Protara Therapeutics (TARA) investor relations material

Key program updates and clinical progress

• Three pivotal clinical programs in oncology and rare disease are ongoing, with key catalysts expected in the next 6–12 months.

• ADVANCED-2 trial for NMIBC (BCG-Unresponsive) is nearing enrollment completion, showing a 66% complete response rate at any time and 33% at 12 months as of January 2026.

• ADVANCED-3 trial is being redesigned to broaden patient inclusion and accelerate data generation for TARA-002.

• STARBORN-1 trial for lymphatic malformations aims to complete enrollment by year-end, with a BLA submission planned for the second half of next year.

• IV Choline Chloride pivotal trial is enrolling, targeting a rare disease population with no current approved IV choline therapy.

Clinical data highlights and safety

• TARA-002 in BCG-Naive NMIBC patients showed a 72% complete response rate at any time and 55% at 12 months as of April 2026.

• Durability of response is strong, with 91% of evaluable BCG-Naive responders maintaining CR from 9 to 12 months.

• Safety profile for TARA-002 is favorable, with most adverse events being mild and transient.

• In STARBORN-1, 83% of LM patients completing treatment achieved clinical success, and 100% of those assessed at 8 weeks post-treatment had clinical success.

• No serious adverse events reported in STARBORN-1; most were mild to moderate.

Market opportunity and strategic positioning

• NMIBC market includes over 25,000 high-risk patients annually in the U.S., with most treated in community practices.

• TARA-002 is positioned as easy to administer, with advantages in safety, efficacy, and workflow integration.

• TARA-002 is the only therapy in development with potential for safe systemic dosing, including subcutaneous maintenance.

• LMs represent a sizable rare disease market, with 20,000 patients in the U.S. seeking treatment for macrocystic and mixed cystic disease.

• IV Choline Chloride addresses an unmet need for 30,000 U.S. patients on long-term parenteral support.