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Ferrer and Verge Genomics Sign Agreement for Clinical Development of ALS Therapy

Orange identification cord on a table, with the "ferrer" logo printed in white and a metal clip in the foreground.

Verge Genomics, a leading biotechnology company in clinical development, and Ferrer, a certified international B Corp pharmaceutical company, with a growing focus on rare neurological disorders and vascular and interstitial lung diseases, have announced a strategic collaboration for the development of VRG50635, a candidate drug 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 small molecule potentially inhibiting PIKfyve, a therapeutic target against ALS discovered in diseased tissues using CONVERGE,® an AI-based Verge Genomics platform that uses human tissue data. 

 

This collaboration agreement combines the technology developed by the US company for drug discovery and development and its leadership in clinical trial innovation with Ferrer's global experience in clinical development, manufacturing and commercialization of medicines. Under the terms of the agreement, Ferrer will obtain exclusive rights to develop and commercialize ALS VRG50635 in various regions outside of the United States. In addition, Verge Genomics will retain the same rights for all uses of the molecule in the United States and in all countries that are not covered by this alliance. 

 

"We are very pleased to be working with Ferrer to advance clinical development and potential commercialization of VRG50635. Ferrer has extensive experience, both in these phases and in the regulatory field around the world, in addition to understanding and knowing very well the complex payment and reimbursement environments in the territories foreseen in the collaboration," said Alice Zhang, CEO and co-founder of Verge Genomics. "This agreement represents another clear recognition of the value of our CONVERGE® platform and its ability to successfully identify therapeutic targets for complex diseases that can be quickly translated to the clinical development phase."

 

"We are delighted to collaborate with Verge Genomics because it is very important news for people living with ALS, their families and their caregivers. In line with our purpose of using the business to fight for social justice, in this way we want to consolidate our commitment to offer transformative therapeutic solutions to people living with serious and debilitating diseases," said Mario Rovirosa, CEO of Ferrer.

 

"We believe VRG50635 represents an entirely new and promising approach to treating this very serious disease, and we hope that with this collaboration we can accelerate the development of a potential treatment. In addition, this new therapy represents a very significant addition to our growing portfolio of treatments for rare neurological disorders, which revalidates our firm commitment to the scientific community and people with ALS," explained Oscar Pérez, Chief Scientific Officer of Ferrer. 

 

VRG50635 is one of the first drugs discovered and developed from an AI-based platform to be a candidate for use in a clinical trial. Specifically, it is an orally bioavailable PIKfyve inhibitor that could improve the survival of neurons in ALS patients.  Inhibition of PIKfyve has demonstrated positive results in preclinical studies carried out in models of motor neuronal degeneration relevant to this disease1,2. Currently, VRG50635 is undergoing a Proof of Concept (PoC) in phase Ib in Canada and different European countries3. This study, with an advanced method and design, incorporates an innovative technology that collects relevant data on the disease to adequately evaluate the safety, tolerability, pharmacological response to different doses and potential efficacy of the drug and even the modification it could exert on the disease during the early phases of clinical development.  

 

About Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis (ALS), the most common neuromotor disease, is a neurodegenerative disorder of the motor neurons of the brain and spinal cord, progressively causing paralysis, until death, usually 2 to 5 years after diagnosis. ALS is a rare disease with a multifactorial etiology whose pathogenic mechanism is not yet precisely understood. ALS typically appears in people between 40 and 70 years of age, being more frequent in men than in women, and a distinction is made between two types: familial ALS, which affects 10% of patients, and sporadic ALS, which affects 90% of them4.

 

References:

  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). Accessed March 2024. Available in: 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.