Tyra Biosciences Inc (TYRA) (Nasdaq: TYRA), a California-based clinical-stage biotechnology company involved in developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, announced on Thursday that the first child has been dosed in BEACH301, a Phase 2 clinical study evaluating the safety and efficacy of dabogratinib in children with achondroplasia, the most common form of dwarfism.

Dabogratinib is a potential first-in-class, once-daily oral FGFR3 selective inhibitor that was discovered using TYRA's in-house SNAP platform.

"Achondroplasia is caused by an alteration in FGFR3, and we believe that precisely targeting the root cause of this condition is the key to transforming care," said Todd Harris, TYRA CEO. "Dabogratinib is the only oral, FGFR3-selective inhibitor in clinical development for achondroplasia, reflecting our commitment to bring forward therapies that are both innovative and accessible for children and their families. Dosing the first child in BEACH301 is more than a milestone for our company, it's a step toward a future where children with achondroplasia can live healthier, fuller lives."

It is estimated that 1 in 15,000 to 40,000 children are born with achondroplasia, and approximately 250,000 individuals are affected worldwide.

BEACH301 is a Phase 2, multicentre, open-label, dose-escalation/dose-expansion study evaluating dabogratinib in children ages 3 to 10 with achondroplasia who have open growth plates. The study will enrol children who are treatment-naive (Cohort 1) and those who have received prior growth-accelerating therapy (Cohort 2) at multiple sites worldwide. Each of these cohorts is expected to enrol up to 10 participants per dose level (0.125, 0.25, 0.375, 0.50 mg/kg) for up to 12 months. The study is also enrolling a safety sentinel cohort of up to 3 participants per dose level in children ages 5 to 10.

The primary objectives of this study will be to assess safety and tolerability in children with achondroplasia and evaluate change from baseline in annualised growth velocity to determine the dose(s) for further development. Secondary objectives will include evaluating change from baseline in height z-score, proportionality and pharmacokinetics.