Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) Q3 2023 Earnings Call Transcript

Unidentified Analyst: All right. Thank you.

Operator: Thank you. One moment for question. Our next question comes from Gena Wang with Barclays. You may proceed.

Unidentified Analyst: Thank you for taking my questions. Two very quick ones. The first one is regarding the Angelman program the update next year. I mean you say you also have 10 patients with a longer follow-up. Will you be able to share the Bayleys other domain? If I look through it, its social emotional and also adaptive behaviour and regarding the multi-domain responder index do you think that that could be a possible index that you could discuss with the FDA about possible approvable endpoint in the future? And quickly regarding Crysvita, so regarding in LATAM now the territory will be much more important for the revenue contribution. Any strategy you can think of can improve the penetration there?

Emil Kakkis: Okay. Well good questions. Maybe I’ll let– but when I get to it you can do the — I’ll let Eric do the Crysvita penetration strategies question. All right, I’ll start with first with the Angelman data. You asked about Bayley other domains of the Bayley. A lot of that means we’re not I think the adapter to social motional are not very well designed for Angelman patients. They’re designed for normal people. So I think they’re not very good. But we will have data on adaptive behaviour. We’ll unbind when we has adaptive behaviour, but we’ll also have another scale called ABC scale and we’ll probably be — I didn’t mention, but is it a barren behaviour scale which I think is maybe even more relevant to these patients which is what’s clinically important when you talk about social emotional or adaptive type things.

So we’ll have some things but I don’t think the Bayley is not the best test for some of those things. We are focusing on the ones where I think it’s sensitive. With regard to the — so that’s 10 patients that we might have longer-term data that is up to 254 longer and 20 that would have day 170. So that’s — we’re trying to give you a better sense. We’ll give you what we have on all of them. Now for MDRI, we believe MDRI is a good strategy. We don’t have FDA’s agreement on that. They’ve seen it. I presented to them including multiple senior leaders in the multiple conferences. We published a good paper on it. It’s a very powerful method. I think it’s a very appropriate method. The key thing with the MDRI is that you really measure endpoints you can agree on any ways endpoints that are meaningful, right?

The only thing the more is an analytical tool, how do you take five different endpoints and add up the results. And this says, look, I’m going to add up how many people responded in each of the end points to a clinically meaningful degree and add up those responses. So it’s not conceptually that hard you do get all the underlying data. So it’s not like FDA is giving up anything by using that endpoint, right? They’re actually getting all the underlying end points. So — if they can get comfort around the Bayley Scale for certain things and the ASA score the parent behavior score Filono others as representative of those functions then I think we can get them comfortable with MBRI as an analysis tool and how to get to the result. So, lastly, let’s talk about Crysvita.

Look, I think Crysvita should be majority of the piece in fact I personally think every single kid who has XLH should be on Crysvita and not oral phosphate if they need treatment. And we still have room to go there. And we’re working with our partners. So I thought maybe Eric maybe you can say anything we might be doing to try to help them maximize the penetration of Crysvita.

Erik Harris : Well, just to reiterate LatAm is doing great. It’s been very successful thus far. We’ll continue with our current commercial efforts. But really what’s going to continue the strong demand there is the — what’s really going to drive the growth there is as we continue to get formal reimbursement across the other countries, right? We’ve seen the significant uptake just recently in Brazil following the formal reimbursement public reimbursement as we work our way through reimbursement through the other countries. We’ll see continued growth in the Latin American region.

Emil Kakkis : Certainly, in countries that have started treating patients they would like what they see they start adding patients. So there is a drive for use of the drug. And hormone reimbursement is definitely one of the pieces that will help get us there. It certainly has picked up a lot since Brazil got approved. So we’re encouraged. I think there’s still more room to grow and Crysvita is still in the middle of launch. It’s not plateauing out it’s continuing to grow.

Unidentified Analyst: Thank you.

Operator: Thank you. One moment for questions. Our next question comes from Laura Chico with Wedbush.

Laura Chico : Good evening. Thanks very much for taking the question. I guess, I wanted to circle back on the potential anylogenics spinout. You showed some intriguing data at the R&D Day with respect to plant reduction in the 5x FAD models. Could you just remind us any data demonstrating effects on either anti-inflammatory or proinflammatory markers there following treatment with the product. And just out of curiosity will there be any further data updates expected prior to the spin out? It sounds like things are moving along pretty quickly. Thanks so much.

Emil Kakkis : Yes. So we focus mostly on the pharmacology of plaque rather than secondary markers of inflammation et cetera. So I don’t have more to offer you at this point. The one challenge I would say is, when you’re doing the way you do it in the mouse we’re injecting directly in the brain there is an effect of putting a needle in the brain how that affects things. To get to those models, I think you need to be doing real intrathecal treatment. And maybe in the rat model where you can do that you can kind of look at those markers. But if you have to inject through the brain, I think it’s — it makes it a little more complicated to get to look at those aspects of the neurologic disease. We won’t likely put out more data until the spinout occurs.