Verge Genomics and AstraZeneca’s Alexion Announce Nearly $900M Multi-Target Collaboration to Advance Therapies for Rare Neurodegenerative and Neuromuscular Diseases

▎WuXi

Edited by Kant Content Team

Verge Genomics today announced a multi-target collaboration with Alexion, a rare disease company under AstraZeneca, to identify novel drug targets for rare neurodegenerative and neuromuscular diseases.Under the terms of the four-year agreement, Verge Genomics will receive up to $42 million in funding, including an upfront payment, and potentially up to $840 million in milestone payments.

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 is based on a proprietary library of genomic datasets derived directly from human tissue, combined with an advanced, human-centered biology platform to rapidly advance new data insights into clinical candidate therapies.The ConVERGE platform aims to address diseases that are biologically challenging to tackle, including:

Due to the complex intersection of various factors (genetic, environmental, and temporal), overt disease eventually occurs.

Diseases for which animal or cell models cannot predict human efficacy.

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

▲Verge Genomics' R&D Pipeline (Image Source:

Verge Genomics

Official Website)

The platform has been validated by the successful advancement of Verge Genomics' lead project in treating Amyotrophic Lateral Sclerosis (ALS). In October last year, the company announced that the first subject had been dosed in a Phase 1 clinical trial for its investigational therapy VRG50635. This was achieved through the evaluation of over 11.4 million data points obtained from ALS patient tissue and genetic databases.The ConVERGE platform discovered that the loss of endosomal/lysosomal function is a new pathogenic mechanism in ALS and identified PIKfyve as a promising new therapeutic target.VRG50635 is a potent inhibitor targeting PIKfyve, capable of restoring endosomal/lysosomal function in neurons of ALS patients. It has demonstrated efficacy in multiple preclinical studies using models related to ALS-associated motor neuron degeneration.

The press release at the time pointed out,VRG50635 is the first PIKfyve inhibitor to enter clinical development. It has been specifically optimized for the treatment of central nervous system diseases, including ALS, and has "best-in-class" potential.

Image Source: 123RF

In this collaboration, the platform will be applied to identify innovative treatment targets for rare neurodegenerative and neuromuscular diseases. Alexion will select high-potential targets for each indication and advance successful targets through clinical development and commercialization.

Alice Zhang (Xinli Zhang), Ph.D., co-founder and CEO of Verge Genomics, mentioned in an exclusive interview with WuXi AppTec in 2018,The more chaotic a field is, the more likely it is to be an area where machine learning and artificial intelligence can make significant strides.Machine learning can make a significant difference in solving the most complex disease problems. The progress made by Verge Genomics in drug development for central nervous system diseases is the best validation of this insight.

▲Dr. Alice Zhang (Image Source: Verge Genomics Official Website)

According to the press release, this is Verge Genomics' second collaboration with a large pharmaceutical company. In July 2021, Verge Genomics entered into a three-year, over $700 million collaboration with Eli Lilly and Company to research and develop new therapies for amyotrophic lateral sclerosis.