Ascendis Pharma A/S (ASND) Earnings

Management highlights

### Core Business and Long-Term Strategy - The company is positioned as a leading global biopharma, driven by its differentiated Transcon technology platform and three approved commercial products. Management expects approved products to drive growth for the next 10-15 years, with the platform enabling at least one new NCE-based IND annually to support long-term, self-sustaining growth and expansion into new therapeutic areas beyond growth disorders and hypoparathyroidism. ### UroPath (Transcon PTH) for Hypoparathyroidism - UroPath is the first and only approved treatment addressing the underlying cause of hypoparathyroidism, and is available commercially or via named patient programs in over 35 countries. New patient demand remained robust in the U.S. during Q2, with broadening physician adoption. - New long-term clinical data presented in Q2 shows sustained 82-86% response rates across multiple organ systems, improved quality of life, and 95% patient retention after five years of treatment, supporting its adoption as a standard of care. The company is advancing label expansion to include adolescents aged 12-18, higher U.S. doses, and development of a once-weekly formulation for stable-dose patients. ### UVWell (Transcon CMP) for Achondroplasia - UVWell launched commercially in the U.S. in Q2 2026, with over 220 enrolled patients and more than 65% reimbursement approval by the end of July 2026. Uptake is coming from three patient groups: those switching from existing therapy, patients returning to treatment after discontinuing prior therapy, and first-time treatment-naive patients, leading to overall expansion of the achondroplasia market. - Long-term clinical data shows durable improvements in growth, bone proportionality, and a favorable safety profile. Regulatory approval in the EU is expected in Q4 2026, and early access programs are active in select international markets using U.S. FDA approval. Enrollment for the 0-2 year old age cohort trial completed faster than expected, and additional trials are ongoing for adults with achondroplasia and children with hypochondroplasia. ### Combination Therapy for Achondroplasia - The combination of Transcon CMP and Transcon Growth Hormone has a strong scientific rationale, with 78-week COAST trial data showing sustained efficacy without safety compromises. A Phase III combination trial is set to begin enrollment later in 2026, and management expects this combination to establish a new treatment standard for achondroplasia. ### Skytropha (Transcon Growth Hormone) for Growth Hormone Deficiency - Skytropha remains the number one long-acting growth hormone by brand value in the U.S., with over 20,000 unique enrolled patients. A Phase 3 basket trial is ongoing for label expansion into ISS, SDA, and Turner syndrome, with planned geographic expansion alongside UVWell. ### Partnership Pipeline - Partners are advancing multiple Transcon-based candidates: the once-monthly Transcon semaglutide program for metabolic disorders/obesity with Novo Nordisk continues to progress, and partner Onconis recently initiated a first-in-human clinical trial for an anti-VEGF Transcon-based therapy for wet AMD.

Guidance

- Management reaffirms its target of reaching 5 billion Euro in total revenue by 2030, with a long-term goal of exceeding 10 billion Euro in revenue in subsequent decades supported by new Transcon product launches.

Segment performance

Total product revenue for Q2 2026 was 315 million Euro, more than doubling year-over-year. Total revenue (including 24 million Euro in non-product collaboration revenue) was 339 million Euro. Breakdown by product segment: - UroPath: 252 million Euro in revenue, accounting for 80% of total product revenue. It reached blockbuster status on a run-rate basis in its second full year of U.S. launch, with steady new patient demand in the U.S. and continued growth internationally. - Skytropha: 55 million Euro in revenue, accounting for 17.5% of total product revenue, with increased demand in the U.S. including sales to a collaboration partner. - UVWell (Transcon CMP): 8 million Euro in revenue, accounting for 2.5% of total product revenue. This was its first commercial quarter on the U.S. market, with strong early demand and rapid conversion to paid therapy.

Risks & headwinds

- There is an ongoing intellectual property dispute (ITC case) with BioMarin over UVWell in the U.S. The relevant patent was fully invalidated in Europe, so the dispute only impacts the U.S. market. A first judge opinion is expected in August 2026, with a full ITC decision expected in December 2026. Management states that even in a worst-case outcome, the dispute will not have a material impact on the company's overall business or its ability to hit 2030 targets, and notes there is strong public interest in keeping the product available to U.S. patients.

Analyst Q&A

• Q: Analysts asked for an update to full-year 2026 operating cash flow guidance (originally guided as greater than 500 million Euro prior to the PRV sale) and an estimate of how much UVWell enrollment comes from market expansion versus patient switches. /

  A: Management reaffirmed the greater than 500 million Euro 2026 operating cash flow guidance (in Euro) and declined to set an upper bound given the early stage of the UVWell launch. Management confirmed they do not track exact split of new UVWell patients by origin, but stated the strong demand indicates most new patients come from previously untreated or lapsed patients, supporting the conclusion that UVWell is expanding the overall achondroplasia market.

• Q: What are the potential outcomes of the ongoing ITC patent dispute over UVWell with BioMarin, what is the worst-case risk of payments to Biomarin, and is a settlement a reasonable possibility? /

  A: The relevant patent has already been fully invalidated in Europe, so the dispute only applies to the U.S. The ITC process is ongoing, with the first ruling expected in August 2026 and a final decision in late 2026. Management reaffirmed the dispute will not have any material impact on the company's business or 2030 targets, and noted that there is strong U.S. public interest in keeping this high unmet need product available. Management stated it is flexible and will always act in the best interest of patients, leaving the door open to a potential settlement.

• Q: Management noted the TransCon platform targets one new NCE IND annually, but there has been no IND yet in 2026. What are the go-forward plans for the early pipeline? /

  A: Management clarified that two NCE TransCon assets advanced by partners (one with Onconis, one with Novo Nordisk) will enter clinical trials in 2026, which counts toward the annual NCE goal. The platform will continue to deliver at least one new NCE for clinical development annually, supporting long-term sustainable growth for the company without reliance on acquisitions of unwanted assets.

• Q: Can you confirm the consistency of UroPath's Q2 growth, and clarify what "consistent with prior quarters" guidance means for the second half of 2026? /

  A: Management stated that the key performance indicators (including steady ~1000 new U.S. patients per quarter) shared in prior quarters remain consistent, and analysts have all the necessary data to model the remainder of 2026 accurately. Management aims to provide transparent guidance that allows analysts to model correctly, rather than providing lowball numbers to beat expectations.

• Q: What is the size of the treatable hypochondroplasia patient population for the upcoming Phase III trial? /

  A: Management explained that broader genetic testing is reclassifying many patients previously diagnosed with idiopathic short stature (ISS) as hypochondroplasia if they have an FGFR3 mutation, expanding the diagnosed population beyond the historically small phenotypically defined severe patient pool. The trial design reflects this updated classification framework.