Taysha Gene Therapies Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Corporate Update
Taysha Gene Therapies (Nasdaq: TSHA) has initiated screening for its Phase 1/2 REVEAL trial targeting Rett syndrome, with the first adult patient anticipated to be dosed in H1 2023. The company plans to submit a Clinical Trial Application to the UK MHRA and an IND to the U.S. FDA for TSHA-102 in pediatric patients in mid-2023 and H2 2023, respectively. Taysha's research and development expenses decreased significantly to $13.9 million in Q4 2022 from $37.9 million a year prior. Despite a net loss of $55.7 million for Q4 2022, the company holds $87.9 million in cash, expected to cover expenses into Q1 2024.
- Decrease in Q4 2022 research and development expenses to $13.9 million from $37.9 million in Q4 2021.
- Cash and cash equivalents of $87.9 million expected to support operations into Q1 2024.
- Initiation of adult patient screening for the REVEAL trial in Rett syndrome.
- Net loss of $55.7 million for Q4 2022, an increase from $50.4 million in Q4 2021.
- Record of a $36.4 million non-cash impairment charge for the North Carolina facility.
Initiated screening of first potential subject in Phase 1/2 REVEAL trial in Rett syndrome; dosing of first adult patient with TSHA-102 expected in H1 2023; submitted protocol amendment to allow for younger patients; initial available Phase 1/2 clinical data, primarily on safety, expected in H1 2023
Clinical Trial Application (CTA) submission to United Kingdom (UK) MHRA for TSHA-102 in pediatric patients with Rett syndrome expected in mid-2023; Investigational New Drug (IND) application to United States (U.S.) Food and Drug Administration (FDA) in Rett syndrome anticipated in H2 2023
FDA feedback for TSHA-120 in giant axonal neuropathy (GAN) suggests consideration of alternative clinical trial designs for clinically meaningful and objectively measured treatment effects; Company plans to request a formal meeting with FDA to discuss final findings from currently ongoing comprehensive data analyses and potential regulatory path forward in Q2 2023
Conference call and live webcast today at 4:30 PM Eastern Time
DALLAS, March 28, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a 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), today reported financial results for the fourth quarter and full-year ended December 31, 2022, and provided a corporate update.
“The actions taken early this year to improve execution and expedite progress on our two lead clinical programs in Rett syndrome and GAN are having a positive effect,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “We recently initiated screening of the first potential adult subject for the REVEAL Rett syndrome trial and remain on track to dose the first patient and deliver initial available first-in-human adult data, primarily on safety, for TSHA-102 in the first half of the year. Additionally, we recently submitted a protocol amendment to allow patients as young as 15 years old to be included in the study, which we believe will further expedite enrollment. We remain on track to submit a CTA to the MHRA in mid-2023 to conduct a pediatric Rett syndrome trial, and plan to submit an IND to the FDA for Rett syndrome in the second half of 2023. For TSHA-120 in GAN, based on the constructive feedback recently received from the FDA in response to our follow up questions to the formal Type B end-of-Phase 2 meeting minutes, coupled with the positive preliminary assessment of the ongoing comprehensive data analyses, we plan to submit a formal meeting request to the Agency in the second quarter of 2023 to discuss the potential regulatory pathway forward for this ultra-rare disease with no approved treatment.”
Recent Corporate Highlights
TSHA-102 in Rett syndrome: a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome, a rare inherited genetic neurodevelopmental disorder. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform designed to regulate cellular MECP2 expression. TSHA-102 has received Orphan Drug and Rare Pediatric Disease designations from the FDA and has been granted Orphan Drug designation from the European Commission.
- Screening initiated for first potential adult patient with Rett syndrome for the Phase 1/2 REVEAL trial
- Submitted protocol amendment expanding enrollment eligibility to include subjects ≥ 15 years
- Dosing of the first adult patient with Rett syndrome anticipated in H1 2023
- Initial available Phase 1/2 clinical data, primarily on safety, for TSHA-102 in adult patients with Rett syndrome expected in H1 2023, with planned quarterly updates on available clinical data thereafter
- CTA submission to UK MHRA for TSHA-102 in pediatric patients with Rett syndrome anticipated in mid-2023
- IND application submission to U.S. FDA for Rett syndrome expected in H2 2023
- Continued dosing of adult patients with Rett syndrome in the REVEAL trial in H2 2023
TSHA-120 for giant axonal neuropathy (GAN): a self-complimentary intrathecally delivered AAV9 gene therapy in clinical evaluation for GAN, an ultra-rare inherited genetic neurodegenerative disorder with no approved treatments. TSHA-120 has received Orphan Drug and Rare Pediatric Disease designations from the FDA and has been granted Orphan Drug designation from the European Commission.
- Completed CMC module 3 amendment submission to FDA detailing commercial process product manufacturing and drug comparability analysis
- Receipt of FDA’s response to Taysha’s follow up questions to the formal Type B end-of-Phase 2 meeting minutes
- FDA clarified MFM32, the primary efficacy scale discussed at the FDA Type B end-of-Phase 2 meeting, as a relevant primary endpoint only in the setting of a randomized double blind placebo controlled trial and acknowledged Taysha's challenge in executing and enrolling such a study design due to the ultra-rare nature of GAN
- FDA is open to regulatory flexibility in a controlled trial setting and willing to consider alternative study designs utilizing objective measurements to demonstrate a relatively large treatment effect that is self-evident and clinically meaningful
- Ongoing comprehensive analyses of functional, biological and electrophysiological assessments as part of totality of data evaluation to inform future interactions with the FDA
- Submission of a formal meeting request to the FDA planned in Q2 2023
Fourth Quarter and Full-Year 2022 Financial Highlights
Research and Development Expenses: Research and development expenses were
General and Administrative Expenses: General and administrative expenses were
Net loss: Net loss for the three months ended December 31, 2022 was
Cash and cash equivalents: As of December 31, 2022, Taysha had
Conference Call and Webcast Information
Taysha management will hold a conference call and webcast today at 4:30 pm ET 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 13736479. 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. 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 TSHA-102 and TSHA-120 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, our corporate growth plans, the forecast of our cash runway. 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, 2022, 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.
Condensed Consolidated Statements of Operations
(in thousands, except share and per share data)
(Unaudited)
| For the three months ended | For the twelve months ended | ||||||||||||
| December 31 2022 | December 31 2021 | December 31 2022 | December 31 2021 | ||||||||||
| Revenue: | |||||||||||||
| Service Revenue | $ | 2,502 | - | $ | 2,502 | $ | - | ||||||
| Operating expenses: | |||||||||||||
| Research and development | 13,861 | 37,918 | 91,169 | 131,943 | |||||||||
| General and administrative | 7,341 | 11,806 | 37,360 | 41,324 | |||||||||
| Impairment of long-lived assets | 36,420 | - | 36,420 | - | |||||||||
| Total operating expenses | 57,622 | 49,724 | 164,949 | 173,267 | |||||||||
| Loss from operations | (55,120 | ) | (49,724 | ) | (162,447 | ) | (173,267 | ) | |||||
| Other income (expense): | |||||||||||||
| Interest Income | 199 | 29 | 249 | 172 | |||||||||
| Interest expense | (796 | ) | (691 | ) | (3,798 | ) | (1,428 | ) | |||||
| Other | (6 | ) | - | (18 | ) | - | |||||||
| Total other income (expense) | (603 | ) | (662 | ) | (3,567 | ) | (1,256 | ) | |||||
| Net loss | $ | (55,723 | ) | $ | (50,386 | ) | $ | (166,014 | ) | $ | (174,523 | ) | |
| Net loss per common share, basic and diluted | $ | (0.99 | ) | $ | (1.32 | ) | $ | (3.78 | ) | $ | (4.64 | ) | |
| Weighted average common shares outstanding, basic and diluted | 56,386,130 | 38,110,597 | 43,952,015 | 37,650,566 | |||||||||
Taysha Gene Therapies, Inc.
Condensed Consolidated Balance Sheet Data
(in thousands, except share and per share data)
(Unaudited)
| December 31, 2022 | December 31, 2021 | |||||
| ASSETS | ||||||
| Current assets: | ||||||
| Cash and cash equivalents | $ | 87,880 | $ | 149,103 | ||
| Prepaid expenses and other current assets | 8,537 | 10,499 | ||||
| Total current assets | 96,417 | 159,602 | ||||
| Restricted cash | 2,637 | 2,637 | ||||
| Property, plant and equipment, net | 14,963 | 50,610 | ||||
| Operating lease right-of-use assets | 10,943 | - | ||||
| Other noncurrent assets | 1,316 | 1,107 | ||||
| Total assets | $ | 126,276 | $ | 213,956 | ||
| LIABILITIES AND STOCKHOLDERS' EQUITY | ||||||
| Current liabilities: | ||||||
| Accounts payable | $ | 10,946 | $ | 21,763 | ||
| Accrued expenses and other current liabilities | 18,287 | 29,983 | ||||
| Deferred revenue | 33,557 | - | ||||
| Total current liabilities | 62,790 | 51,746 | ||||
| Build-to-suit lease liability | - | 25,900 | ||||
| Term loan, net | 37,967 | 37,192 | ||||
| Operating lease liability, net of current portion | 20,440 | - | ||||
| Other noncurrent liabilities | 4,130 | 3,735 | ||||
| Total liabilities | 125,327 | 118,573 | ||||
| Stockholders’ equity | ||||||
| Common stock, | 1 | - | ||||
| Additional paid-in capital | 402,389 | 331,032 | ||||
| Accumulated deficit | (401,441 | ) | (235,649 | ) | ||
| Total stockholders’ equity | 949 | 95,383 | ||||
| Total liabilities and stockholders' equity | $ | 126,276 | $ | 213,956 | ||
Company Contact:
Hayleigh Collins
Director, Head of Corporate Communications
Taysha Gene Therapies
hcollins@tayshagtx.com
Media Contact:
Carolyn Hawley
Evoke Canale
carolyn.hawley@evokegroup.com
FAQ
What is the status of Taysha Gene Therapies' TSHA-102 for Rett syndrome as of March 2023?
When does Taysha plan to submit the Clinical Trial Application for TSHA-102?
What were Taysha Gene Therapies' financial results for the fourth quarter of 2022?
What are Taysha's cash reserves as of the end of 2022?
What feedback did Taysha receive from the FDA regarding TSHA-120 for GAN?
