Nonprofit research foundation Team Sanfilippo (TSF) is preparing to launch a Phase 2 clinical trial to explore the safety and efficacy of a common sugar called trehalose as a treatment for patients with Sanfilippo syndrome.
This follows a collaborative agreement that was initially made with Bioblast Pharma, the therapy’s developer, but has since been acquired by Seelos Therapeutics, who recently obtained the global research and commercialization rights over trehalose from Bioblast.
“The acquisition of Trehalose, along with our collaboration with TSF, are each important to understand in terms of their significance,” Raj Mehra, PhD, chairman, founder, and CEO of Seelos, said in a press release.
“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,” Mehra added.
The new TSF open-label Phase 2 trial plans to enroll approximately 20 patients with confirmed Sanfilippo syndrome and has already received the U.S. Food and Drug Administration‘s seal of approval.
This trial is expected to provide evidence that trehalose, which is often used as a food additive, may be an effective treatment strategy for Sanfilippo patients of all types and ages, according to a post on the foundation’s website.
“We view Trehalose as a great promise to our children, families and community,” Kathleen Buckley, president of New York-based TSF, said in another press release. “We are eager to start the study and plan to do so in the very near future. We have been following the Trehalose potential and programs closely for several years and feel that it could indeed make a difference in the life of our loved ones.”
Sanfilippo syndrome is a genetic disorder characterized by the loss or impaired function of certain enzymes involved in the breakdown of a group of sugar molecules called glycosaminoglycans. As a result, patients accumulate partially broken-down sugar molecules, which, in turn, become toxic to cells and tissues.
Strategies used to restore the levels of the missing enzyme to normal levels, such as enzyme replacement therapies, gene therapy and stem cell transplants, have limitations as well as a risk of severe side effects.
Trehalose is a small compound that can reach the brain when given orally, and is known to promote autophagy — a process used by cells to dispose of their waste. These properties may enable trehalose to enhance cells’ ability to eliminate the abnormally accumulated glycosaminoglycan molecules.
Data from previous Phase 2 trials in more than 70 patients with oculopharyngeal muscular dystrophy (NCT02015481) and spinocerebellar ataxia type 3 (NCT02147886) have already demonstrated that trehalose is safe and well-tolerated.
In addition, these trials showed that treatment with an injectable trehalose solution can effectively prevent the accumulation of toxic molecules by restoring the cells’ recycling system to normal.
“Our team has tirelessly worked to raise proceeds to fund this study. If successful, we will work with the regulatory agencies in the U.S. and E.U. to get this drug approved and into the hands of our families,” Buckley said.