Taysha Gene Therapies Reports Third Quarter 2020 Financial Results and Provides Business Update
Taysha Gene Therapies, a gene therapy company focused on central nervous system diseases, reported financial results for Q3 2020. The company raised over $275 million through its Series B financing and IPO, enhancing its capabilities across 18 gene therapy programs. Significant advancements include securing FDA designations for TSHA-101 (for GM2 gangliosidosis) and TSHA-102 (for Rett syndrome), along with plans for clinical trials. Taysha also strengthened manufacturing partnerships and anticipates tripling its workforce by year-end 2020.
- Raised over $275 million from Series B and IPO for pipeline support.
- Secured rare pediatric disease and orphan drug designations for TSHA-101 and TSHA-102.
- Plans to initiate Phase 1/2 trials for TSHA-101 and TSHA-118 in 2021.
- Strengthened manufacturing partnerships with Invitae and Catalent.
- Anticipates tripling headcount from IPO by the end of 2020.
- None.
DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today reported its financial results for the third quarter of 2020 and provided a business update.
“Just over a year ago, we founded Taysha in partnership with UT Southwestern with a mission to develop gene therapies to address monogenic diseases of the central nervous system, and to that end we have made significant progress in building a talented team that can advance our broad portfolio,” said RA Session, II, President, Founder and CEO of Taysha. “During the third quarter, we raised over
Program Highlights
TSHA-101 for GM2 gangliosidosis – Taysha secured rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-101, its bicistronic vector designed to treat GM2 gangliosidosis, also known as Tay-Sachs and Sandhoff disease. Taysha anticipates initiating a Phase 1/2 clinical trial of TSHA-101 in Canada by end of 2020.
TSHA-102 for Rett syndrome –Taysha announced that FDA has granted rare pediatric disease and orphan drug designation for TSHA-102, which is under development for the treatment of Rett syndrome, one of the most common genetic causes of severe intellectual disability. TSHA-102 utilizes the miRARE platform, which is designed to regulate the expression of therapeutic transgenes on a cellular basis. Taysha remains on track to file an Investigational New Drug (IND) application for the program in 2021.
TSHA-118 for CLN1 – Taysha announced the in-licensing of an AAV9-based gene therapy program for the treatment of CLN1. TSHA-118 was originally developed in the academic lab of Steven Gray, Ph.D., Taysha’s Chief Scientific Advisor. CLN1 Batten disease is a rapidly progressive rare lysosomal storage disease with no approved treatments. The company intends to initiate a Phase 1/2 clinical trial of TSHA-118 in 2021 under a currently open IND.
Recent Business Highlights
In October, Taysha announced a partnership with Invitae to support two different screening programs to enable rapid identification of patients with genetic disorders. DETECT Lysosomal Storage Diseases provides genetic testing for lysosomal storage disorders, including GM2 gangliosidosis and CLN1. The Behind the Seizures® program supports the diagnosis of patients with genetic epilepsies.
In November, Taysha announced a development and manufacturing partnership with Catalent to support future preclinical and clinical supply for several of Taysha’s gene therapy programs, including CLN1 and Rett syndrome.
Taysha has also invested in building its internal management team to advance the development of its broad gene therapy pipeline. The company anticipates more than tripling its headcount from its IPO by the end of 2020 across all areas of the organization.
Financial Results
As of September 30, 2020, cash and cash equivalents totaled
Taysha reported R&D expenses for the nine months ended September 30, 2020 of
Taysha reported G&A expenses of
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,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the conduct or timing of our clinical trials and our research, development and regulatory plans for our product candidates, 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, 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 and the success of our partnerships with Invitae and Catalent. 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, which we intend to file shortly hereafter and will be 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 |
|||||||||||
Selected Condensed Consolidated Financial Information |
|||||||||||
(In thousands, except share and per share data) |
|||||||||||
(Unaudited) |
|||||||||||
|
|||||||||||
Statements of Operations |
|||||||||||
| For the Three Months Ended September 30, 2020 |
For the Nine Months Ended September 30, 2020 |
For the Period from September 20, 2019 (date of inception) to September 30, 2019 |
|||||||||
| Operating expenses: | |||||||||||
| Research and development | $ |
11,057 |
|
$ |
19,633 |
|
$ |
- |
|
||
| General and administrative |
|
3,984 |
|
|
5,002 |
|
|
31 |
|
||
| Total operating expenses |
|
15,041 |
|
|
24,635 |
|
|
31 |
|
||
| Loss from operations |
|
(15,041 |
) |
|
(24,635 |
) |
|
(31 |
) |
||
| Other expense: | |||||||||||
| Change in fair value of preferred stock tranche liability |
|
- |
|
|
(17,030 |
) |
|
- |
|
||
| Interest expense |
|
(1 |
) |
|
(28 |
) |
|
- |
|
||
| Total other expense |
|
(1 |
) |
|
(17,058 |
) |
|
- |
|
||
| Net loss | $ |
(15,042 |
) |
$ |
(41,693 |
) |
$ |
(31 |
) |
||
| Net loss per common share, basic and diluted | $ |
(1.28 |
) |
$ |
(3.73 |
) |
$ |
(0.00 |
) |
||
| Weighted average common shares outstanding, basic and diluted |
|
11,733,170 |
|
|
11,176,429 |
|
|
8,715,999 |
|
||
Balance Sheet |
|||
| September 30, 2020 |
December 31, 2019 |
||
| ASSETS | |||
| Current assets: | |||
| Cash and cash equivalents |
|
$ - |
|
| Prepaid expenses | 604 |
- |
|
| Deferred offering costs | - |
15 |
|
| Total current assets | 279,238 |
15 |
|
| Property and equipment, net | 28 |
- |
|
| Total assets |
|
|
|
| LIABILITIES AND STOCKHOLDERS' EQUITY (DEFICIT) | |||
| Current liabilities | |||
| Accounts payable |
|
$ - |
|
| Accrued expenses | 2,727 |
150 |
|
| Due to related party | 60 |
- |
|
| Total current liabilities | 11,624 |
150 |
|
| Total liabilities | 11,624 |
150 |
|
| Stockholders' equity (deficit) | |||
| Preferred stock, |
- |
- |
|
| Common stock, |
- |
- |
|
| Additional paid-in capital | 310,450 |
980 |
|
| Accumulated deficit | (42,808) |
(1,115) |
|
| Total stockholders’equity (deficit) | 267,642 |
(135) |
|
| Total liabilities and stockholders' equity (deficit) |
|
|
|
