BPS804 Anti‐Sclerostin Antibody in Adults With Moderate Osteogenesis Imperfecta: Results of a Randomized Phase 2a Trial

@article{Glorieux2017BPS804AA,
  title={BPS804 Anti‐Sclerostin Antibody in Adults With Moderate Osteogenesis Imperfecta: Results of a Randomized Phase 2a Trial},
  author={Francis H. Glorieux and J. P. Devogelaer and Michaela Durigova and Stefan Goemaere and Sarah Hemsley and Franz Jakob and Uwe Junker and Jon L. Ruckle and Lothar Seefried and Peter J. Winkle},
  journal={Journal of Bone and Mineral Research},
  year={2017},
  volume={32}
}
This 21‐week, open‐label, phase 2a trial aimed to evaluate the pharmacodynamics and safety of multiple, escalating infusions of BPS804, a neutralizing, anti‐sclerostin antibody, in adults with moderate osteogenesis imperfecta (OI). Patients received BPS804 (three escalating doses each separated by 2 weeks [5, 10, and 20 mg/kg]) or no treatment (reference group). The primary efficacy endpoints were mean changes from baseline to day 43 in: procollagen type 1 N‐terminal propeptide (P1NP… 
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4‐PBA Treatment Improves Bone Phenotypes in the Aga2 Mouse Model of Osteogenesis Imperfecta
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In vivo data demonstrate that a postweaning 5‐week 4‐PBA treatment increased total body length and weight, decreased fracture incidence, increased femoral bone volume fraction (BV/TV), and increased cortical thickness, and the potential use of chemical chaperones as an adjunct treatment for forms of OI associated with ER stress is suggested.
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