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Seelos Therapeutics says FDA accepts Investigation New Drug application for trehalose, can proceed with proposed Sanfilippo patients clinical trial

Seelos’ chief medical officer commented: “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"

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Seelos said it 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

Seelos Therapeutics Inc. (NASDAQ:SEEL) shares rose on Thursday after the firm revealed that the US Food and Drug Administration (FDA) has accepted the Investigation New Drug (IND) application for its SLS-005 (trehalose) treatment and has advised that the company may proceed with the proposed clinical trial on patients with Sanfilippo syndrome.

In morning trade in New York, Seelos shares were 6.8% higher at $1.762.

The clinical-stage biopharmaceutical company said 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 said it 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.

READ: Seelos Therapeutics to embark on two key clinical trials in the third quarter

In a statement, Warren W. Wasiewski, MD, Seelos’ chief medical officer commented: “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.”

“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," he added.

Trehalose is a low molecular weight disaccharide that crosses the blood brain barrier, stabilizes proteins, and importantly activates autophagy which is the process that clears material from cells.

It 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.

Singular focus for Sanfilippo community

Team Sanfilippo Foundation (TSF) is a non-profit medical research foundation founded in 2008 by parents of children with Sanfilippo syndrome (Mucopolysaccharidosis type III). TSF's mission is to fund potential therapies that can be in clinical trials in the near future.

Raj Mehra, chairman and CEO of Seelos Therapeutics said: “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.”

Seelos is focused on the development and advancement of novel therapeutics to address unmet medical needs for the benefit of patients with central nervous system disorders and other rare disorders.

The New York-based company's robust portfolio includes several late-stage clinical assets targeting psychiatric and movement disorders, including orphan diseases.

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Contact the author at [email protected]

Quick facts: Seelos Therapeutics

Price: 0.8736 USD

NASDAQ:SEEL
Market: NASDAQ
Market Cap: $23.54 m
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