Long-Term Benefits of TransCon® PTH Therapy in Adults Unveiled

Recent Findings on TransCon® PTH (Palopegteriparatide) Therapy
New research highlights the sustained effectiveness of TransCon PTH in managing hypoparathyroidism in adults, showcasing findings from Week 214 of the ongoing Phase 2 PaTH Forward Trial. This study is pivotal for understanding the long-term benefits of the treatment on patients’ health outcomes. Presented by Dr. Andrea Palermo from a leading university during a major endocrinology congress, the data provide valuable insights into this therapeutic approach.
Study Overview and Key Results
The PaTH Forward Trial began with a structured approach that included a rigorous 4-week randomized phase before transitioning to an open-label extension. This design aimed to evaluate the ongoing benefits and safety profile of TransCon PTH beyond the initial treatment period. Among the 59 participants initially enrolled, an impressive 95% continued through the trial, indicating strong retention and interest in the treatment.
Positive Outcomes on Serum Calcium Levels
After 214 weeks of treatment, a remarkable 98% of participants showed normal albumin-adjusted serum calcium levels, which is crucial for managing hypoparathyroidism effectively. Furthermore, 93% of these individuals reported independence from conventional therapy, meaning they required less than 600 mg of calcium daily and did not need active vitamin D, demonstrating the profound impact of TransCon PTH on their condition.
Bone Health and Kidney Function Enhancement
The study also tracked essential markers of bone health, revealing that bone turnover markers increased and peaked around week 26, then stabilized well above baseline measurements through week 214. This stabilization is critical for ensuring the ongoing health of bones under treatment. Notably, kidney function improved significantly in over two-thirds of the participants, indicating a clinically meaningful increase in estimated glomerular filtration rate (eGFR) from baseline.
Safety and Tolerability of TransCon® PTH
Throughout the study, TransCon PTH was well-tolerated, with no new safety concerns emerging. Most treatment-emergent adverse events (TEAEs) were reported as mild or moderate. This consistency in safety enhances the treatment's appeal as a long-term option for managing hypoparathyroidism.
About Hypoparathyroidism and Its Challenges
Hypoparathyroidism poses significant health challenges due to insufficient parathyroid hormone levels, which regulates calcium and phosphate in the body. Patients often face a variety of complications, from cognitive challenges to renal issues. With the majority of cases being post-surgical, innovative treatments like TransCon PTH are critical for improving patient outcomes and quality of life.
About Ascendis Pharma A/S
Ascendis Pharma is dedicated to transforming patient care through innovative therapies, and its TransCon technology stands at the forefront of this mission. With a focus on enhancing patient health through science and passion, Ascendis continues to develop therapies aimed at addressing significant medical needs. The company operates out of Copenhagen, Denmark, showcasing a commitment to global healthcare advancements.
Frequently Asked Questions
What is TransCon PTH Therapy?
TransCon PTH is an innovative therapy aimed at treating individuals with hypoparathyroidism, helping to normalize their calcium levels and improve overall health.
How long was the duration of the PaTH Forward Trial?
The trial consisted of a structured treatment approach, with the open-label phase extending to 214 weeks, allowing for detailed monitoring of patient outcomes.
What were the key findings of the study regarding kidney function?
Key findings included a significant increase in eGFR in over two-thirds of the participants, indicating improved kidney function among those treated with TransCon PTH.
Was TransCon PTH well-tolerated during the trial?
Yes, the therapy was generally well-tolerated, with adverse events reported predominantly as mild or moderate, affirming its safety for long-term use.
How does hypoparathyroidism affect patients?
This condition can lead to severe complications, including renal issues, cognitive impairments, and neuromuscular difficulties, making effective treatment essential.
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