Ascendis Pharma A/S (NASDAQ:ASND) shares rose on Friday after the company reported five-year results from its Phase 2 PaTH Forward trial, showing sustained efficacy and safety for TransCon PTH in adults with hypoparathyroidism.

Key Highlights

  • Ascendis Pharma shares rose following five-year PaTH Forward trial results.
  • 82% of patients met a composite response endpoint at Week 266.
  • 96% of patients achieved independence from active vitamin D therapy.
  • No new safety signals were identified over the five-year follow-up period.

Ascendis Pharma A/S (NASDAQ:ASND) shares advanced in Friday trading after the company reported five-year results from its Phase 2 PaTH Forward trial, demonstrating that TransCon PTH, known as palopegteriparatide, delivered sustained efficacy and safety in adults with hypoparathyroidism.

The PaTH Forward trial enrolled 59 adults with hypoparathyroidism, of whom 56 completed the full five-year study period. TransCon PTH is currently approved as YORVIPATH in the United States, the European Union, the European Economic Area, and certain other markets for the treatment of adult hypoparathyroidism patients.

According to the company, the therapy continued to replicate the effects of endogenous parathyroid hormone, supporting stable calcium control, kidney health, bone health, and quality of life, while enabling most patients to discontinue conventional treatment approaches.

Data from Week 266 showed that 82% of patients met a composite response endpoint that included maintaining normal serum calcium levels, avoiding active vitamin D, and limiting calcium supplementation to less than 600 mg per day. Additionally, 88% of patients maintained normal albumin-adjusted serum calcium levels, while 96% achieved independence from active vitamin D therapy. Nearly all patients, at 95%, were able to remain off therapeutic doses of calcium supplements.

The study also demonstrated durable kidney benefits. Mean estimated glomerular filtration rate increased by 9.4 mL per minute per 1.73 square meters from baseline and remained elevated through the full five years of treatment. Mean 24-hour urine calcium levels fell substantially, normalized within 26 weeks, and stayed within the normal range through Week 266.

Beyond clinical markers, patients reported lasting improvements in symptoms, daily functioning, and cognitive well-being over the course of the study, reinforcing the therapy's broader quality-of-life benefits.

On safety, the company said TransCon PTH was generally well tolerated, with no new safety signals identified during the five-year follow-up. Most treatment-emergent adverse events were mild to moderate in severity, and no patient discontinued treatment due to the study drug.

The long-term data adds to a growing body of evidence supporting TransCon PTH's role as a treatment option for hypoparathyroidism, a rare endocrine disorder that requires lifelong management.