Full Press Release Details
Taysha Gene Therapies Reports Full-Year 2020 Financial Results and Provides a Corporate Update
Expects preliminary Phase 1/2 safety and biomarker and preliminary clinical data for TSHA-101 in GM2
gangliosidosis from the Queen s University study in second half of 2021 and by year-end
Plans to initiate Phase 1/2 clinical trial for TSHA-118 in CLN1 disease in the second half of 2021
Phase 1/2 clinical trials for TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh
syndrome by year-end
Plans to initiate a U.S. Phase 1/2 clinical trial for TSHA-101 in GM2 gangliosidosis in
Expects IND/CTA submission from one of the following programs: SLC13A5 deficiency, APBD, Lafora
disease, GM2 AB variant and SLC6A1 haploinsufficiency
Conference call and webcast today at 8:00 AM Eastern Time
Dallas March 3, 2021 Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, clinical-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 full-year ended December 31, 2020 and provided a corporate update.
In 2020, we successfully completed one of the
fastest seed to IPOs in biotech history and made significant strides in advancing our pipeline initiatives, next-generation technology platforms and manufacturing strategy, said RA Session II, President, Founder and CEO of Taysha. We
expect 2021 to be a transformational year for Taysha, as we anticipate reporting first-in-human clinical data for TSHA-101 in GM2
gangliosidosis and initiating Phase 1/2 trials in CLN1 disease, Rett syndrome and SURF1-associated Leigh syndrome. We will be submitting multiple IND/CTAs across three CNS franchises, and advancing four gene therapies into IND-/CTA-enabling studies and initiating work on additional targets. We continue to enhance our platform of next-generation technologies to optimize key components of our
approach with AAV-based gene therapies. With recent additions to our team and Board of Directors and the formation of our Scientific Advisory Board, we believe we have the resources needed to achieve our
corporate initiatives this year. Lastly, we will continue to evaluate other opportunities to maximize the value of our pipeline and technology platforms. We look forward to providing updates on our progress throughout the year and at an R&D day
2020 Corporate Highlights and Updates
Since inception a year ago, the company has achieved its strategic priorities, as highlighted by:
Full-Year 2020 Financial Highlights
Research and Development (R&D) Expenses: R&D expenses were $31.9 million for the year ended December 31, 2020 compared to
$1.0 million for the period from September 20, 2019 (the date of company inception) to December 31, 2019. The increase was primarily related to the company s development programs, as a result of increased manufacturing-related
spend, clinical and preclinical activities, and headcount.
General and Administrative (G&A) Expenses: G&A expenses were $11.1 million
for the year ended December 31, 2020 compared to $0.1 million for the period from September 20, 2019 (the date of company inception) to December 31, 2019. The increase was primarily due to an increase in personnel costs resulting
from increased headcount, professional services fees, and other corporate-related expenses.
Other Expenses: Other expenses were $17.0 million
for the year ended December 31, 2020 which were non-cash in nature and represented the change in fair value of the preferred stock tranche liability associated with the Series A convertible preferred
Net loss: Net loss for the year ended December 31, 2020 was $60.0 million, or $3.40 per share, as compared to a net loss of
$1.1 million, or $0.12 per share, for the period from September 20, 2019 (the date of company inception) to December 31, 2019.
cash equivalents: As of December 31, 2020, Taysha had $251.3 million in cash and cash equivalents, which included $165.9 million in net proceeds from the company s IPO completed in September 2020.
Anticipated Milestones by Program
TSHA-101 for GM2 gangliosidosis: the first bicistronic gene therapy in clinical development designed to deliver two genes HEXA and HEXB intrathecally for the treatment of GM2
gangliosidosis, also called Tay-Sachs or Sandhoff disease
self-complementary AAV9 viral vector designed to express a human codon-optimized CLN1 transgene to potentially treat CLN1, a rapidly progressing rare lysosomal storage disease with no approved treatments
TSHA-102 in Rett syndrome: a self-complementary AAV9 gene
therapy in development for a severe neurodevelopmental disorder, designed to deliver MECP2 as well as a novel miRARE platform that regulates transgene expression on a
TSHA-104 in SURF1-associated Leigh syndrome: a
self-complementary AAV9 viral vector with a codon optimized transgene encoding the human SURF1 protein to potentially treat SURF1-associated Leigh syndrome, a monogenic mitochondrial disorder with no approved treatments
Pipeline programs advancing into
IND-/CTA-enabling studies
Next-generation technology platform
Anticipated Corporate Milestones in 2021
Conference Call and Webcast Information
Taysha management will hold a conference call and webcast today at 8:00 am ET / 7:00 am CT to review its financial and operating results and to provide a
corporate update. The dial-in number for the conference call is 833-614-1477 (U.S./Canada) or 914-987-7215 (international). The conference ID for all callers is 3183829. The live webcast and replay may be accessed by visiting Taysha s website at
https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 30 days.
Taysha Gene Therapies
Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing
curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team s proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program
to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform an engine for potential new cures with a goal of dramatically improving patients
lives. More information is available at www.tayshagtx.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as
anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include
statements concerning or implying the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product
candidates and early-stage programs, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully
distributed and marketed, our corporate growth plans and our plans to establish a commercial-scale cGMP manufacturing facility to provide preclinical, clinical and commercial supply. Forward-looking statements are based on management s current
expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do
not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange
Commission ( SEC ) filings, including in our Annual Report on Form 10-K for the year ended December 31, 2020, which is available on the SEC s website
at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic.
These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.
Taysha Gene Therapies, Inc.
Consolidated Statement of Operations
(in thousands, except share and per share data)
| For the Year Ended December 31, 2020 | For the Period from September 20, 2019 (date of inception) to December 31, 2019 | |||||||
| Operating expenses: | ||||||||
| Research and development | $ | 31,893 | $ | 987 | ||||
| General and administrative | 11,109 | 128 | ||||||
| Total operating expenses | 43,002 | 1,115 | ||||||
| Loss from operations | (43,002 | ) | (1,115 | ) | ||||
| Other (expense) income: | ||||||||
| Change in fair value of preferred stock tranche liability | (17,030 | ) | ||||||
| Interest income | 49 | |||||||
| Interest expense | (28 | ) | ||||||
| Total other (expense) income | (17,009 | ) | ||||||
| Net loss | $ | (60,011 | ) | $ | (1,115 | ) | ||
| Net loss per common share, basic and diluted | $ | (3.40 | ) | $ | (0.12 | ) | ||
| Weighted average common shares outstanding, basic and diluted | 17,665,683 | 9,625,679 |
Taysha Gene Therapies, Inc.
Consolidated Balance Sheet Data
| December 31, 2020 | December 31, 2019 | |||||||
| Cash and cash equivalents | $ | 251,253 | $ | |||||
| Total assets | $ | 258,881 | $ | 15 | ||||
| Total liabilities | $ | 7,579 | $ | 150 | ||||
| Total stockholders equity (deficit) | $ | 251,302 | $ | (135 | ) |
SVP, Corporate Communications and Investor
Taysha Gene Therapies
Canale Communications