Taysha Gene Therapies Announces Publication of Natural History Data for TSHA-120 in Giant Axonal Neuropathy in the Journal, B...
June 21 2021 - 7:00AM
Business Wire
Largest cohort of genetically confirmed
patients with GAN, including patients with the classic
(early-onset) and milder (late-onset) forms of GAN
Largest cross-sectional analysis highlighted
clinical differences in patients with early-onset GAN versus
late-onset GAN based on MFM32 performance as well as other
functional motor scales and disease markers
Robust assessment across clinical outcomes for
GAN, including motor, sensory, respiratory, neurophysiologic, MRI
and biopsy data
First clinical study to evaluate a cohort of
individuals with GAN for autonomic impairment
On track to report clinical data for TSHA-120
from the 3.5x1014 total vg dose cohort in the second half of
2021
Planning to engage with major regulatory
agencies to discuss the approval pathway and expect to provide a
regulatory update by year-end 2021
Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric,
pivotal-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) in both rare
and large patient populations, today announced the publication of
new analyses of natural history data for TSHA-120 in giant axonal
neuropathy, or GAN. The data were published online and will be
included in the June edition of Brain, a highly esteemed
neurological science peer-reviewed journal.
GAN is a progressive neurodegenerative disease that affects both
the central and peripheral nervous systems. The disease is caused
by loss-of-function mutations in the gene coding for gigaxonin,
which results in dysregulation of intermediate filament turnover,
an important structural component of the cell. Although no symptoms
are present in the first few months of life, many children with GAN
do show early symptoms and features before the age of five,
including unsteady gait, frequent falls, and motor weakness.
Symptoms worsen over time and children develop scoliosis,
contractures, atrophy of the spinal cord and abnormalities of the
white matter in the brain. Currently, there are no approved
treatments for GAN, which results in death for patients in their
late teens or early twenties.
In this natural history study, 45 patients, age 3 years to 21
years old, with genetically confirmed GAN were enrolled at NIH and
evaluated at their first enrollment visit. The objective of the
cross-sectional analysis was to identify genetic variants, explore
correlations between genotype and phenotype, identify reliable
markers of disease severity and assess how these markers correlate
with ambulatory function and the impact of the early- and
late-onset phenotypes on these markers.
The two sub cohorts of GAN patients in the study included
thirty-five patients with early-onset GAN and 10 patients with
late-onset GAN. In the early-onset cohort, the mean age of onset of
gait or motor impairment was 2.3 years old whereas the mean age of
onset of symptoms in the late-onset cohort was 5.4 years old. Motor
Function Measure 32 (MFM32), a validated and well-known scale to
measure strength and motor function had the strongest correlation
across outcome measures and age in patients with GAN. Patients with
late-onset GAN had better functional performance compared to
similarly aged patients with early-onset GAN. Ambulatory ability
between the two phenotypes also differed. Disease progression in
early-onset GAN patients occurred in a uniform and homogenous
manner. Autonomic manifestations of the disease did not correlate
with age or motor function.
“The recent publication in Brain serves as the baseline data for
a longitudinal natural history assessment and adds important
context to results from three dose cohorts in the ongoing clinical
GAN trial,” said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM,
Chief Medical Officer and Head of Research and Development of
Taysha. “These results also confirm our findings that there is a
clinical difference between early-onset GAN, a relentlessly
progressive and fatal neuropathy, and late-onset GAN, which has
significant disease morbidity, and underscores the importance for
GAN to be included in genetic screens for hereditary neuropathies.
The estimated prevalence for GAN is 2,400 patients but the GAN
population may be larger than previously appreciated. We view
genetic testing as an important aspect of patient identification
and look forward to leveraging our key collaborations with
companies to increase patient diagnostic efforts and allow for
earlier intervention. Of note, data from today’s publication
provide further support and confidence in the overall clinical
program design for TSHA-120 which include this ongoing,
prospective, natural history and outcomes assessment study, and an
interventional dose selection safety and efficacy study. Building
on the previously presented and favorable interventional study
data, we remain on track to report clinical data from the highest
dose cohort in the interventional clinical trial for TSHA-120 in
the second half of this year. We look forward to engaging with
major regulatory agencies to discuss the approval pathway for
TSHA-120 and to provide a regulatory update by year-end.”
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 to build an extensive, AAV gene therapy pipeline focused on
both rare and large-market indications. Together, we leverage our
fully integrated platform—an engine for potential new cures—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,” 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 TSHA-120, 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, TSHA-120’s eligibility for accelerated
approval in the United States and Europe, 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, 2020, 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.
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Company Contact: Kimberly Lee, D.O. SVP, Corporate
Communications and Investor Relations Taysha Gene Therapies
klee@tayshagtx.com
Media Contact: Carolyn Hawley Canale Communications
carolyn.hawley@canalecomm.com
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