Tyra Biosciences (TYRA) TD Cowen 46th Annual Health Care Conference summary

Strategic program overview

• Lead asset Dabogratinib targets three blockbuster indications: upper tract urothelial carcinoma (UTUC), intermediate-risk non-muscle invasive bladder cancer (NMIBC), and achondroplasia, all driven by FGFR3 alterations.

• Phase 2 and pivotal studies are planned or ongoing, with key data readouts expected mid-year for NMIBC and by year-end for achondroplasia.

• Dose selection is informed by prior studies and aims to balance efficacy (≥70% CR rate) and optimal tolerability, leveraging learnings from other FGFR inhibitors.

• UTUC offers a rapid registrational path due to high FGFR3 positivity and significant unmet need, with a single-arm study design supported by FDA precedent.

• Achondroplasia program seeks to surpass current therapies in annualized height velocity, with a dose-response curve to be established in the Sentinel cohort.

Clinical development and safety insights

• NMIBC phase 2 study is signal-seeking, testing two oral doses with interim data focused on 3-month complete response (CR) rates; 10-15 patients per dose are enrolled.

• Lower doses (40–60 mg) of Dabogratinib show favorable safety: minimal discontinuations, low rates of diarrhea, and rare mild liver enzyme elevations.

• Phase 3 design for NMIBC may include a placebo-controlled arm, leveraging the oral route for robust efficacy and safety demonstration.

• UTUC dosing may be higher (60–80 mg) due to bulkier tumors and greater tolerance, with the goal of maximizing kidney preservation.

• Achondroplasia study will report on annualized height velocity and secondary endpoints like proportionality and tibial bowing, aiming for >7 cm/year at higher doses.

Market access and adoption dynamics

• Oral therapy offers a significant advantage over current intravesical and surgical treatments, simplifying patient experience and reducing procedural burden.

• Community urology practices, which treat 80% of patients, are well-equipped for oral drug dispensing and economically incentivized to adopt oral therapeutics.

• Academic centers may prefer procedures due to different economic structures, but the majority of uptake is expected in the community setting.

• UTUC market, though rare (~3,000 patients/year), is highly concentrated and can be addressed with a small, focused sales force.

• Achondroplasia patients and families seek greater efficacy than current options, with unmet demand for therapies that can deliver typical height outcomes.