Roche, Ionis Partner to Develop Eye Disease Treatment

October 18, 2018

The companies will work together to develop Ionis-FB-LRx for the treatment of complement-mediated diseases, including Geographic Atrophy (GA), the advanced stage of dry age-related macular degeneration (AMD), in a deal worth $760 million.

On Oct. 10, 2018, Ionis Pharmaceuticals announced a collaboration with Roche to develop Ionis-FB-LRx, an antisense drug that targets Factor B (FB) to treat a range of complement-mediated diseases, including Geographic Atrophy (GA), the advanced stage of dry age-related macular degeneration (AMD), in a deal worth $760 million.

Under the terms of 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, as well as 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, according to Ionis. 

Ionis is responsible for conducting a Phase II study starting in early 2019 in patients with dry AMD, as well as exploring the drug in a rare severe renal indication. Roche has the option to license Ionis-FB-LRx at the completion of the studies. Upon licensing, Roche will be responsible for all global development and commercialization activities.

Ionis-FB-LRx is an antisense drug using Ionis' LIgand Conjugated Antisense (LICA) technology. The drug reduces the production of FB, a key protein in the complement innate immune system that 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.

"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-LRx,” said Brett P. Monia, chief operating officer at Ionis, in a company press release. “This new agreement builds upon our productive relationship with Roche on Ionis-HTTRx (RG6042), an antisense drug for the treatment of people with Huntington's disease." 

Source: Ionis Pharmaceuticals