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BioMarin to Highlight Breadth of Innovative Development Pipeline at R&D Day on April 20th in New York
Key development updates will include preliminary data with BMN 270, gene therapy for hemophilia A, and one year data with vosoritide for achondroplasia
Apr 18, 2016

SAN RAFAEL, Calif.April 18, 2016 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) will host an R&D Day from 8am (ET) to 12pm (ET) on Wednesday, April 20, 2016 in New York CityBioMarin management and external experts will provide an update to the investment community on the Company's development portfolio, which is focused on innovative therapies to treat rare and ultra-rare diseases.

Specifically, the BioMarin leadership team will highlight:

  • Preliminary data from BMN 270 (gene therapy) trial in Hemophilia A
  • A review of cerliponase alfa for CLN2, a form of Batten Disease
  • A review of pegvaliase for phenylketonuria (PKU)
  • One year data with vosoritide for Achondroplasia

BioMarin speakers scheduled to present include:

Jean-Jacques Bienaimé, Chairman and Chief Executive Officer
Hank Fuchs, M.D., Executive Vice President and Chief Medical Officer
Wolfgang Dummer, M.D., Ph.D., Vice President, Clinical Development
Sarah Noonberg, M.D., Ph.D., Group Vice President, Head of Global Clinical Development
Len Post, Ph.D., Group Vice President, Chief Scientific Officer
Camilla SimpsonM.Sc., Group Vice President, Regulatory Affairs

External key opinion leaders scheduled to present include:

Cary Harding, M.D., Professor of Molecular and Medical Genetics and Pediatrics at Oregon Health and Science University

  • Lead investigator, pegvaliase pivotal study

John Pasi, Ph.D., Professor of Haemostasis and Thrombosis, Barts and the London School of Medicine and DentistryQueen Mary, University of London

  • Lead investigator, BMN 270 phase 1/2 study

Ravi Savarirayan, M.D., Professor, Department of PaediatricsUniversity of Melbourne

  • Lead investigator, vosoritide phase 2 study

Live Video Webcast
Interested parties may access a live video webcast that will include audio and slides of the presentations via the investor section of the BioMarin website, www.BMRN.com. A replay of the meeting will be archived on the site for at least one week.

For those who choose not to listen and view the event via webcast, dial-in information for the audio portion of the webcast can be accessed using:

U.S. / Canada Dial-in Number: (877) 303-6313
International Dial-in Number: (631) 813-4734 
Conference ID: 93170415

About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of five commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.



Traci McCarty

BioMarin Pharmaceutical Inc.

(415) 455-7558


Debra Charlesworth

BioMarin Pharmaceutical Inc.

(415) 455-7451

Source:  BioMarin Pharmaceutical Inc.