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Analysts Praise BioMarin's Experimental Drugs

Wed, 09/18/2013 - 3:09pm

 

NEW YORK (AP) — Analysts praised BioMarin Pharmaceutical's drug development pipeline on Wednesday, and the company's shares reached another all-time high as the markets rallied in late afternoon.

THE OPINION: BioMarin presented an update on its drug candidates late Tuesday. The San Rafael, Calif.-based company said a Food and Drug Administration panel will discuss its drug Vimizim on Nov. 19.

Investors often respond positively to news that a drug will get a review from an advisory panel because if the review is favorable, it's more likely the drug will be approved.

The FDA is schedule to make a ruling on Vimizim by Feb. 28. The drug is designed to treat a rare disease called mucopolysaccharidosis Type IVA, or Morquio A Syndrome. Morquio A Syndrome is a genetic disease in which the body does not produce enough of an enzyme called GALNS, and a compound called keratan sulfate builds up in organs and tissues. That causes skeletal and joint problems and shortness, and can also cause hearing loss, clouded vision and heart valve disease.

European Union regulators are also reviewing Vimizim.

Cowen and Co. analyst Phil Nadeau said that by the end of 2013 Vimizim will be almost ready to launch worldwide and BioMarin will have three other drugs in pivotal studies. He has an "Outperform" rating on BioMarin stock with a price target of $85 per share.

"We continue to think BioMarin has one of the best pipelines in mid-cap biotech," he said.

Two of BioMarin's approved drugs, Naglazyme and Aldurazyme, treat other types of mucopolysaccharidosis.

The company also said Tuesday that it reached an agreement with the FDA about the primary goal of a study of its drug BMN 701, a potential treatment for a genetic disorder called Pompe disease. The agreement means it is more likely the drug will be approved if the drug meets that goal.

The company also said the FDA is lifting a clinical hold on a study of another drug and allowing it to move ahead with a mid-stage clinical trial. BMN 111 is intended to treat achondroplasia, a condition that is responsible for most cases of dwarfism, which causes disproportionately short arms and legs. BioMarin expects to start a study in pediatric patients in the fourth quarter of 2013 or first quarter of 2014.

Jefferies & Co. analyst Eun Yang maintained a "Buy" rating on the shares, raising her price target to $86 per share from $72.

"(BioMarin's) focus on orphan diseases/biologics, along with one of the richest pipeline in biotech, makes BioMarin one of most attractive long-term investment opportunities, in our view," Yang said.

THE STOCK: Shares of BioMarin Pharmaceutical Inc. rose $1.66, or 2.2 percent to $77.62 in afternoon trading, after topping out at a new high of $77.78 as the markets rallied following word that the Federal Reserve will continue its economic stimulus policies. The stock has nearly doubled in valued over the last year, including a 3.5-percent gain on Tuesday.

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