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 pediatric and adolescent/adult cohorts; both high and low doses were well tolerated with no treatment-related SAEs or DLTs as of February 2025.

• Clinical data showed functional gains in fine/gross motor skills, socialization, communication, and seizure reduction, persisting and strengthening over time.

• Ongoing productive discussions with the FDA through the RMAT mechanism to advance to pivotal Part B trials in 2025, with pivotal trial design updates and additional clinical data expected in H1 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 expanded clinical trials.

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

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

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

Outlook and guidance

• Updates on pivotal Part B trial design and additional clinical data from both dose cohorts expected in the first half of 2025.

• Focus remains on objective, clinically meaningful functional outcomes as primary endpoints for pivotal trials.

• Ongoing regulatory discussions with the FDA, aiming for alignment on trial design and endpoints to coincide with clinical data updates.

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