"The results from our first clinical study in cGVHD demonstrated that belumosudil achieves meaningful, long-lasting responses and is very well tolerated in refractory cGVHD patients," said
Results from KD025-208, the open-label, Phase 2a clinical trial of belumosudil, which enrolled 54 cGVHD patients who had received one to three prior lines of therapy, demonstrated a pooled Overall Response Rate (ORR) of 65% across the three patient cohorts. Responses were achieved across patient subgroups, including in patients with four or more organs involved. Responses were durable, with a median duration of 35 weeks in responders. In addition, 50% of responders experienced a clinically meaningful improvement in symptoms, as measured by at least a 7-point decrease in the Lee cGVHD Symptom Scale score. Belumosudil was also shown to be steroid sparing: 67% of all patients were able to reduce steroid doses and 19% of patients completely discontinued steroids. Belumosudil was well tolerated across all cohorts, with no drug-related serious adverse events and no increased risk of infection observed.
The publication, titled "ROCK2 Inhibition with Belumosudil (KD025) for the Treatment of Chronic Graft-Versus-Host Disease," is available online here as well as on the Kadmon website.
Belumosudil (KD025) is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and pro-fibrotic processes. The FDA granted Priority Review for the NDA for belumosudil for the treatment of cGVHD and assigned a PDUFA target action date of
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 clinical-stage biopharmaceutical company that discovers, develops and delivers transformative therapies for unmet medical needs. Kadmon's clinical pipeline includes treatments for immune and fibrotic diseases as well as immuno-oncology therapies.
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. Among those risks and uncertainties are risks related to market conditions, including market interest rates, and the trading price and volatility of Kadmon's common stock. We believe that these factors include, but are not limited to, (i) the initiation, timing, progress and results of our preclinical studies and clinical trials, and our research and development programs; (ii) our ability to advance product candidates into, and successfully complete, clinical trials; (iii) the impact of the COVID-19 pandemic on our business, workforce, patients, collaborators and suppliers, including delays in anticipated timelines and milestones of our clinical trials and on various government agencies who we interact with and/or are governed by; (iv) our reliance on the success of our product candidates; (v) the timing or likelihood of regulatory filings and approvals, especially in light of the COVID-19 pandemic; (vi) our ability to expand our sales and marketing capabilities; (vii) the commercialization, pricing and reimbursement of our product candidates, if approved; (viii) the implementation of our business model, strategic plans for our business, product candidates and technology; (ix) the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology; (x) our ability to operate our business without infringing the intellectual property rights and proprietary technology of third parties; (xi) costs associated with defending intellectual property infringement, product liability and other claims; (xii) regulatory developments in