Dive Brief:
- An experimental drug from Vertex Pharmaceuticals succeeded in a late-stage trial in a rare kidney disease, positioning the company to quickly finalize plans for an accelerated clearance in the U.S.
- According to a Monday announcement by Vertex, the drug, povetacicept, met its goals in a Phase 3 trial in IgA nephropathy, a chronic condition that can lead to kidney failure. Upon an interim analysis after 36 weeks of treatment, povetacicept was associated with a roughly 50% reduction, versus a placebo, in the amount of protein in participants’ urine, a marker of kidney health and main study objective. Treatment also helped reduce levels of an abnormal antibody as well as blood in the urine, both of which were secondary measures.
- The most common side effects were respiratory infections and injection site reactions, the majority of which were mild to moderate. More people in the placebo group stopped treatment than those given Vertex’s drug, the company added. Vertex has already submitted parts of an accelerated approval application to U.S. regulators and will complete it by the end of the month. The company intends to use a special voucher to shorten the review period from 10 months to six.
Dive Insight:
Vertex has become one of the biopharmaceutical industry’s most valuable companies because of a cystic fibrosis drug business that brings in more than $10 billion each year. But the company has had a tough time recreating that success elsewhere, leading to persistent questions about its growth.
Journavx, a pain drug the company brought to market last year, is thought to be one solution, though some clinical stumbles and a slower-than-expected launch ramp suggest it might not be the big seller Vertex had hoped. Another is povetacicept, a drug Vertex acquired in a roughly $5 billion deal almost two years ago and that the company sees having the potential to treat multiple inflammatory conditions.
Povetacicept is part of a new wave of therapies aimed at immune-mediated drivers of IgA nephropathy, or IgAN, a progressive disease that damages the kidneys. Like some others in development, it targets a pair of cytokines called BAFF and APRIL that help produce the “autoantibodies” that errantly attack the body’s own tissue.
Vertex is behind some top competitors. The Food and Drug Administration last November cleared an Otsuka Pharmaceutical drug that works similarly to Vertex’s. Another, from Vera Therapeutics, could be approved by July 7. Yet Vertex executives have argued their drug has “best-in-class potential,” given its slightly different mechanism than Otsuka’s and potential efficacy and dosing advantages compared to Vera’s.
The Phase 3 data disclosed Monday were Vertex’s first big chance to back up those claims. Analysts at the investment firm RBC Capital Markets predicted in February that anywhere from a 45% to 55% reduction in urine protein levels — which Vertex achieved — with “clean safety” would “further grow enthusiasm about the drug’s multibillion[-dollar] potential.”
“These results are important for patients with IgAN and also bring us one step closer to realizing povetacicept’s pipeline-in-a-product promise,” said Vertex CEO Reshma Kewalramani, in a statement.
Yet more telling results may still lay ahead. Vertex’s trial was designed to have a pre-planned interim analysis when a pre-specified number of patients completed 36 weeks of treatment. A “final” evaluation will occur at two years and evaluate povetacicept’s impact on “eGFR,” a measure of how well the kidneys filter blood.
That goal is the “more commercially meaningful endpoint,” the RBC team wrote. Data on that measure from other IgAN drugs, including Otsuka’s, could come later this year and provide more “definitive competitive comparisons,” they added.
Vertex shares spiked nearly 9% in post-market trading on Monday.
