Full Press Release Details
Taysha Gene Therapies Reports Third Quarter 2021 Financial Results and Provides Corporate Update
Anticipate clinical safety and MFM32 functional data for TSHA-120 from the highest dose cohort of
3.5x1014 total vg in GAN in December 2021
Expect preliminary clinical safety
data and Hex A enzyme activity in the plasma and cerebral spinal fluid for TSHA-101 in GM2 gangliosidosis in December 2021
Anticipate preliminary clinical data for the first-generation construct in CLN7 disease including safety data from first patient ever dosed
intrathecally at 1.0x1015 total vg in December 2021
On track to initiate Phase
1/2 trials for TSHA-118 in CLN1 disease and TSHA-102 in Rett syndrome by year-end 2021
Completed five successful concurrent GMP campaigns for multiple programs to date, sufficient to support clinical-stage programs this year
Received Orphan Drug Designation from the European Commission for TSHA-101 for GM2
gangliosidosis, TSHA-102 for Rett syndrome and TSHA-105 for SLC13A5-related epilepsy
Successfully completed nine regulatory meetings with multiple agencies to support clinical trial initiations; submitted initial request for end-of-Phase meeting to align regulatory pathway for TSHA-120 in giant axonal neuropathy (GAN)
Recent publications of positive preclinical data from an AAV-mediated UBE3A gene replacement
approach for Angelman syndrome and TSHA-104 for the treatment of SURF1-associated Leigh syndrome further support clinical development strategies
Conference call and live webcast today at 8:00 AM Eastern Time
Dallas November 10, 2021 - 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 third quarter ended September 30, 2021 and provided a corporate update.
We made significant advances in multiple
aspects of our business, including nine productive regulatory meetings across several programs, a strategic addition of the clinical-stage CLN7 program and publication of positive preclinical data related to the Angelman syndrome and
SURF1-associated Leigh syndrome programs, noted RA Session II, President, Founder and CEO of Taysha. In September, we submitted an end-of-Phase meeting
request for TSHA-120 with a major ex-US regulatory agency and look forward to submitting additional requests to multiple regulatory agencies by the end of this year. In
GAN, we have up to 6 years of longitudinal data in individual patients and collectively 55-patient years of clinical safety and efficacy data from our ongoing clinical study. We also have eight years of robust longitudinal data from a natural history study being conducted at the NIH by Dr. Carsten
B nnemann. There has been consistency in the strength of data across multiple functional and biomarker endpoints, including the MFM32 scale, ophthalmological assessments and nerve biopsies. We look forward to reporting clinical safety and
functional MFM32 data for TSHA-120 from the high dose cohort of 3.5x1014 total vg in December, where we believe continued clinically meaningful slowing of
disease progression similar to that achieved with the lower dose cohorts would be considered confirmation of disease modification. For TSHA-101 in GM2 gangliosidosis, we remain on track to report preliminary
clinical safety data and Hex A enzyme activity in plasma and CSF in December. Based on natural history, 2% to 4% Hex A enzyme activity in plasma normalizes survival and significantly improves clinical phenotype of GM2 gangliosidosis. For the CLN7
program, we anticipate preliminary clinical data including safety data for the first patient in history to be dosed at 1.0x1015 total vg with the first-generation construct in December. In
parallel, we expect to finalize the second-generation CLN7 construct by year-end and initiate a planned pivotal clinical trial in 2022 with reference to clinical data from the first-generation construct. For TSHA-102 in Rett syndrome, we intend to submit an IND/CTA filing in November followed by initiation of a clinical trial by the end of this year. We have recently obtained preclinical data showing improvement in
survival, and respiratory and motor functions in relevant animal models. Notably, preliminary data from a GLP toxicology study in non-human primates demonstrated no adverse findings at the highest dose tested
suggesting that the miRARE platform is successfully downregulating MECP2 expression to within normal physiological levels. On the manufacturing front, we have completed five concurrent GMP campaigns to support multiple clinical programs this
year, continued Mr. Session.
We believe the combination of our experience in gene therapy drug development, clinical pipeline and a
strong balance sheet provides us with the financial and operational flexibility to achieve numerous value-generating milestones across our programs, and importantly, a potential regulatory approval for
TSHA-120 in GAN in the near term. We look forward to continued execution on our development and regulatory strategies, and will provide updates throughout the remainder of this year, concluded
Recent Corporate Highlights
Anticipated Milestones by Program
TSHA-120 for GAN: an intrathecally dosed AAV9 gene therapy currently being evaluated in a clinical trial for the
treatment of GAN, a rare inherited genetic disorder that affects both the central and peripheral nervous systems and is caused by loss-of-function mutations in the gene
coding for gigaxonin
TSHA-101 for GM2 gangliosidosis: the first bicistronic gene therapy
in clinical development designed to deliver two genes HEXA and HEXB, comprising the alpha and beta sub-units of -Hexosaminidase
A, intrathecally for the treatment of GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease
AAV9 Gene Replacement for CLN7 disease: an investigational AAV9 intrathecally dosed gene replacement
therapy designed to deliver a full-length copy of the CLN7 gene to potentially treat CLN7 disease, a rapidly progressing rare lysosomal storage disease with no approved treatments. The first-generation construct is currently in clinical development
with the next-generation construct anticipated to have improved potency, safety, packaging efficiency and manufacturability.
TSHA-118 in CLN1 disease: a self-complementary AAV9 viral vector designed to express a human
codon-optimized CLN1 transgene to potentially treat CLN1 disease, 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 miniMECP2, as well as a novel miRARE platform that regulates transgene expression genotypically on a cell-by-cell basis
Third Quarter 2021 Financial Highlights
Research and Development (R&D) Expenses: Research and development expenses were $39.5 million for the three months ended September 30,
2021, compared to $11.1 million for the three months ended September 30, 2020. The $28.4 million increase was primarily attributable to an increase of $14.5 million of expenses incurred in research and development manufacturing
and other raw material purchases, which included cGMP batches produced by Catalent and UT Southwestern. There was an increase in employee compensation expenses of $10.7 million, which included $1.9 million of
non-cash stock-based compensation, and $4.9 million in third-party research and development expenses, which includes clinical trial CRO activities, GLP toxicology studies, and consulting for regulatory
and clinical studies. This was partially offset by a decrease in licensing fees of $1.7 million.
General and Administrative (G&A)
Expenses: General and administrative expenses were $11.2 million for the three months ended September 30, 2021, compared to $4.0 million for the three months ended September 30, 2020. The increase of approximately
$7.2 million was primarily attributable to $4.3 million of incremental compensation expense, which included $1.8 million of non-cash stock-based compensation. There was an increase of
$2.9 million mainly in professional fees related to legal, insurance, investor relations/communications, accounting, personnel recruiting, market research, and patient advocacy activities.
Net loss: Net loss for the three months ended September 30, 2021 was $51.2 million or $1.35 per share, as compared to a
net loss of $15.0 million, or $1.28 per share, for the three months ended September 30, 2020.
equivalents: As of September 30, 2021, Taysha had $188.8 million in cash and cash equivalents.
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 877-407-0792 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13723855. 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.
About 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, plans,
and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical 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, 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, the potential market opportunity for these product candidates, and our corporate growth plans.
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 full-year ended December 31, 2020 and our Quarterly Report on
Form 10-Q for the quarter ended September 30, 2021, both of which are 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.
Condensed Consolidated Statements of Operations
(in thousands, except share and per share data)
| For the Three Months | For the Nine Months | |||||||||||||||
| Ended September 30, | Ended September 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 39,528 | $ | 11,057 | $ | 94,025 | $ | 19,633 | ||||||||
| General and administrative | 11,153 | 3,984 | 29,518 | 5,002 | ||||||||||||
| Total operating expenses | (50,681 | ) | 15,041 | 123,543 | 24,635 | |||||||||||
| Loss from operations | (50,681 | ) | (15,041 | ) | (123,543 | ) | (24,635 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Change in fair value of preferred stock tranche liability | (17,030 | ) | ||||||||||||||
| Interest income | 37 | 143 | ||||||||||||||
| Interest expense | (543 | ) | (1 | ) | (737 | ) | (28 | ) | ||||||||
| Total other expense, net | (506 | ) | (1 | ) | (594 | ) | (17,058 | ) | ||||||||
| Net loss | $ | (51,187 | ) | $ | (15,042 | ) | $ | (124,137 | ) | $ | (41,693 | ) | ||||
| Net loss per common share, basic and diluted | $ | (1.35 | ) | $ | (1.28 | ) | $ | (3.31 | ) | $ | (3.73 | ) | ||||
| Weighted average common shares outstanding, basic and diluted | 38,003,954 | 11,733,170 | 37,495,537 | 11,176,429 |
Taysha Gene Therapies, Inc.
Condensed Consolidated Balance Sheet Data
(in thousands, except share and per share data)
| September 30, | December 31, | |||||||
| 2021 | 2020 | |||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 188,785 | $ | 251,253 | ||||
| Prepaid expenses and other current assets | 8,385 | 6,626 | ||||||
| Total current assets | 197,170 | 257,879 | ||||||
| Restricted cash | 2,628 | |||||||
| Deferred lease asset | 691 | 715 | ||||||
| Property, plant and equipment, net | 40,553 | 287 | ||||||
| Total assets | $ | 241,042 | $ | 258,881 | ||||
| LIABILITIES AND STOCKHOLDERS EQUITY | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 22,051 | $ | 1,994 | ||||
| Accrued expenses and other current liabilities | 21,163 | 5,135 | ||||||
| Total current liabilities | 43,214 | 7,129 | ||||||
| Build-to-suit lease liability | 26,607 | |||||||
| Term Loan, net | 27,812 | |||||||
| Other non-current liabilities | 3,015 | 450 | ||||||
| Total liabilities | 100,648 | 7,579 | ||||||
| Stockholders equity | ||||||||
| Preferred stock, $0.00001 par value per share; 10,000,000 shares authorized and no shares issued and outstanding as of September 30, 2021 and December 31, 2020 | ||||||||
| Common stock, $0.00001 par value per share; 200,000,000 shares authorized and 38,473,945 and 37,761,435 issued and outstanding as of | ||||||||
| September 30, 2021 and December 31, 2020 | ||||||||
| Additional paid-in capital | 325,657 | 312,428 | ||||||
| Accumulated deficit | (185,263 | ) | (61,126 | ) | ||||
| Total stockholders equity | 140,394 | 251,302 | ||||||
| Total liabilities and stockholders equity | $ | 241,042 | $ | 258,881 |
SVP, Corporate Communications and Investor
Taysha Gene Therapies
Canale Communications