AbbVie has entered into a global alliance with Belgian biotech company Galapagos NV to discover, develop, and commercialize novel combination therapies for cystic fibrosis (CF). The collaboration, potentially valued at $406 million, includes an initial upfront payment of $45 million from AbbVie, with up to $360 million in additional developmental, regulatory, and sales milestone payments.
The partnership will focus on developing oral drugs that address the main mutations in CF patients, particularly F508del and G551D, which affect the cystic fibrosis transmembrane regulator (CFTR) protein. Both companies aim to initiate Phase 1 clinical trials by the end of 2014.
"Galapagos is very pleased to join forces with AbbVie in this exciting new area of CF. Our programs in CF show promise. Partnering with AbbVie allows us to ramp up our commitment significantly, share development risk and expertise, and increase our chances of bringing best-in-class therapies to CF patients," said Onno van de Stolpe, Chief Executive Officer of Galapagos.
Targeting the Root Cause of Cystic Fibrosis
Cystic fibrosis is a hereditary disease affecting approximately 70,000 people worldwide. It is caused by defects in the gene encoding the CFTR protein, which regulates components of sweat, mucus, and digestive juices. Currently, there is no cure for the disease.
The collaborative research will combine technologies and resources from both companies to develop compounds that correct defects in expression of and/or increase the activity of the main mutations in the CFTR protein.
Jim Sullivan, Ph.D., Vice President of Pharmaceutical Discovery at AbbVie, emphasized the significance of the partnership: "We're pleased to enhance our partnership with Galapagos to include research in cystic fibrosis, a debilitating disease with significant unmet medical need. Our knowledge of the patient experience, combined with innovative advances in the understanding of disease etiology, offers the potential for new transformational treatments."
Growing Industry Commitment to CF Research
This alliance represents part of a broader industry trend of increased investment in cystic fibrosis research. In a separate development, the Cystic Fibrosis Foundation Therapeutics (CFFT), the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation, recently announced a major expansion of its research collaboration with Pfizer.
The CFFT will invest up to $58 million in a new six-year pre-clinical research program with Pfizer, aiming to accelerate the discovery and development of potential therapies targeting patients with the Delta F508 mutation, the most common mutation in cystic fibrosis. The program's goal is to advance one or more drug candidates into clinical trials by the end of the six-year collaboration period.
"We are excited to expand our efforts with Pfizer to accelerate the development of more therapies that treat the root cause of CF and benefit the greatest number of people with the disease," said Robert J. Beall, Ph.D., President and CEO of the CF Foundation. "Pfizer brings impressive technical and scientific expertise, along with its commitment to improving the lives of people with cystic fibrosis."
Strategic Partnerships Driving Innovation
The Cystic Fibrosis Foundation began its research collaboration with FoldRx Pharmaceuticals in 2007, which was later acquired by Pfizer in 2010. The foundation is currently supporting nearly 30 potential new treatments for CF patients and has established successful partnerships with other pharmaceutical companies, including Vertex Pharmaceuticals and Sanofi's Genzyme.
Jose-Carlos Gutierrez-Ramos, Senior Vice President of Pfizer BioTherapeutics R&D, highlighted the importance of such collaborations: "Innovative collaborations between industry and patient organizations are increasingly critical in expediting the translation of science into new treatments. We look forward to continued collaboration with the CF Foundation and to applying our leading science with the goal of identifying novel therapies for the treatment of this devastating disease."
These strategic partnerships between pharmaceutical companies and patient advocacy organizations represent a promising approach to addressing the complex challenges of developing effective treatments for cystic fibrosis, potentially bringing new hope to patients worldwide.
