FDA Greenlights Phase 1/2 Trial of Vertex’s Type 1 Diabetes Cell Therapy
The study of VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy, is already ongoing in Canada. It is expected to kick off in the United States in the first half of 2023.
Bastiano Sanna, PhD
The FDA has cleared an investigational new drug (IND) application from Vertex Pharmaceuticals to proceed with a clinical trial of its stem cell-derived, fully differentiated pancreatic islet cell therapy for type 1 diabetes (T1D). The treatment is currently known as VX-264.1
The planned phase 1/2 clinical trial is expected to be initiated in the first half of 2023 to study the safety, tolerability, and efficacy of VX-264 in patients with T1D. The trial is already ongoing in Canada, where Vertex previously received the go-ahead. Vertex additionally noted that the program for VX-264 does not require immunosuppression use, which the company expressed might broaden the population of patients that might benefit from it.
The trial of VX-264 is a single-arm, open-label study in patients who have T1D. It is planned to be sequential and multipart and seeks to enroll approximately 17 patients globally.
“VX-264 uses the same stem cell-derived pancreatic islet cells as our VX-880 program where we’ve already demonstrated proof-of-concept, with the addition of a proprietary immunoprotective device that allows us to eliminate the need for immunosuppression,” Bastiano Sanna, PhD, the executive vice president and chief of Cell and Genetic Therapies at Vertex, said in a statement.1 “We are excited to see our second program in T1D advancing into the clinic and look forward to bringing transformative, if not curative, therapies to T1D patients who are waiting.”
READ MORE: Estrella Biopharma’s CD19-Targeted T-cell Therapy for B-Cell Lymphomas Gets IND Clearance
VX-880 is another stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for people with T1D, specifically designed to treat those with impaired hypoglycemic awareness and severe hypoglycemia. At the time of a May 2022 update from Vertex,2 the study had been placed on a clinical hold by the FDA based on insufficient data supporting dose escalation.
At the time of the announcement on VX-880, 2 patients in part A of a phase 1/2 trial (NCT04786262) had received half the target dose, the first of whom achieved insulin independence at Day 270 (HbA1c, 5.2%), and the second whom had shown robust increases in fasting and stimulated C-peptide, and reductions in exogenous insulin requirements through Day 150.2 Together, Vertex noted that the data established proof-of-concept for VX-880.
The study’s Independent Data Monitoring Committee reviewed the totality of the safety and efficacy data from the first 2 patients dosed in Part A of the study and recommended advancement to Part B, where patients receive the full target dose. The first patient to do so achieved the Day 29 follow-up milestone.2
This news also follows another IND clearance for Vertex, which it received in December 2022 for its messenger RNA therapy VX-522, which is designed to treat the underlying cause of cystic fibrosis (CF) lung disease for those who do not benefit from CF transmembrane conductance regulator modulator.3
Vertex is not the only company joining the fray in the development of therapies for T1D. In February 2023, an institutional review board (IRB) gave approval to Creative Medical Technology Holdings to proceed with its phase 1/2 CREATE-1 clinical trial (NCT05626712) for the evaluation of CELZ-201 (AlloStem), an investigational cell therapy based on perinatal tissue-derived cells (PRDC), in the treatment of newly diagnosed T1D.4 Creative Medical Technology Holdings had previously received clearance of its IND application for CELZ-201 in November 2022.5 At the time, the company noted that the trial was expected to begin recruiting patients in the first quarter of 2023.
For more on T1D content, check out CGTLive’s sister site, HCPLive. Recently, they brought together Diana Isaacs, PharmD, BCPS, BCACP, BC-ADM, CDCES, and Natalie Bellini, DNP, FNP-BC, to interview Emily Sims, MD, on the latest clinical evidence on teplizumab and other emerging treatments that can delay the onset of type 1 diabetes.
Click here to listen to that MedCast conversation.
