Three years ago, analysts at Jefferies, a prominent Wall Street investment bank, called Vertex Pharmaceuticals "the clearest growth path in biotechnology".
the time, the company had just begun selling its third cystic fibrosis drug and was rapidly advancing the development of a fourth drug that was expected to treat more patients.
analysts had expected Vertex's annual revenue of about $2.5 billion to double over the next three to five years if all goes according to plan.
these predictions seem to be come true.
February 1, Vertex reported net income of $6.2 billion, up more than 50 percent.
this is related to the company's latest approval of the 4th cystic fibrosis drug Trikafta's heavy sales.
Trikafta, a triple therapy, had sales of $3.9 billion in its first full year on the market in 2020, including $1.1 billion in the fourth quarter, above Wall Street's widely expected 4 percent.
but it's worth noting that Trikafta's success also led to a contraction in Vertex's other three CF drugs, with total sales of $2.34 billion, down sharply from $3.591 billion in 2019.
Vertex executives said the company's sales revenue would climb to about $6.8bn in 2021, reflecting a further 10 per cent increase.
conservative for Vertex, it would be ambitious for other biotech companies of its size.
Monday, Vertex chief executive Reshma Kewalramani said on a earnings conference call that "we're really in a different position" than we were three years ago.
growth, investors seem increasingly worried about Vertex's future: How long can the company rely on its cystic fibrosis business to drive growth? On Tuesday, Vertex shares were trading at $219 a share, down 5 per cent from the previous day and well below last summer's $300.
because, while Vertex's cystic fibrosis business is booming, no one knows what its next big product will be.
addition to its cystic fibrosis business, Vertex's fastest-moving project is gene editing therapy for sickle cell disease and another rare blood disease.
the treatment has shown promising results in a small number of patients, it has not produced enough data to win the support of regulators or allay some of the bigger concerns surrounding gene editing.
Behind this project, Vertex has been working to develop a treatment for a genetic disease called α-1 antitrinase deficiency, in which patients do not have enough alpha-antitantase (alpha1-AT) in their bodies, leading to lung and liver damage.
year, Vertex suffered a major setback in the project, with safety issues in a mid-term study leading to the prescription drug being shelved.
, however, the company has two alternatives, one of which will provide proof-of-concept data in the first half of this year.
the Vertex pipeline include treatments for a variety of diseases, from kidney disease and pain to muscular dystrophy and type 1 diabetes.
the company says these goals fit a pattern: a clear understanding of their biology, and new treatments could have a transformative effect on patients.
these assets will further make Vertex an important player in the field of specialized medicines.
, however, are clearly not convinced by this defence.
SVB Leerink analyst Geoffrey Porges wrote in a note to clients: "Although the company's buzzwords are 'cracking creatures' and 'pouring chemistry', there are insidities between their portfolios and strategies."
" Vertex was forced to use its huge cash reserves to expand through acquisitions as doubts admed.
end of last year, Vertex held nearly $7 billion worth of cash and cash equivalents, enough to buy some assets or companies.
Vertex executives have accepted the idea of more deals for the time being.
the past few years, the company has become more active in trading.
in 2019, Vertex acquired Semma Therapeutics and Exonics Therapeutics, acquired cutting-edge technologies such as cell therapy and gene editing, and recently collaborated with Skyhawks on drugs that modify RNA.
at last month's JPMorgan Healthcare conference, Vertex chief executive Reshma Kewalramani said the company was actively looking for "tools and technology" to acquire, particularly mid- to late-stage assets, to strengthen the pipeline.
asked for details, Reshma Kewalramani said only that "any deal must be transformative".
a conference call Monday, Vertex's leadership confirmed that the company remains interested in further building its technology toolkit.
, because of the cash position, the company said it was now more open to trading in mid- to late-stage assets, which was often more expensive than the early projects Vertex has focused on so far.
, Vertex is not very specific about the details of its future plans.