Taysha Gene Therapies(TSHA) - 2025 Q1 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Research and development expenses decreased to $15.6 million for Q1 2025 from $20.7 million in Q1 2024, a reduction of $5.1 million primarily due to lower GMP batch activities [18] - General and administrative expenses increased to $8.2 million for Q1 2025 from $7.1 million in Q1 2024, an increase of $1.1 million mainly due to higher compensation and legal fees [18] - Net loss for Q1 2025 was $21.5 million or $0.08 per share, compared to a net loss of $24.1 million or $0.10 per share in Q1 2024 [19] Business Line Data and Key Metrics Changes - The TATIA-one hundred two program has shown significant regulatory and clinical progress, supporting a clear path to registration [7][10] - The REVEAL Phase one/two trials for TATIA-one hundred two have reported clinical improvements in patients, including functional gains in fine motor, gross motor, and socialization skills [9][16] Market Data and Key Metrics Changes - The company has maintained ongoing discussions with the FDA, which have been productive and supportive of the development approach for TATIA-one hundred two [10][82] - The company is preparing to engage with EMA regarding requirements for potential expansion into European markets, indicating a significant patient population opportunity [66] Company Strategy and Development Direction - The company aims to finalize the protocols and statistical analysis plan for the pivotal Part B trial of TATIA-one hundred two, with an expected submission of the IND amendment in the current quarter [11][103] - The focus remains on advancing TATIA-one hundred two towards registration, with a clear path established through alignment with the FDA on trial design and endpoints [10][32] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of TATIA-one hundred two to provide meaningful benefits to patients with Rett syndrome, emphasizing the importance of early clinical improvements [11][17] - The company anticipates initiating site activities for the pivotal trial as early as summer 2025, with potential dosing beginning in Q4 2025 or Q1 2026 [90][103] Other Important Information - The company has received written alignment from the FDA on key elements of the pivotal trial design, which eliminates the need for a formal end-of-phase meeting [10][56] - The upcoming IRSF Scientific Meeting will feature presentations related to TATIA-one hundred two, providing further updates on clinical data and trial design [11][103] Q&A Session Summary Question: What signals can potentially point to a dose response given the heterogeneity of Rett syndrome? - Management indicated that temporal aspects and qualitative metrics like RMBA and CGI could be used to assess dose response, with expectations of differentiation at higher doses [22][23] Question: Can you frame the bar for success in light of recent regulatory feedback? - Management highlighted the importance of natural history analysis and alignment with the FDA on trial design, endpoints, and statistical significance requirements [30][32] Question: Is there any specific feedback from the FDA post the thirty-day period on the study protocol? - Management confirmed that they have reached alignment with the FDA on key aspects of the trial design, minimizing the likelihood of significant further comments [39][40] Question: What will be the primary endpoint of the trial? - The primary endpoint is expected to be objective and clinically meaningful, focusing on gains of function or restoration of lost function [44][95] Question: How does the aligned design compare with the initial proposal? - Management stated that the design endorsed by the FDA was consistent with the company's initial proposal, with no significant pushback from the agency [50][52] Question: What is the timeline for Part B enrollment and study completion? - Management anticipates initiating site activities and potentially beginning dosing in Q4 2025 or Q1 2026 following the IND amendment [90][103] Question: Can you characterize the level of confidence in TATIA-one hundred two's safety? - Management reported no treatment-related serious adverse events or dose-limiting toxicities, indicating a strong safety profile for the therapy [100][101]