Ascendis Pharma A/S (ASND) 2014 Q4 法說會逐字稿

Thank you for standing by and welcome to the Ascendis Pharma 2014 financial results conference call.

(Operator Instructions)

I must advise you the conference is being recorded today, Wednesday, March 25, 2015. And I'd now like to hand over to your speaker today Tom Soloway, Chief Financial Officer. Please go ahead, sir.

Thank you, operator and good afternoon. Thanks for joining us today to discuss the Ascendis Pharma 2014 full-year financial results.

My name is Tom Soloway, Senior Vice President and Chief Financial Officer of Ascendis. Joining me on the call today are Jan Mikkelsen, President and Chief Executive Officer, and Dr. Martin Auster, our Chief Business Officer.

After market closed this afternoon we released financial results for the year ended December 31, 2014. A copy of this press release is available on our website.

We 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 include but are not limited to our beliefs regarding the clinical and commercial profile of TransCon Growth Hormone including its safety and efficacy, our plans regarding the completion of our Phase 2 pediatric study of TransCon Growth Hormone and the timing and means by which we present top-line and full study results, our plans regarding the timing of the initiation of our planned Phase 3 pediatric study of TransCon Growth Hormone and statements regarding the study design, our plans and the timing of post-Phase 2 study meetings with the FDA and the EMA, our plans regarding the timing of when we announce results from the ongoing Phase 1 study of TransCon Treprostinil and our expectations regarding the timing and amount of expense associated with the completion of Phase 2 and Phase 3 studies of TransCon Growth Hormone. These statements are based on information that is available to us today.

We may not actually achieve our goals, carry out our plans or intentions or are 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.

Actual results or events could differ materially and 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 sections of our Form F-1 filed on January 28 and the Form 20-F that we plan to file on or about March 27, 2014 for the year ended December 31, 2014.

And with that overview of about the call over to Jan Mikkelsen, our President and Chief Executive Officer.

Thank you, Tom. Good afternoon everyone and thank you for joining on our first earnings call as a public company. In 2014 Ascendis achieved important milestones in our two internal clinical programs, both targeting multibillion-dollar orphan indications.

For our most advanced clinical program, our once-weekly TransCon Growth Hormone, we established what we believe to be the best-in-class product profile. And for our second program, TransCon Treprostinil, we initiated the clinical development.

All growth hormone products marketed today are taken as daily injections. The burden of daily administration often results in poor patient compliance which in turn leads to reduced treatment outcome. To address this unmet medical need we are developing TransCon Growth Hormone with an easy-to-remember once-weekly administration.

Our TransCon product technology represents a complete new approach to generate long-acting growth hormone project. The TransCon Growth Hormone product liberates unmodified growth hormone into the bloodstream with the same peak levels and exposure compared to daily growth hormone. Our unique long-acting growth hormone solution, therefore, preserves the same mode of action and distribution within the body as daily growth hormone for this safety and efficacy has been demonstrated over more than 25 years of clinical experience.

Most of the medical and commercial value in growth hormone therapy is in the pediatric segment which comprised about 90% of the total market. We have fully enrolled a six-month Phase 2 study in children with growth hormone deficiency and expect to announce top-line data from this study in mid-2015.

In December we reported a promising interim analysis of this Phase 2 pediatric study. After completion of our Phase 2 pediatric study we plan to schedule an end of Phase 2 meeting with FDA and EMA and that then initiate a Phase 3 study in children with growth hormone deficiency in mid-2016.

In March we presented two posters at the ENDO Conference in San Diego. The first poster was related to the design, characterization and PK/PD profile of TransCon Growth Hormone and the second poster reviewed the released interim results from our ongoing Phase 2 pediatric study. These interim results included height velocity and safety data for the first 25 children who have completed the six-month study.

Some of the encouraging feedback received from physician and key opinion leaders at the ENDO Conference highlighted the best-in-class product properties of our once-weekly TransCon Growth Hormone. Efficacy comparable to active comparator daily growth hormone, no superficial exposure to either growth hormone or IGF-1 and excellent safety, tolerability and immunogenic profile in line with traditional daily growth hormone therapies and the successful enrollment of children with growth hormone deficiency with demographics that are comparable to other US and EU studies of daily and long-acting growth hormone products.

We will continue to be active at medical conferences in the spring and summer of 2015 as we introduce our clinical data to pediatric endocrinologists around the world leading to our presentation of the full result of the Phase 2 pediatric program at the European Society of Pediatric Endocrinology conference in Barcelona in early October.

We continue to actively apply or break through TransCon product technology to expand our internal pipeline. Earlier this year we initiated a Phase 1 study of TransCon Treprostinil in the field of pulmonary arterial hypertension.

This is our second program to advance into the clinic and we expect to announce data from this study mid-2015. Along with our partners we are continuing to advance our product candidate in our diabetes collaboration with Sanofi and our ophthalmology collaborations with Roche Genentech.

Finally in the corporate side we successfully completed a $60 million mezzanine round in December 2014 and then raised additional $111 million of net proceeds in our IPO earlier this year. Ascendis is now a well-capitalized company that is in a position to advance our once-weekly TransCon program through Phase 3 clinical development.

I will now turn the call over to Tom Soloway, our CFO.

Thank you Jan. As Jan mentioned the combined $170 million of net proceeds from our mezzanine financing and initial public offering have created a strong foundation which will allow us to advance TransCon Growth Hormone through Phase 3 development. For previous guidance we plan to initiate a single Phase 3 study in growth hormone deficient children with anticipated enrollment across Europe and the United States in mid-2016.

Similar to our Phase 2 study we anticipate having an interim analysis of a subset of patients that have completed either a portion or all of a Phase 3 study. While we are not in a position to provide cash guidance today for those of you who are building models over the period through which our Phase 3 data becomes available, we would anticipate that our expenses are front-end loaded.

This is due to a substantial CMC effort that is underway in preparation for the Phase 3 study as well as costs that are associated with the early development of our pen device. As such we anticipate that 2015 and 2016 will be higher cash burn years than 2017 and 2018.

Additional financial information was released earlier today after market close and we'll be happy to take any follow-up questions to such information during the Q&A session. And with that brief synopsis, I will now ask the operator to open the line for questions.

(Operator Instructions) Joseph Schwartz, Leerink Partners.

Great, thanks very much for taking the question and congrats on the progress. I was wondering first on the growth hormone program, although it might apply elsewhere as well if you could talk a little bit more about the pen device that you're developing with Bang & Olufsen, who's responsible for which efforts and do they have experience in this area or what experience will you be bringing to bear to ensure that the device is validated the way it will need to be for the regulators? Thanks.

Thanks, Joe. This is really an excellent question and we actually had an extremely fruitful collaboration with our partner in that device area Bang & Olufsen Medicom.

Bang & Olufsen is a company that has been involved in pen devices for many, many years and actually has been the fundament for building out the portfolio of pen devices for Novo Nordisk. So we actually were extremely proud to be working together with them and develop this new pen device that is tailor-made to the pediatric population.

The pen device is being developed as what we call the most advanced with the Danish design as we can hope for. And we will be in a position that we're sure we will come up with a pen that is not only living up to what we call on the optimal features related to design and operation but also being connected to what I call the next generation where there will be automatic connection by Bluetooth to smartphones and other elements which will be an integrated solution.

Just to give you a flavor about Bang & Olufsen's Medicom capabilities they actually had just launched another product here, I think it was last year with one of the big pharma where they actually launched a pen device that actually had some of the same features that is being built into our pen device. Related to device development this is actually Bang & Olufsen that have this expertise and they will also be responsible for the manufacturing.

Great, thanks for all that color. And then could I ask one on the TransCon Treprostinil program?

Treprostinil is a drug which when it's given parenterally results in a lot of tachyphylaxis and personalization of dosing I think. So how do you expect to manage that nuance with the agent together with your longer acting profile that you can offer?

Joe, again an excellent question and is actually very much related to your first question. Because one of the things we want to integrate it in this product opportunity is actually a device strategy and in the device strategy we'll be developing as you have known for insulin area where you also need to have a very tight control of for example the dosing profile of the patient.

And therefore we will actively apply the same dosing assistance that has been developed for this device we are developing for such a product opportunity. So we actually can accommodate exactly and to revise precise dosing of each single patient.

Okay, great. Thanks for taking my questions.

Matthew Andrews, Wells Fargo.

Hey, good afternoon. Hi Jan and Tom. Thanks for the chance to ask a couple of questions here.

So I appreciate your waiting for the final six-month data for TransCon Growth Hormone by mid-2015 but could you just broadly discuss how you think the FDA's 505(b)(2) and Europe's equivalent pathway could be beneficial for your growth hormone development? And in terms of time to market possibly lower development costs and possibility of a very broad label at launch that could include adults or some other pediatric indications?

Thanks, Matt. To recap because we in our way we have designed our TransCon Growth Hormone the active ingredient is an unmodified growth hormone which will be liberated at the same concentrations as daily growth hormone. We actually believe we can apply the 505(b)(2) pathway.

The benefit we could potentially receive with the 505 pathway will be related not to our preclinical safety package or what we will conduct in the growth hormone deficient children but mainly to as you indicated to having as a possibility like it has been done for example for other companies like Sandoz when they made an equivalent trial to broaden out the label when we get the approval. So what we hope we can receive with our 505(b)(2) strategy is to use our reference product which would be Genotropin and then hopeful we can get the same indication that is in the Genotropin label on our label from the beginning.

Can I ask a follow-up question? So thank you. So on the interim six-month data how are you thinking about Phase 3 design?

It looks like you have some safety margin on the high-dose particularly as it relates to your IGF-1 exposure. So do you envision a two or three arm study assessing the mid- and high-dose arms compared to a once-daily growth hormone product?

When we discussed the Phase 3 design I think we are in a situation there we will make the final decision related to the dose that we will apply in our Phase 3 study when we have all the integrated data set. What we have seen until now is a clear dose response related to IGF-1 and we have also seen a small difference in the height velocity. We need still to wait on to get the entire data packet for all the patients which we will have mid this year.

Related to the safety and that is more related to what we have seen during the study we have not seen any kind of indication that we have any kind of safety risk with the doses that have been tested. In the three doses we have tested in our Phase 2 pediatric trial we believe all three doses are extremely safe and we will take a decision of the selection of the dose when we have seen all the integrated data from the entire trial and at the same time we also need to integrate input from both EMA and FDA as we want to design it as one single integrated Phase 3 trial covering both Europe and US.

Great, thank you. Tom, just quickly can you give us the Q4 EPS as well as the basic share count in the fourth quarter?

I actually don't have the fourth quarter right in front of me Matt. But we can follow up with that another time.

Okay, all right, great. Thank you for the chance to ask some questions. Thank you.

(Operator Instructions) We have no further questions at this time.

I'm extremely proud of what Ascendis has achieved in 2014. I'm confident about our plans going forward. We look forward to update you throughout the year about the progress of our programs.

Thanks again for joining us this afternoon. Thanks a lot.

Thank you very much. That does conclude our conference for today.

Thank you for participating. You may all disconnect.