A phase 3 trial of Aurinia Pharmaceuticals’ voclosporin in lupus nephritis has hit its primary endpoint, causing the company's share price to double. The data tee Aurinia up to file for FDA approval next year and go on to address a major unmet medical need.
Voclosporin is a calcineurin inhibitor designed to block IL-2 expression and T-cell mediated immune responses, tamping down the autoimmune attack while stabilizing a type of kidney cell that can be damaged in lupus nephritis patients. Similar drugs, such as tacrolimus, are used in systemic lupus erythematosus but suffer from narrow therapeutic windows and limited data in lupus nephritis.
Now, lupus nephritis patients look set to gain access to a calcineurin inhibitor shown to work in the indication. In the 357-subject phase 3 trial, patients who received voclosporin, rather than placebo, on top of standard immunosuppressants had a higher renal response rate at 52 weeks.
The difference between the renal response rates in the treatment and control arms, 40.8% versus 22.5%, reached statistical significance, resulting in the trial hitting its primary endpoint. Aurinia also reported statistically significant results against a clutch of secondary endpoints, showing voclosporin started outperforming placebo within 24 weeks and brought the urinary protein-to-creatinine ratio down faster.
Voclosporin also emerged from the trial without triggering any safety alarms. In phase 2b, 11% of patients who received the low dose of voclosporin died. The death rate in the placebo arm was 1%. As such, while Aurinia said the deaths were unrelated to voclosporin, safety was one area of concern going into the phase 3.
The mortality signal seen in the earlier trial was absent from the larger phase 3. One patient in the voclosporin arm died, compared to five deaths in the control group. The rate of serious adverse events was nearly identical across the two groups.
Armed with the data, Aurinia plans to file for FDA approval of voclosporin in the first half of next year. Given FDA has put voclosporin on its fast track, the drug looks set to hit the market in 2021. That prospect has been well received in the lupus community.
“The positive results for the AURORA Phase III study of voclosporin is the news the lupus community has been waiting for and represents a remarkable breakthrough in efforts to bring a better quality of life for the more than five million people worldwide living with lupus,” Stevan Gibson, CEO of the Lupus Foundation of America, said in a statement.
Aurinia began preparing for the commercial launch of voclosporin ahead of the phase 3 results, but its possession of a largely derisked, near-approval asset targeting an unmet need means it may be an acquisition target for larger companies that want to add voclosporin to their portfolios.