ORRs of 73% and 74% with Belumosudil (KD025) 200 mg QD and 200 mg BID, Respectively
Kadmon Participating in FDA's Real-Time Oncology Review Pilot Program
Conference Call Today at
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. Today's 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.
Kadmon is submitting its New Drug Application (NDA) to the
"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
"The remarkable results achieved with belumosudil in this advanced patient population demonstrate its broad potential to offer meaningful clinical benefit to cGVHD patients," said
"Treatment with belumosudil has demonstrated compelling results for this major unmet medical need. Importantly, belumosudil has been very well tolerated, which allows trial participants to stay on therapy and achieve meaningful responses," said
Conference Call and Webcast
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To participate in the conference call, please dial (866) 762-3021 (domestic) or +1 (703) 925-2661 (international) and reference the conference ID: 9177687. The accompanying slides will be available for download on Kadmon's website.
ROCKstar (KD025-213) is an ongoing open-label trial of belumosudil (KD025) in adults and adolescents with cGVHD who have received at least two prior lines of systemic therapy. Patients were randomized to receive belumosudil (KD025) at 200 mg QD or 200 mg BID, enrolling 66 patients per arm. The primary endpoint of the study is Overall Response Rate (ORR). Statistical significance is achieved if the lower bound of the 95% CI of ORR exceeds 30%. The ORR endpoint was met at the interim analysis, conducted two months after completion of enrollment, with ORRs of 64% and 67% in the respective arms. At the study's primary analysis, conducted six months after completion of enrollment, belumosudil achieved ORRs of 73% and 74% in the respective arms. Kadmon plans to submit results from the ROCKstar primary analysis for presentation at an upcoming scientific meeting.
About Belumosudil (KD025)
Belumosudil (KD025) is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response. In an earlier Phase 2 study of belumosudil in cGVHD patients (KD025-208), belumosudil achieved an ORR of 65%. Data from this study led to FDA Breakthrough Therapy Designation by the FDA for belumosudil for the treatment of patients with cGVHD who have received at least two prior lines of systemic therapy. The FDA has also granted Orphan Drug Designation to belumosudil for the treatment of cGVHD. Kadmon is submitting its NDA for belumosudil under the FDA's Real-Time Oncology Review (RTOR) pilot program. In addition to cGVHD, belumosudil is being studied in an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209).
cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in
Kadmon is a biopharmaceutical company developing innovative products for significant unmet medical needs. Our product pipeline is focused on inflammatory and fibrotic diseases as well as immuno-oncology.
Forward Looking Statements
This press release contains forward-looking statements. Such statements may be preceded by the words "may," "will," "should," "expects," "plans," "anticipates," "could," "intends," "targets," "projects," "contemplates," "believes," "estimates," "predicts," "potential" or "continue" or the negative of these terms or other similar expressions. Forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. We believe that these factors include, but are not limited to, (i) the impact of COVID-19 on our ability to conduct our business or our clinical trials, (ii) the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; (iii) our ability to advance product candidates into, and successfully complete, clinical trials; (iv) our reliance on the success of our product candidates; (v) the timing or likelihood of regulatory filings and approvals, including an NDA regarding belumosudil (KD025); (vi) our ability to expand our sales and marketing capabilities; (vii) the commercialization of our product candidates, if approved; (viii) the pricing and reimbursement of our product candidates, if approved; (ix) the implementation of our business model, strategic plans for our business, product candidates and technology; (x) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (xi) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xii) costs associated with defending intellectual property infringement, product liability and other claims; (xiii) regulatory developments in