Ascendis Pharma A/S Announces Mean Bone Mineral Density (BMD) Data from Phase 2 PaTH Forward Trial Demonstrating Continued Normalization and Stabilization of BMD Z-scores Between 26 and 58 Weeks
– Data from subjects with available dual energy x-ray absorptiometry (DXA) scans demonstrated stabilization of BMD, in alignment with observed bone turnover markers previously reported –
“HP patients, compared to people with normal parathyroid function, have insufficient parathyroid hormone (PTH) levels and low rates of PTH-driven skeletal remodeling, resulting in above-average bone mineral density and potentially an increased risk of fractures and other negative health effects,” said
About the Week 58 Analysis
BMD was measured with non-invasive DXA, a low-radiation exposure technology widely used to identify individuals with bone fracture risk. Results were read and reported by a central lab.
|Mean Bone Mineral Density Z-scores by DXA*|
|Anatomic region||n||Baseline||Week 26||Week 58|
|Lumbar spine (L1-L4)||42||1.6||1.0||0.9|
|Forearm/ 1/3 radius||41||0.3||0.3||0.3|
*From central lab reading
About TransCon™ PTH1
TransCon PTH is an investigational once-daily long-acting prodrug of parathyroid hormone (PTH[1-34]) in development as a treatment for adult hypoparathyroidism (HP), designed to restore PTH at physiologic levels for 24 hours each day to address both the short-term symptoms and long-term complications of the disease. TransCon PTH has been granted Orphan drug status in
About Hypoparathyroidism (HP) 2,3,4,5,6,7
HP is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH) which plays a critical role in controlling systemic calcium, phosphate, and calcitriol (active vitamin D) levels and is essential to many key biological functions. HP affects approximately 400,000 patients in
HP remains among the few hormonal insufficiency states without an approved replacement therapy that restores the missing hormone at physiologic levels. Standard of care with active vitamin D analogs and calcium supplementation does not fully control the disease and may contribute to risk of renal disease. As a result, patients with HP have an estimated 4-fold to 8-fold greater risk of renal disease compared to healthy controls.
Ascendis is headquartered in
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) Ascendis’ design of TransCon PTH as a once daily injection to restore physiologic levels of PTH, 24-hours a day; (ii) Ascendis’ belief that BMD data observed indicate that TransCon PTH has the potential to be able to normalize calcium metabolism in the body over time; (iii) Ascendis’ ability to apply its platform technology to build a leading, fully integrated biopharma company, (iv) Ascendis’ product pipeline and expansion into additional therapeutic areas and (v) Ascendis’ expectations regarding its ability to utilize its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers to supply TransCon hGH, the SKYTROFA® Auto-Injector and other study drug for commercial sales in the
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1 Karpf DB, et al. J Bone
2 Mannstadt M, et al. Nature Reviews 2017, 3: 17055
3 Ascendis Pharma HP Patient Experience Research.
4 Hadker N, et al. Endo Pract. 2014, 20(7);671-679.
5 Powers J, et al. J Bone
6 Mitchell DM, et al. J Clin Endocrinol Metab 2012, 97(12): 4507-4514
7 Underbjerg L, et al. J Bone
Source: Ascendis Pharma