Verge Genomics Announces Initiation of Proof-of-Concept Study for the Treatment of Amyotrophic ALS

Verge Genomics recently announced the initiation of its Phase 1b proof-of-concept study of VRG50635 for the treatment of sporadic and familial forms of amyotrophic lateral sclerosis (ALS). The study will assess the safety and tolerability of escalating doses of VRG50635, a potential best-in-class, small molecule inhibitor of PIKfyve, a therapeutic target for ALS discovered in diseased human tissues using CONVERGE, the company’s all-in-human, AI-powered platform. VRG50635 is one of the first drugs to enter the clinic that was entirely discovered and developed from an AI-enabled platform.

In traditional ALS clinical trials, a patient’s progress is measured in a clinical setting using poorly responsive physician rating scales, which typically provide sparse data to establish proof of concept early in clinical development. In contrast, Verge’s study harnesses the power of digital devices to collect thousands of objective, quantitative, relevant measurements directly from the patient, including mobility, breathing, and sleeping all from the comfort of their home. This level of patient data greatly increases the ability to detect disease progression and treatment-related changes, allowing to personalize the assessment of change for each study participant as he/she moves from no treatment to increasingly higher doses of VRG50635, and for longer treatment periods.

“Incorporating cutting-edge technology into our ALS proof-of-concept study is a necessity born from the urgent unmet need to efficiently study promising therapeutics for people battling this devastating disease. Our approach and the technology we are incorporating into the VRG50635 proof-of-concept study will make it possible to collect dense amounts of unbiased, objective disease-relevant data to properly assess the safety, tolerability, and potential efficacy, including dose-response, early in clinical development,” said Diego Cadavid, MD, FAAN, FANA, Chief Medical Officer at Verge Genomics.

“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,” added 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.”

“I applaud Verge for creating a bold proof-of-concept design that incorporates cutting-edge technology and scientific innovation in their efforts to create groundbreaking therapies for ALS,” said Dr. Angela Genge, Director, ALS Centre of Excellence for Research and Patient Care, Montreal Neurological Institute-Hospital.

VRG50635 is a potent, orally bioavailable PIKfyve inhibitor that 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. VRG50635 is the only PIKfyve inhibitor in clinical development that has been specifically optimized for the treatment of central nervous system disorders like ALS, with the potential to become a best-in-class therapy.

ALS, also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects motor neuron cells in the brain and spinal cord causing progressive paralysis of voluntary muscles. ALS often strikes people between the ages of 40 and 70, and it is estimated that 100,000 people worldwide may have the disease at any given time. ALS is fatal and there is currently no cure, and available therapies have only modest benefits for the majority of affected patients, who often succumb to the disease within 2 to 5 years after diagnosis.

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 more information, visit https://www.vergegenomics.com/.