Tyra Receives FDA Rare Pediatric Disease Designation, Raises $200 Million

Rare Daily Staff

Tyra Biosciences said it had entered into a private placement financing with institutional and accredited investors for approximately $200 million, which it will use to advance the clinical development of its pipeline, including TYRA-300, and for drug discovery, working capital, and general corporate purposes.

The financing comes as the U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Tyra for its 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 percent 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, 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.”

TYRA-300, the lead candidate stemming from Tyra’s SNÅP platform, is an investigational, oral, FGFR3-selective inhibitor currently in development for the treatment of cancer and skeletal dysplasias, including achondroplasia. In skeletal dysplasias, TYRA-300 has demonstrated positive preclinical results.

Rare Pediatric Disease 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 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. Most recently, Sarepta sold a PRV for $102 million.

Photo: Hiroomi Tada, chief medical officer of Tyra