launched into our current studies with both UGN-103 and UGN-104. The follow-on to Zosduri, UGN-103, is that we have prioritized that. We've already fully enrolled that study, and we'll be talking to the FDA in Q4 of this year because we'll have complete response data from that one. UGN-104, which is the Jelmyto follow-on, will follow as well. The good news is from a strategic standpoint is that we expect both of those medicines to replace our current formulation and then therefore give us the ability to have patent protection to 2041. Maybe Mark gets to go through another ODAC medical. Yes, exactly. Let's focus on the conversation on Zosduri. Sure. Maybe Mark, just starting out with the biology and making sure we're all familiar. What is the difference between low-grade and high-grade NMIBC? What does the treatment paradigm look like in the mid-global skin sector? Sure. Very different diseases. Oncogenic histology is exactly what you'd imagine. These are angry-looking cells with multiple genetic abnormalities that can lead, in some patients, about 30%, in fact, to the development of a more invasive form of cancer that can, if left untreated, become metastatic. The paradigm for the treatment of that population of patients is transurethral resection, which is an operation performed endoscopically under anesthesia, complete resection of all visible tumors, followed by the instillation, typically several weeks following surgery, of a variety of different medications. One that this audience may be well aware of, but is in short supply, is a drug called BCG, a very historically relevant immunomodulator first introduced in the early 1970s. There are a variety of drugs now being developed in this space, particularly for patients who are resistant to BCG. Failing all of that, patients actually can even have their bladders removed before metastasis occurs. That's that group of patients. A larger group of patients have what's called low-grade histology. These people have a genetically different version of bladder cancer. It can look a little like high-grade disease, but behaves very differently. It tends to recur once resected, and historically, patients seem to resect all visible disease and then consider the use of instillation or chemotherapy before small recurrence. You can get a small incremental benefit with aqueous medications when they're used in this setting. Most urology practices in the U.S., however, typically treat these patients with surgical monotherapy. Only about 20% of the American patients get the adjuvant chemotherapy. These patients cycle through practices throughout their lives, once they've been diagnosed, periodically recurring, and the intermediate-risk population we're focused on recurs rather frequently. In fact, the population we studied for the Zosduri approval had a likelihood of recurring at a year of about 50%. A very high likelihood of having multiple surgeries during the lifetime of the patient. Briefly, just recap what you were able to show in your clinical trials with regard to Zosduri. It is important to remember, particularly given the landscape that's currently evolving in the treatment of bladder cancer and distinguishing Zosduri from everything else that's being developed, all the other drugs you hear about are adjuvant therapies. They follow surgery. That is the high-grade space. Eventually, we expect they'll migrate into the intermediate-risk space as well. Zosduri is a primary therapy. For patients who have had a TURBT and then recur, as they would predictably, that primary lesion can be treated primarily with the instillation of Zosduri once a week for six weeks. You can expect what we saw in our clinical development program, an 80% complete response rate with durability beyond a year of over 80%. In fact, we are developing long-term durability data, even more encouraging now, out two years almost to 72%, or actually 72% at two years. We are going to follow that cohort for five years. We haven't reached a median yet for this population. These data look certainly as good and probably better than what can be achieved in this population with surgical monotherapy, which is the standard of care. Is there any other drug therapy that's being developed in this indication? Certainly, there are other competitors who are currently in the high-grade space who are now looking into the intermediate-risk space. J&J, for example, with the TAR programs, and CG Oncology, to name just two, are looking at developing products. What does that mean? They are doing clinical trials, I should say. The answer is there will be competition in the future. It's not here yet, but we expect competition in the future. Who is the ideal patient for Zosduri? The ideal patient is someone who has recurrent low-grade, intermediate-risk disease. That person could be a person who does not want to have a TURBT. That is a person who couldn't have a TURBT for some contributing medical reason. Consider the patient with cardiac stents on anticoagulation who's difficult to manage and having an operation. That's a fairly broad swath of individuals. Remember, the average age of diagnosis is in the mid-70s. These are elderly people with comorbidities. That doesn't take into account the number of patients who, once they hear about a non-surgical option, may be very interested in not having an operation. Angela Sober at UMC and her colleagues have looked intensely at our cohort of patients and discovered that when queried about the experience of having a TURBT, having had the experience of having UGN-102, now known as Zosduri, 90% of patients would, in fact, prefer to have a non-operative Zosduri treatment than a TURBT. That's the experienced patient talking. That will probably pay out as the launch proceeds. Not to force you to relive the May panel, but what was all the controversy about in retrospect? Did it hurt to launch at all that the panel vote was mixed? No, I don't think it does. I think it's particularly because you only had two urologists on the panel, and both of the urologists voted to have it approved, right? That was the part of the conversation we had after. You're in the oncology division, so you're with medical oncologists, and none of the medical oncologists actually treat this disease. There really wasn't a real understanding of that. I think if they had, you know, if you saw after the vote, they went around and asked everybody why. I think if the two urologists had said the thing that they had said before, you probably would have gotten a different answer. The reality of it is, as you're talking to medical oncologists, we also believe that the way the panel, what happened in the sense that they conflated the issue by having a discussion around randomized controlled studies because also, even the medical oncologists couldn't give you a reason why the data didn't demonstrate. It was just more, well, they should do a randomized controlled study. In the end, I think we had a great conversation with the FDA afterward. You saw the results. Not only did we get approval, but we got the label that we expected. No onerous post-marketing commitments or anything. Okay, let's move into commercialization then. I think, Liz, you called this a multi-billion dollar, even $5 billion opportunity here. How did you get there? What needs to happen for? Yeah, what I've said, and we're really clear, is that the market, the TAM, the total addressable market is $5 billion +. It's kind of easy to get to that number, right? You have 60,000 patients. It's about $100,000 net per patient. It's $6 billion, right? That's just the recurrent patient population. That doesn't take into consideration the newly diagnosed. For us, what we've said about Zosduri is Zosduri can be a $1 billion + opportunity there. Back of the envelope math is easy. You have 60,000 patients. You get 20% of that. That's 12,000. You're at $1.2 billion. It's a fairly, I would say, low bar to get us to $1 billion revenue. I think more importantly, what we've seen is physicians and patients both want this alternative, right? There's nothing today, as Mark talked about. There are no other alternatives. We know that 23% of patients have five or more recurrences. 68% have two or more recurrences. Speaking of the ODAC, it was heartbreaking to just listen. I think everybody thinks, surgery, no big deal. It is a really big deal, particularly for those patients who have had multiple surgeries. We're excited about being able to bring this to the market. Talked about a two-stage launch with the J-code, so January 1 being kind of critical to really opening up the gates here. Why is the J-code so important? Yeah, it's really interesting. I think people who know me, if you didn't, I've spent the last 30 years in oncology, so with medical oncologists. In oncology practices, buy and bill drugs are about 70% of their revenue. That's not the case for urology. Buy and bill, so our drug is a buy and bill, and it's also a procedure. It's not, you know, it's an instillation, right? It's not a pill or an infusion that's sort of easy, and you don't have to learn anything. Because it's a buy and bill drug, urologists are just much more trepidatious to use it during this miscellaneous code period. We know that the majority of the patients get seen at the community setting. As you said, we launched the drug. I've been out with customers, been out some organizations where you have a meeting. What's been great about it is every single physician says, yep, can't wait to use it, excited about it, have several patients that I know I'm going to want to use it on as soon as I get a J-code. I love the first part of the conversation. I don't love the second part, but I understand it. We've been messaging that for quite a while, right? The miscellaneous J-code will be a challenge between now and then. They just want this sort of assurance that they're going to get reimbursed. Again, urologists aren't used to dealing with the types of medicines that oncologists are. They don't have the infrastructure in place. Because of that, the permanent J-code becomes so much more important. It's really more of a perception, but it gives them confidence that they'll get reimbursed. Once that J-code is in place, how do the economics work getting it to the practice? Yeah. Buy and bill, PPSP +. Yeah. That's also really important. Physicians do realize and start to see, particularly in the community practice, that this, the practice economics look better for them. With a buy and bill drug, they're going to make an average of 5%. That 5% comes from Medicare, 4.2% commercial, much higher. It sort of averages out to about 5%-5.5%. They're going to make about $5,000 per patient on Zosduri. Today, if they take a patient to the operating room, it's $300- $800 for the professional fee for them to actually do the surgery. They actually can see, plus they get the instillation, you know, the CPT codes. Also, by the way, the doctor doesn't do it. In a community practice, a nurse or an extender can actually give the drug. From all of those perspectives, they learn over time that it's good for the patient and it's good for the practice. Hopefully, it's a win-win. Prior to the J-code, we've got a couple of quarters coming up here in Q4. What kind of metrics do you want us to focus on pre-J-code? Yeah. What are some signs that we're moving in the right direction? That's a great question. We've been having a lot of those conversations the last couple of days. I think, you know, our message is, my message has been twofold. One, what do I really like and what do I feel like we still need to work on? What I really like is what I call the top of the funnel. What we're working on is activating sites, making sure the site is ready with a specialty distributor, making sure that their nurses are trained, all of the things that they need to do, that our drug is set up in their system for them to be able to prescribe the drug. That's probably, you know, that has been happening. We feel like that's right on track, right where we'd like to be, maybe a little bit ahead. Will you be giving metrics? We will be giving metrics on the number of sites. What we probably won't give, but is important, is what we call patient enrollment forms. In a patient enrollment form, you only get a patient enrollment form when a patient has been identified by the doctor and they want to use the drug. It's not like they say, maybe I'll use it. They want to use the drug. A patient enrollment form is filled out, then it goes to our hub, and they do benefit verification. We will do benefit verification. The doctor's office will do benefit verification. With our PEFs, which we call our patient enrollment forms, that's our early indicator of demand. A doctor can say they really want to do it, but until you get a patient enrollment form, that means a patient has been identified. That's an early read. We're also very happy with that metric. Where we need to work with, where we. Will you be giving that metric out or you're not? No, I don't think we're going to get that metric, only because there's so many variables associated. Where we really have to focus and what will take more time is the time from a patient enrollment form to a new patient getting actually dosed, and that time is about 45 days - 60 days. That's really where the funnel starts to get a lot smaller and you really have to get through that funnel. Our caution is more about timing than it is about demand. We want to make sure that everybody understands, like I said, very enthusiastic from a physician standpoint. Patients identified, ready to go, want to use it, want to make sure they get reimbursed, and they need to go through the system. What we've talked about before, which happened also with Jelmyto, is in the beginning, because of the miscellaneous J-code, more will get done in the hospital versus the community. With hospitals, you've got to go through formulary and P&T committee, so that takes a little bit of time. For us, it's really about timing. We want to make sure that we don't get caught up in, you know, what is the revenue number as much as what are the leading indicators to how well the uptake is. We continue to see great uptake in patient enrollment form every week. We get to see those, and we're seeing growth in our patient enrollment forms. The team in the field is really working on how do you pull those through and getting those pulled through as fast as possible. It's great to see. I haven't talked to a physician, and Mark just said, as soon as we got approval, his phone was ringing. That didn't happen with Jelmyto, right? Because patient identification is actually probably our biggest challenge with Jelmyto. It's not a challenge with Zosduri. Would a provider put forth a patient identification form at this stage if he doesn't intend to treat until the J-code, or would they wait until? He or she would wait until maybe December. They would because it takes a couple of weeks, you know, a few weeks to get through the system. Maybe then. Right now, if they put in a patient enrollment form, they intend to use Zosduri. That's why we can confidently use that as a good barometer for how well it goes. As far as investors go, right now, you're only willing to commit to site activation. I don't want to say that right now because, you know, and Chris is here. Chris and I, our CFO, we have a lot of discussion. I think what we'll do is share data that we're confident in at that point in time. We will likely share more granular data in our Q3 earnings because we want to make sure everybody understands the trends. If we need to share earlier data, it would cautiously, then we will likely do that because we want to, again, we want to give everyone, our investors, the confidence that we're on the right track. You know, we're out there excited talking to physicians, talking to investors, and making sure we are managing expectations as to what you'll see the first few months. Remind us what you've done with your commercial infrastructure and sales pitch. Yeah, great. Actually, early, you know, with just Jelmyto, we had 42 reps. We had started to build, so we were at 52 prior to launch. We waited because of the ODAC. We were ready. We actually were interview ready to hire, but then after the ODAC, we waited. We actually didn't add the 30 reps, 30 new territories, and that was just as of August 1. They're just now getting out, doing sort of a warm transfer with the old rep. Now we have 82 reps, 82 territories, but in addition to that, we have operations managers. I talked about the, you know, when I've been out and actually you get this feedback as well, I'm rarely having a clinical discussion with a doctor because they understand the data is very compelling. They're not really asking about that. It's really all about the logistics and reimbursement. We have operations managers. We have field reimbursement managers, which the conversation we've talked about, practice economics, our reps can't do that, but our field reimbursement can have that conversation. We have key account directors who can, you know, call on C-suite in an institution and talk to them or in a large practice to help them understand about the benefits of bringing Zosduri in as a service line in their practice. A lot of the different, and we have nurses, right? We need nurses to sort of train other nurses that are going to give the drug. We have a real, what I call, surround sound when it comes to making sure that we are addressing every barrier that might exist. We've got an answer for that, and that's another important thing. Do you have an opportunity to license additional products, or is this full-on full focus on Zosduri? No, absolutely. We love our BD. We're pretty active, I would say, in a few things. I will say that one of the first things we do want to do is expand on UGN-103. Assuming the data comes out where we expect it to, we're going to want to move into other patient populations in urothelial cancers. We also have UGN-501, which is our oncolytic virus, and we'll want to move that out of just bladder cancer into other cancers. We're definitely open for two things. One, we do have a couple of collaborations where we're using our technology with other drugs that are in different spaces. That's one area of opportunity. The other is we'd love to bring in more medicines, whether they're in urothelial cancers or other specialty cancers. We are a company that's building and growing. We've talked about our ability to get to profitability, where we are today. Once we do, because we have a highly leverageable infrastructure in our organization, once we get to profitability, we start to throw off cash pretty quickly. That will allow us to do even more business development, which we're absolutely interested in. Remind us of your balance sheet, your trajectory to profitability. Most companies that have gone from $5 a share to $19 a share raise capital, you've not done that. Yeah, we did not. We did not do that. We ended Q2 with $162 million in the bank. We chose not to do it. We chose to be patient because we didn't like the price, right? After the, if we had not had the ODAC and the price had been higher, maybe we would have considered it, maybe. The reality of it is that we talked to our board, us as a management team did not recommend it at the time. We don't need the cash. We want to be patient. We have other avenues available to us if we need it. What we've said is with our current cash, we can get to profitability. Obviously, people can do the back of the envelope around what our OpEx is. They can think about what kind of revenue you need to get there. We feel really good about where we are. We have a lot, our phone's ringing with everybody wanting to give us money, which is a good place to be for a change. We have debt. We have $125 million in debt with Pharmakon, who's been a great partner. There's a lot of opportunity for other ways to fund if we need. If we did want to do something in business development, that might be a time where we might need. Other than that, with our current operations and what we have planned, we can get to profitability. We haven't given a date, but I think you people can figure it out pretty easily. I mean, it has to be within the next eight months. I'm not saying a word. Okay. It sounds like you're okay kind of letting the gas tank run on your empty as you get closer and closer. As I said, we have opportunities if we feel like we will have to make that decision with the board on how comfortable we are, how much cushion do we want to have, and at what point in time do we do something if we want to do something. We're out of time, but congrats on all the success. This is a really important product. Appreciate it. I wish you all the best. Yeah. Thanks, Eric. Thanks a lot. Take care.