BioMarin Begins Mid Stage Study on Gene Therapy Candidate

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BioMarin Pharmaceutical Inc.BMRN initiated dosing its first patient in a phase I/II study - BMN 207-203 - evaluating its gene therapy candidate, valoctocogene roxaparvovec, in severe hemophilia A patients with pre-existing AAV5 antibodies.

Meanwhile, BioMarin is already evaluating two different doses of valoctocogene roxaparvovec - 6e13 vg/kg and 4e13 vg/kg - in two separate phase III studies in severe hemophilia A in patients without pre-existing AAV5 antibodies. By studying the candidate in another study in patients that already have antibodies to AAV5, BioMarin aims to develop valoctocogene roxaparvovec with the potential to eliminate the need for chronic treatment in severe hemophilia A across all patient sub-groups

BioMarin's share price has declined 0.6% so far this year, comparing favorably with the industry 's decrease of 9.1%.

The phase I/II study will enroll patients in two cohorts based on AAV5 antibody levels and evaluate a dose of 6e13 vg/kg ofvaloctocogene roxaparvovec. The primary endpoint of the study is the safety of the candidate while secondary endpoints include FVIII activity level, frequency of required FVIII replacement therapy and the number of bleeding episodes requiring treatment after therapy.

The candidate enjoys Breakthrough Therapy designation in the United States. This designation facilitates and expedites development and review of new drugs to address unmet medical need in the treatment of a serious condition. The candidate has also been granted access to Priority Medicines (PRIME) regulatory initiative in Europe.

We remind investors that in December 2017, BioMarin presented updated data from another phase I/II study evaluating valoctocogene roxaparvovec at the American Society of Hemophilia. The findings suggested that a one-time infusion of valoctocogene roxaparvovec has potential to eliminate non-stop bleeding in patients suffering from severe hemophilia A with a very acceptable safety profile.

It is important to note that the current marketed therapies for hemophilia include Bayer AG's BAYRY Kovaltry, approved in both the United States and the EU for children and as well as adults.

Another company, Alnylam Pharmaceuticals ALNY is developing its hemophilia candidate, fitusiran in partnership with Sanofi SNY . Several mid-to-late stage studies are also underway on the candidate.

BioMarin Pharmaceutical Inc. Price

BioMarin Pharmaceutical Inc. Price | BioMarin Pharmaceutical Inc. Quote

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Biomarin currently carries a Zacks Rank #2 (Buy). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here .

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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