VRG50635 is one of the first drugs to enter clinical trials that was entirely discovered and developed using an AI-enabled platform

SOUTH SAN FRANCISCO, CA, USA & BARCELONA, Spain I March 25, 2024 I Verge Genomics, a leading clinical-stage biotechnology company, and Ferrer, an international B Corp pharmaceutical company with an increasing focus in rare neurological disorders, have announced a strategic collaboration to co-develop 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 best-in-class, small molecule inhibitor of PIKfyve, a therapeutic target for 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 and their leadership in clinical trial innovation 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.

“We’re thrilled to work with Ferrer to progress VRG50635 through clinical development and towards potential commercialization. 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,” said Alice Zhang, CEO and cofounder of Verge Genomics. “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.”

“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”, said Mario Rovirosa, CEO at Ferrer.

“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. This is a very significant addition to our growing portfolio of treatments for rare neurological disorders while revalidating our strong commitment to the scientific community and the patients suffering from ALS”, explained Oscar Pérez, Chief Scientific Officer at Ferrer.

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 degeneration1,2. Currently, VRG50635 is undergoing a Phase 1B Proof-of-Concept (PoC) study in Canada and several European Countries3. Verge’s PoC study, which has been applauded for its cutting-edge design, 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.

About Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (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 40-70 years old, slightly more men than women. It is caused by a multitude of factors: 10% familiar ALS, 90% sporadic ALS4.

About Verge Genomics
Verge is focused on developing therapeutics for complex diseases with high unmet need, using human genomics from patient disease tissues and machine learning. Verge has created a proprietary all-in-human CONVERGE® platform, featuring one of the field’s largest and most comprehensive databases of multi-omic patient data. The company is led by experienced computational biologists and drug developers who are successfully advancing clinical and preclinical therapeutic programs in various diseases, including ALS and Parkinson’s disease. For additional information, please follow us on LinkedIn and Twitter.

About Ferrer
At Ferrer we use business to fight against social justice and meet the highest standards of social and environmental performance, transparency, and corporate responsibility. That is why in 2022 we became a B Corp company. In order to fulfil our purpose, we offer transformative therapeutic solutions, with an increasing focus on pulmonary vascular and interstitial lung diseases and rare neurological disorders. We are Ferrer. Ferrer for good.


1 Shi Y, Lin S, Staats KA, Li Y, Chang W-H, Hung S-T et al. Haploinsufficiency leads to neurodegeneration in C9ORF72 ALS/FTD human induced motor neurons. Nat Med. 2018 Mar;24(3):313-325.

2 Hung ST, Linares GR, Chang WH, Eoh Y, Krishnan G, Mendonca S et al. PIKFYVE inhibition mitigates disease in models of diverse forms of ALS. Cell. 2023 Feb 16;186(4):786-802.e28.

3 ClinicalTrials.gov. Identifier NCT06215755, A Study of VRG50635 in Participants with Amyotrophic Lateral Sclerosis (ALS). Consultado en marzo 2024. Disponible en: https://www.clinicaltrials.gov/study/NCT06215755?intr=VRG50635&rank=2

4 Masrori P, Van Damme P. Amyotrophic lateral sclerosis: a clinical review. Eur J Neurol. 2020;27(10):1918-1929.

SOURCE: Ferrer