TPN-101 had lowering effects on key biomarkers of neurodegeneration and neuroinflammation, including neurofilament light chain (NfL) and interleukin 6 (IL-6)
Treatment with TPN-101 also showed clinical benefits on the Revised ALS Functional Rating Scale (ALSFRS-R) and Vital Capacity, key clinical outcome measures for ALS
SAN DIEGO , Oct. 9, 2024 /PRNewswire/ — Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease, today announced that results from its Phase 2 study of TPN-101 in patients with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) related to hexanucleotide repeat expansion in the C9orf72 gene (C9orf72 -related ALS/FTD) have been accepted for both oral and poster presentation at the 2024 Annual Northeastern Amyotrophic Lateral Sclerosis Consortium (NEALS) Meeting . The hybrid meeting will take place October 21-24, 2024 , in Clearwater, Florida , and virtually.
“Given the effects of TPN-101 on key biomarkers of neurodegeneration and neuroinflammation, including NfL and IL-6, and clinical outcome measures of disease progression and respiratory function in patients with C9orf72 -related ALS, we are very pleased these data were accepted for presentation at NEALS,” said Andrew Satlin , M.D., Chief Medical Officer at Transposon. “Based on these promising results, we are advancing TPN-101 into a Phase 3 registration study for the treatment of C9orf72 -related ALS, while also continuing to develop TPN-101 for other neurodegenerative diseases.”
Oral presentation details
Title :
A Phase 2A Study of TPN-101, A Nucleoside Reverse Transcriptase Inhibitor, in Patients with C9ORF72-Related ALS/FTD
Presenter:
Andrew Satlin, M.D.
Session :
NEALS Affiliated Trial Session
Date and time:
Tuesday, October 22, 2024, at 11:15 am EDT
Location:
Opal Ballroom
The poster will also be on view during Poster Session 2 on Wednesday, October 23, 2024 , from 4:00 to 6:00 pm EDT .
Title :
A Phase 2A Study of TPN-101, A Nucleoside Reverse Transcriptase Inhibitor, in Patients with C9ORF72-Related ALS/FTD
Session :
Poster Session 2
Date and time:
Wednesday, October 23, 2024, from 4:00 to 6:00 pm EDT
Location:
Sea Salon & Sand Salon
For more information, please visit the 2024 Annual NEALS Meeting website.
About the Phase 2 Study in C9orf72-related ALS/FTD
The Phase 2 study in patients with C9orf72 -related ALS/FTD was a multi-center, randomized, double-blind, placebo-controlled parallel-group, two-arm study with an open-label treatment period. Participants (n=42) were randomized 3:2 to receive daily doses of 400 mg of TPN-101 or placebo. The study included a six-week screening period, a 24-week double-blind treatment period, a 24-week open-label treatment period, and a follow-up visit four weeks post-treatment. Further information on the study can be accessed at ClinicalTrials.gov .
About TPN-101
TPN-101 specifically inhibits the LINE-1 reverse transcriptase that promotes LINE-1 replication. LINE-1 elements are a class of retrotransposable 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 cDNA, triggering innate immune responses that contribute to neurodegenerative, neuroinflammatory, and aging-related disease pathology.
About ALS and FTD
ALS is a neurodegenerative disease characterized by progressive muscle weakness, and loss of ability to speak, eat, move or breathe. FTD is a progressive frontal/temporal cortex disease associated with behavior and personality changes, emotional problems, and difficulty walking, communicating, or working. A C9orf72 hexanucleotide repeat mutation accounts for 10-15% of both disorders. With onset commonly in middle age or earlier, patients with ALS have a mean survival of two to three years. Patients with FTD have a mean survival of nine years.
About Transposon
Transposon Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a platform of novel therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease. The company’s lead clinical compound, TPN-101, is first-in-class to address LINE-1 reverse transcriptase for treating neurodegenerative and autoimmune diseases. The company also has a discovery platform supporting a deep pipeline of novel therapies to address additional indications.
Contact:
Rick Orr
Transposon Therapeutics, Inc.
(858) 535-4821
rorr@transposonrx.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/transposon-to-present-results-from-phase-2-study-of-tpn-101-for-the-treatment-of-c9orf72-related-alsftd-at-the-2024-annual-neals-meeting-302270774.html
SOURCE Transposon Therapeutics