Program invented in the lab of Dr. Steven Gray,
Taysha’s Chief Scientific Advisor, immediately transforms Taysha
into a sustainable pivotal-stage gene therapy company
Clinical and preclinical data package validates
the scientific approach of Dr. Steven Gray, UT Southwestern, and
Taysha, with readthrough to existing portfolio
Groundbreaking clinical trial run by the NIH is
the first intrathecally dosed gene therapy program in history
Human proof-of-concept data for TSHA-120
demonstrated clear arrest of disease progression and long-term
durability at therapeutic dose levels in patients with giant axonal
neuropathy
Plans to engage with regulatory agencies in the
United States, Europe and Japan as soon as possible
Estimated 2,400 patients in U.S. and Europe
represent potentially greater than $2 billion near-term commercial
opportunity
Program provides basis for accelerating
build-out of commercial infrastructure to support patient
identification, payor engagement and product distribution
Conference call and webcast today at 8:00 AM
Eastern Time
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 acquisition of
exclusive worldwide rights to a clinical-stage AAV9 gene therapy
program, now known as TSHA-120, for the treatment of giant axonal
neuropathy (GAN). TSHA-120 is an intrathecally dosed AAV9 gene
therapy currently being evaluated in a clinical trial for the
treatment of GAN. The trial is being conducted by the National
Institutes of Health (NIH) in close collaboration with a leading
patient advocacy group focused on finding treatments and cures for
GAN. TSHA-120 has received rare pediatric disease and orphan drug
designations from the U.S. Food and Drug Administration (FDA) for
the treatment of GAN.
GAN is a rare inherited genetic disorder that affects both the
central and peripheral nervous systems and is caused by
loss-of-function mutations in the gene coding for gigaxonin. Many
children with GAN show symptoms and features before the age of
five, including progressive scoliosis, contractures, atrophy of the
spinal cord, giant axons – also known as nerve fibers – and
abnormalities of the white matter in the brain. Currently, there
are no approved treatments for GAN, which often results in death
for patients in their late teens or early twenties. TSHA-120 was
originally developed in the laboratory of Taysha’s Chief Scientific
Advisor, Dr. Steven Gray, and advanced in a historic ongoing
clinical trial by the NIH as the first intrathecally dosed gene
therapy study.
“Dr. Steven Gray’s work on the GAN program was the catalyst for
all the other translational research initiatives in his lab and we
are very pleased to continue this important and meaningful work
that has had a significant impact across the entire gene therapy
landscape. As the program that laid the foundation for our robust
pipeline, we believe that TSHA-120 is a seamless strategic fit and
will be immediately value-accretive for Taysha. TSHA-120 clinical
data generated to date is a clear validation of our scientific
approach with read-through to our existing product development
pipeline. Consistent with all of our gene therapy candidates,
TSHA-120 targets a monogenic CNS disease, is delivered
intrathecally using a proven AAV9 vector, and utilizes a highly
scalable HEK293 suspension manufacturing process. Collectively,
these key parallels enable Taysha to leverage synergies across its
core competencies to efficiently develop and commercialize
TSHA-120,” said RA Session II, President, Founder and Chief
Executive Officer of Taysha. “The efficacy data in preclinical
studies with the GAN knockout rodent model were extremely
compelling, and TSHA-120 demonstrated significant improvement in
pathology across a range of tissues, and notably improved the
pathological appearance of the dorsal root ganglia, which is a key
component of disease progression. We look forward to quickly
working with the regulatory agencies on a path forward to approval
of TSHA-120, and in parallel, accelerating the build-out of our
commercial infrastructure to support patient identification, payor
engagement and product distribution. TSHA-120 has the potential to
address a significant unmet need for an estimated 2,400 patients
with GAN across the United States and in Europe, potentially
representing a near-term commercial opportunity of greater than $2
billion.”
The National Institute of Neurological Disorders and Stroke
(NINDS) division of the NIH is conducting the ongoing open-label,
non-randomized, dose-escalation clinical trial of TSHA-120 for the
treatment of GAN. The primary endpoint is safety, with secondary
endpoints measuring efficacy using pathologic, physiologic,
functional, and clinical markers. A primary measure of clinical
efficacy is the Motor Function Measure 32 (MFM32) score, a
quantitative scale designed to assess the severity and progression
of motor function abilities. There is precedent for its use in
multiple clinical studies for neuromuscular diseases, including
spinal muscular atrophy amongst others. To date, 14 patients have
been dosed with one of four dose levels of TSHA-120. TSHA-120 has
demonstrated a dose-response relationship with arrest of disease
progression at the second-highest dose level (1.8x1014 total vector
genomes [vg]) at one-year post-treatment, affecting a statistically
significant 8-point improvement on the MFM32 score. A 4-point
change on the MFM32 score is considered clinically meaningful. Six
of these patients treated at therapeutic dose levels have shown
sustained dose-dependent improvements in MFM32 scores for more than
three years. Long-term results demonstrated that treatment with
TSHA-120 at multiple dose levels was well-tolerated with no severe
drug-related adverse events. Additional data are expected later
this year, including results from the highest dose cohort (3.5x1014
total vg).
“TSHA-120 is the first successful in-human intrathecal gene
transfer in the history of gene therapy and, as such, has had a
significant impact across the field. This program further supports
our approach to treating monogenic diseases of the CNS and may
enable us to pursue proof-of-concept for our redosing platform,”
said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical
Officer and Head of Research and Development of Taysha. “We are
very encouraged by TSHA-120’s halting effect on disease progression
at therapeutic dose levels and long-term durability of effect in
patients living with GAN, and we look forward to highlighting the
initial clinical data in an R&D Day in June 2021. In the
meantime, we plan to engage regulatory agencies in the United
States, Europe and Japan as soon as possible.”
Before the end of the year, Taysha intends to request an
End-of-Phase meeting with the FDA and engage with the European
Medicines Agency (EMA) and the Pharmaceuticals and Medical Devices
Agency (PMDA) in Japan to discuss the regulatory pathway for
TSHA-120. Taysha expects to provide a regulatory and clinical
update on TSHA-120, including data from the 3.5x1014 total vg
cohort by year-end.
Under the terms of the agreement, in exchange for granting
Taysha the exclusive worldwide rights to TSHA-120, the leading GAN
patient advocacy group will receive an upfront payment of $5.5
million and will be eligible to receive clinical, regulatory and
commercial milestones totaling up to $19.3 million, as well as a
low, single-digit royalty on net sales upon commercialization of
the product.
Conference Call and Webcast Information
Taysha management will hold a conference call and webcast today
at 8:00 am ET / 7:00 am CT to review its acquisition of the GAN
program. 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 13718632. 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 60 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 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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version on businesswire.com: https://www.businesswire.com/news/home/20210412005343/en/
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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