I asked a a scientist the other day from Incyte about YMI's anemia effect. He said the molecule was different and gave me a 30 minute lecture. Yes it is real it is different and after his lecture I fully expect anemia to hold up. Other side effects like thrombocytopenia could be an issue though! However he didn't think the molecule was that different enough to overcome Orphan drug status. Going around phase 3 is about the dumbest thing I heard on this board! Are you longs idiots! No way! It is either another huge dilution or buyout. How do you explain to the board of directors that you want to buy a company connected to Cuba? Dilution! dead money for about three years! 70mm cash on hand will go fast really fast.
"However he didn't think the molecule was that different enough to overcome Orphan drug status. "
Did you tell him that some enrolled in the CYT387 trial had previously been in the INCY trials? And that they were receiving benefits from CYT387? Seems to me that the molecules are different enough to matter to them. Give me some time to dig up a link to support this, my memory isn't as good as it was.
I'm going on more of a guess, because I'm not 100% certain, but I do not believe the anemia effect was originally known when YMI acquired the drug from Cytopia. As far as I know, there was nothing to suggest it. It was found along phase I trials and furthered into phase II trials. I do not believe there has been any articles explaining the reason for why the anemia effect exists. This may have changed, but as far as I know, they are not 100% certain why it has the effect.
I believe that there is speculation that the JAK mutation is what causes lowered hemoglobin production in the cells which help to carry oxygen. Without this, anemia develops. Remember in itself that tissue scarring of the bones is the primary culprit behind myelofibrosis. The mutation is caused in the JAK2V617F (had to google that) protein which is what begins the myeloproliferative symptoms.
It's quite possible that Ruxo's ability to delay, or de-intensify (right word?) the signaling of the JAK protein to slow blood proliferation to a norm works, but does not sufficiency allow for the bones to properly create healthy cells with adequate hemoglobin. CYT387 however has shown to do so. It essentially, as of now, is more effective.
I believe Dmattingly will have your answer. If not, there's at least 2 other people on this forum who know a good amount about the condition and drug. I for one feel I know enough, but not nearly as much as some others in here who might as well be doctors.
"...but as far as I know, they are not 100% certain why it has the effect. "
And this could be critical. Cause and effect relationships can be quite difficult to prove scientifically, and when you mix in business and political interests, watch out. It took decades, for example, to establish a cause/effect relationship between smoking and lung cancer.