NEW YORK, Dec. 15, 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 the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital has selected Seelos' Phase IIb/III study of SLS-005 (trehalose) to be included in the HEALEY ALS Platform Trial, the first ever platform trial for the treatment of amyotrophic lateral sclerosis (ALS or Lou Gehrig's disease).
"The inclusion of the registrational phase IIb/III study of SLS-005 in this first ever platform trial is the result of extensive work identifying trehalose as a potential treatment to study in ALS," said Raj Mehra Ph.D., Chairman and CEO of Seelos. "The Healey Center's recognition of this potential therapy is a major validation and being part of this platform trial should help to expedite the trial by helping to provide greater access to patients."
"We are excited to work with Seelos and look forward to studying SLS-005 in an accelerated format through the HEALEY ALS Platform Trial," added Merit Cudkowicz, MD, director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, Chief of the Department of Neurology, and Principal Investigator of the HEALEY ALS Platform Trial. "The design team will work closely with Seelos on their regimen specific protocol as well as completing required steps with the central ethics review board and the FDA."
The HEALEY ALS Platform trial is designed to study multiple potential treatments for ALS simultaneously and expedite the timing from identification of a potential therapy to its testing. The platform trial model, successfully utilized in oncology, aims to greatly accelerate the study of multiple therapies, allowing investigators to test more potential therapies, increase patient access, reduce costs, and shorten development timelines.
SLS-005 was recently granted Orphan Drug Designation for SLS-005 for the treatment of ALS from U.S. Food and Drug Administration (FDA).
The HEALEY ALS Platform Trial's investigational new drug application (IND) was approved by the FDA in January 2020 and is a collaborative effort with the initial goal of 54 clinical trial sites prepared to enroll patients this year across the U.S. to provide greater access to patients wishing to participate.
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.
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 potential for the inclusion of Seelos' Phase IIb/III study of SLS-005 (trehalose) in the HEALEY ALS Platform Trial to expedite the Phase IIb/III trial or provide greater access to patients and other statements relating to the potential to shorten development timelines. 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, but are not limited to, the risk that the platform trial model's success in oncology may not translate to the treatment of amyotrophic lateral sclerosis; 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.