Core Insights - Protara Therapeutics, Inc. is set to present updated interim data from the Phase 2 ADVANCED-2 trial of TARA-002 for BCG-Unresponsive non-muscle invasive bladder cancer (NMIBC) at the ASCO Genitourinary Cancers Symposium in February 2026 [1][4] 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 for NMIBC and lymphatic malformations [8] - TARA-002 is an investigational cell therapy derived from genetically distinct group A Streptococcus pyogenes, which has been granted Rare Pediatric Disease Designation by the FDA [5][8] Clinical Trial Details - The ADVANCED-2 trial (NCT05951179) is an open-label Phase 2 study assessing TARA-002 in NMIBC patients, specifically those who are BCG-Unresponsive or BCG-Naïve [3] - Approximately 25 patients will be evaluated for updated safety and efficacy data in the upcoming presentation [2] - The trial includes an induction course of six weekly intravesical instillations of TARA-002, followed by a maintenance course [3] Presentation Information - The poster presentation titled "ADVANCED-2: Interim efficacy and safety data in BCG-Unresponsive participants with high-grade non-muscle invasive bladder cancer" will take place on February 27, 2026 [4] - Another poster titled "Interim safety and tolerability of TARA-002 in patients with BCG-Naïve and Unresponsive high-grade non-muscle invasive bladder cancer in ADVANCED-2" will also be presented on the same day [5] Mechanism of Action - TARA-002 acts as a first-in-class TLR2/NOD2 agonist, activating both innate and adaptive immune pathways within the bladder wall, leading to a pro-inflammatory response and direct tumor cell death [6] Market Context - NMIBC accounts for approximately 80% of bladder cancer diagnoses in the U.S., with around 65,000 new cases diagnosed annually [8]

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PresentationWelcome, everyone. My name is [ Tai Pavumi ], and I'm welcoming you to the 44th JPMorgan Healthcare Conference. Our next presenting company is Protara Therapeutics. Protara is a clinical stage biotechnology company that's developing transformative therapies for people with rare cancer diseases. And presenting today is CEO, Jesse Shefferman. Please help me welcoming to the stage.Jesse SheffermanCo-founder, CEO, President & Director Thank you. Thanks, [ Tai ]. Thank you to the JPMorgan team for ha ...

Protara Therapeutics Conference Call Summary Company Overview - **Company**: Protara Therapeutics (NasdaqGM:TARA) - **Industry**: Biotechnology, focusing on rare diseases and oncology - **Key Areas**: Non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations Core Points and Arguments Oncology Focus - Protara is developing therapies for non-muscle invasive bladder cancer, with two late-stage programs targeting BCG unresponsive and BCG naive patients [3][4] - The ADVANCED-2 study is a significant registrational study for BCG-unresponsive patients, with expected enrollment completion by the end of 2026 [4] - A randomized controlled trial (RCT) for BCG-naive patients has been approved by the FDA, marking a first in the NMIBC setting [4][11] Lymphatic Malformations Program - Protara's TAR 002 program targets macrocystic lymphatic malformations, showing a 100% clinical success rate at the eight-week evaluation point [7][14] - The company received breakthrough therapy designation and fast-track designation from the FDA, indicating a significant unmet need in this area [15][16] Market Opportunities - In NMIBC, 80% of patients are treated in community settings, where safety and tolerability are critical [9] - Protara's 002 therapy is positioned as a best-in-class option due to its safety profile and ease of administration [10] - The market opportunity for lymphatic malformations is estimated at around 1,000 patients annually in the U.S., with a focus on macrocystic cases [17][20] Clinical Data and Expectations - Upcoming data release in February for the ADVANCED-2 study is anticipated to show a complete response (CR) rate similar to previous findings of 72.4% at six months and 50% at twelve months [6][27] - The company aims to demonstrate that BCG-experienced and BCG-refractory patients respond similarly to TAR 002 [8] Competitive Landscape - Protara aims to position its therapies as alternatives to BCG, particularly for patients who cannot receive BCG [25] - The company is focused on generating level one evidence through its RCT, which will enhance its standing with payers and in treatment guidelines [12][13] Future Developments - Protara expects to begin receiving FDA approvals for its programs starting in 2027, with a robust pipeline of late-stage developments [22] - The IV Choline Chloride program is also in phase three, targeting a patient population of approximately 40,000 in the U.S. [21] Additional Important Content - The company emphasizes the importance of patient advocacy in raising awareness about new treatment options for lymphatic malformations [19] - Protara's leadership reflects on the journey of developing their portfolio, highlighting the progress made since going public in 2020 [34][35]

Core Insights - Protara Therapeutics is on track to report interim results from approximately 25 six-month evaluable patients in the ADVANCED-2 trial in Q1 2026, focusing on BCG-Unresponsive patients [1][5] - The company has received Breakthrough Therapy and Fast Track designations for TARA-002 in lymphatic malformations (LMs), with a regulatory update expected in the first half of 2026 [1][4] - The first patient has been dosed in the THRIVE-3 registrational trial of IV Choline Chloride, with interim results anticipated in the second half of 2026 [1][7] - Protara completed a public offering of approximately $86 million, extending its cash runway into 2028 [1][8] Protara's Pipeline and Progress - The CEO emphasized the company's commitment to delivering transformative therapies for cancer and rare diseases, highlighting the promising clinical data for TARA-002 in non-muscle invasive bladder cancer (NMIBC) [2] - TARA-002 has shown meaningful and durable activity in BCG-Naïve NMIBC patients, with plans to provide an interim update on the registrational BCG-Unresponsive trial later this quarter [2][5] - Positive results from the Phase 2 STARBORN-1 trial of TARA-002 in LMs have led to FDA designations, with a regulatory update on the path to registration expected in H1 2026 [2][4] Clinical Trials and Manufacturing - Protara is evaluating subcutaneous dosing combined with intravesical dosing for TARA-002 in NMIBC patients, as well as exploring combination treatments [5] - TARA-002 has been selected for the FDA's CMC Development and Readiness Pilot Program, aimed at facilitating manufacturing development for therapies with compressed clinical timelines [6] Financial Update - The recent public offering of approximately $86 million is expected to support Protara's operations and extend its financial runway into 2028 [8]

Core Insights - Protara Therapeutics, Inc. has initiated patient dosing in its Phase 3 THRIVE-3 clinical trial for intravenous Choline Chloride, aimed at patients on long-term parenteral support [1][2] - The company believes that IV Choline Chloride could be the first FDA-approved IV choline therapy for patients dependent on parenteral support, addressing a significant unmet medical need [2][4] Company Overview - Protara Therapeutics is a clinical-stage biotechnology company focused on developing transformative therapies for cancer and rare diseases, with its lead candidate being TARA-002 for non-muscle invasive bladder cancer and lymphatic malformations [5][6] - The company is also developing IV Choline Chloride as a phospholipid substrate replacement therapy for patients unable to meet their choline needs through oral or enteral nutrition [4][5] Clinical Trial Details - The THRIVE-3 trial is a seamless Phase 2b/3 study designed to evaluate the efficacy and safety of low and high doses of IV Choline Chloride in patients receiving long-term parenteral support [2][3] - Approximately 105 additional patients will be enrolled in a 24-week Phase 3 double-blinded, randomized, placebo-controlled trial, following an 8-week Phase 2b trial involving 24 patients [2][3] Medical Context - Choline deficiency affects 78% of patients on parenteral support, leading to serious health issues, yet there are currently no approved IV choline products available [2][4] - IV Choline Chloride has been granted Fast Track and Orphan Drug Designation by the FDA, highlighting its potential significance in treating choline deficiency in this patient population [4][5]

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 aimed at treating non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs) [3] - Protara is conducting an ongoing Phase 2 trial for TARA-002 in NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to Bacillus Calmette-Guérin treatment, as well as a Phase 2 trial in pediatric patients with LMs [3] Upcoming Events - Management will present at the 44th Annual J.P. Morgan Healthcare Conference on January 14, 2026, at 3:45 pm PT in San Francisco [1] - A live webcast of the presentation will be available on the company's website, with an archive accessible for a limited time after the event [2] Additional Information - 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]

Core Insights - Protara Therapeutics, Inc. has received Breakthrough Therapy and Fast Track designations from the FDA for TARA-002, an investigational cell-based therapy aimed at treating pediatric patients with lymphatic malformations (LMs) [1][2] - TARA-002 has been selected for the FDA's Chemistry, Manufacturing, and Controls Development and Readiness Pilot Program, which supports expedited clinical development and earlier patient access [1][4] 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 [7] - The company is also developing IV Choline Chloride for patients on parenteral nutrition [8] Product Details - TARA-002 is a genetically distinct strain of streptococcus pyogenes, inactivated to retain immune-stimulating properties, and has been in clinical use in Japan for 30 years [5] - The therapy has previously received Rare Pediatric Disease designation and has shown significant clinical success in a Phase 2 trial involving over 500 patients [5] Disease Context - Lymphatic malformations are rare congenital conditions affecting lymphatic vessels, often diagnosed in early childhood, with serious complications including airway obstruction and recurrent infections [6]

Group 1 - Protara Therapeutics, Inc. has closed its underwritten public offering of 13,043,479 shares at a price of $5.75 per share, generating approximately $75 million in gross proceeds before deductions [1] - The company has granted underwriters a 30-day option to purchase an additional 1,956,521 shares at the public offering price [1] - Protara plans to use the net proceeds from the offering 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 - J.P. Morgan, TD Cowen, and Piper Sandler served as joint book-running managers for the offering, with LifeSci Capital as the lead manager and H.C. Wainwright & Co. as a manager [2] - The shares were issued under an effective shelf registration statement on Form S-3, declared effective by the SEC on November 14, 2023 [3]

Core Viewpoint - Protara Therapeutics, Inc. has announced a public offering of 13,043,479 shares of common stock priced at $5.75 per share, aiming to raise approximately $75 million for clinical development and other corporate purposes [1]. Group 1: Offering Details - The offering includes a 30-day option for underwriters to purchase an additional 1,956,521 shares at the public offering price [1]. - The offering is expected to close on December 8, 2025, pending customary closing conditions [1]. - Protara intends to use the net proceeds for the clinical development of TARA-002 and other clinical programs, as well as for working capital and general corporate purposes [1]. Group 2: Underwriters and Management - J.P. Morgan, TD Cowen, and Piper Sandler are acting as joint book-running managers for the offering [2]. - LifeSci Capital is serving as the lead manager, while H.C. Wainwright & Co. is acting as a manager of the offering [2]. Group 3: Regulatory Information - The shares will be issued under an effective shelf registration statement on Form S-3, declared effective on November 14, 2023, by the U.S. Securities and Exchange Commission [3]. - A final prospectus supplement will be filed with the SEC and will be available on their website [3].