Taysha Gene Therapies(US:TSHA)2025-05-15 12:40PART I. FINANCIAL INFORMATION Condensed Consolidated Financial Statements (Unaudited) This section presents Taysha Gene Therapies' unaudited condensed consolidated financial statements for Q1 2025, covering balance sheets, operations, and cash flows with notes Condensed Consolidated Balance Sheets Total assets decreased to $138.4 million from $160.4 million as of March 31, 2025, primarily due to reduced cash and cash equivalents Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $116,593 | $139,036 | | Total current assets | $120,708 | $142,130 | | Total assets | $138,362 | $160,364 | | Liabilities & Equity | | | | Total current liabilities | $22,557 | $26,227 | | Term loan, net | $42,453 | $43,942 | | Total liabilities | $83,303 | $88,839 | | Total stockholders' equity | $55,059 | $71,525 | | Total liabilities and stockholders' equity | $138,362 | $160,364 | Condensed Consolidated Statements of Operations Q1 2025 revenue was $2.3 million with a net loss of $21.5 million, an improvement from Q1 2024's $24.1 million net loss, driven by reduced R&D Condensed Consolidated Statements of Operations (in thousands, except per share data) | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Revenue | $2,302 | $3,411 | | Research and development | $15,565 | $20,657 | | General and administrative | $8,158 | $7,084 | | Total operating expenses | $23,723 | $27,741 | | Loss from operations | ($21,421) | ($24,330) | | Net loss | ($21,529) | ($24,061) | | Net loss per common share, basic and diluted | ($0.08) | ($0.10) | Condensed Consolidated Statements of Cash Flows Net cash used in operating activities increased to $22.0 million in Q1 2025, with total cash decreasing by $22.4 million to $119.2 million Condensed Consolidated Statements of Cash Flows Highlights (in thousands) | Cash Flow Activity | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($22,020) | ($19,798) | | Net cash used in investing activities | ($371) | ($140) | | Net cash used in financing activities | ($52) | ($22) | | Net decrease in cash, cash equivalents and restricted cash | ($22,443) | ($19,960) | | Cash, cash equivalents and restricted cash at end of period | $119,193 | $126,580 | Notes to Condensed Consolidated Financial Statements Notes detail the company's business, accounting policies, liquidity, Astellas collaboration, term loan, stock compensation, and legal matters - The company is a clinical-stage biotechnology firm focused on AAV-based gene therapies for severe monogenic diseases of the central nervous system24 - As of March 31, 2025, the company had cash and cash equivalents of $116.6 million, which management believes is sufficient to fund planned operations for at least the next twelve months28 - Revenue is derived from the Astellas Option Agreement for the Rett syndrome program, with $2.3 million recognized in Q1 2025 from Rett research and development activities65 - The company is a nominal defendant in a consolidated stockholder derivative lawsuit and has received subpoenas from the SEC, both related to its August 2023 private placement, with possible losses currently inestimable131132 Management's Discussion and Analysis of Financial Condition and Results of Operations Management discusses advancing TSHA-102 for Rett syndrome, Q1 2025 financial results with decreased R&D and revenue, and liquidity into Q4 2026 Overview Taysha is a clinical-stage biotech focused on AAV-based gene therapies, with lead program TSHA-102 for Rett syndrome in Phase 1/2 trials and key regulatory designations - The company's primary focus is advancing TSHA-102, a gene therapy for Rett syndrome, which is currently in REVEAL Phase 1/2 trials for both adolescent/adult and pediatric patients142143 - TSHA-102 has received several key regulatory designations, including Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) from the FDA, as well as ILAP designation in the U.K145146 - As of March 31, 2025, the company had an accumulated deficit of $623.8 million and expects to incur significant operating losses for the foreseeable future150 Results of Operations Q1 2025 revenue decreased to $2.3 million, R&D expenses fell by $5.1 million to $15.6 million, and G&A expenses rose to $8.2 million Comparison of Results of Operations (in thousands) | Line Item | Q1 2025 | Q1 2024 | Change | | :--- | :--- | :--- | :--- | | Revenue | $2,302 | $3,411 | ($1,109) | | Research and development | $15,565 | $20,657 | ($5,092) | | General and administrative | $8,158 | $7,084 | $1,074 | | Net loss | ($21,529) | ($24,061) | $2,532 | - The $5.1 million decrease in R&D expenses was primarily driven by the completion of GMP batch manufacturing activities for TSHA-102 in the prior year period, partially offset by increased headcount183 Liquidity and Capital Resources As of March 31, 2025, cash and cash equivalents totaled $116.6 million, expected to fund operations into Q4 2026, with funding from equity, Astellas, and a $40.0 million term loan - The company's cash and cash equivalents of $116.6 million as of March 31, 2025, are expected to provide a cash runway into the fourth quarter of 2026188202 - In November 2023, the company secured a $40.0 million term loan from Trinity Capital, which is interest-only for 36 months (extendable to 48) and matures in November 2028189 - The company will require substantial additional capital to fund the research, development, manufacturing, and potential commercialization of its product candidates200202 Quantitative and Qualitative Disclosures About Market Risk As a smaller reporting company, Taysha is exempt from providing quantitative and qualitative disclosures about market risk - As a smaller reporting company, Taysha is exempt from providing quantitative and qualitative disclosures about market risk220 Controls and Procedures Management concluded that disclosure controls and procedures were effective as of March 31, 2025, with no material changes to internal control over financial reporting - The CEO and CFO concluded that the company's disclosure controls and procedures were effective as of March 31, 2025221 - No changes in internal control over financial reporting occurred during the quarter that materially affected, or are reasonably likely to materially affect, internal controls222 PART II. OTHER INFORMATION Legal Proceedings The company is a nominal defendant in a stockholder derivative lawsuit and received SEC subpoenas related to its August 2023 Private Placement, with potential losses inestimable - The company is involved in a consolidated stockholder derivative lawsuit alleging breach of fiduciary duty by certain directors and officers related to the August 2023 Private Placement, with litigation currently stayed until September 30, 2025225 - The SEC is conducting an investigation and issued subpoenas for materials related to the company's August 2023 PIPE and certain public offerings, with the company completing its production of materials in April 2025226 Risk Factors This section highlights risks from healthcare legislative and regulatory reforms, including the ACA and IRA, and increased scrutiny on drug pricing, potentially impacting profitability - The company's business is subject to risks from healthcare reform measures, such as the ACA and the Inflation Reduction Act (IRA), which could delay approvals, restrict activities, and affect profitability229231 - There is heightened governmental scrutiny on pharmaceutical pricing, which has led to legislation and executive orders designed to increase transparency and control costs, potentially creating downward pressure on prices for any approved products234 - Changes in regulations in key markets, such as the EU's new Clinical Trials Regulation (CTR), could impose additional costs, lengthen review times, and impact development plans237 Unregistered Sales of Equity Securities and Use of Proceeds The company reported no unregistered sales of equity securities, no use of proceeds from registered securities, and no issuer purchases during the period - There were no unregistered sales of equity securities during the quarter241 Defaults Upon Senior Securities This item is not applicable as the company has not defaulted upon any senior securities - Not applicable244 Mine Safety Disclosures This item is not applicable to the company's operations - Not applicable245 Other Information No other information was reported for the period - None246 Exhibits This section lists exhibits filed with the quarterly report, including corporate governance documents, incentive plan amendments, and officer certifications - The exhibits filed with this report include certifications from the Principal Executive Officer and Principal Financial Officer as required by the Sarbanes-Oxley Act248