BridgeBio Pharma : BBIO – Encaleret – ADH1 – ENDO 2025 – Baseline Characteristics of Pediatric Participants in the CLARIFY Disease Monitoring Study

Published: 2025-07-14 16:57:04 ET BBIO

Published on 07/14/2025 at 16:57

Benson M1, Padidela R2, Bacchetta J3, Ahmad T4, Jacobsen C5, Linglart A6, Tebben PJ7, Kubota T8,9, Ma NS10, Mora S11, Wasserman H12,13, Chen J14, Li D14, Roberts MS14, Adler S14, Mäkitie O15

1Nemours Children's Health, Jacksonville, FL; 2Royal Manchester Children's Hospital & Faculty of Biology Medicine and Health, University of Manchester, Manchester, UK; 3Hospices Civils de Lyon, INSERM1033, Lyon, France; 4UCSF Benioff Children's Hospital, Oakland, CA; 5Boston Children's Hospital, Boston, MA; 6Paris Saclay University, AP-HP Hopital Bicetre, Le Kremlin-Bicêtre, France; 7Yale University School of Medicine, New Haven, CT; 8The University of Osaka Hospital, Suita, Japan; 9Osaka Women's and Children's Hospital, Izumi, Japan; 10Children's Hospital Colorado, Aurora, CO; 11Laboratory of Pediatric Endocrinology, Department of Pediatrics, IRCCS Ospedale San Raffaele, Milano, Italy; 12Cincinnati Children's Hospital Medical Center, Cincinnati, OH; 13University of Cincinnati College of Medicine, Cincinnati, OH; 14Calcilytix Therapeutics, Inc., a BridgeBio Company, San Francisco, CA; 15Children's Hospital, University of Helsinki and Helsinki University Hospital,

Helsinki, Finland

MAT-US--278 05/2028

Dr. Benson: research support from BridgeBio for the CLARIFY trial. I have no other relevant disclosures to this presentation.

The CLARIFY disease monitoring study is a global, multicenter, longitudinal, non-interventional study to understand disease burden, management, and progression in pediatric and adult participants with ADH1 or ADH2 over 5 years.

(N=95 enrolled)

Standard-of-Care (SoC)

Inclusion Criteria

Methods

Documented mutation in the CASR gene (for ADH1) or the GNA11 gene (for ADH2) that is predicted to be either pathogenic, likely pathogenic, or a variant of unknown significance (VUS)

Diagnosis of hypoparathyroidism

Age birth to 90 years

AM, morning; DXA; dual-energy X-ray absorptiometry; CT, computed tomography; Hx, history; SoC, standard-of-care.

SoC treatment on study is as prescribed by treating physicians.

Blood and urine samples collected at pre-specified time points and tested at a central laboratory.

Blood samples collected after a minimum of 4-hr fasting and prior to AM dosing of SoC therapy (if applicable).

Urine samples collected over 24-hrs and assessed at central lab for completeness using pre-specified criteria.

Imaging performed locally and interpreted by central reader.

Characteristic

Analysis Population:

pediatric participants with ADH1/2 (n=25)

ADH Subtype

88% (n=22) ADH1; 12% (n=3) ADH2

Age (years) at enrollment

9.0 (median); 5.0 - 13 (IQR)

Gender

64% (n=16) female; 36% (n=9) male

Age of hypocalcemia dx (mos)

6.0 (median); 0 - 27 (IQR)

SoC Treatment for ADH1/2

64% (n=16) active vitamin D and/or calcium

32% (n=8) both active vitamin D and calcium 24% (n=6) calcium only

8.0% (n=2) active vitamin D only

44% (n=11) native vitamin D (cholecalciferol) 20% (n=5) magnesium

16% (n=4) parathyroid hormone replacement (teriparatide)

20% (n=5) no treatment 12% (n=3) thiazide diuretics 12% (n=3) potassium

12% (n=3) phosphate binder (Sevelamer)

ADH1

ADH2

*Native vitamin D (cholecalciferol) is not accounted for in bar plot

Clinical data cut off 10 FEB 2025.

Dx, diagnosis; IQR, inter-quartile range; n, number of participants; SoC, standard-of-care.

[LEFT] Intact parathyroid hormone (iPTH) concentrations below the limit of detection (<6 pg/mL) are substituted with the constant value 0. Green horizontal line indicate reference range (15-65 pg/mL).

[RIGHT] Green lines indicate reference range of cCa [0-<1y 8.4-11.2 mg/dL (dotted lines); 1-17y 8.6-10.2 mg/dL (solid lines)]; orange line indicates reference limit of weight-adjusted 24-hr uCa excretion (4.0 mg/kg/day). The pts without an evaluable 24-hr uCa result (n=6) are excluded from the plot.

Disclaimer

BridgeBio Pharma Inc. published this content on July 15, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on July 14, 2025 at 20:56 UTC.