“We continued to make tremendous progress towards bringing selinexor, our lead drug candidate, to patients with highly refractory multiple myeloma. Most notably, the U.S.
“As we await the upcoming
Third Quarter 2018 and Recent Events
Selinexor in Multiple Myeloma
- U.S. FDA Accepts Selinexor NDA and Grants Priority Review. The U.S.
FDAaccepted for filing with Priority Review Karyopharm’s NDA seeking accelerated approval for selinexor, its first in class, oral SINE compound, as a new treatment for patients with penta-refractory multiple myeloma. The FDAalso assigned an action date of April 6, 2019under the PDUFA. Provided marketing approval is granted by the FDA, Karyopharm plans to commercialize selinexor in the U.S. in the first half of 2019. The Company also plans to submit a Marketing Authorization Application to the European Medicines Agency(EMA) in early 2019 with a request for conditional approval.
- Phase 2b STORM Data Selected for Oral Presentation at ASH 2018. Additional results from Part 2 of the Phase 2b STORM study have been selected for oral presentation at the upcoming ASH 2018 Annual Meeting in early December. Karyopharm previously reported results from the Phase 2b STORM study evaluating selinexor plus low dose dexamethasone (Sd) in patients with penta-refractory multiple myeloma in September at the
Society of Hematologic Oncology(SOHO) 2018 Annual Meeting. For the STORM study’s primary objective, the overall response rate (ORR) was 26.2%, which included two stringent complete responses (sCRs), six very good partial responses (VGPRs) and 24 partial responses (PRs) in these patients with penta-refractory myeloma. The two sCRs were negative for minimal residual disease, one at the level of 1x10-6 and one at 1x10-4; this is particularly significant in this penta-refractory population. The ORR for Sd in patients who had previously received Darzalex® combination therapy (n=86) was 29.1%. The Disease Control Rate for patients who had achieved stable disease or better was 78.6%. All responses were confirmed by an Independent Review Committee. Median progression-free survival (PFS) was 3.7 months and the median duration of response (DOR) was 4.4 months (range <1 to 9.9 months). Median overall survival (OS) across the study was 8.6 months. Median OS in the approximately 40% of patients with at least a minimal response (MR) on Sd was 15.6 months compared to a median OS of 1.7 months in patients whose disease progressed or was not evaluable (p<0.0001). The short median OS of patients with no response to selinexor is consistent with the lack of available effective therapies for the very heavily pretreated population who entered the study. Across the relevant patient population, side effects of oral selinexor were generally predictable and manageable with dose adjustments and/or supportive care, with safety results that were consistent with those previously reported from Part 1 of this STORM study (Vogl et al., J Clin Oncol, 2018) and from other selinexor studies.
- Two Phase 1b/2 STOMP Abstracts Selected for Presentation at ASH 2018. Two abstracts featuring clinical data from two treatment arms of the ongoing Phase 1b/2 STOMP study in patients with relapsed or refractory multiple myeloma have been selected for oral and poster presentations at ASH 2018. The oral presentation will highlight data from the arm evaluating selinexor in combination with Darzalex (daratumumab) and low-dose dexamethasone (SDd). The poster presentation will provide updated data from the arm evaluating selinexor in combination with Pomalyst® (pomalidomide) and low-dose dexamethasone (SPd). In data reported previously from these arms, selinexor has demonstrated evidence of additive or synergistic anti-myeloma activity when combined with these standard approved therapies.
- Pivotal Phase 3 BOSTON Study in
Progress. Karyopharm’s pivotal, randomized Phase 3 BOSTON study is underway and enrolling patients globally. BOSTONis evaluating 100mg of selinexor dosed once weekly in combination with the proteasome inhibitor Velcade (once weekly) and low dose 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 PFS and ORR. Data from the BOSTONstudy, if positive, would be used to support regulatory submissions to the FDAand EMA requesting full approvals for use of selinexor in second line multiple myeloma, following the Company’s requests for accelerated and conditional approvals, respectively, using data from the Phase 2b STORM study. 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 at the end of 2019.
Selinexor in Diffuse Large B-Cell Lymphoma (DLBCL)
- Received Fast Track Designation from
FDAfor the Treatment of Patients with Relapsed or Refractory DLBCL. In addition to Orphan Drug Designation, selinexor was recently granted Fast Track designation by the FDAfor the treatment of patients with relapsed or refractory DLBCL.
- Phase 2b SADAL Data in DLBCL Selected for Presentation at ASH 2018. An abstract featuring data from the fully enrolled Phase 2b SADAL study has been selected for poster presentation at ASH 2018. The SADAL study is designed to evaluate single agent oral selinexor 60mg for patients with relapsed or refractory DLBCL who are not eligible for stem cell transplantation. The SADAL study has enrolled approximately 125 patients with DLBCL who received two to five lines of prior therapy at single-agent selinexor dosed 60mg twice weekly in patients. Assuming the results from the SADAL study are positive, Karyopharm plans to submit an NDA to the
FDAwith a request for accelerated approval, and a Marketing Authorization Application (MAA) to the EMA with a request for conditional approval, for oral selinexor in this relapsed or refractory DLBCL patient population.
Selinexor in Solid Tumors
- Ongoing Phase 3 Portion of the Phase 2/3 SEAL Study in Liposarcoma. Karyopharm previously reported results from the successful 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. Enrollment and dosing is currently ongoing in the Phase 3 portion of the SEAL study and, assuming a positive outcome on the primary end point of PFS, the Company intends to use the data from the SEAL study to support an NDA and an MAA submission requesting full approval 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. 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, led by Dr.
Ignace Vergote, Head of the Department of Obstetrics and Gynaecologyand Gynaecologic Oncology at the Catholic University of Leuven, Belgium, is currently ongoing. In the U.S., endometrial cancer is the most common gynecological cancer with approximately 58,000 cases expected to be diagnosed and an estimated 10,000 women expected to die from this cancer in 20181, revealing a meaningful patient population in need of novel therapies.
- Phase 1/2 Eltanexor Data in Metastatic Colorectal Cancer (mCRC) Presented at
ESMO2018. At the European Society of Medical Oncology( ESMO) 2018 Congressin October, John Hays. MD, PhD, Ohio State University, Comprehensive Cancer Center, presented preliminary results from the ongoing Phase 1/2 investigator-sponsored study investigating eltanexor in patients with heavily pre-treated (median of 4 prior treatment regimens) metastatic colorectal cancer (mCRC). The presented results showed that 37% of patients experienced disease control at ≥8 weeks on eltanexor and the median preliminary progression free survival (PFS) for all patients in the 30 mg cohort was 3.5 months. Eltanexor was generally well tolerated with manageable adverse events. The highest observed treatment-related grade ≥3 adverse events were hyponatremia (23%), fatigue (20%) and anemia (20%). Karyopharm is encouraged by these preliminary results which demonstrated promising efficacy with a median PFS longer than currently available third line therapies and an acceptable safety and tolerability profile.
Other ASH 2018 Highlights
- Several Investigator-sponsored Trials and Preclinical Abstracts Selected for Presentation at ASH 2018. Three abstracts featuring clinical data from investigator-sponsored clinical studies evaluating selinexor either as a single-agent or in combination with other anti-cancer agents for the treatment of acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and pediatric leukemia have been selected for oral or poster presentations at ASH 2018. Three abstracts describing preclinical research exploring the use of selinexor in models of multiple myeloma and AML have also been selected for poster presentations at the meeting. One abstract featuring preclinical research investigating the use of KPT-9274, Karyopharm’s oral dual inhibitor of PAK4 and NAMPT, for the treatment of AML, has also been selected for a poster presentation. A complete list of the ASH 2018 abstracts can be accessed here.
Carsten Thiel, Ph.D. Appointed to the Board. Karyopharm announced the appointment of Carsten Thiel, Ph.D., to its Board of Directors. Dr. Thiel is currently Chief Executive Officer of Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies. Prior to joining Abeona, Dr. Thiel served as the Executive Vice President and Chief Commercial Officer of Alexion Pharmaceuticals, Inc., a leading global biopharmaceutical company focused on serving patients affected by rare diseases.
Third Quarter Ended
Cash, cash equivalents and investments as of
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
Karyopharm’s current operating plans include the continued clinical development of selinexor in the Company’s lead indications and on preparing the commercial infrastructure and hiring a sales force for the potential launch of selinexor in the U.S. Additional key activities expected in 2018 include preparing for a potential MAA submission to the EMA requesting conditional approval for selinexor in multiple myeloma, topline data from the SADAL study and completion of enrollment in the Phase 3 BOSTON study.
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 our expectations relating to submissions and to the review and potential approval of selinexor by regulatory authorities, including the anticipated timing of such submissions and actions, and the potential availability of accelerated approval pathways, the therapeutic potential of and potential clinical development plans for Karyopharm's drug candidates, especially selinexor, and the plans for commercialization. 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 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 the SADAL study in patients with relapsed or refractory DLBCL or that any of Karyopharm's drug candidates, including selinexor, will successfully complete necessary 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
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Pomalyst® are registered trademarks of
Darzalex® is a registered trademark of
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Karyopharm Therapeutics Inc.
CONDENSED CONSOLIDATED BALANCE SHEETS
(in thousands, except share and per share amounts)
|Cash and cash equivalents||$||101,600||$||68,997|
|Prepaid expenses and other current assets||4,792||1,754|
|Total current assets||211,562||148,423|
|Property and equipment, net||2,914||2,185|
|Liabilities and stockholders’ equity|
|Other current liabilities||556||133|
|Total current liabilities||41,567||49,467|
|Deferred revenue, net of current portion||4,532||—|
|Deferred rent, net of current portion||2,815||1,363|
|Preferred stock, $0.0001 par value; 5,000,000 shares authorized; none issued and outstanding||—||—|
|Common stock, $0.0001 par value; 100,000,000 shares authorized; 60,664,857 and 49,533,150 shares issued and outstanding at September 30, 2018 and December 31, 2017, respectively||6||5|
|Additional paid-in capital||786,763||625,017|
|Accumulated other comprehensive loss||(153||)||(217||)|
|Total stockholders’ equity||171,078||129,464|
|Total liabilities and stockholders’ equity||$||219,992||$||180,294|
Karyopharm Therapeutics Inc.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except share and per share amounts)
|Three Months Ended
|Nine Months Ended
|License and other revenue||$||239||$||—||$||30,130||$||71|
|Research and development||36,427||25,237||122,482||72,440|
|General and administrative||12,966||5,818||30,076||18,717|
|Total operating expenses||49,393||31,055||152,558||91,157|
|Loss from operations||(49,154||)||(31,055||)||(122,428||)||(91,086||)|
|Other income (expense):|
|Total other income, net||1,085||428||2,240||1,196|
|Loss before income taxes||(48,069||)||(30,627||)||(120,188||)||(89,890||)|
|Income tax provision||(14||)||(13||)||(9||)||(54||)|
|Net loss per share—basic and diluted||$||(0.79||)||$||(0.65||)||$||(2.17||)||$||(2.00||)|
|Weighted-average number of common shares outstanding used in net loss per share—basic and diluted||60,586,511||47,141,146||55,465,261||44,974,945|
Source: Karyopharm Therapeutics Inc.