Taysha Gene Therapies Q2 2024 Financial Results and Corporate Update

Taysha Gene Therapies, Inc., a clinical-stage biotechnology firm specializing in adeno-associated virus (AAV) gene therapies for severe monogenic diseases of the central nervous system (CNS), has announced its financial results for the second quarter ending June 30, 2024, along with a corporate update.

During the second quarter, Taysha made significant strides in its TSHA-102 clinical program. Preliminary data from the pediatric trial and long-term data from the adolescent and adult trial indicated encouraging results following the administration of the low dose of TSHA-102. The company initiated the first patient in the high dose cohort of the adolescent and adult trial, with the treatment generally well-tolerated after an initial six-week assessment. Consequently, the Independent Data Monitoring Committee (IDMC) approved the dosing of the second adolescent/adult and first pediatric patient in the high dose cohort. This approval supports the continuation of promising low dose data showing a favorable safety profile and improvements across various clinical domains for patients with Rett syndrome.

CEO Sean P. Nolan emphasized the company's commitment to clinical trial execution and data collection, which will guide discussions with regulatory authorities for the next phase of their studies. Nolan also mentioned that the dosing of additional patients in both the adolescent/adult and pediatric high dose cohorts is scheduled for the third quarter of 2024. Taysha aims to provide comprehensive updates on their clinical data in the first half of 2025.

In financial terms, Taysha completed a follow-on public offering in June 2024, raising net proceeds of $76.8 million and extending its cash runway into the fourth quarter of 2026. Research and development expenses for the quarter amounted to $15.1 million, a decrease from $19.8 million in the same period last year, primarily due to milestone payments made in the previous year. General and administrative expenses increased to $7.3 million from $6.0 million, driven by higher stock-based compensation and consulting fees. The net loss for the quarter was $20.9 million, reduced from $24.6 million in the prior year, with cash and cash equivalents totaling $172.7 million as of June 30, 2024.

Recent corporate highlights include presenting longer-term data from the first cohort of TSHA-102 at the 2024 IRSF Rett Syndrome Scientific Meeting. The data indicated that TSHA-102 was generally well-tolerated with no serious adverse events (SAEs) or dose-limiting toxicities (DLTs). The company observed sustained and new improvements across multiple efficacy measures and clinical domains. The adolescent and adult trial data showed a consistent safety profile, with improvements in motor skills, communication, autonomic function, and seizure events.

In the pediatric trial, Taysha received approval to expand into Canada, complementing ongoing trials in the U.S. and U.K. Preliminary data from the first cohort in the pediatric trial indicated that TSHA-102 was well-tolerated, with initial improvements noted in various clinical domains.

Anticipated milestones include dosing additional patients in the high dose cohorts of both trials and providing updates on safety and efficacy data in the first half of 2025. The company aims to continue its focused efforts on developing transformative gene therapies to address severe unmet medical needs and improve the lives of patients and their caregivers.

Taysha’s lead program, TSHA-102, is an investigational gene transfer therapy designed to treat Rett syndrome by delivering a functional form of the MECP2 gene to cells in the CNS. The therapy has received multiple regulatory designations, including Regenerative Medicine Advanced Therapy, Fast Track, and Orphan Drug designations from the FDA, as well as similar recognitions from the European Commission and the Medicines and Healthcare products Regulatory Agency.

Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the MECP2 gene, characterized by severe intellectual disability, loss of communication and hand function, motor and respiratory impairment, seizures, and shortened life expectancy. Currently, no approved therapies address the genetic root cause of the disease, which affects an estimated 15,000 to 20,000 patients in the U.S., EU, and U.K.

Taysha Gene Therapies is dedicated to leveraging its expertise in gene therapy development and commercialization to rapidly bring treatments from bench to bedside, aiming to significantly improve patient outcomes.