In an interim performance update focusing on the past nine months, dual-listed cellular medicines company Mesoblast (ASX: MSB) reported it had achieved a “strong financial position” and made progress with its lead candidate remestemcel-L, a drug being developed to treat severe cytokine release syndromes.
Mesoblast said it saw its revenues increase by 113% to US$31.5 million, citing a 127% increase in milestone revenues from strategic partnerships as the lead caveat behind the improvement.
Additionally, Mesoblast reduced its loss after tax by 34% to US$45.3 million in the past nine months.
The company reported that it had over US$60 million in cash on hand at the end of Q1 2020 with a successful US$90 million capital raise conducted in recent months adding to its coffers.
Fresh capital is expected to be used to enable a commercial launch of lead drug Ryoncil in the US, treating graft versus host disease (GvHD), manufacturing scale-up and continuing its clinical programs.
With financial performance improving, Mesoblast said it was now focusing its efforts on its lead drug remestemcel-L, better known as Ryoncil for the remainder of this year.
“Our biologics licence application for marketing approval of Ryoncil in children with steroid-refractory acute graft versus host disease is currently under priority review by the FDA, and we hope to be able to make the product available to patients suffering with this life-threatening inflammatory condition during 2020,” Mesoblast chief executive officer Dr Silviu Itescu said.
The FDA set what is known as a “prescription drug user fee act (PDUFA)” action date of 30 September 2020, and if approved, Mesoblast will make Ryoncil immediately available in the US.
A further operational boost could also come in the form of the potential use of Ryoncil in treating COVID-19 patients.
The drug is currently undergoing a phase 3 randomised controlled trial in the US on the back of “remarkable pilot data” from use of remestemcel-L in COVID-19 infected patients with moderate to severe acute respiratory distress syndrome (ARDS).
Mesoblast reported that when its drug was administered to 12 patients under compassionate use, 75% of ventilator-dependent patients were able to come off ventilators within 10 days and were discharged from hospital.
Over the past nine months and based on safety and efficacy data for remestemcel-L in SR-aGVHD, Mesoblast submitted an investigational new drug (IND) application for use of remestemcel-L in the treatment of patients with ARDS caused by COVID-19 and was cleared by the FDA.
Currently, a 300-patient phase 3 randomised controlled trial in ARDS patients from COVID-19 is underway in 30 sites across the US, “with planned interim analyses that may result in stopping the trial early for efficacy or futility”, the company said.
In terms of looking forward and setting targets for the next 12 months, Mesoblast said it intends to focus on launching Ryoncil in the US by the end of 2020 despite the impacts of COVID-19 on all areas of the global economy.
Other milestone targets include completing phase 3 trials for both Ryoncil treating ARDS and Revascor treating advanced and end-stage heart failure.
Furthermore, another phase 3 trial is being conducted of MPC-06-ID for chronic low back pain due to degenerative disc disease.
Mesoblast said final study visits for all patients have been completed, while ongoing quality review of all data is also being finalised at relevant study sites, “with a data readout planned for mid-2020”, the company said.
Earlier this week, Mesoblast told the market that clinical outcomes of its allogeneic mesenchymal stem cell (MSC) medicine in children and adults were published in three peer-reviewed articles and an accompanying editorial in the May issue of Biology of Blood and Marrow Transplantation, the official publication of the American Society for Transplantation and Cellular Therapy.
Mesoblast shares were almost 4% higher to $3.98 in morning trade.