Karyopharm Announces Submission of Marketing Authorization Application to the European Medicines Agency for Selinexor for th...
09 January 2019 - 4:20AM
Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage
pharmaceutical company, today announced that it has submitted a
Marketing Authorization Application (MAA) to the European Medicines
Agency (EMA) for selinexor, the Company’s first-in-class, oral
Selective Inhibitor of Nuclear Export (SINE) compound, requesting
conditional approval for the treatment of patients with relapsed or
refractory multiple myeloma (MM) who have received at least three
prior lines of therapy and whose disease is refractory to at least
one proteasome inhibitor (PI), one immunomodulatory agent (IMiD),
and one anti-CD38 monoclonal antibody (mAb), and to their most
recent treatment regimen (penta-refractory MM). Karyopharm
also announced that the selinexor MAA has been granted accelerated
assessment by the EMA’s Committee for Medicinal Products for Human
Use (CHMP).
“The MAA submission for selinexor is an
important milestone for Karyopharm and the CHMP’s granting of
accelerated assessment further underscores the urgent need to
improve outcomes for patients with highly refractory multiple
myeloma,” said Sharon Shacham, PhD, MBA, Founder, President and
Chief Scientific Officer of Karyopharm. “The results from the
pivotal Phase 2b STORM study provide compelling evidence that
selinexor in combination with low-dose dexamethasone has the
potential to be an effective new treatment option for patients with
this difficult to treat disease. With the filing of the MAA
and an accelerated assessment designation from the EMA, we hope to
make oral selinexor available as quickly as possible to patients
throughout Europe.”
An accelerated assessment is granted to products
deemed by the CHMP to be of major interest for public health and
represent therapeutic innovation. Accelerated assessments can
reduce the active review time of an MAA from the standard 210 days
down to 150 days once it has been validated by the EMA.
Selinexor has also previously received orphan designation in
multiple myeloma from the EMA.
A New Drug Application (NDA) seeking accelerated
approval for oral selinexor with low dose dexamethasone as a
treatment for patients with penta-refractory multiple myeloma is
under Priority Review by the U.S. Food and Drug Administration
(FDA) with an action date of April 6, 2019, under the Prescription
Drug User-Fee Act (PDUFA).
About Selinexor
Selinexor is a first-in-class, oral Selective
Inhibitor of Nuclear Export (SINE) compound. Selinexor functions by
binding with and inhibiting the nuclear export protein XPO1 (also
called CRM1), leading to the accumulation of tumor suppressor
proteins in the cell nucleus. This reinitiates and amplifies their
tumor suppressor function and is believed to lead to the selective
induction of apoptosis in cancer cells, while largely sparing
normal cells. In 2018, Karyopharm reported positive data from the
Phase 2b STORM study evaluating selinexor in combination with
low-dose dexamethasone in patients with penta-refractory multiple
myeloma. Selinexor has been granted Orphan Drug Designation in
multiple myeloma and Fast Track designation for the patient
population evaluated in the STORM study. Karyopharm’s New Drug
Application (NDA) has been accepted for filing and granted Priority
Review by the FDA, and oral selinexor is currently under review by
the FDA as a possible new treatment for patients with
penta-refractory multiple myeloma. The Company has also submitted a
Marketing Authorization Application (MAA) to the European Medicines
Agency (EMA) with a request for conditional approval and was
granted accelerated assessment. Selinexor is also being evaluated
in several other mid-and later-phase clinical trials across
multiple cancer indications, including in multiple myeloma in a
pivotal, randomized Phase 3 study in combination with Velcade®
(bortezomib) and low-dose dexamethasone (BOSTON), as a potential
backbone therapy in combination with approved therapies (STOMP), in
diffuse large B-cell lymphoma (SADAL), liposarcoma (SEAL), and an
investigator-sponsored study in endometrial cancer (SIENDO), among
others. Additional Phase 1, Phase 2 and Phase 3 studies are ongoing
or currently planned, including multiple studies in combination
with approved therapies in a variety of tumor types to further
inform Karyopharm's clinical development priorities for selinexor.
Additional clinical trial information for selinexor is available at
www.clinicaltrials.gov.
About Karyopharm
Therapeutics
Karyopharm Therapeutics Inc. (Nasdaq:KPTI) is a
clinical-stage pharmaceutical company focused on the discovery and
development of novel first-in-class drugs directed against nuclear
transport and related targets for the treatment of cancer and other
major diseases. Karyopharm's SINE compounds function by binding
with and inhibiting the nuclear export protein XPO1 (or CRM1). In
addition to single-agent and combination activity against a variety
of human cancers, SINE compounds have also shown biological
activity in models of neurodegeneration, inflammation, autoimmune
disease, certain viruses and wound-healing. Karyopharm, which was
founded by Dr. Sharon Shacham, currently has several
investigational programs in clinical or preclinical development.
For more information, please visit www.karyopharm.com.
Forward-Looking Statements
This press release contains forward-looking
statements within the meaning of The Private Securities Litigation
Reform Act of 1995. Such forward-looking statements include those
regarding our expectations relating to submissions and to the
review and potential approval of selinexor by regulatory
authorities, including the anticipated timing of such submissions
and actions, and the potential availability of accelerated approval
pathways, the therapeutic potential of and potential clinical
development plans for Karyopharm's drug candidates, especially
selinexor, and the plans for commercialization. Such statements are
subject to numerous important factors, risks and uncertainties,
many of which are beyond Karyopharm’s control, that may cause
actual events or results to differ materially from Karyopharm's
current expectations. For example, there can be no guarantee that
regulators will agree that selinexor qualifies for accelerated
approval in the U.S. or conditional approval in the E.U. as a
result of the data from the STORM study in patients with
penta-refractory myeloma or the SADAL study in patients with
relapsed or refractory DLBCL or that any of Karyopharm's drug
candidates, including selinexor, will successfully complete
necessary clinical development phases or that development of any of
Karyopharm's drug candidates will continue. Further, there can be
no guarantee that any positive developments in Karyopharm's drug
candidate portfolio will result in stock price appreciation.
Management's expectations and, therefore, any forward-looking
statements in this press release could also be affected by risks
and uncertainties relating to a number of other factors, including
the following: Karyopharm's results of clinical trials and
preclinical studies, including subsequent analysis of existing data
and new data received from ongoing and future studies; the content
and timing of decisions made by the U.S. Food and Drug
Administration and other regulatory authorities, investigational
review boards at clinical trial sites and publication review
bodies, including with respect to the need for additional clinical
studies; Karyopharm's ability to obtain and maintain requisite
regulatory approvals and to enroll patients in its clinical trials;
unplanned cash requirements and expenditures; development of drug
candidates by Karyopharm's competitors for diseases in which
Karyopharm is currently developing its drug candidates; and
Karyopharm's ability to obtain, maintain and enforce patent and
other intellectual property protection for any drug candidates it
is developing. These and other risks are described under the
caption "Risk Factors" in Karyopharm's Quarterly Report on Form
10-Q for the quarter ended September 30, 2018, which was filed with
the Securities and Exchange Commission (SEC) on November 8, 2018,
and in other filings that Karyopharm may make with the SEC in the
future. Any forward-looking statements contained in this press
release speak only as of the date hereof, and, except as required
by law, Karyopharm expressly disclaims any obligation to update any
forward-looking statements, whether as a result of new information,
future events or otherwise.
Velcade® is a registered trademark of Takeda
Pharmaceutical Company Limited
Contacts:
Investors:Karyopharm
Therapeutics Inc.Ian KarpVice President, Investor and Public
Relations857-297-2241 | ikarp@karyopharm.com
Media:Argot PartnersDavid
Rosen212-600-1902 | david.rosen@argotpartners.com
Karyopharm Therapeutics (NASDAQ:KPTI)
Historical Stock Chart
From Apr 2024 to May 2024
Karyopharm Therapeutics (NASDAQ:KPTI)
Historical Stock Chart
From May 2023 to May 2024