Full Press Release Details
Karyopharm Reports Second Quarter 2020 Financial Results and
Highlights Recent Company Progress
Second Quarter 2020 XPOVIO Net Product Sales of $18.6 Million; Strongest Quarterly Sales Since July 2019 Launch
XPOVIO Approved by FDA as a New Treatment for Patients with Relapsed or Refractory DLBCL; Commercial Rollout Began Immediately Upon
Supplemental New Drug Application for XPOVIO as a Treatment for Patients with Multiple Myeloma After
At Least One Prior Line of Therapy Accepted by FDA and Assigned a Target PDUFA Date of March 19, 2021
Conference Call Scheduled for Today at 8:30 a.m. ET
NEWTON, Mass. August 4, 2020 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a commercial-stage pharmaceutical company pioneering novel
cancer therapies, today reported financial results for the quarter ended June 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.
ongoing global COVID-19 pandemic, Karyopharm was able to achieve record quarterly XPOVIO sales as well as execute on several important initiatives, including receiving approval of XPOVIO for its second cancer
indication from the U.S. Food and Drug Administration (FDA) to treat patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). XPOVIO is now the only single-agent oral therapy approved in
this indication, including DLBCL arising from follicular lymphoma, and now approved in both multiple myeloma and DLBCL, said Michael G. Kauffman, MD, PhD, Chief Executive Officer of Karyopharm. Other recent highlights include the
reporting of positive clinical results from the Phase 3 BOSTON study in a late breaking oral presentation at the American Society of Clinical Oncology (ASCO) 2020 Virtual Scientific Program. Additionally, a supplemental New Drug Application (sNDA)
was recently accepted by the FDA based on these positive data. Finally, our planned interim analysis of the randomized Phase 2 study of low dose selinexor in patients with severe COVID-19 indicated that while
the agent is unlikely to demonstrate a statistically significant efficacy benefit across the entire patient population studied, it appears to confer clinical benefit in a clearly defined subpopulation of patients. Based on these
results, we expect that future clinical development of low dose selinexor for patients with COVID-19 will focus on this subpopulation.
Second Quarter 2020 and Recent Highlights
XPOVIO in Diffuse Large B-Cell Lymphoma (DLBCL)
Low Dose Selinexor in COVID-19
Second Quarter 2020 Financial Results
Net product revenue: Net product revenue for the second quarter of 2020 was $18.6 million. Karyopharm commenced sales of XPOVIO in the U.S. during
the third quarter of 2019 and therefore did not have net product revenue during the second quarter of 2019.
License and other revenue: License and
other revenue for the second quarter of 2020 was $14.9 million, compared to $9.5 million for the second quarter of 2019. License and other revenue for the second quarter of 2020 included $12.7 million in revenue recognized as a result
of a May 2020 amendment to our license agreement with Antengene Therapeutics Limited (Antengene), coupled with $2.2 million in revenue recognized upon the April 2020 termination of our license agreement with Ono Pharmaceutical Co., Ltd.
Karyopharm recognized $9.4 million in revenue during the second quarter of 2019 pursuant to the terms of the original agreement with Antengene and $0.1 million related to clinical supply provided to various partners, as well as grant
revenue pursuant to a government grant arrangement.
Cost of sales: Cost of sales were $0.4 million for the second quarter of 2020. Cost of
sales reflects the costs of XPOVIO units sold and third-party royalties on net product revenue.
Research and development expenses (R&D):
R&D expenses for the second quarter of 2020 were $42.6 million, compared to $26.5 million for the second quarter of 2019. The increase in R&D expenses compared to the second 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 expenses (SG&A): For the second quarter of 2020, SG&A expenses were $30.8 million, compared to $24.7 million for the second quarter of 2019. The increase in SG&A expenses compared to the second quarter of
last year was due primarily to activities to support the U.S. commercialization of XPOVIO and preparations for the launch of XPOVIO as a treatment for patients with relapsed or refractory DLBCL.
Interest expense: Interest expense for the second quarter of 2020 was $6.8 million, compared to $3.1 million for the second quarter of 2019.
The increase in interest expense was attributable to the imputed interest on the deferred royalty obligation Karyopharm has with HealthCare Royalty Partners.
Net loss: Karyopharm reported a net loss of $46.4 million, or $0.63 per share, for the second quarter of 2020, compared to a net loss of
$43.4 million, or $0.71 per share, for the second quarter of 2019. Net loss includes non-cash stock-based compensation expense of $6.4 million and $4.1 million for the second quarters of 2020
and 2019, respectively.
Cash position: Cash, cash equivalents, restricted cash and investments as of June 30, 2020 totaled
$348.2 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 middle 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, Tuesday, August 4, 2020, at 8:30 a.m. Eastern Time, to discuss the second 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
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
Tel: +1 (888) 209-9326
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 infections were not associated with Grade 3 or higher