Vertex Pharmaceuticals and Lonza have announced a strategic collaboration to support the manufacture of Vertex’s portfolio of investigational stem cell-derived, fully differentiated insulin-producing islet cell therapies for people with type 1 diabetes (T1D), currently focusing on the VX-880 and VX-264 programs that are in clinical trials.
Vertex’s first clinical program, VX-880, has already demonstrated clinical proof-of-concept. In new data released last week, all six patients treated with VX-880 engrafted islet cells, produced endogenous insulin (C-peptide), and had improved glycemic control, while reducing or eliminating insulin use. Patients with greater than 90 days of follow-up had elimination of severe hypoglycemic events in the evaluation period.
Vertex’s second approach, VX-264, utilizes the same fully differentiated insulin-producing islet cells as in VX-880 and is being studied in a Phase 1/2 clinical trial. In VX-264, the cells are encapsulated in a proprietary device designed to shield the cells from the body’s immune system, thereby eliminating the need for immunosuppression. In a third approach, Vertex’s hypoimmune program, the same cells are edited to cloak them from the immune system; this program continues to progress in preclinical development.
Under the terms of the collaboration, Vertex and Lonza will partner in the process development and scale-up for the manufacturing of the product portfolio and co-invest to build a dedicated new facility in Portsmouth, New Hampshire. Operated by Lonza, the facility will span more than 130,000 square feet and is anticipated to create up to 300 new jobs at peak capacity. Construction is scheduled to begin later this year.
The dedicated facility will complement Lonza’s global cell and gene technologies manufacturing network, which supports customers in developing, de-risking, commercializing and scaling their emerging therapies. Lonza’s scientific, regulatory and manufacturing expertise, its focus on operational excellence, and first-hand experience supporting the commercialization of marketed cell therapy products is expected to accelerate the development and commercialization of Vertex’s potentially transformative therapies.
“Vertex’s allogeneic, fully differentiated, insulin-producing islet cells and the results from the Phase 1/2 VX-880 program represent a scientific breakthrough that offers the potential to transform the treatment of T1D,” said Reshma Kewalramani, M.D., Chief Executive Officer and president of Vertex. “Establishing this strategic partnership with Lonza, a world-class manufacturing organization, is a critical milestone in this journey and underscores our long-term commitment to patients with T1D.”
“Lonza has a strong history and established reputation for applying cutting-edge development and manufacturing expertise to enable the progression of exceptional scientific innovations like those developed by Vertex,” said Pierre-Alain Ruffieux, Chief Executive Officer of Lonza. “Vertex has made impressive progress with its T1D cell therapy program, and we are proud to be the company’s partner of choice in supporting its ambition to transform the treatment landscape for this disease.”