Ascendis Pharma A/S

NASDAQ:ASND

Earnings Call Transcript 2019-Q3

[Operator]

Good day, ladies and gentlemen, and thank you for standing by. Welcome to the Third Quarter 2019 Ascendis Pharma Earnings Conference Call. At this time all participants are in a listen-only mode. After the speakers presentation there will be a question-and-answer session. [Operator Instructions] Please be advised that today’s conference is being recorded. [Operator Instructions]

Now, I would now like to hand the conference over to our Senior Vice President and Chief Financial Officer, Mr. Scott Smith.

[Scott Smith, SVP and CFO]

Thank you, operator. Thank you, everyone, for joining our third 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; Tom Larson, Chief Commercial Officer; Juha Punnonen, Head of Oncology and Dr. Dana Pizzuti, Head of Development Operations.

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 that are not limited to our progress on our pipeline candidates and our expectations with respect to their continued progress, statements regarding our strategic plans, our goals regarding our clinical pipeline, statements regarding the market potential of our pipeline candidates, and statements regarding the planned regulatory filings.

These statements are based on information that is 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 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 filed on April 3, 2019.

Please note that our TransCon product candidates are investigational product candidates, and are not approved for commercial use. As investigational products, the safety and effectiveness of the TransCon product candidates have not been reviewed or approved by any regulatory agency. None of the statements made on the conference call regarding our TransCon product candidates shall be viewed as promotional.

On today's call, we will discuss our third quarter 2019 financial results and provide a business update. Following some prepared remarks, we will then open up the call to questions.

I will now turn the call over to Jan Mikkelsen, our President and Chief Executive Officer.

[Jan Mikkelsen, President and CEO]

Thanks a lot, Scott. Good afternoon, everyone. Thank you for joining us today. This quarter was marked by several milestone and achievements in our journey to build a leading fully integrated patient focused biopharma company, as outlined in our Vision 3x3. We are on track towards filing the TransCon growth hormone BLA in the U.S. in the first half of 2020 and the MAA in Europe in the second half of 2020. We’re also advancing our two other endocrinology rare disease programs.

For TransCon PTH we announced last week an expansion of our Phase II trial, which I will discuss in detail and we initiated as planned, our TransCon CMP Phase II trial. We also continue to make progress with our plan to establish global clinical and commercial reach for our endocrinology rare disease pipeline, as our partner in China VISEN Pharmaceuticals initiated a Phase III trial for TransCon growth hormone for pediatric growth hormone deficiency.

Let me start with an update on TransCon growth hormone. Preparation for our BLA filing in the first half of 2020 and MAA filing in the second half of 2020 are both on track. Last quarter, we discussed the two items remaining to be completed prior to filing, finalization of our long-term safety data and completion of our PPQ manufacturing validation batches.

As planned in Q3 we reported the last subject visit for the long-term safety database consisting of around 300 subjects on trial for six months, 120 subjects for 12 months, 45 subjects for two years. We have also now completed the production of our drug product validation batches required for regulatory filings. We are now finalizing the associated analytical and qualification reports for the BLA and MAA filings. Recently, the European Commission granted us orphan designation for TransCon growth hormone for pediatric growth hormone deficiency in Europe.

Orphan designation is provided to therapies for certain health conditions that affect a limited number of patients. And if a treatment already exists, the medicine must be of significant additional benefit. This is an early indication that the European health authorities recognize the unmet need in pediatric growth hormone deficiency and the clinical benefits of our once-weekly TransCon growth hormone therapy.

In addition, in the U.S., in the enliGHTen trial around 150 patients have now successfully been transferred over to our intended commercial presentation of TransCon growth hormone, consisting of our auto-injector and dual chamber cartridge. We believe this patient data will be sufficient to support our auto-injector to be part of our initial BLA submission.

In light of recent clinical development with other long-acting growth hormone products, I would like to address the fundamentals of growth hormone treatment. At successful product needs to have both the direct and indirect benefit of growth hormone to be safe and effective long acting growth hormone therapy.

We have stated for many years that the target product profile for commercial successful long-acting growth hormone must have all the integrated benefits of daily growth hormone therapy. This benefit of growth hormone treatment include not only catch up, sustained high velocity, but also improved body composition, including fatness and muscle PMI [technical difficulty] improved bone health, bone age advancement and improved quality of life.

We believe maintaining the same mode of action and tissue distribution as [daily growth hormone] [ph] is critical to mimic all these effect of native growth hormone. TransCon growth hormone is 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 [endorses] [ph] Somatropin, and Somatropin use in daily growth hormone products.

Our TransCon technology allows the released unmodified growth hormone to diffuse into the target tissue, maximizing its ability to clear out the same effect as [endogenous] [ph] or daily growth hormone. We are also developing our once weekly TransCon growth hormone to have the optimal product features from the daily growth hormone products. We developed our [ultimate] [ph] data to built-in optimal product features such as room temperature storage, small injection volume and a needle and an integrated connected healthcare platform. Also help clinician and the patient with [a great] [ph] adherence and convenience.

We’re also making good progress developing our connected healthcare support program. We expect this program to generate data to support adherence and include apps and interface point for both patient and provider [at lungs] [ph].

We have advanced the TransCon growth hormone program from the idea stage all the way through clinical trials involving around 400 patients and we’re now on the threshold of submitting marketing applications. TransCon growth hormone has a potential not only to match the current daily standard, but if approved may in fact offer great high velocity outcome.

With TransCon PTH, we have followed the exact same development agreement designed to address a major unmet medical need. In hypoparathyroidism patients are in urgent need of a therapy that sustains physiological levels of PTH, 24 hours a day, 7 days a week as a true replacement therapy. The burden of this disease is enormous. With current standard of care patient and physician must balance the trade-off between managing short-term symptoms and reducing risk of long-term complication.

The dilemma is that the treatment of short-term symptoms with calcium and activated vitamin D at the same time also increase the long-term complications. Short-term symptoms have a significant impact on patient productivity and daily lives. In our patient experience research about 30% were no longer able to work and over 76 patient reported interference with work productivity.

Meanwhile the long-term complication act yet another layer of economic burden to the healthcare system. Complication include kidney failure at four to eight fold greater risk, with hospitalization or ER visit experienced by 79% of patients. In recent months, this burden has been at the forefront of our minds, given the recall of the only approved PTH therapy for HP in the U.S. NATPARA.

Since the September record, we have been talking with size and patient about their experience. Originally, we saw that NATPARA might be back on the market and in patients hands quickly. Then at a recent patient conference, hosted by the Hypoparathyroidism Association. Our team learned how the patient community has been significantly affected by this situation.

We heard stories of desperation from patient faces with difficult choices about the care. Importantly, we also understood that it was uncertain when a short acting PTH therapy may again be available for the patient. So, we had been considering, what could we do to help some of these affected patients, while still advancing our clinical program as quickly as possible, and preserving the robustness of our PaTH Forward clinical trial.

Initial in PaTH Forward we were planning to enrolled subjects with a naïve to PTH with patient therapy, all we had undergone a long was out period of at least 12 weeks and a stabilization period. By putting the template in place, we are now able to include patients impacted by the record with only minimal change to our plan timelines.

We concluded this was the best course of action we can take to address the patient need. By reducing the washed out period and over enrolling the current phase to try, we may now obtain data about the safety and efficacy of TransCon PTH in both PTH naïve and experienced subjects.

Depending on our final sample sites across the two groups we can conduct their sub analysis across the Phase II trial in points, including potentially informative data about the relative effects on bone turnover and bone health. We will also look at how the two groups perform during the long-term extension phase in 2020.

Since the attempt will have no other impact on this time, or endpoints of the trial, including no impact on the subject outside of the U.S. it's also a relative streamlet operational process.

Importantly, we will not be expanding the patient population in our Phase II trial, if we did not believe TransCon PTH is function as designed as a true replacement therapy. In addition, one of the key elements for the oral program is building the proper size safety database, both in PTH naïve and PTH experience subjects with a larger Phase II trial we believe be on a good part to realize this objective.

We are very confident in our decision in the potential of TransCon PTH and in the data to be generated from the expanded path forward trial. We are very excited about the potential of TransCon PTH to change patient's life and look forward to the result of the PaTH Forward trial expected in Q1 2020.

Our third endocrinology product candidate TransCon CNP the auto advancing initiation of our Phase II ACcomplisH trial. With TransCon CNP, we again aiming to achieve the optimal balance of safety and efficacy. Upon exposure to the [indiscernible] plate CNP is both modulate of growth and regulate of the FPR3 pathway. In achondroplasia, CNP counterbalance a constant over active signaling pathways caused by the mutation of the FTF receptor 3. Therefore, continuous exposure to CNP at effective levels should result in normalization of the balance between the activity of the FTRF receptor 3 and the CNP signaling pathway, at balance that is insert essential for normal bone growth.

CNP short half-life in humans of two to three minutes had made it impractical to maintain constant drug exposure from one demonstration to the next. With our TransCon technology, we have demonstrated that we can overcome this challenge by providing continuous exposure to CNP for 24 hours a day, seven days a week, we believe we can develop TransCon CNP as a therapeutic option not only for achondroplasia but potentially for various other growth disorder.

Our Phase I clinical trials are TransCon CNP demonstrated we can achieve our target product profile. Now we are evaluating TransCon CNP in the Phase II ACcomplisH trial. We have the aim to evaluate its safety and efficacy in around 60 subjects with achondroplasia from the age 2 to 10 years. In this study, we are looking at a primary input of analyzed height velocity, as well as other key secondary endpoints, which include change in body proportionality, other comorbidities, and patient reported outcomes.

Building from our Phase I data, our goal is to demonstrate that once weekly TransCon CNP affects not only height, but the many comorbidities that can have a life altering implications for children with achondroplasia. During the quarter, we also continue with our global ACHieve study, our natural history study which is now enrolling at around 540 [ph]. ACHieve will provide important inside in the experience of children with achondroplasia. Both ACcomplisH and ACHieve clinical trials demonstrate our commitment to develop a new therapy offering for children with achondroplasia one that is designed to improve the overall health and well-being.

In oncology, we recently presented data from our TransCon TLR7/8 Agonist product candidate at the Society for Immunotherapy in Cancer. The data indicates potent anti-tumors effects in both injected and non-injected tumors. Perhaps more importantly, TransCon TLR7/8 Agonist resulted in lower adoption on systemic cytokines when compared to on conjugated parent TLR7/8 Agonist and triggered strong immunological memory when the animals were returned two months later with a new tumor without any further treatment.

We continue to event several new and exciting programs in this therapeutic area including our receptor bias long acting, TransCon IL-2 beta gamma candidate. In this area, as planned we are working towards our first oncology IND or equivalent filing in 2020. With our progress this quarter, we are one step closer towards achieving our Vision 3x3 of building and leading fully integrated biopharma company.

Now, let me turn the code over to Scott for financial update.

[Scott Smith, SVP and CFO]

Thanks a lot, Jan. Turning to our financial results for the three months ended September 30, 2019 let me review some highlights. For the third quarter we reported a net loss of €25.1 million or €0.53 per basic and diluted share, compared to a net loss of €34 million or €0.81 per basic and diluted share during the same period into 2018.

The third quarter 2019 net loss includes an unrealized non-cash gain of €27.4 million, compared to an unrealized non-cash gain of €3.2 million in the 2018 quarter due to foreign currency exchange rate fluctuations.

Research and development costs for the third quarter were €46.3 million compared to €31.5 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 to support developments of our product candidates.

Then for TransCon growth hormone costs were higher primarily due to increasing costs for manufacturing a validation batches and initial cost of building inventory in anticipation of a commercial launch, which for now will be recognized as R&D costs when incurred. Declining clinical trial costs following the completion of our Phase III heiGHt trial.

For TransCon PTH, costs were slightly lower primarily due to a decline in the preclinical and in -- due to a decline in costs for preclinical research and manufacturing, which were partially offset by higher costs associated with our Phase II PaTH Forward clinical trial.

For TransCon CNP costs were slightly higher primarily due to an increase in clinical trial costs related to our ACHieve study and ACcomplisH trial, which were partially offset by lower manufacturing costs. Other R&D costs were higher primarily reflecting activities within our oncology therapeutic area. As a reminder, our R&D expenses including manufacturing related expenses vary from quarter-to-quarter, reflecting the timing of ongoing development activities.

General and administrative expenses for the third quarter of 2019 were €10 million, compared to €6.8 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 third quarter with cash and cash equivalents of €658.7 million and 47,739,647 ordinary shares outstanding. As a reminder, our quarterly ending cash balance may be impacted by a combination of items including exchange rate and working capital fluctuations, which this quarter led to a net positive impact on the ending cash balance.

During the quarter, we continue to execute on our Vision 3x3 strategic roadmap, including completing our long-term clinical follow up and manufacturing of drug product PPQ batches for TransCon growth hormone and increasing our global reach through VISEN’s initiation of our Phase III trial of TransCon growth hormone in Greater China.

And looking forward over the near-term, we plan to continue progress toward a BLA filing for TransCon growth hormone in the first half of next year and the MAA filing in Europe in the second half of next year. Continued growth of our endocrinology rare disease pipeline through label expansion of TransCon growth hormone into adult GHD and expansion of our Phase II trial of TransCon PTH to include subjects previously treated with short acting PTH.

We report our top line Phase II TransCon PTH data in Q1 2020, an open label extension data for up to six months during 2020. Continue execution on the TransCon CNP ACcomplisH trial, as we march toward identifying an effective dose in 2020, advance our oncology product candidates toward first IND or equivalent filing in 2020 and continue increasing global rates for endocrinology rare disease pipeline.

Operator, we're now ready to take questions.

[Operator]

Thank you. [Operator Instructions] And our first question is from Jessica Fye with JP Morgan. Please go ahead, your line is open.

[Jessica Fye, JP Morgan]

Hi, guys, good afternoon. Thanks so much for taking my questions. First one is on manufacturing, I think you've previously mentioned that you are expanding your manufacturing capacity in order to ensure you have enough drug supply for the launch. Can you just provide a little more of an update on the manufacturing progress and your confidence level that you have enough in place to be ready for the launch?

[Jan Mikkelsen, President and CEO]

Thanks, Jess. What we doing now is actually not really affecting our launch because we already have the capacity really to work and provide necessary material for entire launch phase. And we basically already are manufacturing on the highest speed on every day, we really can do just really to build up as much as possible TransCon growth hormone that can go out to the patients.

What we more or less, really building up the secondary supplier is what we call the global reach, because we now really are implementing clinical trials for rest of the world to ensuring we are not only addressing a U.S. population, but really expanding the TransCon growth hormone to be available basic on a global basis. And this is why we are expanding the capacity and the capacity is built up in such a manner when we starting to get through what I call the clinical operation, the clinical approval process for rest of the world we will be in a position that we also have sufficient manufacturing capacity.

So to make it very short, the extra capacity is not really mainly focused on the U.S. market, is mainly built up to have global capacity.

[Jessica Fye, JP Morgan]

Okay. And then, I know you recently got orphan drug designation in Europe for TransCon growth hormone. And there has been some debate among investors about the regulatory requirements in Europe related to PEGylated products in children. So how much can we or can't we read into the orphan drug designation as kind of a good sign on that front? Did the European Commission review any data in granting that designation? Or was it more just based on the market size and the potential for benefit over existing therapies without a specific read on the data?

[Jan Mikkelsen, President and CEO]

Sure, they're reviewing our data because we are sending in an application that describing the concept of our product compared to the clinical data we have achieved. From that perspective, I think what we're recognizing is basic is that we are in a position that we really have a product opportunities that is really from an initial group it also see it adding a benefit to the patient and that is where we are. We’re still in a continuous dialogue with the European authorities.

And just to recap that discussion is that we’re feeling pretty confident, we are coming as one of the first companies dedicated pediatric development plans have always developing this TransCon growth hormone as a pediatric product. And what we have observed or what we have seen is giving a high confidence that we really have a product opportunity to have a global reach. Just recall, we have been through the entire preclinical passage both in Japan, South Korea and China and [without] [ph] any kind of pushback to our preclinical package.

[Jessica Fye, JP Morgan]

Okay. And last one for me, can you guys talk about the changes at the SVP level, including Jonathan Leff's departure? Are you guys looking for a new CMO at this time?

[Jan Mikkelsen, President and CEO]

We are, from a company policy never comment on single position and single cases. And what we're doing is that we were in a position that Jonathan has left the company for months ago. And we are in a position where we have dedicated a lot of the operations part to three different areas. And it’s really function extremely well. And we have high level of confidence that we now are developing an organization that really can support that we can implement our Vision 3x3.

And I'd say that is really the highly effective thing we want to do. And there has not been any kind of change in timelines or anything. But what we have done in the last 18 months is basically after we implemented our Vision 3x3, we are now in trying to develop the organization so the organization also can be developed to a stage that it can support a successful implementation of our Vision 3x3. And this is what we're doing in a strategic manner to ensure that we can be successful as we want to be.

[Operator]

Thank you. [Operator Instructions] And our next question is from Alethia Young with Cantor Fitzgerald, Please go ahead.

[Alethia Young, Cantor Fitzgerald]

Hey, guys. Thanks for taking my question. I was just curious about maybe digging a little bit more into maybe how opportunistically think about the PTH kind of pathway if you were able to get some patients who had seen that part in the past. And, the follow up is of the 70,000 patients in the United States or so I mean, how many do you really think kind of need it or trying to kind of think about an addressable market size here. Thanks a lot.

[Jan Mikkelsen, President and CEO]

There were a lot of questions. Those are lot of questions. So let me try to take the question in different stages. And if I miss any one of them, please come back. So let me first take the patient population. I think we have it very clearly in our Investor Day, how many patients we believe that have HP in U.S. And we think it's about 70,000 to 110,000. But I think really what the issue is, is the dilemma, the physician -- patient are in because both have an issue with short-term symptom long-term complications. And better you control the short-term symptom you basic worsen the long-term complication.

So definitely you can have a patient that basic feeling I - some way with my disease, but then the basic worsening the long-term complication that is no win here. And from my perspective is I personally believe that the majority of this patient should really be on treatment when there is a true replacement therapy. And how many will be there? I think there's always a good discussion, but I think out from a patient focus, I believe the majority should be on this treatment here.

So coming back to some of the other thing about NATPARA, where we saw the element coming out with the desperation and just recall NATPARA is a compound that basic are not it's a true replacement therapy. And even with that what you should call a compound that's providing some benefit, we really saw a basic desperation by the recall of this product.

For me, it's really illustrate again and again, we really addressing a major unmet medical need. And we will stay focused and focused on how fast can we get the TransCon PTH out to the patient. And this is what we took this initiative is to say, we want also to show then basic and you can say, in a switch therapy, switching for patient that coming nearly of NATPARA transforming over to the product, we also see the same kind of benefit that we received more in a naïve patient population.

[Alethia Young, Cantor Fitzgerald]

Great, thanks.

[Operator]

Thank you. Our next question comes from Tazeen Ahmad. Your line is open, please go ahead.

[Tazeen Ahmad, Bank of America Merrill Lynch]

Hi, good afternoon. Thanks for taking my question. Jan, just wanted to get your thoughts on a bit of the update that BioMarin provided for the achondroplasia drug. Obviously you are waiting for their pivotal results to read out, but would be interested in hearing your thoughts about, what kind of read through we should expect for your product just given the similar mechanisms? Thanks.

[Jan Mikkelsen, President and CEO]

Thanks, Tazeen. I actually think we agree a lot with BioMarin. The CMP is actually optimal way to address this condition or disease and potential, many other areas. Where we someway have a different idea, and it's actually going back to answer your question, if you have a hyper active signaling pathway, 24 hours, seven days a week, how can you really in a meaningful way balance this pathway back to a normal level by only having drug exposure for two to three hours. Basic saying is that 10% of the time you have an active drug.

This is why we believe when we designed our target product profile that we need to have a continuous exposure. They will be like taking a diabetic drug only for two to three hours. No one wants to do that. They want to have a continuous exposure of a basal insulin and this is where we basically want to be in a position that we can provide a continuous exposure.

And we know from basic growth hormone market how difficult it is provide really optimal inherence on any kind of patient group in a pediatric with daily injection. And this is why we also designed it as a once weekly dosing. And that is exactly where we believe we are highly differentiated to the BioMarin product. So I hope for the patients, I hope there will be some clinical effect on it. And I think this is what we -- everyone is waiting forward to see later this quarter.

[Tazeen Ahmad, Bank of America Merrill Lynch]

Okay. And can you just remind us when your next data update us for your program in CNP?

[Jan Mikkelsen, President and CEO]

You’re asking me a question which I really can't answer, because I really do not know exactly when we hitting the effective cohort. We believe we will reach it in for some of the first cohort, but exactly when we getting that, it's really -- I cannot really give you any kind of forward-looking statement related to.

[Operator]

And our next question comes from David Lebowitz with Morgan Stanley. Please go ahead.

[David Lebowitz, Morgan Stanley]

Thank you very much for taking my question. First question, do you have the dimension how many patients you were ultimately examining the Phase II PTH trial to? And how ultimately with that analysis of the separate populations look?

[Jan Mikkelsen, President and CEO]

Thanks, David. I think we need to separate it in different kinds of endpoint, because when we look at the primary endpoint, normalization of calcium, withdrawal of activated vitamin D, withdrawing of calcium supplement, normalization of urinary calcium. I don't think that will have any kind of effect where the patient is coming from.

But if you look an element, like for example, on a long-term extension data, where you think about bone quality, bone density, we know that a naïve HP patient because of lack of bone turnover, basic is building up a much higher bone density than a normal person. That happens not because is really -- is a positive thing because what they do well lack the normal bone turnover, so you're basically building up old bone. And this is where we will potentially see and development that is very different in bone density from a person that have been on a highly product like a short acting product like NATPARA or FORTEO with our basic is also at osteoporosis and therefore you will see that coming from a complete different bone density structure.

And this is where you will segregate the analysis on the primary endpoint no difference, but when we go to element like bone density on other related bone thing you basic, I believe we will see a different for the two different group as why we prepared to make self-analysis of this.

[David Lebowitz, Morgan Stanley]

That makes sense. Really ultimately be about 40 patients in the each site total?

[Jan Mikkelsen, President and CEO]

What we're trying to aim, because we already have randomized large portion of patient, we couldn't change our block randomization. And in the block randomization, we had a ceiling on 64 patients. So from that perspective we are in a position is that it's very, very hard for us to move higher up and around 60. Because we -- it's too high burden to start to really do the entire randomization. So from that perspective, we believe that we hope to have about 40 new patient which we are on track to enroll, and then the 20 patients on previous treated with NATPARA.

[David Lebowitz, Morgan Stanley]

Thanks for taking my question.

[Operator]

Thank you. Our next question comes from Michelle Gilson with Canaccord. Please go ahead.

[Michelle Gilson, Canaccord]

Hi. Thank you for taking my question. I was just hoping you could perhaps elaborate a bit on how the clinical profile of a typical NATPARA patient were at this point of previously on NATPARA patient differs from recombinant PTH naïve patients, perhaps beyond bone turnover. And if you think things like the higher amount of supplement that these patients might be on might affect the Phase II results. And then should we be expecting the reduced washout period to complicate the supplement titration, or how patient should respond to TransCon PTH enrollment?

[Jan Mikkelsen, President and CEO]

There was exactly why we limited down to four weeks, because we believe and I think data showing that after you stop NATPARA for four week, we actually are in a position where we both had dealt with the hungry bone syndrome and other things like that. So we basically are stabilizing, can say the same calcium with supplements in a level where it's really, really are much more reflecting that it's basically will be coming into the study related to the primary endpoint exactly as the more naïve patient.

[Michelle Gilson, Canaccord]

Okay. And then can you just -- after you get the results from the Phase II study, can you just talk about timelines for initiating the Phase III. And if this Phase II change affects the way that you think about that Phase III in any way for TransCon PTH?

[Jan Mikkelsen, President and CEO]

I think for us, it's a really, really, really important to have the two patient group now. And one of the things we also are really excited about in sometime next year both have six months data where really in large patient population. So -- but from related to the Phase III design what we are missing on for example, the six endpoint is exactly the same thing we also will be measuring in the primary endpoint on our Phase III trial. So accessing that is not changing anything, we are utilizing the same primary endpoint that we have agreed with regulatory agencies for as an indication for both our Phase III trial and we’re also testing them in the extension study.

[Michelle Gilson, Canaccord]

Okay, thank you.

[Operator]

Thank you. Our next question comes from Liana Moussatos with Wedbush Securities. Please go ahead.

[Liana Moussatos, Wedbush Securities]

Thank you for taking my question. What are the steps to filing the IND for the oncology program?

[Jan Mikkelsen, President and CEO]

That is a good question where we are in a situation where we basically are finalizing the CMC of scaling to make GMP materials. And we are finalizing and working on our preclinical safety package that is the normal safety package that will be part of the IND or equivalent filing, perhaps Juha you will say something on, we are really excited to go out and showing our data here when for first time in a setting where we really go out and representing a complete new concept of having long-term into intratumoral delivery. And Juha perhaps you can talk a little bit about the excitement that was showing from this poster presentation.

[Juha Punnonen, Head of Oncology]

Yes. And regarding the IND enabling studies, it really is a very standard package for safety and GMP manufacturing, nothing unusual there. We're very excited by the programs. There's a lot of potential in multiple indications and looking forward to the IND filing by the end of the next -- end of next year.

[Liana Moussatos, Wedbush Securities]

Thank you.

[Operator]

Thank you. Our next question is from Josh Schimmer with Evercore ISI. Please go ahead.

[Josh Schimmer, Evercore ISI]

Thanks for taking the question. Jan in your prepared remarks you indicated that you had plans to evaluate the TransCon CNP and settings this beyond achondroplasia, can you give us a sense of timelines to start some of those trials and what the development program might look like will you be pursuing the basket approach similar to what BioMarin discussed on their analyst event, are you going to target individual short stature indication separately? Thanks.

[Jan Mikkelsen, President and CEO]

Yes, it is actually some plan, we really, really are developing a lot now. We have a complete list of lots of interesting indication where we really see huge unmet medical needs that we really, really can address, not only in growth disorder, but it could also be in potential outside of growth disorder. I think CNP from my view it’s one of the most interesting new discovery pathway where you really can see how we can affect a lot of diseases.

But going back to growth disorder, actually I think that potential CNP would be effectively a cornerstone in many growth disorder as you have growth hormone being cornerstone today. And potentially you can also see indication where both of them will be used in combination to get the optimal efficacy. And it was why it was extremely important for us to have really both of them as a once weekly product and potentially have the opportunity to also make combination therapy.

So what you will see later this next year, you will basically see our plans being disclosed about what kind of indication we want to go in and what and when we are going to do it. But just I can guarantee we really going in very broadly with this compound, we would like to see some of the first cohorts now, and then when we have seen that we basically have a strong idea where to position it.

[Josh Schimmer, Evercore ISI]

And then for the TransCon PTH program, what do you ultimately see as the gating steps for approval? And where are you in terms of CMC and characterization and stability testing? And is it more likely to be long-term safety generation that determines the timing of a potential filing? Thank you.

[Jan Mikkelsen, President and CEO]

I think two elements were different compared to the growth hormone. One element that is very, very different for what we did with growth hormone already in the Phase II. We using the commercial presentation. With a major, major difference basic Phase II are being conducted with a commercial presentation we are going to market with. So, we not some way need to bridge or anything that that is always status now.

The second thing is that we are executing on the validation batches on highest speed. We have it up in full scale, we already had it in the scale we want to go from into our Phase II material. So, also because the need for the material is so much, much less in this.

The third element is that we filing under an NDA instead of BLA, meaning is that basic the validation and PPQ batches don't need to be finalized at the day of filing, but you need to have the justification of the process validation at that time. So we are in a must more pull position compared to the TransCon PTH that we ever have been compared to the growth hormone. PTH is also much, much, much simpler to synthesize and produce because it's done by chemical synthesis instead of a biological manufacturing.

[Josh Schimmer, Evercore ISI]

Got it, thanks very much.

[Operator]

Thank you. Our next question comes from Joseph Schwartz with SVB Leerink. Please go ahead.

[Joseph Schwartz, SVB Leerink]

Great, thanks very much. So I noticed that the primary endpoint for the path forward trial is evaluated at four weeks. But it seems to have taken much longer for NATPARA to work on some of the endpoints you're evaluating. So can you give us a sense of the time course that you expect and whether the time will be adequate and which of the endpoints do you think would be more or less challenging to achieve?

[Jan Mikkelsen, President and CEO]

I think the main -- huge difference between NATPARA and what we're doing is that you should must more compares to what you see with an infusion pump, where you providing PTH 134 [ph] infusion pump. What can you do with infusion pump study today? You can withdraw all the supplements basic on day zero. Could I do that in NATPARA no, where we are titrating for ever, ever weeks, for weeks, for weeks.

So what we believe that we need about six to eight days to come to a steady state of PTH level. And we believe after six days, we have really, really mimic what you do with infusion pump. So when we come to six to eight days, we basically should be a position that we can do all the withdrawal of all the supplements. And that is a huge difference to what you saw with NATPARA.

[Joseph Schwartz, SVB Leerink]

Okay. And then as you look at the market for TransCon hGH, what percent of traders are sophistic enough to figure out how to titrate TransCon hGH dosage in patients switching from daily growth hormone in order to keep IGF levels under the threshold of two or three standard deviations.

I ask because a lot of the physicians we speak with at academic sites describe themselves as IGF people who are comfortable that they can push on this growth accelerator pedal appropriately. But they acknowledge that there may be community sites who are less adapt. So if you look at the marketplace and think about who would be ready to adopt TransCon hGH, what do you see?

[Jan Mikkelsen, President and CEO]

I have actually think that what we have done we've done an extensive survey about 120 endocrinologist and we’re asking exactly all these different questions. And sure we seeing exactly the same thing you see different between the academic centers and other ones, this is really 100% right. But what we also know that all the patients that all the physician that used to use daily growth hormone.

And the TransCon growth hormone luckily can be as easily titrated as daily growth hormone. The only difference that need to have to measuring avacitive [ph] we need to measure that on day five. And if measured outside day five that is an correlation factor that correlated back to an average IGF-.

So all the knowledge, all the thinking about how to titrating, you can do exactly the same thing with TransCon growth hormone. Because we have the same unmodified compound as daily growth hormone and basic has the same mode of action and biology with that. So all the learning for the last 20-30 years you can basic transfer all to TransCon growth hormone, only main difference you need to measure at day five where you get the average idea for.

[Joseph Schwartz, SVB Leerink]

So does that entail additional monitoring or is it the same amount of monitoring as currently applied to the daily.

[Jan Mikkelsen, President and CEO]

We believe it will be the same kind of monitoring and it will not be more or less restrictive. Only thing is that you need to have a correlation factor if you measure it, not on day five, but on other days and this correlation factor will transfer your work through on an average idea of one, which are exactly what were used to apply in this.

[Joseph Schwartz, SVB Leerink]

Got it. Thank you.

[Operator]

Thank you. Our next question is from Adam Walsh with Stifel. Please go ahead.

[Adam Walsh, Stifel]

Hey, guys, thanks for taking my questions. On the PTH forward, now that you've increased the enrollment within NATPARA patients, your guidance previously had been to not expect any kind of statistical outcome. Should we get our hopes up there with the trial expansion?

And then are we still on for starting a growth hormone trial next year? And I'm going to sneak one final one in here on the achondroplasia, if vosoritide gets approved, what do you think that means for enrollment for your future trials? How should we think about that? Thank you.

[Jan Mikkelsen, President and CEO]

I got three different questions. I think Adam, if I got all right. The first question is really very simple. It's clear now, I think this expansion has not anything to do with statistic power. I actually think we can achieve the statistic power we want to do with much less than the 40 patient. So it's nothing to do with that. We pretty confident this product will function as a true replacement therapy. Next year, yes, we will plan for additional label expansion of our TransCon growth hormone. And we are working dedicated for that to achieve.

The last one vosoritide. I actually believe every parents, every child should be treated with the optimal product. And I think this is too because of what we also see in all indications. And I think, and I hope we really would be in a position that we can provide an optimal treatment for patients with achondroplasia that is not just giving a small height improvement of potentials, 1 to 2 centimeter, but really can be in position that really also can address the real comorbidity of this condition or disease. Because they don't have all the comorbidities just because of heights, they have a lot of other elements that we would like to address.

[Operator]

And our next question is from Jim Birchenough with Wells Fargo Securities. Please go ahead.

[Nick Abbott, Wells Fargo Securities]

Good afternoon, it's Nick on for Jim. So with the NATPARA outage, if it continues is there – maybe discuss the regulators more opportunity to get a more rapid product to table for TransCon PTH.

[Jan Mikkelsen, President and CEO]

We are coming from that position that we want to really show it in the patients. And that is what we are going to do now. And this is why we have expanded the trial also to that. What we always hope that we can be in a positive dialogue with regulatory agencies and be in a position that the potentially can help this patient group in the best possible manner. And this is what we dedicated to do and this is why we doing this change in this way. Dana, do you have any comments?

[Dana Pizzuti, Head of Development Operations]

Yeah, I think, our plan is as soon as the data are available that we would intend to show those to the regulatory agencies and assuming they come out the way we think they will we are fairly confident that they'll help us to expedite the program. But until the data come out, we can't be any more explicit than that.

[Nick Abbott, Wells Fargo Securities]

And then just a follow-up on that, is the Olympic [ph] chemistry the TransCon loyalty, the same the PTH that you can borrow or walk at the data you have already the growth hormone.

[Jan Mikkelsen, President and CEO]

The entire concept of the TransCon technologies is -- were almost identical across both the growth hormone PTH and CNP. So definitely, there's a lot of learning from the different places. I also believe that when we saw that in TransCon CNP, we basically got the opportunity to move down to children of the age two in our initial Phase II, I think it’s just building on a great knowledge about the safety of the TransCon technology.

[Operator]

And thank you. And with that, we end our Q&A session and program for today and we thank you ladies and gentlemen for participating. You may now disconnect. Have a wonderful evening.

[Jan Mikkelsen, President and CEO]

Thanks a lot.

[Scott Smith, SVP and CFO]

Thanks a lot everyone.