Protara Therapeutics(TARA) - 2024 Q1 - Quarterly Results

Q1 2024 Business and Financial Overview Protara Therapeutics reported strong Q1 2024 progress, securing $45 million in financing and advancing key clinical programs for TARA-002 and IV Choline Chloride Recent Progress and Highlights Protara Therapeutics achieved key milestones in Q1 2024, including a $45 million private placement and positive clinical data for TARA-002 and IV Choline Chloride - In April 2024, Protara successfully closed a $45.0 million private placement led by RA Capital Management and Acorn Bioventures[4] - Current cash resources, combined with private placement proceeds, are expected to fund operations and data milestones into 2026[3][5][13] - Positive three-month data was reported from the TARA-002 clinical program in non-muscle invasive bladder cancer (NMIBC), with interim ADVANCED-2 trial data expected in 2H 2024[3][5] - An agreement was reached with the U.S. FDA on a clear registrational path for IV Choline Chloride in patients dependent on parenteral nutrition (PN)[3][5] Clinical Program Updates Protara Therapeutics provides updates on its TARA-002 program for NMIBC and LMs, and IV Choline Chloride for PN patients, detailing clinical progress and regulatory alignment TARA-002 in NMIBC TARA-002 demonstrated a 38% complete response rate in NMIBC CIS patients with a favorable safety profile, and the ADVANCED-2 trial is progressing - TARA-002 achieved an overall three-month complete response (CR) rate of 38% in 16 evaluable carcinoma in situ (CIS) patients in its high-risk NMIBC program[9] - The CR rate was 63% in CIS-only patients, 13% in CIS +Ta/T1 patients, and 43% in BCG-Unresponsive/Experienced patients[9] - TARA-002 was well-tolerated, with no Grade 3 or greater treatment-related adverse events reported[9] - The Phase 2 ADVANCED-2 trial for TARA-002 in BCG-Unresponsive and BCG-Naïve NMIBC patients expects preliminary results in 2H 2024[9] IV Choline Chloride for Patients on PN Protara aligned with the FDA on a registrational pathway for IV Choline Chloride, broadening its indication for patients on long-term parenteral nutrition - In April 2024, the company aligned with the FDA on a registrational path for IV Choline Chloride for patients dependent on parenteral nutrition (PN)[7] - The indication was broadened from IFALD to a choline source for patients on long-term PN unable to synthesize it orally or enterally[7] TARA-002 in LMs The Phase 2 STARBORN-1 trial for TARA-002 in pediatric lymphatic malformations is actively enrolling, targeting 30 patients with a primary endpoint of significant LM volume reduction - Dosing and enrollment are progressing in the STARBORN-1 Phase 2 trial for TARA-002 in pediatric patients with macrocystic and mixed-cystic LMs[8][3] - The trial plans to enroll approximately 30 patients, with clinical success defined as a 60% to 100% reduction in total LM volume from baseline as the primary endpoint[8] First Quarter 2024 Financial Results Protara Therapeutics reported its Q1 2024 financial results, detailing changes in net loss, operating expenses, and its cash position, along with future funding projections Financial Performance Protara reported an increased net loss of $11.1 million in Q1 2024, primarily due to higher research and development expenses for the TARA-002 program Financial Performance Summary | Financial Metric | Q1 2024 | Q1 2023 | | :--- | :--- | :--- | | Research and development expenses | $7.7 million | $5.1 million | | General and administrative expenses | $4.1 million | $4.6 million | | Total operating expenses | $11.9 million | $9.7 million | | Net loss | $11.1 million | $9.0 million | | Net loss per share | $0.97 | $0.80 | - The increase in R&D expenses was mainly due to a $1.8 million rise in TARA-002 clinical and non-clinical costs and a $1.1 million increase in personnel-related expenses[13] - The decrease in G&A expenses was primarily driven by a $0.5 million reduction in personnel-related costs, including stock-based compensation[13] Financial Position As of March 31, 2024, Protara held $55.2 million in cash and equivalents, projecting sufficient funding into 2026 with recent financing - Cash, cash equivalents, and investments in marketable debt securities totaled $55.2 million as of March 31, 2024[13] - The company expects its cash position, including approximately $42.0 million in net proceeds from the April 2024 private placement, will fund operations into 2026[13] Balance Sheet Overview | Balance Sheet Item | March 31, 2024 | December 31, 2023 | | :--- | :--- | :--- | | Total current assets | $57.9 million | $68.7 million | | Total assets | $68.1 million | $79.0 million | | Total liabilities | $9.7 million | $10.6 million | | Total stockholders' equity | $58.4 million | $68.3 million | About Protara's Pipeline and Disease Areas This section provides an overview of Protara's key pipeline assets, TARA-002 and IV Choline Chloride, and the disease areas they target, NMIBC and LMs About TARA-002 TARA-002 is an investigational cell therapy for NMIBC and LMs, derived from OK-432, hypothesized to activate immune responses and induce immunogenic cell death - TARA-002 is an investigational cell therapy developed from the same master cell bank as OK-432, a marketed immunopotentiator in Japan and Taiwan[11] - The therapy is believed to activate innate and adaptive immune cells, leading to pro-inflammatory cytokine release, and directly kills tumor cells to enhance antitumor immune response[12] About Non-Muscle Invasive Bladder Cancer (NMIBC) NMIBC is the sixth most common cancer in the U.S., accounting for approximately 80% of bladder cancer diagnoses, with 65,000 new cases annually - NMIBC constitutes about 80% of bladder cancer diagnoses in the United States, with approximately 65,000 new patients each year[14] About Lymphatic Malformations (LMs) Lymphatic Malformations (LMs) are rare congenital lymphatic vessel defects, often diagnosed in early childhood, leading to complications like airway obstruction - LMs are rare congenital malformations of lymphatic vessels, with over 50% detected at birth and 90% diagnosed before the age of three[15] - Common morbidities include airway obstruction, intralesional bleeding, and impingement on critical structures[15] About IV Choline Chloride IV Choline Chloride is an investigational intravenous therapy for PN patients unable to synthesize choline, with potential to be the first FDA-approved treatment for this critical deficiency - IV Choline Chloride is being developed for patients on parenteral nutrition (PN) who are unable to synthesize choline and are at risk of liver damage[16] - It has the potential to be the first FDA-approved IV choline formulation for PN patients and has been granted Orphan Drug Designation by the FDA[16] Detailed Financial Statements This section presents the unaudited condensed consolidated balance sheets and statements of operations for Protara Therapeutics, providing a detailed view of its financial position and performance Unaudited Condensed Consolidated Balance Sheets The balance sheet as of March 31, 2024, shows total assets of $68,134 thousand, down from $78,954 thousand at year-end 2023, with total liabilities at $9,728 thousand Unaudited Condensed Consolidated Balance Sheets | | As of March 31, 2024 (in thousands) | As of December 31, 2023 (in thousands) | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $52,231 | $39,586 | | Marketable debt securities | $2,992 | $25,994 | | Total current assets | $57,913 | $68,705 | | Total assets | $68,134 | $78,954 | | Liabilities and Stockholders' Equity | | | | Total current liabilities | $5,501 | $6,149 | | Total liabilities | $9,728 | $10,633 | | Total stockholders' equity | $58,406 | $68,321 | | Total liabilities and stockholders' equity | $68,134 | $78,954 | Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss For the three months ended March 31, 2024, Protara reported total operating expenses of $11,851 thousand, resulting in a net loss of $11,095 thousand or $0.97 per share. The comprehensive loss was $11,065 thousand Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss | | For the Three Months Ended March 31, | | | :--- | :--- | :--- | | | 2024 (in thousands) | 2023 (in thousands) | | Operating expenses: | | | | Research and development | $7,748 | $5,143 | | General and administrative | $4,103 | $4,589 | | Total operating expenses | $11,851 | $9,732 | | Loss from operations | ($11,851) | ($9,732) | | Interest and investment income | $756 | $687 | | Net loss | ($11,095) | ($9,045) | | Net loss per share, basic and diluted | ($0.97) | ($0.80) | | Comprehensive loss | ($11,065) | ($8,826) |