Fantastic clinical trials results for an investigational drug that treats kidney inflammation caused by lupus, an autoimmune disease. Aurinia Pharmaceuticals stock price has shot up 78.8% as a result.
Lupus Nephritis can Kill
Lupus nephritis, affecting about three (3) million people, can lead to kidney damage, kidney failure and ultimately death. It is a type of kidney disease caused by systemic lupus erythematosus (SLE or lupus). Lupus is an autoimmune disease—a disorder in which the body’s immune system attacks the body’s own cells and organs. Kidney disease caused by lupus may get worse over time and lead to kidney failure. If a kidney fails the patient may need dialysis to maintain health reports the National Institute of Diabetes and Digestive and Kidney Disease (NIDDK).
What is Voclosporin?
Aurinia Pharmaceutical’s drug voclosporin has completed Phase III; it was granted Fast Track designation by the FDA in 2016 and Aurinia plans to submit a New Drug Application (NDA) to the FDA in the first half of 2020.
Voclosporin, an investigational drug, is a novel and potentially best-in-class calcineurin inhibitor (“CNI”) with clinical data in over 2,600 patients across indications. Voclosporin is an immunosuppressant, with a synergistic and dual mechanism of action. By inhibiting calcineurin, voclosporin blocks IL-2 expression and T-cell mediated immune responses and stabilizes the podocyte in the kidney. It has been shown to have a more predictable pharmacokinetic and pharmacodynamic relationship (potentially requires no therapeutic drug monitoring), an increase in potency (versus cyclosporine A), and an improved metabolic profile compared to legacy CNIs. Aurinia anticipates that upon regulatory approval, patent protection for voclosporin will be extended in the United States and certain other major markets, including Europe and Japan, until at least October 2027 under the Hatch-Waxman Act and comparable laws in other countries and until April 2028 with anticipated pediatric extension. Further, a U.S. patent has also been issued covering the voclosporin dosing protocol with a term extending to December 2037, if the FDA incorporates the dosing protocol used in both the AURA and AURORA trials into the product label.
The recent stock life resulted from the company’s most recent Phase III clinical trial where 357 patients worldwide evidenced a positive response rate of 40.8%, compared with a control group of 22.5%. The study sought to investigate whether voclosporin, added to the standard of care treatment in active lupus nephritis is able to reduce disease activity over a treatment of 52 weeks. The study utilized up to 187 sites and its principal investigator was Mary Anne Dooley, MPH, University of North Carolina.
The results recently shared by Aurinia Pharmaceuticals:
|Primary Endpoint||Renal Response at 52 weeks||Voclosporin 40.8%Control 22.5%||2.65 [1.64, 4.27]||p < 0.001|
|Secondary Endpoints||Renal Response at 24 weeks||Voclosporin 32.4%Control 19.7%||2.23 [1.34, 3.72]||p = 0.002|
|Partial Renal Response at 24 weeks||Voclosporin 70.4%Control 50.0%||2.43 [1.56, 3.79]||p < 0.001|
|Partial Renal Response at 52 weeks||Voclosporin 69.8%Control 51.7%||2.26 [1.45, 3.51]||p < 0.001|
|Time to UPCR ≤ 0.5||Voclosporin faster|
|2.02 [1.51, 2.70]Hazard Ratio||p < 0.001|
|Time to 50% reduction in UPCR||Voclosporin faster|
|2.05 [1.62, 2.60]Hazard Ratio||p < 0.0|
Comments on the Results
Brad Rovin, chief of nephrology (kidney) division at Ohio State’s Wexner Medical Center called the results “a potential game-changer.” The Phase III results represent “a clinically meaningful leap forward for the treatment of lupus nephritis reported Rovin. Moreover no excess adverse events were reported. He declared, “This confirmatory Phase III result represents a clinically meaningful leap forward in the treatment of lupus nephritis.”
Stevan W. Gibson, President and Chief Executive Officer, Lupus Foundation of America, reports, “The treatment of lupus nephritis has been extremely challenging to date, and people with lupus are in need of innovative treatments for this serious disease. We’re product to have been part of this important achievement through our work educating people with lupus nephritis about the trial and the importance of clinical trial participation.” Gibson continued, “Voclosporin is the first novel treatment that has demonstrated therapeutic efficacy for people living with lupus nephritis and today marks an important advance in the treatment of this potentially life-threatening disease.”
Aurinia Pharmaceuticals is a late clinical-stage biopharmaceutical company focused on developing and commercializing therapies to treat targeted patient populations that are impacted by serious diseases with a high unmet medical need. The Company is currently developing an investigational drug, for the treatment of lupus nephritis, focal segmental glomerulosclerosis and dry eye syndrome. The Company’s head office is in Victoria, British Columbia and focuses its development efforts globally. They currently employ about 50. Their stock price shot up after results: from $8.43 to $17.64 and settling at $15.44 as of this writing.
The AURORA Clinical Trial
The AURORA clinical trial is a global, double-blind, placebo-controlled study to evaluate whether voclosporin when added to background therapy of mycophenolate mofetil (MMF)/CellCept® can increase speed of and overall renal response rates in the presence of low dose steroids. The primary endpoint for the study is complete renal response at 52 weeks, after which patients can choose to enroll into a 104-week blinded extension study. Renal response was defined as UCPR of ≤ 0.5 mg/mg, eGFR ≥ 60 mL/min/1.73 m2, or no confirmed decrease from baseline in eGFR of > 20%, presence of sustained, low dose steroids and no administration of rescue medication. The target enrollment of 324 patients was surpassed with a total of 357 lupus nephritis (LN) patients randomized globally across sites in 27 countries.
AURURA 2 Extension
Eligible patients completing the AURORA trial had the option to roll over into a 104-week blinded extension study (the “AURORA 2 extension study”). A total of 216 patients enrolled into the AURORA 2 extension study. The data from the AURORA 2 extension study will allow the company to assess the long-term benefit/risk of voclosporin in LN patients, however, this study is not a requirement for potential regulatory approval for voclosporin. Data from the AURORA 2 extension study assessing long-term outcomes in LN patients should be valuable in a post-marketing setting and for future interactions with various regulatory authorities.
Lead Research/Investigator AURORA Study
Mary Anne Dooley, MD, MPH University of North Carolina