UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
CURRENT REPORT
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| Item 8.01 | Other Events. |
On September 24, 2019, Urogen Pharma, Ltd. (the “Company”) issued a press release titled “UroGen Reports Positive Data from Two Important Studies: UGN-101 OLYMPUS Pivotal Trial in LG UTUC and UGN-102 Phase 2b OPTIMA II Trial in LG Bladder Cancer” (the “Data Press Release”). A copy of the Data Press Release is filed herewith as Exhibit 99.1 and is incorporated herein by reference.
On September 24, 2019, the Company delivered a presentation to investors via webcast relating to, among other things, the data that is the subject of the Data Press Release. A copy of the presentation is filed herewith as Exhibit 99.2 and incorporated herein by reference.
On September 24, 2019, the Company issued a press release titled “UroGen Investor Day Details Positive Clinical Updates, UGN-101 Launch Preparedness, and Pipeline Advances” (the “Recap Press Release”). A copy of the Recap Press Release is filed herewith as Exhibit 99.3 and incorporated herein by reference.
| Item 9.01 | Financial Statements and Exhibits. |
(d)
| Exhibit |
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| 99.1 |
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| 99.2 |
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| 99.3 |
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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 hereunto duly authorized.
| Date: September 24, 2019 |
UROGEN PHARMA LTD. | |||||
| By: |
/s/ Peter Pfreundschuh | |||||
| Peter Pfreundschuh | ||||||
| Chief Financial Officer | ||||||
Exhibit 99.1
UroGen Reports Positive Data from Two Important Studies: UGN-101 OLYMPUS Pivotal Trial in LG UTUC and UGN-102 Phase 2b OPTIMA II Trial in LG Bladder Cancer
UGN-101:
Consistent Complete Response (CR) Rate of 59 Percent in Patients with Low-Grade Upper Tract Urothelial Cancer (LG UTUC)
Durability of Response Determined to be 89 Percent at Six Months and 84 Percent at 12 Months
Rolling NDA Submission on Track for Q4 2019 with Planned Launch in 1H 2020
UGN-102:
Data Demonstrates CR Rate of 63 Percent in Patients with Intermediate Risk Low-Grade Non-Muscle Invasive Bladder Cancer (LG NMIBC)
Company Completes Enrollment Ahead of Schedule in OPTIMA II Phase 2b Trial
NEW YORK, September 24, 2019 — UroGen Pharma Ltd. (Nasdaq: URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of uro-oncology, today announced updated findings from the UGN-101 Phase 3 OLYMPUS Trial in patients with low-grade upper tract urothelial cancer (LG UTUC), as well as initial CR data from the UGN-102 Phase 2b OPTIMA II Trial in patients with intermediate risk low-grade non-muscle invasive bladder cancer (LG NMIBC).
Results from a final analysis of the primary endpoint for pivotal Phase 3 OLYMPUS showed that investigational UGN-101 (mitomycin gel) for instillation demonstrated a 59 percent CR rate in patients with LG UTUC. Findings were consistent with previously presented results.
The final analysis of the primary endpoint showed that in the OLYMPUS intent-to-treat population, 42 of the 71 patients (59 percent) achieved a CR. Forty-one patients entered follow-up, which is still ongoing. Durability of response was determined by Kaplan-Meier to be 89 percent at 6 months and 84 percent at 12 months after primary disease evaluation (PDE). The estimated median time-to-recurrence was 13.0 months. Thirty four of the 71 patients treated in the study were initially characterized by the treating physician as having endoscopically unresectable tumor at baseline. Twenty of 34 patients (59 percent) achieved a CR at the PDE assessment and 12-month durability was identical for this subgroup.
In OLYMPUS, the most common treatment emergent adverse events (TEAE) included ureteral stenosis, urinary tract infection, hematuria, flank pain, dysuria, renal impairment, hydronephrosis and frequency. Most TEAEs were characterized as mild to moderate and transient. Sixty-seven percent (48/71) of patients in the trial experienced an adverse event involving the renal/urinary tract. Of these, 23 percent (11/48) did not require surgical intervention, 50 percent (24/48) required temporary ureteral stent placement, 23 percent (11/48) required a long-term ureteral stent and 4 percent (2/48) required nephroureterectomy. At the time of database lock, the most common Grade 3 TEAE’s included ureteral stenosis (8.5 percent), hematuria, flank pain, and urinary tract infection (3 percent each). There was one Grade 4 TEAE of subdural hematoma (1.4 percent).
“We are pleased that the six-month durability from this analysis remains consistent with previously presented results and are very pleased with the durability observed at 12 months in evaluated
patients. These findings provide further support for the concept of chemoablation with UGN-101 as an initial kidney-sparing treatment option for patients with LG UTUC,” said Liz Barrett, President and Chief Executive Officer of UroGen. “We are on track to complete our New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in Q4 2019 and will be prepared for anticipated approval and launch in 1H 2020 of the first drug for the non-surgical treatment of LG UTUC.”
The Company also presented interim results from the Phase 2b OPTIMA II trial of investigational UGN-102 (mitomycin gel) for intravesical instillation for patients with intermediate risk LG NMIBC, defined as those with one or two of the following criteria: multifocal disease, large tumors and rapid rates of recurrence. The single-arm, open label study trial recently completed enrollment of 62 patients at clinical sites across the U.S. and Israel. Patients are treated with six weekly instillations of UGN-102 and undergo assessment of CR (the primary endpoint) four to six weeks following the last instillation. In an interim cohort of 32 patients, 63 percent (20/32) achieved a CR.
| Response Rate | ||
| Overall (n=32) | ||
| CR at PDE | 63% (20/32) |
“Achieving our enrollment goal ahead of schedule is a testament to the enthusiasm and need for this type of innovative approach to treatment in LG NMIBC. ‘Intermediate risk’ patients experience what can be viewed as a form of surgical failure, and many undergo multiple surgical procedures, known as transurethral resection of bladder tumor (TURBT), to manage these recurrences. We are encouraged by the data observed in this tough-to-treat population for whom the standard of care is really not effective,” said Mark Schoenberg, MD, Chief Medical Officer of UroGen. “While OPTIMA II remains ongoing and a Phase 3 study is anticipated, the results presented today further support our belief that UGN-102 has the potential to be an effective treatment option for this patient population of approximately 80,000, as there are no other options for these patients aside from repetitive surgical intervention. Based on literature, these patients have a high likelihood of recurrence at one year due to the chronicity of this disease, so we will continue to follow them and assess durability at 12 months.”
In the interim data from OPTIMA II, the most common adverse events observed were dysuria, pollakiuria, fatigue, hematuria and urinary tract infection. The majority of these treatment-emergent adverse events were characterized as mild or moderate and transient.
The Company intends to initiate a pivotal Phase 3 trial in 2020 following discussion with the FDA.
About The Phase 3 OLYMPUS Trial
OLYMPUS (Optimized DeLivery of Mitomycin for Primary UTUC Study) is a pivotal, open-label, single-arm Phase 3 clinical trial of UGN-101 (mitomycin gel) for instillation to evaluate the safety, tolerability and tumor ablative effect of UGN-101 in patients with low-grade UTUC. The trial enrolled 71 patients at clinical sites across the United States and Israel. Study participants were treated with six weekly instillations of UGN-101 administered via a standard catheter. Four to six weeks following the last instillation, patients underwent a PDE to determine response, the primary endpoint of the study. PDE involved a ureteroscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer. Patients who achieved a CR at the PDE timepoint were then followed for up to 12 months to determine the durability of disease control with UGN-101.
About UGN-101
UGN-101 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the
RTGel™ technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-101 is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-101 is delivered to patients using standard ureteral catheters. The Company initiated its rolling submission of the UGN-101 New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in December 2018. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of UTUC. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC.
About The Phase 2b OPTIMA II Trial
OPTIMA II (OPTimized Instillation of Mitomycin for BlAdder Cancer Treatment) is an open-label, single-arm, multi-center Phase 2b clinical trial of UGN-102 (mitomycin gel) for intravesical instillation to evaluate the safety and efficacy of UGN-102 in patients with intermediate risk low-grade non-muscle invasive bladder cancer (LG NMIBC) at intermediate risk of recurrence. Intermediate risk of progression is defined as one or two of the following: multiple tumors, solitary tumor >3 cm, or recurrence (³ 1 occurrence of LG NMIBC within 1 year of the current diagnosis). The trial enrolled 62 patients at clinical sites across the United States and Israel. Study participants were treated with six weekly instillations of UGN-102 administered via a standard intravesical catheter. Four to six weeks following the last instillation, patients undergo a PDE to determine response, the primary endpoint of the study. PDE involves a cystoscopy and wash cytology, a standard microscopic test of cells obtained from the urine to detect cancer. Patients who achieve a CR at the PDE timepoint are then followed for up to 9 months to determine the durability of disease control with UGN-102.
About UGN-102
UGN-102 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin in Phase 2b development for the treatment of low-grade non-muscle invasive bladder cancer (LG NMIBC). Utilizing the RTGel™ Technology Platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using standard intravesical catheters. The Company completed enrollment in the Phase 2b OPTIMA II trial of UGN-102 for the treatment of LG NMIBC in September 2019 and intends to advance the program to a pivotal study to further investigate UGN-102 in the treatment of this condition.
About UroGen Pharma Ltd.
UroGen Pharma Ltd. (Nasdaq:URGN) is a clinical-stage biopharmaceutical company developing advanced non-surgical treatments to address unmet needs in the field of urology, with a focus on uro-oncology. UroGen has developed RTGel™ reverse-thermal hydrogel, a proprietary sustained release, hydrogel-based platform technology that has the potential to improve therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. UroGen’s lead investigational candidates, UGN-101 (mitomycin gel) for instillation, and UGN-102 (mitomycin gel) for intravesical instillation, are designed to ablate tumors by non-surgical means and to treat several forms of non-muscle invasive urothelial cancer, including low-grade upper tract urothelial cancer and low-grade non-muscle invasive bladder cancer, respectively. UroGen is headquartered in New York, NY with operations in Los Angeles, CA and Israel.
Forward-Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation: the potential of UGN-101 for LG UTUC; the timing for completion of the rolling NDA for UGN-101; the potential approval of UGN-101 and the timing thereof; the expectation that UGN-101, if approved, will be the first drug approved for the non-surgical treatment of LG UTUC; the expected readiness of UroGen for a potential commercial launch of UGN-101 in 1H 2020; the potential of UroGen’s proprietary RTGel™
technology platform to improve therapeutic profiles of existing drugs; the opportunity and potential of UGN-102 for LG NMIBC; and the planned initiation of a Phase 3 pivotal study of UGN-102 in LG NMIBC. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: the timing and success of clinical trials, including the OLYMPUS Phase 3 trial and the OPTIMA II Phase 2b trial and potential safety and other complications thereof; the ability to obtain regulatory approval within the timeframe expected, or at all; the ability to maintain regulatory approval; complications associated with achieving commercial readiness for the launch of a new product; the labeling for any approved product; the scope, progress and expansion of developing and commercializing UroGen’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; 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 SEC on August 9, 2019, 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 press release and are based on information available to UroGen as of the date of this release.
UROGEN CONTACT:
Kate Bechtold
Senior Director, Investor Relations
Kate.Bechtold@urogen.com
914-552-0456
Exhibit 99.2 INVESTOR DAY SEPTEMBER 24, 2019Exhibit 99.2 INVESTOR DAY SEPTEMBER 24, 2019
OPENING REMARKS ARIE BELLDEGRUN, MD, FACS Chairman, UroGen PharmaOPENING REMARKS ARIE BELLDEGRUN, MD, FACS Chairman, UroGen Pharma
UROGEN: BUILDING A GROWTH COMPANY LIZ BARRETT Chief Executive Officer, UroGen PharmaUROGEN: BUILDING A GROWTH COMPANY LIZ BARRETT Chief Executive Officer, UroGen Pharma
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 potential of UGN-101 for LG UTUC; the timing for completion of the rolling NDA for UGN-101; the potential approval of UGN-101 and the timing thereof; the expectation that UGN-101, if approved, will be the first drug approved for the non-surgical treatment of LG UTUC; the timing for completion of pre-commercial activities and infrastructure build-out in anticipation of a potential commercial launch of UGN-101; the expected readiness of UroGen for a potential commercial launch of UGN-101 in 1H 2020 and the strength and timing of the potential commercial launch of UGN-101; plans for distribution and product packaging for UGN-101; plans for the retention of field-based personnel in support of the launch of UGN-101; the potential of UroGen’s proprietary RTGel™ technology platform to improve therapeutic profiles of existing drugs; the opportunity and potential of UGN-102 for LG NMIBC; plans to commence a pivotal trial for UGN-102 in LG NMIBC in 2020; UGN-102’s potential to replace current standard of care in LG NMIBC; plans to initiate a Phase 1 study with UGN-201; UroGen’s anticipated status relating to Q3’ 2019 financial guidance; plans to build a sustainable growth company; projections of revenue opportunities in markets of interest; plans to develop a global footprint; plans with Janssen to conduct an early stage feasibility evaluation in an area of mutual interest; and the anticipated completion of a Phase 2 trial of RTGel with Botox. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: the timing and success of clinical trials, including the OLYMPUS Phase 3 trial and the OPTIMA II Phase 2b trial and potential safety and other complications thereof; the ability to obtain regulatory approval within the timeframe expected, or at all; the ability to maintain regulatory approval; complications associated with achieving commercial readiness for the launch of a new product; the labeling and packaging for any approved product; the scope, progress and expansion of developing and commercializing UroGen’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; 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 SEC on August 9, 2019, 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.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 potential of UGN-101 for LG UTUC; the timing for completion of the rolling NDA for UGN-101; the potential approval of UGN-101 and the timing thereof; the expectation that UGN-101, if approved, will be the first drug approved for the non-surgical treatment of LG UTUC; the timing for completion of pre-commercial activities and infrastructure build-out in anticipation of a potential commercial launch of UGN-101; the expected readiness of UroGen for a potential commercial launch of UGN-101 in 1H 2020 and the strength and timing of the potential commercial launch of UGN-101; plans for distribution and product packaging for UGN-101; plans for the retention of field-based personnel in support of the launch of UGN-101; the potential of UroGen’s proprietary RTGel™ technology platform to improve therapeutic profiles of existing drugs; the opportunity and potential of UGN-102 for LG NMIBC; plans to commence a pivotal trial for UGN-102 in LG NMIBC in 2020; UGN-102’s potential to replace current standard of care in LG NMIBC; plans to initiate a Phase 1 study with UGN-201; UroGen’s anticipated status relating to Q3’ 2019 financial guidance; plans to build a sustainable growth company; projections of revenue opportunities in markets of interest; plans to develop a global footprint; plans with Janssen to conduct an early stage feasibility evaluation in an area of mutual interest; and the anticipated completion of a Phase 2 trial of RTGel with Botox. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: the timing and success of clinical trials, including the OLYMPUS Phase 3 trial and the OPTIMA II Phase 2b trial and potential safety and other complications thereof; the ability to obtain regulatory approval within the timeframe expected, or at all; the ability to maintain regulatory approval; complications associated with achieving commercial readiness for the launch of a new product; the labeling and packaging for any approved product; the scope, progress and expansion of developing and commercializing UroGen’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; 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 SEC on August 9, 2019, 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.
AGENDA • Opening Remarks • Management Q & A Arie Belldegrun, MD, FACS, Chairman Liz Barrett, Mark Schoenberg, Peter Pfreundschuh, Jeff Bova + • UroGen: Building a Growth Company Extended Team Liz Barrett, CEO • Closing Remarks • Clinical Update Liz Barrett, CEO Mark Schoenberg, MD, CMO • Innovation in Practice: KOL Panel and Q&A Mark Schoenberg (Moderator), MD, CMO Karim Chamie, MD, MSHS Jennifer Linehan, MD Phil Pierorazio, MD Sandip Prasad, MD Dan Saltzstein, MDAGENDA • Opening Remarks • Management Q & A Arie Belldegrun, MD, FACS, Chairman Liz Barrett, Mark Schoenberg, Peter Pfreundschuh, Jeff Bova + • UroGen: Building a Growth Company Extended Team Liz Barrett, CEO • Closing Remarks • Clinical Update Liz Barrett, CEO Mark Schoenberg, MD, CMO • Innovation in Practice: KOL Panel and Q&A Mark Schoenberg (Moderator), MD, CMO Karim Chamie, MD, MSHS Jennifer Linehan, MD Phil Pierorazio, MD Sandip Prasad, MD Dan Saltzstein, MD
OUR TEAM Liz Barrett Peter Pfreundschuh Mark Schoenberg, MD Stephen Mullennix Jeff Bova Woody Bryan, PhD Chief Executive Officer Chief Financial Officer Chief Medical Officer Chief Operating Officer SVP, Commercial SVP, Business Development Leadership Marina Konorty, PhD Jim Ottinger, RPh Elyse Seltzer, MD John O’Reilly Dalit Strauss-Ayali SVP, R&D & Head of Israel Operations SVP, Regulatory Affairs SVP, Clinical Development VP, Associate General Counsel VP, Non-clinical Research, Science, Discovery Board of Directors Arie Belldegrun, MD, FACS Cynthia Butitta Fred E. Cohen, MD Kate Falberg Stuart Holden, MD Ran Nussbaum Shawn C. TomaselloOUR TEAM Liz Barrett Peter Pfreundschuh Mark Schoenberg, MD Stephen Mullennix Jeff Bova Woody Bryan, PhD Chief Executive Officer Chief Financial Officer Chief Medical Officer Chief Operating Officer SVP, Commercial SVP, Business Development Leadership Marina Konorty, PhD Jim Ottinger, RPh Elyse Seltzer, MD John O’Reilly Dalit Strauss-Ayali SVP, R&D & Head of Israel Operations SVP, Regulatory Affairs SVP, Clinical Development VP, Associate General Counsel VP, Non-clinical Research, Science, Discovery Board of Directors Arie Belldegrun, MD, FACS Cynthia Butitta Fred E. Cohen, MD Kate Falberg Stuart Holden, MD Ran Nussbaum Shawn C. Tomasello
UROGEN: BUILDING A GROWTH COMPANY TM • RTGel Technology • Near-Term Catalysts • UGN-101 • The Road to Anticipated Launch • UGN-102 • UGN-201 • Collaborations/Partnerships • Long-Term Strategy • Leading in Uro-Oncology & BeyondUROGEN: BUILDING A GROWTH COMPANY TM • RTGel Technology • Near-Term Catalysts • UGN-101 • The Road to Anticipated Launch • UGN-102 • UGN-201 • Collaborations/Partnerships • Long-Term Strategy • Leading in Uro-Oncology & Beyond
STRONG MOMENTUM ACROSS PIPELINESTRONG MOMENTUM ACROSS PIPELINE
UNLOCKING THE URO-ONCOLOGY MARKET LOW-GRADE UPPER TRACT LOW-GRADE NON-MUSCULAR UROTHELIAL CARCINOMA (UTUC) INVASIVE BLADDER CANCER (NMIBC) ~80,000 ~6,000 – 7,000 No available Last drug approved intermediate-risk LG addressable FDA-approved >15 years ago NMIBC population medicinesUNLOCKING THE URO-ONCOLOGY MARKET LOW-GRADE UPPER TRACT LOW-GRADE NON-MUSCULAR UROTHELIAL CARCINOMA (UTUC) INVASIVE BLADDER CANCER (NMIBC) ~80,000 ~6,000 – 7,000 No available Last drug approved intermediate-risk LG addressable FDA-approved >15 years ago NMIBC population medicines
KEY ACCOMPLISHMENTS IN 2019 • Completed phase III OLYMPUS trial for UGN-101 in LG UTUC; obtained breakthrough therapy designation and advanced rolling submission with FDA • Fully enrolled phase 2b study of UGN-102 in intermediate-risk low-grade NMIBC and conducted interim analysis • Enhanced the pipeline with UGN-201 preclinical work and announcement of the early-stage feasibility agreement with Janssen • Organizational and commercial readiness for anticipated launch of UGN-101 • Developed long-term vision and strategy for sustainable growth • Strong cash position and delivery of guidance TM Established RTGel as the first innovation UroGen expects to bring to market KEY ACCOMPLISHMENTS IN 2019 • Completed phase III OLYMPUS trial for UGN-101 in LG UTUC; obtained breakthrough therapy designation and advanced rolling submission with FDA • Fully enrolled phase 2b study of UGN-102 in intermediate-risk low-grade NMIBC and conducted interim analysis • Enhanced the pipeline with UGN-201 preclinical work and announcement of the early-stage feasibility agreement with Janssen • Organizational and commercial readiness for anticipated launch of UGN-101 • Developed long-term vision and strategy for sustainable growth • Strong cash position and delivery of guidance TM Established RTGel as the first innovation UroGen expects to bring to market
UROGEN: BUILDING A GROWTH COMPANY OUR PIPELINE: UGN-101UROGEN: BUILDING A GROWTH COMPANY OUR PIPELINE: UGN-101
UROTHELIAL CARCINOMA COMMON, COSTLY CANCER WITH SIGNIFICANT QOL IMPACT LOW-GRADE UPPER TRACT UROTHELIAL CARCINOMA (UTUC) Urothelial carcinoma (UC) Cancer that happens in the lining of the kidneys or the ureters is the 9th most common LOW-GRADE UTUC 1 cancer globally • Kidney-sparing treatments are achievable and may decrease overtreatment and loss of renal units • 70%-80% of LG UTUC patients receive UC is the most costly cancer nephroureterectomies in the US health care system 1 on a per-patient basis Yeung et al. (2014) Pharmacoeconomics UROTHELIAL CARCINOMA COMMON, COSTLY CANCER WITH SIGNIFICANT QOL IMPACT LOW-GRADE UPPER TRACT UROTHELIAL CARCINOMA (UTUC) Urothelial carcinoma (UC) Cancer that happens in the lining of the kidneys or the ureters is the 9th most common LOW-GRADE UTUC 1 cancer globally • Kidney-sparing treatments are achievable and may decrease overtreatment and loss of renal units • 70%-80% of LG UTUC patients receive UC is the most costly cancer nephroureterectomies in the US health care system 1 on a per-patient basis Yeung et al. (2014) Pharmacoeconomics
POTENTIAL OPPORTUNITY IS ~6,000 US PATIENTS Newly Incident: 2,500-2,700 LG UTUC US P op u la tion P reva len ce: Recurrent: 330 M 15,000-18,000 3,000-4,000 Remission: 10,000-11,800 ~6,000 potential patients annually.POTENTIAL OPPORTUNITY IS ~6,000 US PATIENTS Newly Incident: 2,500-2,700 LG UTUC US P op u la tion P reva len ce: Recurrent: 330 M 15,000-18,000 3,000-4,000 Remission: 10,000-11,800 ~6,000 potential patients annually.
UPDATED UGN-101 TOPLINE RESPONSE RATE Endoscopically Unresectable Overall (n=71) Tumors 48% (34/71) CR Rate 59% 59% 6-month CR Durability* 89% 85% 12-month* 84% 84% • Majority of adverse events were reported as mild or moderate • Serious adverse events occurred in 36.6% of patients, most notably ureteric stenosis, hydronephrosis, flank pain, and urosepsis v More complete discussion of UGN-101’s efficacy and safety profile is reserved for clinical presentation. *Kaplan-Meier AnalysisUPDATED UGN-101 TOPLINE RESPONSE RATE Endoscopically Unresectable Overall (n=71) Tumors 48% (34/71) CR Rate 59% 59% 6-month CR Durability* 89% 85% 12-month* 84% 84% • Majority of adverse events were reported as mild or moderate • Serious adverse events occurred in 36.6% of patients, most notably ureteric stenosis, hydronephrosis, flank pain, and urosepsis v More complete discussion of UGN-101’s efficacy and safety profile is reserved for clinical presentation. *Kaplan-Meier Analysis
ON TRACK FOR NDA SUBMISSION: ROADMAP TO ANTICIPATED FDA APPROVAL OF UGN-101 Pre-Clinical Clinical Fast Track Designation NDA NDA Potential 08/2017 12/2019 Q4 2019 1H 2020* UGN-101 09/2014 10/2018 2H 2019 Approval Breakthrough Orphan CMC Drug Therapy NDA Designation Designation *Assumes 6-month PDUFA based on Fast Track & Breakthrough Therapy DesignationsON TRACK FOR NDA SUBMISSION: ROADMAP TO ANTICIPATED FDA APPROVAL OF UGN-101 Pre-Clinical Clinical Fast Track Designation NDA NDA Potential 08/2017 12/2019 Q4 2019 1H 2020* UGN-101 09/2014 10/2018 2H 2019 Approval Breakthrough Orphan CMC Drug Therapy NDA Designation Designation *Assumes 6-month PDUFA based on Fast Track & Breakthrough Therapy Designations
UROGEN: BUILDING A GROWTH COMPANY THE ROAD TO ANTICIPATED LAUNCH: UGN-101UROGEN: BUILDING A GROWTH COMPANY THE ROAD TO ANTICIPATED LAUNCH: UGN-101
ORGANIZATIONAL READINESS: UROGEN IS ON TRACK TO BE LAUNCH READY FOR UGN-101 BY JANUARY 2020 PREPARE THE MARKET ü Field Medical Team hired and active ü Field National Account Directors calling on payers ü Increased awareness in urology community LAUNCH PREPARE THE BRAND READINESS ü Distribution strategy set and brand name selected ü Reimbursement support HUB established PREPARE THE COMPANY ü Full commercial leadership team hired ü Field leadership team hired and recruiting sales forceORGANIZATIONAL READINESS: UROGEN IS ON TRACK TO BE LAUNCH READY FOR UGN-101 BY JANUARY 2020 PREPARE THE MARKET ü Field Medical Team hired and active ü Field National Account Directors calling on payers ü Increased awareness in urology community LAUNCH PREPARE THE BRAND READINESS ü Distribution strategy set and brand name selected ü Reimbursement support HUB established PREPARE THE COMPANY ü Full commercial leadership team hired ü Field leadership team hired and recruiting sales force
UGN-101 OPPORTUNITY: UROLOGISTS RECOGNIZE THE NEED TO DELAY RADICAL SURGERY, AND IDENTIFY MULTIPLE OPPORTUNITIES TO INCORPORATE UGN-101 INTO THEIR TREATMENT OF LG UTUC, FOLLOWING ANTICIPATED FDA APPROVAL Treatment Continuum: Low-Grade UTUC INCIDENCE (NEWLY DIAGNOSED) RECURRENCE 76% RNU New LG UTUC patients 65% 65% 72% Additional Endoscopic Endoscopic RNU Management Management = Percent of urologists who are “likely/very likely to use” UGN-101 in each specific setting.UGN-101 OPPORTUNITY: UROLOGISTS RECOGNIZE THE NEED TO DELAY RADICAL SURGERY, AND IDENTIFY MULTIPLE OPPORTUNITIES TO INCORPORATE UGN-101 INTO THEIR TREATMENT OF LG UTUC, FOLLOWING ANTICIPATED FDA APPROVAL Treatment Continuum: Low-Grade UTUC INCIDENCE (NEWLY DIAGNOSED) RECURRENCE 76% RNU New LG UTUC patients 65% 65% 72% Additional Endoscopic Endoscopic RNU Management Management = Percent of urologists who are “likely/very likely to use” UGN-101 in each specific setting.
CREATIVE SOLUTIONS TO REMOVE BARRIERS • Hire experienced field teams with expertise in uro-oncology, rare disease UGN-101 • Strong marketing awareness efforts Awareness • Real-time patient alerts where possible • Early Payer engagement -- National Account Directors in field 2019 Ensure Reimbursement • Dedicated team of Field Reimbursement Managers + independent HUB services Confidence to support appropriate coding, benefits verifications, issue resolution • National Pharmacy partner will provide pre-mixed formulation to urology clinics Product • All-in-one convenience kit will be provided to hospital pharmacies who prefer to Distribution self-mixCREATIVE SOLUTIONS TO REMOVE BARRIERS • Hire experienced field teams with expertise in uro-oncology, rare disease UGN-101 • Strong marketing awareness efforts Awareness • Real-time patient alerts where possible • Early Payer engagement -- National Account Directors in field 2019 Ensure Reimbursement • Dedicated team of Field Reimbursement Managers + independent HUB services Confidence to support appropriate coding, benefits verifications, issue resolution • National Pharmacy partner will provide pre-mixed formulation to urology clinics Product • All-in-one convenience kit will be provided to hospital pharmacies who prefer to Distribution self-mix
ROLES TO SUPPORT ACCOUNT-BASED APPROACH 33% OF ACCOUNTS HAVE 90% OF THE PATIENT POTENTIAL Regional Business 7 RBM: Regional Business Manager Manager (RBM) Responsible for all commercial activity 48 TBM: Territory Business Manager Customer lead and demand generation Field Certified Nurse Reimbursement 7 FRM: Field Reimbursement Manager Educator (CNE) Manager (FRM) Account experts on billing and coding 7 CNE: Certified Nurse Educator Provide technical training and support for mixing and product instillation Territory Business Manager (TBM)ROLES TO SUPPORT ACCOUNT-BASED APPROACH 33% OF ACCOUNTS HAVE 90% OF THE PATIENT POTENTIAL Regional Business 7 RBM: Regional Business Manager Manager (RBM) Responsible for all commercial activity 48 TBM: Territory Business Manager Customer lead and demand generation Field Certified Nurse Reimbursement 7 FRM: Field Reimbursement Manager Educator (CNE) Manager (FRM) Account experts on billing and coding 7 CNE: Certified Nurse Educator Provide technical training and support for mixing and product instillation Territory Business Manager (TBM)
UGN-101 TREATMENT IS EXPECTED TO FIT WELL INTO EXISTING PHYSICIAN REIMBURSEMENT MODELS • Professional fee: Physicians expected to utilize existing CPT codes for UGN-101 instillation • Technical fee: Hospitals and surgery centers expected to use existing codes for facility reimbursement • Product reimbursement: UGN-101 will ultimately be reimbursed via a product-specific J-Code. In the interim, a C-Code will be utilized to facilitate smooth reimbursement in the hospital/ASC setting Misc C-code assigned Unique C-code Permanent J-code Product ApprovalUGN-101 TREATMENT IS EXPECTED TO FIT WELL INTO EXISTING PHYSICIAN REIMBURSEMENT MODELS • Professional fee: Physicians expected to utilize existing CPT codes for UGN-101 instillation • Technical fee: Hospitals and surgery centers expected to use existing codes for facility reimbursement • Product reimbursement: UGN-101 will ultimately be reimbursed via a product-specific J-Code. In the interim, a C-Code will be utilized to facilitate smooth reimbursement in the hospital/ASC setting Misc C-code assigned Unique C-code Permanent J-code Product Approval
PLANNED UGN-101 ALL-IN-ONE KIT: MAKING UGN-101 PREPARATION AND ADMINISTRATION CONVENIENT FOR PRACTITIONERS All-in-one kit will be available to any practice Network of pharmacies will be established to ensure in the US delivery of pre-mixed product to all practitioners needing mixing services Packaging Prototype UroGen will coordinate all logistics through a reimbursement support center for a seamless customer experience *The UGN-101 All-In-One Kit remains subject to ongoing internal development and future FDA evaluation. FDA approved packaging for UGN-101 may differ from current planned packaging.PLANNED UGN-101 ALL-IN-ONE KIT: MAKING UGN-101 PREPARATION AND ADMINISTRATION CONVENIENT FOR PRACTITIONERS All-in-one kit will be available to any practice Network of pharmacies will be established to ensure in the US delivery of pre-mixed product to all practitioners needing mixing services Packaging Prototype UroGen will coordinate all logistics through a reimbursement support center for a seamless customer experience *The UGN-101 All-In-One Kit remains subject to ongoing internal development and future FDA evaluation. FDA approved packaging for UGN-101 may differ from current planned packaging.
EXPERIENCED TEAM IS PRIMED FOR LAUNCH UPON APPROVAL ü Hired an internal team with a track record ü Launched a successful disease education of success in oncology campaign ü Hired a veteran sales force leadership team ü Tested and validated a launch campaign with deep uro-oncology relationships with urologists ü Consulted with our customers, and we are ü Developed partnerships with seasoned ready to deliver on their needs vendors • HCPs • Pharmacists ü Aligned and prepared for • Payers launch readiness January 2020EXPERIENCED TEAM IS PRIMED FOR LAUNCH UPON APPROVAL ü Hired an internal team with a track record ü Launched a successful disease education of success in oncology campaign ü Hired a veteran sales force leadership team ü Tested and validated a launch campaign with deep uro-oncology relationships with urologists ü Consulted with our customers, and we are ü Developed partnerships with seasoned ready to deliver on their needs vendors • HCPs • Pharmacists ü Aligned and prepared for • Payers launch readiness January 2020
UROGEN: BUILDING A GROWTH COMPANY OUR PIPELINE: UGN-102UROGEN: BUILDING A GROWTH COMPANY OUR PIPELINE: UGN-102
UGN-102 TOPLINE UGN-102 enrollment complete ahead Safety: Most AEs mild to moderate in of schedule severity, related to local tolerability, no related SAEs - CR 63%* *Interim CR based on half of patients v More complete discussion of UGN-102’s efficacy and safety profile is reserved for clinical presentation.UGN-102 TOPLINE UGN-102 enrollment complete ahead Safety: Most AEs mild to moderate in of schedule severity, related to local tolerability, no related SAEs - CR 63%* *Interim CR based on half of patients v More complete discussion of UGN-102’s efficacy and safety profile is reserved for clinical presentation.
BLADDER CANCER MARKET OPPORTUNITY POTENTIAL TO BE THE FIRST PRIMARY NON-SURGICAL CHEMOABLATIVE THERAPY FOR BLADDER CANCER LG NMIBC: Large Surgical SOC with high relapse Drug therapy outdated, unused rate for intermediate risk Patient Population 343 K prevalence 40 K incidence ~80 K intermediate risk High rate of relapse Drugs currently used only after TURBT (SOC) as adjuvant after surgery (10-20% of total LG NMIBC population) UGN-102: POTENTIAL TO REPLACE SOC: Moves care from OR to office/ASC with a potential to decrease cost and morbidity of contemporary therapy SEER Data – US UGN-102 is an investigational product candidate not approved by the FDABLADDER CANCER MARKET OPPORTUNITY POTENTIAL TO BE THE FIRST PRIMARY NON-SURGICAL CHEMOABLATIVE THERAPY FOR BLADDER CANCER LG NMIBC: Large Surgical SOC with high relapse Drug therapy outdated, unused rate for intermediate risk Patient Population 343 K prevalence 40 K incidence ~80 K intermediate risk High rate of relapse Drugs currently used only after TURBT (SOC) as adjuvant after surgery (10-20% of total LG NMIBC population) UGN-102: POTENTIAL TO REPLACE SOC: Moves care from OR to office/ASC with a potential to decrease cost and morbidity of contemporary therapy SEER Data – US UGN-102 is an investigational product candidate not approved by the FDA
UROGEN: BUILDING A GROWTH COMPANY OUR PIPELINE: UGN-201UROGEN: BUILDING A GROWTH COMPANY OUR PIPELINE: UGN-201
UGN-201 PROVIDES US WITH MULTIPLE SHOTS ON GOAL • UGN-201 is a TLR 7/8 agonist that is believed to stimulate innate and adaptive antitumor immunity. It likely works in conjunction with other potent immunoregulatory molecules • Preclinical experiments as monotherapy and in combination with checkpoint inhibitors provide signals of efficacy • Plan is to optimize combinations and move into human studies as soon as is feasibleUGN-201 PROVIDES US WITH MULTIPLE SHOTS ON GOAL • UGN-201 is a TLR 7/8 agonist that is believed to stimulate innate and adaptive antitumor immunity. It likely works in conjunction with other potent immunoregulatory molecules • Preclinical experiments as monotherapy and in combination with checkpoint inhibitors provide signals of efficacy • Plan is to optimize combinations and move into human studies as soon as is feasible
UROGEN: BUILDING A GROWTH COMPANY COLLABORATIONS & PARTNERSHIPSUROGEN: BUILDING A GROWTH COMPANY COLLABORATIONS & PARTNERSHIPS
ADVANCING LOCAL DELIVERY THROUGH STRATEGIC COLLABORATIONS Strategic collaborations in urology and oncology TM TM ALLERGAN JANSSEN Overactive bladder (phase 2) Early-stage feasibility evaluation RTGel has the potential to provide meaningful improvement over the current standard of care across urologic cancers and beyond *The above trademarks are the property of their respective owners.ADVANCING LOCAL DELIVERY THROUGH STRATEGIC COLLABORATIONS Strategic collaborations in urology and oncology TM TM ALLERGAN JANSSEN Overactive bladder (phase 2) Early-stage feasibility evaluation RTGel has the potential to provide meaningful improvement over the current standard of care across urologic cancers and beyond *The above trademarks are the property of their respective owners.
BUILDING UROGEN’S GLOBAL FOOTPRINT EUROPE * Gathering data for EU and Japan * Develop plan and decide on next steps-License/partner JAPANBUILDING UROGEN’S GLOBAL FOOTPRINT EUROPE * Gathering data for EU and Japan * Develop plan and decide on next steps-License/partner JAPAN
UROGEN: BUILDING A GROWTH COMPANY LEADING IN URO-ONCOLOGY & BEYONDUROGEN: BUILDING A GROWTH COMPANY LEADING IN URO-ONCOLOGY & BEYOND
UROGEN LEADERSHIP POSITION IN LOW-GRADE UROLOGIC DISEASE LG NON-MUSCLE INVASIVE HG NON-MUSCLE MUSCLE INVASIVE INVASIVE Upper Tract PD-L1s FKD Therapies BladderUROGEN LEADERSHIP POSITION IN LOW-GRADE UROLOGIC DISEASE LG NON-MUSCLE INVASIVE HG NON-MUSCLE MUSCLE INVASIVE INVASIVE Upper Tract PD-L1s FKD Therapies Bladder
UGN-101 & UGN-102 ADVANCE SOC AND PROVIDE A STRONG FOUNDATION TO BUILD A SUSTAINABLE COMPANY DRIVING INNOVATION IN URO-ONCOLOGY WITH RTGEL LG UPPER TRACT LG NON-MUSCLE INVASIVE UROTHELIAL CANCER BLADDER CANCER UGN-101 (LG) Intermediate-Risk LG- Phase 3 NMIBC Phase 2B Annual US addressable Annual US addressable market: ~6 K market: ~80 K ~$1 BILLION POTENTIAL PEAK REVENUE OPPORTUNITYUGN-101 & UGN-102 ADVANCE SOC AND PROVIDE A STRONG FOUNDATION TO BUILD A SUSTAINABLE COMPANY DRIVING INNOVATION IN URO-ONCOLOGY WITH RTGEL LG UPPER TRACT LG NON-MUSCLE INVASIVE UROTHELIAL CANCER BLADDER CANCER UGN-101 (LG) Intermediate-Risk LG- Phase 3 NMIBC Phase 2B Annual US addressable Annual US addressable market: ~6 K market: ~80 K ~$1 BILLION POTENTIAL PEAK REVENUE OPPORTUNITY
WE BUILD NOVEL SOLUTIONS TO TREAT SPECIALTY CANCERS AND UROLOGIC DISEASES BECAUSE PATIENTS DESERVE BETTER ADDRESS CHALLENGING DISEASE NIMBLE, SOLUTION- MAXIMIZE BENEFIT OF PATIENT CENTRICITY WITH TRANSFORMATIVE LOCAL DELIVERY ORIENTED ORGANIZATION THERAPIES Ensure patients who can benefit from our medicines have Through our nimble Leverage RTGel capabilities and access to them. approach, UroGen is designed to expertise where unique solutions develop and commercialize are needed to overcome Addressing high unmet need Provide a holistic approach with medicines faster and anatomical and biological barriers diseases in Urology & gyn/GI tools that help patients manage more efficiently while creating cancers their disease and live their best a dynamic environment Opportunistically gain access to lives possible for employees additional delivery platforms Must advance SOCWE BUILD NOVEL SOLUTIONS TO TREAT SPECIALTY CANCERS AND UROLOGIC DISEASES BECAUSE PATIENTS DESERVE BETTER ADDRESS CHALLENGING DISEASE NIMBLE, SOLUTION- MAXIMIZE BENEFIT OF PATIENT CENTRICITY WITH TRANSFORMATIVE LOCAL DELIVERY ORIENTED ORGANIZATION THERAPIES Ensure patients who can benefit from our medicines have Through our nimble Leverage RTGel capabilities and access to them. approach, UroGen is designed to expertise where unique solutions develop and commercialize are needed to overcome Addressing high unmet need Provide a holistic approach with medicines faster and anatomical and biological barriers diseases in Urology & gyn/GI tools that help patients manage more efficiently while creating cancers their disease and live their best a dynamic environment Opportunistically gain access to lives possible for employees additional delivery platforms Must advance SOC
CLINICAL UPDATE: UGN-101, UGN-102, UGN-201 MARK SCHOENBERG, MD Chief Medical Officer, UroGen PharmaCLINICAL UPDATE: UGN-101, UGN-102, UGN-201 MARK SCHOENBERG, MD Chief Medical Officer, UroGen Pharma
LASER RESECTION OF LG UTUC Courtesy of Dr. Scott Hubosky, Dept. of Urology, Jefferson University, Philadelphia, PALASER RESECTION OF LG UTUC Courtesy of Dr. Scott Hubosky, Dept. of Urology, Jefferson University, Philadelphia, PA
DEFINING LOW-GRADE VERSUS HIGH-GRADE DISEASE LOW GRADE HIGH GRADE • Chronic relapse • Progression – Metastasis & death • Current treatment – Repetitive surgery • Current treatment: – TURBT – Risks – BCG – Incidence: 42 K – Clinical trials – Prevalence: ~500 K – RCP/TMT • Incidence: ~18 K • Prevalence: ~200 K BCG is not used in low-grade diseaseDEFINING LOW-GRADE VERSUS HIGH-GRADE DISEASE LOW GRADE HIGH GRADE • Chronic relapse • Progression – Metastasis & death • Current treatment – Repetitive surgery • Current treatment: – TURBT – Risks – BCG – Incidence: 42 K – Clinical trials – Prevalence: ~500 K – RCP/TMT • Incidence: ~18 K • Prevalence: ~200 K BCG is not used in low-grade disease
THE UNMET MEDICAL NEED IN LG UTUC Repetitive endoscopic ablation RNU is a major 70%-80% • Average time to surgery with will lose recurrence: 6 months complications their kidney • Treats visible disease but not occult disease Complications associated 45% of patients have Elderly patient with loss of kidney unresectable tumors population • Chronic renal insufficiency at presentation • Risk of dialysis • Exacerbation of comorbidities (eg, cardiac disease)THE UNMET MEDICAL NEED IN LG UTUC Repetitive endoscopic ablation RNU is a major 70%-80% • Average time to surgery with will lose recurrence: 6 months complications their kidney • Treats visible disease but not occult disease Complications associated 45% of patients have Elderly patient with loss of kidney unresectable tumors population • Chronic renal insufficiency at presentation • Risk of dialysis • Exacerbation of comorbidities (eg, cardiac disease)
THE MUTATIONAL LANDSCAPE OF UCB DEMONSTRATES MAJOR DIFFERENCES IN INVASIVE AND NON-INVASIVE DISEASE 90% 80% MIBC NMIBC 70% 60% 50% 40% 30% 20% 10% 0% Hurst & Knowles (2017) Urol OncolTHE MUTATIONAL LANDSCAPE OF UCB DEMONSTRATES MAJOR DIFFERENCES IN INVASIVE AND NON-INVASIVE DISEASE 90% 80% MIBC NMIBC 70% 60% 50% 40% 30% 20% 10% 0% Hurst & Knowles (2017) Urol Oncol
MOLECULAR PROFILING REVEALS: LG BC=LG UTUC LG-NMIBC (n=82) IBC (n=126) Nassar et al. (2019) Clin Ca Res.MOLECULAR PROFILING REVEALS: LG BC=LG UTUC LG-NMIBC (n=82) IBC (n=126) Nassar et al. (2019) Clin Ca Res.
MOLECULAR ANALYSIS REVEALS “NORMAL TISSUE” HARBORS CANCER MUTATIONS Thomsen et al. (2017) Nature/Scientific ReportsMOLECULAR ANALYSIS REVEALS “NORMAL TISSUE” HARBORS CANCER MUTATIONS Thomsen et al. (2017) Nature/Scientific Reports
OLYMPUS TRIAL Enrollment Criteria Rationale • New & recurrent LG UTUC • LG UTUC like LG NMIBC – Solitary or multifocal • Anatomic complexity • Renal pelvis • Limitation of current tools • 5 mm-15 mm • RNU ~70%-80% • No HG, CIS • Cytology negative for HG • Partial resection permitted Goal: Decrease renal loss and avoid repetitive surgeryOLYMPUS TRIAL Enrollment Criteria Rationale • New & recurrent LG UTUC • LG UTUC like LG NMIBC – Solitary or multifocal • Anatomic complexity • Renal pelvis • Limitation of current tools • 5 mm-15 mm • RNU ~70%-80% • No HG, CIS • Cytology negative for HG • Partial resection permitted Goal: Decrease renal loss and avoid repetitive surgery
STUDY FLOW CHART FOR PROTOCOL TC-UT-03 Patients sign Patients undergo informed consent screening procedures Safety lab visit Safety lab visit TR1 TR2 TR3 TR4 TR5 TR6 Primary Disease Evaluation visit (PDE) Visit 1 Visit 2 Visit 3 Visit 4 Visit 5 Visit 6 Visit 9 Visit 7 Visit 8 5±1 wk post last instl 1 week post lab 3 week post lab Visit 0 Wash urine cytology + URS instillation instillation to assess ablative effect of Up to 4 week before V1 UGN-101. In case of lesions, biopsy, or brush biopsy Complete Complete was done response response YES? NO? Study Within 2-3 weeks following PDE visit, 11-monthly administration of UGN-101 completion F/U 12 months study TR9a TR6a TR3a completion TR11a TR10a TR8a TR7a TR5a TR4a TR2a TR1a +F/U +F/U +F/U Visit 21 Visit 20 Visit 19 Visit 18 Visit 17 Visit 16 Visit 15 Visit 14 Visit 13 Visit 12 Visit 11 Visit 10STUDY FLOW CHART FOR PROTOCOL TC-UT-03 Patients sign Patients undergo informed consent screening procedures Safety lab visit Safety lab visit TR1 TR2 TR3 TR4 TR5 TR6 Primary Disease Evaluation visit (PDE) Visit 1 Visit 2 Visit 3 Visit 4 Visit 5 Visit 6 Visit 9 Visit 7 Visit 8 5±1 wk post last instl 1 week post lab 3 week post lab Visit 0 Wash urine cytology + URS instillation instillation to assess ablative effect of Up to 4 week before V1 UGN-101. In case of lesions, biopsy, or brush biopsy Complete Complete was done response response YES? NO? Study Within 2-3 weeks following PDE visit, 11-monthly administration of UGN-101 completion F/U 12 months study TR9a TR6a TR3a completion TR11a TR10a TR8a TR7a TR5a TR4a TR2a TR1a +F/U +F/U +F/U Visit 21 Visit 20 Visit 19 Visit 18 Visit 17 Visit 16 Visit 15 Visit 14 Visit 13 Visit 12 Visit 11 Visit 10
OLYMPUS STUDY DATA SUMMARY (DATABASE LOCK: 5/22/19) RESPONSE RATE* 74 enrolled Endoscopically unresectable 71 treated Overall (n=71) tumors 87% Caucasian 48% (34/71) 68% male CR rate at 75% >65 yo 59% (42/71) 59% (20/34) PDE *76% of CR cohort through 6-month follow-up.OLYMPUS STUDY DATA SUMMARY (DATABASE LOCK: 5/22/19) RESPONSE RATE* 74 enrolled Endoscopically unresectable 71 treated Overall (n=71) tumors 87% Caucasian 48% (34/71) 68% male CR rate at 75% >65 yo 59% (42/71) 59% (20/34) PDE *76% of CR cohort through 6-month follow-up.
KAPLAN-MEIER CURVE OF DURABILITY OF RESPONSE DURING THE MAINTENANCE PERIOD (PDE ANALYSIS SET) CRKAPLAN-MEIER CURVE OF DURABILITY OF RESPONSE DURING THE MAINTENANCE PERIOD (PDE ANALYSIS SET) CR
MOST COMMON GRADE 3 OR HIGHER TREATMENT-EMERGENT ADVERSE EVENTS Graded as Severe Adverse Event N (%) 6 (8.5) Ureteric stenosis UTI 2 (2.8) Hematuria 2 (2.8) Flank pain 2 (2.8) Nausea 1 (1.4) Renal impairment 1 (1.4) Vomiting 3 (4.2) Abdominal pain 1 (1.4) Hydronephrosis 4 (5.6)MOST COMMON GRADE 3 OR HIGHER TREATMENT-EMERGENT ADVERSE EVENTS Graded as Severe Adverse Event N (%) 6 (8.5) Ureteric stenosis UTI 2 (2.8) Hematuria 2 (2.8) Flank pain 2 (2.8) Nausea 1 (1.4) Renal impairment 1 (1.4) Vomiting 3 (4.2) Abdominal pain 1 (1.4) Hydronephrosis 4 (5.6)
RENAL URINARY TOXICITY 48 patients 11 (23%) no surgical intervention 24 (50%) transient stent 11 (23%) long-term stent 2 (4%) RNURENAL URINARY TOXICITY 48 patients 11 (23%) no surgical intervention 24 (50%) transient stent 11 (23%) long-term stent 2 (4%) RNU
PEER REVIEWED LITERATURE: URETERAL STRICTURES FOLLOWING ENDOSCOPIC SURGERY Study N (%) Follow-Up months Complication/Stricture Rate N/% Schmeller et al 1989 16 (50) Median 14 4 (25) strictures Engelmeyer et al 1996 10 (70) Mean 43 2 (20) strictures 9/39 (23) strictures/ureteric Martinez-Pinero et al 1996 59 (24) Mean 31 perforations Daneshmand et al 2003 30 Mean 31 5 (17) strictures Reisiger et al 2007 10 Mean 73 1 (10) stricture Krambeck et al 2007 37 (100) Mean 32 19 (51) overall; 5 (14) strictures 12 (16) strictures; 1x bowel Cutress et al 2012 73 (11) Median 54 perforation with Nd:YAG laserPEER REVIEWED LITERATURE: URETERAL STRICTURES FOLLOWING ENDOSCOPIC SURGERY Study N (%) Follow-Up months Complication/Stricture Rate N/% Schmeller et al 1989 16 (50) Median 14 4 (25) strictures Engelmeyer et al 1996 10 (70) Mean 43 2 (20) strictures 9/39 (23) strictures/ureteric Martinez-Pinero et al 1996 59 (24) Mean 31 perforations Daneshmand et al 2003 30 Mean 31 5 (17) strictures Reisiger et al 2007 10 Mean 73 1 (10) stricture Krambeck et al 2007 37 (100) Mean 32 19 (51) overall; 5 (14) strictures 12 (16) strictures; 1x bowel Cutress et al 2012 73 (11) Median 54 perforation with Nd:YAG laser
OLYMPUS SUMMARY • 71 patients treated • 59% CR –Efficacy consistent in endoscopically unresectable pts • Durability stable at 6 and 12 months (84% per KM analysis) • AE profile –Consistent with upper tract manipulation for cancer –Stricture consistent with published literature • Follow-up ongoingOLYMPUS SUMMARY • 71 patients treated • 59% CR –Efficacy consistent in endoscopically unresectable pts • Durability stable at 6 and 12 months (84% per KM analysis) • AE profile –Consistent with upper tract manipulation for cancer –Stricture consistent with published literature • Follow-up ongoing
UGN-102 PROGRAM FOR INTERMEDIATE-RISK LG UBC • Similarities between LG NMIBC and LG UTUC –Histology –Recurrence pattern –Clinical behavior –High risk of recurrence –Low risk of progression • New molecular data • Sensitivity to locally administered drugs UGN-102 PROGRAM FOR INTERMEDIATE-RISK LG UBC • Similarities between LG NMIBC and LG UTUC –Histology –Recurrence pattern –Clinical behavior –High risk of recurrence –Low risk of progression • New molecular data • Sensitivity to locally administered drugs
CYSTOSCOPIC APPEARANCE OF TURBT Courtesy of Alex Sankin, MD, Montefiore Medical Center & The Albert Einstein College of Medicine, Bronx, NYCYSTOSCOPIC APPEARANCE OF TURBT Courtesy of Alex Sankin, MD, Montefiore Medical Center & The Albert Einstein College of Medicine, Bronx, NY
WHY INTERMEDIATE-RISK NMIBC? • “Surgical failure” cohort: risk of progression low, recurrence high • Cost & morbidity of repetitive surgery How many of the following 4 factors does the patient have? • Multiple tumors • Early recurrence (<1 year) • Tumor size >3cm • Frequent recurrences (>1 per year) 0 1-2≥3 Treatment similar to low Treatment as intermediate Treatment as high risk risk risk • TURBT + BCG induction + maintenance • TURBT + single immediate • TURBT plus adjuvant intravesical post-op chemotherapeutic dose, therapy or • Office fulguration • Intravesical chemotherapy Kamat et al. (2014) J. Urology WHY INTERMEDIATE-RISK NMIBC? • “Surgical failure” cohort: risk of progression low, recurrence high • Cost & morbidity of repetitive surgery How many of the following 4 factors does the patient have? • Multiple tumors • Early recurrence (<1 year) • Tumor size >3cm • Frequent recurrences (>1 per year) 0 1-2≥3 Treatment similar to low Treatment as intermediate Treatment as high risk risk risk • TURBT + BCG induction + maintenance • TURBT + single immediate • TURBT plus adjuvant intravesical post-op chemotherapeutic dose, therapy or • Office fulguration • Intravesical chemotherapy Kamat et al. (2014) J. Urology
OPTIMA II DESIGN • Patients with IR NMIBC (1-2/3: multifocal, lesion >3 cm, recurrence within 12 months) • UGN-102 q wk X 6 • Primary endpoint: CR at 3-month visit –CR pts followed quarterly x 9 months • Secondary endpoint: 12-month durability & safety OPTIMA II DESIGN • Patients with IR NMIBC (1-2/3: multifocal, lesion >3 cm, recurrence within 12 months) • UGN-102 q wk X 6 • Primary endpoint: CR at 3-month visit –CR pts followed quarterly x 9 months • Secondary endpoint: 12-month durability & safety
UGN-102 INTERIM ANALYSIS • The majority of adverse events RESPONSE RATE* were reported as mild or moderate; the most commonly reported AEs Total enrolled (n=32) were: –Dysuria (38%), CR rate 63% (20/32) –Hematuria (16%), –Frequency (13%), –Urinary tract infection (13%) *PDE based on evaluation at 3 months. Patients will continue to be followed for durability at 12 months.UGN-102 INTERIM ANALYSIS • The majority of adverse events RESPONSE RATE* were reported as mild or moderate; the most commonly reported AEs Total enrolled (n=32) were: –Dysuria (38%), CR rate 63% (20/32) –Hematuria (16%), –Frequency (13%), –Urinary tract infection (13%) *PDE based on evaluation at 3 months. Patients will continue to be followed for durability at 12 months.
NEXT STEPS FOR UGN-102 • Continued follow-up of patients in the trial • Discuss with FDA • Scenario planning for phase 3 pivotal study –Streamline & expedite registrational path –Randomized H2H vs TURBT likely • We are prepared to execute • Pivotal study planned for 2020NEXT STEPS FOR UGN-102 • Continued follow-up of patients in the trial • Discuss with FDA • Scenario planning for phase 3 pivotal study –Streamline & expedite registrational path –Randomized H2H vs TURBT likely • We are prepared to execute • Pivotal study planned for 2020
UGN-201 UC is an “immune” 201: TLR 7/8 agonist Hypothesis: responsive tumor • Activates “innate” Combinatorial immune response immunotherapy is • “Early” experience with feasible and clinically BCG (local) • May potentiate “adaptive” meaningful antitumor response • CPI trials for MIBC (systemic) • Phase 1b: “signal” when applied locally to CISUGN-201 UC is an “immune” 201: TLR 7/8 agonist Hypothesis: responsive tumor • Activates “innate” Combinatorial immune response immunotherapy is • “Early” experience with feasible and clinically BCG (local) • May potentiate “adaptive” meaningful antitumor response • CPI trials for MIBC (systemic) • Phase 1b: “signal” when applied locally to CIS
IN MICE: UGN-201 + aCTLA4/RTGEL RESULT IN BETTER SURVIVAL Study I Study II Study III ** 100 100 100 ** ** # 50 50 * 50 0 0 0 0 20 40 60 0 20 40 60 0 20 40 60 80 Days Days Days Vehicle + Isotype (solution) Vehicle + Isotype/RTGel Vehicle + Isotype/RTGel (Alternate) UGN-201 + Isotype/RTGel UGN-201 + Isotype/RTGel UGN-201 + Isotype/RTGel Vehicle + aCTLA4/RTGel UGN-201 + aCTLA4/RTGel UGN-201 + aCTLA4/RTGel UGN-201 + aCTLA4/RTGel *P ≤ 0.05 vs control **P ≤ 0.005 vs control #P ≤ 0.05 vs combo Percent survival Percent survival Percent survivalIN MICE: UGN-201 + aCTLA4/RTGEL RESULT IN BETTER SURVIVAL Study I Study II Study III ** 100 100 100 ** ** # 50 50 * 50 0 0 0 0 20 40 60 0 20 40 60 0 20 40 60 80 Days Days Days Vehicle + Isotype (solution) Vehicle + Isotype/RTGel Vehicle + Isotype/RTGel (Alternate) UGN-201 + Isotype/RTGel UGN-201 + Isotype/RTGel UGN-201 + Isotype/RTGel Vehicle + aCTLA4/RTGel UGN-201 + aCTLA4/RTGel UGN-201 + aCTLA4/RTGel UGN-201 + aCTLA4/RTGel *P ≤ 0.05 vs control **P ≤ 0.005 vs control #P ≤ 0.05 vs combo Percent survival Percent survival Percent survival
SUMMARY OF MAJOR FINDINGS FROM MURINE STUDIES • Intravesical UGN-201+aCTLA4/RTGel: –Smaller tumors and better survival rate –Decreases T regulatory cells –Increases the ratio of CD8+/T regulatory cells • Data support continued progression toward human trials • May represent a novel approach to HG NMIBC SUMMARY OF MAJOR FINDINGS FROM MURINE STUDIES • Intravesical UGN-201+aCTLA4/RTGel: –Smaller tumors and better survival rate –Decreases T regulatory cells –Increases the ratio of CD8+/T regulatory cells • Data support continued progression toward human trials • May represent a novel approach to HG NMIBC
SUMMARY UGN-101: First primary chemoablative therapy for urothelial cancer • Robust efficacy & durability in LG UTUC – CR: 59% – 12-month durability 84% (KM) including “unresectable” 59% pts 63% UGN-102 in IR NMIBC: • Interim data encouraging – CR: 63% – AE profile: mostly mild to moderate, local & transient UGN-201 Immunotherapy: • Local aCTLA4+UGN-201 increased survival UBC (Murine) SUMMARY UGN-101: First primary chemoablative therapy for urothelial cancer • Robust efficacy & durability in LG UTUC – CR: 59% – 12-month durability 84% (KM) including “unresectable” 59% pts 63% UGN-102 in IR NMIBC: • Interim data encouraging – CR: 63% – AE profile: mostly mild to moderate, local & transient UGN-201 Immunotherapy: • Local aCTLA4+UGN-201 increased survival UBC (Murine)
CREDIT WHERE CREDIT IS DUE • Elyse Seltzer, MD • Roman Bromblin • Jim Ottinger, RPh • Diana Licis • Marina Konorty, PhD • Eric Smith • Dalit Strauss-Ayali, PhD DVM • Madlen Malinowski • Ifat Klein, PhD • Tami Gerassi • Yael Agmon • Swati Chiktara • Nimrod Gabai • Ruby Salmo • Elinor Schreiber • Sunil Raju, MD • Dima Zolotaryov • Robert Kirshoff • Baruch Narotzki • Pallavi RajputCREDIT WHERE CREDIT IS DUE • Elyse Seltzer, MD • Roman Bromblin • Jim Ottinger, RPh • Diana Licis • Marina Konorty, PhD • Eric Smith • Dalit Strauss-Ayali, PhD DVM • Madlen Malinowski • Ifat Klein, PhD • Tami Gerassi • Yael Agmon • Swati Chiktara • Nimrod Gabai • Ruby Salmo • Elinor Schreiber • Sunil Raju, MD • Dima Zolotaryov • Robert Kirshoff • Baruch Narotzki • Pallavi Rajput
INNOVATION IN PRACTICE KOL Panel and Q&AINNOVATION IN PRACTICE KOL Panel and Q&A
MANAGEMENT Q&AMANAGEMENT Q&A
UROGEN: BUILDING A GROWTH COMPANY CLOSING REMARKSUROGEN: BUILDING A GROWTH COMPANY CLOSING REMARKS
IN SUMMARY UroGen is committed to leading in challenging areas of high unmet need UroGen is poised for near-term catalysts behind positive data in UGN-101 and UGN-102 UroGen will focus on urology and Gyn/GI oncology UroGen plans to leverage our proprietary expertise in RTGel but grow beyond local delivery UroGen is in a strong financial position that will take us through launch and advance the pipeline UroGen is designed to build a sustainable growth company while maintaining the culture of creativity and problem solving that got us hereIN SUMMARY UroGen is committed to leading in challenging areas of high unmet need UroGen is poised for near-term catalysts behind positive data in UGN-101 and UGN-102 UroGen will focus on urology and Gyn/GI oncology UroGen plans to leverage our proprietary expertise in RTGel but grow beyond local delivery UroGen is in a strong financial position that will take us through launch and advance the pipeline UroGen is designed to build a sustainable growth company while maintaining the culture of creativity and problem solving that got us here
Exhibit 99.3
UroGen Investor Day Details Positive Clinical Updates, UGN-101 Launch Preparedness, and Pipeline Advances
UGN-101 and UGN-102 Demonstrate Positive Clinical Data in Low-Grade Upper Tract Urothelial Cancer (LG UTUC) and Low-Grade Non-Muscle Invasive Bladder Cancer (LG NMIBC)
UGN-101 Final Data Modules for NDA Submission On-Track for Q4 2019
UGN-101 Plans for Launch Readiness by January 2020
UGN-201 Plans for Advancement into Phase 1 for High-Grade NMIBC in 2020
NEW YORK, September 24, 2019 — UroGen Pharma Ltd. (Nasdaq: URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of urology, today presented updates on its advancing pipeline for urologic cancers and UGN-101 launch readiness at its Investor Day in New York on September 24, 2019.
“At UroGen, we are 100 percent committed to overcoming obstacles that have stunted much needed innovation in uro-oncology,” said Liz Barrett, President and Chief Executive Officer of UroGen. “While we prepare for a potential launch of UGN-101, we believe this is just the beginning of what is possible with our pipeline. The data from an interim analysis for UGN-102 unveiled at our Investor Day further highlight the potential of our RTGel™ platform to transform treatments in this space for an even larger patient population with bladder cancer. We look forward to pushing innovation beyond our foundational platform and moving UGN-201, our TLR 7/8 agonist for high-grade bladder cancer, into clinical studies in 2020.”
UroGen detailed data updates for its lead investigational product candidates UGN-101 and UGN-102:
| • | UGN-101 (mitomycin gel) for instillation for patients with low-grade upper tract urothelial cancer (LG UTUC): |
| • | Complete response (CR) rate of 59 percent observed in 71 patients with LG UTUC from Phase 3 OLYMPUS trial. Data remain consistent with previously presented data. |
| • | Durability of response determined to be 89 percent at six months and 84 percent at twelve months. |
| • | In the OLYMPUS trial, the most common treatment emergent adverse events (TEAE) included ureteral stenosis, urinary tract infection, hematuria, flank pain, dysuria, renal impairment, hydronephrosis and frequency. Most TEAEs were characterized as mild to moderate and transient. |
| • | At the time of database lock, the most common Grade 3 TEAE’s included ureteral stenosis (8.5%), hematuria, flank pain, and urinary tract infection (3% each). There was one Grade 4 TEAE of subdural hematoma (1.4%). |
| • | Rolling NDA submission is on track for Q4 2019 with potential approval and launch in 1H 2020. |
| • | If approved, UGN-101 will be the first drug for the primary chemoablative treatment of LG UTUC. |
| • | UGN-102 (mitomycin gel) for intravesical instillation for patients with intermediate risk low-grade non-muscle invasive bladder cancer (LG NMIBC): |
| • | In an interim analysis, 63% (20/32) of patients from the Phase 2b OPTIMA II trial achieved a CR. |
| • | In an interim analysis, the most common treatment emergent adverse events (TEAEs) observed were dysuria, pollakiuria, fatigue, hematuria and urinary tract infection. The majority were characterized as mild or moderate and transient. |
| • | Patients will continue to be followed with 12-month durability to be reported at a later date. |
| • | Trial enrolled patients with intermediate risk LG NMIBC, defined as those with one or two of the following criteria: multifocal disease, large tumors and rapid rates of recurrence. |
| • | Trial completed enrollment of 62 patients ahead of schedule. |
| • | The Company intends to initiate a pivotal Phase 3 trial in 2020 following discussion with the FDA. |
UroGen discussed its ongoing activities to build awareness of unmet needs in UTUC, educate the market and commercialize UGN-101 following anticipated regulatory approval:
| • | Increased scientific awareness of UGN-101 clinical data developments in urologic community. |
| • | Engagement with payers and proactive market access strategy to ensure patient access and reimbursement. |
| • | UGN-101 treatment expected to fit well into existing physician reimbursement models. |
| • | Planned convenience kit for UGN-101 will facilitate preparation and administration for practitioners. |
| • | Experienced commercial team with track record of success in uro-oncology. |
| • | Nimble salesforce with seven Regional Business Managers (RBMs) hired, and 50 sales reps to be hired by end of 2019. |
The Company also provided an update on UGN-201, it’s investigational TLR 7/8 agonist for the treatment of high-grade NMIBC. UGN-201 is believed to stimulate innate and adaptive antitumor immunity. It likely works in conjunction with other potent immunoregulatory molecules. Nonclinical data shows an efficacy signal when UGN-201 is administered locally with anti-CTLA4 antibody in a murine model of high-grade bladder cancer. The Company plans to optimize combinations and advance into human studies.
About UGN-101
UGN-101 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of low-grade upper tract urothelial cancer (LG UTUC). Utilizing the RTGel™ technology platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-101 is designed to enable longer exposure of urinary tract tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-101 is delivered to patients using standard ureteral catheters. The Company initiated its rolling submission of the UGN-101 New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in December 2018. The FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to UGN-101 for the treatment of UTUC. If approved, UGN-101 would be the first drug approved for the non-surgical treatment of LG UTUC.
About UGN-102
UGN-102 (mitomycin gel) for instillation is an investigational drug formulation of mitomycin in Phase 2b development for the treatment of low-grade non-muscle invasive bladder cancer (LG NMIBC). Utilizing the RTGel™ Technology Platform, UroGen’s proprietary sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using standard intravesical catheters. The Company completed enrollment in the Phase 2b OPTIMA II trial of UGN-102 for the
treatment of LG NMIBC in September 2019 and intends to advance the program to a pivotal study to further investigate UGN-102 in the treatment of this condition.
About UroGen Pharma Ltd.
UroGen Pharma Ltd. (Nasdaq:URGN) is a clinical-stage biopharmaceutical company developing advanced non-surgical treatments to address unmet needs in the field of urology, with a focus on uro-oncology. UroGen has developed RTGel™ reverse-thermal hydrogel, a proprietary sustained release, hydrogel-based platform technology that has the potential to improve therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. UroGen’s lead investigational candidates, UGN-101 (mitomycin gel) for instillation, and UGN-102 (mitomycin gel) for intravesical instillation, are designed to potentially ablate tumors by non-surgical means and to treat several forms of non-muscle invasive urothelial cancer, including low-grade upper tract urothelial cancer and low-grade non-muscle invasive bladder cancer, respectively. UroGen is headquartered in New York, NY with operations in Los Angeles, CA and Israel.
Forward-Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation: the potential of UGN-101 for LG UTUC; the timing for completion of the rolling NDA for UGN-101; the potential approval of UGN-101 and the timing thereof; the expectation that UGN-101, if approved, will be the first drug approved for the non-surgical treatment of LG UTUC; the timing for completion of pre-commercial activities and infrastructure build-out in anticipation of a potential commercial launch of UGN-101; the expected readiness of UroGen for a potential commercial launch of UGN-101 in 1H 2020 and the strength and timing of the potential commercial launch of UGN-101; plans for the retention of field-based personnel in support of the launch of UGN-101; the potential of UroGen’s proprietary RTGel™ technology platform to improve therapeutic profiles of existing drugs; the opportunity and potential of UGN-102 for LG NMIBC; plans to commence a pivotal trial for UGN-102 in LG NMIBC in 2020; UGN-102’s potential to replace current standard of care in LG NMIBC; and plans to initiate a Phase 1 study with UGN-201. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: the timing and success of clinical trials, including the OLYMPUS Phase 3 trial and the OPTIMA II Phase 2b trial and potential safety and other complications thereof; the ability to obtain regulatory approval within the timeframe expected, or at all; the ability to maintain regulatory approval; complications associated with achieving commercial readiness for the launch of a new product; the labeling and packaging for any approved product; the scope, progress and expansion of developing and commercializing UroGen’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; 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 SEC on August 9, 2019, 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 press release and are based on information available to UroGen as of the date of this presentation.
UROGEN CONTACT:
Kate Bechtold
Senior Director, Investor Relations
Kate.Bechtold@urogen.com
914-552-0456