Taysha Gene Therapies Announces Second Patient Dosed with TSHA-102 in the REVEAL Phase 1/2 Adult Trial for the Treatment of Rett Syndrome
September 26 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 the second Rett
syndrome patient has been dosed with TSHA-102 in the REVEAL Phase
1/2 adult trial in Canada.
“Dosing the second adult patient in the REVEAL Phase 1/2 adult
trial in Canada marks important progress in the ongoing clinical
evaluation of TSHA-102 for Rett syndrome,” said Sukumar Nagendran,
M.D., President, and Head of R&D of Taysha. “The enthusiasm for
a potential disease-modifying therapy among the Rett syndrome
community is encouraging, and we remain focused on further
evaluating the therapeutic potential of TSHA-102 in adults and
expanding the clinical evaluation to pediatric patients with this
devastating disease. We look forward to reporting initial clinical
data on the second adult patient and providing an update on the
first adult patient in the low-dose cohort at our quarterly
earnings conference call in mid-November, following the
pre-specified IDMC review.”
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 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.
Enrollment in the low-dose cohort is expected to be complete in the
fourth quarter of 2023 with the dosing of the third patient.
The REVEAL adult trial is being conducted at CHU Sainte-Justine,
the Université de Montréal mother and child university hospital
centre in Montreal, Canada, under Principal Investigator Dr. Elsa
Rossignol, M.D., FRCP, FAAP, Associate Professor Neuroscience and
Pediatrics at CHU Sainte-Justine.
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.
Additionally, the Company submitted a Clinical Trial Application to
the United Kingdom Medicines and Healthcare products Regulatory
Agency (MHRA) for TSHA-102 in pediatric patients with Rett syndrome
and expects to receive MHRA feedback in the second half of
2023.
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) platform 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 of TSHA-102, including
the reproducibility and durability of any favorable results
initially seen in our first patient dosed in the REVEAL trial, and
our other product candidates, 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
potential for these product candidates to receive regulatory
approval from the FDA or equivalent foreign regulatory agencies,
whether, if approved, these product candidates will be successfully
distributed and marketed and the potential market opportunity for
these product candidates and the potential benefits of Fast Track,
Orphan Drug and Rare Pediatric Disease designations for TSHA-102.
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 June 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. 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 Collins Director, Head
of Corporate CommunicationsTaysha Gene Therapies,
Inc.hcollins@tayshagtx.com
Media Contact:Carolyn HawleyCanale
Communicationscarolyn.hawley@canalecomm.com
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