Taysha Gene Therapies Announces Positive Recommendation from Independent Data Monitoring Committee of REVEAL Phase 1/2 Trial in Rett Syndrome
31 Julho 2023 - 9:00AM
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), announced today that the
Independent Data Monitoring Committee (IDMC) recommended the
continuation of the REVEAL Phase 1/2 trial and that dosing of the
second patient in the first cohort can proceed. The decision
follows a pre-specified IDMC review of initial clinical data from
the first patient dosed with TSHA-102 following the 42-day
evaluation period.
“We thank the IDMC members for their guidance and are pleased
with their recommendation to continue the REVEAL Phase 1/2 trial,”
said Sukumar Nagendran, M.D., President and Head of R&D of
Taysha. “This recommendation was based on the analysis of initial
clinical data from the first adult patient with Rett syndrome to
receive TSHA-102. A second patient is expected to be dosed in the
third quarter of this year. We are highly encouraged by the initial
clinical observations, which support the transformative potential
of TSHA-102 and mark important progress in our efforts to bring a
gene therapy to patients and families living with Rett syndrome. We
look forward to providing an initial clinical update on the first
patient at our second quarter corporate update conference call in
mid-August."
The REVEAL Phase 1/2 trial is a first-in-human, open-label,
randomized, dose-escalation and dose-expansion study evaluating the
safety and preliminary efficacy of TSHA-102 in adult females 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 will then be administered during dose expansion.
About TSHA-102TSHA-102 is an investigational
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 miRNA-Responsive
Auto-Regulatory Element (miRARE) platform designed to regulate
cellular MECP2 expression. TSHA-102 has received Orphan Drug and
Rare Pediatric Disease designations from the FDA and has been
granted Orphan Drug designation from the European Commission.
TSHA-102 is being evaluated in the first-in-human, open label,
randomized, dose escalation and dose-expansion REVEAL Phase 1/2
trial for adult female patients with Rett syndrome.
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 administration, timing, progress
and results of our preclinical studies and clinical trials of our
product candidates, including TSHA-102, including statements
regarding the dosing of additional patients in REVEAL Phase 1/2
trial, the timing of initiation and completion of studies or trials
and related preparatory work and the period during which the
results of the trials will become available, the potential of our
product candidates, including TSHA-120 and TSHA-102, 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, our plans for regulatory
applications submissions, the potential for these product
candidates to receive regulatory approval from the FDA or
equivalent foreign regulatory agencies, and whether, if approved,
these product candidates will be successfully distributed and
marketed and the potential market opportunity for these product
candidates. 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, 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. Such risks may be amplified by
the impacts of the COVID-19 pandemic. 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 CollinsDirector, Head
of Corporate CommunicationsTaysha Gene Therapies,
Inc.hcollins@tayshagtx.com
Media Contact:Carolyn HawleyCanale
Communicationscarolyn.hawley@canalecomm.com
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