- Ultragenyx Pharmaceutical Inc RARE and Mereo BioPharma Group plc MREO announced data from the dose-selection Phase 2 portion of the Phase 2/3 Orbit study of setrusumab for osteogenesis imperfecta (OI).
- Setrusumab demonstrated statistically significant increases in levels of serum P1NP, a sensitive marker of bone formation, and a substantial and significant improvement in bone mineral density (BMD) by three months.
- In the 20 mg/kg cohort, there was a mean serum P1NP increase of 57% from baseline over the first month.
- Because of the higher baseline, P1NP level in younger patients represents an approximately 8-fold greater increase in serum P1NP over one month in pediatric and adolescent patients compared to adult OI patients.
- The absolute effect of setrusumab on increasing serum P1NP over one month with the 20 mg/kg dose was approximately 80% of the effect achieved with the 40 mg/kg dose, demonstrating a dose response.
- Patients on placebo at the 1-month timepoint (n=4) showed no significant change in mean serum P1NP from baseline.
- The increase in BMD observed in the Orbit patient population over the first three months was consistent with the rapid increase in serum P1NP levels. It was similar to results that took one year to achieve in the ASTEROID study in adult OI patients.
- Treatment with setrusumab for three months increased lumbar spine BMD from a baseline of 9.4% at 20 mg/kg (n=10), representing a substantial mean change in Z-score of +0.65.
- Treatment with 40 mg/kg resulted in a 9.8% BMD increase. Patients on placebo at the 3-month timepoint showed no significant change in BMD or change in lumbar spine Z-score.
- Screening has begun for the Phase 3 portion of the study, designed to enroll approximately 195 patients.
- Price Action: MREO shares are up 11.40% at $1.17 during the premarket session on the last check Tuesday. RARE shares closed 3.86% higher at $53.23 during after-hours trading on Monday.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
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