Ascendis Pharma A / S Announces Mean Bone Mineral Density (BMD) Data from Phase 2 PaTH Forward Trial Demonstrating Further Normalization and Stabilization of BMD Z Scores Between 26 and 58 Weeks
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– Data from subjects with available dual energy x-ray absorptiometry (DXA) scans demonstrated stabilization of BMD, in alignment with previously reported observed bone remodeling markers –
COPENHAGEN, Denmark, September 22, 2021 (GLOBE NEWSWIRE) – Ascendis Pharma A / S (Nasdaq: ASND), a biopharmaceutical company using its innovative TransCon ™ technologies to potentially create new treatments to make a significant difference in the lives of patients, today announced 58-week BMD data from a central lab reading of the PaTH Forward trial, a global Phase 2 trial of its investigational product candidate TransCon PTH in adult subjects with hypoparathyroidism (HP) .
“HP patients, compared to people with normal parathyroid function, have insufficient parathyroid hormone (PTH) levels and low rates of PTH-induced skeletal remodeling, resulting in above average bone mineral density and potentially increased risk of fractures and other negative health effects. Said Aimee Shu, MD, Ascendis Pharma vice president of clinical development, Endocrine Medical Sciences. “We designed TransCon PTH as a single daily injection to restore physiological levels of PTH, 24 hours a day. In PaTH Forward, the mean BMD Z scores, which parallel serum markers of bone turnover, tended towards stabilization and normalization continues at 58 weeks. We believe this is an indicator that TransCon PTH has the potential to normalize calcium metabolism in the body over time.
About the Week 58 AnalysisBMD was measured with non-invasive DXA, a low-exposure radiation technology widely used to identify people at risk for bone fractures. The results were read and reported by a central laboratory.
|Z scores of mean bone mineral density by DXA *|
|Anatomical region||m||Reference||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 the reading of the central laboratory
About TransCon™ PTH1TransCon PTH is a long-acting, once-daily, investigational prodrug of parathyroid hormone (PTH[1-34]) in development as a treatment for hypoparathyroidism (HP) in adults designed to restore PTH to physiological levels for 24 hours a day to treat both short-term symptoms and complications long-term illness. TransCon PTH has been granted orphan drug status in the United States, European Union and Japan.
About hypoparathyroidism (HP) 2,3,4,5,6,7HP is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH) which plays an essential role in controlling systemic levels of calcium, phosphate and calcitriol (vitamin D active) and is essential for many key biological functions. HP affects approximately 400,000 patients in the United States, Europe, Japan, South Korea and Greater China, the majority of whom develop the disease as a result of injury or accidental removal of the parathyroid glands during thyroid surgery. Patients often experience a decrease in their quality of life. Short-term symptoms include weakness, severe muscle cramps (tetany), abnormal sensations such as tingling, burning and numbness (paraesthesia), memory loss, poor judgment, and headache. In the long term, this complex disorder can increase the risk of major complications, such as extraskeletal calcium deposits occurring in the brain, lens, and kidneys, which can lead to kidney failure.
HP remains among the few states of hormonal insufficiency without approved replacement therapy that restores the missing hormone to physiological levels. Standard of care with active vitamin D analogues and calcium supplementation does not completely control the disease and may contribute to the risk of kidney disease. As a result, patients with HP have an estimated 4 to 8 times greater risk of kidney disease than healthy controls.
About Ascendis Pharma A / SAscendis Pharma applies its innovative platform technology to create a leading fully integrated biopharmaceutical company focused on improving the lives of patients. Guided by its core values of patients, science and passion, the company uses its TransCon technologies to create new therapies potentially best in class.
Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Heidelberg and Berlin, Germany; Palo Alto and Redwood City, California; and Princeton, New Jersey. Please visit www.ascendispharma.com to learn more.
Forward-looking statementsThis press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical fact, included in this press release regarding the future operations, plans and objectives of the management of Ascendis are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) the design of Ascendis of TransCon PTH as a daily injection to restore physiological levels of PTH, 24 hours a day; (ii) Ascendis’ belief that the observed BMD data indicate that TransCon PTH has the potential to normalize calcium metabolism in the body over time; (iii) Ascendis ‘ability to apply its platform technology to build a leading fully integrated biopharmaceutical company, (iv) Ascendis’ product pipeline and its expansion into additional therapeutic areas and (v) Ascendis’ expectations regarding its ability to use its TransCon technologies to create new, potentially best-in-class therapies. Ascendis may not carry out the plans, realize the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on such 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 Ascendis’ forward-looking statements, including: reliance on third party manufacturers to supply TransCon hGH, the SKYTROFA® autoinjector and other United States and clinical studies; unanticipated safety or efficacy results in its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to the commercialization of lonapegsomatropin-tcgd in the United States and the further development of TransCon hGH, expenses related to the development and potential commercialization of its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or d ‘other development programs, sales, general and administrative costs, other research and development costs and the activity of Ascedis in general; delays in the development of its oncology, TransCon hGH, TransCon PTH and TransCon CNP programs or other development programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays ; reliance on third party manufacturers to supply study drug for planned clinical studies; Ascendis’ ability to secure additional funding, if necessary, to support its business operations and the effects on its business of the global COVID-19 pandemic. For a more detailed description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as the risks associated with Ascendis ‘business in general, see Ascendis’ annual report on Form 20- F filed with the United States Securities and Exchange Commission (SEC) on March 10, 2021 and other future Ascendis reports filed or submitted to the SEC. Forward-looking statements do not reflect the potential impact of any future licenses, collaborations, acquisitions, mergers, disposals, joint ventures or investments that Ascendis may enter into or make. Ascendis assumes no obligation to update forward-looking statements, except as required by law.
SKYTROFA, Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are registered trademarks of the Ascendis Pharma group. © September 2021 Ascendis Pharma A / S.
— Footnotes: 1 Karpf DB, et al. J Bone Miner Res. 2020; x: 1-11.2 Mannstadt M, et al. Nature Reviews 2017, 3: 170553 Ascendis Pharma HP Patient Experience Research. 4 Hadker N, et al. Endo practice. 2014, 20 (7); 671-679. 5 Powers J, et al. J Bone Miner Res 2013, 28: 2570-2576.6 Mitchell DM, et al. J Clin Endocrinol Metab 2012, 97 (12): 4507-45147 Underbjerg L, et al. J Bone Miner Res 2013, 28: 2277-2285
Source: Ascendis Pharma