BioMarin’s BMN 270 has experts eye thrombosis potential, unknown dose efficacy in next Phase I/II data readout in haemophilia A

18 Nov 2016

BioMarin Pharmaceutical’s (NASDAQ:BMRN) BMN 270’s Phase I/II haemophilia A drug has most experts agreeing its interim factor VIII (FVIII) level increases may cause thrombosis in Phase I/II’s next data read out. Whilst the interim data’s week 20-28 assessment period didn’t report any thrombosis signals, fluctuating FVIII levels can raise concerns for the safety element of its co-primary endpoint, they said. However, one expert said the thrombosis risk could be overstated.

BMN 270’s gene therapy mechanism of action (MOA) caused additional safety concerns, said one expert, as its vector may integrate into the genome and cause adverse events.

Safety concerns aside, most experts were encouraged by the extent of BMN 270’s Phase I/II interim FVIII level increase effects for continued study efficacy but noted the new, unassessed 4x1013 vg/kg dose has unclear outcomes.

Several analyst reports anticipate peak sales could reach up to USD 1bn.

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