Emil Kakkis: So with setrusumab, we haven’t set precise criteria. We really want to look at the gradations of drug concentration and drug effect as we go along. But to give you a rough idea, if there were — if doubling the dose gave you 10% more bone production. I don’t think it’s worth it. But if it gave you 50% more bone production, then I would say that’s worth it. So if that gives you kind of a rough idea I think that would be what we would be looking for is something more meaningful to warrant spending and giving that much more drug. With regard to Evkeeza, we’ve had tremendously positive feedback from doctors on it, both in Europe and we’ve had a few compassionate use requests and patients treated. And I think that we’re excited about potential in really the first really good drug that doesn’t depend on the LDL receptor at all and can be given just as a monthly IV and — so if we can get it reimbursed and get access out there, I think we will see a strong uptick because there is great interest in it.
And part of the reason we decided to partner with Regeneron on the program in the first place.
Operator: Our next question comes from the line of Salveen Richter from Goldman Sachs.
Unidentified Analyst: This is Tommy on for Salveen. So for the UX053 data coming this year, can you remind us what some of the details here are in terms of duration, number of patients and any bars or benchmarks where you would see a positive signal?
Emil Kakkis: Certainly, the UX053 program is an mRNA LNP that’s producing this enzyme that will help restore glycogen metabolism. We’ll be looking for changes in biomarkers and glucose release and biomarkers of glucose fragments that are accumulating as well as safety branders around liver or immune response, etcetera. So it will be primarily a biochemical kind of look at both glucose control as well as release of glycogen fragments. So those two things should help us get an idea about dose. These are all single-dose patients that were escalated at single-dose ascending dose kind of cohort. So it will be a first indication of what we can achieve in terms of metabolic correction based on — primarily biochemical misers.
Operator: And our next question comes from the line of Maury Raycroft from Jefferies.
Maury Raycroft: I was wondering for the setrusumab Phase II update, can you clarify if you’ll have all patients at each of the two doses and the age range is five to 25. But are you breaking out what proportion are older versus younger?
Emil Kakkis: Yes. So for — what we’ll have is for those 24 patients will have all their data through at least two months, including the last patients in. For some of the patients that were started last year will have maybe six months of data, which will include BMDs that were done at interim time points. So that will be kind of a broad set of data. We will look at different age groups, and we haven’t decided how much we need to disclose. We’ll have — we have ages that are distributed around the age range that will allow us to look at different ages and use both PK and the biomarker modeling to try to determine whether we need to increase the drug exposure at the lower doses.
Operator: Our next question comes from the line of Dae Gon Ha from Stifel.
Dae Gon Ha: Maybe a question on GTX-102. Can you maybe provide any updates on the scoliosis patient? Has that patient resolved fully to continue receiving the dose? And any update to the time line of FDA submission of the package for the protocol harmonization?
