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Transposon Announces Interim Results from a Phase 2 Study of TPN-101 for the Treatment of Progressive Supranuclear Palsy to be Presented at the AD/PD™ 2024 International Conference on Alzheimer’s and Parkinson’s Diseases

Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, today announced its abstract of interim results from its Phase 2 study of TPN-101 for the treatment of progressive supranuclear palsy (PSP) has been accepted for poster presentation at the hybrid AD/PD™ 2024: 18th International Conference on Alzheimer’s and Parkinson’s Diseases. The meeting will take place online and in Lisbon, Portugal, from March 5-9, 2024.

The poster presentation will highlight results from the study’s predefined interim analysis, which examined the change from baseline to 24 weeks in biomarkers of neurodegeneration and neuroinflammation, including neurofilament light chain (NfL) and interleukin 6 (IL-6) levels in cerebrospinal fluid (CSF).  In the treatment group receiving 400 mg of TPN-101 once daily for 24 weeks, TPN-101 showed an 18.4% reduction in NfL levels in CSF as compared to placebo. NfL is a biomarker of neurodegeneration that correlates with disease severity and progression in PSP.  In the same 400 mg treatment group, TPN-101 also resulted in a 51.6% reduction in IL-6 levels in CSF as compared to placebo. IL-6 is a biomarker of neuroinflammation that is elevated in PSP patients and correlates with disease severity.  Once-daily oral dosing of TPN-101 was well-tolerated at all dose levels.

“No previously tested drug has shown an effect on NfL levels in patients with PSP, a progressive neurological disorder with no approved treatment options to stop or even slow progression of the disease,” said Andrew Satlin, M.D., Chief Medical Officer at Transposon. “The lowering of CSF NfL levels seen in this interim analysis provides biomarker evidence of a treatment effect on neurodegeneration. We are excited about the potential of TPN-101 as a much-needed treatment option for patients with PSP. In addition, we believe these findings open the door to an entirely new therapeutic approach to treating Alzheimer’s and other neurodegenerative diseases.”

“The Transposon team continues to make excellent progress against our key development milestones and corporate priorities, and we look forward to presenting these interim results from our PSP program at the AD/PD 2024 International Conference on Alzheimer’s and Parkinson’s Diseases,” said Dennis Podlesak, Chairman and Chief Executive Officer of Transposon. “Beyond the promising reduction in NfL levels seen in our PSP study, we also look forward to reporting data from our three ongoing Phase 2 programs of TPN-101 in the first quarter of 2024 and, importantly, to expanding our development efforts for the compound into Alzheimer’s disease and other neurodegenerative and autoimmune disorders.”

Transposon will present results from the interim analysis in a poster presentation (abstract #433) at AD/PD 2024 entitled: “A Phase 2a Study of TPN-101, a Nucleoside Reverse Transcriptase Inhibitor, in Patients with Progressive Supranuclear Palsy.”



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