Taysha Gene Therapies Announces Expanded Eligibility in REVEAL Phase 1/2 Adult Trial to Include Adolescent Rett Syndrome Patients
November 29 2023 - 8:00AM
Taysha Gene Therapies, Inc. (Nasdaq: TSHA), 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 announced that Health
Canada has authorized the protocol amendment to the ongoing REVEAL
Phase 1/2 adult trial evaluating TSHA-102 that expands eligibility
to include patients aged 12 and older with Rett syndrome.
“Following review of the initial clinical data from the first
two adult patients treated with TSHA-102 and Chemistry,
Manufacturing, and Controls (CMC) data, Health Canada has
authorized our protocol amendment to include adolescent patients
aged 12 years and older in the ongoing REVEAL Phase 1/2 adult
trial,” said Sukumar Nagendran, M.D., President, and Head of
R&D of Taysha. “Amending our protocol broadens the patient
population who can potentially benefit from TSHA-102. We look
forward to further advancing the clinical development of TSHA-102
and building on the encouraging data demonstrated in the first two
adult patients treated.”
Rumana Haque-Ahmed, Senior Vice President, Regulatory Affairs of
Taysha, added “Health Canada’s clearance of the protocol amendment
is an important milestone in our quest to develop a potentially
transformative treatment for all patients and families in the Rett
syndrome community. We look forward to future discussions with
Health Canada and other regulatory authorities as we execute on our
development plan to bring TSHA-102 to patients as safely and
expeditiously as possible.”
TSHA-102 is being evaluated in the REVEAL Phase 1/2 adult trial
in Canada, a first-in-human, open-label, randomized,
dose-escalation and dose-expansion study evaluating the safety and
preliminary efficacy of TSHA-102 in females aged 12 and older with
stage four 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 will then be administered
during dose expansion. Dosing of the third adult patient and
completion of dosing in cohort one (low dose) in the adult trial is
anticipated in the fourth quarter of 2023 or the first quarter of
2024.
The United States Food and Drug Administration (FDA) cleared the
Investigational New Drug (IND) application for TSHA-102 in
pediatric patients with Rett syndrome, and the Company expects to
dose the first pediatric patient in the first quarter of 2024.
About TSHA-102TSHA-102 is a self-complementary
intrathecally delivered AAV9 investigational gene transfer therapy
in clinical evaluation for Rett syndrome. 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 Fast
Track designation and Orphan Drug and Rare Pediatric Disease
designations from the FDA and has been granted Orphan Drug
designation from the European Commission.
About Rett SyndromeRett syndrome is a rare
neurodevelopmental disorder caused by mutations in the X-linked
MECP2 gene, which is a gene that’s essential for neuronal and
synaptic function in the brain. The disorder is characterized by
intellectual disabilities, loss of communication, seizures, slowing
and/or regression of development, motor and respiratory impairment,
and shortened life expectancy. 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 UK.
About Taysha Gene TherapiesTaysha 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 StatementsThis 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 benefits and clinical
development of TSHA-102, including the timing of dosing patients in
clinical trials and availability of data from clinical trials.
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.
Company Contact:Hayleigh Collins Director, Head
of Corporate Communications and Investor RelationsTaysha Gene
Therapies, Inc.hcollins@tayshagtx.com
Media Contact:Carolyn HawleyCanale
Communicationscarolyn.hawley@canalecomm.com
Taysha Gene Therapies (NASDAQ:TSHA)
Historical Stock Chart
From Jun 2024 to Jul 2024
Taysha Gene Therapies (NASDAQ:TSHA)
Historical Stock Chart
From Jul 2023 to Jul 2024