TSHA-120 program demonstrated clear arrest of disease progression and long-term durability at therapeutic dose levels in patients with giant axonal neuropathy (GAN); Expects clinical data from highest dose cohort in second half of 2021 and regulatory feedback from agencies by year-end 2021
Expects to have five programs in Phase 1/2 trials in the second half of 2021, including GAN, GM2 gangliosidosis, CLN1 disease, Rett syndrome and SURF1-associated Leigh syndrome
Positive preclinical data for TSHA-102 in Rett syndrome provided quantitative evidence of miRARE’s ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions in both wild type and knockout mouse models of Rett syndrome; Results published in Brain
Treatment with TSHA-102 resulted in a statistically significant survival extension by 56% in 4-5 week-old knockout Rett mice with meaningful accumulated disease, a more translatable model of the disorder in humans
New data for multiple preclinical programs, including tauopathies, SLC13A5 deficiency, SLC6A1 haploinsufficiency, Angelman disease, Adult Polyglucosan Body Disease (APBD), Lafora disease, and GM2 AB variant, highlighted Taysha’s next wave of novel gene therapies that have the potential to impact meaningful patient populations
Plans for IND/CTA submission from one of the following programs by year-end 2021: SLC13A5 deficiency, APBD, Lafora disease, GM2 AB variant and SLC6A1 haploinsufficiency
Advancing development of multiple preclinical programs, including tauopathies and Angelman syndrome
Virtual Research and Development Day on June 28-29, 2021 to feature Key Opinion Leaders and highlight progress across pipeline
Conference call and webcast today at 8:00 AM Eastern Time
DALLAS--(BUSINESS WIRE)-- 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, today reported financial results for the first quarter ended March 31, 2021 and provided a corporate update.
“Our team has ushered in the new year with a continued focus on achieving our corporate objectives and creating value for patients and shareholders,” said RA Session II, President, Founder and CEO of Taysha. “Our recent acquisition of TSHA-120 for GAN immediately transformed Taysha into a pivotal-stage gene therapy company. Based on the compelling clinical and preclinical data package generated to date for this promising product candidate, we intend to engage with regulatory agencies to discuss a pathway to approval and look forward to providing clinical and regulatory updates in the second half of 2021 and by year-end 2021, respectively. We are also extremely pleased to share newly published preclinical data for TSHA-102 in Rett syndrome that provided, for the first time, quantitative evidence of miRARE’s ability to show genotype-dependent regulation of MECP2 gene expression across different regions of the brain in wild type and knockout mouse models. Rett syndrome is an incredibly difficult disease to treat with gene replacement therapy given the challenges of safely regulating the degree of MECP2 expression from the MECP2 gene and we are encouraged that miRARE has achieved this regulation on a cell-by-cell basis without associated toxicities. The built-in self-regulatory feedback loop mechanism is a culmination of approximately 14 years of research and holds great potential for the treatment of what we now consider a reversible disease. Importantly, TSHA-102-treated knockout Rett mice with meaningful disease accumulation experienced a statistically significant survival extension by 56%, which we believe is a more translatable model of the disorder in humans. We believe these data validate our novel approach to treating Rett syndrome, help de-risk the clinical program and support advancement of TSHA-102 into a Phase 1/2 trial by year-end. With these and other recent value-creating achievements, we are even more confident in our outlook for the full year.”
Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha, said, “We expect a steady flow of near-term clinical, regulatory and preclinical catalysts for the remainder of 2021. We anticipate having five programs in Phase 1/2 trials and an additional six programs in IND/CTA-enabling studies by year-end 2021. For the remainder of 2021, we expect data from the highest dose cohort from the ongoing TSHA-120 trial for GAN, a regulatory update on the GAN program, first-in-human Phase 1/2 clinical data from the Queen’s University trial of TSHA-101 in GM2 gangliosidosis and the initiation of Phase 1/2 trials in GM2 gangliosidosis in the U.S., CLN1 disease, Rett syndrome and SURF1-associated Leigh syndrome. For our preclinical programs, we expect to submit an IND/CTA for one of six programs in IND/CTA-enabling studies by year-end 2021. Moreover, we will continue to make advancements on payload design and on the miRARE, mini-gene and vagus nerve redosing platforms, which are expected to drive further innovation. We look forward to providing additional updates at our two-day R&D Day event in June and throughout the year.”
Recent Corporate Highlights
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Published new preclinical data for TSHA-102 in Rett syndrome in Brain journal
- Preclinical data provided quantitative evidence of miRARE’s ability to exhibit genotype-dependent regulation of MECP2 gene expression across different brain regions in both wild type and knockout mouse models of Rett syndrome
- TSHA-102 resulted in a statistically significant survival extension by 56%, whereas unregulated constructs did not extend survival significantly in the validated MECP2 knockout Rett mouse model
- Benefit in 4-5 week-old TSHA-102-treated knockout Rett mice with meaningful accumulated disease should be a more translatable model to humans
- In the pons and midbrain, miRARE inhibited mean expression in a genotype-dependent manner, as indicated by significantly fewer myc(+) cells observed in wild type mice than knockout mice (p