Navigation Links
Results of First Interim Efficacy Analysis for Riquent Phase 3 ASPEN Trial: Continuation of the Trial is Futile
Date:2/12/2009

NOVATO, Calif., Feb. 12 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today 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.

    Contact:

    Investors                            Media
    Eugenia Shen                         Susan Berg
    BioMarin Pharmaceutical Inc.         BioMarin Pharmaceutical Inc.
    (415) 506-6570                       (415) 506-6594

'/>"/>
SOURCE BioMarin Pharmaceutical Inc.
Copyright©2009 PR Newswire.
All rights reserved

Related biology technology :

1. China Kangtai Cactus Biotech Files 2nd Quarter 2007 10QSB and Announces Unaudited Quarterly Results
2. Cephalon Announces Positive Results from a Pivotal Study of FENTORA in Opioid-tolerant Patients with Non-cancer Breakthrough Pain
3. Healthcare Technologies Reports Loss for 2007 Second Quarter & Six Months Results
4. CNS Response Announces Fiscal Third Quarter 2007 Results
5. Skystar Bio-Pharmaceutical Announces Second Quarter 2007 Results
6. China Biopharmaceuticals Holdings Announces Second Quarter 2007 Financial Results
7. XTL Biopharmaceuticals Announces Financial Results for the Six Months Ended June 30, 2007
8. Carrington Reports Second Quarter 2007 Results
9. Avitar Reports Third Quarter Financial Results for Fiscal 2007
10. Response Genetics Reports Second Quarter 2007 Financial Results
11. Martek to Announce Third Quarter 2007 Results on September 5, 2007
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/23/2016)... ... June 23, 2016 , ... ... and Mold) microbial test has received AOAC Research Institute approval 061601. , “This ... introduced last year,” stated Bob Salter, Vice President of Regulatory and Industrial Affairs. ...
(Date:6/23/2016)... , ... June 23, 2016 , ... ... announced the launch of the Supplyframe Design Lab . Located in Pasadena, ... explore the future of how hardware projects are designed, built and brought to ...
(Date:6/23/2016)... Calif. , June 23, 2016  Blueprint Bio, ... biological discoveries to the medical community, has closed its ... Matthew Nunez . "We have received ... with the capital we need to meet our current ... essentially provide us the runway to complete validation on ...
(Date:6/23/2016)... ... June 23, 2016 , ... Regulatory Compliance Associates® Inc. (RCA), ... free webinar on Performing Quality Investigations: Getting to Root Cause. ... no charge. , Incomplete investigations are still a major concern to the Regulatory ...
Breaking Biology Technology:
(Date:6/22/2016)... 2016   Acuant , the leading ... has partnered with RightCrowd ® to ... Management, Self-Service Kiosks and Continuous Workforce Assurance. ... functional enhancements to existing physical access control ... with an automated ID verification and authentication ...
(Date:6/20/2016)... -- Securus Technologies, a leading provider of civil ... investigation, corrections and monitoring announced that after exhaustive ... the final acceptance by all three (3) Department ... (MAS) installed. Furthermore, Securus will have contracts for ... October, 2016. MAS distinguishes between legitimate wireless device ...
(Date:6/9/2016)... innovation leader in attendance control systems is proud to announce the introduction of fingerprint ... sure the right employees are actually signing in, and to even control the opening ... ... ... Photo ...
Breaking Biology News(10 mins):