“2017 was a year of significant achievement for Karyopharm where we reported positive clinical data from our lead selinexor programs, including the Phase 1b/2 STOMP study in multiple myeloma (MM), the Phase 2b SADAL study in diffuse large B-cell lymphoma (DLBCL), and the Phase 2/3 SEAL study in liposarcoma, and the subsequent advancement of each study,” said
Fourth Quarter 2017 and Recent Events
New Strategic Relationships
- Biogen’s Acquisition of KPT-350 for the Treatment of Neurological and Neurodegenerative Diseases. In
January 2018, Karyopharm announced its entry into an agreement with Biogen to acquire Karyopharm’s investigational oral SINE compound KPT-350 targeting certain neurological and neurodegenerative conditions, including amyotrophic lateral sclerosis (ALS). The transaction carries a total deal value of up to $217 million, plus royalties.
- Exclusive License Agreement with
Ono Pharmaceutical Co., Ltd.(Ono) to Develop and Commercialize Selinexor and Eltanexor in Japanand Other Countries in Asia. In October 2017, Karyopharm announced its entry into an exclusive license agreement with Ono for the development and commercialization of selinexor and eltanexor. The agreement includes the development of selinexor and eltanexor for the diagnosis, treatment and/or prevention of all human oncology indications in Japan, South Korea, Taiwan, Hong Kong, and the ASEAN countries (the Territory). The transaction carries a total deal value of up to $193.0 millionbased on the exchange rate on the effective date of the license agreement plus royalties. Karyopharm retains all rights to selinexor and eltanexor outside the Territory.
Selinexor in Multiple Myeloma
- Ongoing Phase 2b STORM Study Expansion in Patients with Penta-refractory MM. Karyopharm expects to report top-line data from the expanded STORM study cohort at the end of
April 2018, and, assuming a positive outcome, intends to use the data from this study to support a request for accelerated approval from the FDAand conditional approval from the EMA for oral selinexor for penta-refractory MM. The Phase 2b STORM study was previously expanded to include 122 additional patients with penta-refractory MM.
- Pivotal Phase 3 BOSTON Study Underway. Karyopharm’s pivotal, randomized Phase 3 BOSTON (Bortezomib, Selinexor and dexamethasone) study is now underway and enrolling patients in 14 countries globally.
BOSTONis designed to evaluate 100mg of selinexor dosed once weekly in combination with the proteasome inhibitor Velcade (once weekly) and dexamethasone (SVd), compared to standard twice weekly Velcade and low-dose dexamethasone (Vd) in patients with MM who have had one to three prior lines of therapy. The primary endpoints of the study are progression free survival and overall response rate. Both the trial design and endpoints have been agreed to by the FDAand the EMA as acceptable to support an application for approval. The Company expects to enroll approximately 360 patients at over 100 clinical sites internationally and is expecting completion of enrollment in 2018, with top-line data anticipated in 2019.
- Positive Phase 1b/2 STOMP Data Presented at ASH 2017. Data presentations featuring clinical results from four treatment arms of the ongoing Phase 1b/2 STOMP study evaluating selinexor in combination with standard therapies for the treatment of patients with MM were presented at the
American Society of Hematology2017 Annual Meeting (ASH 2017). Collectively, the STOMP study data presented at ASH 2017 continue to provide evidence of tolerability and robust anti-myeloma activity when selinexor is combined with the currently available standard myeloma therapies, including proteasome inhibitors including Velcade, immunomodulatory drugs and anti-CD38 monoclonal antibodies. Additional treatment arms with selinexor in patients with newly diagnosed disease in combination with lenalidomide, and in combination with the proteasome inhibitor Kyprolis® (carfilzomib) have been added.
Selinexor in Diffuse Large B-Cell Lymphoma
- Ongoing Phase 2b SADAL Study in DLBCL. Karyopharm is also investigating oral selinexor as a single-agent for the treatment of patients with relapsed or refractory DLBCL. The SADAL study is expected to enroll up to a total of 130 patients in the single-arm cohort evaluating single-agent selinexor dosed 60mg twice weekly in patients with two or more lines of prior therapy. Karyopharm plans to report top-line results by the end of 2018, and assuming a positive outcome, the Company intends to use the data from the SADAL study to support a request for accelerated approval from the
FDAand conditional approval from the EMA for oral selinexor in this relapsed/refractory DLBCL patient population.
Selinexor in Solid Tumors
- Phase 3 Portion of the Phase 2/3 SEAL Study in Liposarcoma Underway. Karyopharm previously reported a successful outcome from the Phase 2 portion of the blinded, randomized Phase 2/3 SEAL study evaluating single-agent selinexor versus placebo in patients with previously treated, advanced unresectable dedifferentiated liposarcoma. The Phase 3 portion is underway and, assuming a positive outcome on the primary end point of progression free survival, the Company intends to use the data from the Phase 2/3 SEAL study to support a New Drug Application in the U.S. and a Marketing Authorization Application (MAA) in
Europefor oral selinexor as a potential new treatment for patients with advanced unresectable dedifferentiated liposarcoma. Top-line data from the Phase 3 portion of the SEAL study are anticipated by the end of 2019.
- Initiation of Investigator Sponsored Phase 2/3 Trial as Maintenance Therapy in Endometrial Cancer Underway. A randomized Phase 2/3 study of selinexor vs. placebo as maintenance therapy in patients with 1-2 prior platinum-based treatments for advanced endometrial cancer lead by Dr.
Ignace Vergote, Head of the Department of Obstetrics and Gynaecologyand Gynaecologic Oncology at the Catholic University of Leuven, Belgium, has been initiated.
- Positive Phase 1/2 Eltanexor Data Presented at ASH 2017. Data from a Phase 1/2 study evaluating oral eltanexor in 34 patients with heavily pretreated MM was also presented at ASH 2017. The data showed that eltanexor, both alone or in combination with low-dose dexamethasone, induced responses or disease control and was associated with prolonged survival. The study has been expanded to evaluate eltanexor in patients with advanced colorectal cancer (CRC), castration-resistant prostate cancer (crPC), and myelodysplastic syndrome (MDS). These are indications where selinexor and XPO1 inhibition has shown clear activity, but where the reduced side effects of eltanexor may permit more extended dosing.
Fourth Quarter and Year Ended
Cash, cash equivalents and investments as of
For the year ended
Karyopharm reported a net loss of
For the quarter ended
Based on current operating plans, Karyopharm expects that its existing cash, cash equivalents and investments will be sufficient to fund its operations through at least the first quarter of 2019. These plans include the continued clinical development of selinexor in the Company’s lead indications with a focus on filing an NDA with the
Conference Call Information
Karyopharm will host a conference call today,
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 the therapeutic potential of and potential clinical development plans for Karyopharm's drug candidates, including the timing of enrollment of certain trials, the reporting of data from such trials and potential regulatory filings, the potential to receive milestone and royalty payments under the arrangements with Ono and Biogen and Karyopharm’s financial outlook and financial projections for Karyopharm. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Karyopharm’s current expectations. For example, there can be no guarantee that any of Karyopharm's SINE compounds, including selinexor (KPT-330), eltanexor (KPT-8602), Karyopharm’s second-generation oral SINE compound, or KPT-9274, Karyopharm's first-in-class oral dual inhibitor of PAK4 and NAMPT, or any other drug candidate that Karyopharm is developing, will successfully complete necessary preclinical and clinical development phases or that development of any of Karyopharm's drug candidates will continue. Further, there can be no guarantee that any positive developments in Karyopharm's drug candidate portfolio will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: Karyopharm's results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the
Velcade® is a registered trademark of
Revlimid® and Pomalyst® are registered trademarks of
Kyprolis® is a registered trademark of
Darzalex® is a registered trademark of
|Karyopharm Therapeutics Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
|Cash and cash equivalents||$||68,997||$||49,663|
|Prepaid expenses and other current assets||1,754||2,084|
|Total current assets||148,423||131,636|
|Property and equipment, net||2,185||2,836|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Other current liabilities||133||83|
|Total current liabilities||49,467||16,476|
|Deferred rent, net of current portion||1,363||1,666|
|Preferred stock, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding||—||—|
|Common stock, $0.0001 par value; 100,000,000 shares authorized; 49,533,150 and 41,887,829 shares issued and outstanding at December 31, 2017 and 2016, respectively||5||4|
|Additional paid-in capital||625,017||528,617|
|Accumulated other comprehensive loss||(217||)||(274||)|
|Total stockholders’ equity||129,464||162,243|
|Total liabilities and stockholders’ equity||$||180,294||$||180,385|
|Karyopharm Therapeutics Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
For the Quarter Ended,
|For the Year Ended
|License and other revenue||$||1,534||$||47||$||1,605||$||154|
|Research and development||34,833||20,671||107,273||86,938|
|General and administrative||6,153||6,541||24,870||23,948|
|Total operating expenses||40,986||27,212||132,143||110,886|
|Loss from operations||(39,452||)||(27,165||)||(130,538||)||(110,732||)|
|Other income (expense):|
|Other income (expense)||(11||)||11||(81||)||10|
|Total other income, net||421||369||1,617||1,294|
|Loss before income taxes||(39,031||)||(26,796||)||(128,921||)||(109,438||)|
|Provision for income taxes||(9||)||(139||)||(63||)||(139||)|
|Net loss per share—basic and diluted||$||(0.80||)||$||(0.65||)||$||(2.81||)||$||(2.92||)|
|Weighted-average number of common shares outstanding used in net loss per share—basic and diluted||48,644,578||41,376,022||45,899,784||37,523,051|
Source: Karyopharm Therapeutics Inc.