Hailshadow/iStock via Getty Images Mereo BioPharma ( NASDAQ: MREO ) traded higher premarket Wednesday after the company and its partner Ultragenyx Pharmaceutical ( NASDAQ: RARE ) announced 14-month data from a Phase 2 portion of an ongoing Phase 2/3 trial for their bone disorder therapy setrusumab (UX143). According to the companies, a data cut on May 24, 2024, suggests that setrusumab continued to significantly cut the rate of fracture occurrence in patients with osteogenesis imperfecta, a genetic disorder affecting bone metabolism. Citing data from 24 patients with an average treatment duration of 16 months, the companies said that setrusumab led to a 67% reduction in the median annualized fracture rate, a measure consistent with the primary efficacy endpoint in the trial's Phase 3 portion.
The decline in annualized fracture rates coincided with a 22% mean increase in lumbar spine bone mineral density (BMD) at month 12 from baseline and a further improvement from 14% seen at six months. There were no treatment-related serious adverse events, and the experimental therapy demonstrated an adverse event profile in line with the findings from a Phase 2b trial. The international trial Orbit enrolled 24 patients aged 5 to 25 with OI, and its Phase 3 component has enrolled an additional 158 patients.
More on Mereo BioPharma, Ultragenyx, etc. Ultragenyx: Modest Sales Growth, With Multiple Inflection Points Approaching Ultragenyx Pharmaceutical, Inc. (RARE) Q1 2024 Earnings Call Tran.
