Vertex news
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Vertex Pharmaceuticals presented positive long-term data for its gene therapy Casgevy and issued a business update on the product. Casgevy is their gene editing therapy, made by editing the BCL11A gene using patients own stem cells. A one time albeit very expensive therapy.
For patients with transfusion-dependent beta thalassemia, or TDT, 98% of patients, or 53 out of 54, who were at least 16 months of follow-up achieved transfusion-independence for at least 12 consecutive months.
The data was presented at the annual meeting of the American Society of Hematology, which is being held Dec. 7-10 in San Diego.
Vertex (VRTX) said that over 45 authorised treatment centres have been activated around the world to support the delivery of Casgevy, with more than 40 patients having had a first cell collection. The therapy costs between 2.2 and 3 million dollars
Vertex (VRTX) added it has received approval for a third manufacturing facility for the product with its partner Lonza
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The FDA decision on whether Vertex's Suzetrigine, a non-opioid medication for moderate-to-severe acute pain, is expected tomorrow, Jan 30. The drug demonstrated positive phase 3 trials, meeting primary endpoint objectives and showed significant pain reduction compared to the control, placebo.
Given the favourable results we expect, approval. This of course is not guaranteed. Watch this space.
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Approval granted, up after hours
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Nice-I did wonder, having not seen anything from the FDA by 10pm +$36/8%. First-in-class status. This is the first drug ever to be close to the effectiveness of an opiod. It's an important step. Whilst opioids work very well, great efficacy, they have well documented side effects and can be deadly if abused.
This discovery has been linked to mutations in the SCN9A gene, which encodes the NaV1.8 sodium channel involved in pain signaling. In other words some people have mutations which block pain naturally. What vertex have achieved is create a drug which targets the same sodium channel as the gene mutation. Very clever.
From their work -A Study documented a Pakistani street performer who could walk on hot coals and stab himself without feeling pain. Genetic analysis revealed that he and some of his family members had a mutation that disabled the SCN9A gene entirely, leading to a complete lack of pain sensation.
Should be a big day today. KLAC/AAPL/VRTX.
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On the BBC today-another Vertex discovery https://www.bbc.co.uk/news/articles/cx2yg9yny0ko
The cost of this one-time drug therapy is > £1.5M per and my understanding is the company doesn't make a lot of money from it but it's a stepping stone in understanding and cracking the path to other discoveries. It's not often you can invest in something, make a profit and make a difference to the wellbeing of so many people.
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On the BBC today-another Vertex discovery https://www.bbc.co.uk/news/articles/cx2yg9yny0ko
The cost of this one-time drug therapy is > £1.5M per and my understanding is the company doesn't make a lot of money from it but it's a stepping stone in understanding and cracking the path to other discoveries. It's not often you can invest in something, make a profit and make a difference to the wellbeing of so many people.
@Adam-Kay said in Vertex news:
On the BBC today-another Vertex discovery https://www.bbc.co.uk/news/articles/cx2yg9yny0ko
The cost of this one-time drug therapy is > £1.5M per and my understanding is the company doesn't make a lot of money from it but it's a stepping stone in understanding and cracking the path to other discoveries. It's not often you can invest in something, make a profit and make a difference to the wellbeing of so many people.
Rarely has an article produced such conflicted feelings in me; while I'm delighted that the drug has been developed and is so effective I'm slightly aghast that it's being offered on the NHS. £1.5m per treatment (article says that the exact cost is confidential but there's not going to be much discount if the company doesn't make much money on it) means that's a big 'ol cost to the state. I guess the flip side is that if my tax dollars are being spent in this way then it's a good thing that I have a (small) investment in the company who is selling it.
However I'm trying to have a political discussion and this is a finance forum, so I'll pipe down.
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The drug cost > $1B to develop so the price (in the US $3.1M) needs to recover this cost and make a profit. Sickle Cell disease is a terrible illness(genetic) often leading to poor quality of life and shortened life expectancy . Many who suffer from it have regular hospitalisation so you need to factor in the large cost over a number of years vs the upfront cost now coupled with the pain and suffering. The reality is, you've read the headline. They will probably only process a handful of patients a year-there will be a budget.
Vertex is not like most drug companies. Their charter is to work in the field of transformative medicines. Medicines that improve healthcare outcomes through discovery and technology. The hard stuff.
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Nice pop for vertex today
Vertex’s appearance at the Citi Healthcare Conference hit the right notes for investors, and the market reaction reflects it. The company reiterated the strength of its core cystic-fibrosis portfolio, which continues to deliver dependable, expanding revenue and remains the backbone of its multi-billion-dollar quarterly performance.
Management reinforced that recent growth trends are holding up well, with momentum expected to persist.
A major positive was the emphasis on financial capacity. Vertex sits on roughly USD 12 billion in cash and marketable securities, giving it the freedom to progress late-stage programmes without taking on debt or issuing shares. That level of balance-sheet strength is uncommon in biotech and signals stability as well as optionality for future deals or accelerated development plans.
Pipeline updates also contributed to the upbeat tone. Programmes in kidney disease, gene editing and other high-value therapeutic areas were highlighted as near- and medium-term opportunities with significant commercial potential. Analysts have already argued that Vertex may be priced below its long-term fair value, and today’s messaging essentially validated that view by showing that both innovation and execution remain firmly on track.
Overall, the presentation delivered a clear story: robust revenue today, meaningful breakthroughs underway and the financial muscle to drive both without compromise.
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Vertex Earnings-stock pops
Vertex Pharmaceuticals delivered a solid, if unspectacular, fourth quarter for 2025. Revenue came in at roughly $3.19 billion, up around 9–10 per cent year on year and slightly ahead of expectations. Full-year revenue was about $12 billion, also representing roughly 9 per cent growth.
The cystic fibrosis franchise remains the core engine of the business. Trikafta and Kaftrio continue to generate strong global sales, supported by label expansions and high margins. There is no sign of erosion in the underlying franchise, which underpins the company’s profitability and cash generation.
Importantly, diversification beyond cystic fibrosis is beginning to show traction. Casgevy, the gene therapy for sickle cell disease and beta thalassaemia, contributed incremental revenue, and Journavx in acute pain is starting to build prescription volume. Pipeline programmes in kidney disease and other areas are advancing, supporting the longer-term growth narrative.
Profitability remains strong, and the balance sheet is robust, with more than $12 billion in cash and equivalents at year-end. The company also continued share repurchases during 2025, returning capital while still funding research and development aggressively.
Earnings per share missed consensus slightly, largely due to higher operating expenses tied to launches and pipeline investment rather than any major one-off charge. Overall, the results reinforced steady growth, strong cash generation and improving diversification, which explains the constructive market reaction.
We originally purchased Vertex in April 22 for $256 and it is $496 today, just under 100% growth in under 4 years which is an 18.8% annual return. It's been a solid return, anchoring the portfolio in quality
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Vertex pops after hours due to very positive phase 3 drug test results. Up 10%. Great day today too generally
Details tomorrow on VRTX
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Vertex Pharmaceuticals reported three very positive clinical updates across its kidney-disease pipeline, with the biggest coming from the Phase 3 RAINIER trial of its drug povetacicept. The trial met its primary endpoint and key secondary endpoints, which is a strong signal for regulators and investors.
The headline result: patients receiving povetacicept showed roughly a 50%+ reduction in proteinuria (excess protein in urine), a major indicator of kidney damage. Proteinuria levels strongly predict progression to kidney failure, so a reduction of this magnitude suggests the drug could substantially slow—or possibly halt—disease progression in some patients.
Diseases targeted
Vertex’s kidney pipeline is focused on several serious conditions:
IgA Nephropathy (IgAN) – the primary target of the latest Phase 3 trial.
Primary Membranous Nephropathy (pMN) – an autoimmune kidney disease.
APOL1-Mediated Kidney Disease (AMKD) – a genetic kidney disorder more common in people of African ancestry.
Autosomal Dominant Polycystic Kidney Disease (ADPKD) – a progressive inherited condition that leads to kidney failure.These are significant markets. IgA nephropathy alone affects roughly 300,000 people across the U.S. and Europe, with many eventually progressing to dialysis or transplant.
Is it a big deal?
Potentially yes. IgA nephropathy currently lacks therapies that directly address the immune cause of the disease. If povetacicept truly modifies the disease rather than just managing symptoms, it could become a best-in-class treatment and possibly delay kidney failure for many patients.Commercial potential
Because these are chronic diseases requiring long-term treatment, analysts estimate multi-billion-dollar annual revenue potential. Across several kidney indications, the drug franchise could realistically reach $3–7+ billion per year.Timeline
Regulatory filings: expected in 2026
Potential accelerated approval: late 2026 to 2027
Commercial launch: likely 2027
Bottom line: The results are strong enough that this could become a major new kidney-disease therapy—and potentially a multi-billion-dollar franchise if subsequent data confirm the benefit. -
Could ... maybe ... potentially ...
I'm mindful that the failure rate of drugs in development is MASSIVE; many start the journey looking promising but most fail along the way. However this is a helpful update, thanks Adam.
Interesting that Vertex is concentrating on kidney disease. I can see the logic in setting up a centre specialising in a particular part of the body and looking at drugs for that part.
Thanks for the news.
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Vertex have a good track record. We can't ever expect anything more that 'could, might, maybe'. It's still a drug trial. Nothing is certain until it's approved and commercialised.
Vertex specialise in what is called Transformative therapies. Drugs which transform the pathway of a disease from mere managing symptoms to a long term cure/outcome. It's nice to know we invest in something that improves peoples quality of like. More specifically Vertex focus on genetic diseases (gene editing)
In terms of success from drug trials. At phase 1 statistically, the odds of success sit at 10-15%. At the conclusion of a successful phase 3 trial-and the results were not just positive, they were extremely positive, the odds increase to 85-90% so I am confident they are onto something.
There are many more drugs in their pipeline-Diabetes is the next potential break-through
