Full Press Release Details
Response Results from the ENVISION Study NASDAQ: URGN June 13, 2024 1
Forward-Looking St at ement s 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 for UroGen to transform bladder cancer treatment; the potential of JELMYTO to change the
treatment paradigm in LG-UTUC; the potential of UGN-102 to transform the treatment paradigm in LG-IR-NMIBC; the potential of UGN-301 to expand to Immuno-Oncology for HG-NMIBC; the estimated patient population in bladder cancer and estimated
addressable patient population for UGN-102 in LG-IR-NMIBC and UGN-301 in HG-NMIBC; the estimated total addressable market opportunity for UGN-102 in LG-IR-NMIBC; the potential for UGN-102 to become the first FDA approved medicine for LG-IR-NMIBC;
the opportunity and potential benefits of UGN-102 for LG-IR-NMIBC and potential advantages over TURBT; the potential NDA completion and review timeline for UGN-102, including the expected completion of the NDA submission to the FDA and the
FDA's potential acceptance thereof and the FDA's potential approval timing; and the potential of UroGen's proprietary RTGel technology platform to improve therapeutic profiles of existing drugs. These statements are subject
to a number of risks, uncertainties and assumptions, including, but not limited to: ENVISION duration of response data may not be sufficient to support an NDA submission for UGN-102; even if an NDA for UGN-102 is accepted by the FDA, there is no
guarantee that such NDA will be sufficient to support approval of UGN-102 on the timeframe expected, or at all; the timing and success of clinical trials and potential safety or other complications encountered therein; results from prior or ongoing
clinical trials 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; competition in UroGen's industry; 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; 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 March 31,
2024, filed with the Securities and Exchange Commission (SEC) on May 13, 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. Not for promotional use with healthcare professionals. 2
UROGEN IS UNIQUELY POSITIONED TO TRANSFORM BLADDER CANCER TREATMENT LIZ
BARRETT, PRESIDENT AND CEO 3
UroGen Overview Panel Discussion Liz Barrett Moderator:
Mark P. Schoenberg, M.D. President and CEO, UroGen Chief Medical Officer, UroGen Max Kates, M.D. The Burden of LG-IR-NMIBC Johns Hopkins School Of Medicine Mark P. Schoenberg, M.D. Jennifer Linehan, M.D. Chief Medical Officer,
UroGen Saint John's Cancer Institute James McKiernan, M.D. Columbia University Irving Medical UGN-102 Clinical Data Center, New York Presbyterian Sandip Prasad, M.D., M.Phil. Angela Stover, Ph.D. Morristown
Hospital/Atlantic Health UNC Gillings School of Global Public System, NJ Today's Health Agenda Patient Perspectives from ENVISION What is Next? Angela Stover, Ph.D. Liz Barrett UNC Gillings School of Global Public President and
CEO, UroGen Health Q&A Patient Interview Julio Lago Patient Liz Barrett President and CEO, UroGen 4
WE ASPIRE TO CHANGE THE Because Patients Deserve Better TREATMENT
PARADIGM SURGICAL CARE MINIMALLY INVASIVE, ORGAN-SPARING THERAPEUTIC OPTIONS JELMYTO UGN-102 UGN-301 (UGN-101) Changing the Potential to Transform Expanding to Treatment Paradigm for the Treatment of Immuno-Oncology for Low-Grade Upper Tract
Low-Grade High-Grade Non- Urothelial Cancer Intermediate Risk Muscle Invasive (LG-UTUC) Non-Muscle Invasive Bladder Cancer Bladder Cancer (HG-NMIBC) (LG-IR-NMIBC)
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 RTGel exists as a therapeutic effects of liquid at lower existing products temperatures and converts to gel form at
body temperature. Leverages physiologic flow of urine to provide natural exit from the body 6
Bladder Cancer Affect s Pat ient s and Families Across The U.S. ~730,000
people in High rates of 1 the U.S. living with bladder cancer 2 recurrence 1. Cancer Stat Facts: Bladder Cancer. National Cancer Institute: Surveillance, Epidemiology, and End Results Program. Accessed June 5, 2024 (data as of 2021).
https://seer.cancer.go v/statfacts/html/urinb.html 2. MBA ASBP PhD. Cancer Recurrence Statistics. Cancer Therapy Advisor. Published November 30, 2018. https://www.cancertherapyadvisor.com/home/tools/fact-sheets/cancer-recurrence- 7
statistics/#:~:text=Some%20cancers%20are%20difficult%20to
High Recurrence Rat e Leads t o Frust rat ing Treat ment Cycle ~68% ~23%
of recurrent patients have 2 of recurrent patients have 5 1 1 or more recurrences or more recurrences 8 1. UroGen projections based on SEER (2016) Babjuk et al. European Urology (2019) and Simon (2019).
UroGen is Uniquely Positioned to 1 Transform the Way ~82,000 #1 Bladder
Cancer is UGN-102 may become the Annual LG-IR-NMIBC Treated first FDA-approved U.S. patient medicine population for LG-IR-NMIBC 2 $5B+TAM UGN-102 can be Compelling clinical data administered by a trained LG-IR-NMIBC market package from 4 trials and
healthcare professional in alone ripe for 593 patients an outpatient setting, or innovation even at home 9 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 TAM: Total Addressable Market
THE BURDEN OF LG -IR-NMIBC MARK SCHOENBERG, M.D., CHIEF MEDICAL OFFICER
NMIBC Has Multiple Stages Before Becoming Muscle Invasive NMIBC MIBC
Tis Ta T1 T2 T3 T4 non-invasive non-invasive tumor invades tumor invades tumor invades tumor invades inner lining and muscle perivesical fat adjacent organs connective and structures tissue G2 G3 moderately differentiated poorly differentiated 11
American Joint Committee on Cancer. AJCC Cancer Staging Manual. Urinary Bladder. 7th edn. New York, NY: Springer; 2010: 497-502.
UGN-102's Proposed Indicat ion: For Treat ment of LG-IR-NMIBC
NMIBC MIBC Tis Ta T1 T2 T3 T4 Low Grade non-invasive non-invasive tumor invades tumor invades tumor invades tumor invades inner lining and muscle perivesical fat adjacent organs ~70% of all bladder cancers connective tissue and structures
High risk of recurrence Low risk of progression Intermediate Risk LG Ta Tumors that are large (> 3cm), multifocal, or recurrent UGN-102 LG T1 Tumors SOC: Repeated Transurethral Resection of Bladder Tumor
(TURBT) G2 G3 moderately differentiated poorly differentiated 12 American Joint Committee on Cancer. AJCC Cancer Staging Manual. Urinary Bladder. 7th edn. New York, NY: Springer; 2010: 497-502. Apollo et al. PLOS ONE (2019)
Repeat Surgery for LG-IR NMIBC Comes wit h Risks for Pat ient s
Patients with LG-IR-NMIBC LG-IR-NMIBC patients who had 2-4 procedures had a who have multiple recurrences carry a ~35% of patients will experience 14% an adverse event 10-20% greater risk of death within 90 days of 1 2 than patients who only
had undergoing a TURBT. risk of progression. 3 one procedure. 1. Sharma V, Aaronson DS, Fero KE, et al. Adverse events after transurethral resection of intermediate-risk non-muscle invasive bladder cancer. J Urol. 2021:206(suppl 3):e122.
doi:10.1097/JU.0000000000001977.08 2. Sharma V, Chamie K, Schoenberg M, et al. Natural history of multiple recurrences in intermediate-risk non-muscle invasive bladder cancer: lessons from a prospective cohort. Urology. 2023;173:134-141.
doi:10.1016/j.urology.2022.12.009 3. Erikson MS, Petersen AC, Andersen KK, Jacobsen FK, Mogensen K, Hermann GG. Do repeated transurethral procedures under general anesthesia influence mortality in patients with non-invasive urothelial bladder 13
cancer? A Danish national cohort study. Scand J Urol. 2020;54(4):281-289. doi:10.1080/21681805.2020.1782978
LG-IR-NMIBC Market : Key Differences Compared t o HG-NMIBC Market
Low-Grade IR-NMIBC High-Grade NMIBC Issues: Chronic recurrence; rarely progresses to Issues: Progression, metastasis & death high-grade disease VS SOC: Repetitive TURBT SOC: TURBT, BCG, radical cystectomy, clinical trials 4 1,2,3 Newly
diagnosed: ~23K/year Incidence: ~25K/year 4 1,2,3 BCG-refractory: 18.7K/year Recurrent: ~59K/year Limited competition: UGN-102 is furthest along Clinical trials ongoing in BCG-refractory populations in clinical development as a non-surgical
chemoablative therapy Significant unmet need given low response BCG is not widely used in low-grade disease rates and durability Goal is to avoid radical cystectomy 1. ACS Cancer Facts & Figures 2023 2. SEER, AUA/SUO joint guideline 3. Babjuk et
al. European Urology (2019), Simon (2019), 14 14 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.
Posit ioning UGN-102 for Success Low-Grade IR NMIBC Issues: Chronic
recurrence; rarely progresses to high-grade disease VS SOC: Repetitive TURBT 1,2,3 Newly diagnosed: ~23K/year 1,2,3 Recurrent: ~59K/year Limited competition: UGN-102 is furthest along in clinical development as a non-surgical chemoablative therapy
BCG is not widely used in low-grade disease 1. ACS Cancer Facts & Figures 2023 2. SEER, AUA/SUO joint guideline 3. Babjuk et al. European Urology (2019), Simon (2019), 15 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: ROBUST AND CONSISTENT CLINICAL RESULTS 16
About Dr. Sandip Prasad Sandip Prasad, M.D., M.Phil. Garden
State Urology Director of Genitourinary Surgical Oncology Vice-Chair of Urology at Morristown Medical Center/Atlantic Health System in New Jersey Clinical Associate Professor at Rutgers NJMS Clinical Assistant
Professor at Thomas Jefferson University Completed residency at the Harvard Program in Urology and an SUO fellowship at the University of Chicago 60 peer-reviewed journal articles and book chapters Associate editor or
editorial reviewer for nine specialty journals in Urology 17
ENVISION DURATION OF RESPONSE RESULTS 18
St rong, Consist ent Complet e Response Rat e At 3 Mont hs OPTIMA II
ATLAS ENVISION (N=63) (N=142) (N=240) 65.1% 64.8% 79.6% (52.0, 76.7) (56.3, 72.6) (73.9, 84.5) UroGen Data on File OPTIMA II was a Phase 2B, single-arm, open-label study in patients with newly diagnosed and recurrent LG-IR-NMIBC. 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. 19 ENVISION is a Phase 3, single-arm, open-label study
in patients with LG-IR-NMIBC.
Robust Durat ion of Response (DOR) Observed in Mult iple Trials OPTIMA
II ATLAS (N=41) (N=92) 69.9% 79.6% (51.8, 82.3) (69.3, 86.8) 9-month DOR KM estimate 12-month DOR KM estimate UroGen Data on File ATLAS DOR estimates based on treatment with UGN-102 alone 20 Based on Kaplan-Meier (KM) Estimates.
ENVISION Single-Arm Pivotal Study Description Patient Population:
Demographics and baseline characteristics reflective of general LG-IR- NMIBC patient population All patients followed for a minimum of 15 months Primary Endpoint: Complete response rate (CRR) at 3-month visit, as defined by
cystoscopy, for cause biopsy, and urine cytology Key Secondary Endpoint: Duration of response (DOR), defined as time from first documented CR until the earliest date of: Recurrence Progression Death UroGen Data on File 21
21 Overall Summary of Demographics in the Appendix
Robust Complet e Response Rat e At 3 Mont hs 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) UroGen Data on File
DOR: 82.3% at 12 Mont hs, Overwhelming Majorit y Remain Disease Free
UGN-102 1.0 + UGN-102 + + + + (N = 191) + + + + + + % % + + + + + + + % 82.1 82.1 + + + + + + + + + + + + + + ++ + 0.8 Number (%) of Patients with 82.3 15mo 18mo 33 (17.3%) Events 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 82.3% 12 months (75.9, 87.1) 80.9% 15 months 0.0 (73.9, 86.2) 0 3 6 9 12 15 18 21 80.9% 18 months Time from 3-Month CR (Months) (73.9, 86.2) 191 181 167 156 108 43 9 0 *Time from 3-Month CR 23
UroGen Data on File Probability of Remaining Event-Free
Large Sample Size Result ed In Tight DOR Confidence Int ervals 1.0 + +
+ + + + 12mo + + 15mo 18mo % + + + % % 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 0 3 6 9 12 15 18 21 Time from 3-Month CR (Months) 191 181 167 156 108 43 9 0 24 UroGen Data on File Probability of Remaining Event-Free
Median DOR Not Est imable Due t o Pat ient s 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 )
25 UroGen Data on File
Predicted Median Duration Of Response (DOR) is 40.0 Months Weibull
Predicted Curve 1.0 0.8 0.6 0.4 0.2 0.0 0 6 12 18 24 30 36 42 48 54 55 Estimate Lower Upper Predicted Median (95% CI): 40.0 months (23.9, 63.9) 26 UroGen Data on File
AEs Generally Mild t o Moderat e in Severit y UGN-102 (N=240)
AEs mainly related to n (% incidence) lower urinary tract Any Adverse Events 140 (58.3) symptoms Any Serious Adverse Events 30 (12.5) Any TEAEs 137 (57.1) Any Grade 3 TEAEs 33 (13.8) The 2 treatment-related SAEs were urethral Any
Treatment or Procedure Related TEAEs 97 (40.4) stenosis and urinary Any Treatment Related TEAEs 81 (33.8) retention (both Any Procedure Related TEAEs 64 (26.7) resolved) Any TEAEs Leading to Treatment Discontinuation 7 (2.9) Any TEAEs Leading to
Study Discontinuation 6 (2.5) The 3 deaths were Any Serious TEAEs 29 (12.1) unrelated to treatment: Any Treatment or Procedure Related Serious TEAEs 4 (1.7) (cardiac event, Any Treatment Related Serious TEAEs 2 (0.8) pneumonia, and not Any
Procedure Related Serious TEAEs 3 (1.3) reported) Any TEAEs Leading to Death 3 (1.3) Any TEAEs of Special Interest 100 (41.7) UroGen Data on File 27 Overall summary of AEs in the Appendix
Consist ent ly High Complet e Response Rat e At 3 Mont hs OPTIMA II
ATLAS ENVISION (N=63) (N=142) (N=240) 65.1% 64.8% 79.6% (52.0, 76.7) (56.3, 72.6) (73.9, 84.5) UroGen Data on File OPTIMA II was a Phase 2B, single-arm, open-label study in patients with newly diagnosed and recurrent LG-IR-NMIBC. ATLAS was a Phase