Protara Therapeutics (TARA) investor relations material

Key product updates and clinical data

• TARA-002, a cell-based immunopotentiator, is in late-stage development for non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LM), with registrational studies ongoing in both indications.

• Interim analysis from the ADVANCED-2 study in BCG-unresponsive NMIBC showed a 68% complete response rate at six months and a preliminary 33% rate at 12 months, with expectations that the 12-month rate will rise as more data matures.

• The STARBORN-1 study for LM has shown promising interim data, with ongoing enrollment and an upcoming FDA meeting to discuss the registrational path.

• IV Choline Chloride is being developed for patients on long-term parenteral nutrition, addressing a significant unmet need due to high rates of choline deficiency and associated liver dysfunction.

• The company holds an Orange Book-listed patent for IV Choline Chloride, providing exclusivity until 2041.

Market landscape and strategic positioning

• NMIBC market strategy focuses on both BCG-unresponsive and BCG-eligible but untreated or exposed populations, with ADVANCED-2 as the fastest path to registration and ADVANCED-3 targeting a larger market segment.

• Approximately 35% of BCG-eligible NMIBC patients in the US do not receive BCG, representing 11,000–15,000 patients annually, with an additional exposed population potentially numbering tens of thousands.

• TARA-002 aims to offer an alternative where BCG is unavailable or unsuitable, with ease of use and supply reliability as key differentiators.

• For LM, the peak opportunity is estimated at 1,000–1,200 patients annually, including both incident and previously untreated patients.

• Pricing strategies will leverage robust benchmarks in NMIBC and allow for differentiated branding and distribution between indications.

Regulatory and commercial outlook

• FDA has allowed comparator studies using chemotherapy rather than BCG for ADVANCED-3, streamlining the path to approval in a larger NMIBC population.

• Ongoing FDA engagement for both NMIBC and LM programs is expected to clarify registrational requirements and support future filings.

• Commercial infrastructure is being established, including J-codes and distribution channels, to support anticipated product launches.

• The company expects to be fully enrolled in ADVANCED-2 before the end of 2026, with ADVANCED-3 completion targeted about a year later.

• A priority review voucher (PRV) is anticipated from the LM program, with recent PRV sales valued at $205 million.