Ascendis Pharma A/S at JPMorgan Healthcare Conference Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Jess Fye - JPMorgan - Analyst : Great. So I guess first question for me is, you gave us that initial stat on YORVIPATH having 324 patients enrolled in the US in the special -- in the ASAP program or with specialty pharmacies. What does that mean? Do those patients have reimbursement? Is the reimbursement still in process? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board So when we take patient in, we take them into our ASAP program, which is our internal hub that basically do everything for the patient or they go with the prescription to the SP. That meaning is that they are starting the reimbursement process. And our way we look on reimbursement is that we expect the vast majority of the patient will receive the level of reimbursement between four and eight weeks. That is the time it takes. Some of them already came after one week, some came after two weeks. But when we look back and see what we have received on other product, the vast majority is between four and eight weeks from the prescription is being written.

Question: Jess Fye - JPMorgan - Analyst : Okay. And then I think the numbers you gave on those initial kind of patient enrollments also suggest that there may be like 50 or 60 YORVIPATH patients from clinical trials or the EAP who have yet to transition. Should we expect those? And if so, when? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board It will come in the first -- next months, we believe. There is about 60, 80 patients that still need to be transferred that also need to be transferred that currently is on YORVIPATH. It's something that just have been running for three or four weeks. So it's really pretty impressive because they need to come into the physician. And typically, what we learn, the big group of patients, the 40,000 patients, when you look on the status being controlled or in a severe or moderate manner, they are seeing the physician about up to four, sometimes more, some of them seeing at least two to three times a year. And we ask the physician, are you starting to stockpiling the patient? They're saying, no, we're not doing that. We wait until they come in because the waiting line just to get an appointment is basically about 8 to 12 weeks. So they're just waiting to have already their regular appointment with the endos and then they'll meet and get the prescription.

Question: Jess Fye - JPMorgan - Analyst : What about ex-US? So you had a really robust rate of patient pickup in Europe already. I'm curious how you think about kind of volume for '25 from this level and also price. What's the right way to think about kind of the evolution of the price in Europe? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. So what we have now, and just give you the background for people that not have so much common knowledge about the European price structure, the key country in what we call outside US as a reference price is Germany. So Germany is a key country that will be taken as a reference for price. And today, we have the EUR105,000 on a list pricing. You have a few percentage, what we call mandatory rebate, in the German market. What we expect to do is, in the middle of the year, to finalize the final price in Germany. And it will be some way you can say the reference price from NATPARA was around EUR80,000, a little bit larger today if you incorporate inflation and other things like that. So it will be between what we call the list price of NATPARA and to the EUR105,000 price. And that will be the final price. We don't need to go to further price negotiation because we are not launching YORVIPATH as an orphan drug product. We take it in as a normal product, so there's only one price negotiation. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. JANUARY 13, 2025 / 4:15PM, ASND.OQ - Ascendis Pharma A/S at JPMorgan Healthcare Conference About the volume, it's really simple. Germany and Austria basically beat NATPARA for four years what they did in the same region. And so we don't have no benchmark more because NATPARA was only in the market for four years in Europe, but we beat that in 11 months. But it's just to illustrate the power of the product.

Question: Jess Fye - JPMorgan - Analyst : Got it. You've got a competitor data readout coming from Eneboparatide at some point, maybe over the next few months, who knows. What are you going to be watching for when we see that clinical data? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board I think, first of all, you know that, yes, I like to go back to the science. How did this product got designed? It got designed out from a fusion peptide between PTH and PTH-related hormone. It's a compound that's very much alike Tymlos but have further mutation in it. Meaning is that it's from a mode of action -- it has not what we call itself a long half-life to give a continuous exposure. It locks one of the mode of action, only one of the mode of actions, and that is the element of intracellular activation in the lysozyme system. It locks there, and therefore, it have a continuous exposure. First of all, let us look at the molecule itself. It got designed as a PTHR1 receptor analogue. We know the benefit we see in all of the patients, mainly for cognitive effect and other things, are to the PTHR2 receptor that's sitting in the brain. And we therefore believe it can never be a replacement therapy because it basically is a hormone that only activates one of the receptors. The other element is that when we look on the data itself, it has not the positive effect on phosphate because it can't do that because it have a different mode of action. It will have a different effect on bones. And I actually think it was best illustrated by a physician that asked the company, when you look on the mode of action of this compound, you will note that hypoparathyroidism is a disease that affect multiple, multiple organs with a lot of different receptors. How can you really show us that you have the right activation on all the different organs in the right manner? And the physician for the trial said, we cannot ever show that. So it's never going to be a replacement therapy. The other element is that when you look on the molecule, when I look at it from the protein side, if you look on the Tymlos of 35% to 40% had neutralizing antibodies after 18 months, but it's not a big issue for Tymlos because it's restrictive to two years treatment. So there's a lot of other things. So when I really look on this compound, I really don't believe that it's -- it's hard for me to see it would be better than YORVIPATH. It would be an approvable product. We need to look at the data.

Question: Jess Fye - JPMorgan - Analyst : Maybe switching to SKYTROFA. When I look at the IQVIA data, and I know it's probably not perfect, it seems like the conversion from dailies to the long-acting category has sort of slowed recently. What can make that pick up again? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board That is why we have such a strong commercial organization. We have Jay now heading up our commercial organization as a president of that. And we have such a strong team, established people in the commercial organization. I'm quite sure that I can get this going again as we did other times. What we did for between '24 and '25, we really didn't really change what we call the element of our contracting. So when we really are building up more demand, we will see much more revenue. And that is our strong focus.