Protara Therapeutics (TARA) Study Update summary

Study design and patient population

• Phase II open-label ADVANCED-2 trial evaluates TARA-002 in high-grade NMIBC, including BCG-unresponsive and BCG-naïve cohorts, with induction and maintenance dosing and reinduction for non-responders.

• BCG-unresponsive cohort is registrational and aligns with FDA guidance; BCG-naïve cohort serves as proof-of-concept.

• 24 subjects enrolled in interim analysis; majority male, median age 71, most with CIS only at baseline.

• Interim dataset includes 20 patients at three months, 18 at six months, and three at nine months.

• The trial aims to enroll 100 BCG-unresponsive and 27 BCG-naïve patients.

Efficacy results

• Six-month landmark complete response (CR) rate was 72% overall; 100% in BCG-unresponsive and 64% in BCG-naïve patients.

• High-grade CR at any time was 70% overall, 80% for BCG-unresponsive, and 67% for BCG-naïve subjects.

• 100% durability of CR from three to six months in all evaluable patients.

• 80% reinduction salvage rate observed among initial non-responders.

• Two of three patients maintained CR at nine months.

Safety and tolerability

• No grade two or higher treatment-related adverse events or drug-related serious adverse events reported.

• Most adverse events were grade one, transient, and consistent with immune potentiation or bacterial immunopotentiation.

• No patient discontinuations due to adverse events.

• Common AEs included dysuria, urinary incontinence, fatigue, chills, and bladder discomfort.

• Most common urinary symptoms were mild and resolved quickly.