Ruxolitinib passes another PhIII test for graft-versus-host disease, a win for Incyte and Novartis
Ruxolitinib has proven itself a stellar moneymaker for Incyte and Novartis, and it should come as no surprise that the drug’s efficacy reached expectations in yet another Phase III trial.
The two companies announced Thursday that ruxolitinib — sold as Jakafi by Incyte in the US and as Jakavi by Novartis elsewhere — met its primary endpoint in a study evaluating treatment in patients with chronic graft-versus-host disease who did not respond well to steroids. Measured against the best available therapy, ruxolitinib showed a significantly improved overall response rate after 24 weeks.
Full data haven’t been released yet, but the drug also met both secondary endpoints improving failure-free survival and patient-reported symptoms. The latter was measured by the modified Lee cGVHD Symptom Scale.
This news comes just a few months after a separate trial illustrated the drug’s efficacy in treating acute GVHD, which manifests much earlier than its chronic counterpart. That study measured overall response rate after only 28 days of ruxolitinib treatment.
Graft-versus-host disease occurs after patients receive a transplant that uses stem cells. The stem cells essentially give the body a new immune system but treat the existing tissue as foreign. GVHD can occur at any time after a transplant, and the risk is greater if the donor is unrelated.
Acute GVHD usually appears within 100 days of a transplant, whereas chronic GVHD takes longer than that benchmark. Patients with chronic GVHD can also exhibit some symptoms similar to autoimmune diseases.
The topline results “underscore the potential for Jakafi to provide a meaningful treatment option, not only for patients with acute GVHD, but also for the chronic form of the condition,” said Peter Langmuir, Incyte’s group VP for oncology targeted therapies.
Ruxolitinib itself is a JAK inhibitor targeting JAK1 and JAK2 and had previously been approved in the US and EU as a second-line treatment for polycythemia vera, as well as a first-line treatment for adults with myelofibrosis, or the buildup of scar tissue in bone marrow. Patients who exhibit MF after being treated for PV can also be prescribed ruxolitinib.
Despite ruxolitinib’s continued success, netting Incyte $1.7 billion in sales last year, the Delaware-based biotech has seen its share of setbacks in recent years. Back in 2018, there was the epacadostat debacle when Incyte had to halt a Phase III trial researching efficacy in metastatic melanoma. And early in 2020, a separate acute GVHD treatment dubbed itacitinib flopped in a first-line, Phase III treatment study.
But ruxolitinib has kept the company afloat for now, and the drug’s continued success in Phase III trials demonstrates its longevity. In the US, a topical Jakafi cream passed two Phase III readouts in treating atopic dermatitis earlier this year.