Ascendis Pharma(ASND) - 2019 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - For Q2 2019, the company reported a net loss of €58.9 million or €1.25 per share, compared to a net loss of €22.8 million or €0.55 per share in Q2 2018 [23] - Research and development costs increased to €43.8 million from €40.2 million in the same period last year, reflecting higher personnel and infrastructure costs [24] - General and administrative expenses rose to €11 million from €5.2 million in the prior year, primarily due to increased personnel and site costs [26] - The company ended Q2 2019 with cash and cash equivalents of €690.4 million [26] Business Line Data and Key Metrics Changes - Costs for TransCon Growth Hormone decreased due to a decline in manufacturing and clinical trial costs following the completion of the Phase III heiGHt trial [25] - For TransCon PTH, costs were slightly lower due to reduced preclinical research and pen device development costs, partially offset by higher Phase II clinical trial costs [25] - TransCon CNP costs increased due to higher manufacturing and clinical trial costs related to ongoing studies [25] Market Data and Key Metrics Changes - The company is focusing on expanding its clinical reach in Asia, particularly in China, Japan, and South Korea, which are among the fastest-growing pharmaceutical markets [17][19] - In China, an IND was filed for a Phase III trial for pediatric growth hormone deficiency, expected to enroll around 75 subjects [18] - The company is also preparing for a Phase III trial in Japan, with plans to initiate in 2020 [19] Company Strategy and Development Direction - The company aims to achieve sustainable growth through its Vision 3 by 3 strategy, focusing on global clinical reach and label expansion programs [15] - The first indication for label expansion of TransCon Growth Hormone will be adult growth hormone deficiency, with a global clinical trial expected to start next year [20] - The company is committed to bringing its product opportunities to patients quickly and broadly, with a goal of filing its first regulatory submission for TransCon Growth Hormone next year [21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the safety and efficacy of TransCon Growth Hormone, anticipating it will set a new standard in treating growth hormone deficiency [11] - The company is preparing for BLA and MAA filings in 2020, with a focus on completing long-term safety data and process validation activities [12][27] - Management highlighted the importance of the Asian market for future growth, emphasizing the potential for accelerated development timelines through local partnerships [17] Other Important Information - The company is leveraging its TransCon technology platform to develop a pipeline of high-value product opportunities in rare disease endocrinology [22] - Management noted that the TransCon platform has validated its ability to move from innovative ideas to successful Phase III development programs [22] Q&A Session Summary Question: What is the unmet need for patients with hypoparathyroidism in the U.S.? - Management provided estimates of 70,000 to 110,000 patients in the U.S. and emphasized the severe impact of the disease on patients' ability to function [29][30] Question: Will the BLA submission for GHD have a favorable outcome? - Management is proactively addressing potential review issues in the BLA and believes the data supports a strong position [34][36] Question: What is the expected calcium dose for patients in the PaTH Forward trial? - Typical calcium requirements for patients are expected to be around 1,000 milligrams or more, with many tapering off supplements after treatment [41] Question: What are the key considerations for regulatory approval of TransCon PTH? - Management highlighted the need to demonstrate normalization of urinary calcium and the ability to withdraw supplements as key outcomes [50][52] Question: How does the company differentiate its TransCon technology from others? - Management emphasized the unique aspects of the TransCon platform compared to other technologies, such as PEGylation and protein fusion [74][75]