NEW YORK, Nov. 8, 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, announced today that the U.S. Food and
Drug Administration (FDA) has accepted Seelos' Investigation New
Drug (IND) application to study SLS-005 (trehalose injection, 90.5
mg/mL for intravenous infusion) for the treatment of
spinocerebellar ataxia (SCA). The FDA has also granted the program
Fast Track designation in the U.S. for SCA, and SLS-005 has
previously received Orphan Drug designation for spinocerebellar
ataxia type 3 (SCA3) from the FDA and from the European Medicines
Agency in the EU.
![(PRNewsfoto/Seelos Therapeutics, Inc.) (PRNewsfoto/Seelos Therapeutics, Inc.)](https://mma.prnewswire.com/media/1244961/Seelos_Therapeutics_Logo.jpg)
"SCA is a highly debilitating neurodegenerative disease that
currently lacks a cure or an approved therapeutic and as such,
patients manage symptoms through physical therapy and other
symptomatic treatments," said Raj Mehra, Ph.D., Chairman and CEO of
Seelos. "SLS-005 has already displayed encouraging open label human
data in SCA3, the most common type of SCA, and our team has taken
that experience and knowledge into the design and plans for our
global Phase IIb/III placebo-controlled study. We look forward to
initiating this study in early 2022 and our recent capital raises
have accounted for the expected development costs for initiating
this study."
Prior to Seelos acquiring the program, SLS-005 had already been
studied in a six-month open label Phase IIa study that also
included an additional six-month follow up in patients with SCA3,
also known as Machado-Joseph Disease (Zaltzman 2020). The open
label study evaluated 14 patients with SCA3 over a six-month period
and found the average score on the Scale for Assessment and Rating
of Ataxia (SARA), a well-recognized clinical tool for measuring
functional impairment associated with the disease, remained stable.
Six patients received treatment for as long as 12 months and
continued to maintain stable SARA scores. In comparison, natural
history data suggests that individuals with SCA3 would be expected
to show a measurable increase on SARA within a 12-month period,
which is indicative of disease progression and worsening of
symptoms.
Additionally, Seelos was named as one of the initial members of
the National Ataxia Foundation (NAF) Drug Development
Collaborative, an industry consortium that has the principal goal
of accelerating the development of treatments for Ataxia.
"On behalf of the National Ataxia Foundation and the ataxia
community we represent, we're thrilled that Seelos Therapeutics has
received both IND acceptance and Fast Track designation from
the FDA," said Andrew Rosen, NAF
Executive Director. "Seelos is a founding member of
the NAF Drug Development Collaborative and NAF looks forward
to working closely with Seelos to support the company as its
program progresses."
About Spinocerebellar Ataxia
Spinocerebellar Ataxia is a serious disease caused by
degeneration of the cerebellum with an onset usually in adult
life. Clinically, it is characterized by progressive
unsteadiness of gait and stance, impaired coordination of limb
movements, slurred speech, and abnormal eye movements.
Spinocerebellar ataxia type 3, also known as Machado-Joseph disease
(MJD), is characterized by progressive cerebellar ataxia and is
known to cause progressively severe disability and often premature
death approximately 10-20 years from onset of symptoms.
About the National Ataxia Foundation (NAF) and the NAF Drug
Development Collaborative
NAF is a nonprofit organization established in 1957 to help
persons with ataxia and their families. The Foundation's mission is
to accelerate the development of treatments and a cure while
working to improve the lives of those affected by ataxia. NAF is
the only organization in the United
States dedicated to the disease that serves all types of
ataxias. NAF works closely with the world's leading ataxia
researchers, promoting exchanges of ideas and innovation in ataxia
discovery.
The NAF Drug Development Collaborative provides a centralized
source for access to resources needed to support research and
development of ataxia therapies. Members will benefit from the
integration of the patient experience with sound ataxia scientific
and clinical expertise. NAF brings more than 60 years of experience
in supporting patients and caregivers and connecting them with
research and clinical trial opportunities. NAF has also funded a
network of ataxia clinicians at sites around the US that will be a
critical component of the Collaborative's work. Specific objectives
of the Collaborative include natural history and biosample data
collection, development of biomarkers, validation of rating scales,
clinical trial design, patient-reported outcomes, and other data
necessary for the development and approval of safe and effective
therapies.
For more information on NAF: https://www.ataxia.org/
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 several animal models of 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 Seelos Therapeutics
Seelos Therapeutics, Inc. is a clinical-stage biopharmaceutical
company focused on the development and advancement of novel
therapeutics to address unmet medical needs for the benefit of
patients with central nervous system (CNS) disorders and other rare
diseases. The Company's robust portfolio includes several
late-stage clinical assets targeting indications including Acute
Suicidal Ideation and Behavior (ASIB) in Major Depressive Disorder
(MDD) or Post-Traumatic Stress Disorder (PTSD), Amyotrophic
lateral sclerosis (ALS), Spinocerebellar ataxia (SCA), Sanfilippo
syndrome, Parkinson's disease, other psychiatric and movement
disorders plus orphan diseases.
For more information, please visit our
website: http://seelostherapeutics.com, the content of which
is not incorporated herein by reference.
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 design and plans for the global Phase
IIB/III placebo-controlled study of SLS-005 in SCA (the "Study"),
the expected timeline for commencing the Study and Seelos'
available capital and expected development costs to initiate the
Study. These statements are based on Seelos' current expectations
and beliefs and are subject to a number of risks and uncertainties
that could cause actual results to differ materially from those
described in the forward-looking statements. Risks associated with
Seelos' business and plans described herein include, but are not
limited to, the risk of not successfully executing its preclinical
and clinical studies, or initiating the Study, and not gaining
marketing approvals for its product candidates, the risk that prior
clinical results may not be replicated in future studies and trials
(including the risk that the results from the prior studies of
SLS-005 may not be replicated or may be materially different from
the results of the Study or other future trials and studies of
SLS-005), 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 most recent 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.
_____________________
Zaltzman R, Elyoseph Z, Lev N, Gordon CR. Trehalose in
Machado-Joseph Disease: Safety, Tolerability, and Efficacy.
Cerebellum. 2020 Oct; 19(5):672-679. doi:
10.1007/s12311-020-01150-6. PMID: 32514820.
Contact Information
Anthony
Marciano
Chief Communications Officer
Seelos Therapeutics, Inc. (Nasdaq: SEEL)
300 Park Avenue
New York, NY 10022
(646) 293-2136
anthony.marciano@seelostx.com
www.seelostherapeutics.com
https://twitter.com/seelostx
https://www.linkedin.com/company/seelos
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