Chronic Transplant Anti-Host Disease (cGVHD) Breakthrough Drug! Total reliever rate (ORR) for oral ROCK2 inhibitor belumosudil treatment s 70%!

May 24, 2020 /
BiovalleyBIOON/ -- Kadmon Holdings Has recently announced the primary analysis of the primary analysis of the primary analysis of the primary analysis of the primary analysis of the oral selective Rho-associated curly helix protein kinase 2 (ROCK2) inhibitor belumosudil (KD025) for the treatment of chronic transplant anti-host disease (cGVHD) criticalclinical
trial spresrocSt (KD025-213), NC0364048This is an ongoing randomized, multi-center, open label Phase II study in adult sGVHD adults and adolescentpatients who have previously received at least 2 systemic therapiesIn the study, patients were randomly assigned to two groups, treated with 200 mg (200mg QD) per day and 200mg (200mg BID) daily, in 66 patients in each groupIn this study, if the lower limit of 95% CI of the total mitigation rate (ORR) exceeds 30%, it is statistically significantpreviously published results show that the study reached the ORR endpoint in a mid-term analysis (October 2019) conducted two months after the completion of the patient's admission to the groupThe data show that the ORR in the KD025 200mg QD group and the 200mg BID group were 64% (95% CI: 51%, 75%, p 0.0001), 67% (95% CI: 54%, 78%, p 0.0001), respectively, which is statistically significant and clinically significantthe results of the positive top line published this time: in the main analysis carried out six months after the completion of the patient's admission, theThe ORR in the KD025 200mg QD group and the 200mg BID group were 73% (95%CI: 60%, 83%, p 0.0001), 74% (95% CI: 62%, 84%, p 0.0001), with clinical and statistical significanceIn addition,were observed in all key patient subgroups for therapeutic remission,were observed in all affected organ systems, including in organs with fibrosis as the data continued to mature, 49 percent of the remissions remained in the initial analysis for at least 20 weeks of therapeutic remission the study, the median treatment time was 29 weeks, and the median remission time had not yet reached In the study, belumosudil had good tolerance, adverse reactions consistent with expectations in the patient population No cytomegalovirus (CMV) infection or reactivation was found, and no significant drug-related cell reduction was found "cGVHD is an area with significant unmet medical needs, and belusudmoil has shown compelling results in treating late-stage cGVHD," said Madan Jagasia, chief medical officer of the Vanderbilt-Ingram Cancer Center (VICC) and a professor of medicine at Vanderbilt University Medical Center, who KD025-208 and ROCKstar research, and chairwoman of the ROCKstar Steering Committee Importantly, belumosudil's tolerance was very good, which enabled the trial participants to adhere to the treatment and obtain meaningful therapeutic remission These results suggest that , if approved, belumosudil has the potential to be the cornerstone of the cGVHD treatment model and will bring meaningful and sustainable benefits to patients with this serious disease "
belumosudil (KD025) chemical structure and mechanism of action
KD025 is a selective oral inhibitor of Rho-associated curly helix protein kinase 2 (ROCK2), a signaling pathway that regulates immune response and fibrosis pathways KD025 inhibits ROCK2 signaling pathways, lowers inflammatory Th17 cells, increases regulatory T (Treg) cells, rebalances immune responses, and treats immune dysfunction In addition to cGVHD, KD025 is conducting a Phase II clinical trial (KD025-209) for adult diffuse progressive progressive progressive sclerosis of Previously, KD025 was granted breakthrough drug eligibility (BTD) and orphan drug qualification (ODD) by the U.S Food and Drug Administration (
FDA ) for the treatment of patients with cGVHD who had previously received at least two systems cGVHD is a common fatal complication after hematopoietic stem cell transplant In cGVHD, transplanted immune cells (grafts) attack the patient's cells (hosts), causing inflammation and fibrosis of several tissues, including the skin, mouth, eyes, joints, liver, lungs, esophagus, and gastrointestinal tract In the United States, there are currently about 14,000 patients with cGVHD, with about 5,000 new cases added each year , Kadmon is currently passing through the U.S Food and Drug Administration (
The FDA ) real-time oncology review (RTOR) pilot project to submit belumosudil's new drug application (NDA), which is expected to be completed by the fourth quarter of 2020 Harlan W Waksal, President and CEO of Kadmon, said: "We are very pleased with the strong and lasting response of belumosudil in this incurable disease We are pleased to participate in the real-time oncology
assessment pilot project the FDA , which aims to accelerate the effective assessment of urgently needed drugs We will continue to work closely with FDA during the submission of the NDA and data review "The remarkable results achieved by belumosudil in this late-patient population demonstrate the broad potential to deliver meaningful clinical benefits to patients with cGVHD," said Dr Sanjay K Aggarwal, Senior Vice President of Clinical Development, Kadmon We look forward to sharing the full data set of ROCKstar research at the upcoming Medical Conference "
(biovalleybioon.com) original origin: Kadmon announces topline from results study results results study out KD025 in GVHD