Press Release

BOSTON--(BUSINESS WIRE)--Jun. 21, 2018-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Senior Vice President Paul Negulescu has been named one of the winners of this year’s Warren Alpert Foundation Prize for “transformative discoveries in the fields of genetics, physiology, pulmonology and pharmacology that led to the development of life-altering precision-targeted treatments for the devastating multi-organ disease cystic fibrosis (CF).” Dr. Negulescu is one of five awardees, alongside Francis Collins, Director, National Institutes of Health; Bonnie Ramsey, Vice Chair and Endowed Professor of Pediatrics, University of Washington School of Medicine, and Director, Center for Clinical and Translational Research, Seattle Children’s Research Institute; Lap-Chee Tsui, founding President, Academy of Sciences, Hong Kong, Professor Emeritus, University of Toronto; and Michael Welsh, Professor of Internal Medicine Pulmonary, Critical Care and Occupational Medicine, University of Iowa.

The prize honors the groundbreaking work of Dr. Negulescu and the entire Vertex team who discovered and developed the first and only approved medicines that address the underlying cause of CF. Vertex has brought three transformative CF medicines to patients in the last six years, and has a pipeline of investigational CF medicines that have the potential to treat 90% of people with this devastating disease.

“It took the work of hundreds of people at Vertex across research, development, regulatory, and commercial to get these medicines from discovery to patients, and this award recognizes all of that hard work and dedication,” said Dr. Negulescu. “We have made great progress, but there are still more patients that need our help. So, we have more work to do.”

This year’s recipients represent the major scientific inflection points in the modern history of CF – from the discovery of the genetic mutations that cause CF all the way through the first FDA-approved medicines that treat the underlying cause of CF.

The Warren Alpert Foundation Prize is awarded annually to individuals whose scientific achievements have led to the prevention, cure or treatment of human diseases or disorders, and/or whose research constitutes a seminal scientific finding that holds great promise of ultimately changing our understanding of/or ability to treat disease. A symposium will be hosted at Harvard Medical School this fall to honor the recipients.

For more information visit the Warren Alpert Foundation Prize website: https://warrenalpert.org/

To read a Q&A with Dr. Negulescu about the Vertex team’s work in CF over the last 20 years, click here.

About Cystic Fibrosis

Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia. CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. This leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median predicted age of survival for a person born today with CF is 41 years, but the median age of death is 27 years.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston'sInnovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada and Australia. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for eight years in a row.

For additional information and the latest updates from the company, please visit www.vrtx.com.

(VRTX-GEN)

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Source: Vertex Pharmaceuticals Incorporated

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