Drug Development, Results

Stem cell co Mesoblast reports PhIII trial success in children with GVHD

An Australian stem cell company tackling a disease that often befalls blood cancer patients says it’s met the primary endpoint of a Phase III trial — and it’s expecting an accelerated FDA OK any day now.

The company, called Mesoblast $MESO, was testing its allogeneic mesenchymal stem cell product called remestemcel-L. The off-the-shelf therapy is meant to treat Graft Versus Host Disease, a condition that often arises in blood cancer patients who’ve had bone marrow transplants. After these transplants occur, donor cells have been known to attack the patient, causing GVHD, which activates an inflammatory response and potential tissue damage in the skin, gut, and liver. When the condition is severe and the patient doesn’t respond to steroids, it can be fatal.

Joanne Kurtzberg

“These children are a very challenging patient population as they suffer from a particularly aggressive and life-threatening disease for which there are currently no available treatments,” Joanne Kurtzberg, the study’s lead investigator from Duke University Medical Center, said in a statement.

Mesoblast’s treatment was evaluated in 55 children enrolled in the company’s open-label Phase III trial. On day 28 after receiving the first dose of remestemcel-L, 69% of children had experienced a complete or partial response. That was deemed a statistically significant increase over historical control rate of 45%, according to data presented at a medical meeting.

The company hopes the therapy might have a longer-term benefit, as 50 patients who had completed 100 days of follow-up after receiving at least one remestemcel-L infusion saw a survival rate of 78%. That’s compared to 100-day survival rates as low as 30% in patients who fail to respond to steroid therapy.

Based on their interactions with the FDA, Mesoblast said it believes the results of the Phase III trial (along with the Day 180 safety and quality of life data) might be enough to get accelerated approval of remestemcel-L in the US.

The company’s stock is up nearly 13% in pre-market trading on the news.


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