(Alliance News) - Mereo BioPharma Group PLC on Friday said it successfully completed a type B end-of-phase 2 meeting with the US Food & Drug Administration over setrusumab.
Shares in clinical stage biopharmaceutical firm Mereo were down 5.8% at 24.03 pence in London in afternoon trading.
Setrusumab is being developed by Mereo to treat children and adolescents with osteogenesis imperfecta, a rare genetic disorder which causes fragile bones which break easily and has no approved treatments.
After a review of data from Mereo's phase 2b Asterios study in adults, the FDA has agreed n the design of a phase 3 paediatric study. This will be completed before Mereo submits a biologics licence application in the US.
Mereo said this is in line with the proposed pivotal paediatric study design already agreed in principle with the European Medicines Agency.
The planned phase 3 study is to include two cohorts in around 160 children and adults aged 2 to 18 years who have osteogenesis imperfecta type 1, 3, or 4 as well as "a confirmed genetic mutation leading to a collagen defect".
A safety cohort with a smaller number of patients will allow Mereo to confirm the setrusumab dose while an efficacy cohort will participate in a 12-month randomised double-blind active control design with two arms.
In one arms of the study, patients will be given setrusumab at a dose equivalent to the high-dose arm from the phase 2b Asteroid study, while the other arm will get "a standardized bisphosphonate".
The primary endpoint of the study will be fracture rate versus active control after 12 months, while the secondary endpoints will be bone mineral density at the lumbar spine after 12 months compared to baseline.
Chief Executive Denise Scots-Knight said: "We are pleased with the productive feedback we received from the FDA during our end-of-phase 2 meeting. Overall, the final pivotal study design will be consistent with what we had previously agreed to in principle with the EMA.
"We now have a clear path forward to initiate a phase 3 study of setrusumab in pediatric [osteogenesis imperfecta] that incorporates feedback from both the FDA and EMA and is intended to support the filings of a [biologics licence application] in the United States and a marketing authorisation application in the EU.
"This is an important milestone for Mereo and we are excited to continue to develop setrusumab as there are no currently approved therapies for OI and treatment options are greatly needed. Preparations for the phase 3 study are underway."
By Anna Farley; annafarley@alliancenews.com
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