European Commission Designates OMS721 as an Orphan Medicinal Product for Treatment in Hematopoietic Stem Cell Transplantation
August 28 2018 - 7:30AM
Business Wire
Omeros Corporation (Nasdaq: OMER) today announced that the
European Commission (EC) has adopted a decision designating OMS721
as an Orphan Medicinal Product in the European Union (EU) for
treatment in hematopoietic stem cell transplantation (HSCT). The
adoption by the EC follows a positive opinion for orphan
designation of OMS721 in this indication by the European Medicines
Agency’s (EMA) Committee for Orphan Medicinal Products (COMP).
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. There are three Phase 3 development programs
ongoing with OMS721 in each of HSCT-associated thrombotic
microangiopathy (TMA), atypical hemolytic uremic syndrome (aHUS),
and immunoglobulin A (IgA) nephropathy.
This represents the second orphan drug designation in Europe for
OMS721, with the first being for OMS721 in the treatment of primary
IgA nephropathy. Orphan drug designation in Europe is
available to companies developing products intended
to treat a life-threatening or chronically debilitating
condition that affects fewer than five in 10,000 persons
in the EU. This designation allows for financial and
regulatory incentives that include a 10-year period of
marketing exclusivity in the EU after product approval, protocol
assistance from the EMA at reduced fees during the
product development phase, and access to centralized
marketing authorization. OMS721 also has been granted orphan
designations from the U.S. Food and Drug
Administration (FDA) for indications related to both
HSCT-associated TMA and IgA nephropathy.
Omeros is in discussions with European regulators and FDA to
discuss full and conditional/accelerated approval for OMS721 in
HSCT-associated TMA.
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,” “could,” “estimate,” “expect,”
“goal,” “intend,” “likely,” “look forward to,” “may,” “plan,”
“potential,” “predict,” “project,” “prospects,” “should,” “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 August 9, 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/20180828005255/en/
Cook Williams Communications, Inc.Jennifer Cook Williams,
360-668-3701Investor and Media Relationsjennifer@cwcomm.org
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