Taysha Gene Therapies (NASDAQ: TSHA) reported Q1 EPS of ($0.87), $0.31 worse than the analyst estimate of ($0.56).

“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.”

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