Positive results from pivotal ROCKstar trial of belumosudil to treat chronic graft-versus-host disease.- Kadmon Holdings Inc.

Kadmon Holdings, Inc. announced positive topline results from the primary analysis of ROCKstar (KD025-213), the pivotal trial evaluating belumosudil (KD 025) in patients with chronic graft-versus-host disease (cGVHD) who have received at least two prior lines of systemic therapy

Belumosudil (KD025) achieved clinically meaningful and statistically significant Overall Response Rates (ORRs) of 73% with 200 mg once daily (QD) (95% Confidence Interval (CI): 60%, 83%; p<0.0001) and 74% with 200 mg twice daily (BID) (95% CI: 62%, 84%; p<0.0001). Responses were achieved across key patient subgroups and complete responses were observed in all organ systems.The study had previously met the primary endpoint of Overall Response Rate (ORR) at the interim analysis, which was conducted as scheduled as two months after completion of enrollment and was previously reported. These results from the primary analysis, which was conducted six months after completion of enrollment, show even greater efficacy of belumosudil (KD025) in cGVHD patients.

While data continue to mature, 49% of responders have maintained their response for at least 20 weeks at the time of the primary analysis. With a median treatment duration of 29 weeks, the median duration of response has not yet been reached in this ongoing study.

Belumosudil has been well tolerated and adverse events have been consistent with those expected in the patient population. No cytomegalovirus (CMV) infection or reactivation has been observed and no significant drug-related cytopenias have been reported.

Comment;Kadmon is submitting its New Drug Application (NDA) to the FDA under the Real-Time Oncology Review (RTOR) pilot program, an initiative of the FDA’s Oncology Center of Excellence. Kadmon is on track to complete its NDA submission for belumosudil (KD025) in the fourth quarter of 2020.