BioMarin's (BMRN) Gene Therapy Enters First Phase III Study

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced initiation of the first phase III study, evaluating the 6e13 vg/kg dose of its investigational gene therapy, valoctocogene roxaparvovec, for treatment of patients with severe hemophilia A. The company plans to enroll the first patient in the second phase III study with the 4e13 vg/kg dose at the beginning of 2018 for the given indication.

The two phase III studies, GENEr8-1 and GENEr8-2, were designed to evaluate the efficacy and safety of valoctocogene roxaparvovec in patients with severe hemophilia A across the two doses mentioned above. While the primary endpoint in both the studies will be factor VIII (a blood clotting protein) activity level achieved following administration of valoctocogene roxaparvovec, the secondary endpoints will measure annualized factor VIII replacement therapy use rate and annualized bleed rate.

BioMarin’s share price has gained 9.7% in the last 12 months, comparing favorably with the industry’s increase of 2.6%.

We remind investors that earlier this month, BioMarin presented an updated data from a phase I/II study on 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 for patients suffering severe hemophilia A with a very acceptable safety profile.

The company is also planning to start another phase I/II trial to evaluate valoctocogene roxaparvovec with the 6e13kg/vg dose on approximately 10 patients, who are adeno-associated virus type 5 (AAV5) positive, in the first half of 2018.

Notably, last March, BioMarin had announced that the FDA granted an Orphan Drug designation to valoctocogene roxaparvovec for treatment of patients with hemophilia A.

Additionally, in February, valoctocogene roxaparvovec has been granted an access to Priority Medicines, a regulatory initiative by the European Medicines Agency. This proposal offers an early and enhanced support to optimize regulatory applications. This would help patients get fast benefits from the therapies that promise to improve their quality of life.

It is important to note that the current marketed therapies for hemophilia include Bayer AG’s (OTC:BAYRY) Kovaltry, approved in both the United States and the EU in early 2016 both for children and the adults.

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

BioMarin Pharmaceutical Inc. Price

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Zacks Rank

Biomarin 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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