Orchard Therapeutics plc (NASDAQ:ORTX) Q2 2023 Earnings Call Transcript

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Braden Parker: Yeah, thank you for that question. In terms of learnings from Europe and how they would apply to a U.S. Launch, I think there are very similar approaches in terms of the market, in terms of the size of the personnel, the number of qualified treatment centers that we would have. You look at the size of the market and applying an incidence rate, they’re pretty similar from that standpoint. I think the main differences that you see in the U.S. versus Europe is there will not be that lengthy period of time where you’re going through health technology assessments in advance of any type of pricing negotiations. The reimbursement will come much sooner in the U.S. than it has in Europe. So as you think about launch and the launch trajectory, if you know, the number of patients will likely be similar and the ramp will likely be similar, but those sales will happen much sooner than they did in Europe, so almost very soon post launch.

But we do anticipate, given the nature of MLD and the challenges in identifying patients within that treatment window of opportunity, that you will see kind of this uneven nature of patient ads and sales in the early years of the launch, as we’ve seen in Europe as well. It gets a little bit lumpy until you have universal newborn screening in place, which will provide much more predictability of patient identification within that treatment window of opportunity.

Brendan Smith: All right, great and then just one quick follow up. Sorry if I missed this. Can you just maybe remind us what’s actually left before initiating a pivotal study in MPS? One, I know you touched on this a bit, but it’s a matter of kind of finalizing design with FDA, or is it really just selecting and getting the sites up and running for the pivotal study?

Bobby Gaspar: Thanks. I’ll take that. The study design is final with the FDA, so we don’t have to do anything further as far as any kind of regulatory path is concerned in order to open the study. So now it is really just the kind of logistics and practicalities of getting sites ready for what will be a multicenter, randomized, controlled trial. So you can imagine the work that goes into that and it is just getting all of that in place and we anticipate opening the study by the end of the year.

Operator: Thank you. I’d now like to turn the call back over to Bobby Gaspar for any closing remarks.

Bobby Gaspar: Okay, thank you very much. And so I just want to. Say. Thank you very much for your for your questions and your time, your attention and your support as we work to realize Orchard’s vision to end the devastation caused by these severe genetic diseases through this platform approach which we think has a curative potential that of HSE gene therapy. So thank you very much.

Operator: Thank you. This concludes today’s conference call. Thank for participating. You may now disconnect.

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