Tyra Biosciences (TYRA) Jefferies London Healthcare Conference 2025 summary

Pipeline and clinical progress

• Lead drug dabogratinib, an FGFR3 selective oral inhibitor, is advancing in phase II trials for upper tract urothelial carcinoma, intermediate risk NMIBC, and achondroplasia.

• FGFR3 is a key genetic driver in bladder cancers and skeletal dysplasias, with high positivity rates in early-stage disease.

• Dabogratinib shows a cleaner safety profile compared to pan-FGFR inhibitors, with significant reduction in typical toxicities.

• Phase II NMIBC study is enrolling, with initial 3-month CR data from 20-30 patients expected by mid-next year.

• Achondroplasia phase II study is progressing through dose cohorts, with six-month AHV data expected in the second half of next year.

Market opportunity and physician adoption

• FGFR3-driven bladder cancer represents a multi-billion dollar market due to high unmet need and patient volume.

• Oral drug dispensing is now a major revenue driver for urology practices, supporting adoption of oral therapies.

• Urologists are enthusiastic about oral options, viewing them as transformative for patient care and practice economics.

• Upper tract urothelial carcinoma is a high unmet need area, with current therapies burdensome and limited in efficacy.

• Oral FGFR3 inhibitors could offer a Gleevec-like opportunity for long-term disease management.

Competitive landscape and differentiation

• Dabogratinib is the only FGFR3 selective oral drug advancing in these indications.

• Competing agents like erdafitinib and infigratinib have efficacy but are limited by off-target toxicities.

• Achondroplasia competitors have achieved up to 6 cm annualized height velocity (AHV); higher doses may yield more growth but with toxicity.

• Preclinical and clinical data suggest selective FGFR3 inhibition can optimize efficacy and safety.

• The company aims to double efficacy in achondroplasia compared to current agents by targeting higher, but safe, FGFR3 inhibition.