UroGen Pharma at H.C. Wainwright Conference: Zosduri’s Market Impact

On Monday, 08 September 2025, UroGen Pharma (NASDAQ:URGN) presented at the H.C. Wainwright 27th Annual Global Investment Conference, detailing both the promising and challenging aspects of its strategic direction. The company highlighted the recent FDA approval of Zosduri, a groundbreaking urothelial cancer treatment, and discussed the significant market potential and operational hurdles.

Key Takeaways

• UroGen’s Zosduri, approved in June, targets a $5 billion market for bladder cancer.
• The company has $162 million in cash, supporting the Zosduri launch and future profitability.
• UroGen plans to expand its drug pipeline with upcoming trials for new cancer therapies.
• The company faces challenges in patient conversion from enrollment to treatment.

Financial Results

• UroGen reported a cash position of $162 million as of the end of June.
• The company believes it has sufficient funds to support the Zosduri launch and achieve profitability.
• UroGen is cautious about future capital formation to maintain financial health.

Operational Updates

• Zosduri, a non-surgical treatment for bladder cancer, has been met with enthusiasm by physicians.
• The sales force increased from 52 to 82 representatives to cover 8,500 physicians.
• 90% of covered lives have open access to Zosduri, with hundreds of sites already activated.
• The company is enhancing support services to improve patient dosing rates.

Future Outlook

• UroGen is developing next-generation formulations, UGN-103 and UGN-104, with IP protection until 2041.
• UGN-103 has completed Phase 3 enrollment, with data expected by year-end and potential approval by 2027.
• The company is also advancing a multi-drug immunotherapy program and an oncolytic virus, UGN-501, with trials planned for 2026.

Q&A Highlights

• CMO Mark Schoenberg emphasized the advantages of UroGen’s RTGel technology for urologic treatments.
• CFO Christopher Degnan highlighted the $5 billion market opportunity for Zosduri and its unique position as the first FDA-approved product in its category.
• Schoenberg noted that 90% of patients prefer Zosduri’s outpatient treatment over traditional surgery.

In conclusion, UroGen Pharma’s presentation at the H.C. Wainwright Conference underscored its innovative approach and strategic focus. For further details, readers are encouraged to refer to the full transcript.

Full transcript - H.C. Wainwright 27th Annual Global Investment Conference:

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Yes, Christopher Degnan.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Now I’m in.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: With my written. Awesome. Let me pull up, scared by a call.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Good morning, everyone, and thank you for joining day one of the H.C. Wainwright & Co. Annual Global Investment Conference.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: My name is Daniel Smith. I’m an H.C. Wainwright Equity Research Associate in Biotechnology. With that said, let me introduce our speakers today for this fireside. We have Dr. Mark Schoenberg, CMO of UroGen Pharma Ltd., and Chris.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Degnan, Chief Financial Officer.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Chris Degnan, CFO. UroGen is a leading biotech company in the urothelial cancer space. UroGen is traded on the NASDAQ under the ticker URGN. I appreciate just a high-level overview. Tell us your UroGen story, where you came from, where you’re going, and we’ll take it from there.

Mark Schoenberg, CMO, UroGen Pharma Ltd.: Thank you very much for having us, and good morning, everybody. It’s a pleasure to be here. Mark Schoenberg, I’m the Chief Medical Officer. I’ve been with the company for about 11 years. Let me tell you the story of how we got to where we are today. UroGen Pharma Ltd. is founded based on research done originally in Israel. It was a small company that developed a technology called RTGel, specifically to deliver drugs to the interior of the urinary tract. The magical technology is a mixture of generally accepted as safe polymers that, when cooled or liquid in form, can be mixed with multiple drugs of varying molecular sizes. When this mixture warms to body temperature, once it’s instilled in the urinary tract, it remains there and slowly releases the drug as a depot for about six hours.

The company was originally founded in 2004 and, over a period of a lengthy development process and multiple clinical trials, brought its first drug to market in 2020 during the pandemic. This drug is called Jelmyto (mitomycin) for pyelocaliceal solution, and it was originally developed for the treatment of low-grade urothelial cancer occurring within the interior of the kidney. The reason that this was chosen as the first project was because patients with cancer occurring in this location historically have been treated by kidney removal. This minimally invasive kidney-sparing therapy was a real advance for the management of this patient population and demonstrated the power of RTGel technology in the context of urologic practice. It has really transformed the way these patients are cared for, and it opened the door for us to then explore a much larger population of patients.

Upper tract urothelial cancer occurs in about 7,000 Americans annually, but many patients in this country have another form of urothelial cancer called bladder cancer, specifically non-muscle invasive bladder cancer, which is, in most patients, a chronically recurring disease. This year, we’re happy to share with you the fact that our product, Zosduri (UGN-102, mitomycin for intravesical solution), which is a product analogous to Jelmyto (mitomycin) for pyelocaliceal solution for the treatment of low-grade intermediate risk non-muscle invasive bladder cancer, was approved by the FDA on June 12, and the launch is underway. You will hear more details about that from Chris. We are an exciting and excited small biotech company with a commercial stage now with a pipeline. We can talk about that later, but we are now in the process of launching our next drug, which is a big drug and a big population of patients need this.

I’ll turn it over to Chris to tell you more about that.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Thanks, Mark. I’m good with this. Just in terms of the story, as Mark mentioned, much bigger patient population. The product was recently approved in June for patients with recurrent low-grade intermediate risk non-muscle invasive bladder cancer. That patient population is about 60,000 patients annually in the U.S. That’s our target patient population. What we said is that target market opportunity is an over $5 billion market opportunity. You take the 60,000 patients, assume about $100,000 per patient, over $5 billion is the opportunity there. That compares to Jelmyto, as Mark mentioned, which is our first product on the market. That target patient population is only about 6,000 or 7,000 patients annually in the U.S. It’s very much more of a rare disease, and it’s a rare disease that’s dispersed across a number of urologists. 10,000 urologists, they only may see one or two of these Jelmyto patients a year.

Compare that to the story where there’s 60,000 across the 10,000. They’re seeing several patients a year. A much bigger opportunity for us as we expand and we scale. The story was approved in June. Product was available in July. We’re in the early stages of the launch. We came into the year with 52 sales reps calling on a target of 5,500 physicians for the Jelmyto opportunity. We have since expanded our sales force. We’re now at 82 reps. We brought 30 reps on on the back of the approval. They’ve been trained and they were in the market starting in August. Those 82 reps are calling on a target universe of 8,500 physicians. Those 8,500 physicians cover about 90% of the target opportunity that we mentioned for Zosduri. Not only do we have the sales reps out there, this is a buy-and-bill product.

As Mark mentioned, it’s an RTGel technology. It’s administered either by a physician or with Zosduri. Given the ease of administration, it can be actually administered by a nurse or an extender. As a buy-and-bill product, we also provide a number of ancillary services to the physician and their offices. We have a hub to help them go through reimbursement support. We have field reimbursement managers that help them go through the reimbursement process and can talk about the economics associated with the product. We have regional operations managers that help sites get onboarded. They have to obviously be able to order the product from the specialty distributor to be able to administer it. They also have to have their staff trained to administer the product through a catheter. We also have nurse educators that help go through the education process.

We have a full support function around, you know, roughly we have about 130 customer-facing colleagues out there supporting Zosduri as well as Jelmyto. As Mark mentioned, we are in the early stages of the launch. We get the question often in terms of what’s going well and kind of where are things within the launch. In terms of what’s going well, first of all, a high level of enthusiasm from the physician community in terms of having another therapeutic option for patients. This is the first therapeutic option available. Right now, these patients get a surgical procedure called TURBT. Zosduri is the first and only FDA-approved product in this space. The other thing from a clinical profile perspective, not only do we have really good complete response data, 80% of the patients had a complete response at three months. We also have really strong durability of response.

We just issued data recently with 24-month follow-up where 72% of the patients were still in complete response following their initial three-month CR. Long-term durability of response data has really resonated well with physicians. In terms of the early launch, in terms of what we’re seeing, we’re doing in terms of the top of the funnel. We define that a couple of different ways in terms of, one, market access. 90% of covered lives have open access to the products. We’ve done a really good job of getting access within the parent community. Two, site activation. We talked about the fact that this is a buy-and-bill product that has to be administered. With that, they have to be able to order and then get all the things necessary to handle the operational aspects of the drug. We’ve done a really good job of opening sites.

We have a few hundred sites already up and available to order and administer Zosduri. The other piece with this is the patient enrollment form. People from the top of the funnel coming in. This is patients that have been identified by a physician where a physician has said, "Here’s a patient I want to administer Zosduri for." We get a patient enrollment form in, and we’re seeing a really good top of the funnel in terms of the number of patients coming through. The piece that we’re working through and some of the headwinds here early on is just the time to conversion, going from a patient enrollment form to the patient actually starting a new patient dose.

When a patient enrollment form comes in, we go through the reimbursement process with the physician, the benefit verification, and then ultimately, once the patient’s through that, the office has been trained, we can administer the product. We’re seeing really good initial demand, and now we’re working on the pull-through in terms of getting patients dosed. Thanks for the really comprehensive overview, guys. In terms of the ideal patient for this drug, could you just kind of describe what they look like?

Mark Schoenberg, CMO, UroGen Pharma Ltd.: Let me take that. I just want to emphasize something that Chris has alluded to. What we’re talking about here is a non-surgical therapy for patients with non-muscle invasive bladder cancer. As many of you probably know, these patients who are typically diagnosed in their mid-70s are historically treated by surgical procedures that require anesthesia and some extended period of convalescence, sometimes even in the hospital. Here, Zosduri is administered in an office setting without anesthesia. It’s entirely ambulatory. It can be given by a nurse. We’re talking about a real paradigm shift in terms of how this population is treated.

We know from patient-centered outcomes research done independently by investigators at the University of North Carolina that when patients who had a TUR are asked after being treated with Zosduri which therapy they would recommend and which they would prefer to have themselves, 90% have emphasized that they would want an outpatient ambulatory intervention like Zosduri as opposed to the traditional surgical procedure. We know this is very appealing to patients. We know this is very consistent with workflows in the doctor’s office.

The ideal patient is the person for whom this label was developed, someone with recurrent low-grade intermediate risk disease, the chronically relapsing individual who is in the office every 9 to 12 months with another tumor who now has to have, or who has had to have surgery in the past, who now can have an office-based therapy, come to the office him or herself, and drive him or herself home or back to work or wherever they’re going without the encumbrance of the traditional surgical approach to this problem.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: That’s really fascinating. When you guys kind of think about that, going from a standard of care that’s surgery to a standard of care that’s just a procedure, can you talk about what the enthusiasm has been like that you’ve seen from urologists and practices?

Mark Schoenberg, CMO, UroGen Pharma Ltd.: Let me just say that as soon as we got approved, my phone lit up with my colleagues calling me, saying, you know, how do I get this drug and how do I get it in my practice? Chris, do you want to talk a little bit more about sort of how we’re dealing with physicians’ enthusiasm and interest in getting this in?

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Yeah. Part of it is there’s a high level of interest. I mentioned earlier with Jelmyto as a contrast. Physicians are only seeing one or two patients on average across the year. When we go in and talk to physicians, one, the clinical data resonates with them. As I mentioned before, just the durability of the response, because what you’re dealing with here is a patient population that’s highly recurrent. They’re having multiple surgical procedures being done. The fact that we can demonstrate long durability of response through a non-surgical procedure is really resonating well with physicians. That aspect is where the high level of enthusiasm is. For us, it’s just getting through the early pieces of launching a product that’s in the buy-and-bill space. It’s getting them set up from a site activation perspective, making sure that they have reimbursement.

The other thing that we’re dealing with with the buy-and-bill product is, for the first six months or so, we have a miscellaneous J code for the physicians in terms of how they submit to get reimbursed. That does take longer for physicians. There’s more of an administrative burden for them. Trying to remove some of those headwinds for them in terms of the support that we provide them, we give them extended dating and things along those lines. January 1, 2026, we’ll have a permanent J code, and that’s where we really expect to see a continued acceleration of growth.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Awesome. When you kind of think about this product, which is, you know, it requires a procedure, it’s also a drug, there’s multiple steps to it. Can you talk about sort of lifecycle management and just the IP life of it?

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Sure. I’ll start. One, our current portfolio, both Jelmyto and Zosduri have IP protection to January 2031. That’s around the RTGel technology. As we were starting to scale the business up to launch Zosduri, given the large patient opportunity, we started looking at alternative supply options. We came across another company that had a proprietary mitomycin that, one, had significant capacity, had supply efficiency. Right now, our product is live for lives. It takes about three weeks to go through that process. The company, Medac, that we identified based in Germany, their process only takes three days. Great supply chain efficiency, COGS efficiency that comes along with that. With that, they also have IP protection for their proprietary mitomycin. Their mitomycin has IP protection to 2035. We then were able to secure IP protection in combination with our RTGel through December 2041.

This is the product portfolio of UGN-103 and UGN-104, which are our next-gen formulation products. Right now, we’re in phase 3 for both products. We have already completed enrollment in the phase 3 for UGN-103, and we’ve started enrolling patients for UGN-104. Maybe I’ll turn it over to Mark to talk briefly about where we sit in terms of the 103 plan.

Mark Schoenberg, CMO, UroGen Pharma Ltd.: Yeah. UGN-103, the successor molecule to Zosduri, is in clinical trial right now in phase 3. The trial is enrolled. We expect to have complete response data in this single-arm trial toward the end of this year. We are going to have a meeting with the FDA to share those data with the FDA. We have a pre-existing agreement with them that the single-arm design of this trial could support approval of this without an additional trial necessitated by other conversations the FDA has been having right now. What we believe we will be talking to the FDA about will be the meaningfulness of the complete response data. We expect those data to be very similar to what we reported for Zosduri.

It will ultimately end up being a conversation about how much durability will be required in order to approve this molecule, which will then be moved into the market in a timely fashion. Chris may want to talk about this more as a substitution for Zosduri to preserve IP protection.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Yeah. The last piece there is, you know, expect to submit the NDA for UGN-103 by the end of next year, approval by 2027. As we talked about before with the miscellaneous JCO and the permanent JCO, we’ll wait to get a permanent JCO for UGN-103 before launching that product into the market. We would, at that point, begin to remove Zosduri from the market and succeed it with 103.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Got it. We got Zosduri, we got 103. Are there any other high-level overviews of the pipeline or any business development ideas that you guys or strategy that you’d love to elucidate on?

Mark Schoenberg, CMO, UroGen Pharma Ltd.: Sure. Just a couple of other points. As Chris was mentioning, we have a successor molecule to Jelmyto that’s called UGN-104 that is also in phase 3, that trial’s open and enrolling. It’s about a year behind in terms of a substitution for the existing drug on the market. It will follow a very similar pathway to what was just described for Zosduri. In addition, we have a multi-drug immunotherapy program that we’ve reported on previously. It includes the intravesical instillation using our gel technology. I’m an anti-CTLA-4 antibody and licensed from a company in Boston. In combination with our own profile TLR7 agonist UGN-201, we have done a phase 1 study. We have actually seen responses in that study in high-grade non-muscle invasive disease. We will be reporting on the durability of those data later this year, probably at the Society for Urologic Oncology.

At that point, we’ll make a go/no-go decision about phase 2 for that program. In addition, we have actually purchased an oncolytic virus called UGN-501. That is in IND enabling phase right now. We expect to do phase 1 for that in bladder cancer in 2026. One thing to remember or to note about UGN-501, it could be used in other cancers. It’s a very flexible and interesting virus. We plan in the future, depending on resources and other obligations, to explore this in the treatment of other cancers beyond the urinary tract. It would give us an opportunity to expand our portfolio further.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Awesome. Maybe to round it out with the last couple of minutes that we have, can you just discuss your cash position and path to profitability?

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Cash at the end of June was $162 million. We have the cash and resources necessary to fully fund the Zosduri launch. In terms of cash runway, based on our expectations for the Zosduri launch, we believe we have cash to profitability. We’ll be thoughtful about capital formation going forward and the cost of that capital as we think about the long term for the business.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Awesome. Mark, Chris, thank you so much for your time. It’s been a pleasure.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Thanks, Daniel.

Daniel Smith, H.C. Wainwright Equity Research Associate in Biotechnology, H.C. Wainwright & Co.: Thank you, everyone, for participating in the conference.

Mark Schoenberg, CMO, UroGen Pharma Ltd.: Thank you very much.

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