Ascendis Pharma A/S

Ladies and gentlemen, thank you for standing by, and welcome to the third quarter 2020 Ascendus Pharma earnings conference call. At this time, all participants are in a listen-only mode. After the speaker presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. Please be advised that today's conference may be recorded. If you require any further assistance, please press star 0. I would now like to hand the conference over to your speaker today, Scott Smith. Senior Vice President and Chief Financial Officer at Ascendus Pharma. Please go ahead, sir.

Thank you, operator. Thank you, everyone, for joining our third quarter 2020 financial results conference call today. I'm Scott Smith, Chief Financial Officer of Ascendus. Joining me on today's call is Jen Mickelson, President and Chief Executive Officer. Dr. Mark Bach, Head of Clinical Development and Medical Affairs for Endocrinology Rare Diseases. Jesper Hoyland, Global Chief Commercial Officer, Dr. Dana Pizzuti, Head of Development Operations, and Dr. Yuha Poonanen, 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 our 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 statement section in today's press release and the risk factor section of our prospective supplement filed on July 9, 2020. 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 has 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 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 Yen Mickelson, our President and Chief Executive Officer.

Thanks, Scott, and good afternoon, everyone. We are now almost to 2020, which has been, again, a very transformative and successful year for Ascendis. Now, 11 months into 2020, we have met or exceeded all of our corporate goals to date. We have advanced our clinical programs, bringing them closer to addressing major unmet needs for patients. And we have built out our first two product opportunities in Oncology that have the potential to represent a new paradigm shift in treatment patients with cancer. Why are we able to execute in this manner day in and day out? It is because we are a company driven by the following fundamentals. Ascendi's three core values, the patience, science and passion, the power of Transcon technology, a clear vision with long-term strategic mindset on how to build a sustainable leading biopharma company. People ask me, how do you motivate Ascendi's employees to take a meeting with their colleagues at 6 a.m. in the morning or stay up until midnight to speak with a patient group? My answer is that it is not me that motivates our people. It is the core values of Ascentis. We put the patient first to drive our decision making. Everything we do is to develop product opportunities that address unmet medical needs for patients as fast as possible. We are dedicated to using science and biological understanding built by the scientific community over many decades to guide our patient focus and decision. And finally, we are passionate about realizing our shared vision and goals. We trust each other's strengths and when we are facing challenges, we remain optimistic and commit to work together as one team to achieve extraordinary results. What enables Ascendis to develop a continuous flow of diversified, highly differentiated product opportunity with a high probability of success as demonstrated by our clinical results? This is the power of the Transcon technology platform and our dedication to science. We have already demonstrated clinical validation of the Transcon technology in three independent endocrinology rare disease programs. Combining our Transcon technologies, the clinical validated parent dog has allowed us to harness well-known biology and the power of mother nature to deliver highly differentiated product opportunity with a high probability of success. We believe we are just getting started with Transcon and that we have a real opportunity to transform patient lives. Now we are embarking on applying this successful approach and our unique algorithm for product innovation to create potential high-value product candidates in multiple therapeutic areas. And we are now getting ready to enter the clinic in oncology. Transcon is a unique technology approach compared to other technologies, as we at Ascentis can create highly differentiated product opportunities not possible by other technologies and at the same time have expected high clinical development success as we are building on scientifically validated biological pathways and parent stock. Last, the value of having a strong vision and a strategic mindset with a clear direction on how to build a sustainable long-term value company is essential for success. Our pipeline strategy has been a key part of our successful vision. The first fundamental in our pipeline strategy is to focus on large orphaned drug product opportunities with a well-established unmet medical need, where the Transcon technology can make a major difference. The second fundamental is that we must build multiple product candidates in each of our therapeutic areas in order to achieve synergies. Economy of scale. and realized the huge advantage from a therapeutic focus in clinical development, regulatory affairs, medical affairs, and commercialization. We believe that combining these two elements provide the fundament for creating economy of scale, achieving long-term sustainable growth to highly differentiated product, and building a leading biopharma company. Our current vision Vision 3x3 provides clear direction and strategic goals year by year on how we want to build a long-term sustainable leading biopharma company through multiple approaches. I have to say, the results we have delivered to date by pulling all these things together, our values, our Transcon technology, and our Vision 3x3 have exceeded my expectations. Not only do we continue to execute year by year, Quarter by quarter, we have accomplished what many companies have tried and failed to do. We continue to push ourselves to deliver not only in endocrinology rare disease, but also in oncology, where I truly believe we have the opportunity to transform the treatment of cancer. We are planning an investor call later this month to share more with you about our vision and strategy in oncology and latest progress. Let me review some of our important achievements in this quarter. Let me start with Transcon growth hormone or Lonapec somatopoeia. All science tells us that growth hormone needs to remain unmodified to achieve the same mode of action as data growth hormone. The same as industrial growth hormone. And you have heard us say this for years. and FDA reviews of long-acting growth hormone analogs further confirm the science and demonstrate that you cannot cheat nature. If you modify a hormone, you will modify its effect, which comes with consequence. We believe transplant growth hormone may provide a major improvement to daily growth hormone therapy, an alternative that maintains the mode of action of daily growth hormone, addresses overall endocrine health and provides convenient weekly administration. Together, this benefit could potentially lead to better outcomes for patients and an expansion of the growth hormone market. In Europe, we submitted our first M&A filing for Transcon Growth Hormone for the treatment of pediatric growth hormone deficiency ahead of schedule. Our submission followed the agreement of EMEA to our proposed pediatric investigation plan, or PIP, covering children from 6 months to less than 18 years of age. We are pleased by the EMEA decision, because we believe it reflects the unique product feature of Lona PEG somatopoeia, which enables the long-acting release of unmodified somatopoeia. To our knowledge, the approval of our PIP is the first time PECCO, the European Committee responsible for overseeing pediatric drug development programs, has concluded that a development program for a long-acting growth hormone treatment supports the clinical development in children. In the U.S., we received notice that the FDA accepted our BLA filing for transplant growth hormone for the treatment of pediatric growth hormone deficiencies. And we now have a PDUFA date of June 25, 2021. We were pleased to hear that there were no filing issue, and we look forward to continue to engage with FDA during its review. We have also completed and submitted the routine day 120 safety and efficacy update from the Enlightenment trial. We are pleased to report that of the 306 children treated with franscon growth hormone in our phase three program 160 children have completed at least two years of therapy and more than 140 children in the u.s have now been using our novel auto injector for at least 26 weeks successfully with this updated safety assessment the overall safety profile has remained consistent with what was reported with the original BLA file. The observed safety profiles continue to be comparable to that observed for daily growth hormone, and no safety information has been identified that would negatively impact the established benefit-risk profile of Transcon Growth Hormone. Updated efficacy analysis showed that the analyzed height velocity was within the expected range for second-year therapy, indicating long-term efficacy with continued treatment. Building on our objective of creating global clinical ways, we recently announced the filing of a clinical trial notification with the PMDA in Japan ahead of schedule. to initiate our phase 3 RITE trial for the treatment of pediatric growth hormone deficiency. The RITE trial will randomize treatment-naive children with growth hormone deficiency in a one-to-one manner to transplant growth hormone or daily growth hormone. As with our pupil-to-height trial, the primary efficacy endpoint is analyzed height velocity at week 52. A third arm will include treatment-experienced children with growth hormone deficiency. The trials will be conducted entirely in Japan. The target enrollment is 40 subjects in the treatment-naive population and more than 10 subjects in the switch arm. An opportunity to continue in an extension phase will be offered. I'm also pleased to tell you that the global four-site trial of transgrown growth hormone in adult growth hormone deficiency is progressing as planned. In adults, We measure body composition, fat mass, lean muscle mass, etc., and we believe that Transcon growth hormone will perform well compared to daily growth hormone. Finally, the commercial team continues the planned preparation for the expected launch of Transcon growth hormone as quickly as practical after approval. During the coming months, we are looking forward to keeping you updated about the progress of Ascendi's first commercial launch. and our vision of how to develop Transcon Growth Hormone to become the leading growth hormone product in the global growth hormone market. Turning to Transcon P-TAIDS. For transcon-PTH, we submitted ahead of schedule an amendment to our IND with the FDA for the part-weight phase 3 clinical trials, evaluating safety, durability, and efficacy of transcon-PTH in adults with hypoparathyroidism, or HP. We have also submitted regulatory findings to enable imitation of European and Canadian sites for part-weight. Partway Trial is a six-month randomized double-blinded placebo-controlled trial with an open-label extension period similar to our Phase II trial. We plan to enroll about 76 adults with chronic HP who are currently on standard of care, randomized in the 3-to-1 fashion to Transcon P2H versus placebo. At the same time we initiated the PART-WELL phase 3 trial, we announced the preliminary six-month results from the open-label extension portion of the phase 2 PART-FORWARD trial. PART-FORWARD is a global phase 2 trial evaluating the safety, tolerability, and efficacy of transcon PTH in adult subjects with HP. These results were better than we could have possibly hoped for. These data indicate that the Transcon PTA can eliminate standard of care treatment for HP, since 100% of patients were able to remove active vitamin D, and 91% of patients were able to stop both active vitamin D and therapeutic calcium supplement. In addition, and very important for the patient, our results demonstrate continued improvement in measure of quality of life in Transcon PTA treated subjects using SF-36. For those subjects we initiated part forward on the placebo arm. When they switched to Transcon PTA, they were also able to normalize all domains and subdomains on their SF-36 scores. Turning to Transcon CMP. The ACOMTIS trial is proceeding as planned, and today we are announcing the filing in collaboration with Wiesen of an IND to initiate the Phase 2 ACOMTIS China trial of Transcon-CMP. The ACOMTIS China trial is a Phase 2 randomized double-blinded placebo-controlled trial evaluating the safety, efficacy, and pharmacokinetics of multiple subcontainer doses of Transcon-CMP administrated once weekly. The primary objectives of the clinical trial are to determine the safety and growth velocity of Transcon-CMP in infants and children of age less than 11 years with acondoplasia and include cohort expansion of optimal doses. All subjects who completed the trial will have the opportunity to receive Transcon-CMP in long-term extension trials. Moving to our second therapeutic area, Oncology, we remain on track to achieve our final corporate goal of the year, to file an IND or similar for Transcon TLR 7.8 Agonist in December. On November 20, we are looking forward to have a virtual Oncology Research and Development Day, where we will provide an update on our vision in oncology and an update on our two most advanced pipeline candidates. Transcon TLR78 Agonist and Transcon ELB IL-2 Beta Gamma. We will share our vision to create potential best-in-class oncology therapeutics by applying our systemic and intratumoral Transcon technologies to clinically validated parent drugs and biological pathways. As we continue to execute our clinical programs, we continue to build out our global clinical development and medical affairs capability with the hiring of Dr. Mark Buck. Mark joins us as Senior VP of Clinical Development and Medical Affairs for Endocrinology Rare Diseases and will report to me. Mark is a pediatric endocrinologist with 30 years of experience building and leaning clinical teams that has successfully launched innovative pharmaceutical products into global markets, including Janssen and Merck. Mark's experience managing global clinical programs across Europe, Asia, and North America aligns well with our vision, three by three, of establishing global clinical ways to bring our endocrinology rare diseases product candidate to market as fast and safe as possible. Each of the milestones we have achieved this quarter and throughout the year represent significant elements of the company's vision 3x3, our vision to create long-term sustainable growth. Our mission is to develop a pipeline of multiple innovative therapeutics, not just a single product opportunity. We have a powerful technology platform. that we can apply to create multiple product opportunities in multiple therapeutic areas. What all these product opportunities have in common is that they can truly address unmet medical needs. That is what motivates us and drives us, seeing our therapeutic providing benefit to patients, improving clinical outcomes, and fighting to bring them to the patient as fast as possible. This is my measure of success, and I think we can truly consider ourselves successful in achieving our goals to date. We look forward to sharing more with you as we move ahead in 2021 to advance our endocrinology rare disease product candidate to patient and bring our oncology pipeline into clinic. Now, let me turn the call over to Scott for a financial review before we open for questions. Thank you, Jan.