NEWTON, Mass., Nov. 2, 2020 /PRNewswire/ -- Karyopharm
Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical
company pioneering novel cancer therapies, today reported financial
results for the quarter ended September 30,
2020. In addition, Karyopharm highlighted select corporate
milestones, including details regarding the ongoing U.S.
commercialization of XPOVIO® (selinexor), and provided an overview
of its key clinical development programs.
"We are delighted to share the significant top-line results from
the Phase 3 portion of the SEAL study, the first, late-stage
clinical data for XPOVIO in a solid tumor indication," said
Sharon Shacham, PhD, MBA, President
and Chief Scientific Officer of Karyopharm. "The top-line results
from the SEAL study are particularly encouraging as advanced
dedifferentiated liposarcoma represents a very difficult to treat
cancer with no established standard of care and limited treatment
options available to patients. XPOVIO may be particularly promising
as it represents the first oral therapy to show activity in
patients with previously treated liposarcoma. We look forward to
presenting the detailed results at the upcoming Connective Tissue
Oncology Society (CTOS) Annual Meeting and plan to submit a New
Drug Application (NDA) to the U.S. Food and Drug Administration
(FDA) in the first quarter of 2021 requesting approval of XPOVIO to
treat the patient population studied in SEAL. If approved, XPOVIO
would represent the first oral, non-chemotherapy agent available
for patients with dedifferentiated liposarcoma. The encouraging
data from the SEAL study also provide additional rationale for
advancing the clinical development of XPOVIO in other solid tumor
indications, including in endometrial, glioblastoma, lung and other
cancers where Karyopharm is currently conducting clinical
studies."
Commenting on additional milestones achieved in the third
quarter of 2020, Michael G.
Kauffman, MD, PhD, Chief Executive Officer of Karyopharm
added, "Karyopharm achieved another strong quarter for XPOVIO
sales, which grew approximately 15% compared to the second quarter
of 2020. Sales growth was driven primarily by an increase in new
multiple myeloma and diffuse large B-cell lymphoma (DLBCL) patient
starts. Looking forward, we plan to continue our ongoing support
for our supplemental New Drug Application (sNDA) seeking approval
for XPOVIO as a treatment for patients with multiple myeloma after
at least one prior line of therapy, for which the FDA has assigned
a target Prescription Drug User Fee Act (PDUFA) action date of
March 19, 2021."
Third Quarter 2020 and Recent Highlights
XPOVIO in Multiple Myeloma and DLBCL
- XPOVIO U.S. Commercialization. Oral XPOVIO became
commercially available to patients with penta-refractory multiple
myeloma in July 2019 and to patients
with relapsed or refractory DLBCL in June
2020. During the third quarter of 2020, XPOVIO generated net
product sales of $21.3 million,
representing a 15% increase compared to the second quarter of 2020.
XPOVIO sales growth was primarily driven by an increase in new
multiple myeloma patient starts compared to the second quarter of
2020. XPOVIO sales also benefited from the initial commercial
launch in patients with relapsed or refractory DLBCL. In the third
quarter of 2020, approximately 1,100 XPOVIO prescriptions were
filled, which represented the highest quarterly level to date and
was 15% higher than in the second quarter of 2020. Over 200 new
physician prescribing accounts were added in the third quarter of
2020, which included both myeloma and DLBCL treating physicians.
Finally, based on data from specialty pharmacies, prescription
refill rates for XPOVIO continued to grow with the average number
of prescriptions per patient reaching 2.9 by the end of
September 2020, compared to 2.0 at
the end of December 2019.
- FDA Accepts sNDA Seeking Expanded Indication For Patients
with Previously Treated Multiple Myeloma. The FDA assigned a
PDUFA action date of March 19, 2021
for Karyopharm's sNDA seeking approval for XPOVIO for the treatment
of adult patients with multiple myeloma after at least one line of
prior therapy. If approved, the Company expects to launch the
expanded indication immediately thereafter. The sNDA is supported
by the positive data from the pivotal Phase 3 BOSTON study, which
was reported at the American Society of Clinical Oncology (ASCO)
2020 annual meeting. The BOSTON
study evaluated once-weekly XPOVIO in combination with once-weekly
Velcade® (bortezomib) and low-dose dexamethasone (SVd) compared to
standard twice-weekly Velcade plus low-dose dexamethasone (Vd) in
patients with multiple myeloma who have received one to three prior
lines of therapy. The BOSTON study
met its primary endpoint with a significant increase in median
progression-free survival (PFS) in patients with multiple myeloma
following one to three prior lines of therapy. The median PFS in
the SVd arm was 13.93 months compared to 9.46 months in the Vd arm,
representing a 4.47 month (47%) increase in median PFS (hazard
ratio=0.70; p=0.0075). There were no new safety signals on the SVd
arm, and deaths were numerically lower on the SVd arm (N=47) as
compared with the Vd arm (N=62).
- Regulatory Strategy Update in Europe. In January
2019, Karyopharm submitted a Marketing Authorization
Application (MAA) to the European Medicines Agency (EMA) requesting
conditional approval for XPOVIO in combination with dexamethasone
as a treatment for patients with heavily pretreated multiple
myeloma based on the results of the Phase 2b STORM study. In January
2020, Karyopharm was granted a three-month extension from
the EMA's Committee for Medicinal Products for Human Use (CHMP) to
provide additional time to respond to the CHMP's outstanding
questions, primarily related to re-monitoring certain clinical
data. Due to the COVID-19 pandemic and the resulting disruption at
many clinical sites, re-monitoring activities requested by CHMP
delayed the review timelines in Europe. Karyopharm submitted the final
requested re-monitoring data in September
2020 and in October 2020,
Karyopharm received a further updated list of outstanding issues
from the CHMP summarizing the remaining topics for Karyopharm to
address and indicating that the CHMP intends to consult its
Scientific Advisory Group for additional advice [in the fourth
quarter of 2020]. Karyopharm continues to expect to receive an
opinion from CHMP with respect to the MAA before the end of 2020.
In addition, contingent upon and following receipt of CHMP's
opinion, we expect to submit a MAA based on data from the
BOSTON study before the end of
2020.
XPOVIO in Development for Solid Tumors
- Twice-Weekly XPOVIO Demonstrates a Statistically Significant
Reduction in the Risk of Disease Progression or Death Compared to
Placebo; Hazard Ratio=0.70, p=0.023. In November 2020, Karyopharm announced positive
top-line results from the Phase 3 portion of the randomized, double
blind, placebo controlled, cross-over, SEAL study evaluating single
agent, oral XPOVIO versus placebo in patients with advanced
unresectable dedifferentiated liposarcoma. The SEAL study met its
primary endpoint of a statistically significant increase in PFS;
hazard ratio=0.70; p=0.023. Among those patients who received
XPOVIO, there was a trend towards an improvement in the median
overall survival compared to those patients who began on the
placebo arm of the study and never crossed over to the XPOVIO
treatment arm. The safety profile for XPOVIO was consistent with
previous clinical studies with fewer hematologic and infectious
adverse events as compared to XPOVIO studies in patients with
multiple myeloma and DLBCL. The clinical data from this study have
been selected for an oral presentation at the CTOS Annual Meeting
on November 20, 2020 at 10:30 AM ET. Additionally, Karyopharm intends to
use the data from the SEAL study to submit an NDA to the FDA in the
first quarter of 2021 requesting approval of XPOVIO to treat the
patient population studied in SEAL.
- Clinical Data from Advanced Solid Tumor Studies Presented at
the European Society for Medical Oncology (ESMO) Virtual
Congress. In September 2020,
encouraging clinical data from investigator-sponsored studies
evaluating XPOVIO in combination with established cancer therapies
were presented at ESMO. Clinical data from XPOVIO studies included:
(i) combination data with pembrolizumab for the treatment of
melanoma, (ii) combination data with carboplatin and paclitaxel for
the treatment of advanced or metastatic solid tumors, and (iii)
combination data with topotecan for the treatment of advanced or
metastatic solid tumors. The Company believes that the encouraging
results from these combination studies warrant further research
into the potential utility of XPOVIO in solid tumors and will help
Karyopharm further prioritize future clinical development
activities.
Low Dose Selinexor in Development for COVID-19
- Presented Phase 2 Data at COVID-19 Focused Medical
Meeting. Selinexor data was highlighted recently in an oral
presentation at the International Society for Influenza and Other
Respiratory Virus Diseases Antiviral Group (ISIRV-AVG) Virtual
Conference on Therapeutics for COVID-19. While the results of the
Phase 2 study demonstrated encouraging anti-viral and
anti-inflammatory activity in an important subset of treated
patients, the trial was discontinued following an interim analysis
which indicated that the trial was unlikely to meet its
pre-specified primary endpoint. While the FDA's opinion was that
the benefit-risk ratio for this study was not favorable, Karyopharm
is encouraged by the potential mechanistic activity of XPO1
inhibition and believes these results warrant further research of
XPO1 inhibitors in COVID-19 and other infectious diseases.
Corporate Updates
- Sharon Shacham, Winner of EY
Entrepreneur of the Year® 2020 New England Award. In
October 2020, Karyopharm's founder,
Dr. Sharon Shacham, was selected as
a winner in the EY Entrepreneur of the Year 2020 New England Awards
Program. For more than 30 years, this award has served as one of
the world's most prestigious business awards recognizing
entrepreneurs who have disrupted industries, created new product
categories and successfully brought innovations that have
transformed our world. Dr. Shacham was recognized for her
scientific research that led to the development and FDA approval of
XPOVIO, as well as for leading Karyopharm from its inception to
what is now a global pharmaceutical company focused on the
discovery, development, and commercialization of novel medicines
for patients with cancer and other major diseases.
- Christy J. Oliger Appointed to the Board. In
August 2020, Karyopharm appointed
Christy J. Oliger to its Board of
Directors. Ms. Oliger is the former Senior Vice President of the
Oncology Business Unit at Genentech, a leading biotechnology
company dedicated to pursuing groundbreaking science to discover
and develop medicines for people with serious and life-threatening
diseases. Ms. Oliger served in a wide variety of commercial
leadership positions at Genentech from 2000 until 2020. Ms.
Oliger's strategic expertise and counsel will be critical to
Karyopharm as it continues to expand the global development and
commercialization of XPOVIO into new patient populations and
potential new indications.
Third Quarter 2020 Financial Results
Net product revenue: Net product revenue for the third
quarter of 2020 was $21.3 million,
compared to $12.8 million for the
third quarter of 2019.
License and other revenue: License and other revenue for
the third quarter of 2020 was negligible, compared to $0.3 million for the third quarter of 2019.
Cost of sales: Cost of sales totaled $0.4 million for the third quarter of 2020,
compared to $1.0 million for the
third quarter of 2019. Cost of sales reflects the costs of XPOVIO
units sold and third-party royalties on net product revenue.
Research and development (R&D) expenses: R&D
expenses for the third quarter of 2020 were $37.0 million, compared to $26.3 million for the third quarter of 2019. The
increase in R&D expenses compared to the third quarter of last
year was primarily attributable to COVID-19 trial activity and
continued activity in our other ongoing clinical trials.
Selling, general and administrative (SG&A) expenses:
For the third quarter of 2020, SG&A expenses were $31.0 million, compared to $25.3 million for the third quarter of 2019. The
increase in SG&A expenses compared to the third quarter of last
year was due primarily to activities to support the U.S.
commercialization of XPOVIO, including the launch of XPOVIO as a
treatment for patients with relapsed or refractory DLBCL.
Interest expense: Interest expense for the third quarter
of 2020 was $6.8 million, compared to
$3.1 million for the third quarter of
2019. The increase in interest expense was primarily attributable
to the imputed interest on the deferred royalty obligation
Karyopharm has with HealthCare Royalty Partners.
Net loss: Karyopharm reported a net loss of $53.5 million, or $0.73 per share, for the third quarter of 2020,
compared to a net loss of $41.4
million, or $0.67 per share,
for the third quarter of 2019. Net loss included non-cash
stock-based compensation expense of $6.5
million and $3.7 million for
the third quarters of 2020 and 2019, respectively.
Cash position: Cash, cash equivalents, restricted cash
and investments as of September 30,
2020 totaled $304.2 million,
compared to $265.8 million as of
December 31, 2019.
2020 Financial Outlook
Based on its current operating plans, Karyopharm continues to
expect its non-GAAP R&D and SG&A expenses, which excludes
stock-based compensation expense, for the full year 2020 to be in
the range of $240.0 million to
$260.0 million. Karyopharm has not
reconciled the full year 2020 outlook for non-GAAP R&D and
SG&A expenses to full year 2020 outlook for GAAP R&D and
SG&A expenses because Karyopharm cannot reliably predict
without unreasonable efforts the timing or amount of the factors
that substantially contribute to the projection of stock
compensation expense, which is excluded from the full year 2020
outlook for non-GAAP R&D and SG&A expenses.
The Company expects that its existing cash, cash equivalents and
investments, and the revenue it expects to generate from XPOVIO
product sales, will be sufficient to fund its planned operations
into the second half of 2022.
Non-GAAP Financial Information
Karyopharm uses a non-GAAP financial measure, including R&D
and SG&A expenses, to provide operating expense guidance.
Non-GAAP R&D and SG&A expenses exclude stock-based
compensation expense. Karyopharm believes this non-GAAP financial
measure is useful to investors because it provides greater
transparency regarding Karyopharm's operating performance as it
excludes non-cash stock compensation expense. This non-GAAP
financial measure should not be considered a substitute or an
alternative to GAAP R&D and SG&A expenses and should not be
considered a measure of Karyopharm's liquidity. Instead, non-GAAP
R&D and SG&A expenses should only be used to supplement an
understanding of Karyopharm's operating results as reported under
GAAP.
Conference Call Information
Karyopharm will host a conference call today, Monday, November 2, 2020, at 4:30 p.m. Eastern Time, to discuss the third
quarter 2020 financial results, recent accomplishments, clinical
developments and business plans. To access the conference call,
please dial (877) 870-4263 (local) or (412) 317-0790
(international) at least 10 minutes prior to the start time and ask
to be joined into the Karyopharm Therapeutics call. A live audio
webcast of the call will be available under "Events &
Presentations" in the Investor section of the Company's website,
http://investors.karyopharm.com/events-presentations. An archived
webcast will be available on the Company's website approximately
two hours after the event.
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. Karyopharm
received 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 in this same RRMM indication. Karyopharm's
supplemental New Drug Application (sNDA) requesting an expansion of
its current indication to include the treatment for patients with
multiple myeloma after at least one prior line of therapy has been
accepted for filing by the FDA. In June
2020, Karyopharm received accelerated FDA approval of XPOVIO
for its second indication in adult patients with relapsed or
refractory diffuse large B-cell lymphoma (DLBCL), not otherwise
specified, including DLBCL arising from follicular lymphoma, after
at least 2 lines of systemic therapy. Selinexor is also being
evaluated in several other mid-and later-phase clinical trials
across multiple cancer indications, including as a potential
backbone therapy in combination with approved myeloma 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.
For more information about Karyopharm's products or clinical
trials, please contact the Medical Information department at:
Tel: +1 (888) 209-9326
Email: medicalinformation@karyopharm.com
IMPORTANT SAFETY INFORMATION
Thrombocytopenia: XPOVIO can cause life-threatening
thrombocytopenia, potentially leading to hemorrhage.
Thrombocytopenia was reported in patients with multiple myeloma
(MM) and developed or worsened in patients with DLBCL.
Thrombocytopenia is the leading cause of dosage modifications.
Monitor platelet counts at baseline and throughout treatment.
Monitor more frequently during the first 3 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, reduce dose, or
permanently discontinue based on severity of adverse reaction.
Neutropenia: XPOVIO can cause life-threatening
neutropenia, potentially increasing the risk of infection.
Neutropenia and febrile neutropenia occurred in patients with MM
and in patients with DLBCL.
Obtain white blood cell counts with differential at baseline and
throughout treatment. Monitor more frequently during the first 3
months of treatment. Monitor patients for signs and symptoms of
concomitant infection and evaluate promptly. Consider supportive
measures, including antimicrobials and growth factors (e.g.,
G-CSF). Interrupt, reduce dose, or permanently discontinue based on
severity of adverse reaction (AR).
Gastrointestinal Toxicity: XPOVIO can cause severe
gastrointestinal toxicities in patients with MM and DLBCL.
Nausea/Vomiting: Provide prophylactic antiemetics.
Administer 5-HT3 receptor antagonists and other anti-nausea agents
prior to and during treatment with XPOVIO. Interrupt, reduce dose,
or permanently discontinue based on severity of ARs. Administer
intravenous fluids to prevent dehydration and replace electrolytes
as clinically indicated.
Diarrhea: Interrupt, reduce dose, or permanently
discontinue based on severity of ARs. Provide standard
anti-diarrheal agents, administer intravenous fluids to prevent
dehydration, and replace electrolytes as clinically indicated.
Anorexia/Weight Loss: Monitor weight, nutritional
status, and volume status at baseline and throughout treatment.
Monitor more frequently during the first 3 months of treatment.
Interrupt, reduce dose, or permanently discontinue based on
severity of ARs. Provide nutritional support, fluids, and
electrolyte repletion as clinically indicated.
Hyponatremia: XPOVIO can cause severe or
life-threatening hyponatremia. Hyponatremia developed in patients
with MM and in patients with DLBCL.
Monitor sodium level at baseline and throughout treatment.
Monitor more frequently during the first 2 months of treatment.
Correct sodium levels for concurrent hyperglycemia (serum glucose
>150 mg/dL) and high serum paraprotein levels. Assess hydration
status and manage hyponatremia per clinical guidelines, including
intravenous saline and/or salt tablets as appropriate and dietary
review. Interrupt, reduce dose, or permanently discontinue based on
severity of the AR.
Serious Infection: XPOVIO can cause
serious and fatal infections. Most infections were not associated
with Grade 3 or higher neutropenia. Atypical infections reported
after taking XPOVIO include, but are not limited to, fungal
pneumonia and herpesvirus infection.
Monitor for signs and symptoms of infection, and evaluate and
treat promptly.
Neurological Toxicity: XPOVIO can cause
life-threatening neurological toxicities.
Coadministration of XPOVIO with other products that cause
dizziness or mental status changes may increase the risk of
neurological toxicity.
Advise patients to refrain from driving and engaging in
hazardous occupations or activities, such as operating heavy or
potentially dangerous machinery, until the neurological toxicity
fully resolves. Optimize hydration status, hemoglobin level, and
concomitant medications to avoid exacerbating dizziness or mental
status changes. Institute fall precautions as appropriate.
Embryo-Fetal Toxicity: XPOVIO can cause fetal harm
when administered to a pregnant woman.
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 (ARs) in ≥20% of patients with
MM are thrombocytopenia, fatigue, nausea, anemia, decreased
appetite, decreased weight, diarrhea, vomiting, hyponatremia,
neutropenia, leukopenia, constipation, dyspnea, and upper
respiratory tract infection.
The most common ARs, excluding laboratory abnormalities, in ≥20%
of patients with DLBCL are fatigue, nausea, diarrhea, appetite
decrease, weight decrease, constipation, vomiting, and pyrexia.
Grade 3-4 laboratory abnormalities in ≥15% of patients included
thrombocytopenia, lymphopenia, neutropenia, anemia, and
hyponatremia. Grade 4 laboratory abnormalities in ≥5% were
thrombocytopenia, lymphopenia, and neutropenia.
In patients with MM, fatal ARs occurred in 9% of patients.
Serious ARs occurred in 58% of patients. Treatment discontinuation
rate due to ARs was 27%. The most frequent ARs requiring permanent
discontinuation in ≥4% of patients included fatigue, nausea, and
thrombocytopenia.
In patients with DLBCL, fatal ARs occurred in 3.7% of patients
within 30 days, and 5% of patients within 60 days of last
treatment; the most frequent fatal AR was infection (4.5% of
patients). Serious ARs occurred in 46% of patients; the most
frequent serious AR was infection. Discontinuation due to ARs
occurred in 17% of patients.
USE IN SPECIFIC POPULATIONS
In MM, no overall difference in effectiveness of XPOVIO was
observed in patients >65 years old when compared with younger
patients. Patients ≥75 years old had a higher incidence of
discontinuation due to an AR than younger patients, a higher
incidence of serious ARs, and a higher incidence of fatal ARs.
Clinical studies in patients with relapsed or refractory DLBCL
did not include sufficient numbers of patients aged 65 and over to
determine whether they respond differently from younger
patients.
The effect of end-stage renal disease
(CLCR <15 mL/min) or hemodialysis on XPOVIO
pharmacokinetics is unknown.
To report SUSPECTED ADVERSE REACTIONS,
contact Karyopharm Therapeutics Inc. at 1-888-209-9326 or
FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see XPOVIO Full Prescribing Information available
at www.XPOVIO.com.
About Karyopharm Therapeutics
Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a
commercial-stage pharmaceutical company pioneering novel cancer
therapies and 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 Selective Inhibitor of Nuclear
Export (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 U.S.
Food and Drug Administration (FDA) in July
2019 in combination with dexamethasone as a treatment for
patients with heavily pretreated multiple myeloma. In June 2020, XPOVIO was approved by the FDA as a
treatment for patients with relapsed or refractory diffuse large
B-cell lymphoma. A Marketing Authorization Application for
selinexor for patients with heavily pretreated multiple myeloma is
also currently under review by the European Medicines Agency. 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 expectations and plans relating to XPOVIO for the
treatment of patients with relapsed or refractory 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 Company's regulatory strategy, 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; the expected design
of the Company's clinical trials; the therapeutic potential of and
potential clinical development plans for Karyopharm's drug
candidates, especially selinexor; Karyopharm's collaboration
efforts with third-parties; 2020 financial expectations, including
forecasted non-GAAP R&D and SG&A expenses; and expectations
of the sufficiency of Karyopharm's existing cash and investments.
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 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: the risk that the
COVID-19 pandemic could disrupt Karyopharm's business more severely
than it currently anticipates, including by negatively impacting
sales of XPOVIO, interrupting or delaying research and development
efforts, impacting the ability to procure sufficient supply for the
development and commercialization of selinexor or other product
candidates, delaying ongoing or planned clinical trials, impeding
the execution of business plans, planned regulatory milestones and
timelines, or inconveniencing patients; 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 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
June 30, 2020, which was filed with
the Securities and Exchange Commission (SEC) on August 4, 2020, 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.
KARYOPHARM
THERAPEUTICS INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(unaudited)
(in thousands, except per share amounts)
|
|
|
|
Three Months
Ended
September 30,
|
|
|
Nine Months
Ended
September 30,
|
|
|
|
2020
|
|
|
2019
|
|
|
2020
|
|
|
2019
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Product revenue,
net
|
|
$
|
21,330
|
|
|
$
|
12,821
|
|
|
$
|
55,992
|
|
|
$
|
12,821
|
|
License and other
revenue
|
|
|
3
|
|
|
|
328
|
|
|
|
16,993
|
|
|
|
9,976
|
|
Total
revenues
|
|
|
21,333
|
|
|
|
13,149
|
|
|
|
72,985
|
|
|
|
22,797
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
sales
|
|
|
438
|
|
|
|
1,013
|
|
|
|
1,653
|
|
|
|
1,013
|
|
Research and
development
|
|
|
37,037
|
|
|
|
26,270
|
|
|
|
113,628
|
|
|
|
90,761
|
|
Selling, general and
administrative
|
|
|
30,967
|
|
|
|
25,267
|
|
|
|
92,488
|
|
|
|
77,032
|
|
Total operating
expenses
|
|
|
68,442
|
|
|
|
52,550
|
|
|
|
207,769
|
|
|
|
168,806
|
|
Loss from
operations
|
|
|
(47,109)
|
|
|
|
(39,401)
|
|
|
|
(134,784)
|
|
|
|
(146,009)
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest
income
|
|
|
600
|
|
|
|
1,137
|
|
|
|
2,424
|
|
|
|
4,320
|
|
Interest
expense
|
|
|
(6,801)
|
|
|
|
(3,093)
|
|
|
|
(20,068)
|
|
|
|
(9,180)
|
|
Other (expense)
income, net
|
|
|
(141)
|
|
|
|
10
|
|
|
|
(177)
|
|
|
|
(36)
|
|
Total other expense,
net
|
|
|
(6,342)
|
|
|
|
(1,946)
|
|
|
|
(17,821)
|
|
|
|
(4,896)
|
|
Loss before income
taxes
|
|
|
(53,451)
|
|
|
|
(41,347)
|
|
|
|
(152,605)
|
|
|
|
(150,905)
|
|
Income tax
provision
|
|
|
(44)
|
|
|
|
(20)
|
|
|
|
(247)
|
|
|
|
(38)
|
|
Net loss
|
|
$
|
(53,495)
|
|
|
$
|
(41,367)
|
|
|
$
|
(152,852)
|
|
|
$
|
(150,943)
|
|
Net loss per
share—basic and diluted
|
|
$
|
(0.73)
|
|
|
$
|
(0.67)
|
|
|
$
|
(2.14)
|
|
|
$
|
(2.46)
|
|
Weighted-average
number of common shares outstanding
used in net loss per share—basic and diluted
|
|
|
73,466
|
|
|
|
62,093
|
|
|
|
71,479
|
|
|
|
61,297
|
|
KARYOPHARM
THERAPEUTICS INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(unaudited)
(in thousands)
|
|
|
|
September 30,
2020
|
|
|
December 31,
2019
|
|
Assets
|
|
|
|
|
|
|
|
|
Cash, cash
equivalents and investments
|
|
$
|
302,415
|
|
|
$
|
263,972
|
|
Restricted
cash
|
|
|
1,806
|
|
|
|
1,831
|
|
Accounts
receivable
|
|
|
11,062
|
|
|
|
7,862
|
|
Property and
equipment, net
|
|
|
2,357
|
|
|
|
3,046
|
|
Other
assets
|
|
|
19,947
|
|
|
|
18,252
|
|
Total
assets
|
|
$
|
337,587
|
|
|
$
|
294,963
|
|
Liabilities and
stockholders' equity
|
|
|
|
|
|
|
|
|
Deferred
revenue
|
|
$
|
297
|
|
|
$
|
4,533
|
|
Convertible senior
notes
|
|
|
115,802
|
|
|
|
109,857
|
|
Deferred royalty
obligation
|
|
|
73,588
|
|
|
|
73,588
|
|
Other
liabilities
|
|
|
64,130
|
|
|
|
57,211
|
|
Total
liabilities
|
|
|
253,817
|
|
|
|
245,189
|
|
Total stockholders'
equity
|
|
|
83,770
|
|
|
|
49,774
|
|
Total liabilities and
stockholders' equity; 73,528 and 65,370 shares
issued and outstanding at September 30, 2020 and December 31,
2019,
respectively
|
|
$
|
337,587
|
|
|
$
|
294,963
|
|
View original
content:http://www.prnewswire.com/news-releases/karyopharm-reports-third-quarter-2020-financial-results-and-highlights-recent-company-progress-301165209.html
SOURCE Karyopharm Therapeutics Inc.