Ascendis Pharma A/S Q2 FY2025 earnings call

Management Statement and Operational Highlights

• YORVIPATH: Strong global launch with EUR 103M in Q2 revenue. In the U.S., over 1,500 prescribers and ~3,100 unique patients, with majority payer approval within 3 months. Ongoing clinical program for label expansion, e.g., PaTHway 60 trial in older children.
• TransCon CNP: FDA granted priority review, interim Phase II results of combination trial with TransCon growth hormone showed boost in linear growth and improved body proportionality with safety/tolerability consistent with monotherapy. Plan to start Phase III study of combination therapy in children with achondroplasia by end of 2025 and initiate pivotal combination trial in hypochondroplasia.
• SKYTROFA: FDA approved for adult growth hormone deficiency, Q2 revenue EUR 51M, with expected long-term growth from label expansion. Phase III basket trial planned later this year for various growth disorder indications.
• Financials: Total Q2 product revenue EUR 153.7M, R&D costs decreased to EUR 72M, SG&A expenses increased to EUR 107.6M due to global commercial expansion. Cash position ended Q2 at EUR 494M, with close to cash breakeven for the quarter.

Segment Performance

• YORVIPATH: Q2 revenue reached EUR 103 million, more than double of Q1 despite currency headwinds. In the U.S., over 1,500 prescribers wrote prescriptions for around 3,100 unique patients, with the majority of U.S. patients receiving payer approval within 3 months. Outside the U.S., steady revenue growth continues with expectations of further acceleration as reimbursement becomes available in additional Europe Direct countries.
• SKYTROFA: Q2 revenue was EUR 51 million. It is established as a high-value brand for pediatric growth hormone deficiency, and recently received FDA approval for adult growth hormone deficiency, with further label expansion planned to drive long-term growth.

Guidance

• Expect continued revenue growth driven by YORVIPATH's global launch. For SKYTROFA, sequential revenue growth to track prescription growth, offset by payer mix and seasonality, with label expansion driving long-term growth. YORVIPATH is seen as having potential for multiple billions in peak sales annually, focusing on building long-term leadership. In the U.S., strong demand and enrollment trends for YORVIPATH, with expectation of continued uptake and reimbursement dynamics.
• For the remainder of 2025, YORVIPATH's launch progressing exceptionally well, with expectations of steady sequential revenue growth globally. SKYTROFA's recent adult approval expected to contribute modestly to 2025 growth, with focus on label expansion for long-term growth.

Risks

• Currency Impact: Potential impact of euro-U.S. dollar exchange rate on reported revenue.
• Regulatory Risks: Uncertainties related to approval timelines and label expansions for product candidates.
• Tariffs and MFN: Potential impact of trade policies, though company believes well-positioned to mitigate such impacts due to flexible operations and import/export strategies within the U.S.

Q: Jessica Fye from JPMorgan asked about continuation of enrollment trends and number of unique patients enrolled.

A: Jan Moller Mikkelsen and Jay Wu discussed steady state growth in enrollments, early stage of launch, and focus on conversion from enrollment to paid prescriptions.

Q: Derek Archila from Wells Fargo inquired about improving the 3-month time from enrollment to conversion.

A: Jan Moller Mikkelsen and Jay Wu discussed payer education, experienced team in managed care, and streamlining processes with specialty pharmacies to improve conversion time.

Q: Tazeen Ahmad from Bank of America asked about the split of patients between severe and moderate hypoparathyroidism.

A: Jan Moller Mikkelsen explained there's no medical definition of severity for hypoparathyroidism, and guidelines focus on broad elements for PTH treatment.

Q: Yaron Werber from TD Cowen asked about Europe revenue expectations and sequential normalized growth in the U.S.

A: Jan Moller Mikkelsen confirmed Europe revenue expectations align with Q1 guidance, and early stage of YORVIPATH's U.S. launch makes it too early to predict long-term sequential growth.

Q: Gavin Clark-Gartner from Evercore ISI asked about conversion rate from enrollment forms to paid drugs and reporting enrollment forms for YORVIPATH.

A: Jan Moller Mikkelsen and Jay Wu discussed difficulty in giving exact conversion rates, focus on helping patients get reimbursed, and plan to continue reporting enrollment forms as necessary for modeling.

Q: Joseph Schwartz from Leerink Partners asked about YORVIPATH's prescribers and TransCon CNP's competitor profile.

A: Jay Wu discussed YORVIPATH's reach across target prescribers, and Jan Moller Mikkelsen commented on TransCon CNP's unique profile and combination therapy's holistic benefit vs. competitors' approaches.

Q: Eliana Merle from UBS asked about TransCon CNP's label indication for achondroplasia and IP landscape for weekly CNP.

A: Jan Moller Mikkelsen discussed TransCon CNP's strong clinical data and benefit beyond linear growth, and confidence in IP due to early development and filings.

Q: Kelly Shi from Jefferies asked about YORVIPATH's patient pull-through and compliance.

A: Jan Moller Mikkelsen discussed low discontinuation rates in Europe, indicating strong patient compliance and addressing unmet medical need.

Q: Paul Choi from Goldman Sachs asked about TransCon CNP's label submission timeline and patient population addressed.

A: Jan Moller Mikkelsen discussed ongoing trial for higher doses, aim to get faster access to patients, and focus on addressing patients needing higher doses than current U.S. label allows.

Q: Leland Gershell from Oppenheimer asked about shift in focus to hypochondroplasia.

A: Jan Moller Mikkelsen explained reallocation of patients from ISS to hypochondroplasia, combination therapy's benefit for heterogenic population, and patient organization input driving focus.

Q: David Lebowitz from Citi asked about evolution of YORVIPATH's market size expectation.

A: Jan Moller Mikkelsen stated belief YORVIPATH could achieve multiple billions in peak sales annually.

Q: Yun Zhong from Wedbush asked about TransCon CNP's launch timeline post-FDA approval and hypochondroplasia combination vs. monotherapy.

A: Jan Moller Mikkelsen discussed waiting for FDA approval to determine launch timeline, and belief combination therapy is best for hypochondroplasia's heterogenic population.

Q: Maxwell Skor from Morgan Stanley asked about pivotal trial for TransCon CNP combination and FDA/EMA feedback.

A: Jan Moller Mikkelsen discussed expectations of a successful pivotal trial and ongoing dialogue with regulatory agencies regarding combination therapy approval.