Full Press Release Details
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- -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,
Changing the Treatment Paradigm for Urothelial Cancers (UGN-101)
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: 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-Gu rin (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.
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):
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
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