Ferrer to co-develop, market PIKfyve inhibitor VRG50635

Pact covers Europe, Central and South America, Southeast Asia, Japan

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Spanish pharmaceutical company Ferrer will co-develop and market Verge Genomics’ investigational PIKfyve inhibitor VRG50635 for the treatment of amyotrophic lateral sclerosis (ALS) in Europe, Central and South America, Southeast Asia, and Japan, under a collaboration agreement announced by the two companies.

Ferrer will have exclusive co-development and commercialization rights to VRG50635 for the sporadic and familial forms of ALS in those regions. Verge will retain the rights to using the medication for all indications in the U.S. and other countries not covered by the agreement.

“We’re thrilled to work with Ferrer to progress VRG50635 through clinical development and towards potential commercialization,” Alice Zhang, CEO and co-founder of Verge, said in a company press release.

“Ferrer has extensive experience navigating clinical development and regulatory landscapes across the globe,” Zhang said. “They also understand the complex and variable payer and reimbursement environments in the territories in which we will collaborate.”

VRG50635 is an experimental oral therapy designed to slow ALS progression and potentially extend survival by boosting nerve cells’ ability to clear the toxic protein clumps that accumulate in virtually all ALS cases.

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PIKfyve inhibitor, lysosome booster

The therapy, identified through Verge’s CONVERGE artificial intelligence platform, works by blocking the PIKfyve protein and increasing the number of lysosomes in cells. Lysosomes are cellular organelles that break down faulty or unwanted molecules. They are essential to prevent the accumulation of toxic proteins, and are usually faulty in ALS.

The collaboration is “important news for people living with ALS, their families, and their caregivers,” said Mario Rovirosa, CEO of Ferrer. “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.”

In preclinical studies, VRG50635 showed success in extending the survival of nerve cells from ALS patients and in models of motor neuron degeneration. It also was deemed safe at varying doses in healthy volunteers who took part in a Phase 1 clinical trial (ISRCTN14792372).

A proof-of-concept Phase 1b study (NCT06215755) is now enrolling people with sporadic and familial types of ALS to assess the safety and tolerability of VRG50635 in patients.

The trial, sponsored by Verge, expects to enroll 50 adult participants at sites in Canada and several European countries. Those taking part in the trial will receive multiple, ascending doses of the oral medication, followed by long-term treatment.

While the trial’s main goal is to determine the treatment’s safety, researchers will also look at secondary measures including changes in disease progression rates, levels of the nerve damage biomarker neurofilament light chain (NfL), and the treatment’s pharmacological properties.

Verge will also use its digital technology to objectively evaluate disease-relevant changes, including changes in speech and language, in patients.

“We believe VRG50635 represents a promising new approach to treating this devastating disease, and we look forward to combining our strengths to accelerate the development of this potential treatment, said Oscar Pérez, chief scientific and business development officer at Ferrer.