Good morning, ladies and gentlemen. Thank you for standing by, and welcome to the UroGen Pharma Third Quarter 2017 Financial Results Conference Call. [Operator Instructions]. It is now my pleasure to turn the call over to Steve Klass, Vice President with the company's Investor Relations firm, Burns McClellan. Please go ahead.

Thanks, [ Carla ]. Good morning, everyone, and welcome to the UroGen Pharma Third Quarter 2017 Financial Results and Business Update Conference Call. Earlier this morning, the company issued a press release providing an overview of its recent corporate highlights and financial results for the quarter ended September 30, 2017. This press release can be accessed on the Investors portion of the company's website at investors.urogen.com. Leading the call today will be Ron Bentsur, Chief Executive Officer of UroGen, who will provide an overview of the company's recent clinical and business progress. Following his remarks, Gary Titus, Chief Financial Officer of UroGen, will provide an overview of the company's financial highlights for the third quarter of 2017 before we turn the call back over to the operator for questions. For Q&A, we're also joined by Dr. Mark Schoenberg, the company's Medical Director. 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 the 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 the final prospectus for UroGen Pharma's initial public offering of securities in the United States filed with the SEC on May 5, 2017, and other filings that UroGen Pharma makes from -- with SEC from time to time. We encourage all investors to read the company's final prospectus 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 like 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 Ron.

Thank you, Steve. Good morning, everyone, and thank you for joining us on our conference call today. This is a very exciting time for UroGen, and we're pleased to be holding this call this morning to discuss our recent progress as well as what to expect as we enter 2018. Following our IPO in May of this year, we have continued to work diligently towards our vision of becoming a leader in the field of urology with a focus on uro-oncology. We believe that our broad clinical pipeline, which is based primarily on our proprietary RTGel sustained-release formulation technology, holds great promise to shift the current treatment paradigms across a number of uro-oncological conditions, including non-muscle invasive upper tract urothelial and bladder cancers. We believe that our RTGel-based drug candidates possess chemoablation properties, meaning that they can destroy tumors on their own without the need for tumor resection surgeries. Therefore, our drug candidates have the potential to transform the existing treatment paradigms and make drug therapy a first-line treatment option in these cancers and, therefore, to provide new therapeutic options for the thousands of patients suffering from these conditions. Importantly, we believe that an effective chemoablation agent may potentially provide better eradication of tumors irrespective of the detectability and location of the tumors as opposed to surgery. Because with surgery, one can only resect the visible tumors, and we know that there's a field effect associated with these cancers. Leading the way in our clinical pipeline is MitoGel, our novel sustained-release RTGel-based formulation of mitomycin C, a well-known chemotherapy currently being evaluated in the ongoing pivotal Phase 3 OLYMPUS trial in patients with non-muscle invasive low-grade upper tract urothelial carcinoma or UTUC. As a reminder, this Phase 3 study is a single-arm, open-label study, and commenced several months ago. UTUC…

Thanks, Ron. We ended the third quarter with $79.2 million in cash and cash equivalents. This includes revenues for the third quarter of 2017 totaling approximately $7.8 million, $7.5 million of which, as Ron alluded to before, was received as a milestone payment from our partner, Allergan. For the third quarter ended September 30, 2017, we reported a net loss of approximately $300,000 or a loss of $0.02 per share. This compares to a net loss of approximately $8 million or a loss of $3.73 per share for the third quarter of 2016. Research and development expenses for the quarter ended September 30, 2017, were approximately $5.6 million compared to $2.9 million for the quarter ended September 30, 2016, and includes $1.5 million in noncash, share-based compensation expenses. The increase from 2016 to 2017 reflects an increase in personnel costs, an increase in share-based compensation and an increase in direct costs associated with the MitoGel Phase 3 clinical trial. General and administrative expenses were $2.2 million for the quarter ended September 30, 2017, as compared to $3.2 million in the third quarter of 2016 and includes approximately $700,000 in noncash share-based compensation expenses. The decrease from 2016 to 2017 is primarily due to recording expenses related to our IPO in the statement of operations in 2016 which is partially offset by an increase in share-based compensation as well as an increase in personnel costs. At September 30, 2017, we had approximately 13.2 million ordinary shares outstanding. With the proceeds from our IPO completed in May of this year, together with the Allergan revenues received this quarter, we believe that we are in a strong financial position to advance our pipeline programs and execute on our business objectives. Based on our current plan and forecasted expenses, we expect the existing cash and cash equivalents to get us through the key anticipated value-creation milestones, including the MitoGel top line Phase 3 data, NDA filing and beyond. This concludes my comments on the third quarter of 2017 financial results. I'd like to now turn the call back over to Ron.

Thank you, Gary. I want to thank everyone for taking the time to join us on the call today. We look forward to updating you during the months ahead as we continue to execute on our near-term objectives and long-term strategy. We plan to be at the Jefferies London Healthcare Conference later this week and hope to see many of you there. Thank you all for your support and interest in UroGen. I would now like to turn the call over to the operator, so we can go ahead with the Q&A session. Thank you.

[Operator Instructions] And our first question comes from Derek Archila with Oppenheimer.

I have 2. So just first off, I think in the prepared remarks, you noted that 2 patients in the MitoGel Compassionate Use study had durability out to 18 months. So I was just curious whether there were any kind of defining characteristics in those 2 patients relative to the other patients?

I will ask Mark to field this question, please.

Thanks for that question. The characteristics of these patients as Ron alluded to in his introductory remarks were that they are patients who had widespread disease more likely and, therefore, scheduled to undergo surgical intervention with kidney removal prior to their enrollment in the study. What we know about mitomycin is that it works on the majority of patients with urothelial cancer as long as the dose and duration of exposure is adequate, which is one of the principal advantages we think of MitoGel in this clinical setting. However, we do know, like all therapies, that it doesn't work on [indiscernible]. So I think that what we could say is that the patients who've achieved CR behave as we might expect. However, we don't expect absolutely every patient to respond with the CR, and that's the characteristic of the performance of this drug.

Got you, and I just -- and out of those 2 patients that saw that like kind of 18-month durability, are they maybe like a younger set in this -- among those patients? I'm just trying to understand whether as you'll have looked and kind of dig into the data at some point, if there is going to be some improvements in patients that are maybe kind of on the younger side or less severe disease. So I'm just kind of curious if there's any other color there.

Sure. I think at this point, it would be premature to give you that. I understand the impulse to look for clues about risk stratification. I think it would be premature, in our experience, to tell you that we know that. I don't think age is going to play a role here. It's probably a characteristic of the disease perhaps in the molecular level that we don't appreciate currently, but it's obviously something we're interested in exploring.

Okay, fair enough. And then just second question, on the hiring of Paul to do some business development, is the focus mainly on working to gain additional agreements, licensing partners for RTGel in new indications? Or is it looking to potentially bring in new assets into the fold? And what type of assets would you guys be looking at?

So the hiring of Paul, and by the way just by way of background, Paul was the project lead at Allergan when we were negotiating the BotuGel deal with Allergan. So I think it is interesting that a few months afterwards, he calls us up and wants to join UroGen. And lo and behold, a few months after that, he's now our VP of Business Development. So I think that's intriguing in its own right. But the idea of bringing Paul into the company is to explore possibilities just like we did with Allergan for our applicability beyond uro-oncology. That's on one hand. On the other hand, it's to try to explore deals outside of the U.S. and also to try to be potentially opportunistic on the in-licensing front. So there's a lot on the plate here, a lot of possibilities. And we felt that we needed someone with experience, someone who understands the landscape in the playing field to help us achieve that.

Our next question comes from Matthew Andrews with Jefferies.

So Mark, could you put the MitoGel data in context for us? What would you expect in terms of response for, to the extent these data are available, water-based mitomycin in upper tract -- low-grade upper tract urinary carcinoma compared to what we've seen with your CUP data? What sort of the bar there in terms of what response would be for water-based mitomycin?

Yes, it's a great question. So the first thing to remember and to remind the audience of is that water-based mitomycin when used in the upper urinary tract has effectively no dwell time in the upper urinary tract, and that has to do with the fact that the kidney constantly produces urine which washes the drug out when it's in a water-based formulation. And humans walk around upright, and so gravity obviously pulls the drug downstream into the bladder. One of the things that we do know about mitomycin is that contact, exposure time and concentration are the keys to facilitating the efficacy of this medication. And that's the basis on which we believe that MitoGel will be a superior performer in this context. When you look at mitomycin use in the upper urinary tract, the -- I think the take-home message is that it's used sporadically for the reasons I've just alluded to. Physicians certainly have tried to inject it and instill it, but it is laborious. It requires significant instrumentation. And because of the adverse physiology and environment as well as the aqueous solution suspension, it's felt to be [ deminimously ] efficacious in that setting. So I think what we would expect is that there might be a little bit of benefit. But remember, you're talking about instilling something that's going to run out in a couple of minutes compared to a drug in the MitoGel formulation that will be present in the upper urinary tract for 6 to 8 hours. So I think it would be hard to give you a number to compare, in large part because the data largely do not exist. But while it is used, if used relatively rarely and one would not expect to see a huge benefit over surgical monotherapy.

Second question on MitoGel, can you just talk about the end point that will be used for the Phase 3 study comparing PDE versus the typical RECIST criteria? And how do you [ write ] it, what that PDE number is for the Phase 3? What is a true complete response for a PDE end point?

Sure. So the way that the trial is constructed, and as Ron previously mentioned, it is an open-label, single-arm. So patients will undergo 6 weekly installations of MitoGel and then 4 to 6 weeks after the last installation will undergo a very standard-of-care evaluation of the upper urinary tract, which will utilize 2 metrics of success. One is direct visualization of the interior of the kidney where the MitoGel was instilled, and in addition, a urine specimen will be obtained from the kidney and sent for what is called a cytology analysis. Cytology is a microscopic evaluation of cells extracted from urine, and it's a standard metric we're looking for evidence of urothelial carcinoma. Both of those evaluations must be normal, must be completely without evidence of pathology in order for a patient to be declared a CR, a complete response, at the primary disease evaluation end point. Our conversations with the agency regarding what success would look like in a larger trial, we were encouraged not to tie ourselves to a specific statistical end point but to bring the agency what they articulated as a clinically meaningful result. And without elaborating further mathematically, we believe, based on our experience in the Compassionate Use program, that we are certainly poised to provide the agency with a clinically meaningful result in our pivotal trial.

And then just, Ron, one on VesiGel, can -- or Mark, can you talk about the strategy -- go-forward strategy there in terms of what your Phase 2 program could look like? Have you finalized it with FDA in terms of the population you'll study in this next Phase 2?

So we're in discussions with the FDA now regarding the next step for VesiGel, and there are several ways of looking at it. And these are not mutually exclusive. We could choose to pursue more than one approach. So one is the more traditional approach. Let's go after the broader patient population of low-grade, non-muscle invasive bladder cancer. Ultimately, that may require some sort of a controlled study versus TURBT or something along those lines, but that is something that we are prepared to do. Certainly, given the Phase 2a study results that we saw, we feel very confident that VesiGel could deliver the goods in a study like that. Another possibility is to try to find what we call subpopulations within that very large patient pool of over 300,000 patients. So for example -- and we may have spoken to you about this before, but for example, patients who are what we call high-risk patients, these are patients who are not recommended for the TURBT procedure because it is not considered safe to put these patients under general anesthesia. So these are patients, for example, that have what we call a very high ASA scores or they have cardiac conditions or on anticoagulant. They have things of that nature. So that's the sub-population that we're looking at very carefully. That dialog with the FDA is ongoing, but the net-net is that the goal for the company is to start a study with VesiGel in the second -- in the first half of 2018.

[Operator Instructions] Our next question comes from Reni Benjamin with Raymond James.

Maybe just a couple of follow-up questions, can you talk a little bit about the patients who did progress in the Compassionate Use program? Have they gone on for subsequent nephrectomies? Or are there other therapies that they're currently being administered, just to get a sense as to what are the options for the indication? And then also, can we talk a little bit about any differences that they might have just between the Compassionate Use study and the ongoing OLYMPUS study?

I'll ask Mark to take this question, please.

So let me comment first on the patients in the compassionate program who did not have a complete response. So there were obviously several patients. And remember, this is a relatively small experience, but there were several patients who had partial responses. And with respect to the options that are available to those patients after MitoGel therapy, they have available to them the standard therapeutic options available to all patients, namely kidney and ureter removal, which is currently the standard of care surgically for the management of patients who cannot be managed endoscopically. We had a very interesting experience with one patient, however, and it's worth mentioning because this gives us a clue that will be useful to urologists in clinical practice once MitoGel hopefully is available for use. One patient achieved a partial response and then received several additional doses of MitoGel in what would effectively be called a maintenance format and went on to achieve a complete response. And while in our lines and certainly within the context of pivotal trial, that patient would be considered a partial but not a complete response to therapy, in clinical practice, it would be very useful for urologists to be able to multiply dose patients and to combine primary ablative therapy with MitoGel with subsequent surgical therapy, if necessary, effectively down-staging or debulking the disease and making a patient who was not endoscopically manageable amenable to endoscopic resection. So while this is a relatively small experience, obviously [ total ], we believe it is emblematic of how MitoGel will fit really nicely into a variety of different clinical scenarios that will be useful to clinicians going forward.

And then, Mark, maybe just some thoughts regarding any differences between the Compassionate Use study and OLYMPUS whether it's patient selection or even dose?

Yes. So important to understand that, as Ron said, the Compassionate Program was a group of patients with very extensive disease and it was an experience, not a clinical trial. In our pivotal clinical trial, we have rigorously controlled the enrollment criteria in order to make sure that we can very accurately assess the response to MitoGel. So the burden of disease is very specifically characterized and measured prior to therapy. The follow-up of these patients is very specifically codified. So rather than being an experience with a fair amount of physician discretion, which is how I would characterize the Compassionate Use program, the clinical trial has all the bells and whistles of a real clinical trial. And in addition to induction therapy in the clinical trial, I should mention that patients who achieve a complete response will also be eligible for maintenance therapy. And maintenance therapy here would be additional dosing on a monthly basis through the duration of the first year of care. And that is an important addition to the program because that's probably what urologists would end up doing to maximize the benefit of this approach and increase the longevity of the complete response rate in this population. And the other thing to remember about this group of patients, just to be very clear, is we actually believe that patients who are enrolled in the clinical trials by virtue of disease burden and other characteristics are actually at lower risk. So this is a more favorable population and likely a more favorable population for a complete response compared to the Compassionate Program, which is really end-stage disease, very -- essentially no clinical options left and really defaulting to surgery. So we believe that we have constructed an experiment that will be very informative with regard to the efficacy of mitomycin and obviously is positioned to succeed.

Got it. And then maybe just as a follow-up, I know Matt asked this, I guess I'll just ask it another way. In terms of what you deem to be a positive study, obviously the FDA has mentioned clinically meaningful, what do you consider, let's say, a low bound of what clinically meaningful is? Obviously, we know how the Compassionate Use study is, what sort of results that's generated. We typically think when we go into Phase 3 studies that the results will vary with one way or the other. How are you guys thinking about what the lower bound could be? And then from a market perspective, Ron mentioned the patients in the prevalence population, if we're thinking about the top line data readout in the second quarter of 2018, potentially a launch in the second half of 2019, how should we be thinking about your commercial strategy, what a sales force looks like and how that works?

Mark, if you can talk about what we believe the lower bound is from a clinical significance perspective, and then I'll touch on the second question.

Sure. The -- in our conversations with the agency and internally, we believe that saving at least 1 in 5 kidneys is a meritorious outcome, and I think that most urologists would agree with that. Now candidly, we believe we're going to do -- be able to do better than that, so we think the numbers will be higher. But anything greater than about a 20%, 25% CR rate in this population would be a victory, and based on our conversations with the agency, we believe that we are aligned with them on that number as the lower bound that I think you're alluding to.

And Ren, to your second question regarding the commercial strategy, so this is an orphan indication. As the numbers would suggest, prevalence of about 15,000, incidents of about 2,500, maybe 3,000 per year, something like that. So it is something that a small focused sales force could certainly do, and that is something that we're planting the seeds for with the hiring of the VP of Commercial. It is that individual's responsibility to start building out that team slowly but surely. Ultimately, I think what it's going to require is a sales force of probably 40, maybe 50 salespeople, probably not more than that. Keep in mind that these patients do tend to get referred to hospitals and to bigger urology centers when UTUC is diagnosed. So that plays to our advantage in terms of being able to cover the hospitals and the clinical sites from a territory perspective, from a geographical perspective and in terms of being able to leverage a small sales force. So I think everything is -- the stars are really aligned for us as a small company to be able to execute this launch properly, and that's what we're focused on doing.

I am showing no further questions at this time. Thank you, everyone, for joining the call today, and you may now disconnect.