Protara Therapeutics, Inc. · NASDAQ:TARA
Protara Therapeutics — Investor Presentation (April 2024)
41 slides from Protara Therapeutics's investor presentation, Apr 5, 2024
Slide 1: Corporate Presentation March 2024
CORPORATE PRESENTATION March 2024
Slide 2: Forward Looking Statements
FORWARD LOOKING STATEMENTS Statements contained in this presentation regarding matters that are not historical facts are "forward looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 . Protara may, in some cases, use terms such as “predicts,” “believes,” “po
Slide 3: Encouraging Interim NMIBC Data; De-risked Rare Disease Programs
ENCOURAGING INTERIM NMIBC DATA; DE - RISKED RARE DISEASE PROGRAMS © 2024 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute 3 *Patient contribution across trials: ADVANCED - 1 Ph 1a = 3 patients; ADVANCED - 1 Ph 1b = 8 patients; ADVANCED - 12 Ph 2 naïve cohort = 5 patients Data cu
Slide 4: Led by a Team of Experienced Professionals
LED BY A TEAM OF EXPERIENCED PROFESSIONALS 4 Jesse Shefferman Co - founder, Director, Chief Executive Officer Pat Fabbio Chief Financial Officer Jacqueline Zummo, PhD, MPH, MBA Co - founder, Senior Vice President, Chief Scientific Operations Officer Justine O’Malley Senior Vice President, Investor R
Slide 5: Multiple Value Creation Opportunities Across Our Pipeline
MULTIPLE VALUE CREATION OPPORTUNITIES ACROSS OUR PIPELINE 5 *Granted Orphan Drug Designations by the U.S. FDA **TARA - 002 granted Rare Pediatric Disease designation by the FDA and orphan drug designation by the European Medicines Agency for the treatment of LMs. Definitions: CPI=Checkpoint inhibito
Slide 6: Financing Takes Us Through Multiple Catalysts by 2026
FINANCING TAKES US THROUGH MULTIPLE CATALYSTS BY 2026 6 © 2024 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute *Designed to be registrational **Financing would support acceleration of these programs 1H’24 2H’24 1H’25 2H’25** NMIBC: BCG - Naïve POC Current pooled preliminary 3 -
Slide 7: Lyophilized, Inactivated Group A Streptococcus pyogenes
TARA - 002 Lyophilized, Inactivated Group A Streptococcus pyogenes
Slide 8: Broad Immunopotentiator with History of Oncology Use in Japan
8 TARA - 002: BROAD IMMUNOPOTENTIATOR WITH HISTORY OF ONCOLOGY USE IN JAPAN TARA - 002 is an investigational, genetically distinct strain of Streptococcus pyogenes that is inactivated while retaining its immune - stimulating properties TARA - 002 is manufactured under cGMP conditions from the same M
Slide 9: Broad Immunopotentiation = Potential for Durable Response
Activates Th1 Immune Cascade (1)(2)(3) BROAD IMMUNOPOTENTIATION = POTENTIAL FOR DURABLE RESPONSE Mechanism similar to BCG, unique to all other agents in development 1. Fujimoto T., et al. J Immunol. 1997: 5619. | 2. Ryoma Y, et al. Anticancer Res. 2004; 3295 - 3298. | 3. Zhao H, et al. Microbiol. Im
Slide 10: Non-Muscle Invasive Bladder Cancer
TARA - 002 Non - Muscle Invasive Bladder Cancer
Slide 11: Interim Data Demonstrates Encouraging Activity
TARA - 002 INTERIM 3 - MONTH DATA DEMONSTRATES ENCOURAGING ACTIVITY 11 CONFIDENTIAL ©2024 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute 100% 63% 0% 14% 13% 33% 38% 60% 50% 43% 0% 20% 40% 80% 100% Unresponsive/Exposed Naïve Total CR % CIS - only CIS +Ta/T1 TOTAL N=5/8 N=2/2 N=
Slide 12: Mouse Model Suggests Durable Effect, Supports Modifications
Apparent 4x clearance to immune exhausting dose TARA - 002 MOUSE MODEL SUGGESTS DURABLE EFFECT, SUPPORTS MODIFICATIONS Kaplan Meyer survival curve. Mantel - Cox Test, Bonferroni correction . *, P<0.05; **, P<0.01; ***, P<0.001 1 1 ) Evidence of delayed onset to peak therapeutic effect – onset at dos
Slide 13: Durable Responses, Sustained Activity Following Onset
TARA - 002, Intravesical instillation 40KE HED Vehicle: 0.9% saline, Intravesical instillation DURABLE RESPONSES, SUSTAINED ACTIVITY FOLLOWING ONSET TARA - 002 exhibited long - term anti - tumor effect in MB49 orthotopic mouse model. In vivo bioluminescence images of tumor development during and aft
Slide 14: Priming, Reinduction, and Maintenance Expected to Enhance Responses
PRIMING, REINDUCTION, AND MAINTENANCE EXPECTED TO ENHANCE TARA - 002 RESPONSES 14 Exploratory Cohorts C and D in ADVANCED - 2 (financing use of proceeds) CONFIDENTIAL ©2024 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute Currently in ADVANCED - 2 P • • REINDUCTION • 5/8 CIS+Ta/
Slide 15: Demonstrated Synergy with Anti-PD1, Preview of Papillary Strategy
15 DEMONSTRATED SYNERGY WITH ANTI - PD1, PREVIEW OF PAPILLARY STRATEGY CONFIDENTIAL ©2024 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute In - Vitro Evidence of Immune - Activation Murine study in non - immunoresponsive cancer model CD4 T cells CD8 + T cells T cells isolated fr
Slide 16: Adverse Events Safety Profile
16 Grade 5 AEs (result in death) Grade 4 AEs (life - threatening) Grade 3 AEs (severe) Grade 2 AEs (moderate) Grade 1 Adverse Events (AEs) (mild) - - 4 22 132 Total AEs for all dosing levels (16 CIS “ Ta/T1 and 6 HGTa) None None All Grade 3 AEs were deemed to be not treatment - related: Hypoxia 1 Cr
Slide 17: Administration Is Among the Easiest of NMIBC Treatments
TARA - 002 ADMINISTRATION IS AMONG THE EASIEST OF NMIBC TREATMENTS 17 TARA - 002 No vial thaw, simple preparation; no pre/post treatment protocol EG - 70 No vial thaw, simple preparation; no pre/post treatment protocol N - 803/BCG - TICE Risk of infection for patient and caregivers; urine decontamin
Slide 18: High-Risk, High Grade NMIBC: A Significant Addressable Market in the US
HIGH - RISK, HIGH GRADE NMIBC: A SIGNIFICANT ADDRESSABLE MARKET IN THE US 18 Even in highly competitive scenarios, the market is large enough to sustain multiple entrants X US HR/HG NMIBC Prevalence - ~45% (1) = X Annual recurrence based on 3 yr US recurrence rate ~80% (2) = Over 90K HR/HG NMIBC ann
Slide 19: Differentiated Profile in NMIBC with Encouraging Interim Data
© 2024 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute 19 TARA - 002 HAS A DIFFERENTIATED PROFILE IN NMIBC WITH ENCOURAGING INTERIM DATA LOGISTIC PROFILE IDEAL FOR COMMUNITY SETTING • No additional administration procedures or safety protocols required vs. other products BROAD
Slide 20: IV Choline Chloride
IV CHOLINE CHLORIDE Phospholipid substrate replacement therapy for patients dependent on parenteral nutrition (PN)
Slide 21: Overview of Parenteral Nutrition
OVERVIEW OF PARENTERAL NUTRITION 21 Crohn's 23% Mesenteric Ischemia 19% Chronic Pseudo - Obstruction 23% Mucosal Disease 5% Radiation Enteritis 5% Familial Polyposis 4% Other 21% Underlying Etiology (1) Over time, dependent on PN Liver disease a well - known consequence of PN Patients are dependent
Slide 22: FDA Has Cleared the Way for PK/PD-Based Endpoints for Pivotal Study
FDA HAS CLEARED THE WAY FOR PK/PD - BASED ENDPOINTS FOR OUR PIVOTAL STUDY 22 1. Buchman et al, Clin Nutr, 1993 | 2. Chawla R, et al. Am J Clin Nutr. 1986 42:577 - 584. | 3. Zeisel S, et al. Neurology. 1980 30:1226 - 1229. Definitions: IV, intravenous; PN, parenteral nutrition. © 2024 Protara Therape
Slide 23: Independent Studies Show IV Choline Restores Choline Levels and Improves Steatosis
INDEPENDENT STUDIES DEMONSTRATE THAT TREATMENT WITH IV CHOLINE RAPIDLY RESTORES CHOLINE LEVELS AND IMPROVES STEATOSIS 23 CONFIDENTIAL ©2024 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute PLASMA FREE CHOLINE LEVELS: ALL PATIENTS (1) Choline supplementation was discontinued at w
Slide 24: Single, Pivotal Seamless 2b/3 Study with PK Endpoint
SINGLE, PIVOTAL SEAMLESS 2B/3 STUDY WITH PK ENDPOINT Protara Confidential Information OLE = open label extension Seamless Phase 2b/3 double - blinded, randomized, placebo - controlled THRIVE - 3 trial to assess the safety and efficacy of IV Choline Chloride in adolescents and adults on long - term P
Slide 25: Choline Replacement Recommended in Key PN Guidelines
CHOLINE REPLACEMENT RECOMMENDED IN KEY PN GUIDELINES 25 Parenteral Nutrition Professional Societies’ Position on Choline Guidelines / Position Paper ASPEN 2012 Position Paper (Vanek et al.) 3 : • Includes recommendations for Multivitamins & Multi - Trace Element PN Products • Recognises the impact o
Slide 26: Lymphatic Malformations (LMs)
TARA - 002 Lymphatic Malformations (LMs)
Slide 27: TARA-002 in Lymphatic Malformations
27 TARA - 002 IN LMs LYMPHATIC MALFORMATIONS Rare, non - malignant lesions consisting of dilated, lymphatic fluid - filled sacs caused by abnormal development of the lymphatic endothelial system (1) EPIDEMIOLOGY Epidemiology: incidence of lymphatic malformations is ≈1,400 - 1,800 LM cases per year (
Slide 28: Clear Evidence of Biologic Activity Observed with OK-432
CLEAR EVIDENCE OF BIOLOGIC ACTIVITY OBSERVED WITH OK - 432* 28 Completed clinical study of OK - 432 (TARA - 002 predecessor therapy) in U.S. suggests effectiveness with strong support for safety profile BEFORE AFTER BEFORE AFTER BEFORE AFTER BEFORE AFTER CONFIDENTIAL ©2024 Protara Therapeutics. All
Slide 29: Robust Clinical Results in Large Academic Study of OK-432
ROBUST CLINICAL RESULTS IN LARGE, ACADEMIC STUDY OF OK - 432 29 69% CLINICAL SUCCESS ǂ IN IMMEDIATE TREATMENT GROUP 6 MONTHS AFTER ENROLLMENT 84% * CLINICAL SUCCESS ǂ IN PATIENTS WITH MACROCYSTIC LESION TYPES 69% 7.5% Immediate Treatment Group (N=110) Delayed Treatment Group Pre - Treatment** (N=40)
Slide 30: In LMs: Phase 2 STARBORN-1 Trial Underway
30 TARA - 002 IN LMS: PHASE 2 STARBORN - 1 TRIAL UNDERWAY Single Arm Open - Label Safety and Efficacy Study of TARA - 002 in Pediatric Patients with Macrocystic and Mixed - cystic LMs (N=29) 6 years to < 18 years 2 years to < 6 years 6 months to < 2 years Phase 2a: Safety Lead - In Phase 2b: Expansi
Slide 31: Summary
SUMMARY
Slide 32: Encouraging Interim NMIBC Data; De-risked Rare Disease Programs
ENCOURAGING INTERIM NMIBC DATA; DE - RISKED RARE DISEASE PROGRAMS © 2024 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute *Patient contribution across trials: ADVANCED - 1 Ph 1a = 3 patients; ADVANCED - 1 Ph 1b = 8 patients; ADVANCED - 12 Ph 2 naïve cohort = 5 patients Data cut
Slide 33: Appendix
V7 Witter Uti aa JE f0 CO 0090000 eee APPENDIX
Slide 34: In NMIBC: Demographics and Disease Characteristics from Pooled Patients
34 [56 - 90] 73 Median age, years [range] Sex, number 81% 13 Men 19% 3 Women Race/Ethnicity 100% 16 White - Black - Asian - Other 6% 1 Hispanic or Latino 94% 15 Non - Hispanic or non - Latino Baseline ECOG 31% 5 1 63% 10 2 6% 1 3 TARA - 002 IN NMIBC: DEMOGRAPHICS AND DISEASE CHARACTERISTICS FROM POO
Slide 35: In NMIBC: Phase 1 Clinical Trial Design
Expansion TARA - 002 IN NMIBC: PHASE 1 CLINICAL TRIAL DESIGN PHASE 1 Dose finding, open - label trial with expansion evaluating intravesical TARA - 002 in adults with high - grade NMIBC Phase 1a: Dose Escalation 1 Phase 1b: Expansion Cohort 1 3+3 Dose Esc alation Design Enroll 10 KE 20 KE 40 KE NMIB
Slide 36: In NMIBC: ADVANCED-2 Trial Design
TARA - 002 IN NMIBC: ADVANCED - 2 TRIAL DESIGN 36 REGISTRATIONAL DESIGN : BCG - Unresponsive (CIS +/ - Ta/T1) Primary endpoint of high - grade complete response (CR) at any time at 6mos; Key secondary of 12 - month DOR Maintenance (months 6 - 18) 3 weekly instillations every 3 months Induction (N=27
Slide 37: Bladder Cancer: Significant Unmet Need with Limited Treatment Options
BLADDER CANCER: SIGNIFICANT UNMET NEED WITH LIMITED TREATMENT OPTIONS 37 Currently approved therapies for BCG - unresponsive NMIBC were approved on the basis of single arm trials FDA BCG - UN; Significant unmet need 1. National Cancer Institute. SEER Bladder Cancer – Stat Facts. Accessed April 25, 2
Slide 38: Manufacturing Is a Potential Competitive Advantage
TARA - 002: MANUFACTURING IS A POTENTIAL COMPETITIVE ADVANTAGE 38 x 20 million vial/year capacity with ability to 5x supply as needed x 47 successful batches run to date, 15 at commercial scale x Capable of manufacturing cGMP commercial product in preparation of Chugai partial change application com
Slide 39: Choline Deficiency Results in Hepatobiliary Pathophysiology
CHOLINE DEFICIENCY RESULTS IN HEPATOBILIARY PATHOPHYSIOLOGY 39 Diminished Choline = Insufficient phosphatidylcholine (PC), resulting in hepatobiliary injury ▪ PC is the primary lipid of the VLDL particle surface mono - layer. Low PC levels inhibit VLDL packaging and secretion. Without sufficient VLD
Slide 40: IV Choline: Supportive Evidence Across Independently Conducted Clinical Studies
IV CHOLINE: SUPPORTIVE EVIDENCE ACROSS 4 INDEPENDENTLY CONDUCTED CLINICAL STUDIES 40 1995 - Choline Pilot Study (2) n=4 PN patients IV Choline replacement reversed steatosis, improved other measures of hepatobiliary injury 1994 – Choline PK Study (1) n=4 PN patients 1 st continuous exposure to IV Ch
Slide 41: Deeply Experienced Board of Directors
DEEPLY EXPERIENCED BOARD OF DIRECTORS 41 Luke Beshar Chairman Roger Garceau, MD Director Barry Flannelly, PharmD, MBA Director Jane Huang, M.D. Director Richard Levy, MD Director Greg Sargen Director Cynthia Smith, MBA Director Michael Solomon, PhD Director CONFIDENTIAL ©2024 Protara Therapeutics. A
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