Vertex Pharmaceuticals has seen promising results from its islet cell therapy, zimislecel, in patients with Type 1 diabetes and severe hypoglycemic events. The treatment, derived from human stem cells, has shown to be effective in producing glucose-responsive endogenous C-peptide and improving glycated hemoglobin (HbA1c) levels.
In a recent phase 1/2/3 study, Vertex reported “unprecedented” data from 12 patients who received the full infusion of zimislecel. The treatment was engrafted successfully to all 12 patients, producing glucose-responsive endogenous C-peptide up to the one-year follow-up. Notably, none of the patients experienced severe hypoglycemic events from day 90 onward.
Ten of the 12 patients no longer required insulin replacement therapy by the 12-month checkup, and glycated hemoglobin (HbA1c) levels were within the recommended level of less than 7%. The treatment was “generally well tolerated,” with no serious adverse events linked to zimislecel itself.
The results are significant, as Vertex’s Chief Medical Officer, Carmen Bozic, said that the data “reinforce the transformative potential” of zimislecel. The company plans to complete enrollment and dosing in the phase 1/2/3 program and submit for regulatory approval next year.
This positive news comes after Vertex discontinued its other islet cell treatment, VX-264, citing disappointing results from a 90-day analysis. Zimislecel, however, has shown to be effective when administered with immunosuppressants to help the cells graft. The phase 3 portion of the zimislecel study is ongoing in multiple countries and approval applications are penciled for next year.
Source: https://www.fiercebiotech.com/biotech/vertexs-remaining-islet-cell-therapy-reduces-dangerously-low-blood-sugar-levels-phase-12