Ascendis Pharma(ASND)
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Ascendis Pharma(ASND) - 2019 Q4 - Earnings Call Transcript
2020-04-02 08:35
Financial 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].
Ascendis Pharma (ASND) Presents At 38th Annual J.P. Morgan Healthcare Conference - Slideshow
2020-01-14 16:33
Endocrinology Rare Diseases Pipeline - Ascendis Pharma expects to submit the BLA for TransCon hGH for pediatric growth hormone deficiency in Q2 2020 and the MAA in Q4 2020[7, 23, 50] - The Phase 3 heiGHt Trial demonstrated superior height velocity (AHV) of TransCon hGH in pediatric GHD, with 112 cm/year compared to 103 cm/year for Genotropin, a treatment difference of 086 cm/year[25, 50] - The adverse event profile of TransCon hGH was comparable to daily hGH across Phase 3 trials, with 77% of patients experiencing treatment-emergent adverse events (TEAEs) in the TransCon hGH 024 mg/kg/week group in the heiGHt trial, compared to 70% in the Genotropin group[42] - Ascendis Pharma plans to initiate a Phase 3 trial for adult GHD in Q1 2020 and a Phase 3 trial for pediatric GHD in Japan in Q4 2020[50, 97] - Top-line Phase 2 data for TransCon PTH in hypoparathyroidism is expected by the end of March 2020, with six-month data from the open-label extension expected in Q3 2020[7, 65, 97] - Preliminary data from the first 8 subjects completing 4 weeks follow-up in open-label extension of TransCon PTH trial shows that all subjects are completely off current standard of care, 8 of 8 subjects no longer require active vitamin D, and 7 of 8 subjects no longer require calcium supplements[65] - Ascendis Pharma expects to initiate a Phase 2 trial for achondroplasia (ACH) in China in Q4 2020 through VISEN Pharmaceuticals[7, 70, 97] Oncology Pipeline - Ascendis Pharma anticipates submitting the first IND or equivalent for its oncology program in Q4 2020[7, 95, 97] - Preclinical data for TransCon IL-2 b/g in cynomolgus monkeys showed a single 1 mg dose/animal (~01 mg/kg) resulted in >3-fold enhancement of lymphocyte counts, with minimal effect on eosinophils compared to Aldesleukin[88] Financial Position - As of September 30, 2019, Ascendis Pharma had approximately €659 million in cash and cash equivalents[7]
Ascendis Pharma(ASND) - 2019 Q3 - Earnings Call Transcript
2019-11-19 02:25
Ascendis Pharma A/S (ASND) Q3 2019 Earnings Conference Call November 15, 2019 4:30 PM ET Company Participants Scott Smith - SVP and CFO Jan Mikkelsen - President and CEO Juha Punnonen - Head of Oncology Dana Pizzuti - Head of Development Operations Conference Call Participants Jessica Fye - JP Morgan Alethia Young - Cantor Fitzgerald Tazeen Ahmad - Bank of America Merrill Lynch David Lebowitz - Morgan Stanley Michelle Gilson - Canaccord Liana Moussatos - Wedbush Securities Josh Schimmer - Evercore ISI Josep ...
Ascendis Pharma(ASND) - 2019 Q2 - Earnings Call Transcript
2019-08-29 21:21
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]
Ascendis Pharma(ASND) - 2019 Q1 - Earnings Call Transcript
2019-05-31 02:12
Ascendis Pharma A/S (ASND) Q1 2019 Earnings Conference Call May 30, 2019 4:30 PM ET Company Participants Scott Smith - Senior Vice President & Chief Financial Officer Jan Mikkelsen - President & Chief Executive Officer Jonathan Leff - Chief Medical Officer Conference Call Participants Tazeen Ahmad - Bank of America Merrill Lynch Jessica Fye - JPMorgan Michelle Gilson - Canaccord Genuity Adam Walsh - Stifel Alethia Young - Cantor Fitzgerald Operator Good day, ladies and gentlemen, and welcome to the First Qu ...
Ascendis Pharma(ASND) - 2018 Q4 - Earnings Call Transcript
2019-04-08 12:37
Ascendis Pharma A/S (ASND) Q1 2019 Earnings Conference Call April 3, 2019 4:30 PM ET Company Participants Scott Smith - SVP and CFO Jan Mikkelsen - President and CEO Jonathan Leff - SVP and Chief Medical Officer Conference Call Participants Michelle Gilson - Canaccord Genuity Yuko Oku - JP Morgan Adam Walsh - Stifel, Nicolaus & Company Liana Moussatos - Wedbush Securities Inc James Birchenough - Wells Fargo Securities Tiago Fauth - Credit Suisse Olivia Yang - Cantor Fitzgerald Joseph Schwartz - SVB Leerink ...
Ascendis Pharma(ASND) - 2018 Q4 - Annual Report
2019-04-03 21:22
Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 20-F (Mark One) ☐ REGISTRATION STATEMENT PURSUANT TO SECTION 12(b) OR (g) OF THE SECURITIES EXCHANGE ACT OF 1934 OR ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2018 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to OR ☐ SHELL COMPANY REPORT PURSUANT TO SECTI ...