Diabetes Research Institute Investigators Will Lead the First Site Activated for the Vertex Study

MIAMI, FL – April 6, 2021 – Researchers with the Diabetes Research Institute (DRI) at the University of Miami Miller School of Medicine and Vertex Pharmaceuticals Incorporated have initiated clinical trials for VX-880, a novel investigational cell therapy with the potential to restore normal glucose control in those suffering from type 1 diabetes (T1D) with severe hypoglycemia and impaired hypoglycemic awareness. The University of Miami Health System was the first clinical site activated for this trial.

The program was granted Fast Track Designation by the U.S. Food and Drug Administration (FDA), which offers an expedited review process to accelerate treatments for urgent and unmet medical needs. It will evaluate the safety, tolerability and efficacy of VX-880 infusion in certain T1D patients. To be considered as a candidate for the trial, participants must have a clinical history of type 1 diabetes of at least five years, be 18-65 years of age, and have multiple episodes of severe hypoglycemia and impaired hypoglycemic awareness 12 months prior to enrollment.

“It’s a remarkable time for T1D research efforts worldwide as this investigational treatment enters the clinic,” said Camillo Ricordi, M.D., Professor of Surgery, Director of the Diabetes Research Institute and the Cell Transplant Center at the University of Miami Miller School of Medicine, Steering Committee Chair for the VX-880 clinical trial and Principal Investigator at the University of Miami Health System clinical site. “The field’s experience with the limited cadaveric islet transplants available, where some patients have experienced prolonged insulin independence for years, provides important proof-of-concept for the potential of cell therapy to be transformative for patients living with T1D.”

The program’s groundbreaking work began in Dr. Doug Melton’s lab, who developed this line of insulin-producing pancreatic cells derived from stem cells. Vertex acquired Semma Therapeutics, the biotechnology company founded by Melton, in 2019.

Type 1 diabetes is an autoimmune disorder that occurs when the body’s immune system attacks its own insulin-producing islet cells. Once all the islets are destroyed, patients must take daily injections of insulin or use an insulin pump to replace the hormone. As one of the largest and most comprehensive research centers dedicated to curing diabetes, the Diabetes Research Institute is aggressively working to develop a biological cure by restoring natural insulin production and normalizing blood sugar levels without imposing other risks.

“I am grateful to the DRI team that will help with this unprecedented, first-in-human clinical trial. Representing the DRI heart of our entire translational and clinical efforts: the cGMP Advanced Cell and Biologic Product Manufacturing Facility (Dr. Elina Linetsky, Xiaojing Wang, Clarissa Lenero), the CCTP Clinical Cell Transplant Program (Dr. Rodolfo Alejandro, Dr. David Baidal, Dr. Raffaella Poggioli, Ana Alvarez) and the regulatory team (Dr. Kemraj Hirani, Luis Roque, Burlett Masters), and Dr. Prasoon P. Mohan, the UHealth interventional radiologist who will infuse the cells,” added Ricordi.

The University of Miami Health System was the first clinical site activated for this trial, that now also includes the University of Pennsylvania and Massachusetts General Hospital. The three clinical sites are open for enrollment, and additional sites will be activated this year. To learn visit clinicaltrials.gov.

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About the Diabetes Research Institute and Foundation
The mission of the Diabetes Research Institute Foundation is to provide the Diabetes Research Institute with the funding necessary to cure diabetes now. The Diabetes Research Institute at the University of Miami Miller School of Medicine leads the world in cure-focused research. As one of the largest and most comprehensive research centers dedicated to curing diabetes, the DRI is aggressively working to develop a biological cure by restoring natural insulin production and normalizing blood sugar levels without imposing other risks. Researchers have already shown that transplanted islet cells allow patients to live without the need for insulin therapy. Some study participants have maintained insulin independence for more than 10 years. The DRI is now building upon these promising outcomes through its BioHub strategy, a multidisciplinary, three-pronged approach for addressing the major challenges that stand in the way of a cure: eliminate the need for anti-rejection drugs, reset the immune system to block autoimmunity, and develop an unlimited supply of insulin-producing cells. For more information, please visit DiabetesResearch.org or call 800-321-3437.

About Vertex and VX-880
For more information, please visit www.vrtx.com.

Media contact:
Lauren Schreier
lschreier@drif.org