--Additional Positive Data Reported from Second Cohort in Phase 2
Trial--
SEATTLE--(BUSINESS WIRE)--Jan. 17, 2019--
Omeros Corporation (Nasdaq: OMER) today announced that it has finalized
its clinical plan for OMS721 submission and approval in immunoglobulin A
nephropathy (IgAN) following a recent meeting with the U.S. Food and
Drug Administration (FDA). OMS721 is Omeros’ lead human monoclonal
antibody targeting MASP-2, the effector enzyme of the complement
system’s lectin pathway. There is no approved treatment for IgA
nephropathy.
FDA Meeting Update
Clinical results from the first
and second cohorts of the Phase 2 trial in IgAN were part of
the background materials provided to FDA for the recent meeting on the
Phase 3 clinical program. During the FDA meeting, the following points
were confirmed:
-
The Phase 3 trial’s primary endpoint of assessment of proteinuria was
extended from 24 to 36 weeks at the company’s request to allow for
additional OMS721 dosing, if needed. This continues to provide OMS721
a path to accelerated, or even full, approval based on those 36-week
proteinuria data in either (i) the entire patient population (patients
with baseline proteinuria greater than 1 gm/24 hours) or (ii) the
high-risk subpopulation (those with baseline proteinuria of at least 2
gm/24 hours).
-
Given investigators’ concerns about extended withholding of OMS721
treatment from any high-risk study patient initially randomized to the
placebo-treated group, patients in that subpopulation will be allowed
open-label treatment with OMS721 after at least 1 year of blinded
treatment.
The OMS721 Phase 3 ARTEMIS-IGAN trial continues to enroll and will
incorporate the beneficial changes noted above without any impact to
study patients already enrolled. The trial is designed based on the
positive results from the two previously reported Phase 2 cohorts – the
first assessing patients who were receiving corticosteroids at time of
enrollment and then tapered off steroids during the study and the second
comprised of patients who were not taking steroids.
Additional Phase 2 Data
Additional data are available
from patients in the second cohort of the Phase 2 trial who entered the
extended follow-up period, all receiving OMS721 treatment during this
period. The 8 patients in the extended follow-up period had longstanding
IgA nephropathy (median time from diagnosis of 11.6 years) with
significant comorbidities and significantly impaired renal function
(median baseline estimated glomerular filtration rate (eGFR) of 35.7
mL/min/1.73 m2) with highly elevated baseline proteinuria
levels (median of 3,786 mg/24 hours). The data, based on the last
observation point for each patient, confirm the positive results seen
earlier:
-
eGFR measurements have remained stable, consistent with preservation
of renal function
-
61 percent median reduction in proteinuria from baseline (across all 8
patients, assessed at 31 weeks to 54 weeks post-baseline)
-
5 of the 8 patients have achieved greater than 50 percent proteinuria
reductions (median reduction of 65 percent), with 2 of those 5 having
received their last OMS721 administration 5 months earlier
-
Across the first (4 patients) and second cohorts, a total of 9 of 12
patients achieved greater than 50 percent reductions in proteinuria
(median reduction of 65 percent)
-
OMS721 continues to be well tolerated and no safety concerns have been
observed
“The data from the Phase 2 study continue to show a consistent and
significant drug effect, the magnitude of which is unparalleled,” stated
Richard Lafayette, MD, Professor of Medicine (Nephrology) and Director
of the Glomerular Disease Center, Stanford University Medical Center and
chair of the OMS721 IgAN Academic Leadership Committee. “Nephrologists
are focused on long-term kidney survival, and the observed effects of
OMS721 on both proteinuria and eGFR are what we would hope to see in an
IgAN treatment. The OMS721 Academic Leadership Committee believes the
data on OMS721 in IgAN are sufficiently groundbreaking and robust to
warrant publication in a major scientific journal and plans to submit
the Phase 2 data for publication in the first half of this year,
together with other publications directed to OMS721 in renal disease
throughout 2019. From the perspective of the ALC, the extended data from
the Phase 2 cohort study strongly support the potential efficacy of
OMS721 in IgA nephropathy, even in patients at the very highest risk of
progression to kidney failure. The data, together with the recently
finalized registration plan, are further consistent with our
expectations that the OMS721 Phase 3 ARTEMIS-IGAN trial will be
successful, and we look forward to making the drug available to our
patients.”
In addition to IgAN, OMS721 is in Phase 3 clinical programs for
hematopoietic stem cell transplant-associated thrombotic microangiopathy
(HSCT-TMA) and atypical hemolytic uremic syndrome. OMS721 holds
breakthrough therapy designations from FDA for IgAN and for HSCT-TMA
and, to the company’s knowledge, no other drug has breakthrough therapy
designation for either of these indications.
“The additional understandings reached with FDA are important, and we
appreciate the Agency’s ongoing engagement around our breakthrough
designation for OMS721 in IgA nephropathy,” stated Gregory A. Demopulos,
MD, chairman and chief executive officer of Omeros. “We’re confident in
the effects seen with OMS721 and in our registration approach for the
drug in IgA nephropathy, and we expect that it will likely follow
stem-cell transplant TMA as the second approved indication for OMS721.”
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 in hematopoietic
stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA),
in immunoglobulin A (IgA) nephropathy, and in atypical hemolytic uremic
syndrome (aHUS). 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 continues to enroll patients with HSCT-TMA and has previously
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
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 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
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,”
“future”, “goal,” “intend,” “likely”, “look forward to,” “may,” “on
track”, “path”, “plan,” “potential,” “predict,” “project,” “prospects,”
“should,” “slated,” “will,” “would” and similar expressions and
variations thereof. Forward-looking statements, including statements
regarding the anticipated basis and prospects for approval of OMS721,
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 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.
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Source: Omeros Corporation
Jennifer Cook Williams
Cook Williams Communications, Inc.
Investor
and Media Relations
360.668.3701
jennifer@cwcomm.org