Both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) had granted Orphan Drug Designation to BPS-804 for osteogenesis imperfecta, a rare, chronic genetic disorder that makes bones to break easily.
The investigational osteogenesis imperfecta drug has also been accepted into the EMA’s Adaptive Pathways Program and was given PRIority MEdicines (PRIME) designation.
Its multi-centre, randomized, double-blind, dose-finding phase 2b study dubbed ASTEROID will evaluate it in a total of 112 adult patients across the US and Europe, said Mereo BioPharma.
ASTEROID will have four patient arms, which includes an open-label arm. The six-month data from the mid-stage study on the top dose of BPS-804 is expected to be reported in H1 2019 while the 12-month data will be released in H2 2019.
On the other hand, Mereo BioPharma expects the top-line 12 month data from the blinded dose ranging part of the ASTEROID trial to come out in the fourth quarter of 2019.
The primary endpoint of ASTEROID is variation from baseline of Bone Mineral Density (BMD) after 12 months as measured by High Resolution peripheral Quantitative Computed Tomography (HR-pQCT) and the secondary endpoints of BMD using traditional two-dimensional dual-energy X-ray absorptiometry (DXA) measurement along with measurement of serum bone biomarkers.
Mereo BioPharma chief medical officer Alastair Mackinnon said: “This is another important milestone in the development of BPS-804 for Osteogenesis Imperfecta, which is a serious, debilitating and painful orphan disease for which there are currently no EMA or FDA approved treatments.
“We believe BPS-804’s mechanism of action is specifically suited to OI and has the potential to become a novel treatment option that could reduce fractures and improve quality of life of these patients.”
BPS-804 is a fully humanized monoclonal antibody, designed to inhibit sclerostin, a protein which in turn inhibits the activity of bone-forming cells, called as osteoblasts.
According to Mereo BioPharma, by blocking sclerostin, BPS-804 will induce or increase osteoblast function and maturation of the cells, leading to increased bone formation and reduction of bone resorption, to eventually reduce fractures in patients with osteogenesis imperfecta.