– EMA Confirmed OMS721 Eligibility for
European Centralized Marketing Authorization Application Review
–
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.
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version on businesswire.com: https://www.businesswire.com/news/home/20181206005287/en/
Jennifer Cook WilliamsCook Williams Communications, Inc.Investor
and Media Relations360.668.3701jennifer@cwcomm.org
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