Core Insights - Protara Therapeutics reported positive six-month data from the ADVANCED-2 trial of TARA-002 in patients with non-muscle invasive bladder cancer (NMIBC) and completed a public offering of approximately $100 million, extending its financial runway into 2027 [1][2][7] - The company anticipates key milestones in 2025, including initial data from 12-month evaluable NMIBC patients in the ADVANCED-2 trial and the commencement of the THRIVE-3 registrational trial of IV Choline Chloride [1][2][3][10] Group 1: TARA-002 in NMIBC - The ADVANCED-2 trial reported a complete response (CR) rate of 72% at six months and 70% at any time for BCG-Unresponsive patients, with a favorable safety profile [3][4] - The company plans to report data on 12-month evaluable patients in mid-2025 and results from a futility analysis of approximately 25 six-month evaluable BCG-Unresponsive patients by the end of 2025 [1][3] - Protara is exploring systemic priming dosing and combination therapy with TARA-002 for NMIBC patients [4] Group 2: IV Choline Chloride - The THRIVE-3 registrational trial for IV Choline Chloride is expected to begin in the first half of 2025, targeting patients dependent on parenteral support [1][10] - Previous studies indicated that 78% of PS-dependent patients were choline deficient, with 63% showing liver dysfunction [10] Group 3: Financial Guidance - Protara reported approximately $81.5 million in cash and equivalents as of September 30, 2024, along with the $100 million from the public offering, which is expected to fund operations into 2027 [7]

Core Insights - Protara Therapeutics, Inc. will present at the 43rd Annual J.P. Morgan Healthcare Conference on January 16, 2025, at 7:30 am PT in San Francisco [1] Company Overview - Protara Therapeutics is a clinical-stage biotechnology company focused on developing transformative therapies for cancer and rare diseases [3] - The company's lead candidate, TARA-002, is an investigational cell-based therapy aimed at treating non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) [3] - TARA-002 is currently being evaluated in a Phase 2 trial for NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to Bacillus Calmette-Guérin (BCG) treatment, as well as in a Phase 2 trial for pediatric patients with LMs [3] - Additionally, Protara is developing IV Choline Chloride, a phospholipid substrate replacement for patients on parenteral nutrition who cannot meet their choline needs through oral or enteral routes [3]

Core Points - Protara Therapeutics, Inc. has closed an underwritten public offering of 13,690,000 shares of common stock at a price of $6.25 per share, along with pre-funded warrants for 2,325,372 shares at $6.249 each, raising approximately $100 million in gross proceeds [1][2][3] Group 1: Offering Details - The offering includes a 30-day option for underwriters to purchase an additional 2,402,305 shares at the public offering price [1] - The net proceeds will be used to fund the clinical development of TARA-002 and other clinical programs, as well as for working capital and general corporate purposes [1] Group 2: Underwriters - TD Cowen, Cantor, LifeSci Capital, Oppenheimer & Co., and Scotiabank served as joint book-running managers for the offering [2] Group 3: Regulatory Information - The shares and warrants were issued under an effective shelf registration statement declared effective on November 14, 2023, by the U.S. Securities and Exchange Commission [3]

Core Insights - Protara Therapeutics, Inc. is advancing TARA002, a cell therapy for non-muscle invasive bladder cancer (NMIBC), with interim data from the Phase 2 ADVANCED-2 trial to be presented at the Society of Urologic Oncology's annual meeting in December 2024 [1][3] - The ADVANCED-2 trial includes BCG-unresponsive and BCG-naïve patients, with a focus on safety and efficacy data from approximately 20 enrolled patients [2][3] - TARA-002 is derived from a master cell bank of genetically distinct group A Streptococcus pyogenes and has shown manufacturing comparability with OK432, an approved immunopotentiator [3][4] Company Overview - Protara Therapeutics is a clinical-stage biotechnology company focused on developing therapies for cancer and rare diseases, with TARA-002 as its lead candidate for NMIBC and lymphatic malformations [6] - The company is also developing IV Choline Chloride for patients on parenteral nutrition [6] Industry Context - NMIBC accounts for approximately 80% of bladder cancer diagnoses in the U.S., with around 65,000 new cases each year, making it a significant area of focus for treatment advancements [5]

NEW YORK, Nov. 06, 2024 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced that management will participate in a fireside chat at the Guggenheim Inaugural Healthcare Innovation Conference on Wednesday, November 13, 2024, at 2:00 pm ET in Boston. A live webcast of the fireside chat can be accessed by visiting the Events and Presentations section of the Company’s website: htt ...

Core Insights - Protara Therapeutics announced the results of the THRIVE-1 study, highlighting a significant prevalence of choline deficiency and liver dysfunction in patients dependent on parenteral support [1][2][4] - The company is developing IV Choline Chloride as a potential treatment to address this unmet medical need, with plans to initiate a pivotal trial in Q1 2025 [1][3][6] Study Findings - The THRIVE-1 study involved 78 patients, revealing that 78% were choline deficient, and 63% of those with deficiency exhibited liver dysfunction, including steatosis and cholestasis [2][4] - The study underscores the critical need for intravenous choline supplementation in this patient population [2][3] Product Development - IV Choline Chloride is an investigational therapy aimed at providing choline when oral or enteral nutrition is insufficient or contraindicated [2][7] - The product has received Orphan Drug Designation from the FDA for preventing choline deficiency in patients on parenteral support [8] Market Opportunity - Approximately 40,000 patients in the U.S. on long-term parenteral support could benefit from an IV formulation of choline [8] - Currently, there are no approved IV choline products for patients dependent on parenteral support, despite recommendations from professional medical societies [5][3] Company Commitment - Protara is dedicated to bringing the first approved IV formulation of choline to the parenteral support community and is preparing for a registrational trial [3][6]

Clinical Trials and Drug Development - TARA-002 is currently in a Phase 1 open-label clinical trial for non-muscle invasive bladder cancer (NMIBC), with positive preliminary data showing favorable tolerability and anti-tumor activity at the 40KE dose[83]. - In the ADVANCED-1 trial, a complete response (CR) rate of 50% was observed in BCG-Unresponsive/Experienced patients with CIS-only, while the overall CR rate across 16 evaluable patients was 38%[87]. - The ongoing ADVANCED-2 trial is assessing TARA-002 in NMIBC patients, with preliminary results from a risk-benefit analysis expected in Q4 2024, involving approximately 10 patients[89]. - TARA-002 has received Rare Pediatric Disease designation for the treatment of lymphatic malformations (LMs) and is currently being evaluated in the STARBORN-1 Phase 2 trial[95][97]. - The STARBORN-1 trial aims to enroll approximately 30 patients, with the primary endpoint being the proportion of participants demonstrating clinical success in treating macrocystic and mixed-cystic LMs[97]. - The company is exploring higher dosing at an 80KE dose and combination therapies to enhance the treatment paradigm for NMIBC[90]. - TARA-002 is being developed for multiple oncologic indications and rare diseases, with worldwide rights secured excluding Japan and Taiwan[81]. Financial Performance and Capital Needs - The company has not generated any revenue from product sales and does not expect to do so in the near term, indicating a need for additional capital to finance ongoing and future clinical trials[99]. - As of June 30, 2024, the company had an accumulated deficit of approximately $221.0 million and expects to continue incurring significant operating losses for the next few years[100]. - The company reported cash, cash equivalents, and marketable debt securities of approximately $89.6 million as of June 30, 2024, an increase from $65.6 million as of December 31, 2023[116]. - Research and development expenses for the three months ended June 30, 2024, were approximately $6.4 million, a decrease of $0.9 million compared to the same period in 2023[111]. - General and administrative expenses for the three months ended June 30, 2024, were approximately $4.3 million, a decrease of $0.6 million compared to the same period in 2023[111]. - The net loss for the three months ended June 30, 2024, was approximately $9.5 million, a decrease of $1.8 million compared to a net loss of $11.3 million for the same period in 2023[109]. - For the six months ended June 30, 2024, the company incurred a net loss of approximately $20.6 million, compared to a net loss of $20.3 million for the same period in 2023[116]. - The company completed a private placement on April 10, 2024, raising approximately $42.0 million in net proceeds by selling 9,143,380 shares of common stock[101]. - Net cash used in operating activities was $18.1 million for the six months ended June 30, 2024, a decrease of $4.2 million compared to $22.3 million for the same period in 2023[121]. - The company reported net cash provided by investing activities of $26.0 million for the six months ended June 30, 2024, compared to $32.2 million for the same period in 2023[122]. - The company believes its current financial resources are sufficient to meet estimated liquidity needs for at least twelve months from the date of the report[117]. Legal and Compliance - The company has operating lease obligations primarily for its corporate headquarters and manufacturing facilities in North America[123]. - There are no recorded liabilities for certain contracts that require indemnification against third-party claims, as amounts cannot be reasonably estimated until a specific claim is asserted[124]. - The company has contingent milestone payments related to development, regulatory approval, and commercialization agreements, which are not included in the balance sheet due to their uncertain timing[125]. - As of June 30, 2024, the company's management evaluated the effectiveness of its disclosure controls and procedures, concluding they were effective at a reasonable assurance level[128]. - There were no changes in internal control over financial reporting during the quarter ended June 30, 2024, that materially affected the company's internal control[130]. - The company is not currently involved in any legal proceedings that are likely to have a material adverse effect on its business[130].

NEW YORK, June 24, 2024 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced the grant of inducement non-qualified stock options to purchase an aggregate of 118,400 shares of common stock to Lisa Schlesinger, Protara's newly appointed Vice President, Head of New Product Development and Market Access. About Protara Therapeutics, Inc. Justine O'Malley Protara Therapeutics Justi ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 | Title of each class | Trading Symbol(s) | Name of each exchange on which registered | | --- | --- | --- | | Common Stock, $0.001 par value per | TARA | The Nasdaq Capital Market | | share | ...

[Q1 2024 Business and Financial Overview](index=1&type=section&id=Protara%20Therapeutics%20Announces%20First%20Quarter%202024%20Financial%20Results%20and%20Provides%20Business%20Update) 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](index=1&type=section&id=Recent%20Progress%20and%20Highlights) 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](index=4&type=chunk) - Current cash resources, combined with private placement proceeds, are expected to fund operations and data milestones into **2026**[3](index=3&type=chunk)[5](index=5&type=chunk)[13](index=13&type=chunk) - 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](index=3&type=chunk)[5](index=5&type=chunk) - 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](index=3&type=chunk)[5](index=5&type=chunk) [Clinical Program Updates](index=2&type=section&id=Clinical%20Program%20Updates) 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](index=2&type=section&id=TARA-002%20in%20NMIBC) 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](index=9&type=chunk) - The CR rate was **63%** in CIS-only patients, **13%** in CIS +Ta/T1 patients, and **43%** in BCG-Unresponsive/Experienced patients[9](index=9&type=chunk) - TARA-002 was well-tolerated, with no Grade 3 or greater treatment-related adverse events reported[9](index=9&type=chunk) - The Phase 2 ADVANCED-2 trial for TARA-002 in BCG-Unresponsive and BCG-Naïve NMIBC patients expects preliminary results in **2H 2024**[9](index=9&type=chunk) [IV Choline Chloride for Patients on PN](index=2&type=section&id=IV%20Choline%20Chloride%20for%20Patients%20on%20PN) 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](index=7&type=chunk) - The indication was broadened from IFALD to a choline source for patients on long-term PN unable to synthesize it orally or enterally[7](index=7&type=chunk) [TARA-002 in LMs](index=2&type=section&id=TARA-002%20in%20LMs) 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](index=8&type=chunk)[3](index=3&type=chunk) - 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](index=8&type=chunk) [First Quarter 2024 Financial Results](index=3&type=section&id=First%20Quarter%202024%20Financial%20Results) 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](index=3&type=section&id=Financial%20Performance) 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](index=13&type=chunk) - The decrease in G&A expenses was primarily driven by a **$0.5 million** reduction in personnel-related costs, including stock-based compensation[13](index=13&type=chunk) [Financial Position](index=3&type=section&id=Financial%20Position) 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](index=13&type=chunk) - 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](index=13&type=chunk) 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](index=3&type=section&id=About%20Protara%27s%20Pipeline%20and%20Disease%20Areas) 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](index=3&type=section&id=About%20TARA-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](index=11&type=chunk) - 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](index=12&type=chunk) [About Non-Muscle Invasive Bladder Cancer (NMIBC)](index=4&type=section&id=About%20Non-Muscle%20Invasive%20Bladder%20Cancer%20%28NMIBC%29) 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](index=14&type=chunk) [About Lymphatic Malformations (LMs)](index=4&type=section&id=About%20Lymphatic%20Malformations%20%28LMs%29) 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](index=15&type=chunk) - Common morbidities include airway obstruction, intralesional bleeding, and impingement on critical structures[15](index=15&type=chunk) [About IV Choline Chloride](index=4&type=section&id=About%20IV%20Choline%20Chloride) 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](index=16&type=chunk) - 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](index=16&type=chunk) [Detailed Financial Statements](index=6&type=section&id=Detailed%20Financial%20Statements) 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](index=6&type=section&id=PROTARA%20THERAPEUTICS%2C%20INC.%20AND%20SUBSIDIARIES%20Unaudited%20Condensed%20Consolidated%20Balance%20Sheets) 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](index=7&type=section&id=PROTARA%20THERAPEUTICS%2C%20INC.%20AND%20SUBSIDIARIES%20Unaudited%20Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) 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)** |