Mesoblast Ltd (NASDAQ:MESO) (ASX:MSB) shareholders have enjoyed a 10-bagger year, with shares in the company continuing up 38.89% intraday.
The leader in allogeneic cellular medicines for inflammatory diseases has announced that the United States Food and Drug Administration (FDA) has approved RYONCIL® (remestemcel-L) as the first mesenchymal stromal cell (MSC) therapy in the country.
The therapy is also the sole FDA-approved treatment for steroid-refractory acute graft-versus-host disease (SR-aGvHD) in children aged two months and older, including adolescents.
Dr Joanne Kurtzberg, a transplant physician and Director of the Marcus Center for Cellular Cures at Duke University Medical (TASE:PMCN) Center, stated, “Steroid-refractory acute graft versus host disease is a devastating condition with an extremely poor prognosis.
"From today we are able to offer RYONCIL, the first FDA-approved treatment which will be life saving for so many children and will have a great impact on their families.”
Impressive results
Approximately 10,000 allogeneic bone marrow transplants are conducted annually in the US, including 1,500 in children. Nearly 50% of these patients develop acute graft-versus-host disease (aGvHD) and about half do not respond to steroids, the current first-line therapy.
A pivotal Phase 3 clinical trial of RYONCIL demonstrated a 70% overall response rate by Day 28 in children with SR-aGvHD, even in severe cases. Furthermore, more than 85% of patients completed the treatment without interruption.
“We are very pleased that the FDA has granted approval of RYONCIL® and are proud of the company’s commitment to the GVHD community in bringing this important new treatment to children and families with no other acceptable options,” Mesoblast CEO Dr Silviu Itescu said.
“With RYONCIL approval by FDA, Mesoblast has demonstrated the ability to bring the first MSC product to market.
"We will continue to work closely with FDA to obtain approval of our other late-stage products, including REVASCOR® for cardiovascular diseases and rexlemestrocel-L for inflammatory pain indications, as well as expanding the indications for RYONCIL in both children and adults with inflammatory conditions.”
RYONCIL will soon be available at US transplant centres and treating hospitals.
Significant turnaround
Today’s news marks a remarkable turnaround for Mesoblast, which raised A$36 million at just A$0.30 per share around this time last year, following a mid-2023 decline in its share price after the FDA requested additional data on RYONCIL®.
Dr Silviu Itescu previously detailed the recovery process in an interview with The Australian, highlighting how the company worked directly with the FDA to address concerns.
In September 2023, Mesoblast representatives travelled to the United States to present their case, ultimately paving the way for the FDA's approval of RYONCIL and restoring confidence in the company.
“Nothing beats face-to-face meetings,’’ Professor Itescu told The Australian.
“We met with them at FDA headquarters in September, we re-presented our data to a group of the leadership around a table, and presented to them both the clinical data and the manufacturing data, the science behind how the product works. And that was clearly a positive meeting.”
The FDA initially wanted more data. However, after fronting up professor Itescu said “instead, what they said was ‘no, we’ve thought this through, and you’ve convinced us that the clinical data are very strong’.
“You’ve got enough to get approved for children – go ahead and file, and then consider how to take the product into adults.”
Mesoblast’s price rise today of $2.75 compares to a 12-month low of just 25.5c.