Protara Therapeutics, Inc. · NASDAQ:TARA
Protara Therapeutics — Investor Presentation (January 2023)
29 slides from Protara Therapeutics's investor presentation, Jan 5, 2023
Slide 1: Corporate Presentation January 2023
CORPORATE PRESENTATION January 2023
Slide 2: Forward Looking Statements
FORWARD LOOKING STATEMENTS 2 © 2023 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute Statements contained in this press release regarding matters that are not historical facts are "forward looking statements" within the meaning of the Private Securities Litigation Reform Act of
Slide 3: Advancing Transformative Therapies for People with Cancer and Rare Diseases
ADVANCING TRANSFORMATIVE THERAPIES FOR PEOPLE WITH CANCER AND RARE DISEASES Applying modern scientific advancements to established mechanisms LEAD PROGRAM: TARA - 002 IN NON - MUSCLE INVASIVE BLADDER CANCER (NMIBC) AND LYMPHATIC MALFORMATIONS (LMS) Cell - based immunopotentiator based on originator
Slide 4: Pipeline Addresses Multiple Indications with High Unmet Need
PIPELINE ADDRESSES MULTIPLE INDICATIONS WITH HIGH UNMET NEED Pre - Clinical IND Cleared Phase 1 Phase 2 Phase 3 IMMUNOLOGY, ONCOLOGY TARA - 002 – Lyophilized, inactivated Group A Streptococcus Lymphatic Malformations (LMs)* Non - Muscle Invasive Bladder Cancer (NMIBC) TARA - 002 combinations HEPATOL
Slide 5: Lyophilized, Inactivated Group A Streptococcus pyogenes
TARA - 002 Lyophilized, Inactivated Group A Streptococcus pyogenes
Slide 6: Cell-Based Immunopotentiator with Significant Potential
TARA - 002: CELL - BASED 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 GMP conditions from the same Master C
Slide 7: Mechanism of Anti-Tumor/Anti-Cystic Activity
TARA - 002: MECHANISM OF ANTI - TUMOR/ ANTI - CYSTIC ACTIVITY Th1 Like Anti - Tumor Cytokine Response IL - 10 7 © 2023 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute TNF - α IFN - γ IL - 2 IL - 6 IL - 8 Multi - Cytokine Inducer (1)(2)(3) IL - 12 GM - CSF G - CSF 1. Fujimoto T.
Slide 8: OK-432: Human Efficacy Data in Multiple Indications
OK - 432: HUMAN EFFICACY DATA IN MULTIPLE INDICATIONS OK - 432 has been approved (ex - US) or studied in multiple indications • Ovarian cancer • Malignant mesothelioma • Pancreatic cancer • Esophageal cancer • Oral squamous cell cancer • Hepatocellular cancer • Ranula • Thyroglossal cysts • Pleurode
Slide 9: Non-Muscle Invasive Bladder Cancer (NMIBC)
TARA - 002 Non - Muscle Invasive Bladder Cancer (NMIBC)
Slide 10: In NMIBC: Profile Supports Potential Cell-Based Immunopotentiator
TARA - 002 IN NMIBC: PROFILE SUPPORTS POTENTIAL CELL - BASED IMMUNOPOTENTIATOR WITH NOTABLE PATIENT EXPERIENCE Proven Anti - Cancer MOA • Elicits Th1 type response inducing multiple cytokines to produce an anti - tumor effect • Mechanistically similar to the current SOC, Bacille Calmette - Guérin (B
Slide 11: In NMIBC: Clinical Evidence from Predecessor Therapy OK-432
TARA - 002 IN NMIBC: CLINICAL EVIDENCE FROM PREDECESSOR THERAPY OK - 432 PROVIDES STRONG RATIONALE FOR DEVELOPMENT IN NMIBC Data across multiple studies in ~150 NMIBC patients treated intravesically shows that OK - 432: • Was generally well - tolerated, with safety and tolerability observed across a
Slide 12: In NMIBC: Target Patient Population
TARA - 002 IN NMIBC: TARGET PATIENT POPULATION NMIBC is categorized and treated based on risk stratification, determined by combination of tumor grade, stage, size, recurrence history and focality Patients diagnosed with bladder cancer annually 1 Patients with NMIBC 2 Initial target patient populati
Slide 13: In NMIBC: Phase 1 Clinical Trial Design
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 Escalation Design Enroll 10 KE 20 KE 40 KE NMIBC HGCIS or
Slide 14: Lymphatic Malformations (LMs)
TARA - 002 Lymphatic Malformations (LMs)
Slide 15: In LMs: Lymphatic Malformations
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 (2)
Slide 16: In LMs: Clear Evidence of Biologic Activity with OK-432
TARA - 002 IN LMs: CLEAR EVIDENCE OF BIOLOGIC ACTIVITY OBSERVED WITH PREDECESSOR THERAPY OK - 432 Completed clinical study of OK - 432 (TARA - 002 predecessor therapy) in U.S. suggests effectiveness with strong support for safety profile Protara Therapeutics data on file BEFORE AFTER BEFORE AFTER BE
Slide 17: In LMs: Robust Results of Completed Clinical Trial in U.S.
TARA - 002 IN LMs: ROBUST RESULTS OF COMPLETED CLINICAL TRIAL (1) IN U.S. OBSERVED WITH PREDECESSOR THERAPY OK - 432 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=
Slide 18: In LMs: Compelling Safety Record of OK-432
TARA - 002 IN LMs: COMPELLING SAFETY RECORD OF PREDECESSOR THERAPY OK - 432 Most common AEs with treatment were local injection site reactions, fever, fatigue, decreased appetite, with resolution within two weeks Treatment emergent SAEs related to OK - 432: reported in 4.1% of patients, with the mos
Slide 19: In LMs: Phase 2 Trial Design
TARA - 002 IN LMS: PHASE 2 TRIAL DESIGN 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 Enroll N = 3 En
Slide 20: IV Choline Chloride: Intestinal Failure Associated Liver Disease (IFALD)
IV CHOLINE CHLORIDE Intestinal Failure Associated Liver Disease (IFALD)
Slide 21: IV Choline in IFALD: Late-Stage Opportunity for an Unmet Medical Need
IV CHOLINE IN IFALD: LATE - STAGE OPPORTUNITY FOR AN UNMET MEDICAL NEED 21 1. Buchman A, et al. JPEN. 2001;5:260 - 268. IFALD, intestinal failure associated liver disease; IV, intravenous; PK, pharmacokinetic; PN, parenteral nutrition. © 2023 Protara Therapeutics. All Rights Reserved – Do Not Copy o
Slide 22: IV Choline in IFALD: Phase 2 Trial Results
IV CHOLINE IN IFALD: PHASE 2 TRIAL RESULTS 22 Improvement in Steatosis and Cholestasis CLINICALLY MEANINGFUL IMPROVEMENT IN STEATOSIS CHOLESTASIS IMPROVEMENT: ALL PATIENTS *(1) At Baseline After 24 Weeks *Mixed model for repeated measurement (MMRM) method used ǂ A placebo subject was excluded from a
Slide 23: Summary
SUMMARY
Slide 24: 2023: Focused Execution Designed to Position Protara for Long-Term Growth
2023: FOCUSED EXECUTION DESIGNED TO POSITION PROTARA FOR LONG - TERM GROWTH STRONG BALANCE SHEET: $107M of cash, cash equivalents and investments as of September 30, 2022 expected to fund operations into 2H 2024 19.3M COMMON SHARE EQUIVALENTS: 11.3M Common + 8.0M Preferred on as converted basis as o
Slide 25: Appendix
APPENDIX
Slide 26: NMIBC Represents the Most Common Form of Bladder Cancer
High rate of recurrence with 3 - year rate estimated at up to 80% (3) NMIBC REPRESENTS THE MOST COMMON FORM OF BLADDER CANCER Bladder Cancer in the U.S. most prevalent cancer in the U.S. 6 th 4x more likely to be diagnosed in men (2) 9 in 10 are age 55+ (1) NMIBC makes up ~80% of all bladder cancer
Slide 27: Current Standard of Care Highlights High Unmet Need for Patients
CURRENT STANDARD OF CARE HIGHLIGHTS HIGH UNMET NEED FOR PATIENTS Initial Presentation Recurrence Repeat TURBT Cystectomy Bladder Removal Intravesical Agents Experimental Agents in Development IV Treatment Transfer to Medical Oncologist • Checkpoint inhibitors Present to PCP : • Blood in urine • Blad
Slide 28: Ongoing BCG Shortage Presents Opportunity for Alternative Treatment Options
ONGOING BCG SHORTAGE PRESENTS OPPORTUNITY FOR ALTERNATIVE TREATMENT OPTIONS AUA GUIDELINES ON BCG SHORTAGE BCG use has been limited to high - risk disease; induction to be prioritized over maintenance MERCK BECOMES SOLE MANUFACTURER Sanofi facility for Connaught strain of BCG goes offline; Merck’s T
Slide 29: IV Choline in IFALD: Prevalence Study
IV CHOLINE IN IFALD: PREVALENCE STUDY 29 © 2023 Protara Therapeutics. All Rights Reserved – Do Not Copy or Distribute PREVALENCE STUDY TO ENHANCE UNDERSTANDING OF THE PATIENT POPULATION DESIGN Retrospective, observational study of patients in both academic & community settings POPULATION Patients de
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