- Initiating Phase 3 Program for OMS721 in IgA Nephropathy with Both
Breakthrough and Orphan Designations -
SEATTLE--(BUSINESS WIRE)--Aug. 4, 2017--
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 Immunoglobulin A (IgA) nephropathy. 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. IgA nephropathy is the most
common primary glomerulopathy globally, with an estimated 120,000 to
180,000 cases in the U.S. alone, and accounts for up to 10 percent of
all dialysis patients. Up to 40 percent of individuals with the disease
develop end-stage renal disease, a life-threatening condition, within 20
years following diagnosis.
As previously reported, Phase 2 clinical trial results with OMS721 in
IgA nephropathy patients show unprecedented reductions in urine protein
levels during and following treatment with OMS721. Elevated urinary
protein is highly correlated with poor outcomes in patients with IgA
nephropathy. Following review of these data, FDA in June granted OMS721
breakthrough therapy designation for the treatment of IgA nephropathy.
Omeros plans to begin enrolling patients in its Phase 3 registration
trial in IgA nephropathy later this year.
“Working with FDA, we are initiating another Phase 3 program for OMS721
– this one in IgA nephropathy, which has been granted both breakthrough
and orphan designations,” stated Gregory A. Demopulos, M.D., chairman
and chief executive officer of Omeros. “This marks the second Phase 3
program for OMS721, joining our ongoing aHUS program that already has
received fast track status from FDA. A third Phase 3 program for OMS721
could be added as well this year in stem cell transplant-associated
thrombotic microangiopathy that, together with aHUS, has been granted
orphan designation. Focused on bringing OMS721 to market as quickly as
possible, we are excited about its prospects and the benefits that we
expect OMS721 will provide for patients across a wide range of serious
and life-threatening disorders.”
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.
To date, more than 150 subjects worldwide have received OMS721, and no
safety concerns have been noted.
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.
Following discussions with both the FDA and the European Medicines
Agency, a Phase 3 clinical program for OMS721 in atypical hemolytic
uremic syndrome (aHUS) is in progress. Also, two Phase 2 trials are
ongoing. One is evaluating OMS721 in glomerulonephropathies, which has
generated positive data in patients with immunoglobulin A (IgA)
nephropathy and with lupus nephritis; the other has reported positive
data both in patients with hematopoietic stem cell transplant-associated
thrombotic microangiopathy (TMA) and in those with aHUS. Based on the
positive Phase 2 data, a second Phase 3 clinical program for OMS721 is
being initiated in IgA nephropathy and a third Phase 3 program could
begin later this year in stem cell transplant-associated TMA. OMS721 can
be administered intravenously, and Omeros also expects to commercialize
OMS721 for one or more therapeutic indications as a subcutaneous
injection. 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, orphan drug status for the
prevention (inhibition) of complement-mediated TMAs 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 is preparing to initiate
manufacturing scale-up of its MASP-3 antibodies in advance of clinical
trials.
About Omeros Corporation
Omeros is a biopharmaceutical company committed to discovering,
developing and commercializing both small-molecule and protein
therapeutics for large-market as well as orphan indications targeting
inflammation, coagulopathies and disorders of the central nervous
system. Part of its proprietary PharmacoSurgery® platform,
the company’s first drug product, OMIDRIA® (phenylephrine and
ketorolac injection) 1% / 0.3%, was broadly launched in the U.S. in
April 2015. OMIDRIA is the first and only FDA-approved drug (1) 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 and (2) that
contains an NSAID for intraocular use. In the European Union, the
European Commission has approved OMIDRIA for use in cataract surgery and
lens 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; Huntington’s disease and
cognitive impairment; and addictive and compulsive disorders. In
addition, Omeros has a proprietary G protein-coupled receptor (GPCR)
platform and 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,” “look forward to,”
“may,” “plan,” “potential,” “predict,” “project,” “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 May 10, 2017. 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: http://www.businesswire.com/news/home/20170804005502/en/
Source: Omeros Corporation
Cook Williams Communications, Inc.
Jennifer Cook Williams,
360-668-3701
Investor and Media Relations
jennifer@cwcomm.org
