A Notice of Allowance allows Seelos Therapeutics to finalize a patent for SLS-005 for the treatment of the disease.
Friedreich’s ataxia is an inherited neuromuscular disease caused by the loss or impaired activity of the frataxin protein. The disease is characterized by the accumulation of high levels of iron in certain areas of the brain, which leads to the formation of damaging reactive oxygen species (ROS) and abnormal protein aggregates that likely leads to the death of affected neurons.
SLS-005 is a disaccharide, a double sugar, whose therapeutic benefits are linked with its ability to inhibit the formation of protein aggregates and enhance cells’ natural cleaning system, called autophagy.
Autophagy is a key process used by cells to degrade damaged materials (such as abnormal protein aggregates) or material no longer required, so that they can be recycled and used in new functions.
By enhancing autophagy’s activity, more proteins are degraded, which again reduces the risk of protein aggregation. SLS-005 also increases the production of proteins that make up acidic compartments inside our cells — the lysosomes — which help degrade material no longer needed.
Importantly, the company states that the compound has been shown to cross the blood brain barrier and penetrate muscle, important for the treatment of neurological conditions.
SLS-005 has already been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) and by the European Medicines Agency (EMA) for the treatment of spinocerebellar ataxia type 3 (also called Machado-Joseph disease) and oculopharyngeal muscular dystrophy, a genetic disorder characterized by slowly progressing muscle disease affecting the muscles of the upper eyelids and the throat.
Seelos is also developing SLS-005 for Sanfilippo syndrome, which comprises a group of four rare genetic disorders caused by a deficiency in one of the enzymes needed to fully break down a complex sugar molecule called heparan sulfate. This leads to the buildup of non-degraded or partially degraded heparan sulfate, particularly at the central nervous system (which comprises the brain and spinal cord), causing neurodevelopmental problems.
Orphan drug designation aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application fees.