UroGen Pharma Ltd. Ordinary Shares 12th Annual Cantor Fitzgerald Global Healthcare Conference

Do you want to start a little bit with the history of the company?

Sure. Nice to be here, and thanks for having us. UroGen comes from an apocryphal conversation 20 years ago between a urologist and a chemist. As the story is told, the urologist says to the chemist, "We put a lot of aqueous drugs in the bladder to treat cancer, and patients just urinate them out very quickly. Is there something you've got in your armamentarium that would help us keep drugs in the bladder longer to treat cancer more effectively?" The result of that conversation was a very elegant material science solution that is our technology platform that has made it possible for us to make the progress we've made to date. It's called RTGel. It is a reverse thermal gel that is very accommodating of molecules of vastly different sizes.

I am sure we will have an opportunity to talk about the success of using that platform and the drugs we have deployed to date to treat cancer.

In the past 12 months, as you asked, the big progress we have made is our first product was approved back in 2020 in the Upper Tract Urothelial Cancer. That is where our focus is in UC. In 2025, we got approved for Zusduri, which is the first and only FDA-approved product in low-grade intermediate-risk non-muscle invasive bladder cancer, so an area of high unmet need. We started with the commercial launch of that product in the second half of last year. We got the permanent J-code at the start of this year, and we are in the early stages of a very promising commercial launch, so a very exciting time for the company.

Yeah. Just the size of the markets, the low-grade UTUC, which is where Jelmyto was indicated, it is a smaller rare disease market. It is about 6,000-7,000 patients in the U.S. With that market, the other part is it is a dispersed rare disease, so these 6,000-7,000 patients present across roughly 10,000 urologists. It is a bit of a different rare disease market opportunity. On the other side is the low-grade intermediate-risk non-muscle invasive bladder cancer market, which is much larger. It is about 10 times the size. Zusduri is indicated in the recurrent low-grade population, which means patients that have previously had a TURBT, generally, or have recurred with their disease, so it is a much larger opportunity.

In terms of why there haven't been good therapeutic approaches in these marketplaces, Mark mentioned, when you try and put aqueous solutions in the urinary tract, obviously the anatomy, it naturally tries to avoid that. Trying to find an option where the medication can dwell in the cavity long enough to provide the therapeutic benefit has been a challenge, and that's where RTGel fits in nicely.

Yeah, I think coming into this year and with the approval of Zusduri, again, there haven't been therapies in this space, so people trying to understand how big truly is this NMIBC market. We've thought coming in that we believe Zusduri has the potential to be over a $1 billion peak sales opportunity. I think coming into the year was a bit of a show-me story. I think people wanted to see the traction and the trajectory towards that opportunity. So I think that was one piece. I think the other piece is just better understanding the durability of revenue. We've done some good things to solidify our intellectual property. We'll talk about it. We have some next-gen formulations that we're developing, with additional intellectual property for both Jelmyto and Zusduri.

I think the other piece, too, is the competitive landscape in this space is really evolving at a fairly rapid pace. So I think people are trying to understand where these competitive data sets are going to land and where we're positioned, and we'll talk about it, but we think we have a very unique value proposition with Zusduri.

Yeah. Well, obviously, just continued strong progress with Zusduri. We're off to a really strong start this year and feel like, again, we're just in the early stages of that launch, so continuing to execute and demonstrate that good launch trajectory are going to be important. The progress that we're making on our next-gen formulations, specifically UGN-103 is our next-gen formulation for Zusduri. We've shown good data, 3-month complete response and 6-month durability of response data that are very similar to Zusduri, which is what our expectations were, and we just submitted the NDA for that drug back in the middle of August. So that's on track for potential approval in the mid part of next year, June of next year.