Q – Tazeen Ahmad: Okay. Thank you.
Operator: Thank you. One moment for questions. Our next question comes from Kristen Kluska with Cantor Fitzgerald. You may proceed.
Kristen Kluska: Hi, everyone. Thanks for taking the question. Can you provide any more color or thoughts around the timing of the interim analysis of the OI study?
Emil Kakkis: Yes. The way the interim will be done, we won’t be disclosing when they’re happening. We have said that we’d expect a first one to happen this coming year. We want to make sure that patients had at least a certain amount of time of treatment before an interim would be done. And the original plan for the program is to operate off of the fracture information how many fractures were essentially event-driven timing. So the precise timing is we haven’t disclosed it depends a little on fracture numbers et cetera. But we said that one will occur next year, but we won’t disclose exactly when. It will happen in a controlled way unblinded assessment by the DMC. So we won’t know when it happens. So at this point, we’ll all be waiting.
But we’re encouraged by what we’re seeing and the potential of the study could end early. But, however, we feel we have a drug that works and we’re going to get through a positive Phase III at least we’re feeling very confident about the ability of the product to get us there.
Kristen Kluska: Thank you.
Operator: Thank you. One moment for questions. Our next question comes from Joon Lee with Truist Securities. You may proceed.
Unidentified Analyst: Hi, everyone. This is Maddy [ph] on for Joon. So on GTX-102 for the three patients who regain some lost clinical benefits after reducing, so how do you best plan to highlight this significant observation for regulatory agencies? And is it at all possible that time to read those could explain some of these clinical benefits? And I have a follow-up question.
Emil Kakkis: Well, first of all, let’s talk about what happened in those patients. Those patients got anywhere from one to four doses, five doses. And had their clinical benefit effect of newer emerging findings they have a safety event. And then they’re off treatment for — on the order of like two years in which they lost all this activity, so two years off they’re pretty much washed out completely. No drugs, no benefit. So when they start up again they’re really starting back from the beginning and regaining the same functions. And in terms of the patient, the one patient has really gained a lot of words who had gained nine to 12 words in the first period and now having been on consistent therapy for a longer period is actually up to 17 words.
That’s the patient also that’s now swimming as well on their own without float support. So we think that the washout period takes them back to the beginning. It’s not really 00 there’s no relationship between what happened there on redosing effect. If you look at what happened with naive dosing patients we showed a graph at Analyst Day that showed a large number of patients having similar first ever emerging skill the first timing they went on treatment. So we think there’s nothing actually special going over those three that’s any different from any of the naive treaties.
Unidentified Analyst: Thank you. The follow-up is on OI. So are there any specific reasons for preferential suitability of setrusumab on AI versus other anticlotting antibodies like Evenity SHR-1222 or the others?
Emil Kakkis: No. Well, the only other one is that they are romosozumab, romo, I guess we call it for short. Both target the same class and there are differences though. Remember our program setrusumab is a fully human antibody, which we think is a better choice for a long-term therapeutic because of the risk of anti-drug, antibodies will be much lower in a fully human antibody. And we haven’t seen them. So we’re — we feel ours is a better choice for a chronic therapy. We will have chronic dosing in our label. That is RS 10. The romo is only 12 months. We think OI patients need continuous — and we’ve shown when you pull them off drug as it was done in the Aster said, they love grown even with the phosphate on board whereas romo is basically given for a year and then lock supposedly the effects are locked in.
But with OI that’s not true. You need chronic dosing. So the chronic dosing story will be ours. And finally when we commercialize, we’re going to provide the first-rate patient support we do to our rare disease patients which is not something that’s going to happen for an osteoporosis drug. The last thing I’ll mention is that the presentation of that drug is a 210 milligrams in a prefilled range, it’s not very adaptive for different size patients and different amounts of drug dosing whereas our presentation will allow weight-based dosing for each patient and optimization which I think will be important in the ultimate care of these patients. So, Atascosa both can work, but I think ours will be a better choice for long-term use.
Unidentified Analyst: Thank you, very much. Thanks for taking our questions.
Operator: Thank you. One moment for questions. Our next question comes from Maury Raycroft with Jefferies. Your may proceed.
Maury Raycroft: Hi, thanks for taking my question. For 701 in Wilsons, what goes into the decision tree for decreasing standard of care? And what is the target reduction in Stage 1? And can you provide more perspective around patient baseline characteristics for the first two cohorts including baselines or oxidase activity?
Emil Kakkis: Yes. Let me provide a little top line. And then Eric, if you want to provide a little bit more detail. After they get the drug, there’s a period of time where we watch and see how they’re doing and then we start titrating down the standard of care. And the goal in the trial to get them off standard care completely, like the first two patients. The others I think are going to get there. I’ll let Erik talk a little bit about what the criteria are. In baseline, we haven’t put out all the information in great detail. But maybe Erik you can provide a little bit a high level on a few things to up with help with Maury’s question. So standard of care strategy and then the baseline.
Eric Crombez: Yes. Great. It’s nice that we’re able to do this in an open-label fashion that gives us the ability to work closely with these investigators. But the goal really is to give all patients a trial of titration off of current standard of care. And what’s nice about this disorder is you can monitor copper in a lot of very different ways and certainly with a very big focus on urinary copper, but we feel with all of the types of way we can measure copper, we really do have a good way to understand the effect of the gene therapy. Certainly, if any patient start seeing any signs and symptoms or biomarkers start trending in the wrong way. We will rescue them back to their original dose, of chelators and thing but we really haven’t seen the need to do that across the board or in a meaningful way.
The ceruloplasm-based activity assay is interesting and again important, because key leaders and zinc have no effect on that. So the only — so the only way for serubaplasin to exist is to have copper onto it. And with this assay specifically measuring the loading of copper and of rural plasm, these patients are coming in really with an activity below the lower limit of detection. So again, measuring the rise from there. So, still early days. Patients are definitely still increasing and benefiting from the transgene, but things seem to be really trending in the right direction for the great majority of these patients who have been dosed so far.
Maury Raycroft: Got it. That’s helpful. Thanks for taking my questions.
Operator: Thank you. One moment for questions. Our next question comes from Salveen Richter with Goldman Sachs. You may proceed.
Unidentified Analyst: Hi. This is Lydia [ph] on for Salveen. Thanks so much for taking our question. So just two on setrusumab. So first, could you just provide a bit more detail on what exactly would trigger an early Phase III readout? And then also, do you have any idea on pricing and reimbursement, given the availability of ifostinates and romo?
