Full Press Release Details
Taysha Gene Therapies Reports Third Quarter 2024 Financial Results and Provides Corporate Update
High dose TSHA-102 was generally well tolerated with no SAEs or DLTs in two adolescent/adult
patients and one pediatric patient as of data cutoff; IDMC approved continued enrollment in cohort two (high dose) across both REVEAL trials; eight patients dosed to date (low dose=4, high dose=4)
Advanced discussions with the FDA on trial design, endpoints and potential use of established natural history dataset for Part B of
REVEAL trials, and aligned on a meeting cadence to expedite the development plan for TSHA-102 following initial RMAT Type B meeting
FDA approved use of pivotal TSHA-102 product in REVEAL trials based on successful demonstration of
analytical comparability; Company released pivotal product manufactured with the final commercial manufacturing process following Type D CMC meeting
Clinical data from cohort two (high dose) and cohort one (low dose) of both REVEAL trials expected in H1 2025
Conference call and live webcast today at 4:30 PM Eastern Time
Dallas November 13, 2024 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 third quarter ended September 30, 2024, and
provided a corporate update.
We are pleased with the progress made with the FDA on further elucidating the potential regulatory pathway for TSHA-102 as we advanced discussions on the trial design, endpoints and potential use of an established natural history dataset for Part B of our REVEAL trials. Additionally, we aligned on a meeting cadence to
expedite the development plan for TSHA-102, said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. We are in a strong position with CMC, having obtained FDA approval to use the pivotal
product in our REVEAL trials based on the successful demonstration of analytical comparability. Subsequently, we released the pivotal product manufactured with the final commercial manufacturing process that we intend to use in Part B.
Mr. Nolan continued, Clinical data presented from the adult patients with the most advanced stage of the disease treated with the low dose of TSHA-102 indicate a pattern of early clinical improvements and functional gains across multiple domains within four weeks post-treatment that persisted and strengthened over time. As the pediatric data mature, we
anticipate that the early clinical improvements and functional gains observed should also persist and strengthen over time in the pediatric patients treated with TSHA-102. We look forward to reporting
longer-term data from the low dose cohort and data from the high dose cohort of both REVEAL trials in the first half of 2025. We plan to continue working closely with the FDA through the RMAT mechanism to solidify the regulatory pathway for TSHA-102 based on the totality of data and remain focused on execution as we prepare for what we expect to be an impactful year ahead.
Recent Corporate and TSHA-102 Program Highlights
Anticipated Milestones
REVEAL Adolescent and Adult
REVEAL Pediatric Trial
Third Quarter 2024 Financial Highlights
Research and Development Expenses: Research and development expenses were $14.9 million for the three months ended September 30, 2024,
compared to $11.8 million for the three months ending September 30, 2023. The $3.1 million increase was driven by an $0.8 million increase in GMP batch activities during the three months ended September 30, 2024, which is
representative of the intended commercial manufacturing process for TSHA-102 in Rett syndrome. Additionally, compensation for R&D employees increased as a result of higher headcount, and this was partially
offset by lower consultant and contractor expenses.
General and Administrative Expenses: General and administrative expenses were
$7.9 million for the three months ended September 30, 2024, compared to $8.6 million for the three months ended September 30, 2023. The decrease of $0.7 million was primarily due to the decrease in issuance costs allocated
to the liability-classified 2023 pre-funded warrants associated with the August 2023 financing.
loss: Net loss for the three months ended September 30, 2024, was $25.5 million, or $0.1 per share, compared to a net loss of $117.1 million, or $0.93 per share, for the three months
ended September 30, 2023. The reduction in net loss in 2024 was primarily due to a non-cash loss of $100.5 million recorded in 2023 from a change in fair value of warrant liability from the 2023 pre-funded warrants associated with the August 2023 financing.
Cash and cash equivalents: As of September 30, 2024, Taysha had $157.7 million in cash
and cash equivalents. Taysha expects that its current cash resources will support planned operating expenses and capital requirements into the fourth quarter of 2026.
Conference Call and Webcast Information
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 13748703. The live webcast and replay may be accessed by visiting Taysha s website.
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
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, including
with respect to functional milestones, 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, the clinical
potential of intrathecal administration 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 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 September 30, | For the Nine Months Ended September 30, | |||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| Revenue | $ | 1,788 | $ | 4,746 | $ | 6,311 | $ | 11,847 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 14,946 | 11,791 | 50,676 | 44,096 | ||||||||||||
| General and administrative | 7,902 | 8,589 | 22,324 | 23,328 | ||||||||||||
| Impairment of long-lived assets | 4,838 | 616 | 4,838 | 616 | ||||||||||||
| Total operating expenses | 27,686 | 20,996 | 77,838 | 68,040 | ||||||||||||
| Loss from operations | (25,898 | ) | (16,250 | ) | (71,527 | ) | (56,193 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Change in fair value of warrant liability | 75 | (100,456 | ) | (67 | ) | (100,456 | ) | |||||||||
| Change in fair value of term loan | (1,703 | ) | (4,035 | ) | ||||||||||||
| Interest income | 2,107 | 1,109 | 5,240 | 1,651 | ||||||||||||
| Interest expense | (24 | ) | (1,471 | ) | (80 | ) | (4,285 | ) | ||||||||
| Other expense | (81 | ) | (19 | ) | (44 | ) | (24 | ) | ||||||||
| Total other income (expense), net | 374 | (100,837 | ) | 1,014 | (103,114 | ) | ||||||||||
| Net loss | $ | (25,524 | ) | $ | (117,087 | ) | $ | (70,513 | ) | $ | (159,307 | ) | ||||
| Net loss per common share, basic and diluted | $ | (0.10 | ) | $ | (0.93 | ) | $ | (0.29 | ) | $ | (1.88 | ) | ||||
| Weighted average common shares outstanding, basic and diluted | 267,824,045 | 125,700,799 | 244,052,057 | 84,630,796 |
Taysha Gene Therapies, Inc.
Condensed Consolidated Balance Sheet Data
(in thousands, except share and per share data)
| September 30, 2024 | December 31, 2023 | |||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 157,688 | $ | 143,940 | ||||
| Restricted cash | 449 | 449 | ||||||
| Prepaid expenses and other current assets | 3,418 | 3,479 | ||||||
| Assets held for sale | 2,000 | |||||||
| Total current assets | 161,555 | 149,868 | ||||||
| Restricted cash | 2,151 | 2,151 | ||||||
| Property, plant and equipment, net | 7,613 | 10,826 | ||||||
| Operating lease right-of-use assets | 8,678 | 9,582 | ||||||
| Other non-current assets | 220 | 304 | ||||||
| Total assets | $ | 180,217 | $ | 172,731 | ||||
| LIABILITIES AND STOCKHOLDERS EQUITY | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 4,932 | $ | 6,366 | ||||
| Accrued expenses and other current liabilities | 12,608 | 12,284 | ||||||
| Deferred revenue | 11,795 | 18,106 | ||||||
| Total current liabilities | 29,335 | 36,756 | ||||||
| Term loan, net | 42,971 | 40,508 | ||||||
| Operating lease liability, net of current portion | 17,751 | 18,953 | ||||||
| Other non-current liabilities | 1,363 | 1,577 | ||||||
| Total liabilities | 91,420 | 97,794 | ||||||
| Stockholders equity | ||||||||
| Preferred stock, $0.00001 par value per share; 10,000,000 shares authorized and no shares issued and outstanding as of September 30, 2024 and December 31, 2023 | ||||||||
| Common stock, $0.00001 par value per share; 400,000,000 shares authorized and 204,943,306 and 186,960,193 issued and outstanding as of September 30, 2024 and December 31, 2023, respectively | 2 | 2 | ||||||
| Additional paid-in capital | 674,643 | 587,942 | ||||||
| Accumulated other comprehensive income | (2,328 | ) | ||||||
| Accumulated deficit | (583,520 | ) | (513,007 | ) | ||||
| Total stockholders equity | 88,797 | 74,937 | ||||||
| Total liabilities and stockholders equity | $ | 180,217 | $ | 172,731 |
Director, Head of Corporate Communications and
Taysha Gene Therapies, Inc.