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BMRN

BioMarin

$81.99

1.09 (1.35%)

10:03
12/11/17
12/11
10:03
12/11/17
10:03

BioMarin highlights new results for valoctocogene roxaparvovec at ASH

BioMarin Pharmaceutical announced updates on valoctocogene roxaparvovec -- formerly BMN 270, an investigational gene therapy treatment for severe hemophilia at ASH. With the 4e13 vg/kg dose, the three patients with the longest follow-up have Factor VIII activity levels that are in or near to the normal range with both median and mean values of 49%. Median annualized bleed and factor VIII use rates for the 4e13 vg/kg cohort were zero after Week 4 and when their Factor VIII activity rose above 5%. Mean annualized bleed and factor VIII use rates for the 4e13vg/kg cohort were 0.6 and 2.0, respectively. With the 6e13 vg/kg dose, at 78 weeks post infusion, the median and mean Factor VIII levels of the 6e13 vg/kg cohort were 90 and 89%, respectively. Median annualized bleed and factor VIII use rates for the 6e13 vg/kg were zero after Week 4. Mean annualized bleed and factor VIII use rates for the 6e13 vg/kg cohort were 0.5 and 6.1, respectively. The company also announced that the New England Journal of Medicine published an independent, peer-reviewed article on the ongoing Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy, in men with severe hemophilia A. The article assessed the safety and efficacy of valoctocogene roxaparvovec at the 6e13 dose, after 52 weeks. As presented at ASH on December 10, the objective of this study was to determine the comparative pharmacodynamics of valoctocogene roxaparvovec when given as a single intravenous bolus injection to cynomolgus monkeys with varying baseline anti-AAV5 total antibody levels and transduction inhibition titers. The results demonstrated no evidence for decreased FVIII expression in animals with non-antibody based transduction inhibition, while baseline AAV5 antibody positive animals had a range of FVIII expression.

  • 08

    Jan

  • 28

    Feb

BMRN BioMarin
$81.99

1.09 (1.35%)

10/19/17
FBCO
10/19/17
NO CHANGE
Target $124
FBCO
Outperform
BioMarin price target raised to $124 from $114 at Credit Suisse
Credit Suisse analyst Alethia Young raised her price target for BioMarin to $124 from $114 as she believes there is a higher chance of success for hemophilia A gene therapy. The analyst reiterates an Outperform rating on the shares.
12/10/17
PIPR
12/10/17
NO CHANGE
PIPR
Piper Jaffray remains bullish on BioMarin, Alexion following ASH
Piper Jaffray analyst Christopher Raymond remains Overweight rated on BioMarin (BMRN) and Alexion (ALXN), and Neutral rated on Celgene (CELG) following the American Society of Hematology Annual Meeting. Most notably, the analyst finds BioMarin's hemophilia gene therapy data with longer term data to be most impactful.
12/11/17
RHCO
12/11/17
NO CHANGE
Target $130
RHCO
Buy
BioMarin price target raised to $130 from $115 at SunTrust
SunTrust analyst Edward Nash raised his price target on BioMarin to $130 after the company's update from the Phase I/II study of BMN270 in patients with severe hemophilia at the annual ASH meeting. Nash says the program "continues to demonstrate a sustained clinical benefit with no safety issues", and its "cytotoxic T-cell response and neutralizing antibodies to Factor VIII further underscore the efficacy and safety of BMN 270". The analyst keeps his Buy rating on BioMarin.
12/11/17
PIPR
12/11/17
NO CHANGE
Target $113
PIPR
Overweight
BioMarin's BMN 270 looks better after Spark data, says Piper Jaffray
Piper Jaffray analyst Christopher Raymond believes the competitive outlook for BioMarin's (BMRN) BMN 270 looks a lot better after Spark Therapeutics (ONCE) reported "underwhelming" data in hemophilia A. Spark's "disappointing" update is likely to provide even more lift to the BMN 270 story, Raymond tells investors in a research note. The analyst notes BMN 270 contributes $12 per share to his sum-of-the-parts analysis for BioMarin. Raymond keeps an Overweight rating on the shares with a $113 price target.

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