BioMarin to Present Phase 1/2 Data for BMN 270 at 2016 Hemophelia Congress

San Rafael, California-based BioMarin Pharmaceutical will present interim data of an open-label, Phase 1/2 clinical trial of BMN 270, an investigational gene therapy treatment for hemophilia at the upcoming XXXII International Congress of the World Federation of Hemophilia (WFH) July 24-28 in Orlando, according to a news release.

The late-breaking abstract has been accepted by the congress, the global biotech said.

John Pasi, professor of Haemostasis and Thrombosis, Barts and the London School of Medicine, and honorary consultant hematologist at The Royal London Hospital, a lead investigator on the clinical trial, will present the data during the Late Breaking Gene Therapy session. Following the presentation, BioMarin management will host an investor event.

The Phase 1/2 clinical trial of BMN 270 gene therapy is evaluating the efficacy and safety of the treatment in up to 12 severe Hemophilia A patients.

The primary endpoints of the trial include the safety evaluation of a single intravenous dose of a recombinant AAV, human-coagulation Factor VIII vector and the baseline change of Factor VIII expression level following 16 weeks post-infusion. The kinetics, duration, and magnitude of AAV-mediated Factor VIII activity are being determined and associated to the proper dose of BMN 270.

The clinical trial is a dose escalation study that aims to assess an increase in the levels of Factor VIII. The secondary endpoints of the clinical trial include the effect of the treatment on the frequency of Factor VIII replacement therapy, the number of bleeding episodes that require treatment, and immune responses. Patients will be followed up for a period of five years.

Patients with hemophilia A do not produce sufficient functional Factor VIII to prevent bleeding. These patients are currently treated with prophylactic or on-demand infusions of plasma-derived or recombinant Factor VIII.

BMN 270 addresses the underlying genetic defect preventing the expression of functional Factor VIII. This is made by using an adeno-associated virus (AAV) vector to deliver a functional copy of the factor VIII gene to patients’ own cells. The goal is that a single infusion of BMN 270 provides a long-lasting increase in levels of Factor VIII.

BMN 270 has been granted orphan drug designation from the U.S. FDA and the European Commission (EC). Phase 3 design preparation and high volume manufacturing plans are underway.