Results Of First Interim Efficacy Analysis For Riquent Phase 3 ASPEN Trial: Continuation Of The Trial Is Futile

Main Category: Lupus
Also Included In: Urology / Nephrology;  Clinical Trials / Drug Trials;  Pharma Industry / Biotech Industry
Article Date: 13 Feb 2009 - 3:00 PDT

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BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that in the first interim efficacy analysis (IEA) for the Riquent(R) Phase 3 ASPEN trial, the Independent Data Monitoring Board (DMB) determined that the continuation of the trial is futile. BioMarin and partner La Jolla Pharmaceutical have decided to stop the study, unblind the data and evaluate all of the clinical results including secondary endpoints such as SLE (Systemic Lupus Erythematosus) disease activity indices and proteinuria.

"While the results of this first interim efficacy analysis are obviously disappointing, we decided to take a calculated risk on this program and with the carefully staged agreement, we have spent a total of $15 million, a relatively modest amount for a late Phase 3 asset addressing a large market opportunity," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "We remain dedicated to advancing our development pipeline by making investments in future growth and continue to look for attractive late-stage in-licensing or acquisition opportunities."

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises three approved products and multiple clinical and preclinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme(R) (laronidase) for mucopolysaccharidosis I (MPS I), a product which was developed through a 50/50 joint venture with Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany. Other product candidates include 6R-BH4 for cardiovascular indications, which is currently in Phase 2 clinical development for the treatment of sickle cell disease, and PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase 1 clinical development for the treatment of PKU. For additional information, please visit http://www.BMRN.com. 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: expectations related to the clinical trials and continued development of abetimus sodium, also known as Riquent. 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: results and timing of current and planned preclinical studies and clinical trials, including the ASPEN study; the enrollment rate of such study and the actual rate of renal flares in such study; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission regarding abetimus sodium; La Jolla Pharmaceutical's ability to and success in executing the ASPEN study and its ability to produce sufficient quantities of material for such study; 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 2007 Annual Report on Form 10-K, and the factors contained in BioMarin's reports on Form 10-Q. 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.

BioMarin(R), Naglazyme(R) and Kuvan(R) are registered trademarks of BioMarin Pharmaceutical Inc

Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC

Riquent(R) is a registered trademark of La Jolla Pharmaceutical

BioMarin Pharmaceutical Inc.
http://www.bmrn.com