“The positive top-line data recently reported from the Phase 2b STORM study evaluating selinexor in patients with penta-refractory myeloma are an important step forward toward the approval of selinexor,” said
First Quarter 2018 and Recent Events
Selinexor in Multiple Myeloma
- Reported Positive Top-line Data from the Phase 2b STORM Study Expansion in Patients with Penta-Refractory Myeloma. Last week, Karyopharm reported positive top-line results from the Phase 2b STORM study evaluating selinexor in heavily pretreated patients with refractory multiple myeloma. For the STORM study’s primary objective, oral selinexor achieved a 25.4% ORR, which included two stringent complete responses (CRs) and 29 partial (PRs) or very good partial responses (VGPRs). One of the stringent CRs was negative for minimal residual disease (MRD), highly significant in this penta-refractory population. The median DOR, a key secondary objective, was 4.4 months. Oral selinexor demonstrated a predictable and manageable tolerability profile. Safety results were consistent with those previously reported from Part 1 of this study and from other selinexor studies and no new safety signals were identified. Karyopharm plans to submit detailed STORM study results for presentation at an upcoming medical oncology meeting.
- Selinexor Receives Fast Track Designation from
FDAfor the Treatment of Patients with Penta-Refractory Myeloma. In addition to Orphan Drug Designation, selinexor was recently granted Fast Track designation by the FDAfor the treatment of patients with penta-refractory multiple myeloma. The FDA’s stated indication is consistent with the design of Karyopharm’s Phase 2b STORM study.
- On Track to Submit NDA for Selinexor in Penta-Refractory Myeloma. Karyopharm plans to submit an NDA to the
FDAduring the second half of 2018, with a request for accelerated approval for oral selinexor as a new treatment for patients with penta-refractory multiple myeloma. The Company also plans to submit an MAA to the EMA in early 2019 with a request for conditional approval for selinexor in the same indication.
- Pivotal Phase 3 BOSTON Study Underway. Karyopharm’s pivotal, randomized Phase 3 BOSTON study is now well underway and enrolling patients in 14 countries globally.
BOSTONis evaluating 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 multiple myeloma who have had one to three prior lines of therapy. The primary endpoints of the study are progression free survival and overall response rate. Assuming a positive outcome, the data from the BOSTONstudy will be used to support regulatory submissions to the FDAand EMA for full approvals. The Company expects to enroll approximately 360 patients at over 100 clinical sites internationally and expects to complete enrollment by the end of 2018, with top-line data anticipated in 2019.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)
- 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 who received 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 positive results 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 SEAL study to support an NDA and an MAA submission for oral selinexor 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.
- Ongoing Investigator Sponsored Phase 2/3 Trial as Maintenance Therapy in Endometrial Cancer Underway. A randomized Phase 2/3 study of selinexor versus placebo as maintenance therapy in patients with one or two 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, is currently ongoing.
- Eight Preclinical Data Posters Presented at the
American Association for Cancer Research(AACR) 2018 Annual Meeting. In March 2018, eight posters were presented at AACR 2018 which featured preclinical data for selinexor, eltanexor and KPT-9274. Collectively, these data continue to provide important insights that Karyopharm believes will help guide the future clinical development of all three product candidates across a wide range of malignancies, including ovarian, prostate and pancreatic cancers and neuroblastoma.
Corporate and Financial
- Strengthened the Balance Sheet with a Public Equity Offering. On
May 7, 2018, Karyopharm completed an underwritten public offering of 10,525,424 shares of its common stock at a price to the public of $14.75per share. The gross proceeds to Karyopharm from the offering were $155.3 million. After deducting the underwriting discounts and commissions and other estimated offering expenses the net proceeds were $145.6 million.
- 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 for Biogen’s acquisition of 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.
First Quarter Ended
Cash, cash equivalents and investments as of
For the quarter ended
For the quarter ended
Karyopharm reported a net loss of
Karyopharm expects its operating cash burn, including research and development and general and administrative expenses, for the year ending
Further Information About Potential Accelerated Approval for Selinexor in Multiple Myeloma
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 timing of submissions to regulatory authorities and the potential availability of accelerated approval pathways, 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 the impact on potential regulatory filings, the potential to receive milestone and royalty payments under third party arrangements 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 regulators will agree that selinexor qualifies for accelerated approval in the U.S. or conditional approval in the E.U. as a result of the data from the STORM study in patients with penta-refractory myeloma or that any of Karyopharm's drug candidates, 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.
CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands, except share and per share amounts)
|Cash and cash equivalents||$||37,499||$||68,997|
|Prepaid expenses and other current assets||2,396||1,754|
|Total current assets||133,313||148,423|
|Property and equipment, net||2,454||2,185|
|Liabilities and stockholders’ equity|
|Other current liabilities||333||133|
|Total current liabilities||46,734||49,467|
|Deferred revenue, net of current portion||2,192||—|
|Deferred rent, net of current portion||1,918||1,363|
|Preferred stock, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding||—||—|
|Common stock, $0.0001 par value; 100,000,000 shares authorized; 49,670,328 and 49,533,150
shares issued and outstanding at March 31, 2018 and December 31, 2017, respectively
|Additional paid-in capital||629,610||625,017|
|Accumulated other comprehensive loss||(286||)||(217||)|
|Total stockholders’ equity||95,529||129,464|
|Total liabilities and stockholders’ equity||$||146,373||$||180,294|
Karyopharm Therapeutics Inc.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share amounts)
|Three Months Ended
|License and other revenue||$||10,000||$||68|
|Research and development||41,321||24,083|
|General and administrative||7,621||6,264|
|Total operating expenses||48,942||30,347|
|Loss from operations||(38,942||)||(30,279||)|
|Other income (expense):|
|Total other income, net||495||385|
|Loss before income taxes||(38,447||)||(29,894||)|
|Provision for income taxes||(12||)||(23||)|
|Net loss per share—basic and diluted||$||(0.78||)||$||(0.71||)|
|Weighted-average number of common shares outstanding used in net loss per share—basic and
Source: Karyopharm Therapeutics Inc.