According to a story from Financial Buzz, the biopharmaceutical company Mereo BioPharma Group plc recently announced that it has successfully concluded its Type B End-of-Phase 2 meeting with the US Food and Drug Administration (FDA). This meeting was in regards to the continued development of the company’s experimental drug setrusumab, which is designed as a treatment for the rare disease osteogenesis imperfecta (OI).
About Osteogenesis Imperfecta
Osteogenesis imperfecta, which is also referred to as brittle bone disease, is a group of genetic disorders which are distinguished by abnormally weak bones which break easily. The effect can range from mild to severe. These disorders are linked to a lack of type I collagen, leading to issues with connective tissue. In the vast majority of cases, this is caused by a mutation affecting either the COL1A1 or COL1A2 genes. Aside from the weakened bones, osteogenesis imperfecta also inflicts other symptoms and signs, such as short stature, breathing issues, dental problems, loosened joints, hearing loss, a blue tinge to the whites of the eyes, and aortic dissection. Currently, there is no cure for the disease and no FDA approved treatments. Avoiding smoking and exercising regularly can help reduce the risk of fractures. Other treatments may include physiotherapy, surgery, and biphosphonates, which can help reduce fractures, particularly in children. To learn more about osteogenesis imperfecta, click here.
Designing Future Trials
The purpose of the meeting was to review data from the recently concluded phase 2b clinical trial of setrusumab, which tested the effects of the treatment on adult patients. In addition, the FDA also agreed to the design for the phase 3 trial, which will test the treatment on pediatric patients. The phase 3 trial is to include two different cohorts with a total of 160 patients. These patients will range from age two to 18 years. One cohort will be the ‘safety’ cohort, that will confirm the dose level. The efficacy cohort will see patients treated with either setrusumab or standard biphosphonates for a 12 month period.
The primary endpoint for the study will be rates of fracture following the 12 month period; secondary endpoints will include quality of life measures, patient reported outcomes, bone mineral density at the lumbar spine, and other bone biomarkers.
With the parameters of the phase 3 study now arranged, the development of this treatment is now on track to continue.