Verge Genomics has forged a partnership with Ferrer, a rare neurological disorders-focused pharmaceutical company, to co-develop the former’s lead amyotrophic lateral sclerosis (ALS) drug candidate, VRG50635.

VRG50635 is an investigational small molecule inhibitor of ALS’ therapeutic target, PIKfyve. The target was found in diseased human tissues using Verge Genomics’ artificial intelligence (AI)-driven all-in-human CONVERGE platform.

VRG50635 is being developed for the treatment of sporadic and familial types of ALS in Europe, Central and South America, Japan, and Southeast Asia.

Under the partnership, Verge Genomics will contribute its all-in-human technology for target discovery and drug development along with its expertise in clinical trial innovation.

On the other hand, Ferrer will support the partnership with its international expertise in clinical development, manufacturing, and commercialisation.

According to the terms of the agreement, Ferrer will get the exclusive rights to co-develop and market VRG50635 for ALS in multiple regions excluding the US.

Verge Genomics will continue having all rights for development and commercialisation for the ALS therapy in the US and all countries outside the agreement.

Verge Genomics CEO and co-founder Alice Zhang said: “Ferrer has extensive experience navigating clinical development and regulatory landscapes across the globe. They also understand the complex and variable payer and reimbursement environments in the territories in which we will collaborate.

“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.”

VRG50635 is said to be one of the first drug candidates to reach the clinical stage that was founded and developed using an AI-enabled platform.

The oral PIKfyve inhibitor has demonstrated effectiveness in several preclinical trials in ALS-relevant models of motor neuron degeneration.

The results suggested its potential use to enhance neuronal survival in ALS patients, Verge Genomics said.

A Phase 1B proof-of-concept study for VRG50635 is now being conducted in Canada and several European countries.

Ferrer CEO Mario Rovirosa said: “We are pleased to partner with Verge Genomics as this is important news for people living with ALS, their families, and their caregivers.

“In line with our purpose to use business to fight for social justice, we are reinforcing our commitment to bring transformative therapeutic solutions for people living with severe and debilitating diseases.”