Commenced site activation for REVEAL pivotal trial in accordance with previously aligned upon key design elements, following receipt of No Objection Letter (NOL) from Health Canada and feedback from the FDA; patient enrollment anticipated to begin in Q4 2025
High dose and low dose TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in the 12 patients treated in Part A of REVEAL Phase 1/2 trials as of August 2025 data cutoff
Part A data from REVEAL Phase 1/2 trials presented at IRSF Scientific Meeting showed 100% response rate for pivotal trial primary endpoint of gain/regain of ≥ one developmental milestone, corroborated by improvements in key secondary endpoints post-TSHA-102
Company anticipates reporting new supplemental REVEAL Part A clinical data supporting TSHA-102 therapeutic impact in Q4 2025
Gross proceeds of $230 million public follow-on offering, including full exercise of the underwriters’ option to purchase additional shares, strengthen balance sheet and extend cash runway into 2028
Conference call and webcast today at 8:30 AM Eastern Time
DALLAS, Aug. 12, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today reported financial results for the second quarter ended June 30, 2025, and provided a corporate update.
“We have made strong progress across the Company supporting the advancement of our TSHA-102 development program toward potential registration for females two years and older with Rett syndrome. In May, we announced alignment with the FDA on key design elements of our REVEAL pivotal trial, and subsequently, submitted our IND application and CTA amendments to both the FDA and Health Canada. I’m pleased to share we have commenced site activation in accordance with the key design elements previously aligned on with the FDA, based on feedback recently received from these regulatory agencies. As a result, we anticipate beginning patient enrollment in the fourth quarter of this year,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “Our frequent and constructive dialogue with the FDA through the Regenerative Medicine Advanced Therapy (RMAT) mechanism has been instrumental in enabling us to navigate this novel regulatory pathway. With a strengthened balance sheet, our pivotal trial underway and a clear path to registration, we believe we are well positioned to advance TSHA-102 to benefit patients living with this devastating disease.”
Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor in Neuroscience and Pediatrics at the Université de Montréal, Director of the Rett Multidisciplinary Clinic of the CHU Sainte-Justine and a Principal Investigator of the REVEAL trials, added, “The enthusiasm generated by the REVEAL Phase 1/2 clinical data presented at the IRSF Scientific Meeting was inspiring and highlights the therapeutic potential of TSHA-102. The developmental milestones achieved across all pediatric, adolescent and adult patients treated with TSHA-102, combined with the positive safety profile to date and minimally invasive intrathecal delivery, reinforces my belief in the potential of TSHA-102 to redefine the treatment landscape for Rett syndrome. I’m pleased to participate in the REVEAL pivotal trial and to contribute to the advancement of a potential therapy for individuals living with Rett syndrome, who continue to face profound unmet need.”
Recent Corporate and TSHA-102 Program Highlights
Anticipated Milestones
Second Quarter 2025 Financial Highlights
Research and Development Expenses: Research and development expenses were $20.1 million for the three months ended June 30, 2025, compared to $15.1 million for the three months ending June 30, 2024. The $5.0 million increase was driven by BLA-enabling process performance qualification (PPQ) manufacturing initiatives, REVEAL clinical trial activities and higher compensation expenses as a result of increased headcount during the three months ended June 30, 2025.
General and Administrative Expenses: General and administrative expenses were $8.6 million for the three months ended June 30, 2025, compared to $7.3 million for the three months ended June 30, 2024. The increase of $1.3 million was primarily due to higher legal and professional fees.
Net Loss: Net loss for the three months ended June 30, 2025, was $26.9 million, or $0.09 per share, compared to a net loss of $20.9 million, or $0.09 per share, for the three months ended June 30, 2024.
Cash and Cash Equivalents: As of June 30, 2025, Taysha had $312.8 million in cash and cash equivalents. This reflects the gross proceeds of $230.0 million from the May 2025 follow-on financing, including full exercise of the underwriters’ option to purchase additional shares. The Company expects that its current cash resources will support planned operating expenses and capital requirements into 2028.
Conference Call and Webcast Information
Taysha management will hold a conference call and webcast today at 8:30 a.m. ET to review its financial and operating results and provide a corporate update. The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13754869. The live webcast and replay may be accessed by visiting Taysha’s website.
About TSHA-102
TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency.
About Rett Syndrome
Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K.
About Taysha Gene Therapies
Taysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company’s management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit www.tayshagtx.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” “plans,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include, but are not limited to, statements concerning the potential of TSHA-102, including the reproducibility and durability of any favorable results initially seen in patients dosed to date in clinical trials, including with respect to functional milestones, to positively impact quality of life and alter the course of disease in the patients Taysha seeks to treat, Taysha’s research, development and regulatory plans for TSHA-102, including the timing of initiating additional trials, reporting data from Taysha’s clinical trials and making regulatory submissions, communications with and feedback from the FDA and Health Canada on the regulatory pathway for TSHA-102, the potential for TSHA-102 to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed and the potential market opportunity for Taysha’s product candidates and Taysha’s anticipated cash runway. Forward-looking statements are based on management’s current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding Taysha’s business are described in detail in Taysha’s Securities and Exchange Commission (“SEC”) filings, including in Taysha’s Annual Report on Form 10-K for the full-year ended December 31, 2024 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, which are available on the SEC’s website at www.sec.gov. Additional information will be made available in other filings that Taysha makes from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and Taysha disclaims any obligation to update these statements except as may be required by law.
| Taysha Gene Therapies, Inc. Condensed Consolidated Statements of Operations (in thousands, except share and per share data) | ||||||||||||||||
| For the Three Months Ended June 30, | For the Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Revenue | $ | 1,986 | $ | 1,112 | $ | 4,288 | $ | 4,523 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 20,141 | 15,073 | 35,706 | 35,730 | ||||||||||||
| General and administrative | 8,598 | 7,338 | 16,756 | 14,422 | ||||||||||||
| Total operating expenses | 28,739 | 22,411 | 52,462 | 50,152 | ||||||||||||
| Loss from operations | (26,753 | ) | (21,299 | ) | (48,174 | ) | (45,629 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Change in fair value of warrant liability | (273 | ) | 195 | (171 | ) | (142 | ) | |||||||||
| Change in fair value of term loan | (1,461 | ) | (1,279 | ) | (2,991 | ) | (2,332 | ) | ||||||||
| Interest income | 1,859 | 1,440 | 3,185 | 3,133 | ||||||||||||
| Interest expense | (17 | ) | (27 | ) | (36 | ) | (56 | ) | ||||||||
| Other (expense), income | (237 | ) | 42 | (224 | ) | 37 | ||||||||||
| Total other (expense) income, net | (129 | ) | 371 | (237 | ) | 640 | ||||||||||
| Net loss | $ | (26,882 | ) | $ | (20,928 | ) | $ | (48,411 | ) | $ | (44,989 | ) | ||||
| Net loss per common share, basic and diluted | $ | (0.09 | ) | $ | (0.09 | ) | $ | (0.17 | ) | $ | (0.19 | ) | ||||
| Weighted average common shares outstanding, basic and diluted | 297,988,978 | 232,821,553 | 283,726,888 | 232,035,448 | ||||||||||||
| Taysha Gene Therapies, Inc. Condensed Consolidated Balance Sheet Data (in thousands, except share and per share data) | ||||||||
| June 30, 2025 | December 31, 2024 | |||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 312,761 | $ | 139,036 | ||||
| Restricted cash | 449 | 449 | ||||||
| Prepaid expenses and other current assets | 3,054 | 2,645 | ||||||
| Total current assets | 316,264 | 142,130 | ||||||
| Restricted cash | 2,151 | 2,151 | ||||||
| Property, plant and equipment, net | 6,957 | 7,485 | ||||||
| Operating lease right-of-use assets | 7,773 | 8,381 | ||||||
| Other non-current assets | 186 | 217 | ||||||
| Total assets | $ | 333,331 | $ | 160,364 | ||||
| LIABILITIES AND STOCKHOLDERS' EQUITY | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 7,713 | $ | 3,592 | ||||
| Accrued expenses and other current liabilities | 12,151 | 12,862 | ||||||
| Deferred revenue | 5,485 | 9,773 | ||||||
| Total current liabilities | 25,349 | 26,227 | ||||||
| Term loan, net | 41,051 | 43,942 | ||||||
| Operating lease liability, net of current portion | 16,808 | 17,361 | ||||||
| Other non-current liabilities | 1,396 | 1,309 | ||||||
| Total liabilities | 84,604 | 88,839 | ||||||
| Stockholders' equity | ||||||||
| Preferred stock, $0.00001 par value per share; 10,000,000 shares authorized, and no shares issued and outstanding as of June 30, 2025, and December 31, 2024 | — | — | ||||||
| Common stock, $0.00001 par value per share; 700,000,000 shares authorized and 272,730,060 issued and outstanding as of June 30, 2025 and 400,000,000 shares authorized and 204,943,306 issued and outstanding as of December 31, 2024 | 3 | 2 | ||||||
| Additional paid-in capital | 900,139 | 677,859 | ||||||
| Accumulated other comprehensive loss | (699 | ) | (4,031 | ) | ||||
| Accumulated deficit | (650,716 | ) | (602,305 | ) | ||||
| Total stockholders’ equity | 248,727 | 71,525 | ||||||
| Total liabilities and stockholders' equity | $ | 333,331 | $ | 160,364 | ||||
Company Contact:
Hayleigh Collins
Senior Director, Corporate Communications and Investor Relations
Taysha Gene Therapies, Inc.
hcollins@tayshagtx.com
Media Contact:
Carolyn Hawley
Inizio Evoke
Carolyn.hawley@inizioevoke.com
