Ionis Pharmaceuticals has signed on to a new collaboration with Roche worth up to $760 million, to help develop its RNA-based drug for advanced dry age-related macular degeneration and other diseases.
Its antisense therapeutic, IONIS-FB-LRx, had been snubbed by GlaxoSmithKline in August 2017, which declined to pick up its option for the drug, as well as inotersen—recently approved and launched as Tegsedi—after CEO Emma Walmsley began moving the Big Pharma away from its rare disease programs.
Now, the Carlsbad, California-based company is set to receive $75 million upfront, plus as much as $684 million in development, regulatory and sales milestone payments and licensing fees. In addition, Roche could pay out royalties ranging from the high teens to 20% if the drug makes it to market, Ionis said in a statement.
Under the deal, Ionis will be responsible for conducting a phase 2 dry AMD study of IONIS-FB-LRx, which aims to interrupt the translation of messenger RNA into Factor B, a complement protein that plays a role in the innate immune system and several diseases. Ionis will also explore the drug’s use against an unnamed, rare renal indication.
Roche will have the option to license IONIS-FB-LRx after the studies are completed, when it will become responsible for all global development and commercialization activities. Ionis previously formed a collaboration with Roche on IONIS-HTTRx (RG6042), an antisense drug for Huntington's disease.
Factor B is mainly produced in the liver and circulates through the bloodstream. When overactivated, it has been associated with several complement-mediated diseases, including geographic atrophy, the advanced stage of dry AMD that can lead to progressive and irreversible loss of retina cells and visual function, and is estimated to affect more than 5 million people worldwide.
“We believe that we have found the right partner whose experience in retinal disease drug development and commercialization will enhance our efforts to effectively develop IONIS-FB-LRx for patients who currently have no adequate treatment options,” said Brett Monia, Ionis’ chief operating officer.
The placebo-controlled phase 2 study in patients with geographic atrophy is planned to begin early next year. Ionis previously completed a phase 1 study in 54 healthy volunteers, showing that IONIS-FB-LRx reduced plasma factor B levels and was well tolerated.