- Aurinia Pharmaceuticals, a small Canadian biotech, reported positive efficacy results from a Phase 2b trial of its lead candidate for lupus kidney disease, but a high number of patient deaths in the treatment arms of the study led to a market sell-off on Monday before the stock recovered somewhat Tuesday.
- Treatment with the lower dose of the drug, an immunosuppressant called voclosporin, led to a statistically significant higher rate of complete remission in patients with active lupus nephritis.
- However, investigators reported 10 patient deaths in the low-dose treatment arm and two in the high-dose group, compared to only one in the control arm. All deaths, most of which happened at trial sites in Asia, were judged to be unrelated to study treatment.
The patient deaths raise important safety questions around the drug, which otherwise showed efficacy in helping treat the condition.
Lupus nephritis is caused by systemic lupus erythematosus and, if untreated, can cause permanent and irreversible damage to tissues within the kidney, and possibly death. Aurinia acknowledged the overall rate of serious adverse events (including deaths) was higher in both treatment arms than in the control but noted "the nature of the SAEs is consistent with highly active [lupus nephritis]."
Aurinia also said disease severity appeared greater in the low-dose voclosporin group, perhaps contributing to the higher number of deaths seem in those patients.
For the study, 265 patients were randomized to either receive a low dose of voclosporin, a high dose or placebo twice daily, along with receiving mycophenolate mofetil and oral corticosteroids as background therapy. Nearly a third of patients on the low dose hit complete remission, compared to 27% on the high dose, and 19% on placebo. The results were statistically significant, but just barely.
Dosing of the oral corticosteroids was tapered over the course of the trial. Given the continued efficacy seen, voclosporin could allow for lower dose treatment with steroids, which have a number of side effects.
Aurinia plans to meet with the Food and Drug Administration sometime in the fourth quarter to discuss further clinical development and registration. The study, analyzed at 24 weeks, will continue for another 6 months.