A late-stage test of Roche and Ionis Pharmaceuticals’ sefaxersen showed that the drug reduced protein in the urine, which suggests the RNA-targeting therapy could improve kidney function and delay transplant.
Roche and Ionis Pharmaceuticals’ RNA-targeted kidney disease therapy improved the reduction of protein in urine during a Phase 3 trial, setting the partners up for a run at an accelerated approval.
The news comes one day after Ionis announced a critical clinical win in ALS for the antisense oligonucleotide ulefnersen, marking a major validation for the company’s RNA-targeting platform.
Sefaxersen achieved the primary endpoint in the ongoing Phase 3 IMAgINATION study of patients with primary IgA nephropathy (IgAN), with a statistically significant and clinically meaningful improvement in proteinuria reduction as compared to placebo at 37 weeks, the partners announced on Wednesday morning. The endpoint was measured by 24-hour urine protein-to-creatinine ratio (UPCR), with a reduction associated with the preservation of long-term kidney function.
Roche did not disclose specific data for UPCR but said the interim results suggest that sefaxersen can modify a key surrogate endpoint of kidney function in patients with IgAN. The drug could delay disease progression and reduce the need for dialysis or kidney transplant, said Roche’s Levi Garraway, chief medical officer and head of global product development, in a statement on Wednesday.
The partners had been hoping the Phase 3 readout would support an accelerated approval for the kidney disease, Ionis CEO Brett Monia told investors earlier this month at the Wells Fargo 21st Annual Healthcare Conference. He promised that once the results were available, Roche would file “soon thereafter.”
Roche did not say on Wednesday when that filing would be submitted but promised to take the data to regulatory authorities.
The IMAgINATION study, meanwhile, will continue on for two years to monitor kidney function. Roche will present more data from the interim readout at an upcoming medical conference.
Sefaxersen was licensed from Ionis in 2022 for the treatment of complement-mediated diseases. Ionis received $55 million upfront plus milestones for this specific drug, but the companies have worked together for years.
The IgAN win follows another milestone for Ionis, which, with partner Otsuka announced on Tuesday that ulefnersen improved functional impairment and survival in a genetic type of ALS. The partners are looking into an expedited approval there as well.
Ionis has endured a bumpy ride of late despite two FDA approvals earned this year alone.
The antisense oligonucleotide Tryngolza was granted an expanded approval for severe hypertriglyceridemia in June while Zanvastro became the first disease modifying treatment for the ultra-rare neurology disorder Alexander disease earlier this month.
But key Phase 3 failures for Novartis-partnered pelacarsen in reducing cardiovascular risk and AstraZeneca-partnered eplontersen in transthyretin amyloid cardiomyopathy (ATTR-CM) have set the RNA company on its backfoot.
Despite the two drug launches, Ionis’ shares have fallen 42% year to date to $45.75.
