Vertex Pharmaceuticals reports ‘positive’ Phase 1/2 VX-880 results

Vertex Pharmaceuticals presented data on all patients dosed in Parts A and B of 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 with impaired hypoglycemic awareness and severe hypoglycemic events. All six patients treated with VX-880 had undetectable fasting C-peptide at baseline, a history of recurrent SHEs in the year prior to treatment and required an average of 34.0 units of insulin per day. Following treatment, all six patients demonstrated endogenous insulin secretion, improved glycemic control as measured by HbA1c, improved time-in-range on continuous glucose monitoring, and reduction or elimination of exogenous insulin use. Patients with greater than 90 days of follow-up had elimination of SHEs in the evaluation period. One patient in Part A received a half-target dose of VX-880 and was followed for approximately nine months, at which time this patient received a second half dose. This patient subsequently withdrew consent and was therefore not evaluable for the primary endpoint. Two patients treated with VX-880 had at least 12 months of follow-up after the last infusion and were therefore evaluable for the study’s primary efficacy endpoint of elimination of SHEs between Day 90 and Month 12 with a reduction of HbA1c. Both of these patients met the criteria for the primary endpoint of the study. In addition, these two patients are insulin independent. Patient A1 had HbA1c of 5.3% at Month 21, compared to 8.6% at baseline, and Patient B1 had HbA1c of 6.0% at Month 12, compared to 7.6% at baseline. Both patients showed over 95% time-in-range based on continuous glucose monitoring. The ADA recommended target is greater than or equal to70%. The three additional patients in Part B, each administered the full target dose of VX-880 given as a single infusion, have follow-up between 29 and 90 days and have shown endogenous insulin secretion, reduction in HbA1c, improvements in glucose time-in-range, and reductions in daily exogenous insulin use. Their trajectory is consistent with that observed in the two patients with more than one year of follow-up at equivalent periods of follow-up after VX-880 infusion. VX-880 has been generally well tolerated in all patients dosed to date. The majority of AEs were mild or moderate, and there were no serious AEs related to VX-880 treatment. The most common AEs were dehydration, diarrhea, hypomagnesemia and rash. As previously reported, one subject had SHEs in the perioperative period. As a result of these safety and efficacy data in Parts A and B, the independent data review committee has recommended moving to Part C of the trial, which allows for concurrent dosing of patients at the full target dose of VX-880.