Mesoblast has partnered with the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) for a clinical trial investigating its Ryoncil (remestemcel-L) in steroid-refractory acute graft versus host disease (SR-aGvHD).
BMT CTN will develop and conduct the trial which will evaluate Ryoncil in patients aged 12 and older who have not responded to previous treatments such as corticosteroids and a second-line agent such as ruxolitinib.
As agreed in the Type A meeting with the US Food and Drug Administration (FDA) in September, Mesoblast will submit the final protocol to the agency after it has been reviewed by two independently appointed committees from the National Heart, Lung, and Blood Institute.
The announcement comes after several setbacks from the FDA, when the agency requested additional data from the company to approve Mesoblast’s biologics licence application (BLA) resubmission for the paediatric population on 4 August 2023.
Mesoblast resubmitted the BLA for Ryoncil in January 2023, with additional data that addressed a complete response letter (CRL) issued by the FDA dating back to September 2020. The resubmitted BLA included survival data from a paediatric Phase III trial, as well as efficacy data.
The single-arm prospective Phase III clinical trial (NCT02336230) for Ryoncil in the paediatric population met its primary endpoints. Results published in the Journal of the American Society for Transplantation and Cellular Therapy showed an overall survival rate of 74.1% at day 100 and 68.5% at day 180.
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In a press release accompanying the announcement, chair-elect of the BMT CTN steering committee and professor of internal medicine and paediatrics John Levine said: “The clinical data from children treated with Ryoncil which support this trial are very compelling.”
Ryoncil is a human bone marrow-derived mesenchymal stem cell (MSC) therapy. It is indicated for treatment in children and adults with SR-aGvHD in Japan, Canada, and New Zealand. Ryoncil is also being developed for ulcerative colitis, Crohn’s disease, and acute respiratory distress syndrome.
According to GlobalData’s Pharma Intelligence Centre, Ryoncil is forecasted to generate $206m in sales in 2029.
GlobalData is the parent company of Clinical Trials Arena.
Several drugs are in development for the condition such as Equillium’s itolizumab in the Phase III EQUATOR clinical trial (NCT05263999), and Takeda’s Entyvio in the Phase III GRAPHITE study (NCT03657160).
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