Screening completed and dosing scheduled for first potential subject in the Phase 1/2 REVEAL trial in Rett syndrome; dosing of first adult patient with TSHA-102 expected in Q2 2023; initial available Phase 1/2 clinical data, primarily on safety, expected in Q2 2023
Clinical Trial Application (CTA) submission to
New preclinical data for TSHA-102 in Rett syndrome to be presented during a poster presentation at the upcoming
R&D Day in
Formal FDA meeting request submission to discuss regulatory path forward for TSHA-120 in GAN expected in Q2 2023; formal meeting anticipated in Q3 2023
Conference call and live webcast today at 4:30 PM Eastern Time
“We continue to make significant progress with our two lead clinical programs and remain on track to deliver on multiple key milestones, including the generation of first-in-human clinical data for TSHA-102 in adult patients with Rett syndrome, the submission of a CTA to the MHRA to initiate expansion of TSHA-102 in pediatric patients, the submission of an IND application to the FDA for TSHA-102, and obtaining further clarity from the FDA on the regulatory path forward for TSHA-120 in GAN,” said
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 genetic neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene. 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
- Phase 1/2 REVEAL trial in adult patients with Rett syndrome
- Completed screening and scheduled dosing for first potential adult patient with dosing anticipated in Q2 2023
- Initial available Phase 1/2 clinical data, primarily on safety, expected in Q2 2023, with planned quarterly updates on available clinical data thereafter
- Continued dosing of adult patients with Rett syndrome in the REVEAL trial in H2 2023
- 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 - New preclinical data for TSHA-102 in Rett syndrome to be presented as a poster presentation at the upcoming ASGCT 26th Annual Meeting on
Friday, May 19 at12-2 PM PT ; these data and available clinical data from Phase 1/2 REVEAL trial will be presented in upcoming R&D Day in June
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
- Completed CMC module 3 amendment submission detailing commercial process product manufacturing and drug comparability analysis; awaiting FDA feedback
- R&D Day in
June 2023 to overview new findings from totality of data evaluation, including comprehensive analyses of functional, biological, and electrophysiological assessments of TSHA-120 in GAN - Submission of a formal meeting request to the FDA in Q2 2023 to discuss alternative study designs, additional objective measures and regulatory path forward; formal meeting anticipated in Q3 2023
First Quarter 2023 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
Cash and cash equivalents: As of
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 855-327-6837 (
About
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, our corporate growth plans, the forecast of our cash runway and the implementation and potential impacts of our strategic pipeline prioritization initiatives. 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
Condensed Consolidated Statements of Operations
(in thousands, except share and per share data)
(Unaudited)
| For the three months ended |
For the three months ended |
|||||
| Revenue: | ||||||
| Service Revenue | $ | 4,706 | $ | - | ||
| Operating expenses: | ||||||
| Research and development | 12,514 | 38,182 | ||||
| General and administrative | 8,751 | 11,469 | ||||
| Total operating expenses | 21,265 | 49,651 | ||||
| Loss from operations | (16,559 | ) | (49,651 | ) | ||
| Other income (expense): | ||||||
| Interest Income | 319 | 14 | ||||
| Interest expense | (1,374 | ) | (672 | ) | ||
| Other expense | (8 | ) | (8 | ) | ||
| Total other expense | (1,063 | ) | (666 | ) | ||
| Net loss | $ | (17,622 | ) | $ | (50,317 | ) |
| Net loss per common share, basic and diluted | $ | (0.28 | ) | $ | (1.32 | ) |
| Weighted average common shares outstanding, basic and diluted | 63,260,905 | 38,174,717 | ||||
Condensed Consolidated Balance Sheet Data
(in thousands, except share and per share data)
(Unaudited)
| ASSETS | ||||||
| Current assets: | ||||||
| Cash and cash equivalents | $ | 63,425 | $ | 87,880 | ||
| Prepaid expenses and other current liabilities | 8,933 | 8,537 | ||||
| Total current assets | 72,358 | 96,417 | ||||
| Restricted cash | 2,637 | 2,637 | ||||
| Property, plant and equipment, net | 14,642 | 14,963 | ||||
| Operating lease right-of-use assets | 10,647 | 10,943 | ||||
| Other noncurrent assets | 1,316 | 1,316 | ||||
| Total assets | $ | 101,600 | $ | 126,276 | ||
| LIABILITIES, CONVERTIBLE PREFERRED STOCK, AND STOCKHOLDERS' (DEFICIT) EQUITY | ||||||
| Current liabilities: | ||||||
| Accounts payable | $ | 9,002 | $ | 10,946 | ||
| Accrued expenses and other current liabilities | 16,602 | 18,287 | ||||
| Deferred revenue | 28,851 | 33,557 | ||||
| Total current liabilities | 54,455 | 62,790 | ||||
| Term loan, net | 38,161 | 37,967 | ||||
| Operating lease liability, net of current portion | 19,928 | 20,440 | ||||
| Other noncurrent liabilities | 4,004 | 4,130 | ||||
| Total liabilities | 116,548 | 125,327 | ||||
| Preferred stock, and outstanding as of |
- | - | ||||
| Stockholders' (deficit) equity | ||||||
| Common stock, and 63,207,507 issued and outstanding as of |
1 | 1 | ||||
| Additional paid-in capital | 404,114 | 402,389 | ||||
| Accumulated deficit | (419,063 | ) | (401,441 | ) | ||
| Total stockholders’ (deficit) equity | (14,948 | ) | 949 | |||
| Total liabilities, convertible preferred stock, and stockholders' (deficit) equity | $ | 101,600 | $ | 126,276 | ||
Company Contact:
Director, Head of Corporate Communications
hcollins@tayshagtx.com
Media Contact:
carolyn.hawley@canalecomm.com
Source: Taysha Gene Therapies, Inc.