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BioMarin Initiates Phase 2 Clinical Study Of PEG-PAL In PKU

September 23, 2009

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that the first patient has initiated treatment in the Phase 2 clinical thought of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). Initial top-line results are expected in mid-2010.

“We remain optimistic on this program and believe that, if approved, PEG-PAL may offer a significant benefit for many PKU patients, especially those who do not respond adequately to Kuvan,” said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. “In our view, the flexible delineate of the Phase 2 protocol provides multiple opportunities to arrive at an optimal dose and dosing oftenness that is ordinary in at least in a sub-segment of the PKU population. We are encouraged by the results of the Phase 1 investigate, that showed a sound blood Phe bring to the same level reduction thwart all patients in the fifth battalions at a dose of 0.1mg/kg, no serious adverse events, and reactions as expected with a protein of this nature.

The Phase 2 clinical trial is an open-label, multi-center study to be conducted in up to 35 patients in a succession of dose-escalating cohorts from 0.001 mg/kg. The primary treatment period of eight one time weekly injections at a fixed dose devise be followed by eight weeks of dose and commonness optimization and an extension period where doses can have being increased up to 2.0 mg/kg/week.

The preparatory objective is to evaluate the effect of PEG-PAL on blood Phe concentrations in subjects with PKU. The secondary objectives are to evaluate the safety and tolerability, immune answer and steady state pharmacokinetics of subcutaneous injections of multiple disagreeable lot levels of PEG-PAL.

About PEG-PAL

PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) is an investigational enzyme substitution therapy with a view to the treatment of PKU. Pharmacology studies conducted in the PKU mouse design demonstrated that weekly subcutaneous administrations of PEG-PAL resulted in a significant and stable decrease of plasma phenylalanine. BioMarin estimates that PEG-PAL could be a possible treatment preference for a significant portion of the PKU number of people.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for momentous diseases and medical conditions. The company’s produce portfolio comprises three approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme(R) (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by the agency of BioMarin; Aldurazyme(R) (laronidase) on this account that mucopolysaccharidosis I (MPS I), a product that BioMarin developed through a 50/50 divide take the liberty through Genzyme Corporation; and Kuvan(R) (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in association with Merck Serono, a es trangement of Merck KGaA of Darmstadt, Germany. Other performance candidates embody PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), what one. is commonly in Phase II clinical development for the management of PKU and GALNS (N-acetylgalactosamine 6-sulfatase), that is currently in Phase I/II clinical development for the treatment of MPS IVA.

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 PEG-PAL, and expectations related to clinical trials of PEG-PAL. 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 current and planned clinical trials related to PEG-PAL; the content and timing of decisions by the U.S. Food and Drug Administration and other regulatory agencies, particularly with respect to PEG-PAL, and those factors detailed in BioMarin’s filings with the Securities and Exchange Commission, including, without restriction, the factors contained under the caption “Risk Factors” in BioMarin’s 2008 Annual Report on Form 10-K. Stockholders are urged not to dispose undue ground of trust on forward-looking statements, which speak only for example of the date hereof. BioMarin is in a state of being liable to not any obligation, and expressly disclaims any one obligation to update or alter any forward-looking statement, whether as a result of new information, future events or in other respects.

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

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

Source: BioMarin Pharmaceutical Inc

View put drugs into notice on Kuvan.

Written by Samanta · Filed Under Pharmacy Industry News