Navigation Links
BioMarin Initiates Phase 1 Trial for BMN 673 in Patients With Advanced Hematological Malignancies
Date:7/14/2011

NOVATO, Calif., July 14, 2011 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the initiation of a Phase 1 trial for BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, for the treatment of patients with advanced hematological malignancies.  A Phase 1/2 trial for BMN 673 for the treatment of patients with solid tumors was initiated in January 2011 and is ongoing.

"We are excited to be at the forefront of studying the potential benefit of PARP inhibitors in hematological malignancies," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin.  "Our approach to the development of our PARP inhibitor is to identify tumor mutations that are more susceptible to treatment with BMN 673 and to target subgroups based on the genetic basis of the disease.  We are committed to the advancement of our pipeline and look forward to many clinical milestones over the next 18 months."

This Phase 1 trial is a two-arm, open-label dose escalation study to determine the maximum tolerated dose and to assess the safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of once daily, oral BMN 673 in patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), chronic lymphocytic leukemia (CLL) or mantle cell lymphoma (MCL).  The study will enroll approximately 80 patients across multiple sites in the U.S. and UK.

About BioMarinBioMarin 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, and 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. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking StatementThis press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the expectations of the development of BMN 673, including the timing of the clinical trials of the candidate, and the possible efficacy of such candidate. 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 content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities, results and timing of current and planned clinical and preclinical studies related to such product; our ability to successfully manufacture the product; 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 2010 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®, Naglazyme® and Kuvan® are registered trademarks of BioMarin Pharmaceutical Inc.

Firdapse™ is a trademark of BioMarin Huxley Ltd.

Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.Contact:Investors

MediaEugenia Shen

Bob PurcellBioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.(415) 506-6570

(415) 506-3267
'/>"/>

SOURCE BioMarin Pharmaceutical Inc.
Copyright©2010 PR Newswire.
All rights reserved

Related biology technology :

1. BioMarin Announces Roll-Out of National PKU Registry
2. BioMarin to Present at the Citi Biotech Day
3. BioMarin to Present at the Morgan Stanley Global Healthcare Conference
4. BioMarin to Present at the Merrill Lynch Global Pharmaceutical Conference
5. BioMarin Corrects Information Included in Bloomberg Article
6. BioMarin Announces Third Quarter 2008 Financial Results
7. BioMarin to Present at the Credit Suisse Healthcare Conference
8. BioMarin Announces the Planned Retirement of Dr. Emil Kakkis, Chief Medical Officer
9. BioMarin to Present at the Piper Jaffray Healthcare Conference
10. BioMarin to Host Fourth Quarter and Full Year 2008 Financial Results Conference Call and Webcast on Wednesday, February 18 at 5:00 p.m. ET (23:00 CET)
11. BioMarin Announces Fourth Quarter and Full Year 2008 Financial Results
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:11/30/2016)... 2016 /PRNewswire/ - Portage Biotech Inc. ("Portage" or "the ... excited to announce the formation of EyGen, Ltd. ... ophthalmology assets through proof of concept. EyGen,s lead ... Portage Pharmaceuticals Limited and being developed for topical ... anterior segment diseases. This agent has the potential ...
(Date:11/30/2016)...  The Allen Institute for Cell Science has ... available collection of gene edited, fluorescently tagged human ... structures with unprecedented clarity. Distributed through the Coriell ... a crucial first step toward visualizing the dynamic ... human cells healthy and what goes wrong in ...
(Date:11/30/2016)... , Nov. 30, 2016  Tempus, a technology ... care, and Penn,s Abramson Cancer Center have partnered ... positive response to immunotherapy treatment based on next ... As part of a research collaboration, Tempus will ... melanoma cancer patient data to Penn. Utilizing next-generation ...
(Date:11/30/2016)... , 30. November 2016   ... hat heute die Unterzeichnung einer Reihe von ... zufolge wird Evotec AG Screeningleistungen für Mercks ... bereitstellen. Der Zugriff auf diese Bibliotheken in ... eröffnet einen schnelleren Weg zur Ermittlung und ...
Breaking Biology Technology:
(Date:6/21/2016)... VANCOUVER, British Columbia , June 21, 2016 ... been appointed to the new role of principal ... has been named the director of customer development. ... , NuData,s chief technical officer. The moves reflect ... development teams in response to high customer demand ...
(Date:6/15/2016)... ALBANY, New York , June 15, 2016 ... published a new market report titled "Gesture Recognition Market ... Trends and Forecast, 2016 - 2024". According to the ... at USD 11.60 billion in 2015 and is ... and reach USD 48.56 billion by 2024.  ...
(Date:6/7/2016)... -- Syngrafii Inc. and San Antonio Credit Union (SACU) ... Syngrafii,s patented LongPen™ eSignature "Wet" solution into SACU,s ... in greater convenience for SACU members and operational ... document workflow and compliance requirements. Logo ... Highlights: ...
Breaking Biology News(10 mins):