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COPENHAGEN - Ascendis Pharma A/S (NASDAQ:ASND), a $10.6 billion biotech company whose shares have surged 33% over the past six months, announced new three-year data from its Phase 3 PaTHway Trial showing that TransCon PTH (palopegteriparatide) provided sustained benefits for adults with hypoparathyroidism regardless of disease cause. According to InvestingPro data, the company maintains an impressive 85% gross profit margin despite being in its growth phase.
According to data presented at the ENDO 2025 conference in San Francisco, patients completing 156 weeks of treatment showed improvements in biochemical markers, kidney function, and quality of life measures.
The trial retention rate was high, with 89% of the original 82 participants completing the 3.5-year study. At Week 156, 88% of patients maintained normal albumin-adjusted serum calcium levels, while 96% no longer required conventional therapy, defined as taking less than 600 mg/day of calcium and no active vitamin D.
Kidney function improvements were maintained through Week 156, with mean estimated glomerular filtration rate (eGFR) increasing by 8.76 mL/min/1.73 m² across all participants. Patients with compromised kidney function at baseline (eGFR
"These responses, including normalization of skeletal dynamics with significant and clinically meaningful improvements in kidney function, demonstrate the long-term benefits of this treatment option for the vast majority of adults with hypoparathyroidism," said Aimee Shu, Executive Vice President and Chief Medical Officer at Ascendis Pharma.
The treatment was generally well-tolerated with no new safety concerns identified during the extended treatment period. Most treatment-emergent adverse events were mild or moderate, with no serious adverse events related to the study drug. This positive development aligns with growing analyst optimism, as InvestingPro reports three analysts have recently revised their earnings expectations upward for the upcoming period.
Hypoparathyroidism is an endocrine disorder caused by insufficient parathyroid hormone, which regulates calcium and phosphate balance. The condition can lead to serious complications including neuromuscular irritability, renal issues, and cognitive impairment.
The information in this article is based on a press release statement from Ascendis Pharma. While the company’s revenue grew 12% in the last twelve months, InvestingPro analysis reveals additional key metrics and insights available through their comprehensive Pro Research Report, one of 1,400+ detailed company analyses designed to help investors make informed decisions.
In other recent news, Ascendis Pharma has been the focus of several analyst reports highlighting its ongoing developments. Morgan Stanley assumed coverage with an Overweight rating, citing the promising commercial trajectory of Yorvipath and the potential of the company’s TransCon pipeline. UBS maintained its buy rating, noting strong demand for Yorvipath in the hypoparathyroidism market, with fewer reimbursement hurdles than expected. Oppenheimer raised its price target to $224, based on strong early demand for Yorvipath, as indicated by a survey of endocrinologists.
TD Cowen reiterated a Buy rating, highlighting positive data from the Phase 2 COACH trial involving achondroplasia treatment, which supports potential growth beyond current estimates. RBC Capital also maintained an Outperform rating, with a survey indicating steady growth for Yorvipath, predicting a significant opportunity for expansion in the patient market. These developments underscore the focus on Yorvipath’s performance and the anticipation of upcoming trial data. Analysts have expressed optimism about Ascendis Pharma’s future, with some noting strategic interests such as the recent deal with Novo Nordisk. Investors are watching closely as the company continues to navigate its promising pipeline and market opportunities.
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