UroGen Pharma Ltd. Q3 FY2023 Earnings Call

Thank you, operator. Good morning, everyone, and welcome to UroGen Pharma's Third Quarter 2023 Financial Results and Business Update Con Call. Earlier this morning, we issued our third quarter press release and filed our 10-Q where you can find details for our financial and operating results. Both documents can be accessed on the Investors portion of our site at investors.urogen.com. Joining me on the call today are Liz Barrett, President and Chief Executive Officer; Dr. Mark Schoenberg, Chief Medical Officer; Jeff Bova, Chief Commercial Officer; and Don Kim, Chief Financial Officer. During today's call, we will be making certain forward-looking statements. These may include statements regarding our ongoing commercialization activities related to JELMYTO, our ongoing and planned clinical trials; commercial and clinical milestones; market and revenue opportunities; our commercial strategy and expectations as well as potential future commercialization activities for UGN-102, if approved; anticipated data; regulatory filings and decisions including UGN-102, potentially receiving priority review. UGN-102 is being positioned as a growth driver for UroGen, if approved; future research and development efforts; our corporate goals and 2023 financial guidance, among other things. These forward-looking statements are based on current information, assumptions and expectations that are subject to change. A description of potential risks can be found in our earnings press release and latest SEC disclosure documents. You are cautioned not to place undue reliance on these forward-looking statements, and UroGen disclaims any obligation to update these statements. I'll now turn the call over to Liz.

Thank you, Vincent, and welcome to everyone joining us today. Before we remark on the quarter, I must mention the October 7 attack in Israel and its impact on our Israeli colleagues, partners, and investors. The safety of our employees is and will continue to be top of mind. With regard to any potential impact to our business operations, I want to assure our shareholders that while we have a portion of our workforce based in Israel, we have robust contingency plans and international partnerships in place to ensure the continued smooth operation of our business. As a result, we do not anticipate any significant impact on our business or operations. Finally, we hope and pray for peace to return to the region as soon as possible. I'll now turn to the quarter. Q3 2023 was one of the most important quarters in UroGen's history. During the third quarter, we shared extraordinary top-line results from our Phase III clinical trials evaluating the use of UGN-102 to treat patients with low-grade intermediate-risk nonmuscle-invasive bladder cancer. Both the ATLAS and ENVISION trials met their primary endpoints, demonstrating meaningful and compelling results overall and compared to the current standard of care, TURBT. This is particularly notable because UGN-102 is being developed as the first nonsurgical therapy for this type of bladder cancer. Mark will talk more about this, but it's important to delineate the various types of bladder cancer and understand that our products are being studied in patient segments that are not being studied by other medicines. Following this announcement, we held a pre-NDA meeting with the FDA to align on the regulatory path forward for UGN-102. As expected, the FDA confirmed that the current clinical development plan for UGN-102, which includes evaluation of duration of response at 12 months following a complete response at 3 months, in the pivotal ENVISION trial, will support submission of an NDA. The FDA also agreed that our NDA can utilize a rolling review, allowing for early submission of CMC sections of the NDA, which is planned for January 2024. Looking ahead, we anticipate sharing data from the duration of response endpoint in the second quarter of 2024. Pending favorable results, we expect to submit the NDA to the FDA a few months later. If granted priority review, we anticipate approval and launch in early 2025. If approved, we believe that UGN-102 would represent a groundbreaking nonsurgical option for approximately 82,000 annual patients suffering from low-grade intermediate-risk nonmuscle-invasive bladder cancer, who currently face frequent recurrences, necessitating the need for multiple surgeries. This potential milestone stands to become a major growth driver for UroGen, with a substantial market in the U.S. exceeding $3 billion. Q3 2023 was also the second strongest revenue quarter for JELMYTO and low-grade upper tract urothelial carcinoma. We're pleased with the pace of growth for JELMYTO, especially when considering this is a rare disease treated in both community and academic centers. We will continue to drive growth and meaningful adoption through increasing sites of care and leveraging the growing body of real-world data, highlighting JELMYTO's meaningful value, and as part of a multimodal kidney-sparing approach to disease management. For the third quarter, we reported $20.9 million in JELMYTO net revenues, an increase of 30% year-over-year. We believe there remains a significant growth opportunity for JELMYTO as the first medicine ever approved for low-grade UTUC, bringing a differentiated chemoablative approach to patients. The closing of our $120 million private placement during the third quarter was an important milestone that significantly strengthened our balance sheet. Given our fortified financial position, we are committed to deploying capital that maximizes shareholder value and plan to utilize proceeds from the raise to develop and execute a comprehensive pre-commercialization and launch strategy for UGN-102, while continuing to grow JELMYTO sales. Importantly, based on our latest financial forecast, we believe our current cash position will support our commercial organization through the prospective launch of UGN-102. Q3 2023 was a transformative quarter for UroGen following strong top-line data from ATLAS and ENVISION and our pre-NDA meeting with the FDA, we have a clear path forward towards an approval for UGN-102 in low-grade intermediate-risk nonmuscle-invasive bladder cancer. Now, JELMYTO continues to grow its footprint in low-grade UTUC. I'm very proud of the dedication and commitment across our organization as we remain focused on pioneering a new era in urologic and specialty cancer care. UroGen is at its strongest and most encouraging point in the company's history. I'll now pass the call to Mark, who will provide a clinical update.

Thank you, Liz, and hello, everyone. I'd like to take a moment to briefly summarize top-line results from the ATLAS and ENVISION trials before commenting on our recent pre-NDA meeting with the FDA. As a reminder, ATLAS was an open-label randomized controlled Phase III study designed to evaluate UGN-102 with or without TURBT versus TURBT alone. The trial enrolled 282 new and low-grade intermediate risk NMIBC patients. ATLAS met its primary endpoint of disease-free survival of UGN-102, demonstrating superiority to TURBT with a 55% reduction of risk for recurrence, progression, or death in patients who received UGN-102. UGN-102 also showed a 65% complete response rate at 3 months for patients who only received UGN-102 compared to a 64% complete response rate at 3 months for patients who only received TURBT. When we evaluate the subgroup of patients in ATLAS with recurrent disease, and a history of at least one prior TURBT, the observed duration of response in the UGN-102 treatment group was a resounding 66.3%, 12 months after achieving a complete response or 15 months post-randomization. This is in comparison to a 40% duration of response observed in the TURBT arm at the same time point. These results offer compelling insight into the effect of UGN-102 in recurrent patients, which is the population studied in our pivotal trial, ENVISION. During our recent pre-NDA meeting, the FDA reaffirmed that ENVISION will serve as the pivotal trial for UGN-102’s NDA. ENVISION, which is a single-arm study of UGN-102, enrolled 242 recurrent low-grade intermediate risk NMIBC patients with a history of at least one prior TURBT. The study met its primary endpoint, demonstrating that patients treated with UGN-102 experienced a 79% complete response rate at 3 months following initiation of treatment. When looking at the totality of clinical data thus far, UGN-102 has demonstrated consistency in the 3-month complete response endpoints across all 3 trials. A consistent and durable response endpoint in ATLAS and OPTIMA, demonstrating a compelling therapeutic and safety profile throughout. For ENVISION, we maintain our view that the rate of duration of response is critical for obtaining meaningful outcomes in this patient population. Given the consistency and the durability endpoints from ATLAS and OPTIMA, we anticipate potential similar outcomes for ENVISION, which we believe supports the approval in low-grade intermediate risk NMIBC. Before turning the call over to Jeff for a commercial update, I'd like to briefly comment on the recently reported clinical data in bladder cancer from several of our peers. As a company, UroGen's mission is to build novel solutions to treat urothelial and specialty cancers because patients deserve better. We recognize the need for innovation in the development of new therapies in our space. Thus, we are encouraged that there are programs in development that may offer patients potentially better options than the current standard of care. However, as we near the final stages of clinical development for UGN-102 and with the prospect of commercialization on the horizon, we are discovering how the significant distinctions between low-grade and high-grade NMIBC and even metastatic bladder cancer may not be widely recognized. Low-grade NMIBC, high-grade NMIBC, and metastatic bladder cancer are distinct types of bladder cancer with significant differences. Low-grade NMIBC is characterized by less aggressive tumors limited to the lining of the bladder and typically carries a better prognosis. High-grade disease, on the other hand, consists of more aggressive cancer cells within the bladder lining, which may have a higher risk of recurrence and progression. In contrast, metastatic bladder cancer represents the most advanced stage where cancer has spread to distant organs, carrying a poorer prognosis and necessitating systemic treatments. The key distinctions lie in tumor aggressiveness, location, treatment approaches, prognosis, and the stage of disease. It's important to understand that UGN-102 is focused on low-grade intermediate risk NMIBC, where the competitive landscape is much less densely populated than in high-grade disease or metastatic disease. Therefore, recent data releases do not impact our current development nor commercial plans nor are expected to encroach on what we believe is a significant market opportunity for this program. We are hopeful that UGN-102 may potentially serve as the first nonsurgical therapy for this indication, which represents a sizable portion of bladder cancer cases each year and is also characterized by a high rate of recurrence. If approved, UGN-102 has the potential to shift the standard of care away from repetitive surgical care and may improve the quality of life for tens of thousands of individuals battling this highly recurrent disease. With that, I'd like to turn the call over to Jeff for a commercial update.

Thanks, Mark. Q3 was another strong quarter for JELMYTO. We had the second strongest quarter in our history with continued momentum in our underlying business. We saw a small decrease from the prior quarter due to typical summer seasonality and continue to see strong double-digit year-over-year growth in JELMYTO sales in what is our third full year of commercialization. JELMYTO net sales for the third quarter were $20.9 million, which represents 30% growth from the same period last year. This growth in our top line reinforces our long-term belief in the low-grade UTUC opportunity. During the third quarter, further strengthening of the JELMYTO ramp and expansion of the JELMYTO user base was the result of several key factors, including continued commercial execution. JELMYTO offers clinical utility alone or following endoscopic management as part of a kidney-sparing treatment regime. The meaningful differentiated treatment profile of JELMYTO and its unique feature of being the only FDA-approved nonsurgical treatment indicated for low-grade UTUC continues to resonate with both patients and physicians. Additionally, the growing body of data from real-world evidence studies continues to strengthen and reinforce JELMYTO's value proposition, supporting its multimodal use across various practice patterns and diverse presentations. Our experience with JELMYTO has given us a foundation with our established use of mitomycin and RTGel. Consistent growth and adoption for this product reinforces our optimism for the significant opportunity in low-grade intermediate-risk nonmuscle-invasive bladder cancer with UGN-102. However, UGN-102 offers several distinct advantages over JELMYTO, including simpler administration and a much lighter operational lift. Delivery of UGN-102, if approved, is expected to be easier for urologists given that it does not require the use of specialized equipment, scheduled time in the OR, will be delivered pre-mixed with an anticipated one-week shelf life, and can be given by a doctor or support staff in a clinic as an outpatient procedure. Importantly, we believe that the reimbursement economics for UGN-102, relative to TURBT, will not be a barrier to adoption. Following the positive ATLAS and ENVISION data, we began executing our pre-commercialization plan in preparation for a prospective UGN-102 launch. With approximately 95% overlap in prescriber base and well-established practice patterns, we expect the seamless integration of UGN-102 into our commercial organization and an expedient launch upon approval. If approved, we anticipate that UGN-102 will be the first-ever nonsurgical treatment option for a disease affecting approximately 82,000 patients in the U.S. each year, with a total market of more than $3 billion in the U.S.

Thank you, Jeff, and thank you to everyone for joining today's call. I'm pleased to review our financial results for the third quarter ended September 30, 2023. We are pleased to report another strong quarter of year-over-year revenue growth. For the third quarter of 2023, we reported JELMYTO net product revenues of $20.9 million, an increase of approximately 30% compared to $16.1 million in the same period last year. For the third quarter of 2023, research and development expenses were $10.2 million as compared to $13.1 million for the same period in 2022. The overall decrease is primarily due to lower expenses related to the conclusion of the ATLAS trial and lower costs of the ENVISION trial for UGN-102, offset by higher R&D expenses related to the Phase I study for UGN-301, and ingredient scale-up and production for UGN-102. Selling, general and administrative expenses for the third quarter of 2023 were $21.8 million compared to $19.1 million for the same period in 2022. The increase in SG&A is primarily due to higher marketing, commercial operations, professional services, and training, offset by lower commercial back-office services and support expenses. UroGen reported a noncash financing expense related to the prepaid forward obligation to RTW Investments of $5.5 million for the third quarter of 2023, compared to $4.8 million for the same period in 2022. Interest expense related to the $100 million term loan facility, with the funds managed by Pharmakon Advisors, was $3.8 million for the third quarter of 2023 compared to $2.7 million for the same period last year. UroGen reported a net loss of $21.9 million or a basic and diluted net loss per ordinary share of $0.68 for the third quarter of 2023, as compared to $25.8 million or a basic and diluted net loss per ordinary share of $1.13 for the same period in 2022. Turning to forward guidance. We reiterate anticipated full year 2023 net product revenues from JELMYTO to be in the range of $76 million to $86 million. We also reiterate the full year 2023 operating expenses to be in the range of $135 million to $145 million. The company anticipates a full year 2023 noncash financing expense related to the prepaid forward obligation to RTW Investments in the range of $21 million to $26 million. Of this amount, approximately $9.9 million to $11.2 million is expected to be in cash. We ended the third quarter with $153.9 million in cash and cash equivalents and marketable securities, which includes proceeds from the $120 million private placement, which closed during the third quarter.

Just a few for me. I want to ask Mark, in the FDA meeting, did you have any discussion around what range of durability from ENVISION the agency would like to see when you have this data?

Leland, thanks for the question. As I think we've discussed previously, our interactions with the FDA indicated that they are interested in, and we are going to provide a totality of the data regarding our data sets. And so no specific numerical bar has been discussed as a bar for approval. But it will be the entirety of clinical meaning, the outcome of these trials that will inform decision regarding approval.

Okay. Regarding the ATLAS data, can you share your perspective on whether they will ultimately be categorized into one or two labels at this time? How much of this is unclear?

Yes. But Leland. Nice to talk to you. Absolutely, the ATLAS data will be in the label and so yes, we believe that the agency and the meeting talked about that data is supportive of our filing. So yes, we expect that to be in the filing, and we expect it to be able to use that data externally.

And just one last question for me. As Liz, we've talked in the past about where you've been doing with respect to lengthening your interest in property runway. Wondering if you might have any update on protections for -102.

Yes. No. Specifically, except to tell you that you'll hear more in the very near future. And we are on track to do that. As what we've stated before is minimally 2035, but we actually believe it will be 2041. So we're working through final details, but no show stoppers, looks really great, and we hope to provide an update very shortly.

Congrats on all the progress recently. I just wanted to get an update on where you folks think you are operationally in terms of identifying any potential efficiencies or cost reductions? And if you think that in particular on the G&A, you might see some additional evidence of that being realized over the course of the coming quarters? Or if you think at this juncture, you've kind of reached optimum operating efficiency?

Yes, that's a great question. To be honest, we are preparing for the launch of a new medicine that is expected to be a major success. Therefore, we are not planning to reduce expenses at this time. We are operating efficiently and currently reviewing all of our operational plans. The key consideration is how much we need to grow for UGN-102 while leveraging our existing organization and shifting some focus from JELMYTO to UGN-102, which presents a broader opportunity for our patients. I don't anticipate significant expense reductions in any areas. For instance, in R&D, we will see some reductions as the ENVISION and ATLAS studies conclude. However, as we've mentioned before, to extend the patent life, we'll be adding a study but at a slower pace. We will likely experience some decreases and efficiencies, but we are not targeting major overall cost reductions. We are committed to maintaining efficiency and are challenging our internal team, especially regarding infrastructure, to ensure we only add what's necessary and shift resources whenever possible. We are also reviewing our inventory levels to determine what we really need. Additionally, in our operational expenses, we are focusing on securing our supply chains and ensuring we have reliable secondary suppliers to prevent any disruptions, which does involve some incremental expenses, though they are not substantial. However, we want to avoid any supply issues.

Okay. And then just a bifurcated question regarding the earlier-stage pipeline and potential additions to the portfolio. If you look at 2024, can you give us a sense of what you expect to be the most important earlier-stage pipeline developments that you're anticipating over the course of next year, particularly as these pertain to zalifrelimab? And also any other potential pipeline programs that you anticipate would likely see notable advancement over the course of 2024 with the exception, of course, of UGN-102, which I think is very much at the forefront of people's minds? And if you could also give a sense of whether you are looking to add anything to broaden the portfolio within oncology via strategic licensing or M&A.

Yes, thanks. So we are advancing the Phase I monotherapy program for zalifrelimab, the anti-CTLA-4 antibody for intravascular treatment of high-grade disease. And as we've discussed previously, this is part of a master protocol that will permit us to study combinations with the antibody. We are already in the process of creating those components of the trial. So we would expect next year to be able to talk about our monotherapy experience and update you on how our combination work is going, particularly with our TLR7 agonist as well as potentially with other drugs of interest such as gemcitabine.

Yes. To address the second question, we are evaluating various products in the market that we would like to integrate with zalifrelimab and our technology. We are actively engaging in discussions with external companies about this, although nothing conclusive has emerged yet. However, it is a priority for us. We are keen to incorporate something in this area, which is currently very dynamic with activity from both large pharmaceutical and biotech companies. It's unlikely that we will initiate this in 2024, but we are open to it in 2025. It’s important to note that this business is highly leverageable. As we expand into uro-oncology, we can add multiple products to our portfolio without significantly increasing our infrastructure. Therefore, one or two years after UGN-102, we expect a considerable enhancement to our bottom line, providing us with the necessary resources to continue growing the company in the long term. Additionally, we are interested in advancing UGN-102 for high-grade disease and for patients who are unwilling or unable to participate in broader low-grade studies. There are many opportunities we will explore moving forward. I would also like to introduce Mike Louie, our new Vice President of Medical Affairs and Clinical Development, who is leading efforts on UGN-102 and looking into a registry for it. Similar to our work with JELMYTO, we are seeing promising data from that registry, which we plan to share, leading to more publications in 2024.

Similar questions for me. First, from the FDA discussion you've had, did they specifically say why they wouldn't accept ATLAS as a pivotal trial to support approval, just given the very strong hazard ratio and just efficacy out of it, that that study has reported?

Yes. Mark, do you want to answer or you want me to?

I'll start out and Liz will undoubtedly comment. I think we were planning to enroll a larger number of patients in the ATLAS trial, substantially larger than we ultimately enrolled. The data are very strong but I think the fact that we halted enrollment would affect the trial with a smaller number of patients than originally anticipated in the original statistical analysis plan, probably informed some of the FDA's position on the aggregate value of the ATLAS data as a stand-alone submission. But Liz may want to comment as well.

No, I think that's right. The data is very compelling. Their initial stance on the data was exploratory, but we made it clear that it is significant data. They ultimately came around to that perspective. However, considering the prospective statistical analysis plan, we didn't meet the necessary endpoints for it to be classified as the pivotal study. They provided very positive feedback. The meeting with them was likely the most positive they have ever been, and they were very impressed with the data. There was no pushback on ENVISION alone; they indicated a desire to see durability, which they have emphasized not just to us, but to others in the field. It's a recurring theme in discussions about bladder cancer. The main issue was related to the prospective analysis plan and the absence of the required data. Nonetheless, the interaction was very positive. They have consistently expressed the importance of durability alongside complete response. We attempted to persuade them, believing our data was compelling, but we also recognized there was a likelihood they would request the ENVISION durability data, which is exactly what they did. We feel we are in a strong position with them, understanding their needs and timeline, and we are working towards that.

Great. And my last question here. You mentioned reimbursement economics that the -102 is more favorable than TURBT. Can you just talk about what those reimbursement economics are in various settings, maybe in academic centers versus private practices or however you want to divide that?

Yes, sure. Jeff, we'll take you through that.

Depending on the size of the tumor for TURBT, the reimbursement amount to the physician is not significant. There may be a perception that the reimbursement is larger than it actually is. However, if you analyze the reimbursement economics, particularly when considering the inclusion of an anti-carcinogenic like UGN-102 and the buy-and-bill component of the drug, which will primarily be administered in the clinic, the financial outlook appears quite favorable compared to TURBT. It's important for people to recognize that reimbursements for surgeries have decreased, which we observe in the context of TURBT, and obviously, this is influenced by the tumor's size. Overall, we anticipate that UGN-102 will have better reimbursement than TURBT.

Yes. And then look, the only thing I'll comment about is we are not going out with a profitability message, right? That's not our message. We can't do that. We won't do that but it will be very right. It will be very important to them that they don't feel like they're losing money that is not a detriment to them, even though we know it's better medicine for the patient, which we will focus on. But we do have field reimbursement managers. We will make sure that our reps are also armed and able to share the information that we can appropriately share because it is an important piece, as you know. Unfortunately, decisions get made that way. But this will be, frankly, a net positive if they did from a financial standpoint. But keep in mind, obviously, our role in how we discuss that to the marketplace.

Congrats on the good quarter. I was just wondering for Mark, if you could maybe share any additional feedback you've had from the physician community since Investor Day, specifically since the Society of Urologic Oncology is coming up here, any additional feedback you've gotten on ENVISION and ATLAS?

Paul, thanks for the question. I may also ask Jeff to provide some insights on this. I believe there is excitement about the potential approval of UGN-102. Everyone, particularly those involved in urologic oncology, recognizes the need and the gaps within that population. What I'm hearing from my colleagues is a positive anticipation. Jeff, do you have anything else to add?

You want to talk about what some of the feedback you got at LUGPA?

Sure. So we just got back, Paul, from LUGPA's Annual Meeting, the larger groups. We had an advisory board there, where Dr. Prasad presented some of the data that we saw out of day. And I think the initial reaction was overwhelmingly positive. It was designed to sort of get their feedback in and around the data. We workshopped a couple of different patient types, but they're eager to have a different option. If you on the conservative side of things, they certainly have a number of patients that they do not want to put under general anesthesia again and have a surgery. So there's that. And then many of them reiterated that essentially what looks like at least in ATLAS, it's the same for TURBT and it's a much longer duration of response, which is obviously a key attribute, if approved, for UGN-102. So that was 12, 13 advisers, very influential in the community that gave us really strong feedback. We'll continue to obviously engage as we need that feedback, but that was just an initial reaction.

And as a follow-up also for you, Jeff. Just regarding your comments on the buy-and-bill process. Can you maybe just give us some sense of what you think the runway will be before reimbursement? And J-Codes and so forth are established for -102 and just how you think the early mechanics might look like starting with the launch in 2025?

Sure. As with any Part B drug, there will be a miscellaneous code for some time. We had this with JELMYTO, and we expect the same with 102. Handling a miscellaneous code is a manual process, so we need to ensure that the office correctly fills everything out associated with that code. I can say that this will be clearer than JELMYTO since, with JELMYTO, you had to bill for both drug and waste, requiring a miscellaneous drug code and a miscellaneous waste code. In this case, there will be fewer line items, making it much more straightforward. We will implement measures to alleviate concerns regarding the miscellaneous code. If approved in the anticipated timeline, the positive aspect is that CMS is now reviewing miscellaneous codes more frequently than once a year. We hope to secure the J-Code sooner due to this review process. However, it’s important to remember that handling a miscellaneous code requires a bit more time from the physician's practice. Early adopters will emerge and hopefully be reimbursed accurately, which will allow them to share their experiences with peers. By that time, we expect to have a permanent J-Code established.

And our first question comes from Leland Gershell from Oppenheimer. That concludes today's conference call. Thank you for participating. You may now disconnect.