Thursday, November 15, 2018 2:25:18 PM
QURE:
I thought the data points quite good... The caveat is why no curves and why present it today and not at R&D Day Monday? They also presented the top line data a little weird. The PR said average at 6 weeks but they give us values at week 6, week 10 and week 8 (which avg to 30 could be 31 with rounding). So its a bit ambiguous were their values the same a few weeks earlier (or one patient up one down)? I'd expect the values to be rising for several more weeks (based on what others have shown).
Graphs should clarify any doubt Monday my concern would be those two who screen-failed another GT (Sparks or Dimension most likely?). Qure may have been too greedy doing an all comers. While its true they did "accidentally" dose I believe a couple patients with low antibody titers in their prior study why risk it here? Developing antibodies to one serotype is known to make a patient develop antibodies against others. BMRN which is also using AAV5 is screening out those with pre-existing antibodies. Of course, they have the dollars so they are doing a separate study for those with low levels.
Here is a nice graph From @AndyBiotech (hope he doesn't mind me stealing it)
and a link to his tweet (note the third company has Nathwani who did the original Hemophilia B studies)
I thought the data points quite good... The caveat is why no curves and why present it today and not at R&D Day Monday? They also presented the top line data a little weird. The PR said average at 6 weeks but they give us values at week 6, week 10 and week 8 (which avg to 30 could be 31 with rounding). So its a bit ambiguous were their values the same a few weeks earlier (or one patient up one down)? I'd expect the values to be rising for several more weeks (based on what others have shown).
Graphs should clarify any doubt Monday my concern would be those two who screen-failed another GT (Sparks or Dimension most likely?). Qure may have been too greedy doing an all comers. While its true they did "accidentally" dose I believe a couple patients with low antibody titers in their prior study why risk it here? Developing antibodies to one serotype is known to make a patient develop antibodies against others. BMRN which is also using AAV5 is screening out those with pre-existing antibodies. Of course, they have the dollars so they are doing a separate study for those with low levels.
Here is a nice graph From @AndyBiotech (hope he doesn't mind me stealing it)
and a link to his tweet (note the third company has Nathwani who did the original Hemophilia B studies)
$QURE vs $ONCE vs Freeline#Hemophilia B Gene therapy data competitive landscape in one chart
— Andy Biotech (@AndyBiotech) November 15, 2018
Freeline is one serious competitor to watch out for $QURE and $ONCE.
Impressive initial data at low dose, more data coming at #ASH18:https://t.co/7lMQ42J1Y6 pic.twitter.com/Wyv18n9Gg7
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