FDA grants TYRA-300 rare pediatric disease status

CARLSBAD, Calif. – Tyra Biosciences, Inc. (NASDAQ: TYRA), a biotech firm specializing in precision medicines for Fibroblast Growth Factor Receptor (FGFR) related conditions, has announced on Thursday that its drug candidate TYRA-300 received the Rare Pediatric Disease (RPD) Designation from the U.S. Food and Drug Administration (FDA). TYRA-300, an oral FGFR3 selective inhibitor, is being developed to treat achondroplasia, the most common form of dwarfism.

Achondroplasia is characterized by severe skeletal abnormalities and currently lacks approved treatments that address both immediate and long-term health complications. TYRA plans to submit an Investigational New Drug (IND) application to the FDA in the latter half of 2024, aiming to commence a Phase 2 clinical trial for children with this condition.

The RPD Designation is granted to drugs that target serious or life-threatening diseases affecting fewer than 200,000 people in the U.S., particularly those under 18 years of age. Should TYRA-300’s New Drug Application (NDA) get FDA approval, TYRA could receive a Priority Review Voucher, which expedites the review of a subsequent marketing application and can be sold or transferred.

TYRA-300 is part of TYRA’s lead precision medicine program, stemming from its proprietary SNÃ…P platform. Aside from its potential application in achondroplasia, TYRA-300 is also undergoing a Phase 1/2 clinical study, SURF301, to treat cancer, specifically advanced urothelial carcinoma and other solid tumors with FGFR3 gene alterations.

The FDA has previously awarded TYRA-300 with Orphan Drug Designation for the treatment of achondroplasia, adding to its recognition as a potential therapeutic option.

This announcement is based on a press release statement from Tyra Biosciences.

InvestingPro Insights

Tyra Biosciences’ recent FDA designation for its drug candidate TYRA-300 marks a significant milestone in its pursuit to address achondroplasia and other FGFR-related conditions. As investors evaluate Tyra’s potential, InvestingPro data provides insights into the company’s financial health and market performance. With a market capitalization of approximately $576.96 million and a price to book ratio of 2.52 as of the last twelve months leading up to Q3 2023, Tyra appears to maintain a solid financial structure. However, the company’s adjusted operating income shows a loss of $69.33 million in the same period, reflecting the high costs associated with drug development.

Investors should note that Tyra holds more cash than debt on its balance sheet and that its liquid assets exceed short-term obligations, which may provide some financial stability as it navigates the costly phases of clinical trials. On the other hand, analysts are cautious, having revised their earnings downwards for the upcoming period and not expecting profitability this year, as indicated by the negative P/E ratio of -9.46. Additionally, the company does not pay dividends, which is not uncommon for biotech firms focused on growth and research.

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