Protara Therapeutics, Inc. · NASDAQ:TARA
Protara Therapeutics — Q1 2026 Earnings Presentation
42 slides from Protara Therapeutics's earnings presentation, May 19, 2026
Slide 1: TARA-002 in Lymphatic Malformations: Targeted Therapy for a Significant Unmet Need
TARA-002 in Lymphatic Malformations: Targeted Therapy for a Significant Unmet Need May 19, 2026
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,” “pot
Slide 3: Agenda
Agenda Jesse G.A. Jones, MD Associate Professor, Dept Welcome and Jesse Shefferman Case Studies with of Neurosurgery and Co-founder, Director, Introduction Chief Executive Officer TARA-002 in LMs Radiology at the University of Alabama Naiem Nassiri, MD The Unmet Need in Vascular & Endovascular Willi
Slide 4: Transformative Investigational Therapies in Oncology and Rare Disease
Transformative Investigational Therapies in Oncology and Rare Disease RARE PEDIATRIC ONCOLOGY TARA-002 in Lymphatic Malformations TARA-002 in Non-Muscle • Pediatric program for indication with no currently approved therapies available in the US Invasive Bladder Cancer • Positive interim data in BCG-
Slide 5: TARA-002 in LMs: Built on the Deep History of OK-432
TARA-002 in LMs: Built on the Deep History of OK-432 for the Treatment of LMs • TARA-002 is an investigational, genetically distinct strain of Streptococcus pyogenes that is inactivated while retaining its immune-stimulating properties • TARA-002 predecessor compound OK-432 is approved in Japan and
Slide 6: Clear Regulatory Path for TARA-002 in LMs
Clear Regulatory Path for TARA-002 in LMs • Review of TARA-002 for LMs has transitioned from the Office of Vaccines Research and Review to the Office of Therapeutic Products Plan to submit a • FDA will evaluate the risk-benefit profile of TARA-002 in LMs BLA in 2H 2027 based on the results of the ST
Slide 7: The Unmet Need in LMs
The Unmet Need in LMs Naiem Nassiri, MD, FSVS, RPVI Vascular & Endovascular Surgery, The Vascular Care Group (TVCG); Founding Director, TVCG CARES (Center for Anomalies & Rare Entities), International and Adjunct / Volunteer Faculty in Surgery (Vascular) Yale School of Medicine & Yale New Haven Hosp
Slide 8: LMs is a Significant Pediatric Rare Disease Opportunity with no Currently Approved Therapies
LMs is a Significant Pediatric Rare Disease Opportunity with no Currently Approved Therapies Healthy Lymphatic Lymphatic Lymphatic Malformations Vessels Malformation • Rare, non-malignant, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain
Slide 9: Three Distinct LMs Subtypes
Three Distinct LMs Subtypes – Today’s Focus is on Macrocystic and Mixed Cystic LMs Incidence: 1,500 cases per year1 47% 32% 21% Macrocystic LMs2 Mixed Cystic LMs Microcystic LMs Mixed Cystic2 Large cysts >1-2 cm Combination of both Small, infiltrative lesions diameter, well-defined macrocystic and w
Slide 10: Developmental Biology & Pathophysiology of Lymphatic Malformations
Developmental Biology & Pathophysiology of Lymphatic Malformations Maturation of Lymphatic Plexus3 Skin Cardinal Vein LECs Form1 LEC Budding and Migration2 PROX1 VEGF-C/ PI3K/ VEGRF3 AKT1/ mTOR Fluid Lymph Sac Drains Healthy Lymphatic Vessels Skin Lymphatic system development begins Upregulation of
Slide 11: Natural History and Age of Diagnosis
Natural History and Age of Diagnosis NATURAL HISTORY OF LYMPHATIC MALFORMATIONS1 • 75% are head and neck • Grow proportionate with child but can suddenly expand, especially in adolescence • Common complications: infections (cellulitis), pain, bleeding, and leakage of fluid from skin vesicles • Sever
Slide 12: Disease Burden in Pediatric Patients
Disease Burden in Pediatric Patients Physical Symptoms & Functional Impact: Psychological and Chronic Nature Healthcare Complications of LMs: Social Quality of Life: & Progression: Utilization: Breathing difficulties, Bleeding, infections, feeding/swallowing Children and their Congenital but often R
Slide 13: Current Management Approaches for Macrocystic LMs
Current Management Approaches for Macrocystic LMs Current treatment is multimodal: → Sclerotherapy or Intralesional ablation Injection with chemicals or UNMET NEED chemotherapeutic agents to collapse the No FDA-approved therapies cyst with limited evidence of efficacy3 exist for the treatment of lym
Slide 14: Sclerotherapy Techniques and Limitations
Sclerotherapy Techniques and Limitations Ablative Agents and Potential Side Effects Efficacy Limitations • Ethanol – high complication rates, including • Limited well-controlled clinical data and blistering, hemoglobinuria, deep ulceration, guidelines and no standardization of dosing nerve injury an
Slide 15: Surgical Approaches and Recurrence Challenges
Surgical Approaches and Recurrence Challenges Surgical Approaches • Complete excision or resection1 1/3 >50% Surgical complications Recurrence rates4,6,7 • Partial resection or debulking 2 occur in up to 1/3 of patients3-5, due to incomplete resection results • Staged surgery (helps protect function
Slide 16: Significant Need for a More Targeted Approach to Treating LMs
Significant Need for a More Targeted Approach to Treating LMs • FDA-approved therapy supported by clinical studies demonstrating efficacy and safety with standardized dosing regimen • Targeted therapy vs. cytotoxic or chemical approach • Minimally invasive and well tolerated • Minimal sedation in an
Slide 17: TARA-002
TARA-002
Slide 18: TARA-002: A Unique TLR2/NOD2 Agonist Derived From Streptococcus pyogenes
TARA-002: A Unique TLR2/NOD2 Agonist Derived From Streptococcus pyogenes That Brings the First Immunologic Mechanism to LMs TARA-002 ignites both innate and adaptive immunity through dual TLR2/NOD2 activation, driving potent, local, cystic responses via fully inactivated bacteria1, 2, 3 References p
Slide 19: Pivotal Phase 2 STARBORN-1 Trial
Pivotal Phase 2 STARBORN-1 Trial STARBORN-1 is a Single Arm, Open-Label Safety and Efficacy Study of TARA-002 in Pediatric Patients with Macrocystic & Mixed cystic LMs (N=29) • Patients receive up to four injections of TARA-002 spaced approximately six weeks apart • DMC review with FDA for each safe
Slide 20: TARA-002 Demonstrated Clinical Success in 83% of Participants that Completed Treatment
TARA-002 Demonstrated Clinical Success in 83% of Participants that Completed Treatment and 100% of Evaluable Participants CLINICAL SUCCESS RATE CLINICAL SUCCESS RATE IN ALL PATIENTS AT 8-WEEKS POST-TREATMENT AT 8-WEEKS POST-TREATMENT* IN EVALUABLE PARTICIPANTS^ 83% 100% 16% 20% 67% 80% 17% N = 12 N
Slide 21: STARBORN-1 Interim Results: Evaluable Participants Across Cyst Types
STARBORN-1 Interim Results: Evaluable Participants Across Cyst Types STRONG RESPONSE ACROSS CYST TYPES IN EVALUABLE POPULATION 100% 100% 100% Macrocystic LMs (N = 7) Mixed-cystic LMs (N = 1) Ranula (N = 2) Complete Response Substantial Response • Clinical success was achieved with one or two doses o
Slide 22: TARA-002 Demonstrated 100% Durable Response at 32-Weeks
TARA-002 Demonstrated 100% Durable Response at 32-Weeks RESPONSE AT 32-WEEKS POST-TREATMENT IN EVALUABLE PARTICIPANTS^ 100% 29% 71% N=7 Complete Response Substantial Response • 7 participants have reached the 32-weeks post-treatment assessment and remain disease free ^Excludes ongoing participants w
Slide 23: TARA-002 Has Demonstrated Meaningful Results in LMs Patients
TARA-002 Has Demonstrated Meaningful Results in LMs Patients Treated in the STARBORN-1 Study Medical photography: Baseline and 8-week post-treatment results for three participants treated with TARA-002 in the STARBORN-1 trial BEFORE BEFORE AFTER BEFORE AFTER AFTER Baseline 1,739mL macrocystic LM rec
Slide 24: TARA-002 Has a Favorable and Well-Tolerated Safety Profile
TARA-002 Has a Favorable and Well-Tolerated Safety Profile • Most AEs were mild to moderate • No serious TEAEs or AESIs have occurred to date Number of Participants, N = 16 Any Grade Grade 1 Grade 2 Grade 3 Grade 4/5 TEAEs, n (%) 11 (69) 11 (69) 9 (56) 1 (6) 0 (0) Related TEAEs, n (%) 10 (63) 10 (63
Slide 25: KOL Discussion
KOL Discussion Jesse G.A. Jones, MD, University of Alabama – Departments of Neurosurgery and Radiology 24
Slide 26: STARBORN-1 Results: Clinical Trial for Significant Unmet Need in LMs
STARBORN-1 Results: Clinical Trial for Significant Unmet Need in LMs •100% of evaluable participants achieved clinical success •TARA-002 demonstrated favorable safety and tolerability profile with no serious adverse events reported •Clinical Success was achieved with one or two doses in 80% of parti
Slide 27: Case Studies
Case Studies 26
Slide 28: Expanded Access Case Review: Patient with LM Overview
Expanded Access Case Review: Patient with LM Overview • 3-year-old male patient with a LM (+ cystic fibrosis) in the left anterior BEFORE TARA-002 neck with recurrences TREATMENT • Prior treatments received: • Sclerotherapy with 2 mL of 40 mg/cc doxycycline and 5 mL Sotradecol and Gelfoam without re
Slide 29: Expanded Access Case Review: Patient with LM – Results
Expanded Access Case Review: Patient with LM – Results • Initial ultrasound showed an ovoid unilocular fluid collection measuring 1.8 x 1.2cm BEFORE TARA-002 AFTER TARA-002 TREATMENT TREATMENT • This patient received one injection of TARA-002 on 03-December-2025 • The aspirated volume was 4 mL and t
Slide 30: STARBORN-1 Case Review: Ranula – Overview
STARBORN-1 Case Review: Ranula – Overview • 7-year-old female participant initially diagnosed with a macrocystic LM located infra and suprahyoid right (de Serres stage III), later diagnosed as a ranula • Prior treatment included: • One dose of 5 mL doxycycline/10 mL sotradecol/10 mL ethanol with no
Slide 31: STARBORN-1 Case Review: Ranula – Results
STARBORN-1 Case Review: Ranula – Results • The 8-week post-treatment MRI demonstrated a substantial response (78.8% reduction) and the investigator assessment of response was substantial resolution BEFORE AFTER • The 32-week post-treatment visit is upcoming • One possibly related TEAE of pain in jaw
Slide 32: LMs Market Overview
LMs Market Overview: TARA-002 Has the Potential to Become the First and Only FDA Approved Treatment for Patients with Macrocystic LMs
Slide 33: Attractive Market Opportunity in Macrocystic and Mixed Cystic LMs
Attractive Market Opportunity in Macrocystic and Mixed Cystic LMs Lymphatic Malformations Incidence: ~1,500 LM cases per year1 Prevalence Seeking Treatment ~20,000 patients Microcystic2 Mixed2 Macrocystic2 ~21% ~32% ~47% Not a target for TARA-002 Total addressable market estimated to be greater than
Slide 34: Patients are Typically Diagnosed in the Community Setting and Referred to Vascular Anomaly Centers
Patients are Typically Diagnosed in the Community Setting and Most are Referred to Vascular Anomaly Centers (VACs) Community ENT, Dermatologist, Pediatrician, Surgeon Referral Sclerotherapy Surgery Complication, Treatment Failure, Referral Recurrence VACs/Centers of Excellence (COE) 1,2Market resear
Slide 35: Real-World Findings from Qualitative Research Study Highlight Burden of LMs
Real-World Findings from Qualitative Research Study Highlight Clinical, Psychological and Structural Burden of LMs1 Patient Perspectives Provider Perspectives 1. Pain, fatigue and disability 1. No FDA approved treatments for macrocystic LMs 2. Functional Issues, including difficulties with mobility,
Slide 36: TARA-002 Has Significant Potential Value for LMs Patients & Providers
TARA-002 Has Significant Potential Value for LMs Patients & Providers Differentiated MOA First targeted immunotherapy in the macrocystic LMs setting Convenience Impressive efficacy to date Most patients only require 1-2 100% clinical success in doses evaluable patients at 8 weeks TARA-002 Emerging,
Slide 37: The Top Accounts for LMs Treated Patients Represent ~80% of the Total
The Top 100 Accounts for LMs Treated Patients Represent ~80% of the Total LM Treated Population % Total LM Patients Account with Sclerotherapy Rank Claims Top 20 54% Top 50 70% Top 100 80% Top 200 86% TARA-002 can achieve a high target penetration with a focused commercial launch © 2026 Protara Ther
Slide 38: Summary
Summary
Slide 39: OK-432 Demonstrated Clinical Efficacy in a Variety of Maxillofacial Cysts
OK-432 Demonstrated Clinical Efficacy in a Variety of Maxillofacial Cysts, Suggesting Significant Future Growth Opportunity for TARA-002 Congenital and acquired cystic masses of the craniofacial region Ranula Thyroglossal Duct Cyst Epidemiology: ~0.2 cases per 1,000 Epidemiology: > 1 million3 person
Slide 40: TARA-002 Provides a De-Risked Opportunity for a Significant Unmet Need
TARA-002 Provides a De-Risked Opportunity for a Significant Unmet Need HIGH UNMET NEED AMONG PATIENTS • No FDA-approved therapies • Current off-label treatment options have significant gaps DE-RISKED PROGRAM WITH SUPPORTIVE CLINICAL DATA • TARA-002 predecessor compound OK-432 is approved in Japan an
Slide 41: Q&A
Q&A 6 Mrota RA THERAPEUTICS
Slide 42: Thank you!
Thank you! 6 Mrota RA THERAPEUTICS