Slide 1: New Horizons in Bladder Cancer: UGN-102 Duration of Response Results from the ENVISION Study
Exhibit 99.1 New Horizons in Bladder Cancer: UGN-102 Duration of Response Results from the ENVISION Study NASDAQ: URGN June 13, 2024 1
Slide 2: Forward-Looking Statements
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 ch
Slide 3: UroGen Is Uniquely Positioned to Transform Bladder Cancer Treatment
UROGEN IS UNIQUELY POSITIONED TO TRANSFORM BLADDER CANCER TREATMENT LIZ BARRETT, PRESIDENT AND CEO 3
Slide 4: UroGen Overview Panel Discussion Agenda
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
Slide 5: We Aspire to Change the Treatment Paradigm
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 Uppe
Slide 6: RTGel Proprietary Reverse-Thermal Hydrogel Technology
® 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
Slide 7: Bladder Cancer Affects Patients and Families Across The U.S.
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
Slide 8: High Recurrence Rate Leads to Frustrating Treatment Cycle
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).
Slide 9: UroGen is Uniquely Positioned to Transform the Way Bladder Cancer is Treated
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 packa
Slide 10: The Burden of LG-IR-NMIBC
THE BURDEN OF LG -IR-NMIBC MARK SCHOENBERG, M.D., CHIEF MEDICAL OFFICER 10
Slide 11: NMIBC Has Multiple Stages Before Becoming Muscle Invasive
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 di
Slide 12: UGN-102's Proposed Indication: For Treatment of LG-IR-NMIBC
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 structur
Slide 13: Repeat Surgery for LG-IR NMIBC Comes with Risks for Patients
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
Slide 14: LG-IR-NMIBC Market: Key Differences Compared to HG-NMIBC Market
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 Newl
Slide 15: Positioning UGN-102 for Success
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 chemoa
Slide 16: UGN-102: Robust and Consistent Clinical Results
UGN-102: ROBUST AND CONSISTENT CLINICAL RESULTS 16
Slide 17: About Dr. Sandip Prasad
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
Slide 18: ENVISION Duration of Response Results
ENVISION DURATION OF RESPONSE RESULTS 18
Slide 19: Strong, Consistent Complete Response Rate at 3 Months
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. A
Slide 20: Robust Duration of Response Observed in Multiple Trials
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.
Slide 21: ENVISION Single-Arm Pivotal Study Description
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 cyst
Slide 22: Robust Complete Response Rate at 3 Months
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
Slide 23: DOR: 82.3% at 12 Months, Overwhelming Majority Remain Disease Free
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 E
Slide 24: Large Sample Size Resulted in Tight DOR Confidence Intervals
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)
Slide 25: Median DOR Not Estimable Due to Patients Remaining in CR
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
Slide 26: Predicted Median Duration of Response is 40.0 Months
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
Slide 27: AEs Generally Mild to Moderate in Severity
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 Tre
Slide 28: Consistently High Complete Response Rate at 3 Months
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. AT
Slide 29: High CR Rates Maintained with Robust Duration of Response
High CR Rat es Maint ained wit h Robust Durat ion of Response OPTIMA II ATLAS ENVISION (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 UroGen Data on File ATLAS DOR estimates based on treatment w
Slide 30: UGN-102 Potentially Addresses the Unmet Need for a Non-Surgical Option
UGN-102 Potentially Addresses the Unmet 82.3% 79.6% Need for a Non- (73.9, 84.5) (75.9, 87.1) Surgical Option Complete Response Rate at Estimated probability of 3 months maintaining Complete Response at 12 months Safety profile Non-surgical treatment characterized primarily by with potential to redu
Slide 31: Patient Perspectives from ENVISION
PATIENT PERSPECTIVES FROM ENVISION ANGELA STOVER, PH.D. ASSOCIATE PROFESSOR DEPARTMENT OF HEALTH POLICY AND MANAGEMENT AT UNC GILLINGS SCHOOL OF GLOBAL PUBLIC HEALTH 31
Slide 32: About Dr. Angela Stover
ABOUT DR. ANGELA STOVER Angela Stover, PhD • Associate Professor, Department of Health Policy and Management at UNC Gillings School of Global Public Health • A health services researcher with expertise in patient-reported outcomes (PRO) methods and implementation science • Co-directs the NC TraCS' I
Slide 33: ENVISION Patient Perceptions of UGN-102 and TURBT: Impact on Daily Activities
ENVISION PATIENT PERCEPTIONS OF UGN-102 AND TURBT: IMPACT ON DAILY ACTIVITIES 33
Slide 34: ENVISION Interviews
ENVISION Int erviews • Patients with NMIBC commonly ask urologists how their daily activities/responsibilities will be affected by treatment(s) • In ENVISION, we interviewed patients about the impact on their daily activities with UGN-102 and their recollection of standard of care (transurethral res
Slide 35: Methods
Methods • Gold standard: content analysis - Data are patient quotes • Semi-structured interview guides at enrollment and 3 months • Transcripts coded by 3 experienced coders with detailed codebooks (software: Dedoose) • Emerging themes and discrepancies were captured and reconciled through consensus
Slide 36: Three Interview Themes
Three Int erview Themes Less impact on activities/ responsibilities (work, recreation & exercise, sexual activity) Less bleeding, catheter UGN-102 issues shorter lasting Patients would recommend because UGN-102 was perceived to be less invasive, painful, and time-consuming N=29 36 36 Stover and Smit
Slide 37: Recovery Time
Recovery Time UGN-102 TURBT “Well, first off, they're [TURBTs] “Yes. I think it took me a gettin' more and more painful, longer time to recover from and it's taken longer and the bladder resection than the longer to recover from them. gel. [For TURBT], maybe at two It's just a little bit of—every ti
Slide 38: No Impact on Daily Activities
No Impact on Daily Act ivit ies UGN-102 TURBT “With the gel, the daily activity was a big difference, and I didn’t worry at [TURBT not discussed] all. Basically, I lived my normal life except the one day [instillation], which was well worth it.” (34) “They [UGN-102] didn’t have any impact on me. I w
Slide 39: Impact on Work
Impact on W ork TURBT UGN-102 “It's [incontinence] mostly a big “The TURBT, I was basically missing embarrassment and an absolute for seven, eight days, I could do no pain in the ass at work…some days work. Here [with UGN-102], really, are much worse than others and work has continued, I have not de
Slide 40: Treatment-Related Side Effects
Treatment-Relat ed Side Effect s UGN-102 TURBT “For six weeks, I was in stages of “I honestly have as much side extreme to moderate to low level of effects from the anesthesia.” pain/itching—extreme internal (06) itching.”(20) “I’ll tell you one thing. I do not “It was very itchy, all my bladder and
Slide 41: Bleeding
Bleeding UGN-102 TURBT “That [TURBT] was a lot of bleeding.” (45) “At least two occasions [for “They removed five polyps at that TURBT], one of which there was time…Then I bled for two weeks. fair amounts of bleeding.” (03) Finally shut off. [My urologist] told me that there would be some It [TURBT]
Slide 42: Impact on Sexual Activity
Impact on Sexual Act ivit y UGN-102 TURBT “I would say that I didn't have any “Basically, I would like to get away difference in performance, but I from having them [tumors] took longer to have relationships extracted from me ’cause my with my wife with TURBT [than doctor told me the more UGN-102].”
Slide 43: UGN-102 meets an unmet need for a non-surgical alternative for NMIBC
From the patient perspective, UGN-102 meets an unmet need in care delivery for a non-surgical treatment alternative for NMIBC 43 43
Slide 44: Patient Interview: Julio Lago
PATIENT INTERVIEW: JULIO LAGO 44
Slide 45: B Yregen
B Yregen
Slide 46: Thought Leader Panel
THOUGHT LEADER PANEL 46
Slide 47: Thought leader panelist biographies
Max Kates, MD, is an Associate Professor of Urology and Oncology in the Brady Urological Institute and directs the Division of Urologic Oncology for the Brady Urological Institute at Johns Hopkins School Of Medicine, where he works with the team at the Johns Hopkins Greenberg Bladder Cancer Institut
Slide 48: What Is Next?
WHAT IS NEXT? 48
Slide 49: Unprecedented Clinical Results Further Support Completion of NDA Submission
Unprecedent ed Clinical Result s Furt her Support Complet ion of NDA Submission Robust Duration of Strong Complete Response Response KM Estimate At 12 Rate At 3 Months Months ENVISION ENVISION (N=240) (N=191) 79.6% 82.3% (73.9, 84.5) (75.9, 87.1) UroGen Data on File 49 49
Slide 50: Potential to Launch UGN-102 in One Year If Approved
Pot ent ial t o Launch UGN-102 in One Year If Approved PRIORITY STANDARD Completion of NDA ENVISION Data Potential Potential Potential FDA Submission Results Approval Approval Acceptance Q3 2024 TODAY Q1 2025 Q2 2025 Q4 2024 50
Slide 51: UroGen is Uniquely Positioned to Transform the Way Bladder Cancer is Treated
UroGen is Uniquely Positioned to Transform the Way Bladder Cancer is Treated 1 2 $5B+TAM ~82,000 #1 Annual addressable U.S. UGN-102 may become LG-IR-NMIBC market population, indicating potential the first FDA ripe for innovation to reduce burden for large approved medicine population of LG-IR-NMIBC
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Slide 55: ENVISION Trial Design
ENVISION Trial Design Informed Consent and Screening Visit to Determine Eligibility (LG-IR-NMIBC and no evidence of HG disease based on cystoscopy, biopsy, and cytology) UGN-102 once -weekly for 6 weeks (total of 6 intravesical instillations) 3-month Visit Complete Response Non-complete Response (Pr
Slide 56: Summary of Demographics and Baseline Characteristics
Summary of Demographics and Baseline Characteristics UGN-102 Characteristic Statistic (N = 240) / n (%) Age Median Age (Min, Max) 70.0 (30, 92) Age Group 2 (Years), n (%) >= 65 162 (67.5) Sex, n (%) Male 147 (61.3) Female 93 (38.8) Prior TURBT, n (%) Yes 232 (96.7) No 8 (3.3) Previous LG NMIBC Episo
Slide 57: Summary of Adverse Events Occurring in >5.0% of Participants
Summary of Adverse Event s Occurring in >5.0% of Part icipant s UGN-102 (N=240) Patients with Any TEAE 137 (57.1) Dysuria 54 (22.5) Haematuria 20 (8.3) Urinary tract infection 17 (7.1) Pollakiuria 16 (6.7) Fatigue 13 (5.4) Urinary retention 12 (5.0) UroGen Data on File 57