Fortune USA - 11.2019

PROFILE 2019 | FUTURE 50

CONTENT FROM VERTEX

BIOTECH COMPANIES DO NOT GROW LIKE

other businesses. In place of linear ad-

vances, there are exhilarating successes

followed in quick succession by crushing

failures. Vertex, a 30-year-old biotech

company based in Boston, knows this

journey well.

In 2012, when current CEO Dr. Jeffrey

Leiden joined the company, it was at a

critical inflection point. That year, the com-

pany introduced a blockbuster drug to

treat hepatitis C, only to be surpassed by

a competitor and have the product taken

off the market less than two years later.

“[Back then] we were losing $300 million to

$500 million every year, and the options

for growth were limited,” Leiden says. “We

needed a way to refocus the company.”

Working with the board of direc-

tors and senior management, Leiden

realigned the company’s mission to

concentrate on “transformative medicines

for serious diseases in specialty markets.”

Vertex wasn’t looking to create the next

flu vaccine. Instead, it would focus on

discovering new medicines to treat the

underlying cause of serious diseases like

cystic fibrosis (CF); Leiden felt that those

medicines would bring the most value to

patients and society.

This strategy has allowed Vertex to

introduce new medicines into the market

with a relatively small sales and market-

ing budget, and then pump revenue back

into research and development—and the

approach is paying off. According to the

company, when Leiden became CEO,

there were no drugs to treat the underlying

cause of CF.

“We’ve done in CF what no one thought

possible 20 years ago,” Leiden says. Today,

Vertex has three CF medicines on the

market, and a fourth is under review by the

Food and Drug Administration. Vertex has

used this same model to build its future

investigational drug pipeline by expanding

research into potential cures for other seri-

ous diseases, such as sickle cell disease

and Duchenne muscular dystrophy, a

genetic disorder marked by progressive

muscle degeneration and weakness. Since

2012, the company’s market cap has

soared from $6.5 billion to $46 billion.

Vertex fortifies its laser focus by relying

on a diverse workforce. More than half of

its global employees are women, and four

of its nine directors are of diverse gender

or ethnicity. It’s that diversity of thought

and experiences that helps Vertex make

the best decisions. “Drug discovery is

incredibly complex,” says Leiden. “It’s

the ultimate team sport.” ■

Innovation and laser focus are helping Vertex find treatments for

some of the most intractable diseases out there.

A Biotech Company

Focused on Finding

Transformative

Treatments for

the Most Serious

Diseases

LEFT: Research scientist Sarah Blair-Reid at work in a Vertex

lab. RIGHT: Jeffrey Leiden, MD, Ph.D., chairman, president,

and CEO, in a Vertex lab.