Full Press Release Details
Karyopharm Announces Preliminary Unaudited Fourth Quarter and Full Year 2019 Product Sales for XPOVIO (selinexor) and Provides Commercial Update
XPOVIO Unaudited Net
Product Sales of between $17 and $18 Million for Fourth Quarter 2019 and between $30 and $31 Million for the Full Year 2019
Approximately 1,400 XPOVIO Prescriptions Fulfilled in the U.S. from Launch Through Year End 2019
NEWTON, Mass. January 13, 2020 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
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
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.
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.
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 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
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.
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.
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.
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.
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.
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