Ascendis Pharma A/S (Nasdaq: ASND) has announced positive three-year results from its Phase 2 PaTH Forward Trial, demonstrating the sustained benefits of TransCon™ PTH (palopegteriparatide) in adults with hypoparathyroidism. The data, presented at the American Society for Bone and Mineral Research (ASBMR) 2024 annual meeting, reveal that long-term treatment with the investigational drug normalized bone remodeling, addressing a key concern in patients with this endocrine disorder.
The trial, involving 57 adults with hypoparathyroidism, showed that treatment with TransCon™ PTH over 162 weeks led to bone remodeling rates within the normal range. Hypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH), leading to low bone remodeling rates, accumulation of overly mature bone, and potentially compromised bone quality.
Key Findings from the PaTH Forward Trial
The study's lead author, Dr. Mishaela Rubin, Associate Professor of Medicine at Columbia University, presented the findings at ASBMR 2024. The data indicated that palopegteriparatide promotes skeletal health parameters similar to those observed in individuals with sufficient parathyroid function. This is particularly significant as hypoparathyroidism often results in reduced bone turnover and increased risk of fractures.
TransCon PTH is designed as a once-daily prodrug of parathyroid hormone (PTH [1-34]), engineered to provide continuous PTH exposure over a 24-hour period. This sustained release mechanism aims to mimic the physiological action of PTH, thereby improving calcium and phosphate balance in the body.
Implications for Hypoparathyroidism Treatment
Hypoparathyroidism, resulting from deficient PTH levels, affects the balance of calcium and phosphate, impacting bone and kidney function. The majority of cases, approximately 70-80%, are a result of post-surgical complications, while other causes include autoimmune and idiopathic factors. Patients may experience neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment.
The positive outcomes from the PaTH Forward Trial suggest that TransCon PTH could offer a significant advancement in the management of hypoparathyroidism, potentially mitigating the long-term complications associated with the disease. The normalization of bone remodeling observed in the study indicates an improvement in overall bone health, which is a critical factor in preventing fractures and maintaining skeletal integrity.
Ascendis Pharma is leveraging its TransCon technology platform to develop new therapies. The company is headquartered in Copenhagen, Denmark, with facilities in Europe and the United States.
