WTX-245 / WaveBreak Therap - LARVOL DELTA

WaveBreak Presents First Preclinical Data Demonstrating Efficacy of Small Molecule WTX-245 Inhibiting Source Mechanisms of TDP-43 Pathology, with Disease-Modifying Potential in ALS and FTD (PRNewswire) - "WaveBreak announced today the presentation of pioneering preclinical data demonstrating that WTX-245, a first-in-class small molecule WaveBreak discovered that targets TDP-43 aggregation for the treatment of ALS, restores normal splicing for multiple mRNAs that are critical to motor neuron function and survival. The WaveBreak data are featured in a poster presentation at the 35th International Symposium on ALS/MND taking place from December 6–8, 2024 in Montreal...WTX-245 was designed to directly inhibit disease-specific nucleation mechanisms at the source of TDP-43 aggregation and was tested in the seeded neuronal cell assay with the following results: WTX-245 significantly reduced cytoplasmic TDP-43 aggregation (p < 0.01) and restored normal transcriptional regulation (p < 0.05), both in a dose-dependent manner; WTX-245 significantly reduced mis-spliced UNC13A and STMN2, coincident with a significant reduction of TDP-43 aggregates."

Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders