Protara Therapeutics (TARA) Guggenheim Securities Inaugural Healthcare Innovation Conference summary

Lead program overview

• TARA-002 is a genetically distinct Strep pyogenes strain, previously approved in Japan for multiple cancers as Picibanil/OK-432, with a large safety database of 75,000 patients.

• Licensed globally (excluding Japan), TARA-002 is positioned as a broad-spectrum immunopotentiator, similar to BCG, with potential for repurposing in new indications.

Differentiation and clinical data in bladder cancer

• TARA-002 offers a broad immune response, potentially leading to durable outcomes in non-muscle invasive bladder cancer (NMIBC).

• Early phase I data showed a 63% complete response rate in CIS-only patients; ongoing phase II ADVANCED-2 trial includes both BCG-unresponsive and BCG-naive cohorts.

• The phase II study is designed for registrational intent, with reinduction and maintenance arms, targeting 127 patients.

• Six-month landmark complete response rates above 50% are considered adoptable, with 60%+ as the threshold for first-line consideration.

• Additional data from at least 10 patients, focusing on six-month durability, is expected later this year.

IV Choline program for parenteral support patients

• IV Choline addresses a significant unmet need in 26,000 US patients on long-term parenteral nutrition, most of whom are choline deficient and at risk for liver dysfunction.

• The THRIVE-1 study found 80% of these patients are choline deficient, with 65% showing liver dysfunction.

• The pivotal phase II-B/III THRIVE-3 trial will use a seamless design, with initial dose confirmation in 24 patients and a primary PK endpoint.

• Initial II-B data is expected in the second half of 2025, with full recruitment over 18 months.

• Fast Track designation from the FDA allows rolling NDA submission and ongoing regulatory dialogue.