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Ferrer and Verge Genomics Partner to Co-Develop Clinical-Stage ALS Treatment, VRG50635

Monday, March 25, 2024

Verge Genomics, a leading clinical-stage biotechnology company, and Ferrer, a pharmaceutical company focusing on rare neurological disorders, have announced a collaboration to co-develop VRG50635, Verge’s lead drug candidate, for treating sporadic and familial forms of amyotrophic lateral sclerosis (ALS) in multiple regions including Europe, Central and South America, Southeast Asia, and Japan. VRG50635 is a potential best-in-class, small molecule inhibitor of PIKfyve, a therapeutic target for ALS discovered in diseased human tissues using CONVERGE®, Verge’s AI-powered platform.

This collaboration combines Verge's technology for target discovery and drug development with Ferrer's global expertise in clinical development, manufacturing, and commercialization. Under the agreement, Ferrer will have exclusive rights to co-develop and commercialize VRG50635 for ALS outside of the United States. Verge retains all rights for development and commercialization in the United States and other countries not covered by the agreement.

Alice Zhang, CEO and co-founder of Verge Genomics, expressed excitement about working with Ferrer and their experience in global clinical development and regulatory landscapes. Mario Rovirosa, CEO of Ferrer, emphasized their commitment to providing transformative therapies for severe diseases like ALS.

VRG50635 is one of the first candidate drugs entirely discovered and developed from an AI-enabled platform. It is a potent PIKfyve inhibitor undergoing a Phase 1B Proof-of-Concept study in Canada and several European countries. The drug's trial design aims to collect comprehensive data to assess safety, tolerability, and potential efficacy.

Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease affecting motor neurons in the brain and spinal cord, resulting in paralysis and typically death within 2 to 5 years of diagnosis. It predominantly affects individuals aged 40-70, with familial ALS accounting for 10% of cases and the remaining 90% being sporadic.



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