Vertex’s Type 1 diabetes cell therapy heads back to clinic after FDA hold lifted

Vertex’s Type 1 diabetes cell therapy heads back to clinic after FDA hold lifted

Two months is evidently all it took for the FDA to investigate Vertex’s phase 1/2 trial for its Type 1 diabetes med, lifting a clinical hold that was placed in early May.

Vertex said Tuesday that the planned 17-person trial for VX-880 is back in business after the agency lifted a clinical hold that was originally placed “due to a determination that there is insufficient information to support dose escalation with the product.” At the time, Vertex’s chief medical officer Carmen Bozic, M.D., said in a statement that the hold was surprising.

Now, the company can exhale as the trial gets back on track. Vertex did not report whether the agency had requested any amendments to the dosing design, and the company hadn’t responded to a request for additional details by the time of publishing.

The med at play is an off-the-shelf stem cell therapy using islet cells, which produce hormones in the pancreas that help control glucose levels. In the phase 1/2 trial, Vertex is homing in on patients with severely low glucose levels and those who have “impaired awareness” of low glucose.

At the time of the hold, the company had completed the first of three phases of the trial testing half the target dose, reporting that one of the two patients achieved insulin independence by nine months post-treatment. The other was showing signs of improvement but hadn’t reached the same milestone.

The second portion of the study, now back underway, is testing the full target dose. One patient had been dosed in that group when the hold was slapped on, and Vertex expects to recruit four more. The final dosing group will be concurrent dosing of the full target dose.

VX-880 is one of two programs Vertex is working on to treat diabetes, although it’s the only one in the clinic. The second, still in a research phase, is encapsulated islet cells, which the company says could “potentially eliminate the requirement for immunosuppression by protecting the cells from the immune system.”

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