▎WuXiEdited by Kant Content Team
Today, Verge Genomics and Ferrer announced a strategic collaboration to co-develop Verge’s lead compound VRG50635 for the treatment of sporadic and familial amyotrophic lateral sclerosis (ALS) in Europe, Central and South America, Southeast Asia, and Japan.VRG50635 is a potential "best-in-class" small molecule inhibitor targeting PIKfyve.The press release pointed out,VRG50635 is one of the first drugs to enter clinical trials that was entirely discovered and developed by an artificial intelligence (AI) platform.
The collaboration combines Verge’s “all-in-human” technology platform for target discovery and drug development, along with its innovations in clinical trials, with Ferrer’s expertise in clinical development, manufacturing, and commercialization. Under the terms of the agreement, Ferrer will obtain exclusive rights to co-develop and commercialize VRG50635 for the treatment of ALS in multiple territories outside the United States. Verge retains all rights to develop and commercialize VRG50635 in the United States and all other countries and regions not covered by the agreement. According to the agreement, Verge is eligible to receive up to 112.5 million euros in upfront and potential milestone payments.
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 ALS-related motor neuron degeneration models.PIKfyve is an ALS treatment target discovered in diseased human tissues by Verge Genomics' AI platform, CONVERGE. Currently, VRG50635 is undergoing a Phase 1b proof-of-concept (PoC) study in Canada and several European countries. Verge’s PoC study employs innovative technology capable of collecting large amounts of unbiased, objective, disease-relevant data to accurately evaluate safety, tolerability, pharmacological dose-response, and potential efficacy in the early stages of clinical development.

▲ Co-founder and CEO of Verge GenomicsDr. Zhang Xinli
For the application of artificial intelligence in the pharmaceutical industry, Zhang Xinli (Alice Zhang) Ph.D. in receivingInterview with the WuXi AppTec Content TeamShe pointed out that the "unbiased" artificial intelligence algorithm systems hold the potential to establish disease models that integrate multiple mechanisms, considering diseases from a broader perspective without allowing human influence to steer the direction of research. In her view, "The more chaotic a field is, the more it may become 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.”




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[1] Verge Genomics and Ferrer Announce Agreement to Co-Develop Clinical-Stage ALS Therapy VRG50635. Retrieved March 25, 2024, from https://www.globenewswire.com/news-release/2024/03/25/2851439/0/en/Verge-Genomics-and-Ferrer-Announce-Agreement-to-Co-Develop-Clinical-Stage-ALS-Therapy-VRG50635.html[2] Ferrer signs $122M biobucks deal for ex-US license to Verge's phase 1 ALS therapy. Retrieved March 25, 2024, from https://www.fiercebiotech.com/biotech/ferrer-signs-122m-biobucks-deal-ex-us-license-verges-phase-1-als-therapyDisclaimer: The content team of WuXi AppTec focuses on introducing the research progress in global biopharmaceuticals and health. This article is for the purpose of information exchange only. The views expressed in the article do not represent the position of WuXi AppTec, nor does it indicate that WuXi AppTec supports or opposes these views. This article is not a recommendation for treatment plans. For guidance on treatment options, please visit a正规 hospital.Copyright Statement: This article is from the content team of WuXi AppTec. Individuals are welcome to share it on their social media circles, but unauthorized reproduction by media or institutions in any form to other platforms is prohibited. For authorization to reproduce, please reply with "reprint" on the "WuXi AppTec" WeChat Official Account to obtain the reprint guidelines.

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