-- Growing Identification of HSCT-TMA a Focus
of Recent Annual Meeting of the European Society for Blood and
Marrow Transplantation --
Omeros Corporation (Nasdaq: OMER) today announced the
recent presentation of a case report describing resolution of
gastrointestinal hematopoietic stem cell transplant-associated
thrombotic microangiopathy (HSCT-TMA) following narsoplimab
treatment under a compassionate-use protocol. The case was
presented at the 2019 Annual Meeting of the European Society for
Blood and Marrow Transplantation (EBMT) held March 24-27 in
Frankfurt, Germany. Omeros is reporting the case to ensure broad
availability of the information presented. Narsoplimab, Omeros’
antibody to inhibit the lectin pathway of complement, has received
breakthrough therapy designation from FDA and is in Phase 3
development for the treatment of HSCT-TMA.
The case was presented by Rafael Duarte M.D., Ph.D., F.R.C.P.,
Associate Professor, Head of Hematopoietic Transplantation and
Hemato-oncology Section, University Hospital Puerta de Hierro
Majadahonda, Madrid, Spain, and Secretary of
the EBMT. Dr. Duarte described an 18-year-old patient with
biopsy-proven HSCT-TMA of the gastrointestinal tract causing severe
gastrointestinal bleeding requiring transfusions. The patient
received narsoplimab, her TMA resolved, and all transfusions have
been discontinued. The patient continues to do well after cessation
of narsoplimab treatment.
Hematopoietic stem cell transplant-associated TMA was a focus of
the EBMT meeting program. In addition to the educational course on
early transplant complications that included Dr. Duarte’s
presentation, the program included a review session on renal
complications of HSCT-TMA and an Omeros-sponsored symposium
entitled “How Do I Diagnose HSCT-TMA.” All of these sessions were
well attended. The meeting program also contained several other
podium and poster presentations directed to complications related
to endothelial injury, which include HSCT-TMA, graft-versus-host
disease and diffuse alveolar hemorrhage.
The interest in HSCT-TMA shown at the EBMT meeting tracks a
broadening recognition of the high incidence and severity of this
transplant complication. This growing awareness within the
stem-cell transplant community is further reflected by the
increasing frequency of compassionate-use requests from physicians
worldwide seeking narsoplimab treatment for adult and pediatric
patients with HSCT-TMA. Historically, the literature-based
incidence of HSCT-TMA has been varied, with some smaller centers
and national registries reporting low numbers of cases while major
research institutions reported incidences in the range of 15-25
percent. More recently, however, as TMA has become better
understood and identified, its reported occurrence has been
approximately 40 percent in patients undergoing allogeneic HSCT in
centers that screen for the disease. The condition is associated
with an increased mortality rate and, even in those patients who
survive, it often causes chronic kidney damage.
Omeros is preparing both a U.S. biologics license application
(BLA) and a European marketing authorization application for
narsoplimab in the treatment of HSCT-TMA. The FDA has agreed that a
rolling BLA submission is appropriate and discussions with European
regulators are ongoing.
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.
Gene-targeted MASP-2-deficient mice and humans with MASP-2 gene
polymorphisms that affect MASP-2 serum levels and MASP-2 functional
activity are generally healthy with no obvious adverse
phenotype.
Phase 3 clinical programs are in progress for narsoplimab,
Omeros’ lead MASP-2 inhibitor also referred to as “OMS721,” in
hematopoietic stem cell transplant-associated thrombotic
microangiopathy (HSCT-TMA), in immunoglobulin A (IgA) nephropathy,
and in atypical hemolytic uremic syndrome (aHUS). Narsoplimab can
be administered both intravenously and subcutaneously,
and Omeros expects to commercialize each formulation of
narsoplimab 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 narsoplimab, which is active in both the U.S.
and Europe. The FDA has granted narsoplimab
breakthrough therapy designation for IgA nephropathy and for
HSCT-TMA, orphan drug status for the prevention (inhibition) of
complement-mediated thrombotic microangiopathies, for the treatment
of IgA nephropathy, for the treatment of HSCT-TMA, and fast track
designation for the treatment of patients with aHUS. The
European Medicines Agency has granted orphan drug designation
to narsoplimab for treatment of primary IgA nephropathy and for
treatment in HSCT.
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, disorders of the central nervous system and
immune-related diseases, including cancers. 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,” “slated,”
“targeting,” “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 Annual Report on Form 10-K filed with
the Securities and Exchange Commission on March 1,
2019. 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/20190403005324/en/
Jennifer Cook WilliamsCook Williams Communications, Inc.Investor
and Media Relations360.668.3701jennifer@cwcomm.org
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