The company said it’s preparing to file a New Drug Application with the FDA on the strength of late-stage study data for its investigational RNA-targeted donidalorsen in patients with hereditary angioedema.
Pictured: Ionis building exterior/Photo courtesy of Ionis
Ionis Pharmaceuticals is making headway with its RNA-targeted hereditary angioedema candidate donidalorsen. The company announced Monday topline Phase III trial results from its Oasis-HAE study, which met the primary endpoint of rate reduction in angioedema attacks in patients with the rare and life-threatening genetic disease.
The results for the four-week injection earned a p-value of p<0.001, while an eight-week injection earned a p-value of p=0.004 against a placebo. Ionis also said that the late-stage trial showed donidalorsen reached statistical significance on all secondary endpoints in the four-week cohort and “key” secondary endpoints in the eight-week group.
No serious adverse events were reported in the trial of donidalorsen, an investigational ligand‐conjugated antisense medicine designed to silence the production of prekallikrein which plays a critical role in activating inflammatory mediators associated with acute attacks of hereditary angioedema (HAE).
Ionis said the data will be presented at an upcoming medical congress and that it is preparing to submit an NDA to the FDA, but did not give a specific date as to when. Donidalorsen has received Orphan Drug Designation from the agency and the pursuit of the equivalent designation process in the EU is ongoing. Otsuka has the right to commercialize the drug in Europe and plans to submit a marketing authorization application to the European Medicines Agency.
“Based on these results and the durable efficacy and favorable safety data seen in the ongoing Phase II open-label extension study, we believe donidalorsen, if approved, could be an attractive new treatment option for patients with HAE, many of whom continue to experience unpredictable, painful and severe breakthrough attacks despite currently available prophylactic treatments,” Kenneth Newman, head of clinical development at Ionis, said in a statement.
Ionis has been on a good run as of late. In December 2023, Ionis and AstraZeneca’s drug Wainua (eplontersen) was approved by the FDA to treat polyneuropathy in hereditary transthyretin-mediated amyloidosis (hATTR-PN). Wainua will now compete with Alnylam’s two approved hATTR-PN therapies.
“Following the recent launch of Wainua (eplontersen), we are now well on our way to independently launching medicines from our wholly owned pipeline with regulatory submissions this year for olezarsen in familial chylomicronemia syndrome and donidalorsen in HAE,” Ionis CEO Brett Monia said in a statement.
Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.
