-- OMS721 now with orphan designation covering both prevention and
treatment of HSCT-TMA in U.S. and in Europe --
SEATTLE--(BUSINESS WIRE)--Oct. 23, 2018--
Omeros
Corporation (NASDAQ:OMER) today announced that OMS721 has received
orphan drug designation from the U.S. Food and Drug Administration (FDA)
for the treatment of hematopoietic stem cell transplant-associated
thrombotic microangiopathy (HSCT-TMA). 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
reported mortality of greater than 90 percent in some high-risk patients.
This most recent orphan designation for OMS721 by FDA is consistent with
Omeros’ Phase 3 program in HSCT-TMA and, therefore, confers to OMS721 on
approval for this indication all the benefits of orphan drug
designation. OMS721 also has orphan designation in the U.S. for
immunoglobulin A (IgA) nephropathy and for the prevention (inhibition)
of complement-mediated TMA, including HSCT-TMA and atypical hemolytic
uremic syndrome (aHUS). Omeros has active Phase 3 programs for OMS721 in
HSCT-TMA, IgA nephropathy, and aHUS.
“With FDA’s granting of an additional orphan drug designation for OMS721
for stem cell transplant-associated TMA, we now have orphan designations
that cover both the prevention and treatment of this disorder with
OMS721 in the U.S. as well as in Europe,” stated Gregory A. Demopulos,
M.D., chairman and chief executive officer of Omeros. “We continue to
work with FDA and European regulators as we pursue expedited pathways to
OMS721 approval in stem-cell TMA. Our objective is to make this drug --
which we and leading experts believe is saving lives – available to
transplanters and their patients as quickly as possible.”
FDA grants orphan designation to promote the development of a drug that
is expected to have significant therapeutic advantage over existing
treatments that target a condition affecting 200,000 or fewer U.S.
patients annually. It qualifies a company for benefits that apply across
all stages of drug development, including seven years of market
exclusivity following marketing approval, tax credits on U.S. clinical
trials, eligibility for orphan drug grants, and waiver of certain
administrative fees.
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,” “objective”, “plan,” “potential,” “predict,” “project,”
“prospects,” “pursue”, “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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20181023005491/en/
Source: Omeros Corporation
Cook Williams Communications, Inc.
Jennifer Cook Williams
Investor
and Media Relations
360-668-3701
jennifer@cwcomm.org
