Full Press Release Details
Taysha Gene Therapies Reports Third Quarter 2023 Financial Results and Provides Corporate and Clinical
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
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 U.S. expected in the first quarter of 2024
Entered into loan and security agreement with Trinity Capital that extends cash runway into 2026 and includes no financial covenants or
Conference call and live webcast today at 4:30 PM Eastern Time
Dallas November 14, 2023 Taysha Gene Therapies, Inc. (Nasdaq: TSHA) ( Taysha or the Company ), a
clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today reported financial
results for the third quarter ended September 30, 2023, and provided corporate and clinical updates.
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 Sean P. Nolan, Chairman and Chief Executive Officer of Taysha.
Importantly, response was seen across multiple clinical domains in both stage four patients with different genetic mutation severity and phenotypic expression, including autonomic function, socialization, and gross and fine motor skills. These
early improvements in both patients, coupled with the sustained response through week 12 in the first patient, support the transformative potential of TSHA-102 across multiple genotypes of Rett syndrome.
Dr. Elsa Rossignol, M.D., FRCP, FAAP, Associate Professor in Neuroscience and Pediatrics at the
Universit de Montr al, and Principal Investigator of the REVEAL trial at the CHU Sainte-Justine added, The two adult patients dosed with TSHA-102 have different mutations in their
MECP2 gene that manifest in different phenotypes and clinical severity. Following treatment, both patients experienced improvement in key clinical domains impacting activities of daily living, including breathing dysrhythmia, autonomic
function, socialization, and gross and fine motor skills. Both patients display significantly reduced breathing dysrhythmia, with less breath holding spells and infrequent hyperventilation, improved limb perfusion and vastly improved interest in
social communication and activities. In addition, the first patient experienced sustained and new improvements, with restored movement in her legs and the gained ability to sit unassisted for up to 15 minutes for the first time in over a decade.
Further, her hand function improved with the gained ability to grasp objects with her non-dominant hand and transfer them to her dominant hand for the first time since infancy. Following treatment, the second
patient s posture, gait and stability improved, resulting in straighter posture and smoother movements when walking. Her hand stereotypies also improved for the first time since regression at age three: she now displays less forceful hand
wringing and her hands are often open and relaxed, providing new opportunities for fine motor skill learning. In addition, her seizures are much less frequent. I m encouraged by the early positive signals and consistent improvement seen in both
patients following treatment.
Recent Corporate Highlights
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 Canada evaluating the safety and preliminary efficacy of TSHA-102 in adult females with Rett syndrome due to MECP2
loss-of-function mutation.
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
Third Quarter 2023 Financial Highlights
Research and Development Expenses: Research and development expenses were $11.8 million for the three months ended September 30, 2023,
compared to $16.8 million for the three months ended September 30, 2022. The net change was due to a $9.3 million decrease due to lower compensation expense as a result of reduced headcount, lower licensing milestone fees, fewer
manufacturing batches and fewer raw material purchases. This was partially offset by a $4.3 million increase in activity surrounding ongoing clinical trial efforts in the Rett syndrome REVEAL adult and pediatric studies.
General and Administrative (G&A) Expenses: General and administrative expenses were $8.6 million for the three months ended September 30,
2023, compared to $8.7 million for the three months ended September 30, 2022. The decrease of $0.1 million was due to reduced compensation expense due to lower headcount of $2.0 million and reduced consulting and professional
fees of $0.7 million, partially offset by $2.6 million issuance costs allocated to the liability-classified pre-funded warrants issued in connection with the private placement financing completed in
Net loss: Net loss for the three months ended September 30, 2023, was $117.1 million, or $0.93 per share, as compared to a
net loss of $26.5 million, or $0.65 per share, for the three months ended September 30, 2022, due to a non-cash expense of $100.5 million recorded in Q3 2023 from a change in the fair value of
warrant liability from pre-funded warrants in connection with the private placement financing completed in August 2023.
Cash and cash equivalents: As of September 30, 2023, the Company had cash and cash equivalents of $164.3 million. The Company expects that
its existing cash and cash equivalents will fund operating expenses and capital requirements into 2026.
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 (U.S./Canada) or 201-689-8263 (international). The conference ID for all callers is 13741244. The live webcast and replay may be accessed by visiting Taysha s website at
https://ir.tayshagtx.com/news-events/events-presentations. An archived version of the webcast will be available on the website for 30 days.
About Taysha Gene Therapies
Taysha Gene Therapies
(Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team s proven experience in gene
therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program. Together, we leverage our fully integrated platform with a goal of dramatically improving patients lives. More information is
available at 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 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 Securities and Exchange Commission ( SEC ) filings,
including in our Annual Report on Form 10-K for the full-year ended December 31, 2022, and our Quarterly Report on Form 10-Q for the quarter ended
September 30, 2023, both of which are 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 Balance Sheet Data
(in thousands, except share and per share data)
| September 30, 2023 | December 31, 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, $0.00001 par value per share; 10,000,000 shares authorized and no shares issued and outstanding as of September 30, 2023 and December 31, 2022 | ||||||||
| Common stock, $0.00001 par value per share; 200,000,000 shares authorized and 186,960,193 and 63,207,507 issued and outstanding as of September 30, 2023 and December 31, 2022, respectively | 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 |
Taysha Gene Therapies, Inc.
Condensed Consolidated Statement of Operations
(in thousands, except share and per share data)
| For the Three Months Ended September 30, | For the Nine Months Ended September 30, | |||||||||||||||
| 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 |
Director, Head of Corporate Communications and
Taysha Gene Therapies, Inc.
Canale Communications