Protara Therapeutics (TARA) investor relations material

Oncology program updates

• Two late-stage programs in non-muscle invasive bladder cancer (NMIBC): ADVANCED-2 for BCG-unresponsive and ADVANCED-3 for BCG-naive patients, both with registrational intent.

• ADVANCED-2 study nearing full enrollment, with data on 25 six-month evaluable patients expected in February; full enrollment anticipated before end of 2026.

• December data in BCG-naive patients showed 72% complete response (CR) at six months and 50% at 12 months; similar results expected in BCG-unresponsive population.

• 002 therapy highlighted for best-in-class safety, ease of use, and no special handling, making it attractive for community urologists.

• FDA agreed to a first-ever RCT in BCG-naive NMIBC, comparing 002 to gemcitabine or mitomycin, with primary endpoint at six months.

Lymphatic malformations (LMs) program

• TARA-002 demonstrated 80% clinical success in all patients and 100% in those evaluable at eight weeks, with most achieving success after two doses.

• Received breakthrough therapy and fast-track designations, plus invitation to FDA's CDRP accelerated review; multidisciplinary meeting with FDA planned to clarify registration path by end of Q2 2026.

• U.S. market estimated at 1,400–1,800 new cases annually, with two-thirds being macrocystic; prevalence model could expand patient pool.

• Pricing expected at the high end of rare disease analogs, justified by functional cure rates and significant quality-of-life improvements.

IV choline chloride program

• Seamless phase II/III trial underway for parenteral support patients; dose confirmation lead-in of 24 patients, followed by phase III expansion.

• Primary endpoint is serum choline elevation post-infusion; interim data from first 24 patients expected mid-2026, with secondary clinical endpoints available by end of 2026.

• U.S. patient population for this indication estimated at 40,000.