Good morning, ladies and gentlemen. Thank you for standing by, and welcome to UroGen Pharma's Fourth Quarter and Full Year 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 Pharma. Please go ahead..

Thank you, operator. Good morning, everyone, and welcome to UroGen Pharma's fourth quarter and full year 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 and full-year ended December 31, 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 Liz Barrett, President and Chief Executive Officer; Dr. Mark Schoenberg, Chief Medical Officer; and Peter Pfreundschuh, Chief Financial Officer.

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 fourth quarter and full year 2018, before we open up the call for questions.

Joining us for the Q&A portion of this call will be Stephen Mullennix, Chief Operating Officer. 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 10-K to be filed with the SEC today, 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. Good morning, everyone, and thank you for joining our conference call. As many of you may know, I joined the UroGen team in the beginning of January. So, this is my first call as CEO of the company. I am absolutely delighted to have joined UroGen at such a defining time in the company's evolution.

Since, I have not had a chance to personally introduce myself to everyone dialed into the call, I'd like to take this opportunity to briefly review my background and experience before diving into UroGen's recent progress and upcoming milestones for 2019.

I've been in this industry for over 30 years, and prior to joining UroGen, I most recently served as CEO of Novartis Oncology.

Prior to Novartis, I was the Global President of Oncology at Pfizer, where I had multiple leadership positions, including President of Global Innovative Pharma for Europe, President of the Specialty Care Business Unit for North America, and President of US Oncology.

I started my healthcare career at J&J leading multiple businesses and built the Oncology Business Unit as Vice President and General Manager at Cephalon. The breadth and depth of my experience has prepared me for this role as President and CEO of UroGen, and I have quickly integrated into the organization.

I'm extremely impressed with the team here and the tremendous progress that has been achieved to advance the potential of our RTGel technology, as we work towards building our company as a global leader in uro-oncology.

Shortly after I joined UroGen, we were pleased to announce positive results of our lead investigational candidate, UGN-101 from our pivotal Phase 3 OLYMPUS trial for the non-surgical treatment of patients with low-grade upper tract urothelial cancer or LG UTUC.

We were very encouraged by the durable complete responses observed and believe that the data continue to support the potential of UGN-101 to change the treatment paradigm for patients with current options consist of repetitive endoscopic surgical intervention or complete loss of a kidney.

We have received Fast Track, Orphan Drug, and Breakthrough Therapy Designation from the FDA for UGN-101. We initiated a rolling submission of our new drug application or NDA in December 2018 and are intensely focused on completing the submission in the second half of 2019 with the potential approval and launch in the first half of 2020.

If approved, UGN-101 would be the first drug approved for the non-surgical treatment of low-grade UTUC. As we work toward a potential approval in the first half of 2020, commercial planning activities are advancing on schedule to ensure that our infrastructure is equipped to support our first commercial launch.

We've hired a team of accomplished senior level executives with experience in commercial, marketing sales, market access and medical affairs, to oversee the efforts for UGN-101. This team has a track record of success with significant experience in urology. In the near-term, we are focused on planning and analysis.

Our commercial strategy will incorporate a multifaceted approach to support uro-oncology practices across the country. Finding urologists who treat UTUC is critical, and our research indicates that approximately 33% of urology accounts actually treat 90% of the patient population.

As a result, we will implement a targeted account-based approach with a small nimble and highly experienced salesforce of approximately 50 individuals to support the unique needs of UGN-101.

We are also very focused on ensuring that we will support the physician office and make UGN-101 a seamless addition to their practice, following potential approval.

Education and scientific exchange are critical components, and our medical affairs team will play a key role to make certain that healthcare professionals are appropriately educated to increase their comfort level with this potentially paradigm shifting therapy and ensure early market adoption upon potential approval.

To that end, our team and medical science liaisons or MSLs have been hired and will be prepared to execute on education, scientific exchange and support of our clinical team. Urothelial cancer is the ninth most common cancer today, and it is the most costly cancer on a per patient basis.

We are conducting a comprehensive analysis to define the cost burden to the system for low-grade UTUC. Remember that the patient population for this disease is elderly.

So, if you think about comorbidities of hypertension and diabetes in these patients, you can imagine that the removal of the kidney, or repetitive endoscopic surgeries is very debilitating, giving us a very compelling case for UGN-101. As we prepare for launch, market access, as well as distribution and logistics are critically important.

Our proactive strategy will help make sure that patients who need UGN-101 will be able to receive it and that physicians will be able to acquire UGN-101 when needed. We are confident in our ability to execute on our current work streams and build a highly efficient commercial organization.

Our team will be fully prepared as we transition into a commercial company. Subject to UGN-101's approval, it is important to note that as we progress our commercial planning, we are also setting the groundwork for UGN-102. Our investigational program for low-grade non-muscle invasive bladder cancer, which is a much larger patient population.

We are already off to a strong start in 2019, with the completion of a follow-on public offering in January, raising approximately $162 million in net proceeds.

With our dynamic leadership team firmly in place, commercial preparation is well under way and with solid financial position, we are prepared for what is poised to be a defining year for UroGen.

We are closing in on potential approval of our first therapy and continue to advance our pipeline programs, as well as to assess partnerships to further enhance the potential reach of our RTGel platform in uro-oncology and beyond.

As we approach completion of our rolling NDA submission later this year, we look forward to updating you on the progress and planning activities. I would now like to turn the call over to Mark to discuss in more detail our progress in clinical development programs.

Mark?.

Thank you, Liz. As Liz just mentioned, we recently reported positive results from our pivotal Phase 3 OLYMPUS clinical trial of UGN-101 for the non-surgical treatment of low-grade UTUC. We observed a 57% complete response rate or CR, which was very consistent with the data that were previously presented in May 2018.

Importantly, all evaluated patients in CR remain disease free at six months. At the time of the announcement, approximately 50% of the patients that were in CR had reached the six-month time point, and we will continue to follow and evaluate these patients.

The safety profile of UGN-101 continues to be acceptable with most treatment-emergent adverse events characterized as mild to moderate and transient and in line with urethral procedures.

We are encouraged by the high response rate and durability observed to date, which provide further evidence that the non-surgical treatment of low-grade UTUC with UGN-101 may result in clinically meaningful recurrence free survival.

The treatment of low-grade UTUC represents a technical challenge for urologists, as the current standard of care requires surgical intervention, which puts patients at risk for the well-known complications associated with repetitive surgical procedures and potential kidney removal.

Approximately 80% of patients who present with low-grade UTUC will undergo kidney removal at some point in their treatment, either at initial presentation or once repetitive endoscopic surgery fails to control the disease.

As we have previously discussed, these patients are generally elderly and are subject to the short-term risks associated with kidney removal surgery such as, bleeding, infection, injury to adjacent organs and the risks of anesthesia, and the effects of chronic renal insufficiency.

Renal preservation in this population would address a significant unmet medical need. In the OLYMPUS trial alone, 45% of patients presented with endoscopically unresectable tumors. In real practice, these patients would have been immediate candidates for kidney removal.

This is another example of how UGN-101 has the potential to transform the front-line management of patients with low-grade UTUC. As a urologist, I believe that UGN-101 has the potential to make a substantial impact on contemporary clinical practice.

In addition to providing physicians with an opportunity to avoid the risks associated with repetitive endoscopic surgery, as well as those associated with kidney removal, UGN-101 fits nicely into current urology practice.

It requires the use of equipment, procedures, and protocols that are familiar to practicing urologic surgeons and aligns well with standard ambulatory practice. Beyond UGN-101, we are equally excited about the potential for our UGN-102 program.

We are currently enrolling patients in our OPTIMA II Phase 2b single-arm open label multicenter trial designed to assess the efficacy and safety of UGN-102 for the intravesical instillation, as a potential first-line chemoablation agent in the treatment of patients with recurrent low-grade non-muscle invasive bladder cancer.

Let me first describe this patient population. We have chosen to study intermediate risk patients. This is a patient population, whereby the current standard of care Transurethral Resection of a Bladder Tumor or TURBT procedure 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 in real life, to manage bladder cancer recurrences. We believe UGN-102 can have an immediate impact on this population and provide a non-surgical alternative for the treatment of chronic relapse.

UGN-102 represents a substantial opportunity for advancing our RTGel technology platform in a larger patient population. Today, there are approximately 700,000 people living with bladder cancer in the United States, and approximately 80,000 who were classified as intermediate risk.

Similar to low-grade UTUC, there are no currently approved drugs by the FDA as first-line treatment for low-grade non-muscle invasive bladder cancer. We are pleased with the enrollment and anticipate a data update in 2019.

While six-month durability is important for low-grade UTUC, we believe that recurrence free survival at 12 months for low-grade bladder cancer is clinically meaningful. Thus, these data would consist of CR rates with durability information to be reported at a later time. We remain on track to complete enrollment this year.

We are also developing UGN-201, an earlier stage program that leverages our platform in immuno-oncology. We are currently evaluating various pathways to advance the program into clinical development for high grade disease. We plan to provide an update on the development path for UGN-201 later this year.

Finally, our last clinical program is a collaboration with Allergan. Allergan is conducting an ongoing Phase 2 trial of BotuGel, a combination of our RTGel and BOTOX for the treatment of overactive bladder or OAB. Current treatment with BOTOX for OAB requires approximately 40 to 60 injections directly into the bladder.

BotuGel has the potential to change this treatment paradigm with a topical therapy versus multiple internal injections in the bladder. If successful, we believe this study will demonstrate the potential of our RTGel technology platform beyond the field of Uro-Oncology.

And with that, I would like to turn the call over to Peter, who will discuss the financials..

Thank you, Mark, and good morning to all of you on today's call. UroGen is entering 2019 well capitalized and ready to support our clinical development programs and commercial planning efforts in preparation for a potential US approval of UGN-101 in 2020.

We ended 2018 with $101.3 million in cash, cash equivalents, and short-term investments, and in January, we successfully completed the public offering of ordinary shares, resulting in net proceeds of approximately $162 million.

For the fourth quarter and full year ended December 31, 2018, we reported net losses of $23.7 million or $1.46 per share and $75.7 million or $4.80 per share, as compared to $10.1 million or $0.74 per share and $20 million or $2.14 per share for the same periods in 2017.

Research and development expense for the fourth quarter and full year ended December 31, 2018 were $11.5 million and $36.9 million as compared to $6.8 million and $18.7 million for the same period in 2017.

Research and development expenses include $3 million and $12 million of non-cash stock-based compensation expense for the fourth quarter and full-year ended December 31, 2018, as compared to $1.4 million and $3.9 million for the same periods in 2017.

The increase in research and development expenses from 2017 to 2018 reflect an increase in direct costs associated with the UGN-101 Phase 3 OLYMPUS trial, cost to initiate the UGN-102 Phase 2b OPTIMA II trial, an increase in personnel costs to support our ongoing clinical and regulatory efforts and an increase in share-based compensation expense.

General and administrative expenses for the fourth quarter and full year ended December 31, 2018 were $12.6 million and $39.6 million as compared to $3.4 million and $8.8 million for the same periods in 2017.

General and administrative expenses include $5.9 million and $18.6 million of non-cash stock-based compensation expense for the fourth quarter and full-year ended December 31, 2018, as compared to $0.9 million and $2.4 million for the same periods in 2017.

The increase in general and administrative expenses from 2017 to 2018 was attributable to an increase in personnel and related costs to support our growing business, an increase in commercial infrastructure and services, an increase consultant and other outside fees, and an increase in share-based compensation expense.

As of December 31, 2018, we had approximately 16.2 million ordinary shares outstanding. Based on our anticipated activities, we project our current cash balance to carry us for the next 24 months to 36 months. Post our January 2019 financing, we refreshed our best business plans to achieve the goals that we have set out for the company in 2019.

Based upon this, the company anticipates a net loss in the range of $100 million to $150 million for 2019, which includes non-cash stock-based compensation expense in the range of $24 million to $27 million subject to market conditions.

The change in net loss for 2019 versus 2018 is largely attributable to the build-out of commercial infrastructure in anticipation of the potential launch of UGN-101. The company has 20.4 million ordinary shares outstanding, post the closing of our most recent financing in January.

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

Thank you. We will now begin the question-and-answer session. [Operator Instructions] And our first question comes from Ram Selvaraju from H. C. Wainwright. Please, go ahead..

Hi. Thanks very much for taking my questions.

I just wanted to know if it would be possible for you to provide us with some additional detail regarding the filing process for UGN-101? Secondly, I wanted to see if you could comment further on how you expect the commercialization strategy for UGN-101 to differ from UGN-102? And if you could clarify for us whether looking forward to the potential future approval of UGN-102, you would be looking to deploy exactly the same sales infrastructure sales personnel for the support of the rollout of both products or if you anticipate there being any notable differences? And finally, with respect to BotuGel, do you have any feedback directly from Allergan regarding what they intend to do if the next set of clinical data is positive, as one might expect it to be? And furthermore, to what extent they anticipate BotuGel being a component of their long-term franchise management strategy for BOTOX overall? Thank you..

Okay, great. Thanks. It's Liz Barrett talking – speaking, just so you understand, I'm going to answer the last one first, because it's easy and we cannot comment on what their expectations are, what their plans are. So, I think that's a question for Allergan.

To talk a little bit more about the filing process, it's in a rolling submission, you basically, as the different components are ready, you're able to submit those to the FDA. We have already submitted the preclinical module.

We are currently working on the CMC module, and that module will go in next, and then the final module that will go in will be the clinical module.

Our timing for this final submission is really driven by the fact that we have to wait for the majority of the patients to reach the six-month durability milestone, because that was the requirement by the FDA that we needed to have the majority of the patients in – at least 75% of the patients at six months.

The six-month – and Mark can obviously add any commentary here, was really driven by the fact that the patient population – average time of recurrence is six months. And so, that's why the FDA felt like six-month data was really critical importance.

So, the only really rate-limiting factor for finalizing the filing, the final submission is just simply as I said, waiting for those patients to reach that milestone. From 101 launch, I would say we're very excited about all of the work that's been done.

I think 2019 is going to be a very busy year for us, as we spin the year, building awareness and educating the physician population through appropriate medical education. When you think about the strategy of 101 and 102, I would say similar in the sense of – obviously, it's the same physician group.

The difference would be that – with 102 it would be a broader physician population that we would be reaching because there obviously are more patients and more physicians treating these patients. So, we do believe at this point in time, we would have to expand the current commercial organization to reach more physicians at that point in time.

So that's – I think that would be a bit how they differ, the actual products are different, the therapies are different and instillation method is different. And so again, similarities – a lot of similarities, but obviously, a lot of differences as well. So, hopefully that answered all your questions.

I don’t think I missed anything, if I did, please let me know..

No, just a point of clarification regarding the filing.

I don't know if you plan to provide specific notifications of the completions of the submissions of different sections of the NDA as they go in, or if you're just going to notify us when the NDA submission is fully complete?.

We would not. There's really no reason, to be honest with you to do so. It doesn't mean that if you're interested in learning, we're happy to let you know individually, but there would be no to public disclosure of that..

Okay. Thank you very much..

Sure. Thank you..

Our next question comes from Boris Peaker from Cowen. Please go ahead..

Great. Good morning. My first question is on 101.

Just curious, when will we see 12 months durability data, or is that something that you anticipate to report altogether?.

Yes. We won't be sharing any additional information until the filing, I mean until the trial is complete. We will show at some point, obviously 12 months durability data, but it's not an endpoint for us. And so, before we would have all of that data, it would really be mid to late 2020.

So – but yes, we will absolutely, you know after we finalize the FDA submission and the data package is final, we will be publishing and hopefully presenting the full dataset that we have, including data on the 12 months and the number of patients we have 12 months data on hopefully by the end of the year..

Great. And my second question on UTUC, and this is just kind of a patient management question that, if a patient comes in with UTUC and their kidney function is fine, both of the kidneys, why not just remove one of the kidneys on the side of the tumor and eliminate any risk of further progression since the other kidney could takeover.

I don't know why wouldn't physician just choose that option?.

Yes. I'm going to let Mark answer that question as a practicing urologist. He has a lot of experience in this area.

Mark?.

Thanks, Liz. Boris, that's a great question. From a clinical perspective, a couple of facts to keep in mind. The risk of the patient developing a similar malignancy in the remaining kidney is somewhere around 2% to 5%. So that kidney remains at risk actually even when the original tumor has been removed with the primary side of development.

So, there is ongoing disease risk that would potentially compromise the remaining kidney. The other thing to keep in mind is really related to the age of the patient population affected by this disease. And I know that you're aware, as our audience on the call that the average age is 74 when this disease is diagnosed.

When you take the kidney – when you take a kidney out of a 74-year-old whose kidney function appears to be normal and perfect for that age, there still is a downstream risk approximately 30% of that person will develop particularly significant renal insufficiency.

So, there is a benefit to preserving kidney mass through a renal preserving therapeutic strategy that we're proposing with UGN-101.

And because of this disease tends to be a chronic relapsing illness, but not one that metastasizes the benefit to the patient of preserving the kidney actually is very significant and the risk of developing a widely metastatic disease is actually very small. So, the overall clinical benefit is to keep the kidney in the person..

Great. Thank you for taking my questions..

Thank you. Next question..

Our next question comes from Derek Archila from Stifel. Please go ahead..

Hi. Bill [ph] on for Derek. Thanks for taking our question. With regards to VesiGel, where you are at in discussions with the FDA? Could the open-label Phase 2 service a registrational study or do you think you'll need to run a Phase 3 versus TURBT or – and if you do, does it have to be superior or just non-inferior? Thanks..

No, it's a great question. I would say that we anticipate that we will have to conduct another study given the feedback that we've had so far from the FDA in the current study.

My only caveat to that, is that always depends on what the data looks like, and as Mark explained and described the patient population, this is an intermediate-risk patient population. So, they are at a higher risk for recurrence than your overall patient population.

So, depending on the results of this study and we will absolutely have a discussion with the FDA to see what study design that might look like. So again, we anticipate, we will have to conduct another study, what that study looks like, will be highly dependent on the data that we see coming out of this current study..

Great. Thank you..

Thank you..

Our next question comes from Leland Gershell from Oppenheimer & Company. Please go ahead..

Great. Thanks. Good morning. Thanks for taking my question. The question for Mark, on 102 and in NMIBC, if I recall correctly, your prior indication has been that we might see the first review from that study in the first half of the year, now it looks like it's simply for 2019.

Just want to ask about that any factors that may have played in, whether it's enrollment or you had made a comment about 12 months durability, the focus on that being more important than six-month. Just wanted to ask about that change in timeline? Thanks..

Yes, Leland. Actually, no change. We're just trying to be appropriately conservative about promising things ahead of actually having them in hand. As I mentioned, the enrollment for the trial is actually going very nicely and we're pleased with the way things are going.

So, we do anticipate providing an update this year, and obviously, Liz will tell us when she thinks it's appropriate to do that..

Okay, thanks. And then I guess it is a question for Liz. In terms of thinking about our modeling for incremental expense as the kind of commercial side begins to ramp up, how we should think about the pattern of spend through the year? Will that be more weighted toward the second half, or is that getting going earlier in the year? Thanks..

I think, what you'll see is that, we already started activity from a commercial standpoint of building awareness and education, but clearly you will see a ramp up in the second half of the year considering, we will be hiring the commercial organization really toward the end of the year.

So, we think about timing for that, but as far as spend from our promotional activities and educational activities, we would see those over the year – throughout the year. Again, little bit heavier in the second half and toward the end of the year..

With more personnel. Okay. Thanks very much..

Yes. Thank you. Thanks for the questions..

Our next question comes from Chris Howerton from Jefferies. Please go ahead..

Thanks. Good morning, and congrats on the progress. Liz, I think most everything has been asked at this point, but I guess one of the things Liz you mentioned was that you're starting to work on the overall economic burden with low-grade UTUC.

And just, I guess the question specifically is, any updates I'm just thinking for pricing for UGN-101 and how is it that you're thinking about it internally and how we might be able to think about it from an external perspective? Thank you..

Look, it's a great question. We're obviously not in a position right now to disclose the price.

We don't have a price, but to your point, one of the things that we are doing is not only looking at the data that is available, but also generating data around pharmacoeconomics, what kind of cost burden exist today with the current standard of care, and therefore have that information.

I would say when you think about pricing, we're thinking about it as an oncology product, and I would say in the range of what you would expect an oncology product to be. But again, until we have all of the pharmacoeconomic data that we're currently pulling together, we really don't have a final price.

But I think it would be fair enough to look at it from that perspective..

Okay.

And when you figured out the ways that this product [indiscernible] would be used in the clinic in terms of multiple instillations per course and potentially multiple courses per patient, how would that factor into your thinking with respect to pricing and what not?.

Yes. I think the way that we would look at pricing is, if you look at the clinical study, it's six instillations is really the treatment and that's how we will be looking at pricing as around the six instillations.

There was in the study the ability if a physician wanted to, to use maintenance therapy once a month after, there was a real heterogeneous approach to how they did that, some physicians did, some did not, there was not a specific amount or number.

So – and then as far as repeating the treatment, we actually do not have data currently on retreating, but it is a good point, because we are currently developing a study for retreatment.

So, for those patients that do recur, can you retreat them and then put them back into the CR, I think that's a very important piece of information that physicians and payers will want to look at and so we think that's an important area for us to generate new data.

So, again and thinking about it, I would think about it as far as the six instillations.

I think you also are going to find that some physicians and patients may not get the full six instillations, but at this point, as we approach the market, our expectation is that that's how we will deliver the message around the therapy being the six weekly instillations..

Okay. That's very helpful. Thank you..

Great, thank you. Thanks, Chris..

I'm 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 want to thank everyone for taking the time to join us on the call today. We look forward to providing further updates throughout 2019 as we work towards advancing the therapeutic potential of our RTGel platform and strengthening our position as the global leader in uro-oncology. Operator, you may now disconnect. Thank you..

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