Protara Therapeutics, Inc. · NASDAQ:TARA
Protara Therapeutics — Investor Presentation (March 2021)
32 slides from Protara Therapeutics's investor presentation, Mar 3, 2021
Slide 1: Corporate Presentation March 2021
Corporate Presentation March 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
Modernizing and Expediting Development of De-Risked Assets Lead programs: TARA-002 in Non-Muscle Invasive Bladder Cancer (NMIBC) ees and Lymphatic Malformations (LMs) * Cell-based immunopotentiator * FDA confirmed initial comparability to Japanese predecessor OK-432 ¢ NMIBC clinical development plan
Slide 4: Pipeline Addresses Multiple Indications With High Unmet Need
Pipeline Addresses Multiple Indications With High Unmet Need Phase 1 Phase 2 Phase 3 IMMUNOLOGY, ONCOLOGY Disease (IFALD)*** Vonapanitase — Recombinant (phase 1 studies completed in fistula ws ROTA RA *TARA-002 Granted Rare Pediatric Disease Designation for the treatment of LMs. OK-432 Granted Orpha
Slide 5: TARA-002
TARA-002 LYOPHILIZED, INACTIVATED GROUP A STREPTOCOCCUS PYOGENES
Slide 6: TARA-002: 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 Cell B
Slide 7: TARA-002: Mechanism of Anti-Tumor / Anti-Cystic Activity
TARA-002: Mechanism of Anti-Tumor / Anti-Cystic Activity Like A umor Cytokine Response IL-2 156 ECE TOR aaa ‘ Abnormal tissue is exposed to Immune cells detect TARA-002 Activated immune cells infiltrate Abnormal cells are TARA-002 and release inflammatory the abnormal cells destroyed > cytokines sy
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 OK-432 CLINICAL RESEARCH CONDUCTED IN: Non-Muscle Invasive Bladder Cancer * Ovarian cancer * Malignant mesothelioma + Pancreatic cancer * Esophageal cancer + Oral squamous cell can
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
TARA-002 in NMIBC: Profile Supports Potential in NMIBC Similar mechanism to BCG, notable patient experience in Asia and manufacturing advantages + Prompts a predominantly Th! type cytokine response * Mechanistically similar to Bacille Calmette-Guérin (BCG) * State-of-the-art U.S. manufacturing facil
Slide 11: Clinical Evidence of OK-432 Provides Strong Rationale for Development
Clinical Evidence of OK-432 Provides Strong Rationale for Development of TARA-002 in NMIBC Was generally well-tolerated, with safety and tolerability observed across a range of doses Demonstrated treatment effect and lower rates of recurrence vs. control group, including in the randomized, controlle
Slide 12: BCG Shortage Causes Significant Impact on Care
BCG Shortage Causes Significant Impact on Care | =BCG Shortage Sanofi production of BCG resumes T 2015 BCG shortages begin after Sanofi lab is shut down due to mold infractions Merck. announces Tice strain BCG shortages of of “..remain extremely concerned about this shortage and its effects on the c
Slide 13: NMIBC Represents the Most Common Form of Bladder Cancer
NMIBC Represents the Most Common Form of Bladder Cancer Bladder Cancer in the US 9 ind 0 gin 1) more nay AX diagnosed % High rate of inmen recurrence most prevalent @ @ age 55+""| with 3-year rate estimated at Cancer in the U.S." " Cs " up 10 80%" NMIBC makes up eae . . . Significant increase in ~80
Slide 14: Current Standard of Care Highlights High Unmet Need for Patients
Current Standard of Care Highlights High Unmet Need for Patients Initial Presentation Present to PCP: *Blood in urine + Bladder irritation w)/ Under Urologist Care High Grade Referral & Testing TURBT | Treatment Recurrence -@-i8- Bladder Removal Repeat TURBT Experimental Agents Urologist tests: Diag
Slide 15: TARA-002 in NMIBC: Overview of U.S. NMIBC & Target Population
TARA-002 in NMIBC: Overview of U.S. NMIBC & Target Population NMIBC is categorized and treated based on risk stratification, determined by combination of tumor grade, stage, size, recurrence history and focality 80K Patients Diagnosed with Bladder Cancer Annually!) 65K Patients with NMIBC |?) Treatm
Slide 16: TARA-002 in NMIBC: Estimated Development Timeline
TARA-002 in NMIBC: Estimated Development Timeline * Complete GMP scale + Complete Phase 1 + Futility analysis in CIS * Complete enrollment up and comparability study cohort of Papillary cohort + Complete non- * Commence Phase 2 * Topline efficacy in CIS clinical studies start-up activities patients
Slide 17: TARA-002 Lymphatic Malformations (LMs)
TARA-002 LYMPHATIC MALFORMATIONS (LMs)
Slide 18: TARA-002 in LMs
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!) Epidemiology Epidemiology: incidence of lymphatic malformations is =1,400-1,800 LM cases per year?) Current Trea
Slide 19: OK-432 in LMs: Clear Evidence of Biologic Activity in Patients
f OK-432 in LMs: Clear Evidence of Biologic Activity in Patients Before PROTARA THERAPEUTICS 12
Slide 20: OK-432 in LMs: Robust Results of Completed Phase 2 Study in U.S.
OK-432 in LMs: Robust Results of Completed Phase 2 Study!) in U.S. 84%" CLINICAL SUCCESS? IN PATIENTS WITH MACROCYSTIC LESION TYPES Complete or Substantial Response by ITT: Observations 6 Months After Enrollment _ Radiographically Confirmed Lesion Type** 69% 22% 60% mComplete Response Substantial Re
Slide 21: OK-432 in LMs: Compelling Safety Record
OK-432 in LMs: Compelling Safety Record Safety Profile* = 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 most severe events b
Slide 22: TARA-002 in LMs: Regulatory Update
TARA-002 in LMs: Regulatory Update 1) IND update completed and filed with FDA Division of Vaccines and Related Products 2] Vaccines division began review of IND update in late Q4'2020 and requested a CSR summarizing the totality of the lowa LMs Phase 2 study 3) CSR has been submitted and dialogue wi
Slide 23: TARA-002 in LMs: Planned Next Steps
TARA-002 in LMs: Planned Next Steps If current data set sufficient for BLA/MAA submission If ad | registrational study required — Apply for Fast-track / Breakthrough Therapy Designation — Agree with FDA on design of any additional study -— Complete GMP comparability (2H-2021) — Initiate enrollment o
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
IV Choline in IFALD: Late-stage Opportunity for an Unmet Medical Need An Essential Molecule in Several Metabolic Processes H3C_CHs / Patients dependent on Parenteral Nutrition (PN) cannot absorb sufficient levels of choline. Ne ~~ Data confirms that choline deficient diets results in steatosis and c
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 1994 - IV PK Study) 2001 - IV Phase 2 RCT(4) n=4 PN patients n=15 PN patients 1st continuous exposure to lV choline, 2g dose confirmed, reversal of steatosis, established safety and 2g dose i
Slide 27: IV Choline in IFALD: Multi-Center Phase 2a POC Study
IV Choline in IFALD: Multi-Center Phase 2a POC Study Randomized, Controlled Study Design & Objective The IV Choline Chloride replacement proof-of-concept, STUDY’ randomized study did not have pre-specified endpoints Study Design Randomized Double-blind Phase 2 Trial The primary objective of the stud
Slide 28: IV Choline in IFALD: Phase 2 Results
IV Choline in IFALD : Phase 2 Results Improvement in Steatosis and Cholestasis CLINICALLY MEANINGFUL a 8 (1) IMPROVEMENT IN STEATOSIS CHOLESTASIS IMPROVEMENT: ALL PATIENTS At Baseline o——— => Placebo = Choline Chloride n=144 (7 active; 7pbo) P0001 P0050 ome prams? pr 00016 +f P pr 0.0768 proses, p=
Slide 29: IV Choline in IFALD: Prevalence Study
IV Choline in IFALD: Prevalence Study Prevalence study underway to enhance understanding of the patient population Retrospective, observational study of patients in both academic & community DESIGN : settings POPULATION Patients dependent on PN for 6 or more months OBJECTIVE Understand presence/inci
Slide 30: Summary
Slide 31: Building Momentum in 2021
Building Momentum in 2021 LMs: e@ Q1'21: Submitted CSR to FDA (Vaccines Division) to support TARA-002 for LMs with the potential to file our BLA in 2H 2021 Near-term Rare Disease Opportunity @ 2H'21: Complete GMP scale up and comparability NMIBC: 8 1H'21: Complete nonclinical studies (tox, MOA, immu
Slide 32: Corporate Presentation
Corporate Presentation March 2021
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