Tyra Biosciences' TYRA-300 promising for hypochondroplasia treatment

Tyra Biosciences has announced promising outcomes from a preclinical proof-of-concept study evaluating its leading drug candidate, TYRA-300, in animal models of hypochondroplasia (HCH). HCH is a form of skeletal dysplasia, a genetic disorder characterized by the underdevelopment of limbs and other body parts. This condition arises from a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, and as of now, there are no approved treatments available for those affected by HCH.

TYRA-300 is an oral inhibitor designed to specifically target the N540K mutation in the FGFR3 gene, which is believed to be responsible for up to 80% of HCH cases. In the preclinical study using a mouse model of HCH, TYRA-300 demonstrated a significant increase in bone length in the limbs of mice with the FGFR3 mutation. Additionally, the study found that the drug candidate was effective in binding the mutated FGFR3 N540K protein and targeting FGFR3 specifically over other isoforms.

These findings were presented at the 6th Annual Achondroplasia & Skeletal Dysplasia Research Conference (Pharmacon 2024) in Baltimore. Todd Harris, the CEO of Tyra Biosciences, expressed optimism about the new preclinical data presented at the conference. He stated that these results reinforce the potential of TYRA-300 to become a leading treatment option for individuals with skeletal dysplasias, addressing unmet medical needs.

The mechanism of action of TYRA-300 suggests it could be a promising treatment for multiple forms of skeletal dysplasia where the FGFR3 gene is mutated. This includes not only HCH but also achondroplasia (ACH), the most common form of dwarfism.

Based on the encouraging results of the preclinical study, Tyra Biosciences is planning to submit an investigational new drug (IND) application in the latter half of 2024. This application aims to initiate a phase 2 clinical trial in pediatric patients with achondroplasia. Dr. Michael Bober, Vice President of Clinical Development and Medical Affairs at Tyra, highlighted the improvements in growth plate function observed in the hypochondroplasia mouse model. He emphasized that this serves as strong proof-of-concept, supporting the further development of TYRA-300 for treating hypochondroplasia. Dr. Bober also noted that these findings contribute to the growing evidence that TYRA-300 could become a precision medicine for FGFR3-mediated skeletal dysplasia.

Tyra Biosciences was established in 2018, with a mission to develop drugs that target the fibroblast growth factor receptor (FGFR). The company utilizes a small molecule drug discovery platform known as SNÅP. This platform focuses on creating therapies for cancer and genetic conditions. TYRA-300 is presently being evaluated in a phase 1/2 clinical trial involving cancer patients with FGFR3 mutations. Additionally, two other drug candidates from Tyra Biosciences, which target different isoforms of FGFR, are currently undergoing preclinical studies.