Ascendis Pharma(ASND) - 2020 Q2 - Earnings Call Transcript

Financial Data and Key Metrics Changes - The company reported a net loss of €94.9 million or €1.97 per share for Q2 2020, compared to a net loss of €58.9 million or €1.25 per share in Q2 2019, indicating a significant increase in losses [26] - Research and development costs rose to €63.6 million in Q2 2020 from €43.8 million in the same period in 2019, driven by advancements in the pipeline and increased clinical trial activities [26] - Selling, general and administrative expenses increased to €20.8 million from €11 million year-over-year, reflecting higher personnel costs and expenses related to commercial capabilities [28] - The company ended Q2 2020 with cash, cash equivalents, and marketable securities totaling €471.6 million, with pro forma cash including subsequent financing reaching approximately €1 billion [29] Business Line Data and Key Metrics Changes - The TransCon Growth Hormone received a positive opinion from the Pediatric Committee of the European Medicine Agency, which is a significant milestone for the product [11] - The company is advancing its clinical trials for TransCon Growth Hormone in both pediatric and adult populations, with ongoing Phase III trials in various regions [12][13] - TransCon PTH demonstrated potential to replace standard care for hypoparathyroidism, with 80% of subjects on TransCon PTH able to discontinue standard care compared to 50% on placebo [16][17] Market Data and Key Metrics Changes - The adult growth hormone deficiency market is under-penetrated, with only 15% to 20% of patients currently receiving treatment, indicating a significant unmet need [54] - The growth hormone market in Greater China is estimated to be around USD 600 million to USD 700 million, with a unique market structure where 75% of sales are direct payments from caregivers [78] Company Strategy and Development Direction - The company aims to build a fully integrated biopharma company with a diversified pipeline, focusing on endocrinology, rare diseases, and oncology [10][31] - The Vision 3x3 strategy emphasizes creating multiple high-value product opportunities using the TransCon technology platform [10] - The company plans to submit regulatory filings for TransCon Growth Hormone and TransCon PTH in the upcoming quarters, indicating a proactive approach to market entry [29][30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of TransCon products to address significant unmet medical needs and improve patient quality of life [10][18] - The company is well-capitalized to execute its strategic plans following a successful financing round, which will support ongoing clinical trials and product development [29][32] - Management highlighted the importance of demonstrating comparable efficacy and safety of TransCon Growth Hormone against daily growth hormone treatments [15] Other Important Information - The company is preparing to report 6-month data from the open-label extension of the PaTH Forward trial, which is expected to provide further insights into the efficacy of TransCon PTH [22][30] - The company has received Orphan Designation for TransCon CMP in both Europe and the U.S., enhancing its market position for this product [23] Q&A Session Summary Question: Can you expand on the SF-36 results and their significance? - Management highlighted the importance of the SF-36 results, noting that the data reflects a significant improvement in quality of life for patients on TransCon PTH compared to placebo, which was not observed in previous studies [33][34] Question: Will the PRO be reported with the 6-month data? - Management confirmed that both SF-36 and the new disease-specific PRO will be reported together with the 6-month data, allowing for a comprehensive analysis of patient outcomes [37][39] Question: What feedback has been received from the FDA regarding the growth hormone submission? - Management indicated that they have not yet shown the data to regulatory agencies but plan to do so alongside the 6-month data, which will include additional insights from the SF-36 results [47][69]