Vertex Pharmaceuticals’ islet cell diabetes med VX-880, which has shown promise in eliminating the need for insulin in a small group of patients, has been placed on a voluntary hold after two deaths deemed unrelated to treatment occurred in the ongoing trial.
The company provided a brief statement on the situation in a pipeline update posted Sunday, noting that two patient deaths had occurred. While they were deemed unrelated to VX-880, the company has placed the program on a protocol-specified pause pending a review of data by an independent data monitoring committee and global regulators.
Vertex said that the med’s safety so far has been consistent with immunosuppressives.
VX-880 is an allogeneic, stem cell-derived insulin-producing islet cell therapy being tested in a phase 1/2 study along with immunosuppressives in patients who have type 1 diabetes and impaired awareness of hypoglycemia and recurrent hypoglycemic events.
Vertex has completed enrollment of parts A, B and C, according to the update. The therapy has so far demonstrated islet cell engraftment and production of endogenous insulin in the 14 patients that have been dosed. All patients in part A and B, except for one who withdrew, demonstrated glycemic control and no longer required insulin.
In a June update with just six patients reporting, Vertex said VX-880 was so far well tolerated and no serious side effects had been reported.
The FDA previously placed a clinical hold on the VX-880 program that lasted two months. Vertex said at the time that the hold was placed “due to a determination that there is insufficient information to support dose escalation with the product.”
The drug made waves in October 2021 when a single patient reportedly began producing insulin after treatment. Vertex is already working on newer versions of the drug including VX-264, a similar therapy that’s delivered via a channel array device to avoid immunosuppressants.
A phase 1/2 trial for VX-264 has initiated with multiple patients dosed already, according to the Sunday update.