Report Highlights and Corporate Update The company advanced its TSHA-102 program for Rett syndrome and extended its cash runway into 2028 with a successful financing - Commenced site activation for the REVEAL pivotal trial for Rett syndrome, with patient enrollment anticipated to begin in Q4 2025, following a No Objection Letter from Health Canada and feedback from the FDA13 - TSHA-102 continues to be generally well-tolerated, with no treatment-related serious adverse events (SAEs) or dose-limiting toxicities (DLTs) in the 12 patients treated in Part A of the Phase 1/2 trials17 - Data from the REVEAL Phase 1/2 trials showed a 100% response rate for the pivotal trial's primary endpoint, which is the gain or regain of at least one developmental milestone1 - Completed a public follow-on offering with gross proceeds of $230 million, extending the company's cash runway into 2028178 TSHA-102 Program Highlights The company finalized the REVEAL pivotal trial design for TSHA-102 following alignment with the FDA on key study parameters - REVEAL Pivotal Trial Design: - Study Type: Single-arm, open-label, with each patient as their own control - Dose: Single high dose of TSHA-102 (1x10¹⁵ total vector genomes) via lumbar intrathecal injection - Patient Population: 15 females aged 6 to <22 years in the developmental plateau stage of Rett syndrome - Primary Endpoint: Response rate, defined as the percentage of patients who gain or regain at least one developmental milestone from a predefined list of 28 - Analysis: A 12-month primary analysis and a 6-month interim analysis are planned4 - The FDA has provided written alignment on an extrapolation approach for a safety-focused study in females aged 2 to <6 years, which is intended to enable access for a broader patient population4 - Data presented at the IRSF Scientific Meeting highlighted positive clinical results, caregiver research supporting the trial's primary endpoint, and preclinical data on biodistribution7 Financial Performance The company reported a higher net loss driven by increased R&D expenses but maintained a strong cash position Q2 2025 vs Q2 2024 Performance | Financial Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Research & Development Expenses | $20.1M | $15.1M | +$5.0M | | General & Administrative Expenses | $8.6M | $7.3M | +$1.3M | | Net Loss | $26.9M | $20.9M | +$6.0M | | Net Loss Per Share | $0.09 | $0.09 | $0.00 | - The increase in R&D expenses was primarily driven by BLA-enabling manufacturing initiatives, REVEAL clinical trial activities, and higher compensation from increased headcount5 - As of June 30, 2025, the company had $312.8 million in cash and cash equivalents, which is expected to support operations into 20288 Condensed Consolidated Statements of Operations Operating expenses of $28.7 million resulted in a net loss of $26.9 million for the three months ended June 30, 2025 | (in thousands) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Revenue | $1,986 | $1,112 | | Total operating expenses | $28,739 | $22,411 | | Loss from operations | $(26,753) | $(21,299) | | Net loss | $(26,882) | $(20,928) | | Net loss per common share | $(0.09) | $(0.09) | Condensed Consolidated Balance Sheet Data Total assets grew to $333.3 million, driven by a significant increase in cash and cash equivalents to $312.8 million | (in thousands) | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $312,761 | $139,036 | | Total current assets | $316,264 | $142,130 | | Total assets | $333,331 | $160,364 | | Total current liabilities | $25,349 | $26,227 | | Total liabilities | $84,604 | $88,839 | | Total stockholders' equity | $248,727 | $71,525 | Outlook and Corporate Information The company outlines key upcoming milestones for its lead candidate TSHA-102, an AAV9-based gene therapy for Rett syndrome - Anticipated Milestones for Q4 2025: - Begin patient enrollment for the REVEAL pivotal trial - Report new supplemental clinical data from Part A of the REVEAL Phase 1/2 trials7 - TSHA-102 is an investigational gene therapy for Rett syndrome that uses a novel miRARE technology to regulate MECP2 gene expression and has received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations from the FDA10 - Rett syndrome is a rare genetic disorder affecting an estimated 15,000 to 20,000 patients in the U.S., EU, and U.K., with no currently approved therapies that treat the genetic root cause11
Taysha Gene Therapies(TSHA) - 2025 Q2 - Quarterly Results