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Islet Cell Therapy Reduces Insulin Dependence in Type 1 Diabetes

The phase 1/2 trial of VX880 remains on clinical hold in the US.

VX880, a pancreatic islet cell replacement therapy from Vertex Pharmaceuticals, reduced insulin dependence in the first 2 participants with type 1 diabetes (T1D) treated in a phase 1/2 study (NCT04786262).1

Data from the study were presented at the American Diabetes Association (ADA) 82nd Scientific Sessions Conference on June 6, 2022 in New Orleans, Louisiana, by James F. Markmann, MD, PhD, chief, division of transplantation, and surgical director, liver, pancreas and islet transplant programs, and clinical director, MGH transplant center.

“The glucose time-in-range data presented today at ADA demonstrate the remarkable glycemic control that can be achieved after treatment with VX-880,” Bastiano Sanna, PhD, executive vice president and chief, cell and genetic therapies, Vertex, said in a statement.2 “Elevations in blood sugar are important to control, as are fluctuations over time, as both increase the risk of complications in patients with T1D. The first two patients treated with VX-880 have not only achieved improved HbA1c and decreased insulin requirements, but also higher time-in-range. Taken altogether, these data provide further evidence of the potential for VX-880 as a functional cure for people living with T1D.”

The multi-center, single-arm, open-label study is enrolling patients with T1D with impaired hypoglycemic awareness and severe hypoglycemia. The study is the first part of a planned multi-part trial in which the first 2 patients received half the target dose. In the second part, 5 patients will receive the full target dose, the first of which has already been dosed. Concurrent dosing at the full target dose will occur in the third part to an ultimate planned enrollment of 17 patients.

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The study is ongoing in Canada but remains on clinical hold in the US due to “insufficient information to support dose-escalation" in the trial, despite a positive recommendation from an Independent Data Monitoring Committee to advance to full dosing.3 VX-880 has been generally well-tolerated in all patients dosed to date, mostly mild-to- moderateadverse events (AEs) and no serious treatment-related AEs.

“As a treating physician, I have seen the profound burden of this disease on patients, especially those who experience severe hypoglycemia. The ability to restore a patient’s islet function and improve glycemic control, and subsequently reduce exogenous insulin dependence, has significant potential to improve patients’ lives,” Steering Committee Chair for the trial Camillo Ricordi, MD, professor, surgery, and director, Diabetes Research Institute and Cell Transplant Center, University of Miami Miller School of Medicine, added to the statement.2 “These results from the first two patients treated with half of the target dose are remarkable and encouraging as we continue investigating treating patients with type 1 diabetes with this stem cell-derived therapy.”

Data presented at the ADA conference demonstrated glucose-responsive insulin production, improvements in glycemic control and reductions in exogenous insulin requirementsin both patients treated with half the target dose of VX-880.1 Investigators found significant increases from baseline in the blood glucose time-in-range between 70 and 180 mg/dL. The accepted ADA/European Association for the Study of Diabetes (EASD) target for time-in-range is 70%.

Blood glucose time-in-range increased from 40.1% at baseline to 99.9% at Day 270 in patient 1, who became insulin independent. Time-in-range increased from 35.9% at baseline to 51.9% at Day 150 in patient 2, who had a 30% reduction in exogenous insulin use. Data is not yet available for patient 3, who has been treated with the full dose.

“The potential impact of this treatment on patients cannot be overstated,” Markmann added.2 “This study shows a significant leap forward in the potential treatment of patients with type 1 diabetes.”

1.Markmann JF, Naji A, Rickels MR, et al. Stem Cell–Derived, Fully Differentiated Islet Cells for Type 1 Diabetes. Presented at: ADA 82nd Scientific Sessions Conference, June 6, 2022, New Orleans, Louisiana. Abstract #259-OR.
2. Vertex Presents New Data from VX-880 Phase 1/2 Clinical Trial at the American Diabetes Association 82nd Scientific Sessions. News release. Vertex pharmaceuticals. June 6, 2022. https://news.vrtx.com/press-release/vertex-presents-new-data-vx-880-phase-12-clinical-trial-american-diabetes-association
3. Vertex provides updates on phase 1/2 clinical trial of VX-880 for the treatment of type 1 diabetes. News release. Vertex Pharmaceuticals. May 2, 2022. https://news.vrtx.com/press-release/vertex-provides-updates-phase-12-clinical-trial-vx-880-treatment-type-1-diabetes