FDA Accepts Investigational New Drug Application and Grants Fast Track Designation of Trehalose for Spinocerebellar Ataxia Treatment 

The Food and Drug Administration (FDA) has accepted the investigational new drug (IND) application to trehalose (SLS-005; Seelos Therapeutics, New York, NY) for spinocerebellar ataxia (SCA) treatment. The FDA has granted the program Fast Track designation for SCA, and trehalose has previously received Orphan Drug designation for spinocerebellar ataxia type 3 (SCA3). Trehalose is administered as a 90.5 mg/mL intravenous infusion.

"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, PhD, chairman and chief executive officer 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. We look forward to initiating this study in early 2022 and our recent capital raises have accounted for the expected development costs for initiating this study."

Trehalose had been studied in a 6-month open-label phase 2a study that also included an additional 6-month follow up in participants with SCA3, also known as Machado-Joseph Disease. In an open-label study, 14 participants with SCA3 treated for 6 months with trehalose had stable Scale for Assessment and Rating of Ataxia (SARA) scores. Out of the 14 participants, 6 received treatment for as long as 12 months and continued to have stable SARA scores. Natural history data for SCA3 suggests a measurable increase on SARA would have been seen within 12 months without treatment.

"On behalf of the National Ataxia Foundation and the ataxia community we represent, we're thrilled that Seelos Therapeutics has received both IND acceptance and Fast Track designation from the FDA," said Andrew Rosen, NAF executive director. "Seelos is a founding member of the NAF Drug Development Collaborative and NAF looks forward to working closely with Seelos to support the company as its program progresses."