Ascendis Pharma AS (ASND) Q4 2024 Earnings Call Highlights: Strategic Growth and Market Expansion

Ascendis Pharma A/S (ASND) recently announced its financial results for the fourth quarter and full year of 2024, highlighting significant revenue growth and strategic advancements. The company reported a full-year revenue of €363.6 million, driven by the successful launch of YORVIPATH® in the U.S. and the continued market leadership of SKYTROFA® in the growth hormone sector. Ascendis also outlined its plans to submit a New Drug Application (NDA) for TransCon™ CNP for treating achondroplasia in children in Q1 2025, with a Marketing Authorisation Application (MAA) to follow in Q3 2025.



The strong early U.S. launch of YORVIPATH® significantly impacts Ascendis Pharma's revenue growth trajectory in 2025 and beyond. As of February 7, 2025, YORVIPATH® had 908 prescriptions, indicating a robust start to its commercialization in the U.S. market. This early success is expected to contribute to the company's revenue growth in 2025 and beyond, as more patients adopt the medication and healthcare providers become familiar with its benefits. Additionally, the drug's full-year 2024 revenue of €28.7 million demonstrates its potential to generate substantial revenue for the company in the coming years.

The key factors driving the anticipated submission of the TransCon™ CNP NDA for achondroplasia in children in Q1 2025 are:

1. Successful Pre-NDA Meeting with FDA: Ascendis Pharma held a pre-NDA meeting with the FDA, which provided a clear path for the submission of the NDA. This meeting likely addressed any potential concerns or requirements, ensuring a smoother regulatory process.
2. Positive Clinical Trial Results: The company presented new data demonstrating significant improvements in leg bowing, a common complication in achondroplasia, observed with TransCon CNP compared to worsening observed with placebo in the pivotal ApproaCH Trial. These positive results support the efficacy and safety of TransCon CNP, strengthening the NDA submission.
3. Market Need and Potential: Achondroplasia is the most common form of short-limbed dwarfism, affecting approximately 1 in 25,000 live births. The current standard of care has limitations, creating a significant market need for a differentiated treatment like TransCon CNP. Approval of TransCon CNP could position Ascendis Pharma as a leader in the treatment of achondroplasia, potentially expanding its market share and revenue.

Regulatory approval of TransCon CNP could significantly enhance Ascendis Pharma's market position by:

1. Expanding Product Portfolio: Approval would add a third product to Ascendis Pharma's portfolio, diversifying its revenue streams and reducing reliance on SKYTROFA and YORVIPATH.
2. Strengthening Market Leadership: As a differentiated treatment for achondroplasia, TransCon CNP could capture a significant share of the market, further solidifying Ascendis Pharma's position as a leader in the Endocrinology Rare Disease sector.
3. Enhancing Stakeholder Value: Approval could lead to increased investor confidence, potentially driving up the company's stock price and attracting new investment opportunities.

The SKYTROFA® full year 2024 revenue, excluding sales deductions related to prior years, was approximately €202 million. This figure reflects the product's strong market performance and competitive position in the U.S. growth hormone market. Here are some specific points to support this analysis:

1. Market Share Increase: SKYTROFA®'s U.S. volume (mg) increased by 84% in 2024 year-over-year, resulting in an estimated 6.5% market share of the total U.S. growth hormone market for 2024. This indicates that the product is gaining traction and becoming more popular among healthcare providers and patients.
2. Revenue Growth: The €202 million in revenue represents a significant increase from the previous year, demonstrating the product's growing demand and market acceptance. This growth is also attributable to the product's single indication in pediatric GHD, which has proven to be a successful market strategy.
3. Competitive Position: SKYTROFA® has achieved a leading position in value in the U.S. growth hormone market. Its revenue growth and increasing market share suggest that it is successfully competing with other growth hormone therapies in the market. The product's unique features, such as its long-acting formulation and convenient dosing schedule, may contribute to its competitive edge.
4. PDUFA Goal Date: The Prescription Drug User Fee Act (PDUFA) goal date for FDA review of the supplemental BLA for the treatment of adults with growth hormone deficiency is July 27, 2025. Pending approval, a U.S. commercial launch is planned for the fourth quarter of 2025. This upcoming milestone further solidifies SKYTROFA®'s position in the market and its potential for future growth.

In conclusion, Ascendis Pharma's strategic roadmap and key milestones for 2025 highlight the company's potential for rapid revenue growth linked to the launch of its first two Endocrinology Rare Disease medicines, SKYTROFA and YORVIPATH, and the planned filings for its third product, TransCon CNP for achondroplasia. With a strong cash balance and established partnerships, Ascendis Pharma is well-positioned to become a leading global biopharmaceutical company with multiple blockbuster products and a strong engine for future innovation.