NEW YORK, Aug. 10, 2020 /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 that, on August 7, 2020, it was notified by the Food and
Drug Administration (FDA) that Seelos may proceed with initiating a
Phase IIb/III trial studying SLS-005 (trehalose) for the treatment
of Amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).
Mutations in the C9orf72, SOD1, FUS, and TARDBP genes can cause
familial ALS and contribute to the development of sporadic ALS.
These mutations contribute to the death of motor neurons and ALS
affected motor neurons develop a buildup of protein aggregates such
as TDP-43 and SOD1. In in-vivo studies of ALS, trehalose has been
shown to increase clearance of TDP-43, decrease SOD1 and SQSM1/p62
aggregates and monomers, delay the progression of the disease,
preserve ventral horn motor neurons and increase muscle fiber
size.
"ALS is a debilitating disease which
currently lacks a cure and there is significant evidence suggestive
of trehalose having the potential to alter or slow the progression
of ALS," said Raj Mehra Ph.D., Chairman and CEO of Seelos.
"Receiving the FDA notice that we may begin a registrational Phase
IIb/III study is a transformative event for Seelos and our hope is
that SLS-005 can offer a potential option for patients. The FDA
signoff to begin this pivotal study allows Seelos to focus on ALS
as the lead indication for SLS-005. We remain committed to our
work in additional indications as well."
"Several preclinical studies have demonstrated the potential of
trehalose as a treatment for ALS, demonstrating preservation of
motor neurons, motor function and prolonged survival. We are excited to start our clinical program for
this devastating disease," said Warren
W. Wasiewski, M.D., F.A.A.P., Chief Medical Officer of
Seelos.
Seelos' Phase IIb/III trial plans to enroll 160 patients with
either familial or sporadic ALS in a double-blind
placebo-controlled trial. Patients will be randomized 3:1 (drug:placebo) and studied with a
primary endpoint measuring change from baseline on Revised
Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
score at 24 weeks. Secondary endpoints will also be measured at 24
weeks, including change from baseline in slow vital capacity,
muscle strength, quality of life measurements as well as additional
signs of disease progression.
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.
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
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 potential for trehalose to alter or
slow the progression of ALS, the focus on ALS as the lead
indication for SLS-005, other potential indications for SLS-005,
the expected number of patients to be enrolled in the Phase IIb/III
trial and other statements relating to the expected design and
endpoints for the trial. 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 of not successfully executing its preclinical and
clinical studies and not gaining marketing approvals for its
product candidates, the risk that prior test results may not be
replicated in future studies and trials, the risks that clinical
study results may not meet any or all endpoints of a clinical study
and that any data generated from such studies may not support a
regulatory submission or approval, the risks associated with the
implementation of a new business strategy, the risks related to
raising capital to fund its 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 Ave., 12th Fl
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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