Ascendis Pharma A / S announces –


– Last-minute oral presentation will include a full 58-week results review of the business phase 2 Path Forward Trial

COPENHAGEN, Denmark, September 30, 2021 (GLOBE NEWSWIRE) – Ascendis Pharma A / S (ASND), a biopharmaceutical company using its innovative TransCon technologies to potentially create new treatments to make a significant difference in the lives of patients, announced today Presentations showcasing new data on its investigational product candidate TransCon PTH at the 2021 American Society for Bone & Mineral Research (ASBMR) annual meeting, to be held October 1-4 virtually and in person in San Diego . The presentations will also include an overview of the basic demographics of the natural history study sponsored by Ascendis Pharma in children with achondroplasia.

“We are delighted to partner with our researchers to share the latest data showing the continued progress of our hypoparathyroidism and achondroplasia development programs,” said Aimee Shu, MD, vice president of clinical development at Ascendis Pharma, Endocrine Medical Sciences. “In particular, we are delighted to have a last minute oral presentation from Dr Aliya Khan, a recognized expert in parathyroid diseases, who will present the results of what we believe to be the most comprehensive dataset generated to date. (58 subjects) long-term continuous administration of parathyroid hormone (PTH) at physiological levels in patients with hypoparathyroidism.

“Over 58 weeks in PaTH Forward, data from study subjects show continuing trends towards normal calcium homeostasis in the absence of standard treatments,” said Aliya Khan, MD, clinical professor of medicine and director of Calcium Disorders Clinic at St. Joseph’s Healthcare, McMaster University. “This was reflected in the normalization of key biomarkers and bone mineral density. Equally important, patients reported a marked reduction in their symptom burden and health-related improvements in their quality of life. The majority of adverse events were mild and unrelated to the study drug, and no adverse events led to discontinuation of the study drug or discontinuation from the trial. These are very encouraging signs that physiological parathyroid hormone replacement is well tolerated and may help meet the unmet needs these patients face.

Oral presentation Date hour
Sustained benefit of TransCon PTH, an investigational hormone replacement therapy for adult patients with hypoparathyroidism, at week 58 in the PaTH Forward trial Last minute oral presentations: clinical treatment
Oral presentation by Aliya Khan, MD # LB-1114
Monday October 4
11:30 am-12:45pm PST
Poster presentations Date hour
ACHieve Baseline Demographics: Five-Year Multinational Observational Cohort Study of Children with Achondroplasia Poster presentation in virtual plenary
Presenter-author: Leanne M. Ward, MD, FRCPC Friday October 1st &
saturday 2 october
Poster # A21023623
A single dose of TransCon PTH in subjects with renal impairment: Poster Session I
A phase 1 trial saturday 2 october
Presenting Author: Aimee Shu, MD 1:00 p.m. to 3:00 p.m. PST
Poster # SAT-272

Ascendis Pharma is also proud to sponsor two continuing medical education (CME) programs at this year’s ASBMR annual meeting:

Ascendis Sponsored CME Programs Date hour
Hypoparathyroidism: changing the treatment landscape Friday October 1
Bart Clarke & Lars Rejnmark 6.30-7.45 am PST
Virtual presentation with live Q&A
Achondroplasia: beyond the small size saturday 2 october
Janet Legare & Michael Bober 6.30-7.45 am PST
Virtual presentation with live Q&A

About hypoparathyroidism16
Hypoparathyroidism (HP) 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 (active vitamin D) and is essential to 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 achondroplasia

Achondroplasia is the most common form of dwarfism, affecting approximately 250,000 people worldwide. People with achondroplasia can have serious skeletal complications and co-morbidities. For example, abnormal development of the vertebra can lead to sleep apnea, chronic back and leg pain due to impingement in the lower spine, and sudden infant death from cervical compression. Chronic ear infections caused by abnormal eustachian tubes can lead to hearing loss and delayed speech.7

The disease is caused by an autosomal dominant activating mutation in the fibroblast growth factor receptor 3 (FGFR3) gene that results in an imbalance in the effects of the FGFR3 and CNP signaling pathways. Preclinical and clinical data show that the CNP pathway stimulates growth. The increase in CNP counteracts the effects of the FGFR3 mutation downstream, thereby promoting bone growth.8

About Ascendis Pharma A / S
Ascendis 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 for more information.

Forward-looking statements
This 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 regarding (i) whether physiological replacement of parathyroid hormone could help meet the unmet needs these patients face, (ii) the ability of Ascendis to apply its platform technology to build a leading, fully integrated biopharmaceutical company, (iii) Ascendis ‘product pipeline and its expansion into additional therapeutic areas and (iv) Ascendis’ expectations regarding its ability to use its TransCon technologies to create potentially best-in-class new 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 Ascedis’ forward-looking statements, including: reliance on third party manufacturers to provide TransCon hGH, SKYTROFA® Auto-injector and other study drug for commercial sales in the 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.

End Notes:

1 Mannstadt M, et al. Nature Reviews 2017, 3: 17055
2 Ascendis Pharma HP Patient Experience Research.
3 Hadker N, et al. Endo practice. 2014, 20 (7); 671-679.
4 Powers J, et al. J Bone Miner Res 2013, 28: 2570-2576.
5 Mitchell DM, et al. J Clin Endocrinol Metab 2012, 97 (12): 4507-4514
6 Underbjerg L, et al. J Bone Miner Res 2013, 28: 2277-2285
7 Ireland PJ, Pacey V, et al. Appl Clin Genet. 2014; 7: 117–25.
8 Horton WA, et al. Lancet. 2007; 370 (9582): 162-172.


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