• VRG50635 was safe and well tolerated in healthy adult volunteers after both single and repeated administration, with favorable pharmacokinetics showing relevant exposures with once-daily oral dosing
• Verge anticipates initiating a proof-of-concept study in ALS patients in Q4 2023

Verge Genomics, a clinical-stage biotechnology company transforming drug discovery using artificial intelligence and human data, today announced the completion of the Phase 1 clinical trial of VRG50635 in healthy adult volunteers. VRG50635 was dosed up to the maximum single and repeated doses planned, demonstrating a favorable safety, tolerability, and pharmacokinetic profile supportive of advancement into a proof-of-concept study in people with amyotrophic lateral sclerosis (ALS). Detailed results from the study will be presented at the upcoming European Network to Cure ALS (ENCALS) 2023 meeting being held in Barcelona, Spain from July 12-14, 2023.

VRG50635 is a potential best-in-class small molecule inhibitor of PIKfyve for treatment of ALS. PIKfyve is a new therapeutic target for ALS discovered using CONVERGE™, the company’s all-in-human, AI-powered platform. VRG50635 is one of the first drugs entirely discovered and developed from an AI-enabled platform to enter clinical trials, and was advanced from discovery to clinic in just four years.

“We are extremely encouraged by these initial results which highlight the favorable safety and tolerability profile of VRG50635, and further validate the novel approach that Verge has taken to identify novel targets for complex diseases with high unmet need using human disease tissues and rapidly advancing compounds into proof-of-concept trials,” said Diego Cadavid, M.D. Verge’s Chief Medical Officer. “We look forward to sharing additional information at the ENCALS meeting in Barcelona next month and anticipate progressing to a proof-of-concept study in patients with sporadic and familial ALS later this year.”

The Phase 1 trial was a randomized, double-blind, placebo controlled, single and multiple ascending dose escalation study designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VRG50635 in 80 healthy adult volunteers.

Topline data from the study demonstrated:

• VRG50635 was safe and well tolerated with no serious adverse events.
• The compound was escalated to the highest single and multiple repeated doses planned.
• The pharmacokinetics showed dose-proportional increases in Cmax and AUC and a terminal half-life of approximately 37 hours for the active metabolite that supports once-daily oral dosing of VRG50635.
• The favorable data supports advancing VRG50635 into a proof-of-concept study in people with ALS.

“The lack of predictive animal models is one of the greatest challenges to developing effective ALS therapies today. This is why we’re excited to advance VRG50635, a drug derived directly from human data, into trials in people with ALS,” said Alice Zhang, CEO and co-founder of Verge Genomics. “Our innovative proof-of-concept ALS study is designed to overcome historical challenges in ALS clinical trials by using state of the art technology, such as digital at-home devices and blood-based biomarkers, that can capture richer, higher-fidelity patient data and have the potential to detect efficacy with greater sensitivity.”

About VRG50635

VRG50635 is a potent, orally bioavailable PIKfyve inhibitor that improves survival in ALS patient neurons and has shown efficacy in multiple preclinical studies in ALS-relevant models of motor neuron degeneration. VRG50635 is the only PIKfyve inhibitor in clinical development that has been specifically optimized for treatment of central nervous system disorders like ALS, and has the potential to become a best-in-class therapy.

About PIKfyve

Verge discovered PIKfyve as a novel therapeutic target in ALS using their AI-powered platform, CONVERGE™, which incorporates large multi-omic data sets directly from CNS tissues from people with the disease. PIKfyve is a kinase that is believed to regulate endolysosomal function within a variety of cells, including neurons. The endolysosomal pathway is a critical cellular process involved in protein homeostasis. Verge and other top research groups have shown that in ALS, this pathway is dysregulated, leading to motor neuronal death and disease progression. After the discovery of PIKfyve as a therapeutic target for ALS, Verge developed novel, orally bioavailable PIKfyve inhibitors optimized for CNS penetration that have been effective in reversing disease-relevant pathology in multiple preclinical models.

About Verge Genomics

Verge is focused on developing therapeutics for complex diseases with high unmet need, using human genomics from patient disease tissues and machine learning. Verge has created a proprietary all-in-human CONVERGE™ platform, featuring one of the field’s largest and most comprehensive databases of multi-omic patient data. The company is led by experienced computational biologists and drug developers who are successfully advancing clinical and preclinical therapeutic programs in various diseases, including ALS and Parkinson’s disease. For additional information, please follow us on LinkedIn and Twitter.

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