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

Financial performance and outlook

• Reported full-year 2025 net product revenue of $1.86 billion, with Elevidys contributing $899 million and PMOs $966 million.

• Ended 2025 with $954 million in cash and cash equivalents, and expects to remain cash flow positive throughout the decade.

• Non-GAAP profit for 2025, excluding Arrowhead transactions, was about $400 million, marking the second consecutive year of profitability.

• Restructured convertible debt, eliminating significant debt overhang for the decade and maintaining an untapped $600 million revolver.

• FY 2025 product revenue estimated at €683M; gross margin ~87%; operating expenses ~€762M; December 2025 cash balance ~€616M; 2026 operating cash flow expected at ~€500M, excluding TransCon CNP contributions; €120M share repurchase program planned for 2026.

Commercial strategy and product updates

• Elevidys has treated over 1,100 patients, with 80% of the ambulatory population still untapped, and a $500 million annual revenue floor reaffirmed.

• Major 2026 initiative is to communicate Elevidys' efficacy, highlighting disease-slowing data from pivotal trials and long-term studies.

• 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.

• PMO therapies (Exondys, Vyondys, Amondys) have over a decade of use, strong safety, high compliance, and real-world evidence of slowing disease progression.

Pipeline and R&D initiatives

• Advancing a next-generation siRNA pipeline targeting muscle and CNS diseases, leveraging Arrowhead's TRiM platform for superior tissue targeting and safety.

• FSHD and DM1 programs use integrin-targeted siRNA for muscle, aiming for best-in-class efficacy and safety.

• CNS programs use a monovalent TFR1 FAB-siRNA construct for blood-brain barrier penetration, with promising preclinical Huntington's data showing >75% knockdown in deep brain regions.

• TransCon platform enables differentiated products and new patents, supporting a robust pipeline in endocrinology, oncology, ophthalmology, and metabolic diseases.

• Key 2026 milestones include biomarker and safety data for FSHD and DM1, first patient dosing in the Huntington's program, and FDA/EMA decisions for TransCon CNP.