<<I thought they found reasonable efficacy with just a single dose.>>
Yes, they THINK that they indeed did found effective single dose.
<<In patients receiving Ad5-FGF4 (1010 v.p.), the mean increase in ETT time was 1.5 min at 4 weeks and 2.0 min at 12 weeks compared to 0.7 min and 1.0 min at 4 and 12 weeks, respectively, in the placebo group. Researchers also observed that patients with a shorter baseline ETT (£ 10 minutes) had a better response to active treatment than placebo.>>
However, it is bit early to translate PI/II data into pivotal medical success. If ETT baseline time is ~12 min, 2.0 min increase (1 min if adjusted for placebo) is ~15%, not that great at all.
I am more curious about relative data for 4, 12 and 24 weeks post dosing, in relation to ETT baseline time of ~12 min?
<<To identify appropriate Ad5-FGF4 doses to carry forward into larger clinical trials, researchers used, in addition to the assessment of safety, a dichotomous qualitative method setting a response level of ETT improvement of 20% at 4 weeks and 30% at 12 weeks after treatment (each about two times placebo). Based on this analysis, in patients treated with an Ad5-FGF4 dose of 1010 v.p., 50% improved at 4 weeks and 45% improved at 12 weeks compared to improvement in 16% and 21% in the placebo group and 42% and 36% in all patients treated with the active product. >>
While they did have better percentage improvement than placebo (Why placebo at all did have improvement? Selective pts???, than those in real world???) I do not think that they are medical significant. Reduce effect by placebo numbers and data are not of the magnitude that one would like to see.
For instance 50% improvement for ETT of 6 min is 3 min, impressive result. But is this real situation in world that we live in?
I am not cardiologist and probably can’t judge data as professional. I would like to read good report on this issue.
Regards the antibody reaction (which is standard thing for AA vector), the point is can antibody neutralize gene transfer (and damage site insertion because of the non-selectivity) before angiogenic effect materialize?
They claim: "Myocardial inflammation was not observed.", which is positive and indicate that there was no strong antibody reaction. They also do claim selective and strong vector uptake by cardiac cells. I like this if true.
As I said, I was impressed with COO position and confidence level. However, he didn't like Schering and their speed. Nothing new here, just how bios can be frustrated when pharma take over development compound.
Hope this help.
Miljenko
PS: <<Do they think they need multi-dose? >> NO! |
