Seelos Therapeutics Receives FDA May Proceed Notice to Initiate a Phase IIb/III Trial of SLS-005 in Amyotrophic Lateral Sclerosis

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
[email protected] 
https://seelostherapeutics.com/ 
https://twitter.com/seelostx 
https://www.linkedin.com/company/seelos

SOURCE Seelos Therapeutics, Inc.

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