Re: Trade - EIGR
Part 1(hope this helps):
1)There are 100K patients in the US with SCD and 60K patients in the EU. The cost of care for the average patient with this affliction is $200K per year.
2)It would seem to me to be able to have patients easily identified. Also, the price for the drug could be very cost effective if they are smart. So cost effective and IMO helpful for the disease that they could outcompete gene therapy. For gene therapy it is hard to see them not charging at least $1 million for the drug. True it MIGHT be a cure for the disease but how do you really know?! If this drug from GBT is priced at like $20K per year and you get 80% of the effectiveness of gene therapy at one-200th the price insurance carriers will IMO go with the GBT drug.
3)The rationale for buying this stock is this medication for SCD. The way this drug will save lives and money is the helping of straightening out red blood cells. The medication makes the cells get more circular rather than stay as the disease is named after, sickle cell shaped. This allows the red blood cells to stay around longer, the numbers being 120 days a normal red blood cell stays around vs. 6 days for a sickle cell. The drug is a small molecule, easy to take and it is once a day.
4)Their study, which IMO was long enough to show significant change but one would expect that over more time you would get better results than what we have seen. Those results include the situation where the red blood cells stay longer so you are not as anemic. Also, with the red blood cells having a more circular shape they will be notably less likely to clog up in small arteries in various organs and reduce vasoclusive crisis(VOC). Their study was not powered for this by I believe from listening to their experts that they did see this effect.
5)It appears to be safe to be started at as earlier as six months of age. That is the time about when fetal hemoglobin that is not affected by SCD gets replaced by the actual sickle cells. The approval if it happens will be down to four years of age but I believe that over time they will try for the younger ages.
6)Almost 60% of the patients had an increase of >1 gm of hemoglobin. I believe that over time both the numbers affected and the amount of hgb increase will go up.
7)The most amazing thing about this drug is that it could stop the large amount of blood transfusions that select patients have to endure. The more blood transfusions that you have to go through the more likelihood that you will develop more and more antibodies making it still harder to get blood that will match that will allow another transfusion.
8)Dr. Robert Adams recently gave and outstanding presentation at one of GBT's CC's. How they were able to figure this out is beyond me. But what he noted is that they started doing transcranial Dopplers(TCD) in children with SCD. Before TCD 100 children transfused to prevent one stroke. Number to treat was then 100. With transcranial Doppler they were able to identify the children at highest risk reducing the number to treat down to ten given blood transfusions to avoid a stroke.
9)What they discovered was the the patients with the highest pulse wave velocity had the highest risk of having a stroke and that patients with the worst anemia had that highest pulsev wave velocity. The vessels are narrower in SCD patients also contributing to this problem.
10)They were able to parse it out in numbers for risk stratification in regard to your pulse wave velocity numbers. Normal was <170 cm/sec and very high risk was >200 cm/sec. An in between category that they don't have direct recommendations on for blood transfusions was the numbers between 17-200. Starting at 140 cm/sec for every 10 cm increase you had a 29% increased risk for a stroke.
This drug if priced right could easily be cost effective:
-Low risk of severe adverse events.
-Numbers so far that show that probably could be used down as low as 6 months....though the approval if given for now would not cover that.
-Greatly reduced need for blood transfusions, and probably a drop in VOC's.
-This drug could usurp the NVS drug which is going for prophylaxis for VOC's, but it would seem that the GBT drug would work farther upstream and will be notably more cost effective as it will be a small molecule vs. Mab.
I'll take a look at the report in detail that you posted and give my opinion....for what it is worth:)) t