Vertex Pharmaceuticals Incorporated presented new data from its Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived, fully differentiated islet cell therapy, in people with type 1 diabetes (T1D) with impaired hypoglycemic awareness and severe hypoglycemic events (SHEs). These updated data on 12 patients who received the full dose as a single infusion in Parts B and C of the trial are consistent with previously reported positive results in the VX-880 trial and reinforce the transformative potential of this therapy. At baseline, all patients in the study had undetectable fasting C-peptide (a marker of endogenous insulin secretion), a history of recurrent SHEs in the year prior to screening, and required an average of 39.3 (min, max; 19.8, 52.0) units of insulin per day.

Following a single infusion of VX-880 at the full dose, all 12 patients demonstrated islet cell engraftment and glucose-responsive insulin production by Day 90. At the latest visit, all patients had improved glycemic control and achieved ADA-recommended targets for both HbA1c below 7.0% and time-in-range above 70% on continuous glucose monitoring. Nearly all participants (11 of 12) had a reduction or elimination of exogenous insulin use at their last visit.

All patients had elimination of SHEs during the evaluation period (from Day 90 onward). Finally, all three patients who had at least one year of follow-up, and are therefore evaluable for the primary endpoint, met the primary endpoint of elimination of SHEs (from Day 90 after infusion) with HbA1c <7.0% and the secondary endpoint of insulin independence. VX-880 has been generally well tolerated.

The majority of adverse events (AEs) were mild or moderate, and there were no serious AEs related to VX-880 treatment. As previously reported, two patient deaths occurred, both unrelated to treatment with VX-880. The safety profile is generally consistent with the immunosuppressive regimen used in the study, the infusion procedure, and complications from long-standing diabetes.

These data were presented during the American Diabetes Association 84th Scientific Sessions Conference on June 21, 2024, in Orlando, Florida at an invited talk, ?Update on Clinical Trials Using Stem Cell Replacement,? as part of the joint ADA/IPITA symposium from 3:45 ? 5:15 p.m. ET.