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.
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-102TSHA-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 SyndromeRett 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-120TSHA-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 TayshaTaysha 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 AstellasAstellas 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
TherapiesAstellas 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 NotesIn
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.
Contacts for inquiries or additional
information:
Astellas Pharma Inc.
Portfolio CommunicationsCassie
Hogenkamp+1-847-942-0980cassie.hogenkamp@astellas.com
Corporate Advocacy &
Relations+81-3-3244-3201
Taysha Gene Therapies, Inc.
Company Contact:Kimberly Lee, D.O.Chief Corporate
Affairs OfficerTaysha Gene Therapiesklee@tayshagtx.com
Media Contact:Carolyn HawleyCanale
Evokecarolyn.hawley@evokegroup.com
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