Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq:
YMAB) a commercial-stage biopharmaceutical company focused on the
development and commercialization of novel, antibody-based
therapeutic products for the treatment of cancer, today announced
that the U.S. Food and Drug Administration (“FDA”) has approved
DANYELZA (naxitamab-gqgk) 40mg/10ml. DANYELZA is indicated, in
combination with granulocyte-macrophage colony-stimulating factor
(“GM-CSF”), for the treatment of pediatric patients 1 year of age
and older and adult patients with relapsed or refractory high-risk
neuroblastoma in the bone or bone marrow who have demonstrated a
partial response, minor response, or stable disease to prior
therapy. This indication is approved under accelerated approval
regulation based on overall response rate and duration of response.
Continued approval for this indication may be contingent upon
verification and description of clinical benefits in a confirmatory
trial. DANYELZA is a humanized, monoclonal antibody that targets
the ganglioside GD2, which is highly expressed in various
neuroectoderm-derived tumors and sarcomas. DANYELZA is administered
to patients three times in a week in an outpatient setting and the
treatment is repeated every four weeks. The product has received
Priority Review, Orphan Drug, Breakthrough Therapy, and Rare
Pediatric Disease designations from the FDA.
“Today is an important day for children living
with refractory/relapsed high-risk neuroblastoma,” said Thomas Gad,
founder, Chairman and President. “It’s very exciting to see this
treatment go from being an experimental therapy used at my
daughter’s bedside to now being FDA approved. On behalf of Y-mAbs,
I want to thank all the patients and physicians who took part in
our clinical trials and our scientific partner, Memorial Sloan
Kettering, for helping us achieve this goal.”
“We believe that DANYELZA in combination with
GM-CSF is a much-needed treatment for patients with
relapsed/refractory high-risk neuroblastoma in the bone or bone
marrow who have historically not had approved treatments available.
This approval of Y-mAbs’ first BLA represents a key step in working
towards our mission of becoming a world leader in developing better
and safer antibody-based oncology products addressing unmet
pediatric and adult medical needs,” said Claus Moller, Chief
Executive Officer.
The FDA approval of DANYELZA is supported by
clinical evidence from two pivotal studies in patients with
high-risk neuroblastoma with refractory or relapsed disease.
DANYELZA appears to be well tolerated with few discontinuations of
treatment in the clinical trials and adverse events were clinically
manageable. See below for information related to adverse
reactions.
The FDA granted approval under the accelerated
approval regulation. The postmarketing clinical trial required by
the FDA to verify and to further characterize the clinical benefit
is the ongoing Study 201, which will enroll a minimum of 80
patients and report overall response rate (“ORR”), duration of
response (“DOR”), progression free survival (“PFS”) and overall
survival (“OS”). The ORR is the primary endpoint for the study, DOR
is the secondary endpoint, PFS and OS are secondary endpoints in
long-term follow up.
DANYELZA is expected to be available in the
United States in the coming weeks. To learn more about DANYELZA,
visit DANYELZA.com. To help patients get started on DANYELZA,
Y-mAbs ConnectTM has been created to answer questions about access,
health insurance coverage, financial support programs and other
resources available for qualifying patients. To learn more about
Y-mAbs Connect, visit ymabsconnect.com.
Researchers at Memorial Sloan Kettering Cancer
Center (“MSK”) developed DANYELZA, which is exclusively licensed by
MSK to Y-mAbs. As a result of this licensing arrangement, MSK has
institutional financial interests related to the compound and
Y-mAbs.
About High-Risk
Neuroblastoma
Neuroblastoma is a solid tumor of childhood that
arises in the nervous system, outside of the brain. The clinical
behavior of neuroblastoma is highly variable, with some tumors
being easily treatable, but the majority being very aggressive. All
patients are staged based on the International Neuroblastoma
Staging System Committee (“INSS”) system, ranging from stage 1
through stage 4S. All patients with stage 4 disease diagnosed after
one year of age are classified in the high-risk category, where the
neuroblastoma tumor cells have already metastasized to other sites
in the body, such as the bone or bone marrow. Essentially all
patients who have tumors with many copies, or amplification, of the
MYCN oncogene also have high-risk disease, even if they do not have
evidence of the tumor having spread.
Important Safety Information and
Indication for DANYELZA® (naxitamab-gqgk)
Indication
DANYELZA (naxitamab-gqgk) is indicated, in
combination with granulocyte-macrophage colony-stimulating factor
(“GM-CSF”), for the treatment of pediatric patients 1 year of age
and older and adult patients with relapsed or refractory high-risk
neuroblastoma in the bone or bone marrow who have demonstrated a
partial response, minor response, or stable disease to prior
therapy. This indication is approved under accelerated approval
based on overall response rate and duration of response. Continued
approval for this indication may be contingent upon verification
and description of clinical benefits in a confirmatory trial.
Important Safety
Information
Please click here to see the full Prescribing Information for
DANYELZA.
Contraindications
DANYELZA is contraindicated in patients with a
history of severe hypersensitivity reaction to naxitamab-gqgk.
Reactions have included anaphylaxis.
Warnings and Precautions
DANYELZA has been approved with a box
warning.
In clinical studies, DANYELZA has been shown to
cause serious infusion reactions including anaphylaxis, cardiac
arrest, bronchospasm, stridor, and hypotension. Infusion reactions
generally occurred within 24 hours of completing a DANYELZA
infusion, most often within 30 minutes of initiation. Infusion
reactions are most frequent during first infusion in each cycle.
Premedicate with an antihistamine, acetaminophen, an H2 antagonist
and corticosteroid as recommended in the label. Monitor patients
closely for signs and symptoms of infusion reactions during and for
at least 2 hours following completion of each DANYELZA infusion in
a setting where cardiopulmonary resuscitation medication and
equipment are available. Reduce the rate, interrupt infusion, or
permanently discontinue DANYELZA based on severity and institute
appropriate medical management as needed.
Based on its mechanism of action, DANYELZA can
cause severe pain. Premedicate with gabapentin and e.g. oral
oxycodone. Treat break-through pain with intravenous hydromorphone
or equivalent.
One case of transverse myelitis (Grade 3) has
been reported. Permanently discontinue DANYELZA therapy in case of
transverse myelitis.
DANYELZA may cause severe hypertension. The
onset of hypertension may be delayed. Monitor blood pressure during
and after infusion. Interrupt DANYELZA infusion and resume at a
reduced rate, or permanently discontinue DANYELZA based on the
severity.
Two cases of posterior reversible encephalopathy
syndrome (“PRES”) have been reported. Monitor blood pressure during
and following DANYELZA infusion and assess for neurologic symptoms.
Permanently discontinue DANYELZA in case of symptomatic PRES.
Adverse ReactionsThe most
common adverse events were mainly mild and moderate and included
infusion-related reaction, pain, tachycardia, vomiting, cough,
nausea, diarrhea, decreased appetite, hypertension, fatigue,
erythema multiforme, peripheral neuropathy, urticaria, pyrexia,
headache, edema, anxiety, localized edema and irritability.
This is not the complete list of Warnings,
Precautions and Adverse Reactions. For further information see
label.
To report suspected adverse reactions, contact Y-mAbs
Therapeutics, Inc., at 1-833-339-6227 (1-833-33YMABS), or FDA at
1-800-FDA-1088 or www.fda.gov/medwatch.
About
Y-mAbs
Y-mAbs is a commercial-stage biopharmaceutical
company focused on the development and commercialization of novel,
antibody-based therapeutic products for the treatment of cancer.
The Company has a broad and advanced product pipeline, including
DANYELZA and omburtamab, which target tumors that express
B7-H3.
Forward-Looking Statements
Statements in this press release about future
expectations, plans and prospects, as well as any other statements
regarding matters that are not historical facts, may constitute
“forward-looking statements” within the meaning of The Private
Securities Litigation Reform Act of 1995. Such statements include,
but are not limited to, statements about our business model and
development and commercialization plans; the benefits, safety and
efficacy of DANYELZA, current and future clinical and pre-clinical
studies and our research and development programs; expectations
related to the timing of the initiation and completion of
regulatory submissions; regulatory, marketing and reimbursement
approvals; rate and degree of market acceptance and clinical
utility as well as pricing and reimbursement levels; retaining and
hiring key employees; our commercialization, marketing and
manufacturing capabilities and strategy; our intellectual property
position and strategy; additional product candidates and
technologies; collaborations or strategic partnerships and the
potential benefits thereof; expectations related to the use of our
cash and cash equivalents, and the need for, timing and amount of
any future financing transaction; our financial performance,
including our estimates regarding revenues, expenses, capital
expenditure requirements; developments relating to our competitors
and our industry; and other statements that are not historical
facts. Words such as ‘‘anticipate,’’ ‘‘believe,’’ “contemplate,”
‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’
‘‘may,’’ ‘‘might,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’
‘‘project,’’ ‘‘should,’’ ‘‘target,’’ “will”, ‘‘would’’ and similar
expressions are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words. Our product candidates and related technologies
are novel approaches to cancer treatment that present significant
challenges. Actual results may differ materially from those
indicated by such forward-looking statements as a result of various
factors, including but not limited to: risks associated with our
financial condition and need for additional capital; risks
associated with our development work; cost and success of our
product development activities and clinical trials; the risks of
delay in the timing of our regulatory submissions or failure to
receive approval of our drug candidates; the risks related to
commercializing any approved pharmaceutical product including the
rate and degree of market acceptance of our product candidates;
development of our sales and marketing capabilities and risks
associated with failure to obtain sufficient reimbursement for our
products; the risks related to our dependence on third parties
including for conduct of clinical testing and product manufacture;
our inability to enter into partnerships; the risks related to
government regulation; risks related to market approval, risks
associated with protection of our intellectual property rights;
risks related to employee matters and managing growth; risks
related to our common stock and other risks and uncertainties
affecting the Company including those described in the "Risk
Factors" section included in our Annual Report on Form 10-K and in
our other SEC filings. Any forward-looking statements contained in
this press release speak only as of the date hereof, and the
Company undertakes no obligation to update any forward-looking
statement, whether as a result of new information, future events or
otherwise.
“DANYELZA” and “Y-mAbs” are registered
trademarks of Y-mAbs Therapeutics, Inc.
“Y-mAbs Connect” is a trademark of Y-mAbs
Therapeutics, Inc.
Contact:
Y-mAbs Therapeutics, Inc. 230 Park Avenue, Suite 3350New York,
NY 10169USA+1 646 885 8505E-mail: info@ymabs.com
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