Tyra Biosciences (TYRA) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Key milestones and pipeline focus

• Lead small molecule drug targets FGFR3, with first-in-human phase 1 data expected this fall and plans to submit an IND for phase 2 in achondroplasia soon.

• FGFR3 is a significant target in both bladder cancer and skeletal dysplasias, with the same molecule being developed for both indications.

• The SNAP Chemistry Platform enables rapid, structure-based drug design, with in-house crystallography and in vivo capabilities for fast iteration.

• The clinical trial for TYRA-300 is in two parts: dose escalation (completed without reaching MTD) and an ongoing efficacy-focused cohort in FGFR3-positive metastatic urothelial cancer.

• Data from the ongoing trial will inform dose selection and efficacy, with a focus on achieving strong disease control and improved tolerability.

Differentiation and selectivity

• TYRA-300 is designed for high selectivity, with a tenfold separation from other FGFR isotypes, aiming to minimize off-target toxicities.

• Dose-limiting toxicity for FGFR3 is overgrowth in animal models with open growth plates, while other FGFRs are associated with hyperphosphatemia, diarrhea, and liver enzyme increases.

• The company leverages deep expertise in FGFR biology and structure-based design, built specifically to address FGFR3 selectivity.

Market landscape and clinical benchmarks

• FGFR3 driver mutations are present in about 20% of late-line bladder cancer patients, with a large early-stage population also FGFR3-positive.

• Benchmark drug erdafitinib (Balversa) achieves a 35% ORR but is limited by tolerability issues, especially after the first month of treatment.

• The goal is to match efficacy benchmarks while reducing toxicities, particularly those seen with pan-FGFR inhibitors.

• Physicians are hesitant to prescribe erdafitinib due to side effects, leading to consideration of alternative therapies.