Ascendis Pharma A/S Announces Presentations for TransCon™ PTH and TransCon™ CNP at the American Society for Bone & Mineral Research 2021 Annual Meeting
– Late-breaking oral presentation will feature a comprehensive review of 58-week results from the company’s Phase 2 PaTH Forward Trial –
“We are excited to partner with our investigators to share the latest data showing continued advances in our hypoparathyroidism and achondroplasia development programs,” said
“Over 58 weeks in PaTH Forward, the data from study subjects show continued trends toward normal calcium homeostasis in the absence of standard of care therapies,” said
|Sustained Benefit of TransCon PTH, an Investigational Hormone Replacement Therapy for Adult Patients with Hypoparathyroidism, at Week 58 in the PaTH Forward Trial
||Late-Breaking Oral Presentations: Clinical Treatment
|Oral presentation by
|Baseline Demographics of the ACHieve Study: A Five-Year, Multi-National Observational Cohort Study of Children with Achondroplasia
||Virtual Plenary Poster Presentation
|A Single Dose of TransCon PTH in Subjects with Impaired Renal Function:
||Poster Session I
|A Phase 1 Trial|
|Ascendis-Sponsored CME Programs||Date/Time|
|Hypoparathyroidism: Changing the Treatment Landscape
|Virtual presentation with live Q&A|
|Achondroplasia: Beyond Short Stature
|Virtual presentation with live Q&A|
Hypoparathyroidism (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.
Achondroplasia is the most common form of dwarfism, affecting approximately 250,000 people worldwide. Individuals living with achondroplasia may experience severe skeletal complications and comorbidities. For example, abnormal development of the vertebra can lead to sleep apnea, chronic back and leg pain from lower spine impingement and sudden infant death from cervical compression. Chronic ear infections due to abnormal eustachian tubes can lead to hearing loss and speech delay.7
The condition is caused by an autosomal dominant activating mutation in the fibroblast growth factor receptor 3 (FGFR3) gene that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways. Preclinical and clinical data show that the CNP pathway stimulates growth. Increased CNP counteracts the effects of the FGFR3 mutation downstream, thus promoting bone growth.8
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) whether physiological parathyroid hormone replacement could help address the unmet needs these patients face, (ii) Ascendis’ ability to apply its platform technology to build a leading, fully integrated biopharma company, (iii) Ascendis’ product pipeline and expansion into additional therapeutic areas and (iv) 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 Mannstadt M, et al. Nature Reviews 2017, 3: 17055
2 Ascendis Pharma HP Patient Experience Research.
3 Hadker N, et al. Endo Pract. 2014, 20(7);671-679.
4 Powers J, et al. J Bone
5 Mitchell DM, et al. J Clin Endocrinol Metab 2012, 97(12): 4507-4514
6 Underbjerg L, et al. J Bone
7 Ireland PJ, Pacey V, et al. Appl Clin Genet. 2014;7: 117–25.
8 Horton WA, et al.
Source: Ascendis Pharma