Seelos Therapeutics, Inc. issued the following announcement on Aug. 22.
Seelos Therapeutics, Inc. (Nasdaq: 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.
About Trehalose:
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.
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