UroGen at TD Cowen’s Summit: Navigating Approval Challenges

On Wednesday, 28 May 2025, UroGen Pharma (NASDAQ:URGN) participated in TD Cowen’s 6th Annual Oncology Innovation Summit, focusing on the strategic path forward for its UGN-102 treatment. The conference call, led by CEO Liz Barrett and CMO Mark Schoenberg, highlighted both optimism and challenges in the approval process, following a surprising outcome from a recent ODAC meeting.

Key Takeaways

• UroGen is seeking FDA approval for UGN-102 by the PDUFA date of June 13.
• The ODAC meeting unexpectedly emphasized the need for randomized clinical trials (RCTs).
• UroGen reported an 80% complete response rate in its single-arm study.
• The company is open to conducting RCTs but faces challenges in agreeing on endpoints with the FDA.
• UroGen aims to avoid pre-approval RCTs, stressing UGN-102’s benefits over surgery.

ODAC Meeting & Outcome

• The ODAC meeting focused on UGN-102 for intermediate-risk non-muscle invasive bladder cancer (NMIBC), especially in recurrent cases.
• The advisory committee’s vote was influenced by a discussion about the general need for RCTs, rather than UGN-102’s specific benefits.
• The vote was split; urologists and a patient advocate supported UGN-102, while medical oncologists favored RCTs.
• UroGen expressed disappointment, emphasizing their study’s compelling efficacy and safety data.

Possible Scenarios & Path Forward

• UroGen’s primary goal is full approval with post-marketing commitments by June 13.
• Options include conducting future RCTs with UGN-102 or its successor, UGN-103.
• Conditional approval, contingent on further clinical trials, is a possibility.
• UroGen prefers post-marketing studies focused on safety and disease history.
• A delay in the PDUFA date is possible if more time is needed for post-marketing study agreements.

Potential for Limited Label

• The impact of a restricted label depends on its limitations.
• A label limited to specific comorbidities would significantly restrict UGN-102’s potential.
• If the label targets patients unwilling or unable to undergo TURBT, the impact would be less severe.

Urologist Feedback

• Urologists expressed disappointment with the ODAC’s decision.
• The urology community supports UGN-102, citing compelling data and alignment with practice needs.

Readers are encouraged to refer to the full transcript for a detailed understanding of the discussions.

Full transcript - TD Cowen’s 6th Annual Oncology Innovation Summit:

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Morning, everyone. I’m Tara Bancroft. I’m one of the senior biotech analysts at TD Cowen. I wanna thank you very much for joining our sixth annual oncology innovation summit. And so to start off the day, in the first session, we have a q and a with UroGen, and it’s my pleasure to introduce Liz Barrett, the president and CEO, and Mark Schoenberg, the CMO.

So it’s a privilege to have you both here. Thank you. Thank you for being here with us.

Liz Barrett, President and CEO, UroGen: Well, thank you for having us.

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Yes. Of course. And so before I get started, I do wanna remind the anyone who’s listening that you can email me questions at any time at terra.Bancroft@tdsecurities.com if you have a question, and I’ll make sure it it gets answered. But I think a really good place to start, Liz, and and then Mark, I I wanna hear both of your thoughts, and and we’ll get into details. But maybe at at a high level, could you give us your your thoughts, your reaction to the ODAC meeting that was that was hosted last week and how the vote went.

Liz Barrett, President and CEO, UroGen: Great question. And, actually, I’m gonna flip it and let Mark answer first, and then I’ll come back and add anything. So, Mark?

Mark Schoenberg, CMO, UroGen: Yeah. Thanks. Good morning, Teri. Thanks for having us. The format of the meeting for those who didn’t have a chance to listen in or read the transcript, was the following.

We presented a dataset that had actually been agreed upon prospectively with the FDA regarding our total programmatic experience with a drug called UGN-one hundred two for the treatment of grade intermediate risk non muscle based bladder cancer. And the agency had specifically asked us to present efficacy data in the subset of patients we’d studied across the program with recurrent disease, which was the highest unmet medical need. And we had talked to the agency about this. In addition to our presentation on that, we had a total programmatic presentation on safety. And prior to our presentation, the agency presented a background talk on non muscle invasive bladder cancer.

We presented, then the agency presented their perspective on our data. There were two questions that the advisory committee was involved. There was one question and one discussion question. So one voting question and one discussion question, the, the advisory was asked to consider. The specific voting question had to do with the benefit risk of our drug in the context of treating non muscle invasive bladder cancer.

The discussion question was quite different than what we had originally anticipated, and it was specifically to address, in a more or less academic manner, the value and need for randomized clinical trials in the future in the setting of treating non muscle invasive bladder cancer, which is an area of interest for a lot of people in our field, including the FDA, but was, frankly, quite disconnected from the matter at hand in considering UGN 102. So after those data presentations, there were relatively few questions about our, our program. We broke, came back for clarifying questions, And then a discussion was conducted, which largely focused on the merits of randomized clinical trials. There was relatively little discussion of our program or the data. And just to be very specific, the the advisory committee was composed predominantly of medical oncologists, a biostatistician, two guest urologists as temporary voting members, an industry representative, not a voting member, and a patient advocate.

And interestingly, in the midst of the conversation where, the clinical trials question was discussed, doctor Pastor interjected just to direct the overall proceedings to remind everyone that the, discussion question was not relevant to, the consideration of Eugene one hundred two or our application. There was then a vote, and it was very clear based on what came out of the vote, I think for those who are listening, that the discussion question had materially influenced many of the members of the advisory committee, particularly the medical oncologists who are obviously very interested in the question of clinical trials in general. The urologists both voted to support the approval as did the patient advocate and one of the medical oncologists. But when asked and Liz will, I’m sure, wanna comment on this as When asked why people voted no, when asked to consider the benefit risk question of UGN one zero two, almost to a person, the, the voters, reference the fact that, randomized clinical trials were necessary even though the the single arm trial that had been presented was prospectively agreed upon with the agency as supportive of an approval. So let me stop there because I’ve said a lot, but I think that was my sort of composite impression of what happened.

And just one final note, in the discussion when FDA was asked what a randomized trial would look like in the context of trust studying non muscle nasal bladder cancer, the FDA couldn’t answer the question directly, but said it would be a matter to discuss with the sponsor at the time such a trial would be designed. And I know Liz is going to probably want to comment on this as well, let me stop talking now.

Liz Barrett, President and CEO, UroGen: Thanks. And I do think that’s sort of the key question here. You know, I think that there was a little bit of misunderstanding of actually what we did because we actually did offer a randomized control study. So they talked about the feasibility of a randomized control study. That was never the issue.

The issue was agreeing on an endpoint in a randomized control study. So we offered up a placebo controlled study, we offered up a non inferiority study, but the agency wouldn’t agree to that and would only agree to a superiority study. And, oh, by the way, in the superiority study, they didn’t want the three month if there was a if there was a recurrence at three months in the TRBT arm, they didn’t want that to be an event. So it it felt like we were you know, the whole trial was being stacked up for failure. And in in case, do you continue with a study that you you know, that the FDA has already told you they don’t agree with the endpoint, or do you try to find another path?

And that is when we got agreement with them that a single arm study could be the basis for an approval. I don’t think that came out in the ODAC, that we actually were willing to do a randomized controlled study, but not a superiority randomized controlled study, because no sponsor had ever been required to demonstrate superiority to a surgical procedure. And so, you know, the complexities of actually showing medicine therapy versus a surgical medicine, I think everybody understands sort of the challenges with doing so. But look, at the end of the day, I think we we have a path forward. We have to work closely with the FDA to see where we go from here.

But we were reassured when Doctor. Pazar did say this is a split vote, and let’s talk. And so that’s kind of where we are right now. You know, we’ve reached out to the agency about having that discussion. We plan to continue, you know, every path that we can see, because our goal is to get an approval for UGN one zero two on, you know, June 13, which is our PDUFA date.

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Okay. Yeah. Thanks for those thoughts, both of you. So I guess before we get into more details on Pazjo’s comments, path forward, I think one of the things that is so confusing about this is really why you think they maybe why they wanted to do this unrelated discussion question because it it it very clearly did confound the committee. And so what what do you think the goal was for having that discussion if it doesn’t pertain to the one zero two review?

Liz Barrett, President and CEO, UroGen: Yeah. I mean I mean, personally, I would prefer not to speculate given given where we are in the conversations with them. To Mark’s point earlier, I do think that the vote was an unintended consequence of that discussion. I don’t think that the FDA purposely wanted to confound the question, but having that discussion right before you take a vote on a single arm study probably, you know, know, wasn’t the best, you know, setup, at least not for us. You know, at the end of the day, I think we know that the FDA’s role is to challenge, to ensure that medicines that are efficacious and safe are delivered to patients.

We feel like we did that, frankly, with a very high bar. I mean, I’ve been in oncology for thirty years, and never have I seen response rate and an 80% durability. And so to to not take into consideration the compelling nature of our data, I think, was short sighted. And so, you know, again, our goal we have one goal, and that goal is to get UGN approved. If we have to do post marketing to talk about safety or to show, you know, continued durability, if that’s the question, we we’ve agreed.

We already agreed, and we already planned a five year follow for Envision. So that will continue to give more data on both durability and safety if there’s still questions. But, you know, I think, you know, again, we probably shouldn’t speculate on what the FDA was thinking at the time. You know, we want to work in partnership with them. We want to find a path forward and and are looking forward to having those discussions.

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Okay. Yeah. And, you know, I think I I wanna get into a little bit more detail on Pazjir and his role and stuff. But can since you just brought up the the five year ENVISION study or the ENVISION follow-up, and that is a possible scenario. I think what would be really helpful is to kind of go through what you envision as the possible scenarios that could come from this.

Like, what are the next steps? What do you think the likelihood is of each?

Liz Barrett, President and CEO, UroGen: Yeah. Mark, do you wanna comment about that? And then

Mark Schoenberg, CMO, UroGen: Yeah. I mean, I you know, I’ll give you my thoughts, and, Liz will reflect on this as well. So, obviously, you know, we are hopeful that we’ll get full approval with some kind of reasonable post market commitment. For example, acknowledging the fact that we’ve already structured Envision to provide very long term safety and efficacy data supporting our contention that this is a reasonable alternative to surgery in this population. We can conceive of other iterations of this that would include possibly the commitment to some form of a randomized trial, either with the current agent or with the successor agent that we’ve talked about, probably one of three.

And then, you know, I suppose that there probably is a possibility because the FDA has the latitude to do this theoretically to provide a conditional approval analogous to accelerated approval contingent upon some additional clinical trial work. But I’ll stop there. Liz may want to comment on that as well.

Liz Barrett, President and CEO, UroGen: No, I think Mark’s right. But the there’s so many different options to your point. It’s hard to sort of peg without having more discussions with the FDA on where they see this going. I think for us, having to do a randomized control study before an approval is not really an option for us. So we really need to find a path forward that allows us to have an approval.

Again, we think that if there’s going to be a focus on post marketing, it should be on safety, it should be understanding the natural history of the disease, But at the same time, we’ll be open minded to whatever, you know, comes forward as long as we can get an approval, you know, prior to that study having having to be done.

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Mhmm. Yeah. So for the RCT, let’s I mean, we can spitball some some potential different options. But, I mean, it seemed like from the discussion, you know, the history that you guys have had with your engagements and what they were discussing at the ODAC, What do you think I mean, if they require an RCT post marketing, do you think it will be a superiority study, noninferiority? And because I think one thing that was very clear is that that’s really difficult to agree upon even even if you get a conditional approval.

And so I’m curious to think what what do you think that would look like?

Liz Barrett, President and CEO, UroGen: Yeah. The only only one that I can think of would be an adjuvant study, which would be very unfortunate. The reason that would be very unfortunate is because one of the greatest benefits to our drug is you got an eighty percent complete response without having surgery. And we all know, you know, the complications that can occur with surgery, even though they may be a small percentage. Do you want to be the one person that has your bladder perforated, right?

Do you want to be the one person that goes back to the hospital? So it’s very unfortunate if that’s the case, but I don’t see any other, to your point, what doesn’t go away if we do a randomized control study is what is the endpoint? You know, what do you have to demonstrate, and how do you do it versus surgery? When you have a surgical procedure where you get a CR at day zero, and then you have a therapy where you get their complete response at three months. So so it you know, how do you reconcile that, from a statistical standpoint?

And I think that’s the biggest challenge. So the challenge of the endpoint doesn’t go away if we have to do a randomized control study, and it’s unfortunate for patients, frankly. Does it matter to us whether it’s an adjuvant or not? Not so much. But boy, it matters to patients, and you heard the patients at you know, you heard the patients there at the ODAC, and that’s typical.

That’s not like those patients are unique. That is typical of what we hear from patients who have gone through both. And in the patient reported outcomes, and in the work that the UNC did, that came out loud and clear. When ninety percent of patients say they preferred, and they’ve all had a TRBT, they would prefer the treatment with UGM one zero two over having another TRBT.

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Mhmm.

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Yeah. No. I I completely agree with that. So, Mark, one of the things that you said in that response that I just kinda wanna go back to, you said correct me if I heard this wrong, that it might be possible to use UGN one zero three as a as a phase three that that could be used post marketing. Did I did I hear that wrong?

Mark Schoenberg, CMO, UroGen: So, again, I’m speculating because, of course, as Liz has pointed out, you know, really, this is contingent upon conversations that we have not yet had with FDA. But as, Liz has said on many times, you know, publicly, we have a successor molecule, the UGN-one hundred two, which is a, another formulation of mitomycin using proprietary methodology that we will be advancing as the successor to UGN-one hundred two and which is currently in clinical trials now on the basis of conversations and implementation agreed upon with FDA. So it is, I suppose, theoretically conceivable that we would arrive at an agreement whereby, given the fact that 103 is already in advanced stages of clinical trials, that it might make more sense to focus on the development of one zero three in the context of a randomized trial, were we to agree to do one and what you know, again, I don’t have the details for what that would look like. But, again, let’s really wanna chime in here.

Liz Barrett, President and CEO, UroGen: No. Agree. I mean, I think until we have those conversations, until we know where we are, you know, it’s it’s it it is speculation. Right? I I I think, you know, the we hope to have a very productive conversation with them.

We hope to have an exchange with them very shortly and look forward to that. And again, with our goal being, you know, we agreed with the FDA that the single arm study could be the basis for an approval if it met certain criteria, and we felt like we met and exceeded that criteria. And so that’s kind of where we are at this point.

Mark Schoenberg, CMO, UroGen: I think our

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Hi. Sorry about that. I, we’ve been having some problems with Zoom at at TD, but okay. Yes. No.

That that definitely makes sense. And I understand you guys you haven’t met with them. It’s really hard to speculate ahead of, ahead of actually having that meeting. So I think one of the things that kind of begs the question is, like, can you meet with them in the in the next two, three you know, before June 13, basically? And so, you know, I’m just trying to get an idea of, like, what you would think if you can have these discussions, plan an RCT ahead of the PDUFA date, and if not, you know, maybe they’ll issue a PDUFA delay and, you know, what could that possibly look like?

Liz Barrett, President and CEO, UroGen: Yeah. I mean, there’s lots of different scenarios like we talked about before. I think, again, I think we will be able to engage with them. It’s hard to say whether it be a meeting, whether it will be an exchange. I think it really depends on what their position is.

We absolutely have time. You know, we, as you’ve noted before, in your in your write ups, we had already been talking to them about the label. So we’re pretty far along from that standpoint. The missing piece is sort of the final labeling as well as what does a post marketing, you know, marketing commitment look like. And so I think we could get that done if if they’re willing to engage with us.

And again, engagement can be a lot of different ways. So so that’s our goal. Our goal right now is to have those, that dialogue, whether the dialogue is a Zoom call or whether it’s in person or whether it’s just an email exchange. It really depends on, you know, kind of what their viewpoint is. And so I think we will be, again, working wanting to work with them very productively and ensuring that we can get an approval.

If there has to be a delay, I’d I’d I’d you know, I don’t foresee that, but if there has to be a delay so that we can come up with something, you don’t have to have the post marketing trial done. You just have to have a framework agreement. So it doesn’t have to be all of the details. It has to be, again, a framework agreement. So I think from that standpoint, we have the time, if they’re willing to engage with us to get that done, and, you know, that would be our goal.

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Okay. Yeah, that definitely makes sense. Okay. So I know, we are we have about five minutes left. So I think one thing that people are playing and replaying and going back over a lot is Pastor’s comments.

So there are a couple components of this that it would be really helpful to get your thoughts on. One is how much influence does he have in this decision, and what did you make of his comments during the ODAC?

Liz Barrett, President and CEO, UroGen: Yeah. I mean, look, and I’ll ask Mark to comment as well. I again, we would be speculating. I I can tell you that, I think that the FDA, I think the health care system, I think patients in general are very fortunate to have someone like doctor Pastor. He is someone who has been very involved in all of the cancer treatments.

I mentioned earlier, I’ve been in cancer for thirty years. He’s been very involved in ensuring that the right medicines get to patients at the right time. I’ve been in situations where drugs have been pulled off and then brought back on because patients needed them. And so personally am very happy that he’s leading the oncology division. I don’t know him personally, but obviously we’ll have and will interact with him.

So again, I don’t want to speculate on Doctor. Pastor. I think it was you know, we we were reassured by his comments that, one, it was a split vote, but, two, trying to to lead the group to understand that it was it was not a, that we were not talking about this application and then and then following up. So so, you know, we we, you know, believe, obviously, he has influence. What what his willingness is to engage, you know, is yet to be determined, and and, again, have a ton of respect for him and believe that that, you know, ultimately, he’ll do the right thing, and that’s our hope.

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Okay. So then I guess another thing that we’re all trying to wrap our heads around, and, again, it is a speculation question. So the possibility for a potentially limited label. So if if you’re if the label is restricted to patients that are either TURBT ineligible, patients with comorbidities, what would that look like? And does that change your outlook for other the initial launch or the peak market at all?

Liz Barrett, President and CEO, UroGen: Yeah, I think it depends, right, on what that label is. I find it to be difficult to have to do that. The reason I find it difficult to do that is you have to look at our clinical study and what data came out of the clinical study. The clinical study wasn’t it was pointed at a patient population where the majority had had one or two TRBTs. So to try to limit it to those who had more, so try to limit it wouldn’t really be reflective of the clinical data.

Now, could they? Sure, they could. And I think it depends. I mean, if it had unwilling and unable to go through a TURBT, patients then can make that decision. Physicians can make that decision.

And if it were relegated to those that have two or more. As we know, we had sixty eight percent of the patient population have had two or more TRBTs. Then of course, you’re talking about sixty eight percent of the patient population. But having said that, we always expected that our drug will initially get used in those patient populations. So it doesn’t really change our outlook, only if they were very limited to patients that had a specific comorbidity.

Comorbidity. That would be, I think, the only way. It kind of would limit the potential of the drug to reach more enough patients and the patients that actually need it. That would be the only way it would be very limited.

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Yeah, no, definitely makes sense. And since it is my fault that I had technical difficulties, I want to ask one more question. Since I know you mentioned before that you have the medical liaisons and your field team out in the field already. And so I’m curious, as they have been at work over the last week, what are they hearing from any feedback from urologist colleagues on what they think about what happened and their support or not support for UGN-one hundred two’s approval and or I’m

Liz Barrett, President and CEO, UroGen: going to ask Mark to comment, because he personally engages with a lot of urologists. As you guys know, he’s still a practicing urologist, but even though knows that and I think he has a lot of insight into what the community is thinking. So Mark?

Mark Schoenberg, CMO, UroGen: Sure. I think the best single word capsule summary of this is disappointment. I think as reflected by the comments on the urologists who were serving on the AdCom, who were supportive of this, if you work in this field and you understand the nuances of this disease, Eugene and one of the team makes complete sense and the data are incredibly compelling. So for the urology community, the response of the AdCom is really not aligned either with practice or patient needs. And I think that was the sense I got from many notes sent to me immediately following the AdCom expressing astonishment at the outcome.

Liz Barrett, President and CEO, UroGen: Yeah. I would use shocked as well as disappointed, too, because I think that it was very unfortunate. I do think the urologist, to your point earlier, that were on the panel, that they understood. I don’t think that the others did. And so it’s unfortunate for our company, of course, but it’s really unfortunate for patients.

Tara Bancroft, Senior Biotech Analyst, TD Cowen: Yeah. I agree. Well, you know, we all wish you luck in in your engagements going forward, and we will obviously all be on the lookout on the PDUFA. And, hopefully, we will talk to you soon. And thank you for joining us, and thank you everyone for listening.

Liz Barrett, President and CEO, UroGen: Thank you. Thanks, Tara. Take care.

Mark Schoenberg, CMO, UroGen: Bye bye.

Liz Barrett, President and CEO, UroGen: Bye bye.

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