Ascendis Pharma(US:ASND)2020-04-02 08:35Financial Data and Key Metrics Changes - The company reported a net loss of €218 million or €4.69 per share for 2019, compared to a net loss of €130.1 million or €3.17 per share in 2018, indicating a significant increase in losses [28]. - Research and development costs rose to €191.6 million in 2019 from €140.3 million in 2018, reflecting ongoing advancements in the pipeline [28]. - General and administrative expenses increased to €48.5 million in 2019 from €25.1 million in 2018, primarily due to higher personnel costs [29]. Business Line Data and Key Metrics Changes - The TransCon Growth Hormone program is on track for a U.S. BLA filing in Q2 2020 and an MAA filing in Q4 2020, with positive clinical data supporting its efficacy [12][14]. - TransCon PTH is in Phase 2 trials, with an expansion of enrollment due to the recall of NATPARA, aiming to establish it as a true replacement therapy for hypoparathyroidism [17][18]. - TransCon CNP is in a Phase 2 trial, with ongoing efforts to expand clinical programs in China, indicating a strategic focus on global reach [25][26]. Market Data and Key Metrics Changes - The adult growth hormone deficiency market is under-penetrated, with estimates suggesting only 15% to 20% penetration, presenting a significant opportunity for the company [49]. - The company is leveraging its global structure to mitigate risks associated with the pandemic, ensuring continued progress across various geographic regions [10][11]. Company Strategy and Development Direction - The company aims to become a leading biopharma company, focusing on a diverse pipeline of innovative drug candidates across multiple therapeutic areas, including endocrinology and oncology [9][26]. - The strategic goal includes establishing TransCon Growth Hormone as a new benchmark in growth hormone replacement therapy, with plans for global clinical reach and new indications [14][16]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving 2020 milestones despite the pandemic, citing a strong organizational structure and adaptability [10][11]. - The company anticipates increased expenses in 2020 as it continues to advance its pipeline and expand its oncology activities [30][31]. Other Important Information - The company ended 2019 with cash and cash equivalents of €598.1 million, providing a solid financial foundation for ongoing projects [29]. - The company is preparing for a potential launch of TransCon Growth Hormone in the U.S. in 2021, indicating readiness for commercialization [16]. Q&A Session Summary Question: Feedback from Pitco on growth hormone and PaTH Forward results - Management indicated they received initial feedback from Pitco, requesting more information, and they expect to hear back by the end of June [38][39]. - They plan to provide analysis related to each element of the composite primary endpoint in the PaTH Forward results [42]. Question: Requirements for European application and adult growth hormone market - Additional submissions for the MAA include an approved PIP and a risk management plan [46]. - The adult growth hormone deficiency market is significantly under-penetrated, presenting a large opportunity for the company [49]. Question: Titration regimen for long-term extension study - The high level of removal of standard of care is attributed to the physiological profile of the PTH product, which mimics continuous PTH levels [52]. - The six-month follow-up data is expected to be essential for assessing treatment effects [54]. Question: Differences in patients previously on NATPARA - Patients previously on NATPARA will be analyzed separately for certain endpoints, but not for the primary endpoint [56]. Question: Frequency of clinical visits in OLE study - The company is implementing procedures to allow for remote monitoring and follow-up, ensuring patient safety during the pandemic [64].