AstraZeneca's Alexion Enters AI-Driven Genomics Collaboration with Verge Genomics to Discover Novel Targets for Rare Neurological Diseases

Sep 10, 2023 16:00 CST Updated 16:00

Verge Genomics

Developer of Neurodegenerative Disease Therapies

Alexion

Developer of New Drugs for Rare Disease Treatment

AstraZeneca

Biopharmaceutical Manufacturer

On September 8, Verge Genomics announced a four-year partnership with Alexion, AstraZeneca Rare Disease, to identify new drug targets.

Verge Genomics is a biotechnology company that uses artificial intelligence to develop therapies for neurological diseases. As part of the agreement,Verge Genomics to Receive Up to $42 Million in Prepayments, Equity, and Near-Term Payments.

The company may also receive up to$840 million in milestone payments, as well as any royalties arising from the products generated thereof.

The partnership aims to identify and validate new drug targets for rare neurodegenerative and neuromuscular diseases.

The two companies plan to achieve this by using Verge Genomics' technology, which uses artificial intelligence and human tissue data to predict which drug targets are most likely to succeed in clinical trials.

AstraZeneca now also holds shares in Verge Genomics.

Favored by multiple MNCs

Verge Genomics was launched in 2015 by founder Alice Zhang.(English name: Zhang Xinli)Born in Virginia to Chinese immigrant parents, Alice Zhang developed an interest in biomedicine from a young age. After graduating from high school, she pursued an undergraduate degree in molecular biology at Princeton University, followed by a Ph.D. in neuroscience at the University of California, Los Angeles.

Alice Zhang, Founder of Verge Genomics

Verge Genomics focuses on neurodegenerative diseases, including Amyotrophic Lateral Sclerosis (ALS), Parkinson's Disease (PD), and Frontotemporal Dementia (FTD), which are clinically unmet and complex conditions.

As of now,Verge Genomics has raised over $100 million from various investors, including asset management company Blackrock and major pharmaceutical companies such as Eli Lilly and Merck & Co.。Verge Genomics uses machine learning to analyze genomic data from human tissue samples or cell models.

In August 2022, Al Sandrock, a former executive of Biogen, joined Verge Genomics. He had led the development of Biogen's Alzheimer's drug Aduhelm.

Four Years, from Target to Clinical

Due to the complexity of the nervous system, animal models have shown poor efficacy in predicting outcomes for neurodegenerative disease clinical trials, leading to the vast majority of drugs failing miserably after entering clinical stages.

In the view of Verge Genomics, these reasons are all due to a lack of comprehensive understanding of diseases and failure to identify optimal drug targets for the best molecules. However, the advent of genomics now allows scientists to study entire organisms through genetic material, providing a more comprehensive understanding of disease biology than ever before, without relying on guesswork or assumptions.

Based on these considerations, the company created a name called"All Humanity"（all-in-human）The method for drug discovery involves abandoning data derived from animal models in the past, opting instead to build their own database specifically targeting neurodegenerative diseases.

Thus, Verge Genomics has established partnerships with more than a dozen institutions, including hospitals, academic centers, and biobanks, building a network of over6,800 human tissue samples(Mainly the brain and spinal cord)The human dataset, in addition to including information on gene expression, genotyping, and patient characteristics.(such as age, demographics, disease progression)...and other types of data, including one of the largest proprietary genomic datasets of human brain tissue in this field.

Human tissue sections collected by Verge Genomics

Due to the vast amount of data, scientists are unable to manually extract insightful information from the massive datasets, so they must rely onArtificial IntelligenceThe method involves using a "training" dataset of known connections between DNA sequences, gene expression patterns, and diseases to learn how to uncover new and powerful biological associations in datasets of interest.

Since scientists did not make assumptions about these algorithms and relied solely on patient data for various analyses, they are also referred to as "hypothesis-free" or "unbiased" algorithms.

Ultimately, Verge Genomics' algorithm identified a network of 200 genes that are consistently suppressed in ALS patients compared to those without the disease. These genes are involved in a biological process known as the lysosomal pathway, which clears cells of the protein aggregates seen in patients with neurodegenerative diseases.

So they identified one namedPIKfyve Phosphoinositide Kinase: A Novel Target for ALS Patients, It plays a key role in the endosomal and lysosomal systems, regulating membrane homeostasis, endosomal trafficking, and autophagy, and is emerging as a target for cancer, viral infections, and neurodegenerative diseases.

PIKfyve as a Drug Target Signaling Pathway

Verge Genomics Designs PIKfyve Inhibitor VRG50635 Targeting This Pathway, Which Restores Endolysosomal Function in ALS Patient Neurons and Demonstrates Efficacy in Multiple Preclinical Studies of ALS-Related Motor Neuron Degeneration Models. At the End of October 2022, Verge Genomics Announced the First Dosing of This Drug in Humans.

Verge Genomics' Clinical Pipeline

Verge Genomics' series of achievements have also drawn the attention of Eli Lilly.

In July 2021, Verge Genomics announced a three-year collaboration agreement with Eli Lilly to jointly develop innovative therapies for ALS. Verge will receive a $25 million upfront payment, with potential milestone payments and royalties worth up to nearly $700 million.

When Company B secured nearly 100 million US dollars in its Series B financing round, Eli Lilly also chose to invest, demonstrating the multinational giant's confidence in Verge Genomics.

Reference link:

https://www.globenewswire.com/news-release/2023/09/08/2739994/0/en/Verge-Genomics-Announces-Artificial-Intelligence-Enabled-Drug-Discovery-Collaboration-with-Alexion-for-Rare-Neurodegenerative-and-Neuromuscular-Diseases.html

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