CARLSBAD, Calif. — Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation to TYRA-300, an oral FGFR3 selective inhibitor, for the treatment of achondroplasia.
Achondroplasia is the most common form of dwarfism with limited therapeutic options. People living with achondroplasia may experience severe skeletal complications including foramen magnum and spinal stenosis, hydrocephalus and sleep apnea. A specific DNA mutation in FGFR3 causes an estimated 99% of achondroplasia. TYRA is planning to submit an Investigational New Drug application (IND) to the FDA in the second half of 2024 for the initiation of a randomized Phase 2 clinical trial evaluating multiple dose cohorts of TYRA-300 for children with achondroplasia.
“The Rare Pediatric Disease Designation recognizes the severity of complications associated with achondroplasia in childhood, and underscores our opportunity with TYRA-300 to develop a highly selective, oral medication that benefits the achondroplasia community,” said Hiroomi Tada, M.D. Ph.D., Chief Medical Officer of TYRA. “Children with achondroplasia face a significant unmet need – and currently, there are no approved treatment options that address the immediate and long-term health complications associated with this condition. We look forward to advancing TYRA-300 to the clinic and pursuing our goal to bring a much-needed therapy to these children.”
Rare Pediatric Disease (RPD) Designation is granted by the FDA to investigational drugs and biologics designed to address serious or life-threatening diseases which affect fewer than 200,000 people in the United States, or for which there is no reasonable expectation that the cost of developing and making the drug or biologic available in the U.S. for the applicable disease or condition will be recovered from sales in the U.S., and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If a New Drug Application (NDA) for TYRA-300 to treat achondroplasia is approved by the FDA, including pediatric populations, TYRA may be eligible to receive a Priority Review Voucher that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred. The FDA has implemented this program to encourage development of new drugs for treatment of rare pediatric diseases.
About TYRA-300
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-center, open label Phase 1/2 clinical study, SURF301 (Study in Untreated and Resistant FGFR3+ Advanced Solid Tumors). SURF301 (NCT05544552) was designed to determine the optimal and MTD and the recommended Phase 2 dose (RP2D) of TYRA-300, as well as to evaluate the preliminary antitumor activity of TYRA-300. SURF301 is currently enrolling adults with advanced urothelial carcinoma and other solid tumors with FGFR3 gene alterations. In skeletal dysplasias, TYRA-300 has demonstrated positive preclinical results, and the Company expects to submit an IND in the second half of 2024 for the initiation of a Phase 2 clinical study in pediatric achondroplasia. TYRA-300 has received Orphan Drug Designation and Rare Pediatric Designation for the treatment of achondroplasia from the FDA.
About Tyra Biosciences
Tyra Biosciences, Inc. (Nasdaq: TYRA) is a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in FGFR biology. The Company’s in-house precision medicine platform, SNÅP, enables rapid and precise drug design through iterative molecular SNÅPshots that help predict genetic alterations most likely to cause acquired resistance to existing therapies. TYRA’s initial focus is on applying its accelerated small molecule drug discovery engine to develop therapies in targeted oncology and genetically defined conditions. TYRA is based in Carlsbad, CA.
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