Protara Therapeutics, Inc. · NASDAQ:TARA
Protara Therapeutics — Investor Presentation (October 2021)
31 slides from Protara Therapeutics's investor presentation, Oct 12, 2021
Slide 1: Corporate Presentation October 2021
Corporate Presentation October 2021
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: Modernizing and Expediting Development of De-Risked Assets
Lead programs: TARA - 002 in Non - Muscle Invasive Bladder Cancer (NMIBC) and Lymphatic Malformations (LMs) • Cell - based immunopotentiator • FDA confirmed initial comparability to Japanese predecessor OK - 432 • NMIBC: Promising existing clinical data in patients and a proven MOA generally similar
Slide 4: Pipeline Addresses Multiple Indications With High Unmet Need
4 Pipeline Addresses Multiple Indications With High Unmet Need *TARA - 002 Granted Rare Pediatric Disease Designation for the treatment of LMs. OK - 432 Granted Orphan Drug Designation by the U.S. FDA for the treatment of LMs, which we believe is applicable under established comparability. **Granted
Slide 5: TARA-002: Lyophilized, Inactivated Group A Streptococcus Pyogenes
T A R A - 002 LYOPHILIZED, INACTIVATED GROUP A STREPTOCOCCUS PYOGENES
Slide 6: TARA-002: Cell-Based Immunopotentiator with Significant Potential
6 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 Ma
Slide 7: TARA-002: Mechanism of Anti-Tumor / Anti-Cystic Activity
7 TARA - 002: Mechanism of Anti - Tumor / Anti - Cystic Activity Th1 Like Anti - Tumor Cytokine Response (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. Immunol. 1994; 183 - 190. T N F - α I F N - γ Mult i - C y t ok
Slide 8: OK-432: Human Efficacy Data in Multiple Indications
• Lymphangiomas (Lymphatic Malformations) • Gastric cancer combo with chemo (post - operative) • Primary lung cancer combo with chemo • Reduction of ascites in gastrointestinal cancer • Reduction of pleural effusion in lung cancer • Unresponsive head, neck & thyroid cancer OK - 432: Human Efficacy D
Slide 9: TARA-002 Non-Muscle Invasive Bladder Cancer (NMIBC)
TARA - 002 NON - MUSCLE INVASIVE BLADDER CANCER (NMIBC)
Slide 10: TARA-002 in NMIBC: Profile Supports Potential in NMIBC
10 TARA - 002 in NMIBC: Profile Supports Potential in NMIBC • ~ 150 NMIBC patients tested with OK - 432 demonstrated promising results ( 2 ) • Treatment generally well tolerated • Elicits Th 1 type response inducing multiple cytokines to produce an anti - tumor effect • Mechanistically similar to th
Slide 11: Clinical Evidence of OK-432 Provides Strong Rationale for Development of TARA-002 in NMIBC
11 Clinical Evidence of OK - 432 Provides Strong Rationale for Development of TARA - 002 in NMIBC Data across multiple investigator - initiated studies in ~150 NMIBC patients treated intravesically shows that OK - 432: Was generally well - tolerated, with safety and tolerability observed across a ra
Slide 12: NMIBC Represents the Most Common Form of Bladder Cancer
NMIBC Represents the Most Common Form of Bladder Cancer N M I B C pa t ien t s are treated by a urologist Bladder Cancer in the US NMIBC makes up ~80% of all bladder cancer with ~65,000 diagnosed per year in the U.S. a r e ag e 55+ 9 i n 10 4x more likely to be diagnosed i n m en ( 2) High rate of r
Slide 13: TARA-002 in NMIBC: Target Patient Population
13 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 Treatment goal is to prevent: 1. Progression 2. Cystectomy 3. Recurrence 1. National Cancer Institut
Slide 14: TARA-002 in NMIBC: Phase 1a/1b Study Design
TARA - 002 in NMIBC: Phase 1a/1b Study Design 1 Subjects will receive weekly intravesical doses of TARA - 002 instillation for 6 weeks 2 Defined as not previously treated with or unable to access BCG. 3 Defined as disease present at last cystoscopic evaluation during the dose expansion phase. Defini
Slide 15: TARA-002 Lymphatic Malformations (LMs)
TARA - 002 LYMPHATIC MALFORMATIONS (LM S )
Slide 16: TARA-002 in LMs: Lymphatic Malformations
16 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 17: OK-432 in LMs: Clear Evidence of Biologic Activity in Patients
Before A f t e r 17 OK - 432 in LMs: Clear Evidence of Biologic Activity in Patients Before A f t e r Completed Phase 2 study of OK - 432 in U.S. provides evidence of treatment effect with support for strong safety profile Protara Therapeutics data on file
Slide 18: IV Choline Chloride: Intestinal Failure Associated Liver Disease (IFALD)
IV CHOLINE CHLORIDE INTESTINAL FAILURE ASSOCIATED LIVER DISEASE (IFALD)
Slide 19: IV Choline in IFALD: Late-stage Opportunity for an Unmet Medical Need
19 IV Choline in IFALD: Late - stage Opportunity for an Unmet Medical Need Clinical History Supporting Choline Substrate Replacement in Patients with IFALD A Phase 2 study demonstrated the clinical potential of choline substrate replacement therapy by reversing certain hallmark pathologies of IFALD
Slide 20: IV Choline in IFALD: Prevalence Study
20 IV Choline in IFALD: Prevalence Study Prevalence study to enhance understanding of the patient population DESIGN Retrospective, observational study of patients in both academic & community settings POPULATION Patients dependent on PN for 6 or more months OBJECTIVE • Understand the incidence of ch
Slide 21: IV Choline in IFALD: Phase 2 Results
IV Choline in IFALD : Phase 2 Results 21 CHOLESTASIS IMPROVEMENT: ALL PATIENTS* (1) CLINICALLY MEANINGFUL IMPROVEMENT IN STEATOSIS At Baseline After 24 Weeks * Mi x e d mo d e l for r e p e a t e d m e asur e m en t ( MM R M) m e t h od us ed ǂ A placebo subject was excluded from all analyses due to
Slide 22: Summary
S u mm ary
Slide 23: Building Momentum in 2021
Building Momentum in 2021 23 NMIBC: Significant Market Potential 2H’21: File IND with GMP scale confirmatory comparability data • Year - End 2021: Initiate Phase 1 study IV Choline: Late - Stage Pipeline Opportunity • 2H’21: Complete retrospective prevalence study to better characterize the unmet ne
Slide 24: Corporate Presentation October 2021
Corporate Presentation October 2021
Slide 25: Appendix
6 S bRoTARA THERAPEUTICS Appendix
Slide 26: Current Standard of Care Highlights High Unmet Need for Patients
Current Standard of Care Highlights High Unmet Need for Patients 26 Primary care / PCP Uro l ogist Medical Oncologist I n i t i al P r esen t a t i on Referral & Testing High Grade TURBT Treatment Recurrence R epeat T URBT Present to PCP : • Blood in urine • B ladder irritation Urologist tests : • C
Slide 27: BCG Shortage Causes Significant Impact on Care
27 BCG Shortage Causes Significant Impact on Care Shortage has prompted major urological associations to issue guidan c e o n pati e nt m anag eme nt (1) “…remain extremely concerned about this shortage and its effects on the care of bladder cancer patients…” - Joint Statement on BCG Shortage, Feb 2
Slide 28: OK-432 in LMs: Robust Results of Completed Phase 2 Study
28 OK - 432 in LMs: Robust Results of Completed Phase 2 Study (1) in U.S. ITT: Observations 6 Months After Enrollment 69% 7 . 5% Immediate Treatment Group (N=110) Delayed Treatment Group Pre - Treatment** (N=40) 69% CLINICAL SUCCESS ǂ IN IMMEDIATE TREATMENT GROUP 6 MONTHS AFTER ENROLLMENT • Patients
Slide 29: Safety Profile of OK-432 in LMs
*Results based on retrospective analysis of source verified data that included the full dataset of subjects enrolled in the P2 randomized study between January 1998 and August 2005, including data in the published study (Smith et al. 2009) which included subjects enrolled between January 1998 and No
Slide 30: IV Choline in IFALD: Informative Clinical History
IV Choline in IFALD: Informative Clinical History A significant body of supportive evidence across 4 studies 1992 - Oral Lecithin Study (1) n=15 PN patients Lecithin did not achieve physiologic levels ; however did reduce steatosis with moderate ALP* improvement 1994 - IV PK Study (2) n=4 PN patient
Slide 31: IV Choline Replacement Proof of Concept Study
IV CHOLINE REPLACEMENT PROOF OF CONCEPT STUDY 1 Study Design Randomized Double - blind Phase 2 Trial Subjects 15 (9 per protocol) Age >16 years old PN Requirement Greater than 80% of all nutrient requirements supplied by PN Randomization 1:1 Usual PN or PN + 2g IV choline/day D u r ati on of Treatme
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