Seelos Therapeutics received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) covering the parenteral, or non-oral administration of SLS-005 (trehalose) for the treatment of neurodegenerative diseases characterized by abnormal protein aggregates.
The Notice of Allowance allows Seelos Therapeutics to finalize a patent — number 10,493,023 — for the treatment of such indications as Parkinson’s disease, amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and Huntington’s disease.
In addition to FA, the new patent also covers diseases like Sanfilippo syndrome, oculopharyngeal muscular dystrophy — a genetic disorder characterized by slowly progressing muscle disease affecting the muscles of the upper eyelids and the throat — and spinocerebellar ataxia type 3 (SCA3), also called Machado-Joseph disease. SCA3 is a form of ataxia caused by mutations in the gene encoding protein ataxin-3 (ATXN3).
The new Notice of Allowance would extend SLS-005’s use to the treatment of several progressive neurodegenerative diseases, including Alzheimer’s, ALS, Parkinson’s and Huntington’s. SLS-005 is given through parenteral administration, or injection. Typical routes of administration of a parenteral dosage include subcutaneous, or under the skin, intramuscular, or into the muscle, and intravenous, or into the blood delivery.
SLS-005 is a disaccharide, a double sugar, whose therapeutic benefits are linked with its ability to inhibit the formation of protein aggregates, or clumps, and enhance a natural cleaning process, called autophagy.
Cells use autophagy 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 reduces the risk of protein aggregation. SLS-005 also increases the production of proteins that make up acidic compartments inside the body’s cells — the lysosomes — which help degrade material no longer needed.
Researchers have noted that SLS-005 was shown to cross the blood-brain barrier, a highly selective membrane that shields the central nervous system — the brain and spinal cord — from the general blood circulation. The ability to cross this barrier is a key property for investigational therapies designed for the treatment of neurological conditions.
SLS-005 was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) and by the European Medicines Agency (EMA) for the treatment of SCA3 and oculopharyngeal muscular dystrophy. The FDA also granted fast track designation to the investigational therapy for oculopharyngeal muscular dystrophy.