Good morning, ladies and gentlemen. Thank you for standing by. And welcome to UroGen Pharma's Second Quarter 2019 Financial Results and Business Update Conference Call. It is now my pleasure to turn the call over to Kate Bechtold, Senior Director of Investor Relations for UroGen Pharma. Please go ahead..

Thank you, operator. Good morning, everyone, and welcome to UroGen Pharma's second quarter 2019 financial results and business update conference call. On Friday morning, we issued a press release providing an overview of our recent corporate highlights and financial results for the quarter ended June 30, 2019.

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; and Peter Pfreundschuh, Chief Financial Officer.

Joining us for the Q&A portion of the call will be Stephen Mullennix, Chief Operating Officer and Jeff Bova, Senior Vice President of Commercial. Liz will provide a summary of our recent corporate developments and Mark will share clinical development and regulatory updates.

Peter will then provide an overview of our financial highlights for the second quarter of 2019 before we open up the call for questions. As a reminder, during today's call, we will be making certain forward-looking statements.

Various remarks that we make during this call about the company's future expectations, plans and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995.

Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the Risk Factors section of UroGen Pharma's quarterly report on Form 10-Q filed with the SEC on August 9 and other filings that UroGen Pharma makes with the SEC from time to time.

We encourage all investors to read the company's Annual Report on Form 10-K and the company's other SEC filings. These documents are available under the SEC filings section of the Investors page of UroGen's website at investors.urogen.com.

In addition, all information we provide on this conference call represents our views only as of today and should not be relied upon as representing our views as of any subsequent date.

While we may elect to update these forward-looking statements at some point in the future, we undertake no obligation to update any forward-looking statements we may make on this call on account of new information, future events or otherwise. I will now turn the call over to Liz..

Thank you, Kate, and good morning, everyone. We hope you're enjoying the summer and appreciate you taking the time to join our call this morning. Before we get into the heart of the quarterly call, we're thrilled to announce that on Tuesday, September 24, we will host our first Investor Day.

The first half of 2019 has set the stage for what is certain to be a pivotal year for UroGen as we approach key milestones over the next few months.

During the event on September 24, we will provide long-anticipated updates on key events, including top line of the final data set for UGN-101 for the treatment of patients with low-grade upper tract urothelial cancer.

We will also share the initial complete response data for UGN-102, our investigational program for low-grade non-muscle invasive bladder cancer; as well as preclinical data for UGN-201, the investigational formulation of our TLR7 agonist. We will outline our long-term strategy and plans for advancing our portfolio.

In addition to updates from management, we will have a panel of urologists with experience using our medicines. They will provide a real-world perspective on how they see our treatments fitting into their practice. We hope you will be able to join us for this exciting event.

For today's call, I am pleased to share that we have submitted the CMC modules of our rolling submission for the new drug application for UGN-101 to the U.S. Food and Drug Administration.

We've locked the database on our Phase III OLYMPUS study and remain on track to complete the submission and prepare for planned acceptance in the fourth quarter of this year. As many of you will recall, UGN-101 has been granted orphan drug, Fast Track and Breakthrough Therapy Designation by the FDA.

As such, following the anticipated completion of our rolling NDA submission, we expect a six month PDUFA date, which sets us up for potential U.S. approval and launch for UGN-101 by June of 2020. We are incredibly excited about the potential of this program to transform the treatment paradigm for patients with low-grade UTUC.

Low-grade UTUC remains an area of high unmet medical need. If approved, UGN-101 would be the first approved therapy for this patient population, which we believe represents around 6,000 to 7,000 patients.

Today, these patients are treated with surgical intervention which puts them at risk for the well-known complications associated with repetitive endoscopic and potential kidney removal. Based on the literature, approximately 80% of these patients would ultimately undergo kidney removal.

We believe, if approved, UGN-101 can provide patients with a treatment option that avoids the risk and downstream sequela associated with surgery. We are continuing to ramp up our precommercial preparation activities and buildout.

Our commercial strategy will ensure a comprehensive understanding of the high unmet need in these patients, will build awareness of RTGel technology of our company UroGen and our mission to provide meaningful advances to patients with urological and oncology conditions.

The commercial team is building innovative solutions for physicians and payers to ensure rapid and broad adoption at launch. UroGen is poised to be the primary resource for all stakeholders in the area of low-grade UTUC, a disease that has previously been ignored. It is also critical that UGN-101 fit seamlessly into the urology practice.

Our market research indicates that 88% of physicians would like to have a treatment the delays or prevents radical surgery interventions for these patients. We understand the current treatment paradigm and the needs of the physicians, practices and patients.

In addition to our field representatives, we'll have a small team of nurse educators to provide training and support around installation. We have hired most of the sales management team, and they will begin recruitment for a 50-person sales team to efficiently reach our target urology accounts.

Given that 33% of urology practices treat 90% of the UTUC patient population, we are confident that a lean team of 50 will be able to effectively navigate the unique needs of UGN-101 across these practices. In addition, given the complexity and importance of reimbursement, we will hire a small team of field reimbursement specialists.

Our multidisciplinary team will be staffed and trained by year-end so that we will hit the ground running upon potential U.S. approval and launch in the first half of 2020. In addition to our specialized team, we have support programs in distribution, reimbursement and pharmacy.

Our commercial team has extensive experience in uro-oncology that's currently finalizing our launch plan to support rapid adoption. We will be prepared for a successful launch and look forward to bringing UGN-101 to patients in the first half of 2020.

In parallel to our commercial preparation activities, we continue to advance our pipeline programs, including UGN-102 for low-grade non-muscle invasive bladder cancer. Patients with low-grade non-muscle invasive bladder cancer have very limited treatment options as there are currently no drugs approved by the FDA as first-line treatment.

We believe that UGN-102 has the potential to replace the current standard of care, which is repetitive surgical resection via transurethral resection of bladder tumor, or TURBT, for up to 80,000 patients a year.

We look forward to sharing early complete response data on a cohort of patients from the Phase II OPTIMA II trial of UGN-102 on the 24th of September at our Investor Day. Beyond our internal pipeline programs, we continue to evaluate business development opportunities to maximize the potential of our RTGel technology in both oncology and urology.

We currently have an early-stage feasibility agreement with Janssen in a therapeutic area of mutual interest as well as a collaboration with Allergan in overactive bladder. Lastly, I'm excited to share that Dr. Robert Uzzo has joined UroGen as a special adviser and will be working closely with our medical affairs team.

Rob is a well-known and well respected leader in the field of urological oncology and has made important scientific and clinical contributions to the field. Rob brings a unique experience and joins a team of accomplished leaders united in the desire to transform the field of uro-oncology.

I'd now like to turn the call over to Mark, who will discuss our clinical development programs and progress in more detail.

Mark?.

Those with high-grade and those with low-grade disease. And we are again focusing on the low-grade disease. Within the low-grade patient population, we are studying a population referred to as intermediate risk. The definition of intermediate risk includes patients with multifocal disease, large tumors and rapid rates of occurrence.

This is a patient population whereby the current standard of care, transurethral resection of a bladder tumor, or TURBT, is used repeatedly to address chronic recurrence of disease.

These patients experience what can be viewed as a form of surgical failure, and many undergo multiple surgical procedures during their lives to manage bladder cancer recurrences.

We believe UGN-102 can have an immediate impact and provide this patient population of approximately 80,000 with a first-line nonsurgical option for the treatment of chronic relapse. As Liz already mentioned, we look forward to providing a data update on this program at our upcoming Investor Day.

The initial data update will consist of complete response rate with durability information to be reported at a later time. We also continue to advance our UGN-201 program, our TLR7/8 immunomodulatory agent for the treatment of high-grade bladder cancer.

Our Phase Ib study of UGN-201 in carcinoma in situ, or CIS, have suggested a preliminary efficacy signal while preclinical models have demonstrated anti-tumor effects for high-grade disease.

We are exploring the utility of UGN-201 in the context of novel combinatorial immunotherapy for non-muscle invasive bladder cancer and look forward to providing you with updates at our Investor Day. And with that, I would like to turn the call over to Peter who will discuss financials..

Thank you, Mark, and good morning to everyone on today's call. UroGen is well-capitalized to further advance our clinical development programs as well as our commercial planning efforts in preparation for a potential U.S. approval and launch of UGN-101 in 2020.

We closed the second quarter of 2019 with $233.3 million in cash, cash equivalents and marketable securities. For the second quarter and six months ended June 30, 2019, we reported a net loss of $22.5 million or $1.08 per share and $43.9 million or $2.19 per share, respectively.

This compares to a net loss of approximately $18 million or $1.14 per share and $31.4 million or $2.02 per share for the same periods in 2018. The net losses for the second quarter and six months ended June 30, 2019, includes $7.2 million and $14.7 million, respectively, in non-cash stock-based compensation expense.

Research and development expenses for the quarter and six months ended June 30, 2019, were $10 million and $19.7 million, respectively, compared to $8.3 million and $15.9 million for the same periods ended June 30, 2018, and included $2 million and $4.3 million in non-cash stock-based compensation expense, respectively.

The increase from 2018 to 2019 was attributable mainly to costs associated with UGN-101 Phase III OLYMPUS trial and an increase in personnel cost to support our ongoing clinical and regulatory efforts and an increase in share-based compensation expense.

General and administrative expenses for the second quarter and six months ended June 30, 2019, were $13.8 million and $26.5 million, respectively, as compared to $10.2 million and $16.3 million for the same periods in 2018 and includes $5.2 million and $10.3 million in non-cash stock-based compensation expenses, respectively.

The increase from 2018 to 2019 was mainly attributable to an increase in personnel and related cost to support our growth in the business and an increase in commercialization infrastructure and services, an increase in consulting and other outside fees and an increase in share-based compensation expense.

As of June 30, 2019, we had approximately 20.8 million ordinary shares outstanding. As you have seen from both our Q1 and Q2 earnings releases, the company continues to achieve its 2019 financial guidance as set forth in February of this year.

Based upon our anticipated activities and business goals, we still plan to have a net loss of approximately $100 million to $115 million, which translates into a cash burn of approximately $76 million to $88 million for calendar year 2019. We still project our current cash balance to carry us for at least the next two years.

With that, operator, I would like to turn the call over to for questions..

Thank you. [Operator Instructions] And our first question comes from Derek Archila from Stifel. Please go ahead..

Great. Thanks, guys. This is actually Ben on the line for Derek. Just wanted to ask what type of feedbacks you guys are hearing from docs right now. Thanks..

Derek, it's Liz, and then I'll turn it over to Mark and he can also add some commentary. We've been doing obviously a few things with physicians in the market. One, we've been doing our own kind of market research out in the field.

And the good news is the market research was coming back about 88% of physicians are interested in -- highly interested in an alternative to kidney removal, so we think that there's a really high unmet need out there.

As we've mentioned before, if you think about the number of physicians that were in -- have experience with UGN-101 and 102, it's a very small portion, so one of the priorities we have for 2019 was around building awareness.

We had a great AUA, which we talked about at our last call, and our sales are out in the field and we're getting very positive feedback. And reason I wanted Mark to sort of also comment is that he has also had a few calls from physicians with patients looking to be able to use it with patients ahead of the approval.

So we've got -- we are getting, I think, more awareness of the -- of our treatment and also a lot of interest.

So Mark, can you just comment?.

Yes.

And just to underscore what Liz was saying, we are continuing to hear from physicians in the community, as well as our academic partners and KOLs, that there's interest in knowing when the drug will actually be available because they are patients that they are lining up for treatment, and now this is part of their thinking in terms of planning therapy for individuals who have low-grade disease.

So we know that the efforts to socialize on the availability of this and the potential of this therapy is beginning to work within the community. So there is interest and we continue to hear this.

We just had -- I actually had a KOL investor -- excuse me, a scientific advisory meeting in Washington this weekend and continue to hear this from interested physicians. So I would say there is a high level of interest..

Great. Thanks for your info guys..

Thanks, Derek..

Our next question comes from Eric Joseph from JPMorgan. Please go ahead..

Hey. Good morning, everyone. This is Turner on for Eric. I'm just curious how you're thinking for UGN-101. How are you thinking about outside U.S.

commercialization, perhaps, in terms of partnerships? And do you have any EU regulatory feedback? And what steps would need to be performed before filing with the EMA?.

Yes, no, sure, Turner. It's a great question and one that we've been looking at and actually just started to work with some consultants on moving that forward outside the U.S. To be honest with you, we don't think it's such a -- as much of a regulatory hurdle in the U.S. as it is -- I mean, in the EU, as it is a reimbursement hurdle.

So what we really need to do is we need -- we have had initial discussions with the regulators. As you know, Europe is usually a lot more difficult to get an approval on an open-label single-arm study, but they also recognize the high unmet need and have been open to regulatory approval based the current -- based on our current 101 study.

Like I said, the bigger challenge though, if you think about how reimbursement occurs in a lot of the countries in EU is they want comparators, right? So they put it in a basket of compares, that's how they price you.

And right now, without head-to-head data, they're likely to run a price at the generic level which obviously doesn't make sense for us or our partner.

So one of the things that we're doing as a next step is gathering some additional information on what type of study would they need to see, and would likely be a superiority study which we believe we could do, and to be able to get a reasonable reimbursement.

So we really -- to be honest, we have been focused on getting the NDA in the U.S., getting the launch in the U.S. and then thinking about outside the U.S. The other market that I just want to highlight for a moment because we've been doing some intelligence work around is Japan.

As you may or may not know, the Asia population has a higher incidence of UTUC. And in a market like Japan obviously, you can get a fairly reasonable reimbursement, and we would need to do a bridging study.

So what we have right now is someone working on getting some meetings set with the regulatory agency in Japan, where we'll do further work on what it looks like. We have had other parties, to be honest with you, interested in partnering certain geographies.

But what we don't want to do at this point, we think is in our best interest and our shareholders' best interest, to maintain our freedom to operate and not license out in individual markets. So I think what we'll do is continue to move that ball forward. And now that we're getting closer to the U.S.

submission, and we can have some time to be able to address those markets. And then -- but your likelihood is that we would probably look to partner outside of the U.S..

Got it. That was helpful. And then for 101 life cycle management, there's a study up on ClinicalTrials.gov for 101 retreatment. And hoping you could just discuss the trial a little bit and what would be viewed as meaningful complete response rate in this population.

And then just a follow-up to that, do you have any additional plans to conduct further studies for expanded use, perhaps as an adjuvant to surgery, for instance? Thank you..

So I think, look, we're open to looking at life cycle management across 101, so absolutely interested in that.

Mark did mention during his remarks in the beginning about the retreatment study, and that is something that we put in place because we do believe, and actually, we've already heard a lot from physicians that -- of their plan to retreat, right? So as long as they get a good response the first time, and with our high CR rate that we've disclosed, with the high CR rate, physicians are interested in retreatment.

And which is why I said Mark can talk to you more about the study specifics, but it's really an extension of our current study. So as these patients recur, which the good news is, is they're not recurring at a high level, we do expect the physicians to be interested in and wanting to retreat.

And we think it's an important piece of data for them to have as well as hopefully included in our label for -- and for reimbursement. So we're moving forward with that. I don't know, Mark, if you want to add anything else to that..

The only thing that I think I would add is that we have some confidence that retreatment would make scientific and medical sense because there is a predicate practice in urology using mitomycin repetitively in responders who have non-invasive bladder cancer.

So the notion and the scientific basis for doing this in the kidney with 101 makes sense to us and makes sense to our colleagues in practice. And I -- as Liz points out, I think we will see physicians retreating patients and we obviously are interested in seeing the data as our experience matures in that area as well..

Great. Thanks so much..

Thank you.

Next questions?.

And our next question comes from Paul Choi from Goldman Sachs. Please go ahead..

Hi. Good morning, everyone. And thanks for taking our questions. Maybe one for Mark, just with regard to the OLYMPIC -- OLYMPUS update, excuse me, that you'll have at your Investor Day.

Can you maybe just confirm for us that all the patients that didn't -- weren't included in six month follow-up at the AUA data will be included at the six month -- will have follow-up there? And then second, for the patients you did have at AUA, can you just maybe confirm for us that -- comment on what the median follow-up will be for that population?.

So the answer to the first question is we're going to tell you that everybody we've got in our locked database at our Investor Day. So we'll we're going to give you what we think is the bottom line story on this trial.

So I hope that's sufficiently informative to give you confidence that you're going to have a lot of information about this trial at that presentation. And then sorry, I'm not sure I understood the second question..

For the patients who were included in the six month follow-up at AUA, I was just wondering, can you may be confirm for us what the median follow-up roughly will be for those patients?.

The median follow-up for those who were not presented? We're going to....

For those who were included in the AUA presentation..

Yes. I can't give that to you right now. But we will certainly present -- we will present a full picture of our six month follow-up data, which will be more than what's presented at the AUA..

So Paul, do I - it's Liz.

Are you asking for the follow-up beyond six months in what will be shared?.

That's correct, right. Not all patients were included in the six month follow-up at AUA. So I was just curious, for the ones that were included, what the median follow-up would be for them..

Right. So just a couple of points on that. One, as Mark stated, we locked the 101 database, but that does not mean that we will 100% of patients in follow-up. As we've communicated earlier, our discussion with the FDA is that they wanted to see at least 75% of the patients. We will not have CR in 100% of patients, and we won't for our submission.

We've been very transparent about that all along in conversations with the FDA because we didn't want -- that would have put us into 2020 with the submission. So we will -- as Mark said, we've locked the database. This will -- the data you'll see on the 24th will be the full data set that we will share with the FDA.

Now what we are -- have not disclosed and likely will not is patients, all of the data beyond that mostly because of publication and we want to make sure we maintain the integrity of the data so that we can -- because what then our next step will be, and we've already begun to work with the investigators, is to ensure that we have a publication prior to launch.

So some of that data will be maintained confidentiality until we get our publication..

Got it. And then on 102, we're looking forward to seeing the initial cohort you described at your Investor Day as well.

But just in terms of progressing with next steps, do you feel that the data you have in hand is enough to inform sort of next steps either with the regulators? Or do you want to see data first from the additional patients?.

No. I think that one of the reasons that we picked September 24 was that we knew that we would have enough of data for 102 that we would have a really, really great signal as to whether it works or doesn't work in this patient population.

The team, together in thinking about the timing of taking an interim look, what we were really close to having about 50% of the patients, so it's important by the time we get to the September 24 day, we'll actual have the initial CR data on almost half of the patient population.

And so because we have -- we will have the data on half of the patient population, depending on the results, if they're positive, our plan is to immediately move to a meeting with the FDA to understand exactly what they would need and want to see for a pivotal study. And we want to move forward and accelerate that as quickly as possible.

And so the plan at this point is as soon as we have the data and look at the data, review it, is to move forward and not to wait. To -- because we think that with half the patients, we think that would be a good projection for what we would see in the final -- with the final data.

This piece we won't have when we talk to the agency, which will be important, but it will give us some level of confidence with the data that we do have, is the durability, right, and the duration of response.

But once we see the current database, I think we'll -- we believe that we'll have enough data to be able to have a meaningful conversation with the agency on what they would want to see because we want to accelerate that and start that study as quickly as possible..

Okay. Thanks for that. Just then turning to your recently announced collaboration with Janssen. Can you maybe just frame for us what's involved with regard to the clinical development? And sort of help us book-end what sort of bio bucks might ultimately be involved with the deal? Thanks for taking our questions..

So look, we've been very clear that we have a limited amount of data that we can actually share. And what we've said is it's a formulation feasibility study.

So the important thing about our proprietary RTGel is that when you -- every time that we develop the gel with an agent, whether it's the mitomycin that we have today or with other novel agents, taking BOTOX for it as an example, we actually to have to customize the formulation.

And so while we believe that there is not an issue to be able to put any novel therapy in our gel, that's something that we obviously have to figure out. So what we're doing with our announcement is we're making sure that we can take the innovative medicine that we have with Janssen and move forward with a formulation.

So that's the work that's ongoing right now, is a formulation feasibility.

Once that data, assuming that it's positive and we're able to develop a new therapy with -- in the -- with our RTGel and their novel medicine, then we would have discussions around how we take that forward and what the clinical development plan looks like and what a partnership may or may not, a collaboration may or may not look like.

So we have not gotten to that point. We've been very clear that what we have right now is a feasibility study. So I hope that's helpful..

It is. Thank you very much for taking our questions..

Thank you..

And our next question comes from Boris Peaker from Cowen. Please go ahead..

Great. Thanks for taking my questions. My first one is on just the low-grade UTUC.

Can you comment what fraction of patients could be resected versus the ones that can't be?.

So I'll let Mark talk about the patients..

So -- and Boris, let me just make sure I'm going to answer your question. Are you talking within the trial or are you talking sort of general within the population for....

Generally within the population..

Yes. So if the trial's any reflection of the general population, which we believe it is, then we think based on what our surgeons have told us in the trial, it's somewhere around 50% of patients are amenable to an endoscopic resection and 50% actually are not.

50% of the participants in this trial -- or just about 50% had tumors that were characterized by the investigators as technically unreachable by laser fiber, so therefore would not have been amenable to endoscopic resection..

And just so if we divide the world into those two groups, resectable and nonresectable, do you see one particular one of them kind of lower-hanging fruit for the drug when you launch it? Or is the need or an adoption, you anticipate be similar in both of those?.

I think the important data that we shared at AUA around the nonresectable and the resectable was that regardless of whether it's respectable or nonresectable, the data was very similar, and that was an important finding for us.

So we don't see, frankly, a difference between the resectable and nonresectable population as far as physician's desire to use the medicine. And so we'll expect that we'll be able to use it and gain adoption in both of those patient populations.

I think the other thing to mention, as Mark said, you have to keep in mind that around 80% of patients, 78% of patients will eventually get a kidney removal. So -- and physicians know this, they're aware of this.

The patients that do get endoscopic resection in the beginning tend to come back again and again for repetitive endoscopic resection before they finally move to a kidney removal.

And so with this in mind, we don't see why physicians wouldn't what to use this in all of their patient populations because they, again, know that eventually, these patients usually end up in kidney removal. So we see that as being an opportunity for all of the patient population..

Great. And my last question is on the sequential development side.

If we think about the bladder strategy there, can you comment just around the Phase III specifically? Would you have to run it against UTUC? And would you need to look at superiority versus will non-inferiority be adequate as an endpoint? And just kind of ballpark, how long would a study like this take?.

Yes. So we're -- actually, the team has been working on developing the -- what a Phase III might look like. Because frankly at this point it's a simulation. It's like if the agency accepts this, it's -- we believe that we would take a noninferiority.

The question is would you want to do with a non-inferiority or superiority study? And it really depends on what the agency has to say. Think one of the things that we've been focused on with 102 is -- and moving forward with potential Phase III, assuming the data's positive, is really gathering.

And one of the things that Mark and his team are working on right now is gathering data around this intermediate-risk patient population. I don't believe that there's a true appreciation by the FDA and the regulatory bodies at this point about this particular patient population.

As Mark has mentioned, we've talked many times, this is a patient -- again, a patient population that ends up with these repetitive surgeries. And they're just not -- their needs are not met by today's standard of care.

So this is a patient population that we want to have more of a dialogue with the FDA, to want to share the data, some data that we're currently generating around that patient population and the high unmet need in that patient population.

And we believe that the agency has -- really understands that, then I think that they will be more flexible, at least we hope that they will be more flexible around what a Phase III pivotal study might look like. But that would be one piece of data that we would want to generate. And so I think we're prepared.

And we've got, like I said, different scenarios of whether we have to do a noninferiority or superiority study.

We feel very confident that if the data, as -- we have to wait to see, but if the data is similar to what we see in our 101 and for the UTUC, then I think the important thing for us is to really sit down with the FDA and have a discussion around with that pivotal study would look like.

So we don't have timing because clearly as you know, it depends on the number of patients that would be in that study, and that really depends on what the FDA wants to see. I think one thing I will tell you is we are ahead of our enrollment in the 102 study, and which is the reason that we will be able to share 50% of the patients.

I think when we initially started talking, we had -- we were hoping we would have 25% to 30% of the patients to be able to share in our first publication of the initial data. And instead, we're going to have 50% of patient population.

So I think that shows you that there's an interest -- a high interest among investigators that believe that will be able to hopefully accelerating, quickly enroll any pivotal study that we need to do for 102..

Great. Thank you for taking my questions..

Thank you..

Our next question comes from Ram Selvaraju from H.C. Wainwright. Please go ahead..

Thanks so much for taking my questions. So I wanted to drill down on whether you see any evidence that the 12-month follow-up data specific to the low-grade UTUC indication would be necessary or potentially awaited by physicians in order to have them fully change their treatment practices in this indication..

Yes. I'll just make a comment and turn it over to Mark, and he can add any additional color. The reason that we are filing with the six month data is this is the average -- current average time to recur. So patients, average time that recur today is six months, so we believe that, that's the most meaningful.

And that's the conversation we've had with the agency. Of course, I think everybody would be interested in the 12-month data, but we don't think that, that data is necessary for adoption, although I'm sure physicians would be interested in. And Mark, I don't know if you want to add anything..

I agree. That's the answer..

Okay. Fantastic.

I also wanted to ask about whether you could just give us a brief overview of the billing process that you envisage for UGN-101? And the potential relevance of the faster J-code assignations that we've been seeing on multiple other fronts from CMS?.

Yes. I'm really thrilled that we have Jeff Bova on the line, and he's our head of commercial. And the team has been working diligently on the issue of reimbursement because we do believe that will be a key success factor for us. So I'm just going to turn it over and let Jeff answer your question.

Jeff?.

The professional fee, which is the fee that the physician actually gets; the technical fee, which will be the fee that the office of the surgery center would get; and then because we are a Part B, as in boy, drug, we will have a buy-and-bill model, where they will be reimbursed on the drug as well.

With regards to the J-code application, we will go through the traditional application timelines, depending on -- and if approved, that will sort of outline when we will have our permanent code. But as most know, before we get a permanent J-code, we will submit for pass-through.

Pass-through comes off, depending on the timing of the approval, anywhere between a month to three months following. That pass-through code will help in the surgery center as well as the hospital to get -- to not -- to no longer have a miscellaneous code.

So we'll go from a miscellaneous code to a pass-through code for a period of time until we get our permanent J-code..

I think, just to add one more comment that you made, as some -- as you know, the CMS right now is considering moving to a quarterly review instead of an annual review for permanent J-codes.

And we're hopeful and believe that by the time we get approved, we're hoping that they have then moved to this quarterly review, and that will obviously expedite our ability to get a permanent J-code.

We don't know yet that and we don't have yet that built in, so Jeff and the team is ensuring that regardless of whether that happens or doesn't happen, that we have everything in place that we need to ensure reimbursement for these physicians because. So that -- because that will obviously be something that will be important to doctors..

Okay. Great. And then I wanted to ask about the UGN-102 sort of long-term treatment regimen. As I understand it, in the OPTIMA study, you're looking at an initial six week regimen where the drug is instilled weekly and then monthly installations after that on a long-term basis.

And just wanted to better understand how you're thinking about this in terms of the context in the real-world setting and real-world practice, potentially in the situation where the drug is approved, whether you think that there is likely to be a need or the long-term sort of monthly recurring installations; if those can be dispensed with after a set period of time; and if so, what that period is likely to be..

Thanks for the question. And this is Mark. And I'll -- let me just clarify one thing. There actually is no maintenance arm in the 102 program. It is an induction arm or an induction protocol once a week for six weeks, and that's the treatment protocol for this group of patients. And then they enter follow-up. So there actually is no maintenance.

That was removed from the protocol. And our expectation is that, that would be the core application in the non-muscle invasive bladder cancer space for this technology. The reason is because maintenance with mitomycin in fact is used very haphazardly in the community and it is entirely clear that it is of therapeutic benefit.

And most physicians would in fact use an induction course as the initial foray, where they're using mitomycin in water as an adjuvant following surgery. So we have no intention at this time of using a maintenance protocol.

And that might be something people would explore later on, but not for this trial or for this initial foray into using the 102 product..

I think one of the things that we've really been focused on is doing what we believe is in the best interest of the patients. And we think that it's burdensome to have monthly, and to Mark's point, we don't really know if there's a benefit or not. So we really have to do a study with and without.

And I think that given the results that we saw in 101 and our expectation around 102 is that patients really don't need the maintenance, and that is though why -- also why we believe that doing a retreatment study, and we shifted away from maintenance and added a retreatment study.

And I think in whatever protocol we move forward with in 102, we would want to do the same as well. So hopefully, that's helpful..

Okay. Just two very quick clarifications on that point, then. If we look at the possibility of applying this solely in a retreatment context, and I think, Liz, you and I had, had discussion about this before.

The possibility might be that you don't necessarily need six installations, that you could potentially get the optimal result with a couple fewer installations. So I was wondering if you could maybe comment on that.

And also, referring to what you were saying earlier regarding the potential design of a pivotal program and the possibility that you would have to do some kind of comparator controlled work.

If we assume what might be the design of a pivotal study involving a comparator control, what might be some options for that comparator control in this study?.

Yes. I think that a comparator control would likely be against TURBT, as Mark stated, because that's the current standard of care. I'll give you my sort of point of view on the six installations and then let Mark comment. At this point, there is no data, right? There's no data that suggest that doing fewer than the six will work.

We do know that some patients actually ended up with a PR.

And one of the questions that we have, one of the things we may want to look into the future is, if those patients had, had a PR after six weeks, would those patients benefit from actually incremental installations? And might a longer treatment help those patients from a -- that were at PR to get to a CR? So I think that you will end up with some patients that have more and some patients that have less.

But our sort of perspective right now is that we really need to do the six installations, that's the data that we have.

Doesn't mean that we won't, in the future, look to do other studies because at the end of the day, we want to make sure that we're doing what's in the best interest of the patients and the physicians, and we believe that, that will ensure rapid and more broad adoption.

But at this point, there's no data that suggests that below six treatments is actually going to be effective. Mark, I don't know if you want to ask -- add....

Yes. I just want to make sure that I respond to one thing that Ram may have brought up here. I think we are mixing products and trials when we're talking about this. So the retreatment programs is for the upper tract.

We don't have any retreatment information about the use of 102 in non-muscle invasive bladder cancer, so that would be a subsequent program. I just want to make sure that, that was clear..

Yes, no, no. That's very clear.

Just one very quick last item for me, which is do you have any indication at this juncture, based on prior experience with mitomycin C, that more installations or longer duration of therapy would be necessary in the retreatment context? Or is that just purely speculative? We just -- as you say, there's very little data here, we can't really hazard a guess on that front..

Well, what we know, and I think that we have talked about this before, and really the fundamental basis for why we believe 101 and 102 are going to be valuable clinically is the because the technology addresses a core requirement for the efficacy or the optimal efficacy of mitomycin, which has really been known since the 1990s, which is duration and high concentration of medication.

So because we're able to deliver to the tumor high concentrations of medication over an extended period of time, we believe that this is going to provide a much better performance profile for the active pharmaceutical agent in this context. So I think in some ways, we're already addressing the question you were asking.

And what you're asking is a very interesting question which we believe the trials are answering, which is, if you provide a higher concentration of drug for a longer period of time, do you see better results? And we are encouraged, based on our results to date that, that in fact is correct..

I think the other thing that's really important is think about it in the context of chemotherapy in patients who -- in other tumors outside of urological area, and if you respond to chemotherapy and you're chemo -- considered to be chemo-sensitive, which is very analogous to this patient population, then the expectation is, and these patients typically get retreated with the same treatment if the treatment worked the first time.

So we actually expect, and so I think to Mark's point, there's a really good rationale for why we believe that retreatment will work. I think the other thing just in the -- to go back to the number of the installations. This -- and Mark, I actually would probably use your prior comment more on this because you were around before I was.

But this is fairly typical for a urology office. And they're used to doing not just this procedure, but other procedures in their office, that the six installations is an area in which they're used to and this is typically what they do. So we don't expect that we will be able -- we don't expect that, that's actually going to be an issue for us.

We think that the protocol, that the data we, and we believe that they will do that. I don't know, Mark, if you want to add anything..

No, I think it's a very important part. One of the constructs that urologists use in thinking about intravesical and intra-catheter-y therapy is this notion of weekly installations for six weeks. And we wanted to make sure that we were doing something in designing this therapy that is comfortable and familiar to urologists.

So that also informs frankly the treatment paradigm, familiarity, the use of the drug in prior settings in this way. And it's -- and we have seen this in our clinical trials that when you're talk to urologists about this type of a treatment paradigm, they are readily accepting of it because it's very familiar..

Great. Thank you very much..

And our next question comes from Chris Howerton from Jefferies. Please go ahead..

Great. Well, I'll try to make this quick. I guess for 102, I think, Liz and Mark, you said a couple of times, if the data are positive. Maybe you could just help us frame what would be positive.

What's a number that would get you excited or at least start the conversation for the next steps?.

Yes.

Mark?.

I think that given the fact that as -- I think Liz has said on a number of occasions, and we've talked before, Chris, before about this. We've chosen people for this trial who are effectively surgical failures.

People who have recurrences, people who have larger tumors, multifocal disease, sort of classic intermediate disease patients are very familiar to urologists in practice and who represent a real challenge because our standards of care just don't work.

And these are elderly individuals for whom repetitive surgery becomes more and more of a challenge as they age and they will have their comorbidities that interfere with safe operations, so to speak.

So for us, I think if we were able to achieve a CR rate after the initial induction course of therapy of 50%, and if at 12 months, 50% of those people were free of disease, that would represent, I think, a significant clinical step forward for this population.

Because remember, these are people who are likely to recur often more than once within the course of 12 months. So that should give you a ballpark for what we are thinking internally and would represent success and a reason for an informed conversation with the regulatory agencies..

Excellent. Okay, yes, no, that's clear. And then I assume we'll discuss this on September 24, but I guess I'd ask now. For 201, I think you're talking about potential combinations.

Are there particular mechanisms that you think are attractive in this case for this high-grade non-muscle invasive bladder cancer?.

Yes. I think that what you'll see on September 24 is some of the preclinical data that we have, and it's in combination with checkpoint inhibitors, which is where we really think that we'll get the most -- patients will get the most benefit.

So I think moving forward, our plan is to do 201, a TLR agonist, in combination with checkpoint inhibitors going forward. So I think that we're continuing to do more preclinical work around there and ensure that we're optimizing the best strategy.

But I think you'll see some of the data that we've seen that makes us really bullish on the opportunity to move 201 forward..

Thank you very much for taking the questions. And obviously look forward to the Analyst Day in just about a month..

Great. Thank you, Chris..

Our next question comes from Matt Kaplan from Ladenburg Thalmann. Please go ahead..

Thank you. Good morning. Wanted to just touch base on the 101 NDA filing, I guess, given that you've submitted the CMC modules.

I guess, what's the rate-limiting step for completing that NDA later this year?.

Yes, the rate-limiting step, as we've talked about all along is six month durability data on 75-plus percent of the patients. So if that was -- it's really, we -- like Mark said and I said earlier in my comments, we've locked the database to ensure that we have at least 75% of the patient population six months.

So it was just making sure that we had 75% patients at six months. So right now, we're just going through your typical cleaning of the database with the clinical data and capturing all of the data and ensuring the quality of the data. Because what's most important to us is that we ensure robust submission.

We've been very happy so far with the response from the FDA. We know that when we put the CMC module in, as we've said, that we've already gotten acknowledgment from that. We know they're reviewing it because we've received a couple of questions from them around our CMC. So we don't actually see any big barriers right now.

It's really simply a matter of getting the clinical data together and submitting that. And we expect to do that in the Q4 time period..

Okay. Great. That's helpful. And then I guess given the relative near term for potential approval of 101 for UTUC. Just wanted to talk a little bit about kind of your commercial preparations that are ongoing.

And kind of you alluded to this a little bit before, but what are you thinking of pricing right now? And can you detail kind of the support systems that you'll have in place to facilitate the reimbursement process for our physicians and patients alike?.

Sure, no -- thanks, Matt. And as I've said before, we have Jeff on the phone. But before I put him on the line, but before I turn it over to Jeff, I'll just reiterate the fact that we're not providing pricing information at this point. We actually haven't even finalized our price.

We're still gathering data, doing pharmaeconomic study around this patient population and current standard of care as well as looking at what we believe the best price will be. What we have said in the past and will continue to say is that "oncology-like" pricing. The team, Jeff and the team have been very busy moving things forward.

And I'll turn it over to him and he can talk to you about specifically what they're doing around reimbursement and other preparations for the commercial launch. But I just want to say that we're really thrilled to have Jeff, and he has built a great team so far of very experienced colleagues in the area of uro-oncology.

So Jeff?.

Sure. Thanks, Liz. So we're working on prior to launch, we have a couple campaigns that we've been focused on. One is primarily for the patients. Prior to UGN-101 coming along, there really wasn't a resource for both the patients and the physician with regards to their disease.

And so we have a website, UTUC.com, that with the help of our advisers, provides a lot of clarity in and around the patient's disease, their options at the current time. And we've had a positive response for that, because as I've said before, before utuc.com, if you did go to try to research the disease, it was very challenging and difficult.

So we've got that for a recourse for both the patients and the physicians. The other is the unmet need campaign. And we're calling it intact-ivism. And the intact-ivism is obviously the importance of keeping your kidney, so keeping your kidney intact. So there's education that's in popular urology journals right now.

This will be something that the rest, once they're brought onboard, will also highlight when they're in the field, just the unmet medical need around keeping your kidney and keeping it obviously intact. The last thing obviously that we're doing is gearing up for launch.

So as Liz alluded to earlier, we are close to hiring that first line sales management team. Once they're onboard, they will begin to hire the representatives. The representatives will have a couple months to discuss the unmet medical need campaign as well as begin to profile the accounts in anticipation of approval..

Thanks for the added detail. Then I guess just shifting gears a little bit, just I guess more for Liz. Kind of plans to explore the use the RTGel technology outside of urology.

Are there indications, are there potential uses of that?.

Yes. No. I think there are a lot of users. We've talked -- one of the things we are really working on right now is a longer term strategy for UroGen Pharma.

I think it's safe to say that we -- our focus is really on uro-oncology, right? In the urologic -- urology space and the oncology space, we would want to own any RTGel or partner and be in a collaboration. I think outside of uro-oncology is something we would look to outlicense to other partners interested.

We have gotten a lot of enthusiasm from physicians that when we sit down and talk to them, they will give you a laundry list of all the potential uses for the gel.

And we have a head of BD that works for -- reports into Peter, and they're obviously fielding any requests that we have, and for this -- and we've been talking about does it make sense for us to be more active in this space? And again, I think right now, we want to make sure that we are all in, in the uro-oncology space.

That's our focus, that's our priority. The team in Israel, so the early clinical team, had been working on some other areas, and we've really focused them on making sure that they're really focused on uro-oncology opportunities. And again, outside of that, we would look to outlicense..

Thanks a lot. Congrats for the progress..

Great. Thank you..

I am showing no further questions at this time. I will now turn the call back over to UroGen's President and CEO, Liz Barrett, for closing remarks..

Thank you, operator. I just would like to say that we think we've made important progress so far this year. It's been a very, very busy year for us in executing some very critical events, i.e., around advancing our portfolio as well as ensuring a robust submission to the FDA.

So we're excited about our meeting on September 24, where we'll be able to share more detailed data around all of these opportunities. And we hope that you'll be able to join us. So appreciate you joining the call today, your continued interest in UroGen, your active questions. And we're always here to answer anything that you might have.

So just want to say thank you to everybody, and we'll talk you soon. Hopefully see you in September. Thanks. You can disconnect now, operator..

Thank you. Ladies and gentlemen, this concludes today's conference. Thank you for participating, and you may now disconnect..