Full Press Release Details
February 2024 For investor audiences only. Not for promotional use with healthcare professionals.
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-UTUC, UGN-102
in LG-IR-NMIBC, and UGN-301 in HG-NMIBC; the potential of UroGen's proprietary RTGel technology platform to improve therapeutic profiles of existing drugs to advance the treatment of specialty cancers and urologic disease; the
expectations regarding the annual and long-term growth of JELMYTO revenue; expected revenue trends forJELMYTO; UroGen's pipeline supporting long-term sustainable growth; the potential ofJELMYTO , UGN-102, and UGN-301 to transform the
treatment paradigm in LG-UTUC, LG-IR-NMIBC, and HG-NMIBC, respectively; the clinical results from ATLAS and ENVISION providing optimism for potential FDA approval of UGN-102; the Company's pending patent applications, may not be successful and
in such event the duration of our intellectual property protection would be more limited; the potential advantages of the antegrade administration ofJELMYTO; the potential prescriber behavior, expected interest in prescribing as well as growing
awareness and adoption of JELMYTO; the expectation that UGN-102 will be a significant driver of UroGen's future growth; the potential of UGN-102 to be the first non-surgical chemoablative therapy in LG-IR-NMIBC; the potential advantages of
UGN-102 over TURBT; plans to submit an NDA for UGN-102 to the FDA in 2024; the expectation of ENVISION duration of response data in 2Q 2024; the expectation of safety and dosing data from the first arm evaluating UGN-301 as monotherapy in mid-2024;
UroGen priorities including the advancement of pre-commercial activities for UGN-102, plans for capital preservation, use of sales strategy to accelerateJELMYTO adoption, a focus on urologic oncology expertise, and focus on UGN-301 as monotherapy
and combination therapy to advance immune-oncology pipeline; the importance of and operational efficiencies created by the 2022 label update that extended the stability period for JELMYTO admixture and its potential to reduce operational hurdles to
uptake upon launch of UGN-102; confidence in the future of JELYMYTO; the potential thatJELMYTO is adopted as a standard of care; the interpretation and summary of results of OLYMPUS Phase 3, OPTIMA Phase 2b, ATLAS, and ENVISION trials; the size and
importance of the sharedJELMYTO and UGN-102 prescriber base; and the encouraging effects of combining UGN-301 with UGN-201 (UGN-302). These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to:
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; potential prescriber behavior is based on preliminary feedback that may change as a result of new data, labeling limitations, or other factors; 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 beyondJELMYTO 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 filed with the Securities and
Exchange Commission (SEC) on November 14, 2023, 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. For investor audiences only. Not for promotional use with healthcare professionals.
UroGen is pioneering new therapies for the unique needs of patients with
urothelial cancers by utilizing proprietary technology to potentially enhance proven and novel medicines and deliver them aligned with the way Urologists practice
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 urinary tract is
designed to void, which poses elderly patients challenges including limited dwell time for chemotherapies and other therapies delivered to the bladder.
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 from at body
temperature. Leverages physiologic flow of urine to provide natural exit from the body
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 Intermediate
Risk Low-grade Upper Tract Urothelial High-Grade Non-Muscle Non-Muscle Invasive Bladder Carcinoma (LG-UTUC) Invasive Bladder Cancer Cancer (LG-IR-NMIBC) (HG-NMIBC) 1. 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.
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 $120 million private NIMBC placement Advanced Immuno-Oncology program
resulting in multi- arm Phase I clinical trial Announced next-generation novel mitomycin-based formulation UGN- 103 and medac GmbH licensing agreement with potential IP protection until 2041
Changing the Treatment Paradigm for Urothelial Cancers JELMYTO
LG-UTUC Is a Rare Disease that Recurs Often 6,000-7,000 2,800-3,200
3,000-4,000 ELIGIBLE PATIENTS IN THE 1 2 Newly Diagnosed Recurrent Patients U.S. ANNUALLY, INCLUDES: Treatment Options Treatment Options RNU RNU Additional Endoscopic Endoscopic Management Management 70%-80% UC is the costliest cancer in the U.S.
healthcare of LG-UTUC patients ultimately 4 system on a per-patient basis 3 receive nephroureterectomies 1. Upfill-Brown 2018, 2. Cutress 2012, 3. Grasso et al. (2012) BJU International, 4. Yeung et al. (2014) Pharmacoeconomics RNU = radical
JELMYTO First & Only FDA-Approved Non-Surgical Treatment for
Patients with LG-UTUC 1 Clinically Meaningful OLYMPUS Phase 3 Data 2 Complete Response Rate at 3-months 58% Durability of Response at 12-months by 82% 2 KM estimate Median Durability of Response (14.6 to 47.6 29 months 3,4 months) data from
long-term follow-up study 1. Important Safety Information and the full Prescribing Information available at https://www.urogen.com/download/pdf/jelmyto_prescribing.pdf 2. Matin, Surena F. J Urol. 2022 Apr;207(4):779-778 3. Pierorazio, Philip M.
Long-term outcomes of treatment with UGN-101. SUO 2022, #158 4. Limitations of long-term follow-up study include N=16. Please refer to the referenced citations for disclosures of such limitations.
JELMYTO Revenue Trend Reflects Long-term Growth Observed QoQ
Variability Is Expected with Summer/Holiday Seasonality Annual JELMYTO WW Revenues ($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 $60 $13.6
$13.0 $48.1 $11.4 $10 $40 $8.0 $7.5 $5 $3.5 $20 $11.9 $0.4 $0 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q $0 2020 2020 2020 2021 2021 2021 2021 2022 2022 2022 2022 2023 2023 2023 2020 2021 2022 2023
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, 2021
to standardize practices/hospitals activated have treated more than one patient and facilitate reimbursement; ASP +6% implemented High repeat use and awareness Majority of large commercial plans ~70% have policies in place, covering
over 150 million lives Expected interest in prescribing 96% 2 JELMYTO over next 12 months Positive reimbursement across all 1 payer types 150 million lives 1. Numbers as of November 1, 2023 2. UroGen market research, 91
urologists surveyed who are not currently prescribing JELMYTO (July 2022)
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 Residual Disease
Following Endoscopic Resection CR Stenosis When JELMYTO As compared to Evaluated Outcomes in Range of Tumor Types; Evidence treated residual 44% in OLYMPUS. for Favorable Response in Patients with Low-Volume disease following laser ~1/2 of patients
Residual Disease ablation (overall CR were treated with 59% in OLYMPUS) antegrade administration. Varied Practice Patterns, with Antegrade Method of Administration via Nephrostomy Tube Shown as Viable Woldu, et al. Early Experience with UGN-101 for
the Treatment of Upper Tract Urothelial Cancer - A MultiCenter Evaluation of Practice Patterns and Outcomes. UrolOncol. *Real world retrospective studies have inherent evidentiary limitations. Please refer to the referenced citations for
disclosures of such limitations.
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 clean after a
nephrostogram confirms placement at the first instillation may limit stricture formation rather than sterile conditions associated with repeated instrumentation of the upper urinary tract Retrospective analyses of real-world data support benefits of
antegrade administration, making it an attractive alternative to retrograde 1,2,3 administration 1. Murray K, et al. J Urol. 2022 Feb 7;101097JU; 2. Rose K, et al. J Urol. 2022 May 1; doi.org/10.1097/JU.0000000000002643.06 3. Rose K, et al. BJUI.
2022 Oct 26; DOI: 10.1111/bju.15925
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)
UGN-102 Potential to be the First Non-Surgical Chemoablative Therapy in
Low-Grade Intermediate Risk Disease High-grade NMIBC Low-grade IR NMIBC Issue: chronic recurrence; rarely Issue: progression, metastasis & death progresses to high-grade disease SOC: TURBT, BCG, radical cystectomy, SOC: repetitive TURBT clinical
trials VS Newly diagnosed: ~22K/year Incidence: ~25K Recurrent: ~60K/year BCG-refractory: 18.7K Limited competition: UGN-102 is Clinical trials ongoing in BCG-refractory furthest along in clinical development populations. as a non-surgical
chemoablative Significant unmet need given low therapy response rates and durability BCG is not widely used in low-grade Goal is to avoid radical cystectomy disease 1. ACS Cancer Facts & Figures 2023 2. SEER, AUA/SUO joint guideline 3. Babjuk et
al. European Urology (2019), Simon (2019),
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 Current Treatment Options A low-grade solitary
tumor >3 cm TURBT TURBT + 4* Recurrence of LG NMIBC within one adjuvant chemotherapy 22,000 year of the current diagnosis 1,2,3 New Patients 1. ACS Cancer Facts & Figures 2023 2. SEER, AUA/SUO joint guideline 3. Babjuk et al. European Urology
(2019), Simon (2019), 4. Tobert et al Urology (2019), Rhijn et al Nature Urology (2016), 4. Bryan et al Ann R Coll Surg Engl (2010) *Adjuvant chemotherapy only used in 0-30% of U.S. eligible population TURBT = trans urethral resection of bladder
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 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, Shore ND, Trainer A, Smith AB,
Saltzstein D, Ehrlich Y, Raman JD, Friedman B, D'Anna R, Morris D, Hu B, Tyson M, Sankin A, Kates M, Linehan J, Scherr D, Kester S, Verni M, Chamie K, Karsh L, Cinman A, Meads A, Lahiri S, Malinowski M, Gabai N, Raju S, Schoenberg M, Seltzer E,
Huang WC. Primary Chemoablation of Low-Grade Intermediate-Risk Nonmuscle-Invasive Bladder Cancer Using UGN-102, a Mitomycin-Containing Reverse Thermal Gel (Optima II): A Phase 2b, Open-Label, Single-Arm Trial. 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), Simon (2019), 5. Simon M, Bosset PO, Rouanne M, et al. Multiple recurrences and risk of disease progression in patients with
primary low-grade (TaG1) non-muscle-invasive bladder cancer and with low and intermediate EORTC-risk score. Real FX, ed. PLOS ONE. 2019;14(2):e0211721. doi:https://doi.org/10.1371/journal.pone.0211721
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 & mutational JELMYTO Routine procedure in driver level clinic that urology offices & UGN-102 are very familiar with Share a 95% prescriber base No special equipment like
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 OCT 2018 RESULTS
PROSPECTIVE NDA PUBLISHED FOR SUBMISSION FOR OPTIMA PHASE 2B UGN-102 IN LG-IR- 1 TRIAL NMIBC 63 Enrolled Patients 1 .J Urol. 2022 Jan; 207(1): 61-69.
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 5 (2.1)
UroGen Data on File Summary of Response Rate At 3-Month Disease Assessment
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-month disease
Similar CRR; offers a less invasive assessment option to patients 2 2 66% vs. 40% 80% vs. 68% Duration of Response (DOR) TBD at 12-months following CR HR = 0.34 (66% Risk Reduction) HR = 0.46 (54% Risk Reduction) 3 3 Disease-Free Survival (DFS) 72%
vs. 37% 72% vs. 50% at 12-months following N/A randomization HR=0.295 (70% Risk Reduction) HR= 0.45 (55% Risk Reduction) Median Disease-Free Survival Not reached vs. 14.8 TBD N/A (DFS) months Prasad et al. JURol, 7Aug2023, 1. Complete Response
defined as having no detectable disease (NDD) in the bladder at 3-month assessment following treatment UroGen Data on File, Source: Table 14.2.2.2.1a 2. Probability of maintaining a durable response at 12-months post CR by Kaplan-Meier analysis
(total of 15 months) 3. Defined as the time from randomization until the earliest date of an event (total of 12-months) 4. Patients in treatment arm received UGN-102 +/- TURBT vs. TURBT alone
Looking Ahead ENVISION DOR data expected in Planned NDA Submission by
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