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Verge Genomics and Ferrer announce agreement for joint clinical-stage development of ALS therapy VRG50635

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VRG50635 is one of the first clinical-stage drugs discovered and developed entirely using an AI-powered platform

Verge Genomics, a leading clinical-stage biotechnology company, and Ferrer, a global pharmaceutical B Corp company with a growing focus on rare neurological disorders, have announced a strategic collaboration for the co-development of VRG50635, Verge’s lead drug candidate for the treatment of sporadic and familial forms of amyotrophic lateral sclerosis (ALS), in Europe, Central and South America, Southeast Asia and Japan. VRG50635 is a potential high-performance small molecule inhibitor of PIKfyve, a therapeutic target for ALS discovered in human tissue affected by disease using CONVERGE ® , Verge’s all-in-human AI-powered platform.

The collaboration combines Verge’s all-in-human technology for targeted drug discovery and development and their leadership in clinical trial innovation with Ferrer’s global expertise in clinical development, manufacturing and market launch. Under the terms of the agreement, Ferrer will acquire exclusive rights to collaborate on the development of VRG50635 for ALS and to commercialize this product in various regions outside the United States of America. Verge has retained all rights to develop and commercialize VRG50635 for all applications in the United States and all non-agreement countries.

“We are excited to work with Ferrer to advance VRG50635 through clinical development and work toward its potential market launch. Ferrer has extensive experience navigating clinical development and the regulatory landscape around the world. They also understand the complex and variable payment and reimbursement environments in place in the territories where we will collaborate,” said Alice Zhang , CEO and co-founder of Verge Genomics. “This partnership is another clear recognition of the value of our CONVERGE ® platform and its ability to more successfully identify novel targets for complex diseases that can be rapidly translated into medicine.”

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“We are excited to partner with Verge Genomics as this is important news for people affected by ALS, their families and their caregivers. In line with our purpose of using business to fight for social justice, we are strengthening our commitment to providing transformative therapeutic solutions to people living with serious, debilitating conditions,” said Mario Rovirosa , CEO of Ferrer.

“We believe VRG50635 represents a promising new approach to treating this devastating disease, and we look forward to joining forces to accelerate the development of this potential treatment. This is a very important addition to our growing portfolio of treatments for rare neurological diseases, while reinforcing our strong commitment to the scientific community and patients suffering from ALS,” explains Oscar Pérez , Chief Scientific Officer at Ferrer.

VRG50635 is one of the first clinical-stage drug candidates discovered and developed entirely by an AI-powered platform. It is a potent, orally bioavailable PIKfyve inhibitor that may improve neuron survival in ALS patients and its efficacy has been demonstrated in several preclinical studies in ALS-relevant models of motor neuron degeneration. Currently, VRG50635 is undergoing a Phase 1B Proof-of-Concept (PoC) study in Canada and several European countries. Acclaimed for its groundbreaking design, Verge’s PoC study integrates innovative technology that enables the collection of large amounts of unbiased, objective disease-relevant data to determine safety, tolerability, pharmacological dose response and potential efficacy, including modification of disease, to be correctly assessed at an early stage in clinical development.

SOURCE: BusinessWire

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