Tyra Biosciences (TYRA) Conference Summary Company Overview - **Company**: Tyra Biosciences (TYRA) - **Focus**: Discovery and development of selective FGFR3 inhibitors for cancer and achondroplasia treatment - **Key Products**: Lead compound is an FGFR3 selective inhibitor, dabogratinib (formerly TYR3-100) Pipeline Initiatives - **Active Phase II Trials**: - **Bladder Cancer**: Targeting intermediate risk FGFR3 positive non-muscle invasive bladder cancer (NMIBC) with a patient population of approximately 700,000 in the US, where FGFR3 positivity is 70-80% [5][11] - **Achondroplasia**: Addressing skeletal dysplasia caused by FGFR3 alterations, which leads to significant health complications [6][11] Study Design and Efficacy - **NMIBC Study**: - Phase II study randomizing patients to receive either 50 mg or 60 mg of dabogratinib daily [12] - Primary endpoint: 3-month complete response (CR) rate; secondary endpoints include duration of response and time to recurrence [12] - Anticipated CR rate benchmark is around 70% based on expert feedback [26] - **Achondroplasia Study**: - Initiated with a focus on pediatric dosing, starting at 0.125 to 0.5 mg/kg [73] - Emphasis on FGFR3 selectivity to optimize growth and reduce complications associated with the condition [78][86] Safety and Tolerability - **Dabogratinib Safety Profile**: - Demonstrated a pristine safety profile with low-grade adverse events (AEs) such as diarrhea and asymptomatic ALT/AST elevations [19][40] - No hyperphosphatemia observed at doses of 50-60 mg, contrasting with other pan FGFR inhibitors [40][64] Competitive Landscape - **Comparison with Other Treatments**: - Dabogratinib shows a significantly improved safety profile compared to erdafitinib, with lower rates of nail toxicity and stomatitis [63] - The oral administration of dabogratinib is expected to enhance patient adherence compared to intravesical therapies [50][51] Market Dynamics - **Community Channel Dynamics**: - The evolution of oral therapies in urology, supported by EuroGPO, has facilitated the adoption of oral drugs in community practices [55][58] - Oral drug spend in urology has reportedly increased from 5% to 15% since 2008, with indications that it may be as high as 40% today [57] Future Outlook - **Prioritization of Trials**: - The company is focusing on the intermediate risk NMIBC and achondroplasia settings due to larger patient populations and unmet needs [70][72] - Future discussions with the FDA regarding the metastatic urothelial carcinoma setting are planned, but current focus remains on ongoing Phase II trials [69][72] Key Takeaways - Tyra Biosciences is advancing innovative treatments for bladder cancer and achondroplasia with a strong emphasis on safety and patient convenience - The company is well-positioned in the market with a promising pipeline and a clear strategy for commercialization and patient engagement - Ongoing studies are expected to provide critical data that will shape the future of treatment options in these therapeutic areas [21][72]

Core Insights - Tyra Biosciences has initiated the BEACH301 Phase 2 clinical study for dabogratinib, the only oral FGFR3-selective inhibitor in development for achondroplasia, marking a significant milestone in the treatment of this condition [1][2][3] Company Overview - Tyra Biosciences, Inc. is a clinical-stage biotechnology company focused on developing precision medicines targeting FGFR biology, utilizing its in-house SNÅP platform for drug design [9] - Dabogratinib is the lead candidate from Tyra's pipeline, designed to be a first-in-class oral FGFR3-selective inhibitor, with ongoing clinical trials for both pediatric achondroplasia and cancer [7][9] Clinical Study Details - The BEACH301 study will evaluate dabogratinib in children aged 3 to 10 with achondroplasia, enrolling treatment-naïve participants and those who have received prior therapies [4][5] - The study aims to assess safety, tolerability, and growth velocity changes, with a focus on improving the quality of life for children with achondroplasia [5][6] Market Context - Achondroplasia affects approximately 1 in 15,000 to 40,000 children, with around 250,000 individuals impacted globally, highlighting a significant unmet medical need [2][6] - The FGFR3 G380R mutation is responsible for about 99% of achondroplasia cases, indicating a targeted therapeutic approach could be beneficial [2][6]

Group 1 - Tyra Biosciences, Inc. is a clinical-stage biotechnology company focused on developing next-generation precision medicines targeting Fibroblast Growth Factor Receptor (FGFR) biology [2] - The company utilizes its in-house precision medicine platform, SNÅP, for rapid and precise drug design, predicting genetic alterations that may cause resistance to existing therapies [2] - Tyra's lead product, dabogratinib, is a potential first-in-class selective FGFR3 inhibitor designed to minimize toxicities associated with other FGFR inhibitors [2] Group 2 - Current clinical development plans for dabogratinib include studies for intermediate risk non-muscle invasive bladder cancer, pediatric achondroplasia, and potential future studies for metastatic urothelial cancer [2] - Tyra is also developing TYRA-430, an oral FGFR4/3-biased inhibitor for FGF19+/FGFR4-driven cancers, and TYRA-200, an oral FGFR1/2/3 inhibitor for metastatic intrahepatic cholangiocarcinoma [2] - The company will participate in investor events, including a virtual fireside chat on August 21, 2025, and the H.C. Wainwright 27th Annual Global Investment Conference on September 10, 2025 [1]