Additional US Patent allowed for Seelos’ Trehalose (SLS-005)

The allowed claims cover a method of using trehalose (SLS-005) to treat several neurodegenerative conditions including spinocerebellar ataxia (SCA), spinal and bulbar muscular atrophy (SBMA), dentatomral-pailidoluyssan atrophy (DRPLA), Pick’s disease, corticobasaldegeneration (CBD), progressive supranuclear palsy (PSP), and frontotemporal dementia. 

Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins, and importantly activates autophagy which is the process that clears material from cells. In several animal models of diseases, associated with abnormal cellular protein aggregation or storage of pathologic material, it has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material.

Seelos gets European ODD for SLS-005 in Sanfilippo Syndrome

Sanfilippo syndrome (or MPS III) is an inherited monogenic lysosomal storage disorder divided into four subtypes. Seelos is currently collaborating with Team Sanfilippo Foundation (TSF). Upon approval by the FDA, TSF will begin a Phase IIb/III clinical trial in up to 20 patients with Sanfilippo syndrome with Seelos providing the clinical supply of trehalose.

To date, SLS-005 has been granted Orphan Drug Designation in the US and Europe for Sanfilippo syndrome, Spinocerebellar Ataxia Type 3 (SCA3) and Oculopharyngeal Muscular Dystrophy (OPMD) as well as Fast Track designation for OPMD.

Seelos gets FDA go-ahead for ph 2b/3 study of SLS-005 in ALS

Seelos’ Phase IIb/III trial plans to enroll 160 patients with either familial or sporadic ALS in a double-blind placebo-controlled trial. Patients will be randomized 3:1 (drug:placebo) and studied with a primary endpoint measuring change from baseline on Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score at 24 weeks. Secondary endpoints will also be measured at 24 weeks, including change from baseline in slow vital capacity, muscle strength, quality of life measurements as well as additional signs of disease progression.  

According to the National Institute of Neurological Disorders and Stroke, Amyotrophic lateral sclerosis (ALS) is a group of rare neurological diseases that mainly involve the nerve cells (neurons) responsible for controlling voluntary muscle movement. In ALS, both the upper motor neurons and the lower motor neurons degenerate or die and stop sending messages to the muscles. Unable to function, the muscles gradually weaken, start to twitch (called fasciculations), and waste away (atrophy). Eventually, the brain loses its ability to initiate and control voluntary movements. The disease is progressive, meaning the symptoms get worse over time. The majority of ALS cases (90 percent or more) are considered sporadic. This means the disease seems to occur at random with no clearly associated risk factors and no family history of the disease. Although family members of people with sporadic ALS are at an increased risk for the disease, the overall risk is very low and most will not develop ALS.

Most people with ALS die from respiratory failure, usually within 3 to 5 years from when the symptoms first appear. However, about 10 percent of people with ALS survive for 10 or more years. Currently, there is no cure for ALS and no effective treatment to halt, or reverse, the progression of the disease.

Trehalose is a low molecular weight disaccharide (0.342 kDa) that crosses the blood brain barrier, stabilizes proteins and importantly, activates autophagy, which is the process that clears material from cells. In animal models of several diseases associated with abnormal cellular protein aggregation or storage of pathologic material, it has been shown to reduce aggregation of misfolded proteins and reduce accumulation of pathologic material. Trehalose activates autophagy through the activation of Transcription Factor EB (TFEB), a key factor in lysosomal and autophagy gene expression. Activation of TFEB is an emerging therapeutic target for a number of diseases with pathologic accumulation of storage material.