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Re: BMRN endpoints article Thank you for helping us all go over the data again. Sorry to belabor the point, but I believe at ASH 2021 they said only 2 of the 5 subjects had bleeds (assume #s 11 and 9, who had lower factor levels). If so, the ABR for the 3 with factor levels above 15 at 2 years would be 0, and the ABR for the other two with factor of <15 from years 1-2 be say 6 and 1, with a resulting average of 1.4. (These are hypothetical numbers for now, since I'm not sure they broke down who bled when.) In any event, continuing on with the point I was trying to make, in my mind, SGMO from the 5 P1/2 subjects (#s7-11) one could perhaps say that 80% had a good response, and 20% (1/5) did not. I think we need lots more data (N>50 vs N=5) to find this true "responder" level. I don't think the mean ABR is as meaningful, since it may be from only one or two subjects in a very small N. The better way to think about it, IMO, is that 60% had excellent outcome (0 bleeds, factor >15 at year 2), 20% had good outcome (~1 bleed/yr, factor of 11 at year 2), and 20% had poor outcome (~6 bleeds/yr, factor of 1 at year 2). and I was speaking more about the BMRN data, since you mentioned how they were so willing to share it. I was just mentioning the format I'd like to see for the data, which I think would be most meaningful to patients. For PFE/SGMO, if the majority of the bleeds were in 1/5 subjects, then mean ABR is not that meaningful. I'd rather see the % with a given factor level, or % with no bleeds, or % with a large reduction in ABR, or % not requiring infusions (none of the 5 Cohort 4 subjects had resumed infusions). That counts for something too. Finally, there was one Grade3 SAE infusion reaction that resolved. Updated P1/2 girfit data could be meaningful for duration of response, but I think the larger number of subjects from P3 trump all. There are different ways to interpret the data, and I don't think mean ABR is all that helpful, per numbers and explanation above. Unfortunately it will be a while until we see that final girfit P3 data with that long trial hold for too high of F8 levels and VTE SAE, and BMRN is likely to have a pretty good head start. However, uptake should not be that fast for valrox in that 1-2 year head start, as many will likely wait to see girfit data and how it compares, since they may only get one shot at it. It may take a while for insurance companies to figure out reimbursement as well. Valrox will likely blaze the trail and girfit will join them on the path relatively soon after. SGMO will obviously benefit greatly if this happens. |
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Msg # | Subject | Author | Recs | Date Posted |
168009 | Re: BMRN endpoints article | rynotheknife | 5 | 9/30/2022 5:48:41 PM |
168012 | Re: BMRN endpoints article | ZFP | 13 | 10/1/2022 9:40:35 AM |