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Oral Abstract Presentation

CSEMO2 - CSEM: Endocrinology & Metabolism Oral Abstracts: Long-Term Efficacy and Safety of Palopegteriparatide Treatment in Adults With Chronic Hypoparathyroidism: 4-Year Results From the Phase 2 PaTH Forward Trial

Friday, November 28, 2025
15:11 - 15:18 Toronto
Location: Room 201 A/B/C/D/E/F

    Presenting Author(s)

  • ALIYA KHAN, MD, FRCPC, FACP,FACE (she/her/hers)

    Professor of Clinical Medicine
    McMaster University
    McMaster University
    Oakville, Ontario, Canada

    Disclosure(s): Amolyt: Consultant (Ongoing), Research Grant (includes principal investigator, collaborator or consultant and pending grants as well as grants already received) (Ongoing), Speaker/Honoraria (Ongoing); Ascendis: Consultant (Ongoing), Research Grant (includes principal investigator, collaborator or consultant and pending grants as well as grants already received) (Ongoing), Speaker/Honoraria (Ongoing); Bridgebio: Consultant (Ongoing), Research Grant (includes principal investigator, collaborator or consultant and pending grants as well as grants already received) (Ongoing), Speaker/Honoraria (Ongoing)

Disclosure(s):
ALIYA KHAN, MD, FRCPC, FACP,FACE: Amolyt: Consultant (Ongoing), Research Grant (includes principal investigator, collaborator or consultant and pending grants as well as grants already received) (Ongoing), Speaker/Honoraria (Ongoing); Ascendis: Consultant (Ongoing), Research Grant (includes principal investigator, collaborator or consultant and pending grants as well as grants already received) (Ongoing), Speaker/Honoraria (Ongoing); Bridgebio: Consultant (Ongoing), Research Grant (includes principal investigator, collaborator or consultant and pending grants as well as grants already received) (Ongoing), Speaker/Honoraria (Ongoing)
Blinded Abstract: Palopegteriparatide is a prodrug of PTH (1-34), administered once daily, designed to provide active PTH within the physiological range for 24 hours/day. It is approved in the US, EU, and several other countries. This analysis investigated the efficacy and safety of palopegteriparatide in adults with chronic hypoparathyroidism through week 214 of PaTH Forward, a phase 2 trial with a 4-week randomized, double-blind, placebo-controlled period, followed by open-label extension through week 266.
At week 214, 95% (56/59) of participants remained in the trial; of those, 93% were independent from conventional therapy (no active vitamin D and ≤600 mg/day elemental calcium) and 98% had normocalcemia (2.07-2.64 mmol/L). Mean bone turnover markers C-terminal telopeptide of type 1 collagen (CTx) and procollagen type 1 N-terminal propeptide (P1NP) increased from low end of normal at baseline, peaked by week 26, and declined thereafter, remaining stable above baseline. Elevated baseline mean BMD Z-scores trended towards age- and sex-matched norms at lumbar spine, femoral neck, and total hip, largely stabilized after week 26 and remained above zero. At week 214, mean (SD) eGFR was 86.0 (21.7) mL/min/1.73 m2, reflecting a mean (SD) increase of 7.6 (13.7) mL/min/1.73 m2 from baseline. Mean (SD) 24-hour urine calcium levels normalized with palopegteriparatide and were maintained (≤6.2 mmol/day). TEAEs were mostly mild/moderate; no new safety signals were identified.
These results demonstrate sustained efficacy and safety of palopegteriparatide in adults with chronic hypoparathyroidism through week 214 of PaTH Forward, suggesting continued benefits in skeletal dynamics and renal function.