Omeros’ Narsoplimab Pivotal Trial Data Shared in Oral Presentation at 2021 Annual Congress of the European Hematology Assoc...
June 14 2021 - 08:30AM
Business Wire
-- Data on Organ Function Improvement Presented
by Chief of Adult Bone Marrow Transplant Service at Memorial Sloan
Kettering --
Omeros Corporation (Nasdaq: OMER) today announced that data on
organ function improvement from its pivotal trial of narsoplimab
for the treatment of hematopoietic stem cell transplant-associated
thrombotic microangiopathy (HSCT-TMA) were shared during an oral
presentation at the virtual edition of the 26th Congress of the
European Hematology Association (EHA). The presentation, entitled
Narsoplimab (OMS721) Treatment Contributes to Improvements in Organ
Function in Adult Patients with High-Risk Transplant-Associated
Thrombotic Microangiopathy, was delivered last Friday by
Miguel-Angel Perales, M.D., Chief of Adult Bone Marrow Transplant
Service at Memorial Sloan Kettering Cancer Center. The organ
function improvement data presented underscore the potential of
narsoplimab as a significant advance in the treatment of often
fatal HSCT-TMA.
The trial’s findings include:
- The study population was high-risk, with 93 percent having
multiple risk factors for poor outcomes, and highly reflective of
“real-world” clinical practice
- At baseline:
- 75% of patients had kidney dysfunction
- 57% had neurologic dysfunction
- 18% had pulmonary dysfunction
- 50% had multiple organ TMA involvement
- 86% had significant infection
- 68% had graft versus host disease (GVHD)
- 61% of the intent-to-treat (ITT) population (any patient
receiving at least 1 dose of narsoplimab) and 74% of the
per-protocol (PP) population (those patients receiving ≥ 4 weeks of
dosing) responded to narsoplimab based on improvement in laboratory
TMA markers (platelet count improvement and reduction in LDH
levels) and clinical status (organ
function or freedom from transfusion)
- 74% of eligible patients in the ITT population experienced
improvement in organ function (67%, 50% and 100% in kidney,
neurologic, or gastrointestinal function, respectively); 77% of
eligible patients in the PP population experienced organ function
improvement
- 48% of eligible patients in the ITT population and 55% in the
PP population experienced freedom from transfusion
- Narsoplimab was well tolerated in this very sick population
- The most common adverse events were pyrexia, diarrhea,
vomiting, nausea, neutropenia, fatigue, and hypokalemia, all common
in HSCT
- Six patients died during the core study period due to causes
common in HSCT
- There were no study discontinuations due to non-fatal adverse
events
Detailed data and findings from the study are being submitted to
a peer-reviewed scientific journal for publication.
Dr. Perales’ question-and-answer panel discussion for his
presentation is scheduled for Tuesday, June 15, at 1:00-1:45 pm
CEST/7:00-7:45 am EDT and will be available to registered attendees
through the EHA Congress virtual platform.
In severe cases of HSCT-TMA, mortality can exceed 90 percent
and, even in those who survive, significant morbidity is common
with chronic organ injury often persisting. There is no approved
treatment for HSCT-TMA. A Biologics License Application for use of
narsoplimab in the treatment of HSCT-TMA is under Priority Review
by the US Food and Drug Administration (FDA) with a Prescription
Drug User Fee Act action date of October 17, 2021.
About Hematopoietic Stem Cell Transplant-associated
Thrombotic Microangiopathy
Hematopoietic stem cell transplant-associated thrombotic
microangiopathy (HSCT-TMA) is a significant and often lethal
complication of stem cell transplantation. This condition is a
systemic, multifactorial disorder caused by endothelial cell damage
induced by conditioning regimens, immunosuppressant therapies,
infection, graft-versus-host disease, and other factors associated
with stem cell transplantation. Endothelial damage, which activates
the lectin pathway of complement, plays a central role in the
development of HSCT-TMA. The condition occurs in both autologous
and allogeneic transplants but is more common in the allogeneic
population. In the United States and Europe, approximately 25,000
to 30,000 allogeneic transplants are performed annually. Recent
reports in both adult and pediatric allogeneic stem cell transplant
populations have found an approximately 40-percent incidence of
HSCT-TMA, and high-risk features may be present in up to 80 percent
of these patients. In severe cases of HSCT-TMA, mortality can
exceed 90 percent and, even in those who survive, long-term renal
sequalae (e.g., dialysis) are common. There is no approved therapy
or standard of care for HSCT-TMA.
About Narsoplimab
Narsoplimab, also known as “OMS721,” is an investigational human
monoclonal antibody targeting mannan-binding lectin-associated
serine protease-2 (MASP-2), a novel pro-inflammatory protein target
and the effector enzyme of the lectin pathway of complement.
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. Omeros controls the worldwide rights to MASP-2 and all
therapeutics targeting MASP-2.
A biologics license application (BLA) is under priority review
by the U.S. FDA for use of narsoplimab in the treatment of
HSCT-TMA, and the drug is in Phase 3 clinical programs for
immunoglobulin A (IgA) nephropathy and atypical hemolytic uremic
syndrome (aHUS). Narsoplimab is also being evaluated for the
treatment of COVID-19 as part of the I-SPY-COVID-19 platform trial
sponsored by Quantum Leap Healthcare Collaborative. The FDA has
granted narsoplimab breakthrough therapy designations for HSCT-TMA
and for IgA nephropathy; orphan drug status for the prevention
(inhibition) of complement-mediated thrombotic microangiopathies,
for the treatment of HSCT-TMA and for the treatment of IgA
nephropathy; and fast track designation for the treatment of
patients with aHUS. The European Medicines Agency has granted
orphan drug designation to narsoplimab for treatment in HSCT and
for treatment of primary IgA nephropathy.
About Omeros Corporation
Omeros is a commercial-stage biopharmaceutical company committed
to discovering, developing and commercializing small-molecule and
protein therapeutics for large-market and orphan indications
targeting inflammation, immunologic diseases (e.g.,
complement-mediated diseases and cancers) and central nervous
system disorders. Its commercial product OMIDRIA® (phenylephrine
and ketorolac intraocular solution) 1%/0.3% continues to gain
market share in cataract surgery. Omeros’ lead MASP-2 inhibitor
narsoplimab targets the lectin pathway of complement and is the
subject of a biologics license application under priority review by
FDA for the treatment of hematopoietic stem cell
transplant-associated thrombotic microangiopathy. 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 in a Phase 1 clinical trial, and the company’s
PDE7 inhibitor program OMS527, targeting addiction and movement
disorders, has successfully completed a Phase 1 trial. Omeros’
pipeline holds a diverse group of preclinical programs including a
proprietary-asset-enabled antibody-generating technology and a
proprietary GPCR platform through which it controls 54 GPCR drug
targets and their corresponding compounds. One of these novel
targets, GPR174, modulates a new cancer immunity axis recently
discovered by Omeros, and the company is advancing GPR174-targeting
antibodies and small-molecule inhibitors. For more information
about Omeros and its programs, visit www.omeros.com.
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Jennifer Cook Williams Cook Williams Communications, Inc.
Investor and Media Relations 360.668.3701 jennifer@cwcomm.org
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