Ascendis Pharma (ASND) 44th Annual J.P. Morgan Healthcare Conference summary

Key financial and operational highlights

• Reported 2025 net product revenue of $1.86 billion, with Elevidys at $899 million and PMOs at $966 million; Q4 revenue exceeded expectations at $370 million.

• Ended 2025 with $954 million in cash and cash equivalents, and no significant debt overhang for the decade after restructuring convertible debt.

• Maintains a $500 million annual revenue floor for Elevidys, with plans to significantly exceed it in 2026.

• Anticipates being cash flow positive throughout the decade, with an untapped $600 million revolver for strategic opportunities.

• Stress tests indicate the ability to meet debt obligations even under adverse scenarios, such as loss of PMO revenue.

Strategic vision and growth targets

• Aims to achieve over €5B annual revenue by 2030, focusing on blockbuster status for multiple products and expanding innovation in endocrinology rare diseases and other therapeutic areas.

• Plans to remain independent and profitable through lean operations and a strong innovation algorithm.

• Pursues accelerated oncology approvals and partnerships to maximize value in large indications.

Commercial and product strategy

• Elevidys has treated over 1,100 patients, with 80% of the ambulatory population still untapped; focus in 2026 is on communicating efficacy and disease-slowing benefits.

• Initiatives include expanding the sales force, launching a promotional campaign, and engaging the patient community.

• Non-ambulatory patient access to Elevidys depends on results from the ENDEAVOR Cohort 8 trial, expected at year-end.

• YORVIPATH approved for adult hypoparathyroidism, with commercial launches in 30+ countries and expansion planned by end of 2026.

• SKYTROFA approved for pediatric and adult growth hormone deficiency, with label expansions and global rollout ongoing.