PIKFYVE

Verge Genomics has touted VRG50635 as “one of the first drugs to enter clinical trials that was entirely discovered and developed using an AI-enabled platform.” Spanish pharma Ferrer is following in Big Pharma’s footsteps by targeting the AI-powered neuroscience R&D work of Verge Genomics. Ferrer has signed off on over 112.5 million euros ($121.7 million) in combined upfront and potential development and commercial milestone payments for the ex-U.S. rights to Verge’s clinical-stage amyotrophic lateral sclerosis (ALS) drug VRG50635. Verge is also entitled to a slice of the royalties should VRG50635 make it to market. Verge is currently assessing the PIKfyve inhibitor in a phase 1b proof-of-concept trial for the treatment of sporadic and familial forms of ALS. Under the agreement, Ferrer will acquire the rights for the drug in Europe, Central and South America, Southeast Asia and Japan, while Verge will retain the license for the U.S. and other territories. VRG50635 will fill an ALS-shaped hole in Ferrer’s pipeline that was created in January when the company’s oral formulation of edaravone—sold by Mitsubishi Tanabe Pharma as Radicava—failed a phase 3 trial. “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,” Ferrer’s chief scientific and business development officer Oscar Pérez said in a March 25 release. Verge has touted VRG50635 as “one of the first drugs to enter clinical trials that was entirely discovered and developed using an AI-enabled platform.” Verge’s CEO Alice Zhang said today’s licensing deal was “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're thrilled to work with Ferrer to progress VRG50635 through clinical development and towards potential commercialization,” Zhang added. “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.” Verge has had plenty of external interest in its R&D work. In September 2023, AstraZeneca signed a $42 million upfront deal to find new targets for rare neurodegenerative diseases. The biotech also inked high-profile deals with Eli Lilly and Merck & Co. back in 2021. While VRG50635 has already entered the clinic, Verge’s Parkinson’s disease, schizophrenia and frontotemporal dementia programs remain in preclinical development.

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, Calif. and BARCELONA, Spain, March 25, 2024 (GLOBE NEWSWIRE) -- 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 co-founder 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 and Business Development 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 degeneration. Currently, VRG50635 is undergoing a Phase 1B Proof-of-Concept (PoC) study in Canada and several European Countries1. 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 ALS2. 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 onLinkedIn and Twitter. About Ferrer At Ferrer we use business to fight for 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. 1ClinicalTrials.gov. Identifier NCT06215755, A Study of VRG50635 in Participants With Amyotrophic Lateral Sclerosis (ALS). Accessed March 2024. Available from: 2Masrori P, Van Damme P. Amyotrophic lateral sclerosis: a clinical review. Eur J Neurol. 2020;27(10):1918-1929. Contact: Verge Genomics Adam Silverstein, Scient PR adam@scientpr.com Ferrer Carlo Ferri, Communication Advisor cferri@ferrer.com

Feb. 15, 2024 -- OrphAI Therapeutics Inc. (“OrphAI”), a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced today that it has received Orphan Drug Designation (ODD) in the European Union (EU) for the treatment of amyotrophic lateral sclerosis (ALS). AIT-101 previously received ODD from the U.S. FDA in June 2023 for the treatment of ALS. “OrphAI Therapeutics now has orphan designation for AIT-101 both in the US and the EU for the treatment of ALS, an important step toward bringing the drug to patients suffering from this neurodegenerative disorder,” said Dr. Brigette Roberts, CEO and Director of OrphAI. “AIT-101 has demonstrated compelling activity in its ability to clear toxic aggregates, which are a hallmark of ALS and other neurodegenerative diseases. We look forward to moving AIT-101 into the next stage of clinical development." AIT-101 is a first-in-class, potent and highly selective inhibitor of the lipid kinase PIKfyve. Inhibition of PIKfyve leads to activation of the transcription factor TFEB which drives the increased clearance of toxic protein aggregates via the lysosomal autophagy pathway. AIT-101 has now been demonstrated to reduce aggregates in human subjects, to improve the survival of motor neurons in in vitro models of familial and sporadic ALS, and to ameliorate functional deficits in a mutant TDP-43 animal model of ALS. AIT-101 is the most clinically advanced PIKfyve inhibitor in development. OrphAI Therapeutics’ mission is to transform the lives of patients with rare diseases. The company is currently developing three investigational therapies across multiple orphan indications: AIT-101, a first-in-class PIKfyve inhibitor, which recently completed a Phase 2a clinical trial in amyotrophic lateral sclerosis (ALS); LAM-001, a proprietary inhaled form of rapamycin, currently in Phase 2 for Group 1 and 3 pulmonary hypertension and bronchiolitis obliterans syndrome (BOS); and AIT-102, a proprietary analogue of mithramycin, in preclinical development, for the treatment of SWI/SNF mutated or dysregulated tumors. The content above comes from the network. if any infringement, please contact us to modify.