FDA Clears TYRA Biosciences' IND for TYRA-300 Phase 2 Trial in Pediatric Achondroplasia
核心洞察
The FDA has cleared Tyra Biosciences' IND application for TYRA-300, allowing a Phase 2 clinical trial (BEACH301) in children with achondroplasia (搜索) to proceed.
TYRA-300 is a potential first-in-class, oral, FGFR3 (搜索)-selective inhibitor designed to avoid toxicities associated with inhibiting FGFR1 (搜索), FGFR2 (搜索), and FGFR4 (搜索).
The BEACH301 trial will be a multicenter, open-label, dose-escalation/dose-expansion study evaluating TYRA-300 in children aged 3-10 with achondroplasia (搜索).
Tyra Biosciences has received FDA clearance for its Investigational New Drug (IND) application for TYRA-300, paving the way for a Phase 2 clinical trial (BEACH301) in children with achondroplasia (搜索). This clearance marks a significant step forward in the development of a potential new treatment option for this genetic condition.
TYRA-300 is an investigational, oral, FGFR3 (搜索)-selective inhibitor. It is designed to selectively target FGFR3 while avoiding the toxicities associated with inhibiting FGFR1 (搜索), FGFR2 (搜索), and FGFR4 (搜索). The drug is also under evaluation for metastatic urothelial cancer in the SURF301 study.
BEACH301 Trial Details
The BEACH301 trial is a Phase 2, multicenter, open-label study that will evaluate TYRA-300 in children aged 3 to 10 with achondroplasia (搜索) who have open growth plates. The trial will include both treatment-naïve children (Cohort 1) and those who have previously received growth-accelerating therapy (Cohort 2). Each cohort is expected to enroll up to 10 participants per dose level (0.125, 0.25, 0.375, 0.50 mg/kg) for up to 12 months. A safety sentinel cohort of up to 3 treatment-naïve participants per dose level in children ages 5 to 10 will be enrolled prior to initiation of Cohorts 1 and 2.
The primary objectives of the study are to assess the safety and tolerability of TYRA-300 and to evaluate changes from baseline in annualized growth velocity to determine appropriate doses for further development. Secondary objectives include evaluating changes from baseline in height z-score, proportionality, and pharmacokinetics (PK). Exploratory assessments of clinical outcomes, such as functional improvements, changes in the spine, and quality of life measures, are also planned.
Clinical Significance
Achondroplasia (搜索) is the most common form of dwarfism, affecting an estimated 1 in 15,000 to 40,000 children worldwide. The condition is primarily caused by a gain-of-function mutation in the FGFR3 (搜索) gene. Current therapies offer limited increases in annualized growth velocity and often require frequent injections.
"IND clearance to proceed with BEACH301 is a significant milestone for the achondroplasia (搜索) community and for TYRA," said Todd Harris, CEO of TYRA. "We believe FGFR3 (搜索) is the right target for achondroplasia, with almost one hundred percent of cases being driven by a specific mutation in the FGFR3 gene. TYRA-300 has the potential to precisely engage FGFR3 to potentially achieve a higher annualized growth velocity, and lead to important functional outcomes and clinical benefits such as improvements in reach, gait and spinal disease."
Development Timeline
TYRA Biosciences anticipates commencing dosing in the BEACH301 study in the first quarter of 2025. The FDA previously granted Orphan Drug Designation (ODD) in July 2023 and Rare Pediatric Disease Designation (RPD) in January 2024 to TYRA-300 for the treatment of achondroplasia (搜索).