BioMarin Receives Favorable Ruling in the Use of Exon 51 Antisense Oligonucleotides Patent Interference
30 September 2015 - 8:10AM
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the
Patent Trial and Appeal Board (PTAB) issued a decision in favor of
BioMarin's claims to the use of exon 51 antisense oligonucleotides
to treat Duchenne muscular dystrophy. The BioMarin patent
application involved is U.S. Patent Application No. 14/198,992
(allowed methods of use claims), which is licensed to BioMarin from
the Academisch Ziekenhuis Leiden (AZL), The Netherlands. The
decision by the PTAB is based on motions filed in the patent
interference proceeding between BioMarin (due to the acquisition of
Prosensa Therapeutics N.V. (Prosensa)) and Sarepta (formerly AVI)
of Cambridge, Mass as licensee under patents filed by the
University of Western Australia (UWA). The allowed claims
cover the use of the antisense oligonucleotide drisapersen, as well
as other antisense oligonucleotides that induce skipping exon 51
for the treatment of Duchenne muscular dystrophy (DMD). BioMarin
also protects drisapersen under U.S. Patents 7,534,879 and
8,759,507, two patents which were not involved in the interference.
The other interference between AZL and UAW related to
composition of matter claims for antisense oligonucleotides for
Exon 51 skipping, Interference 106,008, remains pending.
Companion European Patent
In Europe, BioMarin's method patent EP 1 619 249 B1 was
previously upheld in an amended form after an Opposition procedure
initiated by AVI Biopharma (now Sarepta) before the European Patent
Office Opposition Division. The upheld claims include the use
of 14- to 40-mer antisense oligonucleotides directed to exon
51 in the DMD gene as a potential therapy to treat
DMD. Although an appeal procedure is pending, BioMarin
currently has an issued and enforceable patent, which encompasses
antisense oligonucleotide product/product candidates directed to
exons 51 and 46 in Europe.
About Interference Proceedings in the U.S.
A patent interference is a proceeding conducted by the Patent
Office in instances where two parties claim patent rights to the
same subject matter. The U.S. patent system awards patents to the
first party to invent a particular technology. In an interference,
the Patent Office determines which party invented the technology
first, and awards the patent to that party. In the order entered
today, the PTAB declared that BioMarin's licensor, AZL, was the
first to invent the claimed inventions and therefore entered
judgement against the UWA patent application. Sarepta may appeal
the PTAB decision to the U.S. Court of Appeals for the Federal
Circuit. If they timely file an appeal, it is unlikely that
the Patent Office will issue the allowed claims of the AZL patent
until the appeal is completed.
About the Allowed U.S. Patent Application
claims
U.S. Patent Application No. 14/198,992 (methods of use) belongs
to a portfolio of patents entitled "Modulation of exon recognition
in pre-mRNA by interfering with secondary structure" that was
exclusively licensed from the Leiden University Medical Center
(LUMC, Leiden, The Netherlands), also referred to as Academisch
Ziekenhuis Leiden. The inventor of this application and current
BioMarin employee, Dr. Judith van Deutekom, VP, Discovery in
Research & Development, was former head of the DMD Genetic
Therapy group at LUMC and is one of the pioneers in the development
of genetic therapies for muscular dystrophies.
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.
For additional information, please visit www.BMRN.com.
Information on BioMarin's website is not incorporated by reference
into this press release.
Forward-Looking Statement
This press release contains forward-looking statements about the
business prospects of BioMarin Pharmaceutical Inc., including,
without limitation, statements about: the ongoing patent disputes
in both Europe and the United States and the scope of the patents
and patent applications. 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: future outcomes of
patent proceedings, and any appeals thereto; 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 2014 Annual Report
on Form 10-K, as amended, and the factors contained in BioMarin's
reports on Form 8-K. 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®, Kuvan®, Firdapse® and VIMIZIM® 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
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