▎Edited by the WuXi AppTec content team

Recently, Verge Genomics announced the initiation of a Phase 1b proof-of-concept study for VRG50635 in treating both sporadic and familial amyotrophic lateral sclerosis (ALS). The study will evaluate the safety and tolerability of dose escalation of VRG50635. According to the press release,VRG50635 is one of the first drugs discovered and developed entirely by an artificial intelligence (AI) platform to enter clinical trials.。

Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, also known as "Lou Gehrig's disease."In traditional ALS clinical trials, patient disease progression is measured using physician rating scales in a clinical setting. These scales typically provide only sparse data, making it difficult to conduct proof-of-concept validation early in clinical development. In contrast, the study initiated by Verge GenomicsLeverage the power of digital devices to collect thousands of objective, quantitative relevant measurement data directly from patients, including their activities, breathing, and sleep conditions at home.This type of patient data greatly enhances the ability to detect disease progression and treatment-related changes. As each subject transitions from not receiving VRG50635 treatment to receiving increasing doses of VRG50635 over longer periods, researchers can conduct individualized assessments of their changes.

VRG50635 is a potent, orally available PIKfyve inhibitor that increases the survival rate of neurons in ALS patients and has demonstrated efficacy in multiple preclinical studies using models relevant to ALS-associated motor neuron degeneration.PIKfyve is an ALS treatment target discovered in diseased human tissues using Verge Genomics' AI platform, CONVERGE. VRG50635 is currently in clinical development, specifically optimized for central nervous system diseases such as ALS, and is expected to become a potential "best-in-class" therapy.

Image Source: 123RF

Verge Genomics' proprietary ConVERGE platform combines highly predictive human tissue datasets with machine learning to discover emerging targets with potentially higher clinical development success rates.Verge Genomics' drug discovery platform does not start with cell or animal models,But based on a proprietary library of genomic datasets derived directly from human tissue, combined with an advanced, human-centered biological platform that can rapidly advance new data insights into clinical candidate therapies.The ConVERGE platform aims to address diseases that are biologically challenging to tackle, including:

• Diseases ultimately triggered by the complex interactions of various factors such as genetics, environment, and time.

• Diseases for which human efficacy cannot be predicted through animal or cell models.

• Diseases with high unmet needs, no currently available therapies to alter the disease course, or significant defects in existing treatment modalities.

▲ Co-founder and CEO of Verge GenomicsDr. Zhang Xinli

Regarding the application of artificial intelligence in the pharmaceutical industry, Dr. Alice Zhang (Xinli Zhang), co-founder and CEO of Verge Genomics, stated in an interviewWuXi AppTec Exclusive InterviewShe pointed out that the "unbiased" artificial intelligence algorithm system is expected to establish a disease model that integrates multiple mechanisms, considering diseases from a broader perspective without human influence on the direction of research. In her view, "The more chaotic a field is, the more it could be a place where machine learning and artificial intelligence can make a significant impact. Machine learning can bring important differences in solving the most complex disease problems."To fully realize the potential of AI technology, we need to combine computational power with biology. This proof-of-concept study targeting PIKfyve is the best practice of this insight."

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