Vertex Touts New Construction, Foundation Initiatives and Clinical Trial News

Vertex_David L. Ryan/The Boston Globe via Getty

Courtesy of David L. Ryan/The Boston Globe via Getty Images

Vertex Pharmaceuticals dedicated a new site in Boston’s Seaport, the Jeffrey Leiden Center for Cell and Genetic Therapies. But in a classic fashion, the company seemed to say, “But wait! There’s more!” and announced it plans to build another facility in the area.

The new Jeffrey Leiden Center is named after executive chairman and former CEO Dr. Jeffrey Leiden, M.D., Ph.D. Dubbed Leiden I, it is a state-of-the-art cell and genetic therapies research and clinical manufacturing site. It can support more than 400 staffers. About 70% of the facility is dedicated to lab and research space.

Now, the company announced it plans to build another 344,000 square foot facility in Seaport to support the expansion of its cell and genetic therapies programs. Vertex expects the new site to be completed in 2025, giving the company 1.9 million square feet of real estate across five sites.

“Naming this campus after Jeff is a fitting tribute to his visionary leadership and enormous contributions to making Vertex the company it is today,” said Dr. Reshma Kewalramani, M.D., Vertex’s CEO and president. “Building on our achievements in cystic fibrosis, Vertex is at a new inflection point, and our continued expansion will enable our continued growth as we work to bring many more transformative — if not curative — medicines to patients who are waiting.”

The company also announced a $50 million commitment with the Vertex Foundation for health equity, focused on clinical trial diversity, access to quality care and education of the local health care workforce. The commitment is over five years and is built on three grants.

The first initiative for clinical trial diversity is with JDRF, the leading global type 1 diabetes (T1D) research and advocacy organization. Vertex is supporting its efforts to improve clinical trial diversity.

“Vertex’s support will allow us to expand our efforts to ensure understanding of the disease and knowledge of the most up-to-date treatment options to alleviate daily burdens,” said Aaron Kowalski, Ph.D., JDRF International CEO.”

The second, for access to quality care, is in support of the Mass General Comprehensive Sickle Cell Disease Treatment Center. The funding will advance the Center’s operations and efforts to provide holistic support to improve patient care via expanding social workers, patient coordinators and nurse navigators.

“Massachusetts General Hospital has long been dedicated to identifying and addressing racial and ethnic disparities and achieving equity in our quality of care,” said Dr. Joseph Betancourt, M.D., M.P.H., senior vice president of Equity and Community Health at Mass Gen. “The Comprehensive Sickle Cell Center represents a long overdue step forward in the care and treatment of a serious disorder impacting primarily African Americans and communities of color.”

And third, for the education of the health care workforce, the Vertex Foundation will partner with Boston-based Year Up, a workforce development nonprofit. The funding will support a curriculum to prepare young adults for possible careers in community health, paid internships, and a source of skilled and trained professionals “who reflect the populations they serve.”

Gerald Chertavian, founder and CEO of Year Up, said, “Access to quality health care and the financial sustainability that comes with good jobs go hand in hand in building an equitable and just society. We are proud to partner with the Vertex Foundation to develop career pathways in health care services for underrepresented communities in Massachusetts.”

Earlier this month, Vertex provided updates on its Phase I/II trial of VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for type 1 diabetes. Two patients in Part A received the therapy at half the target dose. The first patient achieved insulin independence at Day 270 with an HbA1c of 5.2%. The second patient demonstrated robust increases in fasting and stimulated C-peptide, with decreases in exogenous insulin requirements through Day 150. The first patient to receive the full dose, from Part B, achieved the Day 29 follow-up milestone.

However, the U.S. Food and Drug Administration placed a clinical hold on the study because it said there was insufficient information to support dose escalation. The two patients from Part A were believed to provide that data sufficient to dose the first patient in Part B.

“We are surprised by the clinical hold placed on the study,” said Dr. Carmen Bozic, M.D., executive vice president, Global Medicines Development and Medical Affairs, and chief medical officer of Vertex. “The results from the first two patients treated with half the target dose establish proof-of-concept by demonstrating that VX-880 can restore glucose-regulated insulin production and improve glycemic control. Indeed, achievement of insulin independence by the first patient is a landmark milestone. Further, the totality of the safety and efficacy data for all three patients dosed to date gives us high confidence in our benefit-risk assessment of VX-880 and its potentially transformative profile. We are committed to working with the FDA to understand and address their questions, so that the trial can resume at U.S. sites as soon as possible.”

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