BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced top-line data from a phase III study and updated long-term data from phase I/II study on its promising gene therapy candidate, valoctocogene roxaparvovec, in patients with hemophilia A.
Data from one cohort of the phase III study — GENEr8-1 — showed that treatment with the gene therapy candidate achieved factor VIII levels of 40 international units per deciliter (IU/dL) in eight patients, a pre-specified criterion for accelerated approval. In this cohort, 20 patients were dosed 6e13 vg/kg of valoctocogene roxaparvovec for 23 to 26 weeks. Annual Bleed Rate (“ABR”) also reduced significantly from baseline.
The company is planning to meet with regulatory authorities to discuss the requirements for a regulatory submission and review of phase III data. The timeline for submission of a regulatory application will be provided by the third quarter of this year.
Separately, data from the long-term phase I/II study study showed that treatment with valoctocogene roxaparvovec (both 6e13 vg/kg and 4e13 vg/kg doses) led to a substantial reduction (in excess of 92%) in bleeding and the need for Factor VIII infusions.
Moreover, data from the early-stage study showed that significant reduction in ABR was sustained in the third yearfollowing infusion of the high dose (6e13 vg/kg) of valoctocogene roxaparvovec. However, data showed that factor VIII levels decreased over the same period. Although the rate of decrease in factor VIII levels slowed in the third year compared to the second year of treatment. The company anticipates bleeding control to be maintained for at least eight years after gene transfer, based on statistical modeling.
Shares of BioMarin fell 5.1% on May 28 following the data readout. This could be because the peak level of factor VIII achieved in the phase III study was lower than that achieved in the phase II study. Moreover, investors were underwhelmed by the expected duration of raised factor VIII levels in hemophilia A patients.
BioMarin shares have increased 4.6% so far this year compared with the industry’s rise of 2.2%
BioMarin is developing valoctocogene roxaparvovec in two separate phase III studies – GENEr8–1 (6e13 vg/kg dose) and GENEr8–2 (4e13 vg/kg dose). Enrollment in both the studies is expected to be completed by the third quarter of 2019. However, BioMarin has completed the enrollment in the initial cohort of patients in the GENEr8–1 study that will be included in a potential accelerated submission.
Meanwhile, approved products and several developing candidates for hemophilia will give competition to valoctocogene roxaparvovec following a potential approval. The currently marketed therapies for hemophilia include Bayer AG’s (BAYRY - Free Report) Kovaltry, approved in both the United States and the EU for children and as well as adults. Alnylam Pharmaceuticals (ALNY - Free Report) is developing its hemophilia candidate, fitusiran in partnership with Sanofi (SNY - Free Report) in a phase III program.
BioMarin currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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