Protara Therapeutics, Inc. · NASDAQ:TARA
Protara Therapeutics — Investor Presentation (February 2021)
32 slides from Protara Therapeutics's investor presentation, Feb 11, 2021
Slide 1: Corporate Presentation February 2021
Corporate Presentation February 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" wi thi n the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as “predicts,” “believes,” “p
Slide 3: Modernizing and Expediting Development of De-Risked Assets
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 clinical developm
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. **Grante
Slide 5: Lyophilized, Inactivated Group A Streptococcus Pyogenes
TARA - 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: 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. Multi - Cytokine Inducer (1)(2)(3) IL
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: Non-Muscle Invasive Bladder Cancer (NMIBC)
TARA - 002 NON - MUSCLE INVASIVE BLADDER CANCER (NMIBC)
Slide 10: TARA-002 in NMIBC: Profile Supports Potential
10 TARA - 002 in NMIBC: Profile Supports Potential in NMIBC • Urologists have been using an attenuated bacteria, BCG, as immunotherapy for decades • Intravesical administration is preferred clinical approach among urologists (1) • ~150 NMIBC patients tested with OK - 432 demonstrated promising resul
Slide 11: Clinical Evidence of OK-432 Provides Strong Rationale 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: BCG Shortage Causes Significant Impact on Care
12 BCG Shortage Causes Significant Impact on Care Shortage has prompted major urological associations to issue guidance on patient management (1) “…remain extremely concerned about this shortage and its effects on the care of bladder cancer patients…” - Joint Statement on BCG Shortage, Feb 2019 “I s
Slide 13: NMIBC Represents the Most Common Form of Bladder Cancer
NMIBC Represents the Most Common Form of Bladder Cancer more likely to be diagnosed in men 4x NMIBC patients are treated by a urologist Significant increase in recurrence, progression & an escalated number of patients needing cystectomies Bladder Cancer in the US NMIBC makes up ~80% of all bladder c
Slide 14: Current Standard of Care Highlights High Unmet Need for Patients
Current Standard of Care Highlights High Unmet Need for Patients 14 Primary care / PCP Urologist Medical Oncologist Initial Presentation Referral & Testing TURBT High Grade Treatment Recurrence Repeat TURBT Present to PCP : • Blood in urine • Bladder irritation Urologist tests : • CT scan • Cytology
Slide 15: Overview of U.S. NMIBC & Target Population
TARA - 002 in NMIBC: Overview of U.S. NMIBC & Target Population 15 5% Metastatic 15% Locally Advanced 80% NMIBC (2) Risk Stratification (4) High Risk Intermediate Risk Low Risk Treatment Guidelines TURBT + BCG Mix of TURBT + Chemo/BCG TURBT + Chemo Goal of Treatment 1) Prevent Progression 2) Prevent
Slide 16: TARA-002 in NMIBC: Estimated Development Timeline
TARA - 002 in NMIBC: Estimated Development Timeline 16 • Complete GMP scale up and comparability • Complete non - clinical studies • Initiate Phase 1 study* • Engage International regulatory authorities • Complete Phase 1 study • Commence Phase 2 start - up activities • Futility analysis in CIS coho
Slide 17: Lymphatic Malformations (LMs)
TARA - 002 LYMPHATIC MALFORMATIONS (LM S )
Slide 18: TARA-002 in LMs
18 TARA - 002 in LMs Epidemiology: incidence of lymphatic malformations is ≈ 1,400 - 1,800 LM cases per year (2) Epidemiology Current treatment options include surgical excision with high complication (33%) and recurrence (55%) rates (3) as well as off - label use of sclerosants Current Treatment Op
Slide 19: OK-432 in LMs: Clear Evidence of Biologic Activity in Patients
Before After 19 OK - 432 in LMs: Clear Evidence of Biologic Activity in Patients Before After Protara Therapeutics data on file
Slide 20: OK-432 in LMs: Robust Results of Completed Phase 2 Study in U.S.
20 OK - 432 in LMs: Robust Results of Completed Phase 2 Study (1) in U.S. 68% 0% Immediate Treatment Group (N=91) Delayed Treatment Group Pre- Treatment** (N=26) ITT: Observations 6 months after enrollment 68% CLINICAL SUCCESS ǂ IN IMMEDIATE TREATMENT GROUP 6 MONTHS AFTER ENROLLMENT • Patients with
Slide 21: OK-432 in LMs: Compelling Safety Record
Smith M, et al. Laryngoscope. 2009;119:107 - 115. Safety Profile ▪ Most common AEs with treatment were local injection site reactions, fever, fatigue, decreased appetite, with resolution within two weeks ▪ SAEs related to OK - 432: re - hospitalization for infection (n=3) and severe edema (n=3), air
Slide 22: TARA-002 in LMs: Regulatory Update
22 IND update completed and filed with FDA Division of Vaccines and Related Products Vaccines division began review of IND update in late Q4’20 and has requested a CSR summarizing the totality of the Iowa LMs Phase 2 study 1 2 3 4 Dialogue with Vaccines Division is ongoing with CSR expected to be su
Slide 23: TARA-002 in LMs: Planned Next Steps
TARA - 002 in LMs: Planned Next Steps If additional registrational study required – Launch in the US – File marketing applications in EU countries 2023 – Complete study – Submit BLA – US Approval Q3’2022 2022 – Apply for Fast - track / Breakthrough Therapy Designation – Agree with FDA on design of a
Slide 24: IV Choline Chloride: Intestinal Failure Associated Liver Disease (IFALD)
IV CHOLINE CHLORIDE INTESTINAL FAILURE ASSOCIATED LIVER DISEASE (IFALD)
Slide 25: IV Choline in IFALD: Late-stage Opportunity for an Unmet Medical Need
25 IV Choline in IFALD: Late - stage Opportunity for an Unmet Medical Need A Phase 2 study demonstrated the clinical potential of choline substrate replacement therapy by reversing certain hallmark pathologies of IFALD (1) Clinical History Supporting Choline Substrate R eplacement in IFALD (intestin
Slide 26: 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 patients
Slide 27: IV Choline in IFALD: Multi-Center Phase 2a POC 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 Duration of Treatment
Slide 28: IV Choline in IFALD: Phase 2 Results
IV Choline in IFALD : Phase 2 Results 28 CHOLESTASIS IMPROVEMENT: ALL PATIENTS* (1) CLINICALLY MEANINGFUL IMPROVEMENT IN STEATOSIS At Baseline After 24 Weeks *Mi xed model for repeated measurement (MMRM) method used ǂ A placebo subject was excluded from all analyses due to likely IV contrast - induc
Slide 29: IV Choline in IFALD: Prevalence Study
29 IV Choline in IFALD: Prevalence Study Prevalence study underway 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 presence/inc
Slide 30: Summary
Summary
Slide 31: Building Momentum in 2021
Building Momentum in 2021 31 NMIBC: Significant Market Potential • 1H’21: Complete nonclinical studies ( tox, MOA, immunogenicity) • 2H’21: File IND • Late 2021: Initiate Phase 1 study LMs: Near - term Rare Disease Opportunity • Q1’21: Expect to submit CSR to FDA (Vaccines Division) to support TARA
Slide 32: Corporate Presentation February 2021
Corporate Presentation February 2021
Protara Therapeutics — Investor Presentation (February 2021) on 6ix