Apolipoprotein C-III and ISIS-APOCIII
Rx
We have obtained patent claims in the United States drawn to the use of antisense compounds
complementary to a broad active region of humanApo C-III including the site targeted by ISIS-APOCIII
Rx
.
Similar claims complementary to any site on humanApo C-III have granted inAustralia. We obtained issued
patent claims to the specific antisense sequence and chemical composition of ISIS-APOCIII
Rx
in the United
States, Australia, and Europe. The issued U.S. claims should protect ISIS-APOCIII
Rx
from generic competition in
the United States until at least 2023. In addition, we will seek additional patent term extension to recapture a
portion of the term lost during FDA regulatory review, extending the term of this patent beyond 2023. We are
also pursuing additional patent applications designed to protect the ISIS-APOCIII
Rx
composition in Canada and
additional methods of use in jurisdictions worldwide. The table below lists the key U.S, European andAustralian
issued patents:
Jurisdiction
Patent No.
Title
Expiration
Description of Claims
United States
7,598,227
MODULATIONOF
APOLIPOPROTEINC-III
EXPRESSION
2023
Methods of treating hyperlipidemia,
lowering cholesterol levels and lowering
triglyceride levels with an antisense
compound comprising an antisense
oligonucleotide 15-30 linked nucleosides
specifically hybridizable within nucleotides
3253-3558 of SEQ ID 4 (apoCIII)
United States
7,750,141
MODULATIONOF
APOLIPOPROTEINC-III
EXPRESSION
2023
Antisense sequence and chemistry of
ISIS-APOCIII
Rx
Europe
EP1622597
MODULATIONOF
APOLIPOPROTEINC-III
EXPRESSION
2023
Antisense sequence and chemistry of
ISIS-APOCIII
Rx
Australia
2004231550
MODULATIONOF
APOLIPOPROTEINC-III
EXPRESSION
2023
Compounds 12-50 nucleobases in length
specifically hybridizable with SEQ ID 4
(apoCIII), the antisense sequence and
chemistry of
ISIS-APOCIII
Rx
and methods
of their use in treating hyperlipidemia,
lowering cholesterol levels and lowering
triglyceride levels
Survival Motor Neuron and ISIS-SMN
Rx
ISIS-SMN
Rx
is protected by a suite of patents in the United States and in Europe from generic competition
in the United States until at least 2030 and in Europe until 2026. These issued patents include: (i) the Bennett
patent related to methods of altering mRNAprocessing (i.e., splicing) with a fully-modified 2’MOE
oligonucleotide, (ii) a patent licensed from the University of Massachusetts drawn to antisense compounds having
the sequence of ISIS-SMN
Rx
, independent of chemical modification and uses of such compounds for treating
SMA, and (iii) a joint patent with Cold Spring Harbor Laboratory claiming fully-modified 2’MOE compositions
targeting SMN2, including the precise composition of matter of ISIS-SMN
Rx
. Those patents should protect
ISIS-SMN
Rx
from generic and antisense innovator competition in the United States until at least 2030 without
patent term extension. The table below lists the key U.S. and European issued patents protecting ISIS-SMN
Rx
:
Jurisdiction
Patent No.
Title
Expiration
Description of Claims
United States
6,210,892
ALTERATIONOF CELLULAR
BEHAVIORBYMODULATION
OFMRNAPROCESSING
2018
Broad claims of altering mRNAprocessing
with a fully-modified 2’MOE
oligonucleotide.
United States
8,361,977
COMPOSITIONSANDMETHODS
FORMODULATIONOF SMN2
SPLICING
2030
Sequence and chemistry (full 2’-MOE) of
ISIS-SMN
Rx
Europe
1910395
COMPOSITIONSANDMETHODS
FORMODULATIONOF SMN2
SPLICING
2026
Sequence and chemistry (full 2’-MOE) of
ISIS-SMN
Rx
United States
7,838,657
SPINALMUSCULARATROPHY
(SMA) TREATMENTVIA
TARGETINGOF SMN2 SPLICE
SITE INHIBITORYSEQUENCES
2027
Oligonucleotides having sequence of
ISIS-SMN
Rx
(chemistry independent)
United States
8,110,560
SPINALMUSCULARATROPHY
(SMA) TREATMENTVIA
TARGETINGOF SMN2 SPLICE
SITE INHIBITORYSEQUENCES
2025
Methods of using antisense
oligonucleotides having sequence of
SMN
Rx
to alter splicing of SMN2 and/or to
treat SMA
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