Protara Therapeutics (TARA) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Oncology and NMIBC Program Updates

• TARA-002 demonstrated a 72% six-month complete response rate in NMIBC, with 100% CR at six months in BCG-unresponsive and 64% in BCG-naive cohorts, and no Grade 2 or higher treatment-related adverse events.

• Phase 2 ADVANCED-2 trial is ongoing for BCG-unresponsive NMIBC, with expansion into BCG-naive, combination, and systemic priming dosing cohorts; interim 12-month data expected mid-2025.

• TARA-002, a fully inactivated bacterial immunopotentiator, offers higher tumor cell killing and cytokine upregulation than BCG, with simpler administration and no special handling required.

• Reinduction salvage rates reach 83%, outperforming competitors, and the safety profile remains clean and well-tolerated.

• International expansion of NMIBC trials is underway, with 25+ US sites targeted by Q1'25 and ongoing expansion in South America and Asia.

Rare Disease Portfolio and Clinical Progress

• IV choline chloride program targets 30,000–40,000 US parenteral nutrition patients, with a phase 3 pivotal study (THRIVE-3) to initiate in 1H'25 and primary endpoint at eight weeks.

• Observational studies show 78% of parenteral support patients are choline deficient, with 63% experiencing liver damage and additional bone and cognitive complications.

• Phase 2 data demonstrate significant improvements in liver fat and cholestatic injury after choline restoration.

• Seamless phase 2b/3 design will confirm dosing and measure serum choline concentration, with initial results expected by year-end.

• Choline supplementation is guideline-recommended in the US and Europe, and IV Choline has patent protection to 2041 with a streamlined FDA pathway.

Lymphatic Malformations and Expansion Opportunities

• STARBORN-1 Phase 2 trial for TARA-002 in pediatric lymphatic malformations is ongoing, leveraging data from a 550-patient study with OK-432, which showed 69% clinical success and 84% in macrocystic cases.

• Drug is safe and appropriate for pediatric use, with high demand and full waitlists at study centers.

• Strategy includes seeking a priority review voucher and expanding into other maxillofacial cystic indications, potentially addressing 12.5 million US patients.

• FDA engagement is ongoing for potential accelerated approval, with interim data and expanded age cohorts expected in 2025.

• Only novel NMIBC agent with systemic dosing potential and first to publish efficacy in BCG-naive patients.