Vertex Pharmaceuticals has shared data from the Phase I/II segment of the Phase I/II/III FORWARD-101 trial, evaluating the stem cell-derived, fully differentiated islet cell therapy, zimislecel (VX-880), for individuals with type 1 diabetes (T1D).
The study is intended for those with severe hypoglycaemic events (SHEs) and impaired hypoglycaemic awareness.
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The data was presented at the American Diabetes Association (ADA) annual conference in Chicago, US.
The data come from 12 subjects who were given a full dose of the single infusion therapy and were then monitored for at least one year, as of October 2024.
These subjects showed engraftment with glucose-responsive endogenous C-peptide production and met the ADA targets of HbA1c and time in range.
They also experienced freedom from SHEs and a mean decrease in daily use of insulin.
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By GlobalDataAt the 12-month mark, 83% of the subjects no longer needed exogenous insulin.
The Phase I/II stage also achieved the primary goal of the elimination of SHEs, with maintained HbA1c levels below 7%.
Zimislecel’s safety profile was found to be favourable, without any serious adverse events linked to the treatment.
Vertex noted that despite two patient deaths reported previously, these were deemed unrelated to the therapy’s treatment.
According to the company, the safety profile is in line with the immunosuppressive regimen in the trial, the infusion procedure, and complications associated with long-standing diabetes.
The trial is now expanding, with additional sites actively enrolling participants in Canada, Europe, and the US.
Vertex Pharmaceuticals global medicines development and medical affairs executive vice-president and chief medical officer Carmen Bozic said: “These data on the first fully differentiated, stem cell-derived, off-the-shelf islet cell therapy continue to be unprecedented.
“The magnitude, consistency, and durability of the results from all 12 patients, with more than one year of follow-up, reinforce the transformative potential of zimislecel for people living with T1D complicated by severe hypoglycaemia.”
The therapy has received fast track and regenerative medicine advanced therapy (RMAT) designations from the US Food and Drug Administration (FDA).
It has also been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA).
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