Taysha Gene Therapies Reports First Quarter 2024 Financial Results and Provides Corporate Update
Taysha Gene Therapies reported its Q1 2024 financial results and provided a corporate update.
The company completed dosing in the first low-dose cohort of the REVEAL Phase 1/2 trial for adolescents and adults, and dosed the second pediatric patient in the same phase.
The FDA granted RMAT designation for TSHA-102, enabling expedited development.
Initial data from both trials is expected in mid-2024, with updates from higher dose cohorts due in 2H 2024.
For Q1 2024, Taysha reported an increase in R&D expenses to $20.7 million and a decrease in G&A expenses to $7.1 million, resulting in a net loss of $24.1 million.
With $124 million in cash, Taysha's resources are expected to support operations into 2026.
- Completed dosing in cohort one of REVEAL Phase 1/2 adolescent and adult trial.
- Dosed second pediatric patient in cohort one of REVEAL Phase 1/2 pediatric trial.
- FDA granted RMAT designation for TSHA-102.
- Initial data from cohort one in REVEAL trials expected mid-2024.
- High dose cohort dosing scheduled for Q2 2024.
- Cash and cash equivalents of $124 million as of March 31, 2024.
- Reduction in G&A expenses to $7.1 million from $8.8 million compared to Q1 2023.
- Current cash resources expected to support operations into 2026.
- Net loss increased to $24.1 million, compared to $17.6 million in Q1 2023.
- R&D expenses rose to $20.7 million from $12.5 million compared to Q1 2023.
Insights
Financial Overview: Taysha Gene Therapies reported an increased net loss of
Cash Position: With
Short-term and Long-term Impact: Short-term, the high cash burn and increased losses could be a concern for investors. However, the long-term view is more positive, with substantial cash reserves supporting ongoing trials and potential expedited approvals due to the RMAT designation.
Key Insight: Investors should weigh the high current expenditure against the potential long-term payoff from successful drug development and potential expedited market entry. While the financials show increased loss, the strategic investments could yield significant future returns.
Clinical Progress: Taysha’s first quarter saw significant advancements in their TSHA-102 program for Rett syndrome, including successful completion of the low-dose cohort and initiation of high-dose cohort trials. The Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA is notable, indicating the therapy's potential to address unmet medical needs in Rett syndrome. Early data showed no serious adverse events and some promising improvements in clinical domains, which is encouraging for the continuation of trials.
Safety and Efficacy: The safety profile of TSHA-102 is crucial. So far, the absence of serious adverse events and some positive preliminary efficacy results are good signs. However, long-term data is essential to fully understand the therapy’s impact.
Industry Context: RMAT designation can expedite development and review processes, potentially bringing TSHA-102 to market faster if further trials support current findings. This can be a significant competitive advantage in the biotech sector.
Key Insight: The positive early-stage results and regulatory support suggest that TSHA-102 has promise, but much will depend on forthcoming data from ongoing trials. Investors should watch mid-2024 updates closely for more definitive insights.
Market Potential: Rett syndrome is a severe condition with limited treatment options, creating a significant unmet medical need. Taysha's focus on AAV-based gene therapies positions it uniquely in the biotech market, where such innovations can command high market value if proven effective. RMAT designation enhances this potential by allowing closer dialogue with the FDA, suggesting a smoother path towards commercialization.
Competitive Landscape: Taysha is not alone in the gene therapy space, but few competitors target monogenic CNS diseases with such specificity. Success in these trials could establish Taysha as a leader in this niche market.
Strategic Positioning: The advancement to higher dose cohorts and ongoing pediatric trials are strategic moves. These steps aim to expand the potential patient base and demonstrate broader applicability of TSHA-102.
Key Insight: From a market perspective, the RMAT designation and ongoing clinical progress position Taysha favorably within the biotech sector. The focus on a high-demand, specialized therapeutic area enhances its growth potential.
Completed dosing in cohort one (low dose, 5.7x1014 total vg) of REVEAL Phase 1/2 adolescent and adult trial with longer-term data supporting the safety profile and durable response of TSHA-102; enrolled first patient in cohort two (high dose, 1x1015 total vg) with dosing scheduled for Q2 2024
Dosed second pediatric patient in cohort one (low dose, 5.7x1014 total vg) of REVEAL Phase 1/2 pediatric trial in Q1 2024
FDA granted Regenerative Medicine Advanced Therapy designation following review of available safety and efficacy data from the first three patients dosed with the low dose of TSHA-102 across both REVEAL trials (adolescent/adult and pediatric)
Initial data from cohort one (low dose, 5.7x1014 total vg) in REVEAL pediatric trial and update from cohort one in REVEAL adolescent and adult trial expected mid-2024; initial data from cohort two (high dose, 1x1015 total vg) in both trials (adolescent/adult and pediatric) expected in 2H 2024
Conference call and live webcast today at 4:30 PM Eastern Time
DALLAS, May 14, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today reported financial results for the first quarter ended March 31, 2024, and provided a corporate update.
“We are pleased with the recent progress we have made to advance our TSHA-102 program in clinical evaluation for Rett syndrome, including enrolling the first patient in the high dose cohort of our REVEAL adolescent and adult trial earlier than planned and dosing the second patient in our REVEAL pediatric trial. We recently received RMAT designation for TSHA-102 following the FDA’s review of safety and efficacy data from the first three patients dosed with the low dose of TSHA-102 across both of our REVEAL trials, which we believe reinforces the therapeutic potential of TSHA-102,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “We look forward to the year ahead as we remain focused on moving to the high dose cohort and generating critical longer-term clinical data across a broad population of patients with Rett syndrome that will guide the next phase of our studies. We expect to report initial clinical data from our REVEAL pediatric trial and provide an update on the completed low dose cohort from our REVEAL adolescent and adult trial in mid-2024.”
Recent Corporate and Program Highlights
Received Regenerative Medicine Advanced Therapy (RMAT) designation for TSHA-102 from the United States Food and Drug Administration (FDA): A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious condition and preliminary clinical evidence indicates the therapy has the potential to address unmet medical needs for such condition. RMAT designation follows the FDA’s review of available safety and efficacy data from the first three patients with Rett syndrome dosed with the low dose of TSHA-102 across the REVEAL Phase 1/2 adolescent and adult trial and the REVEAL Phase 1/2 pediatric trial. RMAT designation will enable increased dialogue with the FDA to support the potential expedited development and review of TSHA-102.
REVEAL Phase 1/2 Adolescent and Adult Trial (Canada and U.S.): a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in adolescent and adult females aged 12 years and older with Rett syndrome due to MECP2 loss-of-function mutation. TSHA-102 is administered as a single lumbar intrathecal injection. Dose escalation will evaluate two dose levels of TSHA-102 sequentially. The maximum tolerated dose (MTD) or maximum administered dose (MAD) established in Part A will then be administered during dose expansion in Part B of the study.
- Completed dosing in cohort one (low dose, n=2) of 5.7x1014 total vg
- Announced longer-term data from cohort one including two adult patients with late motor deterioration stage four Rett syndrome and different genetic mutation severity and phenotypic expression:
- Adult patient one: TSHA-102 was generally well tolerated with no treatment-emergent serious adverse events (SAEs) as of the 35-week assessment, with sustained and new improvement across key efficacy measures at decreased steroid levels six-months post-treatment. The Principal Investigator observed sustained and new improvements across multiple clinical domains including autonomic function, socialization/communication, motor skills and stable seizure events, through 35-weeks post-treatment following completion of steroid taper.
- Adult patient two: TSHA-102 was generally well tolerated with no treatment-emergent SAEs as of the 19-week assessment, with sustained and new improvement across key efficacy measures at 12-weeks post-treatment. The Principal Investigator observed sustained and new improvements across multiple clinical domains including autonomic function, socialization/communication, motor skills and significantly reduced seizures, through 19 weeks post-treatment at decreased steroid levels.
- Enrolled the first patient in cohort two (high dose, n=3) of 1x1015 total vg and scheduled dosing for the second quarter of 2024 following Independent Data Monitoring Committee (IDMC) approval of the Company’s request to dose escalate immediately, which enabled early advancement to cohort two
REVEAL Phase 1/2 Pediatric Trial (U.S. and U.K.): an open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 in pediatric females with Rett syndrome due to MECP2 loss-of-function mutation. TSHA-102 is administered as a single lumbar intrathecal injection. Part A of the study will focus on determining MAD and MTD in patients aged 5 to 8 years old. Part B is the dose expansion phase and will evaluate TSHA-102 at the MAD or MTD in two age cohorts (5 to 8 years and 3 to 5 years).
- Dosed the second pediatric patient in cohort one (low dose, n=3) of 5.7x1014 total vg in the first quarter of 2024 following IDMC review of six-week post-treatment assessment data from the first pediatric patient dosed
Anticipated 2024 Milestones
- REVEAL Adolescent and Adult Trial
- Dosing of the first patient in cohort two (high dose) of 1x1015 total vg scheduled for the second quarter of 2024
- Update on available safety and efficacy data from completed cohort one (low dose) of 5.7x1014 total vg expected in mid-2024
- Initial available safety and efficacy data from cohort two expected in the second half of 2024
- REVEAL Pediatric Trial
- Initial available safety and efficacy data from cohort one (low dose) of 5.7x1014 total vg expected in mid-2024
- Initial available safety and efficacy data from cohort two (high dose) of 1x1015 total vg expected in the second half of 2024
First 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 March 31, 2024, was
Cash and cash equivalents: As of March 31, 2024, Taysha had
Conference Call and Webcast Information
Taysha management will hold a conference call and webcast today at 4:30 p.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 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13745689. The live webcast and replay may be accessed by visiting Taysha’s website at https://ir.tayshagtx.com/news-events/events-presentations.
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 European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency.
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 U.S., EU, and U.K.
About Taysha Gene Therapies
Taysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company’s management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit www.tayshagtx.com.
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, 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 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 Securities and Exchange Commission (“SEC”) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2023, which is available on the SEC’s website at www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.
| Taysha Gene Therapies, Inc. Condensed Consolidated Statements of Operations (in thousands, except share and per share data) | |||||||
| For the Three Months Ended March 31, | |||||||
| 2024 | 2023 | ||||||
| Revenue | $ | 3,411 | $ | 4,706 | |||
| Operating expenses: | |||||||
| Research and development | 20,657 | 12,514 | |||||
| General and administrative | 7,084 | 8,751 | |||||
| Total operating expenses | 27,741 | 21,265 | |||||
| Loss from operations | (24,330 | ) | (16,559 | ) | |||
| Other income (expense): | |||||||
| Change in fair value of warrant liability | (337 | ) | — | ||||
| Change in fair value of term loan | (1,053 | ) | — | ||||
| Interest income | 1,693 | 319 | |||||
| Interest expense | (29 | ) | (1,374 | ) | |||
| Other expense | (5 | ) | (8 | ) | |||
| Total other income (expense), net | 269 | (1,063 | ) | ||||
| Net loss | $ | (24,061 | ) | $ | (17,622 | ) | |
| Net loss per common share, basic and diluted | $ | (0.10 | ) | $ | (0.28 | ) | |
| Weighted average common shares outstanding, basic and diluted | 231,249,344 | 63,260,905 | |||||
| Taysha Gene Therapies, Inc. Condensed Consolidated Balance Sheet Data (in thousands, except share and per share data) | |||||||
| March 31, 2024 | December 31, 2023 | ||||||
| ASSETS | |||||||
| Current assets: | |||||||
| Cash and cash equivalents | $ | 123,980 | $ | 143,940 | |||
| Restricted cash | 449 | 449 | |||||
| Prepaid expenses and other current assets | 4,168 | 3,479 | |||||
| Assets held for sale | 2,000 | 2,000 | |||||
| Total current assets | 130,597 | 149,868 | |||||
| Restricted cash | 2,151 | 2,151 | |||||
| Property, plant and equipment, net | 10,686 | 10,826 | |||||
| Operating lease right-of-use assets | 9,261 | 9,582 | |||||
| Other non-current assets | 304 | 304 | |||||
| Total assets | $ | 152,999 | $ | 172,731 | |||
| LIABILITIES AND STOCKHOLDERS' EQUITY | |||||||
| Current liabilities: | |||||||
| Accounts payable | $ | 10,380 | $ | 6,366 | |||
| Accrued expenses and other current liabilities | 13,562 | 12,284 | |||||
| Deferred revenue | 14,695 | 18,106 | |||||
| Total current liabilities | 38,637 | 36,756 | |||||
| Term loan, net | 40,512 | 40,508 | |||||
| Operating lease liability, net of current portion | 18,499 | 18,953 | |||||
| Other non-current liabilities | 1,502 | 1,577 | |||||
| Total liabilities | 99,150 | 97,794 | |||||
| Stockholders' equity | |||||||
| Preferred stock, | — | — | |||||
| Common stock, | 2 | 2 | |||||
| Additional paid-in capital Accumulated other comprehensive loss | 591,166 (251) | 587,942 — | |||||
| Accumulated deficit | (537,068 | ) | (513,007 | ) | |||
| Total stockholders’ equity | 53,849 | 74,937 | |||||
| Total liabilities and stockholders' equity | $ | 152,999 | $ | 172,731 | |||
Company Contact:
Hayleigh Collins
Director, Head of Corporate Communications and Investor Relations
Taysha Gene Therapies, Inc.
hcollins@tayshagtx.com
Media Contact:
Carolyn Hawley
Inizio Evoke
Carolyn.hawley@inizioevoke.com
FAQ
What are Taysha Gene Therapies' Q1 2024 financial results?
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