Taysha Gene Therapies, Inc. (TSHA), a leader in developing AAV-based gene therapies for severe central nervous system diseases, announced encouraging financial results for the second quarter of 2024, alongside a significant corporate update.
The company showcased promising advancements in its TSHA-102 clinical program, particularly in its ongoing trials for Rett syndrome.
Positive Clinical Data from TSHA-102 Trials
During the 2024 International Rett Syndrome Foundation (IRSF) Scientific Meeting, Taysha presented cohort one (low dose) data from both the adolescent/adult and pediatric trials of TSHA-102.
The results highlighted durable improvements across various clinical domains, including motor skills and communication. The therapy demonstrated a favorable safety profile, with no serious adverse events (SAEs) reported. Following the initial six-week assessment of the first patient in cohort two (high dose) of the adolescent/adult trial, TSHA-102 was generally well tolerated, further supporting its potential in treating Rett syndrome.
Sean P. Nolan, Chairman and CEO of Taysha, expressed optimism regarding the progress made:
“We remain focused on clinical trial execution and data collection that will further inform discussions with regulatory authorities on the development plan for the next phase of our studies.”
The company received approval from the Independent Data Monitoring Committee (IDMC) to proceed with dosing additional patients in both trials, signaling continued momentum in its clinical efforts.
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Strong Financial Position Enhances Growth Potential
Taysha Gene Therapies reported a net loss of $20.9 million for the quarter ending June 30, 2024, an improvement from a net loss of $24.6 million during the same period in 2023. The company’s research and development expenses decreased to $15.1 million, down from $19.8 million year-over-year, primarily due to reduced milestone fees.
General and administrative expenses rose slightly, reflecting increased stock-based compensation and consulting costs. Importantly, Taysha completed a public follow-on offering in June 2024, raising net proceeds of $76.8 million, which extends its cash runway into the fourth quarter of 2026.
As of June 30, 2024, the company held $172.7 million in cash and cash equivalents, positioning it well for future growth and clinical milestones.
Upcoming Milestones and Market Outlook
Looking ahead, Taysha is on track to dose the second patient in the high-dose cohort of the adolescent/adult trial and the first patient in the pediatric trial in the third quarter of 2024.
The company anticipates reporting safety and efficacy data from both cohort one and cohort two in the first half of 2025. With a strengthened balance sheet and ongoing advancements in its clinical programs, Taysha Gene Therapies is poised to make significant strides in the biotech sector, particularly in the treatment of rare neurological disorders.
As Taysha continues to innovate in gene therapy, investors and stakeholders are keenly watching its progress. The company’s commitment to addressing severe monogenic diseases underscores its potential for long-term growth and value creation in the biotech landscape.
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