– EMA Confirmed OMS721 Eligibility for European Centralized
Marketing Authorization Application Review –
SEATTLE--(BUSINESS WIRE)--Dec. 6, 2018--
Omeros Corporation (Nasdaq: OMER) today announced that it has submitted
a pediatric investigational plan (PIP) for the use of OMS721 for the
treatment of hematopoietic stem cell transplant-associated thrombotic
microangiopathy (HSCT-TMA) to the European Medicines Association (EMA).
A pediatric study plan (PSP) is also under development for submission to
the U.S. Food and Drug Administration (FDA). OMS721 is Omeros’ lead
human monoclonal antibody targeting mannan-binding lectin-associated
serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of
the complement system. OMS721 was awarded breakthrough therapy
designation for the treatment of high-risk HSCT-TMA earlier this year.
Thrombotic microangiopathy is a life-threatening complication of HSCT,
with mortality reported to be greater than 90 percent in high-risk
patients.
In addition to the data in adult HSCT-TMA patients forming the basis for
its U.S. Biologics License Application (BLA) and E.U. Marketing
Authorization Application (MAA) currently in preparation, Omeros is
proposing a plan including a small study in pediatric patients that will
accelerate development of OMS721 for the treatment of HSCT-TMA in
children. Rather than deferring initiation of a pediatric clinical trial
until after approval in the E.U. or foregoing the study in the U.S.,
which is an option for an orphan drug, Omeros is proposing to initiate
assessment of OMS721 in the pediatric population prior to approval in
view of the strong OMS721 data observed to date and the significant
unmet medical need. Because OMS721 has been designated as an orphan
medicinal product in the EU, successful completion of an agreed PIP will
provide two additional years of market exclusivity in member states of
the European Union, and in Norway, Liechtenstein, and Iceland.
Omeros is also developing a PSP for submission to the FDA. Successful
completion of an agreed PSP in response to a Written Request from FDA
provides an additional 6 months of market exclusivity in the United
States. OMS721 also has been granted orphan drug designation for the
treatment of HSCT-TMA by the FDA. Although the requirement for pediatric
studies is waived for drugs with orphan drug designation, pursuing a PSP
can help accelerate pediatric treatment in the U.S. and provide
additional market exclusivity.
A large prospective study reported that approximately 40% of pediatric
patients who undergo HSCT will develop TMA and approximately 80% of
these patients will have high-risk features.
“All of us at Omeros and treating physicians are confident that OMS721
is saving lives,” stated Gregory A. Demopulos, M.D., chairman and chief
executive officer of Omeros. “Children, like adults, develop
life-threatening HSCT-TMA and do not have approved therapies available.
Approval of OMS721 for pediatric HSCT-TMA will help remove treatment
barriers for this vulnerable population and we expect will allow OMS721
to save many more lives.”
Omeros has also received notification from EMA of eligibility for the
centralized procedure for submission and review of its MAA for OMS721 in
the treatment of HSCT-TMA. The EMA's centralized procedure allows
submission of a single MAA that, when approved, authorizes the drug to
be marketed in all European Union member states and European Free Trade
Association countries rather than requiring separate national approvals.
About Omeros’ MASP Programs
Omeros controls the worldwide rights to MASP-2 and all therapeutics
targeting MASP-2, a novel pro-inflammatory protein target involved in
activation of the complement system, which is an important component of
the immune system. The complement system plays a role in the
inflammatory response and becomes activated as a result of tissue damage
or microbial infection. MASP-2 is the effector enzyme of the lectin
pathway, one of the principal complement activation pathways.
Importantly, inhibition of MASP-2 does not appear to interfere with the
antibody-dependent classical complement activation pathway, which is a
critical component of the acquired immune response to infection, and its
abnormal function is associated with a wide range of autoimmune
disorders. MASP-2 is generated by the liver and is then released into
circulation. Adult humans who are genetically deficient in one of the
proteins that activate MASP-2 do not appear to be detrimentally affected
by the deficiency. OMS721 is Omeros’ lead human MASP-2 antibody.
Phase 3 clinical programs are in progress for OMS721 in atypical
hemolytic uremic syndrome (aHUS), in immunoglobulin A (IgA) nephropathy
and in hematopoietic stem cell transplant-associated thrombotic
microangiopathy (HSCT-TMA). Also, two Phase 2 trials are ongoing. One is
continuing to enroll IgA nephropathy patients and has already generated
positive data in patients with IgA nephropathy and with lupus nephritis;
the other is enrolling and has reported positive data in patients with
HSCT-TMA and in patients with aHUS. OMS721 can be administered both
intravenously and subcutaneously, and Omeros expects to commercialize
each formulation of OMS721 for different therapeutic indications. In
parallel, Omeros is developing small-molecule inhibitors of MASP-2.
Based on requests from treating physicians, Omeros has established a
compassionate-use program for OMS721, which is active in both the U.S.
and Europe. The FDA has granted OMS721 breakthrough therapy designation
for IgA nephropathy and for high-risk HSCT-TMA, orphan drug status for
the prevention (inhibition) of complement-mediated thrombotic
microangiopathies and for the treatment of IgA nephropathy, and fast
track designation for the treatment of patients with aHUS.
Omeros also has identified MASP-3 as responsible for the conversion of
pro-factor D to factor D and as a critical activator of the human
complement system’s alternative pathway. The alternative pathway is
linked to a wide range of immune-related disorders. In addition to its
lectin pathway inhibitors, the company is advancing its development of
antibodies and small-molecule inhibitors against MASP-3 to block
activation of the alternative pathway. Omeros has initiated the
manufacturing scale-up process of its MASP-3 antibodies in preparation
for clinical trials.
About Omeros Corporation
Omeros is a commercial-stage biopharmaceutical company committed to
discovering, developing and commercializing small-molecule and protein
therapeutics for large-market as well as orphan indications targeting
inflammation, complement-mediated diseases and disorders of the central
nervous system. The company’s drug product OMIDRIA®
(phenylephrine and ketorolac intraocular solution) 1% / 0.3% is marketed
for use during cataract surgery or intraocular lens (IOL) replacement to
maintain pupil size by preventing intraoperative miosis (pupil
constriction) and to reduce postoperative ocular pain. In the European
Union, the European Commission has approved OMIDRIA for use in cataract
surgery and other IOL replacement procedures to maintain mydriasis
(pupil dilation), prevent miosis (pupil constriction), and to reduce
postoperative eye pain. Omeros has multiple Phase 3 and Phase 2
clinical-stage development programs focused on: complement-associated
thrombotic microangiopathies; complement-mediated
glomerulonephropathies; cognitive impairment; and addictive and
compulsive disorders. In addition, Omeros has a diverse group of
preclinical programs and a proprietary G protein-coupled receptor (GPCR)
platform through which it controls 54 new GPCR drug targets and
corresponding compounds, a number of which are in preclinical
development. The company also exclusively possesses a novel
antibody-generating platform.
Forward-Looking Statements
This press release contains forward-looking statements within the
meaning of Section 27A of the Securities Act of 1933 and Section 21E of
the Securities Exchange Act of 1934, which are subject to the “safe
harbor” created by those sections for such statements. All statements
other than statements of historical fact are forward-looking statements,
which are often indicated by terms such as “anticipate,” “believe,”
“confident”, “could,” “estimate,” “expect,” “future”, “goal,” “intend,”
“likely”, “look forward to,” “may,” “on track”, “plan,” “potential,”
“predict,” “project,” “prospects,” “should,” “slated,” “will,” “would”
and similar expressions and variations thereof. Forward-looking
statements are based on management’s beliefs and assumptions and on
information available to management only as of the date of this press
release. Omeros’ actual results could differ materially from those
anticipated in these forward-looking statements for many reasons,
including, without limitation, risks associated with product
commercialization and commercial operations, unproven preclinical and
clinical development activities, regulatory oversight, intellectual
property claims, competitive developments, litigation, and the risks,
uncertainties and other factors described under the heading “Risk
Factors” in the company’s Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission on November 8, 2018. Given these
risks, uncertainties and other factors, you should not place undue
reliance on these forward-looking statements, and the company assumes no
obligation to update these forward-looking statements, even if new
information becomes available in the future.
View source version on businesswire.com: https://www.businesswire.com/news/home/20181206005287/en/
Source: Omeros Corporation
Jennifer Cook Williams
Cook Williams Communications, Inc.
Investor
and Media Relations
360.668.3701
jennifer@cwcomm.org
