UroGen Pharma - Earnings Call - Q1 2025
May 12, 2025
Executive Summary
- Q1 2025 revenue was $20.3M, up 8% year over year, but below Wall Street consensus; diluted EPS was -$0.92, also below consensus. Underlying demand grew 12% but was partially offset by higher 340B chargebacks, pressuring gross-to-net and results.
- Regulatory catalysts: ODAC meeting (May 21, 2025) and PDUFA target (June 13, 2025) for UGN-102; management expects immediate launch readiness post-approval with July product availability.
- Guidance unchanged: FY2025 JELMYTO net product revenue $94–$98M; FY2025 operating expenses $215–$225M (includes $11–$14M SBC), signaling confidence despite Q1 variance versus consensus.
- Strategic spend rose for UGN-102 commercial prep and UGN-103/UGN-501 pipeline work; cash and marketable securities were $200.4M at quarter-end, supporting near-term launch and pipeline execution.
- Near-term stock reaction drivers: ODAC outcome and PDUFA decision; commercial execution plans include expanding sales force to >80 reps, prioritized payer engagement, and misc J-code launch path for UGN-102.
What Went Well and What Went Wrong
What Went Well
- JELMYTO delivered $20.3M in Q1 revenue (+8% YoY), driven by 12% underlying demand growth, evidencing durable franchise momentum despite gross-to-net headwinds.
- Regulatory progress for UGN-102: ODAC scheduled and PDUFA date set, with robust ENVISION durability data (18‑month DOR 80.6% after 3‑month CR) showcased at AUA; management emphasized preparedness and strong clinical package.
- CEO tone confident: “We are entering a pivotal and exciting period…UGN-102 represents a significant commercial opportunity…over $5 billion”.
What Went Wrong
- Revenue and EPS missed consensus; higher 340B chargebacks and elevated SG&A for pre-launch activities weighed on results, with net loss widening to -$43.8M (vs -$32.3M a year ago).
- Sequential revenue decline vs Q4 2024 ($24.6M to $20.3M) as gross-to-net headwinds persisted; management noted stabilization but acknowledged ongoing impact.
- R&D spend increased due to UGN-501 equity consideration and UGN-103 trial costs, contributing to broader operating losses and EPS shortfall versus Street.
Transcript
Operator (participant)
Hello, and welcome to UroGen Pharma first quarter 2025 earnings conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during the session, you will need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 11 again. I would now like to turn the conference over to Vincent Perrone. You may begin.
Vincent Perrone (Senior Director of Investor Relations)
Thank you, Operator. Good morning, everyone, and welcome to UroGen Pharma's first quarter 2025 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 March 31, 2025. The press release can be accessed on the Investors portion of our website at investors.urogen.com. Joining me on the call today are Liz Barrett, President and Chief Executive Officer, Dr. Mark Schoenberg, Chief Medical Officer, David Lin, Chief Commercial Officer, and Chris Degnan, 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-102, regulatory meetings and decisions, our commercialization strategy and expectations, as well as potential future commercialization activities for UGN-102, if approved, market and revenue opportunities, commercialization activities related to JELMYTO, our ongoing and planned clinical trials, commercial and clinical milestones, UGN-102 being the primary growth driver for UroGen, future R&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. 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 (President and CEO)
Thank you, Vincent, and thank you all for joining us this morning. The new drug application for our lead development stage candidate, UGN-102, is now in the final stages of FDA review, with a PDUFA target date of June 13. We designed UGN-102 as a novel and innovative treatment for patients with low-grade, intermediate-risk, non-muscle-invasive bladder cancer. We believe UGN-102 has the potential to change the treatment paradigm and meaningfully improve the standard of care in this patient population. If approved, UGN-102 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-centered solutions to urologic cancers. We have been informed by the FDA that an Oncologic Drugs Advisory Committee, or ODAC, meeting has been scheduled for UGN-102 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 ODAC meeting and believe we are well prepared to present a clear, compelling, and scientifically robust case supporting the approval of UGN-102. In parallel, our regulatory team continues to engage regularly with the agency and has been responding to their information requests. To date, we've encountered no resource or policy-related issues that concern us. The UGN-102 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 80.6% of patients who achieved a CR at three months remained in response at 18 months, per Kaplan-Meier estimate. It's important to highlight that these robust results are with UGN-102 alone and not following a transurethral resection of bladder tumor. Additionally, there are six weekly instillations, then patients are treatment-free until recurrence. As Mark will highlight, these results, along with the broader update on UGN-102 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 UGN-102.
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 pre-launch planning. We are targeting commercial readiness by June, and we will be ready to promote immediately following approval with product availability in July. UGN-102 represents a transformative growth opportunity for UroGen. We estimate an addressable population of approximately 60,000 patients annually with recurrent, low-grade, intermediate-risk, non-muscle-invasive bladder cancer, translating to a market opportunity of over $5 billion. 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. UGN-102 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 UGN-102 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 $20.3 million in first-quarter sales and 8% year-over-year growth compared to the first quarter of 2024, 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-102, as well as our emerging immuno-oncology initiatives.
In February, we acquired product candidate ICVB-1042, a next-generation investigational oncolytic virus from Econovir, which we have assigned an internal code name of UGN-501. This is an important step in expanding our presence in immune-based therapies for urologic cancers. In parallel, we also have multiple strategic 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 million in cash, cash equivalents, and marketable securities as of March 31. 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 Schoenberg, who will provide a clinical update.
Mark?
Mark Schoenberg (Chief Medical Officer)
Thank you, Liz. I would like to echo what Liz shared regarding the strength of the clinical data supporting UGN-102. We have assembled a compelling and comprehensive clinical package that we believe demonstrates both the safety and efficacy of UGN-102. 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. It provided a valuable platform to present the latest data on UGN-102 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 18-month duration of response data from the phase III ENVISION trial, which, as you know, is the pivotal trial that is the foundation of our NDA for UGN-102.
The data were featured in a podium presentation by Dr. Sandip Prasad, the principal investigator on the study. We are highly encouraged by the continued durability of response observed in the phase III ENVISION trial. Among patients who achieved a complete response, the duration of response at 18 months remains strong at 80.6% by Kaplan-Meier estimate. Median follow-up time has now extended to 18.7 months post three-month CR, up from 13.8 months at the previous data cut, and the median duration of response has still not been reached. For reference, the 12-month duration of response was previously reported at 82.5%. These results continue to reinforce the potential of UGN-102 to offer a durable, non-surgical treatment approach for patients with recurrent low-grade, intermediate-risk NMIBC. We also presented a poster featuring patient-reported outcomes from the three late-stage UGN-102 studies, OPTIMA II, ATLAS, and ENVISION.
These assessments used the EORTC 24-item quality of life questionnaire specific to NMIBC to evaluate the symptom burden and overall health status and function. The findings were consistent across trials, showing that treatment with UGN-102 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 I dose escalation study of UGN-301, our investigational anti-CTLA-4 antibody delivered via RTGel. These results were previously presented at SUO in late 2024 and continue to support a favorable safety profile for UGN-301, both as monotherapy and in combination with UGN-201, 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 go-no-go decision on whether to advance UGN-301 into phase II development. Our next-generation candidates are actively advancing through development. Enrollment is ongoing in the phase III Utopia trial, which is evaluating UGN-103 in patients with recurrent low-grade, intermediate-risk NMIBC. Utopia is a single-arm, multi-center 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-104, our next-generation formulation of JELMYTO, and expect to commence a single-arm phase III study by mid-year this year.
Now, over to David Lin for a commercial update.
David Lin (Chief Commercial Officer)
Thank you, Mark. Our organization is now fully engaged in the final stages of pre-launch activities for UGN-102. 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-102 can be a transformative product that will change the standard of care in recurrent, low-grade, intermediate-risk, non-muscle-invasive bladder cancer. 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-102. Second, we are scaling the organization to capture the larger opportunity in low-grade, intermediate-risk, non-muscle-invasive bladder cancer.
This includes expanding our sales force from approximately 50 reps today to over 80 at launch. 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-102. 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-102 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.
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-102 adoption, positioning us for broader expansion once the permanent J-Code is in place. As we enter the final phase of pre-launch execution, we do so with confidence and momentum.
The interest from the healthcare community in UGN-102 has been very encouraging, and we are implementing a robust strategy to support its introduction. Now, turning to JELMYTO, first-quarter sales were $20.3 million, 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. 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 (CFO)
Thank you, David. JELMYTO net product revenues were $20.3 million and $18.8 million for the three months ended March 31, 2025 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&D expenses for the first quarter of 2025 were $19.9 million, including non-cash share-based compensation expense of $0.6 million, as compared to $15.5 million, including non-cash share-based compensation expense of $0.5 million for the same period in 2024.
The year-over-year increase in R&D expenses was primarily driven by the equity consideration issued to IconOVir for the acquisition of UGN-501, which was expensed in the quarter, higher manufacturing costs, and costs associated with the phase III UTOPIA trial for UGN-103, partially offset by lower clinical trial costs and regulatory expenses in connection with UGN-102. Selling, general and administrative expenses for the first quarter of 2025 were $35 million, including non-cash share-based compensation expense of $2.5 million. This compares to $27.3 million, including non-cash share-based compensation expense of $2.2 million for the same period in 2024. The year-over-year increase was primarily a result of UGN-102 commercial preparation activities. We reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $4.6 million in the first quarter of 2025, compared to $5.7 million in the same period in 2024.
Interest expense related to the $125 million term loan facility with funds managed by Pharmakon Advisors was $4.1 million in the first quarter of 2025, compared to $2.4 million in the same period in 2024. The increase was primarily driven by interest expense related to the $25 million third tranche of the loan that was funded in September 2024. Net loss was $43.8 million, or $0.92 per basic and diluted share in the first quarter of 2025, compared with a net loss of $32.3 million, or $0.87 per basic and diluted share in the same period in 2024. As of March 31, 2025, cash, cash equivalents, and marketable securities totaled $200.4 million. 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-$98 million. This implies a year-over-year growth rate of approximately 8%-12% over the $87.4 million in demand-driven JELMYTO sales in 2024, which excludes the $3 million in crates sales reported in 2024. Guidance on full-year 2025 operating expenses is also unchanged. It is expected to be in the range of $215-$225 million, including non-cash share-based compensation expense of $11-$14 million. We're now ready to open the call for questions. Operator.
Operator (participant)
Thank you. Ladies and gentlemen, as a reminder to ask the question, please press star 11 on your telephone. Then wait for your name to be announced. To withdraw your question, please press star 11 again. Please stand by while we compile the Q&A roster. Our first question comes from the line of Tara Bancroft with TD Cowen. Your line is open.
Tara Bancroft (Director and Senior Analyst of Biotech Equity Research)
Hi, good morning, and thanks for taking the question. 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-102 and currently for JELMYTO, and really how you think pricing dynamics could play out? Thanks so much.
Liz Barrett (President and CEO)
Yeah, hi, Tara, It's Liz, and I'll ask David to comment on that in a moment when thinking about the percent of our business, but keep in mind that we're only a U.S.-focused company, so we don't have any issues from a best price perspective, favored nation perspective outside. We don't have any risk associated with that. David, can you just talk about the Medicare?
David Lin (Chief Commercial Officer)
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 and low-grade, intermediate-risk NMIBC. 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 and Senior Analyst of Biotech Equity Research)
Yeah, thank you.
Operator (participant)
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 (VP)
Oh, hey, good morning, and 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 (President and CEO)
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. We've had them with distinguished guests that were our medical oncologists that have been on ODAC, that statisticians. What we've tried to do is really have a situation where we are—it's just like if we were at the ODAC. A lot of the members have been ODAC members in the past. Some have even been leaders of the ODAC. We feel like we've really put ourselves through the paces, and we will continue to do so up until the day of the ODAC. I'm going to give my perspective, and then I'm going to turn it over to Mark and ask him to comment as well.
My perspective is that the biggest question is around the fact that ENVISION is a single-arm study. It's the basis for our approval. How did the results—how do you compare those results? How do you put them in context? In other words, it sounds great, right? Your 80% sounds great, and your 80% sounds great, but I do not have anything to compare it to. Unfortunately, with the exception of our own study, ATLAS, and we can talk about the challenges with ATLAS, except for our own study, ATLAS, there has not been a lot of peer-reviewed studies or data published in this specific patient population, the low-grade, intermediate-risk non-muscle-invasive bladder cancer. It is really about putting it in context, and how do you take a single-arm study and put the data in context. Having said that, we obviously feel really good about it.
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. We give our presentation, we give an FDA presentation, we give them a briefing book. Again, we try to simulate an ODAC meeting. Ultimately, in those situations, we have come out with a positive vote. Mark, will you comment on 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 what you think potential challenges are?
Mark Schoenberg (Chief Medical Officer)
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. The population we're treating and talking about in this meeting are patients who've already had surgery and who have recurred. The agency identified this as the greatest unmet medical need, and we completely agree. These are patients who have failed the standard of care. They have received treatment, primary treatment, as Liz said, with UGN-102, and achieved an 80% complete response rate per the profile, and 18 months later have an over 80% durability of that complete response. 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 did not do well with the standard of care. That is number one. 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. I think Liz has articulated where we think the conversation is going to go, the importance of how to interpret the single-arm trial. We have scoured the literature, and we have 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. We believe those are going to be the areas of conversation.
As Liz pointed out, we have been preparing for months to answer those questions.
Liz Barrett (President and CEO)
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. When questions do get asked, and they are the ones who are standing up answering it, it's very helpful for us. I'm really, really pleased with the two KOLs that are joining us for our presentation during the ODAC.
Kelsey Goodwin (VP)
Great. Thank you so much for all the color, and we're looking forward to watching it. Good luck.
Liz Barrett (President and CEO)
Yeah, thanks. Thanks, Kelsey.
Operator (participant)
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 (Managing Director and Senior Analyst)
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. 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. The second question, I guess, for Mark or David, you want to choose position to become, as you say, a financial therapy in low-grade, intermediate-risk. At the same time, urologist adoption may begin with a certain type of patient profile. Just wondering what you see as kind of that most likely patient profile for urologists to start using 102 out of the 60,000 patients.
What fraction might that be? Thank you.
Liz Barrett (President and CEO)
Sure. 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, all of them asking questions. You can tell from where we are in the questions that they are asking where they are in the review. We have no concerns about the PDUFA date. We don't want to get into a 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? We are in a position, and we'll be in a really good position post-ODAC for that to move very quickly. 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 simplified things, both from our presentation as well as any of the labeling or discussions with the FDA. I think that has helped a tremendous amount. We did not have a late cycle meeting, and we're not going to have a late cycle meeting. When we met with them, I do not know if you recall, the mid-cycle review meeting was really later than it was supposed to be. There has not been a need for a late cycle meeting, and they informed us at that time that there would not be a late cycle review. We will not 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. David.
David Lin (Chief Commercial Officer)
Yeah, thanks, Liz. Thanks, Leland, for the question. Coming out of the gates, first, I'll just share. We've done extensive market research with physicians. Suffice it to say, they are really pleased with the clinical data we've shared on UGN-102. 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. As you know, 70% of the patients have multiple recurrences. The second group are patients who are early recurs. 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.
Those are the primary patient populations that we think are very, very ripe for uptake when we launch UGN-102. Importantly, though, one of the things around UGN-102 is that it fits into the workflow of the urologist office. We are very pleased that with minimal training, we can help onboard UGN-102 into practice and make it a seamless experience. Appreciate the question.
Leland Gershell (Managing Director and Senior Analyst)
Thanks.
Operator (participant)
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 of Healthcare Equity Research)
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-102.
To what extent do you expect the precedent case of JELMYTO to aid and facilitate the discussion around UGN-102 to effectively put the committee in a position to be familiar with how UGN-102 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 (President and CEO)
Okay. Great points, Ram. Mark, why don't you comment, and then I'll add some color as well.
Mark Schoenberg (Chief Medical Officer)
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 sort of 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. We have 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 UGN-102 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 meaningfulness of the trial.
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 do not have a choice of therapies. They only have one therapy. UGN-102 would present to 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. 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 (President and CEO)
It was around the precedent case of JELMYTO.
Mark Schoenberg (Chief Medical Officer)
Oh, I'm sorry, yes. We actually do, as part of our presentation, remind or acquaint the committee with JELMYTO and its history and the fact that it is informative with respect to thinking about upper tract disease. 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, the follow-on conceptually to what we've already had approved by the FDA. Liz may want to comment as well.
Liz Barrett (President and CEO)
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? They will have ad hoc members, urologists. Because medical oncologists are not familiar with either this disease or our treatments, we felt like it was important. Mark put that into the presentation. Great questions because those are exactly why we're actually going to the ODAC and exactly the right questions to ask. Thank you, Ram.
Raghuram Selvaraju (Managing Director of Healthcare Equity Research)
Just one quick follow-up, if I may. Can you give us an update on the current status of the UGN-103 clinical development program and when you expect to report the next material update on the progress of that trial? Thank you.
Liz Barrett (President and CEO)
Yeah, we're almost fully enrolled, and we'll be able to share that soon. 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. 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 2026.
Raghuram Selvaraju (Managing Director of Healthcare Equity Research)
Thank you.
Operator (participant)
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 (Biotechnology Analyst)
Hi. Thank you. Good morning, everyone. I had a question about how maybe you're thinking about the market for 102 here. David provided some nice color on the population size and opportunity, as well as expected greater patient population in the community setting. I was just curious, given the trend you're seeing with 340B utilization for JELMYTO, is that something structural you also expect for the 102 market? Some color there would be helpful in thinking about hospital pharmacy pricing. My second question is for Mark, just in terms of your earlier comments on thinking about advancing 301, maybe can you provide us how you're thinking about the bar for the go or no-go decision for that program for the CTLA-4? Thank you very much.
Liz Barrett (President and CEO)
Yeah, the good news for UGN-102 is the 340B, we expect 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, it will be a sort of 70% of the business will be in the community based off of the comments that David made around that. We are really hopeful and expecting that the 340B will be much less in UGN-102 than it is for. We have been more conservative with our assumptions also as we go into UGN-102 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. Mark, do you want to talk about Paul's second question?
Mark Schoenberg (Chief Medical Officer)
Sure. Thanks, Paul. As the audience will undoubtedly remember, 301 is our intravesical immunotherapy approach to high-grade disease. It is a phase one study, both, as Liz said earlier, a monotherapy study and in combination with our TLR7 agonist, UGN-201, as well as with gemcitabine. As a phase one study, it is primarily focused on tolerability and safety and not on efficacy. That said, we have said publicly that we have seen a few responses, and we are very interested in these responses for obvious reasons. 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. What we are doing now is following patients to look for the durability of these responses.
Later this year, we anticipate presenting those data probably at the SUO at the end of the year, at which time we'll have had a chance to assess both the durability and quality of those responses in the combination arms. At that point, we'll probably be in a position to make a go, no-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. I 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 go, no-go for phase two.
Liz may want to comment as well.
Liz Barrett (President and CEO)
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. Almost all of them are requiring reinductions, continuous dosing. I still think that there's opportunity if you can have a situation like we have with UGN-102 in intermediate risk, where actually we're given six weeks, and then the patients don't have treatment.
I think that's very important for patients to not only be recurrence-free, but also treatment-free. 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. We will be very diligent and will be very critical before we launch into a very expensive phase two or phase three study with UGN-301. I just want to mention 501, right? We're very excited about that. We will have to make a determination for 301 and 501 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 (Biotechnology Analyst)
Great. Thank you for the color.
Liz Barrett (President and CEO)
Thank you.
Mark Schoenberg (Chief Medical Officer)
Thanks, Paul.
Operator (participant)
Please stand by for our next question. Our next question comes from the line of George Farmer with Scotiabank. Your line is open.
George Farmer (Managing Director of Biotechnology)
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. Number two, this 18-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 (President and CEO)
Yeah, great questions, George. Thank you very much. The FDA team at Knoxville Wood is still in place, the same team that has been there since JELMYTO, frankly. Hopefully, over the next few weeks, that team stays in place. They're still the same people that we've been working with along, and we can confirm that they're still employed in there. The 18-month data has already been incorporated. The FDA received that before it was even public. All that data is with the FDA and has been and is part of all of our discussions and presentation with them. I'm sorry, I forgot the last question.
George Farmer (Managing Director of Biotechnology)
Direct-to-consumer.
Liz Barrett (President and CEO)
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 90% of patients prefer UGN-102 to a TURBT. These are patients that have had a TURBT, and they've had UGN-102, and 90% of them prefer UGN-102. 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. When a physician gives them the option, that they understand and know. We will, again, engage a patient, but not expected initially to be in broad-based DTC.
George Farmer (Managing Director of Biotechnology)
Okay. Thanks, Liz.
Operator (participant)
Thank you. Please stand by for our next question. Our next question comes from the line of Aydin Huseynov with Lattenberg. Your line is open.
Aydin Huseynov (Managing Director of Equity Research)
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-102 with 20% or 25%, whatever percentage. So would they switch back to TURBT or would you redose them with UGN-102?
Liz Barrett (President and CEO)
Yeah, Mark, do you want to take that?
Mark Schoenberg (Chief Medical Officer)
Sure. Let me start out by saying that thankfully, very few patients did what's called progress during treatment across UGN-102 program. 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. That eventuality, that rarely occurred in our program. That said, in patients who responded and then recurred or did not respond completely after treatment with UGN-102, the use of transurethral resection to remove any residual or recurrent tumor was not associated with complications or problems that were exceptional or unusual. The standard of care currently, which is our experience in our clinical trials program, is that it is not complicated by prior use or treatment with UGN-102.
As for what you're anticipating, which is if approved and patients are successfully treated initially with UGN-102, and would 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. Liz, I don't know if you want to postulate what you think would happen once approved.
Liz Barrett (President and CEO)
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. We expect that to happen. We will absolutely either launch a phase four study or have a registry or some way to generate data that shows what happens when patients recur and get retreated. The other patient population that we'll want to study as well are partial responders. 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 UGN-102 got 90% of the tumor, but they couldn't be considered a complete responder.
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? 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 UGN-102 once a patient has recurred either on our drug or after a TURBT. Very much expect that you would be able to retreat very much like you do, like I said, in other cancers.
Aydin Huseynov (Managing Director of Equity Research)
Thank you. Appreciate that. Another question, this commercial question. Could you clarify the GTN gross to net for JELMYTO, and what should be the expectation of GTN for UGN-102?
Liz Barrett (President and CEO)
Yep, Chris.
Chris Degnan (CFO)
We've been kind of in the mid-70% net of gross for JELMYTO. 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. 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-102, as Liz mentioned, over time, we do expect the gross-to-net profile to be more favorable for UGN-102 as compared to JELMYTO, namely because mix of business to be more heavily weighted towards community. Initially, at launch, we do expect more utilization in the hospital setting, but over time, that should shift over to the community.
Aydin Huseynov (Managing Director of Equity Research)
Okay. Thanks so much for taking questions.
Operator (participant)
Thank you. Ladies and gentlemen, I am sure no further questions in the queue. I would now like to turn the call back to Liz for closing remarks.
Liz Barrett (President and CEO)
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. We feel really good about it. Most importantly, these patients need new options. We are very much looking forward to next week and then following that, the PDUFA. Thanks, and we'll keep you guys informed of any happening. Thanks a lot. We'll talk to you guys soon. Bye-bye.
Operator (participant)
Ladies and gentlemen, that concludes today's conference call. Thank you for your participation. You may now disconnect.