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 good manufacturing practices batch activities [22] - 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 [22] - 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 [23] Business Line Data and Key Metrics Changes - The TATIA-102 gene therapy program for Rett syndrome has shown significant regulatory and clinical progress, with alignment from the FDA on key elements of the pivotal trial design [8][11] - The REVEAL Phase 1/2 trials have reported clinical improvements in patients, including functional gains in fine motor, gross motor, and socialization skills [10][18] 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-102 [11][20] - The potential market opportunity for TATIA-102 is significant, given the unmet medical need in the Rett syndrome patient population [16] Company Strategy and Development Direction - The company is focused on advancing the TATIA-102 gene therapy program towards registration, with plans to submit the pivotal trial protocol and statistical analysis plan in the current quarter [11][24] - The strategy includes leveraging existing clinical sites for rapid enrollment in the pivotal trial, with a focus on minimizing the time to market [66] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway and the potential for TATIA-102 to provide meaningful benefits to patients with Rett syndrome [13][24] - The company anticipates initiating site activities for the pivotal trial as early as summer 2025, with potential dosing in Q4 2025 or Q1 2026 [93] Other Important Information - The company plans to provide updates on the pivotal trial design and clinical data at the International Rett Syndrome Foundation's scientific meeting in June 2025 [13][24] - The company has $116.6 million in cash and cash equivalents as of March 31, 2025, which is expected to support operations into Q4 2026 [23] Q&A Session Summary Question: What signals can potentially point to a dose response? - Management indicated that temporal aspects and qualitative metrics like RMBA and CGI could help assess dose response, with expectations of differentiation at higher doses [26][27] Question: Can you frame the bar for success regarding recent regulatory feedback? - Management emphasized the importance of natural history analysis and alignment with the FDA on trial design and endpoints, which will provide insights into the probability of success [32][35] Question: Is there any specific feedback from the FDA post-protocol submission? - Management confirmed that they have reached alignment with the FDA on key aspects of the trial design, minimizing the likelihood of significant further comments [41][42] Question: What will the primary endpoint include? - The primary endpoint is expected to be objective and clinically meaningful, focusing on gains of function or restoration of lost function [45][49] Question: How does the aligned design compare with the initial proposal? - Management stated that the FDA endorsed the design proposed by the company, with no significant pushback during discussions [52][56] Question: What is the timeline for Part B enrollment and study completion? - Management indicated that if the protocol submission goes smoothly, site activities could begin shortly after, with potential dosing starting in late 2025 or early 2026 [93] Question: Can you characterize the confidence in TATIA-102's safety? - Management reported no treatment-emergent adverse events or dose-limiting toxicities to date, indicating a favorable safety profile [105]