Protara Therapeutics, Inc. · NASDAQ:TARA
Protara Therapeutics — Investor Presentation (November 2020)
35 slides from Protara Therapeutics's investor presentation, Nov 17, 2020
Slide 1: Corporate Presentation November 2020
6 EROTARA THERAPEUTICS Corporate Presentation November 2020
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: Investment Summary
Investment Summary Lead programs: TARA-002 in Non-Muscle Invasive Bladder Cancer (NMIBC) and Lymphatic Malformations (LMs) + Cellbased immunopotentiator + FDA confirmed initial comparability to Japanese predecessor OK-432 + NMIBC clinical development plan in place following Pre-Investigational New D
Slide 4: Pipeline Addresses Multiple Indications With High Unmet Need
Pipeline Addresses Multiple Indications With High Unmet Need PRE-IND Phase 1 Phase 3 IMMUNOLOGY, ONCOLOGY HEPATOLOGY, GI, METABOLICS in fistula PROTA RA *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 treat
Slide 5: TARA-002
é SeRoTARA THERAPEUTICS 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 Th! Like Tumor Cytokine Response Multi-Cytokine Inducer” IL-8 IL-10 JL-12 GNMEGSE G-CSF TNF-a Abnormal tissue is Immune cells detect Activated immune cells Abnormal cells exposed to TAI TARA-002 and release inflitrate the abnormal cells are de
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 Lymphangiomas (Lymphatic Malformations) * Gastric cancer combo with chemo (post-operative) * Primary lung cancer combo with chemo. * Reduction of ascites in gastrointestinal cancer
Slide 9: TARA-002 Non-Muscle Invasive Bladder Cancer (NMIBC)
é SeRoTARA THERAPEUTICS 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 manufacturing facility , TARA-
Slide 11: Clinical Evidence with OK-432 Provides Strong Rationale for Development of TARA-002 in NMIBC
Clinical Evidence with OK-432 Provides Strong Rationale for Development of TARA-002 in NMIBC Total Pts/ OK-432 Data across multiple studies in ~150 NMIBC Dose Regimen Pts Efficacy Results patients treated intravesically shows that 210 SKE intratumoral, Inpreviously unresected tumors, 5 recuences in
Slide 12: BCG Shortage Causes Significant Impact on Care
BCG Shortage Causes Significant Impact on Care Merck announces = BCG Shortage San ‘ anticipated BCG supply beat pone) constraints despite resumes dir T 2015 2016 i { a Merck ‘Sanofi announces 7 Merck announces plans to BCG shortages begin ig = US Urologists are e iia Gurion ab ie Gnu announces disco
Slide 13: 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 Annvally!") 65K Patients with NMIBC |) Treat
Slide 14: TARA-002 in NMIBC: Highlights of PIND FDA Interaction
TARA-002 in NMIBC: Highlights of PIND FDA Interaction Supportive feedback on multiple facets of NMIBC program CMC: Initial comparability established — GMP scale up in process to complete full comparability testing Modernized release criteria for large scale (3 batch) comparability Non-Clinical: Alig
Slide 15: TARA-002 in NMIBC: Estimated Development Timeline
TARA-002 in NMIBC: Estimated Development Timeline + Initiate GMP scale * Complete GMP scale comparability up and comparability + Initiate agreed-to * Complete non- non-clinical studies clinical studies + Initiate Phase 1 study* + Engage International regulatory authorities + Further FDA engagement (
Slide 16: TARA-002 Lymphatic Malformations (LMs)
é SeRoTARA THERAPEUTICS TARA-002 LYMPHATIC MALFORMATIONS (LMs)
Slide 17: 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 Tr
Slide 18: OK-432 in LMs: Clear Evidence of Biologic Activity in Patients
OK-432 in LMs: Clear Evidence of Biologic Activity in Patients Before After Before After S EROTARA THERAPEUTICS Protara Therapeutics data on file
Slide 19: 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. 94% CLINICAL SUCCESS* IN PATIENTS WITH MACROCYSTIC LESION TY PES Complete or Substantial Response by Radiographically Confirmed Lesion Type ITT: Observations 6 months after enrollment 68%, his ™ Complete Response 83% mSubstantial Res
Slide 20: OK-432 in LMs: Compelling Safety Record
OK-432 in LMs: Compelling Safety Record Long-term safety data in 99 patients with up to 8 years of follow up Safety Profile = Most common AEs with treatment were local injection site reactions, fever, fatigue, resolution within two weeks decreased appetite, with © SAEs related to OK-432: re-hospital
Slide 21: TARA-002 in LMs: Regulatory Update
TARA-002 in LMs: Regulatory Update “i =, IND update completed and filed with FDA Division of Vaccines and Related Products IND update included clarifying questions related to the regulgtory pathway in LMs as well as the confirmation of initial comparability between TA and OK-432 received from FDA's
Slide 22: TARA-002 in LMs: Planned Next Steps
TARA-002 in LMs: Planned Next Steps If current data set sufficient for BLA/MAA submission 2023 _ Launchin the EU and oiher international geographies 2023 P, sTARA THERAPEUTICS Submit BLA — US Approval Q3'2022 = Launch in the US - File marketing applications in EU countries 22
Slide 23: IV Choline Chloride: Intestinal Failure Associated Liver Disease (IFALD)
é SeRoTARA THERAPEUTICS IV CHOLINE CHLORIDE INTESTINAL FAILURE ASSOCIATED LIVER DISEASE (IFALD)
Slide 24: 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 HC CHs Patients dependent on Parenteral Nutrition (PN) cannot absorb sufficient levels of choline. Ne Data confirms that choline deficient diets results in steatosis and cholest
Slide 25: 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‘) n=4 PN patients n=15 PN patients 2g dose confirmed, reversal of steatosis, Ist continuous exposure to IV choline, cholestasis (reduction of ALP*)
Slide 26: 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 IV CHOLINE REPLACEMENT PROOF OF CONCEPT STUDY“ Study Design Randomized Double-blind Phase 2 Trial Subjects 15 @ per protocol Age > 16 years old PN Requirement aed than 80% of all nutrient requirement
Slide 27: IV Choline in IFALD: Phase 2 Results
IV Choline in IFALD : Phase 2 Results Improvement in Steatosis and Cholestasis CLINICALLY MEANINGFUL IMPROVEMENT IN STEATOSIS At Baseline \ PROTARA, ») pistsrnenssucsiocnays HERAPEUTICS CHOLESTASIS IMPROVEMENT: ALL PATIENTS*) === Placebo — Choline Chlatide n=14} (7 active; 7pbo) z Sas £ ¥ 2.00- a J
Slide 28: 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 Understand presence/incidence of liv
Slide 29: Financial Overview
é SeRoTARA THERAPEUTICS FINANCIAL OVERVIEW
Slide 30: Finance Overview
w)/ Finance Overview 11.2M Common plus 8.0. PROTARA THERAPEUTICS $166M Cash position as of September 30, 2020 + $151Mnet proceeds from September 2020 offe 19.2M Common Share Equivalents 30
Slide 31: Investment Summary
Investment Summary Lead programs: TARA-002 in Non-Muscle Invasive Bladder Cancer (NMIBC) and Lymphatic Malformations (LMs) + Cellbased immunopotentiator + FDA confirmed initial comparability to Japanese predecessor OK-432 + NMIBC clinical development plan in place following Pre-Investigational New D
Slide 32: Appendix
é SeRoTARA THERAPEUTICS APPENDIX
Slide 33: OK-432 in LMs: Overview of Completed Phase 2 Study
OK-432 in LMs: Overview of Completed Phase 2 Study Age ontrolled Study (N=117 6 months — 18yrs Inclusion Criteria LMs of the head and/or neck confirmed by MRI or CT = Radiographically confirmed macrocystic LM or mixed macrocystic-microcystic LM with >50% macrocystic component Atleast 6 months since
Slide 34: OK-432 in LMs: Patient Disposition of Completed Phase 2 Study
OK-432 in LMs: Patient Disposition of Completed Phase 2 Study!) 182 patients serolled Instinay” Group Open Label Group (n=182) Completing Completing 4151 patients 31 patients Treatment areatient Complete case report Incomplete case report forms = = forms and imaging and imaging or lost to follow up
Slide 35: Protara Therapeutics
6 EROTARA THERAPEUTICS Corporate Presentation November 2020
Protara Therapeutics — Investor Presentation (November 2020) on 6ix