Seelos Therapeutics (NASDAQ:SEEL) has announced it has submitted an investigational new drug application to the US Food and Drug Administration (FDA) to start a Phase 2b/3 clinical study of SLS-005 to treat Sanfilippo syndrome (SFS).
As quoted in the press release:
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.