- The stock price of Taysha Gene Therapies Inc (NASDAQ: TSHA) increased by over 18% pre-market today. This is why it happened.
The stock price of Taysha Gene Therapies Inc (NASDAQ: TSHA) – a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations – increased by over 18% pre-market today. Investors are responding positively to Taysha Gene Therapies announcing it reported positive clinical efficacy and safety data for the high dose cohort of 3.5×1014 total vg, as well as long-term durability data across all therapeutic doses of TSHA-120 in giant axonal neuropathy (GAN).
Key Clinical Data Supports Clinically Meaningful And Statistically Significant Slowing of Disease Progression Across All Therapeutic Cohorts
(1.2×1014 total vg, 1.8×1014 total vg and 3.5×1014 total vg)
— High Dose Cohort Data (3.5×1014 total vg) by Year 1: 5-point improvement in the change in 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)
— Analysis of All Therapeutic Dose Cohorts by Year 1: 7-point improvement in the change in rate of decline in MFM32 score across all therapeutic dose cohorts compared to natural history decline of 8 points by Year 1 (n=12, p<0.001)
— Analysis of All Therapeutic Dose Cohorts over 3 Years: 10-point improvement in the mean change from baseline in MFM32 score across all therapeutic dose cohorts compared to estimated natural history decline of 24 points by Year 3 (n=5, statistical analysis not performed)
— Disease Stabilization Demonstrated in the Single Patient to Reach Year 5 Visit: 26-point improvement in the change from baseline in MFM32 score for the single patient treated with TSHA-120 compared to estimated natural history by Year 5 (n=1, statistical analysis not performed)
— Bayesian Analysis: Confirmed 97% probability of clinically meaningful slowing of 50% or more in disease progression across all therapeutic dose cohorts
Secondary Endpoints for Pathologic, Physiologic, and Clinical Markers Demonstrated Preservation of Visual Acuity / Stabilization of Retinal Nerve Fiber Layer Thickness / Regeneration of Nerve Fibers
— Biopsies obtained pre- and post-gene transfer in five of six patient samples analyzed confirmed active regeneration of axons with increased number of regenerative nerve clusters (n=6; remaining samples being analyzed)
— Preservation of visual acuity across all therapeutic doses compared to progressive loss of visual acuity observed in natural history (n=12)
— Stabilization of retinal nerve fiber layer (RNFL) thickness and prevention of axonal nerve degeneration compared to diffuse thinning of RNFL observed in natural history (n=12)
Key Long-Term Safety and Tolerability Findings
— 53 patient-years of clinical data support favorable safety and tolerability profile
“The totality of data generated by TSHA-120 to date support our plans to engage with major regulatory agencies in order to discuss pathways for registration and we look forward to providing a regulatory update later this year. In the interim, we are finalizing our commercial strategy with a focus on patient identification, disease awareness and payor engagement for the estimated 5,000 affected patients in addressable markets.”
— RA Session II, President, Founder and CEO of Taysha
“These results are consistent, clinically meaningful and statistically significant. Notably, analysis across all therapeutic dose cohorts confirms the sustained long-term durability of effect for TSHA-120-treated patients. These data also provide new evidence of TSHA-120’s ability to regenerate nerve fibers, demonstrating an improvement in disease pathology, and to preserve visual acuity, which should significantly benefit patients’ and families’ quality-of-life.”
— Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development at Taysha
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