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.