US awards $11.4M for therapeutic to treat Fragile X-associated tremor
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Key takeaways:
- Funding from the Department of Defense will support a phase 2 trial of LYT-300.
- There are no current primary treatments for Fragile X-associated Tremor Ataxia syndrome.
The U.S. Department of Defense has awarded $11.4 million to a Boston-based biotherapeutics firm to advance its novel oral prodrug of endogenous allopregananolone, LYT-300, to treat Fragile X-associated Tremor Ataxia syndrome.
According to a release from PureTech Health plc, these funds will support a phase 2 trial of LYT-300 in collaboration with the University of California, Davis and Randi Hagerman, MD, FAAP, who discovered FXTAS and currently serves as medical director of the UC Davis MIND Institute and is chair in Fragile X research.
“This award from the [Department of Defense] allows us to expand our evaluation of LYT-300, a candidate with a wide variety of potential indications, into [Fragile X-associated Tremor Ataxia syndrome], an area of tremendous need where otherwise normally developed, aging individuals suffer from significant neurodegeneration,” Eric Elenko, PhD, chief innovation officer at PureTech Health, said in the release.
LYT-300 was developed through PureTech's GlyphTM platform, which harnesses the body's natural lipid absorption and transport process to facilitate oral administration of certain therapeutics which otherwise could not be taken by mouth, per the release.
According to PureTech, an exploratory, open-label trial of six men with FXTAS found IV administration of allopregnanolone to be well-tolerated and demonstrated pharmacologic benefit across multiple neurological endpoints, including the Behavioral Dyscontrol Scale which measures executive, cognitive and motor function.
"FXTAS is a devastating, late-onset neurodegenerative condition characterized by cognitive decline, tremors in the hands and balance problems,” Hagerman, co-primary trial investigator, said in the release. “Currently, there are no primary treatments for FXTAS, though IV-administrated allopregnanolone has demonstrated therapeutic potential.”
PureTech additionally stated it plans to evaluate LYT-300 in a placebo-controlled trial to demonstrate the safety, tolerability and efficacy of the drug.