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Mereo BioPharma Group plc

MEREO'S BPS-804 GRANTED EU ORPHAN DRUG STATUS FOR OSTEOGENESIS IMPERFECTA

London, 30 June 2016 - Mereo BioPharma Group plc ("Mereo" or the "Company") (AIM:MPH), a clinical stage, UK-based, biopharmaceutical company focused on rare and specialty diseases, is pleased to announce that the European Commission (EC) has granted BPS-804 orphan drug designation for the treatment of osteogenesis imperfecta (OI), 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.

Commenting on today's announcement, Dr Denise Scots-Knight, CEO of Mereo 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."

The EC may grant orphan drug designation to drugs intended for the treatment of life threatening or chronically debilitating rare diseases where no therapeutic options are either authorised or where the drugs will be of significant benefit to those affected by the condition. Rare diseases are those defined as having a prevalence of no more than five in 10,000 persons in Europe. The designation provides development and commercial incentives, including 10 years of market exclusivity, protocol assistance on the development of the drug, including clinical studies, and certain exemptions from or reductions in regulatory fees.

Enquiries

About Mereo

Mereo is a UK-based specialty biopharmaceutical company focused on the development of innovative medicines that aim to address unmet medical needs in rare and specialty disease areas and improve patient quality of life.

Pharmaceutical companies face increasingly difficult choices in the allocation of internal resources to their drug development programs. With significant P&L constraints and rich pipelines it has become increasingly difficult for many companies to fully fund and advance development all of their drug candidates, especially beyond Phase 2. Mereo has been formed to take advantage of the global pharmaceutical industry's drive for creative ways to progress their clinical development pipelines. 

Mereo's initial mid-late stage portfolio consists of three exceptionally well characterised novel products for the treatment of diseases with considerable unmet medical need, which it acquired from Novartis Pharmaceuticals in July 2015. Each of these programmes has a comprehensive dataset for both pre-clinical and proof-of-concept clinical studies. Since that time the Company has initiated a Phase 2 study and a Phase 2b study in relation to two of the product candidates in 2016 and intends to commence registration studies during H2 2016 for the third product candidate. Additional product opportunities are under evaluation.

Mereo's focus is the development of innovative medicines that have the potential to significantly transform the lives of patients suffering from rare and other specialised conditions around the world. To do this, the Company depends on the combination of its team's expertise in selecting and acquiring product opportunities, creating value in the development pipeline and structuring creative transactions. Mereo's internal expertise is complemented by a unique partnership with a leading global CRO, ICON. 

Mereo combines the operational discipline and efficiency of a small company with the financial resources to conduct comprehensive clinical studies. Mereo will rapidly progress each of the products through further value inflection points before partnering or divesting its products. The Company also has the option to directly commercialise products, for example in orphan disease indications. 

About osteogenesis imperfecta (OI)

OI is an orphan genetic disorder that is characterized by fragile bones that break easily. In addition to fractures, people with OI often have muscle weakness, hearing loss, fatigue, joint laxity, curved bones, scoliosis, and short stature. The majority of cases of OI (estimated at approximately 90 %) are caused by a dominant mutation in a gene coding for type I collagen, a key component of healthy bone. Treatment of OI is supportive, focusing on minimizing fractures and maximizing mobility, but to date, there are no treatments that address the underlying bone weakness.

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