Ionis Pharmaceuticals has entered a deal worth up to $760m with Roche for further development of its antisense drug, IONIS-FB-L, to treat complement-mediated diseases.
IONIS-FB-L is designed using Ionis’ antisense technology platform. It uses the company’s ligand conjugated antisense (LICA) technology to decrease the generation of Factor B (FB) protein.
FB plays an important role in an innate immunogenic cascade, the overactivation of which can lead to development of various complement-mediated diseases such as dry age-related macular degeneration (AMD).
Under the alliance, the partners will leverage Ionis’ expertise in RNA-targeted drugs to advance IONIS-FB-L as therapeutic for multiple conditions.
Initially, Ionis and Roche will investigate the drug for the treatment of geographic atrophy (GA) and advanced dry AMD. They plan to commence a Phase II clinical trial in GA patients early next year.
IONIS-FB-L is said to have minimised plasma FB during a Phase I trial performed in 54 healthy volunteers. The drug was also found to be safe and well-tolerated.
Ionis Pharmaceuticals chief operating officer Brett Monia said: “This new agreement builds upon our productive relationship with Roche on IONIS-HTT (RG6042), an antisense drug for the treatment of people with Huntington’s disease.
“We believe that we have found the right partner whose experience in retinal disease drug development and commercialisation will enhance our efforts to effectively develop IONIS-FB-L for patients who currently have no adequate treatment options.”
As per the agreement, Roche will make an upfront payment of $75m to Ionis, which is also eligible to receive up to $684m in development, regulatory and sales milestones, as well as licence fees.
Ionis may also get tiered royalties on sales of the product, when commercialised. The company will conduct a Phase II trial of IONIS-FB-L for dry AMD and evaluate the drug in a rare severe renal indication.
Roche holds an option to licence the drug at the completion of the studies. If exercised, the company will be responsible to carry out all development and commercialisation activities.