Karyopharm Therapeutics Inc. (Nasdaq:KPTI), an oncology-focused
pharmaceutical company, today announced preliminary unaudited
fourth quarter and full year 2019 net product sales for XPOVIO®
(selinexor), the Company’s first-in-class, oral Selective Inhibitor
of Nuclear Export (SINE) compound, and provided additional updates
on the product’s commercial launch.
Strong XPOVIO Commercial Rollout
Continues in the U.S.
Oral XPOVIO tablets became commercially
available to patients in the U.S. on July 9, 2019. Based on
preliminary unaudited financial information, Karyopharm expects net
product sales of XPOVIO to be between $17 and $18 million during
the fourth quarter and between $30 and $31 million for the full
year 2019. As of December 31, 2019, approximately 1,400 XPOVIO
prescriptions have been fulfilled, driven by strong demand from
both academic and community-based oncologists. In less than 6
months on the market, XPOVIO has been prescribed by more than 550
unique physicians and healthcare accounts. These updates will be
discussed during a webcast presentation at the 38th Annual J.P.
Morgan Healthcare Conference in San Francisco on Tuesday, January
14, 2020 at 10:00 a.m. PT (1:00 p.m. ET). A live webcast of the
presentation and Q&A session can be accessed through the
investor section of the Company’s website at www.karyopharm.com.
Following the live presentation, a replay of the webcast will be
available on the Company’s website for 30 days.
“By all accounts, 2019 was a transformational
year for Karyopharm with the accelerated approval and commercial
launch of XPOVIO, the first and only oral nuclear export inhibitor
approved in the U.S., indicated for patients with heavily
pretreated multiple myeloma,” said Michael G. Kauffman, MD, PhD,
Chief Executive Officer of Karyopharm. “We are extremely pleased
with the commercial launch of XPOVIO thus far, including the
breadth of prescribing physicians and early feedback from patients
who have initiated therapy. We are proud to be making such an
important impact for patients who are battling relapsed or
refractory multiple myeloma.”
The Company intends to provide 2020 financial
guidance on non-GAAP R&D and SG&A expenses and year-end
cash balance in connection with the final financial results for the
fourth quarter and audited financial results for full year 2019
expected to be provided in February 2020.
The financial information presented in this
press release may be adjusted as a result of the completion of
customary quarterly review and audit procedures, and the Company’s
actual financial results may differ materially from the preliminary
estimated financial information set forth above.
About XPOVIO® (selinexor)
XPOVIO is a first-in-class, oral Selective
Inhibitor of Nuclear Export (SINE) compound. XPOVIO functions by
selectively binding to and inhibiting the nuclear export protein
exportin 1 (XPO1, also called CRM1). XPOVIO blocks the nuclear
export of tumor suppressor, growth regulatory and anti-inflammatory
proteins, leading to accumulation of these proteins in the nucleus
and enhancing their anti-cancer activity in the cell. The forced
nuclear retention of these proteins can counteract a multitude of
the oncogenic pathways that, unchecked, allow cancer cells with
severe DNA damage to continue to grow and divide in an unrestrained
fashion. In addition to receiving accelerated U.S. Food and Drug
Administration (FDA) approval of XPOVIO in July 2019 in combination
with dexamethasone for the treatment of adult patients with
relapsed refractory multiple myeloma (RRMM) who have received at
least four prior therapies and whose disease is refractory to at
least two proteasome inhibitors, at least two immunomodulatory
agents, and an anti-CD38 monoclonal antibody, Karyopharm has also
submitted a Marketing Authorization Application (MAA) to the
European Medicines Agency (EMA) with a request for conditional
approval of selinexor. A New Drug Application was recently
submitted to the FDA seeking accelerated approval for selinexor as
a new treatment for patients with relapsed or refractory diffuse
large B-cell lymphoma (DLBCL). Selinexor has received Fast Track
and Orphan designation from the FDA for the patient population
evaluated in the SADAL study. 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
liposarcoma (SEAL) and 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.
IMPORTANT SAFETY
INFORMATION
Thrombocytopenia
XPOVIO can cause thrombocytopenia, leading to
potentially fatal hemorrhage. Thrombocytopenia was reported as an
adverse reaction in 74% of patients, and severe (Grade 3-4)
thrombocytopenia occurred in 61% of patients treated with XPOVIO.
The median time to onset of the first event was 22 days. Bleeding
occurred in 23% of patients with thrombocytopenia, clinically
significant bleeding occurred in 5% of patients with
thrombocytopenia and fatal hemorrhage occurred in <1% of
patients.
Monitor platelet counts at baseline, during
treatment, and as clinically indicated. Monitor more frequently
during the first two months of treatment. Institute platelet
transfusion and/or other treatments as clinically indicated.
Monitor patients for signs and symptoms of bleeding and evaluate
promptly. Interrupt and/or reduce dose, or permanently discontinue
based on severity of adverse reaction.
Neutropenia
XPOVIO can cause neutropenia, potentially
increasing the risk of infection. Neutropenia was reported as an
adverse reaction in 34% of patients, and severe (Grade 3-4)
neutropenia occurred in 21% of patients treated with XPOVIO. The
median time to onset of the first event was 25 days. Febrile
neutropenia was reported in 3% of patients.
Obtain neutrophil counts at baseline, during
treatment, and as clinically indicated. Monitor more frequently
during the first two months of treatment. Monitor patients for
signs and symptoms of concomitant infection and evaluate promptly.
Consider supportive measures including antimicrobials for signs of
infection and use of growth factors (e.g., G-CSF). Interrupt and/or
reduce dose, or permanently discontinue based on severity of
adverse reaction.
Gastrointestinal Toxicity
Gastrointestinal toxicities occurred in patients
treated with XPOVIO.
Nausea/Vomiting
Nausea was reported as an adverse reaction in
72% of patients, and Grade 3 nausea occurred in 9% of patients
treated with XPOVIO. The median time to onset of the first nausea
event was 3 days.
Vomiting was reported in 41% of patients, and
Grade 3 vomiting occurred in 4% of patients treated with XPOVIO.
The median time to onset of the first vomiting event was 5
days.
Provide prophylactic 5-HT3 antagonists and/or
other anti-nausea agents, prior to and during treatment with
XPOVIO. Manage nausea/vomiting by dose interruption, reduction,
and/or discontinuation. Administer intravenous fluids and replace
electrolytes to prevent dehydration in patients at risk. Use
additional anti-nausea medications as clinically indicated.
Diarrhea
Diarrhea was reported as an adverse reaction in
44% of patients, and Grade 3 diarrhea occurred in 6% of patients
treated with XPOVIO. The median time to onset of diarrhea was 15
days.
Manage diarrhea by dose modifications and/or
standard anti-diarrheal agents; administer intravenous fluids to
prevent dehydration in patients at risk.
Anorexia/Weight Loss
Anorexia was reported as an adverse reaction in
53% of patients, and Grade 3 anorexia occurred in 5% of patients
treated with XPOVIO. The median time to onset of anorexia was 8
days.
Weight loss was reported as an adverse reaction
in 47% of patients, and Grade 3 weight loss occurred in 1% of
patients treated with XPOVIO. The median time to onset of weight
loss was 15 days.
Monitor patient weight at baseline, during
treatment, and as clinically indicated. Monitor more frequently
during the first two months of treatment. Manage anorexia and
weight loss with dose modifications, appetite stimulants, and
nutritional support.
Hyponatremia
XPOVIO can cause hyponatremia; 39% of patients
treated with XPOVIO experienced hyponatremia, 22% of patients
experienced Grade 3 or 4 hyponatremia. The median time to onset of
the first event was 8 days.
Monitor sodium level at baseline, during
treatment, and as clinically indicated. Monitor more frequently
during the first two months of treatment. Correct sodium levels for
concurrent hyperglycemia (serum glucose >150 mg/dL) and high
serum paraprotein levels. Treat hyponatremia per clinical
guidelines (intravenous saline and/or salt tablets), including
dietary review. Interrupt and/or reduce dose, or permanently
discontinue based on severity of adverse reaction.
Infections
In patients receiving XPOVIO, 52% of patients
experienced any grade of infection. Upper respiratory tract
infection of any grade occurred in 21%, pneumonia in 13%, and
sepsis in 6% of patients. Grade ≥3 infections were reported in 25%
of patients, and deaths resulting from an infection occurred in 4%
of patients. The most commonly reported Grade ≥3 infections were
pneumonia in 9% of patients, followed by sepsis in 6%. The median
time to onset was 54 days for pneumonia and 42 days for sepsis.
Most infections were not associated with neutropenia and were
caused by non-opportunistic organisms.
Neurological Toxicity
Neurological toxicities occurred in patients
treated with XPOVIO.
Neurological adverse reactions including
dizziness, syncope, depressed level of consciousness, and mental
status changes (including delirium and confusional state) occurred
in 30% of patients, and severe events (Grade 3-4) occurred in 9% of
patients treated with XPOVIO. Median time to the first event was 15
days.
Optimize hydration status, hemoglobin level, and
concomitant medications to avoid exacerbating dizziness or mental
status changes.
Embryo-Fetal Toxicity
Based on data from animal studies and its
mechanism of action, XPOVIO can cause fetal harm when administered
to a pregnant woman. Selinexor administration to pregnant animals
during organogenesis resulted in structural abnormalities and
alterations to growth at exposures below those occurring clinically
at the recommended dose.
Advise pregnant women of the potential risk to a
fetus. Advise females of reproductive potential and males with a
female partner of reproductive potential to use effective
contraception during treatment with XPOVIO and for 1 week after the
last dose.
ADVERSE REACTIONS
The most common adverse reactions (incidence
≥20%) are thrombocytopenia, fatigue, nausea, anemia, decreased
appetite, decreased weight, diarrhea, vomiting, hyponatremia,
neutropenia, leukopenia, constipation, dyspnea, and upper
respiratory tract infection.
The treatment discontinuation rate due to
adverse reactions was 27%; 53% of patients had a reduction in the
XPOVIO dose, and 65.3% had the dose of XPOVIO interrupted. The most
frequent adverse reactions requiring permanent discontinuation in
4% or greater of patients who received XPOVIO included fatigue,
nausea, and thrombocytopenia. The rate of fatal adverse reactions
was 8.9%.
Please see XPOVIO Full Prescribing
Information available at www.XPOVIO.com.
About Karyopharm
Therapeutics
Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is
an oncology-focused pharmaceutical company dedicated to the
discovery, development, and commercialization of novel
first-in-class drugs directed against nuclear export 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). Karyopharm’s lead
compound, XPOVIO® (selinexor), received accelerated approval from
the FDA in July 2019 in combination with dexamethasone as a
treatment for patients with heavily pretreated multiple myeloma. An
MAA for selinexor is also currently under review by the EMA for the
same indication. The Company recently submitted a New Drug
Application to the FDA seeking approval for XPOVIO in patients with
DLBCL. 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
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 Karyopharm’s preliminary financial information for fourth
quarter and full year 2019; Karyopharm’s plans to provide guidance
on its 2020 non-GAAP R&D and SG&A expenses and year-end
cash balance; expectations relating to XPOVIO for the treatment of
patients with heavily pretreated multiple myeloma or relapsed or
refractory diffuse large B-cell lymphoma; commercialization of
XPOVIO or any of its drug candidates and the commercial performance
of XPOVIO; submissions to, and the review and potential approval of
selinexor by, regulatory authorities, including the anticipated
availability of data to support such submissions, timing of such
submissions and actions by regulatory authorities and the potential
availability of accelerated approval pathways; and the therapeutic
potential of and potential clinical development plans for
Karyopharm's drug candidates, especially 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; that regulators
will agree that selinexor qualifies for conditional approval in the
E.U. as a result of data from the STORM study or confirmatory
approval in the U.S. or EU based on the BOSTON study in patients
with relapsed or refractory multiple myeloma, or accelerated
approval in the U.S. for patients with relapsed or refractory DLBCL
as a result of data from the SADAL study, 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 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: items that may be identified during Karyopharm’s
financial statement closing process that cause adjustments to the
estimates included in this press release; 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 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 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, 2019, which was filed with the Securities and
Exchange Commission (SEC) on November 4, 2019, 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
Karp, Vice President, Investor and Public Relations857-297-2241 |
ikarp@karyopharm.com
Media:FTI ConsultingSimona Kormanikova or Robert
Stanislaro212-850-5600 |Simona.Kormanikova@fticonsulting.com or
robert.stanislaro@fticonsulting.com
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