- Taysha Gene Therapies is an emerging leader
in the development of AAV gene therapies; new collaboration aimed
at enhancing development of two of Taysha's novel product
candidates for rare monogenic central nervous system
diseases with serious unmet medical needs -
- Astellas to invest a total of $50 million to acquire 15% of the company and to
receive an exclusive option to obtain an exclusive license for
TSHA-102 for Rett syndrome and TSHA-120 for giant
axonal neuropathy (GAN) -
- Astellas to receive certain rights related
to any potential change of control of Taysha -
- Astellas to receive one Board observer seat
on the Taysha Board of Directors -
TOKYO and DALLAS, Oct. 24,
2022 /PRNewswire/ -- Astellas Pharma Inc. (TSE:
4503, President and CEO: Kenji
Yasukawa, Ph.D., "Astellas") and Taysha Gene Therapies, Inc.
(NASDAQ: TSHA, CEO: RA Session II, "Taysha") today announced a
strategic investment to support the advancement of Taysha's
adeno-associated virus (AAV) gene therapy development programs for
the treatment of Rett syndrome and GAN. The future options to
potentially apply Astellas' global R&D, manufacturing and
commercialization capabilities in gene therapy to Taysha's
innovative AAV gene therapy development programs for genetic
diseases of the central nervous system (CNS) create the opportunity
for the two companies to enhance the development of novel treatment
options for patients with Rett syndrome and GAN, who have serious
unmet medical needs.
![Astellas is excited to announce a new strategic investment to support development of Taysha Gene Therapies, Inc.’s AAV-based gene therapy programs. Astellas is excited to announce a new strategic investment to support development of Taysha Gene Therapies, Inc.’s AAV-based gene therapy programs.](https://mma.prnewswire.com/media/1927550/Taysha_x_Astellas_Logo.jpg)
Under the terms of the agreement, Astellas will invest a total
of $50 million to acquire 15% of the
outstanding common stock of Taysha and to receive an exclusive
option to license two of Taysha's clinical stage programs: TSHA-102
for Rett syndrome and TSHA-120 for GAN. In addition, Taysha
has granted Astellas certain rights related to any potential change
of control of Taysha. Definitive agreements would be executed upon
Astellas' exercise of any such option, and any change of control
transaction would require approval by Taysha's stockholders.
Taysha is engaged in the development of intrathecally-delivered
AAV gene therapies for monogenic CNS diseases. As a part of this
platform approach, Taysha has a promising pipeline, including
TSHA-102, which is the first-and-only gene therapy in clinical
development for Rett syndrome, and TSHA-120, which is in Phase 1/2
development for the treatment of GAN and awaiting regulatory
feedback.
Astellas is continuing to build its capability to bring novel
gene therapies to patients, following the acquisition of Audentes
(now Astellas Gene Therapies, California) in January
2020 and the construction of a
state-of-the-art commercial GMP manufacturing facility in
North Carolina, which was opened
in June of this year.
"Gene therapy is the corner stone of Astellas' Primary Focus,
Genetic Regulation*1; our goal is to bring new
transformative treatment options to patients living with serious
genetic diseases and limited treatment options," said Naoki Okamura, Chief Strategy Officer, at
Astellas. "Taysha is an industry leader in CNS gene therapies and
this partnership fits strategically with our long-term vision of
expanding Astellas' gene therapy capabilities, allowing the company
to impact the lives of a broader range of patients with urgent
unmet medical needs."
"We are excited to enter this strategic investment with
Astellas, a premier biopharmaceutical company with global R&D,
manufacturing and commercial capabilities," said RA Session II,
Taysha's Chief Executive Officer. "We believe this investment not
only further validates the potential of our technology platform,
but also reinforces the therapeutic and market opportunity of our
two lead clinical assets."
To further strategically align Astellas and Taysha, in
connection with its equity investment, Astellas will receive one
Board observer seat on Taysha's Board of Directors, enabling Taysha
to leverage Astellas' gene therapy clinical and commercial
expertise as Taysha advances TSHA-120 and TSHA-102.
*1: Astellas has established a Focus Area Approach for its
research and development strategy. For more information, please
visit our website
at https://www.astellas.com/en/science/focus-area-approach.
About TSHA-102
TSHA-102 is a self-complementary
intrathecally delivered AAV9 gene replacement therapy under
development for the treatment of Rett syndrome. TSHA-102 utilizes
the novel miRNA-Responsive Auto-Regulatory Element (miRARE)
platform to regulate transgene expression genotypically on a
cell-by-cell basis. The miRARE technology is designed to prevent
toxicity associated with transgene overexpression and can be
potentially utilized across other indications. TSHA-102 has
received Orphan Drug and Rare Pediatric Disease designations from
the U.S. Food and Drug Administration (FDA) and Orphan Drug
Designation from the European Commission.
About Rett Syndrome
Rett syndrome is a severe genetic
neurodevelopmental disorder caused by a mutation in the X-linked
MECP2 gene essential for neuronal and synaptic function in
the brain. Primarily occurring in females, Rett syndrome is one of
the most common genetic causes of severe intellectual disability
worldwide. Patients have normal early development, with symptom
onset typically beginning between 6 to 18 months of age. Rett
syndrome is characterized by rapid developmental regression that
leads to intellectual disabilities, loss of speech, loss of
purposeful use of hands, loss of mobility, seizures, cardiac
impairments and breathing issues. Currently, there are no approved
therapies that treat the underlying cause of this progressive
disease.
About TSHA-120
TSHA-120, an intrathecally dosed AAV9
gene replacement therapy delivering the gene gigaxonin for
the treatment of GAN, is currently being evaluated in an ongoing
Phase 1/2 clinical trial. TSHA-120 has received Orphan Drug and
Rare Pediatric Disease designations from FDA and Orphan Drug
Designation from the European Commission.
About Giant Axonal Neuropathy (GAN)
GAN is rare
inherited genetic disorder that 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. Children with GAN present before the age of five with
symptoms including unsteady gait, frequent falls, and motor
weakness. Currently, there are no approved treatments for GAN,
which results in death for patients in their late teens or early
twenties.
About Taysha
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 (Taysha)
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 in GAN and TSHA-102 in Rett syndrome, to
positively impact quality of life and alter the course of disease
in the patients we seek to treat, the potential benefits of
Taysha's collaboration with Astellas, the potential for Astellas to
exercise any of the options granted to it by Taysha, 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,
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, 2021, and our
Quarterly Report on Form 10-Q for the quarter ended June 30,
2022, 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.
About Astellas
Astellas Pharma Inc. is a
pharmaceutical company conducting business in more than 70
countries around the world. We are promoting the Focus Area
Approach that is designed to identify opportunities for the
continuous creation of new drugs to address diseases with high
unmet medical needs by focusing on Biology and Modality.
Furthermore, we are also looking beyond our foundational Rx focus
to create Rx+® healthcare solutions that combine our
expertise and knowledge with cutting-edge technology in different
fields of external partners. Through these efforts, Astellas stands
on the forefront of healthcare change to turn innovative science
into value for patients. For more information, please visit our
website at https://www.astellas.com/en.
About Astellas Gene Therapies
Astellas Gene Therapies
is an Astellas Center of Excellence developing genetic medicines
with the potential to deliver transformative value for patients.
Our gene therapy drug discovery engine is built around innovative
science, a validated AAV platform, and industry leading internal
manufacturing capability with a particular focus on rare diseases
of the eye, CNS and neuromuscular system. Astellas Gene Therapies
will also be advancing additional Astellas gene
therapy programs toward clinical investigation. Astellas Gene
Therapies is based in San
Francisco, with manufacturing and laboratory facilities in
South San Francisco, Calif., and
Sanford, N.C.
Astellas Cautionary Notes
In this press release,
statements made with respect to current plans, estimates,
strategies and beliefs and other statements that are not historical
facts are forward-looking statements about the future performance
of Astellas. These statements are based on management's current
assumptions and beliefs in light of the information currently
available to it and involve known and unknown risks and
uncertainties. A number of factors could cause actual results to
differ materially from those discussed in the forward-looking
statements. Such factors include, but are not limited to: (i)
changes in general economic conditions and in laws and regulations,
relating to pharmaceutical markets, (ii) currency exchange rate
fluctuations, (iii) delays in new product launches, (iv) the
inability of Astellas to market existing and new products
effectively, (v) the inability of Astellas to continue to
effectively research and develop products accepted by customers in
highly competitive markets, and (vi) infringements of
Astellas' intellectual property rights by third parties.
Information about pharmaceutical products (including products
currently in development) which is included in this press release
is not intended to constitute an advertisement or medical
advice.
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