Vertex has regained limited rights to two DNA-dependent protein kinase (DNA-PK) inhibitors from Merck KGaA. Merck licensed the drugs and other assets for $230 million upfront two years ago but has now granted Vertex the right to use them in certain gene-editing applications.
Germany’s Merck bagged the DNA-PK inhibitors to boost its oncology pipeline and has since wrapped up a phase 1 study of the more advanced candidate, M9831, in subjects with advanced solid tumors. However, researchers have also used DNA-PK inhibitors to modulate mechanisms that repair DNA breaks and thereby improve CRISPR/Cas9 genome editing.
Vertex lost the right to use M9831, formerly known as VX-984, and the other preclinical DNA-PK drug in gene-editing applications when it licensed the assets to Merck in 2017. But it has now returned to Merck to gain certain rights to use the molecules.
The new deal gives Vertex the right to use the two DNA-PK inhibitors in gene-editing applications in six specific, but publicly undisclosed, genetic disease areas. Merck retains the rights to the drugs in all other diseases, including oncology, and can use or license them in gene-editing applications outside of those covered by the agreement. Vertex has an option to add more indications to the agreement.
Vertex has put together an undisclosed package of upfront, milestone and royalty payments to land the deal. In doing so, Vertex has again suggested that it sees CRISPR/Cas9 gene editing as important to its future. Vertex has a clinical-phase gene-editing stem cell therapy, CTX001, through its deal with CRISPR Therapeutics.
For Merck, the deal serves as a way to generate immediate and potentially ongoing revenues from a pair of assets while retaining the chance to advance them in oncology. Merck thinks inhibition of the DNA-PK enzyme could make DNA-damaging agents such as radiotherapy and chemotherapy more effective.