Vertex is providing an initial peek at the first patient to receive a new type of diabetes cell therapy that could prevent the need for a cell transplant.
A single patient with type 1 diabetes achieved a “robust” restoration of islet cell function after receiving VX-880, the company said Monday. 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.
RBC Capital Markets said the single-patient data shows proof of concept for Vertex's therapy, which could be a "game-changing off-the-shelf approach." Where data on one patient isn't considered significant when testing traditional drugs, it's considered more significant for treatments such as cell and gene therapies.
The patient, who has been dependent on insulin treatment, received a single infusion of Vertex’s VX-880 at half the target dose as well as immunosuppressive therapy during the phase 1/2 clinical trial.
In the year before receiving VX-880, the patient had five life-threatening hypoglycemic episodes, which is when the blood sugar drops to a dangerous level and was not making insulin.
After treatment, the patient was able to decrease the insulin dose and showed signs that the body was producing insulin on its own. Safety was similar to typical immunosuppressant therapy and no serious side effects related to the treatment were reported. The patient did, however, experience one severe adverse event, which Vertex said was a rash unrelated to treatment.
“While still early, these results support the continued progression of our VX-880 clinical studies, as well as future studies using our encapsulated islet cells, which hold the potential to be used without the need for immunosuppression,” said Bastiano Sanna, Ph.D., executive vice president and chief of cell and genetic therapies at Vertex.
VX-880 is a stem-cell-derived islet cell replacement therapy that Vertex picked up in September 2019 in its $950 million Semma Therapeutics buyout. The therapy won a fast-track tag from the FDA in March.
The treatment is designed to restore the body’s ability to regulate glucose by giving patients new insulin-producing pancreatic islet cells based on donor stem cells. The hope is to replace the insulin injections and chronic care that are the norm for people with Type 1 diabetes.
Results from the single patient mean the phase 1/2 trial will continue, Vertex said. The company plans to enroll up to 17 people with type 1 diabetes who have impaired hypoglycemic awareness and severe hypoglycemia. The initial two patients will receive half the target dose before escalation in the remaining patients.
If the results hold true in further patients and with higher doses, RBC predicts that Vertex could have a $2.2 billion market opportunity on its hands for the initial patient population with immunosuppressant therapy.
But the real prize will be the non-immunosuppressant population, which Vertex is working to get into human studies. The company said its encapsulated islet cell program is expected to be filed with the FDA for clinical testing in 2022.
The diabetes program would help Vertex diversify beyond its successful cystic fibrosis franchise.
The current standard of care for diabetes is a maintenance approach with insulin therapy. No treatment has been approved that addresses the underlying cause of disease.