DOD Awards PureTech up to $11.4 Million to Advance Rare Disease Therapy

Rare Daily Staff

PureTech Health said it has been awarded up to $11.4 million from the U.S. Department of Defense to advance its experimental therapy, LYT-300, for the treatment of Fragile X-associated tremor/ataxia syndrome.

The funds will support a phase 2 trial of LYT-300 in collaboration with the University of California, Davis.

Fragile X-associated tremor/ataxia syndrome (FXTAS) was discovered at the MIND Institute by researcher Randi Hagerman and her colleagues in 2001. It is one of the most devastating of the Fragile X spectrum disorders, which result from a trinucleotide expansion in the FMR1 gene. FXTAS is a late onset condition that can occur in up to 75 percent of males with the premutation by the eighth decade of life and in approximately 16 percent of females, but the clinical signs typically emerge when individuals are in their early 60s. The clinical features of FXTAS include tremor in the hands with action or at rest, balance problems (ataxia) that lead to frequent falling, and cognitive decline that is sometimes misdiagnosed as Alzheimer’s disease. No specific treatment for FXTAS is efficacious, though a variety of medications may improve psychiatric issues or the severity of tremor. Carriers are common in the general population, occurring in 1 in 150 to 200 women and 1 in 400 men, but FXTAS is often mistakenly diagnosed as Parkinson’s disease.

LYT-300, an oral prodrug of allopregnanolone, is PureTech’s wholly owned experimental therapy for the potential treatment of anxiety disorders, postpartum depression, FXTAS, and other neurological and neuropsychiatric indications. LYT-300 was developed using PureTech’s Glyph platform, which harnesses the body’s natural lipid absorption and transport process to enable the oral administration of certain therapeutics that otherwise cannot be administered orally.

“This award from the DoD allows us to expand our evaluation of LYT-300, a candidate with a wide variety of potential indications, [including] FXTAS, an area of tremendous need where otherwise normally developed, aging individuals suffer from significant neurodegeneration,” said Eric Elenko, chief innovation officer at PureTech Health.

Photo: Eric Elenko, chief innovation officer at PureTech Health