Taysha Reports Efficacy, Safety Data From High Dose Cohort For Neurological Disorder Gene Therapy Trial

Taysha Gene Therapies Inc TSHA has reported clinical efficacy and safety data for the high dose cohort of 3.5x1014 total vg and long-term durability data across all therapeutic doses of TSHA-120 in giant axonal neuropathy (GAN).

• 5-point improvement was observed in the rate of decline in MFM32 score for the high dose cohort compared to natural history decline of 8 points by Year 1 (n=3, p = 0.04).
• 32-item Motor Function Measure (MFM32) is an outcome measure used to assess the functional abilities of individuals with neuromuscular diseases.
• A 7-point improvement across all therapeutic dose cohorts was seen compared to a decline of 8 points by Year 1 (n=12, p<0.001).
• 10-point improvement was observed across all therapeutic dose cohorts, compared to the estimated natural history decline of 24 points by Year 3.
• At year 5, a 26-point improvement was seen.
• Related: Taysha Gene Plans Protocol Amendment After Patient Death In Gene Therapy Trial.
• 53 patient-years of clinical data support favorable safety and tolerability profile.
• TSHA-120 is an AAV9 gene replacement therapy delivering the gene gigaxonin to treat GAN, a progressive neurodegenerative disease that affects both the central and peripheral nervous systems. 
• Price Action: TSHA shares closed at $7.18 on Friday.