Parkinson's disease | Acurex Biosciences

15-LO Inhibitor — CU-13001:

Our lead therapeutic candidate, CU-13001, is designed to inhibit 15-Lipoxygenase (15-LO). CU-13001 significantly reduces PD pathologies including LRRK2 kinase hyperactivity and α-synuclein aggregation in cellular models from both genetic and idiopathic PD patients. This novel approach aims to fundamentally alter the progression of the disease. We expect to file an Investigational New Drug (IND) application for CU-13001 in 2025.

Selective CaV3.2 Antagonist:

Acurex has discovered a first-in-class selective antagonist of the low voltage-gated CaV3.2 calcium channel. This channel plays a significant role in Parkinson's disease and other pathologies by dysregulating calcium balance in the cell. In PD, calcium dysregulation leads to mitochondrial stress, neuroinflammation, and dopaminergic deficits, ultimately resulting in neurodegeneration. Due to the wide-ranging impact of CaV3.2 in neurons, this program may offer symptomatic relief and potentially slow or stop disease progression for PD patients.

T-Type Calcium Channel Inhibitor — CU-00048:

Voltage-gated calcium channels have been targets of interest for CNS indications due to their role as essential regulators of neural communication. T-type calcium channels are the only low-voltage activated calcium channels and play a key role regulating calcium oscillations and neuronal excitability. Acurex has developed a best-in-class pan T-type antagonist, CU-00048, that is highly selective to T-type calcium channels, has excellent drug-like properties, and is ready for IND-enabling studies. Acurex plans to partner this program for further development.

Miro1 Biomarker

The progression of Parkinson’s disease is difficult to measure due to the lack of validated biomarkers. This limitation often forces clinical trials to rely on the delayed progression of symptoms as an indicator of disease modification.

Acurex has developed a novel biomarker for PD, based on an initial discovery at Stanford University involving the protein Miro1. This biomarker serves as a tool for measuring mitochondrial dysfunction, a hallmark of PD and many other aging-related diseases. In tests involving over 200 PD patients, the biomarker has shown robust results. It can be detected in peripheral blood mononuclear cells (PBMCs), offering a non-invasive method for patient selection and stratification in clinical trials.

Acurex's proprietary ELISA-based Miro1 biomarker is poised to become the first drug-responsive PD biomarker. Measuring this biomarker in PBMCs from PD patients may enable the early detection of biochemical evidence of disease modification.

Mitophagy Drug Discovery Platform (M3D)

Acurex uses it's high-throughput mitochondrial dysfunction drug discovery platform (M3D) to identify drug targets. Mitochondrial dysfunction and the process of recycling damaged mitochondria (mitophagy) play an early and important role in the development of many diseases, such as Parkinson's disease.

Acurex's drug discovery approach, using the phenotypic screening of Miro1 status, has pinpointed dysfunction in specific signaling pathways as key drivers of mitophagy defects. Miro1 is essential for neuronal health and functions such as mitochondrial trafficking, regulating mitochondrial-endoplasmic reticulum contact sites, calcium buffering, and recycling dysfunctional mitochondria.

Acurex has developed a drug discovery platform that identifies drug targets that can reverse or improve mitophagy dysfunction across various diseases and cell types. To date, our platform has demonstrated consistent utility across different genetic backgrounds and cell types in individuals with PD.