RARE - Ultragenyx Posts New Data From Bone Disease Study | Benzinga
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), along with partner Mereo BioPharma (MREO), announced new positive 14-month data from the phase II portion of the phase II/III Orbit study evaluating setrusumab (UX143) in patients with osteogenesis imperfecta.
The data showed that the large reduction in fracture rates previously reported in patients treated for a minimum of six months was sustained in patients treated for at least 14 months.
OI is a group of genetic disorders that impact bone metabolism. It is characterized by increased bone fragility and a high risk of fractures. Currently, there are no approved treatments for this debilitating condition, representing a highly unmet medical need.
As of the data cut-off date of May 24, 2024, the new data showed that treatment with setrusumab resulted in a large, sustained 67% reduction in annualized fracture rate and persistent median annualized fracture rate of 0.00.
Treatment with setrusumab also resulted in clinically meaningful continued improvement in lumbar spine bone mineral density (BMD) with a mean increase from baseline of 22% at the 12-month timepoint.
Previously, the company reported that treatment with setrusumab for at least six months reduced the annualized fracture rate by 67% in patients with OI.
Per the company, the new data underlines the potential of setrusumab in reducing the incidence of fractures and improving BMD in patients with OI.
No treatment-related ...