Roche and Ionis’ Drug Candidate Succeeds in Phase 3 IgA Nephropathy Trial

A drug candidate developed by Roche and Ionis has met its goal in a Phase 3 trial for kidney disease.

According to BioPharma Dive on the 23rd (local time), Roche and Ionis Pharmaceuticals said their IgA nephropathy treatment, sephaxersen, met the primary endpoint in an interim analysis of a Phase 3 trial.

The companies said sephaxersen led to a “statistically significant and clinically meaningful improvement” in protein levels in urine, a key measure of kidney health. They did not disclose specific figures and plan to present them at a future medical meeting and share them with regulators.

Trial participants will be followed for an additional 2 years to monitor changes in kidney function.

IgA nephropathy is an autoimmune disease in which abnormal antibodies enter the kidneys, causing inflammation and reducing organ function. Drug development in the field has accelerated as understanding of the disease’s mechanisms has improved.

Sephaxersen works through a pathway similar to Novartis’ Fabhalta. Both drugs target complement factor B, which causes inflammation and kidney tissue damage. Fabhalta binds directly to the protein, while sephaxersen binds to a messenger molecule in the liver to inhibit protein production.

Roche and Ionis are positioning sephaxersen as a more convenient alternative to Fabhalta. Sephaxersen is self-injected once a month, while Fabhalta is a pill taken twice daily.

Competition is expected to be fierce even if regulators approve the drug. Since late last year, the U.S. Food and Drug Administration has granted accelerated approval to treatments from Otsuka Pharmaceutical and Vera Therapeutics that target different proteins. A drug from Vertex Pharmaceuticals could also be approved soon.

Myles Minter, an analyst at William Blair, described IgA nephropathy as “a large but highly competitive market across a range of targets and dosing regimens.” He said monthly dosing with sephaxersen’s autoinjector is “relatively competitive,” but added that the data release will be important for assessing the drug’s comparative advantages and safety.

Trung Huynh, an analyst at RBC Capital Markets, named sephaxersen as one of the pipeline assets Roche investors have undervalued. In a recent report, he said sephaxersen could help drive earnings over the next several years, along with the breast cancer drug giredestrant and the multiple sclerosis drug fenebrutinib.

Approval would also be a boon for Ionis. The company, whose stock has been under pressure after several setbacks this year, could receive up to $400 million in milestone payments related to sephaxersen and would also secure sales royalties if the drug is approved.