Urogen Pharma Ltd. · NASDAQ:URGN
Urogen Pharma — Investor Presentation (March 2024)
56 slides from Urogen Pharma's investor presentation, Mar 4, 2024
Slide 1: Developing Innovative Medicines to Treat Urothelial Cancers
Exhibit 99.1 Developing Innovative Medicines to Treat Urothelial Cancers March 2024 For investor audiences only. Not for promotional use with healthcare professionals.
Slide 2: Forward-Looking Statements
Forward-Looking Statements 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 estimated addressable patient population and market and revenue opportunity forJELMYTO in LG-U
Slide 3: UroGen is pioneering new therapies for urothelial cancers
Successful Commercial Product: UroGen is pioneering JELMYTO is the first and only FDA-approved non-surgical new therapies to treatment for patients with LG-UTUC meet the unique needs of patients Late-Stage Clinical Asset: with urothelial UGN-102 being developed as a minimally invasive, non-surgical
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: Repetitive risky surgeries Intolerance of Anatomical foreign materials barriers in the urinary tract Lost kidneys and organs Increased risk of morbidity in The urinar
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 of active drugs Potentially improves the therapeutic RTGel exists as a liquid effects of existing products at lower temperatures and converts to gel fr
Slide 6: Unlocking A Strong Foundational Pipeline Supporting Long-Term Sustainable Growth
Unlocking A Strong Foundational Pipeline Supporting Long-Term Sustainable Growth JELMYTO UGN-102 UGN-301 (UGN-101) Phase 3 Phase 1 ~6K – 7K ~18,700 ~82,000 1 addressable U.S. population addressable U.S. population addressable U.S. population $2B+ Market $3B+ Market $700M Market Low-Grade Intermediat
Slide 7: Changing the Treatment Paradigm for Urothelial Cancers: JELMYTO (UGN-101)
Changing the Treatment Paradigm for Urothelial Cancers JELMYTO (UGN-101)
Slide 8: LG-UTUC Is a Rare Disease that Recurs Often
LG-UTUC Is a Rare Disease that Recurs Often 6,000-7,000 2,800-3,200 3,000-4,000 ELIGIBLE PATIENTS IN THE 1 2 Newly Diagnosed Recurrent Patients U.S. ANNUALLY, INCLUDES: Treatment Options Treatment Options RNU RNU Additional Endoscopic Endoscopic Management Management 70%-80% UC is the costliest canc
Slide 9: JELMYTO First & Only FDA-Approved Non-Surgical Treatment for LG-UTUC
JELMYTO First & Only FDA-Approved Non-Surgical Treatment for Patients with LG-UTUC 1 Clinically Meaningful OLYMPUS Phase 3 Data 2 Complete Response Rate at 3-months 58% Durability of Response at 12-months by 82% 2 KM estimate Median Durability of Response (14.6 to 47.6 29 months 3,4 months) data fro
Slide 10: JELMYTO Revenue Trend Reflects Long-term Growth
JELMYTO Revenue Trend Reflects Long-term Growth Observed QoQ Variability Is Expected with Summer/Holiday Seasonality Annual JELMYTO WW Revenues ($MM) Quarterly JELMYTO WW Revenues ($MM) $100 $25 $86.0 $21.1 $20.9 2023 full-year $20 $80 revenue $18.2 $76.0 guidance $17.2 $16.6 $16.2 $16.1 $64.5 $15 $
Slide 11: Changing the Urologic Cancer Landscape Post Launch
Changing the Urologic Cancer Landscape Post Launch ® Growing Awareness and Adoption of JELMYTO Supports Use of RTGel -based Therapies in Urology Patient Identification Demonstration of Strong Reimbursement Support from Urologists & Adoption Permanent J code effective 296 accounts 1,088 1 1 January 1
Slide 12: Growing Body of Real-World Evidence
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- 69% 23% World Effectiveness, Including as a Chemoablative Agent and Ureteric Treatment of Resid
Slide 13: UGN-102: Anticipated Primary Driver of UroGen Future Growth
UGN-102: Anticipated Primary Driver of UroGen Future Growth UGN-102 Potential to Transform the Treatment Paradigm in Low-Grade Intermediate Risk Non-Muscle Invasive Bladder Cancer (LG-IR-NMIBC)
Slide 14: UGN-102 Potential to be the First Non-Surgical Chemoablative Therapy
UGN-102 Potential to be the First Non-Surgical Chemoablative Therapy in Low-Grade Intermediate Risk Disease Low-grade IR NMIBC High-grade NMIBC Issue: chronic recurrence; rarely Issue: progression, metastasis & death progresses to high-grade disease SOC: TURBT, BCG, radical cystectomy, SOC: repetiti
Slide 15: Bladder Cancer Treatment Algorithm
Bladder Cancer Treatment Algorithm TURBT LOW RISK INTERMEDIATE RISK HIGH RISK TURBT TURBT Multiple or recurrent Solitary Primary Any T1 and/or G3 and/or CIS low-grade tumors Low-grade Ta Single immediate Chemotherapy BCG + Maintenance BCG + Maintenance chemotherapeutic instillation Observations Recu
Slide 16: UGN-102 Focus on Improving Patient Outcomes in LG-IR-NMIBC
UGN-102 Focus on Improving Patient Outcomes with Noninvasive, Durable Option for LG-IR-NMIBC 1 ~82,000 patients in the U.S. annually INTERMEDIATE RISK (IR) PATIENTS ARE CHARACTERIZED BY 1-2 OF $3B+ 4 THE FOLLOWING : Market 60,000 Multiple tumors 1,2,3 Recurrent Patients A low-grade solitary tumor >3
Slide 17: NMIBC Patients Can Find Themselves in a Frustrating Cycle of Treatment
NMIBC Patients Can Find Themselves in a Frustrating Cycle of Treatment ~82,000 ~23% ~68% of recurrent patients of recurrent addressable LG-IR- have 2 or more 2-5 patients have 5 or NMIBC patients 1 1 recurrences more recurrences 1. Babjuk et al. European Urology (2019), Simon (2019), UroGen projecti
Slide 18: 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 3 would used UGN-102 1 Surgically ineligible 1. Areas of greatest unmet need, Qualitative in-depth interviews fielded 2 September 2019 (N = 19 UROs, 8 patients) Early
Slide 19: ENVISION Patient Experience was Positive
ENVISION Patient Experience was Positive Impact to Daily Life Patient Preference TURBTs impacted daily activities for about one week The majority of patients reported they would recommend UGN-102 to UGN-102 didn’t affect daily other patients, citing: responsibilities for most patients • Lack of disr
Slide 20: UGN-102: Leveraging Similarities with Distinct Advantages
UGN-102: Leveraging Similarities with Distinct Advantages ® JELMYTO & UGN-102 UGN-102 ® • RTGel & mitomycin • 10x larger potential formulations patient population ® • Mitomycin RTGel • Simpler administration combinations UGN-102 to bladder than to upper tract • Similar diseases at a genetic & mutati
Slide 21: Overview of UGN-102 Program
Overview of UGN-102 Program ATLAS P3 TRIAL ENVISION P3 TRIAL JULY 2023 LAUNCHED FEB 2021 LAUNCHED MARCH 2022 POSITIVE TOP-LINE RESULTS REPORTED FOR BOTH ATLAS AND ENVISION PHASE 282 242 3 TRIALS Enrolled Patients Enrolled Patients 2023 2024 2022 2018 2021 OPTIMA 2B TRIAL JANUARY 2022 2024 LAUNCHED O
Slide 22: ENVISION: Summary of Response Rate At 3-Month Disease Assessment
ENVISION: Summary of Response Rate At 3-Month Disease Assessment UGN-102 (N = 240) n (%) CRR (95% CI) Complete Response Rate Complete Response 190 (79.2) 79.2 (73.5, 84.1) Non-Complete Response 50 (20.8) 79.2% Residual Disease 35 (14.6) Progression to HG Disease 6 (2.5) Indeterminate 4 (1.7) Missing
Slide 23: UGN-102 Has Demonstrated Compelling Clinical Results in Both Phase 3 Trials
UGN-102 Has Demonstrated Compelling Clinical Results in Both Phase 3 Clinical Trials 4 4 Endpoint ENVISION ATLAS ITT ATLAS Recurrent sub-group Newly diagnosed and Previously diagnosed with prior TURBT recurrent patients with prior TURBT 1 79% 74% vs. 53% 65% vs. 64% Complete Response Rate (CR) at 3-
Slide 24: Looking Ahead
Looking Ahead ENVISION DOR data expected in Planned NDA Submission by 2Q 2024 4Q 2024
Slide 25: UGN-103: Next-Generation Novel Mitomycin-Based Formulation
UGN-103: Next-Generation Novel Mitomycin-Based Formulation POTENTIAL ADVANTAGES Licensing agreement with medac GmbH to commercialize Production a next-generation novel mitomycin-based formulation Combines UroGen’s RTGel® technology with medac’s Supply proprietary mitomycin Cost UroGen plans to initi
Slide 26: Expanding to Immuno-Oncology with Potential Monotherapy and Combination Therapy
Expanding to Immuno-Oncology with Potential Monotherapy and Combination Therapy JELMYTO UGN-102 UGN-301 (UGN-101)
Slide 27: Ongoing Multi-arm Phase 1 Trial of UGN-301 (zalifrelimab)
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 RECURRENT with other agent
Slide 28: Looking Ahead
Looking Ahead
Slide 29: UroGen Priorities
UroGen Priorities Advance pre-commercial activities for UGN-102 in LG-IR-NMIBC; Data from 12-month durability of response data anticipated in 2Q 2024; prospective NDA submission by 4Q 2024 Accelerate JELMYTO U.S. adoption leveraging adjusted sales strategy Support balance sheet with focus on strateg
Slide 30: Q3 2023 Financial Snapshot
Q3 2023 Financial Snapshot Strengthened balance sheet to focus on maximizing shareholder value through disciplined investment supporting clinical and commercial execution $76- $154M $20.9M $120M $86M Cash, cash JELMYTO revenue in Private placement of Full-year 2023 equivalents and Q3 2023 ordinary s
Slide 31: Thank You
Thank You March 2024 For investor audiences only. Not for promotional use with healthcare professionals.
Slide 32: Appendix
APPENDIX
Slide 33: Extended 96-hour Shelf-Life Increases Operational Efficiencies
Extended 96-hour Shelf-Life Increases Operational Efficiencies September 2022 Label Update Extended Stability Period for JELMYTO Admixture from 8 Hours to 96 Hours Reduced gaps in Expanded future Increased flexibility treatment availability opportunities Provides pharmacists more Opportunity to re-e
Slide 34: Confident in JELMYTO's Future Outlook
Confident in JELMYTO’s Future Outlook High Performing and Growing Body of Antegrade Administration JELMYTO 2022 Label Growth in Developing Multicenter Real-World Offers Efficient Mode of Update with Extended Territories Reflect and Long-Term Follow-up Administration and a Shelf Life Increases Potent
Slide 35: Potential to Unlock a Significant Market Opportunity
Potential to Unlock a Significant Market Opportunity in a Very Underserved Patient Population UGN-102 UGN-301 Phase 3 Phase 1 ~82,000 ~18,700 addressable U.S. population 1 addressable U.S. population $3B+ Market $5B+ $2B+ Market combined TAM Low-Grade Intermediate Risk Immunotherapy Targeting High-
Slide 36: OPTIMA II Phase 2b Trial Showed Significant Tumor Response
OPTIMA II Phase 2b Trial Showed Significant Tumor Response and Long-Term Treatment Benefit UGN-102 Phase 2B OPTIMA OPTIMA II Rollover Study 1 Study (LG-IR-NMIBC) Long-Term Follow-Up Initial 3,6 Results These data suggest continued durability in patients treated 2 24.4 65% CR 72.5% with UGN-102 47% m
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Slide 38: ATLAS Trial Design
ATLAS Trial Design Screening* Date of CR recurrence, UGN-102 DFS progression NCR • Suspected • Cystoscopy or death bladder • Cold cup biopsy cancer TURBT • Urine cytology 1:1 • Recurrence • CT urogram, retrograde Date of pyelogram, or CR recurrence, MRI TURBT DFS progression NCR or death TURBT -1 to
Slide 39: ATLAS Study Endpoints
ATLAS Study Endpoints Primary Endpoint (ITT): • Disease-free survival (DFS), defined as the time from randomization until the earliest date of any of the following events: üResidual disease at the 3-month assessment üRecurrence üProgression üDeath Key Secondary Endpoints: • Complete response rate (C
Slide 40: ATLAS Demographics and Safety Profile
ATLAS Demographics and Safety Profile • Treatment-emergent AEs were generally mild to moderate Demographics and baseline characteristics were well balanced • Similar safety profile to other between treatment arms studies of UGN-102 • Any treatment or procedure related serious TEAEs were comparable a
Slide 41: ATLAS Scorecard Highlights Clinically Meaningful, Durable Results
ATLAS Scorecard Highlights Clinically Meaningful, Durable Results for UGN-102 Overall and Relative to TURBT Analysis Assessment UGN-102 +/- TURBT vs TURBT Alone 72% vs. 50% Probability of Disease-Free Survival (KM) at 15 months Primary Endpoint of Disease-Free Survival (DFS) HR = 0.45 (55% Risk Redu
Slide 42: ATLAS DFS - 55% Reduction of Risk in the Intent to Treat Population
ATLAS DFS - 55% Reduction of Risk for Recurrence, Progression, or Death in the Intent to Treat Population in ATLAS H HR R: 0 : 0.4 .46 5 ( (0.2 0.29 4, 0.6 , 0.88 6) ) Prasad et al. JURol 7Aug2023 UroGen Data on File Source: Table 14.2.1.1a Kaplan-Meier Plot of Disease-Free Survival
Slide 43: ATLAS DOR - 54% Reduction of Risk in Patients Who Had a 3-Month CR
ATLAS DOR - 54% Reduction of Risk for Recurrence, Progression, or Death in Patients Who had a 3-Month CR in ATLAS * HR: 0.46 (0.24, 0.86) *UGN Alone Subgroup of the UGN 102 +/- TURBT arm in ATLAS UroGen Data on File Source: Table 14.2.1.1a Kaplan-Meier Plot of Duration of Response in Complete Respon
Slide 44: ATLAS DOR - 66% Reduction of Risk in Recurrent Patients
ATLAS DOR - 66% Reduction of Risk for Recurrence, Progression, or Death in Recurrent Patients Who Received UGN-102 Alone in ATLAS * HR: 0.34 (0.15, 0.75) *UGN Alone Subgroup of the UGN 102 +/- TURBT arm in ATLAS UroGen Data on File Kaplan-Meier Plot of DOR in Complete Responders in the Recurrent Sub
Slide 45: Summary of Disease-Free Survival
Summary of Disease-Free Survival: Significantly More Total Recurrence and Progression in TURBT Alone Arm UGN-102 +/- TURBT TURBT Alone (N = 140) / n (%) (N = 142) / n (%) Patients with Events, n (%) 37 (26.1) 55 (39.3) Recurrence of LG Disease 20 (14.1) 39 (27.9) Progression to HG Disease 17 (12.0)
Slide 46: Three-Month Complete Response Rates Were Similar Between Treatment Arms in ATLAS
Three-Month Complete Response Rates Were Similar Between Treatment Arms in ATLAS UGN-102 Alone TURBT Alone (N = 142) (N = 140) CRR CRR Response n (%) n (%) (95%CI) (95% CI) Complete Response 92 (64.8) 64.8% (56.3, 72.6) 89 (63.6) 63.6% (55.0, 71.5) Non-complete Response 50 (35.2) 51 (36.4) Residual
Slide 47: Summary of Duration of Response in Complete Responders
Summary of Duration of Response in Complete Responders: Longer DOR with UGN-102 Alone UGN-102 Alone TURBT Alone (N = 92) / n (%) (N = 89) / n (%) Patients with Events, n (%) 18 (19.6) 24 (27.0) Recurrence of LG Disease 15 (16.3) 17 (19.1) Progression to HG Disease 3 ( 3.3) 6 ( 6.7) Death 0 1 ( 1.1)
Slide 48: UGN-102 Shows Substantial Reduction of Risk Across Multiple Patient Populations
UGN-102 Shows Substantial Reduction of Risk of Recurrence, Progression, or Death Across Multiple Patient Populations in ATLAS 1 0.45 (0.29, 0.68) ITT – All Patients Complete Responders – 0.46 (0.24, 0.86) UGN-102 Alone Recurrent Complete 0.34 (0.15, 0.75) 2 Responders – UGN-102 Alone 1. Intent to Tr
Slide 49: UroGen is Poised to Transform the Way Bladder Cancer is Treated
UroGen is Poised to Transform the Way Bladder Cancer is #1 95% Treated UGN-102 may become the shared prescriber base first medicine approved ® with JELMYTO to treat LG-IR-NMIBC $3B+TAM $120M Compelling clinical private placement LG-IR-NMIBC market data package from 3 with experienced ripe for innova
Slide 50: 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 (Pri
Slide 51: ENVISION Single-Arm Study Description
ENVISION Single-Arm Study Description Primary endpoint: • Complete response rate (CRR) at 3-month visit Key Secondary endpoint: • Duration of Response (DOR), defined as time from first documented CR until the earliest date of: üRecurrence üProgression üDeath Patient Population: • Previously diagnose
Slide 52: ENVISION Demographics and Safety Profile
ENVISION Demographics and Safety Profile Demographics and baseline characteristics were reflective of • Treatment-emergent AEs were typical LG-IR-NMIBC patient generally mild to moderate population • Similar safety profile to other studies of UGN-102 UroGen Data on File Overall summary of Demographi
Slide 53: Standard of Care for HG-NMIBC
Standard of Care for HG-NMIBC CURRENT TREATMENT TURBT, BCG, Clinical trials, Radical Cystectomy * ANNUAL U.S. PATIENTS HIGH-GRADE ~18,700 BCG Unresponsive Issue: progression, CLINICAL TRIALS ONGOING metastasis & death IN BCG-REFRACTORY POPULATIONS Significant unmet need given low response rates and
Slide 54: Combining UGN-301 with UGN-201 (UGN-302) Shows Encouraging Activity
Combining UGN-301 with UGN-201 (UGN-302) Shows Encouraging Activity as a Novel Agonist / Antagonist Immunotherapy Combination UGN-201 + UGN-301 = UGN-302: Non-clinical data TLR 7 (UGN-201): Anti-CTLA4 (UGN-301): suggest improved survival (murine) and decreased tumor stimulates cytokines blocks ”canc
Slide 55: UroGen has made Significant Progress
UroGen has made Significant Progress KEY ACCOMPLISHMENTS JELMYTO FDA approval and U.S. launch Announced positive topline data from Phase 3 Trials & held successful pre-NDA meeting with Strengthened balance sheet FDA for UGN-102 in LG-IR- via $120 million private NIMBC placement Advanced Immuno-Oncol
Slide 56: Growing Body of Evidence that Nephrostomy Tube Administration of JELMYTO is Efficient
Growing Body of Evidence that Nephrostomy Tube Administration of JELMYTO is Efficient for Doctors and Favorable Safety Profile ANTEGRADE ADMINISTRATION Does not require fluoroscopy Minimizes manipulation of the May be performed by trained ureter during treatment which nursing professionals under cle