BioMarin Halts Phase 3a Study of Neutralase in CABG and Terminates the Neutralase Drug Development Program
Monday September 22, 12:30 am ET
BioMarin Spending Projected to Decrease in Both 2003 and 2004
Conference Call at 9:00 AM ET (15:00 CET)
NOVATO, Calif., Sept. 22 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN; SWX) announced that it halted its Phase 3a study of Neutralase(TM) for the reversal of anticoagulation by heparin in primary Coronary Artery Bypass Graft (CABG) surgery and that it has terminated its Neutralase program for all indications.
The decision to halt the Phase 3 study resulted from a recommendation from an independent Data Safety Monitoring Board (DSMB) and was based on a review of data from enrolled patients, which indicated with high probability that Neutralase would not demonstrate favorable safety and efficacy. Given the expected risk/benefit profile for Neutralase, BioMarin has decided to stop development of the drug for all indications.
As a result of the planned reductions in spending related to the discontinuation of Neutralase development, BioMarin has lowered its projected net loss by $5 million for 2003 and by $13 million for 2004. Revised estimates for both net loss and cash burn for 2003 and 2004 are included below, as are previous forecasts for purposes of comparability:
Changes to 2003 and 2004 Forecast ($ in millions)
2003 2004
Forecast Current Previous Current Previous
Net Loss $76-$78 $81-$83 $68-$70 $81-$83
Cash Burn $74-$78 $80-$84 $69-$73 $80-$84
As of June 30, 2003, BioMarin had approximately $265 million in cash and cash equivalents.
Fredric D. Price, Chairman and Chief Executive Officer of BioMarin stated, "While we are disappointed that Neutralase will not become a successful product, it is better to have learned this relatively early in its development at BioMarin. We have planned BioMarin's product portfolio carefully, and have adequate resources for our other promising product opportunities, including Aryplase(TM), which is in a pivotal Phase 3 trial."
BioMarin's portfolio includes product candidates for genetic diseases, including Aryplase for mucopolysaccharidosis VI that is in a Phase 3 clinical trial expected to be completed at the end of the first quarter of 2004, and a product candidate for Phenylketonuria (PKU) that the company expects to advance into clinical-stage development in 2004. Vibrilase(TM), an enzyme product candidate for treatment of serious burns, is nearing completion of a Phase 1b study, and NeuroTrans(TM) is in preclinical testing as a method to deliver both enzymes and other therapeutic molecules to the brain via traditional intravenous infusion. Aldurazyme® for mucopolysaccharidosis I is approved in both the United States and European Union. Aldurazyme is manufactured by BioMarin and is marketed by BioMarin's joint venture partner, Genzyme Corporation.
BioMarin will host a conference call and webcast to discuss the decision to terminate Neutralase development today at 9:00 AM ET (15:00 CET). This event can be accessed on the BioMarin website at: investor.biomarinpharm.com.
Date: September 22, 2003
Time: 9:00 AM ET (15:00 CET)
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