Ascendis Pharma A/S

— 52-week results from the Phase 3 PaTHway Trial showed that the skeletal dynamics of adult patients with chronic hypoparathyroidism treated with TransCon PTH trended toward a new steady state closer to age-appropriate norms — Bone turnover markers trended toward the normal reference ranges for sex and menopausal status and corresponded to smaller changes in bone mineral density (BMD) through Week 52 — Results were similar to trends in bone turnover markers and changes in BMD previously reported through Week 110 in the Phase 2 PaTH Forward Trial COPENHAGEN, Denmark, Oct. 16, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) shared 52-week data from the open-label extension period of its ongoing Phase 3 PaTHway Trial of TransCon PTH (palopegteriparatide) showing that adults with chronic hypoparathyroidism, whose bones tend to be over-mineralized due to insufficient parathyroid hormone (PTH) exposure, trended toward a new skeletal steady state closer to age-appropriate norms with continued use of TransCon PTH. The results were consistent regardless of sex, menopausal status, or duration of disease and were consistent with results previously reported through Week 110 in the Company’s Phase 2 PaTH Forward Trial. An oral presentation of the data was given today by Aliya Khan, M.D., Clinical Professor of Medicine at McMaster University and Director of the Calcium Disorders Clinic at McMaster University Medical Center, during ASBMR 2023, the annual meeting of the American Association of Bone & Mineral Research in Vancouver, BC, Canada. Reflecting on the clinical data and its potential impact, Dr. Khan said “Treatment with TransCon PTH in this clinical trial showed the positive physiological effects on bone, in patients treated for the full year as well as in those switching from placebo after the 26-week blinded period. These results underscore the importance of providing the missing hormone to address the significant impacts of hypoparathyroidism, including decreased bone remodeling leading to a dense, over-mineralized bone structure.” TransCon PTH (palopegteriparatide) is an investigational prodrug with sustained release of active parathyroid hormone (PTH [1-34]) administered once daily. On September 14, 2023, TransCon PTH received a positive CHMP opinion recommending approval in the European Union for the treatment of adults with chronic hypoparathyroidism. TransCon PTH is also in development in the United States and Japan. PaTHway is an ongoing Phase 3 double-blind, placebo-controlled trial of 82 dosed adults with chronic hypoparathyroidism randomized 3:1 (TransCon PTH:placebo) treated for 26 weeks, followed by a 156-week open-label extension period. Registered attendees of ASMBR 2023 conference can access the abstract, poster, and presentation (#1114) through the conference organizer’s website. 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 Germany (Heidelberg and Munich) and the United States (Palo Alto and Redwood City, California, and Princeton, New Jersey). Please visit 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) the potential approval of TransCon PTH in the European Union; (ii) Ascendis’ ability to apply its platform technology to build a leading, fully integrated, global biopharma company; and (iii) 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, and TransCon® are trademarks owned by the Ascendis Pharma group. © October 2023 Ascendis Pharma A/S. Investor Contacts: Media Contact: Tim Lee Melinda Baker Ascendis Pharma Ascendis Pharma +1 (650) 374-6343 +1 (650) 709-8875 tle@ascendispharma.com media@ascendispharma.com ir@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com

Over the years, medical device design experts have contributed to MD+DI in a variety of ways, including participation in the judging of the Medical Design Excellence Awards (MDEAs) and speaking at our MD&M and BIOMEDevice events. Below are four essential elements of medical device design we've gleaned from these experts over time. Ease of use is critical in medical device design This one may seem obvious, but it's often the first thing past MDEA jurors have noticed when evaluating an entry, particularly as more medical devices transition from being used primarily in a clinical setting to an at-home setting. The idea is that a product that is simple to operate is likely to reduce errors and encourage regular use. Sometimes, the user interface of a medical device is closely linked to the product's performance. For example, a 2017 MDEA finalist in the category of rehabilitation and assistive-technology products developed a fully integrated, microprocessor-controlled lower limb system for above-the-knee amputees. The Linx system was designed to address a well-known drawback of the product category, which is the unnatural gait that often occurs when patients wear prosthetic legs. This was achieved through master control across both joints, hydraulic ankles, sensors in the knee and ankle, and automatic detection and switching for different user modes. A more recent example is the Skytrofa Auto-Injector that Phillips-Medisize designed in collaboration with its customer, Ascendis Pharma. So much thought and usability research went into this device that it has won three industry awards for the design. John Christensen, senior project manager in contract development at Philips–Medisize, gave MD+DI a demonstration of how the Skytrofa Auto-Injector works (see video below). Paul Erik Fabricius, director of front-end innovation at Phillips-Medisize, said there were several key design considerations that went into this device because of the intended user population (parents of young children requiring growth hormone therapy). Ease of use was a key consideration in process of designing the Skytrofa device. "We wanted to facilitate this very complex preparation procedure in a simple and safe manner," Fabricius told MD+DI . "That means both the physical handling of the device, for example it needs to stand up during the reconstitution to ensure that no drug is lost out through the needle, but you also need to have a nice grip when you do the injection." Image courtesy of Phillips-Medisize, a Molex company Color us impressed: Aesthetic considerations in medical device design Choosing a color for a new medical device requires more thought than simply picking one's favorite color. Christine Park, an industrial designer who spoke at MD&M West in 2017, says color can impact everything from brand identity and usability to what kind of emotional response the device could provoke from patients. Color, materials, and finish design specialists like Park use a combination of color psychology and market research to help manufacturers pick the right color scheme for new products. Park used examples from the consumer electronics industry to illustrate how color can impact brand identity. In the 1990s, for example, black and silver was the color scheme of choice for most stereo systems and personal cassette players. Then, in 2001, Apple introduced the iPod in white. Soon it was easy to recognize Apple users by the gleaming whiteness of their earbuds. Similarly, Park noted, Dr. Dre used the color red to build brand identity for his product line of headphones and earphones. Color was also a key design consideration for the Skytrofa Auto-Injector. The team wanted the design to be visually appealing for children and found through usability studies that the white and bright green colors of the device made it more pleasing for the intended user population. Another way color can impact medical device design is through usability, Park said. She explained how color can be used to group related functions and organize buttons on a machine by areas of function and importance. She used the example of an ultrasound machine from Philips that makes use of a muted orange color to draw the user's eye to the more important areas of function, as well as different tones of gray. The keyboard, on the other hand, blends in by being the same color as the background of the system, she said. The user quickly learns to recognize the orange-colored buttons and the trackball as having a unique function, and it's easy for their eye to find as they glance back and forth between the machine and the patient. "You can train the users to associate different colors with different functions," Park said. She cautioned, however, that it's important to test a color decision early in the process through an online color simulator, to see how it would appear to someone with colorblindness, as 8% of the male population is color blind. Females are less likely to be colorblind, with only 0.5% of the female population being affected by colorblindness. Color psychology can also play a key role in color decisions for medical devices, Park said. The color blue, for example, is often associated with tranquility, loyalty, security, and trust, which is why it is a commonly used color for MRI machines. Pink tends to be associated with femininity, nurturing, and compassion, she said, using a pink pregnancy monitoring device as an example. She did point out, however, that color symbolism varies from culture to culture so it's also a good idea to keep that in mind during the color decision process. Park shared a three-step process for choosing the right color of a medical device. First, she said, "understand your users." Second, research market trends not just in that particular device sector, but also look at color trends in other industries that the target user is familiar with. Third, decide on a message and use that to guide color decisions. A device designed to deliver migraine medication, for example, could communicate empathy and calmness through a muted yellow or a calming seafoam color, Park said. Patient-centric medical device design Closely related to ease of use, another essential element of medical device design is keeping the patient at the center of it all. One example, again from the 2017 MDEA finalists, is the Hologic Affirm Prone Biopsy Table. This device was designed to transform the familiar breast biopsy platform by allowing the patient to stay in a prone position during the procedure. In addition to enabling 2D and 3D image-guided biopsies, the Affirm Prone system automates some steps of the biopsy procedure, allows for easier access, and is "designed to reduce the patient's time under compression and deliver a more comfortable biopsy experience," according to the company's submission. "In this day and age of patient satisfaction, the focus of the Affirm system to take a patient-centered approach and couple it with more flexibility in use by practitioners, resulting in improved outcomes, makes this device a standout," said 2017 MDEA juror David Copeland. At the time, Copeland was director of human factors industrial design at Ximedica. Today, he is director of human centered design at Veranex. Medical device design that makes an impact Over the years, many MDEA submissions have emphasized ways the device could impact the overall healthcare system by reducing costs, increasing efficiency, shortening hospital stays, preventing complications, and reducing the threat of hospital-acquired infections (HAIs). The best part of this element of medical device design is that it doesn't always take a high-tech design to make an impact. For example, one 2017 MDEA submission, the Carebag bedpan and commode pail liner from Cleanis, was designed to reduce HAI rates. The product incorporates a pad that is designed to absorb the body fluids and convert it into a gel. Noting that missing HAI objectives can impact a facility's Medicare reimbursement, the submission described the Carebag as "a solution to safely confine and dispose of feces, [a] known vector of transmission of the deadly C. diff infection . . ." How did your answers compare to ours? What four essential elements of medical device design came to mind for you? Share your best design advice with us by emailing MD+DI Senior Editor Amanda Pedersen at [email protected] . Please put "medical device design" in the subject line.

– At Week 26, 81% of patients in Phase 3 PaTHway Trial with chronic post-surgical hypoparathyroidism, the most common cause of this disease, achieved independence from conventional therapy while maintaining normocalcemia following treatment with TransCon PTH – Post-surgical patients treated with TransCon PTH self-reported significant improvements in disease-related symptoms, physical functioning, and daily life compared to those on placebo – Results for post-surgical patients (n=70) comparable to overall PaTHway Trial population (n=82) COPENHAGEN, Denmark, Oct. 02, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) presented a poster with results of a sub-analysis of its Phase 3 PaTHway clinical trial data during the recent American Thyroid Association (ATA) annual meeting, showing that 81% of adults with chronic post-surgical hypoparathyroidism (n = 70) treated with TransCon PTH (palopegteriparatide) achieved independence from conventional calcium and active vitamin D therapy while maintaining normal serum calcium levels during the 26-week blinded portion of the trial, compared to 6% taking placebo. The results were comparable to those from the overall PaTHway Trial population (N = 82), which demonstrated consistency in improved outcomes with TransCon PTH treatment compared to conventional therapy across disease etiologies. TransCon PTH is an investigational prodrug with sustained release of active parathyroid hormone (PTH [1-34]) administered once daily. TransCon PTH (palopegteriparatide) has received a positive CHMP opinion recommending approval in the European Union and is in development in the United States, and Japan for the treatment of adults with hypoparathyroidism. “We are pleased to share with thyroid surgeons and thyroidologists attending ATA this data demonstrating the safety and benefits of TransCon PTH seen in clinical trial patients whose disease arose following neck surgeries,” said Aimee Shu, M.D., Vice President of Clinical Development, Endocrinology Medical Sciences at Ascendis Pharma. “Our goal is to provide a treatment option that can help patients overcome the serious health and quality of life burdens associated with hypoparathyroidism, regardless of its cause. We will continue to advance TransCon PTH globally as a candidate to address the urgent needs of this patient community.” PaTHway is a Phase 3 trial with a completed 26-week randomized, double-blind, placebo-controlled period, followed by an ongoing 156-week open-label extension period, designed to evaluate the efficacy, safety, and tolerability of TransCon PTH in adults with hypoparathyroidism. A total of 84 patients were randomized 3:1 to treatment with TransCon PTH (n = 63) or placebo (n = 21), both co-administered with conventional therapy. The multi-component primary efficacy endpoint of PaTHway was defined as normocalcemia (a normal concentration of calcium in the blood; 8.3–10.6 mg/dL), independence from conventional therapy (no active vitamin D and ≤ 600 mg/day calcium), and no TransCon PTH dose change within the 4 weeks prior to Week 26. Key secondary endpoints included patient-reported assessments of disease impact using the Hypoparathyroidism Patient Experience Scale (HPES) and the 36-Item Short Form Survey (SF-36 version 2). Of the 84 patients randomized, 82 received study drug. Of those, the 70 with post-surgical hypoparathyroidism (52 TransCon PTH, 18 placebo) were included in the sub-analysis population. PaTHway Trial Sub-Analysis HighlightsAcross multiple evaluated parameters in participants with post-surgical hypoparathyroidism, treatment with TransCon PTH enabled independence from conventional therapy (no active vitamin D and ≤ 600 mg/day calcium); improvement of hypoparathyroidism-specific symptoms and their impact on daily life and physical functioning; and normalization of mean 24-hour urine calcium excretion. This includes: At Week 26, 81% of patients with postsurgical hypoparathyroidism treated with TransCon PTH in the trial achieved independence from conventional therapy while maintaining normocalcemia compared to 6% treated with placebo.At Week 26, 81% (42/52) of patients with chronic post-surgical hypoparathyroidism treated with TransCon PTH demonstrated significant improvement compared to those on placebo in disease-related symptoms and the impact of hypoparathyroidism on physical functioning and daily life (all p < 0.01).Health-related quality of life, as measured by the SF-36 physical functioning subscale score, improved significantly with TransCon PTH treatment compared to placebo in participants with post-surgical hypoparathyroidism (p = 0.0063).From baseline to Week 26, patients with postsurgical hypoparathyroidism treated with TransCon PTH in the trial showed a normalization and greater reduction in urine calcium excretion than those on placebo.Treatment with TransCon PTH was generally safe and well-tolerated. Most treatment-emergent adverse events (TEAEs) were mild or moderate, and no patients discontinued study treatment or the trial due to a treatment-related TEAE. About Ascendis Pharma A/SAscendis 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 Germany (Heidelberg and Munich) and the United States (Palo Alto and Redwood City, California, and Princeton, New Jersey). Please visit 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) the potential value of TransCon PTH, if approved, as a treatment option for adults with hypoparathyroidism; (ii) the ability of TransCon PTH to provide stable PTH levels within the physiological range 24 hours/day; (iii) Ascendis’ ability to achieve its goal of providing a treatment option that can help patients overcome the serious health and quality of life burdens seen with hypoparathyroidism; (iv) Ascendis’ plans to continue to advance TransCon PTH globally as a candidate to address the needs of the hypoparathyroidism patient community; (v) the potential approval of TransCon PTH in the European Union; (vi) Ascendis’ ability to apply its TransCon platform technology to build a leading, fully integrated, global biopharma company; and (vii) 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, and TransCon are trademarks owned by the Ascendis Pharma group. © October 2023 Ascendis Pharma A/S. Investor Contacts:Media Contact:Tim LeeMelinda BakerAscendis PharmaAscendis Pharma+1 (650) 374-6343+1 (650) 709-8875tle@ascendispharma.commedia@ascendispharma.comir@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com