Ascendis Pharma A/S Announces Top-Line Results from Week 84 of Its Phase 2 PaTH Forward Trial Demonstrating that TransCon™ PTH Provided Durable Benefit and Was Well-Tolerated in Adults with Hypoparathyroidism
–– 58 of the original 59 subjects continue in the open-label extension portion of the Phase 2 trial.
–– Phase 3 data for TransCon PTH expected in Q1 2022.
“The continued durable improvements observed in this study over a year and a half of treatment with TransCon PTH highlight the importance of sustained restoration of parathyroid hormone to normal physiological levels,” said Professor
After an initial screening and baseline assessment period, patients in the Phase 2 PaTH Forward Trial were randomized to blinded treatment with TransCon PTH at 15, 18, or 21 μg/day or placebo for 4 weeks, followed by a switch to open label treatment, during which physicians could optimize dosing of TransCon PTH to meet individual treatment objectives.
Key Findings at Week 84 of the Phase 2 PaTH Forward Trial:
- 58 out of the 59 original trial participants continued open-label treatment with TransCon PTH.
- Mean serum calcium levels remained stable and in the normal range.
- All study subjects discontinued active vitamin D supplements in the earliest weeks of the trial and have remained off it since then. In addition, 93% of study subjects were taking calcium supplements <600 mg/day.
- Mean urinary calcium excretion remained stable and in the normal range.
- TransCon PTH was well-tolerated at all doses administered. No treatment-related serious or severe adverse events occurred, and no treatment-emergent adverse events (TEAEs) led to discontinuation of study drug.
“We believe that these data, combined with nearly all patients continuing in the open label portion of the trial, indicates the potential for TransCon PTH to become the first hormone therapy to replace active vitamin D and therapeutic doses of calcium for adults living with hypoparathyroidism,” said
The Investor presentation on the Investor & Media section of the
About TransCon PTH 1
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). TransCon PTH is 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 designation in
About Hypoparathyroidism (HP) 2,3,4,5,6,7
Hypoparathyroidism (HP) is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH), resulting in low calcium and elevated phosphate levels in the blood. HP affects approximately 200,000 patients worldwide, most of whom develop the condition following damage to or accidental removal of the parathyroid glands during thyroid surgery. Conventional treatment with calcium and active vitamin D (also called calcitriol) does not effectively address the short-term symptoms, long-term complications, or quality-of-life impacts of hypoparathyroidism.
Short-term symptoms include weakness, severe muscle cramps (tetany), abnormal sensations such as tingling, burning and numbness (paresthesia), memory loss, impaired judgment, and headache. Patients often experience decreased quality of life and, over the long term, this complex disorder can increase risk of major complications, such as calcium deposits in the brain, blood vessels, eye and other soft tissues – including the kidneys, which can lead to impaired renal function.
Current standard of care with active vitamin D analogs and calcium supplementation do not fully control the disease and may contribute to risk of renal disease. Patients with HP have an estimated 4-fold to 8-fold greater risk of renal disease compared to healthy populations. The disease is also associated with a 2-fold increased risk of depression or bipolar disorder compared to healthy populations
HP remains among the few hormonal insufficiency states without a replacement therapy that restores the missing hormone at physiologic levels.
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 expected timing of top-line results from the Phase 3 Phase 3 PaTHway Trial, (ii) Ascendis’ plan to submit an NDA to the FDA, (iii) Ascendis’ ability to apply its platform technology to build a leading, fully integrated biopharma company, and (iv) Ascendis’ use of 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 and distributors 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 Miner Res. 2020; x:1-11.
2 Mannstadt M, et al. Nature Reviews 2017, 3: 17055
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