New Data on VX-880 Brings Hope for T1D Patients

Vertex Pharmaceuticals has presented new data from their Phase 1/2 clinical trial of VX-880, an investigational stem cell-derived islet cell therapy for people with type 1 diabetes (T1D) who have impaired hypoglycemic awareness and severe hypoglycemic events. The 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, reinforcing the transformative potential of this therapy. The data were presented at the American Diabetes Association 84th Scientific Sessions Conference on June 21.

What is VX-880?

VX-880 is an experimental stem cell-derived islet cell therapy for T1D patients who have lost the ability to produce insulin naturally. The therapy provides fully differentiated beta cells that can replace those that have been destroyed by the autoimmune response that characterizes T1D. VX-880 differs from other types of cell therapy in its ability to deliver a large number of healthy insulin-producing cells that can engraft and function within the body.

Study Results: What Do They Mean?

The study evaluated the efficacy and safety of VX-880 in people with T1D who had impaired hypoglycemic awareness and severe hypoglycemic events. The patients received a single infusion of VX-880 at the full dose and were evaluated over the course of several months. In all 12 patients, the therapy demonstrated islet cell engraftment and glucose-responsive insulin production by Day 90. Almost all participants (11 of 12) had a reduction or elimination of exogenous insulin use at their last visit, and all patients had elimination of severe hypoglycemic events (SHEs) during the evaluation period from Day 90 onward.

Three patients who had at least one year of follow-up met the primary endpoint of elimination of SHEs from Day 90 after infusion with HbA1c less than7.0% and the secondary endpoint of insulin independence.

The safety profile of VX-880 has been generally well tolerated, with the majority of adverse events being mild or moderate. There were no serious adverse events 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.

Potential for a Curative Therapy

The results of the VX-880 trial are remarkable and support the potential for a curative therapy for T1D patients. Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, stated, “These remarkable data add to the growing body of evidence for VX-880 as a potentially curative therapy for T1D. As we plan toward pivotal development, we are pleased to have secured regulatory approval to expand study enrollment and look forward to advancing this program for patients who have long awaited a transformative therapy.”

Long-tail Keywords:

VX-880

Type 1 diabetes

Islet cell therapy

Impaired hypoglycemic awareness

Severe hypoglycemic events

Beta cells

Glycemic control

Continuous glucose monitoring

Exogenous insulin

Transformative therapy

Originally Post From https://www.tipranks.com/news/the-fly/vertex-reports-positive-results-from-ongoing-phase-1-2-study-of-vx-880

Vertex Announces Promising Results for VX-880 in Ongoing Phase 1/2 Study