Vertex Stem Cell Study Suffers Setback

Vertex has announced that its VX-880 Phase 1/2 study has been placed on clinical hold in the U.S. by the FDA. VX-880 is an investigational stem cell-derived, pancreatic islet cell replacement therapy for people with type 1 diabetes (T1D). According to Vertex, the FDA took this action due to its determination that there is “insufficient information to support dose escalation” with the product.

 T1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, leading to loss of insulin production and impairment of blood glucose control. The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood glucose levels. High blood glucose can lead to diabetic ketoacidosis and over time, to complications such as kidney disease/failure, eye disease (including vision loss), heart disease, stroke, nerve damage and even death.

VX-880 has the potential to restore the body’s ability to regulate glucose levels by restoring pancreatic islet cell function, including glucose-responsive insulin production. VX-880 is delivered by an infusion into the hepatic portal vein and requires chronic immunosuppressive therapy to protect the islet cells from immune rejection.

“We are surprised by the clinical hold placed on the study. The results from the first two patients treated with half the target dose establish proof-of-concept by demonstrating that VX-880 can restore glucose-regulated insulin production and improve glycemic control. Indeed, achievement of insulin independence by the first patient is a landmark milestone. Further, the totality of the safety and efficacy data for all three patients dosed to date gives us high confidence in our benefit-risk assessment of VX-880 and its potentially transformative profile,” said Carmen Bozic, Executive VP, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “We are committed to working with the FDA to understand and address their questions, so that the trial can resume at U.S. sites as soon as possible.”

Vertex is also pursuing additional programs in T1D, including one in which these same stem cell-derived islets are encapsulated in an immunoprotective device to be surgically implanted without the use of concomitant immunosuppression, and another program where cells are modified to produce hypoimmune stem cell-derived islets. IND-enabling studies for the encapsulated cells program are underway, and the company remains on track to submit an IND in 2022. (Source: Vertex Website, 02 May, 2022)