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BMRN

BioMarin

$84.32

-1.08 (-1.26%)

17:08
10/19/16
10/19
17:08
10/19/16
17:08

BioMarin reports Phase 2 vosoritide data in dwarfism

BioMarin provided an update on its Phase 2 study of vosoritide in children with achondroplasia, the most common form of dwarfism, at the American Society of Human Genetics 2016 Meeting. Results from 8 children in cohort 4, who completed six months of daily dosing at 30 microgram/kg/daily experienced a 46% or 2.1 cm/year increase in mean annualized growth velocity from baseline. These data are comparable to those observed at the lower dose of 15 microg/kg/day in cohort 3, the company said. Results from 10 children in cohort 3, who completed six months of daily dosing at 15 microgram/kg/day experienced a 50% or 2.0 cm/year increase in mean annualized growth velocity from baseline. Vosoritide was generally well tolerated at all doses. All injection site reaction events were mild and transient. No new safety findings were observed at the 30 microg/kg/day dose. "The data support our use of the lower dose of 15 microgram/kg/day in the Phase 3 study," the company said, adding that "the company believes based on discussions with global health authorities that change in growth velocity from baseline as an endpoint could lead to registration. The company plans to augment these data with supportive evidence concerning proportionality and functionality."

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BMRN BioMarin
$84.32

-1.08 (-1.26%)

09/21/16
LEER
09/21/16
NO CHANGE
LEER
Market Perform
Previous PTAB ruling remains overhang on Sarepta eteplirsen, says Leerink
Leerink analyst Joseph Schwartz notes that Sarepta (SRPT) has announced favorable Patent Trial and Appeal Board of the U.S. Patent and Trademark Office decisions in favor of its exon 51 and 53 composition of matter patent interference cases against BioMarin (BMRN). However, he points out that these rulings are subject to appeal and a previous ruling from the U.S. Patent Trial and Appeal Board, or PTAB, in favor of BioMarin's method of use patent remains an overhang on the commercialization of Sarepta's eteplirsen. The analyst reiterates a Market Perform rating on Sarepta's shares.
10/07/16
GSCO
10/07/16
NO CHANGE
GSCO
Smid-cap Biotech sector upgraded to Attractive from Neutral at Goldman
Goldman analyst Salveen Richter upgraded the Smid-cap biotechnology to Attractive and said he favors and companies with near-term pipeline data or regulatory events where he has conviction.Names he recommends are Buy rated BioMarin (BMRN), bluebird bio (BLUE), GW Pharma (GWPH), Incyte (INCY), Kite (KITE), and Sage (SAGE).
10/14/16
BARD
10/14/16
NO CHANGE
Target $115
BARD
Outperform
BioMarin hemophilia drug update encouraging, says Baird
Baird analyst Michale Ulz said he is encouraged by BioMarin's update on its Phase 1/2 study of its Hemophilia A drug, BMN-270. The analyst noted U.K. regulators approved continued enrollment in the gene-therapy study and the study was amended to allow dose optimization ahead of the Phase 2b study expected in 2017. Ulz reiterated his Outperform rating and $115 price target on BioMarin shares.
10/17/16
JANY
10/17/16
NO CHANGE
JANY
PTC's denial has negative read-through for BioMarin, says Janney Capital
Janney Capital noted that PTC Therapeutics' (PTCT) Translarna and Sarepta's (SRPT) Exondys 51 address non-overlapping DMD populations and there was an expectation of flexibility from the FDA following the conditional approval of Exondys 51. However, the FDA has denied the first appeal of the refuse to file letter regarding PTC's NDA for Translarna for the treatment of nonsense mutation DMD, which the firm said has potential negative read-through for BioMarin's (BMRN) attempt to rekindle Kyndrisa, which is a direct competitor to Sarepta's Exondys 51. The firm added that the news might positively impact reimbursement decisions for Exondys 51.

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