Protara Therapeutics Announces Key Milestones for 2025, Including NMIBC and LM Data
Key Insights
Protara Therapeutics anticipates mid-2025 data from the ADVANCED-2 trial of TARA-002 in non-muscle invasive bladder cancer (NMIBC (search)) patients, building on positive six-month results.
The company expects to initiate the THRIVE-3 registrational trial of IV Choline Chloride (search) for parenteral support-dependent patients in the first half of 2025.
By the end of the first half of 2025, Protara plans to release data from additional cohorts of the Phase 2 STARBORN-1 trial of TARA-002 in pediatric lymphatic malformations (search) (LMs (search)).
Protara Therapeutics is poised for a data-rich 2025, with key milestones anticipated across its clinical programs in non-muscle invasive bladder cancer (NMIBC (search)), lymphatic malformations (search) (LMs (search)), and parenteral nutrition. The company is building on positive momentum from 2024, including promising interim data for TARA-002 in NMIBC.
TARA-002 in Non-Muscle Invasive Bladder Cancer
Protara's lead candidate, TARA-002, is under evaluation for the treatment of NMIBC (search). Interim results from the Phase 2 ADVANCED-2 trial, presented in December 2024, demonstrated a 72% complete response (CR) rate at six months and 70% at any time in BCG-unresponsive and BCG-naive patients with carcinoma in situ (search) (CIS). Notably, 100% of patients who achieved a CR at three months maintained it at six months. The treatment also exhibited a favorable safety profile, with no Grade 2 or higher treatment-related adverse events reported.
The company expects to report data on 12-month evaluable patients from the ADVANCED-2 trial in mid-2025. Additionally, results from a futility analysis of approximately 25 six-month evaluable BCG-Unresponsive patients are expected by the end of 2025. This cohort is designed to be registrational, aligning with FDA guidance on developing drugs for BCG-unresponsive NMIBC (search). Protara also plans to provide an update on the design of its planned BCG-Naïve registrational trial following regulatory alignment by mid-2025. The company is also exploring systemic priming dosing prior to intravesical administration, as well as combination therapy with TARA-002.
IV Choline Chloride for Parenteral Support
Protara is advancing IV Choline Chloride (search), an investigational phospholipid substrate replacement, for adolescents and adults dependent on long-term parenteral support (PS) when oral or enteral nutrition is not feasible. The company anticipates commencing the THRIVE-3 registrational trial in the first half of 2025. The FDA previously granted Fast Track designation to IV Choline Chloride for this patient population.
Data from the THRIVE-1 observational study, announced in September 2024, revealed that 78% of PS-dependent patients were choline deficient, and 63% of these choline deficient patients exhibited liver dysfunction (search), including steatosis, cholestasis, and hepatobiliary injury.
TARA-002 in Lymphatic Malformations
Protara is also investigating TARA-002 in pediatric patients with macrocystic and mixed cystic lymphatic malformations (search) (LMs (search)) in the Phase 2 STARBORN-1 trial. Initial results from additional cohorts in the STARBORN-1 trial are expected by the end of the first half of 2025. The company previously reported encouraging efficacy and tolerability in the study's first safety cohort.
Financial Position
Protara's financial position is strong, with approximately $81.5 million in cash, cash equivalents, and investments as of September 30, 2024, supplemented by approximately $100 million in gross proceeds from a December 2024 public offering. The company believes this funding will be sufficient to support its planned operations into 2027.