BLUE fell 20% after mixed Northstar data.
But SCD is the larger market than beta-thalassemia major.
BLUE has one solid patient result so far in SCD, and is enrolling more patients in the trial.
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bluebird bio (NASDAQ:BLUE) fell more than 20% since November 5, on release of the abstracts from the upcoming ASH conference in December. The data showed that while 4 non-β0/β0 genotype patients from Northstar and 2 β0/βE genotype patients from HGB-205 were doing well, 3 β0/β0 patients were not.
This is somewhat mixed data, but what I find even more important is that from the mixed trial HGB-205, one SCD patient -a 13 year old boy - is also doing extremely well, producing 15% more anti-sickling hemoglobin than the 30% required for a disease-modifying effect. That is the only patient whose data seems to be available so far - the data from the pure SCD HGB-206 is from July and does not report how patients are doing. However, one hint of good results visible internally is that BLUE is raising patient enrollment from 8 to 20. One opinion is that higher patient enrollment means there is some suspicion of good data, which will be better tested in a larger cohort. I cautiously agree to that view.
Consider also that BLUE has almost $1bn in cash - more than a third of its market cap - and that Oppenheimer recently increased its stake by over 6o% in the company. Consider also that more detailed SCD results will be available on the bylines of ASH next month, and also how badly the stock is doing right now, and I believe this is a good time for a cautious entry into the stock.
On the flipside, as some have noted, is whether insurers will be willing to pay upfront for longer term effects, but that is a story yet to be written. The cautionary note is about the SCD data. Given that some of its essays in beta-thalassemia is non-effective, and CAR-T is still pre-clinical, SCD is the only deal in town right now. If data from SCD is not good enough, the stock will fall further.