- Primary statistical analysis of overall survival compared
TA-TMA patients treated with narsoplimab in its pivotal trial to a
cohort of over 100 TA-TMA patients not treated with narsoplimab in
an external control stem cell transplant registry; FDA-agreed
analysis was conducted by an independent external statistical
group
- Narsoplimab-treated TA-TMA patients had an over 3-fold
reduction in risk of mortality (hazard ratio = 0.32 [95% confidence
interval: 0.23, 0.44]; p < 0.00001) compared to similarly
at-risk patients without narsoplimab treatment
- Omeros is preparing and will resubmit a BLA to FDA for
narsoplimab to become the first approved therapeutic for TA-TMA, a
life-threatening complication of hematopoietic stem cell
transplantation
Omeros Corporation (Nasdaq: OMER) today announced that an
independent statistical group has completed the primary statistical
analysis agreed with the FDA for narsoplimab, Omeros’
first-in-class monoclonal antibody inhibiting the lectin pathway of
complement, in the treatment of hematopoietic stem cell
transplant-associated thrombotic microangiopathy (TA-TMA), a
life-threatening complication in both adult and pediatric
hematopoietic stem cell transplantation (HSCT). The analysis
compared overall survival in the 28 TA-TMA patients in
OMS721-TMA-001, Omeros’ previously conducted pivotal trial for
narsoplimab in TA-TMA, to that of more than 100 similarly high-risk
TA-TMA patients in an external control registry of HSCT patients
who did not receive narsoplimab treatment. Narsoplimab met its
primary endpoint, with OMS721-TMA-001 patients demonstrating
clinically meaningful and statistically significant superiority in
overall survival – a hazard ratio of 0.32 (95% confidence interval:
0.23 to 0.44) with p-value less than 0.00001 – compared to the
TA-TMA registry patients. Given these results, Omeros will resubmit
to FDA as soon as possible its narsoplimab Biologics License
Application (BLA) for TA-TMA. Omeros aims to make narsoplimab,
which targets the lectin pathway’s effector enzyme MASP-2, the
first approved treatment for TA-TMA.
“The results of this comparative survival analysis for
narsoplimab in TA-TMA are truly outstanding,” stated Alessandro
Rambaldi, M.D., Professor of Hematology at the University of Milan
and Head of the Hematology and Bone Marrow Transplant Unit at ASST
Papa Giovanni XXIII in Bergamo, Italy. “As an investigator in the
pivotal clinical trial and having since requested and used
narsoplimab in a good number of very sick TA-TMA patients through
the expanded access program, we have seen first-hand the impressive
benefits of narsoplimab in these high-risk patients. While we do
not use C5 inhibitors in our transplant patients, nearly 50
patients in the expanded access program received narsoplimab after
failing or stopping C5 inhibitors and other off-label treatments,
with TA-TMA resolving with narsoplimab in 46 percent and 50 percent
of these adults and children, respectively, which is quite
remarkable. There is a tremendous need for an approved therapeutic
for TA-TMA patients, and we are hopeful that narsoplimab will soon
be made available.”
Late last month, Omeros announced that it had received FDA’s
recommendations on the statistical analysis plan (SAP) for the
primary analysis comparing overall survival from time of first
dosing in the 28 narsoplimab-treated TA-TMA patients in the pivotal
trial OMS721-TMA-001 to overall survival, adjusted for immortal
time bias, of the more than 100 TA-TMA patients in the external
control registry, none of whom received narsoplimab. The two
cohorts had similar demographics, diagnostic criteria, baseline
characteristics, underlying diseases, conditioning regimens, and
transplant procedures. All patients in both cohorts met the
criteria for high risk of death as defined by an international
expert panel tasked with reaching consensus on diagnostic and
prognostic criteria and representing the American Society for
Transplantation and Cellular Therapy, the Center for International
Bone Marrow Transplant Research, the Asia-Pacific Blood and Marrow
Transplantation Group, and the European Society for Blood and
Marrow Transplantation. Following receipt of FDA’s recommendations,
the independent statistical group incorporated them into the final
SAP, conducted and validated the primary analysis, and then shared
the results with Omeros.
“TA-TMA is an increasingly recognized and devastating
complication of stem cell transplantation, often leading to
multi-organ failure and death,” stated Rafael Duarte, M.D., Ph.D.,
F.R.C.P.(Lon), Associate Professor, Head of Department of
Hematology and the Hematopoietic Transplantation Program,
University Hospital Puerta de Hierro Majadahonda, Madrid, Spain,
and former Secretary of the European Society for Blood and Marrow
Transplantation and former Chair of the Society’s Transplant
Complications Working Party. “The comparative survival results with
narsoplimab are compelling. TA-TMA is understood to be a disease
driven by microvascular endothelial dysfunction and complement
activation, specifically via the lectin pathway of complement,
which we know is hyperactivated by endothelial damage. By targeting
MASP-2, the key enzyme in the lectin pathway, narsoplimab inhibits
the pathway’s activation and interrupts the cycle of further
endothelial damage. So, mechanistically, this positions narsoplimab
as a potential therapeutic option not only for TA-TMA but also for
other conditions characterized by endothelial dysfunction, such as
ARDS, where it may play a pivotal role in addressing the underlying
pathology.”
Additional analyses are being conducted by the independent
statistical group, including an analysis similar to the primary
analysis but comparing survival in the high-risk TA-TMA patients
enrolled in the global narsoplimab expanded access program (EAP) to
that of similarly at-risk TA-TMA registry patients. Sensitivity
analyses related to each of the primary and EAP comparisons are
also pending. These are expected to support the results of the
primary analysis and will be included in the narsoplimab BLA for
TA-TMA. Representative analyses will be shared publicly.
Across all its clinical trials in various indications to date,
narsoplimab has been well tolerated and has shown no safety signal
of concern.
“We are very pleased with the results of the primary analysis,”
said Gregory A. Demopulos, M.D., Omeros’ Chairman and Chief
Executive Officer. “Our team worked collaboratively with FDA over
many months and incorporated FDA’s recommendations on the
statistical analysis plan comparing overall survival in
OMS721-TMA-001 to that of the external control. Given the primary
analysis results, we will resubmit the narsoplimab BLA for TA-TMA
as quickly as possible followed by our planned submission of the
corresponding European marketing authorisation application in the
second quarter of 2025. Additional sensitivity and other analyses
are pending and should be available over the next few weeks. The
Omeros team and clinical investigators have seen the benefits of
narsoplimab in both adults and children. As demonstrated by the
primary analysis, narsoplimab markedly improves survival, and we
look forward to enabling broad accessibility for TA-TMA patients
and their physicians.”
Two manuscripts – one comparing survival between OMS721-TMA-001
and TA-TMA registry patients and the other comparing survival in
the EAP to that of TA-TMA registry patients – will be authored by
international groups of transplant experts and submitted for
publication to peer-reviewed journals in the first part of the
coming year.
About Narsoplimab
Narsoplimab, also known as “OMS721,” is an investigational fully
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 has
been demonstrated to leave intact the antibody-dependent classical
complement activation pathway, which is a critical component of the
acquired immune response to infection. A biologics license
application (BLA) is pending before the FDA for use of narsoplimab
in the treatment of hematopoietic stem cell transplant-associated
thrombotic microangiopathy (TA-TMA). Omeros will resubmit the BLA
for narsoplimab in TA-TMA followed by our planned submission of the
corresponding European marketing authorisation application (MAA) in
2025. FDA has granted narsoplimab breakthrough therapy and orphan
drug designations for TA-TMA and orphan drug status for the
prevention (inhibition) of complement-mediated thrombotic
microangiopathies as well as for the treatment of TA-TMA. The
European Medicines Agency (EMA) has granted orphan drug designation
to narsoplimab for treatment in hematopoietic stem-cell
transplant.
About Hematopoietic stem cell transplant-associated
thrombotic microangiopathy (TA-TMA)
Hematopoietic stem cell transplant-associated thrombotic
microangiopathy (TA-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 TA-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 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 Omeros Corporation
Omeros is an innovative biopharmaceutical company committed to
discovering, developing and commercializing first-in-class
small-molecule and protein therapeutics for large-market and orphan
indications targeting immunologic disorders, including
complement-mediated diseases and cancers, 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. Omeros’ long-acting MASP-2 inhibitor OMS1029 has
successfully completed Phase 1 single- and multiple-ascending dose
clinical studies. Zaltenibart, Omeros’ inhibitor of MASP-3, the key
activator of the alternative pathway of complement, is advancing
toward Phase 3 clinical trials for paroxysmal nocturnal
hemoglobinuria and complement 3 glomerulopathy. Funded by the
National Institute on Drug Abuse, Omeros’ lead phosphodiesterase 7
inhibitor OMS527 is in clinical development for the treatment of
cocaine use disorder. Omeros also is advancing a broad portfolio of
five novel cellular and molecular immuno-oncology programs. 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 “aim,” “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 resubmission of the biologics
license application for narsoplimab in the United States and the
submission of a marketing authorization application with the EMA,
the timing and outcomes of regulatory events, the availability and
outcomes of additional analyses, the prospects for obtaining FDA or
EMA approval of narsoplimab in any indication, expectations
regarding future cash expenditures, and expectations regarding the
sufficiency and availability of our capital resources to fund
current and planned operations, including the potential
commercialization of narsoplimab if it is approved by FDA or the
EMA, 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, unfavorable, unexpected or
inconclusive results of our statistical analyses relating to an
external registry of TA-TMA patients, potential differences between
the diagnostic criteria used in our pivotal trial and in the
external registry, and whether FDA and the EMA determine the
registry used in our statistical analysis is sufficiently
representative of TA-TMA patients, unanticipated or unexpected
outcomes of regulatory processes in relevant jurisdictions,
unproven preclinical and clinical development activities, our
financial condition and results of operations, regulatory processes
and oversight, challenges associated with manufacture or supply of
our products to support clinical trials, regulatory inspections
and/or commercial sale following any marketing approval, changes in
reimbursement and payment policies by government and commercial
payers or the application of such policies, intellectual property
claims, competitive developments, litigation, and the risks,
uncertainties and other factors described under the heading “Risk
Factors” in our Annual Report on Form 10-K filed with the
Securities and Exchange Commission on April 1, 2024, an in our
subsequently filed Quarterly Reports on Form 10-Q. Given these
risks, uncertainties and other factors, you should not place undue
reliance on these forward-looking statements, and we assume 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/20241219918248/en/
Jennifer Cook Williams Cook Williams Communications, Inc.
Investor and Media Relations IR@omeros.com
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