NEW YORK, March 11, 2020 (GLOBE NEWSWIRE) -- 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 that it has received a written response from the European Medicines Agency (EMA), in lieu of a face to face meeting, offering guidance for Seelos' planned European study of SLS-005 (IV trehalose) in patients with mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome.
Based upon the written response from the EMA, Seelos will design an open-label, non-placebo controlled, Phase IIb/III pivotal study for the treatment of patients with Sanfilippo syndrome types A and B in the European Union (EU).
"The EMA's recommendation to use appropriate natural history data of Sanfilippo syndrome patients allows us to offer active therapy to all children enrolled in the study," said Raj Mehra, Ph.D., Chairman and CEO of Seelos. "This devastating disease currently lacks any approved treatment and we look forward to taking this next step in advancing our SLS-005 program."
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, 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.
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 of the SLS-005 study in the European Union, the use of natural history data of Sanfilippo syndrome patients in the SLS-005 study and the use of active therapy for all children enrolled in the SLS-005 study. 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 clinical studies, including its planned Phase IIb/III study of SLS-005 in the EU, 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 its development plans and ongoing operations, risks related to Seelos' current stock price, 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.
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