Release Date: February 12, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Ascendis Pharma AS (NASDAQ:ASND) achieved significant milestones in 2024, setting the stage for strong growth and value creation.
• Skytrofa has established itself as a high-value growth hormone brand, capturing a 6.5% market share in the US.
• Transcon PTH, the only FDA-approved treatment for hypoparathyroidism in adults, has launched in the US and is gaining traction as a new standard of care.
• The company has a strong financial position with cash equivalents totaling 665 million, including a 100 million upfront payment from Novo Nordisk.
• Ascendis Pharma AS (NASDAQ:ASND) is expanding its TransCon technology platform beyond endocrine diseases, exploring opportunities in metabolic and cardiovascular diseases.

Negative Points

• Revenue for Skytrofa in Q4 2024 was lower than the same period in 2023, despite volume growth.
• The company faces challenges in establishing insurance reimbursement policies for new products like Yorvipa.
• There is uncertainty regarding the timeline for insurance approvals, which are estimated to take 4 to 8 weeks.
• Operating expenses increased by 3% in Q4 2024 compared to the previous year, driven by higher employee and commercial costs.
• The company has not provided specific sales guidance for Yorvipa, creating uncertainty about future financial performance.

Q & A Highlights

• Warning! GuruFocus has detected 2 Warning Sign with ASND.

Q: Can you confirm that the 908 figure is unique patients and not cumulative scripts, including refills? Also, how many of the EAP and OLE rollover patients are included in that 908 number? Lastly, are insurance approvals taking 4 to 8 weeks as expected? A: Yes, the 908 figure represents unique patient enrollments, not cumulative scripts. About 20% of the 908 are existing patients from Transcon PTH EAP or clinical trials, and 80% are new to Yorvipa. Regarding insurance approvals, 4 to 8 weeks is our estimate, but since the drug has been on the market for a short time, we need more time to confirm this timeframe.

Q: Can you provide the Yorvipa patient number outside the US? A: We have not broken down the numbers outside the US yet. We are still in the early stages of commercial availability in Europe, with full commercial launches in Germany and Austria, and a non-promotional program in France. Patient numbers are increasing as expected.

Q: How many of the 908 scripts have been converted to patients on actual therapy, and what proportion of these patients had previous experience with Natpara? A: We don't have all the information yet to provide a concrete answer. However, based on our market research, many of the patients are likely coming from the group we define as uncontrolled on standard therapy. We are still in the initial phase of getting patients fully reimbursed.

Q: Are you considering providing sales guidance for Yorvipa for the full year sometime in the middle of the year? A: We would like to provide guidance when we are confident in the numbers. Depending on how the launch progresses and how different regions perform, we will provide revenue guidance when we feel confident.

Q: How have discussions with payers gone so far for Yorvipa, and what are your assumptions for prior authorization criteria? A: Discussions with payers have been productive, focusing on the clinical value proposition of the drug. We expect prior authorization criteria to be consistent with the label, both in terms of reauthorization timelines and necessary tests. While many policies are still being established, we are encouraged by the conversations.

Q: Can you provide insights on the titration process for Yorvipa in the real world? A: Endocrinologists are familiar with titrating medications, and they have found the process straightforward. We provide guidance as needed, especially when switching between different dose pens. Anecdotally, endocrinologists are comfortable with the titration process.

Q: Are you planning any run-in activities for the hypochondroplasia program to help accelerate the timing of the study and enrollment? A: We are still determining the best pathway forward for hypochondroplasia, whether it will be a standalone Transcon CNP treatment or a combination with Transcon Growth Hormone. We are actively discussing with agencies and considering the unique phenotype of hypochondroplasia to find the best regulatory path forward.

Q: How should we think about the capacity to meet the increasing demand for Yorvipa throughout the year? A: We have a robust supply chain in place, similar to what we use for other products like Skytrofa. We closely monitor inventory levels and are committed to ensuring there is no shortage of supply.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.