Thursday, July 28, 2016 12:36:41 PM
BMRN / Hemophilia-A / Gene-Therapy:
I'll take a stab at some of your questions and raise some of my own
I think the rapid rise in Factor VIII levels being a concern is not as big a deal as some people are making it out to be. At first I thought that and then one must consider there is A LOT of room to dose down with the incredible levels they achieved. What it may say is they still need to work on dosing. Since the mid (and low) doses seems to produce much lower levels so it would not appear to be an issue that can't be addressed. People also need to keep in mind this is just an early 7 high (9 total) patients its an exploratory study not the final registration!
yes there were still bleeding episodes. Look at slide 16 I think
http://files.shareholder.com/downloads/ABEA-3W276N/2477655309x0x901460/C2800939-0F80-4E18-9B69-30EFF1569B04/BMN_270_Data_Update.pdf
The episodes are down to 2 at weeks 9-28 when the Factor VIII levels are much higher than earlier time points AND see the asterisk all but 1 bleed from week 2-28 was in a single patient (the lowest responder)!
I am long BMRN so probably biased but I put the results in the incredible category! The levels are so much better than would be needed to be considered an effective treatment. I don't follow a lot of other Gene Therapies for other Hemophilia's but the few I've seen have much lower levels of production of the deficient factor.
There is still a question of durability and longer term safety.
What I wonder still about is can Gene Therapy be a commodity. It still appears to me there is no shortage of academic researchers and licenses seem to happen frequently. That being said I've been tempted to play QURE and ABEO in that event.
EDIT:
The thrombocytopenia was discussed (briefly) on the call. It is a concern high levels do increase risk but not dramatically and nothing being done at present and again dose may be optimized.
I'll take a stab at some of your questions and raise some of my own
I think the rapid rise in Factor VIII levels being a concern is not as big a deal as some people are making it out to be. At first I thought that and then one must consider there is A LOT of room to dose down with the incredible levels they achieved. What it may say is they still need to work on dosing. Since the mid (and low) doses seems to produce much lower levels so it would not appear to be an issue that can't be addressed. People also need to keep in mind this is just an early 7 high (9 total) patients its an exploratory study not the final registration!
yes there were still bleeding episodes. Look at slide 16 I think
http://files.shareholder.com/downloads/ABEA-3W276N/2477655309x0x901460/C2800939-0F80-4E18-9B69-30EFF1569B04/BMN_270_Data_Update.pdf
The episodes are down to 2 at weeks 9-28 when the Factor VIII levels are much higher than earlier time points AND see the asterisk all but 1 bleed from week 2-28 was in a single patient (the lowest responder)!
I am long BMRN so probably biased but I put the results in the incredible category! The levels are so much better than would be needed to be considered an effective treatment. I don't follow a lot of other Gene Therapies for other Hemophilia's but the few I've seen have much lower levels of production of the deficient factor.
There is still a question of durability and longer term safety.
What I wonder still about is can Gene Therapy be a commodity. It still appears to me there is no shortage of academic researchers and licenses seem to happen frequently. That being said I've been tempted to play QURE and ABEO in that event.
EDIT:
The thrombocytopenia was discussed (briefly) on the call. It is a concern high levels do increase risk but not dramatically and nothing being done at present and again dose may be optimized.
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