Mesoblast boss Silviu Itescu calls for urgent FDA approval of its marquee drug
Mesoblast’s chief executive says its marquee treatment for a rare genetic disease can save children’s lives, releasing phase three clinical trial data to support his claim.
Stem cell-focused biotech Mesoblast has escalated its pursuit of the US Food and Drug Administration in the hope it will finally approve its marquee treatment for children with a rare genetic disorder.
Chief executive Silviu Itescu has called on the FDA to approve its drug, remestemcel-L, highlighting “substantial and durable long-term” survival rates the company achieved in phase three trials for children with acute versus graft host disease.
“The lack of any approved treatments for children under 12 means that there is an urgent need for a therapy that improves the dismal survival outcomes in children,” Dr Itescu said.
“We are at a pivotal juncture, we believe we have appropriately addressed issues raised by FDA in the complete response, and we are well funded in preparation for a potential first product approval and launch by mid-year.”
Mesoblast has been on a rollercoaster ride with US regulators. In August 2020, the US Oncologic Drugs Advisory Committee (ODAC) voted nine to one in favour of the remestemcel-L therapy – a decision that sent Mesoblast shares soaring to a six-year high at $5.22.
But 12 months later, the FDA recommended that Mesoblast conduct at least one additional randomised, controlled study in adults and/or children to provide further evidence of the effectiveness of remestemcel-L for aGVHD, sending the company’s shares into a nosedive.
Dr Itescu believes the company has now satisfied the agency.
“The substantial and durable long-term survival over four years we have reported today in children with SR-aGVHD (acute graft versus host disease) treated with remestemcel-L in our phase three trial underscore the many lives that could potentially be saved by making this therapy available as soon as possible to children with the most common life-threatening complication after bone marrow transplantation”.
Mesoblast shares fell 1.5 per cent to 98c on Wednesday. This compares with a 0.7 per cent gain across the broader sharemarket.
The company has been sweating on FDA approval to lift its fortunes. Overall survival rates in phase three trial showed those who were treated with remestemcel-L, were 63 per cent after one year, 51 per cent at two years, and 49 per cent at four years.
Dr Itescu said this compares with survival rates for those treated with the only FDA-approved drug, ruxolitinib, of 40-49 per cent per cent after one year of treatment and 25-38 per cent at two years.
It released its phase three trial results the same day as its latest quarterly earnings.
Mesoblast’s cash reserves as of September 30 were $US85.5m ($128.78m), with the potential to borrow an extra $US40m from its existing debt facilities.
In the three months to September 30, it has slashed its cash usage 22 per cent to $US14.3m compared with the previous corresponding period.
The company generated $US1.5m in revenue in the quarter, compared with $US3.6m during the same period in 2021. But it managed to reduce its loss from $US22.6m to $US16.9m.
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