Full Press Release Details
Karyopharm Reports Fourth Quarter and Full Year 2020 Financial Results and Highlights Recent Company
Commercial Launch of XPOVIO (selinexor) In Expanded
Multiple Myeloma Indication Fully Underway Following December 18, 2020 FDA Approval
XPOVIO Net Product Sales of
$20.2 Million for Fourth Quarter 2020 and $76.2 Million for the Year 2020; Total Revenues of $35.1 Million for the Fourth Quarter 2020 and $108.1 Million for the Year 2020
Positive CHMP Opinion for NEXPOVIO (selinexor) in Penta-Refractory Multiple
Myeloma Issued; European Commission Decision Expected by April 2021
Conference Call Scheduled for Today at 8:30 a.m.
NEWTON, Mass. February 11, 2021 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical
company pioneering novel cancer therapies, today reported financial results for the fourth quarter and full year ended December 31, 2020. In addition, Karyopharm highlighted select corporate milestones, including details regarding the ongoing
U.S. commercialization of XPOVIO (selinexor), regulatory progress in Europe, and provided an overview of its key clinical development programs.
Karyopharm made substantial progress in 2020 towards its mission of improving the lives of patients with cancer, marked by the FDA approval of XPOVIO in
two additional oncology indications: relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and multiple myeloma in patients who have received at least one prior therapy, said Michael G. Kauffman,
MD, PhD, Chief Executive Officer of Karyopharm. In addition, we were particularly encouraged that the National Comprehensive Cancer Network (NCCN) added three different XPOVIO
combination regimens to its Clinical Practice Guidelines in Oncology (NCCN Guidelines ) for previously treated multiple myeloma. As we begin to execute our plans for 2021, we are focused on
the commercial expansion of XPOVIO into the second- and third-line treatment settings for multiple myeloma, a significantly increased addressable patient population. We are also progressing the international expansion of NEXPOVIO (selinexor), with a European Commission (EC) regulatory decision expected by April 2021 following the recent positive opinion issued from the Committee for Medicinal Products for Human Use
(CHMP). In parallel to our commercial efforts in the hematology space, our clinical development of XPOVIO in solid tumor indications continues to advance, with top-line data from the Phase 3 SIENDO study in
endometrial cancer expected in the second half of 2021.
Fourth Quarter 2020 and Recent Highlights
XPOVIO in Multiple Myeloma and DLBCL
In the fourth quarter of 2020, approximately 1,000 XPOVIO prescriptions were filled, with prescription demand higher in December 2020 compared
to either October or November 2020. Over 170 new physician prescribing accounts were added in the fourth quarter of 2020, which included both myeloma and DLBCL treating physicians. Finally, based on data from specialty pharmacies, prescription
refill rates for XPOVIO remained consistent compared to the third quarter of 2020 with the average number of prescriptions per patient estimated at 2.9 by the end of December 2020, compared to 2.0 at the end of December 2019.
On a quarterly basis, Karyopharm expects XPOVIO sales to return to growth beginning in the first quarter of 2021, compared to the fourth
quarter of 2020, following the expanded U.S. FDA approval of XPOVIO as a treatment for patients with multiple myeloma after at least one prior therapy.
XPOVIO in Development for Solid Tumors
Full Year and Fourth Quarter
2020 Financial Results
Net product revenue: Net product revenue for the fourth quarter of 2020 was
$20.2 million, compared to $17.7 million for the fourth quarter of 2019. Net product revenue for the year ended December 31, 2020 was $76.2 million, compared to $30.5 million for the year ended 2019.
License and other revenue: License and other revenue for the fourth quarter of 2020 was $14.9 million, compared to $0.4 million
for the fourth quarter of 2019. This increase was driven by approximately $10.0 million in milestone payments associated with regulatory filings in Asia from Antengene Therapeutics Limited, as well as a $5.0 million upfront payment from
FORUS Therapeutics Inc. upon the execution of a commercial distribution agreement for Canada. License and other revenue in 2020 were $31.9 million, compared to $10.4 million in 2019.
Cost of sales: Cost of sales for the fourth quarter of 2020 were $1.1 million, compared to $1.4 million for the fourth quarter of 2019. Cost
of sales for the year ended December 31, 2020 were $2.7 million, compared to $2.4 million for the year ended December 31, 2019. Cost of sales reflects the costs of XPOVIO units sold and third-party royalties on net product
Research and development (R&D) expenses: R&D expenses for the fourth quarter of 2020 were $37.2 million,
compared to $31.6 million for the fourth quarter of 2019. R&D expenses for 2020 were $150.8 million, compared to $122.3 million for 2019. The increase in R&D expenses in 2020 compared to 2019 was primarily attributable to
costs incurred related to our COVID-19 trial activity, which are not expected to be incurred in 2021, and continued activity in our other ongoing clinical trials.
Selling, general and administrative (SG&A) expenses: SG&A expenses for the fourth
quarter of 2020 were $33.9 million, compared to $28.4 million for the fourth quarter of 2019. SG&A expenses for the year ended December 31, 2020 were $126.4 million, compared to $105.4 million for the year ended December 31, 2019. The increase
in SG&A expenses compared to the prior year was due primarily to activities to support the U.S. commercialization of XPOVIO, including the launches of XPOVIO as a treatment for patients with relapsed or refractory DLBCL and in combination with
once-weekly Velcade and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy.
Interest expense: Interest expense for the fourth quarter of 2020 was $7.1 million, compared to $6.5 million for the fourth
quarter of 2019. Interest expense for the full year 2020 was $27.1 million, compared to $15.6 million for the full year 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 $43.4 million, or $0.59
per share, for the fourth quarter of 2020, compared to a net loss of $48.6 million, or $0.76 per share, for the fourth quarter of 2019. Net loss includes non-cash stock-based compensation expense of
$6.3 million and $3.6 million for the fourth quarter of 2020 and 2019, respectively. Karyopharm reported a net loss of $196.3 million, or $2.72 per share, for the year ended December 31, 2020, compared to a net loss of
$199.6 million, or $3.22 per share, for the year ended December 31, 2019. Net loss includes non-cash stock-based compensation expense of $24.4 million and $15.3 million for the years ended
December 31, 2020 and 2019, respectively.
Cash position: Cash, cash equivalents, restricted cash and investments as of
December 31, 2020 totaled $276.7 million, compared to $265.8 million as of December 31, 2019.
2021 Financial Outlook
Based on its current operating plans, Karyopharm expects its non-GAAP R&D and SG&A expenses, excluding
stock-based compensation expense, for the year ending December 31, 2021 to be in the range of $280 million to $300 million.
expects that its existing cash, cash equivalents and investments, and the revenue it expects to generate from XPOVIO product sales, as well as revenue generated from its license agreements, will be sufficient to fund its planned operations into late
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.
Karyopharm will host a conference call today, Thursday, February 11, 2021, at 8:30 a.m. Eastern Time, to discuss the fourth quarter
and full year 2020 financial results, recent accomplishments, clinical developments and business plans. To access the conference call, please dial (888) 349-0102 (local) or (412)
902-4299 (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 indication to include the treatment for patients with multiple myeloma after at least one prior therapy was approved by the FDA on December 18, 2020. 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
XPOVIO (selinexor) is a prescription medicine approved:
SELECT IMPORTANT SAFETY INFORMATION
Warnings and Precautions
Use In Specific Populations
Lactation: Advise not to breastfeed.
For additional product
information, including full prescribing information, please visit www.XPOVIO.com.
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.
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), is approved in the U.S. in multiple hematologic malignancy indications, including in combination with Velcade (bortezomib)
and dexamethasone for the treatment of patients with multiple myeloma after at least one prior therapy, in combination with dexamethasone for the treatment of patients with heavily pretreated multiple myeloma and as a monotherapy for the treatment
of patients with relapsed or refractory diffuse large B-cell lymphoma. A Marketing Authorization Application for NEXPOVIO (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