ADA24: Fresh data for Vertex cell therapy reinforce potential in type 1 diabetes

2024-06-21

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并购临床研究细胞疗法引进/卖出临床结果

Vertex Pharmaceuticals' investigational stem cell-derived islet cell therapy continues to show potential in eliminating the need for insulin in type 1 diabetes patients, according to new data from a Phase I/II study.

Results from the FORWARD trial, presented on Friday at the American Diabetes Association (ADA) annual meeting, demonstrated that VX-880 not only reduced or eliminated the need for exogenous insulin in participants, but also restored physiological islet function and improved glycaemic control.

Piotr Witkowski, one of the study investigators, said "this positive data adds to the growing body of evidence for VX-880's potential to revolutionise the treatment of type 1 diabetes that would give patients an alternative solution other than exogenously administered insulin."

The three-part study enrolled adults with type 1 diabetes, impaired hypoglycaemic awareness, and at least two severe hypoglycaemic events (SHEs) in the year before screening.

The latest findings build on previous data from six patients showing islet cell engraftment and endogenous glucose-responsive insulin production, as well as better glycaemic control, after more than 90 days of follow-up. At the time, two patients were evaluable for the main efficacy endpoint, as they had at least one year of follow-up after VX-880 infusion. Both met the primary endpoint criteria by eliminating SHEs between day 90 and month 12, with HbA1c levels below 7%.

Elimination of severe hypoglycaemic events

According to the latest results, all 12 participants in the FORWARD study achieved a reduction in HbA1c to 7% or below. Of 10 who completed the day 180 visit, seven no longer required exogenous insulin, while two had approximately a 70% reduction in their daily insulin use. Moreover, 100% of patients with over a year of follow-up met the primary endpoint of eliminating SHEs with HbA1c

The FORWARD trial has been expanded to enroll approximately 37 participants in total.

The programme suffered a brief setback in 2022 when the FDA placed a clinical hold on the study, citing insufficient information to support dose escalation. Vertex was permitted to resume the trial a few months later.

The company has been actively expanding its cell therapy portfolio for type 1 diabetes. In April, it entered a licensing agreement with TreeFrog Therapeutics, potentially worth over $780 million, to access the latter’s cell manufacturing technology. In 2022, Vertex acquired ViaCyte for $320 million to speed up development of VX-880.

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