Ascendis Pharma (ASND) FY 2025 Conference September 03, 2025 08:00 AM ET Company ParticipantsJan Møller Mikkelsen - President, CEO & DirectorScott Smith - EVP & CFOConference Call ParticipantsDerek Archila - MD & Equity Research Analyst - BiotechnologyDerek ArchilaIt's a long way to say, yeah. All right. Let's get started here. Welcome everyone to the 2025 Wells Fargo Healthcare Conference. My name is Derek Archila. We're going to be kicking off today with Ascendis Pharma. From the company, we have Jan Mikk ...

Financial Data and Key Metrics Changes - The company has achieved revenue generation that is increasing quarter by quarter, indicating a positive cash contribution [3] - The peak sales potential for YORVIPATH is estimated at $5 billion, with a significant portion expected from the U.S. in the first four to five years [10] Business Line Data and Key Metrics Changes - YORVIPATH is positioned as a replacement therapy for hypoparathyroidism, with strong intellectual property protection extending to 2042 [4] - The company has a diversified strategy for ex-U.S. markets, with direct market efforts in 13-14 countries and plans to expand further [7] Market Data and Key Metrics Changes - In the U.S., there are approximately 80,000 to 100,000 diagnosed patients with hypoparathyroidism, highlighting a significant unmet medical need [5] - The competitive landscape for YORVIPATH is clearing, with no other products in clinical development offering similar benefits [6] Company Strategy and Development Direction - The company aims to develop three independent product opportunities addressing major unmet medical needs, leveraging its TransCon technology [2] - Future plans include label expansion and the development of new compounds targeting both rare diseases and broader indications [3][42] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to generate positive cash flow and remain independent of financial markets [3] - The company is focused on building a sustainable growth model through its pipeline and partnerships, particularly in rare diseases and metabolic disorders [43] Other Important Information - The company is preparing for the FDA's priority review of TransCon CNP, which is expected to differentiate itself from competitors like BioMarin's VOXZOGO [21] - The partnership with Novo Nordisk remains strong, with no changes impacting the collaboration on TransCon semaglutide [35] Q&A Session Summary Question: What drives the uptake of YORVIPATH in the U.S.? - The uptake is driven by a steady flow of new prescribers and prescriptions, with improvements expected in the infrastructure for patient transfers from prescription to treatment [11][12] Question: How does the company view competition for longer-acting formulations? - The company has opted for a daily formulation for YORVIPATH to ensure patient stability before considering a weekly product [15][16] Question: What is the differentiation of TransCon CNP compared to competitors? - TransCon CNP offers continuous exposure without injection site reactions and has shown meaningful clinical effects beyond linear growth [22][24] Question: How does the company plan to expand its TransCon platform? - The company is focused on developing multiple product opportunities across various therapeutic areas, including cardiovascular diseases [42][43] Question: What is the company's approach to cost management and earnings power? - The company has maintained cost efficiency and is looking to expand its pipeline while generating cash from operations [44][50]