Urogen Pharma Ltd. · NASDAQ:URGN
Urogen Pharma — Investor Presentation (January 2025)
44 slides from Urogen Pharma's investor presentation, Jan 14, 2025
Slide 1: Developing Innovative Medicines to Treat Urothelial Cancers
Exhibit 99.1 Developing Innovative Medicines to Treat Urothelial Cancers January 2025
Slide 2: Disclaimers
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 w
Slide 3: Investment Highlights
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 Ba
Slide 4: Invasive and Radical Surgery Is the Standard of Care in Urothelial Cancers
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 t
Slide 5: 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 therapeutic effects of ® RTGel exists as a liquid existing products at lower temperatures and converts to gel f
Slide 6: Unlocking a Strong Foundational Pipeline Supporting Long-Term Sustainable Growth
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 $5B+ Potential Market 1,2 Pot
Slide 7: Changing the Treatment Paradigm for Urothelial Cancers (JELMYTO)
Changing the Treatment Paradigm for Urothelial Cancers (UGN-101) JELMYTO
Slide 8: LG-UTUC Is a Rare Disease that Recurs Often
LG-UTUC Is a Rare Disease that Recurs Often 6,000-7,000 2,800-3,200 3,000-4,000 ELIGIBLE PATIENTS IN THE U.S. 1 2 ANNUALLY, INCLUDES: Newly Diagnosed Recurrent Patients Treatment Options Treatment Options RNU RNU Endoscopic Management Additional Endoscopic Management UC is the costliest cancer in th
Slide 9: JELMYTO First and Only FDA-Approved Non-Surgical Treatment for LG-UTUC
JELMYTO First and Only FDA-Approved Non- Surgical Treatment for Patients with LG-UTUC 1 Clinically Meaningful OLYMPUS Phase 3 Data 2 Complete Response Rate at 3 months 58% 2 Durability of Response at 12 months by KM estimate 82% Median Duration of Response (95%CI: 13.1, 57.5; n=41) 3,4 47.8 months d
Slide 10: Growing Body of Real-World Evidence Supports Use Case For JELMYTO
Growing Body of Real-World Evidence Supports Use Case For JELMYTO* Data from 2+ years in market reinforces JELMYTO efficacy and safety Select Results Independent multicenter reviews support JELMYTO real-world effectiveness, including as a chemoablative agent and treatment 69% 23% of residual disease
Slide 11: JELMYTO Retrospective Analysis Results Presented at AUA 2024
JELMYTO Retrospective Analysis Results Presented at AUA 2024* JELMYTO treatment demonstrates favorable Recurrence Free Survival (RFS) rates for patients with LG-UTUC who 1 respond to initial induction Additional Insights No differences in RFS were 1,2 observed regarding : 86% RFS at 24-months for LG
Slide 12: JELMYTO Revenue Trend Reflects Continued Growth
JELMYTO Revenue Trend Reflects Continued Growth Annual JELMYTO WW Revenues ($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 $1
Slide 13: Changing the Treatment Paradigm for Urothelial Cancers: UGN-102
Changing the Treatment Paradigm for Urothelial Cancers UGN-102
Slide 14: UGN-102: The First Potential Breakthrough Localized Therapy for LG-IR-NMIBC
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 mitomyc
Slide 15: LG-IR-NMIBC Market has Key Differences to HG-NMIBC Market
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:
Slide 16: UGN-102 Focuses on Improving Patient Outcomes in LG-IR-NMIBC
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 soli
Slide 17: Approximately 82K Annual Cases of Eligible LG IR NMIBC Patients
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
Slide 18: LG-IR NMIBC Patients Can Find Themselves in a Frustrating Cycle of Treatment
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 pr
Slide 19: Overview of UGN-102 Clinical Program
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 Enroll
Slide 20: ENVISION Phase 3 Summary of Response Rate: CRR of 79.6%
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) Indeterm
Slide 21: ENVISION Duration of Response: 82.3% at 12 months
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
Slide 22: Large Sample Size Resulted In Tight Confidence Intervals
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 Proba
Slide 23: Median DOR Not Estimable Due to Patients Remaining in CR
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 (
Slide 24: Adverse Events Mainly Related To Lower Urinary Tract Symptoms
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 Tre
Slide 25: UGN-102 Has Demonstrated Clinical Results in Both Phase 3 Clinical Trials
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% Sim
Slide 26: Consistently High Complete Response Rate At 3 Months
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 recurr
Slide 27: Robust Duration of Response
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 s
Slide 28: ENVISION Patients Preferred UGN-102 to TURBT
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 St
Slide 29: Projected NDA Review Timeline for UGN-102
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 wit
Slide 30: Field Organization Will Deliver Clinical Education and Operational Support
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) Clini
Slide 31: Patient Populations with Expected Rapid Adoption of UGN-102
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
Slide 32: Urologists' Feedback Reflects Growing Interest for a New Treatment Option
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
Slide 33: Expanding to Immuno-Oncology: UGN-301
Expanding to Immuno- Oncology with Potential Monotherapy and Combination Therapy UGN-301
Slide 34: Ongoing Multi-arm Phase 1 Trial of UGN-301 in High-Grade Bladder Cancer
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, includ
Slide 35: UGN-301 Phase 1 Dose Escalation Study
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
Slide 36: Looking Ahead
Looking Ahead >) UroGen:
Slide 37: UGN-103 & UGN-104: Next-Generation Novel Mitomycin-Based Formulation
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 proprietar
Slide 38: Financial Position
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
Slide 39: In Summary
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 foc
Slide 40: Thank You
Thank You January 2025 >) UroGen:
Slide 41: Appendix
APPENDIX e) UroGen
Slide 42: UGN-102 Potentially Addresses the Unmet Need for a Better Treatment Option
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 mi
Slide 43: UroGen is Striving to Transform the Way Bladder Cancer is Treated
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
Slide 44: Redefine SOC for LG-UTUC as Kidney-Sparing Management with JELMYTO
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-w