Ascendis Pharma reports growth in achondroplasia trial

COPENHAGEN - Ascendis Pharma A/S (NASDAQ:ASND), a biopharmaceutical company with a market capitalization of $9.3 billion and impressive gross profit margins of 85.3%, has announced positive results from its ApproaCH Trial, investigating the efficacy of TransCon CNP (navepegritide) in children with achondroplasia, a genetic bone growth disorder. According to InvestingPro analysis, the company’s stock is currently trading near its Fair Value, while maintaining strong revenue growth of 12.06% over the last twelve months. Data from Week 52 of the trial, presented at the joint congress of the European Society for Paediatric Endocrinology and the European Society of Endocrinology, showed improvements in growth and bone structure among participants.

The trial involved 84 children aged 2-11, randomized to receive either the treatment or a placebo for a year, followed by an open-label extension period. TransCon CNP, a prodrug of C-type natriuretic peptide administered weekly, aims to provide continuous exposure to active CNP, which affects growth plates and skeletal muscle. While the company operates with a moderate level of debt, InvestingPro data reveals analyst consensus remains strongly bullish, with price targets ranging from $191 to $286 per share.

At the one-year mark, the treatment group demonstrated a significant increase in annualized growth velocity compared to the placebo group, without any treatment-related serious adverse events, symptomatic hypotension, fractures, or accelerated bone age. Additionally, improvements were noted in lower limb alignment, proportional growth, and spinal canal dimensions.

Achondroplasia is caused by a variant in the FGFR3 gene and affects over 250,000 people globally. It leads to disproportionate dwarfism and can cause a range of health issues, including spinal deformities and chronic pain, often necessitating multiple surgeries.

Ascendis Pharma, a biopharmaceutical company, focuses on developing new therapies using its TransCon technology platform. The company’s Chief Medical Officer, Dr. Aimee Shu, expressed optimism about the potential of TransCon CNP to provide benefits beyond linear growth and to possibly reduce future complications associated with achondroplasia.

The findings from the ApproaCH Trial support the continued investigation of TransCon CNP as a treatment for children with achondroplasia, aiming to improve their growth outcomes and overall quality of life. Ascendis Pharma plans to further collaborate with medical experts and the achondroplasia community to understand the long-term implications of modifying the progression of skeletal dysplasia from childhood.

This report is based on a press release statement from Ascendis Pharma. For comprehensive analysis and additional insights, including 6 more exclusive ProTips and detailed financial metrics, visit InvestingPro, where you can access the full Pro Research Report covering what really matters about Ascendis Pharma’s business fundamentals and future prospects.

In other recent news, Ascendis Pharma has reported impressive first-quarter results, with revenue from its product Yorvipath™ reaching €44.7 million, significantly exceeding JPMorgan’s estimates. This performance has led JPMorgan to raise its price target for Ascendis Pharma to $245, maintaining an Overweight rating. Similarly, Evercore ISI has increased its price target to $280, citing strong prescription growth and successful transitions to paid drug conversions as key factors. RBC Capital Markets has also initiated coverage with an Outperform rating and a $205 price target, based on the potential for Yorvipath to achieve annual sales of €3 billion.

Additionally, Cantor Fitzgerald has reaffirmed its Overweight rating with a $200 price target, highlighting the upcoming results of the Phase 2 COACH trial in Achondroplasia as a potential catalyst. The trial results are expected to show promising efficacy, potentially impacting the company’s valuation positively. Meanwhile, Ascendis Pharma has announced new data from its Phase 2 PaTH Forward Trial for hypoparathyroidism, showing sustained benefits and a durable response with its TransCon PTH therapy. These developments underscore Ascendis Pharma’s continued momentum and strategic positioning in the biopharmaceutical sector, attracting significant attention from investors and analysts alike.

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