– Marks the next step in bringing the very first potential hormone replacement therapy for hypoparathyroidism to Japan –
COPENHAGEN, Denmark, May 12, 2021 (GLOBE NEWSWIRE) – Ascendis Pharma A / S (Nasdaq: ASND), a biopharmaceutical company that uses its innovative TransCon â¢ technologies to create product candidates that address unmet medical needs, announced today hui Having filed a Clinical Trial Notification (CTN) with the Pharmaceuticals and Medical Devices Agency (PDMA) in Japan to initiate the Company’s Phase 3 clinical trial of TransCon PTH in adult subjects with hypoparathyroidism (HP), the PaTHway Japan trial.
âThe CTN filing for TransCon PTH represents another important step as we continue to lay the groundwork to make this important therapy available worldwide to address a significant unmet need for patients with hypoparathyroidism worldwide. The Phase 3 trial PaTHway Japan, which has been the subject of extensive negotiations with the PDMA, will recruit a predetermined cohort of patients for the combined global program to support the submission of a marketing authorization application to the Japan. This study, along with long-term data from our phase 2 PaTH Forward trial and data from our phase 3 PaTHway trial, have the potential to provide rapid access to important hormone replacement therapy for patients with HP in Japan â , said Jan Mikkelsen, President and CEO of Ascendis Pharma.
âOne goal of our 3×3 Vision is to achieve global clinical reach for our rare disease pipeline in endocrinology. Starting first with North America and Europe, then expanding into Greater China through our investment in VISEN Pharmaceuticals, our recent expansion of our clinical reach in Japan with TransCon hGH and TransCon PTH represents a milestone. important in achieving this vision, âcontinued Mikkelsen.
The Phase 3 PaTHway Japan trial is designed as a single arm study to assess the safety and efficacy of TransCon PTH in adults with hypoparathyroidism.
About TransCon PTH1
TransCon PTH is an investigational, long-acting once-daily parathyroid hormone (PTH[1-34]) in development as a treatment for hypoparathyroidism in adults (HP) designed to restore PTH to physiological levels for 24 hours a day to treat both short-term symptoms and complications in long-term illness. TransCon PTH received Orphan Drug Designation (ODD) by the United States Food and Drug Administration (FDA) in June 2018 and, in October 2020, Orphan Designation from the European Commission for the treatment of hypoparathyroidism. .
About hypoparathyroidism (HP) 2,3,4,5,6,7
Hypoparathyroidism (HP) is a rare endocrine disease characterized by insufficient levels of parathyroid hormone (PTH), resulting in low calcium content and elevated phosphate levels in the blood. HP affects approximately 200,000 patients in the United States, Europe, Japan and South Korea, the majority of whom develop the disease as a result of damage or accidental removal of the parathyroid glands during thyroid surgery. Patients often have a reduced quality of life. Short-term symptoms include weakness, severe muscle cramps (tetany), abnormal sensations such as tingling, burning and numbness (paresthesia), memory loss, poor judgment and headache. In the long term, this complex disorder can increase the risk of major complications, such as extra-skeletal calcium deposits occurring in the brain, lens, and kidneys, which can lead to impaired kidney function.
HP remains among the rare states of hormonal insufficiency without replacement therapy that restores the missing hormone to physiological levels. Standard care with active vitamin D analogues and calcium supplementation does not fully 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 higher risk of kidney disease compared to healthy controls.
About Ascendis Pharma A / S
Ascendis Pharma applies its innovative TransCon technologies to build a leading, fully integrated biopharmaceutical company whose goal is to make a meaningful difference in the lives of patients. Guided by its core values ââof patients, science and passion, the company uses its TransCon technologies to create new therapies potentially best in class.
Ascendis Pharma currently has a portfolio of three independent rare disease endocrinology product candidates and one oncology product candidate in clinical development. The company continues to expand into other therapeutic areas to address unmet patient needs.
Ascendis is headquartered in Copenhagen, Denmark, with additional offices in Heidelberg and Berlin, Germany, Palo Alto and Redwood City, California, and Princeton, New Jersey.
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 relating to (i) Ascendis’ expectations regarding the potential submission by Ascendis of a Marketing Authorization Application in Japan, (ii) to Ascendis’ expectations for the phase 3 trial of PaTHway in Japan, as well as other data, allowing rapid access to hormone replacement therapy for patients living with HP in Japan, (iii) Vision 3×3 (iv) Ascendis , Ascendis ‘ability to apply its platform technology to build a leading fully integrated biopharmaceutical company, (v) Ascendis’ product pipeline and its expansion into other therapeutic areas and (vi) expectations Ascendis regarding its ability to use its TransCon technologies to create new and potentially best-in-class therapies. Ascendis may not actually carry out the plans, achieve 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 the following: 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 development and potential commercialization of its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs, selling, general and administrative expenses, other research and development expenses and the activities of Ascendis 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, timeliness 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 reports of Ascendis 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.
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are registered trademarks belonging to the Ascendis Pharma group. Â© May 2021 Ascendis Pharma A / S.
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