Reporter Noh Shin-young, Biospectator
“Pre-release of single clinical results”.. 90 days after administration of stem cell-derived islet cell treatment ‘VX-880’, blood C-peptide increased, glycated hemoglobin (HbA1c) level and average daily insulin dose decreased
The stem cell treatment ‘VX-880’ from Vertex Pharmaceuticals, a development company for rare disease treatment, showed improvement in diabetic disease by normalizing the function of islet cells in type 1 diabetes patients.
Vertex has been conducting efficacy evaluation clinical trials for cell therapy VX-800 in patients with type 1 diabetes since last March. However, as a result of the initial 90-day clinical data analysis of the first-injected patient, an ‘unprecedented’ blood sugar improvement effect was confirmed, and the single clinical result of the patient was disclosed in advance.
In the case of type 1 diabetes (T1D) patients, normal blood sugar control is impossible because islet cells that produce insulin in the pancreas are destroyed by an autoimmune reaction, so blood sugar is controlled through periodic external insulin injections. . On the other hand, Virtex’s diabetes treatment VX-880 is an allogeneic stem cell-derived insulin-producing pancreatic islet cell treatment.
On the 18th (local time), Vertex disclosed the data of the initial results of the Phase 1/2 clinical trial for ‘VX-880’, a type 1 diabetes stem cell treatment.
The safety and efficacy of ‘VX-880’, a stem cell-derived islet cell treatment for patients suffering from hypoglycemic awareness and severe hypoglycemia due to type 1 diabetes is clinically evaluated. Currently, a clinical trial is being conducted for a total of 17 patients, among which two patients who received the first dose received half of the existing VX-880 dose and then increased the dose to the same dose as the patients who participated in the clinical trial. (dose escalation) was performed (NCT04786262).
The clinical data Virtex disclosed this time is the result of comparing the islet cell function of one patient 90 days after the first administration with that before administration.
The patient who was followed up was diagnosed with type 1 diabetes 40 years ago, and his blood sugar was controlled through external insulin injection. Before treatment, the patient’s insulin dose was 34 units per day, and the level of C-peptide in the blood, which evaluates the secretory ability of insulin, was not detected on an empty stomach or before or after eating. In other words, it indicates that the patient is unable to make insulin on his own.
According to the clinical protocol, the patient received half the dose of VX-880 along with immunosuppressive therapy through a single infusion, and the administered VX-880 cell therapy succeeded in engraftment of the patient. did. Afterwards, Vertex measured the patient’s fasting C-peptide, glycated hemoglobin (HbA1c) level, and average daily insulin dose 90 days after administration of the treatment to evaluate the degree of disease improvement according to the treatment.
As a result of the clinical results, the fasting and postprandial C-peptide of the patient showed an increasing trend immediately after administration of VX-800. After 90 days of administration of VX-880, the patient’s fasting C-peptide was 280 pmol/L and the maximum postprandial C-peptide was 560 pmol/L. Conversely, the glycated hemoglobin level decreased from 8.6% to 7.2%. The daily insulin dose also decreased by 91% from 34 units before treatment to 2.9 units.
The safety evaluation of VX-880 was consistent with the symptoms of general immunosuppressive therapy. There were no serious adverse events (SAEs) associated with VX-880, and most adverse events were reported as mild or moderate. The most frequently reported adverse event was severe hypoglycemia that occurred during surgery unrelated to VX-880. One rash was reported as a confirmed SAE for 90 days after administration of the treatment, and the severity was only mild.
“These results for the first patient treated with VX-880 are unprecedented,” said Bastiano Sanna, vice president and head of cell and gene therapy at Vertex. The clinical results are more noteworthy that the therapeutic effect was achieved with only half the target dose. “We are planning clinical trials for islet cell therapy,” he said.
Although the published clinical results are limited as early (90 days) clinical data from a single patient, Vertex plans to continue conducting phase 1/2 clinical trials to support the positive clinical results of VX-880. In addition, Vertex is planning a clinical trial of a capsule-type islet cell therapy program that does not require a separate chronic immunosuppressive therapy, and plans to submit an IND for the clinical trial in 2022.
▲ Islet cell function evaluation result (Vertex) measured after VX-880 administration
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