FDA lifts hold on Vertex's diabetes stem cell therapy after just two months under the microscope
Vertex Pharmaceuticals has received the FDA go-ahead to continue studies for its diabetes stem cell therapy.
The treatment, which Vertex has attempted to describe as a “cure,” had been on hold at the agency since May, when regulators expressed concerns about a lack of information ahead of the dose escalation portions of the study. With the hold lifted, Vertex will begin enrolling patients again at the high dose level.
Researchers have plans to test the drug in five patients sequentially at the high level before moving on to a concurrent dose cohort, using the high dose. So far, Vertex has administered the high dose to one patient and the low dose to two patients, the company said Tuesday. Vertex aims to enroll 17 total patients across all three parts.
Known as VX-880, the therapy is designed to replace insulin-producing cells that type 1 diabetes patients lose with lab-grown insulin-producing cells. Last October, the company released “extraordinary” results from the first patient, which showed a drop in their HbA1c, a metric for blood sugar levels, from an average of 8.6% to 7.2%.
That patient no longer needs to take synthetic insulin, and results from a second patient showed a HbA1c drop from 7.5% to 7.1%. But the FDA slapped the hold after this result in a move Vertex said was “surprising” at the time. The company has not released detailed safety data.
But now that the hold has been lifted, Jefferies analyst Michael Yee believes the therapy remains on track to read out more data late this year or in early 2023. Yee noted the hold was resolved in two months, a much shorter period than is typical (Yee said the industry average is four to five months).
“The initial clinical hold in May was unexpected and in our view was likely due to FDA conservatism around Cell and Gene Therapy programs and wasn’t driven by any specific safety issue,” Yee wrote. “This is a good sign there weren’t major issues with the clinical or preclinical data (safety, in our view, has been very good).”
Next up for Vertex will likely be data for the first patient at the high dose, which are yet to be reported.
Vertex had racked up a string of recent successes outside its cystic fibrosis franchise before the hold, touting positive results in a mid-stage study on a non-opioid painkiller earlier this year. The diabetes therapy is another of the biotech’s efforts to expand beyond CF.
