Ascendis Pharma A/S Q3 FY2024 earnings call

Management Statement and Operational Highlights

• SKYTROFA: Strong fundamentals with Q3 volume growth; focus on label expansions like adult growth hormone deficiency and a basket trial in established growth hormone indications.
• YORVIPATH: Preparing for US launch in mid-January 2025, with an expanded commercial infrastructure. Estimates 70,000-90,000 adults in US with hypoparathyroidism who could benefit.
• TransCon CNP: Pivotal trial results support leadership in achondroplasia market; plan to submit NDA to FDA Q1 2025 and MAA to EMEA Q3 2025.
• Novo Collaboration: Validation of TransCon platform, lead program is a once-monthly GLP-1 targeting obesity and type 2 diabetes.
• Oncology: Positive results in platinum-resistant ovarian cancer cohort of Phase I/II trial, prioritizing TransCon IL-2 beta/gamma.

Segment Performance

• SKYTROFA: Third quarter 2024 volume increased over 60% year-over-year. Reported revenue in Q3 2024 was EUR 47.2 million (vs EUR 47 million in Q3 2023). First nine months of 2024 SKYTROFA revenue totaled EUR 138.5 million, a 21% year-over-year increase. Full-year 2024 guidance for SKYTROFA revenue, excluding sales deductions related to prior years, is EUR 200 million to EUR 220 million.
• YORVIPATH: Third quarter outside the US revenue increased over 60% to EUR 8.5 million. Preparing for US launch in mid-January 2025, with expanded commercial infrastructure.
• TransCon CNP: Pivotal trial results showed positive growth and other endpoints relevant to achondroplasia. Plan to submit NDA to FDA in Q1 2025 and MAA to EMEA in Q3 2025.
• Novo Nordisk Collaboration: Upfront payment of USD 100 million, with tiered mid-single-digit royalties on global net sales of TransCon products along with development, regulatory, and sales milestones.

Guidance

• Full-year 2024 SKYTROFA revenue expected to be EUR 200 million to EUR 220 million, excluding sales deductions related to prior years.
• Goal to achieve cash flow breakeven by the end of 2025.
• Plan to submit TransCon CNP NDA to FDA in Q1 2025 and MAA to EMEA in Q3 2025.

Risks

• Competition in the growth hormone market with players like Lilly and Genentech exiting, which could impact SKYTROFA's market share.
• Regulatory uncertainties related to product approvals for TransCon CNP and other product candidates.
• Dependence on successful launches and label expansions for SKYTROFA, YORVIPATH, and TransCon CNP to reach blockbuster status.

Q: Jessica Fye of JPMorgan asks about confidence in retaining YORVIPATH patients and if cash flow breakeven by end of 2025 is still the expectation.

A: Jan Mikkelsen discusses YORVIPATH being a true replacement therapy; Scott Smith confirms the cash flow breakeven goal remains.

Q: Tazeen Ahmad of Bank of America Securities inquires about TransCon CNP NDA preparation.

A: Jan Mikkelsen states the integrated data packet is ready for submission to regulatory agencies.

Q: Yaron Werber of TD Cowen asks about YORVIPATH supply and NATPARA inventory timing.

A: Jan Mikkelsen comments on NATPARA inventory running out and expected transition to YORVIPATH.

Q: Gavin Clark-Gartner of Evercore asks about PTH experienced patients in US and uptake timeline for YORVIPATH.

A: Jan Mikkelsen talks about PTH experienced patients and expected uptake of YORVIPATH.

Q: Joseph Schwartz of Leerink Partners asks about YORVIPATH launch comparison to Europe and serum calcium monitoring deterrents.

A: Jan Mikkelsen addresses serum calcium monitoring not being a significant deterrent and differences in US vs European launch curves.

Q: Vikram Purohit of Morgan Stanley asks about Novo collaboration progress and oncology pipeline milestones.

A: Jan Mikkelsen discusses Novo collaboration and oncology pipeline focus on platinum-resistant ovarian cancer.

Q: Kelly Shi of Jefferies confirms SKYTROFA Q4 sales and TransCon CNP NDA submission data requirements.

A: Scott Smith confirms Q4 sales guidance; Jan Mikkelsen states 12-month data is sufficient for TransCon CNP NDA submission.

Q: David Lebowitz of Citi asks about long-acting GLP-1 challenges and SKYTROFA price evolution.

A: Jan Mikkelsen discusses long-acting GLP-1 tolerability and SKYTROFA price stability.

Q: Paul Choi of Goldman Sachs asks about Novo collaboration development timeline and royalty recognition.

A: Jan Mikkelsen agrees 505(b)(2) pathway shortens development, but timelines referred to Novo Nordisk.

Q: Leland Gershell of Oppenheimer asks about European commercialization cadence for YORVIPATH and TransCon CNP.

A: Jan Mikkelsen discusses European commercialization plans for YORVIPATH and TransCon CNP.

Q: Alexander Thompson of Stifel asks about YORVIPATH supply readiness and TransCon CNP basket study plans.

A: Jan Mikkelsen confirms YORVIPATH supply readiness and plans for TransCon CNP basket trial in CNP-related diseases