Protara Therapeutics, Inc. · NASDAQ:TARA
Protara Therapeutics — Investor Presentation (April 2021)
30 slides from Protara Therapeutics's investor presentation, Apr 23, 2021
Slide 1: Corporate Presentation April 2021
Corporate Presentation April 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
3 Modernizing and Expediting Development of De - Risked Assets Lead programs: TARA - 002 in Non - Muscle Invasive Bladder Cancer (NMIBC) and Lymphatic Malformations (LMs) • C e ll - b a sed i mm u no p o t e n t i at o r • FDA confirmed initial comparability to Japanese predecessor OK - 432 • NMIBC
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: 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: 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) F u ji m oto T . , et al. J I mm un ol. 1997 : 5619. (2) Ryoma Y, et al. Anticancer Res. 2004; 3295 - 3298. (3) Zhao H, et al. Microbiol. Immunol. 1994; 183 - 190. T NF - α I FN - γ M ul t i
Slide 8: OK-432: Human Efficacy Data in Multiple Indications
• Lymphangiomas (Lymphatic Malformations) • G a s t r i c c a n c er c o m b o wi t h c h e m o ( p o s t - o p er at i v e) • Primary lung cancer combo with chemo • Reduction of ascites in gastrointestinal cancer • Reduction of pleural effusion in lung cancer • Unresponsive head, neck & thyroid can
Slide 9: Non-Muscle Invasive Bladder Cancer (NMIBC)
TARA - 002 NON - MUSCLE INVASIVE BLADDER CANCER (NMIBC)
Slide 10: TARA-002 Profile Supports Potential in NMIBC
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 re
Slide 11: Clinical Evidence of OK-432 Provides Rationale for Development in NMIBC
11 Clinical Evidence of OK - 432 Provides Strong Rationale for Development of TARA - 002 in NMIBC Data across multiple i n v e s t i g at o r - i n i t i at e d s t ud i e s in ~150 NMIBC patients treated intravesically shows that OK - 432: Was generally well - tolerated, with safety and tolerabilit
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 g u i d a n ce on p a t i e n t m a n a g e m e n t (1) “…remain extremely concerned about this shortage and its effects on the care of bladder cancer patients…” - Joint Statement on BCG Sh
Slide 13: NMIBC Represents the Most Common Form of Bladder Cancer
NMIBC Represents the Most Common Form of Bladder Cancer NMIBC patients 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 a g e 55+ 9 i n 10 4x m o r e li k el y t o be d i a g n o se d in men (2) High rate
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 High Grade TURBT Treatment R ec u rr e n ce Repeat T URBT Present to PCP : • Blood in urine • Bladder irritation U r o l og i st t es t s : • C
Slide 15: Overview of U.S. NMIBC & Target Population
TARA - 002 in NMIBC: Overview of U.S. NMIBC & Target Population 15 5% Metastatic 15 % L o c ally Advanced 80 % N M I B C ( 2 ) Risk Stratification (4) Hi g h R i s k I n te r me di a t e R i sk Low Risk Treatment Gui d e lin es TURBT + BCG Mix of TURBT + Chemo/BCG TURBT + Chemo Goal of Treatment 1)
Slide 16: Lymphatic Malformations (LMs)
TARA - 002 LYMPHATIC MALFORMATIONS (LM S )
Slide 17: TARA-002 in LMs: Lymphatic Malformations
17 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 E p i d e m i o l o g y : in c i d e n c e o f l y m p h at i c m a l f o r m at ion s i
Slide 18: TARA-002 in LMs: Regulatory Path
18 1 2 3 4 TARA - 002 in LMs: Regulatory Path Plan to initiate clinical study in pediatric LMs patients following alignment with FDA on study design Completed Phase 2 study of OK - 432 in U.S. supports treatment effect with support for strong safety profile In April 2021, received feedback from FDA
Slide 19: OK-432 in LMs: Clear Evidence of Biologic Activity in Patients
Before A f t er 19 OK - 432 in LMs: Clear Evidence of Biologic Activity in Patients Before A f t er P r ota r a T h e r a p e u ti c s data on f i le
Slide 20: OK-432 in LMs: Robust Results of Completed Phase 2 Study
20 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 21: Safety Profile
*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 22: IV Choline Chloride Intestinal Failure Associated Liver Disease (IFALD)
IV CHOLINE CHLORIDE INTESTINAL FAILURE ASSOCIATED LIVER DISEASE (IFALD)
Slide 23: IV Choline in IFALD: Late-stage Opportunity for an Unmet Medical Need
23 IV Choline in IFALD: Late - stage Opportunity for an Unmet Medical Need An Essential Molecule in Several Metabolic Processes Patients dependent on Parenteral Nutrition (PN) cannot absorb sufficient levels of choline. Data confirms that choline deficient diets results in steatosis and cholestasis
Slide 24: 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 l e v e ls ; h o w e v e r d i d r e du c e s t e a t os i s with moderate ALP* improvement 1994 - IV PK S
Slide 25: 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 26: IV Choline in IFALD: Phase 2 Results
IV Choline in IFALD : Phase 2 Results 26 CHOLESTASIS IMPROVEMENT: ALL PATIENTS* (1) C L I N IC A LL Y ME AN I N GF UL IMPROVEMENT IN STEATOSIS At Baseline After 24 Weeks *Mixed model for repeated measurement (MMRM) method used ǂ A placebo subject was excluded from all analyses due to likely IV contr
Slide 27: IV Choline in IFALD: Prevalence Study
27 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 28: Summary
Su mm a r y
Slide 29: Building Momentum in 2021
Building Momentum in 2021 29 NMIBC: Significant Market Potential • 1H’21: Complete nonclinical studies (tox, MOA, immunogenicity) • 2H’21: File IND • Late 2021: Initiate Phase 1 study* LMs: Rare Disease Opportunity • 2H’21: Complete confirmatory GMP scale up and comparability • 2H’21: Agree with FDA
Slide 30: Corporate Presentation April 2021
Corporate Presentation April 2021
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