Type 1 diabetes cell therapy of Vertex Pharma shows a potential to obtain the independence of insulin
Type 1 Diabetes Diabetes Green Pharmaceutical Therapy allows patients to produce insulin, eliminating the need for additional insulin therapy in the vast majority of participants in a key clinical trial. With these preliminary results in hand, the drug manufacturer plans to submissions to regulatory therapy, Zimiscel.
The update comes from the 1/2 phase part of a phase 1/2/3 study and covers 12 patients who have been followed for at least one year. Vertex said that the 12 blood sugar levels have reached glucose in the range recommended by the American Diabetes Association (ADA). Treatment of type 1 diabetes requires monitoring of blood sugar and frequent or continuous dosage of insulin. Vertex said that 10 of the 12 patients treated with Zimiscel were free because this insulin exogenous on the date of cutting of the data. The latest results were presented Friday evening at the ADA annual conference in Chicago and published online in the New England Journal of Medicine (NEJM).
Type 1 diabetes is an autoimmune disorder that leads to the destruction of pancreatic island cells that produce insulin. Of the 3.8 million type 1 diabetic patients diagnosed in the United States and Europe, around 60,000 potentially fatal severe hypoglycemic events (SCH) said Green in a presentation of investors. It is the top of the population of patients targeting Zimiscel.
Zimiscel (formerly known as VX-880) is made up of cells of stem cells derived which are administered in a single infusion in the portal hepatic vein. In order to prevent the body from rejecting cell therapy, Zimiscel requires chronic use of immunosuppressive drugs. These drugs introduce risks of complications, such as higher sensitivity to infections.
Vertex had hoped to avoid chronic immunosuppression with a different program, VX-264. This therapeutic candidate encapsulated Zimiscel in an implanted medical device which protected cell therapy from the immune system. In March, Vertex interrupted the VX-264 after failing the effectiveness objectives in a clinical trial of phase 1/2. But the company continued to develop a “naked” zimiscel – cell therapy without protective medical device.
Although the avoidance of immunosuppression would have given to the summit a key advantage in the treatment of type 1 diabetes, the naked zimiscel always has a potential for differentiation. The only cellular therapy approved by the FDA for this disease is Lantidra, which the Celltrans biotechnology company made from islets from deceased donors. Lantidra limits include a guarantee of the quality and supply of cells from this way. Zimiscecel derived from stem cells provides patients with the prospect of an alternative of treatment “out of the shelf”.
The latest results for Zimiscel show that the cells of stem cells were accepted by the body of patients and led to the production of peptide C. This peptide is released alongside insulin and the measurement of its levels in the blood offers a means of evaluating the production of insulin. Vertex said therapy continues to be well tolerated, most of the negative effects classified as light or moderate. The NEJM article indicates that the most common serious adverse event was neutropenia, which is low levels of the white blood cell type called neutrophils. This event was reported in three patients. Vertex said that no serious adverse effects linked to cell therapy has been reported and that the safety profile is consistent with the immunosuppressant diet used in the study. Two deaths by previously reported patients were not linked to treatment with Zimiscel, said the company.
In a note sent to investors on Monday, William Blair Myles Minter’s analyst said Zimiscel’s ability to maintain normal blood sugar while reducing or eliminating the need for exogenous insulin represents a significant lifestyle difference for patients. He also noted that experts say that 35% to 40% of type 1 diabetic patients cannot reach the ADA glucose control guidelines with currently available treatments, which makes the target market Addressable for Zimiscel significantly higher than the 60,000 patients targeted by the summit. But cell therapy always faces obstacles.
“We consider the requirement of immunosuppressants as the largest headwind here for commercial absorption, which can prevent certain populations of patients, and serious adverse events involving neutropenia and acute kidney injury have been noted in the NEJM publication,” Minter.
In the presentation of investors, Vertex said that he is developing immunosuppression alternatives that are currently at the research stage. Meanwhile, the Zimiscel phase 1/2/3 test should register a total of 50 patients. Vertex plans to complete the inscriptions and the dosage this summer.
Photo: David L. Ryan / The Boston Globe, via Getty Images
