Vertex doses first patient with next type 1 diabetes cell therapy program, while padding its coffers with strong Q2


Vertex has dosed the first type 1 diabetes patient with its next cell therapy program, the Boston biotech said Tuesday as part of its quarterly earnings update.

Vertex’s first cell therapy program, VX-880, has impressed with early clinical results, showing a one-time infusion of these insulin-producing cells brought significant effects in the first handful of treated patients. Those results, presented at June’s American Diabetes Association annual conference, included two treated patients with over a year of follow-up who have remained free from using insulin with no severe hypoglycemic events.

Despite the impressive but early efficacy signals, VX-880 requires a lifetime of immunosuppressants to stop the immune system from destroying these islet cells. Irl Hirsch, a professor at the University of Washington’s diabetes institute, told Endpoints News in June that immunosuppression is a big concern because of the elevated risk of infections and side effects.

This newest program, called VX-264, uses a device to protect the lab-grown cells from the immune system rather than drugs.

In March, Vertex announced it had won the FDA’s OK to enter the clinic with VX-264. Both VX-880 and VX-264 come from Vertex’s 2019 acquisition of Semma Therapeutics. Both therapies use the same stem cell-derived islet cells.

The latest update on Vertex’s diabetes research comes as the $89 billion biotech reported another quarter of strong revenue growth, slightly raising its 2023 revenue guidance to $9.7 billion to $9.8 billion. Vertex’s lucrative cystic fibrosis franchise continues to impress, fattening Vertex’s balance sheet. The company ended June with $12.6 billion in cash and equivalents, up from $11.5 billion at the end of March.

If VX-264 succeeds, it could expand the potential patient population beyond Vertex’s estimate of 60,000 type 1 diabetes patients who would meet the criteria for VX-880, which is about 2% of the type 1 population. Vertex did not provide a timeline for when the study may complete, or when it might provide initial clinical results with VX-264.

The biotech is also researching another earlier therapeutic option that uses CRISPR/Cas9 to hide these cells from the immune system by genetically editing them.

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