"At the conclusion of the Phase 2 study, we have achieved our goal of identifying a dosing regimen that allows us to advance to the pivotal Phase 3 trial," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "Based on the Phase 2 experience, we expect that while most patients will experience hypersensitity-type side effects during introduction to the drug, the side effects will be transient and will allow most patients to achieve safe, long-term Phe control. We look forward to meeting with the FDA in the first quarter of 2013 and reviewing the Phase 2 data and our preliminary Phase 3 study design."
Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin added, "We believe PEG-PAL has a strong value proposition in treating adult severe PKU patients, especially in light of the expected ease of self, at-home administration. The initial target market for PEG-PAL is adult PKU patients in our database who are Kuvan non-responders, about 1,500 patients in the U.S. Eventually, we should be able to treat other adult patients and current pediatric patients on Kuvan as they get older."
The preliminary Phase 3 study design includes (1) an open-label study to evaluate safety and blood Phe levels in naive patients and (2) a randomized controlled study in the Phase 2 extension study patients to evaluate blood Phe levels and psychiatric and executive function endpoints. All patients in the Phase 3 trial will be offered the opportunity to remain on drug as part of an extension trial. Based on discussions with the FDA, reduction in Phe levels is expected to be the primary endpoint in the Phase 3 trial. The company expects to initiate the Phase 3 program in the second quarter of 2013 following an end of phase 2 meeting with the FDA in the first quarter of 2013.
Conference Call Details
BioMarin will host a conference call and webcast today, Wednesday, September 26, 2012 at 5:00 p.m. ET. This event can be accessed on the investor section of the BioMarin website at
| Date: September 26, 2012
| Time: 5:00 p.m. ET
| U.S. / Canada Dial-in Number: 877.303.6313
| International Dial-in Number: 631.813.4734
| Conference ID: 30936364
| Replay Dial-in Number: 855.859.2056
| Replay International Dial-in Number: 404.537.3406
| Conference ID: 30936364
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse™ (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include GALNS (N-acetylgalactosamine 6-sulfatase), which is currently in Phase III clinical development for the treatment of MPS IVA, amifampridine phosphate (3,4-diaminopyridine phosphate), which is currently in Phase III clinical development for the treatment of LEMS in the U.S., PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase II clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, and BMN-111, a modified C-nutriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia. For additional information, please visit
. Information on BioMarin's website is not incorporated by reference into this press release.
The BioMarin Pharmaceutical Inc. logo is available at
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the development of BioMarin's PEG-PAL program generally, the timing and design of the planed Phase 3 trial of PEG-PAL, and expectations regarding the final results of the Phase 2 trial following final statistical analysis. 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: differences in the final analysis of the data from the PEG-PAL Phase 2 trial, results and timing of current and planned preclinical studies and clinical trials of PEG-PAL; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; BioMarin's ability to secure clinical trial sites to perform the Phase 3 trial and the ability to enroll patients into those trials; 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 Quarterly Report on Form 10-Q for the Quarter ended June 30, 2012. 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.
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