Protara Therapeutics(TARA) - 2023 Q4 - Annual Results

Business Update and Highlights Protara anticipates a milestone-rich 2024, advancing its pipeline with TARA-002 data for NMIBC and LMs, and FDA engagement for IV Choline Chloride TARA-002 in NMIBC Protara advances TARA-002 for NMIBC with preliminary Phase 1b results expected in 1H 2024 and Phase 2 data in 2H 2024 - Preliminary results from the Phase 1b ADVANCED-1EXP trial are expected in 1H 2024, evaluating TARA-002 in up to 12 CIS patients. The primary endpoint is the complete response (CR) rate at three months[4][5] - Preliminary data from the Phase 2 ADVANCED-2 trial, based on a risk-benefit analysis of approximately 10 patients at the six-month mark, is expected in 2H 2024[5][6] - The ADVANCED-2 trial will enroll at least 102 NMIBC patients with CIS, including both BCG-naïve (n=27) and BCG-unresponsive (n=75-100) cohorts[6] TARA-002 in LMs The Phase 2 STARBORN-1 trial for TARA-002 in pediatric LMs is dosing patients, targeting substantial LM volume reduction - Dosing is progressing in the STARBORN-1 Phase 2 trial, which will enroll approximately 30 pediatric patients with LMs[7] - The primary endpoint is clinical success, defined as a complete response (90-100% volume reduction) or substantial response (60-90% volume reduction) in the LM[7] - The company expects to share Phase 1b proof of concept (POC) and preliminary Phase 2 data for TARA-002 in NMIBC later in 2024[3] - Dosing is underway in the Phase 2 STARBORN-1 trial of TARA-002 for pediatric patients with lymphatic malformations (LMs)[3][7] - The company continues engagement with the U.S. FDA to determine the next steps for the IV Choline Chloride development program[8] Fourth Quarter and Full Year 2023 Financial Results Protara held $65.6 million cash as of Dec 31, 2023, funding operations into Q2 2025, with a reduced $40.4 million net loss for 2023 - Cash, cash equivalents, and investments totaled $65.6 million as of December 31, 2023, expected to fund operations into Q2 2025[5][10] Full Year Operating Expenses (2023 vs 2022) | Expense Category | Full Year 2023 | Full Year 2022 | Change Driver | | :--- | :--- | :--- | :--- | | Research & Development | $25.0 million | $16.8 million | Increase in clinical trial and non-clinical activities for TARA-002. | | General & Administrative | $18.6 million | $20.7 million | Reduction in personnel costs and D&O insurance premiums. | Net Loss and EPS (Q4 & Full Year 2023 vs 2022) | Period | Metric | 2023 | 2022 | Note | | :--- | :--- | :--- | :--- | :--- | | Q4 | Net Loss | $(10.2)M | $(39.0)M | 2022 included a $29.5M non-cash goodwill impairment charge. | | | EPS | $(0.90) | $(3.46) | | | Full Year | Net Loss | $(40.4)M | $(66.0)M | | | | EPS | $(3.57) | $(5.86) | | Product Pipeline and Disease Overview Protara's pipeline features TARA-002 for NMIBC and LMs, and IV Choline Chloride for parenteral nutrition patients About TARA-002 TARA-002, an investigational cell therapy, treats NMIBC and LMs by activating immune cells and inducing tumor cell death - TARA-002 is an investigational cell therapy developed from the same master cell bank as OK-432 (Picibanil®)[11] - It works by activating immune cells to produce pro-inflammatory cytokines and by directly killing tumor cells, triggering an antitumor immune response[12] - TARA-002 has been granted Rare Pediatric Disease Designation by the FDA for the treatment of LMs[11] About Non-Muscle Invasive Bladder Cancer (NMIBC) NMIBC is the 6th most common U.S. cancer, accounting for 80% of bladder cancer diagnoses with 65,000 new cases annually - NMIBC represents approximately 80% of bladder cancer diagnoses in the U.S[13] - There are approximately 65,000 new NMIBC diagnoses in the United States each year[13] About Lymphatic Malformations (LMs) LMs are rare congenital lymphatic malformations, often diagnosed in early childhood, causing serious health issues - LMs are rare, congenital malformations of lymphatic vessels, with 90% diagnosed before the age of 3[14] About Intravenous (IV) Choline Chloride IV Choline Chloride is an investigational orphan drug for choline deficiency in PN patients, vital for liver function - IV Choline Chloride is a replacement therapy for PN-dependent patients, as current PN formulations lack choline[15] - It has been granted Orphan Drug Designation by the FDA for preventing choline deficiency in PN patients[15] Consolidated Financial Statements Protara's total assets were $79.0 million as of Dec 31, 2023, with total liabilities at $10.6 million and equity at $68.3 million Consolidated Balance Sheets Protara's total assets were $79.0 million as of Dec 31, 2023, with total liabilities at $10.6 million and equity at $68.3 million Consolidated Balance Sheets (in thousands) | | Dec 31, 2023 | Dec 31, 2022 | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $39,586 | $24,127 | | Marketable debt securities (current & non-current) | $25,994 | $78,129 | | Total current assets | $68,705 | $86,146 | | Total assets | $78,954 | $113,290 | | Liabilities and Stockholders' Equity | | | | Total current liabilities | $6,149 | $5,740 | | Total liabilities | $10,633 | $11,207 | | Total stockholders' equity | $68,321 | $102,083 | | Total liabilities and stockholders' equity | $78,954 | $113,290 | Consolidated Statements of Operations and Comprehensive Loss Protara reported a $40.4 million net loss for 2023, a reduction from $66.0 million in 2022 due to no goodwill impairment Consolidated Statements of Operations (in thousands, except per share data) | | For the Year Ended Dec 31, 2023 | For the Year Ended Dec 31, 2022 | | :--- | :--- | :--- | | Research and development | $24,989 | $16,808 | | General and administrative | $18,624 | $20,737 | | Loss on impairment of goodwill | - | $29,517 | | Total operating expenses | $43,613 | $67,062 | | Loss from operations | $(43,613) | $(67,062) | | Interest and investment income | $3,193 | $1,110 | | Net loss | $(40,420) | $(65,952) | | Net loss per share, basic and diluted | $(3.57) | $(5.86) | | Comprehensive Loss | $(39,763) | $(66,429) |