Ionis Enters Collaboration with Roche Worth up to $760 Million

Rare Daily Staff

Ionis Pharmaceuticals said it entered into a collaboration with Roche to develop IONIS-FB-L_Rx for the treatment of complement-mediated diseases in a deal worth up to $760 million.

The collaboration will leverage Ionis’ expertise in RNA-targeted therapeutics to develop IONIS-FB-L_Rx targeting Factor B for a broad range of diseases. The first indication the two companies will pursue is the treatment of patients with Geographic Atrophy, the advanced stage of dry age-related macular degeneration.

Under this new collaboration with Roche, Ionis will receive a $75 million upfront payment. In addition, Ionis is eligible to receive up to $684 million in development, regulatory, sales milestone payments, and license fees.

Ionis also has the potential to receive tiered royalties that range from the high teens to twenty percent on sales from the product when commercialized. Ionis is responsible for conducting a phase 2 study in patients with dry AMD and exploring the drug in a rare severe renal indication.

Roche has the option to license IONIS-FB-L_Rx at the completion of the studies. Upon licensing, Roche will be responsible for all global development and commercialization activities.

“Ionis is committed to bringing new therapies to patients living with unmet medical needs. The collaboration is designed to maximize both the potential benefit to patients and the likelihood of success, while optimizing our commercial participation in IONIS-FB-L_Rx,” said Brett Monia, chief operating officer at Ionis. “This new agreement builds upon our productive relationship with Roche on IONIS-HTT_Rx, an antisense drug for the treatment of people with Huntington’s disease.”

IONIS-FB-L_Rx, an antisense drug using Ionis’ advanced Ligand Conjugated Antisense technology, reduces the production of FB, a key protein in the complement innate immune system. 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 pivotal role in an innate immunogenic cascade that, when overactivated, has been associated with the development of several complement-mediated diseases, including dry AMD.

In a Phase 1 study in 54 healthy volunteers IONIS-FB-L_Rx reduced plasma FB and was safe and well tolerated.

October 11, 2018
Photo: Brett Monia, CEO at Ionis