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A breakthrough approval by the US Food and Drug Administration for Mesoblast’s (ASX: MSB) treatment for a common stem cell transplant complication has driven a 40 per cent surge in shares of the Melbourne-based biotech.
Mesoblast shares shot to a peak of $2.90 this afternoon, their highest level of trading in four years, after the FDA approved Ryoncil as the first mesenchymal stromal cell (MSC) therapy in the United States.
Mesoblast says the therapy is the only MSC treatment approved in the US for any indication, and the only approved therapy for steroid-refractory acute graft versus host disease (GVHD) in paediatric patients aged from two months to adolescence.
The treatment provides an alternative to steroid therapy.
Mesoblast has previously noted that there are no treatments available for children under 12 with GVHD, a major complication of allogeneic stem cell transplantation. The disease is typically treated by high-dose steroids in many patients, although the prognosis is poor for patients that don’t respond to steroid treatment.
“Steroid-refractory acute graft versus host disease is a devastating condition with an extremely poor prognosis,” says transplant physician Dr Joanne Kurtzberg, director of the Marcus Center for Cellular Cures at Durham in North Carolina.
“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.”
Mesoblast notes that about 10,000 patients in the US undergo an allogeneic bone marrow transplant every year, among them about 1,500 children.
About 50 per cent of patients develop acute GVHD and almost half of those don’t respond to steroids. This compares with a Phase 3 trial of children that showed 70 per cent achieved an overall response by day 28 of treatment with Ryoncil, a measure that the company says predicts survival in acute GVHD.
“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,” says Dr Silviu Itescu, the CEO of Mesoblast.
“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.”
Mesoblast is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions with Revascor its second product with clinical data sufficient for FDA regulatory review. Revascor targets paediatric congenital heart disease and adults with ischemic HFREF (heart failure with reduced ejection fraction).
Mesoblast says that Ryoncil’s immunomodulatory effects, including inhibition of T-cell activation and secretion of pro-inflammatory cytokines, also position the therapy for potential other indications in diseases with excessive inflammation. T-cells are part of the immune system and develop from stem cells in the bone marrow.
Following the FDA approval announced today, Ryoncil will be available in the United States at transplant centres and hospitals.
Mesoblast announced in its September-quarter update that inventory of Ryoncil has been manufactured and that there is an “established supply chain to ensure cryopreserved product is available for delivery to meet the needs of each site immediately post approval, with ability to scale up as necessary going forward”.
“We have been working diligently to lay the groundwork for a successful launch of Ryoncil, including hiring select senior positions to implement a targeted commercial strategy since 50 per cent of paediatric transplants are performed at just 15 centres,” the company said in its update.
“Post approval implementation will initially target those centres with greatest experience using the Ryoncil product and highest volume, with staged rollout beyond.”
Shares in Mesoblast, which is also traded on the Nasdaq, changed hands for $2.88 – up 90c – at 12.46pm (AEDT).
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