Vertex Pharmaceuticals

Vertex Pharmaceuticals is a biotechnology company headquartered in Boston, Massachusetts, United States. The company specializes in the discovery, development, and commercialization of small-molecule drugs for the treatment of serious diseases. Founded in 1989, Vertex is one of the oldest biotechnology companies in the world.

Vertex's primary focus is on developing treatments for cystic fibrosis, a genetic disease that affects the lungs and digestive system. The company has developed several drugs to treat this condition, including Kalydeco, Orkambi, and Symdeko, which are approved in the United States and Europe. Additionally, Vertex is researching treatments for other conditions such as hepatitis C, alpha-1 antitrypsin deficiency, and sickle cell disease.

Vertex is also involved in developing precision medicines, which are treatments tailored to the genetic makeup of an individual patient. The company has developed gene-editing technology to create treatments that target specific genetic mutations.

Vertex has collaborated with a variety of organizations in the biotechnology and pharmaceutical industries, including Johnson & Johnson, Janssen, and Bristol-Myers Squibb. Additionally, the company has formed a number of strategic partnerships with academic institutions to advance its research and development efforts.

Vertex has received numerous awards and recognitions for its innovative approaches to drug development and its commitment to improving the lives of people living with cystic fibrosis. The company's commitment to innovation has led to its success in the biopharmaceutical industry.

1989: In business since 1989, Vertex has only two minor HIV drugs to its credit and has never turned a profit, but the company has great expectations for two of its hepatitis C drugs in clinical trials and also has a number of cancer drugs in the pipeline. Vertex was founded by Joshua Boger and Kevin J. Kinsella to "transform the way serious diseases are treated."

1993: Vertex signed collaboration agreements with GlaxoSmithKline and Kissei to develop protease inhibitors to treat HIV infection.

1996: Vertex determined the shape of the enzyme: It possessed a shallow pocket, which unfortunately made it difficult to develop an inhibitor to fit it.

1998:  Vertex initiated its cystic fibrosis research program in collaboration with the Cystic Fibrosis Foundation. Clinical trials on this drug were launched, and the research also led to a blocker for a second kinase, which played a role in strokes. In addition, the company opened a research and development facility in the United Kingdom.

1999: Vertex saw the launch of its first product in May, Agenerase.

2000: With more than 3,000 employees and with offices in North America, Europe, Australia and Latin America, Vertex Pharmaceuticals, Inc. is considered one of the Top 2000 Largest Public Companies in the World, according to Forbes.

2001: The year also brought some unwanted publicity in the form of the company's former chief lawyer, Andrew S. Marks, who pleaded guilty to insider trading for dumping stock in September, just prior to an announcement that Vertex was halting clinical trials on its arthritis drug. To this point, all of the growth at Vertex was internal, but the company tried its hand at external growth, using nearly $600 million in stock to acquire San Diego, California-based Aurora Biosciences Corp.

2002: Vertex started with a dozen products in the pipeline and more than $740 million in cash and investments.

2003: The company decided on a more prudent course, focusing its resources on products it believed were the most fully developed.

2006: Heuser, Stephen, "Vertex Forges $545 Million Deal with J&J," Boston Globe, July 1. Early results were such that Vertex decided to make it one of the key drug development projects.

2011: Since late the year, Vertex has ranked among the top 15 best-performing companies on the Standard & Poor's 500.

2014: In January, Vertex completed its move from Cambridge, Massachusetts, to Boston, Massachusetts, and took residence in a new, $800 million complex.

2015: Vertex and CRISPR Therapeutics began a research collaboration focused on the use of CRISPR’s gene-editing technology (CRISPR-Cas9) to discover and develop potential new treatments aimed at the underlying genetic causes of human disease.

2016: Vertex and Moderna began a research collaboration focused on the discovery and development of messenger Ribonucleic Acid (mRNA) Therapeutics for the treatment of cystic fibrosis (CF).

2019: On 23 January, Ian Smith, the COO and interim CFO of Vertex was terminated from his position for undisclosed personal behaviour that violated the established company code of conduct rules. In September 2019 the company announced it would acquire Semma Therapeutics for $950 million in cash.

2020: Former CEO and president Jeffrey Leiden transitioned to the role of executive chairman of the board of directors, as of 1st April.

2021: For the 12th straight year we’re included on the Science magazine’s Top Employer list, ranking #8 in 2021. We’re proud to have placed on both the 2021 Seramount (formerly Working Mother) 100 Best Companies list and the Best Companies for Dads list.

2022: Vertex ranked #6 in the Extra Large Employers Category.

According to Vertex, the company mission is to discover transformative medicines to help people with serious diseases like cystic fibrosis.

“We’re focused on discovering, developing, and bringing innovative medicines to people with serious diseases so they can lead better lives. In pursuit of this mission, we are advancing a robust research pipeline that includes potentially transformative treatments for cystic fibrosis, pain, sickle cell disease, beta thalassemia, alpha-1 antitrypsin deficiency, APOL1-mediated kidney disease, Duchenne muscular dystrophy, type 1 diabetes and more. ”

According to Vertex, the company vision is: 

“To create new possibilities in medicine to cure diseases and improve people's lives. The company works with leading researchers, doctors, public health experts and other collaborators who share our vision for transforming the lives of people with serious diseases, their families and society".

Medicines: In 2012, Ivacaftor was designated as an orphan drug, identifying cystic fibrosis as affecting fewer than 200,000 people in the United States. On 31 January 2012, Vertex gained FDA approval of the first drug, Kalydeco, to treat the underlying cause of cystic fibrosis rather than the symptoms, in patients 6 years or older who have the G551D gene mutation. In the US, 30,000 people have cystic fibrosis.

About 4% of those, or 1,200, have the G551D gene mutation. In 2017 Vertex marketed the drug for $311,000 per year.

Vertex also studied ivacaftor in combination with another drug (lumacaftor) for the most common mutation in cystic fibrosis (CF), known as F508del, and published the first set of results in 2012. Vertex produced the drug after 13 years of research and development, with $70 million in support from the Cystic Fibrosis Foundation.

In the UK, the company provided the drug free for a limited time for certain patients. Subsequently, the hospitals decided to continue to pay for the drug for those patients. UK agencies estimated the cost per quality-adjusted life year (QALY) at between £335,000 and £1,274,000—far above the NICE thresholds of £20,000-£30,000.

On 5 November 2014, Vertex announced the submission of a New Drug Application (NDA) to the FDA for a fully co-formulated combination of lumacaftor and ivacaftor for people with cystic fibrosis ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In 2015, FDA approved the combination of lumacaftor and ivacaftor to treat CF in patients 12 years and older, who have the F508del mutation. The combination drug is marketed under the tradename Orkambi.

On 28 March 2017, Vertex announced Phase 3 data from a dual combination treatment, Tezacaftor plus ivacaftor, in patients with cystic fibrosis. On 12 February 2018, the FDA approved the combination, marketed as Symdeko.

Triple Combination treatments: In 2016, Vertex began developing a new group of CFTR modulators in combination with tezacaftor and ivacaftor. In 2017, the company reported results that showed benefits for patients with different mutations that represent 90% of the CF population. On 22 October 2019, the FDA approved Vertex's Triple-combo therapy Trikafta (elexacaftor/tezacaftor/ivacaftor) for patients 12 and older with at least one F508del mutation. This approval came only 2 months after the new drug application was filed. Trikafta is approved for approximately 90% of patients with cystic fibrosis.

Vertex has refused to make Trikafta available in developing countries and works to block generic alternatives, making it inaccessible for thousands of patients.

Genetic therapies: 

• In 2015, Vertex entered into a research collaboration with CRISPR Therapeutics to develop gene-editing therapies for genetic diseases. In May 2020, CTX001 has received Orphan Drug Designation from the U.S. Food and Drug Administration for transfusion-dependent beta thalassemia and from the European Medicines Agency for sickle cell disease and transfusion-dependent beta thalassemia.
• In 2016, the company entered into a collaboration with Moderna to develop new mRNA-based therapeutics to treat CF; Vertex paid Moderna $20M in cash and provided an additional $20M in cash in exchange for a convertible note that Vertex can cash in for stock. Vertex also promised to pay up to $275M in milestone payments.
• In 2019, Vertex established a research collaboration with a privately held company Arbor Biotechnologies to discover novel proteins to advance the development of new gene-editing therapies for cystic fibrosis and four other diseases. With seven different areas of focus in the deal, Vertex is paying up to $1.2 billion in milestones along with undisclosed up-front payments.
• Vertex's product pipeline includes drug regimens targeting specialty diseases like sickle cell disease, beta thalassemia, Duchenne muscular dystrophy, alpha-1 antitrypsin deficiency, pain, and APOL1-mediated kidney diseases.