Protara Therapeutics, Inc. · NASDAQ:TARA
Protara Therapeutics — Investor Presentation (September 2020)
39 slides from Protara Therapeutics's investor presentation, Sep 8, 2020
Slide 1: Corporate Presentation September 2020
CorporatePresentation September2020
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 thin the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as “predicts,” “believes,” “po
Slide 3: Investment Summary
3 Investment Summary 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 development plan in place following Pre-Investigational Ne
Slide 4: Entrepreneurial Team with Demonstrated History of Uncovering Overlooked Assets
4 Entrepreneurial Team with Demonstrated History of Uncovering and Accelerating Overlooked Assets Blaine Davis Chief Financial Officer • 20 years of experience in the biopharma sector • Experience in operations, finance, corporate strategy and IR • Managed growing portfolio of rare disease products
Slide 5: Deeply Experienced Board of Directors
5 Deeply Experienced Board of Directors Luke Beshar Chairman Michael Solomon, PhD Director Barry Flannelly, PharmD, MBA Director Roger Garceau, MD Director Greg Sargen Director Richard Levy, MD Director
Slide 6: Pipeline Addresses Multiple Indications With High Unmet Need
6 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 Orp
Slide 7: TARA-002 Lyophilized, Inactivated Group A Streptococcus Pyogenes
TARA-002 LYOPHILIZED, INACTIVATED GROUP A STREPTOCOCCUS PYOGENES
Slide 8: TARA-002: Cell-Based Immunopotentiator with Significant Potential
8 TARA-002: Cell-Based Immunopotentiator with Significant Potential ▪ TARA-002 is an investigational, genetically distinct strain of Streptococcus pyogenes that isinactivated while retaining its immune- stimulating properties ▪ TARA-002 is manufactured under GMP conditions from the same Master Cell
Slide 9: TARA-002: Mechanism of Anti-Tumor / Anti-Cystic Activity
9 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-2 IL-6 IL-10
Slide 10: 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 Data
Slide 11: TARA-002: Protara's Planned Strategy
TARA-002: Protara’sPlanned Strategy 11 Reach agreement with FDA that TARA-002 is comparable to OK-432 Accelerate development in LMs Leverage OK-432’s existing safety & efficacy data in NMIBC 1 2 3 4 Explore additional indications / combinations
Slide 12: TARA-002 Non-Muscle Invasive Bladder Cancer (NMIBC)
TARA-002 NON-MUSCLE INVASIVE BLADDER CANCER (NMIBC)
Slide 13: TARA-002 in NMIBC: Profile Supports Potential in NMIBC
13 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 results (
Slide 14: Clinical Evidence with OK-432 Provides Rationale for TARA-002 in NMIBC
Clinical Evidence with OK-432 Provides Strong Rationale for Development of TARA-002 in NMIBC 14 Data across multiple studies in ~150 NMIBC patients treated intravesically shows that OK-432: • Was generally well-tolerated, with safety and tolerability observed across a range of doses • Demonstrated e
Slide 15: BCG Shortage Causes Significant Impact on Care
15 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 se
Slide 16: TARA-002 in NMIBC: Overview of U.S. NMIBC & Target Population
TARA-002 in NMIBC: Overview of U.S. NMIBC & Target Population 16 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 C
Slide 17: TARA-002 in NMIBC: Highlights of PIND FDA Interaction
17 TARA-002 in NMIBC: Highlights of PIND FDA Interaction 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: ▪ Aligned on 6-week local (intravesical instillation)
Slide 18: TARA-002: State of the Art and Scalable GMP Manufacturing
18 TARA-002: State of the Art and Scalable GMP Manufacturing x Successful propagation of working cell bank (WCB) from Chugai OK-432 MCB x TARA-002 WCB -Confirmation of no genetic drift x Modernization of manufacturing and testing techniques (potency, purity, quality attributes) x Proof of concept es
Slide 19: TARA-002 in NMIBC: Estimated Development Timeline
TARA-002 in NMIBC: Estimated Development Timeline 19 2023 •Complete GMP scale up and comparability •Complete non- clinical studies •Initiate Phase 1 study* •Engage International regulatory authorities •Further FDA engagement (SPA request, Fast Track, etc.) 2022 2021 2020 • Initiate GMP scale compara
Slide 20: TARA-002 Lymphatic Malformations (LMs)
TARA-002 LYMPHATIC MALFORMATIONS (LMS)
Slide 21: TARA-002 in LMs
21 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 Options
Slide 22: OK-432 in LMs: Clear Evidence of Biologic Activity in Patients
Before After 22 OK-432 in LMs: Clear Evidence of Biologic Activity in Patients Before After ProtaraTherapeutics data on file
Slide 23: OK-432 in LMs: Robust Results of Completed Phase 2 Study
23 OK-432 in LMs: Robust Results of Completed Phase 2 Study (1) inU.S. 68% 0% Immediate Treatment Group (N=91) Delayed Treatment Group Pre- Treatment** (N=26) ITT: Observations 6 months after enrollment 68% CLINICAL SUCCESS ? IN IMMEDIATE TREATMENT GROUP 6 MONTHS AFTER ENROLLMENT • Patients with rad
Slide 24: 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 relatedto OK-432: re-hospitalization for infection (n=3) and severe edema (n=3), airway obs
Slide 25: 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 – Complete GMP comparability – Initiate enrollment of study (mid-2021) – Engage European regu
Slide 26: IV Choline Chloride Intestinal Failure Associated Liver Disease (IFALD)
IV CHOLINE CHLORIDE INTESTINAL FAILURE ASSOCIATED LIVER DISEASE (IFALD)
Slide 27: IV Choline in IFALD: Late-stage Opportunity for an Unmet Medical Need
27 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 Replacement in IFALD (intestinal
Slide 28: 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 1
Slide 29: 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 Duration of Treatment 2
Slide 30: IV Choline in IFALD: Phase 2 Results
IV Choline in IFALD : Phase 2 Results 30 CHOLESTASIS IMPROVEMENT: ALL PATIENTS* (1) CLINICALLY MEANINGFUL IMPROVEMENT IN STEATOSIS At Baseline After 24 Weeks *Mixed model for repeated measurement (MMRM) method used ?Aplacebo subject was excluded from all analyses due to likely IV contrast-induced im
Slide 31: IV Choline in IFALD: Prevalence Study
31 IV Choline in IFALD: Prevalence Study Prevalence study to be conducted 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 prese
Slide 32: Financial Overview
FINANCIAL OVERVIEW
Slide 33: Finance Overview
Finance Overview 33 FINANCING HISTORY $30M Cash position as of June 30, 2020 9.7M Common Share Equivalents 5.9M Common plus 3.8M Preferred on as-converted basis (as of June 30, 2020) Date Type $M Investors 2017 Seed Round $3 Founders, friends & family 2017-18 Series A $5.5 Smaller dedicated life sci
Slide 34: Key Projected Milestones
Key Projected Milestones 34 TARA-002—NMIBC 2020 1H21 2H21 1H22 Results of Non-clinical Studies Initiate Phase 1 Study* Initial Pharmacodynamic Data from Phase 1 Phase 1 Study Completion TARA-002—LMs 2020 1H21 2H21 1H22 Fast Track Granted Request Meeting with FDA to Discuss Next Steps BLA Submission*
Slide 35: Investment Summary
35 Investment Summary 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 development plan in place following Pre-Investigational N
Slide 36: Appendix
APPENDIX
Slide 37: OK-432 in LMs: Overview of Completed Phase 2 Study
OK-432 in LMs: Overview of Completed Phase 2 Study Randomized, Controlled Study (N=117) (1) Age 6 months –18 yrs 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 At le
Slide 38: OK-432 in LMs: Study Completers and Patient Demographics
Completers (N=119): Completed all protocol visits, received up to 4 injections and evaluated 14 days after last injection, includes non-randomized Open Label Group Patient Demographics Group Patients Enrolled (n=182) Patients Randomized Completing Treatment (n=92) Open Label Group Completing Treatme
Slide 39: Corporate Presentation September 2020
CorporatePresentation September2020
Protara Therapeutics — Investor Presentation (September 2020) on 6ix