Headquartered in New York City, biopharmaceutical company Kadmon Holdings (NYSE: KDMN) announced positive topline results from its pivotal trial of belumosudil (KD025) for the treatment of chronic graft-versus-host disease (cGVHD). Overall response rates of 73% and 74% respectively were achieved when 200mg of belumosudil was used once daily and twice daily. Complete responses were observed in all organ systems.
Chronic graft-versus host disease is a potentially fatal complication that commonly occurs after hematopoietic stem cell transplantation whereby transplanted immune cells (graft) attack the patient’s cells (host). This can lead to inflammation and fibrosis in skin, mouth, eye, joint, liver, lung, esophagus, and gastrointestinal tract tissues. Approximately 14,000 people in the U.S. are living with cGVHD, one of the nation’s major unmet medical needs.
Belumosudil is an oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2) which is a signaling pathway that modulates human inflammatory response. Aside from its potential use with cGVHD patients, the therapy is also being evaluated in a phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis.
Results top prior study further asserting drug’s efficacy
The statistically significant results were from the primary analysis of Kadmon’s ROCKstar trial of belumosudil in adult and adolescent patients with cGVHD who have received at least two prior lines of systemic therapy. They surpassed the results that were seen in the interim analysis that had overall response rates of 64% and 67% in the study’s respective arms. A total of 132 patients were enrolled in the ongoing study and the latest results were attained in a six-month period after enrollment.
Data from the previous phase 2 study earned Kadmon the U.S. Food and Drug Administration’s (FDA’s) Breakthrough Therapy Designation for belumosudil in cGVHD patients in addition to the FDA’s Orphan Drug Designation. The company plans to complete the submission of its new drug application (NDA) for belumosudil by the fourth quarter of 2020 under the FDA’s Real-Time Oncology Review pilot program.
President and CEO Dr. Harlan W. Waksal, stated, “Belumosudil has shown robust and durable responses across the spectrum of this difficult-to-treat disease and we are extraordinarily pleased with this outcome.”
The positive results demonstrate an even stronger efficacy of belumosudil with cGVHD patients compared to previous studies and represent a major step forward in the product’s potential commercialization. Kadmon is planning to submit the results from the trial for presentation at an upcoming scientific meeting.
The small cap biotechnology company has a diverse pipeline focused on inflammatory and fibrotic diseases in addition to immune oncology. Kadmon has received buy ratings from all five analysts that cover the stock with a wide target price range of $7.00 to $25.00. The shares were up 7.5% to $4.72 in midday trading on the day of the announcement. The company is scheduled to report second quarter performance on August 3rd.