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5-Year Phase 2 Data Show TransCon® PTH Replicated Systemic Actions of Endogenous PTH in Adults with Hypoparathyroidism

News Provided by GlobeNewswire2026-06-11

– Multi-organ system and quality-of-life benefits sustained through five years of treatment

– 82% response rate for the multi-component endpoint

– 95% of patients completed five years of treatment

COPENHAGEN, Denmark, June 11, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced 5-year (Week 266) data from its Phase 2 PaTH Forward Trial showing that long-term treatment with TransCon PTH (palopegteriparatide) demonstrated sustained efficacy and safety in adults with hypoparathyroidism. Over the five-year duration of the trial, TransCon PTH replicated the systemic actions of endogenous PTH, with a balanced, beneficial impact on the main target organ systems – kidney, small intestine, CNS, and bone – as demonstrated by normalized and stable urine calcium, serum calcium, quality of life, and bone mineral density. These benefits were sustained while enabling independence from conventional therapy with active vitamin D and calcium.

“Moving from symptom management to addressing the underlying hormone deficiency requires normalization of PTH biology to mitigate the multi-organ impacts of hypoparathyroidism. TransCon PTH has achieved this, meeting the high bar for the treatment of chronic hypoparathyroidism,” said Andrea Palermo, M.D., Ph.D., an endocrinologist at Campus Bio-Medico University (Rome), who presented the five-year data during the European Congress of Endocrinology (ECE) 2026. “These data demonstrate the consistent, long-term benefits of this therapy and reinforce its potential as the emerging standard of care for the treatment of hypoparathyroidism.”

Highlights of Week 266 Results from the Phase 2 PaTH Forward Trial

82% of patients were responders for the multi-component endpoint of (1) serum calcium in the normal range, (2) taking no active vitamin D, and (3) taking <600 mg/day of calcium.
- 88% of patients had normal albumin-adjusted serum calcium levels, with a mean value of 9.0 mg/dL.
- 96% of patients achieved independence from active vitamin D, defined as not taking calcitriol or alfacalcidol.
- 95% of patients achieved independence from therapeutic doses of calcium, defined as taking <600 mg/day of calcium.
Significant improvements in kidney function were maintained, with mean (SE) eGFR of 78.0 (3.0) mL/min/1.73 m² at Week 266, reflecting a mean (SE) increase of 9.4 (1.9) mL/min/1.73 m² from baseline. Improvements were evident as early as Week 4, increased through Week 58, and were sustained over five years of treatment, in contrast to the expected normal age-related decline in eGFR in adults.ⁱ
Mean 24-hour urine calcium decreased substantially, normalized within 26 weeks, and remained normal through Week 266.
As measured by Hypoparathyroidism Patient Experience Scales (HPES), patients reported improvements in symptoms and health-related quality of life across all domains. Hypoparathyroidism-related physical and cognitive symptoms and impacts on physical functioning and daily life improved rapidly with TransCon PTH treatment and were maintained through Week 266.
As measured by SF-36, all mean health-related quality of life subscale and component summary scores rapidly normalized with TransCon PTH treatment and remained in the normative range through Week 266.
Mean BMD Z-scores (matched for age and sex) corrected from high baseline levels through Week 26 and remained above 0 through Week 266.
In the trial, TransCon PTH treatment was generally well-tolerated, with no new safety signals identified. Treatment-emergent adverse events (AEs) were mostly mild or moderate, and no discontinuations were related to study drug.
One patient developed transient, low-titer and non-neutralizing anti-PTH antibodies, with no impact on safety or efficacy. Over five years of treatment, no other patients developed anti-PTH antibodies.

“Across clinical trials and etiologies, TransCon PTH has shown a unique ability to replicate the actions of endogenous PTH, normalizing biochemistries and skeletal health and significantly improving kidney function and quality of life,” said Aimee Shu, M.D., Executive Vice President, Chief Medical Officer at Ascendis Pharma. “We are pleased to see TransCon PTH working as designed to transform treatment for patients living with this often-debilitating chronic disease.”

The PaTH Forward Trial of 59 adults with hypoparathyroidism (80% post-surgical, 20% non-surgical) included a 4-week randomized, double-blind, placebo-controlled period followed by a 262-week open-label extension (OLE) period, and measured a wide array of clinical, biochemical, and quality of life endpoints, consistent with the breadth of negative long-term impacts experienced by patients with hypoparathyroidism. Fifty-six of the original 59 patients enrolled (95%) completed the five-year trial. Endpoints included independence from conventional therapy (defined as <600 mg/day of calcium and no active vitamin D) and maintenance of normocalcemia (8.3 to 10.6 mg/dL). Renal function was assessed by estimated glomerular filtration rate (eGFR). Bone mineral density (BMD) measured by DXA scan was assessed at baseline and regular intervals through Week 266. Hypoparathyroidism-related symptoms and functional impacts were measured using the HPES. Health-related quality of life was measured using the 36-Item Short Form Survey (SF-36 version 2). Safety assessments included treatment-emergent AEs and 24-hour urine calcium excretion.

TransCon PTH is a prodrug of PTH (1-34), administered once daily, designed to provide stable levels of active PTH within the physiological range for 24 hours/day, approved as YORVIPATH^® in the United States, European Union, European Economic Area, and certain other jurisdictions as a treatment for adults with hypoparathyroidism.

A slide presentation with these data will be made available on the Investor Relations & News section of the Ascendis Pharma website: https://investors.ascendispharma.com.

About Hypoparathyroidism
Hypoparathyroidism is an endocrine disease caused by insufficient levels of parathyroid hormone (PTH), the primary regulator of calcium and phosphate balance in the body, acting directly on bone and kidney and indirectly on the intestine. 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%), while other etiologies include autoimmune, idiopathic, and genetic causes, including ADH1.

About Ascendis Pharma A/S
Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States. 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 within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to (i) the consistent, long-term benefits of TransCon PTH and its potential as the emerging standard of care for the treatment of hypoparathyroidism, (ii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients and (iii) Ascendis’ use of TransCon to create new and potentially best-in-class therapies to address unmet medical needs. 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, without limitation: dependence on third‑party manufacturers, distributors, and service providers for Ascendis’ products and product candidates; risks related to regulatory review and approval, including the possibility of delays, requests for additional data or analyses, restrictions or limitations on use, approval with labeling that is more limited than expected, or failure to obtain approval in the United States, European Union, or other jurisdictions; clinical development risks, including that results from ongoing or future trials may not confirm earlier data; unforeseen safety or efficacy findings in development programs or on‑market products; manufacturing, supply chain, quality, or logistics issues that could delay development or commercialization; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen research and development or selling, general and administrative expenses and other costs impacting Ascendis’ business generally; market acceptance, pricing, and reimbursement challenges, including payer coverage decisions and health technology assessments; competitive developments, including new or improved therapies; intellectual property protection, freedom‑to‑operate, and litigation risks; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; cybersecurity, data privacy, and information technology disruptions; and the impact of international economic, political, legal, compliance, public health, and business factors, including tariffs, trade policies, currency fluctuations, and geopolitical events. 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 11, 2026, 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, TransCon, and YORVIPATH^® are trademarks owned by the Ascendis Pharma group. © June 2026 Ascendis Pharma A/S.