-- Targeted Indications for the Company’s MASP-2 Inhibitor Continue to
Expand --
SEATTLE--(BUSINESS WIRE)--Oct. 20, 2016--
Omeros Corporation (NASDAQ: OMER) today announced positive results in
patients with hematopoietic stem cell transplant-associated thrombotic
microangiopathy (HSCT-TMA) from the company's Phase 2 clinical trial of
OMS721 in TMAs, with clinically meaningful improvement in measures of
red blood cell destruction, specifically lactate dehydrogenase (LDH) and
haptoglobin levels (p < 0.01 and p < 0.06, respectively). Severe
HSCT-TMAs carry a high mortality rate. OMS721 is Omeros' lead human
monoclonal antibody targeting mannan-binding lectin-associated serine
protease-2 (MASP-2). In addition to its Phase 2 clinical program in
TMAs, OMS721 is currently in a Phase 3 program for patients suffering
from atypical hemolytic uremic syndrome and in a Phase 2 program for
renal diseases, including immunoglobulin A (IgA) nephropathy (or
Berger’s disease) and membranous nephropathy.
All HSCT-TMA patients in the Phase 2 open-label clinical trial are
adults who had received stem-cell transplantation for hematological
malignancies. Inclusion criteria for these patients included low
platelet count, hemolytic anemia (i.e., destruction of red blood cells
within small blood vessels) and the requirement that the patients had
not responded to attempted treatment of the TMA with modification of
their immunosuppressive drugs. Changing the immunosuppressive regimen is
common practice in HSCT patients who develop a TMA, and those who do not
respond to these drug changes frequently die. Three HSCT-TMA patients,
all of whom had failed immunosuppressive modification, completed
treatment with OMS721 for up to 8 weeks. Two other patients discontinued
OMS721 early in their treatment courses, one later relapsed and the
other’s treatment was changed to palliative care only.
Across all three patients completing treatment, mean TMA markers
improved over time. The mean LDH level decreased from a baseline of 672
U/L by as much as 380 U/L (p < 0.01). The mean haptoglobin increased
over 1.54 g/L from a baseline of 0.33 g/L (p < 0.06). LDH and
haptoglobin are measures of hemolytic anemia. The mean platelet count
increased as much as 57,000/μL from a baseline of 18,000/μL but did not
reach statistical significance in this small number of patients.
Creatinine remained normal or improved, nearly normalizing, in two
patients; one did not show improvement in creatinine but was receiving
concomitant nephrotoxic drugs. On extended follow up, two patients
remain stable and one experienced graft failure, which the investigator
considers unrelated to OMS721 treatment.
No significant safety concerns have been observed. The most commonly
reported adverse events in these patients have been abdominal pain,
diarrhea, neutropenia and hypokalemia, all commonly seen in this patient
population. An abstract has been submitted for presentation at the
February 2017 Tandem Meeting of the American Society of Blood and Marrow
Transplantation and the Center for International Blood and Marrow
Transplant Research.
“These data are exciting,” stated Samer Khaled, M.D., Assistant Clinical
Professor of Hematology and Hematopoietic Cell Transplantation at City
Of Hope in Duarte, California, and OMS721 clinical investigator. “The
response in our patient was impressive, particularly since the patient
had failed previous treatments. Lectin pathway inhibition could play a
role in other post-transplant complications, and I look forward to
continued development of OMS721 in stem cell transplantation.”
Approximately 20,000 HSCT procedures are performed in the U.S. annually,
and TMA is reported to occur in approximately 10 to 25 percent of HSCT
patients. TMA most commonly affects the kidney but can also damage the
lungs, gastrointestinal tract and central nervous system. Mortality in
patients who develop severe HSCT-TMA is greater than 90 percent.
“In addition to the data being collected in aHUS patients, these
HSCT-TMA results expand the potential benefits of OMS721 to multiple
thrombotic microangiopathy indications,” stated Gregory A. Demopulos
M.D., chairman and chief executive officer of Omeros. “Stem cell
transplantation is an expanding field and represents an attractive
medical and commercial opportunity. We plan to discuss with FDA and
international regulatory agencies the design of a single Phase 3 trial
for HSCT-TMA and look forward to OMS721 addressing the significant unmet
needs of patients across a wide range of hematologic, renal and other
therapeutic indications.”
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 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. Omeros has received both Orphan Drug status and Fast
Track designation from the U.S. FDA for its lead human MASP-2 antibody
OMS721. Following discussions with both the FDA and the European
Medicines Agency, a Phase 3 program for OMS721 in atypical hemolytic
uremic syndrome is in progress. Also, two Phase 2 trials are ongoing
with one evaluating OMS721 in glomerulonephropathies, which has
generated positive data in patients with immunoglobulin A (IgA)
nephropathy and with membranous nephropathy, and the other in thrombotic
microangiopathies (TMAs), with positive data reported in patients with
hematopoietic stem cell transplant-associated TMA. In addition to
potential intravenous administration, Omeros plans to commercialize
OMS721 for one or more therapeutic indications as a subcutaneous
injection and is also 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.
Omeros also has identified MASP-3 as the protein that is critical to the
activation of 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 its
development of antibodies and small-molecule inhibitors against MASP-3
to block activation of the alternative pathway.
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
clinical-stage development programs focused on: complement-related
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, which is making
available an unprecedented number of new GPCR drug targets and
corresponding compounds to the pharmaceutical industry for drug
development, and a platform used to generate antibodies.
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, Omeros’ 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, 2016. 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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Source: Omeros Corporation
Cook Williams Communications, Inc.
Jennifer Cook Williams
Investor
and Media Relations
360.668.3701
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