NEW YORK - BioMarin Pharmaceuticals Inc. plans to offer information about its products at an investor meeting Thursday, and a Jefferies analyst said she expects new details about two treatments for phenylketonuria.
| BMRN | 23.66 |
Beginning at 7:30 a.m. EDT, BioMarin will review its products, research and technology and drug development programs. Its products include Kuvan, which treats a genetic disorder that can cause neurological problems, and the enzyme replacement therapies Nalgazyme and Aldurazyme.
Jefferies analyst Salveen Kochnover said she expects more detail about an early-stage clinical trial of the company's drug PEG-PAL, a treatment for phenylketonuria, or PKU. The primary goal is testing the safety and tolerability of the drug, she said, but a second goal involves measuring the effects of different doses on levels of phenylalanine in the blood.
PKU is a rare inherited condition that causes phenylalanine, an amino acid, to build up in the body. If it is not treated shortly after birth, PKU can cause problems including mental retardation, delayed speech and small brain size.
Kochnover also said she expects more information on a trial of Kuvan, and development of drug candidates that treat sickle cell anemia, kidney disorders and cystic fibrosis.
She kept a "Buy" rating on shares of the Novato, Calif., company, with a price target of $45 per share. That implies the stock will rise 20.3 percent over the next year from Wednesday's closing price of $37.40.

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