BioMarin announces positive Phase I/II data for investigational haemophilia A gene therapy

BioMarin Pharmaceutical on Wednesday unveiled "encouraging" preliminary data from a Phase I/II study of the experimental haemophilia A gene therapy BMN 270, an adeno-associated virus (AAV)-factor VIII vector designed to restore factor VIII plasma concentrations. Interim data showed that factor VIII activity levels were increased by as much as 60 percent in patients who received a single dose of the therapy. BioMarin chief medical officer Hank Fuchs remarked "BMN 270 could have the potential to reduce and possibly eliminate the need for infusions of factor VIII."

In the study, eight patients with severe haemophilia A received a single dose of BMN 270, with six patients treated at the highest dose. The primary endpoints assess the safety of a single intravenous administration of a recombinant AAV vector coding for factor VIII and the change from baseline of factor VIII expression at 16 weeks post-infusion. BioMarin added that the kinetics, duration and magnitude of AAV-mediated factor VIII activity in patients with haemophilia A will be examined and correlated to an appropriate BMN 270 dose.

Results so far indicate that five of the six high-dose patients experienced increases in factor VIII of more than 5 percent, with two achieving activity levels of over 50 percent. Moreover, BioMarin noted that all six high-dose patients saw the severity of their disorder improve from severe to moderate, mild or normal range in terms of factor levels based on World Federation of Hemophilia criteria.

Commenting on the findings, Evercore ISI analyst Mark Schoenebaum remarked "while the data presented today were small numbers and of relatively limited follow-up…the data were encouraging, although looks like [there is] significant variability from patient to patient." He said the results "suggest a path forward for BMN 270 in haemophilia A and set a reasonably high bar for competition that will follow." Meanwhile, Michael Yee and Judy Liu of RBC Capital Markets said the data, which look "much better than expected," suggest that given the gene therapy is leading to strong production of factor VIII, there may be no need for recombinant factor VIII, currently a $4-billion market led by Baxalta and Shire. The analysts expect the findings will "be well received by investors for the potential for an important, and possibly very large, growth driver in the future not in consensus estimates."

BioMarin indicated that planning is underway for Phase III testing and high-volume manufacturing of BMN 270. The gene therapy was recently granted orphan drug status for the treatment of haemophilia A by regulators and EU</a> BioMarin Receives European Orphan Drug Designation for BMN 270, First Investigational AAV-Factor VIII Gene Therapy for Patients with Hemophilia A 

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