UroGen Pharma Q1 2025 Earnings Call Transcript

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Provided by Quartr
May 12, 2025
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Operator

Hello, and welcome to UroGen Pharma First Quarter twenty twenty five Earnings Conference Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer session. To ask the question during the session, you will need to press 11 on your telephone. You will then hear an automated message advising your hand is raised.

Operator

To withdraw your question, please press 11 again. I would now like to turn the conference over to Vincent Perrone. You may begin.

Vincent Perrone
Vincent Perrone
Senior Director of Investor Relations at UroGen Pharma

Thank you, operator. Good morning, everyone, and welcome to UroGen Pharma's First Quarter twenty twenty five Financial Results and Business Update Conference Call. Earlier this morning, we issued a press release providing an overview of our recent corporate highlights and financial results for the quarter ended 03/31/2025. The press release can be accessed on the Investors portion of our website at investors.eurogen.com. Joining me on the call today are Liz Barrett, President and Chief Executive Officer Doctor.

Vincent Perrone
Vincent Perrone
Senior Director of Investor Relations at UroGen Pharma

Mark Schoenberg, Chief Medical Officer David Lynn, Chief Commercial Officer and Chris Stegman, Chief Financial Officer. During today's call, we will be making certain forward looking statements. These may include statements regarding our ongoing pre commercialization activities related to UGN-one hundred two, regulatory meetings and decisions, our commercialization strategy and expectations, as well as potential future commercialization activities for UGN-one hundred two, if approved, market and revenue opportunities, commercialization activities related to Jelmyto, our ongoing and planned clinical trials, commercial and clinical milestones, UGN-one hundred two being the primary growth driver for UroGen, future R and D efforts, and our goals and 2025 financial guidance, among other things. These forward looking statements are based on current information, assumptions and expectations that are subject to change. A description of potential risks can be found in our earnings press release and latest SEC disclosure documents.

Vincent Perrone
Vincent Perrone
Senior Director of Investor Relations at UroGen Pharma

You are cautioned not to place undue reliance on these forward looking statements, and UroGen disclaims any obligation to update these statements. I'll now turn the call over to Liz Barrett, Chief Executive Officer. Liz?

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Thank you, Vincent, and thank you all for joining us this morning. The new drug application for our lead development stage candidate, UGN-one hundred two, is now in the final stages of FDA review, with a PDUFA target date of June 13. We designed the UGM one hundred two as a novel and innovative treatment for patients with low grade intermediate risk non muscle invasive bladder cancer. We believe UGM-one hundred two has the potential to change the treatment paradigm and meaningfully improve the standard of care in this patient population. If approved, UGM-one hundred two will be the primary growth driver for our company, And alongside Jelmyto could solidify our leadership in the urothelial cancer space, advancing our mission to bring innovative patient centric solutions to urologic cancers.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

We have been informed by the FDA that an Oncologic Drugs Advisory Committee, or ODAC, meeting has been scheduled for UGM102 on May 21. This is consistent with our expectations, and we look forward to the opportunity to showcase the strength and consistency of our clinical data to the members of the panel and the public. We have been preparing for the ODEP meeting and believe we are well prepared to present a clear, compelling, and scientifically robust case supporting the approval of UGM-one hundred two. In parallel, our regulatory team continues to engage regularly with the agency and has been responding to their information request. To date, we've encountered no resource or policy related issues that concern us.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

The UGN one hundred two NDA is supported by a robust development program, demonstrating meaningful complete response rate, durable responses, and an acceptable safety profile across three late stage clinical trials. In March, we reported updated data from the pivotal ENVISION trial, demonstrating that eighty point six percent of patients who achieved a CR at three months remained in response at eighteen months per Captain Meyer estimate. It's important to highlight that these robust results are with UGM-one hundred two alone and not following a transurethral resection of bladder tumor. Additionally, there are six weekly installations. Then patients are treatment free until recurrence.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

As Mark will highlight, these results, along with the broader update on UGM-one hundred two and Jelmyto, were shared with the urology community at this year's AUA meeting, underscoring our clinical leadership and commitment to advancing innovation in uro oncology. With a PDUFA date goal approximately one month away, our commercial team has been actively preparing for the potential launch of UGM-one hundred two. This launch would mark a pivotal moment in urogen's evolution from a rare disease focused company to a scaled multi product team positioned to serve a significantly broader patient population. We're expanding our commercial footprint accordingly with plans to grow our sales force from approximately 50 reps today to over 80 at launch. Our medical affairs and market access teams are also deeply engaged in prelaunch planning.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

We are targeting commercial readiness by June, and we will be ready to promote immediately following approval with product availability in July. UGM '1 hundred '2 represents a transformative growth opportunity for UroGen. We estimate an addressable population of approximately sixty thousand patients annually, with recurrent, low grade intermediate risk non muscle invasive bladder cancer, translating to a market opportunity of over $5,000,000,000 This is nearly 10 times larger than the Jelmyto market. Critically, this market is highly accessible. Unlike the more fragmented UTUC setting, NMIBC patients are widely distributed and primarily managed by community urologists across the country.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

UGM102 is well aligned with current clinical workflows. It's easy to administer, does not require specialized equipment, and can be delivered by a nurse with minimal additional training. We believe these advantages position UGM102 to become a foundational therapy in the management of low grade intermediate risk non muscle invasive bladder cancer, and a significant driver of long term value creation for urogen. Turning to Jelmyto, we reported $20,300,000 in first quarter sales, an 8% year over year growth compared to the first quarter of twenty twenty four, driven by underlying demand growth of 12%. We continue to advance our pipeline across multiple fronts, including our next generation programs for Jelmyto and UGN-one hundred two, as well as our emerging immuno oncology initiatives.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

In February, we acquired product candidate, ICBB ten forty two, a next generation investigational oncolytic virus from Aconivir, which we have assigned an internal code name of UGN five zero one. This is an important step in expanding our presence in immune based therapies for urologic cancers. In parallel, we also have multiple research collaborations in place aimed at leveraging our proprietary RTGel technology to enhance the delivery and effectiveness of various existing drugs. UroGen is executing with focus and discipline. We remain committed to transforming the treatment landscape in uro oncology, and are supported by a strong balance sheet with just over $200,000,000 in cash, cash equivalents, and marketable securities as of March 31.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

We are investing in innovation with purpose, driven by the opportunity to make a meaningful impact on patients while delivering value to our shareholders. I will turn the call over to Mark Schomburg, who will provide a clinical update. Mark.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

Thank you, Liz. I would like to echo what Liz shared regarding the strength of the clinical data supporting UGN-one hundred two. We've assembled a compelling and comprehensive clinical package that we believe demonstrates both the safety and efficacy of UGN-one hundred and two. This gives us a high level of confidence as we approach the upcoming ODAC meeting and the PDUFA target date. This year's AUA was an important event for UroGen.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

It provided a valuable platform to present the latest data on UGM-one hundred two and Jelmyto with the broader urologic community. We were proud of the six abstracts accepted, reflecting the growing body of evidence behind our programs and the continued momentum of our clinical efforts. The highlight was the updated eighteen month duration of response data from the phase three ENVISION trial, which as you know, is the pivotal trial that is the foundation of our NDA for UGN-one hundred two. The data were featured in a podium presentation by Doctor. Sandeep Prasad, the principal investigator on the study.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

We are highly encouraged by the continued durability of response observed in the phase three ENVISION trial. Among patients who achieved a complete response, the duration of response at eighteen months remains strong at eighty point six percent by Kaplan Meier estimate. Median follow-up time has now extended to eighteen point seven months post three months CR, up from thirteen point eight months at the previous data cut, and the median duration of response has still not been reached. For reference, the twelve month duration of response was previously reported at eighty two point five percent. These results continue to reinforce the potential of UGN-one hundred two to offer a durable non surgical treatment approach for patients with recurrent low grade intermediate risk NMIBC.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

We also presented a poster featuring patient reported outcomes from the three late stage UGN one hundred two studies OPTIMA-two, ATLAS and Envision. These assessments use the EORTC twenty four item quality of life questionnaire specific to NM IBC to evaluate the symptom burden and overall health status and function. The findings were consistent across trials showing the treatment with UGN-one hundred two did not negatively impact symptom burden, patient function, or quality of life. An important consideration for a novel therapy intended for use in routine clinical practice. At the AUA, we also shared data from the phase one dose escalation study of UGN-three zero one, our investigational anti CTLA-four antibody delivered via RTGel.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

These results were previously presented at SUO in late twenty twenty four, and continue to support a favorable safety profile for UGN-three zero one, both as monotherapy and in combination with UGN-two zero one, our TLR7 agonist, and with gemcitabine. We observed clinical responses in both the monotherapy and combination arms, with follow-up on the combination arms ongoing to evaluate durability of response. We expect to share updated data from this program later this year. At that point, we anticipate being in a position to make a gono go decision on whether to advance UGN-three zero one into phase two development. Our next generation candidates are actively advancing through development.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

Enrollment is ongoing in the phase three Utopia trial, which is evaluating UGN-one hundred three in patients with recurrent low grade intermediate risk NMIBC. Utopia is a single arm multicenter study modeled on the ENVISION trial. Efficacy will be measured by complete response at three months following treatment with follow-up focused on assessing durability. Enrollment is progressing ahead of plan, and we expect to complete enrollment by the middle of this year with top line data anticipated in 2026. We are taking a similar approach with UGN-one hundred four, our next generation formulation of Jelmyto and expect to commence a single arm phase three study by mid year this year. Now, over to David Lynn for a commercial update.

David Lin
David Lin
Chief Commercial Officer at UroGen Pharma

Thank you, Mark. Our organization is now fully engaged in the final stages of prelaunch activities for UGN one zero two. The goal is to deliver a seamless and impactful launch that will ensure timely access for patients as soon as possible following approval. Our team is highly energized, and we have the necessary experience, resources, and talent to drive adoption. Our clinical data supports our conviction that UGN one zero two can be a transformative product that will change the standard of care in recurrent, low grade, intermediate risk, non muscle invasive bladder cancer.

David Lin
David Lin
Chief Commercial Officer at UroGen Pharma

There are three ongoing activities that I want to highlight. First, our medical affairs team is leading an educational effort, engaging directly with urologists to highlight the unmet needs in recurrent low grade intermediate risk NMIBC and ensure strong awareness of the clinical evidence supporting UGN one zero two. Second, we are scaling the organization to capture a larger opportunity in low grade intermediate risk non muscle invasive bladder cancer. Cancer. This includes expanding our sales force from approximately 50 reps today to over 80 at launch.

David Lin
David Lin
Chief Commercial Officer at UroGen Pharma

We are also building out the rest of the commercial infrastructure, including a robust patient support and distribution network. Third, we are executing a focused payer engagement strategy. Our market access and medical affairs teams are actively engaging with payers and formulary decision makers to communicate the clinical data supporting UGN one zero two. We recognize that timely access can make a meaningful difference for patients, and our goal is to ensure coverage decisions are aligned closely with approval to support rapid adoption. If approved, UGN one zero two will launch with a temporary miscellaneous j code, and we anticipate securing a permanent product specific j code by January 2026, which will be particularly important for broad adoption in the community setting.

David Lin
David Lin
Chief Commercial Officer at UroGen Pharma

In the first six to nine months, our strategy is to focus on a defined group of urologists who have demonstrated a willingness to adopt new therapies during the miscellaneous J code period. We are also working to identify affiliate sites of care for these physicians to help facilitate access and to support product administration. Our experience with Jelmyto, along with recent customer insights, tells us that many providers prefer to initiate use of new therapies in the hospital outpatient setting where pharmacy budgets are often managed as separate cost centers. During this initial phase, our goal is to establish a strong foundation for UGN one zero two adoption, positioning us for broader expansion once the permanent J code is in place. As we enter the final phase of prelaunch execution, we do so with a confidence and momentum. The interest from the health care community in UGN one zero two has been very encouraging, and we are implementing a robust strategy to support its introduction. Now turning to Jelmyto. First quarter sales were $20,300,000 with demand continuing to grow at a double digit pace. We remain focused on high frequency engagement with our top performing accounts. As we scale our commercial organization in preparation for the anticipated launch of UGN 102, our expanded sales force will also support continued promotion of Jelmyto.

David Lin
David Lin
Chief Commercial Officer at UroGen Pharma

This integrated effort will allow us to deliver broader utilization across both products while maximizing the impact of our commercial infrastructure. I will now turn the call over to Chris Degnan to review our financial results.

Chris Degnan
Chris Degnan
CFO at UroGen Pharma

Thank you, David. Joe Mido net product revenues were $20,300,000 and $18,800,000 for the three months ended March 31, '20 '20 '5 and 2024, respectively. Year over year revenue growth of 8% was driven by underlying demand growth of 12%, partially offset by higher 340B chargebacks. The gross to net rate for Jelmyto has stabilized in recent quarters, and we expect resulting headwinds on year over year growth to be less impactful going forward. R and D expenses for the first quarter of twenty twenty five were $19,900,000 including noncash share based compensation expense of $600,000 as compared to $15,500,000 including noncash share based compensation expense of $500,000 for the same period in 2024.

Chris Degnan
Chris Degnan
CFO at UroGen Pharma

The year over year increase in R and D expenses was primarily driven by the equity consideration issued to Iconovir for the acquisition of UGN-five zero one, which was expensed in the quarter, higher manufacturing costs, and costs associated with the Phase III Utopia trial for UGN-one hundred three, partially offset by lower clinical trial costs and regulatory expenses in connection with UGN-one hundred two. Selling, general and administrative expenses for the first quarter of twenty twenty five were $35,000,000 including noncash share based compensation expense of $2,500,000 This compares to $27,300,000 including noncash share based compensation expense of $2,200,000 for the same period in 2024. The year over year increase was primarily a result of UGN 102 commercial preparation activities. We reported noncash financing expense related to the prepaid forward obligation to RTW Investments of $4,600,000 in the first quarter of twenty twenty five compared to $5,700,000 in the same period in 2024. Interest expense related to the $125,000,000 term loan facility with funds managed by Pharmacun Advisors was $4,100,000 in the first quarter of twenty twenty five, compared to $2,400,000 in the same period in 2024.

Chris Degnan
Chris Degnan
CFO at UroGen Pharma

The increase was primarily driven by interest expense related to the $25,000,000 third tranche of the loan that was funded in September 2024. Net loss was $43,800,000 or $0.92 per basic and diluted share in the first quarter of twenty twenty five, compared with a net loss of $32,300,000 or $0.87 per basic and diluted share in the same period in 2024. As of 03/31/2025, cash, cash equivalents, and marketable securities totaled $200,400,000 Turning now to guidance. We last provided financial guidance with our year end 2024 results in March, and that guidance remains unchanged. We continue to expect full year 2025 net product revenues from Jelmyto to be in the range of $94,000,000 to $98,000,000 and this implies a year over year growth rate of approximately 8% to 12% over the 87,400,000 in demand driven Jelmyto sales in 2024, which excludes the $3,000,000 in Createx sales reported in 2024.

Chris Degnan
Chris Degnan
CFO at UroGen Pharma

Guidance on full year 2025 operating expenses is also unchanged, and is expected to be in the range of two fifteen million dollars to $225,000,000 including noncash share based compensation expense of 11,000,000 to $14,000,000 We're now ready to open the call for questions. Operator?

Operator

Thank Our first question comes from the line of Tara Bancroft with T. D. Cohen. Your line is open.

Tara Bancroft
Director, Senior Analyst, Biotech Equity Research at TD Cowen

Hi, good morning, and thanks for taking the questions. So I was hoping that, you know, in light of today's events, can you possibly give us the breakdown of Medicare and Medicaid exposure that you anticipate for UGN-one hundred two and currently for Jelmyto? Really, you think pricing dynamics could play out? Thanks so much.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Yes.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Hi, Tara, it's Liz. And I'll ask David to comment on that in a moment when I think about the percent of our business. But keep in mind that we are only a U. S.-focused company, so we don't have any issues from a best price favored nation perspective outside. So we don't have any risk associated with that. But David, can you just talk about the Medicare?

David Lin
David Lin
Chief Commercial Officer at UroGen Pharma

Yeah, hi, Tara. It's David. We anticipate that the Medicare population will comprise about 70% of our business, very consistent with the overall, patient demographic in low grade intermediate risk NMIBC. And, as we think about a launch, our principal priority will also be to drive reimbursement confidence with the providers who treat these patients. Thanks very much for the question. Appreciate it.

Tara Bancroft
Director, Senior Analyst, Biotech Equity Research at TD Cowen

Yeah, thank you.

Operator

Please stand by for our next question. Our next question comes from the line of Kelsey Goodwin with Guggenheim. Your line is open.

Kelsey Goodwin
Vice President at Guggenheim Partners

Oh, hey, good morning. Thanks for taking my question. Congrats on getting one step closer to this exciting approval. In terms of preparing for the ODAC, I guess, how are you preparing and where do you expect the most pushback from the panel and what do you think are your strongest arguments there? Thank you.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Yeah, hi Kelsey, it's Liz. Thanks for the question. We have been preparing for quite a few months, frankly since last fall. We have had our mock ODAC panels. We've had several now.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

We've had them with distinguished guests, that are medical oncologists that have been on ODAC, statisticians. So what we've tried to do is really have a situation where we are it's just like if we were at the ODAC. So a lot of the members have been ODAC members in the past, some have even been leaders of the ODAC. So we feel like we've really put our selves through the paces and we will continue to do so up until the day of the ODAC. I'm gonna give my perspective and then I'm gonna turn it over to Mark and ask him to comment as well.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

So, my perspective is that the biggest question is around the fact that ENVISION is a single arm study, it's the basis for an approval, for our approval, and how do the results, how do you those results, how do you put them in context? So in other words, it sounds great, right? Your 80% sounds great and your 80% sounds great, but I don't have anything to compare it to. And unfortunately, with the exception of our own study ATLAS, and we can talk about sort of the challenges with ATLAS, but except for our own study ATLAS, hasn't been a lot of peer reviewed studies or data published in this specific patient population, the low grade intermediate risk non invasive bladder cancer. So, it's really about putting it in context and how do you take a single arm study and put the data in context.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Having said that, we obviously feel really good about it. And I can tell you that in our mock ODACs, you know, we've gotten a positive vote. That's where it comes down to and we are not dealing with people who are being easy on us, trust me. And we give our presentation, we give an FDA presentation, we give them a briefing book. So, again, we try to simulate an ODAC meeting.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

And ultimately, in those situations, we have come out with a positive vote. But, Mark, do you will you comment one at the second part of Kelsey's question, which is why we feel good about where we are and what our arguments are and any other comments you have around you think potential challenges?

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

Sure, thanks Liz. We've got for starters, and actually this came out of extensive conversations with the FDA in preparation for this meeting, are the fact that as everybody listening probably remembers, we are working in a recurrent population. So the population we're treating and talking about in this meeting are patients who've already had surgery and who have recurred. So the agency identified this as the greatest unmet medical need, and we completely agree. These are patients who have failed the standard of care, and then they receive treatment, primary treatment as Liz said, with UGN-one hundred two, and achieve an eighty percent complete response rate profile, and eighteen months later have an over 80% durability of that complete response.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

These are, from a clinical perspective, remarkable results. I think one of our strongest arguments is that we have great results in a population of patients who didn't do well with the standard of care. So that's number one, and I think our experience with the mock panels, as Liz has pointed out, has been favorable precisely because of the encouraging safety profile and the sort of remarkable results of the ENVISION trial and the supportive data from ATLAS and OPTIMA. So think Liz has articulated where we think the conversation's gonna go, the importance of how to interpret the single arm trial. We've scoured the literature, and we've got all the supporting data you can possibly have for the single arm trial, the meaningfulness of ATLAS, the safety profile, and then the unmet medical need, and how to articulate that.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

And we believe those are gonna be the areas of conversation, and as Liz pointed out, we have been preparing for months to answer those questions.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

You know what, one thing I'll just add as well is we have two very strong KOLs that are joining us, they're part of our presentation. They'll be giving a presentation and they'll be also answering some of the questions. They have a lot of credibility, they're well known, again, a lot of credibility. So when questions do get asked, and they are the ones who are standing up answering it, it's very helpful for us. And so I'm really, really pleased with the two KOLs that are joining us for our presentation during the ODAC.

Kelsey Goodwin
Vice President at Guggenheim Partners

Great. Thank you so much for all the color, and, we're looking forward to watching it. Good luck.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Yes. Thanks, Kelsey.

Operator

Please stand by for our next question. Our next question comes from the line of Leland Gershell with Oppenheimer. Your line is open.

Leland Gershell
MD & Senior Biotechnology Analyst at Oppenheimer & Co. Inc.

Hey, good morning. Thanks for taking my questions. Two from us. First, just wanted to ask, in the FDA review process, we are about a month away from the PDUFA date. Just wondering, given the AdCom is still pending, just wanted to ask if you could share any color on your recent directions.

Leland Gershell
MD & Senior Biotechnology Analyst at Oppenheimer & Co. Inc.

Presumably you've had your late cycle meeting. Have you had the chance to discuss proposed labeling? Just wanted to ask if you're able to share any details there. And then the second question, I guess, Mark or David, you know, when I choose physician to become, as you say, a foundational therapy in low grade intermediate risk at the same time, you know, urologist adoption may begin with a certain type of patient profile. Just wondering what you see as kind of that, that most likely patient profile for urologists to start using one hundred two out of the sixty thousand patients, what fraction might that be? Thank you.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Sure. So, thanks Leland for the question. I would say we feel really good about kind of where we are with the FDA. We've had continuous interactions with them, them asking questions. You can tell from where we are and the questions that they are asking where they are in the review.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

And so we have no concerns about the PDUFA date, and we don't want to get into very specific conversation about our conversations with the FDA, but suffice it to say that it's very clear that they're at the end of their review, right? And we are in a position and it will be in a really good position post ODAC for that to move very quickly. So again, no concerns there. I think that as far as I think the conversation we had the mid cycle review around the label and switching it to the recurrent patient population has really made things really simplify things both from our presentation as well as in the labeling or discussions with the FDA. And so I think that has helped a tremendous amount.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

We did not have a late cycle meeting and we're not going to have a late cycle meeting. So when we met with them, I don't know if you recall, the mid cycle review meeting was really late, later than it was supposed to be. There hasn't been a need for a late cycle meeting and they informed us at that time that there would not be a late cycle review. So we won't have that. I'm going to ask David to talk to you about the populations in which we expect will be the kind of low hanging fruit and the first patients that physicians will use this on. So David?

David Lin
David Lin
Chief Commercial Officer at UroGen Pharma

Yeah, thanks Liz, and thanks Leland for the question. Coming out of the gates, first I'll just share, we've done extensive market research with physicians, and suffice it to say they are really pleased with the clinical data we've shared on UGN-one hundred two. They find it very compelling, and they see a need for it in their practices because of the multiple recurrences that their patients experience. Coming out of the gates, there's really three segments that we've teased out in talking to physicians. First, it's those patients who have multiple recurrences.

David Lin
David Lin
Chief Commercial Officer at UroGen Pharma

As you know, seventy percent of the patients have multiple recurrences. The second group are patients who are early recurs. And then finally, there's a small set of patients that are just not able to have surgery for one reason or the other. It could be because of polypharmacy or they can't handle anesthesia. But those are the primary patient populations that we think are very, very ripe for uptake when we launch UGN-one hundred two.

David Lin
David Lin
Chief Commercial Officer at UroGen Pharma

Importantly, though, one of the things around UGN-one hundred two is that it fits into the workflow of the urologist office. So we're very pleased that with minimal training, we can help onboard UGN-one hundred two into practice and make it a seamless experience. Appreciate the question.

Leland Gershell
MD & Senior Biotechnology Analyst at Oppenheimer & Co. Inc.

Thanks.

Operator

Thank you. Please stand by for our next question. Our next question comes from the line of Raghuram Selvaraju with H. C. Wainwright. Your line is open.

Raghuram Selvaraju
Managing Director, Senior Healthcare Analyst at H.C. Wainwright & Co., LLC

Thanks so much for taking my questions. Just in furtherance of what has already been asked on this call regarding the ODAC meeting. I was wondering if you could comment on two aspects in particular. Firstly, to what extent there has been communication regarding the purpose of the ODAC that pertains specifically to the lack of specific precedent for products being approved in low grade intermediate risk NMIBC, if you expect that to be a meaningful topic of discussion at the ODAC meeting with regard to UGN-one hundred two? And then secondly, to what extent do you expect the precedent case of Jelmyto to aid and facilitate the discussion around UGN-one hundred two to effectively put the committee in a position to be familiar with how UGN-one hundred two works, what its benefit level is, and effectively facilitate potentially direction towards a favorable panel vote, because of this precedent example that utilizes the same active ingredient and the same fundamental principle of delivery. Thank you.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Great points, Ram. And Mark, why don't you comment and then I'll add some color as well.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

Yeah, sure. Thanks for the insightful comments. With respect to the first question, I think it's implicit in your observation that this is a therapy that we're presenting to the agency, that the FDA would like a public conversation about what the meaningfulness of this approach to this disease would be in the setting of an expert panel. So we've always thought that that was part of the reason for the ODAC that the FDA has promised us for a very long time would be the case in the approval process for UGM-one hundred two. Because it is a different way of treating patients, because it is a primary therapy, because it does with an option that does not involve surgery, we think a robust discussion about that is going to take place within the context of what Liz was referring to earlier, namely the meaningful addition trial.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

That said, we believe we have ample data to support the meaningfulness of the clinical outcome we've observed in our trials, as well as the benefit to patients of having an option. It is interesting to remember that this would be the only urologic cancer to my knowledge, where patients really don't have a choice of therapies, they only have one therapy. So UGN-one hundred two would present in this population with the type of option that many patients with urologic cancers have in other settings, whether they be prostate or kidney cancer, or more advanced forms of bladder cancer. So, we think we can address that, and we think that is going to be a part of the conversation overall. And then, sorry, the second question is?

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

It was around the precedent case of Jelmyto.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

Oh, I'm sorry, yes. So we actually do, as part of our presentation, we actually remind or acquaint the committee with Jelmyto and its history and the fact that it has informative with respect to thinking about upper tract disease. So we have in fact put forward a segment of our presentation to specifically familiarize the advisory committee with Jelmyto and its history and the similarities with respect to approach and active ingredient to reassure them that this is in fact a follow on conceptually to what we've already had approved by the FDA. But Liz may want to comment as well.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Yeah, no, I think that's great. I think the only other comment I'll make is one of the reasons we want to do that is because the ODAC panel will be made up mostly of medical oncologists, right? So they will have ad hoc members, urologists, but because medical oncologists are not familiar with either this disease or our treatments, we felt like it was important and, Mark put that into the presentation. But great questions because those are exactly why we're actually going to the ODAC and exactly the right questions to ask. So thank you, Ram.

Raghuram Selvaraju
Managing Director, Senior Healthcare Analyst at H.C. Wainwright & Co., LLC

Just one quick follow-up if I may. Can you give us an update on the current status of the UGN-one hundred three clinical development program? And when you expect to report the next material update on the progress of that trial? Thank you.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Yeah, we're almost fully enrolled and so we'll be able to share that soon. So as soon as over the next couple of months, as soon as we're finished enrollment, we'll be able to provide the fact that we've finished enrollment will be by the end of the summer, and then we'll go from there. As far as the timing for the CR, all patients at CR and then all patients in durability. Those would be the next steps. You'll start to see data in '26.

Raghuram Selvaraju
Managing Director, Senior Healthcare Analyst at H.C. Wainwright & Co., LLC

Thank you.

Operator

Please stand by for our next question. Our next question comes from the line of Paul Choi with Goldman Sachs. Your line is open.

Paul Choi
Paul Choi
Analyst at Goldman Sachs

Hi, thank you and good morning everyone. I had a question about how maybe you're thinking about the market for 01/2002 here. David provided some nice color on the population size and opportunity as well as expected greater patient population in the community setting. But I was just curious, given the trend you're seeing with 340B utilization for Jelmyto, is that something structural you also expect for the one hundred two market? Just some color there would be helpful and just thinking about hospital pharmacy pricing.

Paul Choi
Paul Choi
Analyst at Goldman Sachs

And then my second question is for Mark, just in terms of your earlier comments on thinking about advancing 03/2001, sort of maybe can you provide us how you're thinking about maybe the bar for the go or no go decision for that program for the CTLA-four? Thank you very much.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Yes. The good news for UGM-one hundred two is the 340B we expect not the discounts not to be as great as they are for Jelmyto. Because to your point, we do expect that ultimately not at the beginning, but ultimately that it will be a sort of 70% of the business will be in the community based off of the comment that David made around that. So we are really hopeful and expecting that the $3.40 will be much less in UGM-one hundred two than it is for. But we have been more conservative with our assumptions also as we go into UGM-one hundred '2, because as you know, Jelmyto, that's been one of the headwinds we've shared because our data, our actual patient demand has been much greater, but we've seen some real challenges with the 340B.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Mark, do you want to talk about Paul's second question?

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

Sure, thanks Paul. So, as the audience will undoubtedly remember, three zero one is our intravesical immunotherapy approach to high grade disease, and it's a phase one study, both as Liz said earlier, a monotherapy study and in combination with our TLR7 agonist, CGN-two zero one as well as with gemcitabine. So as a phase one study, it's primarily focused on tolerability and safety and not on efficacy. That said, we have said publicly that we've seen a few responses and we are very interested in these responses for obvious reasons. But in a phase one study, it would be a little bit difficult to give you a numeric bar that we are expecting to be meaningful.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

What we are doing now is following patients to look for the durability of these responses. And later this year, we anticipate presenting those data probably at the SUO at the end of the year, at which time we'll have a chance to assess both the durability and quality of those responses in the combination arms. And at that point, we'll probably be in a position to make a gono go decision about a trial that would be able to answer the question that you're asking, namely what numeric bar would be meaningful for us in comparison to the other assets out there currently addressing this population. So, guess my short answer is, it's probably premature for me to give you a number, but we are encouraged that we're seeing responses, we're tracking those, and we'll be able to talk later this year about what would constitute a signal to us for gono go for phase two, and Liz may want to comment as well.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Yeah, my comments are really around, it's very clear that the bar for efficacy and safety, but particularly for efficacy is definitely higher than it was when we started the program, given the results that you've seen. I do think there's still a lot of opportunity in high grade disease for several reasons. One, these patients are not cured unfortunately, so they continue to see recurrences and so they need more treatments because these aren't cures. Two, I think all of the current programs that are out there that are seeing the higher complete response rates and durability are because you're seeing continuous dosing. So, almost all of them are requiring reinductions, continuous dosing.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

And so, I still think that there's opportunity if you can have a situation like we have with UGN-one hundred two in intermediate risk, where actually we're given six weeks and then the patients don't have treatment. And I think that's very important for patients to not only be recurrence free, but also treatment free. And so being able to have a more simple administration and administration schedule, although I do believe that in high grade you're more likely to need to have some sort of maintenance or retreatment, I do think that there's still a lot of opportunity, but we will be very diligent and we'll be very critical before we launch into a very expensive Phase II or Phase III study with UGN-three zero one. And also I just want to mention five zero one, right? We're very excited about that.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

So we will have to make a determination for 03/2001 and '5 zero '1 about where we move and when we move and how we move. Those are all things that we're working on right now. As Mark said, a little too early to comment except to understand that the bar is definitely higher. We believe that both of those approaches actually can easily reach that bar and potentially simplify the administration.

Paul Choi
Paul Choi
Analyst at Goldman Sachs

Great. Thank you for the color.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Thank you. Thanks, Paul.

Operator

Our next question comes from the line of George Farmer with Scotiabank. Your line is open.

George Farmer
Managing Director at Scotiabank

Hi, good morning. Thanks for taking my questions. A few from me. Can you confirm that the FDA representatives you've been speaking with are still employed with the agency? That's number one.

George Farmer
Managing Director at Scotiabank

Number two, this eighteen month data that you presented at AUA, is that going to be included during the discussion at ODAC? And number three, do you see a need or are you planning for any sort of direct to consumer advertising campaign? Thanks.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Yeah, great question, Joyce. Thank you very much. The FDA team and knock on wood is still in place, the same team that have been there since Jelmyto frankly. So hopefully over the next few weeks that team stays in place, but they're still the same people that we've been working with all along and we are we can confirm that they're still employed in there. The eighteen month data has already been incorporated.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

The FDA received that before it was even public. So all that data is, with the FDA and has been and is part of all of our discussions and presentation with them. And I'm sorry, forgot the last question.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

Direct to consumer.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Oh, direct to consumer. I wouldn't expect broad based direct to consumer, but we will absolutely have programs that engage the patient. It's really important, I think you've heard, several times we talk about the fact that ninety percent of patients prefer UGM-one hundred two to a TURBT. These are patients that have had a TURBT and they've had UGM-one hundred two and ninety percent of them prefer UGM-one hundred two. So, we really need to ensure that we engage the patient because we want that physician, we want them to be part of the discussion and part of the treatment decision.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

So when a physician gives them the option that they understand and know. So we will again engage a patient, but not expected initially to be in broad based DTC.

George Farmer
Managing Director at Scotiabank

Okay. Thanks, Liz.

Operator

Thank you. Please stand by for our next question. Our next question comes from the line of Ayden Hausanov with Ladenburg. Your line is open.

Aydin Huseynov
Equity Research Analyst at Ladenburg Thalmann & Co. Inc

Hi, good morning, everyone. Thank you for taking our questions. I got a couple. Maybe this is a little bit unusual question, but curious what would happen to patients who progress on UGN-one hundred two, like twenty or 20 five, whatever percentage. So, they switch back to TURBT, or would you re dose them with UGN-one hundred two?

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Yeah, Mark, do you want to take that?

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

Sure. So, let me start out by saying that thankfully, very few patients did what's called progress during treatment across our program, and when we say progression, what we're talking about is a patient with low grade disease turning into someone with high grade disease, or even more unusually, turning into someone with invasive disease. So, that eventuality, that rarely occurred in our program. That said, in patients who responded and then recurred, or didn't respond completely after treatment with UGN-one hundred two, the use of transurethral resection to remove any residual or recurrent tumor was not associated with complications or problems that were exceptional or unusual. So the standard of care currently, which is our experience in our clinical trials program is that it's not complicated by prior use with, or treatment with UGM-one hundred two.

Mark Schoenberg
Mark Schoenberg
Chief Medical Officer at UroGen Pharma

As for what you're anticipating, is, you know, if approved and patients are successfully treated initially with UGM-one hundred one, they subsequently be treated assuming they had the same disease? I think I'm going to defer to Liz as to how she thinks that would happen. We don't currently have clinical trials data to tell us, specifically about what retreatment would mean or look like. But, Liz, I don't know if you want to, postulate what you think would happen once approved.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Yeah, I mean, I think it's very similar to what you would see in other cancers. If a patient gets a good response and a durable response and then they recur, physicians are likely to retreat with what they used the first time. And so we expect that to happen. We will absolutely either launch a Phase IV study or have a registry or some way to generate data that shows what happens when patients recur and get retreated. And then the other patient population that we'll want to study as well are partial responders.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

So, I don't know if you remember, but when we did the announcement of the durability, one of the doctors talked about the fact that one patient had so much tumor and UTM-one hundred two got 90% of the tumor, but they couldn't be considered a complete responder. So, what would happen if you gave that patient a couple of additional treatments? Could you take these partial responders and put them into complete response? So we will be looking at all of those questions, through either IRRs, Phase four, registry, but we will have a way to generate data that will support the use of UGM-one hundred two once a patient has recurred either on our drug or after a TURBT. So, very much expect that you would be able to retreat very much like you do, like I said, in other cancers.

Aydin Huseynov
Equity Research Analyst at Ladenburg Thalmann & Co. Inc

Thank you, I appreciate that. And another question is commercial question.

Aydin Huseynov
Equity Research Analyst at Ladenburg Thalmann & Co. Inc

Could you clarify the GTN gross to net for Jelmyto and what should be the expectation of GTN for UGN-one hundred 2?

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Yep, Chris.

Chris Degnan
Chris Degnan
CFO at UroGen Pharma

So, Aidan, we've been, you know, kind of in the mid-70s percent net of gross for Jelmyto. And the one thing we said on the call is we do expect that the headwinds we've been experiencing for 340B, some of those headwinds have started to annualize a bit. So from a year over year growth perspective, as we think about the rest of this year, we would expect that impact to be less impactful in terms of our growth rate. In terms of UGN-one hundred two, as Liz mentioned, over time, we do expect the gross to net profile to be more favorable for UGN-one hundred two as compared to Jelmyto, mainly because mix of business to be more heavily weighted towards community. You know, initially at launch, we do expect more utilization in the hospital setting, but over time that that should shift over to the community.

Aydin Huseynov
Equity Research Analyst at Ladenburg Thalmann & Co. Inc

Okay, thanks so much for taking questions.

Operator

Thank you. Ladies and gentlemen, I'm showing no further questions in the queue. I would now like to turn the call back to Liz for closing remarks.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

Well, thanks everybody. As you can imagine, a lot of excitement over here as we prepare and get ready for the ODAC next week. We appreciate all of your support. It's been a long time coming. We're excited about it though.

Liz Barrett
Liz Barrett
President & CEO at UroGen Pharma

We feel really good about it. And most importantly, these patients need new options. So, we're very much looking forward to next week and then following that, the PDUFA. So, thanks and we'll keep you guys informed of any happening. So, thanks a lot. We'll talk to you guys soon. Bye bye.

Operator

Ladies and gentlemen, that concludes today's conference call. Thank you for your participation. You may now disconnect.

Executives
    • Vincent Perrone
      Vincent Perrone
      Senior Director of Investor Relations
    • Liz Barrett
      Liz Barrett
      President & CEO
    • Mark Schoenberg
      Mark Schoenberg
      Chief Medical Officer
    • David Lin
      David Lin
      Chief Commercial Officer
    • Chris Degnan
      Chris Degnan
      CFO
Analysts
    • Tara Bancroft
      Director, Senior Analyst, Biotech Equity Research at TD Cowen
    • Kelsey Goodwin
      Vice President at Guggenheim Partners
    • Leland Gershell
      MD & Senior Biotechnology Analyst at Oppenheimer & Co. Inc.
    • Raghuram Selvaraju
      Managing Director, Senior Healthcare Analyst at H.C. Wainwright & Co., LLC
    • Paul Choi
      Analyst at Goldman Sachs
    • George Farmer
      Managing Director at Scotiabank
    • Aydin Huseynov
      Equity Research Analyst at Ladenburg Thalmann & Co. Inc
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Key Takeaways

  • NDA & ODAC: UroGen’s NDA for UGN-102 is in final FDA review with a June 13 PDUFA date and an ODAC meeting on May 21, supported by trial data showing an 80.6% complete response durability at 18 months in low-grade intermediate-risk NMIBC.
  • Commercial readiness: The company plans to expand its sales force from ~50 to over 80 representatives and target a July launch upon approval, leveraging community urologist workflows to address a $5 billion annual market opportunity.
  • Jelmyto performance: First-quarter Jelmyto net product revenues reached $20.3 million, up 8% year-over-year driven by 12% underlying demand growth, with gross-to-net dynamics stabilizing and continued promotion ahead of UGN-102 launch.
  • Pipeline expansion: UroGen is advancing next-generation Jelmyto (UGN-104) and UGN-103 Phase III trials, has acquired oncolytic virus candidate ICOV-1042 (UGN-501), and continues early immuno-oncology programs evaluating UGN-301.
  • Financial strength & guidance: As of March 31, cash and marketable securities totaled $200.4 million, and full-year 2025 guidance remains unchanged with Jelmyto revenues of $94–98 million and operating expenses of $215–225 million.
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