Ascendis reports sustained benefits in hypoparathyroidism trial

COPENHAGEN - Ascendis Pharma A/S (NASDAQ:ASND), a biopharmaceutical company with a market capitalization of $9.1 billion, announced today new data from its Phase 2 PaTH Forward Trial, indicating long-term treatment with TransCon PTH (palopegteriparatide) continues to show a durable response in adults with hypoparathyroidism. The stock has experienced an 11% decline over the past week, though InvestingPro analysis suggests the company is currently undervalued based on its Fair Value model. The findings were presented at the joint congress of the European Society for Paediatric Endocrinology and the European Society of Endocrinology.

The trial, which reached Week 214, included an initial 4-week randomized, double-blind, placebo-controlled period, followed by an ongoing open-label extension through Week 266. Renal function, bone turnover markers, and bone mineral density were among the parameters assessed. Despite showing promising clinical results, InvestingPro data reveals the company is not currently profitable, with analysts projecting continued losses for the fiscal year 2025.

At this stage, 95% of the original 59 patients remained in the trial. Notably, 98% maintained normal albumin-adjusted serum calcium levels, and 93% were independent from conventional therapy. Bone turnover markers showed an increase from baseline, peaked by Week 26, and then stabilized above baseline levels through Week 214. Bone mineral density stayed within age- and sex-matched norms.

Most participants experienced a clinically meaningful increase in estimated glomerular filtration rate (eGFR) from baseline, with improvements observed as early as Week 4. TransCon PTH was generally well-tolerated, with no new safety concerns arising; treatment-emergent adverse events (TEAEs) were mostly mild or moderate.

Dr. Aimee Shu, Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma, expressed satisfaction with the ongoing safety profile and treatment durability, highlighting sustained normalization of skeletal dynamics and significant, sustained improvements in kidney function.

Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone, leading to complications such as renal issues and cognitive impairment. The majority of cases are post-surgical, with autoimmune and idiopathic causes also identified.

Ascendis Pharma focuses on developing new therapies with its TransCon technology to address unmet medical needs. The company, headquartered in Denmark, operates globally with additional facilities in Europe and the United States. With revenue growth of 12% in the last twelve months and a strong gross profit margin of 85%, the company continues to expand its market presence. For deeper insights into Ascendis Pharma’s financial health and growth prospects, including additional ProTips and comprehensive analysis, visit InvestingPro.

This report is based on a press release statement and contains forward-looking statements that involve risks and uncertainties. Actual results may differ materially from those projected in the forward-looking statements.

In other recent news, Ascendis Pharma’s financial performance and strategic developments have garnered attention from several analysts. The company reported first-quarter results that exceeded expectations, with revenue from its product Yorvipath™ reaching €44.7 million, about 50% higher than anticipated by JPMorgan. This strong performance led JPMorgan to raise its price target for Ascendis Pharma to $245, maintaining an Overweight rating. Similarly, Evercore ISI increased its price target to $280, citing robust prescription growth and a successful transition to paid drug conversions as key factors in the company’s strong quarterly performance.

RBC Capital Markets began coverage on Ascendis Pharma with an Outperform rating and a $205 price target, highlighting the company’s advantageous market position and potential peak sales for its treatments. UBS also maintained a Buy rating with a $196 target, projecting strong performance for the Yorvipath launch and anticipating an increase in patient uptake. Cantor Fitzgerald reaffirmed its Overweight rating with a $200 target, emphasizing upcoming results from the Phase 2 COACH trial as a potential catalyst.

These developments reflect a positive outlook from analysts, with expectations of continued growth and market success for Ascendis Pharma. Investors are closely watching for the company’s next quarterly report, which is expected to provide further insights into its financial trajectory. The company’s strategic initiatives and product portfolio are seen as key drivers of its future performance.

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