Orchard Therapeutics plc (NASDAQ:ORTX) Q4 2022 Earnings Call Transcript

Pete Stavropoulos : And if I can just squeeze one more in. For MPS I, the 203 program, can you just give us a little bit more detail about the composite endpoint of complication-free survival?

Bobby Gaspar : Sure. Let me hand that over to Leslie, who’ll have more detail on that.

Leslie Meltzer : Sure. So this composite endpoint was designed specifically looking at outcomes that are clinically meaningful in MPS I and are particularly suboptimally served by the current standard of care, which for almost all patients is an allogeneic HSCT. So this includes outcomes associated with morbidity and mortality of transplants such as death, failure too in graft, requirement for a follow-up transplant, the need to reinitiate therapy with enzyme replacement and so forth. It also includes components associated with the clinical outcomes of MPS-I that aren’t as well served by HSCT, and that includes incidence of cognitive decline as well as incidence of growth impairment. And so again, we’d be directly comparing outcomes with OTL-203 against outcomes with standard of care and the study is designed to show superiority of OTL-203 relative to that standard of care.

Operator: We have now reached the end of the Q&A session. I will now hand the call back to Bobby.

Bobby Gaspar : Thank you. Thank you, everyone. Thank you for your time, your attention and your support on what I think is a very important day for Orchard as we continue to advance our important mission in bringing this HSC gene therapy to patients with severe devastating diseases. Thank you very much.