Researchers Hit Pause Button On Promising Diabetes Treatment

Researchers Hit Pause Button On Promising Diabetes Treatment


Vertex Pharmaceuticals has temporarily paused a Phase I/II study of its innovative islet cell therapy, VX-880, designed for individuals with type 1 diabetes. This decision was prompted by the unfortunate occurrence of two patient deaths during the trial. 

In a reassuring statement, the Boston-based company emphasized that these fatalities were deemed “unrelated” to the investigational treatment, and the study pause was implemented as a “protocol-specified” precautionary measure.

To ensure a thorough and comprehensive review of the trial data, global regulatory authorities and an independent data monitoring committee will collaborate to assess the totality of information gathered during the study. VX-880 stands out as a stem cell-derived insulin-producing islet cell therapy developed by Vertex. Its primary goal is to restore the pancreas’ ability to release insulin in response to blood glucose levels, offering a potential breakthrough in the treatment of type 1 diabetes.

Administered through the hepatic portal vein and used alongside standard immunosuppression, VX-880 has been undergoing evaluation in a three-part Phase I/II study. This particular trial focuses on patients with type 1 diabetes who experience recurrent hypoglycemic events and an impaired awareness of hypoglycemia. Notably, as of the last data cut-off, Vertex had successfully dosed 14 patients, all of whom exhibited islet cell engraftment and demonstrated the capacity to produce endogenous insulin.

Beyond the temporary pause due to the unfortunate patient deaths, Vertex reported promising results for VX-880. The therapy not only lowered HbA1c levels in patients to below 7% but also eliminated the need for exogenous insulin. The safety profile of VX-880 was consistent with immunosuppressives, indicating its potential as a well-tolerated treatment option.

It’s worth noting that the journey of VX-880 has faced regulatory challenges in the past. In May 2022, the FDA imposed a clinical hold on the therapy, expressing concerns about dose escalation. However, the regulatory pause was lifted in July 2022, allowing the study to proceed.

In addition to the updates on VX-880, Vertex provided insights into several other programs in its recent press announcement. Notably, the absence of highly-anticipated data from the Phase III program of its acute pain candidate, VX-548, has raised curiosity. While the company completed three Phase III studies for VX-548 in acute pain, results are anticipated to be available in early 2024, adding another layer of anticipation to Vertex’s dynamic portfolio.

Are you worried about this setback in diabetes research? Why or why not? Leave your thoughts in the comments below.

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