Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company, announced today 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.
"The acquisition of Trehalose, along with our collaboration with TSF, are each important to understand in terms of their significance," said Raj Mehra, PhD, Chairman, Founder, and Chief Executive Officer, Seelos Therapeutics. "Taken together, these are validating achievements for Seelos' collaborative approach and speaks to the clinical and moral imperative to address the needs of patients with Sanfilippo syndrome, the need to rapidly advance novel therapeutics like Trehalose, and the need to continue to evaluate Trehalose in a range of such rare and devastating diseases including continuing the prior work in oculopharyngeal muscular dystrophy and spinocerebellar ataxia type 3."
"Our hope through this study is to evaluate the safety and efficacy of Trehalose in all forms of Sanfilippo, which is a devastating terminal disease that took the lives of 16 children last year," said Kathleen Buckley, President of the New York-based TSF. "Our team has tirelessly worked to raise proceeds to fund this study. If successful, we will work with the regulatory agencies in the US and EU to get this drug approved and into the hands of our families."
Under the terms of the agreement, Seelos made an upfront payment to Bioblast and will make certain additional milestone payments upon the achievement of clinical and regulatory milestones, plus a royalty upon clinical success, and approval for commercialization. Full terms of this agreement are disclosed in the Current Report on Form 8-K, filed by Seelos with the Securities and Exchange Commission today.
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