TransCon PTH is an investigational once-daily prodrug providing sustained release of active PTH designed to maintain stable PTH levels in the physiological range for 24 hours/day. The PaTHway trial met all primary and key secondary endpoints with statistically significant differences from placebo. Most adverse events (AEs) were mild/moderate. Injection-site reactions and headaches were the most common treatment-related AEs.

In this study, we found that caveolae-mediated endocytic TβRI degradation was reduced in OI due to the impaired regulation of type I collagen on FAK/Src activation-dependent Caveolin-1 phosphorylation, which further enhanced the TGF-β/Smad signaling on the inhibition of osteoblast differentiation. Inhibition of TβRI kinase by SD-208 effectively reduced fractures and improved bone mass and strength in an OI mouse model.