Seelos to Proceed with Trial Enrollment and Dosing for Phase IIb/III Trial for Sanfilippo Syndrome Type A and B Patients
NEW YORK, Aug. 22, 2019 (GLOBE NEWSWIRE) -- Seelos Therapeutics, Inc. (SEEL), a clinical-stage biopharmaceutical company, is pleased to announce the acceptance of the Investigation New Drug (IND) application for SLS-005 (trehalose) by the Food and Drug Administration (FDA). The FDA has advised Seelos that it may proceed with the proposed clinical trial SLS-005-201 for Mucopolysaccharidosis type III (Sanfilippo syndrome).
“We are very pleased to have received the acceptance letter and continue to work closely with the FDA to finalize the details of the analysis methodology for primary and secondary endpoints to demonstrate efficacy of trehalose in Sanfilippo syndrome,” said Warren W. Wasiewski, M.D., F.A.A.P., Chief Medical Officer of Seelos. “Due to support and interest from families of patients with Sanfilippo, we continue to engage with additional clinical trial sites to accommodate enrollment for this trial.”
“This is a great milestone for Seelos and outlines the dedication and hard work of our clinical team under the leadership of Dr. Warren Wasiewski, our CMO,” said Raj Mehra, Ph.D., Chairman and CEO of Seelos Therapeutics. “We are grateful for the detailed guidance from the regulatory agency (FDA) and thank our financial supporter and collaborator, TSF, that has brought a singular focus to helping the Sanfilippo community.”
The clinical trial for SLS-005-201 is a combined Phase IIb/III, multicenter study designed to assess safety, tolerability and efficacy of trehalose IV in Sanfilippo A and B based on functional outcomes, biomarkers, neuro-cognitive assessments and quality of life measurements. Additionally, Seelos intends to expand inclusion of Sanfilippo type C and D patients as well as type A and B patients who do not meet the trial entry criteria into a separate expanded patient access study.
Trehalose is a low molecular weight disaccharide (.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 Team Sanfilippo Foundation:
Team Sanfilippo Foundation (TSF) is a nonprofit medical research foundation founded in 2008 by parents of children with Sanfilippo syndrome. TSF's mission is to fund potential therapies that can be in clinical trials in the near future. Team Sanfilippo is dedicated to providing assistance to families to gain access to clinical trials, treatments and compassionate use. Team Sanfilippo remains dedicated to getting children of all ages access to clinical trials and treatments and assistance to families enrolled in clinical trials. For more information, please visit teamsanfilippo.org.
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 disorders. The Company's robust portfolio includes several late-stage clinical assets targeting psychiatric and movement disorders, including orphan diseases. Seelos is based in New York, New York. For more information, please visit our website: http://seelostherapeutics.com, the content of which is not incorporated herein by reference.
This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and other Federal securities laws. For example, Seelos is using forward-looking statements when Seelos discusses the design of the clinical trial for SLS-005-201; Seelos’ plans to proceed trial enrollment and dosing for such clinical trial; Seelos’ plans to expand such clinical trial to include Sanfilippo type C and D patients; Seelos' future operations and its ability to successfully initiate and complete clinical trials and achieve regulatory milestones and related timing; the nature, strategy and focus of Seelos' business; the development and commercial potential and potential benefits of any of Seelos' product candidates including Trehalose; and that Seelos' product candidates have the potential to address critical unmet needs of patients with serious diseases and conditions. Seelos may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on Seelos' current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Seelos could differ materially from those described in or implied by the statements in this press release, including: the uncertainties associated with the clinical development and regulatory approval of Seelos' product candidates, including potential delays in the commencement, enrollment and completion of clinical trials; the potential that earlier clinical trials and studies of Seelos' product candidates may not be predictive of future results; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including those risks discussed in Seelos' filings with the Securities and Exchange Commission. Except as otherwise required by law, Seelos disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.