Roche has partnered with Ionis Pharmaceuticals, a Carlsbad, California-based pharmaceutical company, to develop Ionis’ antisense drug candidate for treating complement-mediated diseases, in a deal worth up to $760 million.

The collaboration will use Ionis’ experience in RNA-targeted therapeutics to develop the drug targeting Factor B (FB), a protein in the complement innate immune system, for a range of diseases. The first indication the two companies will pursue is treating patients with geographic atrophy (GA), the advanced stage of dry age-related macular degeneration (AMD). A Phase II study in patients with GA is planned to begin in early 2019.

Ionis’ drug, which uses Ionis’ advanced Ligand Conjugated Antisense (LICA) technology, reduces the production of FB, according to information from the company. FB is predominately produced in the liver and circulates throughout the vascular system, including vessels in the eye and kidney. This complement protein plays a role in an innate immunogenic cascade that, when overactivated, has been associated with the development of several complement-mediated diseases, including dry AMD, according to information from Ionis.

Under the agreement, Ionis will receive a $75-million upfront payment. In addition, Ionis is eligible to receive up to $684 million in development, regulatory, and sales milestone payments and license fees. Ionis also has the potential to receive tiered royalties that range from the high teens to 20% on sales from the product when commercialized. Ionis is responsible for conducting a Phase II study in patients with dry AMD and exploring the drug in a rare severe renal indication. Roche has the option to license Ionis’ drug at the completion of the studies. Upon licensing, Roche will be responsible for all global development and commercialization activities.

Source: Ionis Pharmaceuticals