Phase 3 Data Show TransCon® PTH Replicated Systemic Actions of Endogenous PTH Through Week 182 in Adults with Hypoparathyroidism

• Multi-organ system and quality-of-life advantages sustained by way of three and a half years 
• 86% response fee for the multi-component endpoint
• 89% of sufferers accomplished the three-and-a-half-year trial

COPENHAGEN, Denmark, June 13, 2026 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND) at the moment introduced Week 182 knowledge from its accomplished Phase 3 PaTHway Trial displaying that long-term remedy with TransCon PTH (palopegteriparatide) demonstrated sustained efficacy and security in adults with hypoparathyroidism. Over the three-and-a-half-year period of the trial, TransCon PTH replicated the systemic actions of endogenous PTH, with a balanced, useful affect on the principle goal organ techniques – CNS, kidney, small gut, and bone – as demonstrated by improved high quality of life and normalized and steady urine calcium, serum calcium, serum phosphate, and bone mineral density. These advantages have been sustained whereas enabling independence from typical remedy with lively vitamin D and calcium.

“With its distinctive potential to copy the systemic actions of endogenous parathyroid hormone, TransCon PTH has efficiently addressed the bodily and psychological burdens of hypoparathyroidism for almost all of handled sufferers,” stated Aliya Khan, M.D., Scientific Professor of Medication, Division of Endocrinology & Geriatrics, and Director of the Calcium Problems Clinic at McMaster College in Canada. “These Phase 3 knowledge reinforce the fast and sustained advantages seen all through medical trials of TransCon PTH, together with compelling enhancements in high quality of life reported by sufferers beforehand restricted by the fatigue, cognitive challenges, and diminished bodily functioning and well-being which might be the hallmarks of this illness.”

Highlights of Week 182 Outcomes from the Phase 3 PaTHway Trial

• 86% of sufferers have been responders for the multi-component endpoint of (1) serum calcium in the traditional vary, (2) taking no lively vitamin D, and (3) taking ≤600 mg/day of calcium.
  • 89% of sufferers had regular albumin-adjusted serum calcium ranges and a imply worth of 8.8 mg/dL.
  • 100% of sufferers achieved independence from lively vitamin D, outlined as not taking calcitriol or alfacalcidol.
  • 96% of sufferers achieved independence from therapeutic doses of calcium, outlined as taking <600 mg/day of calcium.
• Important enhancements in kidney perform have been maintained, with imply (SE) eGFR of 80.2 (1.8) mL/min/1.73 m² at Week 182, reflecting a imply (SE) improve of 11.0 (1.4) mL/min/1.73 m² from baseline. Amongst sufferers randomized to TransCon PTH, eGFR elevated from baseline by way of Week 38 and stabilized thereafter. After initiation of open-label remedy at Week 26, sufferers who had been receiving placebo in the double-blind interval skilled an identical improve in eGFR. Following these eGFR will increase, imply eGFR values have been maintained by way of Week 182, in distinction to the anticipated typical age-related decline in eGFR in adults.ⁱ
• Imply 24-hour urine calcium decreased considerably, normalized inside 26 weeks, and remained regular by way of Week 182.
• As measured by Hypoparathyroidism Affected person Expertise Scales (HPES), sufferers reported enhancements in signs and health-related high quality of life throughout all domains. Hypoparathyroidism-related bodily and cognitive signs and impacts on bodily functioning and day by day life improved quickly with TransCon PTH remedy and have been maintained by way of Week 182.
• As measured by SF-36, all subscale scores and part abstract scores demonstrated fast and clinically significant enhancements with TransCon PTH remedy which have been sustained by way of Week 182.
• Imply BMD Z-scores (matched for age and intercourse) corrected from excessive baseline ranges by way of Week 26 and remained above 0 by way of Week 182.
• Within the trial, TransCon PTH remedy was typically well-tolerated, with no new security alerts recognized. Therapy-emergent hostile occasions (AEs) have been largely gentle or reasonable, and no discontinuations have been associated to review drug.
• Over three and a half years of remedy, no sufferers developed anti-PTH antibodies.

“Regardless of illness origin, TransCon PTH has normalized key biochemistries and skeletal well being whereas considerably enhancing kidney perform and high quality of life starting on the earliest timepoints and persevering with by way of a number of years of remedy,” stated Aimee Shu, M.D., Government Vice President, Chief Medical Officer at Ascendis Pharma. “We stay dedicated to persevering with our work to advance remedy choices for sufferers all over the world residing with this often-debilitating continual illness.”

The PaTHway Trial of 82 adults with hypoparathyroidism (85% post-surgical, 15% non-surgical) included a 26-week randomized, double-blind, placebo-controlled interval adopted by a 156-week open-label extension (OLE) interval, and measured a wide selection of medical, biochemical, and high quality of life endpoints, constant with the breadth of unfavorable long-term impacts skilled by sufferers with hypoparathyroidism. Seventy-three of the unique 82 sufferers enrolled (89%) accomplished the three-and-a-half-year trial. Endpoints included independence from typical remedy (outlined as <600 mg/day of calcium and no lively vitamin D) and upkeep of normocalcemia (8.3 to 10.6 mg/dL). Renal perform was assessed by estimated glomerular filtration fee (eGFR). Bone mineral density (BMD) measured by DXA scan was assessed at baseline and common intervals by way of Week 182. Hypoparathyroidism-related signs and useful impacts have been measured utilizing the HPES. Well being-related high quality of life was measured utilizing the 36-Merchandise Brief Kind Survey (SF-36 model 2). Security assessments included treatment-emergent AEs and 24-hour urine calcium excretion.

TransCon PTH is a prodrug of PTH (1-34), administered as soon as day by day, designed to supply steady ranges of lively PTH throughout the physiological vary for twenty-four hours/day, accredited as YORVIPATH^® in the USA, European Union, European Financial Space, and sure different jurisdictions as a remedy for adults with hypoparathyroidism.

A slide presentation with these knowledge will probably be made obtainable on the Investor Relations & Information part of the Ascendis Pharma web site: https://traders.ascendispharma.com.

About Hypoparathyroidism
Hypoparathyroidism is an endocrine illness brought on by inadequate ranges of parathyroid hormone (PTH), the first regulator of calcium and phosphate stability in the physique, appearing immediately on bone and kidney and not directly on the gut. People with hypoparathyroidism might expertise a variety of extreme and doubtlessly life-threatening short-term and long-term issues, together with neuromuscular irritability, renal issues, extra-skeletal calcifications, and cognitive impairment. Publish-surgical hypoparathyroidism accounts for almost all of instances (70-80%), whereas different etiologies embrace autoimmune, idiopathic, and genetic causes, together with ADH1.

About Ascendis Pharma A/S
Ascendis Pharma is a worldwide biopharmaceutical firm centered on making use of our revolutionary TransCon know-how platform to make a significant distinction for sufferers. Guided by our core values of Sufferers, Science, and Ardour, and following our algorithm for product innovation, we apply TransCon to develop new therapies that display best-in-class potential to deal with unmet medical wants. Ascendis is headquartered in Copenhagen, Denmark, and has further services in Europe and the USA. Please go to ascendispharma.com to study extra.

Ahead-Trying Statements
This press launch incorporates forward-looking statements that contain substantial dangers and uncertainties. All statements, apart from statements of historic info, included in this press launch relating to Ascendis’ future operations, plans and aims of administration are forward-looking statements throughout the which means of Part 27A of the Securities Act of 1933, as amended, and Part 21E of the Securities Alternate Act of 1934, as amended. Examples of such statements embrace, however aren’t restricted to, statements referring to (i) TransCon PTH’s potential to copy endogenous parathyroid hormone and deal with the bodily and psychological burdens of hypoparathyroidism, (ii) Ascendis’ dedication to persevering with its work to advance remedy choices for sufferers all over the world with hypoparathyroidism, (iii) Ascendis’ potential to use its TransCon know-how platform to make a significant distinction for sufferers and (iv) Ascendis’ use of TransCon to create new and doubtlessly best-in-class therapies to deal with unmet medical wants. Ascendis might not truly obtain the plans, perform the intentions or meet the expectations or projections disclosed in the forward-looking statements and you shouldn’t place undue reliance on these forward-looking statements. Precise outcomes or occasions may differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Numerous necessary elements may trigger precise outcomes or occasions to vary materially from the forward-looking statements that Ascendis makes, together with, with out limitation: dependence on third‑get together producers, distributors, and repair suppliers for Ascendis’ merchandise and product candidates; dangers associated to regulatory evaluation and approval, together with the likelihood of delays, requests for added knowledge or analyses, restrictions or limitations on use, approval with labeling that’s extra restricted than anticipated, or failure to acquire approval in the USA, European Union, or different jurisdictions; medical growth dangers, together with that outcomes from ongoing or future trials might not verify earlier knowledge; unexpected security or efficacy findings in growth packages or on‑market merchandise; manufacturing, provide chain, high quality, or logistics points that might delay growth or commercialization; unexpected bills associated to commercialization of any accredited Ascendis merchandise; unexpected analysis and growth or promoting, common and administrative bills and different prices impacting Ascendis’ enterprise typically; market acceptance, pricing, and reimbursement challenges, together with payer protection selections and well being know-how assessments; aggressive developments, together with new or improved therapies; mental property safety, freedom‑to‑function, and litigation dangers; Ascendis’ potential to acquire further funding, if wanted, to assist its enterprise actions; cybersecurity, knowledge privateness, and data know-how disruptions; and the affect of worldwide financial, political, authorized, compliance, public well being, and enterprise elements, together with tariffs, commerce insurance policies, foreign money fluctuations, and geopolitical occasions. For an additional description of the dangers and uncertainties that might trigger precise outcomes to vary from these expressed in these forward-looking statements, in addition to dangers referring to Ascendis’ enterprise in common, see Ascendis’ Annual Report on Kind 20-F filed with the U.S. Securities and Alternate Fee (SEC) on February 11, 2026, and Ascendis’ different future reviews filed with, or submitted to, the SEC. Ahead-looking statements don’t replicate the potential affect of any future licensing, collaborations, acquisitions, mergers, inclinations, joint ventures, or investments that Ascendis might enter into or make. Ascendis doesn’t assume any obligation to replace any forward-looking statements, besides as required by regulation.

Ascendis, Ascendis Pharma, the Ascendis Pharma brand, TransCon, and YORVIPATH^® are logos owned by the Ascendis Pharma group. © June 2026 Ascendis Pharma A/S.

ⁱGuppy M et al. BMJ Open. 2024;14(11):e089783. doi:10.1136/bmjopen-2024-089783

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