-- PIKfyve Inhibitor Has Potential as a Prophylactic and Preemptive Therapy for SARS-CoV-2 or Other Novel Coronaviruses

-- Abstract Awarded “Top Rated Poster”

-- Data to Be Featured at Fireplace Session on July 12^th

Verge Genomics, a biotech company that has created an industry leading all-in-human, artificial-intelligence-powered drug discovery and development platform focused on therapies for serious genetic diseases, today announced that data from preclinical studies using its novel PIKfyve inhibitor VRG101 showed broad spectrum of antiviral activity. The results will be presented at the 31st European Congress of Clinical Microbiology & Infectious Diseases (ECCMID) online meeting being held July 9-12, 2021.

“There remains an urgent need for oral antiviral treatments for SARS-CoV-2 and its variants,” said Alice Zhang, CEO and Co-Founder of Verge Genomics. “SARS-CoV-2 and other novel coronaviruses are expected to be with us for many years and finding antivirals to combat them is critical to improving public health now and in the future. Based on the results of this study, we believe advancing the development of VRG101 as a prophylactic and preemptive therapy in SARS-CoV-2 patients, and future novel human coronaviruses, is a compelling opportunity.”

Details of Poster Presentation:

TITLE: Novel PIKfyve Inhibitor has Broad Spectrum Antiviral Activity in Preclinical Experiments

ABSTRACT: 04776

PRESENTATION: Monday, July 12, 2021, 16:00-17:00 CEST

FIRESIDE SESSION: Monday, July 12, 2021, 18:15-19:15 CEST

PRESENTER: Michelle I. Mighdoll

AWARDED: Best Rated Poster Award, based on rating by peer reviewers (approximately one percent of submitted abstracts)

About the Preclinical Study

Verge has demonstrated potent antiviral activity in vitro and improvement of SARS-CoV-2-induced pathology in vivo by the PIKfyve inhibitor, VRG101. PIKfyve, a lipid kinase important in endolysosomal maturation, has been reported to be required for SARS-CoV-2 entry into the cell in vitro. VRG101 shows robust antiviral efficacy in vitro against SARS-CoV-2 in multiple cell lines (Vero-E6: EC50 = 0.353 μM, CC50 >100 μM, SI = 283; A549-hACE2: EC50 = 0.0319 μM, CC50 >1000 μM). VRG101 also shows antiviral activity against other viral strains (beta-coronavirus 1 OC-43 in 16HBE/MRC5: EC50 = 0.22 μM, CC50 <10 μM; alpha-coronavirus 229E in 16HBE/MRC5: EC50 = 3.16 μM, CC50 <10 μM), indicative of its potential as a broad-spectrum antiviral. In an in vivo study of SARS-CoV-2 infected hamsters, following four-day treatment, animals treated with VRG101 at 50 mg/kg showed significant improvement in lung pathology compared to vehicle controls. Importantly, VRG101 has a favorable pharmacokinetic profile.

About PIKfyve

Verge originally discovered the relevance of PIKfyve in amyotrophic lateral sclerosis (ALS) using its computational platform, which incorporates large genomic and transcriptomic data sets from patients with the disease. After demonstrating that deficient PIKfyve machinery contributes to ALS, Verge 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.

About Verge Genomics

Verge is focused on developing therapeutics for serious genetic diseases using human genomics and machine learning. Verge has created a proprietary all-in-human platform, featuring one of the field’s largest and most comprehensive databases of neurodegenerative 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 www.vergegenomics.com. Follow us on LinkedIn and Twitter.

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