ADA: Vertex closer to weaning type 1 diabetics off insulin

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ADA: Vertex closer to weaning type 1 diabetics off insulin

A cell therapy developed by Vertex Pharma has allowed 10 of 12 patients with type 1 diabetes to control their blood sugar without insulin, according to new data from a phase 1/2 trial.

One-year results from the company's FORWARD-101 trial of islet cell therapy zimislecel (also known as VX-880) showed that all 12 patients with type 1 diabetes and impaired hypoglycaemia awareness – meaning they find it hard to spot the telltale signs of declining blood sugar levels – showed sustained glycaemic control after the one-shot treatment.

Participants received a full dose of the off-the-shelf islet cells, transplanted into the liver via a portal vein infusion under a standard immunosuppression drug regimen.

Vertex was also developing a version of the therapy called VX-264, in which the cells were encapsulated in a protective device designed to protect them from rejection by the immune system, but that failed to hit the mark in early clinical testing and has been dropped.

All of the patients in the trial started producing endogenous insulin after the procedure and, after 90 days, none of them had a severe hypoglycaemic event throughout the remainder of the year.

They all hit glycaemic control targets – measured by haemoglobin A1C levels reaching the target of less than 7% with a time in range of over 70% – and reduced their need for insulin injections by 92%, according to Michael Rickels of the University of Pennsylvania's Perelman School of Medicine, who presented the results at the ADA congress in Chicago.

Hypoglycaemia remains a dangerous risk for individuals with type 1 diabetes, particularly in those with impaired awareness, as episodes can lead to confusion, coma, seizures, cardiovascular events, and even death, Rickels told delegates.

"As I think about my patients and the unmet need in the type 1 diabetes 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," he added.

Vertex has expanded the FORWARD-101 trial into a phase 3 stage that has a target of enrolling and dosing 50 subjects before the end of the year, and has also started recruiting patients in Europe and Canada, as well as the US.

Meanwhile, the company is preparing a second study of zimislecel in around 10 adults with type 1 diabetes who are already receiving immunosuppressive therapy following a prior kidney transplant.

"[This] data on the first fully differentiated, stem cell-derived, off-the-shelf islet cell therapy continue[s] to be unprecedented," said Vertex's chief medical officer, Carmen Bozic. "We are excited to complete enrolment and dosing in the phase 1/2/3 programme and look forward to potential regulatory submissions next year."