Protara Therapeutics (TARA) Study update summary

Study design and patient cohorts

• The ADVANCED-2 phase II open-label trial evaluates TARA-002 in high-risk NMIBC patients, including both BCG-unresponsive and BCG-naïve cohorts, with induction and maintenance intravesical courses.

• BCG-unresponsive cohort targets 75-100 patients (median age 75, 72% male, 93% CIS only); BCG-naïve cohort includes 31 patients (median age 71, 81% male, 58% CIS only).

• Patients receive six weekly installations of TARA-002, followed by maintenance every three months; trial includes induction, maintenance, and re-induction phases, with a mandatory three-month biopsy.

• Central confirmation of active disease at baseline is required, contributing to a higher screen failure rate.

• About 35% of BCG-unresponsive patients had prior exposure to other investigational or approved therapies.

Efficacy and durability results

• In BCG-unresponsive patients, CR rates were 66%–68.2% at six months and 33%–33.3% at 12 months; in BCG-naïve, CR rates were 66.7%–72% at six months and 57.9%–58% at 12 months.

• Kaplan-Meier estimates for maintaining CR at six months were 71% (BCG-unresponsive) and 73% (BCG-naïve); 100% of evaluable responders maintained CR from month nine to 12 in both cohorts.

• High reinduction success: 61.5%–62% (BCG-unresponsive) and 66.7%–67% (BCG-naïve) of non-CR patients converted to CR at six months after reinduction.

• Durability of response is expected to improve as more patients reach the 12-month time point.

Safety and tolerability

• No Grade 3 or higher treatment-related adverse events reported among 74 participants; most adverse events were mild, transient, and grade one, including dysuria, bladder spasm, fatigue, and micturition urgency.

• No treatment-related discontinuations or serious adverse events occurred in either cohort.

• The therapy is well tolerated in older patients with significant comorbidities.