Jan Mikkelsen - President and CEO Scott Smith - Senior Vice President, Chief Financial Officer Jonathan Leff - Senior Vice President, Chief Medical Officer.

Joe Schwartz - Leerink Partners Alethia Young - Credit Suisse Liana Moussatos - Wedbush Securities Jim Birchenough - Wells Fargo Peter Stapor - Bank of America.

Welcome everyone to the Ascendis Pharma Full Year 2016 Financial Results Conference Call. Following some prepared remarks from the company, we will open up the call to Q&A. I'll now turn the call over to Scott Smith, Senior Vice President and Chief Financial Officer at Ascendis Pharma..

Thank you, operator, and thank you, everyone, for joining our call today. I'm Scott Smith, Chief Financial Officer of Ascendis. Joining me on the call today are Jan Mikkelsen, President and Chief Executive Officer, and Dr. Jonathan Leff, Chief Medical Officer.

Before we begin, I would like to remind you that this conference call will contain forward-looking statements that are intended to be covered under the Safe Harbor provided by the Private Securities Litigation Reform Act.

Examples of such statements may include, but are not limited to, our potential to become a leading integrated rare disease company, our goal to produce multiple potential best-in-class pipeline opportunities, statements regarding our strategic plan, our goals regarding our clinical pipeline of rare disease endocrinology programs, and statements regarding the plans for our Phase 3 heiGHt Trial of TransCon Growth Hormone.

These statements are based on information that's available to us today.

Actual results or events could differ materially from those in the forward-looking statements, and we may not achieve our goals, carry out our plans or intentions, or meet the expectations or projections disclosed in our forward-looking statements, and you should not place undue reliance on these statements.

Our forward-looking statements do not reflect the potential impact of any in-licensing agreements, acquisitions, mergers, dispositions, joint ventures or investments that we may enter into or terminate. We assume no obligation to update these statements as circumstances change except as required by law.

For additional information concerning the factors that could cause actual results to differ materially, please see the forward-looking statements section in today's press release and the risk factors section of our annual report on Form 20-F that was filed with the Securities and Exchange Commission this afternoon.

On today's call, we will discuss our full year 2016 financial results and provide a business update. Following some prepared remarks, we will then open up the call to questions. I will now turn over the call to Jan Mikkelsen, our President and Chief Executive Officer..

Thank you, Scott and thanks everyone for joining our call. 2016 was an important year for Ascendis. We outlined our Vision 2020 for Ascendis to become a leading rare disease company. We expanded our pipeline with two new rare disease endocrinology candidates, TransCon PTH and TransCon CNP.

For TransCon growth hormone we initiated our Phase 3 heiGHt Trial. All three product candidates are highly differentiated presenting potential best in class product profiles addressing unmet medical need. These programs are created by the same clinical validated TransCon technology and built on parent drugs with clinical proof of concept.

We therefore expect to have a higher success rate compared to additional drug development. Each of these pipeline programs is wholly-owned and address a multi-billion dollar market opportunity. Looking ahead in 2017, we are excited about our prospect.

We remain on track for regulatory signing for TransCon PTH in second quarter and to initiate a Phase I clinical trial in the third quarter. We aim to complete PTH enrollment in our Phase 3 heiGHt trial for TransCon Growth Hormone during the fourth quarter.

We also expect to submit an IND or similar filing for TransCon CNP during the fourth quarter and initiate clinical development in early 2018. In a couple of weeks, April 1, we will be presenting data from our three pipeline programs at the ENDO Conference in Orlando. We have eight poster presentation including two late breakers.

In addition to more information about TransCon growth hormone these posters will summarize the broad pre-clinical data supporting the potential best in class profile for TransCon PTH and TransCon CNP. We are also hosting a conference call and webcast on Monday, April 3 to review the ENDO data and we hope that you can get.

Before I turn the call over to Jonathan, I would like to highlight a few key points about our TransCon growth hormone. Since I got first got involved with human growth hormone in the 1980s, there have been many, many learnings from clinical and commercial experience both in daily growth hormone and with long acting growth hormones.

First, to be commercially successful, we believe that a long acting growth hormone needs to demonstrate comparable safety, efficacy and immunogenicity to daily growth hormone.

The goal is to achieve all the integrated effects of growth hormone treatment including catch-up and sustained growth velocity, improved body composition, including fat mass and muscle, better home health and improved quality of life.

We believe maintaining the same mode of action and tissue distribution is critical to mimic all these effects of major growth hormone. We have the only long acting growth hormone product in clinical development that is based on the release of an unmodified human growth hormone the same as endogenous and daily growth hormone.

Our TransCon technology allows unmodified growth hormone to diffuse freely and maximizing this ability to care out the same effect as in endogenous or daily growth hormone. Why this is important? According to the literature GH will certainly express on virtually every cell of the body.

Let me focus on the benefit of this direct effect of growth hormone on two important clinical outcomes. First, related to growth velocity about 15% to 20% of growth velocity is to growth hormones direct action in the growth. Second, for growth hormone to cut down fat mass, it needs to distribute into the fat tissue.

Based on the results from our Phase 2 trial with an actual comparison, we believe TransCon growth hormone will achieve the target product profile, our Phase 2 results demonstrate analyze height velocity of 11.9 centimeter to 13.9 centimeter which compare favorable to the 11.6 centimeter for the Genotropin giving us confidence in our heiGHt trial.

This heiGHt velocity result are also in complete alignment with a positive first year growth velocity seen for recent daily growth hormone Phase 3 program, [indiscernible] including their daily growth hormone compared to our arms Genotropin and Humatrope with all are in the range of 10.5 to 12 centimeter for first year velocity.

Now with the addition of TransCon PTH and TransCon CNP to our pipeline, we are making significant progress to built an internally wholly-owned portfolio of rare disease endocrinology products. We are more confident than ever in our three product candidates and look forward to advancing them towards value creating milestones.

Now, let me turn the call over to Jonathan, our Chief Medical Officer for [indiscernible]..

Thanks Jan. I would also like to reinforce the important transition our company has made during 2016. We have expanded and advanced our endocrinology pipeline to now include three products one of which is in Phase 3. Let me briefly review each programs starting with an update on TransCon growth hormone.

As you know, we continue to enroll patients in the global heiGHt trial, our Phase 3 trial of TransCon growth hormone in children with growth hormone deficiency or GHD.

Enrollment is proceeding well and we have made notable progress in recent months to activate a strong network of international sites in Australia, Canada, Europe, the Middle East, New Zealand and the U.S. We have gained IRB and health authority approvals in multiple countries and conducted site initiation visits worldwide.

We remain on track to complete enrollment during the fourth quarter of 2017. Our investigators are enthusiastic about our TransCon growth hormone program, the underlying technology as well as our pipeline.

Similar to the Phase 2 design, the heiGHt trials are randomized open label, active controls Phase 3 trials, which compares pediatric patients with GHD who receive once weekly TransCon growth hormone to those receiving injections of daily growth hormone.

As Jan referenced, the study objective is to compare our long-acting growth hormone to daily growth hormone from a safety, efficacy and tolerability standpoint with the primary endpoint being height velocity at 12 months.

Subjects in the heiGHt trial will also have the option to enroll in an open label extension to collect long-term safety and efficiency data. In addition to the heiGHt trial, we plan to conduct a switch study, this will enroll approximately 150 subjects previously treated with daily growth hormone who will switch to TransCon growth hormone.

The results of this single arm open label trial will further bolster our safety database more closely aligning it with worldwide recommendations for the clinical safety experience for experimental therapies. Importantly, we plan to conduct the switch study in parallel to the heiGHt trial with no impact on our program timelines.

The time of regulatory submission, we expect to have safety data on approximately 300 patients for six months or more. After recent discussion with the FDA, we feel the size and scope of our pivotal program including the switch study will provide a safety and efficacy database sufficient to support the regulatory filing.

Additionally, we are making progress with the final testing and preparations to introduce the TransCon growth hormone auto-injector during the extension phase of the heiGHt trial. This device administer TransCon growth hormone which is stored at room temperature as a single small volume injection for all patients via small 31 gauge needle.

We expect the device to provide comparable tolerability as daily growth hormone injections and be available at launch. At ENDO, we are delighted to be presenting four posters on our TransCon growth hormone program.

These will describe the Phase 3 heiGHt trial, additional information about the administration device, as well as the bracketed dosing for TransCon growth hormone which is designed to simplify and improve adherence to therapy We expect these four posters to build further enthusiasm for our program in the ongoing Phase 3 heiGHt trial.

Now, turning to TransCon PTH, which has been developed for hypoparathyroidism a rare and serious endocrine deficiency secondly to a variety of neuromuscular, cardiovascular and other symptoms.

TransCon PTH is designed to address limitations of current therapies, which do not address all aspects of the disease by providing constant PTH exposure at physiological levels.

We think this approach may result in an effective PTH replacement therapy that more fully controlled all aspects of the disease including urinary calcium levels and volatility in serum calcium levels. We are on track to submit a regulatory filing for TransCon PTH in the second quarter to initiate clinical development.

Specifically we plan to file with the therapeutic goods administration, the regulatory agency in Australia and initiate the Phase 1 trial during the third quarter. The pre-clinical pharmacology data supporting TransCon PTH are extensive.

We have demonstrated the pharmacodynamic effects in primate studies, showing its infusion like profile following once daily administration. Also relevant animal model show that TransCon PTH normalizes serum calcium and serum phosphate and reduces urinary calcium excretion.

At ENDO, we will be presenting two posters summarizing the TransCon PTH, pre-clinical information. This includes late breaking poster that will detail the effects of TransCon PTH in primates as well as in rats subjected to viral parathyroidectomy who are deficiencies in PTH.

Animal models to be presented at the conference will show the ability of TransCon PTH to normalize serum calcium and phosphorus as well as these positive effects on bone turnover. We are also presenting two posters on our third product candidate.

TransCon CNP which is in development to treat achondroplasia with an IND or equivalent plan in the fourth quarter. Achondroplasia is a serious skeletal disease characterized by disproportionate short stature and is the most common type of dwarfism.

There are currently no approved medical treatments and patients often suffer numerous surgeries to alleviate the skeletal abnormalities. Patients with achondroplasia also experienced higher mortality rates.

Our long acting CNP pro-drug, TransCon CNP, is designed to optimize the therapeutic index by allowing a CNP level high enough to be therapeutic, but without achieving levels that result in hypotension, which has limited current investigational therapies.

In relevant animal models, we have shown no change in blood pressure following once weekly administration of TransCon CNP. In contrast a daily administered CNP which did lead to hypotension.

At ENDO, we plan to present updated results from our primate growth study including six months data points, which will further characterize efficacy and the risk of hypotension.

Also in a late breaking posture we will present data from achondroplasia mass model characterized the effects of TransCon CNP on some of the most common traits associated with achondroplasia. We encourage you to participate in our ENDO conference call where we will review highlights of our various presentations.

Stepping back to reflect on our progress during the past year, I'm very encouraged by the differentiated product profiles for each of our pipeline candidates. These are supported by data we generated in both clinical and non-clinical evaluations. During 2017, we look forward to advancing these candidates one step closer to patients.

Now, Scott will provide the financial updates..

Thank you, Jonathan, and thank you, everyone, for participating in the call today. Turning to our financial results for the full year ended December 31, 2016, let me review some highlights.

With the help of our successful follow-on financing completed in the fourth quarter, we ended 2016 with cash and cash equivalents of €188.3 million and approximately 32.4 million shares outstanding.

For the full year 2016, we reported a net loss of €68.5 million or €2.58 per basic and diluted share compared to a net loss of €32.9 million or €1.39 per basic and diluted share during the same period in 2015. Research and development costs for 2016 were €56 million compared to €40.5 million during 2015.

Higher R&D costs in 2016 reflect greater clinical and manufacturing costs to prepare for the Phase 3 heiGHt Trial, ongoing development of our proprietary auto-injector and preclinical development for the company's two new rare disease pipeline candidates, TransCon PTH and TransCon CNP.

General and administrative expenses for the 2016 year were €11.5 million compared to €9.4 million during 2015. The increase is primarily due to higher general and administrative personnel partially offset by reduced consultancy cost.

During 2017, we expect an increase in R&D expenses as we invest in and advance our wholly-owned internal pipeline program.

R&D expenses will include for TransCon Growth Hormone cost associated with completion of enrollment and follow-up with patients in the Phase 3 heiGHt Trial, the initiation of our switch and extension clinical trials and clinical supply and validation of our commercial manufacturing process.

R&D for 2017 will also include for TransCon PTH, the initiation of the Phase 1 clinical trial and Phase 3 enabling activities including CMC, device development and toxicology studies. And finally for TransCon CNP, we will continue ongoing preclinical development to support an IND or equivalent filing in the fourth quarter.

We look forward to an extremely productive year as we make progress on all three of our wholly-owned rare disease pipeline candidates. We look forward to seeing many of you at the upcoming ENDO conference and sharing new data with you on all three of our programs. Operator, we are now ready to take questions..

[Operator Instructions] And our first question comes from the line of Joe Schwartz of Leerink Partners. Your line is now open..

Great. Thanks very much and congrats on all the progress.

I was wondering if you could give us some more detailed insight into the enrollment status of the heiGHt Trial?.

Really just what I said, we are moving along very nicely. I’m very pleased with the progress. We're opening sites on all continents and as expected we are randomizing patients. We're not disclosing exact numbers yet, but we are confident that we'll finish by the fourth quarter of this year..

Okay.

And there has been much to do about the baseline characteristics of patients in all the areas longer acting growth hormone studies, so I was wondering if you have insight into the baseline characteristics and how they compared to your previous study and thoughts on the relevance of these characteristics or not?.

Sure. So the baseline characteristics are of course very relevant. There are some factors that will drive towards more growth or less growth.

That said, most of the programs in the last 10 years all really seem to be basically pretty similar with one study that's a little bit of our outlier, but in general the baseline characteristics tend to be the typical 7 to 8 years of age, minus 2.5 height SDS scores minus 2 or below IGF-1 scores.

I would highly expect all of the ongoing studies to pretty much gravitate roughly towards those numbers. Of course, we will not look at our baseline characteristics until we are fully enrolled and even then very cautiously, but is to premature for us to evaluate our baseline demographics..

Okay. Thanks for taking my questions..

Sure..

Thank you. And our next question comes from the line of Alethia Young of Credit Suisse. Your line is now open..

Hi, guys, thanks for taking the question and congratulations on the progress. So, this is actually directly on [indiscernible]. So I guess on the comparative landscape, we'll probably see Versatis data in 3Q.

Could you please talk about what's the different scenario, let me put out, safety, had very positive data, how do you position yourself commercially and if they have bad data we'll just say that there is not going to be cost effect. Thank you..

I think it's -- thanks a lot for the question. I think it's a very relevant question. And as I believe when you look on the patient population we mainly are treating. This is children, adult with growth hormone deficiency. Basic this patient group are missing sufficient amount of endogenous growth hormone.

Why do you treat this patient? Children you treat from one parameter you like to see growth velocity and this is mainly the outcome, but you also have the integrated effect of all the other treatments like ensuring they get the right effect, distribution, they get the right muscle exercise capacity, they are getting consideration where they can exercise in a normal manner and the quality of life that they are getting out there.

So when you look at the patient group, you need to look on the entire integrated comprehensive effect of what you're getting with the growth hormone treatment and when you go to adult, you're sure you're not treating them for growth velocity because they are not growing, you've given them a treatment because lot of the effect like cardiovascular risk will suffer under the prophylactic effect and other things like that.

So when you look on the integrated faculties and this is what we wanted to see with us our treatment.

We wanted to ensure that a patient that is moving from a daily growth hormone product or have the alternative to take a daily growth hormone, which gives you all this comprehensive effect having move over to a long acting growth hormone that can give you all the entire comprehensive effect and precision that patient and everyone should expect to get and that was how we designed our product to ensure that we delivered the sale on modified growth hormone will provide the same mode of action will provide the same distribution as endogenous and daily growth hormone.

So therefore, we need to compare the competitive landscape out from who are in the competitive landscape are giving what I call the same efficacy, the same tolerability -- the same safety as you see with daily growth hormone.

If we you can do that, I have to think and this is our belief, this is the only way you can actually have a commercial successful long acting growth hormone and you could go down on single parameter like growth velocity if we go through all the recent Phase 3 trial that has been focused in the latest year related to what is the expected outcome for an analyzed height velocity.

We find everyone go between 10.5 centimeter to 12 centimeter, that is what you expect procedures for height velocity. And we also see for [indiscernible] which are one of the effect where you see the on effect on fat basis very, very clear lot of the trials are giving nearly [indiscernible] 1.0 to 1.5 kilo loss in 26 weeks on transfer.

So I see the competitive landscape is that for being, so first you need to show the same integrated effect after you see the daily growth hormone before you talk about having a commercial success..

With the U.S., there are various scenarios. I would just add that we fully expect Versatis to win and be successful in their pivotal trial and frankly we hope that's the case. That will be good for patients; it will be good for the field..

Okay.

So for the switch study, so what should we expect the timeline, should we expect the same by 4Q for the completion of the enrollment?.

No, no, no. So we're going to start the switch trial in the fall of this year. We expected to enroll relatively quickly since these are pre-identified patients who are already taking daily growth hormone. So it should be relatively swift enrollment, but we'll know more of when we actually start the study to the sites gets them upon running.

More importantly it will not be on the critical path for any filing activities. It will be conducted and completed in parallel to the heiGHt trial..

Okay. That's helpful. Thanks..

Thank you. And our next question comes from the line of Liana Moussatos of Wedbush Securities. Your line is now open..

Hi. Thank you for taking my questions.

What are the expected business development or partnership activities in 2017 and for which product?.

So, Liana, with regard to our existing partnerships with Sanofi in diabetes and Roche Genentech in ophthalmology and we can't comment on those publicly they are totally in the control of our partners, so any developments there at the material we only maybe applies to report on, but we can't comment on them on a forward-looking basis..

Do you anticipate any milestones this year from the existing partnership?.

We can't comment on that, it's totally – they are totally in the hands of our partners..

Okay.

And what about new partnerships?.

That scenario that we're actively evaluating Liana, I think with the great data we generated as well as the publication of our studies recently we're getting some interest actually in leveraging our technology in other areas. And I think where it makes sense and we have the appropriate resources to do that, we will execute some activities..

Okay. Thank you very much..

Thank you. And our next question comes from the line of Jim Birchenough of Wells Fargo. Your line is now open..

Hi, guys, thanks for taking the questions and congrats on the progress. Few questions just on the switch study. We expect those results to end up in the label and what represents a successful switch, is it just maintaining the prior growth velocity, or how should we think about that data and than we expected in the label? And then I have a follow-up..

Sure, thanks, Jim. I would expected to be in the label only because it's a significant number of patients and it will provide significant safety data for the – it's higher safety package.

We're not expecting any efficacy data other than study it really is the safety study, it will be very heterogeneous group of patients who are on various growth hormone products for various lengths of time, various devices. So I think any efficacy data will be very challenging at best to derive from the study.

So it really is a safety study and as such I think it will provide relevant information for the label..

And then, just in terms of the broader growth hormones opportunity, do you think the height study will be used to support used in other situations where you got short stature towards stature [indiscernible] syndrome Prader-Willi, you have a short stature or do you plan to do separate studies for those?.

The current experience is that once you have a product out there, the reimbursement tends to be fairly permissive across indications, of course without an indication we would never promote in those other indications and whether or not we plan to do other studies, we've just not made those decisions quite yet, it's a matter of ongoing discussions..

And then, just a final question on the hypoparathyroidism program, how should we -- as we think about the data we'll see at ENDO, what's a clinically significant reduction in that healthy care and I guess in terms of the end game you want to prevent renal calculi, so there is some threshold do you want to show that you can get below, do you ultimately think you need to show reduction renal calculi for differentiated label or should we be looking at a certain percentage reduction, how do we think about that data?.

Sure. So you think about it in terms of the normal ranges so it's about 250 milligrams for 24 hours in women, 300 milligrams in 24 hours for men.

And you should think about it in patients a many of those if not most like elevations and you should think about it in terms of what proportion of patients are returned into the normal range since that should protect them from the long-term sequel.

Now we're not going to patient data at ENDO, but we will release some animal data that shows that PTH reduces urinary calcium and that is not surprising since that is one of its most important actions on the kidney. It causes re-absorption of calcium.

So we're showing it in animals and you will see the data at ENDO, we would fully expect to see the same effect in humans..

One other thing that someway give us a strong belief that we can achieve this is that those study being conducted with exactly you can nearly say the same profile that we are aiming and it was done in the right patient group from [indiscernible] with the patient with hypoparathyroidism and you can actually show that you can get a normalization of the calcium in the urinary.

So you can say that is clinical data that indicate when you have the PTH in right physiological levels, you can be in a position that you can have and generate calcium down to the normal level..

Great. Thanks for taking the questions..

Sure..

[Operator Instructions] And our next question comes from the line of Tazeen Ahmad of Bank of America. Your line is now open..

Hi. This is Peter Stapor on our Tazeen. Just a couple of questions. So could you comment on how many trial sites are running, I know you talked about it on the last quarterly update 80 to 100 sites that you planned to get enrolling.

And also will you be giving updates on enrollment throughout the year?.

So we plan and still plan for about 100 sites worldwide. So we are making great progress towards activating all of those. It's very likely we will end up at around 100. Right now, we are only giving guidance on what we think is most important which is when the study will end.

We are relatively early on, but we are progressing at a pace that I'm very comfortable with the fourth quarter timelines. And later in the year, we will decide if it's necessary to provide any further updates on that, certainly when we have more patient experience and more experience with screen failure rates that maybe something we would consider.

But, the most important data obviously is when the last patient is enrolled..

Okay. Thanks.

And can I follow-up another -- so, the switch trial was this is a new requirement from the FDA or other regulators or was this something that you guys have been planning anyways to include in the label for safety purposes and maybe some additional cuts of the data on like you said the heterogeneous population that would switch into TransCon growth hormone?.

Right. So this to be clear was not a requirement of any regulatory agency.

Regulatory agencies acknowledged and we of course agreed that our advanced -- our Phase 3 program was on the small side, so we took the decision to bolster the safety database to derisk any regulatory path to add even greater comfort and there was a great deal of comfort to begin with. But it was admittedly a very trim program.

So now with the switch trial, it's a very robust program. It's really essentially in alignment with ICH standards which is not even meant for orphan diseases. So I think we are on very solid footing with the size and structure of our database.

And it will help commercialize as well since in the marketplace there will be some switching and if we can demonstrate the safe switch to TransCon growth hormone that will be a good thing..

Terrific, thanks..

Thank you. And I'm showing no further questions on the phone line at this time. Ladies and gentlemen, thank you for participating in today's conference. This concludes today's program. You may all disconnect. Everyone have a great day..