Aptabio Research on Effective Parkinson’s Disease Treatment Published in International Journal

2023-11-07 04:49:00

Industry | 2023.11.07 14:19

[서울=뉴스핌] Reporter Lee Na-young = Aptabio, a developer of incurable anti-cancer treatments and diabetes complications treatments, announced on the 7th that research on the therapeutic effects of ‘brain disease treatment (APX-NEW)’ in animal models of Parkinson’s disease was published in an international academic journal.

Aptabio announced that the research results, “Excellent Parkinson’s disease treatment effect of APX-NEW (compound-11) in dopamine neurons and old rat alpha-synuclein overexpression Parkinson’s disease animal model,” were published in the International Journal of Molecular Sciences, an international academic journal. This study is the result of joint research with a research team from the University of Delaware, an expert in the field of Parkinson’s disease treatment.

It has been revealed that the core cause of Parkinson’s disease, the second most common degenerative brain disease following Alzheimer’s, is alpha-synuclein, but a fundamental treatment that can prevent the progression of the disease has still not been developed.

Aptabio logo. [사진=압타바이오]

Parkinson’s disease is a degenerative brain disease that affects cognitive function as well as movement disorders such as hand tremors and gait disorders as dopamine neurons in the substantia nigra (SN) of the midbrain gradually die and the concentration of dopamine decreases. The main pathological feature of this disease is the abnormal accumulation of alpha-synuclein in the nervous system, resulting in excessive accumulation of protein aggregates called Lewy bodies.

In a paper published in 2022, it was suggested that oxidative stress caused by NOX plays an important role in the onset and progression of Parkinson’s disease and that regulating NOX might treat Parkinson’s disease by suppressing alpha-synuclein, which is the cause of Parkinson’s disease.

In this paper, we presented the results of APX-NEW (Compound-11) research, which improved brain delivery efficiency and treatment effect through compound optimization research, and induced dopaminergic cells (N27) and aged alpha-synuclein-preformed fibrils (PFF). It was confirmed that the treatment effect for Parkinson’s disease was excellent in the Parkinson’s disease model.

APX-NEW (Compound-11) increased dopaminergic cell viability and reduced cytotoxicity at very low drug concentration (10 nM), and reduced reactive oxygen species (ROS) and protein aggregation (Thyflavine-T staining) in cells. decreased. Oral treatment in an elderly alpha-synuclein-preformed fibrils (PFF)-induced Parkinson’s disease model was found to be effective in protecting brain dopaminergic neurons, reducing alpha-synuclein reduction and protein aggregation, and improving motor dysfunction. It has been done. In addition, the inhibitory effect of NOX1,2,4 was confirmed in the striatum and ventral midbrain, including the substantia nigra of the brain.

As a result of this paper, NOX1,2,4 inhibition can be a treatment option for Parkinson’s disease, and APX-NEW (Compound-11) is evaluated to be a highly competitive drug for development as an oral Parkinson’s disease treatment.

An Aptabio official said, “In a market where antibody treatment research is mainstream, market interest arose in December when Novartis bet $150 million in down payment from UCB for an oral alpha-synuclein inhibitor candidate (UCB0599). “The situation is growing. We plan to further accelerate the pace of development so that APX-NEW can become the ‘first small molecule compound for oral use’ that inhibits alpha-synuclein.”

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