Protara Therapeutics, Inc. · NASDAQ:TARA
Protara Therapeutics — Investor Presentation (January 2025)
35 slides from Protara Therapeutics's investor presentation, Jan 13, 2025
Slide 1: Corporate Presentation January 2025
CORPORATE PRESENTATION January 2025
Slide 2: Forward Looking Statements
FORWARD LOOKING STATEMENTS 2 © 2025 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute 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 1
Slide 3: Promising NMIBC therapy and de-risked rare disease programs
Promising NMIBC therapy C de - risked rare disease programs TARA - 002 in NMIBC • Dosing underway in Phase 2 STARBORN - 1 trial • TARA - 002 predecessor is standard of care in Japan • U.S. FDA granted Rare Pediatric Disease Designation – PRV eligible • Positive interim results from ADVANCED - 2 tria
Slide 4: Multiple upcoming opportunities across our pipeline
Multiple upcoming opportunities across our pipeline 4 *Granted Orphan Drug Designations by the U.S. FDA **TARA - 002 granted Rare Pediatric Disease designation by the U.S. FDA and orphan drug designation by the European Medicines Agency for the treatment of LMs. PK=Pharmacokinetic © 2025 Protara The
Slide 5: Multiple anticipated near-term milestones
Multiple anticipated near - term milestones 5 **Does not include 10.8M common warrants issued with the April 2024 private placement exercisable at a $5.25 per share at the earlier or April 10, 2027 or 90 days after public announcement of a minimum 42% six - month CR rate from at least 25 BCG - Unres
Slide 6: Lyophilized, Inactivated Group A Streptococcus pyogenes
TARA - 002 Lyophilized, Inactivated Group A Streptococcus pyogenes
Slide 7: TARA-002: Broad Immunopotentiator with significant potential
7 TARA - 002: Broad Immunopotentiator with significant potential 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 Master Cell
Slide 8: Activates Th1 Immune Cascade
Activates Th1 Immune Cascade (1)(2)(3) Broad immunopotentiation = potential for durable response Mechanism similar to BCG, unique to 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. Immuno
Slide 9: Non-Muscle Invasive Bladder Cancer
TARA - 002 Non - Muscle Invasive Bladder Cancer
Slide 10: Unique product characteristics anticipated to drive significant adoption
Anticipated low burden on physicians G patients Favorable safety G tolerability Encouraging interim ADAVNCED - 2 data • No additional administration procedures or safety protocols required • Fast administration typically performed by nurse • Dedicated to ensuring access with minimal burden • To date
Slide 11: Bladder cancer: significant unmet need
Bladder cancer: significant unmet need 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, 2023. | 2. Anastasiadis et al. Ther
Slide 12: TARA-002 demonstrated 72% six-month CRR across BCG exposures
TARA - 002 demonstrated 72% six - month CRR and 70% CRR at any time across BCG exposures BCG Unresponsive 12 © 2025 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute BCG Naïve Combined Combined BCG Naïve BCG Un - responsive High - grade CR at Month 6 100 100% 80 72% 60 64% 40 20
Slide 13: TARA-002 demonstrated 100% durability with 80% reinduction salvage rate
TARA - 002 demonstrated 100% durability from 3 months to 6 months with a reinduction salvage rate of 80% 1 Abbreviations: BCG = Bacillus Calmette - Guérin; CR = complete response; CIS = carcinoma in situ NOTES: At the time of data cutoff, of the 24 subjects enrolled, 20 subjects were evaluated for h
Slide 14: Favorable safety and tolerability in interim analysis of ADVANCED-2
14 TARA - 002 demonstrated favorable safety and tolerability in interim analysis of ADVANCED - 2 trial Data cut - off date: 19 November 2024 AEs reflect urinary tract instrumentation effects and known safety profile of an immune - potentiating drug Abbreviations: AE = adverse event; NMIBC = non - mu
Slide 15: BCG-Unresponsive: Accelerating trial enrollment
BCG - Unresponsive: Accelerating trial enrollment 15 Registrational Trial Enrollment International expansion Expansion ongoing across South America and Asia Positive TARA - 002 data Positive data expected to drive participation Increased Capacity in U.S. Other large trials closing U.S. Sites Expect
Slide 16: Preclinical cytokine and cytotoxicity comparison to BCG
TARA - 002 BCG Cytokines + -- - IFN - γ +++ + TNF - α + = IL - 12p70 + + IL - 6 +++ +++ IL - 1β + = IL - 10 + + IL - 4 = = IL - 13 - = IL - 8 -- -- IL - 2 TARA - 002 treatment promotes higher release of pro - inflammatory TH1 - type cytokines than BCG in co - culture =, no change; +: 2 - 5 fold upre
Slide 17: Administration among the easiest of approved and experimental NMIBC treatments
TARA - 002 administration among the easiest of approved and experimental NMIBC treatments 17 Definitions: USPI – U.S. prescribing information; DDM - dodecyl maltoside; Data derived from product SOPIs and clinical trial publications © 2025 Protara Therapeutics. All Rights Reserved – Do Not Copy or Di
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 US HR/HG NMIBC Prevalence - ~45% (1) = Annual recurrence based on 5 yr US recurrence rate ~80% (2) = Over 65K HR/HG NMIBC annual
Slide 19: TARA-002 has a differentiated profile in NMIBC
19 UNIǪUE MOA • Only broad immunopotentiator in the industry pipeline • Non - clinical data points to encouraging durability • No overlapping toxicities with other novel therapeutic in NMIBC OPPORTUNITIES TO EXPAND • First to publish efficacy in BCG - naïve patients; assessing potential next steps •
Slide 20: IV Choline Chloride
IV CHOLINE CHLORIDE Phospholipid substrate replacement therapy for patients dependent on parenteral support (PS)
Slide 21: Overview of Parenteral Support
Overview of Parenteral Support 21 Crohn's 23% Mesenteric Ischemia 1G% Chronic Pseudo - Obstruction 23% Mucosal Disease 5% Other 21% Radiation Enteritis 5% Familial Polyposis 4% Underlying Etiology (1) Over time, dependent on PS Liver disease a well - known consequence of PS Patients are dependent on
Slide 22: Choline deficiency in PS is among the largest rare disease indications
Choline deficiency in PS is among the largest rare disease indications 22 ~30,000 1 PS patients in the U.S. and the majority are choline deficient • 78% of PS - dependent patients are choline - deficient and of those 63% have some degree of liver damage 2 • Data confirm choline deficiency results in
Slide 23: Choline replacement recommended in key PS Guidelines
Choline replacement recommended in key PS Guidelines Parenteral Support Professional Societies’ Position on Choline Guidelines / Position Paper ASPEN 2012 Position Paper (Vanek et al.) 3 : • Includes recommendations for Multivitamins & Multi - Trace Elements • Recognises the impact of long - term ch
Slide 24: 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 PLASMA FREE CHOLINE LEVELS: ALL PATIENTS (1) Choline supplementation was discontinued at week 24. Data are presented for all subjects up until time of withdrawal from the study. PLAS
Slide 25: Pivotal trial with PK-based endpoints expected to initiate in 1H'25
Pivotal trial with PK - based endpoints expected to initiate in 1H’25 25 © 2025 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute • Primary endpoint: Change in plasma choline concentration from baseline to Week 8 • Secondary endpoints: Steatosis and liver function tests THRIVE -
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: incidence of lymphatic malformations is ≈1,400 - 1,800 LM cases per year (2) Current tr
Slide 28: Clear evidence of biologic activity observed with OK-432
Clear evidence of biologic activity observed with OK - 432* 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 28 © 2025 Protara Therapeutics. All Rights Reserved –
Slide 29: Robust clinical results in large, academic study of OK-432
Robust clinical results in large, academic study of OK - 432 6G% CLINICAL SUCCESS ǂ IN IMMEDIATE TREATMENT GROUP 6 MONTHS AFTER ENROLLMENT 84% * CLINICAL SUCCESS ǂ IN PATIENTS WITH MACROCYSTIC LESION TYPES 6G% 7.5% Immediate Treatment Group (N=110) Delayed Treatment Group Pre - Treatment** (N=40) IT
Slide 30: TARA-002 in LMs: Phase 2 STARBORN-1 trial underway
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: Expansion
Slide 31: Appendix
Sy SSNS APPENDIX
Slide 32: Led by a team of experienced professionals
Led by a team of experienced professionals 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 Rel
Slide 33: TARA-002 in NMIBC: ADVANCED-2 clinical trial design
TARA - 002 in NMIBC: ADVANCED - 2 clinical trial design RE - induction (if eligible*) Maintenance (months 6 - 18) Month 3 Month 6 Month 18 Month 60 Maintenance (months 6 - 18) Month 18 Protara Confidential Information Data cut - off date: 19 November 2024 33 © 2025 Protara Therapeutics. All Rights R
Slide 34: ADVANCED-2 demographics and disease characteristics
ADVANCED - 2 demographics and disease characteristics 34 © 2025 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute Data cut - off date: 19 November 2024 N = 24 N = 24 Prior BCG Status, n (%) Age (years) 17 (71) BCG Naïve 71 (10.9) Mean (SD) 2 (8) BCG Exposed 71 Median 5 (21) BCG U
Slide 35: Manufacturing is a potential competitive advantage
20M vial capacity ability to expand capacity 5X Efficient 2 hour doubling time with t wo - week batch completion 47 successful batches to date Completed FDA inspection without Form 483s TARA - 002: Manufacturing is a potential competitive advantage 35 © 2025 Protara Therapeutics. All Rights Reserved
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