FDA Grants VX-880 Fast Track Designation for Type 1 Diabetes

The FDA has granted the allogeneic stem cell-derived islet cell therapy VX-880 a fast track designation as a potential treatment for patients with type 1 diabetes (T1D), according to a statement from Vertex, the company developing the therapy.

In addition to the designation, Vertex simultaneously announced that it was initiating a phase 1/2 clinical study of VX-880 in patients with T1D, following the approval of an investigational new drug application for the therapy in late January. The study plans to enroll 17 patients with T1D and impaired awareness of hypoglycemia (IAH) and severe hypoglycemia (NCT04786262).

“It’s a remarkable time for T1D research efforts worldwide, as this investigational treatment enters the clinic,” steering committee chair for the VX-880 clinical trial Camillo Ricordi, MD, professor of surgery, director of the Diabetes Research Institute and the Cell Transplant Center at the University of Miami Miller School of Medicine, said in a statement. “The field’s experience with the limited cadaveric islet transplants available, where some patients have experienced prolonged insulin independence for years, provides important proof-of-concept for the potential of cell therapy to be transformative for patients living with T1D.”

VX-880 consists of fully differentiated, pancreatic islet cells derived from an allogenic stem cell line. Vertex acquired VX-880 in a 2019 acquisition of Semma Therapeutics. At the time of the acquisition, the stem cell therapy, formally known as STx-02, had demonstrated promising preclinical results in both non-human primates and pigs. Findings from the proof-of-concept trials were presented at the International Society for Stem Cell Research (ISSCR) in June 2019.

In the non-human primate findings, the stem cell-derived islets successfully engrafted and functional cells persisted for 6 weeks. Moreover, the need for insulin was reduced by more than 60% in the study findings. Immunosuppression was used in this study to ensure the cells persisted and to avoid an immune reaction. In the pig study, an immunoprotective device coating was employed to avoid the use of immunosuppression. These device-encapsulated stem cell-derived islet cells persisted and rapidly secreted insulin in response to stimulation.

For the phase 1/2 study, the cell therapy will be infused directly into the hepatic portal vein and will not be encapsulated. To avoid immune rejection, all patients will be administered concomitant immunosuppressive therapy throughout the trial. The open-label study is designed to assess the safety and efficacy of VX-880 at various doses. The co-primary outcome measures are safety and tolerability as well as the proportion of patients free of severe hypoglycemic events. Secondary end points are focused on the percent of patients who achieve insulin independence and changes in stimulated C peptide.

"Ours is the only approach that produces fully differentiated and fully functional insulin-secreting pancreatic islets," Bastiano Sanna, PhD, executive vice president and chief of Cell and Genetic Therapies at Vertex, said in a statement. "We are very pleased to have received FDA’s Fast Track Designation, which facilitates the development and expedites the review of drugs that treat serious conditions and fill an unmet medical need. We continue to work with urgency to bring this innovative therapy to patients.”