BioMarin Pharma (BMRN) Announces MHRA in UK Approves Continued Enrollment in 1/2 Study of BMN 270 in Hemophilia A

October 13, 2016 4:02 PM EDT

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BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom approved continued enrollment into the open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. BioMarin had previously announced that after enrolling the first 9 patients in the study, that dosing of patients had been suspended due to observed increases in alanine aminotransferase (ALT) levels that exceeded a pre specified threshold set by the company. Following study suspension, the company reviewed safety and efficacy data on the 9 patients with the MHRA, and based on its review, the MHRA approved resumption of the study. The agency also approved the company’s proposed amendments to the study, which included eliminating the requirement for prophylactic corticosteroids and increasing potential additional enrollment from up to three additional patients to up to six additional patients.

BioMarin intends to resume enrollment in the Phase I/2 study before the end of 2016. Based on protocol amendments, three patients will be enrolled at a dose of 4 x 1013 vg/kg, and an additional three may be enrolled at this dose or the previously tested high dose of 6 x 1013 vg/kg. In the up to six additional patients, the requirement for prophylactic corticosteroids has been removed and the threshold for starting therapeutic corticosteroids has been increased. Safety and efficacy data from these patients will inform the Phase 2b study planned to begin in the second half of 2017.

“We are pleased that MHRA has approved the resumption of enrollment of the BMN 270 study, as well as the study amendments. We believe that the amendments will allow us to optimize the design of a robust Phase 2b clinical trial, which potentially could support an accelerated approval by health authorities,” said Hank Fuchs, M.D., Chief Medical Officer at BioMarin. “We are grateful to the patients who are participating in this current study, and are encouraged by the results so far for this Phase 1/2 trial.”

Phase 1/2 Study Design

The current Phase 1/2 study is evaluating the safety and efficacy of BMN 270 gene therapy in up to 15 patients with severe hemophilia A defined by the WFH as less than 1% of blood clotting factor. The primary endpoints are to assess the safety of a single intravenous administration of a recombinant AAV vector coding for human-coagulation factor VIII and to determine the change from baseline of factor VIII expression level at 16 weeks after infusion. The kinetics, duration and magnitude of AAV-mediated factor VIII activity in individuals with hemophilia A will be determined and correlated to an appropriate BMN 270 dose.

This is a dose escalation study with the goal of observing an increase in factor VIII levels. Secondary endpoints include assessing the impact of BMN 270 on the frequency of factor VIII replacement therapy, the number of bleeding episodes requiring treatment and any potential immune responses. Patients will be monitored for safety and durability of effect for five years.

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