Taysha Gene Therapies — Third Quarter 2026 Earnings Summary

Financial Performance:

• Revenue was $0 for the three months ended September 30, 2026, compared to $0 for the same period in 2025; revenue was $0 for the nine months ended September 30, 2026, compared to $4.3 million for the same period in 2025.
• Research and development expenses were $28.8 million for the three months ended September 30, 2026, an increase of $3.1 million (12%) from $25.7 million in the prior year period, driven by higher clinical trial expenses and increased headcount.
• General and administrative expenses were $13.1 million for the three months ended September 30, 2026, an increase of $4.8 million (58%) from $8.3 million in the prior year period, primarily due to higher compensation, stock-based compensation, and commercial launch-readiness consulting fees.
• Net loss was $39.1 million ($0.10 per share) for the three months ended September 30, 2026, compared to a net loss of $32.7 million ($0.09 per share) in the prior year period.
• Net loss was $128.2 million for the nine months ended September 30, 2026, compared to $81.1 million for the same period in 2025.
• Cash and cash equivalents totaled $422.8 million as of September 30, 2026, compared to $319.8 million as of December 31, 2025.
• Total liabilities were $78.1 million as of September 30, 2026, down from $96.4 million as of December 31, 2025, with a term loan balance of $47.3 million.
• Stockholders' equity increased to $357.0 million as of September 30, 2026, from $246.9 million as of December 31, 2025.

Guidance and Future Outlook:

• Management expects to report topline data from the six-month interim analysis of the REVEAL pivotal trial and FDA feedback on the BLA submission pathway in the first half of 2027.
• FDA feedback following a CMC Type B meeting is expected in the first quarter of 2027.
• The Company anticipates nominating a second gene therapy development candidate targeting a CNS disorder with high unmet need and commencing preclinical activities in the first half of 2027.
• Current cash resources are expected to support planned operating expenses and capital requirements into the second half of 2028.
• A meeting with the FDA to discuss Chemistry, Manufacturing, and Controls (CMC) data is planned for the fourth quarter of 2026.

Business Segments and Product Lines:

• TSHA-102, an investigational gene therapy for Rett syndrome, continues to be generally well tolerated with no severe treatment-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs) observed across 33 patients in REVEAL Phase 1/2, pivotal, and ASPIRE trials as of the November 2026 data cutoff.
• All patients in the REVEAL Phase 1/2 (n=12), pivotal (n=17), and ASPIRE (n=4) trials have cleared the acute post-dosing safety monitoring period.
• The Company completed a BLA-enabling Process Performance Qualification (PPQ) manufacturing campaign for TSHA-102, meeting all predefined product specifications and comparability criteria for commercial product and clinical material.
• The PPQ campaign demonstrated a consistent single-digit empty capsid percentage, a key indicator of product quality.
• REVEAL Part A data may be included in the planned BLA submission due to comparability between clinical materials used in Part A and Part B pivotal trials.
• TSHA-102 received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA.

Market and Competitive Landscape:

• Rett syndrome is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K., with no currently approved disease-modifying therapies that treat the genetic root cause of the disease.
• The Company presented multiple Encore data supporting the TSHA-102 clinical program at the 55th Child Neurology Society Annual Meeting.

Risks and Challenges:

• The press release includes standard forward-looking statement disclaimers noting that actual results may differ materially due to various risks and uncertainties, including regulatory approval outcomes, manufacturing success, and market acceptance.
• Specific risks are detailed in the Company's SEC filings, including the Annual Report on Form 10-K for the year ended December 31, 2025.

Management Commentary and Tone:

• CEO Sean P. Nolan stated the Company continued to make strong progress across clinical, manufacturing, and commercial readiness activities.
• Management expressed confidence that the first half of 2027 could be a transformative period for Taysha.
• The tone emphasized the robust dataset being built to support the BLA submission and the ability to consistently manufacture high-quality product at commercial scale.

Other Key Points:

• The Company has 325,883,280 common shares issued and outstanding as of September 30, 2026, compared to 285,051,648 as of December 31, 2025.
• A net gain on lease termination of $3.4 million was recorded for the nine months ended September 30, 2025, with no such gain recorded in the current period.
• Interest income was $3.9 million for the three months ended September 30, 2026, compared to $3.2 million in the prior year period.
• The Company plans to discuss CMC data with the FDA in the fourth quarter of 2026 to strengthen the package for the potential BLA submission.