Verge Genomics to advance its inhibitors of PIKfyve for the treatment of ALS and COVID-19

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Verge Genomics announced this week at BIO International its lead program for amyotrophic lateral sclerosis (ALS) is the endosomal lipid kinase, PIKfyve, with plans to enter clinical trials next year. The Company has entered into an agreement with a major pharmaceutical company to screen its library of PIKfyve inhibitors for antiviral activity against SARS-CoV-2.

"The fact that the same PIKfyve inhibitors we have been developing for ALS have the potential to address COVID-19 and future novel outbreaks presents an exciting opportunity to contribute meaningfully to a near-term global health crisis." - Alice Zhang, CEO of Verge Genomics

Verge Genomics, a drug discovery company developing therapies for neurological diseases by integrating a unique all in-human genomic platform with machine learning, announced this week at the BIO International Convention Digital 2020 that their lead program for amyotrophic lateral sclerosis (ALS) is the endosomal lipid kinase, PIKfyve and they plan to enter the clinic next year. Additionally, the company is expanding the development of their potent, selective, orally bioavailable small molecule PIKfyve inhibitors to include the treatment of COVID-19.

Verge discovered the relevance of PIKfyve in ALS using their computational platform, which incorporates large genomic and transcriptomic data sets from patients with the disease. After demonstrating that the deficient PIKfyve machinery contributes to ALS, the company developed highly-specific PIKfyve inhibitors that have been shown to rescue multiple models of ALS. New scientific data shows that many viruses, including COVID-19, hijack that same PIKfyve pathway in order to enter cells and replicate.

“ALS is a devastating, rapidly progressive neurodegenerative disease for which there are few effective treatments. Finding a disease-modifying treatment has been the focus of much of Verge’s research and development,” said Alice Zhang, CEO of Verge Genomics, “The fact that the same PIKfyve inhibitors we have been developing for ALS have the potential to address COVID-19 and future novel outbreaks presents an exciting opportunity to contribute meaningfully to a near-term global health crisis. We are advancing our PIKfyve molecules toward the clinic and expect to begin testing in humans early next year.”

Inhibiting PIKfyve prevents COVID-19 infection in cells as well as blocking various other types of viral entry. Preliminary data from an academic lab has shown positive anti-viral effects of Verge PIKfyve inhibitors in a SARS-CoV-2 cell-based assay.

“After scientists recently published that apilimod, a known PIKfyve inhibitor, decreases COVID-19 infection, we knew we had to test our molecules since compared to apilimod, they have improved pharmacokinetic properties and drug-like characteristics,” said Dr. Jane Rhodes, CBO at the Company. “We are excited about having demonstrated anti-viral effects of Verge compounds and are aggressively pursuing further development of an anti-viral therapy.”

ABOUT THE ENDOLYSOSOMAL PATHWAY
The endolysosomal pathway is a critical cell process that clears the “garbage” within a cell. PIKfyve is an enzyme that regulates endolysosomal function within a variety of cells, including neurons. Verge and other top research groups have shown that in ALS, this “garbage” pathway is dysregulated, which leads to neuronal death and the progression of the disease.

In addition, viruses are able to co-opt PIKfyve in order to gain entry into host cells and replicate, including SARS-CoV-2. Inhibiting this target prevents COVID-19 infection in cells as well as blocking Ebola, MERS, SARS and Marburg virus entry.

ABOUT ALS
ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. ALS often strikes people between the ages of 40 and 70, and it is estimated there are about 16,000 Americans who have the disease at any given time.

ABOUT VERGE
Verge is focused on developing therapeutics for neurological diseases using human genomics to accelerate drug discovery. Verge has created a proprietary all-in-human platform, generating one of the field’s largest and most comprehensive databases of ALS and Parkinson’s Disease patient genomic data. The Company is led by experienced computational biologists and drug developers who are successfully advancing therapeutic programs in ALS and Parkinson’s disease toward the clinic. For additional information, please visit http://www.vergegenomics.com.

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