Ascendis Pharma A/S at Goldman Sachs Global Healthcare Conference Transcript

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

Question: Paul Choi - Goldman Sachs - Analyst : Great. Thank you. Maybe we'll kick it off with what's top of mind with investors and just sort of what happened with the recent PDUFA extension. Can you maybe walk us through the mechanics as to why the FDA considered it major amendment and pushed off the PDUFA to August? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. JUNE 11, 2024 / 12:00PM, ASND.OQ - Ascendis Pharma A/S at Goldman Sachs Global Healthcare Conference Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board It's always an element in any review process that FDA always can decide what they really want to do. We can always speculate, we can always think about it, but I believe -- we believe, as I said that we have a clear pathway. We hope we will get the approvals here in August. And this product opportunity, it's really have proven the benefit when we see it not only in our Phase 2 trial, not only in our Phase 3 trial, we're also seeing it in our EAP program in the US, where we're basically taking more and more patient in every week. We also have seen the benefit of that in our launch in Germany, where we really see the penetration and all the benefit to the product. So I have no doubt that people in the US also should -- with hypoparathyroidism also should get the benefit for that and that is what we're aiming for.

Question: Paul Choi - Goldman Sachs - Analyst : Okay, great. Maybe touching on something you brought up with regard to the EAP. I guess just in terms of sizing and you've talked about patients joining on a weekly basis, where do you think in terms of the sizing of the EAP it might ultimately end up roughly between now and the PDUFA potentially? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah. The US EAP program is very much restricted to a subpopulation of adult patient with hypoparathyroidism. Because you basic will be -- only can be elected in our EAP program if you have previous being treated with PTH therapy. And at the same time, it's a restricted not to every physician, it's restricting to you cite that basic are going to do all the different and necessary contract were to be part of start-up program. So therefore, it's only a subpopulation of the subpopulation we can enroll. We are extremely enthusiastic about what we're seeing of this program. We are extremely enthusiastic to see how we really are providing benefit to this patient group too.

Question: Paul Choi - Goldman Sachs - Analyst : Okay. Bring up something interesting, which is, as people size up the market opportunity, people just think about how many patients are NATPARA, which is relatively small. But you've talked about how patient's adherence here on TransCon PTH has been quite durable and extended and so essentially becomes continuous therapy. So at least as we think about it, seems like the market sizing and opportunity here could be substantial far and above beyond what the NATPARA sales are even at a peak. And so maybe first, how do you and the team internally think about sizing up the PTH opportunity? And it's about the switch opportunity versus the de novo patient opportunity? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah, there is some indication we are getting approved. First is adult growth hormone. Adult hypoparathyroidism, and this is a patient population that really contain two major groups. One coming with a genetic background or hematolotical background and typical will do to either the pediatric phase and when they go off to adult, the identified with the disease. The second part is post-surgery, and this is a population when you go in, typically, to get a head and neck operation. You have an incident of actually ending up with hypo peril because the four different claims is really hard to avoid not to damage. So many cases I have seen frequency down to best cases, 1% to 2%, that patient that go to sites in operation will have chronic hypoparathyroidism. Some cases, I have seen up to 8% to 10% dependent on the speciality, how aggressive they are in the operation. We see a big demographic difference in the number of patients post-surgical. For example, in Germany, that is about 72,000 patients with diagnosed chronic hypopara. And when you go to the US., our best number is between 80,000 to 100,000. And when you look at that demographic between these two regions, you will say something wrong. No. But because if you take the correlation to the number of head and neck operation, it's fit there well. So we see the same pattern in Germany, in Austria, we see it in South Korea. We see it in other countries where there is a different way of treatment. So when we went into our Phase 2 trial and Phase 3 trial, we saw all patient benefit for this treatment. There was no a subpopulation of a hypopara patient that don't have any benefit. All of them have the benefit provided. When we then talk about a target population of 20,000 in Germany is we take the patient group that really are a big burden for the society meeting that -- for example, are coming to a later stage renal diseases that got diagnosed with renal diseases that often go to emergency room because they have high level of cold, that have to manage the quality of life in a normal manner. This is what we call the initial target population. We still believe every patient with chronic hypoparathyroidism will benefit from it. And this is where, I believe, we will see different penetration in different countries where there will be much more openness to take this kind of treatment into a higher population of the patients compared to other places where you only present to see 10% to 20% of the patients being treated and had nothing to do if there is not a treatment benefit, but it's basic is what we call the health economic cost for the society to supported. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. JUNE 11, 2024 / 12:00PM, ASND.OQ - Ascendis Pharma A/S at Goldman Sachs Global Healthcare Conference

Question: Paul Choi - Goldman Sachs - Analyst : Okay.

Question: Paul Choi - Goldman Sachs - Analyst : Okay, great. And Scott, maybe one more for you, you can just remind us what is embedded in the revenue guidance, the mix of SKYTROFA versus and does it includes international sales for YORVIPATH?

Question: Paul Choi - Goldman Sachs - Analyst : Okay.

Question: Paul Choi - Goldman Sachs - Analyst : Okay, great. Question from the audience? Hi. There we go. So my understanding of the FDA review process is that in order for something to be classified as a major amendment, it needs to be submitted within three months prior to the PDUFA date and it's typically either new clinical data or new analyses of the clinical data which necessitates a three-month extension versus manufacturing data, which typically necessitates a two-month expansion. So can you comment on the information requests or the type of information request with regard to which you submitted the response? And then the second question pertains to comparison between NATPARA and TransCon PTH as far as asset utilization rates are concerned that may or may not contribute to a inconsistent delivery of the product. Thank you.

Question: Paul Choi - Goldman Sachs - Analyst : Okay. Maybe turning to the commercial side on SKYTROFA here. Your commercial progress to date on the launch has been solid and you've reached significant dollar share here in the US market. Maybe you can comment on how you think about the trajectory over the intermediate to longer term. And does growth primarily come from market expansion, further share gains, some mix of the both? And how do you sort of think about the longer-term sales growth trajectory for SKYTROFA here?

Question: Paul Choi - Goldman Sachs - Analyst : Okay. You've talked about some interesting expansion opportunities, starting with adult growth hormone deficiency and also Turner's down the road here. And so maybe one question is, I think the IMS data suggests that the growth hormone market is about $3 billion give or take based on sales data, three quarters of which is currently still branded sales approximately. So with regard to the adult growth Hormone opportunity and Turners, can you maybe size up those opportunities there? And maybe as you sort of do your market research, what are some of the potential challenges of developing and growing those particular markets? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Going back to the adult growth hormone market, it's a bit different compared to the pediatric market. And 90% of the growth hormone market is the pediatric product. So 10% is basically adult. And to our best knowledge, we believe that the penetration is between 5% to 10%. Meaning is that adds a huge opportunity still to grow this segment. And I believe one of way we can grow this segment is really proving now with our data, how we really have an improvement in not only the primary endpoint because with primary endpoint is basically not the only reason why we treat the patient. You treat them because they're missing an essential hormone -- growth hormone and you see a positive endocrine effect on multiple organs, it's not only what we call the body composition, this is cardiovascular effect, it's on bone, it's on cognitive effect and et cetera, et cetera. And that is what we really want to indicate on. And having this weekly treatment will, I believe, someway remove one of the limitations on why there only 5% to 10% penetration in this segment here.

Question: Paul Choi - Goldman Sachs - Analyst : Okay. Right. You'll have your Phase 3 data from your pivotal trial for CNP also before year end, so I guess the question here is with a product approved on the market, are you assuming a regular review or potentially a accelerated or faster review with the US FDA? And then can you maybe just remind us what -- how you're thinking about the regulatory timing for European approval? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board I think it's a little bit too early to speculate about what pathway we're getting because I'd like to see the data and the data is always providing you the right pathway. We got accelerated approval for our TransCon PTH. When I look on the element of CNP, we know that the primary endpoint is analyzed height velocity and we have a good feeling where we will end. What we really are looking at is the co-morbidities, which we have built in in a lot of secondary endpoint and we're really looking forward to really coming out and show how we can address this co-morbidity because I believe this is the aim of this treatment. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. JUNE 11, 2024 / 12:00PM, ASND.OQ - Ascendis Pharma A/S at Goldman Sachs Global Healthcare Conference

Question: Paul Choi - Goldman Sachs - Analyst : Okay. Right. We have a few minutes remaining. So I wanted to just briefly touch on oncology updates that might be coming in the next year or two since that often doesn't get as much attention. Can you maybe just remind us in terms of what IL-2 and TLR updates we'll get this year and set the appropriate medical meeting at which we might expect those updates? Jan Moller Mikkelsen - Ascendis Pharma A/S - President, Chief Executive Officer, Executive Director, Member of the Executive Board Yeah, we had our first already here in ASCO where we actually came out and illustrate how for first time there is an IL-2 treatment that both have the right safety, the right efficacy and the right duration. Our first time you have seen the team at three (technical difficulty) you basically have dose stacking in immunological system. We see for first time a clear response (technical difficulty) effect we see it now in what we called well defined subpopulation. We are enrolling about 20 to 30 patients in each single of this subpopulation. We are enrolling them with different speed, and I believe end of '24 will be the year of Ascendis oncology, where you will start the first well defined subpopulation with the patient being treated in this way. And I just (technical difficulty) I really end of the year.