Vertex’s Diabetes Cell Therapy on Track to Meet Endpoint of Eliminating Severe Hypoglycemic Events


All 3 participants who have follow-up of at least 1 year have met the endpoint so far.

Data continue to support VX-880 (Vertex Pharmaceuticals)’s potential to eliminate or reduce the need for insulin use in patients with type 1 diabetes (T1D).1

Updated data from the phase 1/2 FORWARD trial (NCT04786262) were presented at the American Diabetes Association 84th Scientific Sessions, held June 21-24 in Orlando, Florida, and virtually, by investigator Piotr Witkowski, MD, PhD, Professor of Surgery, Director, Pancreatic and Islet Transplant Program, University of Chicago, Illinois.

“This positive data adds to the growing body of evidence for VX-880’s potential to revolutionize the treatment of type 1 diabetes that would give patients an alternative solution other than exogenously administered insulin,” Witkowski said in a statement.1 “These findings will also support further evaluation of VX-880, and we hope to see this treatment become a pivotal development in type 1 diabetes care.”

The open-label 3-part study enrolled adults with T1D with undetectable fasting C-peptide, a history of recurrent severe hypoglycemic events (SHEs) in the year prior to screening, and that required an average of 39.3 (range, 19.8-52.0) units of insulin per day. Participants had an average age of approximately 44 years and a mean HbA1c of 7.8%.1

READ MORE: Kadimastem and iTolerance Work to Bring Immunosuppression-Free Diabetes Cell Therapy to Trials

VX-880 is an investigational, allogeneic, stem cell-derived, fully differentiated insulin-producing islet cell therapy. Following a single infusion of VX-880 at the full dose, all 12 patients demonstrated islet cell engraftment and glucose-responsive insulin production by Day 90. Most adverse events (AEs) were mild or moderate and corresponded to the immunosuppressive regimen used in the study, the infusion procedure, and underlying disease. There were no serious AEs related to VX-880 treatment. Two previously disclosed patient deaths did occur but were unrelated to VX-880 treatment.1

“These remarkable data add to the growing body of evidence for VX-880 as a potentially curative therapy for T1D,” Carmen Bozic, MD, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex, said in a company statement.2 “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.”

All 12 participants that received the full dose had improved glycemic control and achieved ADA-recommended targets for both HbA1c below 7.0% and time-in-range above 70% on continuous glucose monitoring. Most(n = 11) had reduced or no exogenous insulin use at their last visit and all had elimination of SHEs during the evaluation period. All 3 participants who have had at least 1 year of follow-up have met the primary endpoint of no SHEs with HbA1c less than 7.0% and the secondary endpoint of insulin independence. Of 10 participants who completed the Day 180 visit, 7 are no longer using exogenous insulin and 2 had around a 70% reduction in their daily insulin use. Based off these data, Vertex has expanded the FORWARD trial to enroll a total of approximately 37 participants.1

“The data we’ve seen to-date in this study are extremely exciting. Stem cell-derived islets regulate blood glucose control as well as natural human islets,” Witkowski added to the company statement.2 “The marked improvements seen across several key glycemic measures, the elimination of severe hypoglycemic episodes, and the reduction or total elimination of reliance on exogenous insulin have the potential to fundamentally change the treatment landscape for T1D and alleviate the significant burden this disease carries for patients.”

Expanded FORWARD Trial Demonstrates Continued Potential for Stem Cell-Derived Islet Cell Therapy to Eliminate Need for Insulin for People with T1D. News release. ADA. June 21, 2024.
2. Vertex Announces Positive Results From Ongoing Phase 1/2 Study of VX-880 for the Treatment of Type 1 Diabetes Presented at the American Diabetes Association 84th Scientific Sessions. News release. Vertex Pharmaceuticals. June 21, 2024.
Recent Videos
Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells
Subhash Tripathi, PhD, on Developing Safe, Specific Engineered Treg Cell Therapy
Jacques Galipeau, MD, on Highlights from ISCT 2024’s Presidential Plenary
Zheng-Yi Chen, DPhil, on International Collaboration on Clinical Trials
Shankar Ramaswamy, MD, the cofounder, chairman, and CEO of Kriya Therapeutics
Massimo Trucco, MD
Massimo Trucco, MD
© 2024 MJH Life Sciences

All rights reserved.