Urogen Pharma Ltd. · NASDAQ:URGN
Urogen Pharma — Investor Presentation (January 2018)
25 slides from Urogen Pharma's investor presentation, Jan 8, 2018
Slide 1: Changing the Standard of Care for Urothelial Cancers
Changing the Standard of Care for Urothelial Cancers January 2018 Exhibit 99.1
Slide 2: Forward-looking statements
This presentation and the accompanying oral presentation by UroGen Pharma Ltd. (“UroGen”) contains forward-looking statements. All statements contained herein other than statements of historical fact constitute forward-looking statements, including statements regarding UroGen’s anticipated results o
Slide 3: Our Strategy
Our Strategy Obtain first FDA drug approval for the treatment of Upper Tract Urothelial Carcinoma (UTUC) (FDA Orphan Drug & Fast Track Designations Granted) 1 Changing the treatment paradigm for low-grade non-muscle invasive bladder cancer (NMIBC) 2 Enable treatment of high-grade urothelial carcinom
Slide 4: Significant Market Opportunity in Urologic Cancers
Bladder cancer is one of the top 10 most common cancers in the world There is a lack of FDA-approved drugs for the treatment of urinary tract diseases Significant Market Opportunity in Urologic Cancers UTUC: Upper Tract Urothelial Carcinoma No drugs approved by FDA NMIBC: Non-Muscle Invasive Bladder
Slide 5: Limitations of Current Treatment Options & Procedures
Limitations of Current Treatment Options & Procedures UT Physiology Limits Drug Exposure Upper Tract = ~ 5 Minutes Bladder = ~ 30 Minutes Constant urine creation Bladder and upper tract movement Voiding Drug washes out before it has a chance to work properly UT Anatomy Limits Surgical Therapy Upper
Slide 6: Current Standard of Care for UTUC & NMIBC
Current Standard of Care for UTUC & NMIBC Repeated Cycle of Surgical Therapy in Urology
Slide 7: Potential to Create New First-Line Treatment Option
Potential to Create New First-Line Treatment Option Avoids the need for hospitalization, general anesthesia and associated risks of surgery TURBT1 surgery has limitations due to the inability to properly identify, reach and resect all tumors In UTUC, inability to resect tumors often results in kidne
Slide 8: Our Innovative RTGel Platform Technology in Urology
Storage Room temperature Gel form Pre-Instillation Low temperature Gel to Liquid form During Instillation Low temperature Liquid form In Bladder/Upper Tract Body temperature Gel form LT BT (1) RTGel: Reverse Thermal Gelation Hydrogel. RTGel(1): Liquid at low temperature (LT) and converts into gel fo
Slide 9: The Management Team
Arie Belldegrun, MD, FACS Chairman Ron Bentsur Chief Executive Officer Gil Hakim President, Israel Operation Gary Titus Chief Financial Officer Mark Schoenberg, MD Chief Medical Officer Christine Cassiano Corporate Affairs Officer Elyse Seltzer, MD SVP, Clinical Development Paul Chu VP, Business Dev
Slide 10: The Board of Directors
Arie Belldegrun, MD, FACS Chairman Ron Bentsur Chief Executive Officer Kate Falberg Audit Committee Chair Cynthia Butitta Fred Cohen, MD Stuart Holden, MD Ran Nussbaum Pini Orbach, PhD The Board of Directors: Established Industry Leadership
Slide 11: Clinical Development Pipeline
Clinical Development Pipeline Product Candidate Proposed Indication Phase 1 Phase 2 Phase 3 Next Milestone Commercial Rights CHEMOABLATION MitoGel (Orphan) Low Grade Upper Tract Urothelial Carcinoma (UTUC) Ph 3 Data = Q3 2018 expected UroGen VesiGel Low Grade NMIBC Submit IND; Ph 2b Trial Initiation
Slide 12: Validating the RTGel Platform Beyond Oncology
Validating the RTGel Platform Beyond Oncology Exclusive license agreement with Allergan Potential to evolve from multiple injections of BOTOX into the bladder to a single instillation into the bladder Up to $225 million ($25 million already received and $200 million in pending milestones) and tiered
Slide 13: MitoGel for Low Grade UTUC
MitoGel™ For Low Grade UTUC RTGel / MMC1 sustained release formulation Potential to become first drug ever approved as a first-line chemoablation treatment of low-grade UTUC FDA Orphan Drug Designation FDA Fast Track Designation Planned NDA filing in Q1 2019 Phase 3 1 MMC: Mitomycin C
Slide 14: MitoGel: Proof of Concept in UTUC
Localized treatment of hard to resect tumors; extensive tumor burden; some with solitary kidney Conducted in the United States, Europe and Israel >130 instillations performed Observed to be generally well-tolerated and feasible to administer MitoGel: Proof of Concept in UTUC 22 Patients with UTUC tr
Slide 15: MitoGel: Pivotal Trial Design and Expected Timing
Design: Single pivotal, open-label, single-arm, Phase 3 trial Patient Group: Patients with low-grade UTUC Number of Patients: ~70 patients Treatment Regimen: Six weekly instillations of MitoGel MitoGel: Pivotal Trial Design and Expected Timing Recruiting and Treating Patients Trial Starts End Recrui
Slide 16: VesiGel for Low-Grade NMIBC
VesiGel™ For Low-Grade NMIBC RTGel / high dose MMC sustained-release formulation Potential alternative to TURBT Potential first-line chemoablation treatment of low-grade NMIBC Phase 2a
Slide 17: VesiGel: Chemoablation Proof of Concept
Patient Group: Patients with low-grade NMIBC Number of Evaluable Patients: Phase 1: 15 (not shown) Phase 2a: 65 DE: 12 with LG Treatment Regimen: Six weekly instillations No TURBT Primary Efficacy Endpoint: CR at ~four weeks after last instillation Phase 2a Dose Escalation (DE) CR % 20 22 12 VesiGel
Slide 18: VesiGel: Potential Alternative to TURBT
Eligibility for TURBT: Tumor size: > 1cm Multifocal: > 3 tumors Shortcomings of TURBT: Nonvisible lesions Incomplete resection Hospitalization Anesthesia VesiGel vs. Active Control: Higher CR rates in TURBT eligible group VesiGel can allow for increased coverage of the bladder tissue designed to ove
Slide 19: VesiGel: Durable CRs in Clinical Program
% of patients treated with VesiGel with complete response (CR) over 12 months ~80% durability at 12 months No additional treatments were given during this period 26 26 23 20 Durable CR % 22 28 28 25 22 24 100% 100% 89.3% 85.7% 78.6% VesiGel: Durable CRs in Clinical Program
Slide 20: Vesimune for CIS Bladder Cancer
Vesimune For CIS Bladder Cancer Novel Imiquimod formulation for bladder instillation Orphan Drug Designation for CIS bladder cancer Potential local immunotherapy for the treatment of CIS bladder cancer Phase 1b CIS = Carcinoma in Situ
Slide 21: Vesimune: Preliminary Proof of Concept Data
Patient Group: Patients with NMIBC Number of Evaluable Patients: DE: 23 (not shown) Phase 1: 10 (under U.S. IND) Treatment Regimen: Six weekly instillations No TURBT to CIS patients Primary Efficacy Endpoint: CR at 5-7 weeks post last instillation Vesimune: Preliminary Proof of Concept Data Novel lo
Slide 22: 70+ Patents & Patent Applications
70+ Patents & Patent Applications 13 Granted in US 20 Granted Ex-US Broad IP Coverage in Five Interrelated Disciplines 3 4 Pharmaceutical Composition Methods of Treatment 1 Combination Therapy Delivery System for Bladder and Upper UT Manufacturing Process 2 3 4 5
Slide 23: UroGen Highlights
Enable treatment of high-grade urothelial carcinoma with immunotherapy drug, custom made for urology Emerging leader in the treatment of urothelial cancers and other urological indications Potential for first ever FDA drug approval for the treatment of Upper Tract Cancer Changing the treatment parad
Slide 24: Thank you
Thank you
Slide 25: UroGen: Transforming Treatment of Urothelial Cancers
UroGen: Transforming Treatment of Urothelial Cancers UroGen’s Proprietary Sustained Release Technology Improve therapeutic profiles of existing drugs Enable longer exposure to medications and improve localized therapy Treat non-muscle invasive urothelial cancers (LG upper tract carcinoma and bladder