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Mar 31, 2021
Taysha Gene Therapies Q1 2021 Earnings Report
Reported financial results for the first quarter ended March 31, 2021, and provided a corporate update.
Key Takeaways
Taysha Gene Therapies reported a net loss of $32.0 million, or $0.87 per share, for the first quarter ended March 31, 2021. As of March 31, 2021, Taysha had $228.7 million in cash and cash equivalents.
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 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.
Treatment with TSHA-102 resulted in a statistically significant survival extension by 56% in 4-5 week-old knockout Rett mice.
Taysha Gene Therapies
Taysha Gene Therapies
Forward Guidance
Taysha Gene Therapies anticipates several milestones across its programs and corporate development in 2021.
Positive Outlook
- Report clinical data for TSHA-120 from the 3.5x1014 total vg dose cohort in the second half of 2021.
- Engage with major regulatory agencies to discuss the approval pathway and provide a regulatory update by year-end 2021 for TSHA-120.
- Report preliminary Phase 1/2 safety and biomarker data (Queen’s University trial) in the second half of 2021 for TSHA-101.
- Submit an Investigational New Drug (IND) application in the U.S. in the second half of 2021 for TSHA-101.
- Initiate a Phase 1/2 clinical trial in the U.S. in the second half of 2021 for TSHA-101.
Challenges Ahead
- No specific negative aspects were explicitly mentioned in the provided text. Therefore, this section is based on potential risks and uncertainties inherent in the biotechnology industry.
- Clinical trial delays or failures could impact the timeline for bringing therapies to market.
- Regulatory hurdles and the potential for non-approval of product candidates.
- Manufacturing challenges in scaling up production of gene therapies.
- Competition from other companies developing treatments for similar diseases.