Kadmon's graft-versus-host disease med on track for 2020 filing

Kadmon’s belumodsil curbed graft-versus-host disease in nearly three-quarters of patients in a pivotal study, teeing up an FDA filing in the fourth quarter.

The open-label study found that after six months of treatment, the drug improved symptoms of graft-versus-host disease (GVHD) in 73% of patients who took the drug once a day and 74% of patients who took it twice a day. That’s a boost over the response rates from an interim analysis of the study presented earlier this year: 64% and 67%, respectively.

GVHD is a serious complication of bone marrow transplant, occurring when the donor cells mount an immune response against the recipient’s healthy tissues and organs. Steroids may be used to try and limit the immune reaction, but this does not work in all patients. Kadmon tested belumodsil, also called KD025, in patients who had tried two prior treatments.

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Jefferies analyst Biren Amin called the data impressive, noting that "the ORR is higher than those observed with both ibrutinib [Imbruvica] and Jakafi, and recall the ROCKstar trial recruited cGVHD pts with 2-5 prior lines of treatments w/ than a third had prior ibrutinib and around 30% had prior ruxolitinib [Jakafi]." Both Jakafi and Imbruvica are blood cancer drugs that are also used to treat GVHD,.

Of the patients who responded to treatment, nearly half are still responding after 20 weeks. Patients have been taking the drug for a median of 29 weeks and the median duration of response has not yet been reached, suggesting the effects of the drug could be long-lasting. Kadmon plans to share the full dataset at a medical meeting.

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The drug is developed specifically for chronic GVHD. Unlike the acute form of the condition, which comes on within months of the transplant, chronic GVHD generally starts later and can last for years.

The side effects were as expected in the patient population, with no cytomegalovirus (CMV) infections or cytopenias—lower-than-normal numbers of blood cells—reported.

"Belumosudil has shown robust and durable responses across the spectrum of this difficult-to-treat disease and we are extraordinarily pleased with this outcome," said Kadmon CEO Harlan Waksal, M.D., in a statement. "We are excited to participate in the FDA's Real-Time Oncology Review pilot program, which seeks to promote efficient review of much-needed treatments. We continue to work closely with the Agency through their review of our data as we prepare to complete our NDA submission."