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
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
“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
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
- 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
- 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
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 877-407-0792 (
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 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
Condensed Consolidated Statements of Operations
(in thousands, except share and per share data)
(Unaudited)
| For the three months ended | For the twelve months ended | ||||||||||||
| 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 | |||||||||
Condensed Consolidated Balance Sheet Data
(in thousands, except share and per share data)
(Unaudited)
| 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:
Director, Head of Corporate Communications
hcollins@tayshagtx.com
Media Contact:
Evoke Canale
carolyn.hawley@evokegroup.com
Source: Taysha Gene Therapies, Inc.