Urogen Pharma (NASDAQ:URGN) President and CEO Liz Barrett outlined the company’s urothelial cancer focus and provided an update on the commercial launch of its recently approved bladder cancer therapy during a fireside chat.
RTGel platform and two commercial products
Barrett said UroGen was founded in Israel to address an unmet need in treating urothelial cancers, where medicines delivered to the urothelium are often rapidly voided due to urine flow. The company’s RTGel platform—short for reverse thermal gel—is liquid when cold and turns into a gel at body temperature, creating a depot that can sustain-release medicine over several hours.
Unmet need and target population for ZUSDURI
Barrett described low-grade intermediate-risk NMIBC as a disease characterized by recurrence rather than progression. Patients are considered intermediate-risk within the low-grade category based on factors such as multifocal disease, larger tumors, or a history of recurrence. She emphasized that while patients generally do not die from low-grade intermediate-risk disease and only a small percentage progress to high-grade NMIBC, tumors must still be treated.
According to Barrett, the longstanding standard approach has been surgical removal via transurethral resection of the bladder tumor (TURBT), followed by surveillance. She said about 50% of patients recur within the first year after TURBT, creating a cycle of repeated procedures and ongoing recurrence. Barrett positioned ZUSDURI as the first non-surgical treatment option in this setting.
On market size, Barrett said there are about 20,000 “recurrent newly diagnosed” patients, but a larger prevalent pool drives roughly 60,000 low-grade intermediate-risk patients who recur in any given year in the U.S. She said UroGen’s market focus is patients with low-grade intermediate-risk NMIBC who have had at least one recurrence. She added that 68% of these patients have had two or more recurrences, and 23% have had five or more recurrences.
Launch dynamics, J-code, and early physician experience
Barrett discussed early commercial metrics and operational factors affecting launch timing, noting ZUSDURI is administered as a procedure rather than as a pill or infusion. She said there can be a 45-60 day interval between identifying a patient and initiating treatment, reflecting factors such as pharmacy and distribution logistics and buy-and-bill dynamics.
In discussing the early launch period, Barrett referenced UroGen’s third-quarter report, when ZUSDURI revenue was about $1.8 million, and said the company shared October revenue of $4.5 million to illustrate early momentum. She said UroGen expected November and December to be similar to October, given holidays and year-end timing, and noted a permanent J-code became effective January 1.
On reimbursement, Barrett said UroGen had received 100% reimbursement even before the permanent J-code and had not seen denials. However, she said providers had more trepidation and administrative burden before the permanent code, and she expects processes to move faster now that it is in place.
Barrett said the company is seeing an “acceleration” in uptake since the J-code became effective. She described tracking launch progress through patient enrollment forms, conversion to new patient starts, and doses used, and said the company is on track with expectations.
She also described early patient segments where physicians appear to be adopting ZUSDURI, based on anecdotal feedback:
- Patients with multiple recurrences, including those for whom repeated TURBTs have not been effective
- Patients who recur quickly
- Elderly patients (median age at diagnosis cited as 74) and those with comorbidities who want to avoid general anesthesia and repeat operating room procedures
Barrett estimated roughly one-third of the 60,000 recurrent patients fall into these higher unmet-need categories, noting the groups are not mutually exclusive.
Clinical data and positioning versus surgery-based approaches
Barrett said physicians have been enthusiastic about having an alternative to repeated TURBT and pointed to clinical results, including an 80% complete response rate and 80% of those patients still in response at 12 months. She also referenced patient-reported outcomes conducted through the University of North Carolina, saying 90% of patients surveyed preferred UroGen’s treatment over TURBT.
On longer-term durability, Barrett said UroGen has reported that 72% of patients were still in complete response at two years, with median duration of response not reached, implying it extends beyond two years. She contrasted this with the recurrence profile she cited for TURBT, where about half of patients recur within one year.
Barrett said UroGen expects “almost all” patients to receive all six doses of ZUSDURI, and described most discussions with providers as operational and financial rather than clinical. She also highlighted that ZUSDURI is intended as a primary chemoablative therapy that does not require surgery, and argued the six-week treatment course and lack of maintenance can be advantageous compared with approaches that combine surgery with ongoing maintenance therapy.
Commercial outlook and pipeline priorities
Asked about peak sales potential, Barrett said the company believes ZUSDURI can be a “billion-plus” medicine, citing a net price of about $100,000 and estimating that 20% penetration of the 60,000-patient market would imply approximately $1.2 billion in peak sales. She also said UroGen does not plan to provide ZUSDURI revenue guidance yet, explaining the company similarly did not provide guidance at the launch of Jelmyto and wants additional quarters of performance before doing so.
Barrett said the FDA post-marketing commitment for ZUSDURI involves following patients for five years and providing annual updates on durability.
On the pipeline, Barrett discussed UGN-103 as a next-generation formulation and said the company has reported complete response data from the UTOPIA study and is awaiting durability data, with an initial cut expected around mid-year. She said UroGen has spoken with the FDA and plans to file in the second half of the year, with the agency allowing submission before every patient reaches 12 months as long as the company updates the filing during review. Barrett outlined a timeline of potential approval in 2027 and launch in 2028, and said UGN-103 has patent protection through 2041.
Barrett added that UroGen plans to move UGN-103 into additional settings, including high-grade disease and adjuvant use, and said the company is preparing study protocols and expects to start at least one, and possibly two, studies in the second half of this year.
For other programs, Barrett said UGN-104 is a next-generation formulation for UTUC, with enrollment ongoing and expected to complete this year, following a strategy similar to UGN-103. She also described UGN-501 as an oncolytic virus candidate that the company believes could be “best-in-class,” noting it is intended to elicit an immune response and provide direct cell kill. Barrett said UroGen initially plans to study UGN-501 in bladder cancer, with potential to combine it with RTGel for longer exposure, and said the program could eventually extend to cancers outside urothelial disease.
Finally, on Jelmyto, Barrett said the company continues to see single-digit growth and more patients and new treaters. She also described potential commercial synergies from an expense standpoint and suggested that greater physician familiarity with ZUSDURI could lead to increased Jelmyto use, although she said the company has not yet seen that effect. Barrett said UroGen expanded its sales coverage from about 5,500 doctors to 8,500 doctors, which she believes could help support continued growth for Jelmyto and future products such as UGN-104.
About Urogen Pharma (NASDAQ:URGN)
UroGen Pharma is a clinical-stage biopharmaceutical company focused on developing and commercializing novel treatments for uro-oncology and uro-genital diseases. Founded in 2010 and headquartered in Ra’anana, Israel, with offices in New York, UroGen applies its proprietary RTGel® reverse thermal gel delivery platform to create sustained-release formulations designed for in-office use by urologists.
The company’s lead product, Jelmyto® (mitomycin gel), received U.S. Food and Drug Administration approval in 2020 for the treatment of adults with low-grade upper tract urothelial cancer.
