Putting the safety issue to bed, Aurinia's late-stage lupus nephritis data shines
After a checkered mid-stage program marred by patient deaths, Aurinia emerged victorious on Wednesday with Phase III lupus nephritis data that demonstrated its lead drug is safe and effective for a patient population that has few options.
Aurinia’s shares skyrocketed in Thursday morning premarket trading, as the Canadian biotech unveiled plans to submit a marketing application next year.
The 357-patient placebo-controlled AURORA trial tested the company’s drug, voclosporin, in combination with mycophenolate and low-dose corticosteroids to evaluate whether the therapy could improve the speed of and overall renal response rates in patients with lupus nephritis, where the autoimmune disease ravages the kidneys, triggering inflammation that could lead to blood and/or protein in the urine, high blood pressure, poor kidney function, or kidney failure.
Patients given voclosporin recorded renal response of 40.8%, while those on the control arm experienced a rate of 22.5% — meeting the main goal of the study (p < 0.001) at 52 weeks. The Aurinia therapy also induced statistically significant improvements across all prespecified secondary endpoints including renal response at 24 weeks, partial renal response at different time points and time to achieve urinary protein-to-creatinine ratio.
On the safety side, voclosporin data was also decisive.
Six deaths occurred in the study, one in the voclosporin arm and five in the control group. Serious adverse events were reported in 20.8% of voclosporin patients, versus 21.3% in the control arm.
In 2016, Aurinia hit a major roadblock with its voclosporin program after 13 deaths were recorded in its phase IIb study – 10 in the low-dose arm, 2 in the high-dose arm and only one in the control group. However, 40% of the 265 patients in the study were enrolled in sites in Asia, where treatment standards are different, Aurinia had suggested at the time.
On Wednesday, SVB Leerink’s Joseph Schwartz suggested Aurinia’s latest data was in-line with his “optimistic expectations.”
“AUPH has accordingly avoided problematic sites in Ph.3 AURORA and today’s result shows the fruits of that effort,” he said. “With a clear mortality advantage in favor of voclosporin treatment, we think the safety controversy will take a permanent backseat for voclosporin in lupus nephritis (LN).”
Aurinia’s shares flew up 137% to $19.89 in premarket trading.
The systemic autoimmune disease, in which the body’s immune system launches an attack on its own tissues and organs, affects about 1.5 million Americans, according to The Lupus Foundation of America. Roughly 60% of lupus patients develop lupus nephritis and 1 in 4 progress to end-stage renal disease.
Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval, with the field resembling a graveyard in recent years. Last October, UCB and Biogen’s anti-CD40L drug failed in a late-stage study, months after Xencor and Sanofi’s Ablynx also conceded defeat in their programs. In August, however, AstraZeneca offered a glimmer of hope after its drug, anifrolumab, cleared a Phase III study.
The only biologic so far to win approval for lupus is GSK’s Benlysta — which was cleared for adult use in 2011 and for rare cases of childhood lupus this year. GSK is in the midst of testing Benlysta in combination with Roche’s rituximab in the hope the combination will have a more potent effect on the disease versus Benlysta monotherapy. And last month, Roche said it was working on putting together a late-stage study for its monoclonal antibody Gazyva in patients with lupus nephritis.