Verge Genomics has initiated a Phase Ib proof-of-concept study for the treatment of sporadic and familial forms of amyotrophic lateral sclerosis (ALS) with VRG50635.
VRG50635 is a small molecule PIKfyve inhibitor and is reportedly one of the first drugs to enter clinical trials that was entirely discovered and developed using an AI-enabled platform.
Verge’s proof-of-concept study uses a unique design and technology to deliver more precise and comprehensive insights into how VRG50635 may benefit individuals with ALS.
The study uses digital devices to collect thousands of objective, quantitative, relevant measurements directly from the patient, including mobility, breathing, and sleeping.
This level of patient data increases the ability to detect disease progression and treatment-related changes, allowing the researchers to personalise the assessment of change for each study participant.
ALS is a progressive neurodegenerative disease that affects motor neuron cells in the brain and spinal cord causing progressive paralysis of voluntary muscles.
VRG50635 improves survival in amyotrophic lateral sclerosis (ALS) patient neurons and has shown efficacy in multiple preclinical studies in ALS-relevant models of motor neuron degeneration.
“Initiating our proof-of-concept study marks another important milestone for Verge and further demonstrates that by leveraging our AI-enabled platform in combination with patient data using a novel study design we are able to more efficiently discover and develop potential drugs for deadly diseases like ALS,” said Alice Zhang, CEO and co-founder of Verge Genomics. “We are tremendously excited by the potentially transformative power of our CONVERGE platform in redefining the landscape and expediting the journey from lab bench to patients and improving lives worldwide.”