FDA Grants Fast Track Designation to Seelos’s Treatment for Spinocerebellar Ataxia

The U.S. Food and Drug Administration has accepted Seelos Therapeutics’ Investigation New Drug application to study SLS-005 for the treatment of spinocerebellar ataxia and has granted the program Fast Track designation.

The Fast Track process is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.

Spinocerebellar Ataxia (SCA) is a serious disease caused by degeneration of the cerebellum with an onset usually in adult life. Clinically, it is characterized by progressive unsteadiness of gait and stance, impaired coordination of limb movements, slurred speech, and abnormal eye movements. Spinocerebellar ataxia type 3, also known as Machado-Joseph disease (MJD), is characterized by progressive cerebellar ataxia and is known to cause progressively severe disability and often premature death approximately 10-20 years from onset of symptoms.

“SCA is a highly debilitating neurodegenerative disease that currently lacks a cure or an approved therapeutic and as such, patients manage symptoms through physical therapy and other symptomatic treatments,” said Raj Mehra, chairman and CEO of Seelos. “SLS-005 has already displayed encouraging open label human data in SCA3, the most common type of SCA, and our team has taken that experience and knowledge into the design and plans for our global phase 2b/3 placebo-controlled study.”

Trehalose is a low molecular weight disaccharide 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 several diseases with pathologic accumulation of storage material. SLS-005 has previously received Orphan Drug designation for spinocerebellar ataxia type 3 (SCA3) from the FDA and from the European Medicines Agency.

Prior to Seelos acquiring the program, SLS-005 had already been studied in a six-month open label phase 2a study that also included an additional six-month follow up in patients with SCA3. The open label study evaluated 14 patients with SCA3 over a six-month period and found the average score on the Scale for Assessment and Rating of Ataxia (SARA), a well-recognized clinical tool for measuring functional impairment associated with the disease, remained stable. Six patients received treatment for as long as 12 months and continued to maintain stable SARA scores. In comparison, natural history data suggests that individuals with SCA3 would be expected to show a measurable increase on SARA within a 12-month period, which is indicative of disease progression and worsening of symptoms.

Additionally, Seelos was named as one of the initial members of the National Ataxia Foundation (NAF) Drug Development Collaborative, an industry consortium that has the principal goal of accelerating the development of treatments for Ataxia.

Author: Rare Daily Staff