Biotechnology company Transposon Therapeutics announced on May 21 that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for an intervention developed to treat progressive supranuclear palsy (PSP), an FTD disorder that primarily affects movement.

The designation was granted to TPN-101, an orally administered drug that targets LINE-1, a virus-like DNA sequence known as a retrotransposon. Unlike genes that encode proteins critical to cell structure and function, which are copied directly from DNA, retrotransposons can be copied from RNA through a process called reverse transcription. In brain cells, the reverse transcription of LINE-1 can cause cell damage and harmful immune responses that contribute to neurodegeneration. TPN-101 blocks an enzyme necessary for reverse transcription, preventing the detrimental effects on the brain.

The final results of a Phase 2 clinical trial evaluating TPN-101 as a treatment for PSP were released in February. The trial results showed that the drug reduced levels of multiple biomarkers associated with neurodegeneration, with Transposon noting that participants in the trial showed stabilization of their symptoms. A second Phase 2 trial is currently underway to study the drug as a treatment for ALS/FTD caused by C9orf72.

“Fast Track designation for TPN-101 is an important acknowledgement by the FDA of the critical need to find an effective treatment for PSP, a rare and devastating neurological disorder with no approved treatment options,” said Transposon CEO Dennis Podlesak. “We look forward to working collaboratively with the FDA to advance the development of TPN-101 as rapidly as possible for the treatment of PSP and other neurodegenerative diseases, including ALS and Alzheimer’s disease.”

The FDA’s “Fast Track” designation is designed to accelerate the development and regulatory review of drugs or interventions that address an unmet medical need. In addition to increased communication with the FDA, the designation makes TPN-101 eligible for Accelerated Approval and Priority Review.

Researchers studying FTD are making considerable progress in the search for treatments, with an increasing number of experimental therapies and diagnostic tools entering clinical trials. Days before Transposon’s announcement, Aprinoia Therapeutics announced a Fast Track designation of a PET tracer for PSP. In May, Passage Bio highlighted promising clinical trial data in a quarterly update.

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