Full Press Release Details
Karyopharm Reports Third Quarter 2020 Financial Results and
Highlights Recent Company Progress
Phase 3 SEAL Study Meets Primary Endpoint with Significant Increase in Progression-Free Survival in Patients with Unresectable
Dedifferentiated Liposarcoma
Third Quarter 2020 XPOVIO Net Product Sales of $21.3 Million; Quarterly Sales Continue
Strong Momentum, Increasing Approximately 15% Compared to the Second Quarter of 2020
XPOVIO Supplemental New Drug
Application Seeking Approval for Patients with Multiple Myeloma After At Least One Prior Line of Therapy Assigned a Target PDUFA Action Date of March 19, 2021
Conference Call Scheduled for Today at 4:30 p.m. ET
NEWTON, Mass. November 2, 2020 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 in Development for Solid Tumors
Low Dose Selinexor in Development for
Third Quarter 2020 Financial
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
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
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.
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
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