Company Description
Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a commercial-stage pharmaceutical company in the pharmaceutical preparation manufacturing industry. The company focuses on novel cancer therapies that target nuclear export dysregulation, a mechanism linked to oncogenesis. Karyopharm has been described in its public disclosures as an industry leader in oral compounds that address nuclear export dysregulation, and its work centers on developing and commercializing therapies for multiple high unmet need cancers.
The company’s lead compound is XPOVIO (selinexor), a first-in-class, oral exportin 1 (XPO1) inhibitor and the first of Karyopharm’s Selective Inhibitor of Nuclear Export (SINE) compounds for the treatment of cancer. According to company statements, XPOVIO functions by selectively binding to and inhibiting the nuclear export protein XPO1. XPOVIO is approved in the U.S. and marketed by Karyopharm in three oncology indications, including multiple myeloma and diffuse large B-cell lymphoma (DLBCL). The company also reports that XPOVIO (also known as NEXPOVIO in certain countries) has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries.
Karyopharm’s disclosures state that XPOVIO is approved in the U.S. in multiple hematologic malignancy indications, including:
- In combination with VELCADE (bortezomib) and dexamethasone for adult patients with multiple myeloma after at least one prior therapy.
- In combination with dexamethasone for adult patients with heavily pretreated multiple myeloma.
- As a monotherapy for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), including DLBCL arising from follicular lymphoma, after at least two lines of systemic therapy, under accelerated approval based on response rate.
The company states that XPOVIO is approved in more than 50 ex-U.S. territories and countries in various indications, and that it is marketed in those territories by partners such as Antengene, Menarini, Neopharm and FORUS. Karyopharm has indicated that global patient access for selinexor has expanded, with regulatory and reimbursement decisions in multiple countries.
Beyond its commercial product, Karyopharm describes a focused pipeline centered on selinexor and related SINE compounds in multiple high unmet need cancers. The company highlights active Phase 3 clinical programs in:
- Myelofibrosis – including the Phase 3 SENTRY (XPORT-MF-034; NCT04562389) trial, which evaluates once-weekly 60 mg selinexor in combination with ruxolitinib versus ruxolitinib plus placebo in JAK inhibitor–naïve myelofibrosis patients with specified platelet counts. Co-primary endpoints include spleen volume reduction and change in total symptom score.
- Endometrial cancer – including the global Phase 3 XPORT-EC-042 (EC-042; NCT05611931) trial, which evaluates selinexor as a maintenance therapy following systemic therapy in patients with TP53 wild-type advanced or recurrent endometrial cancer. Patients are randomized to receive once-weekly oral selinexor or placebo until disease progression, with progression-free survival as the primary endpoint and overall survival as a key secondary endpoint.
- Multiple myeloma – including the Phase 3 XPORT-MM-031 (EMN29; NCT05028348) trial in previously treated multiple myeloma patients who received an anti-CD38 antibody in their immediate prior line of therapy.
The company also reports clinical activity of selinexor in additional settings, such as myelofibrosis patients who are heavily pretreated, where Phase 2 data from the XPORT-MF-035 trial suggest the potential for single-agent clinical activity, including spleen volume reduction, symptom improvement, hemoglobin stabilization, reduced transfusion burden and evidence of disease modification.
According to its public communications, Karyopharm’s pipeline targets multiple high unmet need cancers, including multiple myeloma, endometrial cancer, myelofibrosis and DLBCL. Selinexor is being investigated in several mid- and late-stage clinical trials across these indications. The company emphasizes the role of TP53 status and mismatch repair status in its endometrial cancer program, and has entered into a global collaboration with Foundation Medicine, Inc. to develop FoundationOne CDx as a tissue-based comprehensive genomic profiling test to identify and enroll patients whose tumors are TP53 wild-type in the EC-042 trial.
Karyopharm’s securities filings and press releases identify it as a Delaware corporation with common stock listed on the Nasdaq Global Select Market under the symbol KPTI. The company has entered into various financing arrangements, including senior secured term loans, convertible senior notes and revenue interest financing agreements, as described in its Form 8-K filings. These financings are intended, according to the company, to provide financial flexibility, additional working capital and to support ongoing and planned clinical trial activities.
Based on its disclosures, Karyopharm derives revenue from U.S. XPOVIO net product sales and from license, royalty and milestone revenue earned from partners. The company has reported that demand for XPOVIO in the U.S. multiple myeloma marketplace is supported by both community and academic settings, and that expanded global patient access for selinexor contributes to royalty revenue from international partners.
In addition to its clinical and commercial activities, Karyopharm uses equity-based compensation, such as restricted stock units and stock options, as inducement awards for newly hired employees under its inducement stock incentive plan, as reported in multiple Form 8-K filings. The company has also disclosed workforce reductions as part of efforts to manage operating expenses while continuing to support its commercial operations and late-stage clinical programs.
Business model and focus
According to its own descriptions, Karyopharm’s business model centers on the discovery, development and commercialization of drugs directed against nuclear export for the treatment of cancer and other diseases. The company’s lead commercial asset, XPOVIO (selinexor), is an oral XPO1 inhibitor that is both marketed in approved indications and used as the backbone of multiple clinical programs. Revenue sources identified by the company include U.S. net product revenue from XPOVIO and license and royalty revenue from partners who commercialize selinexor/NEXPOVIO in ex-U.S. territories.
The company’s clinical strategy, as described in its press releases, involves pursuing pivotal Phase 3 trials in indications such as myelofibrosis and endometrial cancer, alongside continued development in multiple myeloma and DLBCL. These programs are designed to evaluate selinexor in combination regimens, as maintenance therapy, and as monotherapy in specific patient populations defined by prior treatments and molecular characteristics.
Regulatory and clinical context
Karyopharm’s communications provide detailed context for the diseases it targets. For example, myelofibrosis is described as a rare blood cancer associated with bone marrow fibrosis, splenomegaly, progressive anemia and other symptoms, where JAK inhibitors such as ruxolitinib are the only approved class of therapies and where anemia and transfusion dependence are correlated with poor prognosis. In endometrial cancer, the company cites epidemiologic data showing rising incidence and mortality, highlights TP53 as a prognostic marker, and notes an unmet need for targeted therapies in certain mismatch repair–proficient populations.
Through its collaboration with Foundation Medicine on FoundationOne CDx for the EC-042 trial, Karyopharm links its clinical development strategy to genomic profiling to identify TP53 wild-type tumors. The company’s trials specify endpoints such as spleen volume reduction, symptom scores, progression-free survival and overall survival, reflecting standard oncology trial design as described in its public materials.
Capital structure and financing
In a series of Form 8-K filings, Karyopharm outlines financing transactions that include amendments to its credit and guaranty agreement, issuance of 9.00% senior secured convertible notes due 2028 and 2029, exchanges of existing convertible notes for equity and warrants, and a private placement of common stock and warrants. The company states that these transactions are intended to provide approximately $100 million of financial flexibility and additional capital, extend its cash runway into a specified future period based on current operating plans, and support its Phase 3 programs.
The company also reports that it has entered into registration rights agreements and intercreditor arrangements, and that its term loans and convertible notes are secured obligations with specified maturities and interest terms. These details appear in the exhibits and summaries contained in the company’s Form 8-K filings.
Exchange listing and corporate status
SEC filings list Karyopharm’s common stock, par value $0.0001 per share, as registered under Section 12(b) of the Exchange Act and traded on the Nasdaq Global Select Market under the symbol KPTI. The filings identify the company as incorporated in Delaware. There is no indication in the provided materials of a completed merger, acquisition, delisting, bankruptcy filing or liquidation; instead, the company describes ongoing financing activities and exploration of strategic alternatives, including potential mergers or sales, in its Form 8-K disclosures.