Full Press Release Details
European Medicines Agency Grants Orphan Drug
Designation for MT-401 developed by Marker Therapeutics for the Treatment of AML Patients
TX - July 10, 2023 - Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage immuno-oncology company
focusing on developing next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumor indications,
announced that zedenoleucel, its multi-tumor-associated antigen (multiTAA)-specific T cell product candidate, MT-401, was granted
Orphan Drug Designation by the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) for the treatment of patients
with acute myeloid leukemia (AML).
AML is a life-threatening and chronically debilitating
disease that is rapidly progressive and fatal if untreated. Relapse rates after initial treatment are high, and the next step for eligible
patients is an allogeneic hematopoietic stem cell transplant (HSCT). Unfortunately, AML relapse after HSCT is frequent and outcomes are
dismal. Patients who relapse after HSCT have an estimated median survival of less than one year (Estey and D hner, Lancet, 2006),
highlighting the urgent need for new therapies.
MT-401 utilizes a novel non-genetically modified
approach that recognizes multiple antigens expressed on tumor cells, thereby designed to minimize tumor escape. MT-401 is currently being
studied in a Phase 2 clinical trial for the treatment of relapsed AML following allogeneic HSCT, and was designed to specifically target
four different antigens that are upregulated in AML but have limited expression on normal cells.
In the European Union, orphan drug designation
is granted to drugs intended for the treatment of life-threatening or chronically debilitating conditions affecting no more than five
in 10,000 individuals in the European Union. Orphan drug designation by the EMA provides crucial support to expedite the development and
market readiness of necessary drugs for such rare diseases. This designation will help Marker Therapeutics continue to develop MT-401
to fill a significant void in the treatment of AML and provides Marker Therapeutics with a range of potential benefits, including ten
years of market exclusivity following approval, reduced regulatory fees, and invaluable scientific advice from the EMA during the drug
"The orphan drug designation for MT-401 by
the EMA is a significant regulatory milestone," said Nadia Agopyan, Ph.D., RAC, Senior Vice President, Regulatory Affairs of Marker
Therapeutics. "It acknowledges not just the potential therapeutic impact of MT-401, but also the urgent need to deliver innovative
treatment options to patients living with AML. In 2020, MT-401 was also granted orphan designation by the U.S. Food and Drug Administration
for the treatment of patients with AML. We are deeply committed to working with regulatory authorities to expedite the drug development
and approval process."
"We are extremely proud to have been granted
Orphan Drug Designation by the EMA for MT-401," said Juan F. Vera, M.D., President and Chief Executive Officer of Marker Therapeutics.
"In our Phase 2 clinical trial of patients with post-transplant AML, we have observed promising results from patients with measurable
residual disease, suggesting that the unique and differentiated targeting technology of MT-401 can be a potential treatment for patients
with AML before relapse."
"This is an important milestone for Marker
and a significant step forward in our mission to improve the lives of patients with AML, especially of those with relapsed AML where no
therapeutic options have been approved. Our team at Marker is committed to accelerating the development of MT-401 and believes that this
designation brings us one step closer to offering a new, potentially life-altering therapy for relapsed AML patients after stem cell transplant,"
About multiTAA-specific T cells
The multi-tumor associated antigen (multiTAA)-specific
T cell platform is a novel, non-genetically modified cell therapy approach that selectively expands tumor-specific T cells from a patient's/donor's
blood capable of recognizing a broad range of tumor antigens. Clinical trials that enrolled more than 180 patients with various hematological
malignancies and solid tumors showed that autologous and allogeneic multiTAA-specific T cell products were well tolerated and demonstrated
durable clinical responses, and consistent epitope spreading. The latter is typically not observed with other T cell therapies and enables
the potential contribution to a lasting anti-tumor effect. Unlike other cell therapies which require hospitalization and close monitoring,
multiTAA-specific T cells are designed to be administered in an outpatient setting.
About Marker Therapeutics, Inc.
Marker Therapeutics, Inc. is a clinical-stage
immuno-oncology company specializing in the development of next-generation T cell-based immunotherapies for the treatment of hematological
malignancies and solid tumor indications. Marker's cell therapy technology is based on the selective expansion of non-engineered,
tumor-specific T cells that recognize tumor associated antigens (i.e., tumor targets) and kill tumor cells expressing those targets. This
population of T cells is designed to attack multiple tumor targets following infusion into patients and to activate the patient's
immune system to produce broad spectrum anti-tumor activity. Because Marker does not genetically engineer the T cells, Marker believes
that its product candidates will be easier and less expensive to manufacture, with reduced toxicities, compared to current engineered
CAR-T and TCR-based approaches, and may provide patients with meaningful clinical benefit. As a result, Marker believes its portfolio
of T cell therapies has a compelling product profile, as compared to current gene-modified CAR-T and TCR-based therapies.
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Forward-Looking Statements
release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act
of 1995. Statements in this news release concerning the Company's expectations, plans, business outlook or future performance,
and any other statements concerning assumptions made or expectations as to any future events, conditions, performance or other matters,
are "forward-looking statements." Forward-looking statements include statements regarding our intentions, beliefs, projections,
outlook, analyses or current expectations concerning, among other things: our research, development and regulatory activities and expectations
relating to our non-engineered multi-tumor antigen specific T cell therapies; the effectiveness of these programs or the possible range
of application and potential curative effects and safety in the treatment of diseases; the timing, conduct and success of our clinical
trials of our product candidates, including MT-401 for the treatment of patients with AML;
and the potential benefits of orphan drug designation to MT401. Forward-looking statements are by their nature subject to risks, uncertainties
and other factors which could cause actual results to differ materially from those stated in such statements. Such risks, uncertainties
and factors include, but are not limited to the risks set forth in the Company's most recent Form 10-K, 10-Q and other SEC filings
which are available through EDGAR at WWW.SEC.GOV. The Company assumes no obligation
to update its forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press
release except as may be required by law.
TIBEREND STRATEGIC ADVISORS, INC.
Daniel Kontoh-Boateng