-- 15 Year-Old with Stem Cell
Transplant-Associated TMA Had Previously Developed Additional
Life-Threatening Condition while on Soliris® --
Omeros Corporation (NASDAQ: OMER) today announced that,
following physician request, it will provide continued supply of
OMS721 for a pediatric patient with hematopoietic stem cell
transplant-associated thrombotic microangiopathy (HSCT-TMA) who is
currently receiving the drug under the company’s compassionate use
program. The patient’s treating physician requested extended access
to OMS721 for his 15 year-old patient given her positive results
with OMS721 treatment, including discontinuation of dialysis.
HSCT-TMA most commonly affects the kidneys but can also damage the
lungs, gastrointestinal tract and central nervous system, and
severe cases often require dialysis and carry a mortality rate in
excess of 90 percent. 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
TMA, 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 and membranous nephropathy.
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. HSCT-TMA patients frequently have a
complex history of disease. This pediatric patient had received a
stem cell transplant to treat a rare life-threatening anemia.
Following her transplant, she developed TMA. She began treatment
with Soliris and improved but developed pulmonary edema so Soliris
treatment was stopped. Following relapse of her HSCT-TMA, she
received a low dose of Soliris and again developed pulmonary edema,
again requiring discontinuation of Soliris treatment. Her TMA
progressed and she developed renal failure requiring dialysis. When
she began OMS721 treatment, she had been on dialysis for several
months and required daily platelet transfusions.
After three weeks of OMS721 treatment the patient was able to
discontinue dialysis. The frequency of platelet transfusions has
been decreased by more than 50 percent despite bone marrow
suppression caused by other concurrent conditions. Two measures of
red blood cell destruction have also substantially improved on
OMS721 treatment: her haptoglobin has normalized on OMS721
treatment and her lactate dehydrogenase (LDH) has decreased by more
than 50% percent but still remains slightly elevated. All OMS721
administrations have been well tolerated by the patient, and no
side effects have been observed with this treatment. The patient is
also now able to spend weekends at home with her family. Because of
the patient’s improvement on OMS721, her treating physician
requested, and Omeros granted, an extension of the OMS721
compassionate use treatment protocol under which this pediatric
patient is being treated.
“The patient, her family and her team of physicians are
obviously thrilled with her improvement on OMS721,” stated Marco
Zecca, M.D., Director of Pediatric Hematology/Oncology at
Fondazione IRCCS Policlinico San Matteo in Pavia, Italy. “Stem cell
transplant-related TMA is a devastating and often fatal condition,
and this is a patient who had failed other treatment. Her response
to OMS721 is impressive, underscored by her ability to stop
dialysis. We appreciate receiving ongoing access to OMS721 for her
treatment, and we look forward to her continued improvement and a
return to a more healthy and normal life.”
“All of us at Omeros feel privileged to participate in the care
of this young girl,” stated Gregory A. Demopulos M.D., Chairman and
Chief Executive Officer of Omeros. “We remain committed to
supporting both her and her physicians in her care and to her
continued recovery. While treatment results are routinely described
in numbers and statistics, this patient demonstrates the human side
of our work, and it is heartening.”
Omeros’ OMS721 compassionate use program is active
internationally.
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. One is evaluating OMS721 in glomerulonephropathies,
which has generated positive data in patients with immunoglobulin A
(IgA) nephropathy and with membranous nephropathy and the other is
being conducted in patients with 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-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, 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 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
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.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20161206005673/en/
Cook Williams Communications, Inc.Jennifer Cook WilliamsInvestor
and Media Relations360.668.3701jennifer@cwcomm.org
Omeros (NASDAQ:OMER)
Historical Stock Chart
From Apr 2024 to May 2024
Omeros (NASDAQ:OMER)
Historical Stock Chart
From May 2023 to May 2024