Dive Brief:
- An experimental medicine from Vera Therapeutics succeeded in a late-stage trial in a rare kidney disease, positioning the company to discuss an approval application with U.S. regulators in the near future.
- According to Vera, the drug, called atacicept, met its main goal in a Phase 3 trial in IgA nephropathy, a chronic condition that can lead to kidney failure. After 36 weeks of treatment, atacicept was associated with a 42% reduction versus a placebo — and a 46% decline from the study’s start — in the amount of protein in participants’ urine, an important marker of kidney health.
- Without providing specifics, Vera said atacicept’s other efficacy results were “consistent with or better than” what was observed in mid-stage testing and had a safety profile “comparable to placebo.” The company will share the data with the Food and Drug Administration in the coming weeks and intends to file an accelerated approval request in the fourth quarter.
Dive Insight:
Vera is one of the leaders in a push to develop new treatments for IgA nephropathy, or IgAN, a progressive disease that damages the kidneys.
Some estimates hold 130,000 to 150,000 people in the U.S. have IgAN. While several medicines are approved, they don’t totally stop patients’ kidney function from deteriorating. That need, and a better understanding of the disease’s biology, has led to an uptick in dealmaking involving IgAN. Drugs recently launched by Travere Therapeutics and Calliditas Therapeutics have performed well, too.
Vera’s atacicept is part of a newer wave of therapies aimed at immune-mediated drivers of the disease. Like several others in development, it targets a pair of cytokines called BAFF and April that stimulate production of the “autoantibodies” that mistakenly target the body’s own tissue. In Phase 2 testing, the drug stabilized a key marker of disease progression so well that some analysts viewed the findings as the bar for other competitors to meet. The findings also heightened anticipation for Vera’s Phase 3 study.
According to Marshall Fordyce, Vera’s founder and CEO, the drug’s effects on urine protein in the trial significantly “exceed the bar” for what U.S. regulators have accepted in granting an accelerated approval to several other medications. The figure Vera reported also represents the “deepest reduction” in a late-stage stage study in IgAN patients to date, he wrote in an email.
Investors appear to agree, as Vera’s shares climbed by nearly 60%, to over $30 apiece, in early trading on Monday.
Vera has competition ahead, as Otsuka Pharmaceutical could win approval of a similar type of medicine by late November. The company will present detailed Phase 3 study results at a medical meeting on Friday and, though cross-trial comparisons come with caveats, analysts and investors will be paying close attention to how the two treatments stack up.
Vera, for its part, believes the market for IgAN treatments is “ripe for disruption,” according to Fordyce. There are currently “low levels of approved product saturation,” Fordyce wrote, and newer drugs like atacicept “may represent a differentiated approach.” The company is also studying the drug in people whose are at “moderate” or “low” risk of disease progression, which, if successful, could boost its sales potential.
The Phase 3 trial, meanwhile, will continue on while Vera accumulates data on atacicept’s impact on kidney function. Those findings are expected in 2027.
Vera’s results have implications for drugmakers like Biogen and Vertex Pharmaceuticals, which have both cut deals for companies developing IgAN drugs. Vertex has said it could file for approval of a drug that also targets BAFF and April early next year, if data are supportive.
