Ascendis Pharma A/S
Q1 2019 Earnings Call Transcript

Published: 30 May 2019

[Operator:]

Good day, ladies and gentlemen, and welcome to the First Quarter 2019 Ascendis Pharma Earnings Conference Call. At this time, all participants are in a listen-only mode. Later we will conduct the question-and-answer session, and instructions will follow at that time. As a reminder, this call will be recorded. I would now like to introduce your host for today's conference, Mr. Scott Smith, Senior Vice President and Chief Financial Officer of Ascendis Pharma. You may begin.

[Scott Smith:]

Thank you, operator. Thank you everyone for joining our first quarter 2019 financial results conference call today. I'm Scott Smith, Chief Financial Officer of Ascendis. Joining me on today's call is Jan Mikkelsen, President and Chief Executive Officer; and Dr. Jonathan Leff, Chief Medical Officer.

[Jan Mikkelsen:]

Good afternoon, everyone, and thank you for joining us today. In my view 2019 has been the most transformative period in Ascendis history, as we validated our TransCon platform with clinical results from the Phase III heiGHt and fliGHt Trials for TransCon Growth Hormone. For TransCon Growth Hormone not only did the heiGHt Trial receive its primary objectives of non-inferiority compared with daily growth hormone in pediatric growth hormone deficiency, it also demonstrated superior efficacy with comparable safety and tolerability to our daily growth hormone therapy.

[Jonathan Leff:]

Thanks Jan. 2019 is off to a busy start with all of our clinical programs advancing nicely. As Jan described the Phase 3 top line results for heiGHt have strengthened our confidence in the future of the TransCon pipeline. I'll now provide an update on our three endocrinology clinical programs and the key milestones we are working to achieve in 2019. In March, we reported positive top line results for the pivotal Phase 3 heiGHt Trial, which demonstrated the once-weekly TransCon Growth Hormone at comfortable, safety and tolerability of daily Genotropin with a significantly greater increase in annualized heiGHt velocity over the one-year study period in treatment naïve children with growth hormone deficiency. Recently we had the opportunity to discuss our top line results in detail with investigators, following presentation of the results at three relevant conferences, Endo 2019 the Pediatric Endocrine Society and the Pediatric Endocrine Nurses Society. Feedback continues to be positive and we see increasing enthusiasm about the potential of once weekly TransCon Growth Hormone as a potential safe and effective alternative to daily hGH therapy. In addition to height, we recently announced preliminary data from our fliGHt or switch trial evaluating safety and tolerability of TransCon Growth Hormone in subjects to our previously treated with various commercially available daily therapies. Importantly, the trial included some subjects under three years of age, who had not been previously treated with growth hormone therapy.

[Scott Smith:]

Thank you, Jonathan. Turning to our financial results for the three months ended March 31 2019 let me review some highlights. For the first quarter we reported a net loss of €53.6 million or €1.24 million per basic and diluted share compared to a net loss of €41.4 million or €1.07 per basic and diluted share during the same period in 2018. The first quarter 2019 net loss includes an unrealized noncash gain of €3.1 million compared to an unrealized noncash loss of €7 million in the 2018 quarter due to foreign currency exchange rate fluctuations. Research and development costs for the first quarter were €51.3 million compared to €30.5 million during the same period in 2018. Higher R&D costs during the 2019 quarter reflect increased personnel and infrastructure costs due to growth in headcount. For TransCon Growth Hormone costs were higher primarily due to ongoing preparation of validation batches which were partially offset by lower clinical trial costs related to our Phase III program. For TransCon PTH costs were higher primarily due to manufacturing of TransCon PTH for use in our clinical trials and preparation for validation batches. New and ongoing costs associated with our Phase II clinical trial which were partially offset by lower preclinical costs. For TransCon CNP costs were lower primarily due to lower manufacturing costs and preclinical costs which were partially offset by higher clinical trial costs. As a reminder our R&D expenses including manufacturing related expenses vary from quarter-to-quarter reflecting timing of ongoing development activity. General and administrative expenses for the first quarter of 2019 were €10.4 million compared to €4.7 million during the 2018 period. These higher costs primarily reflect an increase in personnel and site costs as well as costs of building out commercial capabilities. We ended the first quarter with cash and cash equivalents of €696.7 million and 46927115 million ordinary shares outstanding which includes a net proceeds from the following of financing completed in March 2019 of approximately $539.4 million or approximately €480.3 million. All of our milestones we laid out in January remain on track. Furthermore with the success of the TransCon platform in the heiGHt and fliGHt trial in our recent financing, we are well positioned and capitalized deliver in our Vision 3x3 strategic road map including initiating plans to expand our product candidates into other endocrinology rare disease indications, advancing our objective of achieving global reach based on ongoing regulatory activities and discussions not only in the U.S. and Europe, but also in Japan, South Korea and through Vision pharmaceuticals in China and planning to establish a global commercial presence for endocrinology rare disease portfolio. In addition we have several significant upcoming milestones expected in the remainder of this year including introduction of the growth hormone auto-injector into the enliGHten Trial, initiation of our Phase II clinical trial of TransCon CNP, top line data from the TransCon PTH Phase II clinical trial and release of further data from our endocrinology rare disease pipeline. We are very excited to be hosting our second R&D day in New York City on June 26 where we will provide a detailed update on our three endocrinology rare disease programs including detailed data on TransCon Growth Hormone from the heiGHt and fliGHt trials, updates on our TransCon PTH and TransCon CNP programs and introduction to our commercial organization and an introduction of our oncology Vision strategic goals, and product candidates. This will be one of the most important events for Ascendis this year for delivering on our vision for achieving sustainable growth and building a leading biopharma company. Operator, we are now ready to take questions.

[Operator:]

Thank you. And our first question comes from Tazeen Ahmad with Bank of America Merrill Lynch. Your line is open.

[Tazeen Ahmad:]

Hi guys. Good afternoon. Thanks for taking my questions. Scott maybe one for you. You provided some color on the R&D expense for the quarter already, but maybe just to give us a better sense of the rest of the year, what proportion of those expenses that you mentioned do you expect to continue throughout the rest of the year?

[Scott Smith:]

I think that in general the expenses will continue throughout the year I think when it comes to just annualizing the first quarter, there was a bolus of PPQ expenses in the quarter for about €10 million, so rather that have that be spread out throughout the year, we took an upfront -- we had an upfront payment for that amount.

[Tazeen Ahmad:]

Just directionally speaking just given the fact that you are going to be having more programs presumably in terms of the clinic, should we think about that run rate should increase as the quarter’s progress throughout the year?

[Scott Smith:]

I would think based on what we disclose today, I would expect even at most continued run rate if not trailing off as we roll off the TransCon Growth Hormone program, but you have to come to the R&D Day on June 26 to get a feel for our oncology pipeline programs as well and start modeling those out.

[Tazeen Ahmad:]

Okay. Thanks Scott.

[Scott Smith:]

Thank you, Tazeen.

[Operator:]

Thank you. Our next question comes from Joseph Schwartz with SVB Leerink. Your line is open.

[Unidentified Analyst:]

Hi, I'm Julie Park dialing in for Joseph Schwartz. Thanks for taking our question. I guess the first question that I have is on TransCon hGH program in particular the adult trial. So, I was wondering if you could provide an overview of your TransCon hGH trial for adults as you plan to initiate in 2020, how much of your trial site overlap is there between the heiGHt Trial and adult study? And can we expect enrollment and trial to go faster in adults due to your heiGHt and fliGHt trial experience?

[Jonathan Leff:]

Okay, this is Jonathan. We previously disclosed we are planning an adult growth hormone deficiency trial to initiate in 2020. We have not given any more specific timelines from that, although we are absolutely sticking to that timeline. It's firmly in our plans. These are a different set of investigators it would be very unusual for pediatric investigators to also see adult patients. So, a different group of investigators in that study. So, essentially zero overlap. We will present a little more detail at our R&D Day in later in June.

[Unidentified Analyst:]

Okay great. And then if I could just have a follow-up -- if I can just a follow-up question. So, for your commercialization a little bit early, but for your two endocrinology programs behind TransCon PTH, I was wondering if there could be benefit from your commercialization infrastructure that you planned and implement with TransCon hGH and how are you strategizing our efforts to launch hGH? Thank you.

[Jonathan Leff:]

I think that what was really one of the core element of our vision in 2015 when we actually decided to focus on one single therapeutic area was to ensuring that we have certainty, the economy of scale really to move forward and develop commercial organization that can advance all three product opportunities under the same umbrella. So, we see lot of synergy in what we are doing. Many basic specified U.S. We have one single commercial organization and it basically can ensuring that we can get this three product opportunities out to the market. And not only that we are talking about the synergy and economy of scale, but we're also talking about potential treatment synergy with some product like both TransCon Growth Hormone and TransCon CNP, so one of the was actually to show that we don't believe in having a single product opportunity and develop an entire commercial organization for just one product and us why we want to do have three independent product opportunities that potentially could go up to more than nine different indication just being commercialized by one single organization. By doing that you are providing a situation where you will see the highest value being creating out from the product opportunities.

[Unidentified Analyst:]

Great. Thank you very much.

[Jonathan Leff:]

Thank you.

[Operator:]

Thank you. And our next question comes from Jessica Fye with JPMorgan. Your line is open.

[Jessica Fye:]

Hey there, good afternoon. Thanks for taking my questions. When we think about oncology and where you may go with that therapeutic vertical. To the extent you are applying the TransCon Technology to somewhat de-risk target. I'm curious how you define a de-risk target, whether that has to be in an approved mechanism or approved molecule or if that could be something that's may be shown promise in the clinic, but has never gotten all the way to market. And then bigger picture can you may be set some more expectations about what we could learn at the R&D Day with respect to the oncology vertical and the kind of the levels detail we'll learn about? Thank you.

[Jan Mikkelsen:]

Thank you. It's one of the key questions we have working with and we actually started to think to move into next therapeutic area. And in -- for about 18 months, 24 months when we have all that discussion, we actually said we will do the same thing that we did in rare disease endocrinology, we will not have one single product opportunity. We believe in pipelines. We believe there’s a way you really get the synergy, economy of scale, but also believe in what I call building up a pipeline with different, kind of, you can say targets. So -- but one thing we have done in common in oncology is that we are not moving away what really makes a great success in rare disease endocrinology validated target, validated parent talks. So what you will see in our oncology is basically the same way of thinking, working on validated target, working on validated parental, and potentially I would like to come back to that, but we will really be focused on making highly differentiated product opportunities that no one else can make really addressing major unmet medical need. So what you will see under the table will be some kind of mimic what you saw in 2016 on our results day when we disclosed our pipeline in rare disease endocrinology. We actually were in a position that we will show you preclinical data that basic support why we are highly differentiated not only the data but also the science behind it and why we really addressing an major unmet medical need. So what we're doing in oncology is basically repeating the success what we have in rare disease endocrinology with the different aspect from a business model perspective have potential a quite different business model, because I choose different to moving to rare disease endocrinology and oncology where rare disease endocrinology had an basic and easy pathway out to commercializing directly to the patient with a few clinical trials. The complexity in going into the highly area like oncology will be that you present to see a different business model and also what you will see when we have our results day in late June.

[Jessica Fye:]

Got it. Thank you.

[Operator:]

Thank you. And our next question comes from Liana Moussatos with Wedbush. Your line is open.

[Unidentified Analyst:]

Hi. This is Shweta for Liana. Thank you for taking our questions, and congrats on all the progress. Can you talk about the physician feedback you have received for the heiGHt and fliGHt trial data? And then also comment on the pricing strategy or TransCon Growth Hormone?

[Jan Mikkelsen:]

I think Jonathan will take the first part of it.

[Jonathan Leff:]

Sure, Jan, I can do the second it's pretty straightforward. The physician feedback has been amazing; not only from the physicians but patients, the patient advocacy groups have been almost exuberant after the results; I mean it was such a remarkable result. I think really striking is how unambiguous it was, it was a very clean data set in terms of both efficacy and safety. So everybody is thrilled. We are hearing more and more anecdotes about the experiences and the extension study enlighten, everyone is just really happy. It is very, very few dropouts in the trial, which I think is exemplary of that. And I think we won't comment on pricing, it's premature to consider a pricing strategy.

[Jan Mikkelsen:]

But I think what we are thinking as a company and with respect to our core values and taking the focus on the patient; we want to be assuring that we believe we really have a unique product opportunities that will provide in real world a much better outcome for the patient. And we believe this is our core value to ensuring as much as possible of the patient population can get assess to our product opportunity, not only in U.S. but also in a global perspective and that is what we want to implement and work on.

[Unidentified Analyst:]

Thank you.

[Operator:]

Thank you. And our next question comes from Michelle Gilson with Canaccord Genuity. Your line is open.

[Michelle Gilson:]

Good afternoon. Thank you guys for taking my question. I guess some high level, can you talk a little bit about how you see the hyperparathyroidism market evolving over time with the potential introduction of TransCon PTH? This is more specifically, maybe, which patients do you think have the highest unmet need in the population for a more physiologic replacement therapy? And maybe help us to understand the market beyond patients treated with net par on daily and twice-daily split dose regimen? And then, how do you build the market from there?

[Jan Mikkelsen:]

I actually think that this is a subject we are talking a lot for the time being and also because just in this stage we have one hyperparathyroidism day and one of the element, which really giving me the comfort that we have developed a product opportunities, really addressing and through major unmet medical need if we are not listening to the patients. When I listen to the patient and hear the story what kind of burden happen in a day-to-day and how basic effect of the long-term complications you have of this disease. And if you want, you can go to our website and see and listen to some of the patients. And I think that really influence me a lot. When we -- no doubt the patient needed when we heal them, we talk, we see the complication everything else, we also done a major research focused on the U.S. related to physician more than 100 physicians and really focus on, would you really prescribe the product that is too replacement therapy, product that really providing physiological PTH 24 hours same days, and more than half of them will do it in the U.S. and this is also why we have the global reach in our Phase 3 program were we have addressed more than 200,000 patients. And I think this is why I personally believe this is not only for the patient and all over in this area will really be a major benefit for hospitals with the high value product opportunities because we really addressing major unmet medical need and when you come to our research day, we actually will continue and we share some of this more in-depth research that we have done in the U.S. market about how the physician see a true replacement therapy.

[Michelle Gilson:]

Okay. And maybe a follow-up. Can you talk a little bit about what you're hoping to learn about the Phase 2 that will inform the Phase 3 other than starting dose will be maybe I guess, specifically from individualized optimization that's planned for the extension? And then can you also just remind us of how you're reducing the supplements in Phase 2 PATH as far as the chasing?

[Jan Mikkelsen:]

So we learned a variety of things from the Phase 2 study. So certainly we will confirm the proof-of-concept which we are highly confident in knowing what we know from Phase 1 where we are simply replacing the missing hormone 24 hours a day. So that's an easy one. We will confirm the starting dose for Phase 3, which we have a very good idea of even now but we will tweak that a little bit and then of course it's titrated. So that's not crucial, crucial. We'll expand our safety database which is always important. And then finally we will be testing out the algorithm for our titration regimen. Our down titration of calcium and vitamin D. We have a very good idea of how to do that. I think it's always good to get a little more experience in the real world out there during clinical trials and will refine that perhaps for Phase 3.

[Jonathan Leff:]

I think in addition the Phase 2 trial, it also gives us an opportunity to take at least 40 patients into long-term extension trial where we basic can follow them, look on long-term function competition like what you see with for example, a bone turnover so we can normalize and get the right bone turnover and also look upon some of the complications like what you put into can follow and avoiding that we have further damage for example, in kidney. And I think this is something that we think is a rather important that we building of not only addressing the short-term symptom but also looking on the long-term complication.

[Jan Mikkelsen:]

Yeah, long-term renal complications in particular.

[Jonathan Leff:]

Exactly.

[Michelle Gilson:]

Thank you.

[Operator:]

Thank you. Our next question comes from Adam Walsh with Stifel. Your line is open.

[Adam Walsh:]

Hi, guys. Thanks for taking my questions. My first one is on the auto-injector. You mentioned that that's going to be introduced into the enliGHten Trial next month and will obviously be part of the BLA filings you along side. Is there a minimum amount of data that the FDA has guided to in order to get the device approved along with the drug like a certain number of patients with the agency would like to see treated with this device prior to filing. I guess the underlying concern is whether the auto-injector could somehow slower delay the TransCon Growth Hormone filing? That's the first question. A - Jonathan Leff Okay. Thanks, Adam. So there is a requirement it's a really minimum requirement it's really just a couple of dozen patients for a couple of months we then want to make sure that the real life patients can actually use the device and it's working properly. Of course we have studied it in well over 100 subjects both -- patients as well as untrained people and it's worked really well and we have iterated the design and instructions over many years. But this is the first time in actual patients with growth hormone. So they just want to make sure that it's working well we will actually greatly exceed the requirements that have been placed on us by FDA. We will have many, many months of exposure in dozens and dozens of patients. Probably close to 100 actually.

[Adam Walsh:]

Hey, that's helpful. In terms of the granularity that you will disclose on the fliGHt Trial at the upcoming R&D day. I know you have emphasized in the past to look at the fliGHt Trial as a primarily a safety trial and you have cautioned against making comparisons on say annual heiGHt velocity between the six months prior to entry in the six months on the TransCon Growth Hormone. I guess my question is beyond safety are there any things -- is there anything on the efficacy side that we really should be looking at when we see those granular data for fliGHt at the R&D day? Thanks.

[A – Jonathan Leff:]

Sure so as you stated clearly a safety trial and that's its main objective. However of course we are measuring heiGHt and we will report heiGHt. So it's important to understand the context so -- if someone is been a treatment naïve patient you're going to expect greater heiGHt velocity in the first year because of all the catch-up growth they get primarily in the first six months. So if someone has been on a growth hormone already for six months or for a year or even for two years or more you’re obviously going to expect less heiGHt. So typically in the second year you would expect about 15% to 20% less heiGHt velocity. So with all of those caveats, we will present some subgroup data to give you a flavor for the heiGHt that we are actually seeing. And put it in context of their prior exposure. So, we are very pleased. And I think you'll be interested in hearing more at the R&D day.

[Jan Mikkelsen:]

Also believe Adam that indicating the Delaware practice how willing to switch already is practice patient over to daily growth is really, really important, so the physician understand the algorithm. And understand how to try treatment. And we can show, real world practice how we get done. There is also a small new subgroup of patients whereas I have a strong, strong feeling for myself. And this is for children under three years that we actually treated with our product there, basically down to nearly the newborn. And I fell really, really thrilled to see that we actually have our product, also being applied in this really subgroup of patients whereas really, really have the high, high, high burden of injection and other things, because basic growth hormone deficient. And I'm really looking forward also to share this data video because I have a strong, strong feeling for what we really do, when we move down to basic newborn children.

[Adam Walsh:]

Looking forward to that and then just one final one if I could, briefly. On enlighten, when and how often will we see updates from that? Thanks.

[Jonathan Leff:]

So, periodically probably at earnings call, we will give you updates there. There won't be a whole lot whereas following this cohort that's pretty large now over time. We see them typically every three months or so. But we can provide updates along the way.

[Adam Walsh:]

Great, thanks so much.

[Jonathan Leff:]

Eventually our medical meetings we present the two-year data for example, that will be an important cut to as a milestone as well.

[Adam Walsh:]

Thank you.

[Operator:]

Thank you. And our next question comes from Alethia Young with Cantor Fitzgerald. Your line is open.

[Alethia Young:]

Hey, guys, thanks for taking my question. So I just wanted to talk a little bit about the profile of TransCon hGH has versus what you guys are trying to construct with your PTH therapy? And also the second question is that, can you talk a little bit about maybe high-level commercial learning from the fliGHt study as well as?

[Jan Mikkelsen:]

I didn't really get the question in that product profile and how we compare?

[Alethia Young:]

Just as kind of …

[Jan Mikkelsen:]

Can you repeat?

[Alethia Young:]

Yeah. How you think what you're trying to construct versus what NATPARA I think is currently like your therapy versus there. And how you think because you are firstly there in the market is the first question? Can you hear me?

[Jan Mikkelsen:]

Yeah. Exactly but I think I'm not really never comparing us to NATPARA, because NATPARA is not providing the same target product profile. So it's not to expand out from NATPARA, because we have a complete different product profile were we really are providing the true replacement therapy, that really gives us benefit for the patient on all aspect of the disease. This is how we got this time. So, when I'm seeing NATPARA, yes, it can increase calcium, but because they increase calcium by taking a lot of calcium supplement and activated vitamin D. Sure you can spare some of them and still have the calcium. But you basically have no real positive impact on urinary calcium. Well illustrated to the FDA document. So there was exactly what we actually got there. We really got clarity about when we went to what I call the FDA briefing documents related to NATPARA. So from that perspective, we see a complete different product profile. We are more focused on the unmet medical need, both from the patient perspective but also talking with the physicians understanding. Yes, this is the solution they have been waiting for. This is the product opportunity that can be in a position, so we basically can normalize. This patient group's PTH levels, as one you actually develop basal insulin and certainly you have diabetes patient that could have basal level of insulin 24 hours, seven days a week.

[Scott Smith:]

And then your second question on commercial insights from flight. fliGHt it turns out with a very valuable experience, because it provided real practical insights in the real world and how subjects might switch coming in on a daily therapy. And we did that in almost all the patients they switched with few exceptions from daily therapies to TransCon. So, how do you switch. What do you switch to? And how do you monitor IGF1, it was very practically helpful in the switching dynamic. And will be very useful to our commercial colleagues.

[Jan Mikkelsen:]

Thank you everyone.

[Operator:]

Thank you. That’s all the time we have for questions, we thank you for participating in today's conference. This concludes today's program. You may all disconnect. Everyone have a great day.