Vertex Pharmaceuticals has shared new Phase I/II data for zimislecel (VX-880), an investigational stem cell-derived, fully differentiated islet cell therapy. 

The FORWARD-101 clinical trial is investigating the treatment in people with type 1 diabetes (T1D) with impaired hypoglycaemic awareness and severe hypoglycaemic events (SHEs). 

All 12 participants: 

• Demonstrated engraftment with glucose-responsive endogenous C-peptide production, which was durable through one year of follow-up. 
• Achieved the ADA targets of HbA1c <7% and time in range of >70%. 
• Were free of SHEs from day 90 onwards. 
• Had a reduction in exogenous insulin use (mean reduction in daily insulin dose: 92%). 
• 10/12 (83%) no longer required exogenous insulin at Month 12. 
• Achieved the Phase I/II primary endpoint of elimination of SHEs with HbA1c <7%. 

Most adverse events (AEs) were mild or moderate, and there were no serious AEs related to zimislecel treatment. 

“It’s remarkable to see 12 out of 12 patients with baseline HbA1c above 7% and multiple severe hypoglycaemic events reach consensus targets for glycaemic control by both HbA1c and time in range as well as elimination of severe hypoglycaemic events,” said Michael Rickels, Medical Director, Pancreatic Islet Cell Transplant Program, Willard and Rhoda Ware Professor in Diabetes and Metabolic Diseases. “As I think about my patients and the unmet need in the T1D community, the results we’ve seen so far for restoring endogenous insulin secretion with a stem cell-derived islet therapy bring me hope and confidence for a transformative treatment option for individuals with type 1 diabetes in the not-so-distant future.” 

Zimislecel has been granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the US Food and Drug Administration, and Priority Medicines (PRIME) designation from the European Medicines Agency (EMA).

Diana Spencer, Senior Digital Content Editor, DDW