UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
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FORM
CURRENT REPORT
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Emerging growth company
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| Item 7.01 | Regulation FD Disclosure. |
Furnished as Exhibit 99.1 to this Current Report on Form 8-K is a presentation of UroGen Pharma Ltd. (the “Company”), all or a portion of which will be presented by the Company beginning at 11:00 a.m. Eastern Time on June 13, 2024 at the Company’s previously announced UGN-102 data event.
The information in this Item 7.01 of this Current Report on Form 8-K, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, or otherwise subject to the liabilities of that section or Sections 11 and 12(a)(2) of the Securities Act of 1933, as amended. The information contained in this Item 7.01, including Exhibit 99.1, shall not be incorporated by reference into any filing the Company makes with the U.S. Securities and Exchange Commission (“SEC”), whether before or after the date hereof, regardless of any general incorporation language in such filing.
| Item 8.01 | Other Events |
On June 13, 2024, the Company announced positive secondary endpoint duration of response (“DOR”) data from the Phase 3 ENVISION trial investigating UGN-102 for intravesical solution in patients with low-grade intermediate risk non-muscle invasive bladder cancer (“LG-IR-NMIBC”).
In the ENVISION trial, the 12-month DOR data by Kaplan-Meier (“KM”) estimate for patients who achieved a complete response (“CR”) at three months after the first instillation of UGN-102 was 82.3% (95% CI, 75.9%, 87.1%). This is in line with the 69.9% (51.8%, 82.3%) nine-month DOR data by KM estimate for patients who achieved a CR at three months after the first instillation of UGN-102 observed in the Company’s Phase 2b OPTIMA II trial and the 79.6% (69.3%, 86.8%) 12-month DOR data by KM estimate for patients who achieved a CR at three months after the first instillation of UGN-102 alone observed in the Company’s Phase 2 ATLAS trial.
The ENVISION trial met its primary endpoint with patients having a 79.6% (73.9%, 84.5%) CR rate at three months after the first instillation of UGN-102. This is in line with the CR rates at three months after the first instillation of UGN-102 of 65.1% (52.0%, 76.7%) and 64.8% (56.3%, 72.6%) observed in the OPTIMA II trial and ATLAS trial, respectively.
Among the patients in the ENVISION trial who achieved a CR at three months, 76.4% (69.8%, 82.3%) maintained a CR at 12 months. Among the 240 patients enrolled in the ENVISION trial, 60.8% (54.3%, 67.0%) were still in response at 12 months.
In the ENVISION trial, DOR KM estimates at 15 (n=43) and 18 (n=9) months were both 80.9% (95% CI, 73.9%, 86.2%). Although median DOR was not estimable due to the number of patients remaining in CR, the predicted median DOR is 40 months based on the Weibull Predicted Curve.
The ENVISION trial demonstrated a similar safety profile to that observed in the OPTIMA II and ATLAS trials, with treatment-emergent adverse events typically mild-to-moderate in severity.
In January 2024, the Company initiated the submission of a rolling New Drug Application (“NDA”) to the U.S. Food and Drug Administration (“FDA”) for UGN-102 as a treatment for LG-IR-NMIBC. The latest DOR data are expected to support the UGN-102 NDA, which the Company plans to complete in the third quarter of 2024, with a potential FDA acceptance in the fourth quarter of 2024 and potential FDA approval in the first quarter of 2025 (assuming priority review) or the second quarter of 2025 (assuming standard review).
If approved, UGN-102 may become the first FDA-approved medicine for LG-IR-NMIBC. The Company estimates that the annual addressable U.S. patient population for LG-IR-NMIBC is approximately 82,000, of which approximately 23,000 are estimated to be newly diagnosed and 59,000 are estimated to be recurrent patients. The Company estimates that the total addressable market opportunity for UGN-102 in LG-IR-NMIBC is potentially over $5.0 billion, assuming an expected pricing range of $16,000 to $19,000 per dose.
UGN-102, if approved, may be an alternative to the current standard of care for LG-IR-NMIBC, trans-urethral resection of bladder tumor (“TURBT”). The Company estimates that approximately 68% of LG-IR-NMIBC patients have two or more recurrences, with approximately 23% having five or more recurrences. Repeated TURBT procedures to treat these recurrences can impact patients’ physical health and quality of life. The Company estimates that around 35% of patients will experience an adverse event within 90 days of undergoing a TURBT, and patients who have had two to four procedures have an estimated 14% greater risk of death than patients who have only had one procedure.
Forward Looking Statements
This Current Report on Form 8-K contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are based upon current expectations or beliefs, as well as a number of assumptions about future events. Although the Company believes that the expectations reflected in the forward-looking statements and the assumptions upon which they are based are reasonable, the Company can give no assurance that such expectations and assumptions will prove to be correct. Forward-looking statements include all statements that are not historical facts and can generally be identified by terms such as “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potentially,” or “will” or similar expressions and the negatives of those terms. These statements include, but are not limited to, statements regarding the potential for the Company to transform bladder cancer treatment; the potential of JELMYTO® to change the treatment paradigm in low-grade upper tract urothelial cancer (“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 high-grade non-muscle invasive bladder cancer (“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 expected price range per dose for UGN-102; 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 FDA’s potential acceptance thereof and approval of UGN-102 and the timing thereof; the potential of the Company’s proprietary RTGel® technology platform to improve therapeutic profiles of existing drugs; and the Company’s sustained release technology making local delivery potentially more effective as compared to other treatment options. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause the Company’s actual results, performance, or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These risks, uncertainties and other factors relate to, among others: ENVISION DOR 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 commercialization activities; the labeling for any approved product; competition in the Company’s industry; the scope, progress and expansion of developing and commercializing the Company’s product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; the Company’s RTGel technology may not perform as expected and the Company may not successfully develop and receive regulatory approval of any product candidate beyond JELMYTO that incorporates RTGel technology; and the Company’s ability to attract or retain key management, members of the board of directors and other personnel. These and other factors are described in greater detail under the “Risk Factors” heading of the Company’s Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2024, filed with the SEC on May 13, 2024. All information provided in this Current Report on Form 8-K is as of the date of this Current Report on Form 8-K, and any forward-looking statements contained herein are based on assumptions that the Company believes to be reasonable as of this date. Undue reliance should not be placed on the forward-looking statements in this report, which are based on information available to us on the date hereof. The Company undertakes no duty to update this information unless required by law.
| Item 9.01 | Financial Statements and Exhibits. (d) Exhibits. |
| Exhibit |
Description | |
| 99.1 | Company Presentation, dated June 13, 2024 | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) | |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
| Date: June 13, 2024 | UROGEN PHARMA LTD. | |||||
| By: | /s/ Don Kim | |||||
| Don Kim | ||||||
| Chief Financial Officer | ||||||
Exhibit 99.1 New Horizons in Bladder Cancer: UGN-102 Duration of 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-à-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 10
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: üRecurrence üProgression üDeath 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 22
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 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. ENVISION is a Phase 3, single-arm, open-label study in patients with LG-IR-NMIBC. 28
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 with UGN-102 alone 29 Based on Kaplan-Meier (KM) Estimates.
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 reduce mild to moderate AEs overall burden on patients 30 UroGen Data on File
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
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' Implementation Science Methods Unit • Associate member of Lineberger Comprehensive Cancer Center • Research program quantifies the impact of treatment for chronic health conditions on symptom burden, identifies important gaps in implementing evidence-based practices in clinics, and determines how those gaps are related to poor patient and clinic outcomes • Dr. Stover’s research program is funded by NIH, PCORI, AHRQ, Pfizer Global, and foundations 32
ENVISION PATIENT PERCEPTIONS OF UGN-102 AND TURBT: IMPACT ON DAILY ACTIVITIES 33
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 resection of bladder tumor [TURBT]) • N=29 U.S. patients (out of 39 eligible [74%]) 34 34 Stover and Smith, under review
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 35 35 Stover and Smith, under review
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 Smith, under review
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 time weeks I felt better and things they do it [TURBT], it's just little like that, and I start to exercise, bit more incontinence. It's but I do believe that I was not at gotten much worse with each my 100 percent until many procedure.” (17) weeks later.” (27) 37 Stover and Smith, under review
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 went in, and I went back to my normal activity…I had no problems at all.” (37) 38 Stover and Smith, under review
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 depending on what I'm doin'—but had an impact. Again, postponing sometimes my pants get wet, and travel, meetings, things like that, I have to go out to the car, get my but I was present, mostly, I was clothes, change, and they're present. I do think the outcome is lookin' for me….Runnin' to the better with the gel than with the bathroom all the time, TURBT.” (27) everybody'll, ‘Well, you’re always in the bathroom’.” (17) 39 Stover and Smith, under review
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 everything. The second time, I look forward to spending the rest knew what it was. Knowing what it of my life attached to a was, the scary part went away. I catheter. That’s a no-no for me.” wasn't worried anymore. It was just (08) toughing it out that one or two days.” (29) 40 Stover and Smith, under review
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] was more painful, bleeding. I don’t think he realized there was a lot of blood came that it was gonna be that long of out — not a little, a lot.” (27) a bleeding…He told me that I would be bleeding for ‘a while’ after the surgery.” (43) 41 Stover and Smith, under review Stover and Smith, under review
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].” (27) surgeries they keep doin’, my bladder—especially since I’ve “You have this medicine in you been doing it so young—that it and you don't wanna be having would mess up good things like sex with your partner…but once erectile function, things like that was done, everything was peeing...” (41) back to normal.” (15) 42 Stover and Smith, under review
From the patient perspective, UGN-102 meets an unmet need in care delivery for a non-surgical treatment alternative for NMIBC 43 43
PATIENT INTERVIEW: JULIO LAGO 44
45 45
THOUGHT LEADER PANEL 46
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 Institute to deliver a personalized approach to bladder cancer utilizing cutting edge precision medicine approaches. Jennifer Linehan, MD is a board-certified urologist, and is an Associate Professor of Urology and Urologic Oncology at the Saint John’s Cancer Institute. She also practices general urology, including both male and female voiding dysfunction and treatment for kidney stones. James McKiernan, MD, the John K. Lattimer Professor of Urology, is the chair of the Department of Urology of the College of Physicians and Surgeons at Columbia University Irving Medical Center and urologist-in-chief at New York Presbyterian/Columbia. Dr. McKiernan is only the sixth physician to hold this title since the founding of the department in 1917. Sandip Prasad, MD, is s a member of Garden State Urology and serves currently as the director of Genitourinary Surgical Oncology and Vice-Chair of Urology at Morristown Medical Center/Atlantic Health System in New Jersey. He is also a Clinical Associate Professor at Rutgers NJMS and a Clinical Assistant Professor at Thomas Jefferson University. He has published over 60 peer-reviewed journal articles and book chapters and serves as an associate editor or editorial reviewer for nine specialty journals in Urology. Angela Stover, PhD, Associate Professor, Department of Health Policy and Management at UNC Gillings School of Global Public Health, is a health services researcher with expertise in patient-reported outcomes (PRO) methods and implementation science. She co-directs the NC TraCS' Implementation Science Methods Unit and is an associate member of Lineberger Comprehensive Cancer Center. 47 47
WHAT IS NEXT? 48
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
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
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 for LG-IR-NMIBC patients Prolonged Generally well RTGel uniquely >86% of patients disease-free tolerated treats what you interviewed would intervals can see and what recommend you can’t UGN-102 51 51 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
Q&A 52
THANK YOU! 53
APPENDIX 54
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 (Primary Endpoint Assessment) Follow -up Period Cystoscopy, for cause biopsy, and urine cytology every 3 months for up to 24 Progression to months and every 6 months thereafter Residual disease HG disease to assess for recurrence or progression First recurrence or progression SOC according to SOC according to following CR or NCR treating physician treating physician SOC according to treating physician End of study visit End of study visit 55 SOC: Standard of Care
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 Episodes, n (%) Yes 229 (95.4) No 11 (4.6) Treatment Course, n (%) 6 instillations 228 (95.0) < 6 instillations 12 (5.0) UroGen Data on File 56
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