WTX-A is a small-molecule oral drug developed by WaveBreak. In a recent pre-clinical study, it efficiently blocked α-synuclein oligomers and aggregates in both cell and mouse models of Parkinson’s disease (PD) in a dose-dependent manner. Oligomers are toxic intermediate forms of abnormally folded proteins directly responsible for neurotoxicity in PD, Alzheimer’s Disease, and Amyotrophic Lateral Sclerosis. WTX-A is highly specific to α-synuclein but not tau and Aβ42 amyloid aggregates.
PD is a neurodegenerative movement disorder affecting over 10 million people worldwide. In PD, dopaminergic neurons of the brain that control movement are preferentially lost. This results in slow movement, rigidity of limbs and trunk, tremors, and postural instability. Inside the affected neurons, inclusion bodies called Lewy bodies containing disaggregated oligomers of proteins accumulate. Presence of aggregated α-synuclein in Lewy bodies is a hallmark of the disease, with progressive development of both motor and non-motor symptoms. Currently available drugs only symptomatically treat a subset of PD symptoms, and there are no treatments available that are disease-modifying and slow the progression of the disease. In the United States alone, a million people suffer from PD. With an annual incidence of nearly 90,000 cases, PD is the fastest-growing neurological disease in the U.S. and expected to affect 1.2 million people by 2030.
Since oligomers are highly transient, targeting them using conventional single-target drug discovery approaches is challenging. WaveBreak’s unique technology platform enables analysis of the inhibition of the source mechanisms that produce oligomer intermediates in complex disease processes.
