FDA Grants Orphan Drug Status to TYRA-300: A Promising Treatment for Achondroplasia

Today, Tyra Biosciences, Inc. (Nasdaq: TYRA), a California-based clinical-stage biotechnology company that’s developing cutting-edge precision medicines targeting large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, has been given a major boost in its goals. The U.S. Food and Drug Administration (FDA) just granted the company’s lead precision drug therapy, TYRA-300, Orphan Drug Designation (ODD) for use in the treatment of achondroplasia, a type of dwarfism. This could be a key step in paving the way for more effective treatments for this and other types of dwarfism.

Achondroplasia is the most prevalent form of dwarfism – yet there are few effective treatments available. Those affected may suffer serious skeletal complications, ranging from cranial and spinal stenosis to hydrocephalus and sleep apnea. Fortunately, TYRA-300, an oral FGFR3 inhibitor, holds the potential to revolutionise the treatment of achondroplasia and other skeletal disorders, as 97% of achondroplasia cases are caused by just one mutation in FGFR3. Could this breakthrough finally bring relief to achondroplasia sufferers?

TYRA is proud to be one step closer to bringing a potential therapy to those who suffer from achondroplasia. Our therapy, TYRA-300, could not only address height issues, but also the long-term health complications that those with achondroplasia face. We have just been granted Orphan Drug Designation from the FDA, a crucial recognition of our ambition to make a difference in the achondroplasia community. With the goal of submitting an IND to the FDA for a Phase 2 study of TYRA-300 in pediatric achondroplasia in 2024, TYRA is proud to be part of a journey to improve the lives of those with achondroplasia.

The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for treatment, diagnosis or prevention of rare diseases and conditions that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits, including tax credits for qualified clinical trials and exemption from certain user fees to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.

TYRA is thrilled to announce the addition of Michael Bober, M.D. Ph.D. to the team as Vice President of Clinical Development and Medical Affairs. Dr. Bober is an expert in the field of skeletal dysplasia, serving on various medical and scientific advisory boards within the community. He brings a wealth of experience from his time as Medical Director of the Skeletal Dysplasia Program at Nemours Children’s Hospital in Delaware. With the valuable skills Dr. Bober brings, TYRA looks forward to continuing their efforts to immediately improve patient care in the field of Skeletal dysplasia.

I am thrilled to be joining TYRA and lend my expertise to the team working to develop TYRA-300 for the patient community. I have faith that TYRA-300 has the capability to enhance functionality and upgrade the quality of life for individuals with achondroplasia. It’s an honor to be part of the team and help make this possible!

About TYRA-300

TYRA-300 is the Company’s innovative precision medicine program, utilizing its in-house SNÅP platform. This investigational, oral, FGFR3-selective inhibitor 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 is currently enrolling adults with advanced urothelial carcinoma and other solid tumors to discover the optimal and maximum tolerated doses (MTD) as well as the recommended Phase 2 dose (RP2D). In addition, promising preclinical results in skeletal dysplasias, such as achondroplasia, have been observed and the Company is aiming to submit an IND for a Phase 2 pediatric clinical study in 2024.

About Tyra Biosciences

Tyra Biosciences, Inc. (Nasdaq: TYRA) is revolutionizing the medical industry with its cutting-edge precision medicine platform – SNÅP. This innovative platform allows for unprecedented drug design through the development of molecular SNÅPshots that identify and predict genetic alterations likely to create resistance to existing treatments. TYRA is laser-focused on applying its accelerated small molecule drug discovery engine to target oncology and other genetically defined conditions. Headquartered in Carlsbad, CA, we are dedicated to providing the world with life-changing medical solutions – discover more about our science, pipeline and people by visiting www.tyra.bio and connecting with us on LinkedIn!

Forward-Looking Statements 

TYRA is working to create revolutionary precision medicines that can potentially offer safer, more effective treatments for conditions such as achondroplasia. Our lead product candidate, TYRA-300, is currently being studied in clinical trials in order to determine its potential therapeutic benefits. We are in the early stages of our development process and are confident that our innovative SNÅP platform will enable the rapid and precise design of future drugs. Though there are risks associated with this process, such as potential delays in clinical trials and unexpected adverse side effects, we remain hopeful for the potential of TYRA-300 to become a safe and effective treatment option for achondroplasia.

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