PureTech to use $11.4M award from US Department of Defense to find cure for 'devastating' neurological condition

By Liza Laws

- Last updated on GMT

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© Getty Images

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PureTech Health plc has been awarded $11.4 million (£8.9 million) from the US Department of Defense (DoD) to advance its therapeutic candidate for the potential treatment of neurological and neuropsychiatric disorders.

PureTech says it is dedicated to changing the lives of patients with devastating diseases made the announcement today (August 1) that its candidate LYT-300 (oral allopregnanolone) for the treatment of Fragile X-associated Tremor Ataxia Syndrome (FXTAS) will now be supported by the funds through a phase 2 trial in collaboration with the University of California, Davis.

LYT-300, an oral prodrug of allopregnanolone, is PureTech’s wholly owned therapeutic candidate for the potential treatment of anxiety disorders, postpartum depression​, FXTAS and other neurological and neuropsychiatric indications.

It was developed using PureTech’s GlyphTM platform, using the body’s natural lipid absorption and transport process to enable the oral administration of certain therapeutics that otherwise cannot be administered orally.

FXTAS, which was discovered by Randi Hagerman and her colleagues at the University of California, Davis, is a devastating neurological condition that is closely related to, but distinct from, Fragile X syndrome (FXS). Both conditions are the result of repeated elements in the Fragile X Messenger Ribonucleoprotein Gene 1 (FMR1) gene. While FXS is associated with intellectual disability and autism, FXTAS leads to neurodegeneration in otherwise normally developed, aging individuals who carry a premutation of the FMR1 gene.

Randi Hagerman is medical director of the UC Davis MIND Institute, chair in Fragile X Research and co-primary investigator for the LYT-300 trial.

She said: “FXTAS is a devastating, late-onset neurodegenerative condition characterized by cognitive decline, tremors in the hands and balance problems. Currently, there are no primary treatments for FXTAS, though IV-administrated allopregnanolone has demonstrated therapeutic potential.

“I am excited to be working with PureTech to evaluate their oral prodrug of allopregnanolone (LYT-300), and I am optimistic that this award will help accelerate the development of this potential first therapy for FXTAS.”

An exploratory, open-label trial of six men with FXTAS, evaluated IV-administration of allopregnanolone across multiple neuropsychological and emotional tests. In addition to being well-tolerated, allopregnanolone showed signals of pharmacologic benefit across multiple neurological endpoints, including the Behavioral Dyscontrol Scale, which measures executive, cognitive and motor function, and demonstrated improvement compared to baseline administration is not feasible in most indications, especially for a chronic therapy, and there remains a need for treatments that can address this debilitating condition.

PureTech plans to evaluate LYT-300 in a placebo-controlled trial to demonstrate the safety, tolerability and efficacy of the drug in people with FXTAS.

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

“We look forward to collaborating with Randi Hagerman and her team at UC Davis to bring the potential of allopregnanolone to the thousands of individuals with FXTAS in need of a treatment.”

In addition to LYT-300, which is being advanced in three indications, PureTech is progressing multiple CNS​-focused programs derived from its Glyph platform. These include LYT-310, an oral form of cannabidiol that is expected to enter a Phase 1 clinical trial in the fourth quarter of 2023, as well as programs that are in various stages of preclinical development.

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