Vertex Gets 'Go' Approval from FDA to Start a T1D Practical Cure Human Trial

Vertex Pharmaceuticals Inc. announced last Thursday that the FDA gave approval to the company to start human trials on a product that has the potential to be a Practical Cure. Referred to as product candidate VX-264, it combines both a source of cell supply (from stem cells) along with cell protection (from encapsulation), which if successful eliminates the need for immunosuppression. Vertex says it plans to initiate the phase 1/2 trial "in the first half of 2023."

Vertex has made a big splash in T1D research over the past several years, after acquiring Semma Therapeutics in 2019 for $950 Million in cash. The acquisition gave Vertex two scientific opportunities: 1) the ability to produce large quantities of functional human pancreatic beta cells from stem cells; and 2) a novel encapsulation device to protect the cells from the T1D autoimmune attack.

The first product that Vertex began testing after the acquisition was called VX-880; a transplantation of the stem cell-derived beta cells that did not include cell protection, making general immunosuppression a daily requirement. This new trial, VX-264, now includes a cell protection encapsulation device that was developed by Vertex specifically for their cells.

A few additional highlights of Vertex's progress to date:

September 2019: 

  • Vertex acquires Semma Therapeutics, gaining the ability to make pancreatic beta cells from embryonic stem cells. LINK. 

October 2021: 

  • Vertex announces early results from the first patient tested with VX-880 (which requires immunosuppression).  At 90 days into the trial, the patient reduced their daily external insulin requirement from 34 units per day to 2.9 units per day, and at the same time, the person’s HbA1C level dropped from 8.6% to 7.2%. LINK.

April 2022:

  • Esteemed Harvard stem cell researcher and founder of Semma Therapeutics, Doug Melton, joins Vertex. LINK.

May 2022:

  • Vertex updates results on the first patient dosed with VX-880. At 270 days in, the patient is off insulin and has a 5.4% HbA1C. A second patient did not have as strong results. 150 days after receiving the treatment, the patient experienced a 30% reduction in external insulin and posted a 7.1% HbA1C. LINK.

July 2022:

  • Vertex announces that it has agreed to acquire ViaCyte, its number one stem-cell technology competitor, in an all-cash deal for $320 million, pending regulatory approval. LINK.