Trehalose, which is currently an investigational molecule, has demonstrated favorable safety and efficacy in 59 patients
across two Phase II studies in rare diseases (OPMD and SCA3).
Additional trehalose studies are ongoing/planned in ALS and Sanfilippo Syndrome in the US
Seelos is currently collaborating with Team Sanfilippo Foundation (TSF). Upon approval by the FDA, TSF will begin a
Phase IIb/III clinical trial in up to 20 patients with Sanfilippo syndrome with Seelos providing the clinical supply of trehalose.