ACP-015

Ascendis Pharma Submits NDA for Novel Achondroplasia Treatment with Multiple Clinical Benefits

2 months ago

• Ascendis Pharma has submitted a New Drug Application to the FDA for TransCon CNP (navepegritide), a once-weekly treatment for children with achondroplasia that demonstrated benefits beyond linear growth. • The application is supported by robust clinical data from three randomized, double-blind, placebo-controlled trials with up to three years of open-label extension data showing improved muscle function and reduced leg bowing. • Achondroplasia affects over 250,000 people globally with complications including spinal deformities, muscle weakness, and respiratory issues, highlighting the significant unmet medical need this therapy aims to address.

Ascendis Pharma Faces Revenue Shortfall, Pipeline Advances Temper Outlook

6 months ago

• Ascendis Pharma's Skytrofa revenue missed estimates due to increased sales deductions, leading to a downward revision of FY24 revenue guidance. • Upcoming milestones include Phase 2 data for Turner syndrome and the initiation of a basket study for other growth hormone indications in 2025. • Regulatory submissions for Ascendis Pharma's C-Type Natriuretic Peptide (CNP) product are on track, with Phase 3 data appearing competitive. • A strategic collaboration with Novo Nordisk offers long-term upside, while the U.S. launch of Yorvipath is anticipated in mid-January.

Ascendis Pharma Announces Progress Across Endocrinology and Oncology Programs in Q3 2024

6 months ago

• 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 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. • A collaboration with Novo Nordisk was established to develop treatments for metabolic and cardiovascular diseases, including a once-monthly GLP-1 receptor agonist.

TransCon CNP Demonstrates Superior Growth in Achondroplasia Trial

8 months ago

• TransCon CNP met its primary endpoint, showing a statistically significant improvement in annualized growth velocity (AGV) compared to placebo at Week 52 (p<0.0001). • Children aged 5-11 years treated with TransCon CNP showed a greater change from baseline AGV compared to placebo, with a statistically significant difference of 1.78 cm/year (p<0.0001). • The trial indicated that TransCon CNP was generally well-tolerated, with a low incidence of mild injection site reactions and a safety profile comparable to placebo. • Ascendis Pharma plans to submit regulatory applications to the FDA in Q1 2025 and to the EMA in Q3 2025, seeking approval for TransCon CNP as a treatment for achondroplasia.

Ascendis Pharma Gains Optimism with Potential Drug Launches and Analyst Upgrade

9 months ago

• Oppenheimer upgraded Ascendis Pharma to Outperform, projecting strong sales for Yorvipath and potential in TransCon CNP. • Yorvipath, a hypoparathyroidism treatment, anticipates a U.S. launch by Q1 2025, driven by positive feedback from key opinion leaders. • Phase 3 trial results for TransCon CNP in achondroplasia are forthcoming, with market sentiment undervaluing its potential impact. • Despite recent sales challenges with Skytrofa, Ascendis aims for approval of all three product candidates by the end of 2025.

Ascendis Pharma Reports Second Quarter 2024 Financial Results and Updates on Clinical Trials

9 months ago

Ascendis Pharma has reported its second quarter 2024 financial results, highlighting progress in its clinical trials for TransCon CNP and oncology programs, alongside a financial update and outlook based on current plans.

Ascendis Pharma Unveils Vision 2030 Strategic Roadmap

a year ago

Ascendis Pharma A/S introduces Vision 2030, a strategic roadmap aiming for blockbuster status for multiple products and expanding future innovation. Key updates include progress on TransCon™ hGH, TransCon PTH, and TransCon CNP, alongside plans for global expansion and new therapeutic areas.

Ascendis Pharma A/S Updates on Vision 3x3 Strategy and 2022 Milestones

3 years ago

Ascendis Pharma A/S provided an update on its Vision 3x3 strategic roadmap and key milestones for 2022 at the 40th Annual J.P. Morgan Healthcare Conference. The company highlighted its progress in 2021, including the U.S. launch of SKYTROFA®, and outlined its plans for clinical data readouts and regulatory submissions across its endocrinology rare disease and oncology portfolios in 2022.

Drug Development Updates