Verge Genomics and Ferrer Announce Agreement to Co-Develop Clinical-Stage ALS Therapy VRG50635
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.
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). 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.
To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.
View source version on businesswire.com: https://www.businesswire.com/news/home/20240325739095/en/
Contact information
Verge Genomics
Adam Silverstein, Scient PR
adam@scientpr.com
Ferrer
Carlo Ferri, Communication Advisor
cferri@ferrer.com
About Business Wire
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Daiichi Sankyo Shines a Light on Inequalities and Unmet Needs in Care as Part of Its Commitment to People With Cardiovascular Disease31.8.2024 18:00:00 EEST | Press release
Daiichi Sankyo Europe, (hereafter, Daiichi Sankyo) today announced its reinforced commitment to addressing key unmet needs and barriers in cardiovascular (CV) care, by shining a light on inequalities in treatment and outcomes experienced by various patient groups. The announcement follows the presentation of data from sub-analyses of the SANTORINI observational study and ETNA-AF (Edoxaban Treatment in routiNe clinical prActice in patients with nonvalvular Atrial Fibrillation) study programme, at the European Society of Cardiology (ESC) Congress 2024. Gender inequalities in CV disease treatment and LDL-C goal attainment Raised low-density lipoprotein cholesterol (LDL-C) is a key modifiable contributor to risk of cardiovascular major events. It has been shown that for every 1 mmol/L reduction in LDL-C, there is a 22% reduction in major cardiovascular events after 1 year.7,8 Female patients in Europe who are at high or very high CV risk were undertreated and less achieved guideline-recomm
Suzano completes purchase of 15% stake in Lenzing AG31.8.2024 01:40:00 EEST | Press release
Suzano, the world's largest pulp producer, today announces the completion of its acquisition of a 15% stake in Lenzing AG, the Vienna-listed leading global supplier of sustainable premium cellulosic fibres for the textile and non-wovens industry, from B&C Group. As announced in June this year, the transaction value is EUR 230 million (or EUR 39.70 per share). The agreement was subject to regulatory approval, which was granted without restrictions. The long-term partnership between Suzano and B&C Group establishes that Suzano has the right to hold two seats on Lenzing’s Board and the option to acquire an additional 15% stake from B&C Group until the end of 2028. Following the sale of the 15% stake, B&C Group now holds 37.25% of Lenzing’s share capital. Lenzing is a leading global supplier of premium wood-based cellulosic fibers (lyocell, modal, and viscose) for the production of clothing, home textiles, hygiene products, and non-woven materials. Beto Abreu, CEO of Suzano, said: “Lenzing
Ant International Collaborates with Brazilian Partners to Drive Financial Inclusion with Innovative Credit Tech Solutions30.8.2024 21:46:00 EEST | Press release
Ant International, a digital payment and financial technology leader, today announced its strategic initiative to provide AI-driven credit technology solutions designed to empower local partners and advance financial inclusions in Brazil. As a major milestone, Ant International has formed a strategic partnership with Dock, a leader in payments and banking technology in Latin America. This collaboration aims to enhance the credit management capability and infrastructure efficiency for Dock’s clients, which include financial institutions, retailers, fintech start-ups, etc. The shared vision is to make credit services more inclusive for market segments that are currently insufficiently served, such as micro, small and medium-sized enterprises (MSMEs). In the initial phase of the collaboration, Ant International’s AI-driven credit tech solutions, including an automated and intelligent risk management engine, will be explored for integration into Dock One platform. This initiative aims to s
Wipro Appoints Srikumar Rao as Global Head of Its Engineering Edge Business Line30.8.2024 17:20:00 EEST | Press release
Wipro Limited (NYSE: WIT, BSE: 507685, NSE: WIPRO), a leading technology services and consulting company, today announced the appointment of Srikumar Rao as Global Head of its Engineering Edge Business Line, effective from October 5, 2024. Srikumar succeeds Harmeet Chauhan, who is stepping down to pursue opportunities outside the organization. Srikumar will report to Srini Pallia. Srikumar has been with Wipro for 26 years and has grown with the organization, serving in various leadership roles. He was most recently the Chief Operating Officer (COO) of Wipro Engineering Edge (WEE) and Global Practice Head of Embedded Systems. His work has spanned several markets, selling and delivering specialized Engineering, Research, and Development (ER&D) services to Software & Internet, Semiconductor, Automotive, Manufacturing, and Communications clients. In his new role, Srikumar will continue to scale and strengthen Wipro’s engineering prowess. He will also be responsible for defining the vision
Alnylam Presents Detailed Results from the Positive HELIOS-B Phase 3 Study of Vutrisiran▼ in Patients with ATTR Amyloidosis with Cardiomyopathy at the European Society of Cardiology Congress30.8.2024 13:00:00 EEST | Press release
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced detailed results from the HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM). The data were presented today in a Hot Line session at the European Society of Cardiology (ESC) Congress 2024, taking place August 30-September 2 in London, United Kingdom. Results from the HELIOS-B study were also simultaneously published in TheNew England Journal of Medicine. As previously reported, the HELIOS-B study met all 10 of its primary and secondary endpoints, across both the overall and monotherapy populations, with statistical significance. Enrolled patients were predominantly New York Heart Association (NYHA) Class I or II with wild-type disease and had been diagnosed by non-invasive methods, with substantial concurrent treatment with available standard of care treatments such as tafamidis and
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.
Visit our pressroom