Ultragenyx, Mereo tout sustained anti-fracture effect of brittle bone disease drug
Clinical ResultLicense out/inPhase 2Phase 3
Ultragenyx and Mereo BioPharma unveiled longer-term results for their investigational anti-sclerostin antibody setrusumab showing sustained reductions in fracture rates and improvements in bone mineral density (BMD) for patients with the rare, inherited brittle bone disease osteogenesis imperfecta (OI).
Previously reported six-month data had shown a 67% reduction in annualised rate of radiologically confirmed fractures across all 24 patients in the Phase II portion of the pivotal ORBIT trial following at least six months of treatment with setrusumab.
The latest results indicate this benefit was maintained in OI patients with at least 14 months of follow-up. Setrusumab also resulted in a mean 22% increase in lumbar spine BMD at 12 months compared to baseline levels across all age groups in the study, which includes patients from 5 to
"The clinically meaningful continued improvement in BMD suggests that new and stronger bone is being created that has resulted in an important reduction in fractures across age groups and types of OI," noted Eric Crombez, chief medical officer at Ultragenyx.
No new safety signals emerged with the longer-term setrusumab exposure. There were no treatment-related serious adverse events (AEs) either, and AEs were "generally consistent" with those seen previously in the Phase IIb ASTEROID study of setrusumab in 112 adults with OI, the companies said. Detailed 14-month data will be presented at a future scientific meeting.
Late-stage testing under way
The Phase III portion of the ORBIT study has enrolled an additional 158 patients aged 5 to 25 years, randomised to receive setrusumab or placebo, with a primary endpoint of annualised clinical fracture rate. Primary completion is set for early next year. Meanwhile, in the open-label Phase III COSMIC study, 69 children ages 2 through 7 years were randomised to setrusumab or intravenous bisphosphonate therapy, assessing reduction in total fracture rate.
Enthusiasm appears to be building for setrusumab. In a recent note, Goldman Sachs analyst Salveen Richter expressed confidence in the drug based on positive feedback from key opinion leaders regarding the Phase II data. She noted "the potential for setrusumab to improve bone formation and strength in addition to reducing fractures via its sclerostin inhibition mechanism, a key differentiator vs. standard-of-care bisphosphonates."
Ultragenyx entered a collaboration and licence agreement with Mereo in 2020 to bolster its existing bone franchise, which includes Crysvita (burosumab-twza) for X-linked hypophosphatemia and tumour-induced osteomalacia. Mereo received $50 million upfront as part of the deal and is eligible for milestones up to $254 million.
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