Ascendis Presents TransCon™ PTH Data at the American Thyroid Association Annual Meeting
– At Week 26, 81% of patients in Phase 3 PaTHway Trial with chronic post-surgical hypoparathyroidism, the most common cause of this disease, achieved independence from conventional therapy while maintaining normocalcemia following treatment with TransCon PTH
– Post-surgical patients treated with TransCon PTH self-reported significant improvements in disease-related symptoms, physical functioning, and daily life compared to those on placebo
– Results for post-surgical patients (n=70) comparable to overall PaTHway Trial population (n=82)
TransCon PTH is an investigational prodrug with sustained release of active parathyroid hormone (PTH [1-34]) administered once daily. TransCon PTH (palopegteriparatide) has received a positive CHMP opinion recommending approval in the
“We are pleased to share with thyroid surgeons and thyroidologists attending ATA this data demonstrating the safety and benefits of TransCon PTH seen in clinical trial patients whose disease arose following neck surgeries,” said
PaTHway is a Phase 3 trial with a completed 26-week randomized, double-blind, placebo-controlled period, followed by an ongoing 156-week open-label extension period, designed to evaluate the efficacy, safety, and tolerability of TransCon PTH in adults with hypoparathyroidism. A total of 84 patients were randomized 3:1 to treatment with TransCon PTH (n = 63) or placebo (n = 21), both co-administered with conventional therapy. The multi-component primary efficacy endpoint of PaTHway was defined as normocalcemia (a normal concentration of calcium in the blood; 8.3–10.6 mg/dL), independence from conventional therapy (no active vitamin D and ≤ 600 mg/day calcium), and no TransCon PTH dose change within the 4 weeks prior to Week 26. Key secondary endpoints included patient-reported assessments of disease impact using the Hypoparathyroidism Patient Experience Scale (HPES) and the 36-
PaTHway Trial Sub-Analysis Highlights
Across multiple evaluated parameters in participants with post-surgical hypoparathyroidism, treatment with TransCon PTH enabled independence from conventional therapy (no active vitamin D and ≤ 600 mg/day calcium); improvement of hypoparathyroidism-specific symptoms and their impact on daily life and physical functioning; and normalization of mean 24-hour urine calcium excretion. This includes:
- At Week 26, 81% of patients with postsurgical hypoparathyroidism treated with TransCon PTH in the trial achieved independence from conventional therapy while maintaining normocalcemia compared to 6% treated with placebo.
- At Week 26, 81% (42/52) of patients with chronic post-surgical hypoparathyroidism treated with TransCon PTH demonstrated significant improvement compared to those on placebo in disease-related symptoms and the impact of hypoparathyroidism on physical functioning and daily life (all p < 0.01).
- Health-related quality of life, as measured by the SF-36 physical functioning subscale score, improved significantly with TransCon PTH treatment compared to placebo in participants with post-surgical hypoparathyroidism (p = 0.0063).
- From baseline to Week 26, patients with postsurgical hypoparathyroidism treated with TransCon PTH in the trial showed a normalization and greater reduction in urine calcium excretion than those on placebo.
- Treatment with TransCon PTH was generally safe and well-tolerated. Most treatment-emergent adverse events (TEAEs) were mild or moderate, and no patients discontinued study treatment or the trial due to a treatment-related TEAE.
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) the potential value of TransCon PTH, if approved, as a treatment option for adults with hypoparathyroidism; (ii) the ability of TransCon PTH to provide stable PTH levels within the physiological range 24 hours/day; (iii) Ascendis’ ability to achieve its goal of providing a treatment option that can help patients overcome the serious health and quality of life burdens seen with hypoparathyroidism; (iv) Ascendis’ plans to continue to advance TransCon PTH globally as a candidate to address the needs of the hypoparathyroidism patient community; (v) the potential approval of TransCon PTH in the
|Investor Contacts:||Media Contact:|
|+1 (650) 374-6343||+1 (650) 709-8875|
|+1 (415) 513-1284|
Source: Ascendis Pharma