Quantcast

BioMarin Begins Mid Stage Study on Gene Therapy Candidate


Shutterstock photo

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

Zacks Rank

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

Today's Stocks from Zacks' Hottest Strategies

It's hard to believe, even for us at Zacks. But while the market gained +21.9% in 2017, our top stock-picking screens have returned +115.0%, +109.3%, +104.9%, +98.6%, and +67.1%.

And this outperformance has not just been a recent phenomenon. Over the years it has been remarkably consistent. From 2000 - 2017, the composite yearly average gain for these strategies has beaten the market more than 19X over. Maybe even more remarkable is the fact that we're willing to share their latest stocks with you without cost or obligation.

See Them Free>>


Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report

Sanofi (SNY): Free Stock Analysis Report

Bayer Aktiengesellschaft (BAYRY): Free Stock Analysis Report

BioMarin Pharmaceutical Inc. (BMRN): Free Stock Analysis Report

Alnylam Pharmaceuticals, Inc. (ALNY): Free Stock Analysis Report

To read this article on Zacks.com click here.

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



This article appears in: Investing , Business , Stocks
Referenced Symbols: SNY , BAYRY , BMRN , ALNY



More from Zacks.com

Subscribe






Zacks.com
Contributor:

Zacks.com

Equity Research










Research Brokers before you trade

Want to trade FX?