Taysha Gene Therapies (TSHA) Q4 2024 earnings summary

Executive summary

• Completed dosing of 10 patients in Part A of both Phase II Reveal trials for TSHA-102 in Rett syndrome, covering high and low dose cohorts across pediatric, adolescent, and adult populations, with no treatment-related SAEs or DLTs as of February 2025 data cutoff.

• TSHA-102 demonstrated an encouraging safety profile and functional gains in core domains of Rett syndrome.

• Ongoing productive discussions with the FDA through the RMAT mechanism, with alignment on focusing pivotal trial endpoints on objective, clinically meaningful functional gains.

• Upcoming milestones include pivotal Part B trial design updates and additional clinical data in the first half of 2025.

Financial highlights

• Research and development expenses were $66 million for FY 2024, up from $56.8 million in FY 2023, mainly due to manufacturing and clinical trial activities.

• General and administrative expenses were $29 million for FY 2024, down from $30 million in FY 2023, primarily due to lower issuance costs related to pre-funded warrants.

• Net loss for FY 2024 was $89.3 million ($0.36 per share), an improvement from $111.6 million ($0.96 per share) in FY 2023.

• Cash and cash equivalents stood at $139 million as of December 31, 2024, expected to fund operations into Q4 2026.

Outlook and guidance

• Expect to provide pivotal Part B trial design and updated clinical data from both high and low-dose cohorts in the first half of 2025.

• Current cash resources projected to support planned operating expenses and capital requirements into Q4 2026.

• Continued focus on advancing TSHA-102 toward pivotal trials and regulatory alignment with the FDA.