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The investigational drug TYRA-300 demonstrated significant increases in growth and bone length in a mouse model of FGFR3-Related Skeletal Dysplasia on recent study.

TYRA-300 is TYRA Biosciences — a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in FGFR biology — lead precision medicine program. An investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasias including achondroplasia.

Tyra building


For skeletal dysplasias, TYRA-300 has demonstrated positive preclinical results and TYRA expects to submit an IND for the initiation of a Phase 2 clinical study in pediatric achondroplasia in 2024.


TYRA-300 is being evaluated in a multi-center, open label Phase 1/2 clinical study, SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors) as well as in preclinical studies with mouse models. Last October, during the American Society for Bone and Mineral Research (ASBMR) 2023 annual meeting, TYRA announced its latest results on TYRA-300 for achondroplasia. You can find a poster with this information here.


tyra poster detailImage credits: Tyra Biosciences



The main conclusions and results are as follows:

  • TYRA-300 increased bone length of the appendicular and axial skeleton in the FGFR3 mouse model
  • Improvements in the foramen magnum diameter were observed with TYRA-300
  • Histological staining indicated that TYRA-300 restored the architecture of the growth plate by improving proliferation and differentiation of chondrocytes
  • TYRA-300 was equally active against the FGFR3 G380R mutant and wild-type FGFR3 in a Nano BRETTM binding assay
  • The FDA granted TYRA-300 Orphan Drug Designation for the treatment of achondroplasia
  • TYRA expects to submit an IND to initiate a Phase 2 clinical study in pediatric achondroplasia in 2024

TYRA-300 aim is to address long-term complications and improve quality of life in affected people with skeletal dysplasias including achondroplasia. Using the data from SURF-301 and the additional preclinical data presented here, TYRA expects to submit an IND (Investigational New Drug Application) to initiate a Phase 2 clinical study in pediatric achondroplasia next year.
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