TSHA-120 treated patients in GAN demonstrated durable improvement and recoverability of sensory nerve amplitude potential (SNAP), a definitive clinical endpoint, compared to natural history
TSHA-120 commercial grade and clinical trial material considered comparable across all key quality attributes as assessed by an extensive panel of release assays and next-generation sequencing
Positive feedback from MHRA supports regulatory strategy and manufacturing approach including potency assay matrix; additional regulatory feedback, including from FDA, expected by year-end 2022
Preclinical data for TSHA-102 in Rett syndrome demonstrated near normalization of survival as well as normalization of behavior in neonatal knockout Rett mice; clinical data expected by year-end 2022
Conference call and live webcast today at 8:00 AM Eastern Time
“We are excited to announce important progress in giant axonal neuropathy (GAN) including stabilization and improvement of sensory nerve function, a definitive clinical endpoint, in patients with GAN following treatment with TSHA-120,” said RA Session II, President, Founder and CEO of Taysha. “In addition, we now have positive comparability data demonstrating that our commercial grade and clinical trial material are comparable across all key quality attributes. Importantly, we believe positive feedback received from the MHRA, in conjunction with robust comparability data for TSHA-120 and comprehensive clinical data generated to date, further support our ongoing regulatory engagement. We expect additional regulatory feedback, including from the FDA, by year-end. In Rett syndrome, we are highly encouraged by late-breaking neonatal data in preclinical mouse models demonstrating near normalization of survival and normalization of behavior. We look forward to reporting preliminary Phase 1/2 clinical data in adult females with Rett syndrome by year-end 2022.”
Recent Corporate Highlights
TSHA-120 for giant axonal neuropathy (GAN): an intrathecally dosed AAV9 gene therapy in clinical development for the rare inherited genetic disorder GAN. TSHA-120 has received orphan drug and rare pediatric disease designations from the FDA and orphan drug designation from the
- TSHA-120-treated patients with GAN demonstrated durable neurophysiological improvements in the sensory nerve action potential (SNAP), a definitive clinical endpoint, compared to rapid and irreversible decline in sensory function early in life in untreated patients based on natural history
- Natural history data suggest that sensory function is unrecoverable once SNAP reaches zero
- All patients who were 9 years and older in the natural history study demonstrated zero SNAP response
- Treatment with TSHA-120 resulted in stabilization and improvement of sensory nerve function with durability of improvement in SNAP
- Pathology from nerve biopsies in all evaluable samples confirmed the presence of regenerative nerve fibers in 100% of TSHA-120-treated patients (n=11)
- TSHA-120 commercial grade and clinical trial material considered comparable across all key quality attributes as assessed by an extensive panel of release assays and next-generation sequencing
- Positive regulatory feedback from MHRA supports regulatory strategy
- MHRA found functional clinical data, eye pathology and visual acuity, and nerve biopsy data compelling. Agency agreed with commercial manufacturing and release testing strategy, including potency assays and recommended dosing a few patients with commercial grade material, which will be released in
September 2022 . MHRA was supportive of Taysha’s proposal to perform validation work on MFM32 as a key clinical endpoint
- MHRA found functional clinical data, eye pathology and visual acuity, and nerve biopsy data compelling. Agency agreed with commercial manufacturing and release testing strategy, including potency assays and recommended dosing a few patients with commercial grade material, which will be released in
TSHA-102 in Rett syndrome: a self-complementary intrathecally delivered AAV9 gene replacement therapy in clinical development for Rett syndrome. TSHA-102 utilizes the novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform to regulate transgene expression genotypically on a cell-by-cell basis. TSHA-102 is the first-and-only gene therapy in clinical development for Rett syndrome. TSHA-102 has received orphan drug and rare pediatric disease designations from the FDA and has been granted orphan drug designation from the
- Late-breaking positive preclinical data for TSHA-102 in Rett syndrome demonstrated near normalization of survival and normalization of behavior in neonatal knockout Rett mice, a model significantly more severe than the human phenotype
Anticipated 2022 Milestones
- Further regulatory update, including from FDA, for TSHA-120 in GAN by year-end 2022
- First-in-human preliminary Phase 1/2 data for TSHA-102 in Rett syndrome by year-end 2022
Second Quarter 2022 Financial Highlights
Research and Development (R&D) Expenses: Research and development expenses were
General and Administrative (G&A) 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 with slides 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 (
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 Six Months Ended |
|||||||||||||||||||||||||
| 2022 | 2021 | 2022 | 2021 | |||||||||||||||||||||||
| Operating expenses: | ||||||||||||||||||||||||||
| Research and development | $ | 23,118 | $ | 30,643 | $ | 60,917 | $ | 54,497 | ||||||||||||||||||
| General and administrative | 9,867 | 10,129 | 21,336 | 18,365 | ||||||||||||||||||||||
| Total operating expenses | 32,985 | 40,772 | 82,253 | 72,862 | ||||||||||||||||||||||
| Loss from operations | (32,985 | ) | (40,772 | ) | (82,253 | ) | (72,862 | ) | ||||||||||||||||||
| Other income (expense): | ||||||||||||||||||||||||||
| Interest income | 27 | 40 | 41 | 106 | ||||||||||||||||||||||
| Interest expense | (912 | ) | (194 | ) | (1,761 | ) | (194 | ) | ||||||||||||||||||
| Other expense | (3 | ) | - | (11 | ) | - | ||||||||||||||||||||
| Total other expense, net | (888 | ) | (154 | ) | (1,731 | ) | (88 | ) | ||||||||||||||||||
| Net loss | $ | (33,873 | ) | $ | (40,926 | ) | $ | (83,984 | ) | $ | (72,950 | ) | ||||||||||||||
| Net loss per common share, basic and diluted | $ | (0.84 | ) | $ | (1.09 | ) | $ | (2.14 | ) | $ | (1.96 | ) | ||||||||||||||
| Weighted average common shares outstanding, basic and diluted | 40,142,403 | 37,479,164 | 39,163,996 | 37,237,115 | ||||||||||||||||||||||
Condensed Consolidated Balance Sheet Data
(in thousands, except share and per share data)
(Unaudited)
2022 |
2021 |
|||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 66,239 | $ | 149,103 | ||||
| Prepaid expenses and other current assets | 10,596 | 10,499 | ||||||
| Total current assets | 76,835 | 159,602 | ||||||
| Restricted cash | 2,637 | 2,637 | ||||||
| Deferred lease asset | 643 | 667 | ||||||
| Property, plant and equipment, net | 61,011 | 50,610 | ||||||
| Other non-current assets | 1,206 | 440 | ||||||
| Total assets | $ | 142,332 | $ | 213,956 | ||||
| LIABILITIES AND STOCKHOLDERS' EQUITY | ||||||||
| Current liabilities | ||||||||
| Accounts payable | $ | 23,967 | $ | 21,763 | ||||
| Accrued expenses and other current liabilities | 18,986 | 29,983 | ||||||
| Total current liabilities | 42,953 | 51,746 | ||||||
| Build-to-suit lease liability | 25,609 | 25,900 | ||||||
| Term Loan, net | 37,580 | 37,192 | ||||||
| Other non-current liabilities | 3,480 | 3,735 | ||||||
| Total liabilities | 109,622 | 118,573 | ||||||
| Stockholders' equity | ||||||||
| Preferred stock, |
- | - | ||||||
| Common stock, |
1 | - | ||||||
| Additional paid-in capital | 352,342 | 331,032 | ||||||
| Accumulated deficit | (319,633 | ) | (235,649 | ) | ||||
| Total stockholders’ equity | 32,710 | 95,383 | ||||||
| Total liabilities and stockholders' equity | $ | 142,332 | $ | 213,956 | ||||
Company Contact:
Chief Corporate Affairs Officer
klee@tayshagtx.com
Media Contact:
Evoke Canale
carolyn.hawley@evokegroup.com
Source: Taysha Gene Therapies, Inc.