Ascendis Pharma (ASND) Goldman Sachs 45th Annual Global Healthcare Conference summary

Strategic pipeline and product updates

• Focus on rare disease endocrinology with three main products leveraging TransCon technology, aiming for global commercialization in up to 50 countries within 3-4 years.

• SKYTROFA, the leading growth hormone product, has surpassed 10,000 patients and is targeting blockbuster status through market access and label expansion, including adult growth hormone deficiency and Turner syndrome.

• YORVIPATH (TransCon PTH) approved in Europe, with successful launch in Germany and plans for direct presence in 12-14 countries by year-end; U.S. FDA review extended to August 14, 2024.

• TransCon CNP pivotal data expected later this year, targeting achondroplasia and aiming to address both growth and comorbidities.

• Oncology pipeline progressing, with promising initial IL-2 data presented at ASCO and further updates expected by end of 2024.

Commercial performance and market dynamics

• SKYTROFA revenue guidance for 2024 is €320–340 million, with operating expenses at €600 million and a goal of quarterly cash flow break-even.

• German launch of YORVIPATH is progressing as planned, with rapid physician adoption and expected acceleration as Natpara supply ends.

• U.S. EAP for TransCon PTH is limited to a subpopulation but shows strong patient retention and benefit, mirroring clinical trial results.

• Market opportunity for PTH therapy in the U.S. estimated at 80,000–100,000 patients, with initial focus on high-burden subgroups but potential for broader penetration depending on health economics.

• Growth hormone market is $3 billion, with adult segment underpenetrated at 5–10%, presenting significant expansion potential.

Regulatory and development milestones

• FDA extended PDUFA date for TransCon PTH to August 14, 2024, with ongoing labeling discussions; no further NDA status updates until material developments occur.

• SKYTROFA previously experienced a similar regulatory delay due to the complexity of combination products.

• Phase III data for TransCon CNP and further oncology updates anticipated by year-end, with regulatory pathways to be determined based on data.

• Expansion into new indications and combination therapies, such as growth hormone plus CNP for catch-up growth in achondroplasia, are in clinical development.

• Ongoing global commercialization efforts include direct launches and distribution agreements for both SKYTROFA and YORVIPATH.