IND Accepted for an Experimental Sanfilippo Syndrome Treatment

According to a story from, the biopharmaceutical company Seelos Therapeutics, Inc. has recently announced that the US Food and Drug Administration (FDA) has accepted their Investigational New Drug (IND) application. This application is in regards to the company’s investigational drug candidate trehalose. This drug is in development as a treatment for the rare genetic disorder Sanfilippo syndrome. This action means that Seelos will now be able to legally proceed with clinical trials focusing on trehalose. Seelos is focused on the development of unique therapeutics for patients with rare diseases affecting the central nervous system and others.

About Sanfilippo Syndrome

Sanfilippo syndrome, which is also known as mucopolysaccharidosis III, is rare genetic lysosomal storage disease. It is linked to a deficiency in the enzyme responsible for the breakdown of heparan sulfate. There are four different types of Sanfilippo syndrome and each one is caused by a different genetic mutation. In type A, the mutation affects the SGSH gene. The mutation caused is the only defining characteristic of the different types, which otherwise present similarly. Symptoms include behavioral abnormalities, dementia, sleep disturbances, difficulty speaking, developmental delays, deafness, and loss of movement. There are currently no disease modifying therapies available for this disorder. However, bone marrow replacement can be useful if implemented early. Most patients do not survive beyond their teenage years, but some can survive into their 30s. To learn more about Sanfilippo syndrome, click here.

Future Trial Plans

Seelos is currently working with the FDA to finalize the final parameters of its clinical trials for the experimental therapy. There are currently plans in the works for a unique combined phase 2b/3 clinical trial that will test in the drug in patients with Sanfilippo syndrome type A and B. The company is also working on an expanded access portion that will included patients with types C and D as well as others that were not eligible for the regular portion of the study.

About Trehalose

Trehalose counteracts the buildup of toxic heparan sulfate by stimulating a process called autophagy, which allows cells to clear waste materials. The drug is also capable of crossing the blood-brain barrier which should allow it to address cognitive symptoms as well.

Follow us