Taysha Gene Therapies(US:TSHA)2025-11-04 12:12FDA Designations and Trials - TSHA-102 received FDA Breakthrough Therapy designation for Rett syndrome, highlighting its therapeutic potential for a significant unmet need[5] - The REVEAL pivotal trial protocol and statistical analysis plan (SAP) have been finalized with the FDA, including a six-month interim analysis that may expedite the BLA submission by at least two quarters[4] - The first patient in the REVEAL pivotal trial is scheduled to be dosed in Q4 2025, with additional patient enrollment expected to continue at multiple sites[10] - In Part A of the REVEAL trials, a response rate of 33% (5 out of 15 patients) was achieved, exceeding the minimum threshold for success[6] - 100% of patients (N=10) achieved multiple additional skills/improvements, totaling 22 developmental milestones and 165 additional skills across the cohort[6] - Taysha regained full rights to the TSHA-102 program, allowing for strategic flexibility and focus on long-term value[5] Financial Performance - Revenue for the three months ended September 30, 2025, was $1.788 billion, compared to $4.288 billion for the nine months ended September 30, 2025[17] - Total operating expenses for the three months ended September 30, 2025, were $34.024 million, an increase from $27.686 million for the same period in 2024[17] - Net loss for the three months ended September 30, 2025, was $32.733 million, compared to a net loss of $25.524 million for the same period in 2024[17] - Research and development expenses for the three months ended September 30, 2025, were $25.745 million, compared to $14.946 million for the same period in 2024[17] - Interest income for the three months ended September 30, 2025, was $3.169 million, compared to $2.107 million for the same period in 2024[17] Cash and Assets - As of September 30, 2025, Taysha had $297.3 million in cash and cash equivalents, expected to support operations into 2028[10] - Cash and cash equivalents increased to $297.344 million as of September 30, 2025, from $139.036 million as of December 31, 2024[19] - Total assets as of September 30, 2025, were $316.554 million, up from $160.364 million as of December 31, 2024[19] - Stockholders' equity rose to $218.989 million as of September 30, 2025, from $71.525 million as of December 31, 2024[19] Liabilities - Total liabilities increased to $97.565 million as of September 30, 2025, compared to $88.839 million as of December 31, 2024[19] Expenses Overview - Research and development expenses for Q3 2025 were $25.7 million, up from $14.9 million in Q3 2024, driven by BLA-enabling initiatives and increased headcount[10] - General and administrative expenses for Q3 2025 were $8.3 million, compared to $7.9 million in Q3 2024, primarily due to debt issuance costs[10] - The net loss for Q3 2025 was $32.7 million, or $0.09 per share, compared to a net loss of $25.5 million, or $0.10 per share, in Q3 2024[10]