NLS Pharmaceutics Reports Promising Preclinical Parkinson's Treatment Results

NLS Pharmaceutics Ltd., a Swiss clinical-stage biopharmaceutical company, has announced promising preclinical results from several in vitro studies targeting alpha-synuclein (α-synuclein) and its A53T mutation. These studies demonstrate the potential of specific compounds in advancing the treatment of Parkinson's Disease (PD). The compounds in question are part of NLS's existing license agreement with Aexon Labs, Inc.

Alex Zwyer, CEO of NLS, highlighted the importance of these findings. He noted that the results, derived from the Aexon Labs' next-generation non-sulfonamide dual orexin agonist (DOXA) platform, provide valuable insights that could lead to more effective treatments for PD patients.

The key study involved using the "Alpha-Synuclein (α-synuclein) A53T Parkinson's Disease Genetic Cell-Based Agonist Neurite Outgrowth Assay" by Eurofins. This assay measured neurite outgrowth, a vital indicator of neuronal health and regeneration, through high-content imaging systems. The study aimed to assess the efficacy of AEX compounds in reducing α-synuclein aggregation or promoting its clearance, thus mitigating its toxic effects on neurons.

Significant findings from the study included:

1. AEX-23: This compound showed strong OX1R agonist activity and positive effects on neurite outgrowth at specific concentrations. These characteristics suggest it could modulate neuronal health by influencing α-synuclein dynamics, making it a potential therapeutic candidate for improving neuronal connectivity and resilience in PD.

2. AEX-19: At low concentrations, this compound demonstrated beneficial effects on neurite growth, OX1R agonist activity, and a moderate increase in Cathepsin D (CTSD) activity, indicating potential neuroprotective benefits for PD.

3. AEX-24: The compound was noted for its increase in CTSD activity and OX1R agonist activity, suggesting it might enhance α-synuclein degradation, thereby offering a potential therapeutic impact on PD.

The study's findings indicate that AEX-23 and AEX-19, which target the Orexin 1 Receptor (OX1R), hold promise for modulating the effects of α-synuclein on neurons. Both AEX-19 and AEX-24 also show potential due to their impact on CTSD activity, which could reduce α-synuclein aggregation. Further research, including in vitro and in vivo preclinical studies, is necessary to understand the precise mechanisms, optimize dosing, and evaluate the long-term efficacy and safety of these compounds in clinical settings.

Eric Konofal, MD, PhD, Chief Scientific Officer of NLS, also announced plans to develop two new chemical successors, AEX-230 and AEX-231. These new compounds will target various cathepsins involved in neurodegenerative disorders, aiming to enhance the understanding and treatment of neurodegenerative processes in PD.

NLS Pharmaceutics Ltd. is a global development-stage biopharmaceutical company focused on discovering and developing innovative therapies for patients with rare and complex central nervous system disorders. Headquartered in Switzerland, NLS collaborates with world-class partners and internationally recognized scientists. Founded in 2015, the company is led by an experienced management team with a proven track record in developing and commercializing product candidates.

Aexon Labs, Inc. is at the forefront of research into new compounds for neurodegenerative disorders, a category that includes diseases like Alzheimer's, Parkinson's, Huntington's, Narcolepsy, and Amyotrophic Lateral Sclerosis. These disorders currently have no cure and existing treatments often fail to address the root causes, lacking in therapeutic effectiveness and safety. Eric Konofal, MD, PhD, also serves as the president and founder of Aexon Labs, and both he and Alex Zwyer are shareholders in the company.

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