Ascendis Pharma A/S Business Update Call Transcript

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

Question: Jessica Fye - JPMorgan - Analyst : Hey, guys. Good morning and congrats on these results. I have one question with two parts. First, I'm curious about the change from baseline AGV for the overall population. Can we just calculate that using the numbers on slide 10 to get an overall change in growth rate for TransCon and for placebo? And then the second part related to this, when we look at slide 11 or the change from baseline ATV for ages five to 11, which I assume would be most comparable to Biomarin's primary endpoint and age group, it looks like the placebo arm here had a positive change in their growth rate, whereas in the Volkswagen pivotal data, the placebo arms growth rate actually decreased during the study period. So I'm curious if you could provide the baseline and on-study AGV inputs for this 5- to 11 age groups so that we can better understand that cross-trial comparison? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. I think I can take the last question, and I think Amy will help me a lot here with all the different numbers on there, early morning. Yes, we tried to do the analysis on slide 11 for the 5- to 11 years to develop the best process for comparison using the same kind of algorithms that really are utilized before. We always know, it's interim trials comparison but we believe that is pretty valid. Why do we also believe it's valid? When I go over and look at all data looking at the 5- to 11 years on height Z-scores, and I actually see exactly the same pattern. So from their perspective, I feel really that is representing and really strong growth in this patient group compared to the rest. Amy, do you want to go into more specific data? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 16, 2024 / 12:00PM, ASND.OQ - Ascendis Pharma A/S Business Update Call

Question: Tazeen Ahmad - Bank of America Merrill Lynch - Analyst : Hi, guys, good morning. Thanks for taking my question. I just wanted to get a sense for how you're thinking about the best population for the drug by age. It did look like kids younger than five. I had laughter, height velocity improvement or slower improvement relative to the older kids? How should we interpret that? Was that expected as part of the study when you designed it? And how should we think about what a label can look like as it relates to age? And then what else do you need to complete in order to get your application together by the first quarter of '25? Thanks. Thanks, Tazeen. I think there's two different way you can look at it. If you look at height, SDS, your basic match, each subject to gender to age. And when we looked at that, we actually see the same increase in height, SDS. So from that perspective, it's really consistent. When you look on annualized height-velocity, it is a little bit different because when you see the age group between two and five, you have an [NABT] acceleration of annualized height velocity. So therefore, you basically have something that going one way. And then we promote growth in the other way. So therefore, the absolute difference in annualized height velocity will be different. But the real way to compare the growth promoting effect on our drug is look on height is between the two-patient subject group and there, your basic see same effect. The last question, we are finalizing all the documentation for US application and its basic is finalizing also the rest of the buyer and a [medical] testing we have done. For example, we are finalizing all the immunogenicity analysis. We know from our Phase 2 data long term, over many, many years, we never have seen really any inorganic development. So but all these elements need to be finalized, and then we are writing. Writing to get this in as fast as possible to ensure we can come out to the market as fast as possible.

Question: Joseph Schwartz - Leerink Partners - Analyst : Great! Congrats on the strong data. So just to follow on the last question. My first question is I'm just wondering if you can give us a sense of your confidence that the review process can advance without a hitch, given the delays which we're seeing in the review for TransCon PTH. Are you taking any particular measures to ensure that you don't experience any such delays again, based on anything that was learned from the last review at the FDA? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Joe, if you look on my agenda, I can guarantee that is one of the thing I really are integrated in the organization. What is the learning from PTH? How can we ensure that we never ending in the same situation, which I don't want able to do in my life. Having a unique product, but some way be stuck in a process. First of all, TransCon CNP is much more simple in a way that is not really being dose titrated. So just from the basic question about dose overlap, it's really hard to imagine when you only have one single dose. So this simple way to look at it is that we will be in an extremely constructive dialogue with the FDA, which we always are. We are already discussing exactly how we are ensuring our CNP section are living up to the latest new requirement from FDA to ensure that we can get the possibility to take it out as fast as possible here in the US.

Question: Joseph Schwartz - Leerink Partners - Analyst : Thanks for taking the question.

Question: Derek Archila - Wells Fargo - Analyst : Hey, good morning and congrats on the data. Just one from us. I was wondering if you could discuss your alignment with the FDA on the regulatory strategy for TransCon CNP. I guess how would it compare to the BioMarin's experience with Seretide and the need for two year data? Thanks. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah, first of all, we have what I call a standard way to look on all primary endpoints that have been utilized in, I think, 20-, 30 years in growth disorder. Looking on change in annualized height velocity for one. It's to cut out to my knowledge, Amy confirms that the impact on linear growth has been really big evaluated in clinical trials with one of the groups. With our interaction not only with the US regulatory agencies. They also account all our record to agencies like EMEA, Japan and a few other places. We never have any kind of request from more than one. Obvious. As you know, we have a lot of supportive data from our long-term trials. I think Amy, do we have three -- four years now?

Question: Derek Archila - Wells Fargo - Analyst : Thank you. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 16, 2024 / 12:00PM, ASND.OQ - Ascendis Pharma A/S Business Update Call

Question: Gavin Gartner - Evercore - Analyst : Hey, guys. Thanks for taking the questions. First, I just wanted to ask on your broader development plans for CNP. So I believe you have an incident study and the schedule of a combo study that's ongoing. But I specifically wanted to ask about hypo achondroplasia and other indications. And I have a follow-up. Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Thanks so much. You know, you are asking a question which we are doing a lot of [irration] of. And Amy, she can help me a little bit because as I said, we want to be the leader in growth disorder and we can be the leader of growth disorder. Because when we just look in an area like hypo achondroplasia, it have a lot of heterogenicity. There's some part of achondroplasia, which are not really affected by high levels of co-morbidities and are affected with low level of co-morbidities. So what we are trying now to potential segregate that out and saying some of them would be best treated with growth hormone and the other one will have the most beneficial, potentially a combination of growth hormone and CNP. But Amy, you you're sitting with all this analysis and trying to build in this kind of visionary thinking in all the growth disorders.

Question: Gavin Gartner - Evercore - Analyst : Great. Very helpful. Just a quick follow up then on the growth hormone Coach combination study as we're looking out to that data next year, what should our expectations be? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board You know, I think that is when I saw this growth data and I say, do we still need that trial? This is the question I asked myself. Because when I look at the data, if you imitate [Sweden] in the first year or two of life, you will never need to have the combination trial. So the combination trial beating to the patient of subjects that feel I didn't start sufficient fast enough any treatment with our TransCon CNP. And therefore from that perspective is I need to have extra cash appraoch. There the combination can help. And this is what we are looking forward to prove later in the year. We basically either enrolling all the patient. It takes us excellent -- we have some excellent clinical operation that really talk with all the site every day and really knew the patients and that everyone so we can get this enrolled in. I think it takes towards 6-, 7 weeks when we finally got all the regulatory approvals. This is how fast our trials get rolled in. Just see the time spent for our pivotal trial here. It was the shortest pivotal trial I have ever been part of. So, yes, you're right. It will be for patients. That basic has not got imitated and CNP treatment when in the first years of life.

Question: David Lebovitz - Citi - Analyst : Thank you very much for taking my question. From a practical speaking, when actually launching, could you run us through the decision making process with respect to treatment experience, patients versus treatment naive patients and how easy it's going to be to transition patients who might actually already be on Box logo given their label starts at this time at a younger age? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Typical. We see elements that could -- I think you need to separate the geographic different way because if you just think US, it's some complete special ecosystem in how you basic have patient treatment. If we go to a other geographic region, if there is an improved treatment and you buy discussion with the national reimbursement system, then any decision always take the best treatment because when you are into the ecosystem where then approved, reimbursed, full commercial product, that's no restriction and it will compound your [presenting]. When you come to the US, on a long-term basis, every patient's basically always get treated with the best-in-class product that penetration can some way be delayed in places in the US as we see that cultural trend where there is a lot of restriction in the payer system to get the best possible treatment because of different revenue recognition for the digital build-out in the system. But I'm convinced that always every patient should get the best and we will work in a positive manner to that to happen. To ensure every learning we did it from scratch over can be integrated in our commercial strategies for our time to consume.