Therapy Areas: Hereditary Disorders
Vertex Receives FDA Clearance of Investigational New Drug Application for VX-264, a Novel Encapsulated Cell Therapy for the Treatment of Type 1 Diabetes
16 March 2023 - - The US Food and Drug Administration has cleared the Investigational New Drug Application for VX-264, a stem cell-derived, fully differentiated pancreatic islet cell therapy encapsulated into a Vertex-developed, immunoprotective device with the potential to treat type 1 diabetes, US-based Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) said.

The VX-264 program does not require the use of immunosuppression, which may broaden the population of people with T1D that this investigational therapy could reach.

Vertex plans to initiate a Phase 1/2 clinical trial in the first half of 2023 to study the safety, tolerability and efficacy of VX-264 in patients with T1D.

The company previously received approval from Health Canada on the Clinical Trial Application for VX-264, and the Phase 1/2 trial is ongoing in Canada.

Vertex is pursuing multiple investigational approaches using stem cell-derived islets with the aim of replacing the insulin-producing islet cells that are destroyed in people with T1D.

Vertex's first clinical investigational program in T1D, VX-880, is a stem cell-derived, fully differentiated, insulin-producing islet cell replacement therapy used in combination with immunosuppression.

Vertex has demonstrated clinical proof-of-concept in the VX-880 program, and the Phase 1/2 clinical study is ongoing in the US and Canada.

VX-264 is an investigational cell therapy in which allogeneic human stem cell-derived islets are encapsulated in a channel array device designed to shield the cells from the body's immune system.

VX-264 is designed to be surgically implanted and is currently being evaluated for patients with T1D.

The clinical trial is a Phase 1/2, single-arm, open-label study in patients who have T1D. This will be a sequential, multi-part clinical trial to evaluate the safety, tolerability and efficacy of VX-264. Approximately 17 patients will be enrolled in the global clinical trial.

T1D results from the autoimmune destruction of insulin-producing islet cells in the pancreas, leading to loss of insulin production and impairment of blood glucose control.

The absence of insulin leads to abnormalities in how the body processes nutrients, leading to high blood glucose levels.

High blood glucose can lead to diabetic ketoacidosis and, over time, to complications such as kidney disease/failure, eye disease (including vision loss), heart disease, stroke, nerve damage and even death.

Due to the limitations and complexities of insulin delivery systems, it can be difficult to achieve and maintain balance in glucose control in people with T1D.

Current standards of care do not address the underlying causes of the disease, and there are limited treatment options beyond insulin for the management of T1D; there is currently no cure for diabetes.

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

The company has multiple approved medicines that treat the underlying cause of cystic fibrosis -- a rare, life-threatening genetic disease -- and has several ongoing clinical and research programs in CF.

Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes and alpha-1 antitrypsin deficiency.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America.

Vertex is consistently recognized as one of the industry's top places to work, including 13 consecutive years on Science magazine's Top Employers list and one of Fortune's Best Workplaces in biotechnology and Pharmaceuticals and Best Workplaces for Women.


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