Earnings Call Transcript - ASND Q1 2020

Operator, Operator

Thank you for joining us for the Ascendis Pharma First Quarter Earnings Conference Call. I would now like to introduce Scott Smith, Senior Vice President and Chief Financial Officer of Ascendis Pharma.

Scott Smith, CFO

Thank you, operator. Thank you everyone for joining our first quarter 2020 financial results conference call today. I am Scott Smith, Chief Financial Officer of Ascendis. Joining me on today’s call is Jan Mikkelsen, Chief Executive Officer; Dr. Dana Pizzuti, Head of Development Operations; and Dr. Juha Punnonen, Head of Oncology. 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 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 expectations or projections disclosed in our forward-looking statements. And you should not place undue reliance on these statements. 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 most recent annual report on Form 20-F. Please note that our TransCon product candidates are investigational product candidates and 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 first quarter 2020 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?

Jan Mikkelsen, CEO

Thanks, Scott and good afternoon everyone. In this quarter, we continue to execute on our strategic goals, advancing towards our vision to build a fully integrated biopharma company. We reported positive data from our Phase 2 PaTH Forward trial demonstrating that TransCon PTH has the potential to be a true replacement therapy in hypoparathyroidism. We submitted an IND amendment to the FDA to initiate our global adult growth hormone deficiency Phase 3 trial, the foresiGHt trial. We continue to build out our global commercial organization with the hiring of Jesper Høiland. We are demonstrating the flexibility and focus of the Ascendis team to execute on the task with minimal impact from the COVID-19 situation. We are happy to see our office in Denmark and research and development facilities in Germany have now reopened for normal operations. In addition, we opened our new research and development facility in Redwood City, California. This facility is dedicated to our oncology division, including specific CMC activities related to the TransCon technology for sustained localized release. To be successful in our strategy to build a leading global biopharma company, we not only need to have the right product, but we also need to have the combined effort of the most talented workforce. Our vision is not just to get a product to the market, but we aim to establish leading market positions for each of our products. As we prepare for the potential launch of TransCon growth hormone, we announced the appointment of Jesper Høiland as Global Chief Commercial Officer. Jesper has 20 years of commercial leadership experience with growth hormone products. Hiring a global leader with therapeutic experience is the logical next step in our preparation for the global launch of TransCon growth hormone and the rest of our endocrinology rare disease portfolio. As part of our Vision 3x3 strategic roadmap, we expect to create sustainable growth through multiple approaches, including global clinical readouts and by pursuing new indications. The milestones for our endocrinology rare disease pipeline reflect our Vision 3x3. The milestones for this year remain on track, all being accelerated. The filing of market application in the U.S. in Q2 and Europe in Q3 for TransCon growth hormone in pediatric growth hormone deficiency, reporting of the 6-month treatment data from the PaTH Forward open-label extension portion in Q3, initiation of a Phase 3 trial for pediatric growth hormone deficiency in Japan in Q4, initiation of a second Phase 2 trial in China for achondroplasia through our partner, VISEN Pharmaceuticals in Q4. For our second therapeutic area, oncology, we are on track to submit an IND or equivalent for the TransCon TLR 7/8 agonist program in Q4. We are continuing to hire key talent and expertise to advance our oncology pipeline forward to clinical trials. With the traditional paradigm for research and development assuming that the development of novel therapeutics addressing significant unmet medical needs comes with high development risk, we are challenging this traditional paradigm with the continuation of positive clinical results from our rare disease endocrinology product candidates. Using the TransCon technology, we are able to leverage the validated biology of existing drugs to create highly differentiated product candidates by identifying significant unmet medical needs. Studying the science underlying the disease and applying our TransCon technologies to an existing clinically validated parent drug, we are able to create new product candidates designed to solve the unmet medical need. We believe we can simply design a highly differentiated product candidate with a potential higher success rate compared to traditional drug development and with a large commercial potential. This is how we have developed our investigative TransCon growth hormone product. First, we took a drug that has been known for many decades, somatropin or human growth hormone. Then we created a long-acting pro drug of somatropin, whereby we preserve the mode of action, the distribution in the body and the physiological properties of somatropin. Then we took the pro drug, TransCon growth hormone, and combined it with a simple, easy-to-use auto-injector with Bluetooth-connected health capabilities to create a product candidate designed to have optimal product features. It is the definition of evolution, building on the growth hormone nature and making it better. TransCon growth hormone has all the elements of what we set out to capture in a potential market-leading product. To date, we have executed a robust Phase 3 program relating the potential of TransCon growth hormone in both treatment-naive and treatment-experienced subjects. From the heiGHt and fliGHt trials, we have seen consistent positive data across the trials, showing comparable safety to daily growth hormone while preserving balance between the direct and indirect effects of growth hormone. And now with our long-term extension trial, enliGHten, we see continued improvement in growth in the arm treated from the beginning with TransCon growth hormone, compared to patients who started treatment with daily growth hormone for one year and then switched to TransCon growth hormone. The heiGHt Trial was the first Phase 3 trial for a long-acting growth hormone product candidate to demonstrate superior efficacy compared to daily administration. The fliGHt Trial was the first growth hormone deficiency registrational trial that included children under 3 years. Together, these trials demonstrate superior efficacy and comparable safety and bone age advancement; our value proposition is strong. So where do we stand with TransCon growth hormone? We now have orphan drug designation in both the U.S. and Europe as a treatment for growth hormone deficiency, which is a recognition of the strong need for a long-acting growth hormone therapy that, in addition to promoting improved growth, also addresses overall endocrine health. In the U.S., we are now working towards submission of our BLA filing as planned during Q2. In Europe, we have accelerated the MAA filing to Q3 instead of the planned Q4 filing. We are currently finalizing discussions with the PDCO on our PIP submission prior to filing the MAA. Globally, our reach continues to expand as well. In China, VISEN Pharmaceuticals is conducting a Phase 3 trial for TransCon growth hormone in pediatric growth hormone deficiency. In Japan, we plan to initiate a pediatric growth hormone deficiency Phase 3 trial in the fourth quarter. We recently announced that we submitted an IND amendment to initiate our global adult growth hormone deficiency Phase 3 trial, the foresiGHt Trial. We plan to begin worldwide enrollment later this year. Turning to TransCon PTH, we recently announced positive top-line data from the fixed-dose, double-blinded portion of the Phase 2 PaTH Forward trial. This trial demonstrated the potential of TransCon PTH to become a replacement therapy for hypoparathyroidism. We believe these data are great news for hypoparathyroidism patients who are in urgent need of therapy that sustains physiological levels of PTH 24 hours a day, 7 days a week. A true replacement therapy should be able to treat the patient to the optimal level of serum calcium, and not just the borderline low end of normal serum calcium that is part of today’s standard of care. We believe that TransCon PTH has the potential to improve all aspects of the disease and potentially restore the patient’s ability to lead a normal life. These 4-week data from the PaTH Forward trial were the first step towards demonstrating that TransCon PTH is not an adjunct to the standard of care, but a potential true hormone replacement therapy for hypoparathyroidism. In the trial, TransCon PTH replaced the current standards of care, active vitamin D and calcium supplements, in 82%, and demonstrated improvements not only in serum calcium, but also in urinary calcium, serum phosphate and calcium phosphate product, which are risk factors for long-term complications. TransCon PTH was well tolerated, with no serious or severe adverse events. There was no discontinuation of study drug and a comparable rate of adverse events compared to placebo. Importantly, the titration algorithm we used to eliminate standard of care did not demonstrate any hypocalcemic episodes, meaning our algorithm will be capable of guiding physicians on safely removing standard of care from patients. 58 of 59 subjects are still in the open-label extension of the TransCon PTH trial, and some subjects have already continued past the 6-month time point. As expected, we’re seeing a variety of doses being utilized in the extension portion, as subjects are allowed to titrate to the optimum TransCon PTH maintenance dose over a range of 6 to 30 micrograms per day. While still early in the process for some subjects, as expected, the mid-dose pen with 15, 18, and 21 micrograms per day is the most commonly used. We are seeing comparable use of the low-dose pen, with doses of 6, 9, and 12 micrograms per day, and the high-dose pen of 24, 27, and 30 micrograms per day. The average dose is around 18 micrograms per day, which aligns with our pharmacokinetic prediction of optimal doses based on our Phase 1 results and literature data for continuous PTH administration. This, again, confirms how TransCon technologies enable the development of a high-value, lower-risk pipeline compared to traditional drug development. After 6 months of treatment on TransCon PTH, we will evaluate the study outcome based on the primary composite endpoint of normal serum calcium, off activated vitamin D, taking less than 500 milligrams of calcium supplement and normal 24-hour urinary calcium or a 50% reduction from baseline. Finally, we are also validating our patient-reported outcome measure in communication with the trial as one of the first hypoparathyroidism-specific measures. It will help us evaluate the broader patient benefit and may strengthen our overall value proposition for TransCon PTH. In summary, the outcome of the fixed-dose blinded portion of the PaTH Forward trial demonstrates the effect we were hoping to see for TransCon PTH over a short 4-week period. What is most impressive for me from the data is that TransCon PTH regulates the average serum calcium concentration up to low levels of the 9th, while at the same time reducing urinary calcium and eliminating standard of care in 82% of subjects. Additionally, we were very pleased to see the expected reduction in serum phosphate and calcium phosphate product as well. We are looking forward to reporting the longer-term 6-month data from the open-label extension portion of PaTH Forward in the third quarter. As planned, we are engaging with regulatory authorities on next steps, including preparation for an end of Phase 2 meeting. We will remain on track with our plan to submit regulatory filings to initiate a global Phase 3 trial in North America, Europe, and Asia in the fourth quarter of this year. We have successfully completed our ethnobridging study, supporting Phase 3 development in Japan, showing no difference in the pharmacokinetic profile between Japanese and non-Japanese subjects and supporting the possibility of including Japan in the global Phase 3 program. We will continue to analyze the results to confirm our accommodating starting dose for the Phase 3 in consultation with regulatory authorities. Moving to TransCon CNP, we continue to work towards escalating dose cohorts in the ongoing ACcomplisH trial, our global Phase 2 trial that is evaluating the safety and efficacy of TransCon CNP at increased doses in children between the ages of 2 to 10 years with achondroplasia. The primary endpoint of achondroplasia is analyzed height velocity. Key secondary endpoints include changes in body proportionality, other co-morbidities, and patient-reported outcomes. Consistent with our global approach, VISEN remains on track to initiate a Phase 2 trial in China for achondroplasia in the fourth quarter of this year. We believe the continuous exposure to TransCon CNP can address the co-morbidities of achondroplasia, and not only treat height. This is how we have designed TransCon CNP and where we continue to see the value. In oncology, we continue to advance multiple programs as we prepare to submit our first IND or equivalent in the fourth quarter for TransCon TLR 7/8 Agonist. Our planned submission for the TransCon TLR 7/8 Agonist program will be followed by expected submission for TransCon IL-2 beta/gamma in 2021. In late June, we plan to present preclinical data from our TransCon IL-2 beta/gamma program at the upcoming virtual AACR conference. Data from this unique program, a novel, long-acting product of a receptor-biased IL-2, reinforce our product development algorithm in combination with the TransCon technology to create truly unique product candidates. Later this year, we will provide an overview of the expected clinical development plans for both our TransCon TLR 7/8 and TransCon IL-2 beta/gamma programs, including combination trials. Now let me turn the call over to Scott before we open up for questions.

Scott Smith, CFO

Thank you, Jan. Turning to our financial results for the quarter ended March 31, 2020. We reported a net loss of €63.3 million or €1.32 per basic and diluted share compared to a net loss of €53.6 million or €1.24 per basic and diluted share during the same period in 2019. Now let me run you through some key components of these results. Research and development costs for the first quarter were €57.5 million compared to €51.3 million during the same period in 2019. Higher R&D costs reflect continued advancement of our pipeline, with the primary drivers including an overall increase in personnel-related costs and higher costs related to the continued build-out of our oncology therapeutic area. For TransCon PTH and TransCon CNP, costs were relatively flat with higher clinical trial costs offset by lower manufacturing costs. For TransCon growth hormone, costs were lower compared to the same period of the prior year due to reduced payments and activities related to the preparation of validation batches. General and administrative expenses for the first quarter were €17.9 million compared to €10.4 million during the same period in 2019. These higher costs primarily reflect an increase in personnel and related costs as well as continued build-out of our commercial capabilities. Other income and expenses included an unrealized noncash gain of €10.3 million compared to an unrealized noncash gain of €3.1 million during the same period in 2019 due to foreign currency exchange rate fluctuations. We ended the first quarter with cash and cash equivalents of €534.4 million. We also remain on track to achieve the following milestones for the remainder of 2020: For TransCon growth hormone, these include submitting the BLA filing in the second quarter; submitting the MAA filing in Europe now in the third quarter; initiating a Phase 3 clinical trial for pediatric growth hormone deficiency in Japan in the fourth quarter; and enrolling subjects in the foresiGHt Trial, a global Phase 3 study for adult growth hormone deficiency. For TransCon PTH, these milestones include reporting 6-month open-label extension data in the Phase 2 PaTH Forward trial in the third quarter and initiating a global Phase 3 clinical trial for adult hypoparathyroidism in the fourth quarter. For TransCon CNP, these include, through our strategic investment, continued support of VISEN Pharmaceuticals as they work to initiate a Phase 2 clinical trial for achondroplasia in China in the fourth quarter. Lastly, in our oncology therapeutic area, we continue to invest in CMC and preclinical activities related to TransCon TLR 7/8 Agonist and TransCon IL-2 beta/gamma. We plan to submit our first IND or equivalent by the end of the year for our TransCon TLR 7/8 Agonist. We continue to execute on our goal of building a leading biopharma company with a diverse pipeline of potential high-value product candidates in multiple therapeutic areas. We remain on schedule, in some cases, even ahead of schedule, with our corporate milestones for 2020. With the upcoming addition of Jesper Høiland to our team, we strengthen our ability to establish global market leadership with the approaching potential launch of our product candidates. We look forward to updating you on our progress as we continue to move forward during the year. Operator, we are now ready to take questions.

Operator, Operator

And our first question is from Tazeen Ahmad with Bank of America.

Tazeen Ahmad, Analyst

Hi, good afternoon. Thanks for taking my question. Jan, I just wanted to ask about your oncology platform. You’ve now chosen the PLR molecule to be the first one that you’re moving into the clinic. Can you just give us some color about the characteristics that make this the right candidate to move into the clinic and what potential indications you could be looking at once it moves into patients? Thanks.

Jan Mikkelsen, CEO

Thanks, Tazeen. How we really look in our oncology? There is not one right product. There are a series of right products, and this is why we’re building up our pipeline in oncology. We are not just coming up with one single product; we’re coming with a pipeline of product opportunities. What we continuously say is, what is the role of product opportunities being launched to INDs through the next multiple years. And that is exactly what we are planning now. So we’re planning for the two. The first one is our TransCon TLR 7/8 and the next one will be our biased best-in-class IL-2 compound. I believe both of them are really unique. Why did we take one in front of the other? Mainly because we don’t really see that it's some kind of exception that we’re utilizing some of the same resources in filing and executing clinical trials, so we took one over the other. From the value perspective, I believe both are high-value product opportunities. The next two will be disclosed next year; we are moving forward will also be in the same group of high value. We’re building a pipeline. It’s because we believe that when you think about what we’re doing with the two different modes of action of these two compounds, TLR 7/8 is a complete paradigm shift, where you place an activator of the tumor inside the tumor, not just for intratumoral injections for about 1 or 2 hours and then it’s gone. We can place it in a tumor for months, activating it. This is why we believe this is a way we can transform a cold tumor into a responsive tumor. It can also be combined with other drugs. I believe that our real, best-in-class biased IL-2 compound can be exactly as valid as we saw checkpoint inhibitors; we're starting to push up the speed of the immune system. This is where I see our value proposition in oncology is not a single drug, but it will be a streamlined dedication to develop best-in-class, unique product opportunities inside oncology.

Tazeen Ahmad, Analyst

So would you want to move multiple molecules into the clinic before determining what indications you choose for each?

Jan Mikkelsen, CEO

When you look at a compound like both of them, there are multiple indications we can address. All kinds of solid tumors we basically should be able to address with our TLR 7/8. This is why we see that as a compound that has broad applicability across a lot of different kinds of tumors, and then you can ask, what exactly are the two or three first tumors we want to eliminate? That is what we will disclose later this year. We’re building a very strong medical team that will come out and disclose that later through the year, where you will get a flavor about the multiple programs we will conduct in oncology.

Tazeen Ahmad, Analyst

Okay, thanks, Jan.

Operator, Operator

Thank you. And our next question comes from Michelle Gilson with Canaccord.

Michelle Gilson, Analyst

Hi, thanks for taking my question. I have one on TransCon PTH. Just a suggestion that hypoparathyroidism patients are very complex to treat on standard of care supplements. Could you just maybe share some of the feedback that you’re getting from the investigators on your Phase 2 study about their experience using TransCon PTH, especially now that we’re starting to get beyond that titration period in the open-label extension? Are you hearing that it’s simplifying treatment for these patients? Are their patients pretty excited about it now that they’ve been moved on to active from placebo for some of them? Would you expect that 58 of 59 that are continuing now to continue based on this feedback from your investigators?

Jan Mikkelsen, CEO

I think, Michelle, you actually had a great interview with one of our key investigators, I think that was this morning. You received a lot of feedback from her related to the treatment of the patient, and you can share that with others. We see the same thing across all the sites, and it’s being confirmed by how we see the retention in the trial. We basically had 58, and all of them stayed in the treatment, despite COVID-19, despite being in Milan, Italy, one of the hot spots for COVID-19. We’re in a position where we see this is providing such a huge benefit to the patient that they are really staying on the treatment. It was not what I typically see just in a diabetes trial or anything like that, where you would typically, at that stage, see a lot of dropouts. I believe that this is truly transformative for the patient, and everything we get back and see in the data only supports that. You also got the feedback this morning.

Michelle Gilson, Analyst

Alright. And just as a follow-up, you mentioned on your last call that you were planning some FDA discussions around breakthrough designation for TransCon PTH. Has anything changed in your thinking on whether you’ll apply for breakthrough designation? Can you walk us through some of the most important data points from the TransCon PTH study that demonstrate that it might have a substantial improvement over standard of care supplements?

Jan Mikkelsen, CEO

What we are doing from a high-level perspective is that we’re trying to get this important treatment out to patients as fast as possible. This involves multiple interactions with regulatory agencies to do that. We just got the data out from some weeks ago; we’re still discussing it. What we would like to do is update you when we get clarity about what the optimal pathway is to get this product to patients as fast as possible.

Operator, Operator

Thank you. And our next question comes from David Lebowitz with Morgan Stanley.

David Lebowitz, Analyst

Thank you very much for taking my question. When patients are titrated to their ultimate dose, how long do you think it might take until they get stabilized with respect to urinary calcium?

Jan Mikkelsen, CEO

That is a question we hope to get more evidence on during the trial. You will see a period where a patient who has not seen PTH for a potential 8 to 10 years will have a calcium deposit that is very different compared to a normal person. There will be a transition phase, which can take months, perhaps 3 to 4 months, where you will see a net loss of calcium from the body. You could also see soft calcification coming from that. You will likely see that happening during this period. When we come to the 6-month point, I believe we will see the best stabilization of the patient, incorporating elements like having sufficient time with a regulated normal bone turnover. This approach results in much more regulated natural homeostasis of calcium metabolism in the body.

David Lebowitz, Analyst

Thanks for answering my question.

Operator, Operator

Thank you. Our next question comes from Josh Schimmer with Evercore ISI.

Josh Schimmer, Analyst

Great. Thanks for taking my question. I would like to come back to the oncology portfolio and maybe ask a little bit of a leading question. I think historically, dose-dense chemotherapy has generally reduced toxicity and improved outcomes in oncology, even with traditional chemotherapeutic agents. The TransCon platform would seem to be uniquely capable of essentially delivering dose-dense chemotherapy, whether traditional agents or the new targets you’re pursuing, either alone or even in combination. As you’re thinking of evolving the portfolio, could you give us a sense of what direction you may be headed in and whether the dose-dense chemotherapy successes of the past might be avenues that Ascendis can pursue?

Jan Mikkelsen, CEO

When we look at the landscape of product opportunities we have in oncology, it's huge. We have two discrete technology platforms. One platform can administer localized injections inside a tumor and keep it active for 2 months, 4 months, 6 months, with anything from cytokines to antibodies or small molecules, and even inside tumors. We’re building the pipeline. We are focusing on how fast we can bring one or two INDs into oncology development every year. This is the flow we will see coming in, and I 110% agree with your thinking. You could theoretically target almost anything, correct? And in theory, you could improve outcomes by recreating dose-dense protocols with everything you choose, either in monotherapy or combination. How do you target this? We spend a lot of time thinking about what the best opportunities are for bringing the most value to the patient. The manual is extremely long, and this is why we're spending many resources on this. We have committed to the two first; we have the next two in line, and we have two more planned. We’re building the pipeline again and again. It’s a great question, but there are many more opportunities than we currently have the capability to pursue.

Josh Schimmer, Analyst

Looking forward to seeing where you take the portfolio. Thank you.

Operator, Operator

Thank you. Our next question is from Alethia Young with Cantor Fitzgerald.

Alethia Young, Analyst

Hey guys. Thank you for taking my question. Congrats on all the progress with PTH over the quarter. I just wanted to ask about the dynamics of people taking vitamin D and calcium. Is there a possibility these data get more robust as time goes on and people become comfortable that TransCon is truly a replacement therapy?

Jan Mikkelsen, CEO

I agree. I think when people see the data, get the story, and hear how we’re really treating patients, it’ll be much easier to titrate away from standard of care, because they are now seeing the clinical evidence that TransCon PTH is a replacement therapy. A hormone replacement therapy restores the missing PTH to physiological levels, and that’s the paradigm shift. People are going to see the results and as we continue sharing them, it becomes easier to understand the benefits. We saw this during the trial; when first-line patients experienced benefits, it made it easier to include them.

Alethia Young, Analyst

And just a follow-up, I guess, psychologically, do you think that the experience from the extension data will lead to some patients being more comfortable with reducing vitamin D and calcium, as they're having a good experience? Is that a fair read?

Jan Mikkelsen, CEO

I think there’s a psychological aspect to moving off standard care for calcium supplements. We believe it will improve with the better treatment experience, data explaining the benefits of using PTH. We expect to see this.

Operator, Operator

Thank you. And our next question comes from Jim Birchenough with Wells Fargo.

Jim Birchenough, Analyst

Yes, hi guys. Congrats on all the progress, not common to see timelines actually improve. Just on the PIP, could you set expectations for what you’re hoping to see around those discussions? What would be a good outcome and how will we hear about that?

Scott Smith, CFO

What we had relayed before was that we’re in the middle of the process with them. We received encouraging information in January when they completed their assessment. They requested some additional information that was straightforward and didn’t involve anything we weren’t already doing in the program. We responded at the end of March. They informed us that the process has started, but it’s not very transparent right now; we expect to hear their assessment at the end of June. We’re moving toward an approved PIP. Everything points in that direction, but we won’t know until the end. All the long-acting compounds received waivers, which recognized some concerns related to long-term benefit risk in children. Tea leaves don’t seem to indicate that for us right now.

Jim Birchenough, Analyst

And just as a follow-up on the growth hormone side of things. If Jesper is on the line, I’d love to hear his thoughts. What are the main barriers to making TransCon growth hormone a commercial success? Is there any inertia or dynamics you’re thinking about in commercialization?

Jan Mikkelsen, CEO

Jesper will be joining us in August, where we expect to have our Q2 call. He will provide great feedback to you regarding his 20 years’ experience in the growth hormone sector, how to build a leading brand. We hope to see Ascendis Pharma in this position someday.

Operator, Operator

Thank you. Our next question is from Tiago Fauth with Credit Suisse.

Tiago Fauth, Analyst

Hey guys. I just have a quick question on the ACcomplisH trial. Could you give us a sense of the number of patients dosed to date or which cohort you might be right now? I know there’s some uncertainty, but trying to get a better sense of the timelines. If that’s not possible, what’s the length of follow-up you’re dosing to help the patients in each cohort before the data monitoring committee allows for dose escalation into the next cohort? Thank you.

Jan Mikkelsen, CEO

In this trial, every time when we have 3 months for a cohort, we have a safety meeting and efficacy meeting where an independent group unblinds the data, reviews it, and then makes recommendations to move forward to the next cohort. We are on track to also initiate our second Phase 2 trial, the ACcomplisH China, where we will have potentially more than 100 children ready to be treated with the new active dose at that time. This will enable us to have two independent Phase 2 trials with potentially greater than 150 patients in total, both of which will be double-blinded and placebo-controlled to evaluate efficacy and safety optimally. This is particularly important as we are treating children aged 2.

Tiago Fauth, Analyst

Got it. Thanks a lot.

Operator, Operator

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

Adam Walsh, Analyst

Hey guys. Thanks for taking my question. My first one is regarding the open-label extension for TransCon PTH. You previously talked about remote nursing visits for collecting urinary and serum calcium. Is that still the case? Are we seeing patients return to standard health care site visits? If it is still the case, how smooth has that process been with respect to collecting all the necessary data?

Scott Smith, CFO

We’re still seeing that evolve right now. Patient access is somewhat restricted in certain sites. So far, the visiting nurse scenario has been pretty successful; we’ve had almost no missed visits. One way or another, we’re collecting these data. I can’t tell you whether there’s been a significant move back to normalcy just yet.

Adam Walsh, Analyst

That’s helpful. A quick follow-up regarding the growth hormone launch in 2021. Given the unknowns around COVID, what might that mean for the commercial launch strategy? What contingency plans are you making to ensure maximal impact on that launch?

Jan Mikkelsen, CEO

This is the question we have been dealing with over the past period of time. We’re building flexibility and the ability to adapt to changing circumstances. What you will see is that, as in our other operations, we are adapting quickly to the circumstances. We are dedicated to get this product out to as many patients as possible, as fast as possible. I’m confident we will achieve that.

Operator, Operator

Thank you. Our next question is from Liana Moussatos with Wedbush Securities.

Liana Moussatos, Analyst

Congratulations on your progress. I have a couple of questions for Scott. €75 million OpEx in Q1; how should we think about Q2, Q3, Q4, and what’s the cash runway?

Scott Smith, CFO

Using the Q1 OpEx is probably a good base for modeling 2020. We have increasing costs throughout the year, including advancing oncology. As we said in the prepared remarks, the costs are falling off regarding the TransCon growth hormone. We previously said we’re comfortable through the launch of TransCon growth hormone. I don’t see any reason to change that.

Operator, Operator

Thank you. Our next question is from Joseph Schwartz with SVB Leerink.

Joseph Schwartz, Analyst

Hi, congrats on the progress as well. Just to follow up on Adam’s question, how do you see endocrinologists managing growth hormone deficiency in an environment where they might have restricted bandwidth? How do you plan to be able to titrate patients to the optimal dose of TransCon hGH if we do get a second wave?

Jan Mikkelsen, CEO

There are multiple facets to your question. As things change, whether that is the current environment in the U.S. or regions like Denmark and Germany, everyone is back to normal. For areas in the U.S. that are highly affected, the reach out to physicians will be done through video calls (like Skype) and other forms of interaction. Medical affairs will use their communication to provide information about our product in different manners. You won’t see changes in how growth hormone deficiency treatment is managed. There will be no difference in measuring IGF-1 or titrating patients with our product compared to daily growth hormone. Both function similarly, except one is taken daily and the other once a week with superior efficacy.

Joseph Schwartz, Analyst

Great. And on TransCon CNP, can you give us an update on the status there regarding IRBs, site activation, and timelines for generating data in achondroplasia?

Jan Mikkelsen, CEO

The timeline is as previously disclosed. I cannot predict what is the best active cohort; I should get a different job. We will do a trial; we will go through the cohorts. Once we proceed through dose escalation and feel it's time to stop, we will. We're hopeful with the ACcomplisH China that we can treat the optimal effective dose and expect to initiate the Phase 2 trial at the end of this year in China.

Joseph Schwartz, Analyst

Got it. Thanks a lot.

Operator, Operator

Thank you. Our next question is from Leland Gershell with Oppenheimer. Please go ahead.

Leland Gershell, Analyst

Hey, good afternoon. Thanks for taking my question. Just a quick question on the oncology pipeline, as you announced the advancement of the TLR 7/8. I wanted to ask about the other two: your TransCon IL-2 beta/gamma selective and the VEGF TKI. How should we think about those heading into the clinic? Can we expect to see that next year for either of those? Will there be additional preclinical data we could see emerging from either of those candidates?

Jan Mikkelsen, CEO

We are planning to present additional data related to TransCon IL-2 beta/gamma at the virtual AACR in June, including preclinical data from mouse and primate studies. We are moving forward with the IND for the TransCon TLR 7/8 Agonist program; studies are progressing well, and we are designing the clinical trial plans to start those studies early next year. In parallel, we are conducting preclinical studies for the early pipeline and adding data to the more advanced programs. Specific to your question, the IL-2 beta/gamma will begin soon after the TLR 7/8 enters the clinic; probably about three to six months after.

Leland Gershell, Analyst

That’s great. Thank you again. That’s very helpful.

Jan Mikkelsen, CEO

Thank you.

Operator, Operator

And this concludes our Q&A session and program for today. We thank you for participating, and you may now disconnect.