Mesoblast’s allogeneic mesenchymal stem cell candidate remestemcel-L showed exceptional promise when given to 12 ventilator dependent COVID-19 patients with acute respiratory distress syndrome (ARDS). The 12 patients were given two intravenous infusions of remestemcel-L and within the first five days, ten patients (83%) survived, while nine patients (75%) were removed off ventilator support after a median follow up of ten days. Seven of the patients (58%) were discharged from the hospital.
The Phase 2/3 study is expected to be a 1:1 randomized, placebocontrolled trial in 240 COVID-19 patients with ARDS with a primary endpoint of 28-day mortality rate. The study is expected to initiate imminently, and management expects to complete patient enrollment in three months. An interim readout is expected at the completion of enrollment of one third of the planned study population.
Treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD)
The FDA has set a PDUFA action date of September 30, 2020 (six-month priority review) and if approved, Mesoblast will make RYONCIL immediately available in the United States.
Enrollment of 566 patients has been completed in a placebo-controlled Phase 3 trial to evaluate a single dose of Revascor in Class II/III CHF patients across multiple sites in North America. Patients with advanced heart failure constitute the majority of the patients enrolled in this clinical trial program.
In April 2017, the trial achieved a successful pre-specified interim futility analysis of the efficacy endpoint in the first 270 patients and an independent data monitoring committee formally recommended the continuation of the trial. Readout mid-2020.
The 404-patient Phase III in lower back pain has completed recruitment and final study visits have been initiated. Data is expected by mid-2020. A confirmatory Phase III is currently being planned with partner Grϋnenthal in Europe.