Announcement • 4h

Ascendis Pharma A/S Announces 5-Year Phase 2 Data Show TransCon PTH Replicated Systemic Actions of Endogenous PTH in Adults with Hypoparathyroidism

Ascendis Pharma A/S 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. 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. 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. 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.

Ascendis Pharma A/S Stock Price

ASND Share Price Performance

ASND Community Narratives

Long Acting TransCon Therapies Will Reshape Rare Endocrine Treatment Over The Coming Years

Long Term TransCon Pipeline Execution Will Shape Future Cash Flows And Business Durability

Long Term TransCon Pipeline Execution Will Shape Future Cash Flows And Business Durability

Long Acting TransCon Therapies Will Reshape Rare Endocrine Treatment Over The Coming Years

Trending Discussion

Recently Updated Narratives

Long Term TransCon Pipeline Execution Will Shape Future Cash Flows And Business Durability

Long Acting TransCon Therapies Will Reshape Rare Endocrine Treatment Over The Coming Years

Snowflake Analysis

Ascendis Pharma A/S Key Details

About ASND

Recent ASND News & Updates

Ascendis Pharma A/S Announces 5-Year Phase 2 Data Show TransCon PTH Replicated Systemic Actions of Endogenous PTH in Adults with Hypoparathyroidism

Ascendis Pharma Reports Strong Q1 Results and Plans New Rare Disease Data at ENDO 2026

Ascendis Pharma: Rare Endocrine Compounder Entering Growth Phase (Initiating Buy)

Recent updates

Ascendis Pharma A/S Competitors

Regeneron Pharmaceuticals

Teva Pharmaceutical Industries

Alnylam Pharmaceuticals

Agilent Technologies