RARE Daily

FDA Grants Fast Track Designation to Transposon’s TPN-101 for Progressive Supranuclear Palsy

May 21, 2024

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation to Transposon Therapeutics’ TPN-101 for progressive supranuclear palsy, a rare neurodegenerative movement disorder.

Progressive supranuclear palsy (PSP) causes slowing of movement, loss of balance leading to falls, impaired eye movements, and disturbances in cognition and behavior. The disease typically affects people in their mid- to late-60s, and the mean survival for individuals with PSP is 6 to 7 years.

TPN-101 specifically inhibits the LINE-1 reverse transcriptase that promotes LINE-1 replication. LINE-1 elements are a class of retro-transposable elements that in humans are uniquely capable of replicating and moving to new locations within the genome. When this process becomes dysregulated, LINE-1 reverse transcriptase drives overproduction of LINE-1 DNA, triggering innate immune responses that contribute to neurodegenerative, autoimmune and aging-related disease pathology.

“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 Dennis Podlesak, chairman and CEO of Transposon.

The designation of TPN-101 as a Fast Track product for PSP is supported by data from a phase 2, randomized, double-blind, placebo-controlled study of TPN-101 in patients with PSP. In that study, TPN-101 was the first treatment for PSP to reduce levels of neurofilament light chain (NfL), a key biomarker of neurodegeneration in tauopathies such as PSP and Alzheimer’s disease. TPN-101 also showed dose-related reductions in interleukin 6 (IL-6) cytokine levels, a biomarker of neuroinflammation that is elevated in PSP and correlates with disease progression and severity. Participants treated with TPN-101 for the entire 48-week trial duration showed a stabilization of their clinical symptoms as measured by the PSP Rating Scale between weeks 24 and 48.

The FDA grants Fast Track designation to facilitate the development and expedite the review of medicines to treat serious conditions and fill an unmet medical need. Fast Track status allows for enhanced communication and collaboration between the FDA and drug developers, potentially speeding up the delivery of life-saving treatments to patients.

Photo: Dennis Podlesak, chairman and CEO of Transposon

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