Cell Therapy News/Articles, page-126

Mesoblast sees US trial success

Mesoblast chief executive and managing director Professor Silviu Itescu.

• SARAH-JANE TASKER
• The Australian
• 3:10PM February 22, 2018
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Stem cell therapy company Mesoblast’s treatment for a potentially fatal disease that impacts paediatric leukaemia patients has met the primary goal of a late-stage US trial.

Shares in the company closed almost 12 per cent higher yesterday at $1.57 after it told shareholders that its phase three trial for its therapy for Graft Versus Host Disease (GVHD) had shown a survival rate of 78 per cent in the children enrolled in the trial.

Billionaire small-cap investor Alex Waislitz’s Thorney Investment Group has a 5.2 per cent stake in the company (MSB), while Mesoblast chief executive Silviu Itescu accounts for 14.5 per cent of the stock.

Dr Itescu said the therapy, remestemcel-L, was well positioned to be Mesoblast’s first revenue-generating commercial product in the US.

GVHD occurs when a cancer patient has a bone-marrow transplant and that transplant attacks their organs. The mortality rate of this disease is as high as 80 per cent and there are no drugs that can effectively treat the condition.

The phase three trial was required by the US regulator, the Food and Drug Administration, ahead of a final submission for drug approval. The trial involved 55 children across 32 sites, and on day 28 after receiving the first dose of remestemcel-L, 69 per cent of children had experienced a complete or partial response. There was a survival rate of 78 per cent in the trial’s first 51 patients who had completed 100 days of follow up, which the company said was an encouraging indicator of potential longer-term benefit. This was in contrast to survival rates as low as 30 per cent at day 100 in patients who fail to respond to initial steroid therapy.

The trial’s senior investigator, Joanne Kurtzberg, said the children were a very challenging patient population as they suffered from a particularly aggressive and life-threatening disease for which there were currently no effective treatments.

“We are now seeing a highly encouraging reduction in the early mortality rate in the vast majority of children who receive remestemcel-L,” Dr Kurtzberg said.

“Furthermore, treatment with remestemcel-L is very well tolerated and does not cause problems with kidney function or decreased immune function which usually occur with other agents.

“This is wonderful news for patients and their families who are in desperate need of an effective therapy for this devastating complication of bone marrow transplantation.”