Galapagos and AbbVie have entered into a global alliance to discover, develop and commercialize novel potentiator and combination therapies in cystic fibrosis (CF), an inherited chronic disease that affects 70,000 people worldwide.
The companies will work collaboratively to contribute technologies and resources in order to develop and commercialize oral drugs that address the main mutations in CF patients, including F508del and G551D. The goal of the collaboration is to identify compounds that correct defects in expression of (corrector) and/or increase the activity (potentiator) of the main mutations in the cystic fibrosis transmembrane regulator (CFTR) protein, including the F508del mutation, which is the most common with 90% prevalence among patients with CF.
The companies will develop potentiators and correctors discovered by Galapagos and expand the range of molecules, with the aim to initiate phase I clinical studies at the end of 2014. Following successful clinical development and regulatory approval, AbbVie will be responsible for commercial activities, with Galapagos retaining exclusive rights in China and South Korea and co-promotion rights in Belgium, the Netherlands and Luxembourg.
AbbVie will make an initial upfront payment of $45 million to Galapagos. AbbVie and Galapagos will share responsibility and funding for phase III clinical development. Galapagos is eligible to receive up to $360 million in total additional milestone payments plus additional double-digit royalty payments on net sales.
Galapagos initiated its research in CF in 2005 as part of a collaboration with the Cystic Fibrosis Foundation. In 2010, Galapagos decided to pursue CF as the first orphan disease for which the company is attempting to discover, develop and launch its own medicines. Galapagos has developed small molecule therapies that can restore the function of the defective CF protein (CFTR). The first preclinical candidate is expected to be nominated this year, with the first clinical trials starting at the end of 2014.