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Ascendis Pharma A/S Announces Top-Line Results from Week 84 of Its Phase 2 PaTH Forward Trial Demonstrating that TransCon™ PTH Provided Durable Benefit and Was Well-Tolerated in Adults with Hypoparathyroidism

Nov 19, 2021

–– 58 of the original 59 subjects continue in the open-label extension portion of the Phase 2 trial. –– Phase 3 data for TransCon PTH expected in Q1 2022. COPENHAGEN, Denmark, Nov. 18, 2021 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND), today announced top-line results from Week 84 of the Company’s Phase 2 PaTH Forward Trial, a global trial evaluating the safety, tolerability, and efficacy of its investigational TransCon PTH product candidate in adult patients with hypoparathyroidism (HP). TransCon PTH is an investigational once-daily prodrug of parathyroid hormone (PTH) designed to restore physiologic levels of PTH 24 hours a day. The week 84 data showed that subjects treated with TransCon PTH had both mean serum calcium levels and urinary calcium excretion that remained stable and in the normal range and that most subjects (93%) continued to be free from taking active vitamin D and were taking < 600 mg/day of calcium supplements. “The continued durable improvements observed in this study over a year and a half of treatment with TransCon PTH highlight the importance of sustained restoration of parathyroid hormone to normal physiological levels,” said Professor Peter Schwarz, M.D., DMSci, Professor of Endocrinology at Rigshospitalet and Copenhagen University. “Parathyroid hormone plays an essential role in the regulation of serum and urine calcium and phosphate, thus impacting the function of nerves and muscles, and I am excited that we may potentially soon be able to offer a new treatment paradigm to address the serious health and quality of life issues these patients face each day.” After an initial screening and baseline assessment period, patients in the Phase 2 PaTH Forward Trial were randomized to blinded treatment with TransCon PTH at 15, 18, or 21 μg/day or placebo for 4 weeks, followed by a switch to open label treatment, during which physicians could optimize dosing of TransCon PTH to meet individual treatment objectives. Key Findings at Week 84 of the Phase 2 PaTH Forward Trial: 58 out of the 59 original trial participants continued open-label treatment with TransCon PTH. Mean serum calcium levels remained stable and in the normal range. All study subjects discontinued active vitamin D supplements in the earliest weeks of the trial and have remained off it since then. In addition, 93% of study subjects were taking calcium supplements <600 mg/day. Mean urinary calcium excretion remained stable and in the normal range. TransCon PTH was well-tolerated at all doses administered. No treatment-related serious or severe adverse events occurred, and no treatment-emergent adverse events (TEAEs) led to discontinuation of study drug. “We believe that these data, combined with nearly all patients continuing in the open label portion of the trial, indicates the potential for TransCon PTH to become the first hormone therapy to replace active vitamin D and therapeutic doses of calcium for adults living with hypoparathyroidism,” said Dana Pizzuti, M.D., Ascendis Pharma’s Chief Medical Officer and Senior Vice President of Development Operations. “We look forward to sharing our top-line Phase 3 data in Q1 2022 and, if positive, plan to complete an NDA submission to the FDA in mid-2022.” The Investor presentation on the Investor & Media section of the Ascendis Pharma website at https://ascendispharma.com has been updated to include the TransCon PTH Path Forward Trial Week 84 Week data. About TransCon PTH 1 TransCon PTH is an investigational once-daily long-acting prodrug of parathyroid hormone (PTH[1-34]) in development as a treatment for adult hypoparathyroidism (HP). TransCon PTH is designed to restore PTH at physiologic levels for 24 hours each day to address both the short-term symptoms and long-term complications of the disease. TransCon PTH has been granted orphan drug designation in the United States and European Union for the treatment of hypoparathyroidism. About Hypoparathyroidism (HP) 2,3,4,5,6,7 Hypoparathyroidism (HP) is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH), resulting in low calcium and elevated phosphate levels in the blood. HP affects approximately 200,000 patients worldwide, most of whom develop the condition following damage to or accidental removal of the parathyroid glands during thyroid surgery. Conventional treatment with calcium and active vitamin D (also called calcitriol) does not effectively address the short-term symptoms, long-term complications, or quality-of-life impacts of hypoparathyroidism. Short-term symptoms include weakness, severe muscle cramps (tetany), abnormal sensations such as tingling, burning and numbness (paresthesia), memory loss, impaired judgment, and headache. Patients often experience decreased quality of life and, over the long term, this complex disorder can increase risk of major complications, such as calcium deposits in the brain, blood vessels, eye and other soft tissues – including the kidneys, which can lead to impaired renal function. Current standard of care with active vitamin D analogs and calcium supplementation do not fully control the disease and may contribute to risk of renal disease. Patients with HP have an estimated 4-fold to 8-fold greater risk of renal disease compared to healthy populations. The disease is also associated with a 2-fold increased risk of depression or bipolar disorder compared to healthy populations HP remains among the few hormonal insufficiency states without a replacement therapy that restores the missing hormone at physiologic levels. About Ascendis Pharma A/S Ascendis Pharma is applying its innovative platform technology 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, the company uses its TransCon technologies to create new and potentially best-in-class therapies. 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 Statement 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) the expected timing of top-line results from the Phase 3 Phase 3 PaTHway Trial, (ii) Ascendis’ plan to submit an NDA to the FDA, (iii) Ascendis’ ability to apply its platform technology to build a leading, fully integrated biopharma company, and (iv) 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 and distributors to supply TransCon hGH, the SKYTROFA® Auto-Injector and other study drug for commercial sales in the U.S. and clinical studies; unforeseen safety or efficacy results in its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs; unforeseen expenses related to commercialization of lonapegsomatropin-tcgd in the U.S., the co-pay program, 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 other development programs, selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its oncology programs, TransCon hGH, TransCon PTH and TransCon CNP or other development programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; dependence on third party manufacturers to supply study drug for planned clinical studies; Ascendis’ ability to obtain additional funding, if needed, to support its business activities and the effects on its business from the worldwide COVID-19 pandemic. 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 March 10, 2021 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 SKYTROFA are trademarks owned by the Ascendis Pharma Group. © November 2021 Ascendis Pharma A/S. Investor Contacts:

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