NEW YORK, NY, USA I July 17, 2019 I Seelos Therapeutics, Inc. (NASDAQ: SEEL), a clinical-stage biopharmaceutical company, announced today that it has submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for the initiation of a Phase IIb/III clinical study of SLS-005 (trehalose) for the treatment of Sanfilippo syndrome (SFS).
Seelos is working in collaboration and under a grant with Team Sanfilippo Foundation (TSF), a nonprofit foundation founded by parents of children with Sanfilippo syndrome. In May, Seelos announced the intention to expand this IND to be 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.
“We are very excited to be working with Team Sanfilippo to offer this innovative therapy to these patients for whom there is no effective treatment,” said Warren Wasiewski, M.D., F.A.A.P., Chief Medical Officer of Seelos Therapeutics. Dr. Wasiewski, who recently joined the company to lead the collaboration with TSF and spearhead SLS-005 development, added, “Trehalose, with its multiple mechanisms of action, has the potential to be effective in a number of Neurologic diseases for which we intend to explore.”
Additionally, based on an overwhelming response from the Sanfilippo community worldwide, TSF, in collaboration with Seelos Therapeutics, has decided 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.
SOURCE: Seelos Therapeutics
Post Views: 915
NEW YORK, NY, USA I July 17, 2019 I Seelos Therapeutics, Inc. (NASDAQ: SEEL), a clinical-stage biopharmaceutical company, announced today that it has submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for the initiation of a Phase IIb/III clinical study of SLS-005 (trehalose) for the treatment of Sanfilippo syndrome (SFS).
Seelos is working in collaboration and under a grant with Team Sanfilippo Foundation (TSF), a nonprofit foundation founded by parents of children with Sanfilippo syndrome. In May, Seelos announced the intention to expand this IND to be 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.
“We are very excited to be working with Team Sanfilippo to offer this innovative therapy to these patients for whom there is no effective treatment,” said Warren Wasiewski, M.D., F.A.A.P., Chief Medical Officer of Seelos Therapeutics. Dr. Wasiewski, who recently joined the company to lead the collaboration with TSF and spearhead SLS-005 development, added, “Trehalose, with its multiple mechanisms of action, has the potential to be effective in a number of Neurologic diseases for which we intend to explore.”
Additionally, based on an overwhelming response from the Sanfilippo community worldwide, TSF, in collaboration with Seelos Therapeutics, has decided 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.
SOURCE: Seelos Therapeutics
Post Views: 915
