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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): January 14, 2025
UROGEN PHARMA LTD.
(Exact name of registrant as specified in its charter)
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| Israel |
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001-38079 |
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98-1460746 |
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(Commission File Number) |
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(IRS Employer Identification No.) |
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| 400 Alexander Park Drive, 4th Floor Princeton, New Jersey |
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08540 |
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(Zip Code) |
Registrant’s telephone number, including area code: +1 (646) 768-9780
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligations of the registrant under any of the following provisions:
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Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
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Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
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Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
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Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
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Trading
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Name of each exchange on which registered |
| Ordinary Shares, par value NIS0.01 per share |
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URGN |
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The Nasdaq Stock Market LLC |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
| Item 7.01 |
Regulation FD Disclosure. |
Furnished as Exhibit 99.1 to this report is a presentation of UroGen Pharma Ltd. (the “Company”), all or a portion of which may be used by the Company in meetings with investors, analysts and others.
The information in this Item 7.01, including Exhibit 99.1, is being furnished and shall not be deemed “filed” for the purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that Section, nor shall it be deemed incorporated by reference in any filing made by the Company under the Securities Act or the Exchange Act, except as shall be expressly set forth by specific reference in such a filing.
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Financial Statements and Exhibits. |
(d)
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
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| Date: January 14, 2025 |
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UROGEN PHARMA LTD. |
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By: |
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/s/ Chris Degnan |
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Chris Degnan |
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Chief Financial Officer |
Exhibit 99.1 Developing Innovative Medicines to Treat Urothelial Cancers
January 2025
Disclaimers This investor presentation contains forward-looking
statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation: the potential of UroGen’s proprietary technology to enhance proven and novel medicines and deliver them aligned with the
way urologists practice; the estimated addressable patient population and market and revenue opportunity for JELMYTO in LG-UTUC, UGN-102 and UGN-103 in LG-IR-NMIBC, and UGN-301 in HG-NMIBC; the potential of UroGen’s proprietary RTGel®
technology platform to improve therapeutic effects of existing products; the expectations regarding the continued growth of JELMYTO revenue; the potential of JELMYTO® and UGN-104, UGN-102 and UGN-103, and UGN-301 to transform the treatment
paradigm in LG-UTUC, LG-IR-NMIBC, and HG-NMIBC, respectively; the potential that JELMYTO and UGN-102, if approved, are adopted as a standard of care; UroGen’s pipeline supporting long-term sustainable growth; the interpretation and summary of
results of OLYMPUS Phase 3, OPTIMA Phase 2b, ATLAS, and ENVISION trials; the potential of UGN-102, including to be the first FDA approved medicine for LG-IR-NMIBC and to set the new standard of care for LG-IR-NMIBC; the potential advantages of
UGN-102 over TURBT; the expected timing for ODAC and PDUFA target action date for UGN-102; the potential launch of UGN-102, increasing adoption of JELMYTO, if approved; the potential of UGN-301 to expand to Immuno-Oncology with potential monotherapy
and combination therapy; the ongoing and planned clinical studies for UGN-301; the potential benefits of and expected patent protection for UGN- 103 and UGN-104; the ongoing Phase 3 UTOPIA study of UGN-103 in LG-IR-NMIBC; UroGen’s plans for
the future including initiating Phase 3 studies to evaluate UGN-104 in LG-UTUC, and the timing thereof, the expansion of the JELMYTO uTRACT registry, publishing OLYMPUS LTFU data, supporting pilot investigator-initiated study of JELMYTO in HG-UTUC
and UroGen’s field organization size, positions and responsibilities; UroGen’s priorities including advancing pre-commercial and launch activities for UGN-102; focusing on strategic and efficient capital deployment, extending
UroGen’s leadership in addressing unmet needs in Urothelial cancers and building a long-term sustainable growth business; and UroGen’s ability to draw down the remaining $75M under its credit facility. These statements are subject to a
number of risks, uncertainties and assumptions, including, but not limited to: there is no guarantee that the NDA will be sufficient to support approval of UGN-102 by the target PDUFA date of June 13, 2025, or at all; UroGen’s pending patent
applications, may not be successful and in such event the duration of its intellectual property protection would be more limited; the timing and success of clinical trials and potential safety or other complications encountered therein; results from
prior or ongoing clinical trials and the real-world retrospective studies of JELMYTO may not be indicative of results that may be observed in the future; unforeseen delays that may impact the timing of progressing clinical trials and reporting data;
the ability to obtain regulatory approval within the timeframe expected, or at all; the ability to maintain regulatory approval; complications associated with product development and commercialization activities; the labeling and packaging for any
approved product; the scope, progress and expansion of developing and commercializing UroGen’s product and product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis
alternative therapies; RTGel technology may not perform as expected and UroGen may not successfully develop and receive regulatory approval of any product candidate beyond JELMYTO that incorporates its RTGel technology; UroGen’s financial
condition and need for additional capital; UroGen’s inability to meet the closing conditions required to draw down additional funds under its credit facility; the impacts of macroeconomic and geopolitical conditions, high inflation, and
uncertain credit and financial markets on UroGen’s business, clinical trials and financial position; and UroGen’s ability to attract or retain key management, members of the board of directors and personnel. In light of these risks and
uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen’s Form 10-Q for the quarter ended September 30, 2024, filed with the Securities and Exchange Commission (SEC) on November 6, 2024, and
other filings that UroGen makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and UroGen’s actual results could differ
materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this presentation and are based on information available to UroGen as of the date of this presentation. 2
Investment Highlights UroGen is pioneering new therapies to meet the
unique needs of patients with urothelial cancers by utilizing proprietary technology with the potential to enhance proven and novel medicines and deliver them aligned with the way urologists practice Commercial Late-Stage Strong Balance
Immuno-Oncology Product Clinical Asset Sheet Pipeline JELMYTO is the first UGN-102 being developed UGN-301 is an anti-CTLA $254.2 million in cash, and only FDA-approved as a minimally invasive, 4 monoclonal antibody for cash equivalents and
non-surgical treatment monotherapy and non-surgical option that marketable securities at combination intravesical for patients with LG- has the potential to set the September 30, 2024. solution for use in high UTUC. new standard of care for grade
NMIBC. LG-IR-NMIBC. Target PDUFA of June 13, 2025. 10x larger potential patient 1 population than LG-UTUC . 1. ACS Cancer Facts & Figures 2023; SEER, AUA/SUO joint guideline; Babjuk et al. European Urology (2019), Simon (2019) 3
Invasive and Radical Surgery Is the Standard of Care in Urothelial
Cancers Urothelial cancers are challenging to treat: Resulting in: Intolerance of foreign Anatomical Repetitive risky surgeries materials barriers in the urinary tract Lost kidneys and organs Increased risk of morbidity The urinary tract is designed
to void, which poses challenges including limited dwell time for chemotherapies and other in elderly patients therapies delivered to the bladder. 4
RTGel® Proprietary Reverse-Thermal Hydrogel Technology Uniquely
Designed to Allow for Local Delivery of Medicines Increases dwell time and exposure to active drugs Potentially improves the therapeutic effects of ® RTGel exists as a liquid existing products at lower temperatures and converts to gel form at
body temperature. Leverages physiologic flow of urine to provide natural exit from the body 5
Unlocking a Strong Foundational Pipeline Supporting Long-Term
Sustainable Growth JELMYTO/UGN-104 UGN-102/UGN-103 UGN-301 Phase 3 Phase 1 ~82K 4,6 ~6K–7K ~18.7K addressable U.S. 1,2 addressable U.S. population addressable U.S. population 3,4,5 population $700M $2B+ 3,4,5 $5B+ Potential Market 1,2
Potential Market Potential 4,6 Market Low-Grade Intermediate Risk Non-Muscle Invasive Bladder Low-Grade Upper Tract Urothelial High-Grade Non-Muscle Cancer (LG-IR-NMIBC) Carcinoma (LG-UTUC) Invasive Bladder Cancer (HG-NMIBC) 1. Upfill-Brown 2018, 2.
Cutress 2012 3. ACS Cancer Facts & Figures 2023 4. SEER, AUA/SUO joint guideline 5. Babjuk et al. European Urology (2019), Simon et al (2019) PLoS ONE 14(2): e0211721 6 6. UroGen commissioned third party assessment (Lion Healthcare Strategies,
Ambaw)
Changing the Treatment Paradigm for Urothelial Cancers (UGN-101)
JELMYTO
LG-UTUC Is a Rare Disease that Recurs Often 6,000-7,000 2,800-3,200
3,000-4,000 ELIGIBLE PATIENTS IN THE U.S. 1 2 ANNUALLY, INCLUDES: Newly Diagnosed Recurrent Patients Treatment Options Treatment Options RNU RNU Endoscopic Management Additional Endoscopic Management UC is the costliest cancer in the U.S. healthcare
system 50%-80% of LG-UTUC patients ultimately 4 1,3 on a per-patient basis receive nephroureterectomies 8 1. Upfill-Brown 2018 2. Cutress et al. (2012 BJU International 2012) 3. Kohut R, Zhu H. JUrol May 2012, Hui Zhu 4. Yeung et al. (2014)
Pharmacoeconomics 5. RNU = radical nephroureterectomy 8 C O N F I D E N T I A L
JELMYTO First and Only FDA-Approved Non- Surgical Treatment for Patients
with LG-UTUC 1 Clinically Meaningful OLYMPUS Phase 3 Data 2 Complete Response Rate at 3 months 58% 2 Durability of Response at 12 months by KM estimate 82% Median Duration of Response (95%CI: 13.1, 57.5; n=41) 3,4 47.8 months data from long-term
OLYMPUS follow-up study 1. Important Safety Information and the full Prescribing Information available at https://www.urogen.com/download/pdf/jelmyto_prescribing.pdf 2. Matin, Surena F. J Urol. 2022 Apr;207(4):779-778 3. UroGen Data on File:
Post-hoc analysis from the OLYMPUS trial that evaluated the long-term efficacy of JELMYTO in patients who experienced a CR 4. Limitations of long-term follow-up study include patient population N=41. Amongst the 41 patients followed after initial
complete response at 3-months median duration of response was 47.8 months (95% CI 9 9 13.0, not estimable) (median follow-up 28.1 months (95% CI: 13.1, 57.5)). Please refer to the referenced citations disclosures of such limitations.
Growing Body of Real-World Evidence Supports Use Case For JELMYTO* Data
from 2+ years in market reinforces JELMYTO efficacy and safety Select Results Independent multicenter reviews support JELMYTO real-world effectiveness, including as a chemoablative agent and treatment 69% 23% of residual disease following endoscopic
resection Ureteric CR Stenosis When JELMYTO As compared to 44% Evaluated outcomes in range of tumor types; evidence for treated residual in OLYMPUS. ~1/2 of favorable response in patients with low-volume residual disease following laser patients
were treated ablation (overall CR with antegrade disease 58% in OLYMPUS). administration. Woldu, et al. Early Experience with UGN-101 for the Varied practice patterns, with antegrade method of Treatment of Upper Tract Urothelial Cancer – A
MultiCenter Evaluation of Practice Patterns and Outcomes. Urol Oncol. administration via nephrostomy tube shown as viable *Real world retrospective studies have inherent evidentiary limitations. Please refer to the referenced citations for
disclosures of 10 such limitations.
JELMYTO Retrospective Analysis Results Presented at AUA 2024* JELMYTO
treatment demonstrates favorable Recurrence Free Survival (RFS) rates for patients with LG-UTUC who 1 respond to initial induction Additional Insights No differences in RFS were 1,2 observed regarding : 86% RFS at 24-months for LG-UTUC patients who
were ✓ Usage of 1 complete responders to induction therapy chemoablation vs. post- endoscopic resection ✓ Tumor size 100% ✓ Multifocality RFS at 24-months in patients who received maintenance 2 therapy of JELMYTO, compared to
61% in those who did not ✓ Tumor location 1.Woldu et al. Exploring Recurrence After Initial Response to UGN-101 Induction in Expanded Settings. AUA 2024 Presentation 2.Woldu et al. Longitudinal Follow Up of Multicenter Study of UGN-101 for
Upper Tract Urothelial Cancer. AUA 2024 11 Presentation
JELMYTO Revenue Trend Reflects Continued Growth Annual JELMYTO WW
Revenues ($MM) Quarterly JELMYTO WW Revenues ($MM) $30 $100.0 +28% $25.2 $82.7 $25 $23.5 $80.0 $21.8 +34% $21.1 $20.9 $64.5 $20 $18.8 $18.2 $17.2 $60.0 $16.6 $16.2 $16.1 $48.1 $15 $13.6 $13.0 $11.4 $40.0 $10 $8.0 $7.5 $20.0 $5 $3.5 $11.9 $0.4 $0 $-
2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 2020 2021 2022 2023 2020 2020 2020 2021 2021 2021 2021 2022 2022 2022 2022 2023 2023 2023 2023 2024 2024 2024 12
Changing the Treatment Paradigm for Urothelial Cancers
UGN-102
UGN-102: The First Potential Breakthrough Localized Therapy 1,2 For
Patients with LG-IR-NMIBC in Over 30 Years UGN-102 represents a new approach specifically for LG-IR-NMIBC, 1 with strong efficacy and safety data in the ENVISION phase 3 trial 1 Innovative reverse-thermal hydrogel containing mitomycin offers potent
tumor ablation: † 1 complete response at 3 months* (95% CI: 73.9, 84.5; n=191/240) 79.6% of patients who achieved CR estimated to remain tumor free at 12 months 82.3% 1 (95% CI: 75.9, 87.1; n=108/191) 1 Can be administered intravesically in an
outpatient setting †:Complete response was defined as negative white light cystoscopy, negative urine cytology, and when indicated, a negative for-cause biopsy at 3 months.1 CI=confidence interval. 1. Prasad et al. JUrol, 25Feb2024; 2.
Steinberg RL, Thomas LJ, O’Donnell MA. Bacillus Calmette-Guérin (BCG) treatment failures in non-muscle invasive bladder cancer: What truly constitutes unresponsive disease. 14 Bladder Cancer. 2015;1(2):105-116. doi:10.3233/blc-150015
LG-IR-NMIBC Market has Key Differences to HG-NMIBC Market Low-Grade
IR-NMIBC High-Grade NMIBC Issues: Chronic recurrence; rarely progresses to high- Issues: Progression, metastasis & death grade disease VS SOC: Repetitive TURBT SOC: TURBT, BCG, radical cystectomy, clinical trials 4 1,2,3 Incidence: ~25K/year
Newly diagnosed: ~23K/year 4 1,2,3 BCG-refractory: 18.7K/year Recurrent: ~59K/year Limited competition: UGN-102 is furthest along in C Clini linic ca al l t tr rials ials ongoing ongoing in in BCG BCG- -r refr efracto actor ry y p populatio
opulations ns clinical development as a non-surgical chemoablative S Signif ignifican icant t un unmet met need need given given llow re ow resp spon onse se r rates ates an and d therapy d du urab rability ility BCG is not widely used in low-grade
disease G Goa oall is is to to av avoi oid d r radical adical cystect cystectom omy y 1. ACS Cancer Facts & Figures 2023 2. SEER, AUA/SUO joint guideline 3. Babjuk et al. European Urology (2019), Simon (2019), 15 4. SEER*Stat Database (2019)
Surveillance Research Program; Curr Urol Rep (2016) 17: 68; Ther Adv Urol. 2012 Feb; 4(1): 13–32; UroGen Market Research.
UGN-102 Focuses on Improving Patient Outcomes with Non-Invasive,
Durable Option for LG-IR-NMIBC 1 ~82,000 patients in the U.S. annually Intermediate risk (IR) patients $5B+ are characterized by 1-2 of the Potential 4 following : Market 59,000 1,2,3 Recurrent Patients Multiple tumors A low-grade solitary tumor
>3 cm 23,000 Recurrence of LG NMIBC 1,2,3 New Patients within one year of the current diagnosis 1. ACS Cancer Facts & Figures 2023 2. SEER, AUA/SUO joint guideline 3. Babjuk et al. European Urology (2019), Simon (2019) 4. Chang et al. JUiI
2016 Diagnosis and Treatment of NMIBC AUA SUO Guideline 16
There Are Approximately 82K Annual Cases of Eligible LG IR NMIBC
Patients 1 Total US Population 340M (2025) Bladder Cancer 85,000 2 Incidence Non-Muscle 63,000 3 Invasive BC Low Grade 35,000 4 NMIBC Newly Diagnosed 23,000 5,6,7 LG IR NMIBC 59,000 Recurrent LG IR NMIBC 5,6,7 LG IR NMIBC patients per year 1. US
Census; 2.NIH; 3. SEER; 4. SEER; 5. ACS Cancer Facts & Figures 2023; 6. SEER, AUA/SUO joint guideline; 7. Babjuk et al. European Urology (2019), Simon (2019) 17
LG-IR NMIBC Patients Can Find Themselves in a Frustrating Cycle of
Treatment ~68% ~23% ~82,000 of recurrent patients of recurrent addressable LG-IR- 2-5 have 2 or more patients have 5 or NMIBC patients 1 1 recurrences more recurrences 1. Babjuk et al. European Urology (2019), Simon (2019), UroGen projections based
on SEER (2016) 2. Cancer Stat Facts: Bladder Cancer. National Cancer Institute Surveillance, Epidemiology, and End Results Program. Accessed July 10, 2023. https://seer.cancer.gov/statfacts/html/urinb.html 3. Chevli KK et al.. J Urol. 2022
Jan;207(1):61-69. doi: 10.1097/JU.0000000000002186. Epub 2021 Aug 26. PMID: 34433303; PMCID: PMC8667793. 4. Babjuk et al. European Urology (2019), 18 5. Simon M et al. ed. PLOS ONE. 2019;14(2):e0211721.
doi:https://doi.org/10.1371/journal.pone.0211721
Overview of UGN-102 Clinical Program ATLAS P3 TRIAL ENVISION P3 TRIAL
LAUNCHED FEB JUNE 2024 LAUNCHED MARCH JULY 2023 2021 2022 12-MO. DURABILITY POSITIVE TOP-LINE RESULTS REPORTED RESULTS REPORTED FOR ENVISION FOR BOTH ATLAS PHASE 3 TRIAL AND ENVISION 282 240 PHASE 3 TRIALS Enrolled Patients Enrolled Patients 2023
2024 2018 2021 2022 OPTIMA 2B TRIAL OCTOBER 2024 JANUARY 2022 LAUNCHED OCT 2018 FDA ACCEPTED RESULTS NDA SUBMISSION PUBLISHED FOR FOR UGN-102 IN OPTIMA PHASE 2B 1 LG-NMIBC TRIAL 63 Enrolled Patients 19 1 .J Urol. 2022 Jan; 207(1):
61–69.
Envision Phase 3 Summary of Response Rate At 3-Month Disease
Assessment: CRR of 79.6% UGN-102 (N = 240) Complete n (%) CRR (95% CI) Response Rate Complete Response 191 (79.6) 79.6 (73.9, 84.5) Non-Complete Response 49 (20.4) 79.6% Residual Disease 35 (14.6) Progression to HG Disease 7 (2.9) Indeterminate 2
(0.8) Missing 5 (2.1) 20 Prasad et al. 25Feb2024, JUROL
Envision Phase 3 Duration of Response (DOR): 82.3% at 12 months 1.0
UGN-102 (N = 191) % Number (%) of Patients with Events 33 (17.3%) 82.3 0.8 12mo Median (Months) Estimate: NE (NE, NE) 0.6 KM Estimates at*: 3 months 96.8% 0.4 6 months 91.9% 9 months 86.9% 0.2 12 months 82.3% (75.9, 87.1) 15 months (n=43) 80.9%
(73.9, 86.2) 0.0 9 0 3 6 18 months (n=9) 80.9% (73.9, 86.2) 12 Time from 3-Month CR (Months) *Time from 3-month CR 156 108 191 181 167 21 Prasad et al. JUrol, 25Feb2024 Pro ba bility of R e m aining E vent - Fre e
Large Sample Size Resulted In Tight Confidence Intervals 1.0 12mo 18mo
15mo % % % 82.3 80.9 80.9 0.8 (75.9, 87.1) (73.9, 86.2) (73.9, 86.2) UGN-102 0.6 0.4 0.2 0.0 9 12 15 18 21 0 3 6 Time from 3-month CR (months) 156 108 43 9 0 191 181 167 22 Prasad et al. JUrol, 25Feb2024 UroGen Data on File Probability of Remaining
Event-Free
Median DOR Not Estimable Due to Patients Remaining in CR UGN-102
(N=191) Kaplan-Meier Estimates of Duration of Response (months) 1st Quartile (95% CI) Not Estimable ( 14.7, Not Estimable ) Median (95% CI) Not Estimable 3rd Quartile (95% CI) Not Estimable Median Follow-up Time, months (95% CI) 13.8 ( 12.2, 14.5 )
UroGen Data on File 23
Adverse Events (AEs) Mainly Related To Lower Urinary Tract Symptoms
UGN-102 (N=240) n (% incidence) Treatment-emergent AEs (TEAEs) Any Adverse Events 140 (58.3) were generally mild to moderate in Any Serious Adverse Events 30 (12.5) severity Any TEAEs 137 (57.1) Any Grade >=3 TEAEs 33 (13.8) Any Treatment or
Procedure Related TEAEs 97 (40.4) The 2 treatment-related SAEs Any Treatment Related TEAEs 81 (33.8) were urethral stenosis and urinary Any Procedure Related TEAEs 64 (26.7) retention (both resolved) Any TEAEs Leading to Treatment Discontinuation 7
(2.9) Any TEAEs Leading to Study Discontinuation 6 (2.5) Any Serious TEAEs 29 (12.1) The 3 deaths were unrelated to Any Treatment or Procedure Related Serious TEAEs 4 (1.7) treatment: (cardiac event, Any Treatment Related Serious TEAEs 2 (0.8)
pneumonia, and not reported) Any Procedure Related Serious TEAEs 3 (1.3) Any TEAEs Leading to Death 3 (1.3) Any TEAEs of Special Interest 100 (41.7) 24 Prasad et al. JUrol, 25Feb2024
UGN-102 Has Demonstrated Compelling Clinical Results in Both Phase 3
Clinical Trials 4 4 ATLAS ITT ENVISION ATLAS Endpoint Newly diagnosed and Previously diagnosed Recurrent sub-group recurrent patients with prior TURBT with prior TURBT 65% vs. 64% 1 Complete Response Rate (CR) 79.6% 74% vs. 53% Similar CRR; offers a
less invasive 3-month disease assessment option to patients 2 2 Duration of Response (DOR) 66% vs. 40% 80% vs. 68% 82.3% 12-months following CR HR = 0.34 (66% Risk Reduction) HR = 0.46 (54% Risk Reduction) 3 3 Disease-Free Survival (DFS) 72% vs. 37%
72% vs. 50% N/A 12-months following randomization HR=0.295 (70% Risk Reduction) HR= 0.45 (55% Risk Reduction) Not reached vs. 7.2 Median Disease-Free Survival (DFS) Not Reached Not reached vs. 14.8 months months Prasad et al. JURol, 7Aug2023; Prasad
et al. JUrol, 25Feb2024 1. Complete Response defined as having no detectable disease (NDD) in the bladder at 3-month assessment following treatment UroGen Data on File, Source: Table 14.2.2.2.1a 2. Probability of maintaining a durable response at
12-months post CR by Kaplan-Meier analysis (total of 15 months) 3. Defined as the time from randomization until the earliest date of an event (total of 12-months) 4. Patients in treatment arm received UGN-102 +/- TURBT vs. TURBT alone 25
Consistently High Complete Response Rate At 3 Months 1 1 1 OPTIMA II
ATLAS ENVISION 2 3 4 (N=63) (N=142) (N=240) 65.1% 64.8% 79.6% ( 52.0, 76.7 ) ( 56.3, 72.6 ) ( 73.9, 84.5 ) 1. UroGen Data on File 2. OPTIMA II was a Phase 2B, single-arm, open-label study in patients with newly diagnosed and recurrent LG-IR-NMIBC.
3. ATLAS was a Phase 3, randomized, controlled trial of UGN-102 +/- TURBT vs TURBT alone in patients with newly diagnosed and recurrent LG-IR-NMIBC. ATLAS complete response is based on treatment with UGN-102 alone. 26 4. ENVISION is a Phase 3,
single-arm, open-label study in patients with LG-IR-NMIBC.
Robust Duration of Response 1 1 1 OPTIMA II ATLAS ENVISION 2 3 4 (N=41)
(N=92) (N=191) 69.9% 79.6% 82.3% ( 51.8, 82.3 ) ( 69.3, 86.8 ) ( 75.9, 87.1 ) 9-month DOR KM estimate 12-month DOR KM estimate 12-month DOR KM estimate 1. UroGen Data on File 2. OPTIMA II was a Phase 2B, single-arm, open-label study in patients with
newly diagnosed and recurrent LG-IR-NMIBC. 3. ATLAS was a Phase 3, randomized, controlled trial of UGN-102 +/- TURBT vs TURBT alone in patients with newly diagnosed and recurrent LG-IR-NMIBC. ATLAS complete response is based on treatment with
UGN-102 alone. 4. ENVISION is a Phase 3, single-arm, open-label study in patients with LG-IR-NMIBC. 27
ENVISION Patients Preferred UGN-102 to TURBT Less impact on activities/
responsibilities (work, recreation & exercise, sexual activity) Less bleeding, catheter issues shorter lasting UGN-102 Patients would recommend because UGN-102 was perceived to be less invasive, painful, and time-consuming 28 Stover and Smith,
under review (n=29)
Projected NDA Review Timeline for UGN-102 ENVISION Data Completion of
Acceptance Anticipated Results NDA Submission 1 2 Of NDA ODAC PDUFA June 2024 August 2024 October 2024 Q2 2025 June 13, 2025 1. ODAC (Oncologic Drugs Advisory Committee) anticipated in Q2 2025 based on most recent interactions with the U.S. Food and
Drug Administration (FDA) 29 2. PDUFA Target Action Date
Field Organization Will Deliver Industry-Leading Clinical Education and
Operational Support, Covering 85% of Market Opportunity Regional Business Director (RBD) Sales Force Territory Business Manager (TBM) Target HCPs ~9,000 (includes physicians National Business Executive (NBE) and extenders) Clinical Nurse Educator
(CNE) Field Support Regional Operation Manager (ROM) Key Account Director (KAD) Market Access Territory Business ~85 Field Reimbursement Manager (FRM) Managers (TBM) at launch Medical Medical Science Liaisons (MSL) 30 Note: Based on management
expectations and latest forecast
Patient Populations with Expected Rapid Adoption of UGN-102 1 Multiple
Recurrences In a recent survey, 92% of Urologists stated they 1 Surgically ineligible 3 would use UGN-102 2 Early Recurrences 1. Areas of greatest unmet need, Qualitative in-depth interviews fielded September 2019 (N = 19 UROs, 8 patients) 2.
Highest likelihood of use, Quantitative surveys fielded September 2023 (N = 111) 31 3. Based on survey conducted by UroGen in Q3 2023 of 111 board-certified urologists. Vendor IQVIA
Urologists’ feedback reflects growing interest for a new,
innovative treatment option in LG-IR-NMIBC • Urologists acknowledge the rapid and frequent recurrences and numerous procedures patients face, in addition to 1 potential increased morbidity • Excitement around promising and impressive
ENVISION data as the first potential product indicated for LG-IR- 2 NMIBC • Urologists consider UGN-102 a paradigm shifting novel therapeutic with almost 80% of patients achieving CR at 3 2 months with an estimated DOR at 12 months around 82%
2 • Durability is “meaningful and differentiating” when compared to TURBT in this LG-IR-NMIBC patient population 3 • UGN-102 has a favorable safety profile • Urologists find the prolonged dwell time a benefit over other
intravesical therapies; great option for “difficult to reach” 2 tumors; for small, multifocal, LG, IR disease or for LG recurrence • Challenges urologists highlight include cost, delayed reimbursement, pharmacy logistics, and slow
clinical and 2 operational adoption, satisfaction and habit of TURBT 1. Babjuk, 2019; Simon, 2019 2. UroGen Market Research and based on feedback from Ad Board comprised of Urology KOLs (n=21) held August and October 2024 3. Prasad et
al, JUroL 25Feb2024 32 LG-IR-NMIBC = low-grade intermediate-risk non-muscle invasive bladder cancer, TURBT = transurethral resection of bladder tumor, CR = complete response, DOR = durability of response, LG = low-grade, IR =
intermediate-risk
Expanding to Immuno- Oncology with Potential Monotherapy and
Combination Therapy UGN-301
Ongoing Multi-arm Phase 1 Trial of UGN- 301 (zalifrelimab) Anti-CTLA4
Antibody for Use in High-Grade Bladder Cancer Phase 1 clinical study utilizes a Master Protocol, evaluates safety, tolerability, and the potential Phase 2 dose of UGN-301 as monotherapy and in combination with other agents, including UGN-201
RECURRENT NMIBC Arm A Arm B Arm C Safety and dosing data from the first arm HG Ta /T1 &/or HG Ta/T1 &/or HG Ta &/or HG Ta /1 &/or CIS or IR LG Ta CIS CIS or IR LG Ta CIS evaluating UGN-301 as monotherapy presented late 2024
Additional UGN-301 UGN-301 UGN-301 arms to be monotherapy + UGN-201 + gemcitabine added Initiated combination therapy arms 1 evaluating UGN-301 + UGN-201 and UGN- 301 + gemcitabine in HG-NMIBC patients Dose Dose Dose Cohort 1: escalation to
escalation to Safety run-in escalation to ID RP2D ID RP2D ID RP2D RP2D -1 34 1. UGN-201 is UroGen’s proprietary formulation of imiquimod, a toll-like receptor 7 (TLR 7) agonist C O N F I D E N T I A L
UGN-301 Phase 1 Dose Escalation Study • UGN-301 is well tolerated
and has a favorable safety profile at all dose levels • No DLTs & no TEAEs leading to treatment discontinuation • Mild or moderate TRAEs • Local delivery of UGN-301, formulated in an RTgel, allows sustained exposure of
zalifrelimab in the bladder while limiting systemic exposure • Among the evaluable patients, 46% (6 of 13) and 33% (2 of 6), respectively, were recurrence-free or had a complete response at Week 12 Cohort 1 Cohort 2 Cohort 3 Cohort 4 Total
Dose level 100 mg 300 mg 500 mg 700 mg Evaluable @ wk 12 N=3 N=5 N=3 N=2 N=13 Ta/T1 Recurrence Free 1 3 2 0 6 patients Recurrence 2 2 1 2 7 Evaluable @ wk 12 N=0 N=1 N=4 N=1 N=6 CIS +/- Complete Response N/A 0 2 0 2 Ta/T1 Non-Complete Currently
evaluating safety of UGN-301 as a combination N/A 1 2 0 3 patients Response therapy with intravesical UGN-201 or gemcitabine in HG Indeterminate 0 0 0 1 1 recurrent NMIBC to establish RP2D 35 Dose limiting toxicity (DLT); Treatment emergent adverse
event (TEAE); Treatment related adverse event (TRAE); Recommended Phase 2 dose (RP2D)
Looking Ahead
UGN-103 & UGN-104: Next-Generation Novel Mitomycin- Based
Formulation Potential Advantages Received New U.S. Patent Allowance for Next- Generation Mitomycin-Based Products Expected Production to Provide Protection Until December 2041 Combines UroGen’s RTGel® technology with Supply medac’s
proprietary mitomycin Initiated Phase 3 UTOPIA trial of UGN-103 in Cost recurrent LG-IR-NMIBC patients, with UGN-104 Phase 3 trial expected to start in 1H 2025 Product convenience 37
Financial Position $254.2M $25.2M $200M $123.6M Cash, cash JELMYTO
revenue in Public offering of Term-loan facility 2 equivalents, and Q3 2024 common stock and with Pharmakon marketable pre-funded warrants 1 3 securities completed Q2 2024 1. Cash, cash equivalents, and marketable securities as of 09/30/2024.
Excludes restricted cash on Balance Sheet 2. In Q1 2024, UroGen entered into an amended and restated loan agreement with Pharmakon for an additional third and fourth tranche of senior secured loan. The third tranche of $25 million was drawn on
September 23, 2024. The fourth tranche of $75 million may be drawn down at UroGen’s discretion if UGN-102 is approved in the U.S. on or before June 30, 2025 3. The closing of the sale of $16.1 million of common stock pursuant to the
underwriters' option to purchase additional shares was completed in July 2024 38
In Summary.... With unprecedented data in LG-IR-NMIBC, we are focused
on pre-commercial and launch activities for UGN-102 with a target PDUFA date of June 13, 2025 We continue to increase adoption of JELMYTO with recent Durability Data supporting continued use We have a strong balance sheet with focus on UGN-102
commercial execution, and strategic and efficient capital deployment Our next generation novel mitomycin formulations will provide an opportunity to extend our leadership in addressing unmet needs in Urothelial cancers Through Organic and In-Organic
opportunities, we plan to build a long-term sustainable growth business 39
Thank You January 2025
APPENDIX
UGN-102 Potentially Addresses the Unmet Need for a Better Treatment
Option 79.6% 82.3% (73.9, 84.5) (75.9, 87.1) Complete Response Rate Estimated probability of maintaining at 3 months durable response at 12 months Safety profile characterized primarily Non-surgical treatment with potential to by mild to moderate
AEs reduce overall burden on patients UroGen Data on File 42
UroGen is Striving to Transform the Way Bladder Cancer is Treated 1
$5B+ ~82,000 #1 2 Potential TAM Annual addressable U.S. population, UGN-102 may become the first LG-IR-NMIBC market FDA approved medicine for indicating potential to reduce burden for ripe for innovation LG-IR-NMIBC large population of LG-IR-NMIBC
patients >86% of patients RTGel is designed to Prolonged disease-free Generally interviewed would uniquely address what you intervals well tolerated recommend UGN-102 can see and what you can’t 1. ACS Cancer Facts & Figures 2023; SEER,
AUA/SUO joint guideline; Babjuk et al. European Urology (2019), Simon et al (2019) PLoS ONE 14(2): e0211721 2. UroGen estimates based on market research 43
Redefine SOC for LG-UTUC as Kidney-Sparing Management with JELMYTO
Amplify real-world experience with JELMYTO and generate additional data to inform clinical practice Expected to Initiate Expand JELMYTO uTRACT Registry and support data Phase 3 Trial of UGN- collection around: 104 in 1H 2025 ▪ Real-world
durability and safety OLYMPUS-like trial to ▪ Effectiveness in broad patient and tumor types determine efficacy and ▪ Adjunctive use after endoscopic ablation safety of UGN-104 ▪ Outcomes following retreatment and maintenance
therapy UGN-104 to simplify Publish OLYMPUS long-term follow up data: In those reconstitution procedure achieving a CR, median DOR was ~4 years and shorten the Support pilot investigator-initiated study of JELMYTO in high- manufacturing process
grade UTUC 44 C O N F I D E N T I A L
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