SEATTLE, Nov. 19, 2014 /PRNewswire/ -- Omeros
Corporation (NASDAQ: OMER) today announced positive data using
OMS721, the lead human monoclonal antibody for its mannan-binding
lectin-associated serine protease-2 (MASP-2) program, to inhibit
thrombus formation in an ex vivo
pathophysiologic system of human atypical hemolytic uremic
syndrome (aHUS), a form of thrombotic microangiopathy (TMA). TMAs
are a family of rare, debilitating and life-threatening disorders
characterized by excessive thrombi (clots) in the microcirculation
of the body's organs, most commonly the kidney and brain. OMS721 is
currently in a Phase 2 clinical program evaluating the drug's
efficacy in treating TMAs, including aHUS.
The data announced today resulted from studies in a
well-established experimental model of TMA aimed at assessing the
potential therapeutic benefits of OMS721 in TMA using serum samples
from aHUS patients with different etiologies obtained during the
acute phase of disease as well as during remission. The studies
were conducted by Prof. Giuseppe
Remuzzi and colleagues Marina
Noris and Miriam Galbusera at
the Mario Negri Institute for Pharmacological Research in Bergamo,
Italy, and the Clinical Research
Center for Rare Diseases "Aldo e Cele Dacco" of the same institute,
a European center for the study of TMA. The experimental model is
based on the finding that sera from aHUS patients promote the
formation of thrombi on human microvascular endothelial cells, the
defining pathological feature of TMA.
The studies reported today showed that OMS721 significantly
inhibited thrombus formation when added to serum samples from aHUS
patients obtained during the acute phase of disease (p<0.01) as
well as during remission (p<0.0001). OMS721 was as effective at
inhibiting thrombus formation as the positive control in the
studies – soluble complement receptor 1, an agent that completely
blocks the complement system.
Earlier this year, Omeros reported that OMS721 also
significantly inhibited complement deposition compared to control
treatment in an experimental ex vivo
pathophysiologic system of complement activation in TMA, again
using serum samples from aHUS patients obtained during the acute
phase of disease (p<0.01) and during remission (p<0.001).
Prof. Remuzzi's laboratories have previously shown in the same
complement-activation model that eculizumab (Soliris®),
a monoclonal antibody blocking the complement factor C5 that is
approved by the FDA and the European Medicines Agency to treat
patients with aHUS, also inhibited complement deposition. Data
recently published indicate that the ex vivo
pathophysiologic system established by Prof. Remuzzi and his
colleagues may be useful in assessing the clinical effectiveness of
anti-complement therapy in aHUS (Noris et al., Blood (2014)
124:1715).
"We believe that the notable anti-complement and anti-thrombotic
activities of OMS721 in serum samples from aHUS patients bode well
for the therapeutic potential of OMS721 in thrombotic
microangiopathies," stated Prof. Remuzzi, MD, FRCP, Research
Coordinator at the Mario Negri Institute and international expert
in the study of kidney disease who has contributed major advances
to the understanding of the pathophysiology of hemolytic uremic
syndrome.
Having demonstrated that the lectin pathway, and MASP-2
specifically, are involved in the pathophysiology of aHUS, Prof.
Remuzzi and his team are currently conducting additional
experiments to understand the mechanism by which OMS721 blocks
ex vivo thrombus formation in aHUS and its effect on
endothelial cells, platelets and the alternative pathway of
complement.
"Thrombus formation is the central pathological feature of aHUS
and other TMAs, so we are particularly pleased with the robust
anti-thrombotic activity in serum samples from aHUS patients,"
stated Gregory A. Demopulos, M.D.,
chairman and chief executive officer of Omeros. "We believe that
these most recent data from Prof. Remuzzi's laboratories
meaningfully de-risk our '721 Phase 2 program in TMAs, including
aHUS, and we look forward to reporting preliminary efficacy and
safety data from the ongoing clinical trial."
About Omeros' MASP-2 Program
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 appears to
be unique to, and required for the function of, one of the
principal complement activation pathways, known as the lectin
pathway. 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 the 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. Therefore, Omeros believes that it may be possible
to deliver MASP-2 antibodies systemically and OMS721, its lead
MASP-2 antibody, is designed to be self-administered by
subcutaneous injection.
Omeros also believes that it has identified the proteins that
activate the complement system's alternative pathway in humans,
which is linked to a wide range of immune-related disorders. In
addition to its lectin pathway inhibitors, the Company is advancing
the development of antibodies that would block activation of the
alternative pathway alone or in combination with the lectin
pathway.
About Omeros Corporation
Omeros is a biopharmaceutical company committed to discovering,
developing and commercializing small-molecule and protein
therapeutics for large-market as well as orphan indications
targeting inflammation, coagulopathies and disorders of the central
nervous system. Derived from its proprietary
PharmacoSurgery® platform, the company's first drug
product, Omidria™ (phenylephrine and ketorolac
injection) 1%/0.3%, has been approved by the FDA for use during
cataract surgery or intraocular lens replacement (ILR) to maintain
pupil size by preventing intraoperative miosis (pupil constriction)
and to reduce postoperative ocular pain. Omeros is completing
preparations for a planned U.S. product launch in early 2015.
Omidria is currently under review for marketing approval by the
European Medicines Agency. Omeros has six clinical-stage
development programs focused on: complement-related thrombotic
microangiopathies; Huntington's disease, schizophrenia, and
cognitive impairment; addictive and compulsive disorders; and
preventing problems associated with surgical procedures. In
addition, Omeros has a proprietary GPCR platform, which is making
available an unprecedented number of new GPCR drug targets and
corresponding compounds to the pharmaceutical industry for drug
development.
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," "look forward to," "may," "plan," "potential,"
"predict," "project," "should," "will," "would" and similar
expressions. 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 effectiveness of
Omidria™ sales and marketing efforts, Omidria market
acceptance, product pricing and reimbursement, Omeros' ability to
obtain regulatory approval for its Marketing Authorization
Application and to partner in the EU for the commercialization of
Omidria, Omeros' unproven preclinical and clinical development
activities, regulatory oversight, product commercialization,
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 10, 2014. 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.
SOURCE Omeros Corporation