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EC grants orphan drug status to Mereo’s BPS-804 for osteogenesis imperfecta

Mereo BioPharma Group announced that its drug BPS-804 has been granted orphan drug designation by the European Commission (EC) in the treatment of osteogenesis imperfecta (OI).

Osteogenesis Imperfecta (OI) is a rare, chronic genetic disorder that results in bones that can break easily. The Company intends to initiate a potential registration trial of BPS-804 in the second half of 2016.

In March 2016, BPS-804 was granted orphan drug designation for the treatment of OI by the U.S. Food and Drug Administration (FDA).

OI is a rare condition that is estimated to affect a minimum of approximately 20,000 and possibly as many as 50,000 patients in the United States. In Europe, approximately 7.5 out of 100,000 people have the condition.

Current treatment largely relies on the acute management of fractures as they occur and the use of bisphosphonate drugs, although the Company believes there is no clear data demonstrating that bisphosphonate drugs reduce fractures. BPS-804 aims to demonstrate a benefit compared to placebo in terms of fractures in OI patients.

BPS-804 works by inhibiting sclerostin, which inhibits the activity of bone-forming cells, known as osteoblasts. The Company believes that by blocking sclerostin, BPS-804 will induce or increase osteoblast function and maturation of these cells, increasing bone formation and reducing bone resorption, thereby reducing fractures in OI patients. Phase 2 proof of concept data in OI patients demonstrated a statistically significant improvement in bone biomarkers and bone mineral density.

Mereo CEO Dr Denise Scots-Knight said: "Osteogenesis imperfecta is a debilitating and painful disease. The EC orphan drug designation grant follows the recent grant from the FDA.

"It is another important regulatory milestone and marks a significant step forward for BPS-804. We believe BPS-804 has the potential to become the first therapy to reduce fractures and improve quality of life in osteogenesis imperfecta patients.

"Initial clinical data generated with BPS-804 has been highly encouraging and we look forward to progressing this product candidate towards registration."

