Vertex Provides an Update on the VX-880 Clinical Trial

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Update: On July 5th, the FDA Clinical Hold was lifted. The trial will now be reopened for screening, enrollment and dosing at multiple sites in the U.S.

Vertex published a press release with many updates on their Phase 1/2 Clinical Trial of VX-880. The good news? The clinical trial participants are showing positive results. The surprising news? The trial has been put on Clinical Hold by the FDA. Let’s dive in.

What Is VX-880

VX-880 is the name of the Vertex therapy undergoing clinical testing. It uses stem cell-derived beta cells to try to restore the body’s ability to produce insulin, in combination with immunosuppressive therapy to protect the cells from rejection.

These cells are administered through the portal vein in the liver. After receiving the cells, study participants are on immunosuppressive therapies akin to those taken by someone who has received an organ transplant.

Some Encouraging Data

Results from the first person to receive VX-880 (at half the target dose) have been covered extensively both here and elsewhere. Vertex has intermittently released data showing a simultaneous increase in C-peptide (an indicator that the body is making insulin) and reduction in external insulin requirements. This latest update is the biggest yet: Patient #1 is 100% insulin independent 270 days after receiving the therapy with an HbA1c of 5.2%. He is on immunosuppressive therapy, but no longer needs to administer insulin.

Patient #2 also received a half dose of VX-880 and, like Patient #1, is seeing a simultaneous increase in C-peptide and a reduction in insulin requirement, but to a smaller extent than Patient #1. After 150 days, they have a 30% reduction in insulin requirements.

A third patient has received the full dosage of VX-880. They are experiencing an increase in C-peptide and improving glycemic control after 29 days. Patient #3 will be more fully evaluated at their 90-day visit later this summer.

There have been no serious adverse events with any of the three participants, meaning none have experienced a serious and undesirable experience because of VX-880.

“JDRF is encouraged to learn that Patient #1 is fully insulin independent and that VX-880 is showing meaningful insulin production in Patient #2,” says JDRF Chief Scientific Officer Sanjoy Dutta, Ph.D. “JDRF is looking forward to seeing further data from these patients and, hopefully, more patients in the future, as soon as the FDA’s questions have been addressed by Vertex.”

What is a Clinical Hold?

Update: the Clinical Hold was lifted on 7/5 and the trial has resumed. 

A Clinical Hold is a mechanism through which the FDA can suspend an ongoing clinical trial for a multitude of reasons. In this case, Vertex has stated that the hold is due to “insufficient information to support dose escalation with the product.” FDA has up to 30 days to send Vertex a written explanation of the decision. Vertex has stated that they are committed to working through this with FDA and want to resume this clinical trial as soon as possible.

In the meantime, Vertex will not be administering VX-880 to any trial participants or enrolling new participants in the study, but they will continue to follow the three patients who have already been enrolled. We will update this blog with more information as we learn more.

“It is not uncommon for clinical trials of such novel therapies to be suspended for the company and FDA to carefully consider the data,” says JDRF Vice President of Regulatory and Health Policy, Campbell Hutton. “We hope that process can happen quickly so the research can continue.”


Simply put, VX-880 would not be possible without JDRF. Douglas Melton, Ph.D., who just departed Harvard to work at Vertex, received a JDRF grant to make insulin-producing beta cells from stem cells in 2004. Since then:

JDRF fully supports a variety of cell therapy approaches, investing in creating an unlimited supply of cells and finding ways to keep them safe without immunosuppressive therapies. Learn more about our efforts in Cell Therapies here.

JDRF will also continue to proactively work with the FDA to keep them current on the latest in cell therapy technology so that these therapies, when ready, can progress through the regulatory process as quickly and safely as possible.

What’s Next

JDRF has confidence Vertex will work with the FDA to address any concerns they have so this important clinical trial can continue as soon as possible. JDRF has no insight into the specific reason for the hold or when the clinical trial will resume in the US.

In the meantime, Vertex has reaffirmed their commitment to developing therapies for T1D and is also working on at least two other therapies as a part of their Cell Plus program. This includes one that takes the cells from VX-880 and encapsulates them in a device that keeps them safe from the immune system. They’re also working on genetically modifying cells to make them hypoimmune, meaning immune evasive. Both therapies are intended to function without the need for chronic immunosuppression, unlike VX-880. Vertex plans to file an Investigative New Drug (IND) application on the encapsulated cells in 2022.

Read more at Vertex