Seelos Therapeutics announced it will meet with the European Medicines Agency (EMA) in March to seek scientific advice and assistance for a European clinical study of SLS-005 (trehalose) in patients with Sanfilippo syndrome types A and B.
The EMA supports the research and development of medicines for rare diseases in the European Union (EU) through regulatory guidance and incentives.
“We are truly excited to be working with the EMA to formulate the Sanfilippo study in the EU,” Raj Mehra, PhD, chairman and CEO of Seelos, said in a press release. “There are currently no approved therapies in the EU for this devastating neurological condition.”
Sanfilippo syndrome is a genetic disorder characterized by the loss or impaired function of enzymes that break down a group of sugar molecules called glycosaminoglycans.
There are four types of Sanfilippo syndrome, designated A, B, C, and D. Each type is distinguished by a mutation in a different gene containing the instructions for making one of these enzymes. A person with one of these mutations will accumulate partially broken-down sugar molecules that become toxic to cells and tissues.
SLS-005 is a natural type of sugar (trehalose) synthesized by some bacteria, fungi, plants, and invertebrate animals as an energy source.
As a medicine, it is given intravenously (in a vein) and can reach the brain to promote a process used by cells to eliminate waste products known as autophagy. For those with Sanfilippo syndrome, trehalose can boost this process to dispose of the accumulated glycosaminoglycan molecules.
Preclinical studies in a Sanfilippo type B mouse model demonstrated that SLS-005 reduced inflammation in the brain and retina, lessened nerve cell degeneration, and extended patients’ lifespans.
SLS-005 was shown to be safe and well-tolerated in two Phase 2 clinical trials in patients with oculopharyngeal muscular dystrophy (NCT02015481) and spinocerebellar ataxia type 3 (NCT02147886). Treating these patients with injectable SLS-005 prevented the accumulation of toxic molecules by stimulating autophagy.
Encouraged by these results, Seelos will begin a multi-center Phase 2b/3 trial to test the safety, tolerability, and effectiveness of SSL-005 in patients with Sanfilippo syndrome types A or B.
The company is collaborating with a nonprofit organization founded by parents of children with Sanfilippo syndrome, Team Sanfilippo Foundation (TSF). Its mission is to provide funding to support research into potential therapies and help families gain access to treatments and clinical trials.
Based on the response from the global Sanfilippo community and TSF, Seelos has submitted a protocol to the U.S. Food and Drug Administration for its planned expanded access study for Sanfilippo syndrome types C and D patients, as well as type A and B patients who do not meet the Phase 2b/3 trial criteria for entry.