Data from first adult patient in REVEAL Phase 1/2 trial showed TSHA-102 was well-tolerated with no treatment-emergent SAEs as of 20-week assessment with sustained improvement across key efficacy measures and new improvement in R-MBA, PGI-I and hand function, a hallmark characteristic of Rett syndrome at week 12
Data from second adult patient showed TSHA-102 was well-tolerated with no treatment-emergent SAEs as of six-week assessment with improvement across key efficacy measures, including CGI-I, R-MBA, PGI-I and RSBQ at week four
Notable differences in genetic mutation and phenotypic expression reported between patient one and two; Principal Investigator (PI) observed improvements in both patients across multiple domains, including autonomic function, socialization, and gross and fine motor skills, including further improvement in ability to sit unassisted at week 12 in patient one and improved posture, gait and stability at week four in patient two
IDMC provided clearance to dose third adult patient based on available data; dosing of third adult patient and completion of cohort one (low dose) expected in the fourth quarter of 2023/first quarter of 2024; dosing of first pediatric patient in the
Entered into loan and security agreement with Trinity Capital that extends cash runway into 2026 and includes no financial covenants or warrants
Conference call and live webcast today at 4:30 PM Eastern Time
“Prior to initiating the REVEAL trial, the expectation of seeing a clinical benefit in adults with stage four Rett syndrome was low due to the advanced and relentless progression of the disease. We are highly encouraged by the positive 12-week data from the first adult patient and initial four-week data from the second adult patient in the low dose TSHA-102 cohort,” said
Dr.
Recent Corporate Highlights
- Presented two posters at the
European Society of Gene & Cell Therapy (ESGCT) 30th AnnualCongress on new preclinical in vitro data supporting the miRARE technology, and initial clinical data from the first adult patient dosed in the REVEAL Phase 1/2 trial United States (U.S.) Food and Drug Administration (FDA) granted Fast Track Designation to TSHA-102 for Rett syndrome- Entered into a loan and security agreement with Trinity Capital and terminated existing loan and security agreement with
Silicon Valley Bank , extending cash runway into 2026; no financial covenants or warrants associated with the loan and security agreement with Trinity Capital
Recent Clinical 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 miRARE technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression.
TSHA-102 is being evaluated in the REVEAL Phase 1/2 adult trial, a first-in-human, open-label, randomized, dose-escalation and dose-expansion study in
Results from the first patient (large MECP2 deletion; associated with severe phenotype) and second patient (missense MECP2 mutation; associated with milder phenotype) with late motor deterioration stage four Rett syndrome dosed with TSHA-102 in the low dose cohort:
- Generally well-tolerated with no treatment-emergent serious adverse events (SAEs) as of 20-week assessment post-treatment for patient one and six-week assessment for patient two
- Based on clinical observations by the Principal Investigator (PI), both patients demonstrated improvement in multiple clinical domains, with sustained and new improvements in patient one 12-weeks post-treatment and initial improvements in patient two four-weeks post-treatment, including:
- Autonomic function: improved breathing patterns and sleep quality/duration (patient one) reduced seizures and improved breathing patterns (patient two)
- Socialization: improved social interest and vocalization (patient one) improved social interest (patient two)
- Gross motor skills: gained ability to sit unassisted and move legs (patient one) improved posture, gait and stability (patient two)
- Fine motor skills: improved hand function (patient one) improved hand stereotypies (patient two)
- Seizure Diary demonstrated comparable seizure events relative to baseline through 20-weeks post-treatment in patient one and reduced seizure events relative to baseline through day 33 post-treatment for patient two, based on caregiver-reported medical history
- Clinical improvements demonstrated in both patients across key efficacy measures include:
- Patient one: sustained improvement through 12-weeks in Clinical Global Impression–Improvement (CGI-I), Clinical Global Impression–Severity (CGI-S) and Rett Syndrome Behavior Questionnaire (RSBQ), with new improvements in Revised Motor Behavior Assessment (R-MBA), Parental Global Impressions–Improvement (PGI-I) and Rett Syndrome Hand Function Scale (RSHFS)
- Patient two: improvement four-weeks post-treatment in CGI-I, PGI-I, RSBQ and R-MBA
- Figure accompanying this announcement is available at: https://www.globenewswire.com/NewsRoom/AttachmentNg/9b39103b-685c-4849-9072-97f32658320c. Additional information on available clinical data is available in the Company’s quarterly report on Form 10-Q for the quarter ended
September 30, 2023 , to be filed with theSEC . - Independent Data Monitoring Committee (IDMC) provided clearance to dose third adult patient based on available data
Upcoming Milestones
- Dosing of third adult patient and completion of dosing in cohort one (low dose) in the adult trial in
Canada anticipated in the fourth quarter of 2023/first quarter of 2024 - Further updates on available clinical data from the low dose cohort expected in the first quarter of 2024
- Dosing of first pediatric Rett syndrome patient in the
U.S. anticipated in the first quarter of 2024 U.K. Medicines and Healthcare products Regulatory Agency (MHRA) response to Clinical Trial Application (CTA) for TSHA-102 in pediatric patients with Rett syndrome expected by year-end 2023
Third Quarter 2023 Financial Highlights
Research and Development 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 today at 4:30 pm ET to review its financial and operating results and to provide corporate and clinical updates. 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 the reproducibility and durability of any favorable results initially seen in our first and second patients dosed in the REVEAL trial and 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 timing of our clinical trials, including reporting data therefrom, the forecast of our cash runway and the Company’s expectations regarding funding, operating and working capital expenditures. 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 Balance Sheet Data | ||||||||
| (in thousands, except share and per share data) | ||||||||
| (Unaudited) | ||||||||
2023 |
2022 |
|||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 164,278 | $ | 87,880 | ||||
| Prepaid expenses and other current assets | 5,529 | 8,537 | ||||||
| Assets held for sale | 2,000 | — | ||||||
| Total current assets | 171,807 | 96,417 | ||||||
| Restricted cash | 2,637 | 2,637 | ||||||
| Property, plant and equipment, net | 11,169 | 14,963 | ||||||
| Operating lease right-of-use assets | 9,852 | 10,943 | ||||||
| Other non-current assets | 304 | 1,316 | ||||||
| Total assets | $ | 195,769 | $ | 126,276 | ||||
| LIABILITIES AND STOCKHOLDERS' (DEFICIT) EQUITY | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 7,520 | $ | 10,946 | ||||
| Accrued expenses and other current liabilities | 13,638 | 18,287 | ||||||
| Deferred revenue | 18,759 | 33,557 | ||||||
| Warrant liability | 140,534 | — | ||||||
| Total current liabilities | 180,451 | 62,790 | ||||||
| Deferred revenue, net of current portion | 2,951 | — | ||||||
| Term loan, net | 38,548 | 37,967 | ||||||
| Operating lease liability, net of current portion | 19,101 | 20,440 | ||||||
| Other non-current liabilities | 3,832 | 4,130 | ||||||
| Total liabilities | 244,883 | 125,327 | ||||||
| Stockholders' (deficit) equity | ||||||||
| Preferred stock, |
— | — | ||||||
| Common stock, |
2 | 1 | ||||||
| Additional paid-in capital | 511,632 | 402,389 | ||||||
| Accumulated deficit | (560,748 | ) | (401,441 | ) | ||||
| Total stockholders’ (deficit) equity | (49,114 | ) | 949 | |||||
| Total liabilities and stockholders' (deficit) equity | $ | 195,769 | $ | 126,276 | ||||
| Condensed Consolidated Statement of Operations | ||||||||||||||||
| (in thousands, except share and per share data) | ||||||||||||||||
| (Unaudited) | ||||||||||||||||
| For the Three Months Ended |
For the Nine Months Ended |
|||||||||||||||
| 2023 | 2022 | 2023 | 2022 | |||||||||||||
| Revenue | $ | 4,746 | $ | — | $ | 11,847 | $ | — | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 11,791 | 16,774 | 44,096 | 78,462 | ||||||||||||
| General and administrative | 8,589 | 8,683 | 23,328 | 30,019 | ||||||||||||
| Impairment of long-lived assets | 616 | — | 616 | — | ||||||||||||
| Total operating expenses | 20,996 | 25,457 | 68,040 | 108,481 | ||||||||||||
| Loss from operations | (16,250 | ) | (25,457 | ) | (56,193 | ) | (108,481 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Change in fair value of warrant liability | (100,456 | ) | — | (100,456 | ) | — | ||||||||||
| Interest income | 1,109 | 9 | 1,651 | 50 | ||||||||||||
| Interest expense | (1,471 | ) | (1,078 | ) | (4,285 | ) | (2,493 | ) | ||||||||
| Other expense | (19 | ) | (1 | ) | (24 | ) | (12 | ) | ||||||||
| Total other expense, net | (100,837 | ) | (1,070 | ) | (103,114 | ) | (2,455 | ) | ||||||||
| Net loss | $ | (117,087 | ) | $ | (26,527 | ) | $ | (159,307 | ) | $ | (110,936 | ) | ||||
| Net loss per common share, basic and diluted | $ | (0.93 | ) | $ | (0.65 | ) | $ | (1.88 | ) | $ | (2.79 | ) | ||||
| Weighted average common shares outstanding, basic and diluted | 125,700,799 | 40,937,808 | 84,630,796 | 39,761,764 | ||||||||||||
Company Contact:
Director, Head of Corporate Communications and Investor Relations
hcollins@tayshagtx.com
Media Contact:
carolyn.hawley@canalecomm.com
REVEAL Phase 1/2
REVEAL Phase 1/2
Source: Taysha Gene Therapies, Inc.