– Regulatory Designation Includes Primary
Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis and
Post-Polycythemia Vera Myelofibrosis –
– Pivotal Phase 3 Study of Selinexor and
Ruxolitinib in Treatment-Naïve Myelofibrosis Initiated in
June 2023 –
NEWTON,
Mass., July 17, 2023 /PRNewswire/ -- Karyopharm
Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical
company pioneering novel cancer therapies, today announced that the
United States Food and Drug Administration (FDA) has granted Fast
Track Designation to the development program of selinexor for the
treatment of patients with myelofibrosis, including primary
myelofibrosis, post-essential thrombocythemia myelofibrosis, and
post-polycythemia vera myelofibrosis.
"Fast Track Designation for selinexor highlights its potential
to address the unmet medical need in myelofibrosis, an important
acknowledgement as we continue our pivotal Phase 3 study," said
Reshma Rangwala, MD, PhD, Chief
Medical Officer of Karyopharm. "Selinexor's unique mechanism of
action, XPO1 inhibition, is a novel and potentially fundamental
mechanism in myelofibrosis. We have been highly encouraged by the
efficacy and safety data observed to date [in our Phase 1 study]
with selinexor in combination with ruxolitinib in patients with
treatment-naïve myelofibrosis and believe selinexor has the
potential to shift the treatment paradigm. We look forward to
continued interaction with the FDA as we advance the development of
this promising treatment for patients in need."
In June 2023, Karyopharm initiated
a pivotal Phase 3 clinical trial (XPORT-MF-034) (NCT04562389) to
assess the efficacy and safety of once-weekly selinexor 60 mg in
combination with ruxolitinib in JAKi-naïve patients with
myelofibrosis. Updated data from the Phase 1 study were
presented at the American Association for Cancer Research
Annual Meeting 2023, American Society of Clinical Oncology 2023 and
European Hematology Association 2023, which showed rapid, deep and
sustained spleen responses and robust symptom improvement in
patients treated with selinexor 60 mg in combination with
ruxolitinib as of the April 10, 2023
cut-off date. Top-line data from the Phase 3 study is
expected in 2025. The Company plans to expand its clinical
development program in myelofibrosis by investigating selinexor in
other JAKi-naïve settings, such as novel combinations, to benefit
the greatest number of patients.
Fast Track Designation is intended to facilitate development and
expedite review of drugs to treat serious and life-threatening
conditions so that an approved product can reach the market
expeditiously. Features of Fast Track Designation include frequent
interactions with the FDA review team, and if relevant criteria are
met, eligibility for Priority Review and Rolling Review.
Further information about the Phase 3 study can be found at
www.clinicaltrials.gov.
About Karyopharm Therapeutics
Karyopharm Therapeutics
Inc. (Nasdaq: KPTI) is a commercial-stage pharmaceutical company
pioneering novel cancer therapies. Since its founding, Karyopharm
has been an industry leader in oral Selective Inhibitor of Nuclear
Export (SINE) compound technology, which was developed to address a
fundamental mechanism of oncogenesis: nuclear export dysregulation.
Karyopharm's lead SINE compound and first-in-class, oral exportin 1
(XPO1) inhibitor, XPOVIO® (selinexor), is approved in the U.S. and
marketed by the Company in three oncology indications and has
received regulatory approvals in various indications in a growing
number of ex-U.S. territories and countries, including Europe and the United Kingdom (as NEXPOVIO®) and China. Karyopharm has a focused pipeline
targeting multiple high unmet need cancer indications, including in
multiple myeloma, endometrial cancer, myelodysplastic neoplasms and
myelofibrosis. For more information about our people, science and
pipeline, please visit www.karyopharm.com, and follow us on Twitter
at @Karyopharm and LinkedIn.
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 the ability of selinexor to
treat patients with myelofibrosis; and expectations related to the
clinical development of selinexor and potential regulatory
submissions of selinexor. 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
Karyopharm will successfully commercialize XPOVIO or that any of
Karyopharm's drug candidates, including selinexor and eltanexor,
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 the development or commercialization of
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: the adoption of XPOVIO in the
commercial marketplace, the timing and costs involved in
commercializing XPOVIO or any of Karyopharm's drug candidates that
receive regulatory approval; the ability to obtain and retain
regulatory approval of XPOVIO or any of Karyopharm's drug
candidates that receive regulatory approval; 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; the ability of Karyopharm or its third
party collaborators or successors in interest to fully perform
their respective obligations under the applicable agreement and the
potential future financial implications of such agreement;
Karyopharm's ability to enroll patients in its clinical trials;
unplanned cash requirements and expenditures; development or
regulatory approval of drug candidates by Karyopharm's competitors
for products or product candidates in which Karyopharm is currently
commercializing or developing; the direct or indirect impact of the
COVID-19 pandemic or any future pandemic on Karyopharm's business,
results of operations and financial condition; and Karyopharm's
ability to obtain, maintain and enforce patent and other
intellectual property protection for any of its products or product
candidates. These and other risks are described under the caption
"Risk Factors" in Karyopharm's Quarterly Report on Form 10-Q for
the quarter ended March 31, 2023,
which was filed with the Securities and Exchange Commission (SEC)
on May 4, 2023, 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.
XPOVIO® and NEXPOVIO® are registered trademarks of Karyopharm
Therapeutics Inc. Any other trademarks referred to in this release
are the property of their respective owners.
View original content to download
multimedia:https://www.prnewswire.com/news-releases/karyopharm-receives-fda-fast-track-designation-for-selinexor-for-the-treatment-of-myelofibrosis-301878818.html
SOURCE Karyopharm Therapeutics Inc.