FDA Grants Orphan Drug Designation to Omeros’ MASP-3 Inhibitor OMS906 for Treatment of Paroxysmal Nocturnal Hemoglobinuria
July 29 2022 - 8:45AM
Business Wire
-- Enrollment of PNH Patients
Expected to Begin this Summer --
Omeros Corporation today announced that OMS906 has received
orphan drug designation from the U.S. Food and Drug Administration
(FDA) for the treatment of paroxysmal nocturnal hemoglobinuria
(PNH). OMS906 targets mannan-binding lectin-associated serine
protease-3 (MASP-3), the key activator of the alternative pathway
of the complement system. MASP-3 converts pro-complement factor D
(pro-CFD) to mature CFD.
PNH is a rare, life-threatening disease characterized by red
blood cell destruction, blood clots and impaired bone marrow
function. Based on its mechanism of action as well as the
pharmacokinetic/pharmacodynamic (PK/PD) profile shown in a
completed Phase 1 study, OMS906 has the potential to offer a
favorable safety profile and more convenient dosing than other
drugs on the market or in development for PNH. Also, different than
other enzymes in the alternative pathway targeted by competitors’
agents, MASP-3 does not appear to be an acute phase reactant,
meaning that the concentration of MASP-3 – and the effective dosing
level of OMS906 – do not change in the setting of inflammation.
Omeros continues to build a strong and exclusive intellectual
property position around therapeutics targeting MASP-3.
“We are excited to begin clinical studies to demonstrate the
efficacy of OMS906 in alternative pathway-related disorders,” said
Gregory A. Demopulos, M.D., chairman and chief executive officer of
Omeros. “Based on our preclinical and Phase 1 trial data, we expect
that OMS906 will perform well. With the potential advantages of
MASP-3 inhibition and OMS906 – decreased infection risk, better
dosing profile, and the ability to avoid ‘breakthrough’ disease
seen with agents targeting acute phase reactants – we believe that
OMS906 could become first-line therapy in alternative pathway
disorders.”
FDA grants orphan designation to promote the development of a
drug that is expected to have significant therapeutic advantage
over existing treatments that target a condition affecting 200,000
or fewer U.S. patients annually. It qualifies a company for
benefits that apply across all stages of drug development,
including seven years of market exclusivity following marketing
approval, tax credits on U.S. clinical trials, eligibility for
orphan drug grants, and waiver of certain administrative fees.
Omeros recently completed a Phase 1 trial of OMS906 in healthy
subjects and expects to begin enrollment this summer in a clinical
trial assessing OMS906 in PNH patients who have had an
unsatisfactory response to the C5 inhibitor ravulizumab. The
company is also targeting efficacy data in populations of
treatment-naïve PNH and C3 glomerulopathy patients by early 2023.
Based on the results of the completed Phase 1 trial, administration
of OMS906 is expected to be once monthly to once quarterly
intravenously or subcutaneously.
About OMS906
OMS906 is an investigational human monoclonal antibody targeting
mannan-binding lectin-associated serine protease-3 (MASP-3), the
key activator of the complement system’s alternative pathway. The
complement system plays a central role in inflammation and becomes
activated as a result of tissue damage or microbial infection.
Responsible for the conversion of pro-complement factor D to
complement factor D, MASP-3 is believed to be the premier target in
the alternative pathway – it has the lowest native circulating
level and low relative clearance compared to the other alternative
pathway proteins and, unlike C5 and C3 blockers, MASP-3 inhibition
leaves intact the lytic arm of the classical pathway, important for
fighting infection. Also, unlike other components of the
alternative pathway, MASP-3 is believed not to be an acute phase
reactant, which could provide a significant advantage to MASP-3
inhibitors, like OMS906, over other alternative pathway inhibitors.
MASP-3 inhibitors are thought to have preventive or therapeutic
effects across a broad range of diseases including paroxysmal
nocturnal hemoglobinuria (PNH), hemolytic uremic syndrome (HUS),
atypical HUS, traumatic brain injury, arthritis, wet age-related
macular degeneration, ischemia-reperfusion injury,
transplant-related complications and other immune-related
disorders. Through its growing and exclusive intellectual property
position, Omeros controls the use of MASP-3 inhibitors across a
wide range of alternative pathway-related and other diseases and
disorders.
About Omeros Corporation
Omeros is an innovative biopharmaceutical company committed to
discovering, developing and commercializing small-molecule and
protein therapeutics for large-market and orphan indications
targeting immunologic diseases, including complement-mediated
diseases and cancers related to dysfunction of the immune system,
as well as addictive and compulsive disorders. Omeros’ lead MASP-2
inhibitor narsoplimab targets the lectin pathway of complement and
is the subject of a biologics license application pending before
FDA for the treatment of hematopoietic stem cell
transplant-associated thrombotic microangiopathy (HSCT-TMA).
Narsoplimab is also in multiple late-stage clinical development
programs focused on other complement-mediated disorders, including
IgA nephropathy, atypical hemolytic uremic syndrome and COVID-19.
OMS906, Omeros’ inhibitor of MASP-3, the key activator of the
alternative pathway of complement, is initiating Phase 1b clinical
programs in paroxysmal nocturnal hemoglobinuria (PNH) and
complement 3 glomerulopathy (C3G). For more information about
Omeros and its programs, visit www.omeros.com.
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,” “objective,”
“plan,” “potential,” “predict,” “project,” “should,” “slate,”
“target,” “will,” “would” and similar expressions and variations
thereof. Forward-looking statements, including statements regarding
the anticipated therapeutic advantages of Omeros’ drug candidates,
expectations regarding the initiation or continuation of clinical
trials evaluating Omeros’ drug candidates and the anticipated
availability of data therefrom, 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,
unanticipated or unexpected outcomes of regulatory processes in
relevant jurisdictions, unproven preclinical and clinical
development activities, events negatively affecting our financial
condition or results of operations, regulatory processes and
oversight, challenges associated with manufacture or supply of our
investigational or clinical products, 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 1, 2022. 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,
whether as a result of new information, future events or otherwise,
except as required by applicable law.
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version on businesswire.com: https://www.businesswire.com/news/home/20220729005194/en/
Jennifer Cook Williams Cook Williams Communications, Inc.
Investor and Media Relations IR@omeros.com
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