Ionis hands off an antisense drug for kidney disease, giving all rights to Roche
Ionis is handing Roche a drug on the cusp of Phase III.
Ionis announced early Monday that Roche is in-licensing and advancing the antisense drug candidate known as IONIS-FB-LRx into a Phase III clinical study. The drug is being investigated for immunoglobulin A nephropathy (IgAN), a kidney disease that can lead to renal failure. The disease is marked by immunoglobulin A buildup in the kidneys, resulting in inflammation and ultimately kidney failure if left untreated.
Roche and Ionis have been partners for some time now after the Big Pharma shelled out $75 million to license the drug candidate in 2018, after reaching a previous deal on a Huntington’s program. Roche only got its hands on the drug after GSK, Ionis’ earlier partner on IONIS-FB-LRx, dumped its option in a move to revamp its R&D pipeline after Emma Walmsley became CEO in 2017.
Ionis originally had tested the candidate in dry age-related macular degeneration, or AMD. However, the companies left the option for Roche to license the drug altogether in 2018 after Ionis did some more work in dry AMD and an at-the-time unnamed rare, severe renal indication.
Roche decided to advance the program after a positive Phase II readout, where the drug met the primary endpoint of “change in 24-hour urinary protein at 29 weeks compared to baseline.” Those Phase II study data have been submitted for presentation at an unnamed medical conference.
Ionis noted, in a release, that Roche will lead and be responsible for the Phase III study, on top of future global development, regulatory and commercialization activities.
A Roche spokesperson told Endpoints News that “Roche’s collaboration with Ionis will help to maximize our chances of success for developing therapies in two devastating conditions, where only few options are available.”
As for Roche’s next steps, the Phase III study will aim to confirm the benefit seen in the earlier trial. On top of that, Ionis is conducting a Phase II study in geographic atrophy due to late-stage age-related macular degeneration (AMD). Roche added that if the outcome of the study is positive, it will look at potentially developing the drug for that indication.
According to ClinicalTrials.gov, the Phase II study in question on geographic atrophy is scheduled to finish sometime in October this year. Ionis received $55 million from Roche for licensing IONIS-FB-LRx for IgAN and achieving a yet-unspecified development milestone in that aforementioned Phase II study.
Ionis and Roche have not been the only players in the IgAN space. Angion Biomedica scrapped a Phase II trial looking at its oral TKI inhibitor in patients with IgAN and focal segmental glomerulosclerosis (FSGS) after an unexpected safety event just two weeks ago. Alnylam, in collaboration with Regeneron after inking a blockbuster R&D deal in 2019, recently touted data on investigational RNAi drug cemdisiran from a Phase II descriptive trial — with no statistical testing conducted.
Ionis did not respond to a request for comment from Endpoints before publication, but this story will be updated when we hear back.