Omeros Corporation Streamlines Path to Biologics License Application for OMS721 in HSCT-TMA Following Meeting with FDA

Patient Data from Existing Single-Arm Study Will Form the Basis for
BLA; No Historical Control Required

SEATTLE–(BUSINESS WIRE)–Omeros Corporation (Nasdaq: OMER) today announced that, based on a
recent meeting with the U.S. Food and Drug Administration (FDA), the
company has streamlined the required work and further advanced its
preparations for its Biologics License Application (BLA) for OMS721 in
the treatment of hematopoietic stem cell transplant-associated
thrombotic microangiopathy (HSCT-TMA). OMS721 holds FDA’s breakthrough
therapy designation as well as orphan drug designations from FDA and the
European Medicines Agency (EMA) for HSCT-TMA. OMS721 is the company’s
Phase 3 human monoclonal antibody targeting mannan-binding
lectin-associated serine protease-2 (MASP-2), the effector enzyme of the
lectin pathway of the complement system.

Agreements reached during the recent FDA meeting are the following:

  • A response-based analysis is the most appropriate and expeditious
    assessment for inclusion in an OMS721 BLA for the treatment of
    HSCT-TMA. Criteria for this primary-endpoint analysis were proposed by
    Omeros and discussed with FDA and are expected to be finalized in the
    near term. Overall and 100-day survival will now be secondary
  • Using the response-based analysis as the primary endpoint eliminates
    the need for a historical control, and Omeros is no longer conducting
    its chart review-based historical-control data collection.
  • Patient data from the existing single-arm OMS721 HSCT-TMA trial will
    form the clinical basis for the BLA, and Omeros will collect
    additional data from patients currently in the trial for inclusion in
    the BLA.
  • FDA will consider not only accelerated approval but also regular
    (full) approval for OMS721 in HSCT-TMA, with the determination based
    on the submitted data.
  • A rolling BLA submission is appropriate for OMS721 in this indication.
    Omeros plans to submit first the nonclinical sections, which were
    completed late last year.

“We appreciate FDA’s collaborative approach to the development of OMS721
for patients with stem cell transplant-associated TMA. We now have an
even clearer and more streamlined path to submitting our BLA, saving
time and crucial resources as we advance toward regulatory review and
approval,” stated Gregory A. Demopulos, M.D., chairman and chief
executive officer of Omeros. “There’s no FDA-approved treatment for stem
cell transplant-associated TMA, and severe cases progress rapidly with a
very high mortality rate. We have seen in our ongoing clinical trial the
life-saving effects of OMS721 in these patients, a good number of whom
had not only TMA but also other components – such as graft-versus-host
disease and diffuse alveolar hemorrhage – of broader endothelial-injury
syndrome. We remain committed to working closely with FDA and European
regulators to make OMS721 available as quickly as possible to patients
who need it.”

Omeros plans to meet with European regulators and expects to harmonize
the marketing authorization application (MAA) for OMS721 in HSCT-TMA in
Europe with the U.S. BLA. Omeros has received positive feedback from
EMA’s Pediatric Development Committee on its Pediatric Investigational
Plan for OMS721, a required element for an MAA, and intends to meet in
the second quarter of this year with the rapporteurs assigned to the
company’s application.

In addition to its HSCT-TMA registration program, Omeros is enrolling
its OMS721 Phase 3 clinical trials for immunoglobulin A (IgA)
nephropathy and atypical hemolytic uremic syndrome (aHUS). OMS721 also
holds FDA’s breakthrough therapy designation for IgA nephropathy, FDA’s
and EMA’s orphan drug designations for IgA nephropathy, and FDA’s
fast-track designation for aHUS.

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 OMS721, Omeros’ lead
MASP-2 inhibitor, in hematopoietic stem cell transplant-associated
thrombotic microangiopathy (HSCT-TMA), in immunoglobulin A (IgA)
nephropathy, and in atypical hemolytic uremic syndrome (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
HSCT-TMA, orphan drug status for the prevention (inhibition) of
complement-mediated thrombotic microangiopathies, for the treatment of
IgA nephropathy and 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 OMS721 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 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

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,” “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, the ability for OMIDRIA to obtain separate reimbursement as
part of CMS’ OPPS, 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 8, 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.


Jennifer Cook Williams
Cook Williams Communications, Inc.

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