A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD

Summary

This is a Phase 2a study to assess the the safety and tolerability of TPN-101 in patients with Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated with Hexanucleotide Repeat Expansion in the C9orf72 gene (C9ORF72 ALS/FTD).

Timeline

Start

2021-10-01

Primary completion

2023-08-17

Completion

2023-09-01

Outcome

Met primary endpoint

release “本試験の複数の主要エンドポイントで TPN-101（OBP-601）が有効であったことは” oncolys.com ↗

Publications

• Nicodemus J, Liu CS, Ransom L, Tan V, Romanow W, Jimenez N, Chun J. Sequence Diversity and Encoded Enzymatic Differences of Monocistronic L1 ORF2 mRNA Variants in the Aged Normal and Alzheimer's Disease Brain. J Neurosci. 2025 Jun 18;45(25):e2298242025. doi: 10.1523/JNEUROSCI.2298-24.2025.

Drugs

Indications