Vertex Pharmaceuticals’ blockbuster cystic fibrosis (CF) drug, Trikafta, is so effective that studies predict it could extend the lives of some young patients up to 45 years. As such, it’s not cheap: in the United States, it costs $300,000 per patient per year. But given the transformative nature of treating a disease with a poor prognosis, the high list price has not slowed the pharmaceutical group’s sales.
And now the same team of scientists at San Diego’s Vertex lab has a second, decades-long research project coming to fruition that could pave the way for another potentially huge market: non-opioid painkillers.
If patients live longer, “it’s good for business,” says David Altshuler, Vertex’s chief scientific officer. The number of people with cystic fibrosis worldwide now stands at 105,000, up from 70,000 in 2012, according to estimates by the Cystic Fibrosis Foundation, a US non-profit organization – an increase that experts attribute to both to Vertex drugs and improved diagnostics.
“If you take a life-shortening, life-threatening disease and you have a transformative drug, not only do you get short-term benefits, but people then live a long time and that’s a positive thing for everyone.” , adds Altshuler. These positive effects are evident in Vertex’s revenues, which more than doubled between 2019 and 2022, from $4.2 billion to $8.9 billion, the period covered by the FT-Statista 500 rankings. fastest growing in the Americas. Revenues then increased again, reaching $9.9 billion, in 2023.
Since its launch in 2019, Trikafta has generated $26 billion in revenue and is expected to reach $10 billion in annual sales next year. The Boston-based biotech is also poised to seek regulatory approval this year for its fifth CF treatment, known as Vanza Triple, which is also expected to generate nearly $10 billion in annual sales, according to the investment bank William Blair.
Vertex “will likely be one of the fastest growing lines outside of weight loss drug makers.” [Eli] Lilly and Novo [Nordisk]predicts Debjit Chattopadhyay, healthcare analyst at Guggenheim Securities. “I would place Vertex in my top 3 pharmaceuticals [to invest in].” This year, Vertex’s market capitalization exceeded $100 billion for the first time.
Daniel Lyons, portfolio manager at Janus Henderson, one of Vertex’s top 25 shareholders, says the biotech faces “no real competitive threat” to its dominance in the CF space, with patents extending to ‘in 2039 at least.
“It gives the company an incredibly profitable core business and it has essentially reinvested a significant portion of those profits back into its pipeline.”
Altshuler says Vertex learned the importance of continued investment in research through the troubled rollout of its hepatitis C treatment, Incivek. After becoming the fastest drug to reach $1 billion in annual sales, it was quickly supplanted by a superior treatment from rival Gilead. “The first or second drug is never the best, but most companies think no one will follow them, so they stop,” he says. “We learned from this: never assume you are going to win; continue to build better and better medicine.
However, Vertex has been criticized for the high price of Trikafta, which means only 12% of cystic fibrosis patients worldwide have been prescribed the treatment, according to a study published in the Journal of Cystic Fibrosis in 2022.
The researchers concluded that “the drugs are so expensive that they are practically only available if they are reimbursed by the government or health system authorities.” Altshuler says the “vast majority” of cystic fibrosis patients worldwide who meet the criteria for prescribing Trikafta have been prescribed the drug.
The challenge for Vertex is whether it can replicate its success in cystic fibrosis in other diseases. The company received regulatory approval last year for the first-ever gene-edited treatment Crispr, which targets sickle cell disease and beta thalassemia. It also paid $4.9 billion this month for Alpine Immune Sciences, which is developing a treatment for autoimmune kidney disease.
But it’s a new generation of non-addictive painkillers that analysts say are the most promising drugs in Vertex’s pipeline.
Vertex hopes to receive approval from the U.S. Food and Drug Administration this year for a non-opioid painkiller, known as VX-548, after a late-stage clinical study showed it reduced pain with a much lower incidence of side effects such as nausea and vomiting. — and without risk of dependence.
“Every year we accumulate evidence that we are going to eradicate another disease,” says Stuart Arbuckle, Vertex’s chief operating officer. He claims that Vertex’s $4.8 billion R&D budget is not about maximizing “shots on goal”; rather, it focuses on “a set number” of diseases for which Vertex has “a very strong belief that they will work.”
Vertex’s acute pain drug is expected to generate $2.3 billion in annual sales by 2030, according to analysts’ consensus forecasts, but the much bigger opportunity lies in an approved non-opioid painkiller that can serve as a alternative to prescription opioids that have led to the deaths of hundreds of thousands of Americans.
“Wall Street is everywhere asking questions about how to deal with chronic pain, because of the filth of the opioid crisis and because people became optimistic about pain launches and those launches failed,” said one person from a biotechnology fund with a stake. at the top.
To overcome competition from cheap generic opioids, Vertex hired a sales force of several hundred people – far larger than its cystic fibrosis-focused team – to work on launching the product.
Some analysts say non-opioid painkillers could be the next class of blockbuster drugs, with an impact on the industry similar to that of weight-loss drugs. “Pain could be analogous to another GLP-1 scenario,” says Hartaj Singh, a biotechnology analyst at equity research firm Oppenheimer, referring to the hormone targeted by weight-loss drugs. “If we can move from acute pain to chronic pain, we could be talking about a revenue potential of $10 billion to $20 billion. »
Altshuler recalls how experts dismissed GLP-1. “What they said… ten years ago there was no need for diabetes and obesity drugs: they’re all generic, nothing really works, it’s not going to happen. It happened. turned out to be false. I can’t promise it, but for me. [the pain franchise] looks like [CF] in 2012.”
