-- Additional Positive “Challenge-Rechallenge” Data Reported in
Patient with Stem Cell Transplant-Associated TMA --
SEATTLE--(BUSINESS WIRE)--Mar. 28, 2017--
Omeros Corporation (NASDAQ: OMER) today announced presentation of a case
report describing resolution of hematopoietic stem cell
transplant-associated thrombotic microangiopathy (HSCT-TMA) in a
dialysis- and transfusion-dependent adolescent girl who was treated with
OMS721 under a compassionate-use protocol. The presentation “Resolution
of acute kidney injury secondary to TA-TMA by the anti-MASP-2 monoclonal
antibody OMS721 in a pediatric HSCT recipient” occurred at the 43rd
Annual Meeting of the European Society for Blood and Marrow
Transplantation in Marseille, France on Monday, March 27, 2017. Marco
Zecca, M.D., Director of Pediatric Oncology at the Fondazione IRCCS
Policlinico San Matteo, presented the data. 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.
The presentation describes a girl who developed HSCT-TMA following stem
cell transplantation at 14 years of age. She was initially treated with
eculizumab but did not tolerate treatment, developing pulmonary edema
that recurred on retreatment with eculizumab. The HSCT-TMA became
life-threatening and she required hemodialysis and daily platelet
transfusions. Dr. Zecca, the patient’s physician, requested OMS721 for
compassionate-use treatment and Omeros complied. Following OMS721
treatment, the patient was able to discontinue hemodialysis and to
decrease substantially her platelet transfusion requirements. Recently,
the patient’s dose of OMS721 was tapered, but she developed a viral
infection that reactivated her HSCT-TMA. Her TMA was again successfully
treated with restoration of the original OMS721 dose. To date, she has
remained free of both dialysis and transfusions.
“This patient had severe TMA that I believe would have caused her
death,” stated Dr. Zecca. “Her positive response to OMS721 treatment,
both initially and following her virus-induced relapse during tapering,
was impressive – the results of OMS721 treatment in this
challenge-rechallenge scenario underscore the important effects of the
drug. Since the poster was produced, her TMA has remained in remission
and we have been able to discontinue her platelet transfusions. Her
rapid response has been heartening, and we all are grateful for this
remarkable outcome.”
Thrombotic microangiopathy is a potentially life-threatening
complication of HSCT. Approximately 20,000 HSCT procedures are performed
in the U.S. annually, and TMA is reported to occur in up to 30 percent
of HSCT patients. Although the kidney is the most commonly affected
organ, HSCT-TMA is a multi-system disorder and can also manifest
clinically in the lungs, gastrointestinal tract and central nervous
system. Reported mortality in patients with multi-organ involvement is
greater than 90%. Even in patients who survive acute episodes, HSCT-TMA
increases the risk for chronic kidney disease and end-stage renal
disease.
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 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; the
other has reported positive data both in patients with hematopoietic
stem cell transplant-associated thrombotic microangiopathy (TMA) and in
those with aHUS. 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. The FDA has granted
OMS721 both orphan drug status for the prevention (inhibition) of
complement-mediated TMAs and fast track designation for the treatment of
patients with aHUS.
Omeros also has identified MASP-3 as the critical activator of the human
complement system’s alternative pathway, 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 Annual Report on Form 10-K filed with the
Securities and Exchange Commission on March 16, 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/20170328005644/en/
Source: Omeros Corporation
Cook Williams Communications, Inc.
Jennifer Cook Williams
Investor
and Media Relations
360.668.3701
jennifer@cwcomm.org
