Healthcare Industry News:  PKU  


 News Release - August 13, 2007

Positive Kuvan(TM) Pivotal Phase 3 Trial Results Published in The Lancet

NOVATO, Calif., Aug. 13 (HSMN NewsFeed) -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN News) announced today that final results from the Kuvan(TM) (sapropterin dihydrochloride) pivotal Phase 3 clinical trial were published in the August 11, 2007, issue of The Lancet. The study suggests that treatment with Kuvan results in significant reductions in blood Phe levels in some phenylketonuria (PKU) patients. Kuvan, an investigational oral small molecule for the treatment of PKU, is being developed in partnership with Merck Serono, a division of Merck KGaA, Darmstadt, Germany.

"This marks the first published Kuvan clinical study, and we expect additional publications in the coming months to build on the collection of data related to Kuvan," said Emil Kakkis, M.D., Ph.D., Chief Medical Officer of BioMarin. "We recently received priority review from the FDA and hope to receive FDA approval by late November. In the interim, we are pleased to be able to provide Kuvan to PKU patients prior to commercial availability through our expanded access program."

The Phase 3 clinical study enrolled 89 patients with elevated blood Phe levels aged eight years and above at 29 sites in the United States, Europe and Canada. All patients enrolled had demonstrated a reduction in blood Phe levels following treatment with Kuvan in a Phase 2 screening study.

The patients were randomly assigned to receive placebo or 10 mg/kg of Kuvan daily for six weeks. Patients were evaluated every two weeks for changes in blood Phe levels and adverse events. The primary objective was to assess the efficacy of Kuvan compared with placebo for reduction of blood phenylalanine in patients with PKU. The secondary objective was to assess the safety of Kuvan compared with placebo. A total of 87 patients completed six weeks of treatment.

Highlights from the Phase 3 double-blind study are summarized below:

* Patients treated with Kuvan for six weeks had a mean decrease in
blood Phe level of 236 uM/L (29 percent) compared to a mean increase
of 3 uM/L (3 percent) in the placebo group (p<0.0001). Prior to
treatment, patients in the Kuvan group and placebo group had mean
blood Phe levels of 843 uM/L and 888 uM/L, respectively.

* After six weeks of treatment, 54 percent of patients treated with
Kuvan and 23 percent of patients in the placebo group had a blood
phenylalanine concentration below the recommended 600 uM/L level

* Blood phenylalanine concentrations fell by about 200 uM/L after one
week in the Kuvan group, and this reduction persisted for the
remaining five weeks of the study (p<0.0001).

* The type and incidence of adverse events was similar in the Kuvan
and placebo groups. Kuvan was well tolerated and investigators
reported that no serious adverse event occurred.

About Kuvan

Kuvan is an investigational oral small molecule therapeutic for the treatment of PKU. The active ingredient in Kuvan, sapropterin dihydrochloride, is the synthetic form of 6R-BH4 (tetrahydrobiopterin), a naturally occurring enzyme cofactor that works in conjunction with phenylalanine hydroxylase (PAH) to metabolize Phe. Clinical data suggest that treatment with Kuvan results in significant reductions in blood Phe levels in BH4-responsive patients. It also may enable some patients to minimize or eliminate highly-restrictive dietary constraints by increasing Phe tolerance levels. BioMarin and Merck Serono estimate that Kuvan could be a potential treatment option for approximately 30 percent to 50 percent of the estimated 50,000 identified PKU patients in the developed world.

Kuvan has received orphan drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMEA). If approved, it will receive seven years of market exclusivity in the United States and 10 years in the European Union for this indication. Additionally, the FDA has granted Kuvan Fast Track designation, which is designed to facilitate the development of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

About PKU

PKU, a genetic disorder affecting approximately 50,000 diagnosed patients in the developed world, is caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). PAH is required for the metabolism of phenylalanine (Phe), an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe mental retardation and brain damage, mental illness, seizures, tremors, and limited cognitive ability. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all PKU patients under the age of 40 in developed countries have been diagnosed at birth. Currently, PKU can only be managed by a Phe- restricted diet, which is supplemented by nutritional replacement products, like formulas and specially-manufactured foods; however, the strict diet is difficult for most patients to adhere to the extent needed for achieving adequate control of blood Phe levels. To learn more about PKU, please visit Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises two approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin, and Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation. Investigational product candidates include Kuvan(TM) (sapropterin dihydrochloride), a Phase 3 product candidate for the treatment of phenylketonuria (PKU), and 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of peripheral arterial disease and sickle cell disease. Both products are being developed in partnership with Merck Serono, a division of Merck KgA of Darmstadt, Germany. For additional information, please visit Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the development of its product candidate Kuvan; expectations regarding the Kuvan expanded access program; expectations regarding filings with regulatory agencies; and the development of 6R-BH4 for other indications. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: the results of ongoing clinical trials related to Kuvan; the content and timing of decisions by the U.S. Food and Drug Administration, the European Medicines Agency and other regulatory authorities concerning Kuvan; results and timing of current and planned clinical trials of 6R-BH4 for other indications; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2006 Annual Report on Form 10-K, as amended, and the factors contained in BioMarin's reports on Form 8-K. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

Source: BioMarin Pharmaceutical

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