Ascendis Pharma Announces Progress Across Endocrinology and Oncology Programs in Q3 2024
核心洞察
Ascendis Pharma's YORVIPATH is set to launch in the U.S. by mid-January 2025, with significant revenue growth reported ex-U.S. in Q3 2024.
The company plans to submit an NDA for TransCon CNP to the FDA in Q1 2025 for treating achondroplasia (搜索) in children, followed by an MAA submission to the EMA in Q3 2025.
SKYTROFA revenue reached €47.2 million in Q3 2024, with full-year revenue expected to be between €200-€220 million, excluding prior sales deductions.
Ascendis Pharma A/S (Nasdaq: ASND) has announced its financial results for the third quarter of 2024, highlighting significant advancements across its endocrinology and oncology programs. The company is preparing for the U.S. launch of YORVIPATH, anticipates regulatory submissions for TransCon CNP, and reports continued growth in SKYTROFA revenue.
YORVIPATH Launch and Expansion
YORVIPATH (palopegteriparatide) is set to launch in the U.S. with product availability expected by mid-January 2025. Ascendis Pharma's expanded U.S. field teams are engaging with healthcare providers, and the Ascendis Signature Access Program will begin accepting prescriptions and enrolling patients in December. In the third quarter, YORVIPATH revenue outside the U.S. reached €8.5 million, marking a 60% sequential quarter-over-quarter increase, driven by growing physician and patient demand, with over 600 patients currently on treatment.
TransCon CNP for Achondroplasia
Ascendis Pharma is advancing its TransCon CNP (navepegritide) program for achondroplasia (搜索). Following positive topline data from the pivotal ApproaCH Trial in children aged 2-11 years, the company plans to submit a New Drug Application (NDA) to the FDA in the first quarter of 2025. A Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) is planned for the third quarter of 2025. Topline Week 26 data from the COACH trial, evaluating the combination of TransCon hGH and TransCon CNP in children with achondroplasia, are expected in the second quarter of 2025.
SKYTROFA Performance and Growth
SKYTROFA (lonapegsomatropin) revenue for the third quarter of 2024 totaled €47.2 million, compared to €47.0 million in the same period in 2023. Volume growth was offset by higher sales deductions and a negative adjustment to prior period sales deductions of €2.5 million. For the first nine months of 2024, SKYTROFA revenue reached €138.5 million, a 21% year-over-year increase compared to €114.4 million in 2023. Ascendis Pharma submitted a supplemental Biologics License Application (sBLA) to the FDA for TransCon hGH for treating adults with growth hormone deficiency (搜索). Topline results from the Phase 2 New InsiGHTS Trial in Turner syndrome (搜索) are expected in the fourth quarter of 2024. The company anticipates initiating a basket trial evaluating SKYTROFA in other established daily growth hormone indications in the first half of 2025.
Strategic Collaboration with Novo Nordisk
In November, Ascendis Pharma entered a strategic collaboration with Novo Nordisk A/S, granting an exclusive, multi-product, worldwide license to the TransCon technology platform for developing, manufacturing, and commercializing Novo Nordisk proprietary products in metabolic diseases and a product-by-product exclusive license in cardiovascular diseases. The lead program is a once-monthly GLP-1 receptor agonist product candidate initially targeting obesity (搜索) and type 2 diabetes (搜索). Ascendis Pharma is eligible to receive up to $285 million in upfront, development, and regulatory milestone payments for the lead program, plus sales-based milestone payments and escalating tiered, mid-single-digit royalties on global net sales. Novo Nordisk will be responsible for Ascendis’ early development costs, as well as clinical development, regulatory, commercial manufacturing, and commercialization activities.
Financial Position
As of September 30, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €625.5 million, compared to €399.4 million as of December 31, 2023. Full-year 2024 SKYTROFA revenue, excluding sales deductions related to prior years, is expected to be between €200-€220 million. Total operating expenses for 2024 are projected to be approximately €600 million.