Ascendis Pharma Releases Week 104 Data for TransCon CNP
Ascendis Pharma announced new radiographic data from Week 104 from the completed pivotal ApproaCH Trial of once-weekly TransCon CNP in children with achondroplasia. In the trial, TransCon CNP-treated children demonstrated continued improvements in lower extremity alignment through up to two years of treatment, including improvements in tibial-femoral angle. As previously reported, improvements in annualized growth velocity were maintained and ACH-specific height Z-score increased with TransCon CNP treatment through Week 104. Through up to two years of treatment, TransCon CNP was generally well tolerated, with a low rate of ISRs, no symptomatic hypotension, and no acceleration of bone age. Most adverse events in TransCon CNP-treated children were mild or moderate, with none leading to treatment discontinuation or withdrawal from the trial.
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- Clinical Trial Results: The completed ApproaCH trial demonstrated that children treated with TransCon CNP showed continued improvements in lower extremity alignment through Week 104, indicating the drug's potential to reduce chronic pain and surgical needs associated with skeletal dysplasia.
- Significant TFA Improvement: The average tibial-femoral angle (TFA) in all children treated with TransCon CNP decreased from 9.1° at baseline to 6.9° at Week 104, reflecting a mean absolute change of -2.2°, with even greater improvements observed in children with baseline TFA ≥ 5°, underscoring the drug's efficacy.
- Good Safety Profile: Over the two-year treatment period, TransCon CNP was generally well tolerated, with a low incidence of injection site reactions and no serious adverse events such as accelerated bone age, indicating a high safety profile for pediatric use.
- Future Outlook: TransCon CNP has received FDA approval for promoting growth in children with achondroplasia, and its marketing authorization application is under review in Europe, which is expected to further enhance market acceptance and utilization of the drug.
- Data Presentation: Ascendis Pharma will present pivotal trial data for YUVIWEL at the 2026 International Conference on Children's Bone Health, which is expected to attract industry attention and enhance the company's market position in rare endocrine diseases.
- Clinical Trial Results: In a study involving 84 children with achondroplasia, YUVIWEL demonstrated a mean annualized growth velocity of 5.71 cm over 52 weeks, significantly outperforming the placebo group by 1.78 cm, indicating strong therapeutic efficacy that may enhance product acceptance.
- Combination Therapy Study: The COACH trial revealed that the combination of YUVIWEL and SKYTROFA achieved annualized growth velocities of 8.80 cm in treatment-naïve children, showcasing the potential of combination therapy to improve growth and body proportionality, which may attract more patients to this treatment option.
- Strong Financial Performance: Ascendis Pharma reported total revenue of €246.6 million in Q1, a 143% increase from €100.95 million in the same period in 2025, primarily driven by SKYTROFA sales, demonstrating the company's competitiveness in the rapidly growing skeletal dysplasia market.
- Inclusion in Russell Indexes: Ascendis Pharma (ASND) announced on Friday that it will be added to the Russell 3000, Russell 1000, Russell 2500, and Russell Midcap indexes as part of the 2026 Russell U.S. Indexes annual reconstitution, effective after the close of trading on June 26, which is expected to enhance its market visibility and liquidity.
- Automatic Style Index Inclusion: The inclusion in the Russell 1000, Russell 2500, and Russell Midcap indexes will also result in automatic inclusion in the corresponding growth and value style indexes, potentially attracting more institutional investors and driving stock price appreciation.
- Market Reaction Expectations: This inclusion is likely to trigger positive reactions from investors, particularly among index funds and ETFs that follow passive investment strategies, which is expected to increase demand for Ascendis Pharma shares and enhance its market performance.
- Long-Term Growth Potential: With a unique product portfolio in the endocrine space and ongoing clinical data supporting its growth potential, Ascendis Pharma is poised to achieve higher revenues and profitability as market attention increases.
- Inclusion in Russell Indexes: Ascendis Pharma A/S has been added to the Russell 3000®, Russell 1000®, Russell 2500®, and Russell Midcap® indexes, marking an increase in the company's market recognition and is expected to attract more investor attention.
- Market Coverage Expansion: The Russell 3000® index tracks the largest 3,000 U.S. stocks, representing approximately 98% of investable U.S. equities, and Ascendis' inclusion will provide greater exposure within a broad investment portfolio.
- Benchmark Asset Impact: As of December 31, 2024, approximately $11.8 trillion in assets are benchmarked against Russell U.S. indexes, and Ascendis' inclusion may enhance its stock liquidity and market participation, thereby increasing the company's overall valuation.
- Strategic Implications: The inclusion not only boosts Ascendis' visibility among investors but may also facilitate future financing and collaboration opportunities, further driving the company's innovative development in the biopharmaceutical sector.
- Sustained Long-term Efficacy: In the three-and-a-half-year PaTHway trial, TransCon PTH demonstrated an 86% response rate for the multi-component endpoint, successfully replicating endogenous parathyroid hormone actions and significantly improving patients' quality of life.
- Independence from Conventional Therapy: The trial showed that 100% of patients achieved independence from active vitamin D, and 96% maintained calcium intake below 600 mg/day, indicating TransCon PTH's effective replacement of traditional therapies.
- Improved Kidney Function: At Week 182, the mean estimated glomerular filtration rate (eGFR) reached 80.2 mL/min/1.73 m², reflecting an increase of 11.0 mL/min/1.73 m² from baseline, showcasing TransCon PTH's positive impact on renal function.
- Good Safety Profile: Throughout the trial, TransCon PTH was well-tolerated with no new safety signals identified, and all adverse events were mild or moderate, ensuring patient safety during treatment.
- Sustained Efficacy: Ascendis Pharma's TransCon PTH demonstrated sustained efficacy in the five-year Phase 2 trial, with 82% of patients achieving normal serum calcium levels by Week 266 without active vitamin D, indicating its potential as a new standard of care for hypoparathyroidism.
- Kidney Function Improvement: The trial showed a sustained increase in mean eGFR from baseline, reflecting the therapy's effectiveness in improving calcium levels while countering age-related kidney function decline, thereby enhancing patients' quality of life.
- Stable Bone Density: Bone mineral density scores were corrected early and remained stable over five years, highlighting TransCon PTH's long-term benefits for bone health and reinforcing its position as a new standard treatment for chronic hypoparathyroidism.
- Good Safety Profile: The therapy was generally well tolerated with no new safety signals identified; most adverse events were mild or moderate, and no patients discontinued treatment, demonstrating the safety and efficacy of TransCon PTH.








