Protara Therapeutics(TARA)

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Protara Therapeutics to Present Interim Analysis from the Phase 2 ADVANCED-2 Trial of TARA-002 in Patients with NMIBC at the American Urological Association Annual Meeting
Globenewswire· 2025-04-10 20:05
Core Insights - Protara Therapeutics, Inc. is presenting findings from the ongoing Phase 2 ADVANCED-2 trial of TARA-002 for non-muscle invasive bladder cancer (NMIBC) at the AUA 2025 Annual Meeting [1][2][3] Company Overview - Protara Therapeutics is a clinical-stage biotechnology company focused on developing transformative therapies for cancer and rare diseases, with TARA-002 as its lead candidate [10] - TARA-002 is an investigational cell therapy for NMIBC and lymphatic malformations (LMs), having received Rare Pediatric Disease Designation from the FDA [6][10] Trial Details - The ADVANCED-2 trial is assessing TARA-002 in NMIBC patients who are Bacillus Calmette-Guérin (BCG)-unresponsive or naïve, with approximately 100 patients in the BCG-unresponsive cohort and 30 in the BCG-naïve cohort [3] - The trial is designed to align with the FDA's guidance for developing treatments for BCG-unresponsive NMIBC [3] Presentation Information - Two presentations and a panel discussion on the ADVANCED-2 trial will occur at the AUA 2025 Annual Meeting from April 26 to April 29, 2025 [1] - The interim analysis abstract for the ADVANCED-2 trial is available on the AUA website, featuring updated safety and efficacy data [2] Mechanism of Action - TARA-002 is hypothesized to activate immune cells within the tumor, producing a pro-inflammatory response and releasing cytokines, while also directly killing tumor cells and inducing immunogenic cell death [8] Disease Context - NMIBC accounts for approximately 80% of bladder cancer diagnoses, with around 65,000 new cases diagnosed annually in the United States [9]
Protara Therapeutics (TARA) Moves to Buy: Rationale Behind the Upgrade
ZACKS· 2025-04-09 17:00
Protara Therapeutics (TARA) appears an attractive pick, as it has been recently upgraded to a Zacks Rank #2 (Buy). This upgrade primarily reflects an upward trend in earnings estimates, which is one of the most powerful forces impacting stock prices.The Zacks rating relies solely on a company's changing earnings picture. It tracks EPS estimates for the current and following years from the sell-side analysts covering the stock through a consensus measure -- the Zacks Consensus Estimate.The power of a changin ...
Protara Therapeutics Announces Encore Presentation of Results from THRIVE-1 Prospective Observational Study Evaluating the Prevalence of Choline Deficiency and Liver Injury in Patients Dependent on Parenteral Support
Newsfilter· 2025-03-19 12:00
Data to be featured during oral presentation at the American Society for Parenteral and Enteral Nutrition 2025 Nutrition Science & Practice ConferenceDosing of first patient in THRIVE-3 registrational trial of IV Choline Chloride in patients dependent on parenteral support expected in 1H 2025 NEW YORK, March 19, 2025 (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 da ...
Protara Therapeutics(TARA) - 2024 Q4 - Annual Report
2025-03-05 13:16
Clinical Trials and Development - TARA-002 is currently in the ADVANCED-2 Phase 2 trial for non-muscle invasive bladder cancer (NMIBC), with Cohort A expected to enroll 27 patients and Cohort B expected to enroll 75-100 patients[22][23] - The company plans to share 12-month interim data from Cohort A by mid-2025 and complete an interim analysis for Cohort B by the end of 2025[23] - The THRIVE-3 Phase 2b/3 trial for IV Choline Chloride is set to initiate in the first half of 2025, targeting 100 patients[28][39] - In September 2024, interim data from the STARBORN-1 trial showed that 2 out of 3 patients achieved a complete response after one injection of TARA-002 for lymphatic malformations[32] - The ongoing Phase 2 ADVANCED-2 trial reported a CR rate of 72% (13/18) at six months for BCG-exposed patients, with 100% (4/4) CR in BCG-Unresponsive patients[55] - The company plans to share interim 12-month data from the ADVANCED-2 trial by mid-2025 and is in discussions with the FDA regarding a registrational trial for TARA-002 in BCG-Naïve patients[57] - TARA-002 is currently undergoing a Phase 2 clinical trial (STARBORN-1) with approximately 30 pediatric patients aged 6 months to less than 18 years[88] - In the first cohort of the trial, 66.7% of patients treated with TARA-002 achieved a complete response after one dose[88] Product Designations and Regulatory Status - TARA-002 has received Orphan Drug Designation for lymphatic malformations and Rare Pediatric Disease Designation from the FDA[30][36] - IV Choline Chloride has been granted Orphan Drug Designation by the FDA for the prevention and/or treatment of choline deficiency in patients on long-term PN[74] - The FDA has granted Fast Track Designation to IV Choline Chloride as a source of choline when oral or enteral nutrition is not possible[74] - The FDA granted Rare Pediatric Disease Designation for TARA-002 in July 2020, which may provide eligibility for a priority review voucher[93] - The European Commission granted Orphan Drug Designation to TARA-002 in May 2022[94] Financial and Operational Status - 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 clinical trials[34] - The company has a limited operating history and has never generated any revenues, focusing on organizing, staffing, and developing pipeline assets like TARA-002 and IV Choline Chloride[218] - The company has not yet demonstrated the ability to successfully complete registrational clinical trials or commercialize any product candidates[218] - As of December 31, 2024, the company had 33 employees, with 20 engaged in research and development activities[210] - The company has undergone a merger, completing the reorganization with ArTara Subsidiary, Inc. on January 9, 2020, structured as a reverse merger[211] - The company has no products approved for commercial sale and has not generated any revenue from product sales or otherwise[218] Manufacturing and Capacity - TARA-002 manufacturing facility has an annual capacity of 20 million vials, with potential to expand capacity by 500%[63] - 47 successful consecutive batches of TARA-002 were manufactured by the end of 2024[63] - The manufacturing process for TARA-002 is modernized but equivalent to that of OK-432, with no Form 483s issued during the latest FDA inspection[95] Intellectual Property and Agreements - The company holds U.S. Patent 8,865,641 B2 for IV Choline Chloride, which provides protection until 2035, and additional patents expiring in 2041[121][135] - The agreement with Chugai Pharmaceutical provides exclusive access to materials and technical support for the development of TARA-002, valid through June 17, 2030[44] - Under the Chugai Agreement, the company is responsible for the development and commercialization of TARA-002 outside of Japan and Taiwan[96] - The company will pay Chugai Pharmaceutical a low single-digit million fee for each additional indication approval under the Chugai Agreement[97] - The company has a sponsored research agreement with The University of Iowa, providing $30,000 annually for project funding[103] - The company entered into a license agreement with The Feinstein Institute for Medical Research, granting an exclusive worldwide license for the treatment of fatty liver disease, with tiered royalties of 1.0% to 1.5% on net sales[114] Safety and Efficacy - TARA-002 demonstrated a favorable safety profile, with the majority of adverse events being Grades 1 and 2, and no Grade 3 or higher treatment-emergent adverse events reported[53] - Non-clinical studies indicate that TARA-002 may have superior tumor cell killing compared to BCG, with significant upregulation of pro-inflammatory cytokines[58] - In the ADVANCED-1 trial, TARA-002 showed a complete response (CR) rate of 38% across 16 evaluable patients, with a CR rate of 63% in CIS-only patients[53] Market and Competitive Landscape - The company is building its commercial infrastructure to launch and commercialize oncology and rare disease programs in key geographies, focusing on areas with high patient concentration[41] - The company faces competition from various pharmaceutical and biotechnology companies in the commercialization of its products[131] - The marketability of approved products may suffer if adequate coverage and reimbursement are not provided by government and third-party payors[199] Regulatory Compliance and Challenges - The FDA regulates the company's drug development processes, and failure to comply with regulations may result in sanctions[136][137] - The FDA requires completion of preclinical laboratory tests and animal studies in accordance with cGLP regulations before marketing biopharmaceutical products in the U.S.[138] - An IND must be submitted to the FDA and becomes effective 30 days after receipt unless safety concerns are raised, allowing clinical trials to commence[141] - Human clinical trials are conducted in three phases, with Phase 3 typically requiring two adequate and well-controlled trials to demonstrate efficacy[144] - The FDA aims to review standard applications for new molecular entities within ten months and priority reviews within six months after filing[149] - The NDA or BLA submission requires a substantial application user fee, which is typically increased annually[148] - Compliance with various federal and state healthcare regulations is essential, including adherence to the Anti-Kickback Statute and the False Claims Act, which impose significant penalties for violations[181][183] Pricing and Reimbursement - The company faces uncertainty regarding coverage and reimbursement for product candidates, which is critical for new product acceptance in the market[193][194] - Third-party payors increasingly challenge pricing and medical necessity, making reimbursement for branded drugs particularly difficult[197] - Legislative changes in the U.S. healthcare system may impact the marketing approval and profitability of product candidates[200] - The company must navigate complex pricing and reimbursement schemes in foreign markets, which may include mandatory clinical trials for cost-effectiveness[198]
Protara Therapeutics(TARA) - 2024 Q4 - Annual Results
2025-03-05 13:07
Financial Performance - Protara reported a net loss of $12.8 million, or $0.48 per share, for Q4 2024, compared to a net loss of $10.2 million, or $0.90 per share, in Q4 2023[14]. - For the full year 2024, the net loss was $44.6 million, or $2.17 per share, compared to a net loss of $40.4 million, or $3.57 per share, in 2023[14]. - The company reported a net income (loss) per share of $(0.48) for the three months ended December 31, 2024, compared to $(0.90) for the same period in 2023[21]. - Comprehensive loss for the year ended December 31, 2024, was $44,563,000, compared to $39,763,000 in 2023, reflecting an increase of 12%[21]. - Net loss for the year ended December 31, 2024, was $44,596,000, compared to a net loss of $40,420,000 in 2023, indicating a deterioration of 10%[21]. Cash and Investments - Cash, cash equivalents, and investments totaled $170 million as of December 31, 2024, expected to support operations into 2027[5]. - Cash and cash equivalents rose significantly to $162,798,000 in 2024, up from $39,586,000 in 2023, marking an increase of 311%[19]. - Interest and investment income for the year ended December 31, 2024, was $4,171,000, up from $3,193,000 in 2023, an increase of 30%[21]. Research and Development - Research and development expenses for Q4 2024 increased to $9.5 million from $6.4 million in the prior year, and for the full year increased to $31.7 million from $25.0 million in 2023[14]. - Research and development expenses increased to $31,704,000 in 2024 from $24,989,000 in 2023, a rise of 27%[21]. - Protara continues to explore combination treatment opportunities with TARA-002 in NMIBC patients, indicating significant potential for systemic dosing and combination therapy[8]. - The complete response (CR) rate in the ADVANCED-2 trial for TARA-002 was 72% (13/18) at six months and 70% (14/20) at any time[4]. - Initial data from 12-month evaluable patients in the ADVANCED-2 trial is expected to be reported by mid-2025[3]. - Protara plans to initiate the THRIVE-3 trial of intravenous Choline Chloride in the first half of 2025, with a total of 100 patients expected to be enrolled[6]. - The company expects to provide an interim update from the STARBORN-1 trial of TARA-002 in pediatric lymphatic malformations by the end of the first half of 2025[5]. Operating Expenses - Total operating expenses for the year ended December 31, 2024, were $49,154,000, compared to $43,613,000 in 2023, reflecting an increase of 13%[21]. - General and administrative expenses for Q4 2024 increased slightly to $4.8 million from $4.7 million in the prior year, while full-year expenses decreased to $17.5 million from $18.6 million in 2023[14]. Assets and Equity - Total assets increased to $181,454,000 in 2024 from $78,954,000 in 2023, representing a growth of 130%[19]. - Total stockholders' equity rose to $167,134,000 in 2024, up from $68,321,000 in 2023, an increase of 144%[19]. - The number of common shares outstanding increased to 35,044,772 in 2024 from 11,364,903 in 2023, representing a growth of 208%[19].
Protara Therapeutics Announces Fourth Quarter and Full Year 2024 Financial Results and Provides a Business Update
Newsfilter· 2025-03-05 13:00
Core Insights - Protara Therapeutics, Inc. is focused on developing transformative therapies for cancer and rare diseases, with significant progress reported in their clinical pipeline [2][16] - The company announced positive six-month data from the Phase 2 ADVANCED-2 trial for TARA-002 in non-muscle invasive bladder cancer (NMIBC) and plans to report initial 12-month data by mid-2025 [2][5] - Protara is set to initiate the pivotal THRIVE-3 trial for intravenous Choline Chloride in the first half of 2025, targeting patients on parenteral support [4][5] Recent Progress and Highlights - Positive interim results from the ADVANCED-2 trial showed a complete response (CR) rate of 72% at six months for NMIBC patients [6] - The company has a cash position of $170 million as of December 31, 2024, which is expected to fund operations into 2027 [5][14] - The THRIVE-3 trial will assess the efficacy and safety of IV Choline Chloride over 24 weeks in patients unable to receive oral or enteral nutrition [4][15] Financial Results - For Q4 2024, Protara reported a net loss of $12.8 million, or $0.48 per share, compared to a net loss of $10.2 million, or $0.90 per share, in Q4 2023 [14][20] - Research and development expenses increased to $9.5 million in Q4 2024 from $6.4 million in the prior year, primarily due to clinical trial activities [14][20] - Total operating expenses for the full year 2024 were $49.2 million, up from $43.6 million in 2023 [20]
Protara Therapeutics to Present at the TD Cowen 45th Annual Health Care Conference
Globenewswire· 2025-02-26 13:00
Company Overview - Protara Therapeutics, Inc. 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] - Protara is conducting ongoing Phase 2 trials for TARA-002 in NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to Bacillus Calmette-Guérin (BCG) treatment, as well as in pediatric patients with LMs [3] Upcoming Events - Management will participate in a fireside chat at the TD Cowen 45 Annual Health Care Conference on March 5, 2025, at 9:50 am ET in Boston [1] - A live webcast of the fireside chat will be available on the company's website and will be archived for a limited time after the presentation [2] Additional Developments - Protara is also developing IV Choline Chloride, an investigational phospholipid substrate replacement for patients on parenteral nutrition who cannot meet their choline needs through oral or enteral routes [3]
Protara Therapeutics to Present at the Oppenheimer 35th Annual Healthcare Life Sciences Conference
Globenewswire· 2025-02-05 13:00
Company Overview - Protara Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing transformative therapies for cancer and rare diseases [3] - The company's lead candidate is TARA-002, an investigational cell-based therapy targeting non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) [3] - Protara is conducting a Phase 2 trial for TARA-002 in NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to Bacillus Calmette-Guérin (BCG) treatment, as well as a Phase 2 trial in pediatric patients with LMs [3] - Additionally, Protara is developing IV Choline Chloride, a phospholipid substrate replacement for patients on parenteral nutrition [3] Upcoming Events - Management will participate in a virtual fireside chat at the Oppenheimer 35th Annual Healthcare Life Sciences Conference on February 12, 2025, at 12:40 pm ET [1] - A live webcast of the fireside chat will be available on the company's website and archived for a limited time [2]
Protara Highlights Recent Updates and Anticipated 2025 Milestones
Globenewswire· 2025-01-13 13:00
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]
Protara Therapeutics to Present at the 43rd Annual J.P. Morgan Healthcare Conference
Globenewswire· 2025-01-08 13:00
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]