2nd time unlucky for Mesoblast as FDA rejects cell therapy again, sinking shares

Mesoblast’s second attempt to win over regulators to its off-the-shelf cell therapy has had no more success than the first try, sinking the Australian biotech’s stock.

The company had asked the FDA in February to reconsider approval of remestemcel-L for children with steroid-refractory acute graft-versus-host disease (SR-aGVHD). The reworked application contained what the company described as “substantial new information,” including efficacy and biomarker data, some of which were compared to a database of patients with aGVHD that the Mount Sinai medical system maintains.

But Mesoblast revealed today that it has received another complete response from the regulator, requiring yet more data. Undeterred by the previous knockbacks, the company said it’s still willing to do what it takes.

An FDA inspection of the biotech’s manufacturing process had “resulted in no observed concerns” and the regulator hadn’t raised any safety issues with the more than 1,300 patients who have received remestemcel-L across various trials, CEO Silviu Itescu pointed out in the release.

“We remain steadfast in making remestemcel-L available to both children and adults suffering from this devastating disease and have received substantial clarity in how to bring this much-needed product to these patients,” Itescu added.

When it turned down Mesoblast’s initial application, the FDA requested an additional trial. The company ignored that recommendation, which no doubt played a part in the latest rejection.

Now, it seems Mesoblast has got the message. The biotech said it will hold a Type A meeting with the FDA within the next 45 days to finalize the details of the trial, for which the company plans to enroll adult patients at highest risk of dying from SR-aGVHD.

“Mesoblast has generated pilot data through its emergency IND program in adults showing a survival benefit with remestemcel-L in this target population,” the company explained. “In line with our overall commercial strategy to expand into the adult SR-aGVHD indication, Mesoblast has already been working with leading investigators at various U.S. centers of excellence to establish the adult follow-on study protocol, potentially utilizing established clinical trials networks.”

Investors didn’t seem convinced, sending the biotech’s stock crashing 56% to just 47 cents on the Australian stock exchange over the course of Friday's trading. The company had already halted trading of its shares on the Nasdaq on Tuesday “until the earlier of the commencement of normal trading on Friday … or when the announcement is released to the market.”

The company ended March with just $48.8 million available as cash reserves, with another $40 million since raised from a private placement of its shares and potentially $40 million that could be drawn down from existing financing facilities if needed “subject to certain milestones,” Mesoblast revealed in its most recent earnings report.