Protara Therapeutics(TARA) - 2025 Q2 - Quarterly Results

Executive Summary & Business Outlook This section summarizes Protara Therapeutics' Q2 2025 financial results, business updates, and pipeline milestones Q2 2025 Financial Results and Business Update Protara Therapeutics announced Q2 2025 financial results and business updates, outlining pipeline progress and key upcoming milestones - Protara Therapeutics is a clinical-stage company developing transformative therapies for cancer and rare diseases[2] - CEO Jesse Shefferman stated the company is well-positioned to advance its pipeline in the second half of the year[3] - Interim results from the ADVANCED-2 trial of TARA-002 in non-muscle invasive bladder cancer (NMIBC) are expected in 1Q 2026[3] - The company is on track to dose the first patient in the THRIVE-3 registrational trial of IV Choline Chloride by end of 3Q 2025[3] - An interim update from the STARBORN-1 trial in pediatric lymphatic malformations (LMs) is expected in 4Q 2025[3] Recent Progress and Pipeline Updates This section details recent advancements and updates across Protara Therapeutics' clinical pipeline, including TARA-002 and IV Choline Chloride programs TARA-002 in Non-Muscle Invasive Bladder Cancer (NMIBC) Protara Therapeutics expects interim analysis from approximately 25 six-month evaluable BCG-Unresponsive NMIBC patients in the ADVANCED-2 trial in 1Q 2026, while also exploring subcutaneous dosing and combination treatments - Interim analysis from approximately 25 six-month evaluable BCG-Unresponsive NMIBC patients in the ADVANCED-2 trial is expected at a medical conference in 1Q 2026[5][6] - An update on next steps in the BCG-Naïve program is expected in 2H 2025, following discussions with the U.S. Food and Drug Administration (FDA)[6] - Protara continues to investigate subcutaneous dosing and explore combination treatments with TARA-002 in NMIBC patients with CIS[6] IV Choline Chloride for Patients on Parenteral Support (PS) The company is on track to dose the first patient in the THRIVE-3 registrational Phase 3 clinical trial in 3Q 2025, with EU enrollment commencing soon, and IV Choline Chloride holding Fast Track designation - First patient dosing in THRIVE-3, a registrational Phase 3 clinical trial, is on track for the third quarter of 2025[7] - Enrollment across the EU is expected to begin in the coming months following recent clinical trial approval by the European Union Clinical Trials Regulation[7] - THRIVE-3 is a seamless Phase 2b/3 trial, including a dose confirmation portion (n=24) and a double-blinded, randomized, placebo-controlled portion (n=105)[7] - IV Choline Chloride was previously granted Fast Track designation by the FDA[7] TARA-002 in Lymphatic Malformations (LMs) Dosing continues in the Phase 2 STARBORN-1 trial for pediatric LMs, with an interim update anticipated in 4Q 2025, following promising safety cohort results - Dosing remains ongoing in the Phase 2 STARBORN-1 trial of TARA-002 in pediatric patients with macrocystic and mixed cystic LMs[8] - An interim update from the STARBORN-1 trial is intended to be provided in the fourth quarter of 2025[8] - The study's first safety cohort showed promising results, and TARA-002 was generally well-tolerated[8] Corporate Update & Financial Highlights This section covers Protara Therapeutics' recent operational achievements and its financial position as of Q2 2025 Operational Highlights Protara strengthened its leadership with the appointment of William 'Bill' Conkling as Chief Commercial Officer and gained broader market recognition by joining the Russell 3000 Index - William 'Bill' Conkling was appointed as Chief Commercial Officer in June 2025, bringing over two decades of experience in developing and commercializing novel cancer and rare disease therapeutics[11] - Protara was added as a member of the broad-market Russell 3000 Index, effective June 30, 2025[11] Financial Position & Outlook As of June 30, 2025, Protara reported approximately $146 million in cash, cash equivalents, and investments, providing an expected funding runway into mid-2027 Cash, Cash Equivalents and Investments | Metric | Amount (as of June 30, 2025, in millions) | | :----- | :--------------------------------------- | | Unrestricted Cash, Cash Equivalents and Marketable Debt Securities | $145.6 | | Expected Funding Runway | Into mid-2027 | Second Quarter 2025 Financial Performance Protara experienced increased operating expenses in Q2 2025, leading to a higher net loss of $15.0 million, though net loss per share decreased to $(0.35) due to increased shares outstanding Operating Expenses (Q2 2025 vs. Q2 2024) | Expense Category | Q2 2025 (in thousands) | Q2 2024 (in thousands) | Change (YoY, in thousands) | % Change (YoY) | Primary Reason for Increase | | :---------------- | :--------------------- | :--------------------- | :------------------------- | :------------- | :-------------------------- | | Research and development | $10,770 | $6,387 | +$4,383 | +68.6% | Clinical trial activities for TARA-002 and IV Choline Chloride (+$3.9M) | | General and administrative | $5,816 | $4,274 | +$1,542 | +36.1% | Personnel-related expenses (+$0.6M), market development-related expenses (+$0.5M) | Net Loss and EPS (Q2 2025 vs. Q2 2024) | Metric | Q2 2025 (in millions) | Q2 2024 (in millions) | Change (YoY, in millions) | | :----- | :-------------------- | :-------------------- | :------------------------ | | Net Loss | $(14.960) | $(9.513) | $(5.447) | | Net Loss per Share (in dollars) | $(0.35) | $(0.45) | +$0.10 | | Weighted-average shares outstanding | 42,270,855 | 21,233,163 | +21,037,692 | Product and Disease Overviews This section provides detailed information on Protara Therapeutics' investigational products, TARA-002 and IV Choline Chloride, and the diseases they target About TARA-002 TARA-002 is an investigational cell therapy for NMIBC and LMs, derived from the same master cell bank as OK-432, hypothesized to activate immune responses - TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and LMs[10] - It has been granted Rare Pediatric Disease Designation by the U.S. Food and Drug Administration for LMs[10] - TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432 (Picibanil®), with manufacturing comparability shown[10] - It is hypothesized to activate innate and adaptive immune cells, producing a pro-inflammatory response and directly killing tumor cells to enhance the antitumor immune response[12] About Non-Muscle Invasive Bladder Cancer (NMIBC) NMIBC is the 6th most common cancer in the United States, accounting for approximately 80% of bladder cancer diagnoses, defined as cancer confined to the bladder's inner lining - Bladder cancer is the 6th most common cancer in the United States, with NMIBC representing approximately 80% of bladder cancer diagnoses[13] - Approximately 65,000 patients are diagnosed with NMIBC in the United States each year[13] - NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle[13] About Lymphatic Malformations (LMs) LMs are rare, congenital lymphatic vessel malformations, often diagnosed in early childhood and primarily located in the head and neck, causing significant morbidities - LMs are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system[14] - Most LMs are present in the head and neck region, with over 50% detected at birth and 90% diagnosed before the age of three years[14] - Common morbidities include compression of the upper aerodigestive tract, intralesional bleeding, impingement on critical structures, recurrent infection, and cosmetic/functional disabilities[14] About IV Choline Chloride IV Choline Chloride is an investigational intravenous phospholipid substrate replacement therapy for choline-deficient patients on parenteral support, holding FDA Orphan Drug and Fast Track designations with patents expiring in 2041 - IV Choline Chloride is an investigational, intravenous phospholipid substrate replacement therapy for patients receiving parenteral support (PS)[15] - Choline is an important substrate for phospholipids critical for healthy liver function, gene expression, cell membrane signaling, brain development, neurotransmission, muscle function, and bone health[15] - Approximately 78% of patients dependent on PS are choline-deficient, and of those, approximately 63% have some degree of liver dysfunction[15] - It has been granted Orphan Drug Designation by the FDA for prevention/treatment of choline deficiency in long-term PN and Fast Track Designation as a source of choline[15] - U.S. patents claiming a choline composition and method for treating choline deficiency with a choline composition have terms expiring in 2041[15] About Protara Therapeutics, Inc. Protara Therapeutics is a clinical-stage biotechnology company dedicated to advancing transformative therapies for cancer and rare diseases, with a portfolio including TARA-002 and IV Choline Chloride - Protara is a clinical-stage biotechnology company committed to advancing transformative therapies for people with cancer and rare diseases[16] - The company's portfolio includes lead candidate TARA-002 for NMIBC and LMs, and IV Choline Chloride for patients on parenteral nutrition[16] Forward-Looking Statements This section provides a disclaimer regarding forward-looking statements, highlighting inherent risks and uncertainties that could cause actual results to differ materially Disclaimer and Risk Factors Statements regarding future events are "forward-looking statements" subject to risks and uncertainties, and Protara disclaims any obligation to update them, except as required by law - Statements regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995[17] - Such statements are subject to risks and uncertainties, and actual results may differ materially from those expressed or implied[17] - Factors contributing to uncertainty include risks related to financial guidance, development programs, market conditions, strategic plans, financing, and general economic, industry, market, regulatory, political or public health conditions[17] - Protara undertakes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law[17] Financial Statements This section presents Protara Therapeutics' unaudited condensed consolidated financial statements, including balance sheets and statements of operations, for relevant periods Unaudited Condensed Consolidated Balance Sheets The balance sheet shows Protara's financial position as of June 30, 2025, with total assets decreasing by $24.5 million to $156.9 million, primarily due to reduced cash and cash equivalents, partially offset by increased marketable debt securities Unaudited Condensed Consolidated Balance Sheets (Selected Items, in thousands) | Item | June 30, 2025 | December 31, 2024 | Change (vs. Dec 31, 2024, in thousands) | | :-------------------------------- | :------------ | :---------------- | :--------------------------------------- | | Cash and cash equivalents | $31,496 | $162,798 | $(131,302) | | Marketable debt securities (current) | $90,720 | $7,494 | +$83,226 | | Marketable debt securities (non-current) | $23,392 | $- | +$23,392 | | Total current assets | $125,091 | $172,155 | $(47,064) | | Total assets | $156,933 | $181,454 | $(24,521) | | Total current liabilities | $9,761 | $10,961 | $(1,200) | | Total liabilities | $12,510 | $14,320 | $(1,810) | | Total stockholders' equity | $144,423 | $167,134 | $(22,711) | Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss For the three months ended June 30, 2025, Protara reported a net loss of $14.960 million, an increase from the prior year, driven by higher operating expenses, though net loss per share decreased to $(0.35) due to increased shares outstanding Unaudited Condensed Consolidated Statements of Operations (Selected Items, Three Months Ended, in thousands) | Item | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change (YoY, in thousands) | % Change (YoY) | | :-------------------------------- | :------------------------------- | :------------------------------- | :------------------------- | :------------- | | Research and development | $10,770 | $6,387 | +$4,383 | +68.6% | | General and administrative | $5,816 | $4,274 | +$1,542 | +36.1% | | Total operating expenses | $16,586 | $10,661 | +$5,925 | +55.6% | | Interest and investment income (expense) | $1,626 | $1,148 | +$478 | +41.6% | | Net income (loss, in thousands) | $(14,960) | $(9,513) | $(5,447) | +57.3% (loss increased) | | Net income (loss) per share, basic and diluted (in dollars) | $(0.35) | $(0.45) | +$0.10 | -22.2% (loss per share decreased) | | Weighted-average shares outstanding, basic and diluted | 42,270,855 | 21,233,163 | +21,037,692 | +99.1% | Unaudited Condensed Consolidated Statements of Operations (Selected Items, Six Months Ended, in thousands) | Item | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change (YoY, in thousands) | % Change (YoY) | | :-------------------------------- | :----------------------------- | :----------------------------- | :------------------------- | :------------- | | Research and development | $19,918 | $14,135 | +$5,783 | +40.9% | | General and administrative | $10,792 | $8,377 | +$2,415 | +28.8% | | Total operating expenses | $30,710 | $22,512 | +$8,198 | +36.4% | | Interest and investment income (expense) | $3,355 | $1,904 | +$1,451 | +76.2% | | Net income (loss, in thousands) | $(26,874) | $(20,608) | $(6,266) | +30.4% (loss increased) | | Net income (loss) per share, basic and diluted (in dollars) | $(0.65) | $(1.26) | +$0.61 | -48.4% (loss per share decreased) | | Weighted-average shares outstanding, basic and diluted | 41,493,714 | 16,327,056 | +25,166,658 | +154.1% | Company Contact This section provides contact information for Protara Therapeutics Investor Relations Contact Information This section provides the contact details for Protara Therapeutics' investor relations - Company Contact: Justine O'Malley, Protara Therapeutics, Justine.OMalley@protaratx.com, 646-817-2836[22]