Vertex’s type 1 diabetes cell therapy turned heads when one patient’s islet cell function was restored. Now, the pharma is readying version 2.0 for the clinic after the FDA cleared a similar treatment that uses a device to shield the cells from the body’s immune system.
VX-264 is expected to arrive in the clinic in the U.S. in the first half of this year, after the FDA cleared Vertex’s investigational new drug application, according to a Thursday press release. The off-the-shelf therapy uses the same cell-derived pancreatic islet cells that VX-880 does but is delivered via a channel array device meant to avoid the need for immunosuppressive drugs.
Vertex made a splash in October 2021 with the announcement that a single patient had seen restored islet cell function after treatment with VX-880. The company said the results showed proof of concept for the approach, which uses insulin-producing pancreatic islet cells from donor stem cells. Restoration of these cells could allow patients to regulate insulin or even produce it on their own, rather than use daily insulin treatment or seek an islet cell transplant. Vertex has positioned the two therapies as potentially “curative” for the common and stubborn condition.
VX-880 must be administered along with immunosuppressive drugs. VX-264, meanwhile, uses the device to avoid this extra treatment. That could broaden the population of patients who can take the cell therapy, Vertex said.
The earlier treatment VX-880 has had a bit of a bumpy ride in the clinic since the fanfare surrounding the initial patient’s data. The FDA placed a two-month clinical hold on the med in mid-2022. Vertex said the FDA had issues with the justification for escalating the dose, which were resolved by July 2022.
In February, Vertex reported that enrollment had wrapped up for the B portion of a phase 1/2 trial for VX-880, which is testing the full target dose. Updated data is expected to be presented at a scientific conference this year. Once part B is done, Vertex plans to move on to part C, which uses concurrent dosing. Vertex picked up VX-880 in the $950 million buyout of Semma Therapeutics in September 2019.
The company will now initiative a phase 1/2 for VX-264 to examine safety, tolerability and efficacy. The therapy was previously approved by regulators in Canada where a trial is ongoing.
Not content with these two novel diabetes treatments, Vertex acquired ViaCyte for $320 million in July 2022, bringing another stem cell-based treatment into the fold.