Protara Therapeutics (TARA) TD Cowen 46th Annual Health Care Conference summary

Program overview and clinical data

• TARA-002 is a bacterial therapeutic engineered for inactivation, retaining immunogenicity without toxicity, and is being developed for NMIBC and lymphatic malformations.

• Historical use in Japan (as OK-432) showed survival benefits in over 65,000 cancer patients, with modernized understanding and manufacturing.

• In BCG-unresponsive NMIBC, six-month CR rate is 68%, the highest published, and 12-month CR is 33% in early data; BCG-naive cohort shows 68% CR at six months and 58% at 12 months.

• The product is positioned as a broad-spectrum immunopotentiator with strong safety and tolerability, aiming to address unmet needs in both BCG-unresponsive and BCG-naive populations.

• Enrollment for the BCG-unresponsive study is expected to complete in the second half of the year, with ongoing data maturation and updates planned at academic conferences.

Regulatory and trial design updates

• Phase 3 for BCG-naive/alternative population will be a randomized controlled trial (RCT) against investigator's choice of gemcitabine or mitomycin, with a total N under 500.

• FDA agreed to landmark six-month CR as the primary endpoint, with durability of response (DOR) at 12 months as a key secondary measure.

• The agency supports inclusion of BCG-exposed patients in the trial population, with study initiation targeted for the second half of the year.

• The company leverages real-world claims data to define the eligible patient population and demonstrate significant unmet need.

• Commercial readiness includes manufacturing scalability, ease of administration, and cost advantages, supporting broad adoption in community urology settings.

Lymphatic malformations and pipeline catalysts

• TARA-002 is also being developed for macrocystic and mixed lymphatic malformations, with a Type C FDA meeting scheduled and regulatory clarity expected in Q2.

• The addressable US incident population is estimated at 1,400–1,800 births annually, with about two-thirds being macrocystic or mixed; no approved product currently exists.

• Differential pricing strategies are planned for NMIBC and LM indications, leveraging different dosage forms and health economics value.

• Patient advocacy and targeted center coverage are key to reaching both incident and prevalent LM populations.

• Additional pipeline includes IV choline chloride for parenteral support patients, with a pivotal study underway and a primary endpoint of serum choline elevation.