RARE Daily

Verge Genomics and Ferrer to Co-Develop Clinical-Stage ALS Therapy

March 25, 2024

Rare Daily Staff

Verge Genomics and Spanish biopharma Ferrer entered a strategic collaboration to co-develop VRG50635, Verge’s lead drug candidate for the treatment of sporadic and familial forms of amyotrophic lateral sclerosis in 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 amyotrophic lateral sclerosis (ALS) discovered in diseased human tissues using CONVERGE, Verge’s all-in-human, AI-powered platform.

The collaboration combines Verge’s all-in-human technology for target discovery and drug development with Ferrer’s global expertise in clinical development, manufacturing, and commercialization. Under the terms of the agreement, Ferrer will obtain the exclusive rights to co-develop and commercialize VRG50635 for ALS in multiple regions outside of the United States of America. Verge has retained all rights to development and commercialization for VRG50635 for all uses in the United States and all countries outside the agreement.

“This partnership is another clear recognition of the value of our CONVERGE platform and its ability to identify novel targets more successfully for complex diseases that can be rapidly translated to the clinic,” said Alice Zhang, CEO and cofounder of Verge Genomics.

ALS, the most common motor neuron disease, is a progressive neurodegenerative disease of motor neurons in the brain and spinal cord, resulting in progressive paralysis, with death typically within 2 to 5 years of diagnosis. ALS is a rare disease with multifactorial etiology, and the precise pathogenic mechanism is still unknown. ALS typically occurs in people between 40 and 70 years old, slightly more men than women and is caused by a multitude of factors with 10 percent familiar ALS and 90 percent sporadic ALS.

VRG50635 is one of the first candidate drugs to enter the clinic that was entirely discovered and developed from an AI-enabled platform. It is a potent, orally bioavailable PIKfyve inhibitor that could improve survival in ALS patient neurons and has shown efficacy in multiple preclinical studies in ALS-relevant models of motor neuron degeneration.

Currently, VRG50635 is undergoing a phase 1b proof-of-concept study in Canada and several European Countries and incorporates innovative technology that makes it possible to collect dense amounts of unbiased, objective disease-relevant data to properly assess safety, tolerability, pharmacological dose response, and potential efficacy, including for disease modification, early in clinical development.

Photo: Alice Zhang, CEO and cofounder of Verge Genomics

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