Tyra Biosciences Inc (Nasdaq: TYRA), a California-based clinical-stage biotechnology company, announced on Tuesday that it had presented its preclinical proof-of-concept results for TYRA-300, an investigational oral FGFR3 selective inhibitor, in hypochondroplasia (HCH) at the sixth Annual Achondroplasia & Skeletal Dysplasia Research Conference (Pharmachon 2024) scheduled from 28 to 30 June 2024.

HCH is a skeletal dysplasia closely related to achondroplasia (ACH), the most common form of dwarfism. HCH is most commonly caused by the N540K mutation (~70-80%) in the FGFR3 gene. There are currently no approved therapeutic options for HCH. The design of TYRA-300 may inhibit the alteration driving FGFR3-related skeletal dysplasias including ACH, HCH and others.

TYRA-300 is the company's lead precision medicine program stemming from its in-house SNÅP platform. TYRA-300 is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasias, including achondroplasia. In oncology, TYRA-300 is being evaluated in a multi-centre, open label Phase 1/2 clinical study, SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors), which was designed to determine the recommended Phase 2 dose (RP2D) of TYRA-300, as well as to evaluate preliminary antitumor activity. The company says that in skeletal dysplasias, TYRA-300 has demonstrated positive preclinical results in achondroplasia and hypochondroplasia, and it expects to submit an IND in the second half of 2024 for the initiation of a Phase 2 clinical study in paediatric achondroplasia. In July 2023 and January 2024, the FDA granted Orphan Drug Designation (ODD) and Rare Paediatric Designation (RPD) to TYRA-300, respectively, for the treatment of achondroplasia.

Todd Harris, TYRA CEO, said: "The new preclinical data presented at Pharmachon 2024 are very encouraging and continue to support our belief that TYRA-300 has the potential to become a best-in-class agent with the potential to address unmet medical needs for people with skeletal dysplasias. Developing TYRA-300 in HCH is a natural extension of our plans in ACH, and we look forward to submitting our IND in the second half of this year to support our planned Phase 2 study in paediatric achondroplasia."