Ascendis Pharma (ASND) investor relations material

Strategic overview and product pipeline

• TransCon technology underpins internal and external product development, including collaborations in metabolic diseases and obesity.

• Three independent products target major unmet medical needs, with label expansion and new chemical entities planned over the next 3–4 years.

• Revenue is increasing quarter by quarter, leading to financial independence and positive cash generation.

• Eight to ten product opportunities are in development, with a focus on rare disease endocrinology and future expansion into cardiovascular diseases.

• Commercial infrastructure is 90% established globally, with further expansion tied to additional country launches.

Yorvu Path launch and market strategy

• Yorvu Path targets hypoparathyroidism with strong IP protection through 2042 and a combination product design for long-term durability.

• U.S. market expected to drive initial revenue, with ex-U.S. growth accelerating after 4–5 years as more countries come online.

• Commercialization ex-U.S. follows a three-pillar strategy: direct markets, distribution agreements, and partnerships, with Japan launch imminent.

• Uptake is driven by steady new prescribers and improved prescription-to-treatment infrastructure, with future growth expected from expanding prescriber base.

• Barriers to treatment are low due to user-friendly delivery devices, and adherence rates are high.

Competitive landscape and product differentiation

• Yorvu Path faces limited competition, with no comparable products in clinical development offering similar benefits.

• Decision to launch with a daily rather than weekly formulation was based on safety, titration needs, and patient stability.

• Once-weekly options may be considered for stable patients, but daily remains optimal for most.

• TransCon CNP offers continuous exposure, reducing injection site reactions and hypertension risk, and shows benefits beyond linear growth, such as improved muscle strength and quality of life.

• Differentiation is recognized by patients and physicians, with priority review status and plans for standard-of-care positioning.