Protara Therapeutics (TARA) investor relations material

Key program updates and regulatory progress

• TARA-002 is advancing in both lymphatic malformations (LM) and non-muscle invasive bladder cancer (NMIBC), with actionable FDA feedback and streamlined review under the Office of Therapeutic Products.

• No changes were requested by the FDA to STARBORN-1 trial design, endpoints, or sample size for LM; non-clinical requirements are complete.

• Enrollment for STARBORN-1 is expected to complete in H2 this year, with interim data showing strong efficacy and durability.

• ADVANCED-2 enrollment for NMIBC is also targeted for completion in H2, with regulatory endpoints aligned with approved products in the BCG-unresponsive setting.

• A pivotal trial in the BCG-naive NMIBC population is set to start in H2, with FDA agreement on sample size and comparator arm.

Clinical efficacy and differentiation

• TARA-002 demonstrated 83% clinical success in LM, with 100% success at 8 weeks and all patients disease-free at 32 weeks post-treatment.

• Efficacy in LM exceeds historical OK-432 data, with potential advantages attributed to manufacturing processes.

• TARA-002 shows strong results in patients with prior treatment failures, including those who failed multiple chemoablative agents.

• In NMIBC, complete response rates are in the high 60s to 70s for both BCG-naive and unresponsive populations, with competitive durability.

• TARA-002 offers a differentiated mechanism, favorable safety, and dosing profile, and is positioned as a potential backbone therapy in NMIBC.

Market opportunity and pricing

• LM market includes an incident population of 1,500 new cases per year and a prevalent population of about 20,000 actively seeking treatment.

• TARA-002 is expected to address unmet needs in patients who have exhausted current sclerotherapy and surgical options.

• Pricing is anticipated to reflect enhanced value over PI3K inhibitors, which are priced up to $400,000 annually.

• NMIBC market opportunity is significant, with up to 600,000 new and 400,000 recurrent cases annually; bladder preservation is a key driver for therapy adoption.

• TARA-002's lack of overlapping toxicities enables combination with other agents, supporting broad adoption as the treatment landscape evolves.