Vertex surprised by FDA hold on diabetes trial

Trials for Vertex Pharmaceuticals' investigational stem cell-derived diabetes therapy have been halted by the U.S. FDA, taking the biotech by surprise.

The agency placed a clinical hold on the phase 1/2 clinical trial of VX-880, saying there was not enough information to support giving patients the full dose of the drug. The biotech said it was “surprised by the clinical hold placed on the study” and that the patients dosed so far had reported positive reports. VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using Vertex's proprietary technology.

So far, there have been three patients dosed in the study. The first two received a half-dose of the treatment. The first patient was able to stop using insulin less than a year after dosing, with data indicating his body is now able to control its blood sugar levels. The second patient in the study is also responding well to the treatment and has been able to cut insulin use by one-third.

The third patient was given a full dose of the treatment after an independent monitoring committee gave the nod to up the dosage. This patient is only 29 days out from dosing and has experienced no adverse events so far.

No serious adverse events have been reported in response to treatment. Side effects experienced included headache and hypomagnesemia, which were unrelated to treatment, according to Vertex.  

Wall Street investors have kept a close eye on VX-880, as the drug has the potential to reach blockbuster status, according to Bloomberg Intelligence. Type 1 diabetes is a genetic condition where the pancreas stops producing insulin. Left untreated, it can lead to serious problems in the kidneys, heart and eyes. The condition affects about 1.6 million Americans and there is currently no permanent cure.