Vertex Presents Positive VX-880 Results From Ongoing Phase 1/2 Study in Type 1 Diabetes at the American Diabetes Association 83rd Scientific Sessions
– All six patients treated with VX-880 engrafted islet cells, produced endogenous insulin (C-peptide) and had improved glycemic control while reducing or eliminating insulin use –
– The two patients with at least one year of follow-up met the criteria for the primary endpoint of elimination of severe hypoglycemic events (SHEs) and HbA1c <7.0 –
– Both of these patients also achieved insulin independence with HbA1c values of 5.3% and 6.0% –
– VX-880 generally well tolerated in all patients dosed to-date –
– Based on these results, trial advanced to Part C with concurrent dosing –
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 (not related to adverse events [AEs]) 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 (<7.0% or a decrease of at least 1% compared to baseline). 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
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.
“These data represent a foundational advance in the potential treatment of T1D, bringing us one step closer to a potentially curative therapy for patients who are waiting,” said
“The reproducible efficacy across multiple patients and endpoints, including the level of glucose control and the elimination of SHEs, observed in this trial is highly unusual in T1D patients treated with exogenous insulin, wherein only ~25% of people with T1D meet the recommended HbA1c target of 7.0%, and is truly remarkable,” said
These data were presented during the
VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy manufactured using proprietary technology. VX-880 is being evaluated for patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. VX-880 has the potential to restore the body’s ability to regulate glucose levels by restoring pancreatic islet cell function, including glucose responsive insulin production. VX-880 is delivered by an infusion into the hepatic portal vein and requires chronic immunosuppressive therapy to protect the islet cells from immune rejection.
The VX-880 trial has expanded to additional sites that are active and enrolling in
VX-880 was recently granted PRIME designation by the
About the VX-880 Phase 1/2 Clinical Trial
The clinical trial is a Phase 1/2, multi-center, single-arm, open-label study in patients who have T1D with impaired hypoglycemic awareness and severe hypoglycemia. This study is designed as a sequential, multi-part clinical trial to evaluate the safety and efficacy of VX-880. Approximately 17 patients will be enrolled in the clinical trial. Enrollment is ongoing in this study.
VX-264 is an investigational cell therapy in which allogeneic human stem cell-derived islets are encapsulated in a channel array device designed to shield the cells from the body’s immune system. VX-264 is designed to be surgically implanted and is currently being evaluated for patients with T1D.
About the VX-264 Phase 1/2 Clinical Trial
The clinical trial is a Phase 1/2, single-arm, open-label study in patients who have T1D. This will be a sequential, multi-part clinical trial to evaluate the safety, tolerability and efficacy of VX-264. Approximately 17 patients will be enrolled in the global clinical trial. Enrollment is ongoing in this study.
About Type 1 Diabetes
T1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, leading to loss of insulin production and impairment of blood glucose control. The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood glucose levels. High blood glucose can lead to diabetic ketoacidosis and, over time, to complications such as kidney disease/failure, eye disease (including vision loss), heart disease, stroke, nerve damage and even death.
Due to the limitations and complexities of insulin delivery systems, it can be difficult to achieve and maintain balance in glucose control in people with T1D. Current standards of care do not address the underlying causes of the disease, and there are limited treatment options beyond insulin for the management of T1D; there is currently no cure for diabetes.
Founded in 1989 in
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, (i) statements by
International: +44 20 3204 5275