Ascendis Pharma Launches 2nd TransCon™ Product: YORVIPATH® Now Available in Germany and Austria for Adults with Chronic Hypoparathyroidism

COPENHAGEN, Denmark, Jan. 31, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that YORVIPATH^® (palopegteriparatide, developed as TransCon PTH), a parathyroid hormone (PTH) replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism, is now available in Germany and Austria. YORVIPATH is the second product developed with Ascendis Pharma’s TransCon technology platform to be launched commercially.

“We are pleased to initiate the launch of YORVIPATH to address the needs expressed within the hypoparathyroidism community for a new PTH-based treatment option,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “In addition to addressing a significant unmet medical need, this launch of our second TransCon product represents a key milestone in our goal to launch the three Endocrinology Rare Disease products for which, under Vision 3x3 we target achieving regulatory approvals by 2025 and for each of which, under Vision 2030, we aim to achieve blockbuster status by 2030. With an increasingly broad commercial infrastructure in place, we are well positioned to accomplish this by expanding the availability of YORVIPATH and our other approved products to meet the needs of physicians and patients around the world.”

About YORVIPATH
YORVIPATH (palopegteriparatide, developed as TransCon PTH) is a prodrug of parathyroid hormone (PTH 1-34) administered once daily, designed to provide parathyroid hormone levels within the normal physiological range across the 24-hour dosing period. YORVIPATH was granted marketing authorization by the European Commission (EC) in November 2023 as a PTH replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. A decision on YORVIPATH in the United Kingdom is expected from the Medicines & Healthcare Products Regulatory Agency (MHRA) during the first quarter of 2024. In the United States, the U.S. Food & Drug Administration (FDA) has set a PDUFA date of May 14, 2024 to complete their review of Ascendis Pharma’s New Drug Application for TransCon PTH for adults with chronic hypoparathyroidism. TransCon PTH is also in development in Japan through Teijin Ltd. and China through VISEN Pharmaceuticals. Internationally, Ascendis is working to expand the global reach of its Endocrinology Rare Disease portfolio, including YORVIPATH, through exclusive distribution agreements, three of which have been established to date.

About Hypoparathyroidism
Hypoparathyroidism is an endocrine disease caused by insufficient levels of PTH, the primary regulator of calcium/phosphate balance in the body, acting directly on bone and kidneys and indirectly on intestines. Hypoparathyroidism is considered chronic if it persists >6 months following surgery per the 2016 Endocrine Society Guidelines, 2019 Canadian and International Consensus Statement, and 2022 European Society of Endocrinology Consensus Statement. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%); other etiologies include autoimmune disorders, familial disorders, and idiopathic causes.

About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit https://ascendispharma.com to learn more.

Forward-Looking Statements
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) YORVIPATH’s ability to address the need for a new PTH-based treatment option, (ii) Ascendis’ goal of obtaining approval for, launching and achieving blockbuster status for three Endocrinology Rare Disease products by 2030, (iii) Ascendis’ ability to expand the availability of YORVIPATH and its other approved products, (iv) the timing of the MHRA’s decision on YORVIPATH in the United Kingdom, (v) the FDA’s PDUFA date for TransCon PTH, (vi) Ascendis’ plan to expand the global reach of its Endocrinology Rare Disease portfolio, including YORVIPATH, through exclusive distribution agreements, (vii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (viii) 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, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including inflation, the effects on its business from the worldwide COVID-19 pandemic and ongoing conflicts such as that in the region surrounding Ukraine and Russia. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 16, 2023 and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law.

Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, and YORVIPATH^® are trademarks owned by the Ascendis Pharma group. © January 2024 Ascendis Pharma A/S.