BioMarin Announces Agreement With Repligen for Pre-clinical Compounds

BioMarin Announces Agreement With Repligen for Pre-clinical Compounds

Includes Compounds for Potential Treatment of Friedreich's Ataxia

SAN RAFAEL, Calif., Jan. 21, 2014 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical
Inc. (Nasdaq:BMRN) announced today that it has entered into an agreement to
purchase Repligen Corporation's histone deacetylase inhibitor (HDACi) library
and related intellectual property. Potential applications of the HDACi
portfolio include Friedreich's ataxia and other neurological disorders.Under
the terms of the agreement, BioMarin will provide Repligen with an upfront
payment of $2 million and potential future milestone and royalty payments for
the development, regulatory approval and commercial sale of compounds included
in this agreement. 

"This agreement represents a natural extension of BioMarin's business strategy
of acquiring promising pre-clinical programs that we can successfully develop
and commercialize for people with serious, unmet medical needs," said
Jean-Jacques Bienaime, Chief Executive Officer of BioMarin."Fredreich's
ataxia is a neurological disorder that affects tens of thousands of young
people in the developed world and we look forward to the opportunity to move a
compound into the clinic in this indication."

About Friedreich's ataxia

Friedreich's ataxia (FA) is a progressive, neurological disorder that affects
approximately 20,000 people in the United States and Europe, typically
resulting in wheelchair dependence in young adulthood and early death due to
cardiac failure. It is caused by mutations in the FXN gene, and is inherited
in an autosomal recessive manner. FXN mutations result in reduced expression
of frataxin protein, manifesting in progressive neurological and cardiac
damage. Major neurological symptoms include muscle weakness and ataxia, a loss
of balance and coordination. These symptoms typically appear between 10 and 15
years of age, but FA has been diagnosed in people from ages 2 to 50 with
earlier onset associated with a more severe course.

About BioMarin

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 MPS VI, a
product wholly developed and commercialized by BioMarin; Aldurazyme®
(laronidase) for 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 VIMIZIM™ (N-acetylgalactosamine 6-sulfatase), formally referred to as
GALNS, which successfully completed Phase 3 clinical development for the
treatment of MPS IVA, PEG PAL (PEGylated recombinant phenylalanine ammonia
lyase), which is currently in Phase 3 clinical development for the treatment
of PKU, BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is
currently in Phase 3 clinical development for the treatment of germline BRCA
breast cancer, BMN 701, a novel fusion of acid alpha glucosidase (GAA) with a
peptide derived from insulin like growth factor 2, which is currently in Phase
1/2 clinical development for the treatment of Pompe disease, BMN 111, a
modified C-natriuretic peptide, which is currently in Phase 2 clinical
development for the treatment of achondroplasia and BMN 190, a recombinant
human tripeptidyl peptidase-1 (rhTPP1) for the treatment of late-infantile
neuronal ceroid lipofuscinosis (CLN2), a form of Batten Disease. For
additional information, please visit www.BMRN.com. Information on BioMarin's
website is not incorporated by reference into this press release.

Vimizim™ is our trademark, and BioMarin^®, Naglazyme^®, Kuvan^®, Firdapse^®
are registered trademarks of BioMarin Pharmaceutical Inc.

Aldurazyme^® is a registered trademark of BioMarin/Genzyme LLC.

CONTACT: Investors:
         Traci McCarty
         BioMarin Pharmaceutical Inc.
         (415) 455-7558
        
         Media:
         Debra Charlesworth
         BioMarin Pharmaceutical Inc.
         (415) 455-7451

BioMarin Pharmaceutical Inc. Logo
 
Press spacebar to pause and continue. Press esc to stop.