Protara Therapeutics (TARA) 44th Annual J.P. Morgan Healthcare Conference summary

Key program updates and clinical progress

• Two late-stage oncology programs in non-muscle invasive bladder cancer (NMIBC): ADVANCED-2 for BCG-unresponsive and a new RCT for BCG-naive patients, both with anticipated significant data releases and regulatory milestones through 2026.

• ADVANCED-2 study expects to complete enrollment before end of 2026, with upcoming data on 25 evaluable patients in February; prior data in BCG-naive patients showed 72% CR at 6 months and 50% at 12 months.

• BCG-naive RCT will compare 002 to gemcitabine or mitomycin, targeting a patient population five times larger than BCG-unresponsive; enrollment expected to be rapid due to higher eligible patient numbers.

• Lymphatic malformations (LM) program with TARA-002 in children shows 80% clinical success and 100% in evaluable patients at 8 weeks; received breakthrough therapy and fast-track designations, with regulatory clarity expected by end of Q2 2026.

• IV choline chloride program in phase III for patients on parenteral support, with interim data from the dose confirmation lead-in expected mid-2026 and secondary endpoints by year-end 2026.

Market opportunity and strategic positioning

• NMIBC therapies focus on safety, tolerability, and ease of use, with 002 positioned as best-in-class for community urologists; no special handling, simple administration, and no post-procedure requirements.

• 002 aims to be the first choice for payers and physicians, supported by level one evidence from the RCT, which is expected to influence guidelines and reimbursement.

• LM market opportunity estimated at 1,000+ patients per year in the U.S., with pricing expected at the high end of rare disease analogs due to functional cure rates and significant quality-of-life improvements.

• IV choline chloride targets a U.S. patient population of about 40,000, representing a substantial rare disease market.

• Multiple late-stage programs provide diversified opportunities for success, with potential FDA approvals anticipated annually from 2027 through 2030.

Regulatory and clinical outlook

• ADVANCED-2 NMIBC study to complete enrollment by late 2026, with pivotal data readouts expected to support regulatory filings.

• LM program benefits from expedited FDA pathways, with a multidisciplinary meeting planned to define the registration path; clarity expected by Q2 2026.

• IV choline chloride pivotal study uses a seamless phase II/III design, with primary endpoint data in mid-2026 and secondary clinical outcomes by year-end.

• No head-to-head trial with BCG required for FDA approval of 002; focus is on providing an alternative for patients ineligible for BCG.

• Leadership expresses optimism as all programs approach key inflection points, with the portfolio expected to yield major clinical and regulatory milestones over the next several years.