Our Acquisition of Trehalose and Collaboration with Team Sanfilippo Foundation (TFS) by Anthony Marciano
On February 19, 2019, Seelos Therapeutics, Inc. (Nasdaq: SEEL) announced a definitive agreement with Bioblast Pharma Ltd., (Nasdaq: ORPN), whereby Seelos acquired all development and commercial rights to Bioblast’s proprietary trehalose 90 mg/mL IV (Trehalose) solution as well as the existing inventory of the drug which should be sufficient to fulfill its current research needs. Trehalose, which is currently an investigational molecule, has been studied in prior phase 2 clinical studies in over 70 patients with two rare diseases and demonstrated a favorable safety profile and promising efficacy signal.
Under the terms of the acquisition, Seelos assumed a collaborative agreement with Team Sanfilippo Foundation (TSF), a nonprofit medical research foundation founded by parents of children with Sanfilippo syndrome. TSF, upon approval by the FDA, plans to begin an open label, Phase 2(b) clinical trial in up to 20 patients with Sanfilippo syndrome and Seelos will provide the clinical supply of trehalose. The terms of the agreement entitle Seelos access to all clinical data from this trial. Based on the pre-clinical and in-vitro studies, there is a sound scientific rationale for developing trehalose for the treatment of Sanfilippo syndrome.