New Data Strengthens Vertex Cell Therapy's Type 1 Diabetes Potential

Vertex Pharmaceuticals' investigational stem cell-derived islet cell therapy, VX-880, is showing significant promise in reducing or eliminating the need for insulin among patients with type 1 diabetes. Recent data from a Phase I/II study, named the FORWARD trial, was presented at the American Diabetes Association (ADA) annual meeting and highlighted the potential of VX-880 in transforming type 1 diabetes treatment. This therapy aims to restore natural islet function and improve glycemic control, thus providing an alternative to exogenous insulin.

The study involved adults diagnosed with type 1 diabetes who also had impaired hypoglycemic awareness and experienced at least two severe hypoglycemic events (SHEs) in the year prior to screening. The trial is structured in three parts and aims to assess the efficacy and safety of VX-880.

Initial data from the FORWARD trial, which included six patients, showed that the therapy enabled islet cell engraftment and endogenous insulin production responsive to glucose. This led to better glycemic control after more than 90 days of follow-up. At that time, two patients had sufficient follow-up duration to be evaluated for the main efficacy endpoint. Both patients succeeded in eliminating SHEs between day 90 and month 12 and had their HbA1c levels drop below 7%.

The latest results from the study further support these findings. All 12 participants in the FORWARD study achieved a reduction in HbA1c levels to 7% or below. Among the 10 participants who completed their day 180 visit, seven no longer required exogenous insulin, while two showed approximately a 70% reduction in daily insulin use. Furthermore, all patients with over a year of follow-up met the primary endpoint of eliminating SHEs and maintaining HbA1c levels below 7% at month 12. These patients also achieved insulin independence, a secondary target of the study.

This positive data reinforces VX-880's potential to revolutionize type 1 diabetes treatment, providing patients with a viable alternative to constant insulin injections. Piotr Witkowski, one of the investigators, emphasized the therapy's potential to offer a new solution for patients with type 1 diabetes.

The FORWARD trial has now been expanded to include approximately 37 participants. The program did face a temporary halt in 2022 when the FDA put a clinical hold on the study, citing a lack of sufficient information to support dose escalation. However, Vertex was allowed to resume the trial a few months later.

Vertex Pharmaceuticals has been proactive in enlarging its cell therapy portfolio for type 1 diabetes. In April, the company entered into a licensing agreement with TreeFrog Therapeutics to access their cell manufacturing technology, a deal potentially worth over $780 million. Additionally, in 2022, Vertex acquired ViaCyte for $320 million to accelerate the development of VX-880.

Overall, these advancements indicate substantial progress in the realm of type 1 diabetes treatment, potentially marking a significant shift in how the disease is managed. The ongoing success of VX-880 in clinical trials brings hope to many patients looking for a more sustainable and less invasive treatment option.