NEW YORK, May 27, 2021 /PRNewswire/ -- Seelos
Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage
biopharmaceutical company focused on the development of therapies
for central nervous system disorders and rare diseases, today
announced it has received European Orphan Drug Designation for
SLS-005 in amyotrophic lateral sclerosis (ALS) from the European
Medicines Agency (EMA) Committee for Orphan Medicinal Products
(COMP).
Under orphan designation in the European Union (EU), Seelos
stands to benefit from several incentives such as protocol
assistance, reduced regulatory fees and market exclusivity.
European guidelines for Orphan Drug Designation are for diseases
affecting not more than five in 10,000 people in the EU.
In November, SLS-005 was granted Orphan Drug Designation from
the U.S. Food and Drug Administration (FDA) for ALS. SLS-005 was
previously granted Orphan Drug Designation from the FDA and EMA for
Spinocerebellar Ataxia Type 3 (SCA3), Sanfilippo syndrome and
Oculopharyngeal Muscular Dystrophy (OPMD). SLS-005 has also been
granted Fast Track designation from the FDA for OPMD.
About Amyotrophic Lateral Sclerosis (ALS)
According to the National Institute of Neurological Disorders
and Stroke, amyotrophic lateral sclerosis (ALS) is a group of rare
neurological diseases that mainly involve the nerve cells (neurons)
responsible for controlling voluntary muscle movement. In ALS, both
the upper motor neurons and the lower motor neurons degenerate or
die and stop sending messages to the muscles. Unable to function,
the muscles gradually weaken, start to twitch (called
fasciculations), and waste away (atrophy). Eventually, the brain
loses its ability to initiate and control voluntary movements. The
disease is progressive, meaning the symptoms get worse over time.
The majority of ALS cases (90 percent or more) are considered
sporadic. This means the disease seems to occur at random with no
clearly associated risk factors and no family history of the
disease. Although family members of people with sporadic ALS are at
an increased risk for the disease, the overall risk is very low,
and most will not develop ALS.
Most people with ALS die from respiratory failure, usually
within 3 to 5 years from when the symptoms first appear. However,
about 10 percent of people with ALS survive for 10 or more years.
Currently, there is no cure for ALS and no effective treatment to
halt or reverse the progression of the disease.
About Trehalose
Trehalose is a low molecular weight disaccharide (0.342 kDa)
that crosses the blood brain barrier, stabilizes proteins and,
importantly, activates autophagy, which is the process that clears
material from cells. In animal models of several diseases
associated with abnormal cellular protein aggregation or storage of
pathologic material, it has been shown to reduce aggregation of
misfolded proteins and reduce accumulation of pathologic material.
Trehalose activates autophagy through the activation of
Transcription Factor EB (TFEB), a key factor in lysosomal and
autophagy gene expression. Activation of TFEB is an emerging
therapeutic target for a number of diseases with pathologic
accumulation of storage material.
Forward Looking Statements
Statements made in this press release, which are not
historical in nature, constitute forward-looking statements for
purposes of the safe harbor provided by the Private Securities
Litigation Reform Act of 1995. These statements include, among
others, those regarding the incentives that Seelos expects to
receive in connection with receiving Orphan Drug Designation for
SLS-005. These statements are based on Seelos' current expectations
and beliefs and are subject to a number of factors and
uncertainties that could cause actual results to differ materially
from those described in the forward-looking statements. Risks
associated with Seelos' business include, but are not limited to,
the risk that Seelos does not receive the incentives associated
with receiving Orphan Drug Designation for SLS-005, the risk of
Seelos not successfully executing its preclinical and clinical
studies and not gaining marketing approvals for its product
candidates, the risks associated with the implementation of a new
business strategy, the risks related to raising capital to fund
Seelos' development plans and ongoing operations, risks related to
Seelos' current stock price, risks related to the global impact of
COVID-19, as well as other factors expressed in Seelos' periodic
filings with the U.S. Securities and Exchange Commission, including
its Annual Report on Form 10-K and Quarterly Reports on Form
10-Q. Although we believe that the expectations reflected in
our forward-looking statements are reasonable, we do not know
whether our expectations will prove correct. You are cautioned not
to place undue reliance on these forward-looking statements, which
speak only as of the date hereof, even if subsequently made
available by us on our website or otherwise. We do not undertake
any obligation to update, amend or clarify these forward-looking
statements, whether as a result of new information, future events
or otherwise, except as may be required under applicable securities
laws.
Contact Information:
Anthony Marciano
Head of Corporate Communications
Seelos Therapeutics, Inc. (Nasdaq: SEEL)
300 Park Avenue
New York, NY 10022
(646) 293-2136
anthony.marciano@seelostx.com
https://seelostherapeutics.com/
https://twitter.com/seelostx
https://www.linkedin.com/company/seelos
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