Matthew Klein: Kristen, thank you very much for the question. So as I mentioned in our prepared comments, in the previous procedure, there were two opinions. There’s actually two procedures in one. One was the conversion of the conditional marketing authorization to full marketing authorization. And the other was continuation of the conditional authorization. Through nuances of the procedural elements in Europe, they had brought that together and said, we’re going to issue two opinions that really get together. So, when they went negative on the conversion to full authorization, they elected to go negative as well for the renewal of the condition. Then they gave us the option to say, do you want to pursue one or both? Look, our priority here is to make sure this drug stays on the market, stays available for the boys in Europe, when there’s no other targeted therapy for nonsense mutation available.
And we believe that best chance of doing that is to focus solely on maintenance of the conditional authorization for now. We also believe in a way that by not asking for a conversion to a full approval, that the issues — some of the issues with having a negative primary analysis population analysis in Study 041 may become less of an issue. Really, you can focus on the other important and strong data in Study 041, including in the ITT population, where we have clear evidence of benefit over a number of different endpoints, as well as obviously the long-term STRIDE data. So we really feel that by focusing this question on just renewal of the conditional authorization, that we can provide the necessary evidence that can get this opinion from negative to positive.
Now, as you mentioned, what happens from — as you asked, what happens from there? Well, if you have a conditional marketing authorization in Europe, you are required to conduct or collect additional evidence to continue to support the benefit. Now, we believe there’s many potential sources of this, including continuing to collect data and STRIDE. We’ve obviously been able to show clear benefit in delaying time to loss of ambulation by 3.5 years in the most recent analysis and continue to show meaningful multi-year delay in loss of pulmonary function. As we can continue to collect more data over time, there’ll be even more patients informing the loss of ambulation analysis, even more patients informing the pulmonary function analysis and hopefully ultimately cardiac function and mortality.
So, there’s still a lot more meaningful data that can truly inform the long-term benefit of Translarna from STRIDE. There’s also the possibility to talk about bolstering STRIDE, either with additional analyses or in second registry, that’s a possibility. I think in the universe of possibilities, could they ask us to do another clinical trial? I think that’s possible, probably not likely, given a lot of the feedback that the scientific experts gave in the scientific advice portion of the procedure. Last time was that clinical studies for genetic-directed therapies in DMD are really hard and really difficult to produce meaningful data. The experts have told the agency they believe that these longer-term data collection mechanisms that are really most useful for understanding the true benefit.
So, that’s a long answer. The short answer is, look, we’ll have to collect additional data. It will be well-positioned and quite honestly, pleased to do so. The priority now is to keep this therapy on the market in Europe. And we believe by pursuing just the renewal of the conditional, puts us in the strongest position to do that.
Kristen Kluska: And just second part of that, I would imagine since you had the announcement that the community was probably pretty upset, especially the ones that have been on the therapy long-term. So wondering if there’s anything you can collect from them, anecdotes, stories, et cetera, and if they can be of any help ahead of this upcoming meeting and decision.
Matthew Klein: Yeah, Kristen, good question. I think needless to say, the physician and the patient community was surprised, disappointed, and quite honestly scared for the patients. Obviously, this is the only therapy they have that is directed for nonsense mutation patient. Many of them have been on the therapy for years and observed the benefits that we’ve been reporting. For physicians, they understand the context of the disease. They understand the strength of the data that we produce, and they can’t, as many have said, they can’t imagine having to take patients off a drug that’s safe and effective. The patients on their own have the ability on their own to reach out and let their voice be heard. I’m sure in many parts of Europe that will happen given the fear and concern they have.
And I think similarly for the physician community, they’ll want to speak on that. Our sense here is that most people, as we were, we never thought this date would come or this would be a reality. So I think this is a little bit for them, a call to speak up and let their voice be heard. And importantly, as you say, share their story, share their experience, particularly the physicians who are experts and can truly articulate the benefits they’ve observed and the importance of this therapy for patients with nonsense mutation DMD.
Operator: Thank you. One moment please. Our next question comes from the line of Sami Corwin of William Blair.
Samantha Corwin: On the topic of Translarna, I guess since the negative opinion, have you seen any change in how physicians are writing scripts or if patients are accumulating medication ahead of the potential that the opinion does not reverse? And then can you speak a little bit as to if you’ve increased your sales efforts in non-EU areas since the negative opinion as well?
Matthew Klein: Yeah, Sami, thank you for the question. Again, I think in the physicians we’ve spoken with, the first response was clarifying and you confirmed that in no way does things change. Patients can stay on the drug, they can write new prescriptions and obviously everyone’s going to do everything they can to make sure patients stay on therapies. But let me take it over to Eric and if you want give some more detail on prescriptions.
Eric Pauwels: First of all, we haven’t actually seen an impact since CHMP has issued their opinion. In fact, we’ve seen physicians who have actually been very — they’ve been very, very supportive. In fact, our customer-facing team has actually stepped up their interactions with healthcare providers, the advocacy groups and others to emphasize that the drug is actually available. And I think that’s one of the most important things. We’ve made specific calls to every prescribing physician and we’ve worked with them, because we’ve had relationships now for many, many years with many of these physicians. They know about the treatment, they’ve been treating patients for many years and we’ve been reaffirming that Translarna is not only just available, it’s available for existing patients, but it’s also, we’ve seen new prescriptions as well.
