Urogen Pharma Ltd (URGN) 2018 Q3 法說會逐字稿

Good morning, ladies and gentlemen. Thank you for standing by, and welcome to UroGen Pharma's Third Quarter 2018 Financial Results and Business Update Conference Call.

It is now my pleasure to turn the call over to Kate Bechtold, Director of Corporate Communications and Investor Relations for UroGen. Please go ahead.

Thank you, operator. Good morning, everyone, and welcome to UroGen Pharma's Third Quarter 2018 Financial Results and Business Update Conference Call. Earlier this morning, we issued a press release providing an overview of our recent corporate highlights and financial results for the quarter ended September 30, 2018. The press release can be accessed on the Investors portion of our website at investors.urogen.com.

Joining me on the call today are: Ron Bentsur, Chief Executive Officer; Dr. Mark Schoenberg, Chief Medical Officer; and Peter Pfreundschuh, Chief Financial Officer. Joining us for the Q&A portion of this call will be Stephen Mullennix, Chief Operating Officer. Ron 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 third quarter of 2018 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 annual report on Form 20-F filed with the SEC on March 15, 2018, 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 20-F 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 Ron.

Thank you, Kate. Good morning, everyone, and thank you for joining us on our conference call. We look forward to providing you with an update on the continued clinical and operational progress that we have made during the third quarter, but I have no doubt that the update on the UGN-101 OLYMPUS trial is of most interest to those on the call.

As noted in our quarterly press release this morning, following our Breakthrough Therapy Designation, known as BTD, in October, our team met with the FDA to reach an agreement on the required information for our NDA submission, so we could ensure that it would be accepted, reviewed in a timely manner and would have the greatest potential for approval. We are very pleased with the outcome from this meeting. As you'd likely recall, the OLYMPUS trial is an open-label, single-arm study. At this pre-NDA meeting, we presented the data that we have to date to the FDA, following which the decision was made that we would no longer need to recruit additional patients and that the complete response rate seen in approximately 65 patients would be sufficient to support submission.

As a result, patient enrollment for this study is now complete. We are pleased by what we believe is the FDA's support for UGN-101. Recently manifested by the granting of the Breakthrough Therapy Designation as well as the outcome from the recent pre-NDA meeting, we believe that there is recognition of a need for potentially organ-sparing, noninvasive therapy in low-grade upper tract urothelial cancer. Our current plan is to present the top line data in January of next year and the full data at a medical meeting in the second quarter of 2019. Durability updates will be provided in both presentations.

Importantly, we remain on track to initiate the UGN-101 rolling NDA submission this quarter and complete the submission in the second quarter of 2019. We continue to target potential approval in 2019 and are building out our infrastructure to support a launch next year, subject to such approval. We look forward to sharing additional details of our commercial strategy in the coming months.

Beyond our clinical programs and commercial planning, we continue to keep a close eye on future growth opportunities. In September, we welcomed Dr. Jones Woody Bryan to our team as Senior Vice President of Business Development. Woody brings over 25 years of industry experience to UroGen. Prior to joining the company, Woody served as Senior Vice President of Business Development at the Sucampo Pharmaceuticals, Inc., where he worked closely with our Chief Financial Officer, Peter Pfreundschuh, and spearheaded the business development effort for its acquisition by Mallinckrodt Pharmaceuticals for $1.2 billion earlier this year. His primary focus at UroGen will be on the integration of corporate strategy and business development to assess partnerships, both inbound and outbound, and bolster our product portfolio.

This is an incredibly exciting time at UroGen as we sit on the cusp of an NDA submission to the FDA for our first therapy. We remain intensely focused on our corporate milestones and look forward to providing updates on our progress.

I will now turn the call over to Mark. Please go ahead.

Thanks, Ron. Given the encouraging interim analysis from the OLYMPUS trial that was presented at the AUA earlier this year, the granting of Breakthrough Therapy Designation and the positive outcome of our recent meeting with the FDA, we are excited about the potential of UGN-101 to change the treatment paradigm for low-grade UTUC.

When we reported our interim analysis in May, 20 of 34 patients, or 59%, had achieved a complete response with 5 of 34 patients, or 15%, achieving a partial response. At the time of the announcement, of the 13 and 7 patients who had reached 3- and 6-month follow-up, respectively, all remained disease-free. UGN-101 appeared to be safe and well-tolerated with most adverse events reported as mild or moderate and transient. It's important to note that more than 1/3 of the patients in this analysis presented with unresectable tumors, which means that the tumors could not be reached using conventional endoscopic technology. In a real-life setting, these patients would be candidates for kidney removal.

As Ron alluded to earlier, an important component of our NDA submission will be durability. In addition to the high complete response rate observed in the interim analysis, we were very pleased with the durability. It has now been 6 months since we presented the interim analysis at the AUA, and we continue to be encouraged by the recurrence-free survival rate observed in the trial. We are cautiously optimistic that based on the peer-reviewed literature, the durability observed following treatment with UGN-101 could potentially exceed the durability associated with primary and surgical intervention.

If approved, UGN-101 would be the first primary nonsurgical therapy for low-grade UTUC and the first drug ever approved for this indication. Current treatment for UTUC requires surgical intervention, which puts patients at risk for the well-known complications associated with repetitive surgical procedures and the potential for kidney removal. UGN-101 would provide patients living with UTUC a much-needed nonsurgical means to treat tumors, which would be especially meaningful for patients with rapidly and chronically recurrent cancer and for those whose disease cannot be visualized or resected using contemporary endoscopic technology.

As a urologist, I am particularly excited about UGN-101, given the potential impact it may have on contemporary clinical practice. If approved, UGN-101 would provide physicians in general urological practice with a simple-to-use kidney-sparing approach to the management of low-grade UTUC. In addition, it would aid urologists in avoiding the risks associated with repetitive endoscopic surgery as well as those associated with kidney removal, such as bleeding, infection, injury to adjacent organs and the risks of anesthesia.

In addition to UGN-101, UGN-102 represents a substantial opportunity for advancing our RTGel technology platform. We have leveraged the significant experience from our 101 program and are pleased to announce that we are enrolling patients in our OPTIMA II open-label, single-arm, multicenter Phase IIb clinical trial of UGN-102 for the treatment of patients with recurrent, low-grade, non-muscle-invasive bladder cancer. The multicenter trial was designed to assess the efficacy and safety of UGN-102 as a potential first-line chemoablation agent in the treatment of approximately 60 patients with low-grade, non-muscle-invasive bladder cancer at risk for recurrence.

The current standard of care for low-grade NMIBC is transurethral resection of bladder tumor, or TURBT, followed by adjuvant chemotherapy. In 2012, the annual incidence of urethral bladder cancer was 80,000 in the United States with a prevalence of 700,000. NMIBC accounts for approximately 80% of all new cases of bladder cancer diagnosed in the United States each year with most patients experiencing life-long, repetitive surgical treatment for cancer recurrence.

UGN-102 has the potential to initially address the unmet medical need in up to approximately 85,000 patients for whom TURBT is no longer an effective approach for the control of cancer recurrence. Like UTUC, there are no currently approved drugs by the FDA as first-line treatment for NMIBC and only 3 drugs have been approved by the FDA, all as adjuvant to treatments following TURBT. We look forward to presenting initial data from the OPTIMA II trial in the first half of 2019.

And with that, I would like to turn the call over to Peter.

Thank you, Mark, and good morning to everyone on today's call. UroGen is well capitalized to further advance our clinical development programs and support our commercial planning efforts in preparation for potential U.S. approval of UGN-101 in 2019.

We closed the third quarter of 2018 with $109.5 million in cash and cash equivalents on the books. For the third quarter of 2018, we recorded a net loss of $20.5 million or $1.28 per share. This compares to a net loss of approximately $300,000 or a loss of $0.02 per share for the third quarter of 2017. This net loss includes a $9.5 million charge in noncash-based stock compensation expense during the third quarter of 2018. Additionally, in the third quarter of 2017, the company had a $7.5 million milestone revenue from Allergan as compared to Q3 2018, where we did not have a milestone revenues from Allergan.

Research and development expenses for the quarter-end September 30, 2018, were $9.6 million as compared to $5.6 million for the quarter ended September 30, 2017, and included $3.8 million in noncash share-based compensation expense. The increase from 2017 to 2018 was attributable mainly to the increase of $2.3 million of share-based compensation expense and an increase of $1.7 million in headcount and related costs to support increased clinical trial activities.

General and administrative expenses were $10.7 million for the third quarter of 2018 as compared to $2.2 million for the same period in 2017 and included $5.7 million in noncash share-based compensation expense. The increase in general and administrative expenses resulted primarily from an increase in share-based compensation expense of $5.1 million and an increase in headcount and related costs to support our growing business and an increase of commercial services and an increase in consulting and director fees.

As of September 30, 2018, we had approximately 16.1 million ordinary shares outstanding. Based on the anticipated activities, we are preparing for potential approval and launch of UGN-101. In addition, we plan to advance ongoing clinical programs and other ongoing and new research initiatives. Based on this, we project our current cash balance to carry us into 2020. Finally, I'd like to note that the SEC is closed in observance for Veterans Day today. Our Form 6-K will be filed prior to the open of market tomorrow.

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

(Operator Instructions) We have a question from Chris Howerton from Jefferies.

Just a real quick one in terms of the timeline for the OLYMPUS data and kind of the interaction with the FDA. So I think the original timeline was that the top line results were going to be presented by the end of this year. Obviously, you stated this morning that you're now expecting to do that in January. And I suspect that had something to do with the pre-NDA meeting and kind of the breakthrough designation. So just how did that all work in terms of the timing? And as a kind of corollary to that, I think the original enrollment was expected to be 74 and now you're working on 65 patients. So is that an increased confidence based upon the CR rate that you've historically seen and the data to date?

Chris, it's Peter Pfreundschuh. I'm going to play quarterback for today's Q&A session of the call, largely because the team is located in a number of different locations across the globe for various conferences and meetings this week. So I'll first tee up the question to Ron and then that will be followed up by Mark. Ron?

Thank you for the question. So regarding the timing for the top line release, we just decided to push it out to January because we felt that in January, we'll get more attention, certainly more eyes and ears as opposed to a timeline that was converging on the second half of December. So we felt that it's just better to push it out to January. Obviously, there is the JPMorgan conference, that's one possibility, and some other possibilities as well. And it's just a matter of getting just more people to focus on it. That's the only reason for that.

With respect to the enrollment and the discussions with the FDA, so as we said on the call, the FDA, when we had the meeting with the FDA recently, this pre-NDA meeting, the number that the FDA saw was -- for enrolled patients was 65. But obviously, that was a few weeks ago and that's outdated now. And right now, we're hovering at around 70. So we'll probably end the study with around 70, maybe even a little bit more. So right at the number that we had initially targeted, which was 74. Keep in mind that whenever you have a patient who signs an informed consent, you really can't turn that patient back and that patient needs to go through screening. And if the patient passed the screening, they need to be enrolled. So at the time of the discussions with the FDA, we had a few patients in the hopper. And fortunately, some of those patients made it in and they will be included.

But going back to what you said in the body of your question, certainly the fact that the FDA had agreed to allow us to stop recruitment based on a number that they had seen, which was around 65, I think, is a confidence booster in and of itself because they felt it was the data that we had available. Certainly, it looked very convincing and unequivocal. So we're very, very pleased by that. And the rest are just timing issues and all -- these things that happen at the tail end of really any study. Mark, I don't know if you want to add anything to that?

Yes, Chris, it's a great question. And I think the only thing I would emphasize, which Ron has really alluded to already, is that the early completion of the trial, which is what the agency signaled to us in our conversation, is very validating and, I think, reflects the fact that the data available are very strong and very clinically convincing.

Sure. Yes, okay. Appreciate that. If you don't mind, maybe just one quick one on 102. So I know obviously the OPTIMA II trial is going on. And there's been some discussions on whether or not that could potentially serve as a pivotal trial. And I'm just wondering if you're able to provide any updates on registrational path for 102 and particularly concerning that trial.

So Chris, I'll direct that question to Mark.

Chris, thanks. So as you pointed out, the 102 trial is up and running. And I think as we have previously discussed, this trial, which is a trial in patients who effectively represent surgical failures, people with rapidly recurring and frequently recurring noninvasive, low-grade bladder cancer are a special niche population that are very problematic, represent an unmet medical need that all urologists struggle with. And this is a group of people who are not well served by the standard of care.

That said, the trial, we hope, will show that the approach using 102 is effective in decreasing the recurrence rate in this population. And depending on the extent to which that is achieved, we will then, I'm sure, go back and discuss how those data could be used along a regulatory pathway with the agency. I think it would probably, from my perspective, be premature to predict whether or not an approval could be based upon those data because we don't have them in hand yet. But obviously, that's a subject for further discussion. Ron may also want to comment on this as well.

Yes, Mark, I completely agree. It's premature to make that determination. Obviously, the strength of the data will determine what approach we would want to take with the FDA and to be continued. But the most important thing is that we will have initial data to present in the first half of 2019. So hopefully, we'll get people to focus on the program and on the potential as that moves along and to give people a sense, hopefully, of the strength of UGN-102 in this indication, in these tough-to-treat patients, the high-recurring patients. So that's -- the first stake in the ground is to be able to present some preliminary data from the study.

The next question comes from Boris Peaker from Cowen.

More questions on the OLYMPUS study. Maybe we'll start with that. I'm just curious, was the response assessed by investigator or centrally reviewed? And is that something that came up in the FDA discussions?

So Boris, I'll direct that question initially to Mark with a follow-up by Ron.

Boris, thank you for the question. So the response rates are actually adjudicated at the site, which reflects a desire on the part of the sponsor to create a protocol that would be reflective of real-life practice. We do, in the case of disagreements amongst pathologists, have a central pathology adjudicator, if that's required. For the most part, it has not been. And again, just to specifically answer the question, the response rates are actually assessed and recorded by the investigators. And it did not come up. That specific point did not come up, to my memory, in our conversations with the FDA.

Great.

Yes, I'll just add that the FDA is in full agreement that the visuals can be locally assessed. And in addition to that, keep in mind that in order to confirm a complete response, we also have a cytology test, which is empirical. It's a 0 or 1. And the patients need to be negative on cytology. So that's another added measure that we have to confirm complete response.

Great. My next question though on manufacturing, just curious, what's the status of manufacturing approval and make sure that everything is -- that, that's not -- doesn't become a limiting issue?

Good question, Boris. I'm going to direct that question to Stephen first. And then I'll do some follow-up on it and possibly Ron.

Yes, I mean, just to remind folks, we have a network of CMOs we've been working with through the clinical setting. And so we're well into working with them closely with their QA departments, our QA department. And all of that is feeding into the CMC module that will go into the FDA. So I think we feel positive on where we stand on those conversations from the manufacturing side.

Yes. And the things I would add to that, Boris, is that we're also looking at downstream supply chain, so 3PLs and as well as specialty distributors. I think that process is well underway. We've got a very clear view as to how we want to go to market with those parties, which is very important. That kind of sets the stage then as to how the final product is packaged, delivered to those parties, then sub-delivered to the final physicians, investigators who are going to be utilizing the product. So both upstream and downstream from a manufacturing perspective, we're pretty far along in terms of that process, both from the CMO side as well as the final distribution side. And that's obviously plugged in very closely with our commercial guys, who ultimately will be out there in the streets selling this thing. So that's kind of a big, broader view from that perspective. Ron, any further comments there?

No, nothing to add. Thank you.

Great. And lastly, I just wanted to quickly ask, maybe this is for Ron, have you met with the EMA? And I'm curious what the status for Europe then is.

We will be meeting with the EMA by the end of the year. So we'll be able to provide an update shortly thereafter.

The next question comes from Derek Archila from Stifel.

I guess my first, just on the OLYMPUS study. I think you guys noted about 1/3 of the patients were presenting with unresectable tumors. Just want to get a sense of how representative of that number is with kind of the overall UTUC population? And how -- again maybe from a commercial perspective and working with payers, how you view kind of unresectable and resectable and again the commercial opportunity kind of in both of those populations?

So Derek, I'm going to direct that question to Mark first. Ron will chime in and then I'll add a few points at the end.

So thanks. That's an interesting question. And to give you a sense of what's driving the real issue we're addressing with this therapy, let me step back and tell you that we know that people with this disease, low-grade UTUC, are having their kidneys removed in as many as -- inasmuch as 80% of the cases. The things that drive kidney removal are recurrence, rapid and multiple recurrences, multifocal recurrences and unreachable, and that's what we mean by unresectable disease, disease that you can't get the scope all the way next to, so you can put the laser fiber next to it to get rid of the tumor. We know that, that's a common occurrence. We know that, that is a driver of kidney removal. And I think the study reflects the fact that it is a common problem in clinical practice. So I think it is one of the important contributors to the aggregate population or defining the aggregate population that will benefit from UGN-101 therapy.

Yes. So yes, I'll just add that we believe that the FDA was clearly intrigued by this unresectable patient population within our study, which is hovering a little bit above 35%, as we speak, of the patients in the program. And this is just based on the fact that as part of the breakthrough designation, they asked specific questions about those patients. So again, we can only deduct that the FDA certainly was intrigued by that subpopulation. Clearly, the ability to generate complete responses in these types of patients is a major attribute of the drug and, we believe, is going to be obviously -- can provide us with significant leverage, as we discussed, the value proposition that we bring to the table with payers and physicians alike.

And going back to your question regarding the -- basically, the proportion of patients that are unresectable in our study versus the overall population. Again, it's very hard to find literature that draws a direct comparison. We're probably slightly above the average in the population. So if you look at the population at any given point in time, probably 1/4 to 1/3 would have unresectable tumors at any given point in time. We're hovering right there, maybe slightly above. But as Mark mentioned, what's very important to mention is that if you look at these patients and you follow them out for a number of years, inevitably almost everyone will have his or her kidney removed.

So this particular study that Mark was alluding to looked at a very large cohort of UTUC patients, about 16,000 of them that were followed for a period of about 8 years. And about 80% of them within that 8-year period had their kidney removed, which essentially means that you can dodge the bullet once by having a resectable tumor, maybe twice, but it's not going to happen forever. And eventually, these patients will present with unresectable tumor. So the ability to generate complete responses and in essence, spare organs in this patient population, we believe, is tremendous. Peter?

Yes. So I think Ron hit on all kind of the dovetailed commercial points that then lead to kind of where does this thing play commercially and from a pricing perspective and those types of thoughts relative to this patient setting. I think from our perspective, at the end of the day, and we've been saying this to you guys all along, we want to see the final target product profile based upon the clinical data that comes out of the ultimate program here. And based upon that and the submission package that then goes into the FDA sometime next year, clinically speaking, that then sets the stage commercially as to kind of how this thing is going to present itself and exactly how we're going to go to market with regards to the product and price it, so on and so forth.

I can tell you that we've been doing a tremendous amount of work internally in the company as we speak with regards to looking at the continuum of care, both from a resectable as well as unresectable perspective. But with the kind of term of these patients' lives from the time they're initially diagnosed to, call it, 10 years out, which is typically for the most part when a lot of these patients perish. And a lot of times they're perishing not from the disease itself, but they're perishing from other comorbidities associated with the disease. So we're really trying to understand what that looks like. Obviously, the final target product profile will ultimately dictate where we go with the product and what we get approved on. But we think that there is a very viable and compelling story here from a value proposition perspective relative to what it currently costs to treat these patients over that continuum of care. So it's a good story.

Great. And just one quick one on OPTIMA II. So I guess, in -- as we think about that population of patients, these surgical failure patients, I guess, are these patients still obviously being treated with TURBT now? And I guess, the 2 questions would be, first, what kind of durability do you see for the complete responses for TURBT in those types of patients? And then in kind of a registrational setting, potentially in a registration trial, would you have to have that as kind of the active treatment arm, TURBT versus VesiGel?

So I'll turn that question over to Mark and then we'll go from there.

So since we've acknowledged that these are people who are surgical failures and we're sort of selecting an adverse population for the trial in order to make the point, we do expect the recurrence rate in this group, which is, as you pointed out, currently treated with TURBT, to be very high in 1 year. If you look across the nation, if you look at this population, recurrence rate is certainly at a year likely to be 40% or maybe even higher. So we would imagine, and this is again to some extent hypothesis generating, that a trial with the 102 product generating a recurrence-free survival rate a year of 50% or 60% would be very validating for the technology. Again, we have to do the trials and see what the data look like. But I think those general parameters would give you a sense of what we think internally success would look like. And Ron may want to comment on that as well.

Yes, I'll just add, we're looking at what we call surgical failures. These are patients that recur frequently. So as you can imagine, just by doing back-of-the-envelope analyses, the patients who are going -- on average that we're going to be looking at in the study will probably have recurrences of within 6 months or so on average, so -- and when you want to compare that to our data, obviously if we can beat their historical recurrence, I think we're going to be off to a great start. And based on the data that we've seen in the past and the European experience, we're seeing some very prolonged responses with 102, albeit in an all-comers population and this is a little bit more specific, these high-recurring patients. But we believe that the drug is going to perform well in this study. And hopefully, we can present preliminary data that already starts to hint to that effect in the second half -- in the first half of 2019.

The next question comes from Leland Gershell from Oppenheimer.

First question, I think, for Mark. Your comment about the durability we're seeing in the OLYMPUS trial as it continues. Perhaps, showing what may be a disease recurrence beyond what we see historically with surgical removal. If you could just remind us kind of what the numbers are around the data historically from the nephrectomy case?

Sure, Leland. Thanks for the question. So when you look at the peer-reviewed literature on endoscopic resection of these tumors and you look at a large body of literature, what you come up with is that with endoscopic ablation for a low-grade UTUC, the recurrence rate is around 6 months. So as a sort of target point for us, we believe and are encouraged by our data and believe that our data are going to compare very favorably to that historical benchmark.

Okay, great. And then with the hiring of Woody, how should we think about business development activities for the platform as we head into 2019?

Yes, I'll take that one. It's Pete. So I think we -- first off, we think the platform has got a lot more applicability than kind of beyond what we've done so far with UGN-101, 102 and 201. We know of a number of opportunities that are out there in the marketplace that potentially could play, call it, in our space, the space that we're practicing in, in urology, oncology, other drugs that either have not as good, call it, a side effect profile because they're dosed in other forms or because from a clinical efficacy perspective potentially could be a little bit better. So we kind of view that as an opportunity to bolt on to the platform whether those are through partnerships or those are through situations where we continue to advance the science and do it in a collaborative manner. So that's an opportunity, I think, that we're looking at very actively.

We also know of other plays outside of our space. So obviously, our first focus is always kind of within our space. The next focus is kind of outside of our space. Other applicabilities of the gel technology in other areas in the body, and I know we highlighted a number of those as part of our overall corporate presentation. Those are, I'd call, Allergan-like-type agreements, where Allergan is looking obviously to advance BOTOX in the area of overactive bladder, get it into a better dosage form than it is currently dosed today, which is via catheter and injections into the muscle wall. So those are other opportunities that we're obviously looking at pretty actively. But that's kind of, I'd call that, secondary versus the primary. And then just kind of expanding our overall platform as a company. I think the other side of the equation is positioning the company for the best possible position in terms of we're a urology-oncology company.

So maybe it's beyond our platform technology and the products and drugs that we're already currently delivering and some other potential applicability in terms of other technologies that might come down the road that could help round us out in that area. So again, we're kind of taking it in a stepwise fashion there. But we're being very thoughtful about what we do there because our first and primary focus, obviously, is around low-grade, upper-tract urinary cancer, so -- and bladder cancer. So it's a -- our focus is to kind of stay in that zone and leverage our platform and then also leverage other products that can come in the platform as well as possibly some other platforms and drugs that might be very beneficial to putting a couple products in, call it, our commercials group's bag. So that's the idea there.

Great. And then just a timing question with the -- I guess, back to Mark or Ron.

No. Yes, I'll just -- I'll add -- Leland, I'll add one more thing. Woody is a very seasoned business development professional and has done a lot of inbound and outbound deals. And just look at his recent experience with Sucampo, where he spearheaded the sale of that company to Mallinckrodt. So obviously, we need to build internal inherent value in the company. So we're doing a lot of things that we think are going to be smart in terms of building out the strength within the company. And that's all of the things that Peter alluded to. And so we're very keen and focused on building the value and building the leadership position that we hope to establish in this world of uro-oncology and possibly beyond.

But obviously, we're also aware of the fact there are certain, for example, ex U.S. territories where it would make sense to partner. And obviously, we also want to stay opportunistic on some other business development fronts. So that's why we brought in a very seasoned professional who can kind of help shepherd all these processes. So I think we've hired one of the best in the business, and we'll see what happens. But we're going to be -- we're going to be remain very focused on building the inherent and internal value within the company.

That sounds terrific, good. And one last question, Ron, just with the top line data from OLYMPUS now being January but then your hopes to have the full data at the meeting, would that make it possibly for ASCO GU? Or is it more of an AUA type of venue?

It could very well be AUA. Obviously, we can't guarantee anything at the moment and time will tell. And obviously, we would need to have all the data in place in order to file for late-breakers and abstracts and things like that. But we are -- AUA is certainly a possibility. But if it won't be AUA, I'm sure we'll be able to find some other venue.

We have a question from Reni Benjamin from Raymond James.

I apologize, I jumped on to the call a little late, so I'm apologizing if you've already answered these in the prepared remarks. But can you just tell us, when was the last patient enrolled in the OLYMPUS study? And can you just remind us the sort of the process of what's involved in locking the data and undergoing the analysis? And is that done in-house? Or is that done by a third party?

So Reni, I'll first put that to Ron and then it will be followed up by Mark.

So Reni, we talked a little bit about that. So right now, we've got approximately 70 patients enrolled in the study. Keep in mind that once you essentially announce that you're going to be closing the study for enrollment, which we did last week, you still have patients who have signed an informed consent and you give -- you need to give these patients a fair chance of making into the study, meaning to either pass screening or fail and then, of course, they're excluded. So after we put out that notice, we still had a couple of patients like that. I don't know the final status on each and every one of them. But essentially, we're at about 70 patients as we speak. We could have 1 or 2 that will be officially enrolled/treated within the next week or 2. And that will round it out. So that's basically what we're looking at right now.

So at the time of our discussions with the FDA or even a little bit before that, when we had sent them the briefing documents and so on, we had about 65 patients. Now that number has gone up. And so in fact, we're actually pretty much at the number that we had initially anticipated, which was that same as 74. But the most important thing to understand is that the timelines are remaining steadfast in terms of the submission. We're starting the rolling submission in this quarter. We will finish the submission in the second quarter. And obviously, we still anticipate launch in the fourth quarter of 2019. So everything is still very much on track.

In terms of the database lock and what that entails, so obviously we've got several vendors that are involved. But what you need to do in order to make that as efficient as possible is to do as much of a clear resolution as you go because obviously patients come in, in a staggered fashion. So you're able to do that bit to a large extent. And obviously, you have to be very vigilant with the sites to make sure they capture the data in timely fashion, that they are very proactive about bringing patients for the study visits on time. And if you're able to do that, then you're in a position where you can lock the database within a relatively short time after that last patient visit, so to speak. And then it's a matter of, of course, finishing up the clinical study report, the tables, listings and figures, the integrated reports and so on. But a lot of those skeletons, if you will, of these documents can be written well in advance. So there are a lot of things that are happening in parallel. And that's how we're going to move forward between now and the completion of the submission.

Got it. I didn't know if Mark wanted to come in on this, but I guess, can you talk a little bit -- or do you have any color regarding the patients that have been enrolled into the trial since the interim results were announced? And the reason why I'm asking is the interim results actually came in much better than at least I was expecting. I always think about phase -- well, pivotal studies as getting a more heterogeneous group of patients and response rates going down. And we actually saw the opposite. So is it fair to assume that, that exact sort of character group of patients are what came in afterwards as well and that this new kind of response rate and duration of responses is what to expect when the final results are coming out?

Mark?

Thanks, Ren. The answer is we didn't change the protocol at all. So we've been doing the same thing over and over again throughout this entire trial with the same inclusion and exclusion criteria. And I think the results are going to speak for themselves. The reception by the FDA, I think, is very validating. And I would expect that the data would look very similar without a change because we've been doing the same experiment the whole time through.

Got it. Then as one final question for me, you may have talked about this already. But precommercialization activities, can you talk a little bit about what that will look like, what a potential sales force ramp might look like? And I know, Peter, you talked about the burn and how long the cash would last. But should we be seeing a significant bump-up in the first half of 2019?

Yes. So I'll take that and then Ron can round it out. So I think you should naturally believe that the burn will kind of increase a bit, especially in 2019 relative to the precommercialization/commercialization process. I mean, as we've touched upon a number of times during the course of the call, it is our intent, plans, timelines, everything is still kind of adding up for a Q4 approval, Q4 commercialization at the end of next year. With that being said, the real buildup in terms of a commercial sales force and infrastructure will take place, call it, in Q2, Q3. That would be really kind of when the uptick starts taking place in terms of the buildout of the organization respectively for next year. I don't think -- this is really, for all intents and purposes, a rare disease kind of commercialization play, strategy. It's -- you also look at kind of the accounts that we want to go to and centers that we'll be selling to. And it will be an account-based approach in terms of the commercialization path going forward.

The reality is you might be looking at a commercial sales force somewhere in the 40 to 50 realm, not including some back-office infrastructure that we put in place in the organization. We've been pretty consistent kind of in the messaging around kind of what that looks like in the buildout of that. So really as we kind of move from this quarter to, call it, a year from now, this year, the team is -- it's grown to about 4. We've got a Head of Commercial. We've got a Head of U.S. Sales, Marketing and Patient Access. We've got some infrastructure there now. We'll continue to build it out with medical liaisons through the course of the end of this year. But the real kind of start to build a few more heads will be in the first quarter. We're not going to go hog wild. We really want to make sure that the whole overall clinical submission and rolling NDA process is completed before we really kind of pull the trigger on that one.

So again, timeline-wise, first quarter still is going to be a little bit of an uptick but not huge. And then it's really a second, third quarter filled, with the fourth quarter, we're in place, we've got everything done. Everything from -- as we touched upon earlier, once we have the final data package submission to the FDA, we'll know the target product profile that we're going to go to market with. We'll know how to price that. We've obviously got some work already well underway with regards to that, looking at the patient journey, looking at the cost, all those things. So it's -- I think, it will add up for, call it, a Q4 launch and a bit of an uptake Q2, Q3 and then really Q4 ready for commercialization.

We have a question from Jonathan Aschoff from National Securities.

And I wondering, getting back to an earlier question about 102, would you definitely need a control group for a pivotal 102 trial if Phase IIb is successful and not deemed pivotal by the FDA? Or would you not need a control group? I'm asking because the absence of a control group for 101 is a major point in that trial's low risk of a negative outcome. And I was just kind of hoping you could replicate that highly favorable pivotal trial environment for 102 as well.

So Jonathan, I'll direct the question at Mark first and then Ron.

It's a really interesting question. And I think the problem that I have answering it is I'd like to see some data before I tell you. I think that if we are pleasantly surprised by what we see along the lines of what we saw in the 101 program, we may need to meet with the agency to discuss what you're describing. But I think it would be premature to contemplate at single arm at this point in this indication because it is a somewhat different disease with a somewhat different profile. Ron may want to comment on that as well.

Yes, I'll just add, Jonathan, that there's a reason why we chose this particular patient population, which is the subset of the overall low-grade, non-muscle-invasive patient pool, which is, as you know, enormous. We specifically chose the tough-to-treat patients because hopefully that will allow us to pursue a single-arm type of an approach. So that was really the thought process that went into the study design for this Phase IIb to begin with. So hopefully, as Mark said, if the data plays out in our favor, that will give us a lot of leverage as we head into those discussions with the FDA to talk about next steps.

Okay. And given the similarity between 102 and 101, are you pursuing breakthrough for that as well?

Mark? Ron?

Yes, I'm going to defer to Ron on that.

Yes. No, still too early to tell, Jonathan. And we would need some data in our hand in order to pursue that anyway. We could not approach the FDA with something theoretical. They would need to see some clinical data. So that's to be determined at a later stage. But initially, we would need some data for that.

I am showing no further questions at this time. I will now turn the call back over UroGen's CEO, Ron Bentsur, for closing remarks.

Thank you, operator. I want to thank everyone for taking the time to join us on the call today. We're very excited about the progress we've made toward the potential approval of UGN-101, and thank you for your support as we prepare for an exciting year ahead for UroGen. Operator, you may now disconnect.