Presented cohort one (low dose) data from both trials of TSHA-102 at 2024 IRSF Rett Syndrome Scientific Meeting that demonstrated durable improvements across consistent clinical domains and an encouraging safety profile in adult (up to 52 weeks) and pediatric (up to 22 weeks) patients
TSHA-102 was generally well tolerated with no SAEs or DLTs as of initial six-week assessment in first patient in cohort two (high dose) of adolescent/adult trial; IDMC approved dosing of second (adolescent/adult trial) and first (pediatric trial) patient in cohort two, which is scheduled for Q3 2024
Cohort two (n=3) and cohort one (n=2) data from both trials (adolescent/adult and pediatric) expected in 1H 2025
Conference call and live webcast today at 8:30 AM Eastern Time
“In the second quarter, we made strong progress across our TSHA-102 clinical program, including reporting encouraging preliminary data in our pediatric trial and longer-term data in our adolescent and adult trial following the low dose of TSHA-102. We dosed the first patient in the high dose cohort of our adolescent and adult trial, and TSHA-102 was generally well tolerated as of the initial six-week assessment. Subsequently, we received IDMC approval to proceed with dosing the second adolescent/adult and first pediatric patient in the high dose cohort of our REVEAL trials. This progress enables us to build on the promising low dose data that demonstrated an encouraging safety profile and improvements across consistent clinical domains impacting daily activities in adult and pediatric patients suffering from Rett syndrome,” said
Recent Corporate and Program Highlights
- Completed public follow-on offering with total net proceeds of
$76.8 million with anticipated cash runway into the fourth quarter of 2026
REVEAL Phase 1/2 Adolescent and Adult Trial (
- Presented longer-term data from cohort one (low dose, n=2) of 5.7x1014 total vector genomes (vg) at the 2024
International Rett Syndrome Foundation (IRSF) Rett Syndrome Scientific Meeting:- Generally well tolerated with no serious adverse events (SAEs) related to TSHA-102 or dose-limiting toxicities (DLTs) as of the 52- and 36-week assessment for patient one and two, respectively
- Sustained and new improvements across multiple efficacy measures and clinical domains relative to baseline, including fine and gross motor skills, communication/socialization, autonomic function and seizure events, through 52- and 25-weeks post-treatment for patient one and two, respectively
- Dosed the first patient in cohort two (high dose, n=3) of 1x1015 total vg, and TSHA-102 was generally well tolerated with no SAEs or DLTs as of the initial six-week assessment
- Enrolled the second patient in cohort two and scheduled dosing for the current quarter, following Independent Data Monitoring Committee (IDMC) review of initial six-week clinical data from the first patient dosed in cohort two
REVEAL Phase 1/2 Pediatric Trial (
Health Canada cleared the pediatric clinical trial application (CTA), enabling expansion of the ongoingU.S. and U.K. REVEAL pediatric trial intoCanada - Presented preliminary data from cohort one (low dose, n=2) of 5.7x1014 total vg at the 2024 IRSF Rett Syndrome Scientific Meeting:
- Generally well-tolerated with no SAEs related to TSHA-102 or DLTs as of the 22- and 11-week assessment for patient one and two, respectively
- Initial improvements across multiple efficacy measures and clinical domains relative to baseline, including fine and gross motor skills, communication/socialization, autonomic function and seizure events, as of 12- and eight-weeks post-treatment for patient one and two, respectively
- Enrolled the first pediatric patient in cohort two (high dose, n=3) of 1x1015 total vg and scheduled dosing for the current quarter, following IDMC approval to proceed with the Company’s request for early advancement to cohort two after review of the initial six-week safety data from the first patient treated with the high dose of TSHA-102 in the adolescent and adult trial
Anticipated Milestones
- REVEAL Adolescent and Adult Trial
- Dosing of the second patient in cohort two (high dose) scheduled for the third quarter of 2024
- Safety and efficacy data from cohort two (n=3) and an update on safety and efficacy data from cohort one (n=2) expected in the first half of 2025
- REVEAL Pediatric Trial
- Dosing of the first patient in cohort two (high dose) scheduled for the third quarter of 2024
- Safety and efficacy data from cohort two (n=3) and an update on safety and efficacy data from cohort one (n=2) expected in the first half of 2025
Second Quarter 2024 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 8:30 a.m. ET to review its financial and operating results and provide a corporate update. The dial-in number for the conference call is 877-407-0792 (
About TSHA-102
TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the
About Rett Syndrome
Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the
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, but are not limited to, statements concerning the potential of TSHA-102, including the reproducibility and durability of any favorable results initially seen in patients dosed to date in clinical trials, and our other 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, including the timing of initiating additional trials and reporting data from our clinical trials, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and our current cash resources supporting our planned operating expenses and capital requirements into the fourth quarter of 2026. 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) |
||||||||||||||||
| For the Three Months Ended |
For the Six Months Ended |
|||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| Revenue | $ | 1,112 | $ | 2,395 | $ | 4,523 | $ | 7,101 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 15,073 | 19,791 | 35,730 | 32,305 | ||||||||||||
| General and administrative | 7,338 | 5,988 | 14,422 | 14,739 | ||||||||||||
| Total operating expenses | 22,411 | 25,779 | 50,152 | 47,044 | ||||||||||||
| Loss from operations | (21,299 | ) | (23,384 | ) | (45,629 | ) | (39,943 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Change in fair value of warrant liability | 195 | - | (142 | ) | - | |||||||||||
| Change in fair value of term loan | (1,279 | ) | - | (2,332 | ) | - | ||||||||||
| Interest income | 1,440 | 223 | 3,133 | 542 | ||||||||||||
| Interest expense | (27 | ) | (1,440 | ) | (56 | ) | (2,814 | ) | ||||||||
| Other (expense) income | 42 | 3 | 37 | (5 | ) | |||||||||||
| Total other income (expense), net | 371 | (1,214 | ) | 640 | (2,277 | ) | ||||||||||
| Net loss | $ | (20,928 | ) | $ | (24,598 | ) | $ | (44,989 | ) | $ | (42,220 | ) | ||||
| Net loss per common share, basic and diluted | $ | (0.09 | ) | $ | (0.38 | ) | $ | (0.19 | ) | $ | (0.66 | ) | ||||
| Weighted average common shares outstanding, basic and diluted | 232,821,553 | 64,244,531 | 232,035,448 | 63,755,435 | ||||||||||||
Condensed Consolidated Balance Sheet Data (in thousands, except share and per share data) |
||||||||
2024 |
2023 |
|||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 172,743 | $ | 143,940 | ||||
| Restricted cash | 449 | 449 | ||||||
| Prepaid expenses and other current assets | 3,278 | 3,479 | ||||||
| Assets held for sale | 2,000 | 2,000 | ||||||
| Total current assets | 178,470 | 149,868 | ||||||
| Restricted cash | 2,151 | 2,151 | ||||||
| Property, plant and equipment, net | 10,513 | 10,826 | ||||||
| Operating lease right-of-use assets | 8,971 | 9,582 | ||||||
| Other non-current assets | 288 | 304 | ||||||
| Total assets | $ | 200,393 | $ | 172,731 | ||||
| LIABILITIES AND STOCKHOLDERS' EQUITY | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 8,718 | $ | 6,366 | ||||
| Accrued expenses and other current liabilities | 11,875 | 12,284 | ||||||
| Deferred revenue | 13,583 | 18,106 | ||||||
| Total current liabilities | 34,176 | 36,756 | ||||||
| Term loan, net | 37,835 | 40,508 | ||||||
| Operating lease liability, net of current portion | 18,134 | 18,953 | ||||||
| Other non-current liabilities | 1,380 | 1,577 | ||||||
| Total liabilities | 91,525 | 97,794 | ||||||
| Stockholders' equity | ||||||||
| Preferred stock, |
— | — | ||||||
| Common stock, |
2 | 2 | ||||||
| Additional paid-in capital | 664,457 | 587,942 | ||||||
| Accumulated other comprehensive income | 2,405 | — | ||||||
| Accumulated deficit | (557,996 | ) | (513,007 | ) | ||||
| Total stockholders’ equity | 108,868 | 74,937 | ||||||
| Total liabilities and stockholders' equity | $ | 200,393 | $ | 172,731 | ||||
Company Contact:
Director, Head of Corporate Communications and Investor Relations
hcollins@tayshagtx.com
Media Contact:
Inizio Evoke
Carolyn.hawley@inizioevoke.com
Source: Taysha Gene Therapies, Inc.