Douglas Ingram: Goodness, I am so sorry, Rye. I heard the beginning, but I don’t hear the question itself. I’m very apologetic.
Unidentified Company Representative: I think he asked if it included data on microdystrophin expression.
Douglas Ingram: Oh, well, I imagine that we’ll have that data available during the review process with the FDA. As Louise said, the expression we’re seeing is in the hunted range of what we’d normally seen. So there’s nothing unusual about it. In fact, the P value on it is .0, many zeros. It’s very strongly, robustly, as you would expect, the limit is robustly mixed. That’s the question, though.
Unidentified Analyst: That helps. Thank you.
Douglas Ingram: Thank you so much, Rye. Apologies, I couldn’t hear you. I’m sorry about that.
Operator: Please stand by for the next question. The next question comes from Brian Skorney with Baird. Your line is open.
Brian Skorney: Good afternoon. Thanks for taking the question. I guess it wasn’t something we looked at very closely before, but I’m starting to tell, I think, time to rise had a non-significant difference in favor and treatment, but not that different in terms of magnitude. I think maybe just under half a second difference. You can tell me if that’s right or not. So I know you’ve talked extensively about the baseline imbalance issue here, and it seems particularly acute in the case of baseline time to rise, where the active arm was 5.1 at baseline and placebo was 3.6. So I was just wondering if you’ve gone back and looked at making adjustments for baseline imbalances to evaluate the time to rise differences in study 102, and in particular, if the four to five-year-old subgroup looks the same in 102 as it did in EMBARK.
Douglas Ingram: Yes, thank you for that question, Brian. I’ll turn this to Louise to respond.
Louise Rodino-Klapac: I’m going to speak generally, because what we did was take the inclusion criteria that we’ve generated for EMBARK and applied it to our previous data when we compared it to the external control. And what we found there is a difference when you exclude those patients that would have been excluded by that criteria in 102, where you had those rapid decliners. So in that case, we saw a more significant difference, but the specific numbers are escaping back right now. But we did do that analysis where we kind of applied the same exclusion criteria and did see a difference.
Brian Skorney: Great. Thank you.
Douglas Ingram: Yes. And maybe just one thing to add in that analysis to Louise’s point, we saw a really good consistency between what we observed in 102 and what we observed in 301.
Operator: Please stand by for the next question. The next question comes from Salveen Richter with Goldman Sachs. Your line is open.
Salveen Richter: Good afternoon. Thanks for taking my question. With regard to the regulatory submission, are there formal or regulatory procedures involved with revisiting an accelerated approval after the primary endpoint fails and in a confirmatory trial here? And then just any preliminary feedback on your data from payers and how that might impact the existing label or from physicians with regard to how they think about use in patients here?
Douglas Ingram: Again, on the first question, I’m not a 100% confident I understand the question. Let me be very clear, the standard for confirmation of an accelerated approval is looking at the totality of evidence and determining whether the benefits of that therapy have been confirmed by the entire data set, not just the confirmatory data, but all of the surrounding evidence that would exist, including other studies. And I would strongly argue that not only EMBARK, but all of the supporting evidence as well, has strongly confirmed the benefits of this therapy. So I think we’re in very good shape there. The focus of our review with this division is going to be on the breadth of the expansion of this label. That, I am quite confident, is going to be the review focus.
And as relates to that, as you know, our strong view is that having confirmed these results, having confirmed them across patients and looking at the totality of these evidence and looking at the forest plot as an example, and looking at the statistical analysis of the forest plot adjusted for multiplicities, it is quite clear that this therapy is arresting the decline in these patients and deserves to be made available to the patients without limitation to age or artificial restrictions around angulation. As it relates to payers, this is additional evidence in our armamentarium with payers. Things have gone very well. Dallan and his team, Medical Affairs, our commercial, our field force, access to reimbursement and the like have just done a fabulous job supporting the launch of the ELEVIDYS.
And I hope everyone will agree with me that it shows in our performance this quarter and this bolsters the discussion that the team can have. Now we can give as an example. We now have a really powerful metric that it’s compelling on the speed with which one you put a kit on therapy. So as I will remind you on the time to rise, not only is the P value, I think, 0.002, if I am not mistaken — but in time to rise is the single greatest prognosticator of loss of ambulation and a rise time above 5 seconds, as we’ve talked about often and is in the literature robustly is the single greatest predictor of early loss of ambulation. And EMBARK has shown that using ELEVIDYS reduces the odds of that occurring in a 52-week period by over 90%. So this provides an additional compelling point with payers who, frankly so far have done a really good job of providing access.
This provides additional evidence that it really is important to get kids on this therapy as soon as possible. And I would argue looking forward to label expansion and is a compelling argument for why this therapy should be — this label should be expanded as soon as possible as well. So everyone has access to it as well.
Operator: Please standby for the next question. The next question comes from Uy Ear with Mizuho. Your line is open.
Uy Ear: Hey guys thanks for taking the question. Congrats on the great quarter for ELEVIDYS. So I guess my first question is, were those — the patients with dose were they primarily those who were pretty much anticipated the approval the accelerated approval, and they get them more like the bolus. And like how many would you be able to share how many patients are waiting to be dosed in the coming quarter? And just continue on this theme. Now that the EMBARK data has read out, do you have any sense of any shift in patients receptivity to the product at all? I know its early days. Thanks.
